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It’s going to take a lot more ice buckets to fill the NIH funding gap

This scene should look familiar to you by now. (AP Photo/David J. Phillip)

In the weeks since Facebook has been filled with ice bucket challenge videos, there's been a robust public debate over what it means that people are suddenly donating to ALS research at record rates. But this latest viral sensation is also highlighting how much philanthropy is having to step in to make up for the declining public investment in research to cure some terrible diseases.

The ALS Association reports that the ice bucket challenge has netted $31.5 million in donations in just the past three weeks, compared to $1.9 million during the same period last year. That windfall is almost as much as the $40 million the National Institutes of Health expects to spend on ALS research this year, which is down from $59 million in the 2010 fiscal year.

There's no cure for ALS, a disease that kills most infected people within three to five years of the onset of symptoms. The disease, which eats away at its victims' nervous systems, fortunately has a relatively limited scope — as many as 30,000 Americans are thought to have it at any time.

It's hard to predict what it's going to cost to eventually find a cure. But the ALS Association's donation surge in recent weeks — and whatever future donations the ice bucket challenge likely will generate — won't be enough on its own to fund the research and development that's needed to eliminate this disease.

"It's not enough money," said Mary Woolley, chief executive of Research!America. "It takes the level of funding on a sustained basis that very few foundations can provide. And then the federal government really has to sustain that process."

So what could all that ice bucket money do?

"It makes it possible for some young scientist to get a start to try out their new hypothesis before they are ready to submit a proposal to NIH," Woolley said. "It may also bridge some dry spells they're feeling right now because the NIH is really strapped."

The decline in NIH funding isn't unique to ALS research. The agency budget, after reaching a peak of $31.2 billion in 2010, fell to $30.2 billion in 2014. And the NIH says its budget has effectively been cut by 22 percent in the past decade when accounting for medical inflation. Further, the sequester's automatic 5 percent cut to the NIH resulted in 8 percent fewer research grants in the 2013 fiscal year compared to the previous year.

The success rate for NIH grant applications has fallen from about 30 percent in 2000 to 15 percent last year. Jeffrey Rothstein, who heads the Packard Center for ALS Research at Johns Hopkins, said NIH's smaller budgets has made it noticeably more difficult for medical researchers to get government funding.

"To get money from the NIH, it takes a long time, and because it's so competitive, you actually have to have a lot of the work done to get funded," said Rothstein, who's been studying ALS for nearly 20 years. "How do you get to that point? That's where the philanthropy is critical."

Research is just one part of the equation. Then there's actually the cost of developing drugs.

Right now, there's only one FDA-approved drug to treat ALS, but it only slows down the disease a bit. Clinical trials are taking place for other treatments.

ALS is one of 7,000 types of so-called orphan diseases that affect just small parts of the population, fewer than 200,000 people each. But all those orphan diseases add up to 25 million affected Americans.

The United States recognized the special challenge of funding cures for orphan diseases in a 1983 law providing drugmakers with a number of advantages for pursuing treatments — faster drug approval times, tax benefits and stronger patent protections. That's led to the FDA approval of about 350 drugs for orphan diseases in the past 30 years, compared to just a handful in the decade leading up the 1983 law.

MIT Sloan School of Management professor Andrew Lo said drugmakers more than ever are seeing the financial benefits of orphan disease products, which could cost hundreds of millions of dollars to develop. But Lo, who's proposed a type of super fund to finance orphan drug development, said he's worried about the declining public investment in research for these rare diseases.

"Without the research, you can't develop the drugs," Lo said. "The private sector funding is not going to be of any use unless you have the basic research."