Patients Seek Therapies That Might Stop Progression and Ease Burden of ALS, Online Survey Finds

Patients Seek Therapies That Might Stop Progression and Ease Burden of ALS, Online Survey Finds

The majority of patients with amyotrophic lateral sclerosis (ALS) report wanting new treatments to stop their disease’s progression and so improve breathing function, muscle strength, and mobility, according to the results from a self-reported online survey of ALS patients and caregivers.

These findings may help define future guidelines for the development of therapies for ALS and fulfill the needs of the ALS community, the IMPACT-ALS survey showed.

The results of the survey were discussed at the 29th International Symposium on ALS/MND in Glasgow, Scotland. The presentation, “Updated results from a US focused online survey of patient and caregiver perspectives on disease burden in ALS,” was given by Amy Laverdiere, director of new product planning and business analysis at Cytokinetics.

A total of 1,534 people participated in the U.S. online survey — including 813 ALS patients, 74 people who assisted ALS patients, and 647 caregivers — who provided their views on disease burden, functional outcomes, treatment, and clinical trial participation. They were recruited at patient meetings and clinics, and online by email and social media notifications to members of local support groups and patient registries. The average ALSFRS-R score reported by 242 participants was 32.

The online survey was anonymous and conducted between October and November 2017. People with ALS were represented from every state in the U.S., with representation across states comparable to the portion of the U.S. population in each state.

ALS patients were asked 30 questions about their experiences over the past two weeks, while caregivers were asked 43 questions about the experiences of their patients as well as about their burden over the same period of time.

The survey results showed that nearly every ALS patient experienced at least one symptom during the prior two weeks. These included weakness in limbs (89%), fatigue (79%), muscle strength loss (76%), cramps and twitches (63%), balance issues (58%), speech problems (57%), muscle stiffness (53%), and shortness of breath (52%).

When asked about the most wanted outcomes in a new treatment, respondents selected stopping the progression of the disease (48%) — in particular, they wanted improvements in breathing/respiratory function (86%), muscle weakness (70%), and mobility (67%).

“These results may inform drug development in areas of greatest patient burden and highest unmet need, decision-making by the FDA and health insurers and other policy issues,” the researchers wrote. “We plan to submit these results in a Voice of the Patient Report for ALS to the FDA (U.S. Food and Drug Administration).”

About 71% of ALS patients who were offered participation in a clinical trial did take part, with many stating as their reason a willingness “to contribute to the greater good.” Those who did not participate cited ineligibility as their reason.

More than half of caregivers (65%) rated their health as somewhat or much worse since they began caring for a person with ALS. In addition, 94% reported medium, high, or maximum stress levels during the previous two weeks.

The survey did not require patient and caregiver travel, compared with the in-person meetings of the U.S. Food and Drug Administration (FDA) patient-focused drug development (PFDD) initiative.

Biogen, Ionis Pharmaceuticals, and Cytokinetics provided financial support for the development of the survey, which resulted from a collaboration of the ALS Association with regulatory and methodology experts, ALS clinical leaders, an ALS patient, a caregiver, and industry representatives.

A similar survey is currently under development in Europe to study the experiences of people with ALS and their caregivers, and to inform global ALS drug development.


  1. Richard Stefanczyk says:

    Dear sir or madam,

    I lost my wife on 8/31/18 from ALS BULPAR. My wife was a very strong and healthy person until this hit her. My wife followed doctors advice and fought this disease with all her might. I believe since there is no cure or even any medication to really slow down this disease your information in your letters are giving people false hope. It’s a terrible disease as you know. I can’t say anymore.

  2. Allan Frederick Moore says:

    ALS is a terrible chronic disease. I would like a cure found but is important to help just to survive. I need financial help me with ADLs so I won’t bankrupt my disabled wife. Please fix medicare to help me quick. If you don’t know anyone with ALS, come visit me and hear my story.

  3. Suzanne Harford says:

    My sister has had ALS limb onset. She has been fighting for 2 1/2 years now. Your survey sounds good but what these ALS patients need progression stopage, ALS reversal or develop a cure. Much more decent places for ALS patients to be cared for besides their own house.

  4. John Preuss says:

    I have ALS and I have lost my voice, have trouble swallowing, and muscle mass. I am willing to participate in clinical trials that slow the progression or cure ALS.

