On the same day in August that he and three other researchers published their breakthrough explanation of the cause of ALS (also known as Lou Gehrig’s Disease) in Science, Johns Hopkins University scientist Jonathan Ling did a Q&A session on Reddit. He said he wanted to set the record straight on whether the ALS Ice Bucket Challenge, which raised $115 million for disease research in six weeks back in 2014 (and then tens of millions more in repeat donations since), actually accomplished anything.
Ling recounted “reading a lot of stories about people complaining that the ice bucket challenge was a waste and that scientists weren’t using the money to do research, etc. I assure you that this is absolutely false.” The surge of research funding from the ALS Association allowed his Johns Hopkins team to conduct high-risk, high-reward experiments that were crucial to their discovery, he reported.
At any given time, about 30,000 people suffer from ALS—which kills most of its victims within a couple years of diagnosis. There is currently only one drug for treating the disease, and it quickly loses its effectiveness, extending a patient’s life only three to six months. It is hoped that lifesaving therapies based on the new JHU findings could enter clinical trials within two or three years. And thanks to the money raised in the Ice Bucket Challenge, those expensive trials are already paid for.
Another charity that recently hit pay dirt with a creative approach to disease research was the Cystic Fibrosis Foundation. Its leaders had become frustrated that only incremental improvements in treatment were in place by the late 1990s, despite discovery of the genetic cause of cystic fibrosis more than a decade earlier. Most cystic fibrosis sufferers continued to pass away by their 30s, even after decades of traditional research investment.
So in 1999 the foundation shifted gears. Instead of just funding academic investigators, it would put money directly into pharmaceutical companies, seeking to speed useful drugs and also to interest the industry in making its own larger investments in lifesaving treatment. The Cystic Fibrosis Foundation eventually poured $450 million of donations into investments in a variety of small and large drug companies.
CFF bets paid off in a big way. In 2012, a breakthrough drug developed with the foundation’s funding was approved by the FDA. It treats underlying causes of the disease rather than symptoms. Then in 2015 an even more broadly effective drug was approved; it offers significant relief to half of all sufferers. These compounds may double the life expectancy of some patients.
In addition to catalyzing these clinical triumphs, the foundation more than recouped its investments—by selling its rights to future royalties from the drugs for $3.3 billion. Those proceeds will allow the Cystic Fibrosis Foundation to maintain and expand existing patient-support services while directing additional money into a “supercharged” scientific search for a long-term CF cure for all patients.
This is an excerpt from Briefly Noted in the Fall 2015 issue of Philanthropy magazine.