StemCell Therapy

Because gene therapy involves making changes to the bodys set of basic instructions, it raises many unique ethical concerns. The ethical questions surrounding gene therapy include:

How can good and bad uses of gene therapy be distinguished?

Who decides which traits are normal and which constitute a disability or disorder?

Will the high costs of gene therapy make it available only to the wealthy?

Could the widespread use of gene therapy make society less accepting of people who are different?

Should people be allowed to use gene therapy to enhance basic human traits such as height, intelligence, or athletic ability?

Current gene therapy research has focused on treating individuals by targeting the therapy to body cells such as bone marrow or blood cells. This type of gene therapy cannot be passed on to a persons children. Gene therapy could be targeted to egg and sperm cells (germ cells), however, which would allow the inserted gene to be passed on to future generations. This approach is known as germline gene therapy.

The idea of germline gene therapy is controversial. While it could spare future generations in a family from having a particular genetic disorder, it might affect the development of a fetus in unexpected ways or have long-term side effects that are not yet known. Because people who would be affected by germline gene therapy are not yet born, they cant choose whether to have the treatment. Because of these ethical concerns, the U.S. Government does not allow federal funds to be used for research on germline gene therapy in people.

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What are the ethical issues surrounding gene therapy ...

In 1990, geneticist William French Anderson injectedcells with altered genes into a four-year-old girl with severe immunodeficiency disorder. This was the first sanctioned test of gene therapy, in which genetic material is used to treat or prevent disease.

If were lucky, Anderson told The Chicago Tribune, with this little girl weve opened the door for genetic engineering to attack major killers and cripplers, particularly AIDS, cancer and heart disease.

Gene therapy has never fulfilled these grand hopes. In the decades since Andersons experiment, thousands of clinical trials of gene therapies have been carried out. But the first gene therapy was only approved for sale in the U.S. this week. The Food and Drug Administration announced its approval of Kymriah, a gene therapy produced by Novartis for a form of childhood leukemia. A few gene therapies have previously become available in Europe and China.

An FDA press release emphasizes the historic nature of the approval. Were entering a new frontier in medical innovation with the ability to reprogram a patients own cells to attack a deadly cancer, FDA Commissioner Scott Gottlieb says.

As I have noted previously, for gene-therapy proponents have long predicted that it will eliminate diseases such as cystic fibrosis and early-onset breast cancer, which are traceable to a defective gene. Enthusiasts also envisioned genetically engineered "designer babies" who would grow up to be smarter than Nobel laureates and more athletic than Olympians.

Gene therapy turned out to be extremely difficult, because it can trigger unpredictable, fatal responses from the body's immune system.The National Institutes of Health warnsthat gene therapy can have very serious health risks, such as toxicity, inflammation, and cancer.

Kymriah is a case in point. The FDA press release warns that Kymriah can cause life-threatening immune reactions and neurological events, as well as serious infections, low blood pressure (hypotension), acute kidney injury, fever, and decreased oxygen (hypoxia). According to The New York Times, the FDA is requiring that hospitals and doctors be specially trained and certified to administer [Kymriah], and that they stock a certain drug needed to quell severe reactions.

Kymriah illustrates another problem with gene therapy: high cost. Novartis estimates the cost of its treatment at $475,000 per patient. As a recent Reuters article notes, over the past five years two gene therapies have been approved for sale in Europe, one for a rare blood disease and the other for the bubble-boy immunodeficiency disorder. The therapies cost $1 million and $700,000, respectively. So far, the companies that make the therapies have achieved a total of three sales.

As journalist Horace Freeland Judson points out in this excellent 2006 overview, The Glimmering Promise of Gene Therapy, most individual diseases caused by single-gene defectsthe kind that seem most likely to be cured by gene therapyare rare. (Sickle-cell anemia and some other hemoglobin disorders are among the few exceptions.)

Judson adds that because different diseases have different genetic mechanisms and affect different types of tissue, each presents a new set of research problems to be solved almost from scratch. As the millions burned away, it became clear that even with success, the cost per patient cured would continue to be enormous. And success had shown itself to be always glimmering and shifting just beyond reach.

The advent of CRISPR, a powerful gene-editing technique, has aroused hopes that gene therapy might finally fulfillexpectations. Researchers recently reported that they employed CRISPR to counteract a mutation that causes heart disease. Potentially, The New York Times reported last month, the method could apply to any of more than 10,000 conditions caused by specific inherited mutations.

CRISPR has also renewed concerns about the ethics of producing designer babies with enhanced physical and mental traits. But as Science noted recently, CRISPR poses some of the same risks as gene therapies. CRISPR still has a long way to go before it can be used safely and effectively to repairnot just disruptgenes in people.

So to answer the question posed in the headline: No, the gene-therapy era has still not arrived.

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Has the Era of Gene Therapy Finally Arrived? - Scientific American (blog)

Carrying a $475,000 price-tag

AP

By Mallory Locklear

A CAR-T treatment a type of gene therapy for cancer has been approved for use in the US. Announced by the US Food and Drug Administration (FDA) on Wednesday, this is the first approval anywhere in the world for a type of CAR-T therapy, although the techniques have been used experimentally for some time.

CAR-T therapy made headlines earlier this year, when it was announced a CAR-T approach had saved the life of Layla, a young child in the UK who had leukaemia. The approach involves reprogramming a persons own immune cells to make them better at targeting cancerous ones.

The drug that has been approved by the FDA is Kymriah, a treatment for B-cell acute lymphoblastic leukaemia, the most common childhood cancer in the US.

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To synthesise Kymriah, a patient first has a type of immune cell, called T-cells, removed from their body and transported to a facility in New Jersey operated by the pharmaceutical firm Novartis. Here, viruses will be used to insert a gene into these cells. The gene codes for a protein called a chimeric antigen receptor (CAR).

These cells are then reinfused back into the person. The added protein helps these modified T-cells home in on and fight leukemia cells.

In a trial, this approach achieved an 83 per cent remission rate over a period of three months in people who hadnt responded to other treatment options. The FDA has approved Kymriah for people aged 25 or under who have not responded to other treatments, or who have relapsed.

Nearly half the people in the trial experienced a side effect caused by an unwanted immune response triggered by the altered T-cells. Because of this, the FDA is requiring staff at the 32 facilities approved for this treatment to undergo specific training to recognise this response, called cytokine release syndrome.

Kymriah will cost $475,000. This sounds high, but its lower than some analysts expected, and unlike many expensive cancer drugs, it is a one-off treatment that could result in years, not months, of extended lifespan.

The FDAs decision has been hailed as the first approval for a gene therapy in the US. Some argue that this isnt a true gene therapy, as the genes introduced into the T-cells are not the treatment themselves it is the transformed T-cells that go on to fight the cancer. But the FDA defines human gene therapy as products that introduce genetic material into a persons DNA to treat a disease, so has classified Kymriah as such.

Europe has already approved two gene therapies for inherited diseases, while China approved a gene therapy for cancer treatment in 2004.

As for CAR-T therapies, other firms have similar treatments in the works, while Novartis also plans to get Kymriah approved for treating lymphoma.

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Pioneering gene therapy approved for leukaemia in the US - New Scientist

MarketWatch rounded up 10 of its most interesting topics over the past week.

Campbell Soup Co. CPB, -0.22% had a rough quarter, but the company is facing a dire long-term problem.

Novartis AG NVS, -0.08% received FDA approval for the first cancer gene therapy available in the U.S. Emma Court explained how important this is for young people suffering from a type of acute lymphoblastic leukemia (ALL), and she interviewed Janney analyst Paul Knight, who made recommendations for investors on how to play a potential decade-long growth cycle for gene therapy.

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The damage from Hurricane Harvey to the Houston area has been devastating. The coming flurry of activity as the damage is repaired might cause a rise in U.S. GDP, but Caroline Baum calls claims of real economic benefits predictable nonsense.

