Harvard Research Team Competing for $1M ALS Treatment Prize Discover New Biomarker Along the Way

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In a strange quirk of fate, Prize4Life, a non-profit organization dedicated to accelerating the discovery of treatments and a cure for Amyotrophic Lateral Sclerosis (ALS), who last year awarded $1M to a Harvard researcher for the identification of a novel ALS biomarker, is pleased to congratulate one of the teams competing for its other $1M prize for their identification of another promising new ALS biomarker.

Prize4Life

“As an ALS patient, I am encouraged by such progress. As the CEO of Prize4Life, I am proud that the resources we provide are making a real difference for ALS researchers and that we could play a small part in such a significant finding,” said Avi Kremer.

In a strange quirk of fate, Prize4Life, a non-profit organization dedicated to accelerating the discovery of treatments and a cure for Amyotrophic Lateral Sclerosis (ALS), who last year awarded $1M to a Harvard researcher for the identification of a novel ALS biomarker, is pleased to congratulate one of the teams competing for its other $1M prize for their identification of another promising new ALS biomarker.

The team of researchers led by Dr. Howard Weiner, director of the Brigham and Women’s Hospital Multiple Sclerosis Program and the senior author on the study, has been competing for Prize4Life’s $1M ALS Treatment Prize since 2008. Complementing the $1M ALS Biomarker Prize designed to accelerate clinical trials, the $1M ALS Treatment Prize aims to fill the drug development pipeline with promising new therapeutics.

To facilitate and support research efforts, Prize4Life offers teams competing for the Treatment Prize a supply of quality controlled, genetically modified SOD1 mice from the Prize4Life colony at The Jackson Laboratory, the world’s leading provider of mouse models for research. In addition to the mice, interested researchers receive guidance to assure that pre-clinical protocols are properly designed. Prize4Life believes that easier access to these resources removes some of the various obstacles researchers face in identifying a strong therapeutic candidate worthy of advancing to clinical testing in ALS patients.

“We are delighted to congratulate the BWH research team on their important discovery of a blood based biomarker for ALS,” said Avi Kremer, co-founder and Chief Executive Officer of Prize4Life. “As an ALS patient, I am encouraged by such progress. As the CEO of Prize4Life, I am proud that the resources we provide are making a real difference for ALS researchers and that we could play a small part in such a significant finding.”

First author Oleg Butovsky discovered that changes in the immune system were causing damage to the spinal cord in SOD1 mice. His team identified that the SOD1 mice had an increased production of monocytes, a type of white blood cell. These cells were flooding into the spinal cord, where they damaged and killed neurons. Monocytes are produced in the spleen, and once the researchers injected the mice with antibodies to slow monocyte production they observed fewer monocytes entering the spinal cord, less neuronal cell death, and extended survival of the mice.

Remarkably, this immune response and overproduction of monocytes could be detected two full months before visible symptoms of ALS appeared in the blood monocytes from ALS patients. The researchers hope that testing for an increase in this type of monocyte could help identify individuals shortly before they develop ALS, as the changes in monocyte production in the mouse model are nearly identical to changes observed in ALS patients. “Affecting the abnormalities in monocytes could also serve as a means to test for candidate drugs to treat ALS,” Butovsky said. Although researchers do not know what causes the overproduction of inflamed monocytes, the immune system has previously been implicated in ALS, an idea that this study supports.

These findings also identify a potential new target for developing treatments for ALS patients, and although it may not mean a cure is imminent, the discovery does open the door to a new avenue for therapies and the possibility that a simple blood test could measure the efficacy of future ALS treatments. Further research is needed to determine what type of immunotherapy might best inhibit the inflammation of the spleen and overproduction of monocytes, but Prize4Life remains hopeful that its incentive prizes, mouse colony, and informational resources will continue to help the BWH team, as well as other research groups, reach our shared goal of finding an effective ALS treatment.

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Sarah Groothuis
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