By Dr. Mindy Aisen
The field of stem cell research offers enormous promise for the treatment of human disease. Stem cells are “undifferentiated” cells; they have the potential to develop into virtually any specialized type of cell in the body. So theoretically, stem cells might be used to regenerate human tissue or organs damaged by disease. There is hope that some day stem cells may provide effective restorative therapy for many human conditions, including those caused by damage the nervous system such as stroke, Parkinson’s Disease, spinal cord injury and cerebral palsy. At the present time, however, the field of stem cell research is in its infancy, and there are few effective uses of this type of therapy.
Stem cells can be derived in several ways. Embryonic stem cells (removed from early stage embryos) can be maintained in the laboratory and coaxed into differentiating into various cell types. Fetal cord blood contains human stem cells that can also be grown in the laboratory or frozen for later use. A recent technique for generating human stem cells involves obtaining cells from skin biopsies and “deprogramming” them into stem cells that can then be differentiated into various human cell types. The adult human brain contains viable neural stem cells; it may be possible to use medications to stimulate these natural stem cells into regenerating damaged brain tissue.
Stem cell scientists are currently working on the optimal conditions to maintain stem cells in the laboratory, the precise methods necessary to convert stem cells into the specific cells that might reconstitute damaged human tissues, and the techniques required to direct such cells to function effectively together. At the present time, much work remains to be done before the practical application of stem cells to the treatment of human disease becomes feasible.
In cerebral palsy, brain tissue is damaged at a very early stage of development: in utero, at or around the time of birth, or up until the age of 2 years. The brain is, of course, the most incredibly complex of all human organs. The brain consists of many different cell types interacting in a precisely organized fashion to produce the different aspects of thought and behavior in different brain regions. Before stem cells can be used to repair damaged brain tissue in cerebral palsy or any other brain disorder, scientists will have to discover how to turn stem cells into various specific types of brain cells, and induce them to form the precise connections and organization necessary for meaningful brain function. It is likely that stem cells converted into brain cells will have to be implanted in the precise areas that they are needed in order to provide effective treatment.
Specific Types of Approaches: Cord Blood Infusions
It is extremely unlikely that the administration of stem cells by peripheral infusion of cord blood can effectively treat cerebral palsy. Such cells have not been “taught” to form the necessary types of brain cells, and they will not be able to enter the brain, because there is an anatomic and physiological barrier which prevents certain medications and cells from reaching the brain, known as the blood brain barrier.
How can one explain the reports of improvements in the symptoms of cerebral palsy (CP) following cord blood infusions? Medicine is filled with such “anecdotal” reports of improvements when novel treatments are applied to chronic conditions. The symptoms of all chronic conditions fluctuate, and subjective factors, including the optimistic expectations that accompany novel therapies, often seem to alleviate disease manifestations. But in almost all cases, the underlying disease remains unchanged, and there is no meaningful long-term benefit. Indeed, in most cases, the risk of harm outweighs the chance of benefit when unproven novel therapies are used.
CPIRF strongly supports research into the use of stem cells to treat CP. But at present, CPIRF, in consultation with leading stem cell scientists, has reached the firm conclusion that use of cord blood or other forms of stem cell treatments for CP is inadvisable. Basic and applied research into various approaches to stem cell neural regeneration therapy must be vigorously pursued, and CPIRF will continue to fund such efforts. But at present, administration of cord blood to people with CP offers no meaningful chance of benefit.
Furthermore, the long term risks of cord blood infusions have not been studied, receiving infusions in other countries from donated cord blood may have significant risk and certainly present substantial financial strain for the families of those being treated.
We strongly endorse an organized scientifically rigorous initiative focused on rapidly identifying the most effective methods for using stem cell treatments to help repair the damaged brains of children and adults with developmental brain conditions.
Most treatments for cerebral palsy (CP) are initially directed toward children. What is not clearly established is the long- term effects of such treatments. Many appear helpful in the short term but prove to be disadvantageous in the long run. Selective dorsal rhizotomy (SDR) is a permanent, irreversible neurosurgical procedure for reducing spasticity in cerebral palsy. Parents contemplating SDR for their child would like assurance that that there will not be harmful complications from it as the child ages into adolescence and adulthood. We now have new evidence...
Hello Dr. Aisen
Given encouraging results being conducted at Duke University (Dr. Kurtzberg) and numerous other published (granted, non scientific journal) results of individuals with CP and brain trauma having received autologous (one’s own) stem cell transplants, have you considered clarifying your position on stem cell transplants? Your write-up of December 2008 seems to specifically address allogenic (foreign cell) transplants but not autologous (one’s own cell) transplants.
Your position discusses allogenic transplants where of course there may be rejection risks. However, what are the risks of an autologous transplant? There is strong evidence that autologous transplants seem to have been beneficial for some patients. What is not known, is whether this is a statistical minority or majority of patients being treated. But, so what? If the risks of autologous transplant are negligible, even if a minority of transplants were an evident success, wouldn’t it be valuable for parents to consider this?
I’d be grateful for your thoughts.
