Brainstorm Announces Positive Results for NurOwn Phase II U.S. Clinical Trial

Today, leadership from Brainstorm Cell Therapeutics announced results from their recently completed U.S. phase II stem cell study of NurOwn® in patients with ALS through a press release and webinar. Below we provide some detail on this study.

Background:

The Brainstorm stem cell trial is based off of NurOwn, which is a cell therapy platform centered on mesenchymal stem cells derived from bone marrow samples given by the participants in the trial. They are induced to secrete neurotrophic factors (MSC-NTF), which are a type of nutrient for cells that was previously show to have protective effects in animal models of neurodegenerative disease. Continue reading Brainstorm Announces Positive Results for NurOwn Phase II U.S. Clinical Trial

Neuralstem Phase II Stem Cell Trial Appears Safe

Researchers at Neuralstem Inc. are investigating a potential ALS therapy that involves injecting stem cells into a person’s spinal cord. This week, the results of the phase II study that tested the safety of this type of stem cell transplantation were published in the journal Neurology. Below we give some background on this latest study. Continue reading Neuralstem Phase II Stem Cell Trial Appears Safe

Choice of Stem Cells Can Improve Therapeutic Potential

Stem cell therapy could represent an effective and comprehensive approach to treating ALS. Stefania Corti, M.D., Ph.D., Assistant Professor of Neurology at the University of Milan, and her colleagues set out to find ways to improve stem cell therapy. Their work, supported by The ALS Association, was published on June 6, 2016 in the journal Human Molecular Genetics.

Stem cells are cells that are capable of developing into different cell types, including neurons (brain cells) and glia (brain support cells). Not all stem cells are the same and choosing stem cell populations with specific desirable properties could in fact improve the therapeutic potential of stem cell therapy. Continue reading Choice of Stem Cells Can Improve Therapeutic Potential

Mitsubishi Tanabe Pharma Submits New Drug Application for Edaravone to Treat ALS in the US

Mitsubishi Tanabe Pharma Corporation (MTPC), which is locally based in Jersey City, N.J. with a head office in Osaka, Japan, announced yesterday that a New Drug Application has been submitted to the U.S. Food and Drug Administration (FDA) for edaravone (MCI-186) for the treatment of amyotrophic lateral sclerosis (ALS).

Those in the ALS community may have questions about what this means for them. Below we provide some background information on edaravone.

What is edaravone?

Edaravone is believed to act as a free radical scavenger, a compound that works by getting rid of toxic waste generated as a normal by-product of cell function. In ALS it is thought that these by-products are not as effectively removed and the compound may be neuroprotective by relieving the effects of this oxidative stress. The increase in oxidative stress is thought to damage motor neurons (cells that die in ALS). Continue reading Mitsubishi Tanabe Pharma Submits New Drug Application for Edaravone to Treat ALS in the US

Targeting Neuro-inflammation to Treat ALS

In 2011, The ALS Association, Wisconsin Chapter began supporting the work of Howard Weiner, M.D., Co-director of the Ann Romney Center for Neurologic Diseases, Brigham and Women’s Hospital around the role of inflammation in ALS. In March 2016, The ALS Association awarded a new Translational Research Advancing Therapy for ALS (TREAT ALSTM) Drug Development Contract  to Dr. Weiner and David Rodman, M.D. at miRagen Therapeutics to continue this work targeting inflammation in ALS.

The goal of their project is to accelerate a novel microRNA (miRNA) therapeutic approach to reduce neuro-inflammation, in order to bring it into clinic trials and meet a significant medical need in ALS. Below we provide some background on this exciting work. Continue reading Targeting Neuro-inflammation to Treat ALS

Targeting Stress Granules: Harnessing Biology to Develop ALS Therapies

Aquinnah Pharmaceuticals, in partnership with researchers at Boston University, are targeting stress granules to design new therapies for amyotrophic lateral sclerosis (ALS). The goal is to advance promising new drug leads aimed at providing disease-modifying treatments for patients that will slow the clinical progression of this devastating disease.

One of the cornerstones of The ALS Association’s global research program is to fund milestone-driven projects to push research efforts more rapidly toward effective treatments and cures. The Association successfully accomplishes this is through fostering partnerships between academic laboratories and industry, and then funding them through The Association’s TREAT ALS™ Drug Development Contract program grants.

Wolozin Head Shot 2012 (002)
Dr. Ben Wolozin
Larsen photo-sm
Dr. Glenn Larsen

One great example of this type of collaboration is a partnership between Dr. Ben Wolozin, Professor of Pharmacology and Neurology at Boston University School of Medicine and Dr. Glenn Larsen, CEO of Aquinnah Pharmaceuticals based out of Cambridge, Mass. Dr. Wolozin also serves as Aquinnah Pharmaceutical’s Chief Scientific Officer.

Continue reading Targeting Stress Granules: Harnessing Biology to Develop ALS Therapies

New Medicine Report Brings Hope

When I was diagnosed with ALS, I was hoping to see my sons graduate from high school. As it turns out, I’ve been blessed to have seen them graduate from high school and college.

I know I’m one of the lucky ones. ALS has progressed fairly slowly in me and I don’t take that for granted. From day one of learning I have this disease, I’ve done what I can to advance ALS research and the discovery of new treatments.

All of us living with ALS have to have hope: hope for a treatment and hope for a cure. That’s why I’m excited about a new report that came out today from the Pharmaceutical Research and Manufacturers of America (PhRMA) and The ALS Association, “Medicines in Development for Rare Diseases.”

This new report, which also shares my story, finds that America’s biopharmaceutical research companies are currently developing more than 560 medicines for patients with rare diseases, 38 of which are for people with neurological disorders, including ALS.

Among the new therapies in development for ALS is antisense technology against SOD1. Antisense technology is an important step toward helping patients and their families manage ALS and something The ALS Association has supported since 2003.

There are about 7,000 known rare diseases that affect fewer than 200,000 people in the U.S. every year. This report gives me hope that industry is focusing on these diseases, including ALS, more than ever before.

Lorri Carey