Category Archives: Cell Therapy

More than one-third of patients with metastatic uveal melanoma had objective tumor regression when treated with adoptive transfer of autologous tumor-infiltrating lymphocytes (TILs), according to results of an unplanned interim analysis published recently in Lancet Oncology.

We observed that a single infusion of TILs after a non-myeloablative lymphodepleting conditioning regimen could induce objective tumor regression, wrote Smita S. Chandran, PhD, of the Center for Cancer Research at the National Cancer Institute in Bethesda, Maryland, and colleagues, including individuals whose disease was refractory to immune checkpoint blockade.

Unlike cutaneous melanoma, use of immune-based therapies in the rare uveal melanoma have been disappointing. Recent studies have shown that adoptive T-cell therapy led to salvage responses in a variety of refractory solid tumors. Therefore, with this study, Chandran and colleagues tested whether adoptive transfer of TILs could mediate tumor regression in uveal melanoma.

The phase II study included patients aged 16 or older with metastatic uveal melanoma. Metastasectomies were performed to obtain tumor tissue and generate the autologous TIL cultures. Patients were given lymphodepleting chemotherapy. The primary endpoint was objective tumor response.

The trial included 21 consecutive patients who received TIL therapy, of which 20 were evaluable. Of the 20 patients, seven (35%) had objective tumor regression.

These results challenge the belief that uveal melanoma is a cancer resistant to immunotherapy, wrote Chandran and colleagues.

One patient achieved a complete response of hepatic metastases. The additional six patients had partial responses, two of which are ongoing. Three of the responders were refractory to previous immune checkpoint blockage.

The precise mechanism for the anti-tumor responses observed in this study is still under investigation, the researchers wrote. All patients received a single cycle of fludarabine and cyclophosphamide, not intended as a direct cytotoxic therapy, but rather as a lymphocyte-depleting regimen before cell transfer to enhance T-cell engraftment and efficacy. Although neither of these chemotherapies has shown activity in metastatic melanoma, we cannot completely exclude their possible role in the tumor responses.

The most common grade 3 or worse chemotherapy-related adverse effects were lymphopenia, neutropenia, and thrombocytopenia.

In an editorial that accompanied the study, Kimberly M. Komatsubara, MD, and Richard D. Carvajal, MD, of Columbia University Medical Center in New York, wrote that the proportion of patients achieving a response as a measure of clinical efficacy is not an established surrogate for progression-free or overall survival in uveal melanoma and must be interpreted with caution. They added that further data will be important in interpreting the clinical effect of this therapy.

Although promising, the results reported by Chandran and colleagues are based on 20 assessable patients and thus must be considered preliminary, requiring confirmation in a larger patient population, they wrote. Overall, however, these data provide important evidence that the immune system can be harnessed to treat uveal melanoma and serves to identify adoptive transfer of TILs as a high-priority avenue of further research for patients with this disease

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Adoptive T-Cell Therapy Induced Response in Metastatic Uveal ... - Cancer Network

At the forefront of regenerative medicine, Kenneth Pettine, MD, has participated in three FDA biologic studies. He works with Jeffery Donner, MD, at the Colorado Spine Institute . Dr. Pettine is the founder of the Orthopedic Stem Cell Institute, and is a pioneer in the field, with the only Stem Cell methods patent procedure in the nation.

"I'm convinced your body wants to heal itself," says Dr. Pettine. "The problem in orthopedics and spine is there's a paucity of blood supply to the joints or the disc in your back. If you injure your cartilage or disc, it has very little capacity to heal itself."

The key to regenerative medicine in orthopedics and spine lies in the mesenchymal stem cell, because it has the ability to differentiate into osteoblasts, chondroblasts or fibroblasts.

"This may be the most important stem cell in your body," Dr. Pettine explains. "The MSC is the cell that modulates your immune system through its paracrine ability to release numerous growth factors, cytokines, chemokines and inhibitorsIt's the conductor and your body is the orchestra."