  5. Blane Ebersold says:

    I don’t see that this survey is contributing anything that isn’t obvious to anyone who has or is in contact with anyone who has ALS. It doesn’t take a study to know that patients want to stop the progression of the disease and the regain functionality it progressively takes away. In fact, I’m shocked that dysphagia (the loss of the ability to swallow) and is a major contributor to fatality from ALS, isn’t even on their list.

    • Dave Reckonin says:

      “I don’t see that this survey is contributing anything that isn’t obvious to anyone who has or is in contact with anyone who has ALS. It doesn’t take a study to know that patients want to stop the progression of the disease and the regain functionality it progressively takes away.”

      You are 100% correct.

  6. Kenneth Erickson says:

    I have ALS (C9ORF72 gene). Instead of spending money on a survey put it towards finding some hope in slowing or even stopping progression.

  7. Danielle Riddell says:

    I was just diagnosed with bulbar ALE on 11/14/18 I am 49 years old. I have already lost my ability to speak and I just had a peg tube placement done since I can’t eat without choking. I am currently taking rilutek I know this is not a cure but would be interested in any clinical trials.

  8. NeuroSense Therapeutics were also in Glasgow at the MND symposium.
    One conclusion – things are not moving fast enough.
    But – there is a progress, and if we accelerate the development of new potential drugs, we may have an effective treatment in the very near future (2-3 years).
    There is definitely HOPE for the patients.
    A year ago we have held a conference in Israel. Now we have more supportive data that the combination drug (PrimeC) can be beneficial for ALS patients.
    Here is a link to the conference:

  9. Dave Reckonin says:

    “…and if we accelerate the development of new potential drugs…”

    Once again we have lip service paid to ‘acceleration ‘ in developing drugs.

    We all can see that this is empty of meaning as Phase 1,2 and 3 are ‘progressing’ at the same snail-lie rate as was always the case.

  10. Dave Reckonin says:

    This is a ridiculous survey and a waste of time and money and energy for all concerned.
    It’s conclusions are a bit like saying that cancer patients would like to see a simple universal cure for all types of tumour or people who have a cold would like to see a treatment which instantly stops all cold symptoms.

    Coming soon: A survey which asks people if they would like a 50% pay-rise this year.

  11. Dave Reckonin says:

    This has been a poor year for new ALS drugs. Tirasemtiv failed this time last year and Pimozide is still being experimented with.
    Radicava is not all it is marketed to be and no-one is publishing what outcomes are for people who have been taking it for a year.

    What are they afraid of telling us? That is is just expensive gloop ?

    I think so.
    Willing to be proved wrong.

  12. David Buschhorn says:

    “The presentation was given by Amy Laverdiere, director of new product planning and business analysis at Cytokinetics.”

    That explains why no one said, “We want a cure, not a treatment.” Why would someone in that position even consider wasting time on a cure when they could inject us with something every day for the rest of our lives?

    “Don’t put THAT question on the questionnaire or we might all share a Nobel prize… that’s not a lot of money once divided up. No… we need a MONEY MAKER. And look! The data says people want a TREATMENT, not a cure!”

    Don’t get me wrong, but to deliberately not research the cause or a cure, is purposefully going after income over cure.

    Outside of new antibiotics and some pretty awesome chemo drugs, it seems to have been a LONG time since someone cured something. 🙁

  13. Christopher says:

    So…let me get this straight. This lady (Amy Laverdiere) from Cytokinetics, gave a presentation about this online survey to a room of folks. Did anyone in this room have a pulse? And this was the 29th International Symposium on ALS/MND. What did the first 28 International Symposiums discuss? I have 28 ideas, but probably can’t list them here. Mr. Alon Ben-Noon…”One conclusion – things are not moving fast enough” and “we may have an effective treatment in the very near future (2-3 years)”
    Thank you for your brilliant insight and hopeful thoughts. So, this is what is considered ALS News? Having ALS has really opened my eyes to the world. How nasty, greedy, slimy, ignorant, cowardly, deceptive, etc… human beings can be. It makes me sad.

  14. Dave Reckonin says:

    The real money is in chemo drugs and radiation for cancer patients, plus monitoring and managing diabetes. Tens of Millions of people have these disorders throughout the world.
    pAls are relatively few in number. There is no money in finding either a treatment or a cure for ALS because so few, ie so relatively few, people develop ALS.
    We have had around 50 years of the technology age. Technological techniques are, we are told, changing the face and pace of treatment development….oh really? Result?
    We have Riluzole and Radicava, carpet dust and gloop respectively.

