StemCell Therapy

Opening a new era in cancer care, the Food and Drug Administration on Wednesday approved the first treatment that genetically engineers patients' own blood cells into an army of assassins to seek out and destroy childhood leukemia.

The CAR-T cell treatment developed by Novartis Pharmaceuticals Corp. and the University of Pennsylvania is the first type of gene therapy to hit the U.S. market and one in a powerful but expensive wave of custom-made living drugs being tested against blood cancers and some tumors.

FDA called the approval historic.

This is a brand new way of treating cancer, said Dr. Stephan Grupp of Children's Hospital of Philadelphia, who treated the first child with CAR-T cell therapy, a girl who had been near death but now has been cancer-free for five years. That's enormously exciting.

CAR-T treatment uses gene therapy techniques not to fix disease-causing genes but to turbocharge T cells, immune system soldiers that cancer too often can evade. Researchers filter those cells from a patient's blood, reprogram them to harbor a chimeric antigen receptor that zeroes in on cancer, and grow hundreds of millions of copies. Returned to the patient, the revved-up cells can continue multiplying to fight disease for months or years.

Novartis said it would charge $475,000 for the treatment, made from scratch for every patient. But the company said there would be no charge if the patient didn't show a response within a month.

We're entering a new frontier in medical innovation with the ability to reprogram a patient's own cells to attack a deadly cancer, FDA Commissioner Scott Gottlieb said.

This first use of CAR-T therapy is aimed at patients desperately ill with a common pediatric cancer acute lymphoblastic leukemia that strikes more than 3,000 children and young adults in the U.S. each year. While most survive, about 15% relapse despite today's best treatments, and their prognosis is bleak.

In a key study of 63 advanced patients, 83% went into remission. It's not clear how long that benefit lasts: Some patients did relapse months later. The others still are being tracked to see how they fare in the long term.

Still, a far higher percentage of patients go into remission with this therapy than anything else we've seen to date with relapsed leukemia, said Dr. Ted Laetsch of the University of Texas Southwestern Medical Center, one of the study sites. I wouldn't say we know for sure how many will be cured yet by this therapy. There certainly is a hope that some will be.

Most patients suffered side effects that can be grueling, even life-threatening. An immune overreaction called cytokine release syndrome can trigger high fevers, causing plummeting blood pressure and, in severe cases, organ damage, requiring special care to tamp down those symptoms without blocking the cancer attack. Also Wednesday, the FDA designated a treatment for those side effects.

The new CAR-T therapy might replace bone marrow transplants that cost more than half a million dollars, said Grupp, who led the Novartis study.

I don't want to be an apologist for high drug prices in the U.S., Grupp stressed. But if it's the last treatment they need, that's a really significant one-time investment in their wellness, especially in kids who have a whole lifetime ahead of them.

Initially, Novartis' CAR-T version to be sold under the brand name Kymriah will be available only through certain medical centers specially trained to handle the sophisticated therapy and its side effects. Patients' collected immune cells will be frozen and shipped to a Novartis factory in New Jersey that creates each dose, a process the company says should take about three weeks.

While this first use of CAR-T therapy only is aimed at a few hundred U.S. patients a year, it's being tested as a treatment for thousands more.

Kite Pharma Inc.'s similar CAR-T brand, developed by the National Cancer Institute, is expected to win approval later this year to treat aggressive lymphoma, and Juno Therapeutics and other companies are studying their own versions against blood cancers including multiple myeloma.

On Monday, Gilead Sciences Inc. announced that it was buying Santa Monica-based Kite in an $11.9-billion deal.

Analysts said the eventual pricing of the Novartis treatment could be an advantage for Kite.

Since these therapies are unbelievably effective for leukemia, Novartis pricing power is high, said Thomas Shrader, biotechnology analyst at Stifel. That means Kite could piggyback off Novartis price, even though its therapy is aimed at non-Hodgkins lymphoma, which has a lower response rate to the therapy than leukemia does.

Scientists around the country also are trying to make CAR-T therapies that could fight more common solid tumors such as brain, breast or pancreatic cancers a harder next step.

Times staff writer Samantha Masunaga contributed to this report.

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UPDATES:

9:55 a.m.: This article was updated with the price of Novartis therapy.

9:45 a.m.: This article was updated with comments from medical experts and a financial analyst, as well as more details about CAR-T therapy.

This article was originally published at 8:25 a.m.

More:
FDA approves historic 'living drug' treatment to fight childhood leukemia - Los Angeles Times

Nanoparticles (orange) deliver temporary gene therapy to immune cells (blue) to give them disease-fighting tools. (Fred Hutch Illustration / Kimberly Carney)

CAR T immunotherapies are all the rage in the medical community, reprogramming a patients immune system to fight cancer. For some patients, theyve produced near-miraculous recoveries, and they could be a huge breakthrough in cancer treatment.

The business community is taking note as well: Kite Pharma, a biotech company developing these therapies, announced a deal to be acquired for $11.9 billion on Monday, sending stock prices of Seattle immunotherapy developer Juno Therapeuticsskyrocketing.

But there are still giant pitfalls to using the therapies on a large scale because they are incredibly complex and expensive to produce. Researchers from Seattles Fred Hutchinson Cancer Research Center are taking the problem head-on with new hit-and-run gene editing technology.

In a study published Wednesday in the journal Nature Communications, researchers led by Dr.Matthias Stephan reported they have developed a nanoparticle delivery system that can temporarily alter cells so they are able to fight cancer and other diseases.

The best part? The treatment is a powder that just needs to be mixed with water to activate and even better, it could be an essential breakthrough in making cutting-edge medical technology affordable for patients.

Stephan told GeekWire in a previous piece on the technology that his goal is to make immunotherapy so easy to access that it replaces chemotherapy as the front-line treatment for cancer.

What I envision is like the Walgreens flu shot scenario, or you go to your doctor and you get hepatitis B shot, he said at the time. You go there every Friday, and thats it.

We realized in order to outcompete chemotherapy, we have to design something that is at least as affordable and can be manufactured at large scale by one biotech company and shipped out to local infusion centers, Stephan said. At the moment, CAR T cell therapies must be made individually for each patient in specialized labs.

Heres how the new tech works: The nanoparticles designed by Stephan and his team act like shipping containers for bundles of mRNA, the molecules that tell cells how to build disease-fighting proteins. The nanoparticles also have molecules attached to the outside to help them find the right kind of cells, like a shipping label on a package.

When the mRNA is delivered to the cell, it prompts the cell to grow disease-fighting features, like the chimeric antigen receptor in CAR T cells that help them identify and kill cancer.Researchers said the technology could potentially be used to develop treatments for HIV, diabetes and other immune-related diseases.

In the short run, the tech could help researchers discover new treatments and therapies in the lab. It could one day be used in hospitals and clinics around the world, but will first need to undergo extensive clinical trials to ensure the tech is effective and safe to use in humans.

View post:
New 'hit-and-run' gene editing tool temporarily rewrites genetics to treat cancer and HIV - GeekWire

31 August 2017 08:22

Almost all men over 60 and women over 75 are eligible for statins

A common genetic variant could be the reason some people suffer from aches and pains when taking statins, according to new research.

