Posts Tagged: safety

NEW YORK (Reuters Health) – To test whether a new drug is an improvement over existing treatments, the ideal clinical trial would compare the medications head to head, but few trials of rheumatoid arthritis treatments happen that way, according to a new study.

Instead, researchers found that for certain new rheumatoid arthritis medications, subjects in the comparison groups were often assigned to continue taking a drug that didn't help them or to take a fake drug called a placebo — in both cases effectively depriving those patients of treatment for their disease.

“Of course it's easier to compare to a placebo than an active treatment but in no way can it justify exposing patients to irreversible morbidity,” said Dr. Candice Estellat at the French national medical research institute, INSERM, who led the study.

Her concern is that if studies don't compare a new drug to other effective ones, people's conditions will persist or even worsen throughout the study.

Additionally, without so-called head-to-head trials, doctors will have little evidence to go on to determine whether one treatment is better than another, Estellat said.

Rheumatoid arthritis is an autoimmune disease that causes inflammation and damage to joints.

About 1.5 million adults have the painful disorder, and they typically require lifelong treatment with physical therapy or medications, such as methotrexate.

The newest forms of rheumatoid arthritis medications to become available are called biologic disease-modifying antirheumatic drugs (DMARDs).

These include the brand-name medications Enbrel and Humira. They come in the form of an injection, and cost around $15,000 per year.

Estellat and a colleague gathered information on all clinical trials of biologic DMARDs registered with the U.S. government's clinicaltrials.gov website and that were ongoing between 2002 and 2009.

She said they decided to do the study after hearing from specialists about the lack studies comparing these new drugs against other biologic DMARDs.

Of the 91 trials they identified, just five studies compared one biologic medication to another.

“Unfortunately we were not surprised as it confirms rheumatologists' feeling(s),” Estellat said.

The remaining trials will not provide doctors and patients with information for making evidence-based decisions, she and her coauthor write in the Archives of Internal Medicine.

“There (are) plenty of studies to show that A is better than placebo, B is better than placebo, C is better than placebo but so few to know which one is the best between A, B or C,” Estellat told Reuters Health by email.

Not only are placebo-compared experiments less useful in understanding how well a drug works compared to others, but, the report points out, they may violate international ethical research standards and specific guidelines from the American College of Rheumatology if people are not given treatment for a serious condition like rheumatoid arthritis.

The 91 trials included 102 comparisons of a biologic medication against something else — in most cases that something else was a placebo or a treatment previously shown not to work for them.

“Despite recommendations to give biologic treatments to patients with an inadequate response to conventional treatment, 9,879 patients were or will be randomized to control arms to receive no treatment or their previous ineffective treatment,” Estellat said.

Studies on humans have to go through ethical scrutiny by independent bodies called institutional review boards, and in the U.S. they must also be approved by the Food and Drug Administration.

A spokesperson for the pharmaceutical trade group, PhRMA, wrote in an email to Reuters Health that “much of that decision-making is done under the guidance of FDA, whose experts are able to work with companies to evaluate the pros and cons of different types of trials.”

Dr. Steven Pearson, the president of the Institute for Clinical and Economic Review in Boston, explained the possible reasons for not using head-to-head trials for certain drugs in an editorial accompanying Estellat's study.

He agreed that using placebos is a less desirable trial design to ultimately help physicians determine which medication they should prescribe for their patients, but said it's a trade-off for making an experiment less difficult.

“For one, having an active agent against a comparator would require many more patients,” he told Reuters Health.

“In order to be able to get clear signals of the safety and effectiveness of new agents it's going to be easiest to compare it to a placebo, in terms of costs and duration,” Pearson added.

To be most useful to patients and physicians, clinical trials need to compare one drug to another, and Pearson said that companies, regulators and researchers should think of creative ways to do so without having the studies become prohibitively expensive or unwieldy.

“I think the study is helpful to (the Food and Drug Administration) and others to take stock and see if there are other innovative study designs and approaches that allow more head-to-head trials,” Pearson said.

Estellat said that people involved in clinical studies “have to imagine original designs to conciliate ethics and scientific requirements.”

SOURCE: http://bit.ly/yh1orf Archives of Internal Medicine, February 13, 2012.

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Few rheumatoid arthritis clinical trials compare drugs: study

LOS ANGELES–(BUSINESS WIRE)–

ImmunoCellular Therapeutics, Ltd. (“ImmunoCellular” or the “Company”) (OTCBB: IMUC –News), a biotechnology company focused on the development of novel immune-based cancer therapies, today announced that John Yu, MD, Chairman and Chief Scientific Officer of ImmunoCellular Therapeutics, will deliver a presentation at the Cambridge Healthtech Institute’s inaugural Targeting Stem Cells Symposium as a part of the 19th Annual Molecular Medicine Tri-Conference from February 19-23, 2012. Dr. Yu will present during a session highlighting Emerging Cancer Stem Cell Therapeutics, featuring the Company’s discovery and development of cancer stem cell therapy.

The Cambridge Healthtech Institute’s Targeting Cancer Stem Cells Symposium reflects a growing interest in cancer stem cells and their developing importance in the field of oncology, as more pharmaceutical and biotech companies have begun to focus on cancer stem cells as oncological drug targets. The symposium will feature case studies from those working with cancer stem cells, a history of the role of cancer stem cells in treatment resistance, as well as highlights from ongoing novel cancer stem cell therapeutic development programs and platforms.

About ImmunoCellular Therapeutics, Ltd.

IMUC is a Los Angeles-based clinical-stage company that is developing immune-based therapies for the treatment of brain and other cancers. The Company recently commenced a Phase II trial of its lead product candidate, ICT-107, a dendritic cell-based vaccine targeting multiple tumor associated antigens including those associated with cancer stem cells for glioblastoma treatment. To learn more about IMUC, please visit www.imuc.com.

Forward-Looking Statements

This press release contains certain forward-looking statements that are subject to a number of risks and uncertainties, including the risk that any patents issued covering IMUC’s vaccine technology will not provide significant commercial protection for IMUC’s technology or products; the risk that the safety and efficacy results obtained in the Phase I trial for the dendritic cell- based vaccine will not be confirmed in subsequent trials; the risk that the correlation between immunological response and progression-free and overall survival in the Phase I trial for ICT-107 will not be reflected in statistically significant larger patient populations; the risk that IMUC will not be able to secure a partner company for development or commercialization of ICT-107. Additional risks and uncertainties are described in IMUC's most recently filed SEC documents, such as its most recent annual report on Form 10-K, all quarterly reports on Form 10-Q and any current reports on Form 8-K. IMUC undertakes no obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise.

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ImmunoCellular Therapeutics To Present at Targeting Stem Cells Symposium during 19th Annual Molecular Medicine Tri …

By David Voreacos – Thu Feb 16 18:24:47 GMT 2012

Johnson & Johnson’s anti-psychotic drug Risperdal wasn’t a substantial factor in causing a Nebraska man’s diabetes, even though J&J failed to adequately warn his doctor about the drug’s risks, a New Jersey jury ruled.

Jurors in state court in New Brunswick, where J&J is based, today found by a 5-1 vote that the company didn’t adequately warn the doctor for Gary Skala of the risk of diabetes. Because they ruled 5-1 against Skala, 56, on the causation question, they didn’t award him any damages.

Skala’s lawsuit was the first of more than 400 personal- injury lawsuits over Risperdal to go to trial. J&J lawyers said the company properly warned of the drug’s risks after its introduction in 1994. They said Skala was an obese “couch potato” whose disease was caused by his weight, heavy drinking, sedentary lifestyle and other risks, not Risperdal.

“Doctors used the medicine because it worked,” J&J attorney Jeffrey Peck told jurors yesterday in his closing argument. “In this case, Dr. Skala’s doctors prescribed it and it saved his life.”

Skala, who has chronic major depression, first took Risperdal in 1996 after attempting suicide by taking an overdose of anti-anxiety medicine, said Peck. He later drank as many as 10 beers a day, and anxiety, stress, sleep problems and family history contributed to his diabetes, Peck said. Skala, who is 5- feet, 8-inches tall, weighed as much as 240 pounds, Peck said.

‘Contributing Factor’

An attorney for Skala, Fletch Trammell, argued to jurors that Risperdal was a “substantial contributing factor” in his diabetes by helping to cause his obesity. While the drug may have helped his mental illness, Trammell said, J&J’s Janssen unit failed to warn Skala’s doctor of the diabetes risk.

“We’re not suing them for helping him with depression, we’re suing them for giving him diabetes,” Trammell said in his closing arguments yesterday. “Risperdal can be a good drug, but they still have to warn you about the safety risk.”

