When you want to conduct research on human subjects at a university or a hospital, you must first pass rigorous inspection by an IRB (Institutional Review Board). The job of the IRB is to look at the ethics of your proposal as well as weigh the potential risks vs. the potential benefits to the participants. Naturally, when we began to consider stem cell treatments for Evangeline we knew there would be a lot of information to go over so we formed our own family “IRB” which would consist of voting members.
The information compiled here is not intended to serve as either medical advice or an endorsement or rejection of the idea of using stem cell transplants as offered abroad. The subject of stem cells is highly charged. It is not my intention to be offensive or to cast any disparagement on anybody’s deeply held beliefs. I am simply being a scientist, putting forth ALL the research I could find on the subject as it pertains to what we are considering for Evangeline. At the time I write this our family IRB board has not voted. According to the rules we set forth before our scheduled meeting date, in the interest of erring on the side of caution we need a unanimous vote in order to proceed with the treatment.
· First, I could not find ANY research existing ANYWHERE about stems cells and spina bifida. This doesn’t mean such research doesn’t exist but it does mean it’s not readily found via typical search routes.
· The research I could find was based on Spinal Cord Injury (SCI). SCI is not analogous to spina bifida. In SCI the individual had a perfectly formed and functional spinal cord that was damaged. In spina bifida the cord is not fully formed or functional. Though it varies on a case by case basis, many children and adults with spina bifida have nerves that don’t connect to anything or never formed at all. There is nothing in science anywhere that says stem cells can grow or reconnect nerves. However, in addition to having nerves that are not there/not connected, individuals with spina bifida (to varying degrees) do have formed and functional nerves that were damaged either during prenatal exposure to amniotic fluid or during closure surgeries/tethered cord surgeries. So, if stem cells ever fully “cure” SCI, they will not ever fully “cure” spina bifida (and not just because SB isn’t a disease to start with). The only parts of spina bifida that we can say stem cells will address (based on SCI research) will be those formed but damaged nerves.
· Most research on SCI (here in the states) is based on “animal models” meaning research involving human subjects is only just beginning. Animal models don’t tell us as much as a human model would, but they do tell us some things.
· As I mentioned before research is just now really being done in the US on stem cells. This is likely because stem cells became a “hot topic” in the abortion debate. Some people thought that the only source for stem cells was aborted fetuses. This is not the case. Stem cells originating from a fetus are called “embryonic” stem cells. “Adult” stem cells can come from the umbilical cord and placenta of a full term baby. You can also get stem cells from bone marrow and fat cells. Regardless of the reasons, stem cell research was banned for a time and slowed by the controversy surrounding it. If you want to see what clinical trials are being done on stem cells here in the US go to http://clinicaltrials.gov/ and do a search for stem cells. You will see that there are a couple of thousand underway this time (for SCI as well as some for diabetes, cancer, autism, traumatic brain injury, Alzheimer’s and others). A large one that will eventually be of interest to the SB community when the results return is on “Safety and Efficacy of Autologous Mesenchymal Stem Cells in Chronic Spinal Cord Injury” (this means they are testing how safe and effective stem cells they get from the patient’s own bone marrow are in treating spinal cord injury). Most studies are only for those 18 and up and none (as I mentioned before) include individuals with SB…but the results of this study will answer 1) if stem cells are safe and 2) if they help improve function and sensation.
· Many countries have USED stem cells but have not systematically studied or regulated their effectiveness. Japan has many places for people to get stem cell treatments (from everything to looking younger and growing hair to genuine medical reasons to seek treatment). Here is an interesting scholarly article on the need to regulate stem cells use in that country http://www.practicalethics.ox.ac.uk/__data/assets/pdf_file/0009/29727/Akabayaski-Fujita.pdf You will see that some side effects from treatment are mentioned such as brain tumors and one individual who had cells injected into her kidney only to grow bone marrow and blood cells there. The article addresses and dismisses many of these cases as either the patient health before injection (one man was 71 years old) or the fact that the wrong kind of stem cells/unprimed cells were injected into the patient. This article demonstrates that while stem cells are generally low-risk, there can be rare but serious side effects if the practitioner administering the cells is hap hazard in how they are handled or does not properly make sure the patient is healthy.
· There is a FREE on-line peer reviewed scholarly journal called Journal of Stem Cell Research and Therapy which does update with new articles http://www.omicsonline.org/stem-cell-research-therapy.php
· I got a LOT of info from this article. http://www.omicsonline.org/stem-cell-research-therapy.php It is a meta analysis of several hundred animal studies on SCI. A meta analysis is where they look at many studies and create complex mathematical models that show trends and explain the differences in study results. Because meta analysis includes multiple studies it is considered the gold standard of research statistics and often tell us more than any single study. I was very pleased to find this article. Its also recent, being published in 2013. This study found that over all stem cells lead to some improvement in motor and sensation, although for whatever reason males had greater improvement than females. When researchers didn’t know which group of mice got the stem cells, efficacy scores were lower. Injection of cells by IV vs direct injection to the spine showed slightly more improvement. While the type of manipulation to the stem cells before injection DID NOT affect motor scores, it DID affect sensory scores. Animals injected with cells which were pre-differentiated (primed to be a specific kind of cell) had a greater increase in sensation after injection with the cells. Even the type of anesthetic used during the various studies seemed to have an effect on how much the cells impacted recovery.
· The conclusion we can draw is that YES, stem cells are beneficial in SCI. The degree to which they are beneficial varies greatly and how that translates into helping spinal nerve damage in spina bifida would also likely vary greatly.
· We cannot ignore what science call “anecdotal evidence” or “case study”. This is considered the least generalizable form of research in science (meaning it doesn’t tell you much about how everybody else will respond to a treatment, only how one individual does. Enough case studies taken together though can be more generalizable). Many individuals have taken their children (or themselves) over to other countries for stem cell treatment. Most have reported some improvement ranging from mild to WOW. On one hand this confirms the research studies- stems cells help but results vary. One the other hand, you cannot ignore what is called “confirmation bias”. That’s a fancy psychological term that means people generally find what they are looking for. Since these programs cost so much, the individuals who go for the treatment are invested and looking for results. There is a chance that those seeing only very mild results do not actually have any documentable change in either sensation or movement. This is likely NOT because stem cells do nothing, but may be because some places that administer them are less than reputable. 60 minutes did an investigation of certain companies offering stem cell therapy and found that many had stem cells that were not even ALIVE. They were administering useless dead cells that wouldn’t do more than grow your fingernails. http://www.cbsnews.com/news/stem-cell-fraud-a-60-minutes-investigation-26-08-2012/ So a big part of the stem cell process is finding an agency that you can TRUST- one that will not take your cash and leave you with nothing.
· So the information/questions before the Kugler IRB is as follows:
o Yes, stem cells help SCI; Do we believe we have enough info to say stem cells may help SB?
o How can we know the company we are using will deliver the product it says it will? (here we would need to set forth guidelines)
o Do we believe we have the necessary information to minimize “rare but serious side effects” to our daughter through stem cell treatment? (here we need to set forth criterion to minimize risks)
· After we have addressed the above issues/questions, each member will vote on whether or not we proceed for stem cells for Evangeline at this time.
I hope this information is helpful to you and a place to start your own further research if you want to look into stem cells for your child.