Two Hearts, The FDA and Stem cells

By Amy Price PhD
My email informs me people are hungry for real help on finding medical providers who can offer stem cells. They want to know are stem cells a viable alternative, Can you find me quality information instead of hype, and what is your view on the FDA in this regard?

This field is moving very rapidly and what was not possible only a year ago is happeneing today.I have sen great and not so great results for orthopedic, heart, blood and some wound healing applications but it is important to realize that this therapy is still in experimental stages and there are no guarantees.

A few pioneers have moved through the opposition and fear of new therapies to pave the way for university hospitals who are now offering up stem cells in the way of research. One good example is new heart tissue grown out in Los Angeles "We seek to actually reverse the injury that has been caused by the heart attack, by re-growing new heart muscle to at least partially replace the scar that's formed," says Dr. Eduardo Marban of Cedars-Sinai Heart Institute

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A tale of two hearts summarizes this nicely. Both hearts were damaged. The first heart was in a young man whose parents are dedicated medical professionals. They waited eagerly for FDA clearance so they could take their son for a legal stem cell repair. The wait was too long and emergency surgery ensued. The young man lived but sustained brain damage and paralysis. This heart could have been repaired with adult stem cells. A company that developed the stem cell technology was less than one hour from them. The cultural mindset opinion that procedures performed outside of North America because of FDA restrictions were too risky cost them deeply.

On the other spectrum was a banker friend well known in the diplomatic community? His heart was damaged and he was urged to change his lifestyle and lose weight. He picked up over the counter diet pills later banned by the FDA and marched to the treadmill like a soldier in boot camp to the acclaim of family and treating professionals. Noticing his lips and finger tips had an ominous bluish tinge, I asked to see the pills. They contained amphetamines in the form of herbs. We urged him to see his cardiologist and to stop the pills but he chose to soldier on until a month later he collapsed on the treadmill and was taken to hospital where his chest was sawed open and major heart surgery took place as it was the only way to save his life.
The FDA found a niche because when humans are physically vulnerable they will pay or do almost anything for survival. They keep us safe from predators in the marketplace. They help us with food labels and medical warnings. Dieters have been known to ingest tapeworms, destroy their stomachs, livers, kidneys, hearts and intestines to lose a few pounds. Doctors who see patients on whom they conduct treatment for a fee will say I have to be honest with you the chances aren’t great but if it is your choice we can proceed. They are applauded for their ‘honesty’ whether the treatment succeeds or not. We say go ahead because even false hope is better than none at all. Likely none of us would hire a roofer who said I will work on your roof but because of pre-existing structural damage it likely won’t work out! The FDA has a tough job and emotional stakes run high.

On the other hand the FDA is a bureaucracy with allegiances and political agendas, not because they are necessarily evil but because this is a given for group behavior. They are shaped by society and moved by mass public opinion. They also make mistakes and if some of the lifesaving drugs we have today would have needed FDA approval masses would be dying while wars were fought that have little to do with how well something works.

Do I think most people or their doctors have enough medical training to make an informed choice about stem cells? No…but I am not sure the FDA is in a position to make this choice for the best interests of the people and I would welcome some of the transparency they have promised.

Would I consider not having FDA approval for a therapy before using it on myself or family? I would not consider a banned or illegal substance but I would have non FDA sanctioned treatment or off label use of pharmaceuticals. It has in the past improved my quality and likely my length of life. I would not consider a choice like this lightly. The risks need to be weighed against the benefits and I would have to know and trust the doctor involved whether in this country or somewhere else on the globe.

Parkinson's Disease Stem Cell Sequel

By Amy Price PhD

I found an interesting addition to the Parkinson's post's in the form of an article from Nature. Many thanks from helpful individuals at the Open University in the UK for passing this on.

In the article in Nature Suchowersky O. Transplantation therapy for Parkinson disease: the good, the bad and the enigmatic. Nature clinical practice. Neurology. 2008 Sep;4(9):465
Parkinson disease (PD) affects an estimated 1 million cases in North America . Motor symptoms in PD initially
respond well to dopaminergic replacement medications, but because PD progresses the drugs gradually lose effectiveness after about 10 years resulting in gait dysfunction and complications such as dyskinesias
PD is not just a disorder of the dopaminergic system but involves other neurotransmitter systems which inform autonomic dysfunction, mood disorders, fatigue, pain, sleep disorders and cognitive function. Dopaminergic cell replacement, even if successful is thought capable of treating only motor symptoms.
Over 20 years ago, transplantation of fetal ventral mesencephalic cells into the putamen was
performed on a small sample of participants with reportedly good results. Regrettably further studies with larger participant pools failed to confirm the initial reports (Freed CR et al. [2001] N Engl
J Med 344: 710–719). PET and pathological analyses revealed adequate survival of grafted
neurons, and there was untreatable runaway dyskenesia. However clinical benefit was seen in
a small subset of patients, 16 years on autopsy results are available Mendez et al. showed survival of
grafts without PD pathology for 14 years in five patients (Mendez I et al. [2008] Nat Med14: 507–509). Another patient showed good clinical improvement l 5 years plus, before gradual worsening of motor function and development of gait and balance problems (Kordower JH et al. [2008] Nat Med 14: 504–506). Autopsy results from this patient 14 years after transplantation showed great graft survival but grafted neurons had pathological
changes typical of PD, including Lewy bodies and activated microglia (cell scavengers) were seen in large numbers
Liet al. reported similar graft pathology in three patients up to 16 years after transplantation (Li JY et al. [2008] Nat Med 14: 501–503). These results indicate that for stem cell therapy to be effectual long term in D research needs to be initiated to investigate the spread to youn grafted neurons and determine strategies to resolve this issue. I would hope that advancement in the area of Adult Stem Cell research will make this possible in the near future.