Trammell said he was disappointed with the verdict, which he called a “Pyrrhic victory.”

“It’s just a reflection of the way that people who are mentally ill live, and it’s a judgment on his lifestyle,” Trammell said.

Teresa Mueller, a spokeswoman for J&J, the world’s second- biggest health-products company, said in an e-mail: “We are pleased with the jury’s decision to reject the plaintiff’s claims. Since the early 1990s, Risperdal has improved the lives of countless people throughout the world who suffer from debilitating mental illnesses.”

Texas Settlement

J&J has faced other legal challenges over Risperdal, which was once its best-selling drug. On Jan. 19, it ended a trial in Austin, Texas, agreeing to pay $158 million to settle claims by the state attorney general and a whistle-blower that it fraudulently marketed Risperdal.

J&J has agreed to pay more than $1 billion to the U.S. and a number of states to end a civil investigation into Risperdal marketing practices, Bloomberg News reported on Jan. 6, citing people familiar with the matter. The settlement hasn’t been made public.

In June, a South Carolina judge ordered J&J to pay $327 million after a jury found the drugmaker liable for damages over its Risperdal marketing. The drugmaker vowed to appeal that award.

The company also lost a Risperdal case in Louisiana in October 2010, where on top of a $257.7 million jury award, a judge ordered the company to pay $73.3 million in attorneys’ fees and costs.

A Pennsylvania judge threw out the state’s case against J&J and Janssen in June 2010. J&J and Janssen also have been sued over their Risperdal marketing by Alaska, Arkansas, Louisiana, Montana, New Mexico, Pennsylvania and Utah. The Arkansas case is set for trial in March.

The New Jersey case is Skala v. Johnson & Johnson (JNJ), MID- L-6820-06 (MT), Superior Court of New Jersey, Law Division, Middlesex County (New Brunswick).

To contact the reporter on this story: David Voreacos in Newark, New Jersey, at dvoreacos@bloomberg.net

To contact the editor responsible for this story: Michael Hytha at mhytha@bloomberg.net

Originally posted here:
J&J’s Risperdal Not Substantial Factor in Diabetes, New Jersey Jury Says

By Dr Ananya Mandal, MD

Researchers from United States have successfully healed the scars caused by a heart attack using stem cells gathered from the patient's own heart. The amount of scar tissue was halved in the small safety trial reported in the Lancet medical journal. The authors said there was also an “unprecedented” increase in new heart muscle.

A heart attack happens when the organ is starved of oxygen, such as a clot blocking the flow of blood to the heart. As the heart heals, the dead muscle is replaced with scar tissue, but because this does not beat like heart muscle the ability to pump blood around the body is reduced. About 1.3 million Americans have a heart attack each year. Doctors around the world are looking at ways of “regenerating” the heart to replace the scar tissue with beating muscle. Stem cells, which can transform into any other type of specialized cell, figure prominently in their plans.

For the new study the researchers conducted a trial at the Cedars-Sinai Heart Institute. It was designed to test the safety of using stem cells taken from a heart attack patient's own heart. Within a month of a heart attack, a tube was inserted into a vein in the patient's neck and was pushed down towards the heart. A sample of heart tissue, about “half the size of a raisin”, was taken. This was taken to the laboratory where the stem cells were isolated and grown. Up to 25 million of these stem cells were then put into the arteries surrounding the heart.

Twenty five patients took part in the trial. Before the treatment, scar tissue accounted for an average of 24% of their left ventricle, a major chamber of the heart. It went down to 16% after six months and 12% after a year. Healthy heart muscle appeared to take its place. The study said the cells, “have an unprecedented ability to reduce scar and simultaneously stimulate the regrowth of healthy [heart] tissue”.

One of the researchers Dr Eduardo Marban said, “While the primary goal of our study was to verify safety, we also looked for evidence that the treatment might dissolve scar and regrow lost heart muscle. This has never been accomplished before, despite a decade of cell therapy trials for patients with heart attacks. Now we have done it. The effects are substantial, and surprisingly larger in humans than they were in animal tests.”

“If we can regenerate the whole heart, then the patient would be completely normal,” Marban said. “We haven’t fulfilled that yet, but we’ve gotten rid of half of the injury, and that’s a good start.”

However, there was no increase in a significant measure of the heart's ability to pump – the left ventricle ejection fraction: the percentage of blood pumped out of the left ventricle. Prof Anthony Mathur, who is co-ordinating a stem cell trial involving 3,000 heart attack patients, said that even if the study found an increase in ejection fraction then it would be the source of much debate. He argued that as it was a proof-of-concept study, with a small group of patients, “proving it is safe and feasible is all you can ask”. “The findings would be very interesting, but obviously they need further clarification and evidence,” he added.

“The findings in this paper are encouraging,” Deepak Srivastava, director of the San Francisco-based Gladstone Institute of Cardiovascular Disease, said in an interview. “There’s a dire need for new therapies for people with heart failure, it’s still the No. 1 cause of death in men and women.”

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Heart attack scars mended using stem cells

CLEVELAND, Feb. 14, 2012 /PRNewswire/ – Milo Biotechnology will receive $250,000 from JumpStart Inc., a nonprofit investing intensive business assistance and some capital into early stage Northeast Ohio-based tech companies.  A clinical stage startup developing a therapy to increase muscle strength and improve the quality of life of muscular dystrophy patients, Milo is the 61st company in JumpStart's portfolio.  ”There are limited treatment options for muscular dystrophy and approved therapies are inadequate or can cause significant side effects,” says JumpStart's Mike Lang. “Since most muscular dystrophy patients are children, the medical community is interested in a safer and more targeted therapy.”

Milo Biotechnology's lead product is an adeno-associated virus (AAV) delivered follistatin protein.  Follistatin inhibits the activity of myostatin, a protein that impedes muscle differentiation and growth.  That makes follistatin a very potent stimulator of muscle growth and prevention of muscle scarring after injury.  “The translational research team at the Research Institute at Nationwide Children's Hospital has harnessed the power of follistatin and increased its specificity for muscle,” explains Al Hawkins, CEO of Milo Biotechnology and CEO-in-Residence at BioEnterprise in Cleveland. “The efficacy and safety results thus far have been remarkable.” Pre-clinical studies in mice and non-human primates demonstrated considerable increases in muscle size and strength.  FDA approved the investigators' IND in October 2011 and a clinical trial evaluating the safety and efficacy of Milo's follistatin therapy began last month at Columbus-based Nationwide Children's Hospital, where the technology was developed.  The Phase I/II trial, funded by a grant from Parent Project Muscular Dystrophy, is enrolling patients with Becker muscular dystrophy and inclusion body myositis.

Milo Biotechnology was founded by Hawkins and one of the lead inventors of the follistatin technology, Brian Kaspar, PhD. 

MILO BIOTECHNOLOGY  Milo Biotechnology is a Cleveland-based company dedicated to improving the lives of patients with neuromuscular diseases.  Milo's early clinical stage follistatin program, exclusively licensed from Nationwide Children's Hospital, is designed to increase muscle size and strength.

JUMPSTART INC  JumpStart Inc. accelerates the successes of entrepreneurs, their companies, and the ecosystems supporting them. JumpStart has given intensive business assistance to more than 400 entrepreneurial clients and invested in 61 early stage Northeast Ohio companies. For more information, visit www.jumpstartinc.org and follow @JumpStartInc on Twitter.

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JumpStart Invests $250,000 in Milo Biotechnology

February 15, 2012, 12:06 AM EST

By Ryan Flinn

(Adds comment from researcher in 13th paragraph.)

Feb. 14 (Bloomberg) — Stem cells grown from patients’ own cardiac tissue can heal damage once thought to be permanent after a heart attack, according to a study that suggests the experimental approach may one day help stave off heart failure.

In a trial of 25 heart-attack patients, 17 who got the stem cell treatment showed a 50 percent reduction in cardiac scar tissue compared with no improvement for the eight who received standard care. The results, from the first of three sets of clinical trials generally needed for regulatory approval, were published today in the medical journal Lancet.

“The findings in this paper are encouraging,” Deepak Srivastava, director of the San Francisco-based Gladstone Institute of Cardiovascular Disease, said in an interview. “There’s a dire need for new therapies for people with heart failure, it’s still the No. 1 cause of death in men and women.”

The study, by researchers from Cedars-Sinai Heart Institute in Los Angeles and Johns Hopkins University in Baltimore, tested the approach in patients who recently suffered a heart attack, with the goal that repairing the damage might help stave off failure. While patients getting the stem cells showed no more improvement in heart function than those who didn’t get the experimental therapy, the theory is that new tissue regenerated by the stem cells can strengthen the heart, said Eduardo Marban, the study’s lead author.