Amazon.com Inc. AMZN, +0.11% was called the weakest major U.S. retailer this week by Moodys Investors Service. But T. Rowe Price Media and Telecommunications Fund PRMTX, +0.69% is a big believer. The fund, which had more than quadrupled the S&P 500s return over the past 15 years, had more than 10% of its assets in Amazons shares as of July 31.

If you are retired, you might think it will be very difficult to get a mortgage loan because of low income. But there are many financing options available for those without a steady monthly income, according to Darrow Kirkpatrick.

Jeff Reeves weighs the pros and cons of scooping up shares of Apple Inc. AAPL, +0.05% right now.

If you get excited by Labor Day sales, you might be missing out on bigger savings later.

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Weekend roundup: Campbell in the soup | New cancer gene therapy | Exposing bad investment advice - MarketWatch

MINNEAPOLIS (WCCO) Colin Cooley of Burnsville beat lymphoma four years ago, but the lymphoma came back in a different spot two years later.

Chemo wasnt cutting it, Cooley said. It was keeping it in check, but it wasnt getting rid of it.

He decided to undergo a clinical trial at the University of Minnesota. He received a gene therapy called CAR-T and is now cancer-free.

The FDA approved CAR-T Wednesday as the first type of gene therapy in the United States.

The treatment has been called a breakthrough in the fight against cancer. It is only approved right now to treat children with acute lymphoblastic leukemia, but doctors are excited about its potential for other cancers and diseases.

Doctors at the University of Minnesotas Cancer Day at the Minnesota State Fair called the therapy a major leap.

(credit: CBS)

Were able to take a patients own cells and turn them into something that can actually attack their specific cancer, said Dr. Edward Greeno, medical director of the University of Minnesotas Masonic Cancer Clinic. Many people have referred to this as living cancer because were taking live cells and turning them into your treatment.

First, a patients blood is drawn and their T-cells, or immune cells, are separated out. Those T-cells are then sent to a laboratory to be genetically modified and reprogrammed to zero in on the cancer.

Those modified cells are then multiplied in the lab before being returned to the patient via blood. They are essentially revved-up cells that are missiles for the cancer.

In one significant study, 83 percent of the patients who received CAR-T went into remission.

This treatment is expected to be offered for lymphoma patients next year. Dr. Greeno says it could be decades, though, before its offered to patients with other types of cancer.

Right now, its expensive almost $500,000 and used mostly on patients when other methods of treatment, like chemotherapy, have failed.

Before I didnt know if Id be here in three or four or five years, I didnt know, Cooley said. Now I feel like I have a new lease, some minor issues, but a new lease on life, and thats pretty exciting.

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How Does Gene Therapy Work? - CBS Minnesota / WCCO

Nightstar Therapeutics has filed to raise up to $86 million in a Nasdaq IPO. The money will equip Nightstar to complete a phase 3 trial of its choroideremia gene therapy and advance two other eye disease candidates through early-stage clinical studies.

London-based Nightstar, also known as NightstaRx, is set to move the choroideremia asset into phase 3 in the first half of next year. The therapy, NSR-REP1, is advancing into the 140-patient trial on the strength of data on 32 subjects treated in investigator-sponsored studies. Those trials found 90% of patients either maintained or improved their visual acuity in the year after receiving the gene therapy.

Given choroideremia causes currently-untreatable progressive vision loss, Nightstar sees the data as supporting further development. The asset is moving forward with a fairly clean safety profile in the 50 people treated to date. Investigators have seen one adverse eventtransient intraocular inflammationthat may have stemmed from treatment with NSR-REP1.

Challenges await Nightstar as it scales up for the phase 3 trial and potentially commercial sales, though. The biotech acknowledges the administration of NSR-REP1 requires significant skill and training, potentially creating a bottleneck to use of the gene therapy. And as a small, unpartnered player in a new field, any number of events could knock it off course.

What Nightstar does have is a head start. Spark Therapeutics has the most advanced challenger to NSR-REP1 but its program is yet to move past phase 1/2. The field is similarly clear for Nightstars follow-up candidate NSR-RPGR, which moved into the clinic just ahead of MeiraGTxs rival X-linked retinitis pigmentosa gene therapy. AGTCs Biogen-partnered candidate is close behind.

Nightstar has reached this point using money from a succession of private rounds, starting with the 12 million Syncona invested when the biotech spun out of the University of Oxford in 2014. The biotech pulled in a further 5 million when it named former Johnson & Johnson VP David Fellows as CEO early in 2015. A $35 million series B round followed late in 2015. And Nightstar broadened its investor base and raised a further $45 million in a series C round a few months ago.

Along the way, Nightstar has built out a team in preparation for the broadening of its clinical trial program and life on public markets. Last month, Ex-Pfizer clinical lead Tuyen Ong, M.D., left PTC Therapeutics to serve as chief development officer. And in April, Nightstar hinted at its IPO plans by recruiting the man who led Intercept Pharmaceuticals repeated public raises, Senthil Sundaram.

The question now is how receptive public investors are to gene therapy biotechs. The companies in the sector to go public to date have delivered mixed returns, with the successes of bluebird bio and Spark offset by the steady decline of uniQure and implosion of Dimension Therapeutics.

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Nightstar files for $86M IPO to fund gene therapy trials - FierceBiotech

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Dr. Gregory Yanik, clinical director of the Pediatric Blood and Marrow Transplantation Program at C.S. Mott Children's Hospital in Ann Arbor, works with Maryam Rasheed of Macomb Township. Maryam was part of a clinical trial using gene therapy to successfully treat her leukemia.(Photo: Sophie Masson/Michigan Medicine)

The U.S. Food and Drug Administrationapproved on Wednesdaythe first-ever gene therapytotreat children and young adults withleukemia.

Called Kymriah, but better known as CAR T-cell treatment, the therapy is being hailed by doctors as revolutionary. Itinvolves genetically modifyinga patient's own T-cells, which thencantarget and kill a form of acute lymphoblastic leukemiacells.

This new treatment has the potential to change the face of cancer therapy for years to come, not just in childhood acute lymphoblastic leukemia but in other cancers in which a patients own T-cells can be collected, genetically modified and redirected to kill a patients tumor," said Dr.Gregory Yanik, clinical director of the Pediatric Blood and Marrow Transplantation Program at the University of Michigan's C.S. Mott Children's Hospital. Mottwas one of a few hospitals nationally to take part inclinical trials of the treatment.

"This allows us to turn patients own cells into a powerful weapon to fight the disease a weapon that does not rely on chemotherapy but takes a whole new approach to attacking childhood leukemia, Yanik said.

The CAR T-cell treatmentoffers new hope for children like Maryam Rasheed, 10, of Macomb Township.

Maryam was diagnosed with B-cell acute lymphoblastic leukemia at age 4, when her family was seeking refuge from religious persecution in Turkey, said Maryam's mother, Asmaa Rasheed.

Maryam Rasheed (right) with her brother, Rashid, and sister Samantha. Maryam, 10 of Macomb Township, survived acute lymphoblastic leukemia.(Photo: Rasheed family photo)

"My country is Iraq," Asmaa Rasheedsaid. "It wasnt safe. We are Christian. It was so hard over there in Baghdad. We run away to Turkey.

"We take her to hospital the first timebecause ... she stopped eating, stopped walking, stopped talking. We bring her to emergency. The doctor decided to take her bone marrow to do tests. Then the results came back, and she have leukemia."

Maryam underwent her firstchemotherapy treatment in Turkey.

"Over there, it was so hard," Rasheed said. "The doctors dont speak English over there. We know English a little bit. We speak Arabic."

Maryam Rasheed of Macomb Township undergoes treatment for acute lymphoblastic leukemia. She is now in remission.(Photo: Rasheed family photo)

Rasheed stayed with her daughter for two months in the Turkish hospital. A few months later,the Rasheed family was able to immigrate to the U.S. and settled in Michigan.

But Maryam's cancer returned. She was treated at Children's Hospital of Michigan with more chemotherapy and radiation. In 2013,her younger brother, Rashid, proved to be a match for a bone marrow transplant.

Still, the cancer wouldn't relent.

