Die Trying - The Battle For ALS Treatment (VICE on HBO: Season 4, Episode 16)

Summary

The Vice documentary explores the urgent search for an ALS cure, highlighting the personal stories of patients like Beth and Matt, who face the devastating progression of the disease. It delves into the challenges of clinical trials, the FDA's cautious drug approval process, and the desperate measures patients take, such as seeking experimental treatments abroad. The documentary underscores the need for a reevaluation of drug approval protocols to expedite access to potential treatments and give hope to those battling this fatal disease.

Takeaways

• 🕒 The urgency of finding a cure for ALS is underscored by the fact that patients are losing their abilities rapidly, and time is of the essence.
• 💉 Despite the Ice Bucket Challenge raising over 220 million dollars for ALS research, no one in the U.S. is receiving the experimental drug discussed in the script due to FDA safety concerns.
• 📉 The disease progresses quickly, leading to unrelenting muscle loss and eventual paralysis, while cognitive function remains largely intact.
• 🧬 ALS patients face significant barriers to accessing experimental treatments, with clinical trials being the primary way to access potential drugs, but strict eligibility criteria often exclude patients.
• 🌐 Some patients are forced to travel abroad to access experimental treatments, highlighting the limitations of the U.S. healthcare system in providing cutting-edge care for ALS.
• 🔬 Research centers like the ALS Therapy Development Institute are working tirelessly to understand the disease and find treatments, focusing on pharmacology and testing various compounds.
• 💼 The FDA's cautious approach to drug approval can be detrimental to ALS patients who are willing to take risks on unproven treatments in the hope of slowing or stopping their disease.
• 💊 Even when effective treatments are found, the high cost and regulatory hurdles can prevent patients from accessing them, as illustrated by the story of a drug company unable to provide their treatment in the U.S. due to financial constraints.
• 🌟 The potential for ALS research to benefit other neurodegenerative diseases like Alzheimer's, Parkinson's, and Huntington's is highlighted, emphasizing the broader implications of finding a cure.
• 🚫 The script calls for a reevaluation of the FDA's drug approval process for diseases with no viable treatment, advocating for a more patient-centered approach that acknowledges the desperate need for solutions.

Q & A

What is the primary focus of the documentary 'Vice' this week?

The primary focus of the documentary 'Vice' this week is the search for a cure for ALS (Amyotrophic Lateral Sclerosis) and the challenges faced by patients and researchers in finding effective treatments.

How much money was raised for ALS research through the ice bucket challenge in 2014?

The ice bucket challenge raised more than 220 million dollars for ALS research in 2014.

What criticism did the ice bucket challenge face?

Critics of the ice bucket challenge dismissed it as viral narcissism, suggesting that people were more interested in showing off than actually helping the cause.

What is the current situation regarding the availability of the drug discussed in the script in the U.S.?

The drug discussed in the script is not available in the U.S. due to FDA regulations and concerns about safety. No one in the U.S. is receiving the drug at the moment.

What is the role of the FDA in the approval process for drugs like those for ALS?

The FDA is responsible for ensuring the safety and efficacy of drugs before they are released to the public. It has a standard drug approval process that can be a contentious issue for terminally ill patients who are willing to accept risks associated with unapproved drugs.

What is the significance of the ALS Therapy Development Institute mentioned in the script?

The ALS Therapy Development Institute is the world's first research center solely focused on ALS. It plays a crucial role in researching and developing potential treatments for the disease.

What is the current status of clinical trials for ALS treatments?

Clinical trials for ALS treatments are ongoing, but they are limited in scope and often have strict exclusion criteria, making it difficult for many patients to participate.

Why do some ALS patients travel abroad for treatment?

Some ALS patients travel abroad for treatment because they are unable to access experimental treatments or clinical trials in the U.S. due to strict regulations and limited availability.

What is the 'expanded access' program mentioned in the script?

The 'expanded access' program is an FDA initiative that allows patients to try unapproved drugs. However, the process can be lengthy and may not be feasible for ALS patients who deteriorate rapidly.

What is the potential impact of changing the drug approval process for ALS as suggested by Dr. Andrew Lo?

Changing the drug approval process for ALS, as suggested by Dr. Andrew Lo, could lead to faster approvals and more trials, potentially increasing access to treatments and accelerating the development of cures for ALS and other progressive neurological diseases.

What is the current reality for ALS patients who are severely disabled, as depicted in the script?

The current reality for severely disabled ALS patients is grim. They often require 24/7 care, face the decision of whether to get a tracheotomy and use a ventilator, and live with the constant threat of choking or suffocating due to their inability to move or communicate effectively.