The study could lead to a screening method to help identify those patients who are most likely to have a bad reaction to the drugs.

Statin intolerance doubles

Millions of Britons take statins every year to lower cholesterol and reduce their risk of heart attacks and strokes. However, between 7% and 29% of users suffer from sore muscle symptoms, which can in some cases stop them using the pills.

Research undertaken at the University of Dundee found that statin intolerance was doubled when patients carried two identical copies of a common variant of the LILRB5 gene, which has an immune system and muscle repair role.

The team also confirmed that some people are genetically more likely to suffer from aching muscles regardless of whether they are taking statins.

Lead scientist Professor Colin Palmer said: "We found that there are people in the general population who carry a genetic factor that predisposes them to muscle aches. If these people are put on statins, they might discontinue their medication in the erroneous belief that it is the statin that is making their muscles ache."

Sub-group of patients

He added that the researchers also identified a genetic sub-group of patients who are susceptible to statin-specific muscle ache, however, at this stage the reason for this is not understood.

Professor Palmer suggested that in the future prospective statin users could be tested for key genetic variants, including LILRB5.

Almost 12,000 statin users took part in the Genetics of Diabetes Audit and Research Tayside Scotland (GoDARTS) study. The findings will appear in the European Heart Journal.

People with long-term, pre-existing conditions can arrange medical travel insurance should they need to travel.

Continued here:
Genetics could be behind statin side effects - World First Travel Insurance

Spinal injuries are oftenpermanent, but new research suggests such injuries may be healed, at least in part.Researchers were able to stimulate limb function in paralyzed mice by implanting human stem cells into theirspinal cords. We're not close to repeating the test in people, but the study shows it may be possible some day with further research.

The University of California-San Diego team grafted human neural stem cells (NSCs) into the spinal cord injuries of mice who were purposely injuredto impair the use of their front legs. The stem cells grew slowly, yet steadily, over the course of 18 months, retaining their original function despite being in a strange and challenging environment for an extended period of time. Whats more, eventually the rodents were able to use their front legs again.

"The bottom line is that clinical outcome measures for future trials need to be focused on long time points after grafting," said study researcher Mark Tuszynskiin a recent statement. Relying on shorter time frames might produce misleadingly negative results considering how long it takes neural stem cells to develop, he added.

For the study, the team used H9 human NSCs, which are a type of stem cell derived from human embryonic stem cells, as commonly used in scientific research, the statement reported. They then grafted these human stem cells into the spinal injuries of mice. The researchers observed the rodents recovery over the course of 18 months, noting that significant cellgrowth did occur soon after grafting, and continued up to a year after the initial implantation.

The most important observation was that these cells were able to continue to do what they were designed to doregrow neural cellsdespite the fact that they were transplanted into an entirely different species. This suggests the cells have resilience and similar experiments mayalso work in human subjects.

Before you get too excited about these results, the researchers emphasized that there were a number of caveats. First, humans and mice are entirely different species, and though the results observed in the rodents are promising, we don't know if they could be repeated in people.

Also, the researchers observed that some astrocytes, star-shaped neural cells associated with electrical impulse transmission, did migrate from the original implantation site to other areas of the rodents. These brain cells are classified as glial cells, which are noted to lead to devastating and difficult to treat cancers when they are dysregulated, Harvard University reported. However, there were no tumors or abnormal growths observed in the mice in the study and the researchers are trying to figure out way to make sure cancer doesn't develop.

Ultimately, the team believe that these results stand as a good foundation on which to buildfurther research.

Success, it would seem, will take time," concluded Tuszynski.

Source: Lu P, Ceto S, Wang Y, et al. Prolonged human neural stem cell maturation supports recovery in injured rodent CNS. The Journal of Clinical Investigation . 2017

See the original post:
Stem Cell Graft Repairs Spinal Cord Injury, Helps Paralyzed Mice Move Again - Medical Daily

AsianScientist (Aug. 30, 2017) - In a study published in Cell Stem Cell, scientists in Japan and Switzerland have found that bacterial infections can stress blood-producing stem cells in the bone marrow and reduce their ability to self-replicate.

When a person becomes infected with a virus or bacteria, immune cells in the blood or lymph react to the infection. Some of these immune cells use sensors on their surfaces, called Toll-like receptors (TLR), to distinguish invading pathogens from molecules that are expressed by the host. By doing so, they can attack and ultimately destroy pathogens thereby protecting the body without attacking host cells.

Bone marrow contains hematopoietic stem cells which create blood cells, such as lymphocytes and erythrocytes, throughout the lifetime of an individual. When infection occurs, a large number of immune cells are activated and consumed. Hence, it is necessary to replenish these immune cells by increasing blood production in bone marrow.

Recent studies have revealed that immune cells are not the only cells that detect the danger signals associated with infection. Hematopoietic stem cells also identify these signals and use them to adjust blood production. However, little was known about how hematopoietic stem cells respond to bacterial infection or how it affected their function.

In this study, researchers from Kumamoto University and the University of Zurich analyzed the role of TLRs in hematopoietic stem cells upon bacterial infection, given that both immune cells and hematopoietic stem cells have TLRs.

To generate a model of bacterial infection, researchers injected one of the key molecules found in the outer membrane of gram negative bacteria and known to cause sepsislipopolysaccharide (LPS)into lab mice. They then analyzed the detailed role of TLRs in hematopoietic stem cell regulation by combining genetically modified animals that do not have TLR and related molecules, or agents that inhibit these molecules.

The results showed that LPS spread throughout the body, with some eventually reaching the bone marrow. This stimulated the TLRs of the hematopoietic stem cells and induced them to proliferate. They also discovered that while LPS promoted stem cell proliferation, it also induced stressed the stem cells, impairing their ability to successfully self-replicate and resulting in diminished blood production. Similar results were obtained after infection with Escherichia coli bacteria.

Fortunately we were able to confirm that this molecular reaction can be inhibited by drugs, said Professor Hitoshi Takizawa of Kumamoto University who led the study. The medication maintains the production of blood and immune cells without weakening the immune reaction against pathogenic bacteria. It might be possible to simultaneously prevent blood diseases and many bacterial infections in the future.

The article can be found at: Takizawa et al. (2017) Pathogen-Induced TLR4-TRIF Innate Immune Signaling in Hematopoietic Stem Cells Promotes Proliferation but Reduces Competitive Fitness.

Source: Kumamoto University.Disclaimer: This article does not necessarily reflect the views of AsianScientist or its staff.

Originally posted here:
Bacterial Infection Stresses Blood Stem Cells - Asian Scientist Magazine

Gene therapy is the use of DNA as a potential therapy to treat a disease.In many disorders, particularly genetic diseases caused by a single genetic defect, gene therapy aims to treat a disease by delivering the correct copy of DNA into a patients cells.The healthy, functional copy of the therapeutic gene then helps the cell function correctly.

In gene therapy, DNA that encodes a therapeutic protein is packaged within a vector, often a naked virus, which is used to transfer the DNA to the inside of cells within the body. Gene therapy can be delivered by a direct injection, either intravenously (IV) or directly into a specific tissue in the body, where it is taken up by individual cells. Once inside cells, the correct DNA becomes expressed by the cell machinery, resulting in the production of therapeutic protein, which in turn treats the patients disease and can provide long-term benefit.

