When I was diagnosed with multiple sclerosis in the June of 2005, there were four treatment options available for managing the disease. These treatments were considered disease modifying agents intended to increase the time between relapse. Each of these treatments also required the patient to inject themselves, which was something that I could never do. I remember the most popular question at the MS support groups was, “When will there be an MS pill?” Most of us thought that day would never come.
In the next nine years since my diagnosis, the landscape of MS treatments has changed substantially. The number of FDA disease modifying agents has grown to ten. Of the ten FDA approved treatments, there are now three approved oral treatments. All of the treatments have different levels of efficacy and allow your neurologist to personalize a treatment strategy for managing your MS.
The challenge is that out of all the treatments available to those living with MS, none of the treatment options show the promise to repair damage to the myelin sheaths caused by multiple sclerosis. The questions at the support groups now became, “Is stem cell treatment the answer?”
Dr. Violaine Harris and the team at the Tisch MS Research Center of New York (Tisch MSRCNY) seek to answer that question. Dr. Harris joined the laboratory at Tisch MSRCNY in 2004 and after a decade of research, the FDA approved a Phase 1 clinical trial to test the safety of their adult stem cell treatment. I recently sat down with Dr. Harris to learn more about stem cell therapy and MS.
Thank you for joining me Dr. Harris and thank you for all the hard work and research that you and your team have done to make a difference in the lives of people living with MS. This is truly an historic, exciting day in the history of MS research!
Dr. Harris: It’s my pleasure to have this opportunity to answer some questions about this research. The reason this clinical trial is so exciting and ‘groundbreaking’ is because it represents the beginning of a new era of treatments that can repair and possibly even reverse disability in MS.
MC: Why did you become interested in multiple sclerosis research?
Dr. Harris: My background is in cancer research and pharmacology, with a focus in stem cell biology. The regenerative capacity of stem cells was very interesting to me, especially in a disease like MS. When I entered the field of MS, I was immediately struck by how little we know about what triggers the disease and what drives disease progression. However, we do know quite a bit about what ultimately contributes to disability in MS patients, which is loss of myelin, impaired nerve conduction, and ultimately neurodegeneration and scar formation. Why not harness the potential of stem cells to try to repair that damage? Of course, there have been many challenges along the way.
MC: Many people living with MS believe stem cells can help, but the average person isn’t really sure what a stem cell is. Could you explain in layman’s terms what a stem cell is and how stem cell therapy could benefit someone living with MS?
Dr. Harris: Stem cells are unspecialized cells. An embryo is full of unspecialized cells (called embryonic stem cells) that ultimately specialize into different tissues, organs, etc. It turns out that the adult organism retains pools of unspecialized stem cells throughout the body that function to replenish tissues on a regular basis and particularly in response to damage. For MS, an optimal source of stem cells would be able to replenish and repair neurons and myelin-forming cells called oligodendrocytes.
MC: In your research, has stem cell therapy shown the potential to repair, renew or regenerate the damage to the myelin caused by MS?
Dr. Harris: When we tested our stem cells in mice, we noticed that the injected stem cells could find their way to lesion areas and send out certain signals that triggered repair, which included shutting down immune cells and increasing myelin. We were also able to replicate that process in a petri dish. Whether or not stem cells can promote myelin repair in MS is one of the questions we will be asking in this clinical trial.
MC: Conceptually, how would stem cells be gathered and administered as a treatment?
Dr. Harris: From a single bone marrow sample, we grow and freeze an entire batch of the patient’s own stem cells. The treatment consists of three doses of 10 million cells spaced three months apart. Each dose is administered into the cerebrospinal fluid through a lumbar puncture.
MC: What are the next steps in moving this trial forward?
Dr. Harris: Every regulatory hurdle has been cleared and we are ready to start data collection. At this point, the speed at which we can do the study and develop this therapy is completely dependent on the amount of funding we get. Unlike the majority of clinical trials, this study is not being supported by a pharmaceutical company.
On behalf of the entire MS community, I would like to thank Dr. Harris for joining me and sharing her research. The hard work and dedication of the entire Tisch MS Research team offers hope to all those affected by Multiple Sclerosis. For this research to continue, they need your help. Tisch MSRCNY has not received any state, federal or corporate funding to continue this important research. Tisch MS Research Center of New York, is a 501(c)(3), independent, non-profit research center meaning that any contributions to this research is considered a charitable donation. If you are going to give to one cause this year, consider giving to this important clinical study which has the potential to reverse disability and make a difference in the lives of those affected by Multiple Sclerosis.
To learn more about the research study and what you can do to help, please visit: http://igg.me/p/696737