I am finding the stem cell story is not so new. I did hear about a very young women stricken with metasticized spinal cancer who was treated at Sloan Kettering with grown out adult stem cells taken from her own bone marrow over fifteen years ago. She is now a successful professional with no trace of malignancy in her body. I also remember twenty five years ago in Canada I knew three terminal patients who underwent adjustments to the bone marrow transplant protocol that involved growing out their own bone marrow cells. One person enjoyed a year free from blood cancer and then relapsed but the other two lived. It is strange that when I go to Pub Med , Google Scholar or even the University library there is no trace of this research performed for "humanitarian reasons"

There is also a lot of interest in the London Project where stem cell trials are close to human trials for those with macular degeneration. Pfizer is quoted as expected to announce backing of stem cell therapy, apparently with joint labs in Cambridge Mass and Cambridge UK but on the Pfizer site I didn't see this in place.

Individuals are reporting encouraging success for adult stem cell treatment where they are thier own donors particularly for orthopedic and cardiac applications. These treatments are largely privately funded. It would be great to see this in the mainstream covered by insurance and available for people regardless of income levels

Parkinson's Adult stem cell Breakthrough and more

More about Parkinson’s and stem cells….Keeping you posted as I learn too… The photo showing where damge occurs is from web MD (2002)

Would you be surprised that the technology to implant neural tissue in humans to alleviate Parkinson’s Disease was available as early as 2001 and in case studies has produced more successful remission than any drug on the market and with fewer side effects?

Michel F. Levesque, M.D., F.R.C.S.(C), F.A.C.S.Cedars-Sinai Medical Center in Los Angeles, Associate Clinical Professor of Neurosurgery at the UCLA School of Medicine, and member of the UCLA Brain Research Institute applied for Phase 11 FDA approval in several years ago. This was granted but sponsor complications and new rules by the FDA have put this phase on hold until Levesque and company can jump through the new hoops and negotiate more sponsors. This is tragic for Parkinson’s patients who could die or degenerate significantly before help is available.

It seems that the FDA would apply the same standard to our own cells that they do to foreign drugs. Pharmaceuticals take a ten year, 750,000 million dollar investment on average to bring to market and to start to make a profit. I do wonder about the stability of stem cell companies that have not counted the cost or prepared a strategy and stored up ammunition for battle. What did they think was going to happen? Nevertheless it makes one long for the days when scientists bit the bullet and practiced on themselves and family members. The results produced their own script. It seems today the trend is to scorn the establishment while begging to get into ‘the inner circle’. This cannot in all fairness be blamed on the FDA but rather on the inconsistencies of human nature and the need to belong. It is tragic that ethics and human decency for ‘the little people’ are tossed aside in the parade of blind ambition that ignores the simple concept that if you take away from the foundation (those that have supported your growth) the structure will destroy you…but I digress, on with the story….

It does seem strange that it is a woman’s ‘right’ to terminate a child but it is not an individual’s right to choose their own stem cells. The right to terminate brings death and is endorsed but the right to life through the harvesting of our own stem cells is denied by an outside source.

It is not like the FDA is even particularly good at culling out the problem children in the industry or adding to medical knowledge. A simple Google search on major drugs and problems in approved FDA pharmaceuticals is an eye opener in politics and commerce rather than the mainstream of patient safety for which they purport to represent.

