Are Remyelination Strategies Realistic?

At the 2017 Rare Neuroimmune Disorders Symposium, Dr. Benjamin Greenberg from the University of Texas Southwestern Medical Center gave a talk about remyelination strategies and announced the beginning of a Phase I trial in transverse myelitis. This is the first FDA sanctioned cell therapy to promote repair through myelin regeneration in Transverse Myelitis, which is set to begin in 2018. The University of Texas Southwestern, Q Therapeutics, Inc. and The Siegel Rare Neuroimmune Association are collaboratively sponsoring this first human safety study to treat central nervous system disease.

How did this trial become reality?

Dr. Greenberg presented the stages of clinical research. The first step is preclinical development to develop the agent, whether it’s a cell or a molecule that then has to be tested for efficacy. The next step is to prove that there is no obvious or expected toxicity from that therapy. This preclinical development can literally take years or even decades to complete. In the United States, regulatory approval is required through the FDA where they grant what’s called an IND, or an Investigational New Drug application. A phase I trial is primarily meant to measure safety. As long as we make sure an intervention is safe, then phase II and phase III studies get approval to market that therapy to the rest of the world.

How does this cell therapy remyelinate?

Q therapeutics developed a glial-restricted precursor cell that develops into oligodendrocytes. Oligodendrocytes are cells that produce myelin, the insulation around nerves. In mice that are born with nerve cells without myelin, these cells have been shown to produce myelin.

Where are we now?

UT Southwestern and Q Therapeutics, with input from SRNA, collaborated and put in an IND application to the FDA, that was granted in July 2017. This means the FDA gave us approval to move into human remyelinating trials for transverse myelitis in a Phase I trial. This type of study requires funding. The CONQUER program at UT Southwestern secured a one and a half million-dollar gift to fund the clinical portions of the phase I trial. SRNA has been working through The Pauline H. Siegel Eclipse Fund to raise money to cover non-clinical costs. If we raise all the money needed, we expect to begin enrollment in mid-2018. The phase I trial will include nine non-ambulatory adult transverse myelitis patients who are more than a year out from their diagnosis.

How can you get involved?

If you are interested in learning more about this trial and other research, please fill out this form. You can also contribute to The Pauline H. Siegel Eclipse Fund to raise money to cover the cost of the trial. Every donation received will be matched dollar for dollar up to $150,000 by a generous anonymous donor.