Learning from Those Who Know—Latest ALS Focus Survey Needs to Hear from Current or Past Caregivers

Making assumptions can lead to trouble, but we all do it from time to time. You might assume that since the weather was nice yesterday it will be so again today, but if you don’t bother to find the weather forecast you may end up with some soggy clothes at the least. Annoying to be sure, but not really that big of a deal.

However, assumptions about the impact of a disease as serious as ALS can have far reaching implications. ALS researchers around the world work hard to challenge assumptions and find the facts behind the causes of ALS and how to best treat—and someday cure—the disease. But even in assessing the impact of ALS on day-to-day living, there is the danger of assuming.

The goal of the ALS Focus survey project is to move beyond those assumptions. It is a patient- and caregiver-led survey program that asks people with ALS and current and past caregivers about their needs and burdens. The goal is to learn as much as possible about individual experiences throughout the disease journey so that the whole ALS community can benefit.

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What a Year It Has Been

There is no doubt, 2020 will be a year not soon forgotten. We all have had our lives turned upside down in ways big and small, and we are all hoping for 2021 to turn things right-side up.

And while 2020 has been for many a year we’d be fine forgetting, there has been much worth remembering for our Chapter and the ALS community. For while 2020 has been a struggle, we are inspired by those who have struggled to overcome the obstacles in the path. And we are reminded that for people with ALS the struggle never ends. ALS does not stop for global pandemics, or for the challenges created by them.

Like so many others, when the scope of the COVID-19 pandemic became clear, our Chapter was forced to reimagine how to provide services to the community we serve. Our Care Service team worked with our partners to find new and different ways to meet the needs of people with ALS. Visits that would normally have been in person now took place over the phone or internet. Here on ALS Connect, we tried to keep you informed about how we and others in our community we meeting these challenges each and every day.

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More Encouraging News about AMX0035—And Still time to Sign the Petition to Make this Promising Treatment Available as Quickly as Possible

In September, we shared with you the exciting news that AMX0035, a trial ALS treatment drug developed by Amylyx Pharmaceuticals that had just completed the phase 2 trial, showed a statistically significant benefit to people living with ALS. This promising news lead The ALS Association and I AM ALS to launch a petition asking the FDA and Amylyx to work together to make AMX0035 available to the ALS community as soon as possible.

And while the news of a possible significant benefit to people living with ALS has created much optimism in the ALS community, the story doesn’t end there. All patients who completed the Phase 2 study were eligible to enroll in an “open label extension” (OLE) study to receive AMX0035 with no placebo comparator for up to an additional 30 months. Out of the 98 participants who were eligible for OLE enrollment, 92 percent (or 90 participants), opted to enter the OLE.

The primary goal of an OLE is to gather information about safety and tolerability of the new drug in long term, day to day use outside the trial environment, but they also can and do provide information on the effectiveness of the drug being tested. In this case, those findings make an even more compelling case for providing AMX0035 to the ALS community while Phase 3 trials take place, not after.

Continue reading More Encouraging News about AMX0035—And Still time to Sign the Petition to Make this Promising Treatment Available as Quickly as Possible

ALS Virtual Town Hall Brings Community Together

In a lot of ways, the origins of the “town hall” meeting are uniquely American. Sure, community gatherings have been happening from the time there have been communities to gather, but since the early days of colonial America, the gathering of a group of concerned individuals to be updated on current events has been a time honored tradition that persists to this day across the country. The idea has become so much a part of us that we don’t think twice that “town hall” can easily refer as much to a meeting as it does to a building.

But just because the town hall concept is old doesn’t mean it has stayed the same, and in 2020 when nothing seems normal the concept has had to evolve even more. What might have in the past been in-person gatherings have become virtual, expanding the scope and reach even further. But the basic idea of gathering a community to be updated on current events remains.

In just such spirit, our Chapter hosted a virtual town hall meeting in September to discuss the current state of ALS care services, research, and advocacy. Members of our community were able to hear from experts in these areas and ask question to the speakers. The full 90 minute recording is available for all to view.

