Benjamin Murdock, PhD, the Robert A. Epstein and Joan M. Chernoff-Epstein Emerging Scholar first worked with Target ALS in 2016 after the foundation provided a grant for him to study NK cells as they relate to ALS. Murdock recalls that this was one of the earliest supports he received for his work on the connection between the immune system and ALS, which has now become one of the most promising avenues of study at the ALS Center of Excellence.
Target ALS describes itself as “champions for the power of collaboration.” Its annual conference brings people together from different disciplines, notably from academia and industry, with the idea of generating new drugs and therapies, eventually leading to clinical trials. Of course, all with a focus on ALS.
Early this month, Dr. Murdock journeyed to Cambridge, MA, for the Target ALS Annual Meeting to present his latest work funded by the foundation, promising to do just that – move a drug, tofacitinib, which is already FDA-approved for other conditions, closer to a clinical trial for ALS patients.
A Magic Meeting:
If we rewind a few years, it was actually another Target ALS meeting that led to the identification of this new, possibly game-changing drug therapy for patients. Dr. Murdock had always been told by foundation leadership that it was the cocktail hours, coffee breaks and similar opportunities where the magic really happens because they allow potential partners to chat and brainstorm.
That is exactly what happened during one such breakout session after Dr. Murdock presented his findings on NK cells' association with ALS and identified them as a therapy target. A person from biotech approached him and said he had a few potential drugs in mind that might be worthy of study but needed to explore further and get back to him. When he did, his recommendation was tofacitinib, and the rest is history.
Since then, Dr. Murdock and the research team at the ALS Center of Excellence, directed by Eva Feldman, MD, PhD, confirmed that tofacitinib in fact was a very promising therapy to target NK cells in ALS patients and have moved steadily closer to investigating it in human clinical trials.
Current Research:
Dr. Murdock’s current collaboration with Target ALS gives him the ability to test the drug on special C9orf72 ALS mouse models, which bear a faulty variant of that gene. When the C9orf72 gene is not working properly, it can result in familial (or genetic) ALS. As Dr. Murdock told the audience at the annual meeting, he believes that this form of ALS is a particularly promising target for tofacitinib, a theory he will now begin to validate.