“What our trial was designed to do is to reverse the injury once it’s happened,” said Marban, director of Cedars- Sinai Heart Institute. “The quantitative outcome that we had in this paper is to shift patients from a high-risk group to a low- risk group.”

Minimally Invasive

The stem cells were implanted within five weeks after patients suffering heart attacks. Doctors removed heart tissue, about the size of half a raisin, using a minimally invasive procedure that involved a thin needle threaded through the veins. After cultivating the stem cells from the tissue, doctors reinserted them using a second minimally invasive procedure. Patients got 12.5 million cells to 25 million cells.

A year after the procedure, six patients in the stem cell group had serious side effects, including a heart attack, chest pain, a coronary bypass, implantation of a defibrillator, and two other events unrelated to the heart. One of patient’s side effects were possibly linked to the treatment, the study found.

While the main goal of the trial was to examine the safety of the procedure, the decrease in scar tissue in those treated merits a larger study that focuses on broader clinical outcomes, researchers said in the paper.

Heart Regeneration

“If we can regenerate the whole heart, then the patient would be completely normal,” Marban said. “We haven’t fulfilled that yet, but we’ve gotten rid of half of the injury, and that’s a good start.”

While the study resulted in patients having an increase in muscle mass and a shrinkage of scar size, the amount of blood flowing out of the heart, or the ejection fraction, wasn’t different between the control group and stem-cell therapy group. The measurement is important because poor blood flow deprives the body of oxygen and nutrients it needs to function properly, Srivastava said.

“The patients don’t have a functional benefit in this study,” said Srivastava, who wasn’t not involved in the trial.

The technology is being developed by closely held Capricor Inc., which will further test it in 200 patients for the second of three trials typically required for regulatory approval. Marban is a founder of the Los Angeles-based company and chairman of its scientific advisory board. His wife, Linda Marban, is also a founder and chief executive officer.

“We’d like to study patients who are much sicker and see if we can actually spare them early death, or the need for a heart transplant, or a device,” Eduardo Marban said.

–Editors: Angela Zimm, Andrew Pollack

#<184845.409373.2.1.99.7.25># -0- Feb/14/2012 17:13 GMT

To contact the reporter on this story: Ryan Flinn in San Francisco at rflinn@bloomberg.net

To contact the editor responsible for this story: Reg Gale at rgale5@bloomberg.net

Originally posted here:
Scarred Hearts Can Be Mended With Stem Cell Therapy

13 February 2012 Last updated at 19:52 ET By James Gallagher Health and science reporter, BBC News

Damage caused by a heart attack has been healed using stem cells gathered from the patient's own heart, according to doctors in the US.

The amount of scar tissue was halved in the small safety trial reported in the Lancet medical journal.

The authors said there was also an “unprecedented” increase in new heart muscle.

The British Heart Foundation said it was “early days”, but could “be great news for heart attack patients”.

A heart attack happens when the organ is starved of oxygen, such as a clot blocking the flow of blood to the heart.

As the heart heals, the dead muscle is replaced with scar tissue, but because this does not beat like heart muscle the ability to pump blood around the body is reduced.

Doctors around the world are looking at ways of “regenerating” the heart to replace the scar tissue with beating muscle. Stem cells, which can transform into any other type of specialised cell, figure prominently in their plans.

Heart to heart

This trial, at the Cedars-Sinai Heart Institute, was designed to test the safety of using stem cells taken from a heart attack patient's own heart.

Continue reading the main story Healing the heart

This is the second group of doctors to report using cells taken from a heart to heal a heart.

In November 2011, another safety trial showed the cells could be used to heal the hearts of heart failure patients who were having heart bypass surgery.

The heart is not the only source for these stem cells and other fields are much further ahead.

The largest ever trial of stem cell therapy in heart attack patients is about to get under way in Europe.

The BAMI trial will inject 3,000 heart attack patients with stem cells taken from their bone marrow within five days of the heart attack.

Within a month of a heart attack, a tube was inserted into a vein in the patient's neck and was pushed down towards the heart. A sample of heart tissue, about “half the size of a raisin”, was taken.

This was taken to the laboratory where the stem cells were isolated and grown. Up to 25 million of these stem cells were then put into the arteries surrounding the heart.

Twenty five patients took part in the trial. Before the treatment, scar tissue accounted for an average of 24% of their left ventricle, a major chamber of the heart. It went down to 16% after six months and 12% after a year.

Healthy heart muscle appeared to take its place. The study said the cells, “have an unprecedented ability to reduce scar and simultaneously stimulate the regrowth of healthy [heart] tissue”.

One of the researchers Dr Eduardo Marban said: “While the primary goal of our study was to verify safety, we also looked for evidence that the treatment might dissolve scar and regrow lost heart muscle.

“This has never been accomplished before, despite a decade of cell therapy trials for patients with heart attacks. Now we have done it.

Continue reading the main story “Start Quote

These cells have been proven to form heart muscle in a Petri dish but now they seem to be doing the same thing when injected back into the heart as part of an apparently safe procedure”

End Quote Prof Jeremy Pearson British Heart Foundation

“The effects are substantial, and surprisingly larger in humans than they were in animal tests.”

However, there was no increase in a significant measure of the heart's ability to pump – the left ventricle ejection fraction: the percentage of blood pumped out of the left ventricle.

Prof Anthony Mathur, who is co-ordinating a stem cell trial involving 3,000 heart attack patients, said that even if the study found an increase in ejection fraction then it would be the source of much debate.

He argued that as it was a proof-of-concept study, with a small group of patients, “proving it is safe and feasible is all you can ask”.

“The findings would be very interesting, but obviously they need further clarification and evidence,” he added.

Prof Jeremy Pearson, associate medical director at the British Heart Foundation, said: “It's the first time these scientists' potentially exciting work has been carried out in humans, and the results are very encouraging.

“These cells have been proven to form heart muscle in a petri dish but now they seem to be doing the same thing when injected back into the heart as part of an apparently safe procedure.

“It's early days, and this research will certainly need following up, but it could be great news for heart attack patients who face the debilitating symptoms of heart failure.”

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Cells 'heal' heart attack scars

MARLBOROUGH, Mass.–(BUSINESS WIRE)–

Advanced Cell Technology, Inc. (“ACT”; OTCBB: ACTC), a leader in the field of regenerative medicine, announced today the dosing of third patient in its Phase 1/2 trial for Stargardt’s macular dystrophy (SMD) using retinal pigment epithelial (RPE) cells derived from human embryonic stem cells (hESCs). The patient was treated on Monday (Feb. 6) by Steven Schwartz, M.D., Ahmanson Professor of Ophthalmology at the David Geffen School of Medicine at the University of California, Los Angeles (UCLA) and retina division chief at UCLA’s Jules Stein Eye Institute. The outpatient transplantation surgery was performed successfully and the patient is recovering uneventfully.

“With the treatment of this third Stargardt’s patient at Jules Stein Eye Institute, we have now completed the treatment of the first cohort of patients under our clinical protocol for phase I/II of our U.S. SMD trial,” said Gary Rabin, chairman and chief executive officer of ACT. “We will continue to regularly monitor the three SMD patients in this trial, and by early spring anticipate review of their progress and safety-related data by the Data and Safety Monitoring Board (DSMB). With approval of the DSMB, we would then advance to the next cohort of patients and administer a higher dosage of RPE cells. In the context of all three trials we have running, this patient is the fifth person worldwide to be treated with our hESC-derived RPE cells. To date, there have been no complications or side effects due to the RPE cells, and we remain cautiously optimistic that our ongoing clinical programs will demonstrate the safety and tolerability of ACT’s stem cell-derived RPE cells.”

Each of the three clinical trials being undertaken by the company in the U.S. and Europe will enroll 12 patients, with cohorts of three patients each in an ascending dosage format. These trials are prospective, open-label studies, designed to determine the safety and tolerability of hESC-derived RPE cells following sub-retinal transplantation into patients with SMD or dry age-related macular degeneration (dry AMD) at 12 months, the study’s primary endpoint. Preliminary results relating to both early safety and biological function for the first two patients in the United States, one SMD patient and one dry AMD patient, were recently reported in The Lancet. On January 20, 2012, the first SMD patient to be enrolled in the Company’s U.K. clinical trial was treated at Moorfields Eye Hospital in London.

Further information about patient eligibility for the SMD study and the concurrent study on dry AMD is also available on www.clinicaltrials.gov; ClinicalTrials.gov Identifiers: NCT01345006 and NCT01344993.