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New Drug Treatment Shows Significant Benefit for People with ALS

These days, it seems everything is “news.” Or at least there needs to be enough “news” to fill the 24 hour news channels, your Facebook and Twitter feeds, and who knows how many website devoted to covering the “news.”

But in a time when everything claims to be “news,” it can be hard to tell when something genuinely newsworthy occurs. When we really ought to break out the “Breaking News” banners and pay attention.

For the ALS community, just such an event took place earlier this month, when the New England Journal of Medicine published encouraging clinical trial results showing that Amylyx Pharmaceuticals’ AMX0035 brought statistically significant benefit to people living with ALS. The study showed that AMX0035 decreased the rate of decline in the Revised ALS Functional Rating Scale (ALSFRS-R). The trial included 137 people with ALS and was conducted across 25 top medical centers through the Northeast ALS (NEALS) consortium.

People with ALS who received AMX0035 performed 2 points better on the ALSFRS-R compared to those who received the placebo. This is a statistically significant result, and in the real world could mean the difference between a person with ALS being able to feed themselves versus being fed, or the difference between needing a wheelchair versus not needing one.

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ALS Biomarker Research — One More Reason to Hope

The ALS Association funds millions of dollars in research every year, in a variety of scientific focus areas critical to advancing the search for treatments and a cure for ALS. In the nearly 150 years since ALS was first described in 1874, the search for new treatments and a cure has been frustratingly slow. However, the last decade has seen promising acceleration in progress, and in the last few years, five genes related to ALS have been identified. The ALS Association is currently funding a total of 169 active research projects in 16 different focus areas, and for everyone involved, critical discoveries cannot come soon enough. The promise shown in ongoing biomarker research gives reason for hope.

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Adding to Research Momentum—The First ALS Platform Trial Takes Shape

We’ve written before here about how there are real, tangible discovers being made in ALS research, but for everyone involved the pace of discovery can’t move fast enough. As such, when a new idea presents itself that has a real chance to move the needle it worth noting. Such an idea is taking shape right now with the first ALS platform trial at The Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital.

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The Year That Was—Looking Back

It’s the time of year where you almost can’t help looking back. In a few short days 2019 will be no more. The year has been one of milestones and new beginnings for us here at the ALS Association St. Louis Regional Chapter, and while we are looking back we wanted to include you and remember what a year it’s been.

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A Clinical Trials Primer

First, the obvious: everyone wishes there were more effective ALS treatments found already. Progress is being made, with five new genes discovered and two new treatments in the last five years—we are closer than ever to the possibility of a cure. But, even as we talk about how there have been real, tangible discoveries in ALS research, we cannot yet point to a reliable treatment to dramatically slow progression of the disease, let alone a treatment that stops progression or acts as a cure. It is heartbreaking for people with ALS and their families.

But for people with ALS, there is an active role they can take in fighting the disease: by participating in a clinical trial. For while the search new therapies begins in the laboratory, where ideas for new treatments are tested in cell cultures or test tubes, if a treatment shows enough promise it must eventually be tested on the intended end user, meaning human beings—living, breathing people.

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ALS Research—The Pace of Discover Is on the Rise

From the outside, research can seem like an endless process. As much as we all yearn for the “AH HA!” moment of discovery, more often than not the gains from any sort of research are incremental. Not so much finding a needle in a haystack as a slow, methodical, documented labeling of this piece of hay, then this piece, then this piece….As Thomas Edison said about the process of researching and creating the lightbulb: “I have not failed 10,000 times. I have not failed once. I have succeeded in proving that those 10,000 ways will not work. When I have eliminated the ways that will not work, I will find the way that will work.”

ALS research has proven to be just such a challenge to the best minds in the medical and scientific communities. But recent advances are creating excitement about new ideas and opportunities, aided in no small part by the influx of funding created by the Ice Bucket Challenge. With all that is going on, we wanted to take a moment to look at what is happening in ALS research—to see what is new, what is promising, and what the future may hold.

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