Lambert-Eaton myasthenic syndrome (LEMS) is an autoimmune disorder that affects the patient’s pelvic and thigh muscles, gradually weakening them.
It’s closely tied with small cell lung cancer because it affects this patient population more than any other. In many cases, LEMS manifests before a cancer diagnosis is made. For cases in which no cancer is present, the cause is unknown. LEMS is a progressive disorder, gradually weakening muscle groups in other areas of the body such as the shoulders, neck, and arms. It’s worth mentioning that LEMS is exceedingly rare, with only 400 known cases in the United States.
So, this is where I start to get excited.
Most very rare diseases don’t get the attention they deserve in terms of research. But the planets must have aligned somewhere in the multiverse because the FDA just cleared Catalyst Pharmaceuticals to conduct the second Phase 3 clinical trial for a drug called Firdapse.
Most pharma companies salivate over their next blockbuster drug coming out of the so-called pipeline, so I have great admiration for companies like Catalyst that offer hope to patients who previously had none.
Final details for the study will be available at https://clinicaltrials.gov in the near future. In closing, I’d like to offer a round of applause to the brave people who participate in clinical trials, and for the pharma pioneers who keep on searching for answers.
