The goal of Target ALS is to increase the number, robustness and intensity of ALS drug development programs in the pharma/biotech industry by supporting the best preclinical research and fostering interactions between academia and industry. Given our success in building this bridge over the past two years, our central strategy now is to validate novel therapeutic targets – molecular steps in the disease pathway which, when blocked by a drug, can slow or prevent disease progression – to be built upon by the industry.
Targets are the common currency between researchers focused on mechanistic studies and drug development specialists. Yet the recent upsurge in ALS biology has led to an embarrassment of riches: there are more candidate targets than can ever be tested in the clinic and too little is known about most of them to allow for rational selection.
We see the role of Target ALS in this process as three-fold:
- Fill the funnel: fund academic and industry consortia projects to exploit the full range of modern biology to identify the best and most novel potential targets. This process is called “target discovery”.
- Filter targets to select the best: set up community-wide core facilities that allow diverse targets to be compared head-to-head in an objective manner. This is “target validation”.
- Turn the best targets into drugs: create the conditions for pharma and biotech to move ALS drugs forward from initial concept to clinical reality. This should foster productive “drug discovery”.
Unique features of Target ALS
- Our focus on therapeutic targets, the most productive interface between academia and industry
- Our unique collaborative structure: only preformed consortia are funded, with the exception of the Springboard Fellowships for emerging future leaders in ALS research
- Our nationwide core facilities in contract research organizations to remove bottlenecks in translational ALS research
- Together with the ALS Association, we are the largest philanthropic funding source for ALS, with completely independent review of all funding decisions
- Direct involvement of industry scientists at all levels of strategy, review and performance