The use of the MSC to treat orthopedic injuries is standard of care in veterinary medicine, with a good amount of Class 1 data proving safety and efficacy. Dr. Pettine believes humans could also potentially benefit from the use of the MSC to treat orthopedic and spine pathology.

Throughout his career, Dr. Pettine has served as principle investigator for 15 FDA IDE studies focused on non-fusion technology.

He helped with the ISTO Technologies FDA phase one study, which was the first biologic study ever conducted in the human spine in the United States. Using juvenile cartilage cells, the study saw significant reduction in patients' back pain and one-year results have been published.

Dr. Pettine also conducted an IRB study similar to the ISTO trial, utilizing autologous bone marrow concentrated cells to treat discogenic low back pain in 26 patients. This treatment has no FDA issues, as autologous bone marrow concentrated (BMC) cell therapy falls under "the practice of medicine" by the FDA under Section 361 of the Public Health Service Act's provisions.

The 30-minute procedure can be performed in an office or ambulatory surgery center with IV sedation or local anesthetic. Dr. Pettine has published one- and two-year results, and plans to publish three-year follow-up results soon.

The one-year results revealed the cell therapy "significantly reduces lumbar discogenic pain," according to Pettine et.al., Stem Cells 2015; 33:146-156. Out of the 26 patients, only six received surgery 36 months post-injection. Dr. Pettine reported a 72 percent average reduction in Oswestry Disability Index scores and 75 percent average decrease in Visual Analog Scales scores at 36 months.

"It seems to be long lasting," says Dr. Pettine. "We only re-injected two of the 26 patients at three-year follow up."

Of 210 patients with cervical degeneration Dr. Pettine has injected with BMC, about 70 percent reported a 65 percent improvement in pain at one year follow up. Any arthritic joint can be injected with BMC.

Although seeing positive results, Dr. Pettine notes this BMC cell therapy is not intended to replace surgery, but rather serve as a treatment for chronic conditions in patients who want an option prior to surgery. He believes this therapy will become more prevalent in the industry within three years to five years.

"I think it's important for surgeons to be more proactive with [stem cell therapy], because I promise this will not go away," cautions Dr. Pettine. "And if surgeons don't get involved in this, it will be taken over by non-surgeons."

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Dr. Kenneth Pettine: Stem cell therapy is here to stay - Becker's Orthopedic & Spine

A SCOT with crippling multiple sclerosis who planned to end his life at a Swiss suicide clinic has revealed he is applying to undergo an experimental stem cell treatment abroad in a last ditch attempt to reverse his symptoms and prolong his life.

Colin Campbell, from Inverness, said he would postpone his appointment at the LifeCircle clinic in Basel, where he had expected to end his life on June 15, if he was accepted for the pioneering therapy by medical chain, Swiss Medica. A 12-day course at its facility in Belgrade, Serbia costs around 15-16,000 and floods patients with up to 300 million stem cells which have been shown to restore myelin - a fatty coating around nerve cells destroyed by multiple sclerosis - leading to improved brain function and mobility.

Several clinical trials worldwide are exploring stem cell therapy as a means of "pausing" the degeneration associated with MS, but it cannot cure the condition and the treatment is not available on the NHS or privately in Britain.

Former IT consultant Mr Campbell praised his "very kind" landlord, Robert More, for persuading him to try out the procedure.

Mr Campbell, 56, said: "Robert said 'I don't want you to die - you can go abroad and try this. If it works, great; if it doesn't, it doesn't. There's nothing to lose'. So I would say I've moved into a new territory where I'm a 'deferred' suicide, but not a cancelled suicide.

"I will hopefully get onto the treatment programme, but if not then June 15 goes ahead as planned. So I'm in a limbo situation. I'm still holding on to June 15 because I don't want to knock that back and find that the time passes and I'm thinking 'why didn't I get out when I could?'. I've got no desire to spend another winter in the UK with MS - death would be preferable for me.