    We are being played.

  15. Barbara McLean says:

    I too am flustered about how long it takes. I have started water therapy. It makes me Happy. I can do anything in the water and I don’t know how to swim. I do range of motion exercises at home to help with the stiffness. I have the C90RF72 gene. The worse part of this for me is that I have familial ALS so I worry about passing on to my sons. I live alone. I have fallen 20 times resulting a sprained wrist, sprained ankle twice, 10 stitches in my forehead. Every time I fall a small voice – hope- says get up your journey is not over yet. I have right drop foot, partial loss of right hand, slurred speech, and 69% respiratory. I have had ALS since at least August of 2017. I have not been in any clinical trials yet. But I do help research with a company in Boston MA. I live in NC. I know new small molecule targets root cause of ALS for C9ORF72. I know mostly likely it will take too long for a medicine to be found and approved for me. But hopefully, my sons will be spared. I have not given up hope yet. I face fear everyday of my life now. I try to count the things I can still do not what I have lost. Like the songs Fear you don’t own, Fearless,
    Fear is a Lier, Fight song I try to conquer it. I never allow myself to dwell on negative emotions more than 10 minutes a day. Life is now precious and I don’t want to waste it being negative. I find the small joys of everyday. I draw to release my negative emotions (I could never draw before), sing songs that help, listen to sermons on you tube, and dance for first time holding on to desk. I nap when I am tired.
    I live on a very limited income and insurance has now changed.

  16. John Gilmer says:

    I have to agree with several prior postings here – this survey is absolutely ridiculous and I would say a complete waste of time and money! PEOPLE WANT A CURE and short of that, something to slow the progression to buy some time FOR A CURE. I find the thought of this being a topic at a conference completely asinine. I can’t imagine a group of everyday people being surprised to find these results, much less people in this field! If you were hanging by a thread off a cliff, would you would greatly appreciate someone throwing you a rope and pulling you up? Of course! Possibly the largest “DUH” I’ve ever heard of.

    I have ALS and have participated in several studies – I mostly cannot do so any longer because I’ve had it too long. THROW ME A ROPE!

  17. Dino says:

    The system is a joke…the foundations skim…the doctors are there for imprimatur.

    Goldman Sachs Says Curing Diseases May Not Be Economically Viable

    A leaked report has stated what many in the health industry have whispered privately; there is a lot less money in curing people than in long-term management of disease.
    The issue is brought into focus by the possibility of a gene therapy that could soon cure a large variety of genetic conditions by changing individual units of DNA. However, the huge potential boon for society could be blocked without changes to the way health is funded. Countries with public health systems may be able to reap the benefits of the new technologies, leaving the US trailing in their wake.

    Goldman Sachs analyst Salveen Richter reportedly set out the issue in a note to clients; “The potential to deliver ‘one-shot cures’ is one of the most attractive aspects of gene therapy, genetically-engineered cell therapy, and gene editing. However, such treatments offer a very different outlook with regard to recurring revenue versus chronic therapies.”

    Someone who is rich enough may be willing to pay hundreds of thousands of dollars to be cured of a debilitating disease. Most people, however, coudn’t afford this, especially if their condition affected their earning capacity. Meanwhile, the same person might be able to scrape together tens of thousands of dollars a year to keep the worst aspects of the disease at bay, in the long run paying far more than they would have for a cure. It’s a medical equivalent of Terry Pratchett’s “boots theory”.

    Richter points to the sofosbuvir treatment of hepatitis C marketed by Gilead Sciences. In 2015 this brought the company $12.5 billion, mostly in the United States, as people suffering liver damage from the virus rushed to take up the new drug. However, the therapy cures most patients so thoroughly they’re finished treatment within 12 weeks. Better still from a social perspective, but worse for profits, once treated people no longer transmit the virus, preventing new infections. This year Richter expects American sales to fall to less than $4 billion.

    “While this proposition carries tremendous value for patients and society, it could represent a challenge for genome medicine developers looking for sustained cash flow,” Richter’s note, which is not online, reportedly concludes.

  18. Jeremy Jones says:

    Why are time and resources being wasted on surveys that come to an obvious conclusion. Those making these decisions either have poor management skills or lack the sense of urgency that those of us affected by ALS have. Please rethink and focus resources on making progress and not on irrelevant surveys.

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