Abeona is developing next generation adeno-associated virus (AAV) gene therapies. Viruses such as AAV are utilized because they have evolved a way of encapsulating and delivering one or more genes of the size needed for clinical application, and can be purified in large quantities at high concentration. Unlike AAV vectors found in nature, the AAV vectors used by Abeona have been genetically-modified such that they do not replicate. Although the preclinical studies in animal models of disease demonstrate the promising impact of AAV-mediated gene expression to affected tissues such as the heart, liver and muscle, our programs use a specific virus that is capable of delivering therapeutic DNA across the blood brain barrier and into the central nervous system (CNS), making them attractive for addressing lysosomal storage diseases which have severe CNS manifestations of the disease.

Lysosomal storage diseases (LSD) are a group of rare inborn errors of metabolism resulting from deficiency in normal lysosomal function. These diseases are characterized by progressive accumulation of storage material within the lysosomes of affected cells, ultimately leading to cellular dysfunction. Multiple tissues ranging from musculoskeletal and visceral to tissues of the central nervous system are typically involved in disease pathology.

Since the advent of enzyme replacement therapy (ERT) to manage some LSDs, general clinical outcomes have significantly improved; however, treatment with infused protein is lifelong and continued disease progression is still evident in patients. Thus, viral gene therapy may provide a viable alternative or adjunctive therapy to current management strategies for LSDs.

Our initial programs are focused on LSDs such as Mucopolysaccharidosis (MPS) IIIA and IIIB, also known as Sanfilippo syndromes type A and type B. MPS III is a progressive neuromuscular disease with profound CNS involvement. Our lead product candidates, ABO-101 and ABO-102, have been developed to replace the damaged, malfunctioning enzymes within target cells with the normal, functioning version.

Delivered via a single injection, the drug is only given once.

Here is the original post:
Gene Therapy - Abeona Therapeutics

Personalized cancer treatments known as CAR-T cells (chimeric antigen receptor T cells) have dominated the headlines lately, thanks to Novartis tisagenlecleucel, which won an early approval from the FDA for the treatment of leukemia on Aug. 30. But CAR-T treatments are labor-intensive and expensive to make, and they can attack healthy tissues in the body, leading to dangerous side effects.

Scientists at the Fred Hutchinson Cancer Research Center have developed a tool that they believe could address both those shortcomings of CAR-T and other forms of cell engineering. They have invented nanoparticles that deliver proteins to cells, which in turn edit those cells genes temporarily. Lead author and bioengineer Matthias Stephan describes it as hit-and-run gene therapy, and he believes the technique will streamline the manufacturing of cell-based therapies.

Heres how it works: The nanoparticles home in on specific cells, such as the T cells in the immune system. They then deposit messenger RNA (mRNA) to those cells, which triggers short-term changes in the proteins the genes produce. The technology does not permanently change the DNA, but it makes enough of an impact on it to produce a therapeutic outcome.

RELATED: Can CAR-T cancer treatments be fine-tuned to avoid toxic side effects?

Whats more, the nanoparticles can be freeze-dried and then activated with a small amount of water. They really let you fulfill all your wishes as a genetic engineer because you can pack in all your different [gene-therapy] components and further improve the therapeutic potential of your cell product without additional manufacturing steps, Stephan said in an article posted on Fred Hutchs website.

Stephans team proved out their concept by testing the nanoparticles in three different cell-engineering applications, one of which was CAR-T. Currently, CAR-T treatments are made by giving T cellsgenes that teach them to destroy cancer cells. The Fred Hutch scientists used their nanoparticles to remove a different gene from T cellsone that normally prompts them to attack healthy tissue.

Then they tried enhancing the CAR-T cells in a different manner. They temporarily gave them genes that have the potential to make central memory T cells, which are able to survive over the long term, remembering their cancerous targets and attacking them should they ever resurface.

The scientists tested their engineered CAR-T cells in mouse models of leukemia and found that the animals that received them lived twice as long as mice that got conventional CAR-T cells. They also tested the nanoparticles in two other cancer-related applications of gene therapy.

Despite all the excitement over CAR-T, concerns about side effects continue to dog the field. A dangerous immune reaction known as a cytokine storm has been seen in trials of both Novartis treatment and Axi-Cel, a CAR-T from Kite Pharma, which is being acquired by Gilead. The third player in the CAR-T field, Juno Therapeutics, saw its late-stage trials delayed when some patients died of neurological side effects.

Fred Hutch scientists have been working on other techniques for improving CAR-T. In December, a set of researchers there who receive funding from Juno announced positive results from a trial of a fine-tuned CAR-T treatment in patients with chronic lymphocytic leukemia (CLL). Instead of using just one type of CAR-T, the team combined two specially selected cell subtypes into one treatment. They also announced they had identified biomarkers that they believe can be used to predict which patients are likely to have severe reactions to the treatment.

Stephans team is now collaborating with several companies to fine-tune CAR-T treatments for cancer, according to Fred Hutch. And they believe their freeze-dried nanoparticles may prove useful in developing treatments for a range of other diseases, too, including HIV and blood disorders caused by defective hemoglobin.

Read the original:
'Hit-and-run' gene therapy nanoparticles could enhance CAR-T ... - FierceBiotech

(KMSP) - Charlotte Gifford is almost 5 years old with dreams of looking like her favorite Disney princess.

Rapunzel, because she has the long hair, says Charlotte.

Charlotte lost her own long locks after being diagnosed last March with b-cell acute lymphoblastic leukemia, the most common form of leukemia to develop in children and young adults.

Once we got the word it was leukemia. Our world came down it crumbled, says Erica Gifford, Charlottes mom.

Erica and Adam Gifford are thrilled the Food and Drug Administration announced approval of the first gene therapy in the United States. Car t-cell gene therapy can be used for Charlottes type of leukemia, affecting anyone under 25 years old.

Its a new treatment with an entirely different flavor, says Dr. Daniel Weisdorf, Professor of Medicine and Chief Division of Hematology, Oncology and Transplantation. Its not a drug, but a way to take the patients own cells, their normal cells, and engineer the cells so they will specifically and uniquely attack the leukemia cells.

Weisdorf witnessed success from this treatment, as some patients from University of Minnesota Health and Masonic Cancer Center were part of the clinical trials.

The treatments are remarkably effective but complicated to administer because the patients get very sick, says Weisdorf.

The Leukemia and Lymphoma Society has helped fund research for decades, and today, Executive Director Teri Cannon is celebrating a major medical milestone.

Nowadays 90 percent of young people diagnosed with leukemia survive, says Cannon, Those kids that relapse and standard therapy doesn't work for them, 83 percent of the kids who have used car t-therapy in the clinical trial have survived. So that's going to bring us a lot closer to that 100 percent that is going to make parents happy.

As for Charlotte, she's doing well and in remission. Hopefully she'll never need the this newly approved gene therapy reserved for patients whose cancer has returned. For the Giffords, this major medical advancement offers their family and others optionsand hope.

You dont know how important it is until your own child is diagnosed with cancer, says Gifford.

Charlotte continues steroid, chemo and physical therapy. Her parents have started a Go Fund Me Page.