About Stargardt's Disease

Stargardt’s disease or Stargardt’s Macular Dystrophy is a genetic disease that causes progressive vision loss, usually starting in children between 10 to 20 years of age. Eventually, blindness results from photoreceptor loss associated with degeneration in the pigmented layer of the retina, called the retinal pigment epithelium, which is the site of damage that the company believes the hESC-derived RPE may be able to target for repair after administration.

About Advanced Cell Technology, Inc.

Advanced Cell Technology, Inc., is a biotechnology company applying cellular technology in the field of regenerative medicine. For more information, visit www.advancedcell.com.

Forward-Looking Statements

Statements in this news release regarding future financial and operating results, future growth in research and development programs, potential applications of our technology, opportunities for the company and any other statements about the future expectations, beliefs, goals, plans, or prospects expressed by management constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Any statements that are not statements of historical fact (including statements containing the words “will,” “believes,” “plans,” “anticipates,” “expects,” “estimates,” and similar expressions) should also be considered to be forward-looking statements. There are a number of important factors that could cause actual results or events to differ materially from those indicated by such forward-looking statements, including: limited operating history, need for future capital, risks inherent in the development and commercialization of potential products, protection of our intellectual property, and economic conditions generally. Additional information on potential factors that could affect our results and other risks and uncertainties are detailed from time to time in the company’s periodic reports, including the report on Form 10-K for the year ended December 31, 2010. Forward-looking statements are based on the beliefs, opinions, and expectations of the company’s management at the time they are made, and the company does not assume any obligation to update its forward-looking statements if those beliefs, opinions, expectations, or other circumstances should change. Forward-looking statements are based on the beliefs, opinions, and expectations of the company’s management at the time they are made, and the company does not assume any obligation to update its forward-looking statements if those beliefs, opinions, expectations, or other circumstances should change. There can be no assurance that the Company’s clinical trials will be successful.

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ACT Announces Third Patient with Stargardt’s Disease Treated in U.S. Clinical Trial with RPE Cells Derived from …

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Washington, Feb 13 : Children with juvenile idiopathic arthritis (JIA) are four times likelier to have cancer than those without the disease, researchers have revealed.

The findings suggest JIA treatment, such as tumor necrosis factor (TNF) inhibitors, does not necessarily explain the development of cancer in this pediatric population.

Children with JIA experience symptoms similar to adults with arthritis including joint pain, swelling, tenderness and stiffness.

JIA is a general term used to describe the various chronic arthritis diseases in children.

One of the drug types used to treat childhood and adult arthritis, along with a number of other rheumatic conditions, is TNF inhibitors.

Studies have reported that more than 600,000 people worldwide have received anti-TNF therapy since their introduction 15 years ago. However, possible cancer risk has been associated with treatment, prompting the U.S. Food and Drug Administration (FDA) to place “black box” warnings of the potential malignancy risk on TNF inhibitors labels.

In the present study Dr. Timothy Beukelman from the University of Alabama at Birmingham and colleagues conducted one of the largest investigations into the rates of incident malignancy among JIA pediatric patients relative to their treatment.

Using data from the U.S. Medicaid records from 2000 through 2005, researchers identified 7,812 children with JIA and two comparator groups without JIA; one group with asthma (652,234 children) and the second with attention-deficit hyperactivity disorder (ADHD; 321,821 children).

The team categorized patients' treatment with methotrexate and TNF inhibitors as “ever” or “never” used, though many children with JIA did not receive either of these treatments during the study.

The research team did not have access to detailed medical records, and therefore categorized the identified incident malignancies as “possible,” “probable,” or “highly probable.”

The team determined that among all children with JIA compared to those without JIA, the incidence rate was 4.4 times higher for probable and highly probable malignancies.

Pediatric JIA patients treated with methotrexate without TNF inhibitors had a similarly increased incidence of cancer, which was 3.9 times higher than children without JIA. No probable or highly probable malignancies were identified in patients following any use of anti-TNF during the study period.

“While our findings show children with JIA have a higher incidence of cancer compared to peers without JIA, the greater frequency of malignancy does not appear to be necessarily associated with treatment, including use of TNF inhibitors,” Dr. Beukelman stated.

“This highlights the critical importance of appropriate comparator groups when evaluating the safety of new medications. Further confirmation of our findings with large-scale and long-term investigation of the association between cancer and JIA, and its treatment is needed,” he added.

The findings are available in Arthritis and Rheumatism, a journal published by Wiley-Blackwell on behalf of the American College of Rheumatology. (ANI)

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Yearly Horoscope of 2012 for the Zodiac Sign:

Sagittarius     Scorpio     Libra    Virgo    Leo     Cancer     Gemini     Taurus     Aries     Pisces     Aquarius     Capricon

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Juvenile arthritis quadruples cancer risk in kids

Public release date: 13-Feb-2012
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Contact: Dawn Peters
healthnews@wiley.com
781-388-8408
Wiley-Blackwell

New research reports that incident malignancy among children with juvenile idiopathic arthritis (JIA) is four times higher than in those without the disease. Findings now available in Arthritis & Rheumatism, a journal published by Wiley-Blackwell on behalf of the American College of Rheumatology (ACR), suggest JIA treatment, such as tumor necrosis factor (TNF) inhibitors, does not necessarily explain the development of cancer in this pediatric population.

Children with JIA experience symptoms similar to adults with arthritis including joint pain, swelling, tenderness and stiffness. JIA is a general term used to describe the various chronic arthritis diseases in children and affects roughly 294,000 under the age of 17 in the U.S. according to a 2008 report from the National Arthritis Data Workgroup.

One of the drug types used to treat childhood and adult arthritis, along with a number of other rheumatic conditions, is TNF inhibitors. Studies have reported that more than 600,000 people worldwide have received anti-TNF therapy since their introduction 15 years ago. However, possible cancer risk has been associated with treatment, prompting the U.S. Food and Drug Administration (FDA) to place “black box” warnings of the potential malignancy risk on TNF inhibitors labels.

In the present study Dr. Timothy Beukelman from the University of Alabama at Birmingham and colleagues conducted one of the largest investigations into the rates of incident malignancy among JIA pediatric patients relative to their treatment. Using data from the U.S. Medicaid records from 2000 through 2005, researchers identified 7,812 children with JIA and two comparator groups without JIA; one group with asthma (652,234 children) and the second with attention-deficit hyperactivity disorder (ADHD; 321,821 children).

The team categorized patients' treatment with methotrexate and TNF inhibitors as “ever” or “never” used, though many children with JIA did not receive either of these treatments during the study. The research team did not have access to detailed medical records, and therefore categorized the identified incident malignancies as “possible,” “probable,” or “highly probable.”

Children diagnosed with JIA had a total follow-up time of 12,614 person-years with 1,484 children in this group contributing 2,922 person-years of anti-TNF exposure. The team determined that among all children with JIA compared to those without JIA, the incidence rate was 4.4 times higher for probable and highly probable malignancies. Pediatric JIA patients treated with methotrexate without TNF inhibitors had a similarly increased incidence of cancer, which was 3.9 times higher than children without JIA. No probable or highly probable malignancies were identified in patients following any use of anti-TNF during the study period.

In a related editorial published today in Arthritis & Rheumatism, Dr. Karen B. Onel and Dr. Kenan Onel from the University of Chicago state that the Beukelman et al. study indicates that children with JIA may be at increased cancer risk from the disease, but suggests that anti-TNF therapy may not be associated with a further increased cancer risk. Dr. Kenan Onel cautions, “Larger studies in different populations and with longer follow up are required to confirm Dr. Beukelman's findings.”

The editorial authors point out that most patients in this study were treated with etanercept, a soluble TNF receptor blocker, and the investigation of other anti-TNF agents working by different mechanisms may yield different results. Nonetheless, Dr. Onel argues, “By focusing on the possible cancer risk associated with the use of TNF inhibitors, the underlying cancer risk associated with JIA may have been understated, and it is important to make patients, families, and physicians aware of the possible late consequences of this disease.”

Dr. Beukelman concludes, “While our findings show children with JIA have a higher incidence of cancer compared to peers without JIA, the greater frequency of malignancy does not appear to be necessarily associated with treatment, including use of TNF inhibitors. This highlights the critical importance of appropriate comparator groups when evaluating the safety of new medications. Further confirmation of our findings with large-scale and long-term investigation of the association between cancer and JIA, and its treatment is needed.”

###

This research was supported by grants from the Agency for Healthcare Research and Quality (AHRQ), the Food and Drug Administration (FDA) U.S. Department of Health and Human Services (DHHS), and the National Institutes of Health (NIH).

