ALS: A Pressing Human Need
Amyotrophic lateral sclerosis (ALS; aka Lou Gehrig’s disease) remains a terrible disease for patients and their families, and for doctors trying to help. Patients typically survive 3-5 years after diagnosis and during their illness they will become progressively paralyzed, losing speech and all independence. This reflects the unstoppable degeneration of motor neurons – the nerve cells in our spinal cord and brain that move the muscles we need for walking, lifting, swallowing and breathing. Finding an effective treatment for ALS is a pressing human need and would be a remarkable triumph for modern medicine. There are already ~25,000 patients battling with the disease in the US, and many more worldwide, and this number will increase significantly as the first treatments prolong lifespan.
Where We Stand
Over most of the 150-year period since ALS was first described, little happened because of our lack of knowledge of the nervous system or of the disease itself. More recently, and particularly in the last 5 years, there has been a literal explosion of discovery of new ALS genes, new disease mechanisms involving motor neurons and many other cell types in the brain and spinal cord, new human and animal models and new candidate therapeutic targets (steps in the ALS disease process at which it can be slowed or halted). Moreover, novel clinical trial strategies mean the community is ready to evaluate new treatments.
The major bottleneck delaying clinical trials in ALS is that despite the justified excitement around the new science, novel findings have not been transformed into validated therapeutic targets. Yet an identified target is essential for focused drug development programs and robust clinical trials. We therefore need to increase the bandwidth of both target discovery and validation. This can only be achieved by iterative interactions and collaborations between scientists in academia and their pharma/biotech colleagues. Over the last 3 years, Target ALS has achieved this to an unprecedented extent.
ALS: The Immediate Future
Through the efforts of researchers involved in Target ALS and other initiatives, ALS research is now in an exciting position. We are poised to evaluate multiple novel therapeutic targets in animal models of increasing relevance to the disease, and to push the most promising toward clinical development through the strong partnerships that have been established with the biotech/pharma industry. We urgently need to amplify our support for promising new research consortia and the core facilities that we have made available to ALS researchers worldwide. We also need to develop biomarkers that will allow us to identify the most appropriate treatment for a given patient, to rigorously monitor the effects of each new drug, and to ensure the safety of each new approach. To reach this goal will require more support from all sources: philanthropists, the industry itself, foundations and the NIH, and those in the general public who want to be part of this game-changing initiative.