Clinical trials

09
Aug 2016
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Dr. Molly Shoichet

Breakthroughs can produce a lot of hope, but also frustration

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You read about a stem cell breakthrough that could lead to a whole new way of treating — maybe even curing — a disease. …

You read about a stem cell breakthrough that could lead to a whole new way of treating — maybe even curing — a disease. Then … nothing happens.

The gap in time between a medical science discovery and actual clinical application can seem like a millennium, especially if you are living with the disease in question.

This week in the Toronto Star, Dr. Molly Shoichet articulately — and compassionately — explains why it takes so long to move from the “Eureka!” moment in the research lab to a “We can treat this” scenario at the hospital bed.

She knows first-hand, having worked with a University of Toronto team including Drs. Derek van der Kooy,  Cindi Morshead, Brian Ballios and Michael Cooke that created a hydrogel to help transplanted stem cells thrive in the brain and eye. By injecting photoreceptor cells, the team was able to restore vision by approximately 15% in blind mice.

Their 2015 discovery garnered plenty of media attention, sparking hope that people with macular degeneration — a currently incurable condition — could have a new treatment. More than a year later, no such treatment is available or even on the horizon.

“For many people with macular degeneration, or any degenerative disease for that matter, studies like mine often produce a lot of hope, but also frustration that the ‘product’ is not available to them,” she writes in the Star.

As Dr. Shoichet explains, it can easily take 17 years or more to move a discovery from the lab to the patient.  It takes lots of testing with animals before a new treatment can safely move to the three stages of human clinical trials, with all the attendant regulatory controls and approvals.  It involves plenty of hard, painstaking work. And it costs lots of money.

But Dr. Shoichet remains optimistic. The opportunity to improve the lives of people with deadly diseases is what drives scientists to get up in the morning and get to work.  As she writes, “I am optimistic we will deliver on the promise of regenerative medicine — but it won’t happen right away.”

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20
Jul 2016
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Dr. Duncan Stewart

Q&A with Duncan Stewart, new head of the Ontario Institute for Regenerative Medicine

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Dr.  Duncan Stewart is one of the busiest people in regenerative medicine in Canada.  An internationally respected cardiac specialist, he leads a three-city clinical trial investigating the potential benefits of genetically enhanced stem cells in healing severe heart attacks. …

Dr.  Duncan Stewart is one of the busiest people in regenerative medicine in Canada.  An internationally respected cardiac specialist, he leads a three-city clinical trial investigating the potential benefits of genetically enhanced stem cells in healing severe heart attacks.  He also heads research at The Ottawa Hospital and teaches at the University of Ottawa.  Dr.  Stewart was recently named President and Scientific Director of the Ontario Institute for Regenerative Medicine (OIRM).

Q: Along with your research, clinical work and teaching, you’ve now taken on the role of Scientific Director for OIRM.  How do you juggle it all?

A: It’s a matter of prioritization.  My day job is here at the Ottawa Hospital Research Institute (OHRI) and that’s a fairly significant operation because we’re one of the largest in Canada now.  I have a wonderful team here; it’s well set up and it can be done reasonably.

My clinical practice is really a hobby right now.  I see patients in a clinic that specializes in what I’m interested in: pulmonary hypertension and serious cardiac heart problems.  It’s personally rewarding; it keeps me sane and I can do something where I think I’ve made a difference.  But it’s about a half-a-day a week.  I don’t do much teaching … two or three or four courses a year.

I love doing research.  I thought that when I took on these other responsibilities maybe I’d have to reduce that, but I’ve been successful at getting funding and the projects have been successful, so it’s larger.  It’s aligned with the other things I do.  The priorities of the OHRI are well aligned with the kind of research I do and the new opportunity with the OIRM, again, is very aligned.

Q: What are you excited about in the field of stem cells and regenerative medicine?

A: I’m excited about moving it more into the clinic.  But we have to be realistic about the expectations.  When we’re doing these clinical trials, we are doing them to learn.  There are all kinds of issues we have to get right in order to unlock the real potential of these therapies.  It’s going to take a fair bit of work and a number of clinical trials and going back to the bench to refine approaches before we get to the point where we can do the dramatic things we hope to do.

