One month ago on May 25, 2021, representatives from the FDA joined a We Can’t Wait Action Meeting. During this meeting, eight ALS advocates from across the US shared their personal experience with the disease to highlight why those with ALS need expedited access to experimental therapies.
Following the We Can’t Wait Action Meeting, the ALS Association sent a letter to the FDA that requests the agency to reaffirm their commitment to the 2019 ALS Guidance and provide details regarding how they have implemented the guidance.
The FDA’s Response to the Letter
Dr. Patrizia Cavazzoni, Director of the Center for Drug Evaluation and Research sent a response letter to the ALS Association on June 15, 2021. The letter acknowledges the “burden of ALS for patients, their families and caregivers,” noting there is an “unmet need for treatments” for the disease.
We can also assure you that we are exercising the regulatory flexibility described in FDA’s 2019 guidance for industry titled Amyotrophic Lateral Sclerosis: Developing Drugs for Treatment (ALS Guidance) and we continue to commit to doing so,” Dr. Cavazzoni shared in the letter.
The FDA responded positively to the We Can’t Wait Action Meeting, but the ALS community still has grave concerns. The FDA has not clearly communicated what they are doing to expedite the approval of promising treatments for ALS patients. Yet they allow regulatory flexibility for other disease treatments.
The FDA recently used its regulatory flexibility to approve Aduhelm to treat Alzheimer’s. While this is great news for those living with Alzheimer’s and their loved ones, those living with ALS are left to wonder why the same urgency is not granted to them.
In the letter from the FDA, Dr. Cavazzoni shared that they are “committed to exercising [the guidance] when appropriate.” However, the agency does not appear to make any of those considerations for AMX0035. The drug met its primary and secondary endpoints in The Phase 2 Trial and is generally safe. Yet the FDA requires another clinical trial.
Health Canada is already working with Amylyx to bring the drug to market for Canadians with ALS in 2021. Yet Americans with ALS will have to wait years to access the drug. That is if the FDA even allows it to hit the market.
How We’re Moving Forward
The ALS Association is deeply committed to expediting the access to promising drug treatments to those living with ALS. We hope to achieve this by:
- Continuing to put public pressure on the FDA
- Work behind the scenes to expedite change
- Seek greater public accountability for the FDA
Read more about this response and how the ALS Association is implementing the above action items here. Our organization will continue to share important updates as they arise as we continue the fight to end ALS. We will not rest until we find a cure!
Read the Full Letter from the FDA
Even from a young age, Norman’s grandmother instilled in him the importance of faith. It kept him strong when he moved to the U.S. from Jamaica as a child, and it led him to become a Chaplain in the Army. Norman served in the army for 32 years, 24 of those being as Chaplain, before retiring in 2016.
Today, Norman and Dee, his wife of 41 years, live in San Antonio. They have two daughters—one who lives with them at home and the second who recently got married and is teaching in Korea.
During this time, virtual support groups kept the Jones family connected with the ALS community. “You’re able to express your emotion and your struggle. And then you get advice from others how they handle their struggles,” Norman shared.
Besides the hope of finding a cure, Norman, and Dee hope that researchers will discover ways to detect ALS early and provide treatments to improve quality and length of life with the disease. “I have a lot of hope,” Norman shared.
The future is an illusion and only a concept in your head. That is how our minds trick us; we are promised that we’ll get something that we already have. The future begins today, right now-but how do we stay present in the here and now, and invite happiness into our lives? Try these tips as a start!
John Robinson from Parish, FL, a member of the board of trustees of the ALS Association and retired military veteran of nearly 32 years, was diagnosed with ALS in 2019. ALS not only affects the person with the disease but also their family caregivers. Both he and his wife can no longer serve their communities in the same way because of ALS. “The loss to society of their skills and humanity leaves holes never filled the same way again.”
Sunny Brous, a Texan with ALS, joined the call to share her perspective. Sunny was diagnosed with ALS in 2015 at the young age of 27. For Sunny, access to experimental treatments go beyond extending her own life. “Expanded access is how I can contribute to finding a cure for ALS. It takes my love for others and makes it a quantifiable action combining research and science and my dedication to the greatest good of people living with ALS.”
Sandy Morris, from Truckee, CA played a recorded message. She tearfully shared how many of her friends with ALS died waiting for access to life saving drug treatments. “Think about how much better it would be if we were able to die trying rather than die waiting,” she shared, urging the FDA to consider how their delays harm those living with ALS. “It’s not within my power to save my life—it’s within yours.”
Troy Fields from Champa Bay, FL was diagnosed with ALS in 2018. He noted how the considerations of benefits versus risk should be put in the hands of those living with ALS. “Because unless it is your own life on the line, you simply do not get it.”