View post:
FDA approves gene therapy that could fight type of leukemia - KMSP-TV

The US Food and Drug Administration (FDA) on Wednesday approved a "historic" treatment that genetically engineers a patient's own immune cells to fight childhood leukemia.

The procedure, known as CAR-T cell therapy, takes a patient's immune cells, or T-cells and white blood cells, and genetically modifies them to give a power boost to viciously target leukemia.

Once transfused back into the patient, the immune cells then attack leukemia cells for months or even years.

"We're entering a new frontier in medical innovation with the ability to reprogram a patient's own cells to attack a deadly cancer," said FDA Commissioner Scott Gottlieb. "New technologies such as gene and cell therapies hold out the potential to transform medicine and create an inflection point in our ability to treat and even cure many intractable illnesses."

Read more:Fighting cancer with methadone - making chemotherapy more powerful

Last resort

The treatment was developed by Novartis Pharmaceuticals and the drug is known as Kymriah (tisagenlecleucel).

The immune system-altering treatment was approved for patients aged3 to 25 with a blood cancer called acute lymphoblastic leukemia (ALL),the most common form of childhood cancer in the US.

About 3,100 people under the age of 20 are diagnosed with ALL each year in the United States.Around20 percent of those with ALL fail to respond to traditional drug, bone marrow transplants and chemotherapy treatments. Typically, young patients with ALL who fail chemotherapy have only a 30 percent chance of survival.

The breakthrough treatment would only be used if the disease failed to respond to standard treatment.

The therapy would cost $475,000 (400,000 euros) per dose, but Novartis said anyone who didn't respond within a month would not have to pay. Typical treatments of ALL, such as bone marrow transplants, can run up to $800,000 in the first year.

Application to be filed in EU

According to studies, about 83 percent of patients responded to CAR-T cell therapy went into remission after three months.

The treatment does carry potential side effects, including an immune overreaction, fever, high blood pressure, neurological reactions, acute kidney injury and decreased oxygen.

Questions also remain about the drug's possible long-term side effects and ability to preventALL from coming back.

Due to their age, many of those suffering from the disease would be covered by their parent's health insurance or Medicare in the US.

The procedure is best known for treating a now 12-year-old girl named Emily Whitehead, who has been cancer-free for five years after being the first child to receive the experimental therapy.

An application for CAR-T is expected to be filed with the European Medicines Agency by the end of the year.

cw/cmk(AP, AFP)

Read the rest here:
US approves breakthrough gene therapy for childhood leukemia - Deutsche Welle

Hurricane Harvey Flooding Claims 6 Members Of Same Family

Members of the Saldivar family ranging from 6 to 84 perished inside a van swept by currents as they sought higher ground. (Patch)

U.S. OK's First Gene Therapy Treatment For Childhood Leukemia

The Food and Drug Administration on Wednesday signed off on the first gene therapy treatment in the U.S. that doctors say uses a patient's own cells to seek out and destroy deadly childhood leukemia. (Patch)

Legendary Basketball Coach Rollie Massimino Dead At 82

Legendary college basketball coach Rollie Massimino, who led the Villanova Wildcats to a storied NCAA Championship in 1985 against Georgetown University, has died, according to the chancellor of Keiser University, who expressed "heartfelt sadness" on behalf of the school. Massimino served as head coach of the Keiser men's basketball team for the past 12 years. He was 82. (Patch)

World's Worst Babysitters Leave 9-Month-Old At Long Island Casino

Three people, including two teens, were arrested after they left a 9-month-old baby at a Long Island casino Tuesday, police said. The trio was supposed to be babysitting the child, but a dispute with the baby's mother quickly sent things haywire. (Patch)

Indiana Couple Completes Decades-long Goal To Visit Every Cracker Barrel

An elderly couple from Goshen finished a decades-long mission over the weekend: to visit every Cracker Barrel location, WGN-TV reports. Ray and Wilma Yoder flew out to Tualatin, Oregon, so they could visit and eat at their 645th Cracker Barrel on Monday morning. (Patch)

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Deer In The Headlights Detained On San Francisco-Oakland Bay Bridge (Patch)

Girl, 7, Halfway Through Hug-A-Cop Tour Across USA

Rosalyn Baldwin is on a mission. Her goal? To hug a law enforcement officer in every state of the USA. Spanning 21 states so far, the seven-year-olds journey has been shared via her website, which is maintained by her two sisters, OceanRose and Scholar24, as well as her Facebook page. (Patch)

Harvey Slams Southeast Texas: Helicopter Rescues; Heartbreaking Scene As Toddler Clings To Mother

People are being plucked from homes by helicopter, boats are going door to door and the governor is sending the National Guard to the area. And in Beaumont, one of the more heartbreaking scenes of the storm unfolded when rescuers saved a toddler who was clinging to the body of her mother, who had died. (Patch)

All Houston ISD Students To Receive 3 Free Meals Per Day This School Year

All Houston ISD students will eat for free during the 2017-18 school year thanks to a waiver of the required application process for the National School Lunch/Breakfast Program from the United States Department of Agriculture and the Texas Department of Agriculture. (Patch)

Hurricane Harvey Scam Alert: Beware Of ICE, HSI Impersonators

Houston Police Chief Art Acevedo said there are reports that people impersonating Homeland Security Investigations special agents. Acevedo said they are knocking on doors in the Houston area telling residents to evacuate in the aftermath of Hurricane Harvey presumably so these impostors can rob the empty homes, he said. (Patch)

Gas Prices Rising Thanks To Harvey, Says AAA

While Hurricane Harvey was bearing down on Texas and the Gulf Coast, drivers looking to fill up their tanks were greeted at the gas station with one of the largest one-week surges in gas prices this summer, according to AAA. Prices increased 4 cents to a nationwide average of $2.37 per gallon. (Patch)

Parade For Purple Heart Recipient Planned In Illinois

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Photo of Overjoyed Mom Becomes A Social Media Sensation

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This Day In History

1422 King Henry V of England dies of dysentery while in France. His son, Henry VI becomes King of England at the age of 9 months.

1864 During the American Civil War, Union forces led by General William T. Sherman launch an assault on Atlanta.

1897 Thomas Edison patents the Kinetoscope, the first movie projector.

1962 Trinidad and Tobago becomes independent.

1993 Russia completes removing its troops from Lithuania.

Famous Birthdays

AD 12 Caligula, Roman emperor

1928 James Coburn, American actor

1944 Roger Dean, English illustrator and publisher

1946 Tom Coughlin, American football player and coach

1970 Debbie Gibson, American singer-songwriter, producer, and actress

Photo: Former Villanova head coach Rollie Massimino looks on prior to the 2016 NCAA Men's Final Four National Championship game between the Villanova Wildcats and the North Carolina Tar Heels at NRG Stadium on April 4, 2016, in Houston. Credit: Streeter Lecka/ Staff/ Getty Images Sport/ Getty Images

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Originally published August 31, 2017.

Read more from the original source:
Harvey Haunts Family Leukemia Gene Therapy Massimino Dies: Patch Morning Briefing - Patch.com

FINDINGS

Scientists from UCLA and the Howard Hughes Medical Institute successfully used a gene that suppresses cholesterol levels as part of a treatment to reduce plaque in mice with a disorder called familial hypercholesterolemia. In a preclinical study, researchers found that the gene, LeXis, lowered cholesterol and blockages in the arteries, and the treatment appeared to reduce the build-up of fat in liver cells.