"That's the thing about not having [voluntary assisted suicide] in Scotland though. If I could do it here I wouldn't have to be too concerned about a date because it would be available to me whenever I choose, whereas when you have the travel to Switzerland and you've got a progressive illness you probably don't want to plan it too far ahead because you might not be up to the journey."

He added that MS patients were also let down because doctors did not routinely highlight the options for treatment outside the NHS.

He said: It would be nice after a diagnosis if a neurologist would go through your options - if they said look, you cant get this on the NHS at the moment, but you can get this abroad, but they dont even have this discussion with you. You get nothing, and thats the experience I hear from talking to other MS sufferers."MS

Mr Campbell was a keen footballer, swimmer and tennis player before being diagnosed aged 34 with primary progressive MS, a rarer form of the disease which results in a steady deterioration without any periods of remission. He now relies on walking aids to move around his first-floor flat and a wheelchair outdoors.

He revealed his plan to end his life in Switzerland to the Herald's sister paper, the Sunday Herald, in April as part of a campaign calling on MSPs to bring a new Bill on assisted dying to Holyrood. The proposed Bill would bring Scotland into line with Canada and parts of the US and Australia by allowing terminally ill people with less than six months to live the right to be prescribed a lethal dose of medication which they can then self-administer.

Mr More, who has rented a flat to Mr Campbell for three years, said: "He's a decent man and, quite frankly, he was depressed with his condition and all he was getting was tea and sympathy. There's nobody doing anything to really help him. Nobody is giving him options and in those situations there are always options.

"There was a 36-year-old woman that was at this [Swiss Medica] clinic and she went in in a wheelchair and when she came out her only complaint was she got tired after long walks. Stem cell therapy doesn't offer a cure, but it might make his life better and that's what I want for the man."

Mr More said he felt compelled to help after his own experience 32 years ago when his youngest daughter was diagnosed with spina bifida and the family were told she would never walk again.

He said: "If someone tells me that I try to do something about it so I took her to the Peto Institute in Budapest. I took her there for four years running and when she came out she could walk. So just because people tell you it's a death sentence, I don't believe it. She's alive and well - the Hungarians did a magnificent job with her."

Mr Campbell said he had also been boosted after being contacted by a fellow MDS sufferer, Rona Tynan, who encouraged him to test out a mobility scooter after seeing reports about his plans to end his life.

He said: "This has given me some kind of optimism which I definitely didn't have - so I owe that to Rona."

Mrs Tynan said: "What alarmed me about Colin was, I felt he was more able than myself."

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MS patient reveals he may 'defer' assisted suicide to undergo stem cell therapy in Serbia - Herald Scotland

H

e made the emotional plea to his colleagues: Pass this bill.

It might give somebody like my wife a chance to walk, Texas Representative Drew Springer said through tears late Thursday at the state Capitol in Austin. Id trade every one of my bills Ive passed, every single one of them, to get the chance to hear HB 810.

HB 810 is one of three bills being considered in the Texas Legislature that would make it easier for sick people to try unproven therapies at their own risk, and cost. Springers bill would allow clinics offering unapproved stem cell treatments to treat patientsin Texas. HB 661 would permit people with chronic illness to get therapies in early-stage clinical trials not just terminally ill patients, as the states current right-to-try law does. And HB 3236 would allow companies to charge patients for unproven therapies.

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The debate in Texas echoes a national discussion over how much access patients should have to experimentaldrugs. For the lawmakers supporting the measures, the issue is about the ability to make ones own decisions about health care and not let bureaucracy get in the way of that. But for stem cell researchers and many patient advocates, the bills are dangerous; they make it easier for people to be fleeced or potentially harmed by treatments with little evidence suggesting that they work, orare safe.

With patients demanding experimental drugs, right to try is becoming the law of the land

When patients get desperate, they have a capacity to suspend disbelief, said Sean Morrison, a stem cell biologist at the University of Texas Southwestern Medical Center in Dallas. When offered the opportunity of a therapy they believe in, even without data and if the chances of benefit are low, theyll fight for access to that therapy. The problem is there are fraudulent stem cell clinics that have sprung up to exploit that.

