I recently conducted an interview with Dr. Joanne Kurtzberg of Duke. You can see Part 1 of the interview here.
Today in Part 2, I asked Dr. Kurtzberg some broader questions about the stem cell field.
1. How concerned are you about non-compliant stem cell interventions here in the US as well as stem cell tourism? Perhaps because of my blog, I get contacted by parents of pediatric patients lately at least once a week about possible stem cell treatments for CP, autism, and other diseases. They generally are not asking about clinical trials, but rather expensive, dubious treatments abroad. What can we do about this situation?
I share your concern. I think the biggest risks associated with these therapies are infection, tumors, and immune mediated reactions, particularly when cells are delivered directly into the brain or spinal fluid. The financial risks are high too. Desperate families are not likely to respond to someone saying the treatment is not promising. As an alternative, I take the approach of educating parents who approach me with similar questions about the questions they should ask about these therapies. These questions include:
- 1. What kind of cells are being used in this therapy?
- 2. Where do the cells come from?
- 3. How are they tested for the risks of transmitting infectious and genetic diseases? Specifically are they tested for HIV and hepatitis?
- 4. How are the cells prepared/manufactured?
- 5. How many patients have been treated with these cells?
- 6. How have these patients done? What kind of follow-up is practiced/required?
- 7. Are there any toxicity/adverse reactions associated with the therapy?
2. On the positive side, what excites you the most about the stem cell field today?
I think the stem cell field has enormous potential particularly in the emerging fields of cellular therapies and regenerative medicine. I think that development of this field will take 10-20 years and that it will take this long to see stem cells used in routine medical care. I think it will be necessary to develop third party, ‘off the shelf’ products to fully realize the potential of cellular therapies.
3. Where do you think the field will be in 5 years? 10 years?
In 5 years, I think we will be conducting early phase trials in some disease at the proof of concept stage. I also think we’ll see major advances in immunotherapies, e.g. tumor and antiviral vaccines, cells to enhance immune reconstitution. In 10 years I think we’ll have evidence from these trials to justify further development of specific cell therapies for specific diseases. My best guess is that we’ll first see cells used to treat patients with stroke, ischemic heart disease, newborn and fetal brain injury and traumatic brain injury.
Thank you to Dr. Kurtzberg for the interview.
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A researcher has a different philosophical approach in any new treatment (drug) than a medical doctor (medical procedure). A researcher has FDA protocols that are mostly geared towards massively produced chemical drugs that can reach 100s of thousands of patients in a short period of time, so justifiably, the drug or in this case autologous stem cells have to be deemed potentially harmful first, and then proven safe. Their “guilty first until proven innocent” approach is not the same for medical procedures. Autologous stem cells have been shifted away from medical state board’s jurisdiction that considers patient’s risk to the level of riskiness in a unique procedure into a more cumbersome FDA guilty first protocol. A perfect example of this is which defies that logic is the first heart transplant performed. I think you cannot get more venturesome than having heart from another person to a different host. Besides, autologous stem cells are uniquely yours and not massively produced even after minimal manipulation threshold (UNDER PROPER PROTOCOLS), so they are a lot safer.
Below is an example of what I was referring to when parents are duped and the snake oil clinic tries to shut them up. This is like many of the stories I have been told by parents here in the US and I sent the story to Doug. However this mother paid the ultimate price and was brave enough to speak out.
Click on the links in the text to see just how evil this “Dr” was. http://sctmonitor.blogspot.com/2012/02/reader-alerted-me-to-another-tragic.html#!/2012/02/reader-alerted-me-to-another-tragic.html
ICMS is an illiterate scam in every sense of the word.
10-20 years? Ahem… That’s what they used to say about fusion reactors (60 years ago).
There are already many proof of concept publications — and clinical trials — regarding the application of MSCs to orthopedic injuries. But I guess that’s called the “practice of medicine” and so it has to be shunned, banned and ignored?
Funny you would mention that Brian. During a baby shower for her grandchild, I recently spoke with my cousin, a nuclear physicist that was called in to evaluate and report on 3 mile island. The one of many things that we both agreed upon in science was safety. Boom.
Safety First? How the Current Drug Approval System Lets Some Patients Down
Andrew Culliford, whose story is featured in the Daily Mail, is one of the estimated 7 in 100,000 people living with Motor Neuron disease, a progressive degenerative disease which attacks muscles, leaving those affected eventually unable even to breathe unassisted. For Andrew, a young father who has a severe form of the disease, it means a two to five year life expectancy.
Like Les Halpin and Jenn McNary, the mother of twins afflicted with a similar rare disease, he has a simple request: earlier access to medicines that might help improve or extend his life.
The US introduced a mandatory pre-approval process for pharmaceutical drugs after over 100 people were killed by an untested drug formulation . Today, each drug must go through a series of strictly controlled trials, including Phase 1 tests on healthy volunteers, followed by Phases 2 and 3 which test the drug and dosages on smaller and then larger patient groups. The process is estimated to cost $500 million per drug and to take 8 – 12 years.
But it has been a failure for one small group of patients: those with rare, imminently lethal diseases, for whom there are no existing good treatments. Those who will die in less than 8 years. It is these patients who are asking to have access to untested medicines, and to avoid placebo controlled trials, where half the participants are given no drug at all. Les has proposed innovative methods of patient recording data. I have discussed this proposal previously on this blog , and in a joint paper with Les Halpin and clinicians.
http://blog.practicalethics.ox.ac.uk/2013/08/safety-first-how-the-current-drug-approval-system-lets-some-patients-down/
I agree with Dr. Kurtzberg and the questions to ask the clinics are great. I know she had all the correct answers and kept my child safe.
The only problem is the dubious clinics will lie, cheat and steal to get a parent’s dollar and the answers will not be truthful. It is really important for parents to put the safety of their child first and not get wrapped up in a sales pitch and false hope. Parents, and anyone they are asking for money from, need to do their due diligence and Google the heck out of any clinic, digging deep to see what’s really going on before they even make that first phone call or open their wallet. I have heard first hand some very sad stories from parents that raised money through their churches/communities (charlatans will provide a huge game plan on how to do this) to go down the snake oil road.
When the truth came out later about the how the family had been duped, the child put at risk, had become much sicker and the money wasted, the parent found that the support they once had from the donors was gone leaving them alone and embarrassed. Not to mention their personal funds gone and unable to afford the medication and therapies that were helping previously. The legal threats from these dubious clinics to the parents if they ever said anything was somehow even worse, allowing the scam to continue on.
Thankfully blogs like Paul Knoepfler’s, Doug Sipp’s http://sctmonitor.blogspot.com/2010/12/public-safety-announcement-stem-tech.html#!/2010/12/public-safety-announcement-stem-tech.html and several others have helped many avoid that fate, but there is always more work to be done to educate the public and continue to expose snake oil salesmen for what they are.