ALS Texas

We Can’t Wait: ALS Advocates Urge the FDA to Expedite Access to Experimental Therapies

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The average life expectancy for someone after an ALS diagnosis is only 2 – 5 years. However, the process of developing, testing, and approving new treatments takes far longer. For those living with ALS, time is limited—We Can’t Wait.

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.

Compelling Testimonials from the ALS Community

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.

Where Do We Go from Here?

In 2021, the ALS Association is committed to accelerating the development, approval, and access to affective new treatments. We will do this by re-introducing and passing the Accelerating Access to Critical Therapies (ACT) for ALS Act and the Promising Pathway Act, so the FDA is fully equipped to speed ALS trials and drug approvals. We will discuss these matters further during the annual ALS Advocacy Conference in June.

It is our sincere hope that the testimonials of these individuals and those who sent written stories will compel the FDA to make actionable changes for the ALS community. Time is off the essence—We Can’t Wait.

Submit your testimony to the FDA

Register for the Virtual ALS Advocacy Conference

Read Our 2021 Advocacy Priorities

Because of You: 2020 in Review

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This year our ALS community has come together in new ways, connecting from afar and working together, even when we are physically apart. In the midst of a pandemic your support never ceased. And because of you, we are now serving more people with ALS and their families than ever before.

Because of you, family caregivers are receiving the support and resources they need. Medical equipment is being loaned to people in need and alleviating their financial burden. Youth are sharing their voices and finding strength through shared experiences. ALS clinics are continuing to deliver specialized care and conducting clinical trials. New treatments are on the horizon.

Because of you, our Texas Chapter Events continue to raise crucial funds while moving to socially distanced and virtual fundraisers. And our Walk to Defeat ALS at Home brought Texans together from across the state, allowing us to gather safely even from afar.

This is how your support has made an impact on the lives of Texans with ALS.

Local Care Overview

Our team provides resources and support to people with ALS and their families. Your generosity enables us to continue this critical work, counseling newly diagnosed patients and helping Texans with ALS navigate the disease.

1,004 People with ALS are currently Registered with Our Chapter
(compare to 929 at this time in 2019)

From February to November 2020:

278 New Patients
(compare to 169 at this time in 2019)

1,165 Total Patients Served
(compare to 1,207 in all of 2019)

Learn more about our Local Care Programs.

Equipment Loans

Our Equipment Loan Program provides crucial medical equipment to people with ALS, without charge. COVID-19 impacted this program significantly and due to safety concerns we were unable to access and deliver much of our equipment in our loan closet. We were purchasing new equipment to continue to meet the needs of people with ALS, but saw our funding dwindle rapidly. We asked for your help, and through your support, we were able to get the funding needed to continue this vital program. Now, we’re working with our partners to deliver needed equipment in a safe manner and ensure people with ALS have the equipment needed to navigate their everyday lives.

From February to November 2020:

331 Pieces of Equipment Loaned to People with ALS

If you are a person with ALS or caregiver who needs help with equipment, please contact us.

ALS Multidisciplinary Clinics

In the early days of the pandemic, many of our ALS Multidisciplinary Clinics had to cancel regular clinic days. As time has progressed, each clinic has modified their operations to best serve the safety of their community. Most have now resumed normal operations or offer a combination of in-person and virtual visits. We’ve been working closely with our clinic partners to help keep our ALS community safe and ensure our presence as a resource for Texans with ALS and their families.

We partner with 11 ALS clinics across the state of Texas. You can see the latest update about clinics here.

Staying Connected

Gathering in-person is not an option for our ALS community who are at greater risk to infection during this pandemic. To stay connected we moved all support groups to virtual platforms, which have continued to meet monthly. In addition, we transitioned in-person educational presentations and workshops to virtual platforms and provided statewide access. Seeing an emerging need, we also added a Veterans support group.

From March to November 2020:

120 Virtual Support Groups, Workshops, and Presentations

1,336 People Engaged in Virtual Events

You can view our upcoming virtual events here, and watch recordings of past events.

Community Education

Community support has continued for Texans with ALS and their families. We worked with partners across the state to develop an educational series of presentations and interactive workshops. This included a clinical series, focused on different aspects of the ALS clinic, connecting healthcare professionals with the patients they serve.

View the Clinical Series

New Family Programs

We’ve been listening closely to our ALS community to discover where additional resources are needed. Two big needs have emerged: greater support for caregivers and programs for youth and children. This year we have begun rolling out these virtual programs.

