ALS patients’ experimental treatment gains FDA attention, but agency remains skeptical of its effectiveness

The Food and Drug Administration is set to convene this week to deliberate on the approval of an experimental treatment for Lou Gehrig’s disease, marking the culmination of a tireless lobbying campaign by patients afflicted with this fatal neurodegenerative condition.

However, despite the efforts of these advocates, they still face a significant obstacle in the form of the FDA’s skepticism regarding the efficacy of the proposed treatment.

In fact, the regulatory body has recently reiterated its long-standing position that the study conducted by Brainstorm, the manufacturer of the stem cell-based therapy in question, does not provide sufficient evidence to support the claim that it can effectively treat ALS, or amyotrophic lateral sclerosis.

Despite the hopes of those affected by this debilitating illness, it remains to be seen whether the FDA will ultimately grant approval for this experimental treatment.

The Food and Drug Administration (FDA) has been consistent in its message to company executives regarding the treatment known as NurOwn.

This message was initially delivered in early 2021 when the FDA shared data on the treatment with the executives.

The same message was reiterated in November of the same year when the FDA refused to accept the company’s application for review.

The FDA’s stance on NurOwn is clear and unwavering, and it underscores the importance of adhering to the regulatory standards and guidelines set forth by the agency.

The FDA’s refusal to accept the company’s application for review is an indication that the treatment does not meet the necessary criteria for approval, and it underscores the need for companies to conduct rigorous clinical trials and provide robust data to support their claims.

The FDA’s role in ensuring the safety and efficacy of treatments cannot be overstated, and companies must work closely with the agency to ensure that their products meet the necessary regulatory standards.

However, despite facing numerous challenges and obstacles, Brainstorm, a pharmaceutical company specializing in the treatment of ALS, made the bold decision to proceed with filing their application for approval, even in the face of protest.

This move was unprecedented and put immense pressure on the FDA to make a decision regarding the drug.

Recognizing the significant influence and authority that the FDA holds, Marc Scheineson, a former associate commissioner of the FDA who now works as a consultant for drugmakers, acknowledged the difficulty in changing the agency’s opinion once they have deemed a drug ineffective.

In the recently released documents, FDA reviewers expressed their major concerns regarding the evidence presented by Brainstorm, shedding light on the potential roadblocks the company may face in gaining approval for their drug.

Despite the challenges faced by ALS patients, there are reasons for optimism on the horizon.

Recognizing the pressing need for new treatments for ALS, the FDA has responded to the demands of the ALS community and Congress by expressing their commitment to expedite the review process and exercise regulatory flexibility.

This commitment has already resulted in the approval of two new ALS drugs within the past year, even though they did not meet the agency’s traditional approval standards.

However, the true litmus test for the FDA’s willingness to bend its rules lies in the evaluation of NurOwn, a potential breakthrough medicine for this rare and fatal condition that currently has limited treatment options available.

ALS is a devastating disease that progressively damages the nerve connections essential for basic bodily movements and functions, including the ability to breathe. Sadly, the majority of individuals diagnosed with ALS do not survive beyond five years.

In an effort to thoroughly assess the effectiveness of NurOwn, federal advisers will convene a meeting where they will hear from FDA scientists, company researchers, and patients. Ultimately, the FDA will make the final decision regarding the approval of this therapy later this year.

The convening of the meeting came about as a result of ALS advocates successfully delivering a petition with an impressive 30,000 signatures, urging for a public evaluation of the treatment in question.

Brian Wallach, one of the co-founders of the esteemed advocacy group I AM ALS, believes that even if the NurOwn treatment only offers a modest benefit to certain patients, it should still be made accessible.

Wallach, a former member of the Obama White House staff, was diagnosed with ALS in 2017, lending a personal perspective to his passionate advocacy.

Speaking through an interpreter, he emphasized the importance of not allowing the pursuit of perfection to hinder the pursuit of progress.

Wallach stressed the need for treatments that can transform ALS into a manageable chronic condition, allowing all patients to lead longer lives and, ideally, witness a cure.

However, it is worth noting that there is a lack of consensus within the typically close-knit ALS community regarding the merits of the NurOwn treatment.

