Who Cares About ALS?

1,100 days. That’s the average amount of time a person survives following a diagnosis of ALS. That is because ALS is a progressive neurodegenerative disorder defined by the loss of both upper and motor neurons which in the clinic is characterized by the atrophy of nearly all a person’s muscles.  These 1,100 days aren’t pretty. Each person will progressively lose their ability to speak, move or eat on their own. Most people will die from respiratory failure as the disease strips away the muscles that control the diaphragm. There is nothing forgiving about this disease. It doesn’t care how rich or poor you are or what your background is. It can take you before your 21st birthday or soon after retiring in your 60s. Make no mistake, ALS is a killer.

May in the United States is ALS Awareness Month. There are local gatherings, fly-ins to DC and capitals all across the country. We take to the streets for fundraising walks, rides and other events on a mission to educate people about ALS. Each day this month, including today, more than a dozen people in the US alone will be told by their doctor that they have a confirmed diagnosis of ALS. By the end of the month 600 more people begin their 1,100 day count down. Today is someone’s Day 1.

As drug developers, what can we offer in 1,100 days? What can we do in just three years? We all know the statistics from Tufts University’s comprehensive annual review; it takes about 12 years and roughly $2 billion to develop a new treatment. In ALS terms, 12 years is 70,000 people in the US alone and hundreds of thousands more worldwide.  Anyone that still thinks that the market in ALS is a barrier for entry simply doesn’t understand the economics of drug development and healthcare.

The state of ALS research is strong. We have incredible partners in this fight. The field was buoyed with the news that Bloomberg and Doctoroff would be making $25 million in grants to ALS research.  That type of bold leadership and funding works, and I speak from a position of experience. Together with our Chairman and ALS patient, Augie Nieto, and our incredible partners at the Muscular Dystrophy Association, we have raised tens of millions of philanthropic dollars and invested it in a single purpose: discover and develop effective treatments for ALS patients today as quickly as possible.

In addition to these philanthropic and private dollars, the federal government continues to provide support for ALS research even as it wades through difficult economic times (Note: ALS strikes members of the armed forces in the US at significantly higher rates than the civilian population).

About three years ago our team made a discovery that led to the identification of a drug validated to hit its target and produce efficacious results in rigorous preclinical cell and animal-based testing. That drug, Gilenya, was approved for clinical trial in ALS by the FDA earlier this year. All of the preclinical and clinical work was paid for directly by the ALS community. We will complete the first clinical trial in the next few months. Although this trial is still ongoing, this example highlights what can be done when patients and drug developers are aligned on a single goal.

ALS patients and drug developers share a common motivator: speed. We are aligned on this point.  The goal is to go as fast as you can from the “aha!” moment to actually creating medicine that helps people. Similarly, both are willing to take enormous risks with their lives and fortunes.

The ALS Therapy Development Institute, the world’s first non-profit biotech, has raised around $100 million from the ALS community, and we are not alone. Biogen Idec, UCB, GlaxoSmithKline, Novartis, Cytokinetics, Sanofi, all have skin in the game, so to speak.

According to two different reviews on the ALS market completed earlier this year (NeuroPerspective and The Pink Sheet), there are nearly 40 other smaller biotechnology companies with products either being directly or co-developed as potential treatments for ALS. R&D activities on ALS are conducted on every continent except Antarctica. Simply put, ALS is no longer just a niche.

This past year there were both advancements and set-backs. We found new genes that cause ALS, including an unexpected hexanucleotide repeat, which could help explain the largest percentage of familial forms of the disease. In addition, there have been several launches of international biomarker efforts, including an ambitious program to collect thousands of patient MRIs. Our partners at Prize 4Life launched PRO-ACT, which is a public database of clinical trial data from the last 20 years of ALS research.

This past year saw its share of disappointments as well, the most high-profile of which was the negative read out of dexpramipexole by Biogen Idec. The innovative trial design worked, the drug did not. Hope lost? Absolutely not. Biogen isn’t turning away from ALS; they have actually renewed their investments to drive research forward and have several leads on candidate therapeutics as was noted in the two articles printed earlier this year.

This month, as the ALS community takes to the streets to raise awareness of this horrific disorder, I ask that you take a moment at your own organization to think about what you may be able to do to help.  Perhaps you have someone on staff living with ALS or has a loved one living with the disease; offer them extra support. Find a local ALS event to sponsor or volunteer. Make a contribution with your time, expertise, and yes, your wallet. Today is someone’s Day 1 with ALS.

Steve Perrin, Ph.D., is the CEO and CSO of the ALS Therapy Development Institute.

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4 Responses to Who Cares About ALS?

  1. LARRY SENN says:

    I am Larry Senn, retired from the US ARMY in 1992. Short comment. I lost my voice March 6,2011. I was diagnosed/confirmed with Bulbar ALS Onset on Aug,4,2011. I was put on Rilutek ALS medicine. I was told that I would never talk again and as thr diseased progressed I would eventually be in a wheelchair. About 2 months later I was provided a DynaVox+ with Eyemax System for eye communication. Later MIRACLES happen. By the Mercy and Grace of God I was healed of ALS and Dec 13,2011, Jesus touch my voice and healed it. DIVINE HEALING. I talk for the first time in 10 months. MIRACLES.MIRACLES. I had been told that there was nothing else the doctor could do for me BUT GOD DID. I am free of ALS and my voice is great. PRAISE GOD. GLORY TO GOD.GOD CAN HEAL ALS. JESUS IS MY HEALER. JESUS WAS MY PHYSICIAN.

  2. Charlie Guenard says:

    ALS is brutal and for many PALS it isolates you and depression can set in. I’ve met sooo many people who have helped in both small and large ways and I am so grateful – more than I can ever express. Sometimes though it feels like Eddington writing Einstein regarding Mercury not following Isaac Newton laws of gravity meanwhile a giant meteor is about to hit lower Manhattan. Good science is great but as a resident of Manhattan (1,100 day club) the rubber hitting the road is a treatment of any sort the rest is “just” interesting science.
    Ever respectfully yours, Charlie

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