Target ALS, AFTD Award $5M to Support Research Projects

Grant awards totaling $5 million will support six academic-industry projects into potential biomarkers and treatments for amyotrophic lateral sclerosis (ALS) and frontotemporal degeneration (FTD), Target ALS and the Association for Frontotemporal Degeneration (AFTD) announced.

The two groups joined to support this collaborative work as ALS and FTD, the most common dementia of people under age 60, share genetic causes and biological mechanisms.

Disease biomarkers are key tools for guiding therapy development and mapping disease progression, with their discovery informing treatments for ills that range from cardiovascular disorders to cancer.

Target ALS and the ALS Association recently joined to financially support two research projects focused on the development of new tests to assess TDP-43’s potential as an ALS biomarker.

“Funding collaborative efforts has led to the most promising research coming out of our Innovation Ecosystem, the model we created in 2013 to bring the best minds together and achieve impactful results,” Manish Raisinghani, PhD, Target ALS CEO, said in a press release. “Partnering with AFTD has been an incredible step forward in that commitment, now reaching a critical milestone.”

Innovation Ecosystem brings together scientists, funding, critical tools, and resources to speed the discovery and development of breakthrough ALS therapies.

Those with the newly funded research teams will have access to Innovation Ecosystem tools and resources including, human biosamples, genomic datasets, and stem cells.

FTD and ALS overlap clinically, imaging-wise, and pathologically. For example, it’s been discovered that mutation of the C9orf72 gene is the most common genetic cause of both disorders. In ALS, the mutations account for up to 40% of familial ALS cases.

The $5 million will be shared among these groups and projects:

• Eikonizo Therapeutics, VIB-KU Leuven Center for Brain & Disease Research, Mayo Clinic, UZ/KU Leuven. This project will attempt to validate enzyme histone deacetylase 6 (HDAC6) inhibitors as a disease-modifying therapy for ALS and FTD. Inhibiting the HDAC6 enzyme is thought to rescue the transport of several molecules within nerve cells, and to lessen disease-associated symptoms.
• Novation Pharmaceuticals, Universite de Montreal/Centre de recherche du CHUM. This project will attempt to identify small molecules that can influence gene expression — the process by which information in a gene creates, say, a protein — and rescue deficits in stress granules (cellular structures that are essential for cell survival) that may contribute to ALS/FTD.
• Expansion Therapeutics, Scripps Research. This  team will attempt to identify small molecules that interact with RNA as a therapeutic strategy for ALS/FTD caused by mutations in the C9orf72 gene.
• Merck, University of Pennsylvania, University of Pittsburgh. They hope to identify selective inhibitors of aberrant forms of the TDP-43 protein as a novel treatment approach for ALS and FTD.
• QurAlis, Harvard University, University of Massachusetts Medical School. Researchers will develop an animal model that can be used to screen new therapeutic compounds for ALS and FTD, evaluate new therapeutic candidates, and develop a biomarker assay to determine these compounds’ effectiveness.
• Biogen, University of Massachusetts Medical School, University of Michigan Medical School. In this project, researchers will attempt to identify compounds that can protect against C9orf72 mutation-induced toxicity in animal and cellular models of genetic forms of ALS and FTD.

“We’re proud to join Target ALS in announcing funding for these crucial funding efforts,” said Susan L-J Dickinson, the AFTD’s CEO. “With the challenges that the COVID-19 pandemic is adding for the families we serve, we want them to know that we are working as hard as ever to bring momentum for a day when treatments can halt FTD and ALS in their tracks.”

Visit this site for more information about the Target ALS and AFTD collaboration.

Target ALS funds collaborative research consortia that pursue novel approaches in ALS treatment discovery and development. The AFTD seeks to improve the quality of life of people affected by FTD, and drive research toward a cure.