FDA Backs Development of Blood Biomarker for Clinical Trials in Progressive MS

An urgent unmet need in progressive MS is the lack of effective treatments. This is an important focus of MS Research Australia. Back in 2014 MS Research Australia and a number of other MS organisations around the world came together to form The International Progressive MS Alliance (Alliance).  The Alliance focuses and funds the world’s best and brightest to accelerate the development of new therapies. However one major hurdle in developing therapies is there are no quick, reliable measures (or “biomarkers”) to tell whether a therapy is working or not in progressive MS.

The Alliance has been working for several years to investigate neurofilament light chain (NfL) as a potential biomarker for progressive MS. NfL is a protein fragment produced when nerve fibres are damaged. These fragments then enter the spinal fluid and blood. Studies have investigated how NfL could detect and predict disease activity, including response to treatments. NfL may reflect ongoing disease pathology and may be a good sign to show whether a treatment is working or not.

These research efforts recently had a huge boost when the Alliance received a Letter of Support from the U.S. Food and Drug Administration (FDA).  The FDA, in a rare show of support, encouraged the Alliance to continue the exploration and development of NfL as a potential, rapid indicator of the value of an experimental therapy in trials for people with progressive MS.

This is the first time the FDA has provided a Letter of Support for further research of any MS-specific biomarker for use in clinical trials. Further work is needed to validate NfL as a clinical trials outcome measure. This letter signals to industry and other researchers that drug regulators are aware of the evidence supporting this biomarker. The Alliance has also submitted support materials to the European Medicines Agency to request similar support.

The FDA’s Letter of Support resulted from an application that included a review of published research results on NfL. The review was compiled by a panel that includes experts from the pharmaceutical/biotech industry, academic research and people living with progressive MS. The team previously published recommendations outlining evidence and priority research for developing NfL as a biomarker for progressive MS.

“Receiving this Letter of Support is a critical step that signals to industry sponsors and academic researchers the potential value of including serum neurofilament light as an exploratory outcome measure in clinical trials focusing on progressive MS,” said Dr. Robert J. Fox, Vice-Chair of the Alliance’s Scientific Steering Committee and senior author of the paper.

“Having a simple blood test to quickly track the potential benefits of experimental therapies would be an enormous step forward for people with progressive MS, for whom there are too few therapies.”

The Alliance is also investing in other initiatives to develop biomarkers of therapies for progressive MS, and recently announced funding 19 new Research Challenge awards to improve the understanding of mechanisms that drive progression, an area where the current lack of knowledge is hindering treatment development.

Find out more about the International Progressive MS Allianceits goals, track record and international partners.


Read the FDA’s Letter of Support

Read more about FDA Letters of Support

Read “Serum neurofilament light as a biomarker in progressive multiple sclerosis” in Neurology

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FDA Backs Development of Blood Biomarker for Clinical Trials in Progressive MS