This study is published in Arthritis & Rheumatism. Media wishing to receive a PDF of this article may contact healthnews@wiley.com

Full citations: “Rates of Malignancy Associated with Juvenile Idiopathic Arthritis and Its Treatment.” Timothy Beukelman, Kevin Haynes, Jeffrey R Curtis, Fenglong Xie, Lang Chen, Christina J. Bemrich-Stolz, Elizabeth Delzell, Kenneth G Saag, Daniel H Solomon, James D Lewis on behalf of the Safety Assessment of Biological thERapeutics (SABER) Collaboration. Arthritis & Rheumatism; Published Online: February 13, 2012 (DOI: 10.1002/art.34348).

URL Upon publication: http://doi.wiley.com/10.1002/art.34348.

Editorial: “TNF inhibitors and Cancer in JIA: Disentangling the Web.” Karen B Onel and Kenan Onel. Arthritis & Rheumatism; Published Online: February 13, 2012 (DOI: 10.1002/art.34349).

URL Upon publication: http://doi.wiley.com/10.1002/art.34349.

Author Contacts: To arrange an interview with Dr. Beukelman, please contact Bob Shepard with the University of Alabama at Birmingham at bshep@uab.edu . Media wishing to speak with Dr. Karen B. Onel or Dr. Kenan Onel may contact John Easton with the University of Chicago Medical Center at John.Easton@uchospitals.edu .

About the Journal:

Arthritis & Rheumatism is an official journal of the American College of Rheumatology (ACR) and the Association of Rheumatology Health Professionals (ARHP), a division of the College, and covers all aspects of inflammatory disease. The American College of Rheumatology (www.rheumatology.org) is the professional organization who share a dedication to healing, preventing disability, and curing the more than 100 types of arthritis and related disabling and sometimes fatal disorders of the joints, muscles, and bones. Members include practicing physicians, research scientists, nurses, physical and occupational therapists, psychologists, and social workers. The journal is published by Wiley-Blackwell on behalf of the ACR. For more information, please visit http://onlinelibrary.wiley.com/journal/10.1002/(ISSN)1529-0131.

About Wiley-Blackwell:

Wiley-Blackwell is the international scientific, technical, medical, and scholarly publishing business of John Wiley & Sons, with strengths in every major academic and professional field and partnerships with many of the world's leading societies. Wiley-Blackwell publishes nearly 1,500 peer-reviewed journals and 1,500+ new books annually in print and online, as well as databases, major reference works and laboratory protocols. For more information, please visit www.wileyblackwell.com or our new online platform, Wiley Online Library (wileyonlinelibrary.com), one of the world's most extensive multidisciplinary collections of online resources, covering life, health, social and physical sciences, and humanities.

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AAAS and EurekAlert! are not responsible for the accuracy of news releases posted to EurekAlert! by contributing institutions or for the use of any information through the EurekAlert! system.

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Cancer rate 4 times higher in children with juvenile arthritis

Children with juvenile idiopathic arthritis (JIA), the most common form of childhood arthritis, may be at higher risk of developing cancer than children who do not have the condition, according to a new study published in the journal Arthritis & Rheumatism.

Researchers identified 7,812 children with JIA and compared them with thousands of children not affected by JIA and found that the arthritic children developed about four times as many new growths considered likely to be cancerous as children who did not have arthritis. They followed the children for about 18 months.

“Based on the data, it appears that being diagnosed with JIA increases the likelihood of developing malignancies,” said Dr. Timothy Beukelman, the study's lead author and an associate professor of pediatrics at the University of Alabama at Birmingham.

But despite the statistically higher risk, Dr. Sampath Prahalad, associate professor of pediatrics and human genetics at the Emory University School of Medicine, said cancer in children with JIA is rare.

“The risk is very low, and it's more common for children with JIA to not get cancer,” he said. The study included only 7,812 children with JIA, and nationwide, there are about 300,000. Prahalad was not involved with the research.

Beukelman said there are a number of possible explanations for the finding, but so far, no one knows why JIA may predispose children to cancer. He stressed, however, that there was no association between cancer risk and any treatments for the condition, including the use of drugs known as tumor necrosis factor (TNF) inhibitors.

TNF inhibitors, including the brand-name drugs Enbrel and Humira, are considered by doctors to be revolutionizing treatments that can sometimes stop rheumatoid arthritis in its tracks.

But there have been a number of case reports linking the use of TNF inhibitors to an increased cancer risk in children, prompting the Food and Drug Administration (FDA) to add a black box warning to these drugs in 2009. A black box warning is the strongest warning used by the FDA and indicates a drug has potentially life-threatening effects.

“The initial concern about TNF inhibitors may have been overstated, since some of the risk likely comes from the disease itself,” Beukelman said.

Beukelman explained that when the FDA made its decision to include black box warnings on TNF inhibitors, the only data available compared children with JIA who took these drugs with healthy children.

“When we're evaluating the safety of these drugs, it's important to make comparisons to other children with the disease who didn't get the drugs and not comparisons to children in the general population,” he said.

Does Disease Itself Predispose to Cancer?

Previous studies have found a link between elevated cancer risk and certain inflammatory conditions in adults.

“There are studies that show that in adults with rheumatoid arthritis, the more severe the disease, the higher the disease activity, so the more likely the patient is to develop malignancy,” Beukelman said.

It could be, he explained, that because diseases like JIA attack the body's own immune system, the body isn't able to mount defenses against invading cancer cells.

Another possibility is that the persistent inflammation characteristic of JIA could lead to cancers, particularly in the blood cells, he said.

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Does Arthritis Raise Kids' Cancer Risk?

MONDAY, Feb. 13 (HealthDay News) — The cancer rate in children with juvenile arthritis is four times higher than in other children, a new study says.

This increased risk of cancer isn't necessarily linked to arthritis treatments, such as tumor necrosis factor (TNF) inhibitors, according to the study published online Feb. 13 in the journal Arthritis & Rheumatism.

In the United States, TNF inhibitors carry a “black box” warning about the potential cancer risk associated with the drugs.

In this study, the researchers analyzed 2000-2005 Medicaid data from more than 7,800 children with juvenile arthritis and comparison groups of about 650,000 children with asthma and nearly 322,000 children with attention-deficit hyperactivity disorder (ADHD).

The incidence rate of probable and highly probable cancers in children with juvenile arthritis was 4.4 times higher than in the other groups of children.

Juvenile idiopathic arthritis (JIA) in children is a general term covering different types of chronic arthritis. Symptoms, similar to adult arthritis, include joint pain, swelling, tenderness and stiffness.

“While our findings show children with [juvenile idiopathic arthritis] have a higher incidence of cancer compared to peers without JIA, the greater frequency of malignancy does not appear to be necessarily associated with treatment, including use of TNF inhibitors,” concluded Dr. Timothy Beukelman, of the University of Alabama at Birmingham, in a journal news release.

“This highlights the critical importance of appropriate comparator groups when evaluating the safety of new medications. Further confirmation of our findings with large-scale and long-term investigation of the association between cancer and [juvenile arthritis] and its treatment is needed,” he added.

Most of the children with juvenile arthritis in the study were treated with injections of etanercept, a soluable TNF-receptor blocker. Other anti-TNF drugs that work by different mechanisms may yield different results, Dr. Karen Onel and Dr. Kenan Onel from the University of Chicago noted in an accompanying journal editorial.

But, “By focusing on the possible cancer risk associated with the use of TNF inhibitors, the underlying cancer risk associated with [juvenile arthritis] may have been understated, and it is important to make patients, families and physicians aware of the possible late consequences of this disease,” they added in the news release.

More information

The U.S. National Institute of Arthritis and Musculoskeletal and Skin Diseases has more about juvenile arthritis.

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Higher Cancer Rate Seen in Children With Juvenile Arthritis

February 14, 2012, 12:22 AM EST

By Ryan Flinn

Feb. 14 (Bloomberg) — Stem cells grown from patients’ own cardiac tissue can heal damage once thought to be permanent after a heart attack, according to a study that suggests the experimental approach may one day help stave off heart failure.

In a trial of 25 heart-attack patients, 17 who got the stem cell treatment showed a 50 percent reduction in cardiac scar tissue compared with no improvement for the eight who received standard care. The results, from the first of three sets of clinical trials generally needed for regulatory approval, were published today in the medical journal Lancet.

“The findings in this paper are encouraging,” Deepak Srivastava, director of the San Francisco-based Gladstone Institute of Cardiovascular Disease, said in an interview. “There’s a dire need for new therapies for people with heart failure, it’s still the No. 1 cause of death in men and women.”