The personal appeal from Springer, whose wife is paralyzed from the waist down, has worked, at least for now. HB 810 and the other two billspassed the House on Friday with no opposition. They have now moved to the Senate, which only has two weeks to take them up before the Legislature breaks on May 29 for two years.Governor Greg Abbott has indicated he supports HB 810.

Stem cells hold tremendous promise as therapies, but experts say they are still experimental and are not ready to be widely deployed outside regulated and limited trials. Yet clinics offering unproven, and sometimes dangerous, stem cell treatments to eager patients have proliferated around the country in recent years even without the state law, there areat least 71 clinics selling unapproved stem cell therapies in Texas alone.Stem cell scientists fear that the Texas bill would lend legitimacy to the field, provide false hope to patients, and even embolden hucksters touting stem cells as miracle cures for everything from diabetes to multiple sclerosis to spinal injuries.

It may sound like an appealing idea to allow seriously ill patients accelerated access to experimental therapies, Sally Temple, the president of the International Society for Stem Cell Research, wrote to Texas lawmakers this month. But in the absence of full clinical testing, these bills will allow snake oil salesmen to sell unproven and scientifically dubious therapies to desperate patients.

When offered the opportunity of a therapy they believe in, even without data and if the chances of benefit are low, theyll fight for access to that therapy.

Sean Morrison, stem cell biologist at the University of Texas Southwestern Medical Center

In the letter, Temple also wrote that the bills would cost more lives than they save and will undermine confidence in Texas medical system. She cited the three women who were blinded after receiving stem cell procedures at a Florida clinic. At least one of the women thought she was participating in a clinical trial.

For the most part, stem cell clinics and their claims are unchecked. Theyhave largely avoided regulatory scrutiny because they typically take a patients own stem cells and inject them back into the person, meaning the cells are considered minimally manipulated,taken, perhaps, from belly fat, purified, and injected near the persons knee. Plus,stem cell clinics typically do not publish data about their interventions and their patients results, so outside researchers have not been able to verify even their supposed successes.

If these clinics really did have a cure for something, you think they would collect systematic data and publish it in a journal, so people would know, Morrison said.

In a phone interview the morning after his speech, Springer, a Republican who represents a North Texas district, said he wanted to maintain some level of oversight for stem cell therapies and that the state attorney generals office or health department could step in should problems arise. But he said he leaned toward letting people have treatments they think can help them, especially because the drug approval process takes so long.

Stem cell clinics hawking unproven therapies sprout up across US

Springers wife was injured in a diving accident when they were dating and has been in a wheelchair since. He said they stored cord blood from when one of their children was born 16 years ago in hopes that the stem cells from that could one day help his wife. For now, he wants the Texans who head to places like Panama and China for stem cell therapies to be able to get them in their home state, under state law.

We do have a responsibility not to let every snake oil salesman come in, Springer said, but when we do have these rays of hope, we have to make sure theyre available.

Springer is only an author of HB 810, not the other two measures. The lead authors of the other two measures, Republican Representative Tan Parker for HB 661, and Republican Representative Kyle Kacal for HB 3236, did not respond to requests for comment.

HB 810 would give some legal recognition to the stem cell clinics that are already operating in Texas, an indication that troubles some researchers. Paul Knoepfler, a stem cell scientist at the University of California, Davis, co-led a nationwide survey that found Texas has more stem cell clinics than many other states, but that the businesses were part of a national pattern. But he said he hasnt seen other states consider the types of policies Texas is weighing now.

The kind of murky status quo that exists now for regulating stem cell clinics is quite different than there being laws on the books that explicitly say that what the clinics are doing is legal at the state level, Knoepfler wrote in an email.