The Rasheed family learned of a clinical trial for CAR T-cell therapy under way atMott. It was the family's last chance,Rasheed said.

Maryam Rasheed, 10, of Macomb Township holds up her arms joyfully. She's surrounded by her sister Samantha (left), brother, Rashid, and baby sister Annabell.(Photo: Rasheed family photo)

"There was nothing to do," her mother said."In Detroit, there was chemo, radiation, bone marrow transplant. It returned back three times. She lose her hair three times. It was so hard for her and my family."

She remembers the date Maryam started the clinical trial at Mott: Dec. 17, 2014. Maryam spent Christmas and her seventh birthday in the hospital.

"I think we waited like 100 days,I dont remember exactly, and they did a bone marrow test, and the medicine, it work!" Rasheed said.

"It was like a dream, you know, like light coming from far away when youre in the dark. Theres nothing else we could do. But the CART-cell was like a shining light from far away."

Maryam has been in remission two years, andis starting fourth grade next week at Shawnee Elementary School in Macomb Township.

"Now, shes start her life, and doing everything a little kid is doing," said Rasheed, who says she hopes the treatment helps other children, too.

So does Yanik.

"Acute lymphoblastic leukemia is the most common form of cancer in children, accounting for approximately25% of all childhood cancers," Yanik said. "This particular therapy utilizes a childs own immune system to target their leukemia."

Theclinical trials focused on the 15% to 20% ofchildren whoseB-cell acute lymphoblastic leukemia had either relapsed or who had residual leukemia cells in their bone marrow after treatment.

"Historically, such patients would have an estimated cure rate of approximately 10%," Yanik said. "The two trials were groundbreaking. In the most recent trial, 52 of 63 patients with childhood leukemia successfully entered complete remission with this therapy."

Novartis Pharmaceuticals Corp. got the FDA approval for the gene cell therapy, whichinvolves drawing blood from childrenwith B-cell acute lymphoblastic leukemia. The T-cellsin the child's blood are thenshipped to a lab where they are genetically engineered so theywillseek outa particular protein in the leukemia cells and attack. Patients are then infused with the modified blood, and the T-cells go to work to find and kill the leukemia.

The New York Times reported Wednesday that the therapy will cost $475,000 for the initial treatment, with additional treatments administered at no cost.

Although 83% of the children in the clinical trials for CAR T-cell therapy went into remission, Yaniksaid it's too early to tell howcurative treatmentswill prove in the long run. And, its use will be limited to only a few medical centers in the U.S.

"The University of Michigan is the only site in the state and within this region that is licensed to administer these cells for childhood leukemia," he said.

Offering the treatment at a large medical center like U-Mis essential, said Dr. Rajen Mody,a pediatric oncologist at Mott, because of the severity ofpotential side effects.

"It can cause serious side effects, especially within the first 21 days," said Mody, who is Mott's director of pediatric oncology. "Patients can have high fevers, bleeding complications, trouble breathing, infections. ... Thats why a hospital like the University of Michigan is the ideal place. ... Patients who undergo this treatment are usually so sick after an infusion of the CAR-T cells, that they can't be safely treated at smaller hospitals."

Dr. Rajen Mody, a pediatric oncologist at the University of Michigan's C.S. Mott Children's Hospital.(Photo: University of Michigan)

Yanik is hopeful that successful treatment with CAR T-cell therapy in children with leukemia will open the door for similar therapies targeting other cancers.

"Aseparate CAR T-cell trial targeting diffuse large-cell lymphoma was recently completed with the results in that clinical trial now under review at the FDA," he said. That trial alsoincluded adult patientsat the University of Michigan.

Mody called the gene therapy revolutionary.

"This is clearly a life-saving and potentially curative therapy," he said."Its being tested in other types of leukemia and solid tumors. Its too early to say whether its going to work as well for other cancers.... We are not there yet."

Still, he said, it's made all the difference for Maryam and her family.

"She was one of the lucky ones coming from Iraq, and with all the things she has survived. And then coming here and surviving this,... she clearly has some goodluck.

"I think she should do very well. Patients who actually survive the first six months and still have CAR T-cells detected in their systems tend todo very, very well."

Contact Kristen Jordan Shamus: 313-222-5997 or kshamus@freepress.com. Follow her on Twitter @kristenshamus.

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First gene therapy to treat cancer gets FDA approval; UM only Michigan hospital to use it - Detroit Free Press

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KANSAS CITY, Mo. -- Lucas Novick, 27, has been in a battle with leukemia since his freshman year of college.

"I was having headaches that were so bad that they were causing vomiting pretty regularly and I couldn`t see straight well enough that I felt safe driving myself to school," Novick said.

Since 2009, Novick has endured a number of treatments including chemotherapy and a bone marrow transplant. The treatments have taken a physical and mental toll on Novick's body.

"The transplant that was supposed to save my life also nearly took it from me," Novick said. "The damage chemotherapy did to my body when I was first treated in 2009 and 2010 was such that I was walking with a cane after my 21st birthday. It did so much damage to my hip joints that they were replaced in 2011."

But after Novick's leukemia returned for a second time, he went to Children's Mercy Hospital where doctors were performing an experimental treatment.

"The approval of the CTL019 product for pediatric patients with relapsed refractory acute lymphoblastic leukemia is really exciting for us," Doctor Doug Myers, of Children's Mercy Hospital, said. "We`ve spent a lot of time working on ways to get the immune system into the fight against cancer because we think it can decrease toxicity, decrease the amount of chemotherapy and radiation that we use for these cancers."

Dr. Myers said the treatment helped Novick, a musician, back onto the stage and has held his leukemia awayfor two years.

"Those are really special rewards for us in this field that have seen so many failures of this type of therapy in the past. To see this go forward, move forward, do well enough for a pharmaceutical company will pick this up and take it the rest of the way, that`s a really special time for us," Dr. Myers said.

While doctors believe it's too early to call the new treatment a cure, many agree this is the first step to a new generation of cancer treatment.

"I know at the end of the day that this is the future of medicine," Novick said.

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Man describes new FDA-approved gene therapy for leukemia that ... - fox4kc.com

Erlanger hopes to bring the treatment to the patients who need it most within the next few years . (Coutesy: WTVC)

Wednesday, the Food and Drug Administration cleared the way for a ground breaking cancer treatment in the United States.

It's a gene therapy treatment named CAR-T therapy.

Dr. Meghann McManus is a Pediatric Hematologist Oncologist at Erlanger in Chattanooga.

On Thursday, Dr. McManus described the treatment as the "first gene therapy treatment for any type of cancer in the pediatric world or adult world."

In layman's terms, Dr. McManus said the treatment allows doctors to remove a patients "T-cells, which are a type of white blood cell," send them to a lab "where their genetically modified to fight their certain type of leukemia."

"It uses the patients own immune system, their own cells from their own body, to fight their leukemia. Which is not something that we do with any other disease," Dr. McManus said.

At this time, Dr. McManus said CAR-T therapy is approved for "children that have relapsed or with refractory disease."

"We may not be able to stop cancers from happening... but if we could treat it in a way to get rid of the cancer without big side effects or tolls on patients, it would change the way we do our job," Dr. McManus said.

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Local pediatric oncologist hopeful new gene therapy will save lives - WTVC

A new study has discovered a genetic mutation on growth hormone receptors that they believe could expand our lifespan by an average of up to 10 years.

A new study led by Prof. Gil Atzmon head of the Laboratory of Genetics and Epigenetics of Aging and Longevity at the University of Haifa and his team have discovered a genetic mutation on growth hormone receptors that they believe could expand our lifespan by an average of up to 10 years. "We were aware before that variants involved with genetic paths related to the growth hormone are also associated with longevity. Now we have found a specific variant whose presence or absence is directly connected to it," Prof. Atzmon explained.

According to other research its well known that IGF-1 (insulin-like growth factor-1) contributes to longevity as well as associated with HGH and other growth hormones.

