ALS The Road Ahead
Toward Therapies that Will Save Lives
Over the last few years, Target ALS has succeeded in dismantling many of the long-standing barriers to progress, and creating what we call, the Target ALS Innovation Ecosystem. By bringing together the right people, funding, and resources, this new research ecosystem has successfully accelerated the development of an array of potential therapeutic approaches.
In 2018/19, Target ALS undertook a comprehensive assessment of the state of ALS research to determine how to leverage progress thus far to reach the next step – therapies that will save lives. Based on this analysis and conversations with over 100 scientists, industry executives, patients and caregivers, we identified three priority areas. We are expanding our Innovation Ecosystem to take on these three areas.
- Biomarker Discovery and Development Develop the first set of ALS biomarkers to enable earlier diagnosis and more effective tracking of disease progression.
- Target Discovery
- Continue to strengthen the therapeutic pipeline by supporting early stage drug target identification and development.
- Drug Development
- Accelerate the translation of promising ideas into clinical trials.
Priority Area:Clinical Biomarker Discovery and Development
Target ALS will drive the development of an optimal set of biomarkers and clinical outcome measures to confirm early diagnosis of ALS, track its progression, and transform the nature of the clinical trial process. We have developed a three-pronged strategy to accelerate biomarker development:
We now understand that we are not just looking for one drug. Given the heterogeneous nature of ALS, an array of therapies will be necessary. While we have made tremendous progress in the last few years, we need to continue to increase our understanding of the disease and identify new levels at which we can intervene to either halt or slow disease progression.
To further strengthen the therapeutic pipeline, Target ALS is expanding its signature Target Discovery Consortia Grant Program.
Drug development is an undertaking that can run into the billions. Corporations and institutions are therefore reluctant to develop new therapeutics without a strong risk-to-reward ratio. We are expanding our Innovation Ecosystems’ tools, resources, and funding to accelerate the translation of promising therapeutic strategies into clinical trials.