Case Study: Unprecedented Biomarker Initiative Offers Optimism for People with ALS

Clinical biomarkers have been critical to achieving major breakthroughs and lowering the barriers to drug approval in a range of deadly diseases.

Currently, there are no reliable biomarkers to diagnose ALS early, track disease progression or group ALS patients with similar forms of the disease. After launching an Innovation Ecosystem largely focused on therapeutic targets, we turned our attention to biomarkers. In 2017, Target ALS brought together an unprecedented collaboration—11 pharma/biotech companies, four foundations, and over 20 academic experts—to identify, validate and further develop promising biomarker candidates on a pre-competitive basis.

Biomarkers can take many forms. Our collaboration’s first step was to fund a study that focused on proteins in either cerebrospinal fluid (liquid that bathes the spinal cord and brain) or blood. Our aim is to understand how they change during the course of the disease. Over the next five years, we aim to discover and validate the first set of biomarkers and clinical outcome measures in the history of the disease.

Usually, research like this takes years to become available to other scientists through peer-reviewed journals. In a historic first, the data from this new initiative will be shared immediately with the ALS research community worldwide. The field will benefit from the work and paving the way for accelerated biomarker development.