Familial hypercholesterolemia is an inherited condition characterized by extremely high levels of low-density lipoprotein cholesterol (commonly referred to as bad cholesterol) and an increased risk of early heart disease.

The LeXis gene belongs to a unique group of genes that until recently were considered junk DNA because scientists believed they served little purpose. However, evidence from the human genome project led to the discovery that genes like LeXis are actually active. The study of these genes,now referred to as long noncoding ribonucleic acids, or lncRNAs, is a rapidly evolving area in biology.

Researchers wanted to test whether a single injection of LeXis could slow the development of heart disease. To do so, they gave the mice either LeXis or a control gene, and fed them a 15-week diet consisting of food high in sodium and cholesterol the mouse equivalent of fast-food hamburgers and french fries. Researchers then measured the progression of heart disease.

In the next phase of the study, researchers intend to confirm the findings in larger animals and test the therapy in combination with currently available treatments.

Although previous research has shown that lncRNAs can be important, this is the first study to show that they could potentially be used to treat a human disease using gene therapy. Junk genes could one day offer a framework for treating people with familial hypercholesterolemia and other conditions that are otherwise very difficult to treat.

The papers authors are Xiaohui Wu, Zhengyi Zhang and Dr. Tamer Sallam of UCLA; and Dr. Peter Tontonoz, Marius Jones and David Salisbury of the Howard Hughes Medical Institute.

The study waspublished onlinein the journal Circulation.

The research was supported by grants from the National Heart, Lung, and Blood Institute; the American College of Cardiology; and the Lauren B. Leichtman and Arthur E. Levine UCLA Cardiovascular Discovery Fund.

Learn more about the cardiovascular research theme at UCLA.

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Gene therapy using 'junk DNA' could lower risk for heart disease - UCLA Newsroom

Newswise BIRMINGHAM, Ala. Biomedical engineering researchers will attack two banes of cardiovascular disease heart failure after heart attacks and the scourge of resistant high blood pressure with $4.8 million in National Institutes of Health grants that begin this fall.

One sign of the clinical significance of this research by the University of Alabama at Birmingham investigators are the percentile scores that Jianyi Jay Zhang, M.D., Ph.D., and Gangjian Qin, M.D., received in those two NIH grant applications.

Zhangs plan to dissect the mechanisms of electromechanical integration of a human heart-muscle patch to aid survival and stability of the patch garnered a 1 percentile score, the highest possible. Qins plan to dissect a novel molecular pathway in endothelial cells of arteries that appears to regulate contractile function and blood pressure has significant potential to improve human health from the disease and death caused by high blood pressure, NIH reviewers said, and Qin received a 2 percentile score.

Zhang, chair and professor of the UAB Department of Biomedical Engineering and holder of the T. Michael and Gillian Goodrich Endowed Chair of Engineering Leadership, will receive $2.5 million over four years. Qin, professor of biomedical engineering and director of the Molecular Cardiology Program, will receive $2.3 million over four years.

Zhang came to UAB in 2015 from the University of Minnesota Medical School with the goal of moving his work with engineered heart patches into human use within seven years. As chair of Biomedical Engineering, a joint department of the UAB School of Medicine and the UAB School of Engineering, Zhang has recruited top researchers, and he also was awarded $11 million of NIH funding in 2016 $8 million of which is shared in collaborations with University of Wisconsin and Duke University researchers.

One of the recent recruits to biomedical engineering is Qin, who serendipitously discovered a novel and fascinating line of research that may lead to new drugs for treatment-resistant high blood pressure, where existing blood pressure drugs are ineffective. People with resistant high blood pressure have increased risk of strokes, heart attacks, heart failure and arterial aneurysms, and high blood pressure is a leading cause of chronic kidney failure. Even moderately elevated arterial blood pressure shortens life expectancy.

At the time, Qin was interested in the often fatal heart failure that occurs months or years after heart attacks. He reasoned that growth of new blood vessels into the damaged heart tissue of the left ventricle could be boosted by altering the amounts of cell-cycle regulators in the E2F family of transcription factors, to speed division of cells in the endothelial tissue of arteries.

When he deleted one of the eight E2Fs that are found in mice and humans E2F2 it had no effect on cell growth. But unexpectedly, we found a striking function, Qin said. If you delete E2F2, the vessel is more contractile. It becomes rigid and hard, and this contributes to high blood pressure.

So we had a question: How does E2F2 interact with other molecules to regulate blood pressure? Qin did pull-down experiments with E2F2, where other proteins are flowed past tethered E2F2 molecules to see if any would bind. He found that a kinase enzyme called Sam68 did bind to the transcription factor.

When he knocked out the gene for Sam68 in mice, they had low blood pressure.

Ultimately, a series of experiments in Qins lab and observations of other laboratories suggested a previously unknown mechanism of blood pressure control that involves E2F2/Sam68 and the expression of endothelial converting enzyme 1b. ECE-1b affects the levels of peptides that constrict blood vessels and raise blood pressure. Dysregulation of this pathway may contribute to blood pressure disorders, especially hypertension.

Despite a strong correlation, the E2F2/Sam68-ECE-1b pathway has not explicitly been linked to blood pressure regulation, and the mechanisms of how Sam68/E2F2 signaling regulates ECE-1b expression and blood vessel function remain uncharacterized.

Qin will use his new grant to search for the link to blood pressure regulation and characterize the mechanisms. His research could provide the missing links between the results of large-scale genomewide association studies of human high blood pressure and its pathogenesis namely how dysregulation leads to refractory hypertension.

Detailed knowledge of those steps would offer new targets for potential new drugs, which are especially needed to prevent or treat resistant hypertension.

Qin says he was attracted to UAB by the strong focus of clinicians and basic scientists on solving the clinical problem of hypertension, as well as the depth and breadth of cardiovascular disease research in biomedical engineering, the UAB Department of Pathology and the UAB Division of Cardiovascular Disease. He also has great interest in Zhangs research, where Qins past work in stem cell biology and cardiovascular science can contribute.

As measured by NIH funding, the UAB Department of Biomedical Engineering is the fourth-ranked biomedical engineering department among all departments that are jointly led by schools of medicine and engineering, according to the 2016 Blue Ridge NIH database.

The joint biomedical engineering departments ahead of UAB are at Stanford University, Johns Hopkins University, and Oregon Health and Science University. Those trailing UAB in the funding ranking are at the University of North Carolina-Chapel Hill, Emory University, University of Virginia, Case Western Reserve University, University of Colorado-Denver, University of Rochester, the University of Illinois-Chicago, Wake Forest University Health Sciences and State University of New York-Stony Brook.

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New Biomedical Engineering Grants Aim at Heart Failure and Resistant High Blood Pressure - Newswise (press release)

Researchers have discovered that a vaccine for shingles should be given to arthritis patients before they take drugs for the joint condition.

This is because sufferers are at a higher risk of developing shingles than other adults.

Furthermore, taking common arthritis drugs - such as tofacitinib and other antirheumatic drugs - can further increase this risk.

