If you haven’t taken the Ice Bucket Challenge or seen dozens of videos of others pouring a pail of frigid water over their heads, you’re likely reading this in print because you don’t own a computer or smartphone. For the other 85 percent of Americans, ice bucket fatigue is beginning to set in and one wonders whether the $94 million donated as of last Wednesday to the ALS Association will provide a shot at curing ALS, also known as Lou Gehrig’s disease, or at least accelerate the discovery of better treatments.
Barbara Newhouse, president and CEO of the ALS Association, said the nonprofit group is “absolutely committed to transparency” and will “invest these dollars wisely in areas that will have maximum impact on the fight against this devastating disease.” The group has the highest four-star rating from Charity Navigator , a nonprofit watchdog group. In 2013, the ALS Association used 79 percent of donated funds on research grants, education efforts, and patient services, according to the association’s website. The rest of the funds went toward fund-raising efforts and administrative costs.
ALS, short for amyotrophic lateral sclerosis, strikes 1 in 1,000 Americans and destroys motor nerve cells in the brain and spinal cord causing progressive paralysis and death, typically within two to five years. How the Ice Bucket Challenge got started remains a matter of debate, but 29-year-old ALS patient Peter Frates , a former captain of the Boston College baseball team, was instrumental in helping the fund-raising effort go viral through a social media campaign he initiated in mid-July.
Boston ALS researchers, who have received grants from the ALS Association, say the influx of donations couldn’t come at a better time. Recent discoveries of more than 35 gene mutations linked to ALS and new therapies in the pipeline to target those mutations have left scientists more optimistic than they were a decade ago about potential new treatments that could significantly slow or halt the progression of the disease, said Dr. Robert Brown, chair of neurology at UMass Medical School in Worcester.
“The most exciting, high-risk, high-gain projects are often funded by private agencies like the ALS Association rather than the federal government,” Brown added.
His research has focused on genetic discoveries, and he’s been experimenting with gene therapy in mice and monkey studies to silence the SOD1 gene mutation, which he helped discover two decades ago. “If all goes well, we hope to try the gene therapy in ALS patients in the fall of 2015,” Brown said.
Brown is also conducting a trial in coordination with Dr. James Berry, co-director of the ALS clinic at Massachusetts General Hospital, to recruit about 45 ALS patients to test an experimental Israeli drug produced from stem cells. “The hope is that these stem cells will secrete proteins that will create a much better environment for the neurons,” Berry said, “helping to protect those that are still functioning well and possibly repair those that are deteriorating.”
Retigabine, an antiseizure drug, and mexiletine, used to treat irregular heartbeats, are both being tested in large clinical trials to determine if they can slow the progression of ALS. Both work similar to riluzole (Rilutek), the only drug approved by the FDA to treat the disease itself, rather than to manage symptoms. It was found to extend patients’ lives by two to three months in manufacturer clinical trials used to gain FDA approval. “Riluzole has a modest effect, and we’re hoping these two other drugs offer more benefits,” Berry said.
More than likely, Berry and other researchers acknowledge, the most promising treatments for ALS have yet to be discovered and will result from basic research efforts — perhaps through surprising findings made in labs that weren’t pursuing ALS treatments.
For example, the ALS gene discoveries of the past five years wouldn’t have been possible without data generated by scientists undertaking the giant human genome project, pointed out Avital Rodal, a Brandeis University neuroscientist studying ALS mutations in fruit flies.
Deborah Kotz can be reached at firstname.lastname@example.org.