The study, by researchers from Cedars-Sinai Heart Institute in Los Angeles and Johns Hopkins University in Baltimore, tested the approach in patients who recently suffered a heart attack, with the goal that repairing the damage might help stave off failure. While patients getting the stem cells showed no more improvement in heart function than those who didn’t get the experimental therapy, the theory is that new tissue regenerated by the stem cells can strengthen the heart, said Eduardo Marban, the study’s lead author.

“What our trial was designed to do is to reverse the injury once it’s happened,” said Marban, director of Cedars- Sinai Heart Institute. “The quantitative outcome that we had in this paper is to shift patients from a high-risk group to a low- risk group.”

Minimally Invasive

The stem cells were implanted within five weeks after patients suffering heart attacks. Doctors removed heart tissue, about the size of half a raisin, using a minimally invasive procedure that involved a thin needle threaded through the veins. After cultivating the stem cells from the tissue, doctors reinserted them using a second minimally invasive procedure. Patients got 12.5 million cells to 25 million cells.

A year after the procedure, six patients in the stem cell group had serious side effects, including a heart attack, chest pain, a coronary bypass, implantation of a defibrillator, and two other events unrelated to the heart. One of patient’s side effects were possibly linked to the treatment, the study found.

While the main goal of the trial was to examine the safety of the procedure, the decrease in scar tissue in those treated merits a larger study that focuses on broader clinical outcomes, researchers said in the paper.

Heart Regeneration

“If we can regenerate the whole heart, then the patient would be completely normal,” Marban said. “We haven’t fulfilled that yet, but we’ve gotten rid of half of the injury, and that’s a good start.”

While the study resulted in patients having an increase in muscle mass and a shrinkage of scar size, the amount of blood flowing out of the heart, or the ejection fraction, wasn’t different between the control group and stem-cell therapy group. The measurement is important because poor blood flow deprives the body of oxygen and nutrients it needs to function properly, Srivastava said.

“The patients don’t have a functional benefit in this study,” said Srivastava, who wasn’t not involved in the trial.

The technology is being developed by closely held Capricor Inc., which will further test it in 200 patients for the second of three trials typically required for regulatory approval. Marban is a founder of the Los Angeles-based company and chairman of its scientific advisory board. His wife, Lisa Marban, is also a founder and chief executive officer.

–Editors: Angela Zimm, Andrew Pollack

-0- Feb/13/2012 22:32 GMT

To contact the reporter on this story: Ryan Flinn in San Francisco at rflinn@bloomberg.net

To contact the editor responsible for this story: Reg Gale at rgale5@bloomberg.net

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Scarred Hearts Can Be Mended With Stem Cell Therapy, Study Shows

MENLO PARK, Calif., Feb. 9, 2012 /PRNewswire/ – Auxogyn, Inc., a privately-held company advancing women's reproductive health, announced today that the Personalized Medicine World Conference (PMWC) and Prescience International selected the Auxogyn as the 'Most Promising Company' at PMWC2012 annual conference, held January 23-24, 2012.

“We are honored to be recognized by industry leaders and our peers as the most promising company for PMWC2012,” said Lissa Goldenstein, president and chief executive officer of Auxogyn.  “We look forward to bringing the promise of personalized medicine to the field of assisted reproduction through Eeva, our proprietary non-invasive system that provides objective information on embryo viability to guide patient treatment paths.”

Auxogyn was chosen from a group of 31 companies by a group of industry leaders including Paul Billings, M.D., chief medical officer, LIFE Technologies, Rowan Chapman, Ph.D., partner, Mohr Davidow Ventures, Edgar Engleman, M.D., managing partner, Vivo Ventures and Drew Senyei, M.D., managing director, Life Science Practice, Enterprise Partners, as well as audience polling.

“Auxogyn stood out among the presenting companies for its revolutionary approach to improving patient outcomes during in vitro fertilization (IVF) procedures and reproductive health as a whole,” said Tal Behar, co-founder of the Personalized Medicine World Conference. “The ability to provide quantitative information for selection of embryos during IVF procedures holds unique promise for the millions suffering from infertility today. We expect novel products like Eeva to play an increasingly significant role in delivering high-quality, cost-effective healthcare in the years to come, and we extend our sincere congratulations to Auxogyn on this premier award.”

To qualify for the Most Promising Company Award, a company must be privately-held and working the areas of therapeutics, diagnostics or platform and information technologies. Selection is based on how a company's innovation is disruptive to current standard-of-care, both in cost and effectiveness; strength of team and ability to execute; barriers to competition and strength of intellectual property and expected financial returns for investors.

About Eeva™

Auxogyn's non-invasive early embryo viability assessment (Eeva) system may improve assisted reproduction outcomes by providing IVF clinicians and patients with objective information on embryo viability.  Eeva's proprietary software automatically analyzes embryo development against scientifically and clinically validated cell-division timing parameters.  With Eeva's quantitative data on each embryo's potential development, IVF clinics may be able to select the best embryo(s) for transfer or optimize the treatment path for their patients undergoing IVF procedures. Auxogyn is completing a multi-center, 150-patient clinical trial to validate the safety and efficacy of Eeva, and plans to use the results of the study for regulatory filings in Europe and the United States in 2012.

About Auxogyn

Auxogyn, Inc. is focused on advancing the field of reproductive health through its uniquely-combined knowledge of early human developmental biology, advanced computer vision technology and best clinical practices. The company's first product, Early Embryo Viability Assessment (Eeva), provides quantitative information regarding embryo development, to assist IVF clinics in potentially selecting the best embryo(s) for transfer and optimizing the treatment path for their patients undergoing IVF procedures.  Auxogyn is privately held and funded by Kleiner Perkins Caufield & Byers, TPG Biotech and Merck Serono Ventures. For more information regarding Auxogyn please visit www.auxogyn.com.

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Auxogyn Takes Top Honor as 'Most Promising Company' at Personalized Medicine World Conference 2012

WASHINGTON–(BUSINESS WIRE)–

Biotechnology Industry Organization (BIO) President and CEO Jim Greenwood issued the following statement regarding the newly released U.S. Food and Drug Administration (FDA) guidance for the review and approval of biosimilars:

“Today the FDA announced specific guidance for the review and approval of biosimilars, medicines that are similar to, but not exact replicas of, innovator biologics. Biologic therapies save, extend and improve the quality of life for patients living with debilitating diseases including cancer, HIV/AIDS, multiple sclerosis, and a host of rare and orphan diseases.

“We are pleased that the FDA has developed these important guidances, marking another important step forward on the pathway to biosimilars. We look forward to reviewing the drafts in detail to ensure that they comply with our key principles, particularly ensuring patient safety, recognizing scientific differences between drugs and biologics, maintaining the physician-patient relationship and preserving incentives for innovation.

“In addition, BIO believes that careful post-market monitoring of the safety of a biologic, whether an innovator or biosimilar, is critical and should be funded by user fees. BIO supports timely authorization of the biosimilars user fee agreement, which will provide FDA with the resources and capacity to evaluate biosimilars products and support biosimilar post-market safety activities, while continuing to prioritize the review of innovative new medicines for unmet medical needs.

“BIO encourages the FDA not to delay approval for new innovator biologics as it undertakes a pathway for approving biosimilars. The needs of patients awaiting innovative cures and therapies for unmet medical needs must remain paramount.

“BIO looks forward to continuing to work with the FDA on finalizing the pathway for biosimilars.”

About BIO

BIO represents more than 1,100 biotechnology companies, academic institutions, state biotechnology centers and related organizations across the United States and in more than 30 other nations. BIO members are involved in the research and development of innovative healthcare, agricultural, industrial and environmental biotechnology products. BIO also produces the BIO International Convention, the world’s largest gathering of the biotechnology industry, along with industry-leading investor and partnering meetings held around the world. BIO produces BIOtech Now, an online portal and monthly newsletter chronicling “innovations transforming our world.” Subscribe to BIOtech Now.

Upcoming BIO Events

BIO CEO & Investor Conference
February 13-14, 2012
New York, NY

BIO IP Counsels Committee Conference
April 16 – 18, 2012
Austin, TX

World Congress on Industrial Biotechnology & Bioprocessing
April 29-May 2, 2012
Orlando, FL

2012 BIO International Convention
June 18-21, 2012
Boston, MA

BIO Business Forum
June 18 – 21, 2012
Boston, MA

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Release of FDA Guidance for Biosimilars Pathway Marks Important Step Forward, Says BIO

20-10-2011 12:44 Eric Nelson, Center for Veterinary Medicine This presentation discusses FDA’s recent strategic review, known as the Animal Feed Safety System Initiative. It provides information on FDA’s early efforts to ensure feed safety for animals and people, with animal feed regulations designed to ensure safe drug use in feed, resulting in Good Manufacturing Practice (GMP) regulations. Later regulations addressed Salmonella, mycotoxin, BSE, and melamine contamination of feed; and recent significant food borne human health issues are discussed.

