A Breakthrough Opportunity for ALS
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, Target ALS undertook a deep analysis, with input from more than one hundred researchers, industry executives and philanthropic leaders, around how to leverage progress thus far to reach the next step – therapies that will save lives. The final result is a multi-year strategy to produce the first viable treatments for this disease. All of our collaborative grant-making and programmatic activities will be focused on advancing these three priorities:
- Develop an optimal set of biomarkers and clinical outcome measures to confirm early diagnoses and track disease progression, and transform clinical trial performance.
- Discover the next 20+ therapeutic approaches to address the heterogenous nature of the disease and identify new ways to intervene.
- Facilitate the launch of 10+ clinical trials to ensure the first viable treatments become accessible to patients as soon as humanly possible.
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 want to provide the community with the means of identifying those patients best-suited for each trial, and tools to gauge the potential efficacy of a treatment more efficiently – and with smaller trial groups.
We have developed a three-pronged strategy to accelerate biomarker development:
- A Pre-Competitive Biomarker Initiative: In the Spring of 2018, Target ALS launched the first comprehensive, collaborative effort to validate promising biomarker candidates that were recently discovered. This validation work is critical to accelerating the process of confirming whether a biomarker candidate is indeed relevant to disease progression. The initiative is being done in partnership with four foundations, 11 pharma/biotech companies, and a cadre of academic researchers and all results and any tools created, will be made available to investigators worldwide with no delay.
- A Longitudinal Biofluid Core: In partnership with research institutions and clinical centers across the nation, Target ALS is developing the first freely accessible longitudinal biofluid repository. The core facility will enable scientists worldwide to test their theories using biofluid samples. Without this type of resource, scientists can spend months and even years trying to access or collect relevant biofluid samples to test their theories.
- Competitive Grant-Making for Cross-Sector Research Consortia: To further spur innovative thinking, Target ALS will fund large-scale biomarker discovery, development collaborative projects that will work to identify novel biomarker and clinical outcome measures. These consortia will be comprised of both industry and academic researchers.
Priority Area: Target Discovery
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 better understand the disease and identify new levels at which we can intervene. Our initial $30 million investment in target discovery generated more than 12 novel approaches. Given the fact that the ecosystem is now in place, we believe a similar investment could yield as many as 20+ therapeutic approaches.
Target ALS is launching two initiatives to strengthen the therapeutic pipeline:
- Target Discovery Consortia Grants: Target ALS will issue a series of grant-making calls to support the same kind of academic and industry-led multi-investigator research consortia that developed a dozen therapeutic approaches in five years.
- Novel Idea Grants: Target ALS will support proof-of-concept work on emerging ideas that are promising but not yet ready for a comprehensive consortia grant. Target ALS will issue these grants on a rolling basis.
Priority Area: Drug Development
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. Building on the huge success of the scientific tools and resources Target ALS has provided to the community, we will expand their range and depth, and provide new funding packages to accelerate the translation of promising therapeutic strategies toward the clinic.
Target ALS will expand two initiatives to fast track promising therapeutic approaches into the clinic:
Core Facilities Expansion: We will ensure the following resources are accessible to ALS researchers worldwide through our Core Facilities.
- Human biospecimens and genomic datasets
- Animal models and In vivo target validation platforms
- Human stem cell models and stem cell based high throughput screening assays
- Antibodies and associated validated assay systems
- Medicinal chemistry support, PK/PD and safety pharmacology evaluation
Drug Discovery Acceleration Packages: Provide comprehensive support to consortia poised to translate promising therapeutic approaches into viable drug candidates.