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Mar 28
2022

A statement from Jeffrey D. Rothstein MD, PhD, founder and director of the Robert Packard Center ALS Research at Johns Hopkins

ALS Headlines, Packard Center News

A statement from Jeffrey D. Rothstein MD, PhD, founder and director of the Robert Packard Center ALS Research at Johns Hopkins

I was quoted in a recent article about a possible new drug for the treatment of ALS. While I'm glad to see attention regarding any new promising ALS therapeutics, only a few of my comments were part of the article and unfortunately it did not capture the complete conversation. Let me be clear, I am excited about the possibility of another drug – this drug appeared safe within the first trial. ALS is a uniformly fatal disease so any drug that shows benefits, even if those benefits are limited, is fantastic for patients who essentially have no clinical options. Most diagnosed with ALS will die relatively soon after diagnosis and a drug that even “marginally” delays disease is better than nothing, and also adds to the cabinet of drugs clinicians can offer (e.g., riluzole, radicava).

Additionally, I am already giving the drug mentioned in the article to patients, outside of the trial — TUDCA via shipping service and sodium phenylbutyrate by prescription. I do this because I believe that the data is encouraging.

When I mentioned I was in favor of a second trial it is because the vast majority of phase 2 trials fail in repetition with a phase 3. I do, however, support the FDA possibly approving this drug, even after one trial, because the drug company is carrying out that very important phase 3 trial now. So, presumably, there will be two possible outcomes: 1) the phase 3 is positive- which means by approving the drug “now” more patients gain access to it, thus helping to slow their disease and give them more time with family, their job, or anything else valuable to their lives. Or 2) the phase 3 trial fails and the FDA halts approval. I and the ALS community have been down this road before, but as long as the drug is safe, at least patients could have the opportunity to possibly benefit from it (and no harm- as our core mission as physicians is to help and do no harm!)

Please understand that I wear two hats. As a clinician, patient advocate, and friend of hundreds of people living with ALS and having lost many 1000's over the years, I deeply support access to any promising and safe therapeutics. As a researcher, I must also look at the data and results. Past experiences with many failed trials may have created some bias, but that does not change the fact that I and my colleagues are all fighting for effective treatments. My opinions on the science do not negate the need for patients to have access to these new therapeutics.

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