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: http://www.regulations.gov/#!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…”

Diverse Stem Cell Person of the Year 2014 Award Finalists

Stem Cell Award Poll 2014With more than 4,142 votes cast, the readers of this blog have chosen the top 12 finalists for the Stem Cell Person of the Year Award for 2014 from the 27 nominees.

You can see the final vote tallies at left. The votes came from more than 50 countries with some interesting geographic patterns (I may do a post on that as a follow up).

I’ve pasted the brief bios of the twelve finalists below at the end of this post.

Now comes the tough task for me to pick a single winner from this amazing group. I will announce the Stem Cell Person of the Year 2014 within 1-2 weeks.

The finalists are a diverse group. They include scientists from academia and industry, patient advocates, a blogger, and the Pope. We have six male and six female finalists who live all around the world including in the US, Japan, Sweden, Canada, and Vatican City.

I’m happy to see both some familiar faces from nominees and finalists from past years and new ones too.

Who would you pick as the one winner and why? Post in the comments.

Finalists Bios (including in bold quotes from nominators)

Chris Fasano. A principal investigator at the Neural Stem Cell Institute where he uses stem cells to study early nervous system development. “Chris stands out for his energy, enthusiasm, dedication to the field, creativity and accomplishments.”

Don C. Reed. Long-time stem cell research advocate who played a key role in the success of Prop 71 and the creation of CIRM. “A tireless stem cell advocate always there to make a positive difference.”

Janet Rossant. Professor, University of Toronto. Stem Cell Researcher and Past President, ISSCR. “She works tirelessly to create new opportunities and collaborations…globally respected for her work in early development and embryonic stem cells”

Judy Roberson. Long-time Huntington’s Disease patient advocate. “She makes concrete positive developments happen such as millions of dollars in research funding for HD.”

JuuichiJigen. Japanese blogger who investigates scientific misconduct and played a key role in revealing the STAP scandal. He was the first to investigate and bring to the public of problems with STAP papers. His investigations demonstrated the role of social media and post-publication peer review in rapid self-correction of science.”

Malin Parmar. Associate Professor, Developmental and Regenerative Neurobiology, Lund University. Top neural regeneration scientist. “Young, hard worker who is doing very well”.

Masayo Takahashi. Stem cell researcher leading the team that is doing the first ever clinical study based on human iPS cells. “Creative and courageous clinical stem cell researcher.”

Pope Francis. Leader of Worldwide Catholic Church. “Strong supporter of adult stem cell biotechs and research”.

Robert Lanza. CSO of Advanced Cell Technology, which has multiple ES cell-based clinical trials ongoing. “Visionary and practical so makes the impossible possible with stem cells”.

Susan Solomon. Co-Founder and CEO of The New York Stem Cell Foundation (NYSCF). Remarkably effective advocate for stem cell research. “Not many leaders have created their own research laboratories and raised $100 million plus. Seriously, what an accomplishment!”

Ted Harada. Leading stem cell research advocate and very effective ALS patient advocate. “An Energizer Bunny for the ALS community and stem cell advocate”

Tory Williams. Stem cell advocate and author of the 2014 book, Inevitable Collision. Co-Founder and Executive Director of the Alabama Institute of Medicine (AIM). “A true hero who inspires and makes real things happen like AIM”.