Barriers to Progress
Over most of the 150-year period since ALS was first described, little happened because of our lack of knowledge of the nervous system and of the disease itself. In the last two decades, a veritable explosion of promising basic science took place including the discovery of new ALS genes and new candidate therapeutic targets.
Yet despite these discoveries, ALS remained an incurable disease. Why? A series of mutually-reinforcing barriers, were standing in the way, including:
- Gaps in Funding: Few funding opportunities supported the translation of basic science ideas into validated drug targets and treatments.
- Siloed Research: ALS researchers were operating in traditional silos, competing with each other rather than collaborating.
- Lack of Scientific Resources: Researchers lacked access to the tools and resources necessary to advance promising ideas.
- Limited Industry Investment: Given the limitations of translating ideas into the clinic, industry saw ALS as a high-risk area.
- Bias in Funding Decisions: ALS research funding relied on a peer review process with inherent conflicts of interest.
- Intellectual Property Restrictions: ALS scientists needed access to data and resources with no strings attached.
Enter Target ALS
With support from Bloomberg Philanthropies and David Rubenstein, Daniel L. Doctoroff founded Target ALS to break down these barriers and build an innovative framework under which scientists could be brought together and real progress could be achieved.
We created a global network of pharma/biotech, academic, and foundation partners, and sparked collaboration through joint funding opportunities. We identified and funded the best ideas through an objective review process.
We developed a range of cutting-edge scientific tools and resources for the research community to use freely, without intellectual property restrictions. What emerged was the Target ALS Innovation Ecosystem—a radically different approach to driving breakthroughs in ALS research.