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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26 Responses to ALS Awareness Month: 10 Things You Should Know About ALS

  1. Lynne de Villiers says:

    ALS sufferers are needed to be supported at all times. How do we do this?

  2. Judith Hains Ryan says:

    My brother John died of ALS in 2010 and now my sister has been diagnosed.
    Thank you for all you do to help bring a cure.I am trying to make people aware.

    • Judith, I am sorry to hear that your family has now been touched twice by ALS. Some forms of the disease are considered to be “familial” and there are specific clinical trials aimed at certain ones of those. If you are interested, please contact me at rgoldstein@als.net.

  3. Phyllis Dezember says:

    I am amazed that everybody who works in ALS research don’t at least give all ALS patients a chance to add time on to their lives by Stem-Cell infusions. All these patients know that researchers are working on cures, they also know with the system we have in the us most of them will be dead while they wait. All they hear is HOPE, HOPE, HOPE, and there never is any. No wonder the ones who can afford it go out of the country to get them.

    • Patty Rolow says:

      I would like to know about the stem cell I fusions that you are talking about. This is the first I’ve heard of it. I am in the “diagnostic process,” which is inconclusive at the moment, but I’m not receiving any type of care whatsoever, other than told to come back in 3 months to re test to see if this has progressed. I am already suffering definate symptoms and I have to be proactive with a uoung son. Thank you!

  4. Ernie Brandt says:

    I have had two friends contract this condition and my objective is go get inside the nuts and bolts of what is going on so we can brainstorm a solution.

  5. Ernie Brandt says:

    question… what is the thinking on what is happening at the cellular level causing this condition. E Brandt

    • Ernie, thanks for the question and I am very sorry to hear that you have seen two friends develop this disease. At the cellular level, we see lots of things go wrong including oxidation, mito dysfucntion, ER stress and protein aggregation. ALS is both clinically heterogeneous as well as non-cell autonomous making it a very challenging one to figure out. But maybe we don’t need to “know” everything about what “causes” it to find a way to fight back…..

  6. Larry Johnson says:

    I have ALS I’m in a nursing home on life support. I would love to see a cure or something to. Stop the progression. I pray day and night
    For help!

  7. Flavia Tafner Martins dos Santos says:

    I am from Brazil and my husband was diagnosed in October, 2010 and so far his sickness is developing quite slow. I would like to thank you all for the effort and I pray daily that someone will find something to hold the development completely as we have 2 kids to finish educating and because I love him dearly. Here in Brazil there are many cases and we are all hopeful that soon this torture will be over.

    God bless you all!

  8. JY says:

    Pretty amazed with the work done by team of ALS research. Work must be continue since so many people in the whole world are diagnosed with ALS.
    Question; ALS is a hereditary disease??

  9. Ronnie Dunbar says:

    I am a caregiver for a man named Joe. He has ALS and is in the very late stages. He is non verbal and has no mobility but his eyes do move and his head does follow my movement. I am wanting to know if he is completely aware of what I say? What can I do to comfort him more? I want him to feel I’m his friend not just a caregiver. Thank you for your comments and help. I look forward to response. Help me help Joe.

  10. lindashriver says:

    I also have als and it took two years to diagnose,so I’m in my fourth year. I have to use. A cane to walk and my speech is,not very good but I keep plugging a long.every day is a challenge but l am not as bad as some. I just keep the faith and I enjoy reading about other people who have the same symptoms as I do and it helps me to cope with this awful disease. I pray they find a cure and l don’t mean I enjoy there pain my heart goes out to everyone who is dealing with this disease.

  11. Eddy L says:

    Been living with this disease for 23 years, diagnosed at 23… it sure isn’t easy! But hey, that’s life eh!?

    Let’s Roll…

    • Marysue says:

      Hey Eddy,
      Do you know what gene was involved in your case? Do you have the S0D1 misfold gene? I am curious because you have been living so long with ALS and I want to know if your case is different? What treatments or medicines did you use? Thanks so much for your reply.

  12. sara says:

    Hi,
    It is interesting that in this article they mention ALS and MS in incident rate are similar, because my mom has MS and her sister has ALS. I wonder some how this is related, and may be used in research. I really hope the cure for both to be discovered soon.It is so sad to see the person you love the most in the world suffer like this.

  13. James McKay says:

    Hi I’m James from Australia, my family is ALS 20 of the 50 familys in Australia ALS is genetic. We have the c9orf72 gene, but unfortunately in our family it is bifolded mutated gene, I am 46 diagnosed August last year, my Sister is 38 and diagnosed September last year, as my Neurologist said if you have the gene you get it, because being mutated it sits in the general DNA.

  14. Kay Risch says:

    My dad, his brother, my sister and my brother, all died of ALS. I lost my dad when I was 12, and I’ve tried to learn about it since then. I even became a nurse to learn more. Sad thing about it though, when my dad died in 1967, there was very little known about it. This was before ventilators to assist breathing, and dad was going to be put in an iron lung, the primitive ventilators. He passed away before that happened though. It seems only palliative care is what is needed more than anything else. The people with ALS need all the help they deserve to live as normal a life as possible.

  15. Cindy Harfst says:

    We lost our Mother to this devastating disease, September will be a year now. She suffered with it for 2 years…

  16. Andrew Parker says:

    My father died of ALS in 1998. I truly admired his drive to live every day as fully as he was capable, driving for as long as he could, going to work, enjoying theater from his special chair. I see it as a common thread among ALS sufferers – a commitment to living life as fully as possible. My admiration to all of you and my hope that research will indeed discover the treatments and cures we all pray for.

  17. Catherine vandervort says:

    I too have been diagnosed with this disease. However, it didn’t take a year or more to come to this conclusion. I question my diagnosis. I do have all the symptoms..the twitching, slurred speech, swallowing issues, weakness in my hands and other muscles. Drs seem to think als is what I have. Of course anger and sadness has been my battle thus far. I’m terrified.. I will fight as long as possible, I pray a cure is on the way…god bless all of us who push forward!

  18. Kenzie Smith says:

    Hello all! I am a high school student. I don’t know anyone with ALS. But I have read a few stories from people who have it and it touched my heart. My prayers go out to all diagnosed with ALS and their families! May God bless you!

  19. Nicole Campbell says:

    Hi all,
    I was diagnosed with ALS November 2013, but the symptoms have been around October 2012. I live in India and don’t have research happening here. So we seeked an appointment in the USA my diagnostics were reconfirmed in January of 2014. Today, four months down the line I have undergone a surgery for the G-Tube, my speech is now slurred and I soon will be in a wheel chair.

    I pray through all the research trials being conducted they find a cure for patients with ALS. God Bless us all.

  20. Emma Morgan says:

    My dad died from ALS when I was 8!

  21. Julie says:

    Hello, My mother has just been diagnosed with ALS. My family and I are devastated. I love my mother more then anything we are so close. I hope a cure can be found soon. Nobody should have to go through this.

  22. Brenda Merrick says:

    My mother died 8 yrs ago. I have a back issue, so I have to have a Dna work up. To find my odds. I was my mothers care giver.Lord help!

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