If you look at the pace of development for other disruptive technologies, in the early stages they didn’t look that fantastic.  It’s similar to the early days of the personal computer.  It was very frustrating.  You did it for fun, really, for word processing, but it was hardly earth-shaking.  Now you look at a PC and it can do what NASA used to do with a great big mainframe.  A smartphone can do absolutely everything.  The technology has evolved to the point where it’s changed the whole landscape.

I think that’s true also in the cell and gene therapy space.  When I look at what’s going on in our lab and what’s going on globally, there is so much opportunity to advance this and improve the sophistication.  The potential is just huge.  What we’re doing in clinical trials, because we’re doing things that are well-tested and safe, tends to be using technologies that were developed 10 years ago.  So it’s well behind the cutting edge of where the field is going.  It takes that much time to get to clinical trials.  I think what we’re looking for is more proof of principle, where we can — in a modest way — achieve some of our goals and learn where the opportunities are to make things better.

Q: So the key is to move into clinical trials?

A: It’s important even when you’re at the early stages of development of technologies, in a safe and responsible way, to get the experience in the context of the patient population that has the disease.  Because you can never model all this completely in the lab.  You may have a great technology, but unless you can use it in the real world patient population you could have a problem.

A good example is autologous cell therapy (therapies in which the patient’s own stem cells are used).  For the chronic diseases we want to treat with regenerative approaches, we’re usually dealing with older patients who have risk factors and various diseases.  All that reduces their regenerative activity of their own cells: their stem cells aren’t very good.  So, you can do all the testing you want in young, healthy models but you’ve got to get at that problem.  We need to find why these cells don’t work and what we can do to recover activity.  Induced pluripotent stem cell technology (drawing cells from skin and reprogramming them back to an embryonic stem cell-like state) not only produces a pluripotent stem cell, it turns the age back to zero.  There are other issues with that in terms of safety and whether it’s ready for prime time, but there are already clinical trials starting with that technology.  That’s one way.  There are other ways, maybe more subtle ways, to recover regenerative activity.  These are the issues that come to the fore when you start to translate therapies into clinical trials.

Q: There is some frustration that things haven’t moved faster.  Stem cells have been touted as being able to cure a number of diseases and that it’s going to happen soon.  What’s holding the field back?

A: The frustration is probably our own doing: we bought into the hype or generated the hype too much.  We need to set the expectations at a realistic level.  It does no good to say we’re going to cure a disease with an approach when we’re not really there yet.  It’s important that we do the clinical trials.  It’s important to understand what benefit we get, if any, with a simple, early therapy so we can start improving it.  But we need to understand that this is going to be a somewhat incremental process at the beginning.  It’s not going to be earth-shattering and necessarily curative in the first iteration.  Setting that expectation will decrease that frustration.

The reality is, this takes time.  It’s a very different activity to be moving these approaches into clinical trials than doing the fundamental research.  People who have made major discoveries are not necessarily equipped or have the ability or interest to move them into clinical trials.

What does work well is a team approach.  You have the innovative scientist who makes the discovery as an important part of that team.  But a lot of what has to be done to translate that into a clinical trial is the preclinical research needed for regulatory submissions — which is not the exciting kind you publish in Nature.  It’s a different skill set than is normal in a discovery lab.  And you need people who understand the disease and where the therapy should best be targeted.  You need people who understand what kind of trial needs to be done to get the right answers.  It takes a lot of people who have complementary expertise who come together in a translational research program and say ‘OK, we think this is potentially going to make a big difference.  This is how we’re going to move it forward in a safe, responsible and effective manner.’ Coincidentally, this is exactly what OIRM is doing, funding these team grants to develop translational teams.

Q: What do you see as OIRM’s role?

A: Ontario is a big player in Canada in this space.  We’re not the whole story but were a big part of the story.  There is tremendous talent.  What OIRM can do is help people understand how to move this forward, how to build these teams.  It’s education: bringing in experts who have had success and helping these teams move forward, helping them build in the areas where they need to build.  It’s not always intuitive.  I see OIRM as a real catalyst.  It also has a role to shepherd these activities in the right direction so there’s the greatest chance of success.