The results of two studies, published in the journal Arthritis & Rheumatology, found that the live varicella-zoster vaccine given to protect against shingles can induce an effective immune response in those suffering rheumatoid arthritis.

Researchers gave patients the vaccine several weeks before they started treatment with tofacitinib.

In one of the studies, 112 patients with rheumatoid arthritis were vaccinated and then split into two groups.

One group was given tofacitinib, and the other group received a placebo.

The vaccine triggered a robust immune response in all patients.

In those who received tofacitinib after vaccination there was no negative impact.

Whats more, patients treated with tofacitinib had a similar or even higher immune responses to the vaccine compared with patients given a placebo.

"We showed that the vaccine was adequately immunogenic in patients whether they were starting tofacitinib or placebo in a few weeks, and the immunogenicity and the response to the vaccine were similar to what we've seen outside the rheumatoid arthritis setting in general population studies," said Dr Kevin Winthrop, of Oregon Health and Science University.

However, the researchers also discovered that the vaccine should no be given to patients who have not had the chicken pox in the past.

In the second study, researchers looked to see if conventional synthetic disease-modifying antirheumatic drugs - such as csDMARDs, including methotrexate and chloroquine - or corticosteroids contributed to the increased risk of shingles linked to tofacitinib.

Research involved looking at 6192 patients with rheumatoid arthritis across 19 clinical trials.

They found shingles rates were lowest for patients taking tofacitinib without csDMARDs or corticosteroids and highest for those taking tofacitinib with csDMARDs and corticosteroids.

"If you want to lower shingles risk for rheumatoid arthritis patients, there are two strategies: one is vaccinating them and the other is getting them off steroids and methotrexate if you can," said Dr Winthrop.

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Rheumatoid arthritis treatment: Shingles vaccine should be given to ... - Express.co.uk

Xconomy Indiana

Eli Lilly is preparing to file again for FDA approval of rheumatoid arthritis drug baricitinib (Olumiant) by the end of Januarythis time with new information about the drugs safety and efficacy.

The FDA rejected the Indianapolis pharmaceutical companys initial drug application in April. In July, Lilly (NYSE: LLY) said discussions with the regulator included the possibility of requiring an additional study. Lilly said the FDA had expressed concern about blood clots observed during clinical trials and it asked the company to provide more information about dosing and safety.

Lilly will now try to provide the data that the FDA wants without conducting another study. The company said Wednesday that its new January goalmonths earlier than the 18-month delay that the company forecast in Julywas set after additional discussions with the regulator late this month. The filing will be a Class 2 resubmission, an agreement between a company and the FDA to start a six-month review cycle for a drug after the FDA receives the application. Under that timeline, an FDA decision could come by the end of July 2018.

Rheumatoid arthritis is an autoimmune disorder in which the immune system mistakenly attacks joint tissues, causing swelling and pain. Baricitinib is a once-daily pill that blocks proteins associated with this inflammation. In 2009, Lilly licensed global rights to the drug from Wilmington, DE-based Incyte (NASDAQ: INCY).

The European Commission approved baricitinib in February. That month, the New England Journal of Medicine published results from a Phase 3 clinical trial that compared the Lilly drug to adalimumab (Humira), an injectable biologic drug that generates blockbuster sales for AbbVie (NYSE: ABBV). Baricitinib beat AbbVies drug in the study, and Lilly was widely expected to win FDA approval.

In the months following the FDAs surprise rejection of baricitinib, regulators in Europe have agreed to update the drugs label with additional warnings for patients at risk for deep vein thrombosis and pulmonary embolism. Baricitinib was approved in Japan last month. Lilly says that the drugs label in that country includes precautions about the cardiovascular risks.

Photo by Flickr user tr0tt3rvia a Creative Commons license.

Frank Vinluan is editor of Xconomy Raleigh-Durham, based in Research Triangle Park. You can reach him at fvinluan [at] xconomy.com

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Eli Lilly Preps Rejected Rheumatoid Arthritis Drug for Resubmission - Xconomy

Provided by Harvard Health Publications

By Robert H. Shmerling

When I see patients with rheumatoid arthritis, Im often asked about diet. Are there foods that can help? Should I avoid certain foods?

The role of diet in arthritis

Whenever Im asked about diet for arthritis, my short answer is that with a few exceptions, there is no proven role for making dietary changes. In the case of rheumatoid arthritis, there is no clear evidence that eating more (or less) of any particular food will improve (or worsen) their symptoms or protect their joints. Of course, new research could change my answer.

And what about the exceptions? The most important is probably gout. Certain foods and beverages (such as organ meats and alcohol) can predispose to the condition or make it worse. Most people with gout are told to modify their diets, although for most the impact is small.

What about the microbiome?

There is enormous interest now in the role of the microbiome the vast numbers of microorganisms living within us in health and disease. And we know that diet affects the microbiome within the intestinal tract. It could turn out that what you eat may cause certain populations of bacteria in the intestinal tract to rise or fall. Since these bacteria may affect immune function, and since rheumatoid arthritis is an autoimmune condition, its possible that changes in the microbiome will affect the activity and severity of rheumatoid arthritis. Researchers are working hard to understand how the microbiome might affect autoimmune diseases, and how this new perspective could lead to better control of conditions such as rheumatoid arthritis.

Fish, fish oil, and rheumatoid arthritis

A number of studies have found that fish oil supplements or a diet rich in fish oils may be helpful in controlling the inflammation of rheumatoid arthritis. In addition, its possible higher intake of fish oil actually reduces the likelihood of developing the disease. Some studies have found that rheumatoid arthritis is less common in places where fish consumption is highest. However, fish oil is not routinely recommended, because its effect is modest and medications tend to be much more effective.

A new study resurrects the idea that fish oil (or at least fish consumption) might suppress the joint inflammation of rheumatoid arthritis. Researchers publishing in the medical journal Arthritis Care & Research report that the more fish a person consumes, the better the control of their arthritis. In this study, researchers analyzed data from 176 people with rheumatoid arthritis, comparing their reported intake of non-fried fish with the results of their joint examinations and blood tests. Heres what they found:

Those with the highest fish consumption (more than two servings per week) had the best control of their arthritis.

There was a dose effect. For minimal, low, or high fish consumption, the higher the intake, the better the arthritis.

The findings were noted even after accounting for other factors that might affect arthritis control, such as duration of disease and fish oil supplement use.

The fine print

This was a small study that found an association between fish consumption and control of rheumatoid arthritis. Thats not the same as finding that fish intake actually caused the improvement in arthritis. This is an important point because factors other than diet could explain the findings. For instance, its possible that people who eat fish regularly are generally more attuned to their health and take their medications more reliably than people who eat fish less often. Thats why the authors of this study do not conclude that everyone with rheumatoid arthritis should start eating more fish. What they do say is that additional research is needed.

One other point: the improvement in arthritis control noted among those who ate the most fish was modest, and so small that most patients probably wouldnt notice. Still, small improvements can add up, so even a small effect from a natural remedy that poses minimal risk is worth consideration.

What now?

Im hopeful that in the near future well have more definitive, larger, and long-term studies that examine the role of diet on rheumatoid arthritis and other types of joint disease. Until then, I think people with rheumatoid arthritis should consider increasing their intake of non-fried fish. It might be good for the joints. And eating fish may have other health benefits as well, especially if it replaces less healthy choices.