View original post here:
Animal Feed Safety – Video

The phase I trial follows promising studies on mice showing that such transplants were able to rebuild the structures of the inner ear, and some anecdotal evidence from humans, sparking hope of a cure for some forms of deafness.

One of those people is two-year-old Finn McGrath, who suffered brain damage after being deprived of oxygen during a prolonged and complicated delivery, according to his mother, Laura.

“His doctors told us he was at high risk for cerebral palsy, vision issues, hearing problems and mental retardation,” she said in an interview with AFP.

Finn's early days were an all-out struggle to survive, so for his parents, learning that he had failed his hearing tests and had damaged hair cells — the sensory receptors in the inner ear that pick up sounds — was almost an afterthought.

He had organ failure, breathing problems, and his cerebral palsy left him unable to roll, crawl or walk, hold his head up, talk or eat.

As his parents searched for ways to help him, they came upon stories online that told of studies using cord blood to help children with cerebral palsy and other disorders.

Prior to his birth, the McGraths had arranged to privately bank his umbilical cord blood, a procedure that costs around $2,000 plus storage fees, and remains controversial among pediatricians.

Private companies such as the Cord Blood Registry, which is funding the Texas study on hearing loss, urge expecting parents to bank their umbilical cord blood and reserve it for personal use as a way to protect their family.

That advice runs counter to the guidelines issues by the American Academy of Pediatrics in 2007, which calls such claims “unsubstantiated” and says banking for personal or family use “should be discouraged” but is “encouraged” if it is to be stored in a bank for public use.

Since Finn's parents had already banked his, they enrolled him in cord blood trial for cerebral palsy in North Carolina and he received his first transplant in November 2009 when he was about seven weeks old.

A second transfusion followed and by May, his parents began to notice a change.

Nighttime noises, like an alarm on his food pump or the sound of ripping medical tape, would suddenly startle him awake, his mother recalled.

“He started vocalizing sounds and we could tell that he was anticipating things that we would say. Like, if he had heard a story a number of times or a song, he would smile like he recognized the song or the story.”

Finn had a third infusion in September 2010, when he was one year old. Four months later, an otoacoustic emissions test (OAE), which plays a sound and picks up vibrations in the cochlea and hair cells, came back normal.

The early hearing tests that showed hearing loss were not exactly the same as the later tests that came back normal, so McGrath is cautious about comparing them directly, but she believes the cord blood transfusions may have helped.

“All I can tell you is anecdotally he was not able to hear for probably the first three or four months of his life, and then when he was about six to eight months old, he started hearing.”

The hearing trial in Texas aims to take a first step in testing the safety, and later the efficacy, of transfusing cord blood in children age six weeks to 18 months who have sustained post-birth sensorineural hearing loss.

Some reasons that children lose their hearing at or after birth may include oxygen deprivation, head injury, infection, strong doses of antibiotics or loud noises.

Sensorineural hearing loss affects approximately six per 1,000 children, and there is no available medical treatment. Hearing aids or cochlear implants are typically offered to boost the ability of the damaged tissues.

“Stem cell therapy may potentially repair the damaged structures of the inner ear and restore normal hearing,” lead investigator Sami Fakhri told AFP.

“We are at the initial stages of this process and the results are looking promising,” Fakhri added.

Research using stem cells in cord blood, known as hematopoietic cells, is already under way on some types of brain injury, cerebral palsy, juvenile diabetes, kidney and lung disease, he said.

The new study at Memorial Hermann-Texas Medical Center is being funded by the Cord Blood Registry, a private bank, and those eligible must have already banked their own umbilical cord blood with CBR.

But to Stephen Epstein, an otolaryngologist in Maryland, that does not pose a conflict of interest, because separate medical institutions in Texas and Georgia are conducting the Food and Drug Administration-approved research.

“If both of them can reproduce the same results then I would say it has some validity to it,” said Epstein, who is not involved in the study.

“This is certainly a welcome, acceptable experiment, but it should be looked at with caution and time will tell.”

One patient is already enrolled and the study, which runs for one year, has room for nine more.

While Finn McGrath still faces many challenges due to his cerebral palsy, his mother is grateful for the things he can do.

“I don't know how much worse off he would have been without the stem cell transfusion,” McGrath said, pointing to his normal cognition, lack of seizures, good hearing and vision.

“We remain hopeful that he will continue to improve.”

(c) 2012 AFP

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US begins stem cell trial for hearing loss

ScienceDaily (Jan. 30, 2012) — Mouse skin cells can be converted directly into cells that become the three main parts of the nervous system, according to researchers at the Stanford University School of Medicine. The finding is an extension of a previous study by the same group showing that mouse and human skin cells can be directly converted into functional neurons.

The multiple successes of the direct conversion method could refute the idea that pluripotency (a term that describes the ability of stem cells to become nearly any cell in the body) is necessary for a cell to transform from one cell type to another. Together, the results raise the possibility that embryonic stem cell research and another technique called “induced pluripotency” could be supplanted by a more direct way of generating specific types of cells for therapy or research.

This new study, published online Jan. 30 in the Proceedings of the National Academy of Sciences, is a substantial advance over the previous paper in that it transforms the skin cells into neural precursor cells, as opposed to neurons. While neural precursor cells can differentiate into neurons, they can also become the two other main cell types in the nervous system: astrocytes and oligodendrocytes. In addition to their greater versatility, the newly derived neural precursor cells offer another advantage over neurons because they can be cultivated to large numbers in the laboratory — a feature critical for their long-term usefulness in transplantation or drug screening.

In the study, the switch from skin to neural precursor cells occurred with high efficiency over a period of about three weeks after the addition of just three transcription factors. (In the previous study, a different combination of three transcription factors was used to generate mature neurons.) The finding implies that it may one day be possible to generate a variety of neural-system cells for transplantation that would perfectly match a human patient.

“We are thrilled about the prospects for potential medical use of these cells,” said Marius Wernig, MD, assistant professor of pathology and a member of Stanford's Institute for Stem Cell Biology and Regenerative Medicine. “We've shown the cells can integrate into a mouse brain and produce a missing protein important for the conduction of electrical signal by the neurons. This is important because the mouse model we used mimics that of a human genetic brain disease. However, more work needs to be done to generate similar cells from human skin cells and assess their safety and efficacy.”

Wernig is the senior author of the research. Graduate student Ernesto Lujan is the first author.

While much research has been devoted to harnessing the pluripotency of embryonic stem cells, taking those cells from an embryo and then implanting them in a patient could prove difficult because they would not match genetically. An alternative technique involves a concept called induced pluripotency, first described in 2006. In this approach, transcription factors are added to specialized cells like those found in skin to first drive them back along the developmental timeline to an undifferentiated stem-cell-like state. These “iPS cells” are then grown under a variety of conditions to induce them to re-specialize into many different cell types.

Scientists had thought that it was necessary for a cell to first enter an induced pluripotent state or for researchers to start with an embryonic stem cell, which is pluripotent by nature, before it could go on to become a new cell type. However, research from Wernig's laboratory in early 2010 showed that it was possible to directly convert one “adult” cell type to another with the application of specialized transcription factors, a process known as transdifferentiation.

Wernig and his colleagues first converted skin cells from an adult mouse to functional neurons (which they termed induced neuronal, or iN, cells), and then replicated the feat with human cells. In 2011 they showed that they could also directly convert liver cells into iN cells.

“Dr. Wernig's demonstration that fibroblasts can be converted into functional nerve cells opens the door to consider new ways to regenerate damaged neurons using cells surrounding the area of injury,” said pediatric cardiologist Deepak Srivastava, MD, who was not involved in these studies. “It also suggests that we may be able to transdifferentiate cells into other cell types.” Srivastava is the director of cardiovascular research at the Gladstone Institutes at the University of California-San Francisco. In 2010, Srivastava transdifferentiated mouse heart fibroblasts into beating heart muscle cells.

“Direct conversion has a number of advantages,” said Lujan. “It occurs with relatively high efficiency and it generates a fairly homogenous population of cells. In contrast, cells derived from iPS cells must be carefully screened to eliminate any remaining pluripotent cells or cells that can differentiate into different lineages.” Pluripotent cells can cause cancers when transplanted into animals or humans.

The lab's previous success converting skin cells into neurons spurred Wernig and Lujan to see if they could also generate the more-versatile neural precursor cells, or NPCs. To do so, they infected embryonic mouse skin cells — a commonly used laboratory cell line — with a virus encoding 11 transcription factors known to be expressed at high levels in NPCs. A little more than three weeks later, they saw that about 10 percent of the cells had begun to look and act like NPCs.