 

 

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27
Jun 2016
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Putting a face on the dangers of stem cell tourism

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Jim Gass, a 66-year-old lawyer who now lives in San Diego, has inadvertently become the human face of the dangers of stem cell tourism.…

Jim Gass, a 66-year-old lawyer who now lives in San Diego, has inadvertently become the human face of the dangers of stem cell tourism.

A feature in the New York Times headlined “A Cautionary Tale of Stem Cell Therapy Abroad” tells of how Mr. Gass spent $300,000 by travelling to clinics in Mexico, China, and Argentina in search of a miracle stem cell cure after he had a stroke. Recently, his American doctors found a huge mass of someone else’s cells growing aggressively in his lower spine.

As the article explains, Mr Gass travelled from clinic to clinic believing that the worst that could happen was there would be no improvement in his post-stroke condition, which left his left arm useless and his left leg weak. Except for being able to use his right arm, he is now paralyzed from the neck down.

In Mexico he received an injection of fetal cells shipped from Russia that, for a time, seemed to improve his ability to walk: “Then something disturbing happened,” Mr. Gass told the Times. “I felt pain when I would lie down, like I was lying on a tumor. “I started to lose my ability to walk and I fell down a lot.”

That’s when Boston doctors found the mass containing someone else’s primitive cells. They treated it with radiation, which seemed to slow its growth, but the mass is growing again.

As discussed on the Treatments Abroad page on our website, scientific discoveries and innovations surrounding the potential of stem cell science have led to great enthusiasm about potential benefits to patients.  But the dark side of that enthusiasm is the hype, exaggerated publicity and inaccurate claims in the interest of financial gain.

Many unregulated clinics around the world offer treatments that are simply not based on sound scientific evidence. If you or someone you know is considering such a therapy, you’d be wise to consult a booklet produced by the University of Alberta Faculty of Law, Albany Medical College and the Stem Cell Network entitled What you need to know about stem cell therapies.  A PDF version is available here.

An excellent web resource is the International Society of Stem Cell Research Web page: A Closer look at Stem Cells. It includes a Patient Handbook downloadable in several languages.

Both sources advise potential patients to be wary of clinics that offer patient testimonials in place of evidence gathered through rigorous clinical trials.  Or, as Mr. Gass now says: “Don’t trust anecdotes.”

 

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13
Jun 2016
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CBC program delves deeply into stem cell treatment for MS

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Want to know more about last Thursday’s announcement that two Ottawa doctors have found a way to shut down aggressive MS?…

Want to know more about last Thursday’s announcement that two Ottawa doctors have found a way to shut down aggressive MS?

In case you missed it, Rita Celli, host of CBC’s Ontario Today, dedicated her entire hour-long program on Friday to discussing the report. Published in the Lancet, the paper details how Drs. Harry Atkins and Mark Freedman were able to halt the progression of the disease for the 24 patients in the study. Some patients, like Jennifer Molson, saw their MS symptoms disappear entirely over time.  You can access the podcast here.

Ms. Celli features Dr. Freedman who explains how the treatment destroys the patient’s immune system through chemo and then rebuilds a new MS free one using their own previously harvested bone marrow stem cells. He also takes calls from listeners, some of whom tell their stories of life with MS.

The program also features clips of Ms. Molson explaining how the treatment freed her from life in a wheelchair, and Dr. Atkins declaring that “MS can be stopped in its tracks.”

The program provided a comprehensive look at the treatment, which is considered high risk (one patient died) and is only for those MS patients for whom nothing else is working.

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09
Jun 2016
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Drs. Harry Atkins and Mark Freedman of The Ottawa Hospital

Ottawa team proves stem cell/chemo combo halts MS

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A team led by two doctors from The Ottawa Hospital has proved that stem cells, used in combination with chemotherapy can halt the progression of aggressive multiple sclerosis (MS) and — in some cases — help patients recover from the autoimmune disease.…

A team led by two doctors from The Ottawa Hospital has proved that stem cells, used in combination with chemotherapy can halt the progression of aggressive multiple sclerosis (MS) and — in some cases — help patients recover from the autoimmune disease.

