ALS Network collaborates on $300K drug-repurposing study
Goal is to ID existing medications that could slow disease's progression
The ALS Network will support a collaborative research effort, at California’s Lawrence Livermore National Laboratory, that will use electronic health records (EHRs) to identify existing medications that could slow the progression of amyotrophic lateral sclerosis (ALS).
The $300,000 project is aimed at drug repurposing — finding medications already approved for other conditions that may have off-target effects beneficial in ALS. Because these therapies have well-established safety profiles, they could potentially be repurposed for use in ALS with less testing needed. That would allow these drugs to move more quickly through the development process for ALS than newly developed therapies.
The project will be led by Priyadip Ray, PhD, of Lawrence Livermore, as well as three other California-based researchers: Richard Reimer, MD, of Stanford University and Veterans Affairs; Jennifer Wilson, PhD, of the University of California, Los Angeles; and Kevin Grimes, MD, of Stanford University.
“Medications typically have well-defined therapeutic targets, but often also have off-target effects,” Ray said in a press release from the ALS Network. “Our long-term goal is to identify associations between medications and progression of neurodegenerative diseases through the analysis of electronic health records. As an initial step, we have focused on ALS as a proof of principle study.”
US nonprofit contributes $35K for drug-repurposing research
The ALS Network is providing about $35,000 in funding from its multifaceted research program for the study. Other funding will come from the ALS CURE Project, the Livermore Lab Foundation, the RDM Positive Impact Foundation, and Stanford University.
“The ALS Network’s research-funding model is an ambitious program driven by urgency and innovation,” said Sheri Strahl, president and CEO of the ALS Network. “Providing funding to these experts will lead to greater discovery and progress critically needed to find better treatments and cures.”
The collaborative project builds on a previous study that examined EHR data from more than 20,000 U.S. military veterans with ALS. Funded by the Livermore Lab Foundation with support from the ALS CURE Project, that research found several drugs and drug classes associated with longer survival in these patients.
However, veterans have characteristics that differ significantly from the general population, the researchers noted. For example, they are mostly male and with military experience — both known risk factors for ALS — and this may prevent findings from being generalized to other ALS patient groups.
Providing funding to these experts will lead to greater discovery and progress critically needed to find better treatments and cures.
To address that, researchers will use the Optum EHR dataset to assess data from a more diverse population of patients. Access to the database will be provided by Stanford, with machine learning tools from the Lawrence Livermore National Laboratory used to scan through the data and find potential drug candidates.
While the initial focus is on ALS, the RDM Positive Impact Foundation will also support similar efforts in Parkinson’s disease. Once the research techniques are established in ALS, the team will apply them to the same Optum EHR dataset to look for drugs that may slow Parkinson’s progression.
Ultimately, similarities across the two diseases may be used to accelerate the development of therapies for a broader range of neurodegenerative diseases, according to the researchers.
About the Author
