ALS Texas

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.

Serving in the Military Poses 60% Higher Risk for ALS

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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

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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.

Frequently Asked Questions about Radicava™ (Edaravone)

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Based on information as of 5/5/2017

What is Radicava?

Radicava™ (edaravone) is a prescription medicine approved by the U.S. Food and Drug Administration (FDA) to treat people with amyotrophic lateral sclerosis (ALS).1

In clinical trials, some people given Radicava showed significantly less decline in physical function compared to placebo as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R), a validated rating instrument for monitoring the progression of disability in patients with ALS.1,2

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FDA Approves New ALS Drug

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Radicava is First Approved Treatment for ALS in Decades

The Food and Drug Administration’s (FDA) announced today that it has approved Radicava (edaravone), the first new treatment approved specifically for ALS in 22 years. The FDA approved Radicava less than a year after Mitsubishi Tanabe Pharma Corporation submitted a New Drug Application. The only other approved treatment specifically for ALS, riluzole, was approved in 1995.

“We thank the FDA and MT Pharma for working together to expedite the approval of the first new ALS-specific treatment in decades,” said Barbara Newhouse, president and CEO of The ALS Association. “We hope today’s announcement signals the beginning of a new chapter in the fight against this terrible disease. There are several drugs to treat ALS currently in clinical trials and we are hopeful that people living with ALS have even more therapies available to them sooner rather than later.”

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New Gene Discovered that Contributes to ALS

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$1 Million Grant from Ice Bucket Challenge Helped Spur Discovery

Today, researchers from Project MinE announced that they have identified a new gene NEK1 that ranks among the most common genes that contribute to ALS. The study revealed an association between mutations in the gene and ALS. The discovery of NEK1 gives scientists an exciting new target for drug development.

Approximately 10 percent of ALS cases are familial, meaning genes are inherited from a family member. The other 90 percent of ALS cases are sporadic, or without a family history. The NEK1 gene is present in approximately 3 percent of all ALS cases.

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Congress Takes Action on ALS Priorities

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Thanks to nationwide advocacy efforts, Congress is taking action on several priorities for the ALS Community.

The Senate Appropriations Committee passed the fiscal year 2017 Health and Human Services spending bill and included $10 million to continue funding to the National ALS Registry. The National ALS Registry is a congressionally directed registry for people in the U.S. with ALS. It is a program to collect, manage, and analyze data about people with living with ALS, which helps doctors and scientists learn more as they work toward a cure. It is the only population-based registry in the U.S.

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FDA Approval Sought for New ALS Drug

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Pharmaceutical company Mitsubishi Tanabe Pharma Corporation submitted a formal proposal to the United States Food and Drug Administration for approval of a drug to treat ALS. The drug, Edaravone, gets rid of toxic waste that is a normal by-product of cell function. It is thought that this waste is not as effectively removed in ALS patients, causing damage to motor neurons, which are the cells that die in ALS patients. Edaravone would protect these neurons by helping rid the cells of this waste.

The Edaravone new drug application is supported by clinical research with ALS patients in Japan, and in 2015, Edaravone was approved as a treatment for ALS in Japan and South Korea. After being submitted to the FDA for consideration, it could either be automatically approved for use in the U.S., or it could require a Phase III study conducted in the U.S.

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