In a paper published today in the prestigious medical journal The Lancet, Drs. Harry Atkins and Mark Freedman show that bone marrow stem cell transplants can eliminate all signs of damaging brain inflammation by rebuilding a patient’s immune system.

MS occurs when a person’s own immune system attacks the myelin protective sheath that covers nerves, causing inflammation and damage that inhibits communication between cells in the nervous system — the brain and spinal column.

The researchers took bone marrow stem cells from patients with aggressive MS and purified them in a laboratory. After the patients underwent extreme chemotherapy, their robust stem cells were returned to rebuild new immune systems.

The clinical trial involved 24 patients and tracked their progression over several years. According to The Ottawa Hospital’s news release, after the treatment:

  • Not a single participant experienced a clinical relapse (zero relapses in 179 patient-years), whereas before treatment, the participants experienced an average of 1.2 relapses per year.
  • Not a single new active inflammatory lesion could be detected in the brains of any of the participants.
  • Not a single participant required MS-specific drugs to control their disease.
  • 70% of participants experienced a complete stop in disease progression.
  • The average rate of brain shrinkage, typically a measure that correlates with MS progression, returned to levels associated with normal aging.
  • 40% of participants experienced some lasting reversal of such symptoms as vision loss, muscle weakness and balance problems.
  • Some participants were able to return to work or school, regain the ability to drive, get married and have children.

In Jennifer Molson’s case, the treatment eradicated all traces of the MS that had taken over her life. Prior to taking part in the study, she was receiving 24-hour care at the Rehab Centre at the Ottawa Hospital, “learning to how to live with my disability.” She had quit her job and could only walk with the help of forearm crutches or a walker. Life in a wheelchair was imminent.

Now free of MS symptoms for more than a dozen years, Ms. Molson has resumed a demanding career and a busy schedule. As she described in the book Dreams & Due Diligence:  Till and McCulloch’s Stem Cell Discovery and Legacy, “I downhill ski, I drive a standard. I can skate. I can dance … Am I cured? I like to use that word. They (Drs. Atkins and Freedman) don’t like to use that word. They’re calling it a lasting remission. I’m very lucky. I got a second chance at life.”

While his recovery was less dramatic, Vancouver’s Aaron Prentice said his quality of life is much improved: “I am now five years post-transplant,” he wrote in the Foundation’s Cellections newsletter last year. “I have not had a relapse and no longer require a cane. My gait has improved significantly and continues to do so. My arms are symptom free.”

The Ottawa Hospital, the MS Society of Canada and the University of Ottawa have produced a video about the clinical trial that can be seen here: https://www.youtube.com/watch?v=vW86owclZes&feature=youtu.be

Marjorie Bowman, trial coordinator and advanced practice nurse at The Ottawa Hospital, expressed her admiration for the remarkable “courage and dedication” demonstrated by the trial’s participants. “We thank the patients from across Canada who participated in this clinical trial, as well as their family members,” she said.

The treatment, Dr. Atkins explained in Dreams & Due Diligence, is a variation on the bone marrow ablation that leukemia patients undergo.

“MS is an autoimmune disease where the immune system is attacking a patient’s brain. The simple concept behind our treatment is, ‘Let’s just get rid of the old immune system and put back the seeds, let a new one grow and hope that it won’t learn the same lesson.’

“Because stem cells don’t carry over immunologic memory. That’s really what we have tried to do. We had a track record for doing transplants for leukemia and knew how we could damage the immune system to remove it. We just applied the lessons we learned in care of patients with leukemia and applied them to this new setting.”

Not for everyone

However, the therapy is not for all MS patients.

“It is only used in very severe cases because participants face a significant risk of infection and other side-effects, including death,” Dr. Atkins said in the media release. “The risks are similar to those faced by leukemia patients undergoing this kind of treatment.”

Indeed, one participant in this study died of liver failure due to the treatment and another required intensive care for liver complications. The treatment regimen was modified to reduce toxicity, but all participants still developed fevers, which were frequently associated with infections.

People who are interested in this therapy should speak with their own neurologist, who can request a referral to The Ottawa Hospital MS Clinic or another major hospital with experience in this area. The Ottawa Hospital cannot treat people without valid Canadian health coverage.

