ALS Biomarker Research — One More Reason to Hope

The ALS Association funds millions of dollars in research every year, in a variety of scientific focus areas critical to advancing the search for treatments and a cure for ALS. In the nearly 150 years since ALS was first described in 1874, the search for new treatments and a cure has been frustratingly slow. However, the last decade has seen promising acceleration in progress, and in the last few years, five genes related to ALS have been identified. The ALS Association is currently funding a total of 169 active research projects in 16 different focus areas, and for everyone involved, critical discoveries cannot come soon enough. The promise shown in ongoing biomarker research gives reason for hope.

Promise in Blood Plasma Analysis

A biomarker is any measurable substance in the body that changes in quantity or that appears or disappears with a change in the body’s state. These measurable indicators of health and disease, such as blood, chemical changes in the blood, cerebral spinal fluid, and urine, can be tracked over time.

There are currently no known biomarkers to diagnose ALS. However, that may soon change. Biomarker research led by Dr. Michael Bereman, associate professor of biological sciences at North Carolina State University, and supported by a $100,000 grant from The ALS Association recently found that blood plasma analysis could be key to speeding up the process of ALS diagnosis and monitoring disease progression. Researchers analyzed the metabolites in blood samples from both people with ALS and healthy individuals to look for patterns connected to ALS progression in individuals.

Living with ALS himself, Dr. Bereman has a very personal stake in biomarker research, which he hopes will one day lead to a cure. “Since disruptions in metabolism are hallmark features of ALS, we wanted to investigate metabolite markers as an avenue for biomarker discovery,” he says.

According to the study, the most significant metabolite markers were elevated levels of creatine and decreased levels of creatinine and methylhistidine, all associated with muscle activity.

“These results are strong evidence that a panel of plasma metabolites could be used both for diagnosis and as a way to monitor disease progression,” says Gilles Guillemin, professor of neurosciences at Macquarie University and co-author of the paper describing the research. “Our next steps will be to examine these markers over time within the same patient.”

The Effectiveness of NurOwn

In addition to diagnosing ALS more quickly, the search for biomarkers is vital in helping to measure the effectiveness of potential treatments. The ALS Association and I AM ALS have recently awarded a $500,000 grant to BrainStorm Cell Therapeutics, a biotechnology company, to support its ALS biomarker research study, which focuses on measuring the effectiveness of NurOwn, an investigational therapy that uses a mesenchymal stem cell platform as a means to treat patients with ALS.

“This grant to BrainStorm marks an important step forward in establishing how exactly NurOwn works in the body,” says Calaneet Balas, President and CEO of The ALS Association. “We hope NurOwn is ultimately proven effective in treating ALS, and we stand ready to support BrainStorm in its plan to apply for a biologics license for NurOwn.”

If successful, this study will help determine whether NurOwn works in the way it is intended, in addition to informing our larger understanding of ALS biomarkers. Danielle Carnival, CEO of I AM ALS, notes, “This biomarker research will help us more expeditiously understand the effectiveness of NurOwn, while possibly unlocking discoveries that provide clues for other promising treatments.”

Biomarker discovery is extremely important to move the ALS field forward, and the ALS Association is committed to this search. “The ALS Association supports a number of biomarker research studies, in addition to a TDP43 Biomarker Grand Challenge program that our Chapter is currently supporting at Washington University in St. Louis, led by Dr. Timothy Miller, all aimed at moving the ALS biomarker field rapidly forward,” says Maureen Barber Hill, President and CEO of the ALS Association St. Louis Regional Chapter.

Right now, we may not see the exact path this research will lead to in the search for a cure because, as with all research, where the path leads is unpredictable. But because of every donation contributed, understanding of this disease is increasing every day. ALS biomarker research is one more reason to hope that effective treatments for ALS are on the horizon. ALS does not stop, and neither do we.

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