Cari Meystrik from Nashville Tennessee was diagnosed with ALS in 1998 when she was only 28 years old. She has watched hundreds of friends die from this awful disease. “Would I jump to try a drug that might help me live longer of a disease that I am dying from if it has already proven to be sage and well tolerated but isn’t approved yet? Absolutely. Give me that chance.”
Jay Quinlan from New Orleans joined with his wife and caregiver Stephanie. “You have shown through your recent handling approvals for COVID vaccines that flexibility and expediting processes are possible.” He asked the FDA to use the same urgency when it comes to approving drug treatments for those living with ALS.
Tommy May from Pine Bluff, AR was diagnosed with ALS in 2005. Due to slow progressions, he was both unable to participate in clinical tries and frustrated with the lack of access to treatments. He discussed how those living with ALS are desperate for HOPE on their path. “We are not interested in the long-term. We are desperate for the here now.”
Larry Falivena of Apex, NC, has ALS caused by a mutation of the SOD1 gene. The fight for treatment access is not just for himself—it is also for his children. “I’m asking the FDA to help make sure my boys don’t have to worry about ALS.” He shares that even if the opportunity exists for just a small number of patients to have extended time on this earth that they need that chance.
In January 2019, after a year of doctor’s appointments, increased symptoms, referrals and second opinions, Jack Mercz was diagnosed with ALS at the age of 53. In a recent ALS Texas interview with his widow Jennifer Mercz, Jennifer shared “our whole relationship pretty much changed in a day.” Jennifer and Jack had a then two-year old son, and Jennifer added, “I had been with my husband for 10 years and suddenly found myself not only caring for my husband but also my young child.” She recalled moments that included putting her son in the bath and helping her husband in the shower at the same time, and at the end of the day being too tired to even take a shower herself.
Jennifer shared that Jack was not very accepting of any kind of help; he wanted to be stronger in every way and help Jennifer at home when he was not traveling for work. Frustration over his declining physical abilities and strength led to anger and depression and Jennifer shared that she “became very standoffish emotionally-clinical with him.” While Jennifer did reach out to ALS Texas and the community as much as she could, she added that “things were moving so quickly that I didn’t seem to have time” to do more-such as accessing our ALS Texas equipment loan program sooner. “The ALS Association delivered a Hoyer lift to our home-it was great, and we needed it. Jack was able to get up (after weeks of being in bed) and sit on the couch with our son.” He told me that he wished we would have had that [Hoyer lift} earlier. In retrospect, she added that she would do things differently in making some “executive decisions” about Jack’s needs; equipment and care that would have also benefitted her. She urges all family caregivers to seek help with caregiving if at all possible-from anyone who is able to help, including friends and family members, and when someone says, “Hey, do you need help?” the answer is “YES! Here’s how you can help me.”
This year, we are honoring Sarah Durand as our ALS hero during the Under Ballou Skies Golf Classic & Party. In the last year, Sarah has not only had to navigate life during a pandemic, but also an ALS diagnosis.
Once the COVID-19 pandemic began, resorts closed, giving Sarah several months off, yet she saw no improvement with her finger. She went to a Rheumatologist and was diagnosed with rheumatoid arthritis in early 2020 and started doing injection treatments. However, after three months of injections, her condition continued to worsen.
In the meantime, Sarah is finding a new normal, from adjusting her home to make it accessible to applying for disability. “Once I get adjusted to one thing, I lose control of something else.”
Sarah finds joy in time spent with her boyfriend, Danny, her friends, or her two dogs and cat. She cheerfully shared how Danny is incredibly supportive of her ALS journey, “He’s really been with me day in and day out.” From cooking together, to enjoying a relaxing day at the pool, Sarah and Danny are making the most of every moment they have together.
Before being diagnosed with ALS, Sarah ran races and supported fundraising events for breast cancer research. Her mom had fought the disease, which made it a cause dear to her heart. However, she noted that there is not the same kind of widespread awareness surrounding ALS. “I never see anything for ALS like pins, hats, clothing”.
This made me think of the phrase “bird’s nest on the ground”. It refers to something being very easy and not at all a challenge-offering at least some advantage with little effort or risk. Do you ever seek the bird’s nest on the ground-especially when it comes to FEAR and uncertainty? While the temporary fix may seem easier in the moment, it often creeps back up again until we put on our shield and truly face FEAR, rise above it and through it, and begin the much brighter journey of courage and peace. We can’t do that alone, and our monthly newsletter shares just some of the ways that you connect with others and face your fears within our ALS Texas community, and with a whole lot of encouragement and support along the way.