While The Lancet paper is focused strictly on the MS patients, Dr. Atkins has also seen some success treating patients with other immunological disorders such as Stiff Person’s Syndrome, neuromyelitis optica and Crohn’s disease.

And Dr. Freedman has gone on to co-lead a new chemo-free MS clinical trial using mesenchymal stem cells. “These cells have been shown, at least in early studies in humans, to repair — period,” he explained recently. “But they happen, at the same time, to have an anti-inflammatory effect. So they may be able to accomplish both things together. Without the need of any chemo, there is very little risk to the people taking it.”

A turning point for MS

Yves Savoie, President and CEO, MS Society of Canada, called publication of the paper “a turning point” in MS care. “What started as a bold idea has translated into a treatment option for people living with highly active, relapsing MS.”

The $6.47 million trial was funded by the MS Society of Canada and its affiliated Multiple Sclerosis Scientific Research Foundation. The research was also supported by The Ottawa Hospital Foundation, The Ottawa Hospital Department of Medicine and Canadian Blood Services.

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06
May 2016
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Potent cells could spare preemies from long-term lung troubles

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Dr. Bernard Thébaud

When babies are born prematurely — especially at fewer than 28 weeks in the womb — their lungs are not fully formed and they need help to breathe. …

Dr. Bernard Thébaud

Dr. Bernard Thébaud

When babies are born prematurely — especially at fewer than 28 weeks in the womb — their lungs are not fully formed and they need help to breathe.  At neonatal units in hospitals around the world, doctors put these babies on ventilators to give them additional oxygen.

Unfortunately, the very device that keeps these tiny babies alive can create life-long consequences by triggering bronchopulmonary dysplasia (BPD).

“Approximately 40% of extreme pre-term babies will get BPD,” says Dr. Bernard Thébaud, a researcher and clinician with The Ottawa Hospital and the Children’s Hospital of Eastern Ontario. “They will have a higher risk of developing asthma and of being re-hospitalized after being sent home. They may be at risk of early onset aging of their lungs — something similar to smoker’s lung.”

There is no treatment for BPD.  Steroids that decrease the inflammation it causes can also inhibit brain development.  “So we’re very cautious in administering steroids,” says Dr. Thébaud, who is working on a stem cell solution to the ventilator/BPD conundrum.

In mesenchymal stem cells extracted from Wharton’s jelly, a gelatinous substance within the umbilical cord, Dr. Thébaud hopes to find a BPD treatment that will also help other organs thrive.

“These cells will do several things.  They will attenuate the inflammation, but also promote lung growth. In addition, we believe their benefits will not be limited to the lung.  They will also have some beneficial effects on the brain, the gut and other organs in preterm babies that still need to grow and need to be protected from injury. “

So far, Dr. Thébaud, who is also a professor of pediatrics at the University of Ottawa where his holds the uOttawa Partnership Research Chair in Regenerative Medicine, has only tested the mesenchymal stem cells on animals. But the results have been encouraging, with the stem cells protecting the lungs from oxygen induced injuries. “We still have to do further tests with the cells made in a Good Manufacturing Process facility for a clinical grade product.”

He looks forward to conducting a Phase 1 clinical trial in the near future to test the feasibility and safety in newborns.  “If it’s safe, we’ll move to Phase 2 and enroll more babies.”

As a researcher who cares for these fragile babies every day, Dr. Thébaud is both frustrated and hopeful.

“When we see these babies in our unit and we discuss what we can offer, we can see how the disease evolves and we know that we will be stuck at one point. Sometimes a nurse will come up to me and say, ‘Oh, Bernard, this baby needs your stem cells. Can you bring some over from our lab?’  On one hand it’s frustrating. On the other, it’s very rewarding to see that everyone recognizes the potential of this therapy and is waiting for it.”

The beauty of these mesenchymal stem cells, also called stromal cells, is that they are easily harvested from umbilical cords that would otherwise be discarded as medical waste. They are also easy to expand in a lab, generating the millions of cells required for transplant.  Because no genetic match is required between donor and recipient, they can be supplied off-the-shelf. And they are powerful.

