In the face of adversity, Nancy Poon, a lifelong runner, finds solace in embracing each day with gratitude. Diagnosed with amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease, in December 2018, she has learned to cherish the simple joys in life. Despite the challenges posed by this devastating illness, Nancy’s resilient spirit shines through as she navigates her journey with optimism. In this article, we explore the positive developments in ALS treatment, shedding light on the progress being made to extend and improve the lives of ALS patients.
ALS Treatment Breakthroughs
ALS patients, including Nancy Poon, have reason to be hopeful as advancements in treatment offer glimpses of a brighter future. Several positive developments have recently emerged:
1. Prolonged Survival with a New Drug
Thanks to funds raised during the 2014 Ice Bucket Challenge, a new drug has been developed that shows promising results. In a clinical trial, this drug increased the average survival of patients by an impressive seven and a half months.
2. Innovative Clinical Trials
For the first time, a clinical trial is being conducted to test multiple ALS treatments simultaneously. This innovative approach aims to expedite the discovery of effective therapies for this debilitating disease.
3. Increased Research Funding
Record levels of federal research funding have been allocated to ALS studies this year. This financial support fuels ongoing research efforts, fostering progress in understanding and treating ALS.
Amylyx’s AMX0035: A Ray of Hope
One drug, AMX0035, has shown remarkable potential in prolonging the lives of ALS patients. Developed with a $2.2 million grant from the Ice Bucket Challenge, AMX0035 has extended patients’ survival by seven months—a significant increase considering the average life expectancy of two to five years for ALS patients.
Encouraged by the promising results of AMX0035’s Phase 2 trial, which assesses a drug’s efficacy and side effects, the ALS community is urging the Food and Drug Administration (FDA) to fast-track its approval. The ALS Association has even requested that a Phase 3 trial be skipped—an unprecedented move that underscores the urgent need for effective ALS treatments. However, it is important to note that the FDA typically requires completion of a large Phase 3 trial before approving drugs, making the path to fast-track approval challenging.
A Push for Faster Drug Approval
Recognizing the urgency of the situation, the FDA has prioritized ALS treatment. The agency has the authority to expedite drug approvals, especially in cases where there is a substantial unmet need and compelling evidence of efficacy and safety. While specific drugs cannot be commented on, discussions are underway between Amylyx and the FDA regarding paths to approval and expanded access to AMX0035.
Balancing Speed and Solid Evidence
Despite the pressure to accelerate drug approval, it is crucial to adhere to the rigorous standards of large Phase 3 trials. These trials play a vital role in identifying potential safety concerns and verifying the efficacy of a drug. Rushing into using new drugs without solid evidence can have unintended consequences, as demonstrated by past instances in which preliminary treatments failed to deliver the expected benefits.
Dr. Joel Lexchin, a health policy expert, emphasizes the value of robust evidence before embracing new drugs. He cites cautionary examples where treatments were widely adopted based on preliminary data but ultimately proved ineffective or even harmful. While the desire for immediate breakthroughs is understandable, thorough evaluation is essential to ensure patient safety.
ALS Research Acceleration
The landscape of ALS research has evolved significantly in recent years, with an estimated 80 ongoing clinical trials worldwide. This surge in research activity marks a substantial increase compared to the number of completed, terminated, or suspended ALS trials from 2007 to 2018. Notably, only one treatment, Edaravone, received FDA approval during this period to slow ALS progression.
Stacy Lindborg, executive vice president of Brainstorm Cell Therapeutics, has a personal connection to ALS, having witnessed her uncle’s suffering and ultimate demise from the disease. She highlights the relentless and brutal nature of ALS, where patients remain mentally aware while their bodies progressively deteriorate. Despite the challenges, she echoes the sentiment that the ALS community, with its tireless efforts and advocacy, has spurred hope for progress in neurodegenerative diseases.
The Unyielding Spirit of Nancy Poon
Nancy Poon, deeply inspired by neurosurgeon Paul Kalanithi’s memoir “When Breath Becomes Air,” embarked on her ALS journey after retiring from a fulfilling career as an elementary school teacher. Determined to make the most of her time, Nancy immersed herself in activities and experiences that brought her joy. Although her retirement plans took an unexpected turn, she remains grateful for the love and support she receives from her friends and family.
A Glimpse of Hope
The ALS community, researchers, and regulatory authorities are collaborating to accelerate ALS research and bring effective treatments to the forefront. While challenges persist, the dedication and determination of those involved instill hope for a brighter future. As Nancy Poon continues her daily battle against ALS, she serves as a testament to the strength and resilience of the human spirit in the face of adversity.