ALS Awareness Month: 10 Things You Should Know About ALS

ALS Awareness Month: 10 Things You Should Know About ALS

By Steve Perrin, Ph.D., Chief Executive Officer and CSO, ALS Therapy Development Institute

May is a month full of many exciting and celebratory days: college graduations, Mother’s day and Memorial Day.  It is a month typified by excitement, renewal, anticipation and remembrance.  It is also the month in the United States where we recognize all those living with ALS, commonly known as Lou Gehrig’s disease.  If you don’t know someone already living with this currently incurable, progressive neurodegenerative disease, the odds are that you will during your lifetime, as a new person is diagnosed with ALS in the US every 90 minutes.  This month, upwards of 500 people will be diagnosed with ALS.  People living with ALS have been waiting for an effective treatment for way too long.  This month, as we raise awareness of ALS, we ask that the entire biotechnology and pharmaceutical industry join us in thinking about what we can do to help end ALS.

Steve Perrin

Steve Perrin, Ph.D.

As a community, biotechnology is leading the way, with hundreds of millions of dollars already invested in ALS research – including major late stage clinical trials pipelines rich with promising preclinical compounds.  While that is a sizable investment for an “orphan disease”, it isn’t enough because the job is far from complete for patients today.  However, we are grateful for the work being done at Biogen Idec, UCB Pharma, Sanofi, GlaxoSmithKline, Knopp Biosciences, Neuraltus, and all the others across the globe that give patients hope by taking on leadership roles as public supporters of ALS research.

There are similarly vast resources being expended in the academic setting on ALS research and multi-disciplinary teams caring for people living with ALS. These programs exist at places like the Methodist Neurologist Institute in Houston, UMass Medical and MGH is Massachusetts, California Pacific Medical Center in the Bay Area, and John’s Hopkins in Baltimore. There are hope inspiring new programs being spearheaded at the National Institutes of Health, such as NeuroNext and the National Center for Advancing Translational Sciences that have the potential to streamline processes and remove barriers to drug development for ALS.

And then there are the nonprofit organizations, such as ours, that fill the gap between the “ah-ha” moments in the lab to moving a potential treatment into the clinic. Since the ALS Therapy Development Institute (ALS TDI) was founded in 1999 we have raised and spent nearly $100 million on developing potential treatments, two of our which are in crucial stages of clinical development.   This is an exciting time for ALS research, but we temper that excitement in our lab every day with the simple fact that patients are waiting. We all must move faster.  We have to continue to innovate and take more risks. These are things that we value at TDI and I know they are shared throughout our industry.

Every organization working on behalf of ALS patients recognizes ALS awareness month in the US a little differently and I’ll provide just a couple of examples for you.  Our partner, the Muscular Dystrophy Association (MDA) is the world’s largest private-funder of ALS research and all month long they conduct an “ALS: Anyone’s Life Story®” series which highlights a different family touched by ALS.  Another organization, the ALS Association, hosts an Advocacy Conference in Washington, DC every year, helping to raise awareness in Congress about the importance to fund more research.  ALS TDI participates in both of these activities and many others around the country all month long and this is in no way meant as an exhaustive list of ALS Awareness month activities.

In addition to working together with other organizations on those and other activities, ALS TDI uses social media to help raise awareness of ALS, posting a new picture (example to left) or YouTube® video  from our lab on Facebook® every day during May of a scientist working in our Institute on potential treatments.  We at ALS TDI are also honored to be able to participate in several 4tALS Awareness days held in partnership with Major League Baseball, including one next week at Fenway Park in Boston.

Ten things you should know about ALS:

  1. Every 90 minutes someone in the US is diagnosed with ALS
  2. The incidence rate is nearly identical for ALS and MS (2 per 100,000)
  3. More than a dozen different familial genetic mutations have been linked to onset, however, the vast majority of ALS is considered sporadic with the exact cause(s) unknown
  4. ALS is also considered by most to be a heterogeneous disorder, with site of onset, progression rates and overall survival varying dramatically from patient to patient
  5. Because the process is one only of exclusion, it can take a year or more to get a confirmed diagnosis of ALS on average
  6. While the reason is unknown, military vets are twice as likely to develop ALS
  7. There are more than 150 potential treatments being looked at for ALS in the US today
  8. ALS is NOT just an old person’s disease, many people in their 20s and 30s are diagnosed
  9. The ALS clinical network is incredibly supportive for trial initiation, enrollment and retention
  10. People living with ALS are incredibly engaged and eager to help advance research and take risks

I ask that you take a moment to think about what you can do to help end ALS.  Each of us in the biotechnology industry can make a difference in the battle to end ALS.  I ask that you join us during ALS Awareness month in recognizing the estimated 450,000 people living with this disease worldwide today waiting for an effective treatment.  You can help them battle back by making a donation to an ALS organization or simply by taking a moment in your own organization this month or this year in drawing attention to this disease.

On behalf of all of us at ALS TDI, thank you for showing your support this month for our PALS and CALS everywhere.  The biotechnology community has been an incredible partner and asset in the battle to end this disease not just this month, but all year long.  We need your continued support and commitment and together we will end ALS.

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