“Because the cells are young, they might be more potent than bone-marrow-derived mesenchymal stem cells,” says Dr. Thébaud.  “So it could be that the product we are developing for the babies could also be beneficial in treating adult diseases.” and superior to current bone marrow-derived stem cells.”

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31
Mar 2016
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Dr.Lauralyn McIntyre

Man makes remarkable recovery from septic shock after stem cell transplant

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A 73-year-old man from Hawkesbury, Ontario, survived a deadly infection after receiving millions of mesenchymal stem cells in a world-first trial at The Ottawa Hospital.…

A 73-year-old man from Hawkesbury, Ontario, survived a deadly infection after receiving millions of mesenchymal stem cells in a world-first trial at The Ottawa Hospital.

Charles Berniqué developed severe septic shock in June of last year after his esophagus burst, likely from food poisoning. Septic shock is a deadly condition in which rampant infection triggers hyper-activation of the immune system, causing the cardiovascular system and organs to fail.

Mr. Berniqué was first treated by thoracic surgeons, who restored his fluids, repaired his esophagus and started antibiotic therapy. He was placed into a coma in the intensive care unit where mechanical ventilation and dialysis supported his heart, lungs and kidneys.

During this time, his wife Maureen consented to his participation in the clinical trial led by Drs. Duncan Stewart and Lauralyn McIntyre.

Within 24 hours, Mr. Berniqué received an intravenous infusion of 30 million mesenchymal stem cells originally extracted from the bone marrow of a healthy Ottawa volunteer.

“Our laboratory studies that showed that mesenchymal stem cell therapy tripled survival in a mouse model of septic shock,” Dr. Stewart, Executive Vice-President of Research and senior scientist at The Ottawa Hospital, said in a media release. “The cells also reduced damaging inflammation and helped the mice eliminate the bacteria.”

Dr. McIntyre, an intensive care physician and senior scientist at The Ottawa Hospital, was impressed by the results. “Researchers around the world have spent decades trying to find a therapy that will treat the root causes of septic shock rather than just the symptoms, but so far, none of these therapies have improved survival,” she said. “We don’t know whether the cell therapy played any role in Mr. Berniqué’ s remarkable recovery, but the cells were very well tolerated and we are excited to continue to study this promising therapy in more patients.”

Mesenchymal stem cells have been studied extensively in human clinical trials for other conditions, but The Ottawa Hospital trial is the first in the world to evaluate the cells specifically for the treatment of septic shock. The main goal of the Phase I trial is to evaluate the tolerability and feasibility of the cells. However, the researchers have already received funding from the Ontario Institute for Regenerative Medicine to begin scaling up their cell bank for a larger Phase II trial, which will help determine if the therapy is effective against septic shock.

As for Mr. Berniqué, he’s grateful to be alive and happy that the care he received may do the same for others. “It is tremendous what The Ottawa Hospital did for me,” he said. “I was so close to death, but I received the best care in the world and got to participate in this study which could help many people.”

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14
Mar 2016
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Stem cell treatment for bad knees lacks high-quality clinical proof

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Recently we blogged about the proliferation of clinics offering an expensive, unproven stem cell treatment for arthritic and damaged knees.

In the current edition of the Clinical Journal of Sport Medicine, the Australasian College of Sports Physicians (ACSP) published a position paper on the use of mesenchymal stem cell therapies in sports and exercise medicine.…

Recently we blogged about the proliferation of clinics offering an expensive, unproven stem cell treatment for arthritic and damaged knees.

In the current edition of the Clinical Journal of Sport Medicine, the Australasian College of Sports Physicians (ACSP) published a position paper on the use of mesenchymal stem cell therapies in sports and exercise medicine. You can find the full paper here. It’s well worth reading if you are thinking about spending thousands of dollars for such a treatment or know someone who is.

Mesenchymal stem cells are commonly extracted from adipose (fat) tissue, skeletal muscle, umbilical cord blood, placenta, and bone marrow. In our blog post we dealt mainly with a same-day treatment known as the BMAC (bone marrow aspirate concentrate) procedure. It involves extracting the mesenchymal stem cells from a patient’s pelvis, running them through a centrifuge to concentrate them, and then re-injecting them into the bad knee to reduce inflammation and repair damage. The ACSP, which represents sports physicians in Australia and New Zealand, also considered a treatment in which cells are extracted via liposuction from fat tissue and then put through the centrifuge — a procedure the U.S. Food and Drug Administration is cracking down on.

