Project ALS, Medidata Join to Speed Personalized ALS Therapies

The Medidata Institute and Project ALS have announced a research partnership aimed at better understanding amyotrophic lateral sclerosis (ALS) and developing new and personalized treatment approaches.

With a focus on rare diseases, the institute works with scientists, regulatory agencies, patient advocates, life science companies, and research organizations to help solve challenges in clinical trials of potential treatments, and in therapy development and delivery. Project ALS identifies and funds promising research into this progressive disease.

“The partnership with Medidata will bring both talent and technology resources crucial to our efforts,” Meredith Estess, president of Project ALS, said in a press release.

Research work will utilize Medidata’s Rave Omics, which uses machine-learning to identify patient groups based on “omic” biomarkers integrated from across life science fields. The biomarker discovery solution streamlines omic data capture within the clinical study process.

Cutting across fields like  genomics, proteomics, and transcriptomic (gene expression) data, Rave Omics’ integration is said to support a better understanding of biological systems with the potential to advance the development of precision medicines.

Rave Omics is part of Medidata’s Acorn AI, created to speed production of analytics and artificial intelligence solutions that are being developed clinically. Built upon Medidata’s cloud-based platform — composed of nearly 20,000 clinical trials and more than 5.8 million patients — Acorn products are said to feature the largest structured, standardized clinical trial data collection linked to real world, translational, and other datasets.

For diseases such as ALS, identifying biomarkers that groups patients into subtypes to determine optimal treatments could  improve outcomes.

“ALS is a complex, heterogeneous disease, and treatment of its various forms may require specific therapies targeting distinct ALS subtypes,” said Neil Shneider, MD, PhD, Project ALS research advisory board member, and director of the Eleanor and Lou Gehrig ALS Center. “I’m eager to begin working with Medidata’s Acorn AI team to define these subtypes, and to develop a precision medicine approach to our treatment of ALS.”

A subsidiary of Dassault Systemes, a French company that develops 3D design and digital markup management software, Medidata focuses on solutions supporting the clinical trial process. Through its platform, designed to help trials run faster, smoother and more safely, Medidata reports having helped biopharma companies conduct more than 1,200 rare disease studies involving some 190,000 patients.

“We are proud to partner with Project ALS on such an important quest,” said Glen de Vries, Medidata co-founder and co-CEO. “The patients and the researchers inspire us … We look forward to applying Medidata’s next-generation tools to deepen our understanding of this devastating disease, and accelerate the pace of new discoveries.”

Project ALS also teamed with Columbia University recently to open an initiative aimed at discovering more effective treatments for ALS, and advancing to clinical trials the most promising candidates.

By 2025, it is projected that 1 in 25 U.S. residents will be diagnosed with a neurodegenerative disorder, at a cost of more than $120 billion annually. According to the ALS Association, about 5,000 Americans are diagnosed with ALS annually, and at any given time, up to 20,000 U.S. residents are estimated to have the disease.