A few years ago, in one famous case, a Houston company, Celltex Therapeutics, moved its treatment operations to Mexico after a warning from the Food and Drug Administration. But experts wonder if the FDA would take such an action again if the bills became law in Texas, even though the agency would still maintain its authority to do so under federal law. That concern also stems from the feeling that the regulation-averse Trump administration wouldnt endorse such actions, especially because Vice President Mike Pence is a proponent of right-to-try measures and Energy Secretary Rick Perry, the former Texas governor, credits a stem cell treatment from Celltex for helping relieve his back problems.

Perrys story and Springers emotional testimony highlight the uphill battle scientists have faced in recent years as right-to-try laws have been passed around the country. Powerful personal stories of patients cured by unapproved drugs or who die before they can get access to an experimental drug have swayed many lawmakers from both parties.

They look at us like were the devil, which pisses me off because were doing it the right way, said David Bales, the chairman of Texans for Cures, a stem cell research advocacy group that opposes the three bills as written.

Baless group wants to fund legitimate clinical trials involving stem cell treatments to help determine in what ways the cells can help patients. But for now, its top target is stopping HB 3236, which would open the door to patients paying for experimental therapies. Virtually all reputable clinical trials provide experimental treatments to patients at no cost and ofteneven pay participants for their effort.

We dont think that patients in the most vulnerable positions should pay for an unproven drug, Bales said.

Springer, the state representative, said he had not spoken to the governor about HB 810. But Abbott, who has been paralyzed from the waist down since a 1984 accident, when a tree branch fell on him while he was out for a run, tweeted a message of support to Springer early last Friday.

I look forward to signing HB 810, the tweet said.

Andrew Joseph can be reached at andrew.joseph@statnews.com Follow Andrew on Twitter @DrewQJoseph

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Texas leans into unproven stem cell treatments, to the dismay of scientists - STAT

ReNeuron has won a 1.8 million grant from Innovate UK to advance its next generation commercial cell therapy manufacturing capabilities.

The award will support work being undertaken by the UK stem cell company and the Cell & Gene Therapy Catapult.

In particular it will fund key process development activities relating to up-scaled commercial manufacture of ReNeurons cell therapy candidates.

Sharon Grimster, general manager, Wales, at ReNeuron, said: We are delighted to have won this prestigious and highly competitive grant from Innovate UK. It enables us to further pursue and optimise our cell therapy manufacturing processes as our therapeutic programmes get closer to market.

The grant award will also assist ReNeuron in the execution of its strategy to ultimately bring the manufacture of its cell therapy candidates in-house to meet demand following market approval.

ReNeurons lead stem cell candidate is for patients left disabled by a stroke. Last year a Phase I trial of showed its CTX cell therapy candidate improved neurological function in patients with stable motor disability following a stroke for at least 24 months.

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ReNeuron wins 1.8 million for cell therapy development - PharmaTimes

Grupp has been an attending physician and oncology researcher at CHOP since 1996, after earning his MD and a PhD in Immunology from the University of Cincinnati. He completed his clinical training in pediatrics and pediatric hematology/oncology at Boston Children's Hospital, Dana Farber Cancer Institute, and Harvard Medical School, performing postdoctoral research in immunology at Harvard. He joined the University of Pennsylvania medical faculty in 1996, and is now a Professor of Pediatrics in Penn's Perelman School of Medicine.

Since arriving at CHOP two decades ago, Grupp has led a basic science lab studying cell therapy, signal transduction and novel therapies in ALL and other pediatric cancers.Reflecting the translational focus of his work, he also developed and led clinical trials that established tandem stem cell transplantation (two separate transplant procedures) as a standard of care for children with high-risk neuroblastoma. He helped to develop and lead clinical trials that resulted in FDA approval of a new treatment for veno-occlusive disease, a deadly complication of stem cell transplantation. In addition, he led the pediatric hematology/oncology fellowship training program at CHOP for almost a decade. Eight current CHOP faculty trained in his lab.

His outstanding contribution to oncology is in the clinical development of chimeric antigen receptor (CAR) T cell therapy for relapsed and refractory ALL in children and young adults. Collaborating with Dr. Carl June's team at the University of Pennsylvania, Grupp's lab did many of the preclinical in vivo studies, and then developed the first pediatric trial and treated the first pediatric patient at CHOP with CAR T cell therapy in 2012.

