MDA – a cure-driven initiative to find treatments and eventually a cure for ALS – is the world leader in ALS research and services. Funding the full therapeutic development spectrum from basic research to clinical trial infrastructure to drug development and Phase III trials, MDA funding has contributed to many important research breakthroughs including two potential ALS therapeutics in development that may reach clinical trials by 
the end of the year.

With a rapidly progressive disease like ALS, people don’t have time to wait for a treatment to be found through traditional drug approval processes. Therefore, this February MDA led an effort which helped to persuade the FDA to hold a first-ever public hearing about the ALS drug development process. At the hearing, people with ALS, researchers and others strongly urged the regulatory agency to accelerate the clinical development and review process for ALS therapeutics. Because understanding what causes ALS is an essential step in discovering new therapies, MDA has partnered with the National Institutes for Health (NIH) to launch a large-scale “exome sequencing” project, which will result in a database of valuable information about the genetics of ALS. The exome sequencing data will be made freely available to researchers around the world in order to accelerate the pace of ALS research.

In 2013, MDA launched the U.S. Neuromuscular Disease Registry, layering it over the expansive clinical reach of its national clinic network. By gathering and analyzing patient data over time, MDA aims to improve survival and quality of life in ALS and other diseases, optimize clinical trial infrastructure, and leverage existing resources with those of pharmaceutical industry partners.

In 2012 MDA helped bring together ALS clinicians to develop a clinical trial of the diaphragm pacing system that was recently approved for humanitarian use by the FDA. For this kind of approval, the FDA requires evidence that the probable benefit of the device outweighs the risk of injury or illness. However, it does not require evidence that shows that the device is beneficial (in this case, improves respiration, quality of life or survival). The MDA sponsored trial aims to determine whether there is a benefit to using the system – as the system is surgically inserted, if there is no benefit patients should not be using the device.