Treatment & Support Resources

What Is ALS?

Amyotrophic lateral sclerosis (ALS) is also known as Lou Gehrig’s disease, named after the New York Yankees baseball player with ALS. ALS is a disease that affects the motor neurons, the nerves that control movement. Over time it leads to muscle weakness, affecting the ability to walk or use one’s hands and arms and eventually difficulty swallowing and breathing. It also causes uncomfortable muscle twitches and pain due to reduced mobility, muscle cramps and tightness. It is a fatal disease, but the timeline of the disease can be very different for different people. One in every 300 people will receive a diagnosis of ALS in their lifetime. Only about 10 percent of people with ALS have a genetic or familial type. U.S. military veterans are twice as likely to develop ALS.

Connecting with a multidisciplinary, certified ALS clinic is important to assure the highest quality of care.

Certified Treatment Centers of Excellence

To become certified as a center of excellence, an ALS clinic must meet rigorous clinical care and treatment standards, participate in ALS-related research and successfully complete a comprehensive site review. Achieving certification through successful completion of the program’s robust clinical and administrative reviews confirms the validity and comprehensiveness of their standards of quality and implementation of best practices. Below is a list of Certified Treatment Centers of Excellence in the VA/DC/MD Area.

ALSA Recognized Treatment Centers)

The ALS Association Recognized Treatment Centers have the same high quality approach to multidisciplinary care as The ALS Association Certified Treatment Centers of Excellence; however, they may not offer onsite research or opportunities to participate in clinical trials. This designation assures patients of the very best standard of care, while also communicating the fact that research or clinical trial involvement may not be part of the institution’s offerings. Below is a list of ALSA Recognized Treatment Centers of Excellence in the VA/DC/MD Area.

Synapticure

Synapticure is a new care coordination and telemedicine program available to ALS patients and their caregivers. Synapticure brings personalized care online and to your home. Built by patients, caregivers and clinicians to transform care for those living with ALS and PLS.

 

NOTE: If there is not a multidisciplinary ALS clinic near you, contact your local ALS Association or Muscular Dystrophy Association chapter to ask about other options. They may also have travel grants or resources if transportation is an issue. Even if you can make it to an ALS clinic every 3 months or so, we strongly recommend being under the care of one of these clinics.

A second opinion can help you decide which treatment is most appropriate for your particular situation. It’s important to make sure that any second opinion you seek is from a physician who has broad experience and expertise in treating ALS. Below is a list of some (but not all) opportunities for a second opinion.

ALSA offers support groups for both patients and caregivers. Below is a list of a few support groups in the area. These support groups offer a variety of services, including care coordination, remote clinical care, caregiver support groups, traditional support groups, and more.

Resources:

  • Everything ALS offers regular “Expert Talks.” Guests have included Dr. Richard Bedlack, Dr. Eduardo Locatelli, and many others. Their YouTube channel includes recordings of the Expert Talks on a variety of subjects. Click here to visit that YouTube channel.
  • Hope & Housing ALS Renovation Series: A home renovation series meant to serve as a guide for remodeling your home for people living with ALS. Learn more here.
  • I’m Dying to Tell You podcast, hosted by Lorri Carey, who herself is living with ALS. She created the podcast to find and share stories of inspiration. Jim is featured on season 4, episode 80.
     

Multiple other drugs are being studied in trials and showing promise. See below for links to the clinical trials available:

There is currently no cure for ALS. However, there are medications that slow the progression of the disease or show a survival benefit for a certain period of time. These include riluzole, edaravone, and AMX0035. A brief synopsis of these treatment options is below.

FDA Approved Treatments

Riluzole (Rilutek) is an oral medication believed to reduce damage to motor neurons by blocking the glutathione-mediated neuroexcitation. Clinical trials in people with ALS showed the riluzole prolonged survival by a few months in clinical trials, but newer “real world” evidence suggests the survival effect may be greater than previously thought. (Hinchcliffe et al 2017)

Edaravone (Radicava) is an IV medication (given daily) that has been shown in clinical assessments of function to slow the functional decline in patients with ALS. Edaravone works as an antioxidant, reducing the toxic oxidative stress that can lead to cell death of the motor neuron. An oral form of this medication was approved in May 2022 by the FDA. This vastly enhances access to this drug, since daily administration of an IV medication is challenging

AMX0035 (Relyvrio) Based on top-line results from the Phase 3 PHOENIX trial of AMX0035 in ALS, Amylyx Pharmaceuticals (the manufacturer of Relyvrio) has started a process with the FDA and Health Canada of voluntarily discontinuing the marketing authorizations for Relyvrio. This drug is no longer available for new patients as of April 4, 2024. Patients currently on therapy in the U.S. and Canada, who, in consultation with their physician, wish to continue can be transitioned to a free drug program.

Supplements

ALSUntangled™ reviews alternative and off label treatments, with the goal of helping people with ALS make more informed decisions about them. ALSUntangled is a website created by Duke ALS physician Richard Bedlack, who has focused his research on alternative and complementary therapies in ALS treatment. Bedlack has a large scientific advisory team that does a deep dive, reviewing the evidence for various therapies and grades each therapy based on the evidence for safety, efficacy etc. For the more promising therapies, he tries to develop clinical trials to further evaluate efficacy.

ALS requires constant adaptations as the course of disease progresses. Some adaptations are small, but some are major and require planning and preparation. Below are websites of organizations dedicated to helping ALS patients and their caregivers meet these challenges.

Make changes before they are absolutely necessary: it gives everyone time to practice

When I casually mentioned to my daughter-the-young-doctor that I was thinking about buying a cane, as a souvenir, while we were on vacation together, she gently reminded me what she has learned as a doctor: patients do better when they have a chance to practice with the cane before they absolutely need one. Following her sage advice, I picked up a collapsible cane before our trip, and brought a painting home as my souvenir.

