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:

A Pre-Competitive Biomarker Initiative:

In the Spring of 2018 and 2020, we launched funding calls, representing the first comprehensive, collaborative efforts to validate promising ALS biomarker candidates. All results and tools created are, and will be, made available to investigators worldwide with no delay or IP restrictions.

A Longitudinal Biofluid Core:

In partnership with research institutions and clinical centers across the nation, We are leading the first freely accessible longitudinal biofluid repository. The core facility will enable scientists worldwide to test their theories using biofluid samples from living patients, complemented by insights provided through postmortem tissue and genomics. 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, we are funding large-scale biomarker discovery, development collaborative projects to identify novel biomarker and clinical outcome measures. These consortia are be comprised of both industry and academic researchers, a requirement to receive funding from Target ALS and a representation of our committment to unprecedented collaboration.

Priority Area:
Target Discovery

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. 

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. We are expanding our Innovation Ecosystems’ tools, resources, and funding to accelerate the translation of promising therapeutic strategies into clinical trials.

  • Expansion of Tools and Resources: We will ensure the following tools and 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
  • Target Validation: Drugs often fail in the clinical trial phase due to inadequate pre-clinical target validation. Target ALS aims to address this issue by providing both the models and the funding for scientists to ensure targets reach acceptable levels of validation before moving on to the next phase of the drug development process.