i’m going to cut right to the chase here guys. today was really hard. in fact, one of the most difficult days i’ve ever had to endure. for the past 16 months, i’ve been working on a drug discovery platform related to a drug called dexpramipexole that was in phase III clinical trials. this is the final trial a drug goes through before becoming approved by the FDA for use in patients. it’s a test of how well it works (versus rate of decline), if it causes any toxicity and also looks at mortality.
the drug wasn’t toxic but it didn’t prove to be better than the placebo so it was a failed study. the press release is here: top line data via biogen
i got a call late last night to come into work early this morning to be present when the top line data came across the wire during pre-market activity. i tried my best to keep calm and get a good night’s rest. i slept pretty well and woke very early today and made the drive into the dark, cold morning. i could feel the butterflies building up as i entered the building and prepared a quick cup of tea to calm my upset stomach.
the energy in our conference room was high, we’re a small team but extremely proud of the solid science we put out on a day to day basis. we knew that regardless of the outcome of the study, it was not a reflection of our experiments or hard work over the past year and a half.
as soon as the results were delivered, i felt like someone had just punched me in the gut. it was difficult to breathe for a moment and i felt like everything was standing still. in that instance, i chose to breathe deeply and felt incredibly focused. my heart immediately went out to the patients who are still without a treatment and their families. this was devastating news!
the next few hours were spent in meetings but we are a strong and dedicated team. while ALS is still a disease with unmet needs, i still remain confident in the work that continues to be done in neurodegenerative disease. we continue to be humbled everyday by shortcomings, but also encouraged at the discoveries that we have made along the way. each setback is another opportunity to learn. we will never know everything, but great minds and thinkers will continue to press on and challenge the conventional thinking about disease models.
let’s keep pressing on. . .x thanks for reading and supporting me along the way-M