The Next Ice Bucket Challenge? Making NIH Whole | Commentary

Posted September 10, 2014 at 5:33pm

I’ve seen a hockey player do it on the top of a glacier and a senator do it in a freezing Alaska lake. I’ve done it. My kids have done it. These days, it’s hard to turn on the television or log on to the computer without seeing a friend, family member or celebrity dunk themselves with icy cold water in the name of ALS awareness.

Although the ice bucket challenge did not start out as an ALS fundraiser, all that changed when Pete Frates, a young man with ALS, did it on the hallowed grass of Fenway Park. The question is, why are so many people getting so cold and wet for a disease no one can pronounce, let alone spell? The answer, it turns out, is pretty simple: because people are dying and it’s long past time we found a cure.

Amyotrophic lateral sclerosis is a progressive neurodegenerative disease that causes muscle weakness, paralysis and, ultimately, respiratory failure. As the disease ravages their bodies, ALS patients lose the ability to walk, talk, swallow and breathe on their own.

I know. I watched my father fight ALS with every ounce of strength he could muster for three years, until he passed away last summer. And I watched my mother care for him with such grace and love that it breaks my heart just thinking about it.

But it is not just the memory of my father that breaks my heart; it is also the knowledge that ALS patients like him can do little more today than he did, namely live with the disease instead of beat it. Someone diagnosed today is offered basically the same treatments that we had 80 years ago. The truth is ALS has no cure and the only government-approved therapy extends life by three months.

Three months.

That is not acceptable to me, the 30,000 Americans living with ALS or the nearly 6,000 who will be diagnosed next year alone. Once diagnosed, the average survival time for an ALS patient following diagnosis is just two to five years. Yet that is the fate they face.

These people deserve more, which is why organizations such the ALS Therapy Development Institute are so important. Built by and for ALS patients in 1999, ALS TDI is the leading nonprofit biotechnology organization dedicated to developing effective treatments for ALS. It runs the largest dedicated ALS research lab in the world and it has emerged as the global leader in pre-clinical drug screening for ALS. But as committed as its scientists are, and as much progress as ALS TDI has made, it cannot do it alone.

The tragic fact is this nation could do so much more for patients suffering from so-called orphan diseases, such as ALS, with relatively low incident rates but catastrophic results for patients and families. For example, the National Institutes of Health currently spends roughly $80,000 per patient death on arthritis research. By contrast, it spends $8,000 per patient death on research for ALS — a far deadlier disease.

The ice bucket challenge, which has resulted in an extraordinary outpouring of funds from private citizens, can help but it cannot close the gap. That is where government can and should come in. Indeed, the NIH exists with the express purpose of conducting and supporting the kind of basic scientific research that is needed to find medical cures and improve people’s lives. But after adjusting for inflation, NIH funding today is 25 percent less than it was in 2003 and Congress has shown no willingness or ability in recent years to reverse this shortfall.

The impact of the NIH funding crisis has been dramatic. The world’s best scientists are left to chase a continually shrinking pool of dollars. This environment has led to many promising researchers to either leave the country in search of better opportunities or, worse, leave the medical research field entirely. The result is that the critical bench science that can lead to medical breakthroughs is either being done in other countries, or simply not at all.

One thing that surprised me about the ice bucket challenge is how many people had someone to dedicate their involvement to — someone they knew who had died of ALS. This disease does not affect nearly as many people as cancer or heart disease, but when it does, it is profound for family and friends.

We can do better for them. In fact, we have to do better. Congress should act immediately to increase NIH funding and put the country back on track to finding cures for orphan diseases like ALS.

Robert Sepucha is senior vice president of corporate affairs at Fresenius Medical Care and serves on the board of directors of ALS Therapy Development Institute.