Learn More About our Caregiver Program

Learn More About Our Youth & Children Program

Walk to Defeat ALS at Home

The Walk to Defeat ALS 2020 looked very different this year. Although we were unable to all gather in-person, we still stayed connected. Our Live Opening Ceremony featured teams from across the state and conveyed the incredible energy of our ALS community. Big News: We met our goal of raising $1 million. Thank you to everyone who participated in the Texas Walk to Defeat ALS !

Watch the Texas Walk to Defeat ALS Live Opening Ceremony

Research Progress

Because of you, research is advancing and a potential treatment is on the horizon. In September, the New England Journal of Medicine published the results of a phase 2 trial (known as CENTAUR) of the compound AMX0035, produced by Amylyx, a Massachusetts-based pharmaceutical company. The results of the trial were very promising – people with ALS receiving this drug experienced a significantly slower decline in disease progression and lived an average of six months longer, compared to those on a placebo.

The ALS Association and I AM ALS have issued a petition calling on the drug company and the FDA to make the treatment widely available as soon as possible. To date, the petition has received more than 50,000 signatures.

Advocacy

On November 11, the Senate recommended $40M for the Department of Defense ALS Research Program (double the previous year’s amount) and $10M for the CDC National ALS Registry in their FY2021 appropriations bills. These victories help us better support the ALS community and bring us one step closer to defeating the disease.

Next, we wait for the House and Senate to agree on final funding levels. We’re cheering on our ALS community who are continuing to fight to make this a reality.

Looking to the Future

Because of you, we have a lot to look forward to in the new year. In January, we will launch new support groups for elementary and middle school children, as well as teen and young adult groups. We will also kick-off an educational series for people with ALS who have been newly diagnosed and their families, providing them with the resources needed for their journey. This will include information about current research, multidisciplinary care, employment and insurance, and navigating the disease.

Because of you, we have been able to increase access to support groups, presentations and workshops via virtual platforms. As we continue to navigate the pandemic and look beyond this crisis, virtual programming will continue to be a critical part of our work, enabling us to reach Texans with ALS and their families in all corners of the state. Even when we can safely resume in-person gatherings, we’ll continue to provide a place to virtually connect, learn, and share.

In the face of the pandemic, you made this crucial work possible. Thank you.

ALS Drug Shows Further Promise, Slowing Progression and Increasing Survivability

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Muscle and Nerve has published the results of the open label extension study of AMX0035, a promising new drug therapy for people with ALS developed by Amylyx. The study showed that people who received the drug lived about 6.5 months longer than people who received the placebo during the trial. This finding is in addition to the findings from the phase 2 trial, showing that people with ALS receiving this drug experienced a significantly slower decline in disease progression, compared to those on a placebo.

As quoted in the New York Times article, Neil Thakur, chief mission officer of the ALS Association, states, “That is a very meaningful benefit for people affected by this devastating, fatal disease.”

These findings further show that the treatment should be made available to people with ALS as soon as possible. We urge the FDA and Amylyx to work together to bring this treatment to market as soon as possible and we stand ready to help ensure that happens. You can help by signing our petition to the FDA and Amylyx at als.org/petition.

ALS Drug Slows Progression, Petition for FDA Approval

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The New England Journal of Medicine (NEJM) has published the results of a phase 2 trial (known as CENTAUR) of the compound AMX0035, produced by Amylyx, a Massachusetts-based pharmaceutical company. The results of the trial were very promising – people with ALS receiving this drug experienced a significantly slower decline in disease progression, compared to those on a placebo. Additionally, this drug shows to be safe and well-tolerated indicating a positive benefit and risk consideration for people with ALS.

The ALS Association and I AM ALS have now issued a petition calling on the drug company and the FDA to make the treatment widely available as soon as possible. The ALS Association and I AM ALS are asking that Amylyx and the FDA move with urgency to ensure this drug is a treatment option for all people living with ALS.

Click to expand

The ALS Association committed $750,000 to Amylyx in funding for this research, and $1.4M to the NEALS clinical trial consortium to support the trial. These grants were made possible through the ALS Ice Bucket Challenge. The trial included 137 people with ALS and was conducted across 25 top medical centers through the Northeast ALS (NEALS) consortium. All of the participants enrolled in the trial were diagnosed with sporadic or familial ALS within the previous eight months and had a rapid progression of the disease.

With a typical survival time of between 2 to 5 years, people with ALS cannot wait for the full experimental process of this drug to continue. In light of the substantive data of AMX0035’s effectiveness and safety, it has been requested that the FDA and Amylyx to work together to bring this to people with ALS as soon as possible by approving the drug prior to a Phase 3 trial. To access and sign the petition, visit www.als.org/petition.