From theSciencegroup in the journalScience Advances,scientists have discovered a variation in genes that changes hormones in men that extend their life-span. Professor Nir Barzilai at Einstein, compared 3 groups of 100-year-old men from around the world with 100 American Jewish men with a control group of 70-year olds.

They discovered that men without Exon 3 on growth hormone receptor genes were more likely to live up to 10 years longer. According to Prof. Atzmon, this variant ensured longevity absolutely. "This study nicely wraps up the connection between growth hormone function and longevity. Our goal now is really to understand the mechanism of the variation we found, so that we can implement it and enable longevity while maintaining quality of life," Prof. Atzmon concluded.

Coincidently the research found that men with this genetic variation were also about 3 cm taller than their counter-parts. This is in opposition to other species in nature wherein that smaller of the strains usually live longer. This phenomenon holds true in insects, rodents and other mammals. In human males however the receptor anomaly caused less growth hormone absorption.

This research is very encouraging for World Health and A4M members. It further explains what we have hypothesized for years, that certain genetics allow for longer life. Our entire existence at A4M and World Health is devoted to Anti-Aging and how to enhance life and longevity.

Article by: Dr. Michael J. Koch, Editor withwww.WorldHealth.net and for Dr. Ronald Klatz, DO, MD President of the A4M has 28,000 Physician Members, has trained over 150,000 Physicians, health professionals and scientists in the new specialty of Anti-aging medicine. Estimates of their patients numbering in the 100s of millions World Wide that are living better stronger, healthier and longer lives.

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Growth Hormone Receptor Gene Mutation Discovered - Anti Aging News

A spate of new laws goes into effect today following the passage of about 1,200 pieces of legislation by the state legislature during its regular session.

Texans as a whole will be able to text less unless they find themselves in a city like Amarillo where the new state law will trump more restrictive city ordinances and force a more relaxed approach.

That guy walking down the street with a sword wont be subject to arrest.

Jails will follow new mental health procedures and indigent citizens will have new pathways for dealing with minor criminal offenses.

Public safety was at the top of legislators minds during the session that ran from mid-January until the end of May and saw more than 10,000 bills filed for consideration.

Here are a few of the most watched bills that become law today and some insight into how they apply to Amarillo.

Texting and driving (HB 62)

The push for a statewide texting and driving ban has been an ongoing battle for nearly a decade, and this year it was approved by both chambers and signed into law by Gov. Greg Abbott.

The bill bans drivers from texting while a vehicle is moving and makes doing so a misdemeanor offense punishable by a fine of up to $99 on the first offense and up to $200 for repeat offenders. The law only addressees reading, writing or sending electronic messages. So theres going to be a learning curve for police, because certain defenses such as using the phone for a map, for music or for anything other than texting could be used.

Amarillo adopted a hands-free ordinance in September 2012 that was enacted on Jan. 3, 2013. Amarillos local ordinance only allowed drivers to send or view text messages while legally parked, not stopped at a red light, and banned all use of mobile or electronic devices while driving unless they are connected to a hands-free device or for certified emergencies.

The texting and driving law will be uniform for the whole state. This way people can travel from town to town and not have to worry about the laws being different, Amarillo Police Department Office Jeb Hilton said of the new law. The major difference in the state law and the previous city ordinance is that you can now text while stopped at a stop sign or stop light. Our officers will continue to do their best to enforce the law.

Hilton said that from January 1 through July 31, APD wrote 619 citations and issued 244 warnings for use of wireless comunication devices.

While APD will enforce the new law, not everyone associated with the city was happy with the development while it was in special session.

Im really proud of the leadership our city showed five years ago when we enacted that law, Amarillo Mayor Ginger Nelson said of the citys ban on texting and driving. Because this is a public safety issue that will actually decrease the level safety, I am concerned about that.

Large knives (HB 1935)

Its not getting the same chatter as gun laws, but starting next month citizens can openly carry large knives.

State Rep. John Frullo, R-Lubbock, championed a bill that got passed allowing citizens to openly carry large knives in most areas of the state. The bill allows individuals to now carry knives with blades longer than 5 inches, except in certain areas like schools, hospitals and places of worship.

The new knife laws change the wording form illegal knives to location-restricted knives. What this means is that every knife is now legal to carry whether concealed or in the open, Hilton explained. There will still be places that you cannot carry a knife that has a blade that is over 5 inches in length. These include schools, polling places, secure areas of airports, hospitals, churches and bars.

Swords, machetes, Bowie knives and sabers will now be perfectly fine to tote around.

We might see people carrying swords or machetes when the law first goes into effect, but I think once they realize how inconvenient it is things will change, Hilton added. APD wants to stress that there is no reason to call the police just because someone is carrying a large knife or sword in the open, but if they are carrying or using the weapon in a threatening manner be sure to give us a call.

Misdemeanor fines (HB 351)

State lawmakers passed a law aimed at keeping low-income individuals who commit minor offenses out of jail trying to prevent whats often referred to as debtors prisons.

The new law gives judges more leeway in issuing fines and costs for things such as failing to pay parking or speeding tickets, and even the ability to substitute community service for legal fees.

The law aims to make it easier for low-income and low-level offenders to get out of jail, something that local attorneys and prosecutors support with some reservations.

Randall County Distrct Attorney James Farren said he thinks getting low-level and low-income offenders out of jail quickly could help to ease costs by freeing up jail cells, which are a daily expense for taxpayers when occupied. It would also free up space for those who commit the more serious offenses.

Potter County Attorney Scott Brumley agrees with the bills intent but still has some reservations about what costs the city and county might incur.

As I understand, the major impact would be on misdemeanors and non-violent crimes, Brumley said earlier this year when the bill was passed by the House. My reaction I dont oppose looking at the way we improve criminal cases at the pre-trial stage, our office is aware and in agreement, but at some point the county will need a pre-trial services center.

That pre-trial services center or the lack of one in Potter County is one reason Brumley and Farren remain skeptical. The law does not address assisting counties financially to help make pre-trial arrangements for people who could be released early under the bill.

Sandra Bland Act

Lawmakers approved a bill in response to the death of Sandra Bland, who was found dead in a county jail after being held there following a routine traffic stop.

Lawmakers passed a watered-down version of what was originally discussed. The bill that goes into effect Friday mandates that county jails divert people with mental health and substance abuse issues toward treatment and mandates that independent investigations be had if a person dies in custody. The Sandra Bland Act also makes it easier for defendants to receive a personal bond if they have a mental illness or intellectual disability.

Ten other new Texas laws starting today

(HB 25) Eliminates straight-ticket party voting when casting an election ballot.

(HB 29) Allows state lottery winners who win more than $1 million to remain anonymous and prohibits the release of all personal information to the public.

(HB 1424) Prohibits drones and other small aircraft from flying over correction facilities like jails and prisons, and sports venues such as stadiums or facilities with more than 30,000 seats.

(SB 693) Mandates that a school bus be equipped with a three-point seat belt for every passenger. The bill only applies to buses that are 2018-and-newer models.

(SB 16) Reduces the first-time fee for a license to carry from $140 to $40 and the annual renewal fee from $70 to $40.

(HB 810) Allows patients with a severe chronic disease to use stem cell treatment.

(SB 179) Mandates that schools adopt policies related to cyberbullying and requires that schools report offenses. This law also created a new definition of cyberbullying.

(HB 478) If a person enters into a motor vehicle to remove a vulnerable individual, such as a child, that person is immune from civil liability for damage that may occur from entry.

(HB 214) Requires the Supreme Court of Texas and the Court of Criminal Appeals to have audio and video recordings of oral arguments and public meetings available if funds are made available.

(HB 1099) Says landlords cannot prohibit a tenants right to call police or emergency assistance.

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Of cell phones and swords things Amarilloans should know about new state laws - Amarillo.com

The guidelines are expected to have a negative list of around 10 items in which research would be prohibited. Besides, outlining areas in which research is permitted, detailed regulatory and technical guidelines would also be laid out.

Indian Council of Medical Research (ICMR) is set to come out with guidelines for stem cell research in two weeks.

