A panel of leading amyotrophic lateral sclerosis (ALS) researchers recently published a review study, supported by the ALS Association, detailing the state of disease biomarker development and calling for the formation of an ALS Biomarker Consortium to speed up research into more effective disease treatments.
The review, titled “ALS biomarkers for therapy development: State of the field and future directions,“ was published in the journal Muscle and Nerve.
The ALS Association is in the process of developing a Guidance Document for Biomarker Development, using funds from the ALS Ice Bucket Challenge. The document, which will be submitted to the U.S. Food and Drug Administration (FDA), intends to help the industry navigate the development process and provide the FDA with the view of the ALS community regarding approaches to new ALS therapies.
Lucie Bruijn, chief scientist for the Association, said in a press release, “The ALS Association has invested significant funds towards the development of biomarkers for ALS and will continue its commitment by investing further funds to establish the ALS Biomarker Consortium, and seeks dedicated involvement from all stakeholders.”
The review and its calls for a biomarker consortium are the result of a series of meetings, including a biomarker workshop co-sponsored by the ALS Association and the ALS Therapy Development Institute. The earlier emergence of another biomarker-focused consortium, Clinical Research in ALS and related disorders for Therapeutic Development (CReATe), also contributed.
The review was penned by Michael Benatar, MD, PhD, of the University of Miami; Kevin Boylan MD, of the Mayo Clinic in Jacksonville, Florida; Andreas Jeromin, PhD, of Iron Horse Diagnostics in Phoenix, Arizona; Seward B. Rutkove, MD, of Beth Israel Deaconess Medical Center in Boston; James Berry, MD, and Nazem Atassi, MD, both of Massachusetts General Hospital in Boston; and Lucie Bruijn, PhD, MBA, of the ALS Association. The group was also instrumental in the formation of the FDA Guidance Document for ALS clinical trials, the Association said in its release.
The authors urge the development of an ALS Biomarker Consortium that includes all relevant stakeholders— including those with competing interests, like academic researchers and private biotech and pharmaceutical companies. “This is an opportune time for the establishment an ALS Biomarker Consortium that includes all relevant stakeholders. If it is to succeed, the structure and governance of this consortium must recognize and accommodate the complexity of the ALS research landscape, including the number and diversity of stakeholders,” they wrote.
Biomarkers, according to the ALS Association, are measurable quantities — often captured in ALS through brain scans, blood tests, or muscle strength measurements — that can be used to diagnose disease, track its progression, and, importantly, monitor response to therapy. In other words, they are objective disease measures of use in clinical trials.
ALS, a progressive neurodegenerative disease that affects nerve cells in the brain and the spinal cord, severely impacts a person’s ability to initiate and control muscle movement. The disease, prevalent in military veterans for unknown reasons, has no cure and only one FDA-approved drug that “modestly” extends survival, the Association reported.