Despite having provided Brainstorm, the company responsible for NurOwn, with a substantial $400,000 in research funding, the ALS Association, the largest organization in the field, has refrained from endorsing Brainstorm’s bid for approval.

It is noteworthy that Brainstorm has declined to release its complete dataset for external review, as confirmed by a spokesperson.

The controversy surrounding the approval of NurOwn, a stem cell therapy for ALS patients, has reached a boiling point, with conflicting data and emotional testimonials fueling the debate.

A group of skeptics has challenged the validity of the positive testimonials posted online, arguing that they do not match the scientific evidence presented by Brainstorm, the company behind the drug.

However, the overwhelming support from patients and their families, who have been waiting for years for a viable treatment, is expected to dominate the upcoming meeting with the FDA.

The frustration and desperation of those affected by ALS are palpable in the thousands of written comments received by the agency, with many expressing regret that NurOwn was not approved earlier, when it could have made a difference in their lives.

The therapy, which involves injecting stem cells collected from the patient’s bone marrow into the spinal column, has shown mixed results in a clinical trial involving 200 patients.

While the drug did not demonstrate a statistically significant difference compared to placebo, Brainstorm and the researchers involved in the study attribute this to the high number of patients with advanced disease who participated in the trial.

The fate of NurOwn remains uncertain, as the FDA weighs the conflicting data and the emotional pleas of those affected by ALS.

Amyotrophic lateral sclerosis (ALS), a devastating neurodegenerative disease, is commonly assessed and monitored through the utilization of a comprehensive 48-point questionnaire.

This meticulously designed questionnaire serves as a reliable tool in evaluating the progression and severity of ALS by tracking various essential functions such as walking, swallowing, and handwriting.

By encompassing a wide range of physical abilities and tasks, this questionnaire provides an encompassing assessment of the impact of ALS on the patient’s daily life and functional capabilities.

Each point within the questionnaire represents a specific aspect of bodily function, enabling healthcare professionals to obtain a detailed and comprehensive understanding of the individual’s condition.

Through the systematic evaluation of key areas affected by ALS, this questionnaire plays a crucial role in the diagnosis, treatment, and management of this debilitating disease.

Its efficacy lies in its ability to capture the nuanced changes in functional abilities over time, allowing for a more accurate assessment of disease progression and the development of tailored treatment plans.

Ultimately, the utilization of this 48-point questionnaire serves as an invaluable tool in the ongoing battle against ALS, ensuring that patients receive the necessary care and support to maintain their quality of life in the face of this challenging condition.

During a meeting scheduled for Wednesday, researchers will present their findings to the FDA, highlighting the fact that the study failed to demonstrate the effectiveness of Nurown in slowing the progression of the disease due to the rapid decline experienced by many patients.

However, they argue that when analyzing data from a small subset of healthier patients in isolation, NurOwn did show a significant slowdown in the disease. Dr. Anthony Windebank, a neurologist from the Mayo Clinic who will be presenting on behalf of Brainstorm, emphasized that experts in the field have acknowledged that there is more to this story than simply concluding that the treatment does not work. Nevertheless, FDA reviewers expressed skepticism on Monday, stating that the company’s theory does not adequately explain the study’s failed results. D

espite this, some physicians who were not involved in the research suggest that regulators may be open to a compromise by approving NurOwn for certain patients while awaiting more conclusive evidence. Dr. Terry Heiman-Patterson of Temple University, for instance, expressed the need to strike a balance between not losing a potential ALS treatment and not subjecting the public to an expensive treatment that lacks efficacy.

This potential compromise would bear similarities to the FDA’s approach with Relyvrio, another ALS drug with questionable data, which was approved while the drugmaker conducted a follow-up study that had already been initiated.

However, unlike Relyvrio, Brainstorm has not yet commenced a second study due to insufficient funding. Consequently, there are concerns among FDA observers regarding the long-term consequences if regulators continue accepting weaker evidence from drug manufacturers.

Lowering the standards could inadvertently send a message to the industry that rigorous efficacy testing is no longer necessary.

Holly Fernandez Lynch, a bioethicist at the University of Pennsylvania, warns that while it is understandable that some patients may be willing to accept this compromise, adopting such a regulatory standard could ultimately hinder progress in the field of ALS treatment.