The guidelines are expected to have a negative list of around 10 items in which research would be prohibited. Besides, outlining areas in which research is permitted, detailed regulatory and technical guidelines would also be laid out.

The research wing of ministry of health & family welfare is also going to reduce the time taken for approvals in cord and adult stem cell research.

"These two areas would be decentralized at the institutional level to reduce the bureaucratic hurdles. The ethics committee within each institution can look into it and would be required to report to ICMR," said an ICMR official.

However, for embryonic stem cell research, the National Apex Committee would scan every proposal. "This is so because we want to know how the embryos are procured, what's the procedure etc. This is the area that is most vulnerable to abuse", added the official.

The team of experts are meeting in January-end to finalize the guidelines.

In 2002, draft guidelines were issued that ICMR should be the regulatory authority for biological research and a National Apex Committee for cell-based research & therapy should be set up.

The latter should be vested with powers to examine scientific, technical, ethical, legal and social issues in all the three types of stem cell.

See the article here:
ICMR to release stem cell research guidelines soon - BSI bureau (press release)

About Cellect Biotechnology Ltd.

Cellect Biotechnology is traded the NASDAQ (NASDAQ: "APOP", "APOPW). The Company has developed a breakthrough technology for the isolation of stem cells from any given tissue that aims to improve a variety of stem cells applications.

The Company's technology is expected to provide pharma companies, medical research centers and hospitals with the tools to rapidly isolate stem cells for in quantity and quality that will allow stems cell related treatments and procedures. Cellect's technology is applicable to a wide variety of stem cells related treatments in regenerative medicine and that current clinical trials are aimed at the cancer treatment of bone marrow transplantations.

Forward Looking Statements

This press release contains forward-looking statements about the Company's expectations, beliefs and intentions. Forward-looking statements can be identified by the use of forward-looking words such as "believe", "expect", "intend", "plan", "may", "should", "could", "might", "seek", "target", "will", "project", "forecast", "continue" or "anticipate" or their negatives or variations of these words or other comparable words or by the fact that these statements do not relate strictly to historical matters. For example, forward-looking statements are used in this press release when we discuss the anticipated benefits of a sole listing on Nasdaq. These forward-looking statements and their implications are based on the current expectations of the management of the Company only, and are subject to a number of factors and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. In addition, historical results or conclusions from scientific research and clinical studies do not guarantee that future results would suggest similar conclusions or that historical results referred to herein would be interpreted similarly in light of additional research or otherwise. The following factors, among others, could cause actual results to differ materially from those described in the forward-looking statements: changes in technology and market requirements; we may encounter delays or obstacles in launching and/or successfully completing our clinical trials; our products may not be approved by regulatory agencies, our technology may not be validated as we progress further and our methods may not be accepted by the scientific community; we may be unable to retain or attract key employees whose knowledge is essential to the development of our products; unforeseen scientific difficulties may develop with our process; our products may wind up being more expensive than we anticipate; results in the laboratory may not translate to equally good results in real clinical settings; results of preclinical studies may not correlate with the results of human clinical trials; our patents may not be sufficient; our products may harm recipients; changes in legislation; inability to timely develop and introduce new technologies, products and applications, which could cause the actual results or performance of the Company to differ materially from those contemplated in such forward-looking statements. Any forward-looking statement in this press release speaks only as of the date of this press release. The Company undertakes no obligation to publicly update or review any forward-looking statement, whether as a result of new information, future developments or otherwise, except as may be required by any applicable securities laws. More detailed information about the risks and uncertainties affecting the Company is contained under the heading "Risk Factors" in Cellect Biotechnology Ltd.'s Annual Report on Form 20-F for the fiscal year ended December 31, 2016 filed with the U.S. Securities and Exchange Commission, or SEC, which is available on the SEC's website, http://www.sec.gov. and in the Company's period filings with the SEC and the Tel-Aviv Stock Exchange.

ContactCellect Biotechnology Ltd.Eyal Leibovitz, Chief Financial Officerhttp://www.cellect.co+ 972-9-974-1444

SOURCE Cellect Biotechnology Ltd.

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Cellect Shares Will Be Traded From Next Week Exclusively on NASDAQ - PR Newswire (press release)

A new genetics study adds fuel to the debate about muscle aches that have been reported by many people taking popular cholesterol-lowering drugs called statins.

About 60 percent of people of European descent carry a genetic variant that may make them more susceptible to muscle aches in general. But counterintuitively, these people had a lower risk of muscle pain when they took statins compared with placebos, researchers report August 29 in the European Heart Journal.

Millions of people take statins to lower cholesterol and fend off the hardening of arteries. But up to 78 percent of patients stop taking the medicine. One common reason for ceasing the drugs use is side effects, especially muscle pain, says John Guyton, a clinical lipidologist at Duke University School of Medicine.

It has been unclear, however, whether statins are to blame for the pain. In one study, 43 percent of patients who had muscle aches while taking at least one type of statin were also pained by other types of statin (SN: 5/13/17, p. 22). But 37 percent of muscle-ache sufferers in that study had pain not related to statin use. Other clinical trials have found no difference in muscle aches between people taking statins and those not taking the drugs.

The new study hints that genetic factors, especially ones involved in the immune systems maintenance and repair of muscles, may affect peoples reactions to statins. This is a major advance in our understanding about myalgia, or muscle pain, says Guyton, who was not involved in the study.

People with two copies of the common form of the gene LILRB5 tend to have higher-than-usual blood levels of two proteins released by injured muscles, creatine phosphokinase and lactate dehydrogenase. Higher levels of those proteins may predispose people to more aches and pains. In an examination of data from several studies involving white Europeans, people with dual copies of the common variant were nearly twice as likely to have achy muscles while taking statins as people with a less common variant, Moneeza Siddiqui of the University of Dundee School of Medicine in Scotland and colleagues discovered.

But when researchers examined who had pain when taking statins versus placebos, those with two copies of the common variant seemed to be protected from getting statin-associated muscle pain. Why is not clear.

People with double copies of the common form of the gene who experience muscle pain may stop taking statins because they erroneously think the drugs are causing the pain, study coauthor Colin Palmer of the University of Dundee said in a news release.

The less common version of the gene is linked to reduced levels of the muscle-damage proteins, and should protect against myalgia. Yet people with this version of the gene were the ones more likely to develop muscle pain specifically linked to taking statins during the trials.

The finding suggests that when people with the less common variant develop muscle pain while taking statins, the effect really is from the drugs, the researchers say.

But researchers still dont know the nitty-gritty details of how the genetic variants promote or protect against myalgia while on statins. Neither version of the gene guarantees that a patient will develop side effects or that they wont. The team proposes further clinical trials to unravel interactions between the gene and the drugs.

More study is needed before doctors can add the gene to the list of tests patients get, Guyton says. I dont think were ready to put this genetic screen into clinical practice at all, he says. For now, its much easier just to give the patient the statin and see what happens.

More here:
Muscle pain in people on statins may have a genetic link - Science News Magazine

New Delhi [India], Aug 31 (ANI-BusinessWireIndia): PerkinElmer Health Sciences Pvt Ltd (PEHS), a screening and diagnostic laboratory of PerkinElmer, Inc. today announced that it has kicked off a series of seminars for neurologists, paediatricians and gynaecologists in Delhi, Mumbai, Hyderabad, Chennai and Mangalore, India.

These events serve as an ideal platform for discussing PerkinElmers recently launched affordable gene panels, whole exome sequencing (WES) and whole genome sequencing (WGS) services using next generation sequencing and other complementary assays to address the broad range of genetic disorders.

At the first conference, which took place in Delhi, Dr. Madhuri Hegde, Vice President and Chief Scientific Officer, PerkinElmer Diagnostic Laboratory Services, delivered a talk, Simplifying Genomics: Transforming Complexity into Meaning to a group of clinicians.

