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What will the ALS Association do with that $100 million?

Ice-Bucket-Challenge2By Todd Wasserman From Mashable

The $100 million that the ALS Association raised with its viral Ice Bucket Challenge is certainly an impressive figure. But those who suffer from the disease and those who took the challenge may wonder if it’s actually a drop in the bucket.

Amyotrophic lateral sclerosis is one of the world’s most horrifying diseases. Sufferers maintain full cognitive functions while their bodies atrophy. Within a relatively short time, patients lose their ability to walk, use their hands, swallow food and speak. The survival rate, on average is 39 months, though some outliers — such as physicist Stephen Hawking, who has had the disease for more than 50 years — have defied the odds.

As the ALS Association’s website points out, amyotrophic is Greek for “no muscle nourishment,” which is a shorthand description of how the disease operates: Motor neurons connecting the brain to the spinal cord to the muscles throughout the body degenerate, leaving the sufferer fully paralyzed or close to it.

Thankfully, the disease affects relatively few of us — just two deaths for every 100,000 people. Still, there’s little historical tracking of ALS — so there’s no way to tell whether the disease is affecting more people now than in, say 1920.

Genetic Silencing and Drugs
Nor is much known about the cause of the disease. The biggest breakthrough in the past 20 years has been the introduction of Riluzole, a drug that modestly slows the progression of the disease and adds about two or three months to the lifespan of sufferers.

That said, there have been breakthroughs. Dr. Steve Perrin, CEO of the ALS Therapy Development Institute, pointed out that GSK is executing a phase 3 trial for Ozanezumab, a drug that may be effective in treating ALS. Perrin said there are a bunch of similar drugs in the pipeline.

While Perrin dismissed the notion of a cure for ALS, he foresees a cocktail approach in which various drugs are used.

“If Drug A doesn’t work for you, then you can go to Drug B,” Perrin said. “Ultimately, we’ll get to a combination therapy.”

Such a cocktail approach has been used to treat other diseases, notably AIDS — but also Multiple Sclerosis, which like ALS is a neurogenerative disease that affects the central nervous system. There are now nine FDA-approved drugs that treat MS. As a result, Perrin said patients are often relieved to hear they have MS instead of ALS.

One crucial difference: with MS, imaging technology can let researchers know if a drug is working. No such technology exists for ALS, which makes drug development a lot slower.

There have been breakthroughs though, that make effective drug treatments more likely. Over the last 30 years, researchers have discovered that the immune system plays a prominent role in ALS — meaning cancer drugs might be used to nourish dying motor neurons, extending the life of a patient.

Another promising area of research is genetics.

Some 27 genes have been identified in in playing a role in the development of ALS. Valerie Estess, founder of Project ALS believes there is a possibility that such genes could be “silenced” in carriers, which could conceivably prevent the occurrence of ALS. Estess declined to give a time frame, but said we’re within a “hair’s breadth” of starting clinical trials for such genetic manipulation.

It’s unclear, however, if the genetic silencing would achieve the goal of preventing ALS — and if it could do so without harming the patient.

The use of stem cells has been a huge step forward in such research, Estess said.

“I would really underscore the importance of using stem cells,” she said. “You can create ALS in a dish.”

Perrin wasn’t as hopeful about genetic research.

“I’m very excited about genetics, but there’s no magic linear line to treatment,” he said.

ALSA’s windfall
The $100 million raised by the Ice Bucket Challenge will doubtlessly help some of this research along, but the money isn’t necessarily a game changer. That’s because ALSA’s mission is both to research a cure for the disease and help those afflicted with it.

The two are related, since it’s necessary to get ALS sufferers to take part in clinical trials in order to let the research progress.

A good portion of ALSA’s money goes toward helping sufferers of the disease. A wheelchair for an ALS patient can cost $30,000, noted Carrie Munk, chief communications and marketing officer at the ALSA. While a patient’s family is wrangling with insurance companies to get that paid for, the ALSA provides them free.

The organization also offers financial help for sufferers, and provides support groups to help victims and their families, among other services.

Last year, 28% of ALSA’s budget went toward research. Since the organization’s budget has now quadrupled, that may change, but here was the breakdown last year:

Screen Shot 2014-08-29 at 3.48.37 PM
Even assuming that the ALSA put every dollar toward research, that’s not a huge amount in the world of disease prevention and research.

“It can take up to a billion dollars to develop one drug,” Munk said. “While this is an incredible amount of money and will do much good, people need to focus on the progress being made.”

Perrin was a bit more upbeat: “It’s going to have an amazing effect,” he said. “Smaller organizations focused on a single disease can really move the needle.”

IMAGE: Ice-bucket-challenge Former Tennessee Titans linebacker Tim Shaw is dunked with water as he takes the ALS ice bucket challenge in the second quarter of a preseason NFL football game between the Titans and the Minnesota Vikings Thursday, Aug. 28, 2014, in Nashville, Tenn. Shaw has announced that he has ALS.IMAGE: MARK ZALESKI/ASSOCIATED PRESS


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