Kind regards,
Eugenio Ramirez
I don’t understand the hesitation to use stem cells as a treatment for people with cp. These individuals, and their families suffer enormously, and not enough is being done to improve quality of life. It seems you have to meet a certain criteria in the US to even be considered for an exam, yet countries around the world are moving beyond the mental block that seems to prevent progress in America, and success is being reported in every case. I have 3 grandchildren with different levels of cp. The 9 year old is the most severe, not sitting, non-verbal, etc. However, very aware of his surroundings, and what is happening. I am preparing to get stem cell treatment this summer for all 3 of the children. We are not necessarily looking for a cure, but quality of life. I don’t see any reason not to go forward. I wonder if some of this uncertainty being spread is to keep things as they have been. Comfortable. You go to therapy, physical,speech, occupational. Wheelchairs, standers, braces, etc. are continued purchases. No one gets better, and everyone keeps their income. The thing that saddens me the most is the fact that I am going to have to travel outside the US to get the treatment that my grandchildren deserve. The money that I could be spending here, is going to be spent in another country. Time is not on our side. I can’t wait on the US to get through 30 more years of testing. Every day I wait is another day they are denied. Thank God, and the websites this treatment is available to those who don’t meet the guidelines in the US.
Is there any treatment programs for adults with CP? It seems like all the research is on children. I am 33-years old and I would go anywhere to attempt a better quality of life.
Artical was very clear. I have a 8 yr. old granddaughter with cp. We are also waiting, and waiting for stem cell treatments to come to USA. I have emailed about 15 stem cell treatment places off shore. Each one states that their treatment is the best, even though they also advise that there are no garanties, each patient will get different degrees of improvement. That seems to me that they have such a gimick going. If my granddaughter doesn’t improve the stem cell place can say, well each patient is different. So there’s no proof of what stem cells they used and where it should be injected. I have spoken to Dr. Evan Snyder and he does agree that stem cell treatments for cp is very difficult. He also states that the brain has so many types of brain cells, each doing their own special job, that first they have to find this out before any transfusions can go on. Then there is Dr. Kurtberg who uses autologous cord blood stem cells for cp. I have only heard of one patient, Dallas Hextell who was on TV stating how much better he got. Then I have not heard any other results. I don’t think she is finished with the investigation, but this would be so helpful to know. Then there’s the great possibiity that we didn’t save the cord blood. It can only be saved at birth and who knew to save the cord blood 8 yrs. ago. I am killing myself over that. And she is not infusing the cord blood right near the damaged cells in the brain. There are so many, many opinions out there. So, I chose to wait for fear of spending great deals of money for a treatment that’s not going to work. I contacted a famous doctor in Israel and he wanted $1250 dollars for reviewing the history alone and then another $32,000 for treatment. That does not include the plane fare. After I requested a little discount and for him to research our doctors here in the USA he refused to treat my granddaughter. The best of the scientists and researchers here in the US advise not to go off shore. That what they are doing off shore will not work for cp. Oh, these off shore places will take any age patients. Here we are waiting. It’s certainly not the researchers in US fault. It’s just that the brain is the hardest organ because it has many types of neurons with specific jobs and they all interact with one another. You can reply to my email. Thanks, Shelly
Thank you for your inquiry. The article by Dr. Aisen, our former Medical Director, provides sound advice and summarizes well the state of stem cell therapy for cerebral palsy. It is frustrating for families to be cautious and patient by one’s doctors here in the US on the one hand but be offered the prospect of cure by facilities outside the United States. It is the consensus among cerebral care physicians in the US that this therapy is not ready for general availability and should be restricted to carefully designed research studies.
An additional more recent source of information about stem cell therapy in cerebral palsy can be found on the following website by our colleagues in Canada.
http://www.neurodevnet.ca/kt/stem-cells
James A. Blackman, MD, MPH
Medical Director
Cerebral Palsy International Research Foundation
186 Princeton Hightstown Road; Building 4; 2nd Floor
Princeton Junction, NJ 08550
This is Shelly Ratner again. I am still researching and researching, waiting and waiting. Who knows what decade the stem cell scientists will agree that it is finally safe to do the treatment. I want to hear results from Dr. Kurtzberg at Duke and Dr. James Carroll at Georgia University who are both using autologeous (one’s own cord blood) stem cells for cp. What are their results? Speak up already. And what if you didn’t save the cord blood at birth. Who knew back then. So many questions. Then there is a Dr. Margolis who swears that the off shore treatment places he recommends to go to really work. Where is that proof? Can you rely on families telling you the truth or is that a scam? We need an investigation or someone willing to get to the very truth here. Then the off shore places say that the only reason that they have to perform these treatments off shore is that the US is not ready yet. Who do we believe? I really do respect Dr. Evan Snyder at Sanford University who says that stem cells for cp will not work yet because stem cells have to pass the blood brain barrier in order to repair the damaged neurons. And that stem cells from other parts of the body will not be able to break into the blood brain barrier. So, where do we get healthy neurons who will be able to pass the blood brain barrier and repair damaged neurons in the brain. Reply, please.
HELLO AGAIN, JUST TO LET YOU KNOW THAT I DECIDED AGAINST GOING ELSEWHERE FOR STEM CELL THERAPY. I REALLY WANT TO GO AHEAD, AND DO IT, BUT HAVE DECIDED TO TRY AND WAIT. THE THING THAT IS REALLY UPSETTING TO ME IS I CAN’T FIND ANY TRIALS BEING DONE FOR STEM CELL RESEARCH FOR CP KIDS WHOSE CORD BLOOD WAS NOT SAVED. HOW LONG WILL IT BE BEFORE THESE KIDS WILL GET A CHANCE? EVERY DAY PROLONGED IS ANOTHER DAY THAT THEY CAN NOT BE A CHILD. HOW UNFAIR IS THAT? THESE RESEARCHERS NEED TO GET ON WITH IT. I WOULD LOVE TO SPEND MY MONEY HERE IN THE U.S. FOR MY GRANDCHILDREN TO BE HELPED.