Starting her presentation, Dr. Hegde said, A growing interest in personalized medicine calls for genome sequencing in clinical diagnostics, but major challenges must be addressed before its full potential can be realized. This talk on a medical genetic testing algorithm will help clinicians select the most appropriate molecular diagnostic tool for each scenario. Dr. Hegde also serves on the board of ACMG Foundation for Genetics and Genomic Medicine and is an Adjunct Professor of Genetics and Paediatrics at Emory University and Georgia Institute of Technology.

Dr. IC Verma, a pioneer in the field of Genetic Medicine joined the session in Delhi and commented: This is a most exciting time in genetics. As a result of the new genomic sequencing technologies, we can arrive at a diagnosis in many more patients than before. Finding the variation in genes is leading to the development of new treatments for the genetic disorders. The medical professionals must take advantage of the genomic tests being offered in India at an affordable rate. The genetic tests enable screening of couples for being carriers of genetic disorders, genetic counseling and prenatal diagnosis to prevent disease and the possibility of new treatments.

Dr. Verma is a renowned medical geneticist. He received genetics training in the UK, USA & Switzerland. He is a Fellow of the Royal College of Physicians, London, the American Academy of Pediatrics, and the National Academy of Medical Sciences, New Delhi. He has received a number of national awards Ranbaxy Science Award, ICMR, NAMS and BC Roy Medical Council award. He is a Member and Vice-chairman of the Ethics Committee of the International Human Genome Organization (HUGO) and serves as an adviser in genetics to the WHO in Geneva, and to Roche Genetics in Basel.

The launch of our genetics service is all about providing quality and specialized service to clinicians in India. Dr. Hegde brings our customers high confidence in PerkinElmers quality sample analysis and reporting, said Jayashree Thacker, President, PerkinElmer India. We have been observing a high demand of sequencing services for rare inherited disease. Combining these offerings with our current portfolio will help address the evolving needs of our customers.

PerkinElmer now offers its customers a global genomic lab testing platform that performs screening and diagnostic testing, specializing in newborn screening and high throughput next generation sequencing for rare inherited diseases. (ANI-BusinessWireIndia)

This is published unedited from the ANI feed.

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PerkinElmer hosts Medical Genomics Seminars in India - India.com

In a step that heralds a new era in cancer treatment, the U.S. Food and Drug Administration said Wednesday it has approved a form of gene therapy that is highly effective at fighting an aggressive form of leukemia in young patients with no other options.

The treatment, to be marketed under the name Kymriah, is neither a pill nor an injection, but a personalized medicine service that functions as a living drug. Patients would have their bodys own disease-fighting T cells fortified and multiplied in a lab, then get the cells back to help them fight their cancer.

In clinical trials of 88 patients with a relapsing or treatment-resistant form of acute lymphoblastic leukemia, 73 went into remission after receiving the experimental treatment.

FDA Commissioner Scott Gottlieb, himself a survivor of blood cancer, predicted that this new approach to cancer treatment will change the face of modern medicine.

Cancer researchers and physicians outside the agency shared Gottliebs enthusiasm.

Dr. Crystal L. Mackall, associate director of Stanford Universitys Cancer Institute, called Kymriah a transformative therapy. It represents an entirely new class of cancer therapies that holds promise for all cancer patients.

Acute lymphoblastic leukemiais the most common form of pediatric cancer, affecting some 3,000 children and young adults yearly in the United States. Though it is considered highly curable in most patients, about 600 each year either do not respond to chemotherapy or see their leukemia return after an initial round of successful treatment.

Those patients dont make it none of them do, said Dr. Stephan A. Grupp, director of the cancer immunotherapy program at Childrens Hospital of Philadelphia, who administered the first course of Kymriah five years ago when it was an experimental treatment called CTL019.

That initial patient, 7-year-old Emily Whitehead of Philipsburg, Pa., saw her leukemia remit completely within three weeks of getting the treatment. Now 12, she was among those calling on the FDA to approve Kymriah for other patients like her.

Certainly for blood cancers, this is a game-changer, Grupp said. Adapting this therapy for patients with solid tumors, he said, will be the work of the next five years.

The new approach was designed to fight some of the most stubborn cancers by giving the bodys immune system a very specific assist.

It starts by harvesting a cancer patients T cells, the warriors of the immune system. The cells are delivered to a specialized lab where scientists alter their DNA, essentially reprogramming them to target cancer cells. These reengineered cells are called chimeric antigen receptor T cells, or CAR-T cells.

The new and improved cells are copied millions of times before theyre sent back to the patient. Once infused into the bloodstream, the CAR-T cells are much better equipped to hunt down and kill cancer cells, wherever they may hide.

Novartis, the company that developed Kymriah, intends to have 32 certified treatment centers up and running by the end of 2018. Patients up to the age of 25 would go to one of these centers to have their T cells harvested and later reintroduced in their modified form.

The cells themselves will be genetically engineered at a Novartis manufacturing facility in Morris Plains, N.J.

Kymriah is the first CAR-T treatment to come before the FDA, but it wont be the last. No fewer than 76 CAR-T treatments are currently under review at the FDA, and Gottlieb predicted that other approvals would follow.

Therapies that would operate in similar ways engineering the immune systems T cells to fight disease more effectively are under investigation for a host of other conditions, including HIV/AIDS, genetic and autoimmune disorders and other forms of cancer.

Todays FDA ruling is a milestone, said Dr. David Maloney, medical director of cellular immunotherapy at Fred Hutchinson Cancer Research Center in Seattle. This is just the first of what will soon be many new immunotherapy-based treatments for a variety of cancers.

Novartis, the Swiss pharmaceutical company that is gearing up to provide Kymriah to as many as 600 patients a year, said it would charge $475,000 for the treatment.

Novartis representatives said they calculated a cost-effective price for the therapy that fell between $600,000 and $750,000. But the company chose instead to charge a price that it said would cover costs, and to introduce a novel approach to billing. Chief Executive Joseph Jimenez said the company will not charge hospitals for the therapy if the patient does not fully respond in a given period of time.

The company also said it will launch a patient assistance program for those who are uninsured or underinsured, and provide some travel assistance for patients and caregivers seeking the treatment.

Gottlieb touted Kymriahs approval as a turning point for the FDA as well. Novartis application for Kymriah came just seven months ago. The agency tagged the application with two designations that ensured its speedy review.

First proposed in 1972, the idea of correcting or enhancing genes to treat disease has a history buoyed by promise but also buffeted by failures. With recent advances in genomic medicine, cell biology and genetic engineering, efforts to locate and edit the genes and cells that play a key role in disease have injected new hope for such treatments.

Gene and cell therapies that target the immune system for enhancement have been particularly promising. They do, however, come with risks specifically, that the activation of immune cells will run amok, sparking reactions ranging from rash and itching to fever and flu-like symptoms that can lead to death.

In approving Kymriah, the FDA warned that it has the potential to cause severe side effects, including cytokine release syndrome, an overreaction to the activation and proliferation of immune cells that causes high fever and flu-like symptoms, and neurological events. Both can be life-threatening. Kymriah can also cause serious infections, low blood pressure, acute kidney injury, fever and low oxygen levels.

The FDA called for continuing safety studies of the new therapy.

melissa.healy@latimes.com

@LATMelissaHealy

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Hailing a breakthrough in fighting cancer, FDA approves gene therapy that functions as a 'living drug' - Los Angeles Times

B. Bick, . Poindexter, UT Med. School/SPL

A depletion of brain cells that produce dopamine is responsible for the mobility problems seen in people with Parkinsons disease.

Japanese researchers report promising results from an experimental therapy for Parkinsons disease that involves implanting neurons made from reprogrammed stem cells into the brain. A trial conducted in monkeys with a version of the disease showed that the treatment improved their symptoms and seemed to be safe, according to a report published on 30 August in Nature1.

The studys key finding that the implanted cells survived in the brain for at least two years without causing any dangerous effects in the body provides a major boost to researchers hopes of testing stem-cell treatments for Parkinsons in humans, say scientists.