The paper suggests technology is running ahead of scientific knowledge:  “A contemporary development in manufacturing techniques uses far shorter preparation times (often less than two hours) at far less cost, producing a non-expanded mixed cell population. However, mesenchymal stem cell numbers are low in these preparations and the presence of multiple cell types raises questions about the efficacy of these techniques. Although the progress in biotechnology has resulted in commercially attractive outcomes, the science substantiating their effectiveness has lagged behind … No published randomized controlled trials for ‘same-day bone marrow stem cell concentrates’ are available as of 2015.”

This is not to say such treatments don’t work, but that much more study is needed. And it needs to meet the gold standard of a double-blind, randomized, placebo-controlled clinical trial in which some patients get the mesenchymal stem cells and others get a placebo treatment. The ACSP found only one such trial has been done and it did not involve BMAC. Rather, it demonstrated no difference in outcomes between the treatment group who received adipose-derived mesenchymal stem cells and those who got a saline injection. The full study has yet to be published.

According to the ACSP, using stem cells to treat damaged knees should be initiated under only two conditions:

  • As part of a rigorous clinical research trial.
  • As an individualized innovative therapy where there is a commitment to the transparent collection of data for analysis by a research body external to the clinic.

“Innovative therapy” refers to a treatment undertaken by a physician in the hope it will help the patient, though its effectiveness has yet to be proven.  The upside is that this is how medical practice improves: doctors try something and it works.  The downside, as the ACSP notes, is that such treatments may become adopted practices with no proof they actually do any good. The worst case scenario is that an unproven treatment actually does harm.

Randomized, placebo-controlled clinical trials can take years and cost hundreds of thousands of dollars. To someone with arthritic or damaged knees — or hips or shoulders for that matter — it’s cold comfort to hear that more study is needed before such stem cell treatments get the thumbs up or thumbs down.   In the meantime, we don’t know.

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24
Feb 2016
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FDA to crack down on clinics peddling unproven stem cell treatments

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Federal regulators in the United States are about to crack down on hundreds of clinics peddling pricey stem cell therapies to treat a laundry list of diseases and conditions without scientific evidence to back them up.…

Federal regulators in the United States are about to crack down on hundreds of clinics peddling pricey stem cell therapies to treat a laundry list of diseases and conditions without scientific evidence to back them up.

According to a report by STAT, an online health and medicine news service, some 200 stem cell clinics have cropped up in recent years, selling injections, facelifts, and treatments that have not undergone clinical trials.

Many of the clinics use a procedure called stromal vascular fraction (SVF) in which cells are extracted from a patient by liposuction, run through a centrifuge to collect the adipose (fat) stem cells and returned to the patient intravenously or injected at the site of the condition. Clinics promote the SVF procedure to treat a variety of conditions such as arthritis, Parkinson’s disease, pulmonary fibrosis, chronic obstructive pulmonary disease, multiple sclerosis, cerebral palsy, and amyotrophic lateral sclerosis. Treatments can cost anywhere from $5,000 to $25,000.

The American Food and Drug Administration (FDA) recently issued draft guidelines on the use of human cells, tissues, or cellular or tissue-based products and will hold a public hearing on April 13 at Silver Spring, Maryland to help finalize its stand on such treatments.

As STAT reported,  the FDA already sent a warning letter  to a network of stem cell clinics in California, New York, and Florida advising the owner that he needed FDA approval to sell and use stem cells, which the agency classified as biological drugs.

FDA approval requires evidence that stem cell treatments are safe and effective, which, as the article points out, “takes drug companies many years of clinical trials to obtain, at a cost of millions of dollars.”

Canadian bioethicist Dr. Leigh Turner of the University of Minnesota has long criticized the FDA for failing to crack down on the clinics as the mushroomed across the U.S. “If it’s not safe and it’s not going to help patients,” Dr. Turner told STAT. “It’s just predatory behavior.”