Dr. Robert H. Shmerling is Faculty Editor of Harvard Health Publications. Follow him on Twitter @RobShmerling.

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Fish consumption and rheumatoid arthritis: Natural remedy or just ... - KPCnews.com

However now experts have struck upon a new way to predict the progression of osteoarthritis in individual patients which could help improve treatment.

Scientists from the University of Eatern Finland said a cartilage degeneration algorithm can predict the onset of the condition.

They said the algorithm could facilitate clinical decision making in the treatment of osteoarthritis.

Osteoarthritis (OA) is a joint disease that deteriorates the articular cartilage.

Experts said the most important risk factors are ageing and overweight, and osteoarthritis is common especially in joints that are subject to heavy loading.

Osteoarthritis cannot be cured and the condition often leads to joint replacement.

Currently methods of looking at osteoarthritis in patients dont predict the progression of the condition.

Scientists tested the ability of a cartilage degeneration algorithm to predict the progression of osteoarthritis in patients and to grade the severity of their disease by using the Kellgren-Lawrence classification.

The algorithm was applied to 21 patients who were divided into three groups - those without osteoarthritis, those with mild osteoarthritis and patients with severe osteoarthritis.

The patients were divided into the groups based on their Kellgren-Lawrence grades defined experimentally after a four-year follow-up.

The algorithm was applied at the start of the follow-up, and the findings were compared against the four-year follow-up data.

Based on this information, the researchers found that the algorithm was able to categorise patients into their correct groups.

The degeneration algorithm is based on stresses experienced by the knee joint during walking, and these were simulated on a computer.

Scientists said the algorithm assumes that stresses exceeding a certain threshold during walking will cause degeneration in the cartilage of the knee.

Experts said this degeneration algorithm shows great potential in predicting progression of osteoarthritis in the knee in individual patients.

The new algorithm could help doctors make clinical decisions for patients with osteoarthritis.

Experts said it could help doctors slow down symptoms and even stop the progression of the disease.

The findings were published in Scientific Reports.

This comes after a woman hailed a miracle device she said stops arthritis pain.

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Arthritis symptoms: Algorithm could predict progression of ... - Express.co.uk

Acupuncture

Acupuncture is a technique in which practitioners stimulate specific points on the body - most often by inserting thin needles through the skin. It is one of the most effective practices used in traditional Chinese medicine. Acupuncture stimulates nerve fibers to transmit signals to the spinal cord and brain, activating the bodys central nervous system. The spinal cord and brain then release hormones responsible for making us feel less pain while improving overall health. Acupuncture may also: increase blood circulation and body temperature, affect white blood cell activity (responsible for our immune function), reduce cholesterol and triglyceride levels, and regulate blood sugar levels.

Aquatherapy

Aquatic Physical Therapy is the practice of physical therapy in a specifically designed water pool with a therapist. The unique properties of the aquatic environment enhance interventions for patients with neurological or musculoskeletal conditions. Aquatic therapy includes a wide range of techniques allowing patients to improve their balance, muscle strength and body mechanics. Aquatic therapy works to enhance the rehabilitation process and support effectiveness of stem cell treatment.

Epidural Stimulation

Hyperbaric Oxygen Therapy

Hyperbaric Oxygen Therapy (HBOT) is the medical use of oxygen at a level higher than atmospheric pressure. The equipment required consists of pressure chamber, which may be of rigid or flexible construction, and a means of delivering 100% oxygen into the respiratory system. Published research shows that HBOT increases the lifespan of stem cells after injection and provides an oxygen-rich atmosphere for the body to function at optimum levels.

Nerve Growth Factor (NGF)

Nerve growth factor (NGF) is a member of the neurotrophic factor (neurotrophin, NTFS) family, which can prevent the death of nerve cells and has many features of typical neurotransmitter molecules. NGF plays an important role in the development and growth of nerve cells. NGF is synthesized and secreted by tissues (corneal epithelial, endothelial, and corneal stromal cells), and it can be up-taken by sympathetic or sensory nerve endings and then transported to be stored in neuronal cell bodies where it can promote the growth and differentiation of nerve cells.NGF can exert neurotrophic effects on injured nerves and promote neurogenesis (the process of generating neurons from stem cells) that is closely related to the development and functional maintenance and repair of the central nervous system. It is also capable of promoting the regeneration of injured neurons in the peripheral nervous system, improving the pathology of neurons and protecting the nerves against hypoxia (lack of oxygen)/ischemia (lack of blood supply).

Nutrition Therapy

Occupational Therapy

Occupational therapy interventions focus on adapting the environment, modifying the task and teaching the skill, in order to increase participation in and performance of daily activities, particularly those that are meaningful to the patient with physical, mental, or cognitive disorders. Our Occupational Therapists also focus much of their work on identifying and eliminating environmental barriers to independence and participation in daily activities, similar to everyday life.

Physiotherapy

Physical therapy or physiotherapy (often abbreviated to PT) is a physical medicine and rehabilitation specialty that, by using mechanical force and movements, remediates impairments and promotes mobility, function, and quality of life through examination, diagnosis, prognosis, and physical intervention. We combine our PT with stem cells for maximum physical rehabilitation improvements.

Transcranial Magnetic Stimulation

Research has shown that TMS can effectively treat symptoms of depression, anxiety, neurological pain, stroke, spinal cord injuries, autism and more. This procedure is very simple and noninvasive. During the procedure, a magnetic field generator or coil is placed near the head of the person receiving the treatment. The coil produces small electrical currents in the region of the brain just under the coil via electromagnetic induction. This electrical field causes a change in the transmembrane current of the neuron which leads to depolarization or hyperpolarization of the neuron and the firing of an action potential.

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Stem Cell Treatment for Spinal Cord Injury - Beike ...

Against all odds, stem cell therapies are now getting significant attention from the pharmaceutical industry.

This means that Big Pharma is now seeing that the upside potential of stem cell therapies outweighs the downsides.

While certain things about the stem cell initiative remain controversial - notably embryonic stem cells - some interesting events have occurred recently. These events will, perhaps, encourage more companies to consider investing in stem cell research.

For example, a heart attack victim in London had an experimental surgery that involved the injection of stem cells from his hip into his heart to encourage his heart to repair itself.

The experiment has been successful so far with 37 percent of the victims heart muscle now functioning, up from 21 percent after the heart attack.

Researchers behind this new technique hope that it will increase survival rate from heart attacks by a quarter. Since the Centers for Disease Control and Prevention has said that about 600,000 people die of heart attack in the U.S. every year, this would mean that 150,000 more people could still be alive every year in the U.S.

In light of this development, here are three companies that are investing in stem cell research.

Related:Why Investors Should Closely Watch Celgene

Novartis (NYSE: NVS)

This health care giant has been involved in helping stem cell therapies for about 30 years now, having developed ciclosporin 31 years ago.

Novartis recently broadened its position in the stem cell space by agreeing to take a 15-percent stake in Gamida Cell, an Israeli developer of stem cell therapies. The terms of the agreement has Novartis investing $35 million in Gamida immediately for the stake.