Jun Takahashi, a stem-cell scientist at Kyoto University in Japan who led the study, says that his team plans to begin transplanting neurons made from induced pluripotent stem (iPS) cells into people with Parkinsons in clinical trials soon.

The research is also likely to inform several other groups worldwide that are testing different approaches to treating Parkinsons using stem cells, with trials also slated to begin soon.

Nature breaks down the latest research and what it means for the future of stem-cell treatments.

Parkinsons is a neurodegenerative condition caused by the death of cells called dopaminergic neurons, which make a neurotransmitter called dopamine in certain areas of the brain. Because dopamine-producing brain cells are involved in movement, people with the condition experience characteristic tremors and stiff muscles. Current treatments address symptoms of the disease but not the underlying cause.

Researchers have pursued the idea that pluripotent stem cells, which can form any cell type in the body, could replace dead dopamine-making neurons in people with Parkinsons, and thus potentially halt or even reverse disease progression. Embryonic stem cells, derived from human embryos, have this capacity, but they have been the subject of ethical debates. Induced pluripotent stem (iPS) cells, which are made by coaxing adult cells into an emybronic-like state, have the same versatility without the associated ethical concerns.

Takahashis team transformed iPS cells derived from both healthy people and those with Parkinsons into dopamine-producing neurons. They then transplanted these cells into macaque monkeys with a form of the disease induced by a neuron-killing toxin.

The transplanted brain cells survived for at least two years and formed connections with the monkeys brain cells, potentially explaining why the monkeys treated with cells began moving around their cages more frequently.

Crucially, Takahashis team found no sign that the transplanted cells had developed into tumours a key concern with treatments that involve pluripotent cells or that they evoked an immune response that couldnt be controlled with immune-suppressing drugs.

Its addressing a set of critical issues that need to be investigated before one can, with confidence, move to using the cells in humans, says Anders Bjorklund, a neuroscientist at Lund University in Sweden.

I hope we can begin a clinical trial by the end of next year, says Takahashi. Such a trial would be the first iPS cell trial for Parkinson's. In 2014, a Japanese woman in her 70s became the first person to receive cells derived from iPS cells, to treat her macular degeneration.

In theory, iPS cells could be tailor-made for individual patients, which would eliminate the need to use drugs that suppress a possible immune response to foreign tissues.

But customized iPS cells are expensive to make and can take a couple months to derive and grow, Takahashi notes. So his team instead plans to establish iPS cell lines from healthy people and then use immune cell biomarkers to match them to people with Parkinsons in the hope of minimizing the immune response (and therefore the need for drugs to blunt the attack).

In a study described in an accompanying paper in Nature Communications2, Takahashis team implanted into monkeys iPS-cell-derived neurons from different macaques. They found that transplants between monkeys carrying similar white blood cell markers triggered a muted immune reaction.

Earlier this year, Chinese researchers began a Parkinsons trial that used a different approach: giving patients neural-precursor cells made from embryonic stem cells, which are intended to develop into mature dopamine-producing neurons. A year earlier, in a separate trial, patients in Australia received similar cells. But some researchers have expressed concerns that the immature transplanted cells could develop tumour-causing mutations.

Meanwhile, researchers who are part of a Parkinsons stem-cell therapy consortium called GForce-PD, of which Takahashis team is a member, are set to bring still other approaches to the clinic. Teams in the United States, Sweden and the United Kingdom are all planning trials to transplant dopamine-producing neurons made from embryonic stem cells into humans. Previously established lines of embryonic stem cells have the benefit that they are well studied and can be grown in large quantities, and so all trial participants can receive a standardized treatment, notes Bjorklund, also a consortium member.

Jeanne Loring, a stem-cell scientist at the Scripps Research Institute in La Jolla, California, favours transplanting iPS-derived neurons made from a patients own cells. Although expensive, this approach avoids dangerous immunosuppressive drugs, she says. And because iPS cells are established anew for each patient, the lines go through relatively few cell divisions, minimizing the risk that they will develop tumour-causing mutations. Loring hopes to begin her teams trial in 2019. This shouldnt be a race and were cheering for success by all, she says.

Lorenz Studer, a stem-cell scientist at the Memorial Sloan Kettering Cancer Center in New York City who is working on a trial that will use neurons made from embryonic stem cells, says that there are still issues to work out, such as the number of cells needed in each transplant procedure. But he says that the latest study is a sign that we are ready to move forward.

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Reprogrammed cells relieve Parkinson's symptoms in trials - Nature.com

Increased DNA double-strand breaks in Pol-deficient neural progenitors. Credit: Osaka University

DNA is the computer code that programs every event in the body. Despite the importance of DNA fidelity, as the body develops, cells grow and replicate, DNA is constantly turned over. This repeated process can compromise the DNA, so cells have many DNA repair mechanisms. Using mice, Osaka University scientists report a defect in one type of machinery. DNA polymerase (Pol) causes underdevelopment of the brain's cortices and axonal network. The findings could explain cortical development disorders such as autism and microcephaly.

"Pol is responsible for repairing DNA base damage in the brain. Because many neurological disorders are associated with de novo mutations, we wanted to study how loss of Pol affects neuronal development," said Assistant Professor Noriyuki Sugo, an expert in the study of Pol in brain development.

"We found evidence that Pol has a role in the development of the brain but not other organs, and that its defect causes catastrophic DNA double strand breaks (DSBs) and consequent cell death in certain regions of the developing cortex," he said.

These regions represent one of the earliest stages of cortical development, and the generation of cortical neurons is fundamental for proper neural networking.

In the present study, Sugo and his team prepared mutant mice deficient in Pol. These mice showed a large number of DSBs in neural progenitors, the stem cells that eventually produce neurons. Consequently, many immature neurons died through apoptosis. Furthermore, the mice showed defects in the development of specific brain anatomy and the growth of axon in specific cell types, suggesting both an underdevelopment of the cortex and of neural networking.

"We found that Pol deficiency led to higher neuronal cell death in deeper layers than upper layers of the cortex. The deeper layers were thinner," said Sugo. He added that deeper-layer neurons were marked by a higher rate of DSBs.

Neurons formed in these layers are thought essential to the early stages of neural networking. Thus, even if the cells manage to escape death, the brain circuitry is likely compromised.

Finally, proper development depends on both genetic and epigenetic factors. The correction of DNA damage by Pol is an example of genetic regulation. In addition, the researchers found DNA demethylation, an example of epigenetic regulation, is also abnormal in mice deficient of Pol. Together, Sugo argues the findings are strong evidence for the importance of Pol on proper gene expression in cortical development and provide a new target for the study of associated syndromes and disorders.

"The brain is actively constructed in embryonic stages. Neural progenitors produce many neurons, and their genomic DNA is constantly processed. Defects in Pol function could be a new target for explaining cortical developmental disorders."

Explore further: CD38 gene is identified to be important in postnatal development of the cerebral cortex

More information: Kohei Onishi et al, Genome Stability by DNA polymerase in Neural Progenitors Contributes to Neuronal Differentiation in Cortical Development, The Journal of Neuroscience (2017). DOI: 10.1523/JNEUROSCI.0665-17.2017

Excerpt from:
Faulty DNA repair depresses neural development - Medical Xpress

Michael R. Bishop, MD, specializes in the diagnosis and treatment of lymphomas and leukemias. In particular, he cares for patients with hematologic malignancies that have not responded to first-line treatments. An expert in hematopoietic stem cell transplantation (bone marrow transplantation), Dr. Bishop and his team are working to address the unique social, economic, physiological and biological issues that patients face while undergoing this treatment.

His research focuses on the prevention and treatment of relapse after stem cell transplantation. Relapse is the primary cause of treatment failure and death after stem cell transplantation. He has served as the primary investigator on studies designed to prevent and treat disease recurrence after transplantation. Specifically, he works on ways to enhance immune effects of the transplanted cells against cancer.

Bishop has authored more than 150 peer-reviewed articles, in addition to more than 30 book chapters and two books on cancer treatment and research. He also serves on the editorial board of numerous scientific journals, including Biology of Blood and Marrow Transplantation.

