2020 was a year unlike any other, and so was this years’ ALS TDI Summit. Formerly known as the ALS TDI Leadership Summit, this annual event serves as a community update on the science behind the ALS Therapy Development Institute’s (ALS TDI) mission to find effective treatments for Amyotrophic Lateral Sclerosis (ALS).
Observing the need for social distancing surrounding the COVID-19 pandemic, this year’s Summit took place online, rather than in Boston, MA, as it has in past years. However, this year’s edition was no less informative or inspiring. Unique to the online format, there were several video presentations featuring interviews with scientists describing how ALS TDI kept the pace of research going despite the many challenges presented by the pandemic. Additionally, there were three feature presentations by members of ALS TDI’s science leadership team and one of our key collaborators for a new project in 2021.
Pharmacology in the Time of COVID-19
Theo Hatzipetros, Ph.D., ALS TDI’s Director of Preclinical Pharmacology described his team’s recent progress. He talked about how ALS TDI has expanded our pharmacology efforts to include multiple models (cells, fish, mice) and multiple endpoints (symptom progression, electrophysiology, RNA, protein, and histology). He highlighted drugs that we have worked on in recent years that have advanced into clinical development, including AP101, a drug being developed by AL-S Pharma LG, and CuATSM, a drug being developed by Collaborative Medicinal Development, LLC. He went further to describe new findings from ALS TDI around CuPTSM, an analog of CuATSM, that appears to compare favorably with CuATSM in the SOD1 mouse model.
Finally, Dr. Hatzipetros described a cell-based screening system established by the ALS TDI Translational Research Team in which human induced pluripotent stem cells are used to execute a drug screening campaign, identifying new leads for advanced testing in SOD1 mice. Dr. Hatzipetros highlighted one such compound, TDI1793, that is already showing promising signs in preclinical testing.
ALS Reversals: What we know so far and what comes next
Richard Bedlack, M.D., Ph.D., Director of the Duke Neurology Clinic, presented his fascinating experiences investigating and validating extremely rare instances of ALS reversals. Dr. Bedlack highlighted examples in other diseases, like HIV/AIDS, where the identification and study of spontaneously resistant individuals resulted in the identification of important therapeutic targets and the development of effective treatments. He described a rationale for utilizing the same approach in instances of ALS Reversals.
Dr. Bedlack announced a pending collaboration with ALS TDI to enroll people who had experienced ALS Reversals into the Precision Medicine Program Precision Medicine Program (PMP) in 2021 to explore their blood transcriptomes and proteomes.
ALS TDI Research Update and Outlook
ALS TDI’s Chief Scientific Officer Fernando Vieira, M.D. presented on a series of topics. First, he described a set of obstacles that make it challenging to identify effective treatments for ALS. He followed by highlighting ALS TDI’s specific approach to address each of those obstacles simultaneously. Second, Dr. Vieira described recent findings from ALS TDI identifying a family of enzymes, protein arginine methyltransferases (PRMTs), as therapeutic targets for C9orf72 ALS, as well as making a case for the broader involvement of these enzymes in other forms of ALS. Finally, Dr. Vieira presented innovations from the PMP and Google collaborators using digital outcome measures like voice recordings and accelerometers to predict ALS Functional Rating Scale scores.
2020 Leadership Award Winners
To conclude the ALS TDI Summit, ALS TDI's Senior Director of Development, Carol Hamilton, honored the individuals that were chosen by the community to be our 2020 Leadership Award winners! We were pleased to present these awards the individuals below.
Interconnected Award Winner:
Stephen Heywood Patients Today Award Winners:
To learn more about our 2020 Leadership Award winners, read our blog here.
Watch the Summit Recording
As in past years, the presentations at the 2020 Summit highlighted ALS TDI’s approach to finding effective treatments for ALS from every level – including basic research, industry quality drug discovery and pharmacology, clinical research, and translational research.
To watch a recording of this year’s Summit, click here.