ALS Drug Discovery

The Harvard NeuroDiscovery Center has joined forces with a number of partners nationwide to focus on two key areas of ALS research: genetics and drug discovery.

Drug Discovery

The number of new drugs delivered to the market to treat severe and fatal neurological diseases is declining. This decline is likely due to a combination of factors including the pharmaceutical industry becoming increasingly risk adverse, regulatory hurdles becoming more onerous, and the relatively small size of the ALS market. ALS receives little attention from the biotechnology and pharmaceutical industries. To address this problem and to focus drug discovery efforts on ALS and other neurodegenerative diseases, the NeuroDiscovery Center established in 2001 the Laboratory for Drug Discovery in Neurodegeneration (LDDN). The LDDN integrates the best of industry and academia to drive the discovery of potential new drug treatments for neurodegenerative disease. Learn more about our Laboratory for Drug Discovery in Neurodegeneration.

The Harvard NeuroDiscovery Center has recently launched an ALS-specific drug discovery program at the LDDN called CADDI (Collaborative ALS Drug Discovery Initiative). This new program of early stage ALS drug discovery projects combines the biological and mechanistic expertise of investigators around the world with our dedicated drug discovery team. This major not-for-profit ALS drug discovery effort will help ensure 1) an ongoing pipeline of early stage ALS drug discovery projects through collaborations with ALS researchers, and 2) the further development of the most promising of these projects to the point that they will be of interest to industry.

We are currently requesting proposals for novel targets or approaches to ALS drug discovery. Click here for further details and here for the RFA.

Genetics of ALS susceptibility

Most cases of ALS are referred to as sporadic because they do not run in families. Nonetheless, crucial genetic elements do play a role. The presumption is that a constellation of normally occurring gene variants acts in concert with environmental triggers to cause an apparently sporadic disease. The gene variants that predispose to sporadic ALS are likely to provide insight into biochemical pathways and events that cause the disease. By understanding these biochemical and molecular pathways, we will be able to design drugs that target key parts of the disease-causing pathways.

Led by Harvard NeuroDiscovery Center member Dr. Robert Brown (Massachusetts General Hospital and Harvard Medical School), we are engaged in a program that draws on the expertise of many ALS investigators in the United States, Europe and elsewhere to develop a comprehensive genetic map of ALS. The genetic map will be available to any person or organization studying ALS and will serve as a catalyst for many new investigations into the causes and treatments of ALS for years to come.