People with ALS get a shot at experimental drug

Dec 05, 2023

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By Katherine Ellen Foley and Lauren Gardner

Presented by

With Ben Leonard and Chelsea Cirruzzo

Driving The Day

A laboratory technician handles human blood tests | Getty Images

The NIH's new program allows people with ALS to be treated with experimental therapies. | Getty Images

NIH GRANTS FOR EXPERIMENTAL THERAPIES — In early 2024, hundreds of patients with a rare, fatal neurodegenerative disease will begin receiving treatments not yet approved by the FDA, thanks to a new grant program from the National Institutes of Health.

The NIH allocated $33 million this year to academic institutions, including Massachusetts General Hospital and Columbia University, that are partnering with biopharma companies to fund expanded access programs for patients with amyotrophic lateral sclerosis, or ALS.

The disease affects about 30,000 people annually and is usually fatal within two to five years of diagnosis. The FDA-approved treatments for the disease temporarily relieve disease symptoms or moderately slow its progression. And because failures in the field have been so prominent, developers often limit clinical trial access to only a small number of patients who they think have the best shot at improvements.

The FDA’s expanded access program allows some patients to receive experimental treatments with the agency’s blessing. But the obstacle to getting the drugs has been funding at the drugmaker level; most drug development for ALS is by small biotech companies that can’t afford to provide the usually pricey doses for patients not in clinical trials.

Congress directed NIH to bridge the funding gap with the ACT for ALS, which President Joe Biden signed into law at the end of 2021. The law authorized the NIH to establish grants for expanded access programs for ALS. It also directed the NIH and the FDA to establish a public-private partnership to further support research into developing treatments for ALS and similar diseases.

The hope is that if the NIH program can work for ALS patients, it can work for other patients with rare diseases in the future.

“We are starting something that was never done before,” Dr. Sabrina Paganoni, co-director of the Massachusetts General Hospital Neurological Clinical Research Institute and recipient of some of the NIH grants, said. “You have to start by establishing the framework.

IT’S TUESDAY. WELCOME BACK TO PRESCRIPTION PULSE. 'Tis the season of holiday parties and end-of-year celebrations — which ones are you most excited to attend?

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TODAY ON OUR PULSE CHECK PODCAST, host Ben Leonard talks with POLITICO health care reporter Alice Miranda Ollstein, who explains why calls by former President Donald Trump to replace the popular Affordable Care Act, which insures more than 40 million people, could fall on deaf ears.

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Eye on the FDA

Peter Marks of the FDA speaks.

The FDA's Peter Marks said the agency has plans to help make gene therapy production more efficient. | Jim Lo Scalzo/AP

EASING GENE THERAPIES — The FDA is taking multiple approaches aimed at getting innovative gene therapies to more rare disease patients faster and helping biotechs recoup their research and development costs, a senior agency official said Monday.

Manufacturing challenges and clinical development timelines have prevented biotechs making gene therapy candidates from “quick wins,” Dr. Peter Marks, director of the agency’s Center for Biologics Evaluation and Research, said at an Alliance for a Stronger FDA meeting. The group advocates for congressional appropriations to the FDA.

The small patient pool for many gene therapies constrains drug companies’ ability to profit from their innovations. Marks said the FDA is working with other public and private partners to make developing and manufacturing gene therapies more efficient.

Agency interaction: The FDA aims to have regular discussions with gene therapy manufacturers about products in development. In January, the agency will request companies to participate in a pilot program, with CBER working on three products targeting rare diseases.

“We will give these products the same type of service that we did during [the pandemic’s] Operation Warp Speed,” Marks said.

The agency is also considering ways to collaborate with its counterparts in other high-income locations, such as in Europe, to harmonize their approaches to gene therapies, Marks said. Regulatory red tape in certain countries has kept some companies from seeking approvals, he said, which limits their market and makes it harder to recoup costs.

“You might be able to bring benefit to more people globally” and help biopharma drug developers if regulatory work didn’t have to be duplicated across borders, Marks said.

GENE THERAPY FEEDBACK — Senate HELP Committee ranking member Bill Cassidy (R-La.) will request information today on bolstering access to gene therapies, Ben reports.

The move comes as the FDA is expected to decide by the end of the week whether to approve the nation's first gene-editing therapy. Cassidy wants feedback from industry players for crafting future legislation to ensure commercial insurance can cover the pricey treatments.

What’s next: Cassidy will take comments through Jan. 22.

DRUG SHORTAGES IN THE SPOTLIGHT — Pharmaceutical supply experts and generic manufacturers will testify before the Senate Finance Committee today about drug shortages, which have struck treatments for a number of conditions, including cancer, diabetes and ADHD over the past year.

“Shortages disproportionately affect low-cost therapeutics, which operate in a largely different — and increasingly challenging — economic environment,” said ranking member Mike Crapo (R-Idaho) in opening remarks. “The average shortage affects at least half a million consumers, forcing them to scramble for viable alternatives or else forgo treatment entirely.”

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In the Courts

SCOTUS SPLIT ON PURDUE — Some members of the Supreme Court sounded unconvinced Monday by the Biden administration argument that Purdue Pharma’s multibillion-dollar bankruptcy settlement should not shield the family that owns the company from lawsuits. But the justices also seemed uneasy with protecting the family from litigation.

Why it matters: The 2022 settlement includes billions of dollars in compensation for those harmed by Purdue Pharma’s opioid pain medication, OxyContin, a restructuring of the company and immunity from future litigation for the Sackler family.

Background: The administration argues that the Sackler family immunity provision of the settlement violated the law. On Monday, however, Curtis Gannon, a deputy solicitor general representing the Department of Justice, struggled to persuade the justices that yanking immunity from the Sacklers would lead to further payout for victims of the opioid crisis — many of whom have agreed to the settlement.

Purdue’s lawyer, Pratik Shah, seized on the justices’ concerns in his argument.

Without immunity, he argued, the Sackler family will face a “tsunami” of claims, Shah said, the cost of which will reduce what’s available for the victims and states party to the settlement.

Justice Elena Kagan questioned whether the settlement had cost the Sacklers enough — but also asked the federal government whether there would ever be enough for the Sacklers to pay.

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WHAT WE'RE READING

STAT’s Jason Mast chronicles how sickle cell disease became the first target for a CRISPR gene-editing therapy.

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Document Drawer

Lisa McClain (R-Mich.), chair of the House Oversight and Accountability Subcommittee on Health Care and Financial Services, asked the FDA to provide a briefing on the efficacy of over-the-counter drugs. The request to FDA Commissioner Robert Califf on Sunday comes after the agency’s external advisory committee recently agreed that a commonly used OTC nasal decongestant is ineffective.

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