Pete Frates, the ALS patient behind the fundraising juggernaut known as the “ice bucket challenge,” has been hospitalized — news that touched off a spate of appreciative tweets on Monday lauding his contributions to the efforts to find a cure for amyotrophic lateral sclerosis, also known as Lou Gehrig’s disease.
A former Boston College baseball player who was diagnosed with ALS at the age of 27, Frates didn’t dream up the ice bucket challenge, but he was its most effective marketer. When it took off in the summer of 2014, videos of people pouring freezing water on their heads spread like wildfire on social media. The efforts raised $115 million for the ALS Association, along with millions more to other ALS charities.
Where has all that money gone? Here’s a partial list:
Finding a new genetic clue
The ALS Association dedicated two thirds of the fundraising haul — $77 million — to research. A recent payoff: the discovery of a variant of a gene called NEK1 that’s linked to 3 percent of ALS cases and could offer a potential new target for drug development. The association is now funding research in mice to better understand how that defective gene plays out in the progression of ALS.
To try to make more such discoveries, the association is pouring money into tracing other genetic variants linked to the disease. (Such mutations are believed to be responsible in about 10 percent of ALS cases.) Researchers at Columbia University got $3.5 million to do genetic sequencing of 1,500 people with ALS. Another project got a $2.5 million grant to sequence the tissues of deceased ALS patients.
Investing in community support
The ALS Association has dedicated $23 million, or one-fifth of the ice bucket money, to support patients and their communities. In Tennessee, that means grants to give patients more sessions with speech pathologists and iPads to help them communicate when speech becomes difficult. In Rhode Island, it means modest grants to help patients pay for handicapped ramps and stair lifts. And in western Pennsylvania, it means free training for caregivers and grief counseling for people who’ve lost a loved one with ALS.
Fueling the hunt for biomarkers
There are not yet any validated biomarkers for ALS that could be monitored through simple tests and used to track progression of the disease. Part of the ice bucket money is supporting such research. Scientists in St. Louis got $1 million to try to figure out how to track a protein known as TDP-43, which is almost always found in ALS cases. And a Boston researcher got $1 million to upgrade a repository of blood and spinal fluids being tested as part of the search for biomarkers.
At Cedars-Sinai in Los Angeles, a researcher got a $1.9 million grant to try to identify inflammatory biomarkers by collecting blood and skin samples from people with and without ALS. That same researcher got another $400,000 to work with General Electric to try to develop a nanoparticle for magnetic resonance imaging that has potential as a biomarker.
Pushing for better communication technology
Investments in early-stage research won’t help today’s patients, so some of the fundraising haul went to tech research with potential for nearer-term payoff. Last December a $400,000 prize went to two groups: a team of Dutch researchers working on an interface to allow ALS patients to communicate by what’s known as selective attention, and a Massachusetts company working on a system to help patients who can’t move control their laptops, phones, and home robotics.
Shepherding promising compounds along
There are plenty of compounds that have impressed scientists with what they can do in the Petri dish or in mice. But they won’t get anywhere near human trials without more funding. Ice bucket money is helping provide that boost. A biotech called miRagen Therapeutics, for instance, has been awarded $400,000 to try to move forward a compound targeting neuro-inflammation.
And a University of Massachusetts researcher got $2.5 million for a project that aims to deliver DNA molecules to the brain and spinal cord using transport viruses. The goal is to silence genes that sometimes trigger the toxicity involved in ALS.
Inspiring similar efforts by other charities
The spectacular success of the ice bucket challenge has made it a case study for other charities. Experts have identified key factors that made it work: the spirit of competition. The perfect timing in the dog days of summer. The interactive nature, ideal for social media and alluring to celebrities.
But some of it was pure serendipity — as illustrated by the fact that ALS charities themselves haven’t been able to recapture the magic in trying to make the challenge an annual August event. Despite a corporate partnership with Major League Baseball, the ALS Association raised just $500,000 through nearly the full month of August 2015.
As for Frates, now 32, he responded playfully to inaccurate reports on Monday morning that he had died — by posting a video on Twitter of himself moving his eyes accompanied by the soundtrack of Pearl Jam’s “Alive.”
My Mom was diagnosed with ALS (Parkinson’s ) also known as PD) or Lou Gehrig’s disease in November of 2012. I was told that she had a year to live, no more than two at best. I believed the doctors for only a short time, then decided that dying of PARKINSON’S was not acceptable. My Brother, Williams and I, embarked on a journeinfoy to heal. We were introduced to an African herbalist whose herbs caused a turn around in my life. I’ve been on these African herbal drugs and i’m doing greatly. no more saliva and now i can use my arms and feet. all thanks to [email protected] the great African Herbalist….. If I can do it, others can too.
: [email protected]
My father was diagnosed with ALS in the summer of 2013; His initial symptoms were quite noticeable. He first experienced weakness in his right arm and his speech and swallowing abilities were profoundly affected. We all did our best to seek help for this disease no medications they prescribe worked ,we were all scared we might lost him due to his condition, as he had been his brother’s caregiver a few years earlier for the same disease before he past. doctor recommend nuatural treatment from total cure herbal foundation for his ALS we have no choice to give a try on natural organic treatment ,this herbal cure has effectively reverse my father condition ,losing his balance which led to stumbling and falling stop after the completing the herbal supplement which include his weakness in his right arm and his speech, home remedies from totalcureherbsfoundation com is the best although their service is a little bit expensive but it worth it, they save lives.
No money was spent to detect the presence of Borrelia and for effective treatment. 130 years of misdirected research
It is such a pleasure to read about a health charity that does good work.
Investigative reporters fill the news with negatives: The bloated salaries, the expensive hotels, the starving kids that didn’t get food, etc
Thanks for this TRUE story.
ALS is caused by a Borrelia infection. The money should go to better detection of this bacteria and implementing an appropriate treatment program with antibiotics covering all co-infections.
And this has been determined how? I have not heard that the cause of ALS had been discovered. Please tell us more.
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