Sign the Petition Today!

ALS Association Makes Multi-Year Commitment to HEALEY ALS Platform Trial

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Photo provided by Texas Tech University Health Sciences Center in El Paso

This week a $3 million commitment was made by The ALS Association to the first platform trial for ALS. The platform trial will be taking place at the Sean M. Healey & AMG Center for ALS at Mass General in collaboration with the Barrow Neurological Institute and the Northeast ALS Consortium (NEALS). A “platform trial” is a clinical trial in which multiple treatments are tested and evaluated simultaneously. The Association’s commitment is $1 million per year for 3 years.

“This platform trial will significantly speed efficacy trials for ALS research, and is a major advancement for ALS research,” said Calaneet Balas, President, and CEO of The ALS Association. “In addition to supporting the HEALEY ALS Platform Trial, we will be working to ensure the rest of the research pipeline is moving fast enough to keep up. We are delighted to be partnering with Dr. Merit Cudkowicz and her team on this project.”

The platform trial model will greatly accelerate therapy development, allowing investigators to test more drugs, increase patient access to trials, and reduce the cost by quickly and efficiently evaluate the effectiveness of multiple therapies. This trial mode has already been proven successful in the cancer field. New treatments will be added to the trial as they become available which will decrease the gap in time from identification of an exciting therapy to testing. Having a shared infrastructure, common data & sample collecting processes, and centralized governance within the platform trial will help save time and money and led to more operational efficiencies.

“Working closely together, we will bring new therapies forward faster for people with ALS,” said Cudkowicz. “The platform trial builds on previous ALS Association supported research on many new ALS targets and trial innovations. The HEALEY ALS Platform Trial initiative can only succeed with the support and involvement of the entire ALS community. We look forward to ongoing partnerships with people and families with ALS, all ALS foundations, our colleagues in the NEALS Consortium, industry partners and regulatory agencies,” Cudkowicz added.

This funding from The ALS Association will directly support numerous parts of the platform trial. The following will directly benefit from this funding: the Clinical and Data Coordination Center activities at Mass General, the Biomarker Development Endpoint Engine, and the Monitoring and Outcome Measures Core at Barrow Neurological Institute.

The ALS Association will become an integral part of the Foundations and Benefactors Committee responsible for advising on study progress, data sharing, budget matters, fundraising, patient recruitment/retention and variety of other scientific and operational topics. In addition, the trial will be supported by the enrollment of patients at ALS Association Certified Centers of Excellence, Recognized Treatment Centers, and other affiliated centers.

This award to the Healey Center builds on a long relationship The ALS Association has had with Massachusetts General Hospital, Dr. Cudkowicz, and other researchers. The Association has committed over $9 million since 2010 to ALS researchers at Massachusetts General Hospital.

For more information, please email healeycenterforals@mgh.harvard.edu and visit the center’s website www.massgeneral.org/als/. To read the full press release, visit www.alsa.org/.

Clinical Trials Give Hope to Texans with ALS

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The ALS Association of Texas invests in research to discover effective treatments and a cure for ALS. One of the ways we support research locally is through clinical trials, which are conducted at our ALS clinics across the state.

Dr. Carlayne Jackson, Medical Director of the ALS Clinic at UT Health San Antonio, first became interested in clinical trials in neuromuscular disease as a medical resident at the clinic. She would be the first to tell you she “fell in love with the patients” she treated during her residency, fellowship and eventually her first two years of leading the clinic’s ALS studies.

Founded in 1996, the clinic is designated as an ALS Association Certified Treatment Center. Dr. Jackson and her staff have used their multidisciplinary approach to offer hope for the future of ALS to their South Texas community and the 250 ALS patients they serve. Through the generosity of the ALS Association and the Glenn Biggs Institute for Alzheimer’s and Neurodegenerative Diseases at UT Health San Antonio, the clinic has recently been able to add neuropsychological testing and the services of a Nurse Research Coordinator to their offerings.

The Clinical Trials Process

The Research Coordinator meets with patients interested in participating in a clinical trial, sharing helpful information, including the number of visits, what to expect at each visit, potential side effects and treatment effectiveness. Once they have a thorough understanding of the risks and benefits, patients are screened to determine if they meet the criteria defined by the company or group sponsoring the trial. It is important to note that most of the criteria are put in place to protect each patient’s safety. Researchers must quickly determine if an intervention is working or not.

