ALS Texas

ALS Drug Shows Further Promise, Slowing Progression and Increasing Survivability

by Leave a Comment

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

by 3 Comments

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

by Leave a Comment

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

by 2 Comments

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

by Leave a Comment

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

by 64 Comments

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

by 2 Comments

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

by 1 Comment

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.

Serving in the Military Poses 60% Higher Risk for ALS

by 1 Comment

For decades, researchers have been searching for correlations that may give insight into what causes ALS. While the direct cause remains elusive, there have been interesting findings over the years that could possibly bring us closer to a cure.

The ALS Association has released a new report highlighting the prevalence of ALS among military service members. In the 2019 ALS in the Military report, it was found that those who have served in the military are at a nearly 60% greater risk of developing ALS than those who have no history of military service. In addition, the data shows that ALS is connected to military service regardless of the branch of service or whether serving during peacetime or in war.

The report compiled findings from research conducted from the early 1990’s up until recent years with one of these studies examining  military members going back as far as 1910.  Finally in 2008, after many studies and decades of research,  the Department of Veterans Affairs established ALS as a service-connected disease. Since then, the average number of veterans living with ALS has increased by a staggering 30 percent.

“Study after study has shown that our military heroes are more likely to develop ALS, which is why it is critical that Congress fully funds the ALS Research Program at the Department of Defense, as well as research at the National Institutes of Health,” said Calaneet Balas, president and CEO of The ALS Association.

We’ve made strides and progress but we need more support to fund research that could help both veterans and non-veterans alike. ALS is a devastating disease but with your help, we’re well on our way to a deeper understanding and possibility of a cure.

ALS Association Awards $1 Million to Fund Potential ALS Treatment

by 2 Comments

Today marks a monumental day for ALS research as the first in-human clinical trials of AT-1501 have begun. AT-1501 is an antibody therapeutic developed by the ALS Therapy Development Institute (ALS TDI), with support from The ALS Association, ALS ONE, and ALS Finding a Cure®. The drug blocks immune cell activation and protects nerves against the progression of ALS. As of today researchers at Anelixis Therapeutics, lead by Dr. Steve Perrin, have successfully administered a dose of AT-1501 to the study’s first participant.

This is a great progressive step on our journey for a cure, and we’re happy to announce that the ALS Association awarded an additional $1 million to Anelixis to help fund this groundbreaking research.

“We are proud to partner with Anelixis and other ALS organizations to help advance this promising compound into clinical trials,” said Calaneet Balas, President and CEO of The ALS Association.

The safety trial of this potentially new treatment is currently enrolling volunteers, both those with ALS and those without, to participate in the study. In total, eight people with ALS will receive AT-1501 and will be monitored for safety , tolerability, and how the drug moves through the body. If it’s proven safe the trial will then move on to Phase II.

We’re excited for the prospect of this new treatment and we remain hopeful that together with patients, caretakers, donors, and researchers we can create a world without ALS.

Help us create a world without ALS by donating today.