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Reason for Hope: The State of ALS Research

In the past decade, major changes in the pharmaceutical industry and the drug development landscape have taken place. The severity of ALS, the absence of effective therapy, and the importance of finding treatments for all neurodegenerative diseases have combined to make ALS an attractive target for new approaches to drug discovery and development.

Changes in the drug development landscape
Most major pharmaceutical companies have reached an impasse in their efforts to develop new drugs for central nervous system (CNS) disorders like Alzheimer’s, Parkinson’s and ALS. It costs an estimated $1-2 billion to develop a new drug; the development process takes a decade or more; and the failure rate for drug development is approximately 95 percent. As a consequence, many pharmaceutical companies have been forced to downsize their operations, especially in the areas of early drug discovery, and in the highest-risk areas, including CNS research. 

New models for discovery are emerging
But new models for drug discovery are emerging as a result. The reliance on outsourcing for the earlier stages of drug development means that small biotechnology companies and academic medical institutions have an increasingly important role in setting priorities for therapy development. And many of these organizations place a high priority on understanding and finding new treatments for ALS. These organizations also rely heavily on the non-profit health organizations such as The ALS Association to provide scientific expertise, an established network of ALS scientists, and a clinical and patient community through our chapter network that are primed for clinical trials. 

Catalyzing new discoveries
The ALS Association has been instrumental in keeping the flow of therapy development strong throughout this transitional period in the pharmaceutical industry. Our TREAT ALS™ program catalyzes important new discoveries that are now being used to design new treatments, as we facilitate partnerships among academic medicine, biotechs, and large pharmaceutical companies. Because of these efforts, we are poised for major progress in the near future in the development of effective therapies for ALS.

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