After a particular therapy has gone through Phase I testing on a healthy population, the San Antonio clinic conducts Phase II and Phase III trials on ALS patients accepted into the studies. Phase II trials focus on safety and dosing of the medication. Next comes the pivotal trial – Phase III – which the FDA reviews in order to determine the drug’s approval for patients outside of the trial. Phase II trials typically take six to 12 months, whereas Phase III trials can last 12-18 months.

The Importance of Clinical Trials

Dr. Jackson believes clinical trials are vital.

We will never be successful in bringing effective therapies to ALS patients without them first being studied in trials. We can’t prove what does or doesn’t work without a trial,” she says.

Through clinical trials at ALS clinics, such as the one at UT Health San Antonio, we are one step closer to finding treatments and a cure for ALS.

ALS Association Awards Joint Grant to Help Fund Houston Neurologist’s Groundbreaking Research

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The ALS Association, ALS Finding a Cure (ALSFAC), and MDA have jointly awarded a $2.5 million clinical trial grant to Dr. Stanley Appel and his team of researchers at the Houston Methodist Neurological Institute, and Dr. Merit Cudkowicz and his team at Massachusetts General Hospital.

The funding award will allow Dr. Appel to expand the first-in-human study that demonstrated promising results leveraging patients’ own immune cells to treat ALS. [Read more…]

Houston Neurologist Discovers Treatment That Could Halt the Progression of ALS

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Research Study Information and Photos Provided by Methodist Houston Hospital

Dr. Stanley Appel, neurologist and co-director of the Houston Methodist Hospital Neurology Institute, has been leading the ALS Clinic at Houston Methodist Hospital since its founding in 1982. This was the first multi-disciplinary clinic dedicated to the treatment of and research on ALS. More than 35 years later, he continues to innovate in the field.

His latest research? An immunotherapy treatment that could stop the progression of ALS in its tracks. [Read more…]

Closer to a Cure: Researchers Open Enrollment for Tofersen Phase 3 Clinical Trial

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Our “closer to a cure” segment will highlight research news and important milestones made by researchers as they work towards finding a cure for ALS.

Exciting news! Neuroscience collective Biogen has initiated a phase 3 clinical trial to evaluate Tofersen (previously called BIIB067), a type of antisense drug meant to target superoxide dismutase (SOD1), for the potential treatment of ALS. 

It’s approximated that two percent of people living with ALS have a mutation in the SOD1 gene- the second most common cause of genetic ALS. An SOD1 mutation leads to abnormal protein, which is likely toxic to cells and could lead to the death of motor neurons- the cells that die in ALS. Tofersen has been designed to reduce levels of SOD1 protein in people with ALS caused by an SOD1 gene mutation.

The trial is currently enrolling with 60 spots available for people with SOD1 ALS.

Interested in enrolling and helping researchers test the safety and efficacy of Tofersen? The ALS Association has worked with Biogen to create a FAQ surrounding the trial. The FAQ covers how to enroll, where to get more information, study design and endpoints, expanded access, and more. See the FAQ here.

For more information about the clinical trial and its currently enrolling locations, visit: https://clinicaltrials.gov/ct2/show/NCT02623699.

Closer to a Cure: What is the KIF5A ALS gene & why is it important

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Our “closer to a cure” segment will highlight research news and important milestones made by researchers as they work towards finding a cure for ALS.

While ALS is currently a disease with no cure and limited treatment options, the ALS Association continues to fund groundbreaking research to bring us closer to a cure. Last March, researchers from collaborative initiatives funded by The ALS Association, with money raised through the ALS Ice Bucket Challenge, announced the discovery of a new ALS gene- KIF5A.

You may have heard the news in the past year, but do you truly understand what the KIF5A gene is and why it’s important? If your answer is “no” that’s ok! That’s what we’re here for.

What is the KIF5A ALS Gene?

The KIF5A ALS gene was discovered through a unique collaborative effort of over 250 researchers, led by Dr. John Landers at University of Massachusetts Medical School and Dr. Bryan Traynor at the NIH. Researchers analyzed genetic data from over 101,000 samples to discover the KIF5A gene and it’s actually the fifth new ALS gene discovered since the 2014 Ice Bucket Challenge.

KIF5A, or kinesin family member 5A, is a motor protein involved in the protein cargo transportation in cells. Kinesins play an important role in transporting along a motor neuron axon, which is crucial to motor neuron health. Mutations identified in KIF5A may cause disease by interrupting that axonal transport.

Why is the KIF5A ALS Gene Important?

Identifying common pathways leading to ALS is an important part of creating new treatments to slow or reverse the progression of ALS. The more we understand about the mechanics of the disease, the closer we get to finding a cure.

ALS is a disease that’s incurable for now, but with more research we have hope that a cure will be discovered.