In 2020, ALS TDI scientists announced the discovery of a previously unexplored drug target for ALS subtypes that are caused by the C9orf72 mutation. They are now testing drugs against this new target to see if they can find one that is protective against C9orf72 ALS, and potentially against other forms of ALS as well.

ALS TDI has established a platform that uses human induced pluripotent stem (iPS) cells to identify potential ALS treatments. Through this platform, they have recently discovered a collection of drug-like molecules that appear to be protective against ALS-related cell death.

ALS TDI’s Precision Medicine Program was the first program of its kind in ALS – partnering with people with ALS around the world and using their data to learn more about the disease. By analyzing this program data, ALS TDI is able to better understand causes of ALS and is working to create new measures for clinical trials.

In 2013, ALS TDI collaborated with Neurimmune to help validate AP101, a potential ALS treatment that they were trying to accelerate to trials. ALS TDI researchers saw encouraging results after testing the drug in our SOD1 mouse model of ALS. Our results contributed to the collection of data that was essential for moving AP101 into a Phase 1 trial that began enrolling in 2019 and is still ongoing.

Tegoprubart (formerly known as AT-1501) is the first potential ALS treatment to be invented by a nonprofit biotech, the ALS Therapy Development Institute (ALS TDI), and advance to human clinical trial. AT-1501 (antiCD40L) is an antibody therapeutic with comprehensive and promising preclinical data. It blocks specific immune cell activation and protects nerves against the progression of ALS and Alzheimer’s disease. It is currently enrolling for Phase 2 trials.

In 2007, ALS TDI published a landmark paper with guidance on execution of preclinical ALS studies. Lessons from this paper are now applied broadly in many areas of biomedical research – resulting in improved research standards and more accurate study results.

Since 1999, ALS TDI has evolved from a small research effort started in a family basement in Newton, MA, to become what is now the most comprehensive ALS research lab in the world. Learn how community support and research innovation have helped the ALS TDI lab continue to grow and evolve over the years.