Responding to a life-threatening complication of T cell therapy, called cytokine release syndrome, he and his team pioneered a successful treatment for this complication, and thereby revolutionized T cell immunotherapy. Grupp and his colleagues at CHOP have since treated over 150 children with CAR T cell therapy, and he recently led the first multicenter global study of an engineered CAR T cell product that is expected to lead to FDA approval later this year.

About Children's Hospital of Philadelphia: Children's Hospital of Philadelphia was founded in 1855 as the nation's first pediatric hospital. Through its long-standing commitment to providing exceptional patient care, training new generations of pediatric healthcare professionals, and pioneering major research initiatives, Children's Hospital has fostered many discoveries that have benefited children worldwide.Its pediatric research program is among the largest in the country. In addition, its unique family-centered care and public service programs have brought the 546-bed hospital recognition as a leading advocate for children and adolescents. For more information, visit http://www.chop.edu

Contact: Amy Burkholder The Cancer Center, Children's Hospital of Philadelphia Phone: (267) 426-6083 BurkholdAS@email.chop.edu

To view the original version on PR Newswire, visit:http://www.prnewswire.com/news-releases/dr-stephan-grupp-to-lead-cellular-therapy--transplant-section-in-chop-cancer-program-300457482.html

SOURCE Children's Hospital of Philadelphia

http://www.chop.edu

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Dr. Stephan Grupp to Lead Cellular Therapy & Transplant Section in CHOP Cancer Program - PR Newswire (press release)

A fascinating documentary that is making the rounds at film festivals like Tribeca and Cannes gives a rare view of a controversial treatment that more and more Americans are paying up to $50,000 to receive. Stem cell therapy is widely considered to be the next big hope in medicine, with researchers everywhere from Stanford to Johns Hopkins investigating the technologys potential to treat seemingly every ailment known to mankindAlzheimers, cancer, joint injuries, even basic signs of aging. The only hitch: With one tiny exception, it isnt legal in the United States.

We all know the stem cell revolution is occurring outside the U.S., says Brian Mehling, M.D., a Manhattan-based orthopedic surgeon who is certainly doing his part to foment the insurgency. A coproducer of the film, as well as its charismatic recurring subject, Mehling is bringing stem cell tourism into the spotlight and determined to lift the curtain on a medical field that remains mysterious to most. His Blue Horizon medical clinics, with locations in China and Slovakiaand three more set to open in Mexico, Israel, and Jamaicacater to American tourists looking to cutting-edge therapy for help when traditional medicine fails.

Stem cells are the undifferentiated cells that abound in newborns and have the ability to transform into blood, nerve, or muscle cells and aid the body in self-repair. Proselytizers like Mehling say they constitute the latest in holistic medicine, allowing the body to healwithout drugs, surgery, or side effects. At clinics such as Mehlings, doctors either inject the cells, which are generally obtained from umbilical cords during C-sections, into a patients spinal cord (much like an epidural), or administer them via IV drip. The process is alarmingly quick, and patients can typically check out of the facility by the end of the day. One of the few stem-cell therapies approved for use in the United States is one used to treat the blood disease known as beta thalassemia; in that instance, the treatment replaces damaged blood in the immune system and saves tens of thousands of lives each year. Few other stem cell applications, however, have been proven effective in the rigorous clinical trials the Food and Drug Administration requires before signing off on any treatment.

In fact, stem cell clinics remain completely unregulated, and there have been incidents of related troubles. In one recent report , Jim Gass, a resident of San Diego who traveled to stem cell clinics in Mexico, China, and Argentina to help recover from a stroke, later discovered a sizable tumor on his spinal columnand the cancerous cells belonged to somebody else. Troubling cases also emerged at a loosely regulated clinic in Sunrise, Florida where, earlier this spring, three women suffering macular degeneration reported further loss of vision after having stem cells, extracted from their belly fat via liposuction, injected into their eyes. Though, on the whole, reports of treatments at clinics gone awry remain relatively few.

