The ALS Association

ALS Ice Bucket Challenge Progress

Research Conference on Rapid Therapy Development Explores Most Promising Developments

November 10, 2015

In late September, The ALS Association convened leaders in ALS research for a three-day conference to discuss and collaborate on the most promising therapeutic developments: antisense oligonucleotides, gene therapy, and stem cell therapy. Researchers from across the country turned out for the conference, which was sponsored by The Greater New York Chapter and held at the Banbury Center at Cold Spring Harbor Laboratory in Huntington, New York. The event was a prime opportunity for these researchers to offer updates on their progress, some of which are described below.

2015-banbury-group
ALS Researchers gathered for a three-day conference at the Banbury Center at Cold Spring Harbor Laboratory in Huntington, New York.

Antisense Therapy

Antisense oglionucleotide therapy is believed to offer the best hope for the two major genetically-based forms of ALS, those caused by mutations in the SOD1 gene and in the C9orf72 gene. The goal of this therapy is to destroy these genes’ messenger RNA before it can further the disease process and damage a person’s motor neurons.

At the conference, researchers from the University of Washington in Saint Louis discussed their work testing the efficacy of a form of antisense treatment that targets SOD1 and is delivered by injection into the space surrounding the spinal cord. This method has so far proven safe in people with ALS and researchers are moving forward with plans for a clinical trial. Researchers from the University of California at San Diego also reported that an antisense trial against the C9orf72 mutation is in development and is expected to begin in late 2016.

Gene Therapy 


In gene therapy, a gene is introduced into the nervous system that enters cells and is then expressed (that is, churns out RNA working copies) to create a therapeutic substance. One advantage of gene therapy is that the gene is expressed for long periods of time, likely for months but perhaps as long as five years.

Several researchers described their work in developing ALS gene therapy. At Voyager Therapeutics in Cambridge, Massachusetts, a gene that targets SOD1 has been created and is being tested in animal models. Researchers have plans to move this therapy to human trials in the future. Researchers at Ohio State University are also working on a therapy that may slow the progress of spinal muscular atrophy, a motor neuron disease similar to ALS. If supported by further tests, this could provide an important proof of principle for the gene therapy approach.

Stem Cell Therapy


Stem cells are being used in many laboratories today for research into the causes of and treatments for ALS. Researchers at the conference reported that an ongoing trial that sees stem cells implanted into the spinal cord to improve the health of deteriorating motor neurons has so far proved to be safe, with the cells remaining alive. However, this treatment has not led to a measurable change in disease progression.

Additionally, researchers from Cedars-Sinai Hospital in Los Angeles have engineered stem cells to release excess growth factors designed to help motor neurons overcome the toxic effects of the ALS disease process. An initial safety trial of 18 individuals is likely to commence early in 2016.

What’s Next?

“Our purpose in holding these small scientific meetings is to encourage key researchers to engage in a free flow of dialogue that can bring out important ideas, point out weaknesses in approach that need strengthening, and promote collaboration to push the field forward,” said Lucie Bruijn, Ph.D., M.B.A., Chief Scientist for The ALS Association. “Judged by those criteria, the meeting was a terrific success.” “The excitement among our researchers about the progress of therapy for ALS is what drives the field forward,” Dr. Bruijn commented. “We saw at this meeting, as we see every day, their commitment to finding the most effective treatments in the shortest possible time frame. We are all encouraged by the advances being made in the field, and know that we must continue to work even harder to move them forward even faster.”

For more information on the conference, read The Association’s press release and view the meeting agenda.

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