Swing For Life Against ALS

03/01/2026

Here’s a current overview of new and emerging treatments for amyotrophic lateral sclerosis (ALS) — a progressive neurodegenerative disease — with a focus on research progress, potential therapies, and clinical trials. ALS remains difficult to treat, but several promising approaches are advancing: 

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✅ Currently Approved and Recent Therapies

These aren’t cures but can slow progression or target specific forms of ALS:

**• Qalsody® (tofersen) — *Genetic-specific therapy
• An antisense oligonucleotide (A*O) that reduces toxic SOD1 protein production in people with SOD1 mutation ALS (about ~2% of cases). It is FDA-approved and represents the first gene-targeted therapy for ALS. 

**• Riluzole — long-used therapy
• Modestly slows disease progression by reducing glutamate-induced excitotoxicity. 

**• Edaravone (Radicava®)
• May slow decline by reducing oxidative stress; available in IV and oral forms. 

Note: Relyvrio (AMX0035) was previously approved to treat ALS but was voluntarily withdrawn from the U.S./Canada after a late-stage trial failed to meet key endpoints. 

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⭐ Emerging Treatments & Clinical Advances

🧬 1. New Drug Candidates in Clinical Trials

**• IPL344 (Immunity Pharma) — Phase 2a
• Showed significant slowing of ALS progression (~58–64% by ALSFRS-R) and reduced neurodegeneration markers. 

**• TPN-101 (Transposon) — HEALEY ALS Platform Trial
• Has shown proof-of-concept signals of slowing progression (reduced vital capacity decline and biomarkers). Now moving into Phase 2/3 testing. 

**• **Pridopidine — PREVAiLS Trial
• A new Phase 3 trial starting 2026 to test this compound in early, fast-progressing ALS. 

**• COYA-302 (Coya Therapeutics) — immunomodulation
• Targets T cell regulatory mechanisms to reduce neuroinflammation; Phase 2 expected data in 2026. 

**• NP001 (Neuvivo) — FDA submission in progress
• Aims to rebalance immune/inflammatory responses and may preserve motor and respiratory function if approved. 

🧠 2. Regenerative & Gene-Based Approaches

• Gene therapy & antisense strategies
• Building already on tofersen’s success, broader gene-editing and RNA-targeting approaches (like CRISPR and next-gen A*Os) are under early development. 

• Stem cell and regenerative medicine
• Cell therapies (including neural stem cells and iPSC-derived motor neurons) aim to protect or replace affected neurons — some early phase trials have started globally. 

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🧪 Innovative Research Tools & Platforms

Not direct treatments yet, but critical for accelerating discovery:

• HEALEY ALS Platform Trial
• Adaptive platform trial testing multiple therapies simultaneously, speeding data collection and decision-making. 

• Disease models (“ALS-on-a-chip”)
• Microfluidic chips using patient cells improve early drug screening and understanding of disease mechanisms. 

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🔎 What’s the Big Picture?

Researchers are moving beyond general neuroprotective drugs into targeted, mechanism-based therapies:
• Genetic therapies for specific mutations (e.g., SOD1)
• Immune and inflammation modulation
• Neuroprotection and cellular health
• Advanced trial designs to speed validation
• Biomarker-guided precision treatment 

This multi-pronged strategy increases the chances of finding effective treatments and eventually cures — though no definitive cure exists yet. Continued clinical trial participation and research funding are crucial.

02/17/2026

"Sandra Bullock stepped away from her film career in 2022 following the releases of The Lost City and Bullet Train, explaining to reporters that she simply needed to be home with her family. At the time, most people assumed it was just a typical break. What the public didn't know was that her partner, Bryan Randall, had been privately diagnosed with ALS in 2020, and she had devoted herself to caring for him full-time.
ALS (Amyotrophic Lateral Sclerosis) is a progressive neurological disease that gradually weakens muscles, affecting speech, mobility, and breathing. According to statements shared with People magazine in 2023, Randall chose to keep his diagnosis private from the start, and Bullock honored that wish. There were no public campaigns or interviews about hospital visits—only a small, guarded circle of family and friends who knew what was happening behind closed doors.
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By 2021, as Randall’s condition progressed, Bullock completely reorganized her life. Industry insiders noticed she was turning down scripts and postponing commitments after her promotional duties for The Lost City ended. Her choice was a deliberate move to prioritize caregiving over the spotlight. Friends noted that her daily life became centered on medical consultations, home support, and the specialized routines that ALS patients rely on.
Those close to the couple shared how deeply involved she became in the medical side of the disease. She researched treatments, worked with specialists, and did everything possible to keep Randall comfortable and independent. Caring for someone with ALS requires constant adaptation as mobility fades and daily tasks become more difficult. Bullock reportedly handled much of this herself, determined to keep his life as dignified and familiar as possible.
The couple had been together since 2015, building a quiet life away from Hollywood. Randall, a professional photographer, always valued his privacy, which shaped how they managed his illness. When Bullock told CBS Sunday Morning in 2022 that she was taking time to be present with her children and family, the comment held a much deeper meaning than anyone realized.
During her absence, there was plenty of speculation. Some suggested she was tired of her career, while others thought she was reassessing her future after massive hits like The Blind Side (2009) and Gravity (2013). The truth was much more personal; caregiving at that level is emotionally and physically exhausting, often making professional ambitions feel secondary.
In August 2023, Bryan Randall passed away at the age of 57 after his three-year battle with ALS. His family released a statement asking for privacy and thanking the medical teams who helped him. Only then did the world understand the scale of Bullock’s quiet commitment. For three years, she had built her world around appointments, adaptive equipment, and the unpredictable nature of a terminal illness.
Friends described her dedication as unwavering. They said she wanted Randall’s final years to be filled with love and comfort. Rather than seeing it as a sacrifice, they saw it as a moment of total clarity: she knew exactly where she needed to be.
Bullock has said very little publicly about the experience. Her focus has always been on honoring Randall’s wishes and protecting her children. While the industry wondered where she went, her priority never wavered. Caring for a partner with ALS takes immense patience and resilience, and she gave both freely. Her three-year withdrawal from Hollywood wasn't just a career pause; it was a deeply personal mission of loyalty and love.
"