The agreement also gives Novartis the option - which expires in the first half of 2016 - to fully acquire the company, provided certain milestones are achieved regarding the development of NiCord. In this case, the acquisition could come up as early as 2015.

Norvatisannounced on September 6 that it partnered with Regenerex to gain access to Regenerexs stem cell technology, which would help the company develop groundbreaking therapies for underserved diseases.

Johnson & Johnson Company (NYSE: JNJ)

Johnson & Johson kicked off the year by betting $12.5 million on theCapricor Therapeutics(OTC: CAPR) cell therapy program for cardiovascular applications - notably CAP-1002 - through its subsidiary,Janssen Pharmaceuticals, Inc.

Through Janssen Pharmaceuticals, J&J invested in ViaCytes VC-01 combination product being developed for type 1 diabetes. This came after the ViaCyte obtained approval from the FDA to start a clinical trial of the candidate.

J&J is to provide ViaCyte with $20 million.

Celgene Corporation (NASDAQ: CELG)

Celgene is perhaps the boldest big pharmaceutical company out there at the moment, in terms of commitment in stem cell research.

A previousarticleon Benzinga discussed how a robust drug portfolio qualifies the company to be on investors watch list. Its commitment in stem cell adds another reason to watch this company.

Late last year, Celgene entered into an agreement with OncoMed (NASDAQ: OMED) for the development of six anti-cancer stem cell antibodies and certain small molecule stem cell drugs based on OncoMeds study. Celgene committed $155 million in upfront cash to the project.

Under certain conditions in the agreement, the deal could increase to $3.3 billion. The company also entered into an agreement with Human Longevity Inc recently to license, develop and co-promote Celgenes placental cell population, PSC-100. This collaboration also goes to show just how serious Celgene is in holding a significant position in the stem cell space.

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2014 Benzinga.com. Benzinga does not provide investment advice. All rights reserved.

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3 Companies Investing In Stem Cell Research - Yahoo Finance

Story highlights

There's great potential in the field of regenerative medicine, but doctors caution against seeking experimental treatments in an unregulated environment.

Colts head coach Jim Caldwell declined to discuss Manning's health situation when asked about the stem cell treatment at a press conference Monday. Further details of Manning's treatment are not available.

Discussing the choice to go abroad for stem cell treatments puts researchers in an awkward position, because the therapies offered abroad are unregulated and not confirmed to work, but they are based on concepts that the American medical community believes have potential. Generally, however, the consensus is that patients should not try to seek experimental stem cell treatments elsewhere, as there's no telling if they will work or have serious side effects.

"We believe that there is merit to this approach, we just want to see it (carried out) well, ethically and rigorously," said Dr. Joshua Hare, director of the Interdisciplinary Stem Cell Institute at the University of Miami's Miller School of Medicine. Scientists like Hare are trying to gather medical evidence through clinical trials to ensure that these treatments are safe and effective.

Besides orthopedic injuries like Manning's, there are stem cell therapies available elsewhere aimed at heart disease and neurological conditions, even autism. Germany, Panama and Thailand are all popular countries for seeking these kinds of treatments, Hare said.

The basic idea is that stem cells have regenerative potential, and can even form new tissues. Mesenchymal stem cells are a particular kind that can become a variety of cell types, including bone cells and cartilage cells.

And earlier this year, authorities closed down a large stem cell clinic in Germany called the XCell-Center, which had operated through a loophole in the country's regulations regarding unapproved experimental treatments. The clinic had been implicated in the death of an 18-month-old boy after a stem cell treatment; a 10-year-old almost died after receiving something similar.

One problem with going abroad for these unapproved treatments is that there's no follow-up; you won't have anyone back home to take care of any complications that might result, Hare said. In cardiac stem cell trials conducted in this country in people with advanced heart disease, medical professionals do follow up with patients and treat any complications that may arise.

Another concern is that, without proper regulation, any given stem cell therapy abroad may actually be different every time you receive a treatment, said Jeffrey Karp, director of the Laboratory for Advanced Biomaterials and Stem-Cell-Based Therapeutics at Brigham & Women's Hospital in Cambridge, Massachusetts.

There also may not be any regulations on the quality of the treatment, so you really don't know what you're getting, he said.

"Regulatory agencies such as the FDA can ensure that cell therapy that reaches patients is safe, effective, and that quality control is established for isolating the cells, manipulating them outside of the body, and delivering them," Karp said.

A lot of parents believe that stem cell therapies could help their children with autism, although there haven't been any clinical trials in the U.S. to substantiate this, Hare said. In orthopedics and cardiology, on the other hand, there are ongoing investigations; someone like Manning could have potentially joined a clinical trial instead of going abroad, although there's no guarantee that he would have received the treatment rather than a placebo.

The idea of placebo also comes into play here, as with any therapy -- sometimes, just believing strongly that a treatment will heal you actually does change your body, even if that "treatment" isn't actually doing anything.

But there are still significant risks from anything that's unregulated, Karp said.

"While stem cell treatments have been shown to have significant placebo effects during controlled clinical trials, I think patients considering unregulated stem cell treatments need to be aware that they are not optimized and may even do harm," he said.

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Experts caution against stem cell treatments abroad - CNN

Representative imageBy Priyanka V Gupta

New Delhi: Indian Council of Medical Research (ICMR) will soon release the final document on guidelines for stem cell research, the draft of which was available on the ICMR and the DBT (Department of Biotechnology) websites for public reviews till July 31 this year. The guidelines are expected to help in curbing the unethical practices in regenerative medicine. The information was shared by Dr Geeta Jotwani, deputy director general, ICMR, at a recent event where MoU was signed between ABLE (Association of Biotechnology Led Enterprise) and FIRM (Forum for Innovative Regenerative Medicine) for industry research collaborations.

Dr Jotwani said, On the directives of DCGI (Drug Controller General of India), ICMR has been framing the guidelines for stem cell research and therapy since 2001. Unfortunately, there is no therapy available other than bone marrow transplantation, for which also no standard of care has been laid out. In that direction, we have been making periodic efforts by releasing the guideline documents in 2002, 2007, 2013 and now the updated documentation for 2017 is under finalization.

ICMR has been proactively working towards educating the stakeholders about the ethical practices in stem cell research and therapy, for which a special committee, called National Apex Committee for Stem Cell Research and Therapy (NAC-SCRT), has been formed to advise the scientists community. Regenerative medicine is an innovative science. As part of ICMR, more research is involved than getting into conclusion that we are ready for application. We are proactively making efforts to educate, create awareness and give directions to our scientists community and clinicians on how they should go about the research part of stem cell therapy, said Dr Jotwani.

There are many clinicians entering into unethical practices and promising general public about the available care in almost all sorts of incurable conditions, including autism, according to Dr Jotwani.

She said, We are always concerned about what the end users are getting and the promises that are being made to them. Hence, we are proactively being involved in interacting with different government agencies as well as the industry to curb the unethical practices for which we also established NAC-SCRT under the Department of Health Research, Government of India. The committee, which comprises of different government agencies as well as ethics and social groups, legal experts, representatives of drug controllers office and CDSCO (Central Drug Standard Control Organization), deliberates on the issues of upcoming technologies and takes proactive role in the regenerative medicine.

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ICMR's stem cell research guidelines soon to be released - ETHealthworld.com