Patients talk stem cells. The FDA listens.

By Jeanne Loring

Last Tuesday I visited the FDA headquarters in Maryland, for a meeting called Public Meeting on Patient-Focused Drug Development for Huntington’s and Parkinson’s Diseases. The FDA holds about 6 meetings on different diseases each year, inviting patients and advocates to speak while a panel of FDA directors listens. The FDA representatives at this meeting included Billy Dunn and Eric Bastings, (Director, and Deputy Director, respectively, Division of Neurology Products [DNP], Office of New Drugs), Susanne Goldstein (Medical officer, DNP), and Theresa Mullin (Director, Office of Strategic Programs). All of these people are from the Center for Drug Evaluation and Research (CDER), which is the branch of the FDA responsible for evaluating new drugs. Biological therapies, including stem cell therapies, are evaluated by another branch of the FDA, the Center for Biologics Evaluation and Research (CBER). These CDER members will not be directly examining stem cell therapies, but it’s still useful for them to be aware of them – and they may have friends at CBER.

Summit for Stem CellsThe morning session, on Huntington’s disease, was both disheartening and inspiring: disheartening to hear about how horrible the disease is, and how it affects families – inspiring to see how hope still lives among the stricken and their family members. There are no treatments- none. HD is caused by a trinucleotide repeat in an essential gene that changes its function; the man next to me introduced himself as “a 43 CAG”, which means that he has 43 repeats of the trinucleotide CAG, enough to cause moderate pathological symptoms. His son, he said, is a 46.

Jenifer RaubThe afternoon was for Parkinson’s disease. I was there with two members of the Summit for Stem Cell Foundation, which is supporting development of a patient-specific stem cell therapy in my lab in collaboration with Dr. Melissa Houser at Scripps Clinic: Sherrie Gould, a nurse practitioner who treats PD patients is the heart of the foundation, and Jenifer Raub (pictured in photo from the meeting at left from Jeanne Loring) is a patient who speaks eloquently about the impact of the disease on her and her family. Jenifer was one of 5 patients who were chosen to speak directly to the FDA on a panel about therapies. If you want to add your own comments, the FDA is taking comments until November 23 at this link:!documentDetail;D=FDA-2012-N-0967-0747 .

Jenifer’s words will appear in the transcript and are far more meaningful than anything I could say- here is some of what she said:

“My Name is Jenifer Raub. …I have Parkinson’s disease….Every day is a struggle with the ups and downs of Parkinsonian symptoms and at the end of each day I am exhausted and frustrated.

I never know what tomorrow brings. Another day filled with the same unpredictability – Can I stand? How much pain will there be? How long will the medications that are working today continue to do so???

Acceptance of this instability is often frustrating. I heard a lot of frustration in persons here today…I have days I’m reminded of the fact that the medicines I take will not be effective forever. Parkinson’s disease is progressive. Those are the days I look at my children and grandchildren and wonder: How long will the medications be effective? How much time do I have?

My life has a new definition of “normal”. Parkinson’s disease has redefined my family, friends and marital relationships. There is NO cure for Parkinson’s disease today.

And, yes, I have looked for alternate treatments for my condition. I do not want to be dependent on medications for the remainder of my life… I strongly believe that there is great promise in the NEAR future for stem cell therapy for the treatment of Parkinson’s disease -specifically induced pluripotent stem cells, which are patient specific and DNA matched…Stem cell therapy in Parkinson’s disease has a 30 year history. Decades ago studies were done using fetal cells. Those participating in those studies are now dying of natural causes and the dopamine cells at the time of death were still viable! Fast forward 30 years – today a simple skin cell can be made into a pluripotent stem cell and consequently a dopamine producing neuron…. [Summit for Stem Cell hopes to file an application with the FDA in 22-36 months. I know the risks and side effects of the medications I take. I know the risks of stem cell therapy. I am willing to take the risk rather than acquiesce to Parkinson’s disease. Let me – Let US have the right to take the risk and the right to choose HOW we fight the symptoms of Parkinson’s.

I fight EVERY day to move. I am fighting for a life with my family…I want a future with the simple joy of being with my husband, my children and my grandchildren. I want to see my grandchildren graduate college. I want to attend their weddings and I want to meet their children and kiss their foreheads.

Time is limited for someone with Parkinson’s.

MY time is limited.

Give me a chance to choose. Give all of us a choice. I would like to ask the FDA to consider supporting and furthering this new and revolutionary field of regenerative medicine that will change therapies and treatment for not only Parkinson’s disease but for so many diseases that there is NO cure for today.

Thank you for your time…I appreciate you listening to me…”

5 thoughts on “Patients talk stem cells. The FDA listens.

  1. What a brave lady to stand up and say these things. What she said is what I truly believe too. “Let US have the right to take the risk and the right to choose HOW we fight the symptoms of Parkinson’s” and many other medical problems.

  2. The regenerative medicine field shows so much promise, it has been proven safe and in many cases shows a high degree of efficacy. It is unfortunate the lobbyists that represent Big Pharma are able to suppress the hopes and dreams of so many suffering from diseases with unmet medical needs.

  3. Kevin – “The regenerative medicine field…has been proven safe” You see, these are the kind of loose statements that create expectations in patients. A whole field can never be proven safe and for stem cells acceptable safety has been shown for some diseases but not (yet) in others. There are still reports of tumorigenesis and ectopic growth in some preclinical studies. I hope many more diseases will be tackled soon but we have be realists in the clinic.

  4. Jeanne – thanks for posting. This is an important move for both patients, the FDA and for stem cell therapy advocates. I realize the FDA has been running such public meetings for a while, but this one incorporates several aspects in one meeting.
    In taking action to speed stem cell therapies to patients, FDA cannot simply reduce the standards set for drug approval but may have to create new definitions for approval. This may also help them come to some decision soon on how to regulate different types of stem cell clinics, as patients are also at risk through non-activity in this regard.

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