02/09/2026

11/25/2025

https://www.tiktok.com/t/ZTMvHeh2N/

7891 likes, 311 comments. “Paula Trefiak was one of the first ALS patients to take the drug Tofersen as part of a clinical trial. The Regina resident was diagnosed with ALS in 2016 and told she only had another two to five years to live. Instead, thanks to the new drug, she's still living an activ...

08/25/2024

Pictures of the Wildwood ALS Ride for a Cure in memory of Kay Peachey, a Louisiana Girl in a New Jersey world.

02/21/2024

The ALS Town Hall, hosted by ALS TDI, is a virtual forum for that allows the ALS community to safely convene while social distancing and discuss ALS research.

02/13/2024

12/18/2023

Urge your Members of Congress to cosponsor the ALS Better Care Act (H.R. 5663 / S. 3258) today!

03/15/2023

10/15/2022

09/30/2022

ALS

FDA Approves New Treatment Option for Patients with ALS

For Immediate Release:
September 29, 2022
The U.S. Food and Drug Administration today approved Relyvrio (sodium phenylbutyrate/taurursodiol) to treat patients with amyotrophic lateral sclerosis (ALS), commonly referred to as Lou Gehrig’s disease.

“This approval provides another important treatment option for ALS, a life-threatening disease that currently has no cure,” said Billy Dunn, M.D., director of the Office of Neuroscience in the FDA’s Center for Drug Evaluation and Research. “The FDA remains committed to facilitating the development of additional ALS treatments.”

ALS is a rare disease that attacks and kills the nerve cells that control voluntary muscles. Voluntary muscles produce movements such as chewing, walking, breathing, and talking. ALS causes the nerves to lose the ability to activate specific muscles, which causes the muscles to become weak and leads to paralysis. ALS is a progressive disease that continues to get worse over time. Most cases will result in death from respiratory failure, usually within three to five years from when the symptoms first appear. Approximately 5,000 individuals in the United States are diagnosed with ALS annually, and approximately 20,000 Americans are currently living with the disease.

Relyvrio can be taken orally by combining one packet in 8 ounces of room temperature water. It can also be administered through a feeding tube. The recommended dosage for the first three weeks is one packet (3 grams sodium phenylbutyrate and 1-gram taurursodiol) daily. After three weeks, the dosage increases to one packet twice a day. The medication can be taken before a snack or meal.

The efficacy of Relyvrio for the treatment of ALS was demonstrated in a 24-week, multicenter, randomized, double-blind, placebo-controlled, parallel-group study. In the trial, 137 adult patients with ALS were randomized to receive either Relyvrio or placebo. The patients treated with Relyvrio experienced a slower rate of decline on a clinical assessment of daily functioning compared to those receiving a placebo. Additionally, longer overall survival was observed in a post hoc, long-term analysis of patients who originally received Relyvrio versus those who originally received placebo.

The most common adverse reactions experienced with Relyvrio were diarrhea, abdominal pain, nausea and upper respiratory tract infection. Relyvrio contains taurursodiol, a bile acid, which may cause worsening diarrhea in patients with disorders that interfere with bile acid circulation. These patients should consider consulting with a specialist before taking Relyvrio. The prescribing information includes additional information on risks associated with Relyvrio.

The FDA granted this application Priority Review designation. It also received orphan drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases. The FDA granted the approval of Relyvrio to Amylyx Pharmaceuticals Inc.

08/04/2022

IMPACT STORY, NEWS from the ALS ASSOCIATION
Tofersen NDA Provides Hope for Thousands and Shows Promise of Antisense Technology
Research
When the FDA recently accepted Biogen’s new drug application for tofersen, it signaled hope for thousands of people with SOD1 mutations that cause ALS.

Tofersen works by blocking the production of the SOD1 protein using a molecule called an antisense oligonucleotide. An antisense oligonucleotide is a small string of synthetic DNA that, in this case, is believed to prevent the SOD1 protein from getting made. We were early supporters of antisense research, with investments starting in 2004 and totaling more than $1.5M, and are excited that our investment has led to this and several other significant milestones in ALS.

In October 2021 Biogen announced that the phase 3 VALOR study failed to reach it’s primary endpoint, though there were some signs of reduced disease progression. Then, in June 2022, Biogen released results from a follow-on open label extension study which showed that earlier use of tofersen slowed declines in clinical function, respiratory function, muscle strength and quality of life compared to people who originally were assigned to a placebo but who began taking tofersen several months later during the open label extension study.

The FDA has granted priority review to this new drug application and is expected to decide on whether or not to approve tofersen by January 25, 2023.

July 28, 2022
ARTICLE TOPIC
Research
SCIENTIFIC FOCUS AREA
Drug Development