He previously served as a senior investigator and as the clinical head of stem cell transplantation for the National Cancer Institute at the National Institutes of Health.

Two of his patients, now in remission, have told their story publicly:

Motivating a Malignant Immune System

The CAR T-cell Chicago story: One year later

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Dr. Bishop @Uchicagomed Knows CAR T-Cell Therapy Backward and Forward - Newswise (press release)

The US Food and Drug Administration indicated Monday that it will be increasing oversight and enforcement to prevent the use of potentially dangerous and unproven stem cell treatments.

The US Food and Drug Administration indicated Monday that it will be increasing oversight and enforcement to prevent the use of potentially dangerous and unproven stem cell treatments.

The US Food and Drug Administration indicated Monday that it will be increasing oversight and enforcement to prevent the use of potentially dangerous and unproven stem cell treatments.

On its website, the agency posted awarning letterthat it sent last week to U.S. Stem Cell Clinic of Sunrise, Florida, accusing the clinic of selling unapproved and nonsterile stem cell treatments and injecting them intravenously or directly into patients spines.

The FDA also said Monday that it sent US marshals last week to StemImmune Inc. of San Diego to seizefive vials of a live virus vaccinereserved for people at high risk of smallpox. After being mixed with stem cells, the unapproved concoction was injected directly into the tumors of cancer patients at California Stem Cell Treatment Centers in Rancho Mirage and Beverly Hills, the FDA said.

Chief Science Officer Kristin Comella of U.S. Stem Cell wrote in astatementthat the company is not violating the law as it is currently written.

It is inappropriate and harmful to state that our clinic is not sterile as we are completely compliant with the regulations for surgical procedures, she wrote. The strict regulations mentioned in the warning letter required to manufacture drugs are not applied to clinics or hospitals.

Comella wrote that the surgical procedure used by the clinic is not subject to the rules for tissue banks which include minimal manipulation and homologous use as described in current federal regulations. She concluded, our clinic is not violating the law as it is currently written.

StemImmune is fully cooperating with the FDA about the development of its stem cell-based investigational cancer therapy, Ulrike Szalay, a spokeswoman for the company, wrote in an email. We look forward to continuing our dialogue with the FDA.

Dr. Elliot B. Lander, co-founder of the California Stem Cell Treatment Center, said it was voluntarily participating in studies for late-stage, no-options cancer patients that had been approved by institutional review boards. The boards are basically bioethics committees, he said, and the one overseeing this study was formed by the International Cell Surgical Society.

We provided our services gratis, for compassionate purposes, and no patient was ever charged, he said. Everything to protect patient safety was done appropriately.

Though the vaccine seized by the FDA falls under the domain of StemImmune, Lander said, we provided autologous stem cells to help carry a viral agent into the cancers. All of the early safety study patient data was submitted in detail to the FDA several months ago. It did show tremendous safety and no adverse events related to the vaccine or cell therapy.

The FDA commissioner, Dr. Scott Gottlieb, issued astatementMonday warning of additional actions in the coming months against a larger pool of actors whose unproven and unsafe products put patients at significant risk.

The International Society for Stem Cell Research commended the FDA for its policy direction and enforcement efforts. President Hans Clevers said the society has been very concerned about reports of patients using unproven stem cell therapies.

Many of these patients have suffered great harm, and even death as a result of using unproven stem cell therapies, Clevers said in a statement. We are hopeful that increased regulatory enforcement against clinics offering unproven treatments will deter this practice and help protect patients.

Ive directed the FDA to launch a new working group to pursue unscrupulous clinics through whatever legally enforceable means are necessary to protect the public health, Gottlieb wrote. We have examples where some of these unproven treatments have clearly harmed patients.

This year, a paper published in the New England Journal of Medicine recounted how three women, ages 72 to 88, with macular degeneration were left blind after a stem cell treatment at an unnamed clinic in Florida in 2015.

I wish it hadnt taken this long, said Leigh Turner, associate professor at the Center for Bioethics at the University of Minnesota. This is a space where the FDA could have taken action four or five years ago as far as making this a policy priority.

Turner said he sees the steps announced Monday as both important and necessary, yet he remains skeptical.

There are important distinctions to be made, and the FDA seems to be making these distinctions in terms of suggesting that they are putting together this working group, a task force, going after businesses marketing unproven interventions, going after businesses making illegitimate or unwarranted claims about stem cell treatment, he said.

Stem cells, like other medical products, generally require FDA approval before they can be marketed. The FDA has not approved any stem cell-based products for use other than cord blood-derived cells, which are blood-forming stem cells, for certain diseases, according to theagencys website.

Gottlieb wrote in his statement its incumbent upon the FDA to make sure the existing legal and regulatory framework is properly defined, with bright lines separating individualized or tailored therapies surgeons are permitted to use from new treatments subject to regulation. Because the field of regenerative medicine is rapidly evolving, he said, close calls may be frequent between what constitutes an individualized treatment and what constitutes an unapproved, possibly harmful medical product.

Turner said Gottliebs statement allowed for a bit of slippage as far as what exactly the FDA is going to do and which businesses they are going to target.

Questions remain as to whether the warning letter is a sign of more letters to come and whether we will see a dramatic increase in such activities from the FDA, Turner said.

FDA spokeswoman Lyndsay Meyer wrote in an email that the agency will seek to take additional actions in the coming months as we address this field, and target those who are clearly stepping over the line.

Yet, Turner asked, what is enough to trigger FDA regulation? Are marketing campaigns and commercial activity enough?

Or do we actually require people being blinded before the FDA does something? he asked, noting that theres a considerable amount of uncertainty in terms of what we should expect in the months ahead. The statement itself doesnt provide clear answers to all those questions.

Susan L. Solomon, CEO of the New York Stem Cell Foundation, a nonprofit research organization, said via a spokesman that the regulation of these clinics is very difficult, so the announcement today that the FDA will be stepping up their oversight should be welcomed and applauded.

Overall, Turner said, the agencys actions should not give all stem cell treatments or doctors performing these regenerative therapies a bad name. There are already effective treatments. If we think about bone transplants as stem cell transplants, its standard of care for certain diseases, he said.

Solomon agreed: There are extremely promising studies and research using stem cells to treat macular degeneration, multiple sclerosis, diabetes and many other devastating diseases. I cannot emphasize enough how exciting and promising the research is.

However, anyone advertising a cure today is simply taking advantage of patients for their own financial gain, she said.

Turner acknowledges the difficulty for patients, who may not easily recognize which stem cell therapies are approved and beneficial and which are not.

If you see a business thats making all sorts of dramatic marketing claims across disease categories, claiming to use fat as a treatment for all sorts of indications, these are all signs to be wary of, he said.

The FDA offersadvice for consumers, Meyer noted, adding that anyone who exploits and deceives patients puts the entire field at risk.

Turner acknowledged stem cell treatments as a very promising area of research, and over time, he expects to see more FDA-approved therapies in the marketplace. The problem, he said, is that many American businesses making claims about stem cell treatments lack proper scientific safety and efficacy data.

Why, for example, didnt California Stem Cell Treatment Centers get a warning letter for all the other treatments they are doing? It leaves me a bit perplexed, he said. Why is the FDA so focused on these vials and not on the broader array of marketing claims that California Stem Cells was making?

Solomon said that by providing unproven treatments to chronically ill or injured patients, these clinics are not only taking advantage of patients, they are muddying the scientific waters of clinical trials that are trying to show whether a treatment does or does not work.

In its statements Monday, the FDA notes the handful of bad actors in the stem cell space, Turner observed. (Meyer repeated the FDAs assertion that its only a small number of unscrupulous actors who have seized on the clinical promise of regenerative medicine.)

Whereas I look and I see hundreds of companies, said Turner, who published apaperon the practice.

Ultimately, Turner is glad for the FDAs actions.

I hope this is a sign that the FDA is going to do a lot more and better regulate this market space so well see whether or not that happens, he said. Its easy to make these bold announcements. The question is going to be whether anything really comes of it.

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