150 Years Later, Still No Effective Treatment for ALS. New Research Model Offers Hope

In our world of incredible scientific advancements, it seems inconceivable that this year marks 150 years since ALS was first described, yet there remains no effective treatment for the disease. However, an innovative approach—created by nonprofit medical foundation Target ALS—is disrupting the status quo, spurring innovation that shows promise for the first successful therapies to combat the disease.

The Target ALS model, or “Innovation Ecosystem,” removes roadblocks that traditionally slowed or inhibited drug development, despite how promising the research may have been. In the past, advancement stalled due to long-standing hurdles including limited funding or access to research tools, as well as lack of necessary partnerships across the industry. Target ALS removes these hurdles to fuel unprecedented collaboration, which eases the path from drug concept through to clinical trials. They fundraise for strategic grants that are then issued to new cross-industry collaborations between scientists in academia and the pharmaceutical/biotech industry—two groups that previously competed for funding instead of working together. The organization also opens up access to high-value research tools, such as human biosamples needed by academic and pharma/biotech partners to fuel drug discovery.

As a result, Target ALS’ Innovation Ecosystem has led to more than 150 new scientific collaborations in just six years, resulting in more than 12 new potential drugs—including three now in clinical trials.

Target ALS is building on this momentum by doubling the size of the Innovation Ecosystem over the next five years to reach even more scientists, foster more collaborations, and ultimately accelerate the development of the first effective treatments for the disease. By doing this, the organization is essentially building a multiplier effect, knowing that the more promising ideas that are funded will lead to the more promising drug options. Their goal is for this work to yield at least 20 additional drug options and 10 new clinical trials by 2024.

Collaboration is Key to this New Approach.

ALS is a neurodegenerative disease that affects motor neurons, paralyzing patients as they progressively and irreversibly lose their ability to move or speak. On an average, patients survive approximately three to five years after diagnosis. It is a devastating experience for friends and family to see their loved ones face this cruel disease with no options for effective treatments. The disease places an enormous emotional and financial burden on the caregivers. This narrative has remained unchanged for 150 years.

While some drug options have made it to clinical trials over the years, they failed to prove effective, leaving patients without options or hope as their health declined. Target ALS’ approach, however, expands the drug discovery pipeline, offering new hope for a future in which no one dies from ALS.

For more information about Target ALS or to make a donation visit www.targetals.org.