In his film, Stem Cells: The Next Frontier , which is set to appear at Cannes Film Festival this month, Mehling offers a persuasive side of the story, with rapturous testimonials from patients, some of whom who have regained the ability to walk after their stem cell vacations. Added bonus: They come home with better skin, bigger sex drive, and (in the case of at least one balding patient) more hair.

However compelling, there is scant evidence that the injections actually make a difference, and most American doctors caution against buying into the hype. Stem cell researcher Jaime Imitola, M.D. and Ph.D, director of the progressive multiple sclerosis clinic research program at Ohio State University, says he is impressed by the evidence that stem cells can help with neurological disorders in animals. But the question is how can you translate it into clinical trials? We still dont know what were doing when we put stem cells in people.

David Scadden, a professor of medicine and stem cell and regenerative biology at Harvard, and the director of Harvards Stem Cell Institute, says that stem cell tourism is a waste of money for the time being. A world-renowned expert in stem cell science, he remains optimistic about its future applications. Researchers are currently looking into reprogramming, for instance, which effectively converts a mature cell into a stem cell. You rewind its history so it forgets its a blood cell or a skin cell and it rewinds back in time and it can become any cell type, he says. Youd be able to test drugs on these cells, and it could be used to reverse Type 1 diabetes.

For now, though, he does not recommend experimenting with stem cells before we understand them well enough to properlyand safelyharness their benefits. People call me about it all the timethey say, I have this knee thats bugging me, Im going to one of these clinics, he says. His response? For the most part they dont do harm. But nobody Ive spoken with has come back to me and said, You Harvard docs have to get on this . . . . Not yet.

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Stem Cell Tourism Is the Controversial Subject of a New Cannes Documentary - Vogue.com

Capricor Therapeutics said it would have to cut jobs and the scope of its operations after interim data from a Phase II study evaluating its lead allogeneic cardiosphere-derived cell therapy CAP-1002 in heart attack patients showed that the trial was unlikely to meet its primary 12-month efficacy endpoint. The firm said it would now focus its resources on its Duchenne muscular dystrophy (DMD) program for CAP-1002.

The 142-patient ALLSTAR trial was evaluating CAP-1002 in adults who have experienced a large heart attack with residual cardiac dysfunction. The prespecified interim analysis on 6 months of follow-up data indicated that the study wasnt going to demonstrate a statistically significant difference in the efficacy endpoint of percent change from baseline infarct size as a percentage of left ventricular mass, measured by cardiac magnetic resonance imaging (MRI).

"The lack of a clear difference in the change in scar size from baseline to 6 months between the active and control groups in the interim observations from ALLSTAR was unexpected, admitted Linda Marbn, Ph.D., president and CEO of Capricor. These results diverge from the consistent and extensive record of activity observed with our cell technology in the setting of cardiac fibrosis as demonstrated by both preclinical and clinical studies, and we hope to gain an understanding of the factors that led to these observations through the conduct of further analyses.

Raj Makkar, M.D., co-principal investigator of the ALLSTAR Trial, and associate director for interventional technologies in the Heart Institute at Cedars-Sinai Medical Center, added, "We believe it is important to note that the observed improvements in scar size in the placebo group are markedly inconsistent with the well-established natural history of this disease process. It is certainly possible that, for a variety of reasons, the greater number of sites involved in the conduct of ALLSTAR contributed to an increase in variability seen in the scar measurements as determined by MRI."

Carpricor said it would continue to analyze cumulative ALLSTAR data, which showed no notable differences between treatment groups in ejection fraction, but did show near statistically significant reductions in mean end-diastolic volume and trends toward reduction in mean end-systolic volume associated with CAP-1002 therapy.

