A letter from the ALS Therapy Development Institute's (ALS TDI) Chief Scientific Officer, Fernando Vieira M.D.

Dear ALS Community,

Happy holidays and thank you for continuing to support our mission to end ALS.
The past year has been unique in so many ways. Certainly, we have all confronted uncertainties and hardships.
But 2020 has been unique in positive ways as well. I have been deeply inspired to see the team of scientists at ALS TDI persevere through the year – adapting to new safety protocols, working odd hours, adjusting to working from home, and pivoting on projects to advance our mission as quickly as possible.
In speaking with members of the science team throughout the year, I heard a consistent refrain: any challenges our scientists have faced were negligible compared to the challenges of the people with ALS who we aim to serve.
And so, thanks to your unwavering support, your team of scientists at ALS TDI accomplished our goals in 2020. I’d like to share some highlights with you here.
  • We began a drug screening initiative testing our library of 36,000 small molecules using neural cells derived from human induced pluripotent stem cells.
  • We have begun lead discovery assessments of early hits from the drug screening campaign using in vivo models with promising molecules emerging.
  • We have been analyzing blood samples from the Precision Medicine Program’s (PMP) In-Home Blood Collection Initiative to discover and validate blood-based biomarkers of ALS disease progression.
  • We enrolled more than 85 new participants into the PMP to allow for the comprehensive study of ALS symptom progression, genetics, risk factors, biomarkers, and outcomes from interventions.
  • We published a paper in Frontiers in Pharmacology where we shared our discovery of a previously unexplored drug target for C9ORF72-mediated ALS.
I am so proud of what we accomplished in 2020 despite the circumstances, but it would be disingenuous for me to say that we moved as quickly as I had hoped. 

I am excited to seize on the opportunities that we have created through our recent discoveries, I am inspired by the team of scientists that I get to be a part of, and I am buoyed by the support of the ALS community and your faith in us. I look forward to sharing our continued progress with you.
This is your lab, and we will continue our relentless research for all of you.
With sincere gratitude, 

Fernando Vieira, M.D., CSO
The ALS Therapy Development Institute 
If you would like to make a donation to support our ALS research, please click here.
ALS is not an incurable disease. It is an underfunded disease.