Repeated experiments allowed them to winnow the original panel of 11 transcription factors to just three: Brn2, Sox2 and FoxG1. (In contrast, the conversion of skin cells directly to functional neurons requires the transcription factors Brn2, Ascl1 and Myt1l.) Skin cells expressing these three transcription factors became neural precursor cells that were able to differentiate into not just neurons and astrocytes, but also oligodendrocytes, which make the myelin that insulates nerve fibers and allows them to transmit signals. The scientists dubbed the newly converted population “induced neural precursor cells,” or iNPCs.

In addition to confirming that the astrocytes, neurons and oligodendrocytes were expressing the appropriate genes and that they resembled their naturally derived peers in both shape and function when grown in the laboratory, the researchers wanted to know how the iNPCs would react when transplanted into an animal. They injected them into the brains of newborn laboratory mice bred to lack the ability to myelinate neurons. After 10 weeks, Lujan found that the cells had differentiated into oligodendroytes and had begun to coat the animals' neurons with myelin.

“Not only do these cells appear functional in the laboratory, they also seem to be able to integrate appropriately in an in vivo animal model,” said Lujan.

The scientists are now working to replicate the work with skin cells from adult mice and humans, but Lujan emphasized that much more research is needed before any human transplantation experiments could be conducted. In the meantime, however, the ability to quickly and efficiently generate neural precursor cells that can be grown in the laboratory to mass quantities and maintained over time will be valuable in disease and drug-targeting studies.

“In addition to direct therapeutic application, these cells may be very useful to study human diseases in a laboratory dish or even following transplantation into a developing rodent brain,” said Wernig.

In addition to Wernig and Lujan, other Stanford researchers involved in the study include postdoctoral scholars Soham Chanda, PhD, and Henrik Ahlenius, PhD; and professor of molecular and cellular physiology Thomas Sudhof, MD.

The research was supported by the California Institute for Regenerative Medicine, the New York Stem Cell Foundation, the Ellison Medical Foundation, the Stinehart-Reed Foundation and the National Institutes of Health.

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The above story is reprinted from materials provided by Stanford University Medical Center. The original article was written by Krista Conger.

Note: Materials may be edited for content and length. For further information, please contact the source cited above.

Journal Reference:

E. Lujan, S. Chanda, H. Ahlenius, T. C. Sudhof, M. Wernig. Direct conversion of mouse fibroblasts to self-renewing, tripotent neural precursor cells. Proceedings of the National Academy of Sciences, 2012; DOI: 10.1073/pnas.1121003109

Note: If no author is given, the source is cited instead.

Disclaimer: This article is not intended to provide medical advice, diagnosis or treatment. Views expressed here do not necessarily reflect those of ScienceDaily or its staff.

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Skin cells turned into neural precusors, bypassing stem-cell stage

WASHINGTON, D.C. — Researchers at the U.S. Department of Energy’s (DOE’s) Joint BioEnergy Institute (JBEI) recently announced a major breakthrough in engineering systems of RNA molecules through computer-assisted design.

This could lead to important improvements across a range of industries, including the development of cheaper advanced biofuels. 

Scientists will use these new “RNA machines,” to adjust genetic expression in the cells of microorganisms. 

This will enable scientists to develop new strains of Escherichia coli (E. coli) that are better able to digest switchgrass biomass and convert released sugars to form three types of transportation fuels — gasoline, diesel and jet fuels.

“This is a perfect example of how our investments in basic science innovations can pave the way for future industries and solutions to our nation’s most important challenges,” Energy Secretary Steven Chu said in a news release.

“This breakthrough at the Joint BioEnergy Institute holds enormous potential for the sustainable production of advanced biofuels and countless other valuable goods.”

A breakthrough with E. coli could make it cheaper to produce fuel from switchgrass or other non-food biomass plants to create advanced biofuels with the potential to replace gasoline. 

While the work at the JBEI remains focused on the development of advanced biofuels, its researchers believe their concepts may help other researchers to develop many other desired products, including biodegradable plastics and therapeutic drugs. 

For example, some researchers have started a project to investigate how to use the “RNA machines” to increase the safety and efficacy of medicine therapies to treat diseases, including diabetes and Parkinson’s.

Biological systems are incredibly complex, which makes it difficult to engineer systems of microorganisms that will produce desired products in predictable amounts. 

JBEI’s work, featured in the Dec. 23 issue of “Science” magazine, is the first of its kind to set up and adjust a RNA system in a predictable way. 

Specifically, researchers focused their design-driven approach on RNA sequences that can fold into complicated three dimensional shapes, called ribozymes and aptazymes. 

By using JBEI-developed computer-assisted models and simulations, researchers then created complex RNA-based control systems that are able to program a large number of genes. 

In microorganisms, “commands” that are sent into the cell will be processed by the RNA-based control systems, enabling them to help develop desired products.

One of the major goals of synthetic biology is to produce valuable chemical products from simple, inexpensive and renewable starting materials in a sustainable manner. 

Computer-assisted models and simulations like the one JBEI developed are essential for doing so. 

Up to this point, such tools for biology have been limited, and JBEI’s breakthrough in applying computer assisted design marks an important technical and conceptual achievement for this field. 

To view additional details about this research, visit http://newscenter.lbl.gov/news-releases/2011/12/22/cad-for-rna/.

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Genetic breakthroughs help develop cheaper biofuels: DOE

LOS ANGELES–(BUSINESS WIRE)– ImmunoCellular Therapeutics, Ltd.(“ImmunoCellular” or the Company) (OTCBB: IMUC.OB – News), a biotechnology company focused on the development of novel immune-based cancer therapies, today announced that its president and CEO, Manish Singh Ph.D., will be presenting at the Immunotherapeutics Partnering and Deal Making Conference, which will take place in San Diego, California on January 30-31, 2012. Dr. Singh’s presentation will be titled “Targeting Cancer Stem Cells Via Immunotherapy” and will include an overview of ImmunoCellular’s clinical activities to date including ICT-107, its lead program targeting cancer stem cells (CSC) in Glioblastoma. ICT-107 is currently in a randomized phase II clinical trial at multiple centers in the US. Company is also developing two additional products targeting CSCs in tumors via modulating immune system.

Dr. Singh will also be moderating two of the panel discussions with high level executives from a number of major biotech/pharmaceutical companies. The panel discussions Dr. Singh will be moderating are “Strategic Trends in Immunotherapeutics” at 10:30 am on January 30th and “Investments and Partnership Opportunities in the Immunotherapeutics Space” at 1:30 pm on January 31st.

The Immunotherapeutics Partnering and Deal-making Conference is an immunotherapeutics business development conference that gives global biotechnology and pharmaceutical companies an opportunity to network with high-level executives from top pharma and various biotech/pharmaceutical companies, as well as explore potential collaborations, and learn about relevant immunotherapeutics issues and partnerships that will affect the industry. This event also provides a unique venue for attendees to learn about immunotherapeutics business development trends, the market, and novel technologies that shape up the industry.

This conference is also part of the Novel Immunotherapeutics Summit, which consists of this track and three other tracks:

1) 4th Immunotherapeutics and Immunomonitoring

2) 10th Cytokines and Inflammation

3) Allergy Drug Discovery and Development

For more information, please visit www.gtcbio.com

About ImmunoCellular Therapeutics, Ltd.

IMUC is a Los Angeles-based clinical-stage company that is developing immune-based therapies for the treatment of brain and other cancers. The Company recently commenced a Phase II trial of its lead product candidate, ICT-107, a dendritic cell-based vaccine targeting multiple tumor associated antigens for glioblastoma. To learn more about IMUC, please visit www.imuc.com.

Forward-Looking Statements

This press release contains certain forward-looking statements that are subject to a number of risks and uncertainties, including the risk that the safety and efficacy results obtained in the Phase I trial for the dendritic cell- based vaccine will not be confirmed in subsequent trials; the risk that the correlation between immunological response and progression-free and overall survival in the Phase I trial for ICT-107 will not be reflected in statistically significant larger patient populations; the risk that IMUC will not be able to secure a partner company for development or commercialization of ICT-107. Additional risks and uncertainties are described in IMUC's most recently filed SEC documents, such as its most recent annual report on Form 10-K, all quarterly reports on Form 10-Q and any current reports on Form 8-K. IMUC undertakes no obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise.

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ImmunoCellular Therapeutics To Deliver Presentation on Cancer Stem Cells at Prestigious Immunotherapy Conference