My ALS friends have told similar stories about their rollators, wheelchairs, and bath chairs. They use them, for practice, before they are absolutely essential.

Not long after diagnosis, our kind and concerned friends wondered when and how we would manage in our home with the master bedroom and bath on the second floor. Would we need a ramp for the three steps at the entrance? Although home renovations can be overwhelming, getting them organized, and completed before they are critical to daily life, has allowed us to enjoy them as improvements to our home. Having the renovations completed early also allows for gradual transitions.

Working together creates power for change: wheelchair consultations return to UVA ALS clinic

Up until several years ago, ALS patients at UVa could get their repairs and other services for their motorized wheelchairs onsite, during their visit to the ALS clinic. One-stop shopping. Whether newly-diagnosed or managing advanced disability, the onerous logistics of using a power wheelchair were efficiently addressed during a routine visit to the ALS clinic. However, in one our initial meetings with the Dart Clinic for ALS at UVa–while standing up the Patient Advisory Council to better integrate patient needs into the care provided at Dart –a clinician told Peggy and Jim that the clinic had been forced to end this program.

It immediately dawned upon everyone in the meeting that this is precisely the kind of bureaucratic blip that becomes a logistical nightmare for patients. How many extra hours and trips to Richmond must this mean for routine adjustments or repairs? And how hard would it be to fix this once and for all?

This was why we created the Patient Advisory Council (PAC)–to fix problems for patients once and for all. Within a month, Peggy, representing the PAC, and working with clinic staff and institutional leadership, had the comprehensive wheelchair services returned to UVA as a routine part of its interdisciplinary care team.

The pharmacist is your new best friend. Consult them regarding high co-pays, prior-authorizations, off-label medications, mechanisms and side effects, the pharmaceutical literature, and clinical trials

On December 2, 2021 Jim was diagnosed with ALS at UVa. Leaving the medical center that day, he drove with his family to the Kroger Pharmacy to fill a prescription for the only available oral medication for ALS, called Riluzole. At that time, Jim was taking no medications and had not had a prescription filled in years. He was delighted to learn they had Riluzole in stock, and was shocked to find out the co-pay for a 3 month supply would cost almost $6,000. Astoundingly, and without being asked, the pharmacist found a GoodRX coupon that brought the cost down to about $90.

Time and again, the pharmacist has saved the day. They have helped us to figure out complicated prior-authorizations, explained drug mechanisms and side effects, sleuthed out off-label medications, discussed clinical trials, and communicated with clinicians at distant tertiary centers to help coordinate my care. I am grateful to have a pharmacist on my interdisciplinary team. I feel fortunate that my pharmacist is directly accessible to me through the EPIC MyChart patient portal.

Receiving an early diagnosis promotes key treatment options for ALS: use the ALSA algorithm

There are perks to being married to a brilliant doctor when you develop signs and symptoms of ALS. Jim’s diagnosis came with lightning speed compared to most patients. ALS is a diagnosis of exclusion. Making the ALS diagnosis requires an EMG, and often multiple MRI’s and lab tests. All of that takes time. When ALS is neglected as a possible diagnosis, the signs and symptoms of ALS often lead doctors to an incorrect diagnosis, like carpal tunnel, or spinal stenosis, delaying a diagnosis of ALS. A delayed diagnosis wastes precious time that can be used to try meaningful and life-extending treatments for ALS. A delay in treatment can mean that a patient is no longer eligible to enter key, experimental clinical trials.

The ALS Association has developed a concise algorithm to help clinicians remember to consider ALS in their differential diagnosis.
thinkALS | The ALS Association
Think ALS Tool

Grassroots political action works: The Amylex success story

ALS is a daily barrage of firsts and workarounds. Most systems we interact with are not built for this pace of adaptation, least of all federal bureaucracies like the FDA. We discovered this firsthand in the push to approve AMX-0035, now RELYVRIO.

As a rule, rigid consistency is good at FDA–they are a rigorous gatekeeper of safe and effective therapies upon which the entire world relies. But sometimes a disease like ALS requires the same thing from regulators as it does from patients–adaptation. ALS is 100% fatal; it is fast-moving; it has no current cure. In these ways, it is unlike almost every other disease with which the FDA deals. When it comes to new treatments for ALS, the meaning of “safe and effective” is more nuanced.

In the Spring of 2022, the FDA issued its initial denial of the drug Relyvrio, and the ALS community was unanimously opposed to the decision. The FDA had denied the drug because while it was safe and effective, its effectiveness was not statistically robust enough. The number of months potentially added to the life of someone living with ALS was not significant enough for FDA approval. As we looked into the decision, we realized that it had been made by a room full of biostatisticians, most of whom had no experience with ALS or neuro-degenerative conditions.

The FDA had no malice nor corrupt intent. It was just that their decision lacked the obvious perspective of patients living with ALS. Something safe that potentially adds 15 months to someone’s life is better than nothing. Denying the drug and giving patients nothing was an error of omission.

The Hummingfbird Fund joined an effort led by I AmALS to speak up and counter the thinking and decision at FDA. We called on our followers to write to the FDA in public comment, and to lobby legislators. In the end, a significant percentage of the public comment came from Hummingbird contacts.

The many thousands of public comments and emotional testimonies illuminated the error of omission. Doing nothing was actually doing something potentially harmful: denying people with ALS the opportunity to take a safe medication that could extend their lives for many meaningful months.

In the fall of 2022, the FDA finally approved AMX-0035. The approval injected hope into the ALS community. Once again, the power of patients’ voices prevailed.

Recently Diagnosed?

Connect here for help and support, including a comprehensive toolkit available through I AM ALS.