Advancing Potential ALS Treatments Is Like Accelerating a Glacier
Glaciers are known for their slow movement and the transformative change that they leave in their wake. Most move only a few centimeters a day. Yet they produce lakes, cliffs, moraines, valleys, mountain arêtes and horns, and pronounced landscape striations. Perhaps stem cell research will prove analogous.
BrainStorm Cell Therapeutics is enrolling a Phase 3 study that uses bone-marrow-derived cells extracted from the patient. These cells can differentiate into bone, muscle, fat, and cartilage. Prior to reintroduction, the cells are cultured to produce higher levels of neurotrophic factors — chemicals that protect neurons. The trial is attempting to gather 200 participants who are declining at least one point per month as measured by the ALS Functional Rating Scale (ALSFRS).
Preclinical research into the use of stem cells in ALS was first done in mice in 2006. Based on these mice studies and evidence that stem cell injection was safe, BrainStorm began a clinical program called NurOwn. A Phase 1 safety and tolerability trial demonstrated both. The company then sponsored a Phase 2 trial, and no serious adverse events were observed. It was reported that the treatment slowed disease progression as measured by ALSFRS. Increases in muscle strength and bulk, and also arm and leg circumference, were observed.
BrainStorm then conducted a larger Phase 2 trial. The primary goal was to confirm safety and tolerability, and a secondary objective was disease modification. Again, there were no serious adverse events. More importantly, once the study participants were divided into fast and slow progressors, the fast-progressing subgroup was shown to have an improved rate of decline.
Topline data from the Phase 3 trial is expected in 2020. That means, pending positive results, a stem cell treatment for ALS could be approved in 2021. And there may be more coming. Q Therapeutics has plans to launch a clinical trial this year. Neuralstem has developed a spinal-cord-derived neural stem cell line, currently in clinical-stage testing, for the treatment of ALS and other nervous system diseases and injuries. The Mayo Clinic and Cedars-Sinai also have undertaken research initiatives into the efficacy of stem cell treatment relative to ALS.
That is all exciting and promising. But if an ALS stem cell treatment is commercially available in 2021, it will mark 15 years since it showed potential in the lab. To anyone with ALS, the pace of progress feels glacier-like. However, if the impact on the patient population is equally glacial, the time to market will be quickly forgotten.
It is impossible to predict the gains to be had in modifying disease progression via stem cell therapy. The median benefit as reported in both of BrainStorm’s Phase 2 trials were modest. The optimum amount and frequency of dosing has yet to be established and may vary from person to person. Once this is better understood, the expected benefit will only increase. Still, the stem cell saga had left me mostly nonplussed.
That is, until I saw footage from ABC News. It shares the experience of two patients whose improvements since receiving BrainStorm’s NurOwn have been striking. Both have regained function. One can stand on his own and even walk a bit, after more than two years of being unable to do either. If their progress is maintained and replicated in others, we may be on the cusp of a glacier-led retreat of ALS.
Continuing the metaphor, there is a rare phenomenon called a galloping glacier. About 1 percent of the world’s glaciers exhibit significant bursts of relative speed. One in northern Pakistan “gallops” every 20 years, moving nearly 1,500 times its normal crawl. Scientists are left to speculate why, but most theories include a reduction in friction.
We can be a virtual frictional agent of change. First by rushing to participate in any open stem cell trials. Second, by being vocal. After a Phase 3 trial is complete, the FDA must approve the therapy, insurance must deem it inclusively coverable, and manufacturing and delivery must be scaled up. We must collectively scream that all tasks necessary to afford treatment access be attended to with life-or-death urgency.
A glacier is like a moving mountain. In this case, it just needs to move faster than normal. Jesus said that faith can move mountains. My pastor describes faith as belief plus trust. I believe that WE can. I trust that WE will.
Keep the faith!
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