The crowd pitches in $243K (and counting) for clinical trials of a stem cell therapy for MS

While donation-based crowdfunding has taken some industries, like consumer technology, by storm, its been dismissed by some as a source of early-stage life science funding because of the sky-high price tag that biotech and medical device R&D carries. Raising a few thousand dollars wont really do much to get a company off the ground.

But a few recent campaigns are challenging that idea, pulling in more than $100,000 in donations through Indiegogo. The latest is Tisch MS Research Center, a nonprofit research center in New York thats so far collected more than $243,000 for a Phase 1 clinical trial of a stem cell treatment for multiple sclerosis.

Tisch researchers have gotten the green light from the FDA to proceed with a safety and tolerability study of a treatment it says has the potential to reverse damage done by MS. The autoimmune disease attacks the protective coating of axons (the myelin sheath) in the brain and spinal cord, disrupting the ability of nerve signals to travel properly. That can cause numbness, pain, vision loss, difficulty walking and even paralysis.

Stem cells are hypothesized to promote repair in MS by migrating to areas of demyelination, blocking damage-forming events and enabling repair, Tisch researchers explain on their campaign page.

Their proposed therapy uses neural progenitors that are derived from adult stem cells isolated from bone marrow to promote repair in MS. Bone marrow is taken from a patients hip or sternum and sent to a lab, where stem cells are isolated and neural progenitors are derived from the sample. Then theyre administered in multiple rounds intrathecally into the cerebrospinal fluid, rather than into the bloodstream every three months.

The researchers have demonstrated proof-of-concept and feasibility in preclinical studies and are planning a Phase 1 trial that will enroll 20 progressive MS patients. They would receive six months of treatment, followed by up to 27 months of follow-up by the researchers, who say enrollment would begin once they obtain the necessary funding and IRB approval.

According to most recent estimates, the number of people living with MS has gone up 10 percent over the last five years, reaching 2.3 million. A new drug made by Biogen Idec was FDA approved last year for treating relapsing MS, and a number of treatments are in late-stage development. Cell-based therapies, though, havent advanced quite that far yet. But in a paper published in The Lancet last fall, researchers expressed optimism about the evolution and translation of such therapies for treating MS.

Tischs researchers have been working for more than 10 years on their investigational drug.

[Image credit: Tisch MS Research Center]

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The crowd pitches in $243K (and counting) for clinical trials of a stem cell therapy for MS