On Friday, November 8, 2019, the ALS Therapy Development Institute (ALS TDI) hosted the 15th Leadership Summit. The Leadership Summit is a full-day conference featuring keynote presentations that provide an in-depth look at the most advanced ALS research. This year, the Summit focused on the groundbreaking research being done at ALS TDI, as well as our exciting collaboration with Google.

The day started with a welcome address from Steve Perrin Ph.D., the President and CEO of ALS TDI. The morning session began with talks from Fernando Vieira M.D. and Theo Hatzipetros Ph.D. The afternoon session included a talk from guest speaker Michael Brenner Ph.D., a research scientist from Google who is working with data from our Precision Medicine Program (PMP) participants. The day wrapped up with the presentation of the Leadership Awards and a poster session with scientists from ALS TDI.

Fernando Vieira, M.D. – Confronting ALS: Our Comprehensive Research Strategy

Dr. Vieira described an overall framework of the ALS Therapy Development Institute’s research strategy to discover and develop treatments for ALS. Focusing on the past year’s advancements and the 30+ researchers who assemble daily at the lab in Cambridge, MA to execute the experiments and conduct the studies, Dr. Vieira described the following:

• ALS TDI’s drug screening platform designed to discover treatments worthy of advancement into human clinical development for ALS.

• ALS TDI’s commitment to the development, validation, and application of experimental model systems that adequately address ALS in all its complexity.

• ALS TDI’s efforts to discover and develop alternate clinical outcome measures that can accelerate the ALS clinical trial process.

• ALS TDI’s commitment to partnering with, and learning from, people with ALS through the Precision Medicine Program to better understand the disease, its drivers, and its possible treatments.

Theo Hatzipetros, Ph.D. – Advancements in Preclinical Drug Development of ALS

Dr. Hatzipetros gave an overview of the ALS TDI preclinical drug development program focusing on the development of an early disease readout in the SOD1G93A mouse model. This was made possible by the adoption of a technique known as compound muscle action potential (CMAP), which is related to human nerve conduction studies for ALS diagnosis.

CMAP allowed ALS TDI researchers to detect early changes in the SOD1G93A mouse model as early as age day 50, which is more than a month earlier than was possible to detect by typical symptom assessments. Additionally, CMAP at age day 50, significantly predicted the age of disease onset and survival.

To determine whether CMAP was sensitive enough to detect treatment effects, a study was designed using TDI-1762, a drug-like molecule that was recently identified as an effective molecule in an iPS cell-based phenotypic assay. TDI-1762 significantly decreased the decline in CMAP values as compared to untreated mice, just after two weeks of dosing, and ultimately extended the survival of the SOD1G93A mouse. Dr. Hatzipetros closed his talk by saying that despite its efficacious properties TDI-1762 is not currently considered a clinical candidate, due to solubility and toxicity concerns, however, he was hopeful that ongoing efforts will result in better TDI-1762 analogs.

Watch the Leadership Summit

You can view the live recordings from the morning session of the Leadership Summit here and of the afternoon session here. You can view the Leadership Award presentations here.

We would like to thank everyone in our community for supporting us on our mission to #EndALS. Our ALS research is only possible thanks to the support of community members like you.