The firm hasnt disclosed how many jobs will have to go, but said it would now concentrate on developing CAP-1002 for the DMD indication, including the ongoing Phase I/II HOPE trial evaluating skeletal muscle performance and cardiac biomarkers in boys and young men with DMD treated using CAP-1002. Positive 6-month data from the single-dose HOPE study were reported last month.

Capricor also plans to start a repeated-dose study with intravenous CAP-1002 therapy in DMD during the second half of 2017, primarily to evaluate skeletal (noncardiac) muscle function.

Although we are disappointed, the favorable safety profile demonstrated by CAP-1002 in ALLSTAR supports the prospect of its chronic, repeat administration in patients with DMD, Dr. Marbn added. Also, the potent anti-inflammatory properties of CAP-1002 may be well-suited to mitigate DMD progression, for which chronic inflammation is believed to play a causative role."

Earlier this week Capricor confirmed raising $3.7 million in a private placement, which the firm said it would use to progress its products and for general corporate purposes. In February, Capricor reported that it was dropping its Phase II-stage heart failure drug Cenderitide and would end its license agreement with the Mayo Clinic for the natriuretic peptide receptor agonist program so that it could concentrate on its cell- and exosome-based programs.

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Capricor to Cut Jobs as Cardiac Cell Therapy Falters in Heart Attack Study - Genetic Engineering & Biotechnology News

May 12, 2017

Researchers have pinpointed a key enzyme that is vital for the production of fresh blood cells in the body. The enzyme is essential for the survival of specialised stem cells that give rise to new blood cells, the study found. Experts say the findings could help to improve the success of stem cell therapies that are being developed to treat some blood cancers and disorders of the immune system.

Enzyme

Scientists focused on an enzyme called fumarase, which is known to play a key role in the generation of energy inside cells. Children with gene mutations that affect fumarase have blood defects, which prompted researchers to investigate its function.

The mouse study found that deletion of fumarase from blood cells causes major defects in new blood cell production. These defects could be traced back to defects in the specialised stem cells that give rise to new blood cells. Blocking the enzyme causes a molecule called fumarate to build up inside the cells, which has wide-ranging toxic effects.

Stem cells

The study sheds new light on the conditions that blood stem cells need to survive, which could help to boost the success of stem cell therapies, the researchers say. Stem cell metabolism is an emerging field of research with an immense therapeutic potential. In future, we hope to identify the biochemical pathways affected by fumarate in stem cells and, by manipulating these pathways, improve the success of stem cell transplant therapies.

Professor Kamil Kranc says, "The research was prompted when researchers noticed that children with genetic mutations in the fumarase gene have blood defects."

The research was prompted when researchers noticed that children with genetic mutations in the fumarase gene have blood defects.

Scientists from the Medical Research Council Centre for Regenerative Medicine at the University of Edinburgh led the study.

The research is published in the Journal of Experimental Medicine and was funded by The Kay Kendall Leukaemia Fund, Cancer Research UK, Bloodwise, Tenovus Scotland and Wellcome.

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Blood study insight could improve stem cell therapy success - Medical Xpress

Celyad has received Fast Track Designation from the FDA for C-Cure, a cell therapy aimed to treat heart failure with the patients own stem cells.

Celyad is confident that the announcement of Fast Track Designation from the FDA will help it accelerate the search for a partner to develop C-Cure, a cell therapy for ischemic heart failure. After running a Phase III trial in Europe that didnt meet its primary endpoint, the Belgian biotech identified a subpopulation of more of 60% of patients that did show a significant improvement and is now determined to try again once it finds financial support.

CelyadsC-Cure technology consists in the harvest of the patients bone marrow, which is treated with growth factors and then re-injected into the heart to grow new functional muscle in the area lost due to infarction. The therapy is complementary to Celyads main focus, immuno-oncology. The company is well-known for developing an allogeneic version of the promising CAR-T technologythat recently triggered a license agreement with Novartis.

Image viaJolygon/Shutterstock

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Celyad gets FDA Fast Track to Tackle Heart Failure with Cell Therapy - Labiotech.eu (blog)