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
January 3, 2013 at 4:10 pm
Wow that was intense. So long story short it got rejected? What is ALS disease? Sorry to hear that
but again there’ll always be time to improve the drug so hopefully in the future it’ll be passed.
January 3, 2013 at 6:03 pm
basically it did no better than the placebo so yes a complete failure. ALS is also known as Lou Gehrig’s disease, it’s a progressive neurodegenerative disease that affects nerve cells in the brain and the spinal cord. you start to lose function of your muscles, even the tongue and eventually die from your diaphragm not being able to contract so you suffocate to death
January 4, 2013 at 3:07 pm
Omg that sounds so scary! Is it similar to Alzheimer?
January 4, 2013 at 3:08 pm
no…patients with ALS are of sound mind and aware that they are losing functionality of their limbs and other parts of their body. that to me is even scarier!
January 3, 2013 at 4:11 pm
Oh, Melissa, how devastating. I’m so sorry the trial “failed,” but as you said, each experience is an opportunity to learn more and continue striving to find a cure. Hugs.
January 3, 2013 at 6:07 pm
yeah i’m pretty gutted right now. hopefully i will find some clarity this weekend after some soul searching…
January 4, 2013 at 3:14 pm
thanks katie, i’m just trying to focus right now on moving forward and not burying my head and wallowing. i reminded myself that i can put the project away on a shelf but these people are still sick and dying with this disease. clearly, there’s no choice on what end of the spectrum i would rather be on right now. i need to acknowledge my feelings about this and not dismiss that it is difficult to process emotionally, but continue making paths forward.
January 3, 2013 at 5:36 pm
I’m sorry to hear that things didn’t turn out well with this trial. This has to be an incredibly intense and emotional type of experience to go through. It sounds like you’re doing such important work – you will get there!
January 3, 2013 at 7:10 pm
wow, you are amazing. I could not do what you do, but we need YOU! thank you, keep working, it will payoff. Shortcomings and all!
January 4, 2013 at 3:12 pm
thank you lindsay, i know it’s not ‘personally’ my fault but i feel responsible in a way that i’ve let these people down. it really sucks right now :/
January 3, 2013 at 8:56 pm
Hang in there, Melissa.
January 4, 2013 at 3:12 pm
thanks :/
January 3, 2013 at 9:57 pm
Melissa, I am so sorry. I can not imagine after all of the hard work and dedication you put into this project to have it rejected. You are so strong and amazing and wonderful. Thank you for all of the work you put into this for people suffering from that horrible disease. Sending you hugs <3
January 4, 2013 at 3:11 pm
yeah it’s pretty crushing. i woke up today definitely not wanting to go into work but i knew my team would all be there. we can’t bury our heads in the sand but it’s one of the hardest things i’ve gone through professionally. not many people get to experience the scenario i just encountered and it sort of feels like i’m grieving a loss… hopefully the weekend unplugged will help!
January 4, 2013 at 3:16 am
girl you are amazing!! do not let this bring you down. just keep going!! you are doing more than you know
January 4, 2013 at 3:10 pm
:hugs:
January 4, 2013 at 5:55 am
Ah, science research. I know it means little now, but every study has a positive side. Every “negative” result means you are one step closer to the cure. You tried option A, and you learned that it doesn’t work. So, you still learned something.
That said, I feel you. Wallow for a bit, you’ve earned that right.
January 4, 2013 at 3:09 pm
i knew you would understand the painful reality of negative data. it’s just devastating because the phase II trial showed a 35% increase over placebo
so what happened?
January 4, 2013 at 2:10 pm
I’m so sorry
I know how that feels – I spent the first 1.5 years at my current company in an ‘innovation’ group where we were pretty much constantly piloting new approaches and ideas. Even though we knew that not everything we launched was going to work, it still sucked when something we poured a ton of blood, sweat and tears into did not get the results we expected and we ended up having to kill it. Like you said, your team is awesome and your work was solid – you guys rock and you are working for such a good cause. Hang in there!
January 4, 2013 at 3:09 pm
thanks for that
January 4, 2013 at 3:26 pm
I’m sorry to hear. I agree with Katie, so I hope that you and your team will get closer to getting the correct formula. Although I can understand why you’re thinking about shifting careers. Whatever your decision is, for as long as you’re at peace, then you’re doing the right thing. Good luck.
P.S. You look so cool in that working in the hood photo.
January 5, 2013 at 1:31 am
I can imagine how devestating it is to work so hard on something and dedicate so much time to it and then have it fail. Never be a pessimist though because like you said, you have to look at all failures as a lesson and just try again next time. Everyone who succeeds have more failures.
January 5, 2013 at 12:12 pm
So sorry to read this….you are an amazingly bright person and I admire you for changing lives. And you will! Katy said it best, “everyone who succeeds have more failures.”
Happy New Year!
January 8, 2013 at 4:08 pm
thank you ameena x
January 6, 2013 at 1:18 pm
I can really only imagine the disappointment and sadness, Melissa. I’m so, so sorry. But I remain confident that your lab, and that science more broadly, can and will find answers. Nothing is more awful than starting from scratch, but I have faith in the work you’re doing and encourage you to press forward. I know you’ve expressed some doubts about your career in the past. But your heartfelt response to this news shows me how deeply you care about your work. Sending you love.
January 8, 2013 at 4:07 pm
gena, thanks so much for stopping by. i know your heart is full of compassion. these are definitely times that i wish science and medicine in general were more clear cut. sometimes, it feels as though, the more we know the less we understand a disease. i keep reminding myself that if no one faced failure, there would never be any success.
January 7, 2013 at 3:58 pm
many hugs, M! so glad the world has you working hard for such an important cause. we need more people like you! one step at a time, keep on walking!
January 8, 2013 at 4:05 pm
thank you so much! this means more to me than you know! x
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