Levesque and his colleagues did a successful case study with a man suffering from Parkinson’s in 2001 and yet in 2009 Parkinson’s patients are still dying needlessly. The FDA is still a bureaucracy which people are distracted into attacking while Scientists followed by the masses line up on the adult stem cell or embryonic bandwagon instead of putting partisan economics and politics aside to join together as scientists to promote healing and answers for those without hope.Here is a little about the study below:
“MATURE NEURONS DERIVED FROM THE PATIENT’S OWN BRAIN CAN BE TRANSPLANTED BACK SAFELY AND IMPROVE SYMPTOMSWe recently presented the clinical outcome of our autologous method at the International Congress of Parkinson’s disease and Movement Disorders in Rome. In accordance with our institutional review board, we transplanted a patient with advanced Parkinson’s disease with differentiated neurons derived from an initial needle biopsy. At three years post-operatively, the overall Unified Parkinson’s Disease Rating Scale (UPDRS) improved by 81% while “on” medication and 83% while “off” medication. We demonstrated here the long-term clinical remission of Parkinson’s disease symptoms in a single patient.Because of their biocompatibility, safety and potential integration into the host striatum, autologous adult neural stem cells and stem cell-derived neurons represent an effective alternative to current cell therapy aimed at the restoration of dopamine neuronal loss in Parkinson’s disease. Under the guidance and supervision of the Food and Drug Administration (FDA) office of Cellular, Tissues and Gene Therapies and the Center for Biologics Evaluation and Treatment (CBER) we are about to begin Phase II trials using this promising cell therapy.
CONCLUSION
Degenerative and traumatic disorders of the brain represent an enormous burden to the patient, their family and health care providers. The current debate between the embryonic stem cell proponents and those who are opposed to their use distracts from other avenues with promising outcome, such as adult stem cell therapy. It also overlooks other important issues of resource allocation between basic and clinical research, health insurance, and patient care. Scientific knowledge has rapidly progressed in the last five years (written in 2004) and stem cell research and therapy remains a very promising field for treatment of neurological disorders. In a recent biotechnology industry meeting, a presentation had the approximate title: “Businesses are from Mars, Academics are from Venus”. What was forgotten there is that patients are from planet Earth and this is what should guide our efforts “ (Levesque, 2004)For the complete story read here and for an update on where things are today check out this link

Love S et al., Glial cell line-derived neurotrophic factor induces neuronal sprouting in human brain, Nature
Medicine 11, 703-704, July 2005
Slevin JT et al., Improvement of bilateral motor functions in patients with Parkinson disease through the unilateral intraputaminal infusion of glial cell line-derived neurotrophic factor, Journal of
Neurosurgery 102, 216-222, February 2005
Gill SS et al.; “Direct brain infusion of glial cell line-derived neurotrophic factor in Parkinson disease”;
Nature Medicine 9, 589-595; May 2003 (published online 31 March 2003)

Stem Cells For Parkinson's

By Amy Price PhD

Can Parkinson's patients benefit for stem cell therapy? The answer is likely that in the future this will be possible. Right now stem cells for orthopedic applications are a working reality and cardiac
applications are showing great success.

It was interesting to see a company with customer testimonials claiming success for Parkinson's . What happens is they are injecting stem cells intravenously and claiming these cell are migrating to the brain to "fix" Parkinson's. There are big problems with this...some cells migrate but the number of cells that would actually make it to the brain are fewer than finding a friend in a 50,000 seat foot ball stadium, the rest of the cells go the the lungs and then are reabsorbed by the body and filtered out through the liver and excreted by body wastes. Patients are paying thousands of dollars and travel expenses for cells they will end up flushing.

Think about it this way. If cultured cells could just fix things intravenously why would leukemia
patients even need bone marrow transplants? With Parkinson's the underlying cell structure in the brain has been compromised and unless that is restructured new cells will be taken over like ants on a chocolate cake. For stem cells to work treatment needs to be targeted and care needs to be given as to how to best treat the underlying problem.

So why are some Parkinson's "patients" saying it could be working for them?
First of all Parkinson's is a dopamine deficiency issue. Anytime we do something
that brings pleasure or excitement, dopamine is circulated. For a short period of time patient's actually feel better. This principle is biological not psychosomatic. Plus the cells have some anti inflammatory properties so this benefit is felt as a temporary relief from pain.

Just after Thanksgiving a family member ran over our cat in full view of three small children. Having done some veterinary work and some first aid I could see the cat was not long for this world. She was bleeding from her mouth and her lungs were filling with blood and yet I held her in my arms and my son and I drove to the vet in hopes that someone bigger and smarter could do something...they couldn't. I guess I would do the same for a person I cared about and so would a lot of people. This makes us vulnerable in spite of knowledge so even when a celebrity claims to be healed it may not be the whole story. About the Parkinson's stem cells.... no fMRI or electrical studies of before and after treatments are shown and a search on PubMed turned up nothing.

This is in stark contrast to stemcell company scientists who publish research results and actual case studies to show where and how treatment is progressing. One question to ask when you are considering a new medical treatment is do the people I am dealing with offer any other options in case of candidates that are not suitable for the procedure?

What can you do right now until stem cells catch up with the ability to treat
brain disorders? There are meds, deep brain stimulation and brain training
options that can all help. The love and care we surround people with also makes
maximum use of the dopamine available and can enhance the quality of life for a
loved one with Parkinson's.....keep posted as soon as we see safe viable
treatment it will be posted here!