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03
Dec 2015
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Dr. Eva Feldman

Major trial for ALS therapy in the works

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Dr. Eva Feldman devoted 12 years to working on a drug-based cure for amyotrophic lateral sclerosis (ALS).  It was, she says, “a very big endeavour.”  It failed.…

Dr. Eva Feldman devoted 12 years to working on a drug-based cure for amyotrophic lateral sclerosis (ALS).  It was, she says, “a very big endeavour.”  It failed.

So, in 2006 the University of Michigan clinician/researcher took a sabbatical to rethink her approach to fighting ALS, the cruel, fatal condition that attacks the nerve cells (neurons) that control muscle movement.  “I wanted a break,” she says.  In California, Dr. Feldman found scientists doing interesting animal studies on treating spinal cord injury with stem cells. It changed her perspective entirely.

Today, with two early-stage human studies behind her, Dr. Feldman hopes to soon begin a large-scale clinical trial to test whether human neural stem cells injected into the spinal columns of ALS patients can stop the disease from stealing their ability to walk, talk, eat and breathe.

“We inject the cells into the high part of the spinal cord of patients with ALS with the goal of protecting the large motor neurons that are necessary to maintain normal breathing.  Our goal is for the stem cells to go into that area, surround the neurons that are starting to get ill and nurse them back to health.  We do very similar injections in the lumbar area of the spine to preserve the neurons that go to the muscles that allow patients to walk.”

Preclinical studies she and her team conducted on rats and pigs showed that the stem cells “take a really bad environment and clean it up.” Inflammation is ameliorated and the stem cells surround the large, ailing motor neurons and nurse them back to health. “The cells go from looking like they are about to die to being quite healthy and robust,” says Dr. Feldman.

Phase I and II clinical trials involving 30 patients went “extremely well,” she says, with the procedure proven to be safe and the patients able to tolerate the accompanying immuno-suppressant therapy. “We have good preliminary data,” she says.

Neuralstem Inc., Dr. Feldman’s industry partner in the project, is organizing a large, multi-centre trial in 2016 to test whether the procedure truly works. Richard Garr, the company’s Chief Executive Office, is understandably guarded about the details, saying via email that his company is working with the U.S. Food and Drug Administration and that “all of the issues with respect to the scope and nature of the trial are still being determined.”

Dr. Feldman, who has been down this road before with the failed ALS drug, is cautiously optimistic.  “As enthusiastic as I am about the therapy, until we do a very large trial we simply won’t know with certainty that this is the hopeful home run that we want it to be.”

For Ted Harada, a 43-year-old former FedEx manager in Atlanta, Dr. Feldman’s stem cell therapy has been a life-saver. The recipient of two stem cell implant surgeries, he has seen his decline from ALS virtually stopped.  The normal survival period for ALS, which is sometimes called Lou Gehrig’s disease after the New York Yankee slugger who succumbed to it, is about 36 months. He is now five years out and feeling good, although he still has the disease.

“I put my cane down two or three weeks after the (second) surgery and I haven’t picked it back up,” he says.  “When I had my fifth year anniversary, my doctor said ‘Ted, I would have guaranteed you’d be dead within two or three years when I first met you.’  I like to say that the surgeries set the clock back to what I call onset.”

Dr. Feldman says other patients in the studies also have done well but “the numbers are small … until our numbers are larger we can’t say with certainty.”

While criteria haven’t been set, participants in the larger trial likely will need to be in the early stages of the disease, with the ability to breathe reasonably well and speak and swallow without difficulty. Dr. Feldman says Canadian patients might be eligible if they can travel to a surgical site — but, again, details are still being worked out.

Dr. Feldman is also excited about the possibility of using the same kinds of stem cells to treat the dementia disease.

“I have beautiful preclinical data in animal models of Alzheimer’s. We’ve shown that the injection of stem cells into the selected areas of the brain that are required to form new memories rescues the animals and they are able to function normally. We see the accumulation of amyloid, which is the build-up of plaque that patients get, gone. The stem cells go in and they are just like garbage disposals, cleaning up all the garbage.  It’s remarkable.”

 

 

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