Montana Opens Experimental Drug Access Through Privately Run Review Board

Montana has begun implementing a state law that expands access to experimental treatments far beyond the FDA's framework, relying on a privately operated review board to decide which unapproved therapies may be sold to patients — including those who do not have life-threatening illnesses.

The law, which took effect in July, was drafted with input from investors on the libertarian fringe of the biotech industry, including Niklas Anzinger, a 37-year-old German venture capitalist and founder of Infinita City Inc. (搜索)搜索该词条 Anzinger's company operates the only review board established so far, the five-member Montana Experimental Treatment Review Board (搜索)搜索该词条, which charges companies a $12,500 application fee to sell experimental treatments in the state.

First Approval Targets Hearing Loss

Last month the board authorized its first treatment: a 30-day regimen from biotech company Parley Neurotech (搜索)搜索该词条 that combines an off-label, FDA-approved antihistamine taken daily with a proprietary sound mix delivered for 60 minutes a day, aimed at reversing hearing loss (搜索)搜索该疾病. The treatment has not been cleared by the FDA and was not reviewed by the state.

Patients cannot yet receive it because Montana has not issued a license to an experimental treatment center. The state health department said two applications for a center are pending completion.

Sam Budoff, Parley's CEO, said sales through the Montana program would help fund future clinical trials and give access to patients who would not qualify for those trials. He declined to share the cost of treatment but said it would reflect out-of-pocket prices in the hearing aid market. He described the law as the "right balance of regulation" and said he was confident the Phase 1 requirement and review board structure would keep out bad actors.

A Lower Evidence Bar Than the FDA

Under Montana's rules, any experimental review board with five members — including a Montana-licensed physician, a researcher with expertise in clinical outcome data, and an ethicist — can approve treatments. Unlike Institutional Review Boards used by pharmaceutical companies and academic medical centers to oversee clinical trials, Montana's boards are not supervised by the FDA.

Montana allows access to therapies that have passed small Phase 1 trials establishing that a product probably will not kill patients, not that it works. FDA rules limit experimental treatments to people with serious conditions, whereas Montana requires only that patients give informed consent, have the money to pay, and obtain a provider's recommendation. More than 40 US states have adopted measures to increase access to therapies not approved by the FDA, and two federal programs also allow some patients to access unapproved treatments, but Montana's law permits a much broader patient population.

Ethics and Conflict-of-Interest Concerns

The industry's role in administering the program has drawn scrutiny. Arthur Caplan, former head of the division of medical ethics at NYU Grossman School of Medicine, called it a "risky endeavor" and said the program is closer to a prediction market than medical research. "This thing they set up in Montana is really a program to facilitate access to anybody and everybody who wants it, to novel experimental interventions for anything," he said. "It's like, you want to gamble? OK, go ahead."

The law barely passed the state legislature last year amid intense opposition. The Alliance for Longevity Initiatives (搜索)搜索该词条, a Virginia-based biotech industry group, helped draft the legislation along with Anzinger, who is a member of the alliance, according to the alliance and the bill's sponsor, Republican state senator Kenneth Bogner. Anzinger said Bogner, the state Senate president pro tempore, invited him to Montana and asked what the law needed to look like to attract biotech companies. Bogner said Anzinger "came out to Montana to learn how to make Montana a future home" and described the longevity law as a test case he hopes will "spur changes to the U.S. health care system that, in my opinion, isn't sustainable."

The review board's chair is longevity scientist Matt Kaeberlein, who also serves on the board of directors of the Alliance for Longevity Initiatives (搜索)搜索该词条, a volunteer role from which he said he planned to resign soon. Kaeberlein said the Montana review board "has no financial incentive in whether a determination is yes or no." Dylan Livingston, founder of the alliance, said the group has no financial stake in any review board or treatment center, that board members are unpaid, and that their outside roles are independent of their alliance work.

Stephen Martin, who leads US initiatives at Infinita and handles intake for the Montana review board, said board members must disclose conflicts each time they review an application, and that no conflicts have occurred yet. Infinita cannot direct or overrule board decisions under the board's rules, he said. "If you think we are not doing a good job at oversight, come compete against us, do it better, and put us out of business," Martin said.

Anzinger told Business Insider that none of Infinita VC's portfolio companies — which include Minicircle (搜索)搜索该词条, which sells a gene therapy intended to increase muscle mass, and Unlimited Bio (搜索)搜索该词条, which is testing a gene therapy for circulation, endurance and hair growth — have submitted an application to the review board or indicated plans to do so. The fund has no plans for further investments, he said.

Board Members Cite Longevity Field's 'Snake Oil'

Some board members said they joined to broaden access to investigational drugs beyond one-off compassionate use cases; others said they wanted to bring rigor to a field plagued by risky, unproven longevity treatments. As injectable peptides have grown in popularity, so have peptide injuries, according to the report.

"The field of longevity is plagued right now by snake oil," said board member Filipe Sierra, a former director of the Division of Aging Biology at the National Institute on Aging. He said trials in experimental treatment clinics could generate efficacy data on antiaging therapies, which can be challenging to run through the FDA's normal clinical trial pipelines because the agency considers aging a natural process rather than a disease with defined clinical endpoints.

Jamie Justice, who holds a PhD in neurophysiology and the physiology of aging and is executive director of a competition that awards money for longevity startups, said she joined the board to help Montana's initiative succeed and to ensure there were "some adults at the table" when ethically thorny questions arose. "There are a million ways that this could fail and only a few that could go right," she said. "And so I think part of it really is taking our job seriously and thinking about the safety."

FDA Enforcement Uncertainty Clouds the Program

The Montana experiment hinges on whether the Trump administration's FDA chooses to take enforcement action against participating companies. In June, a group including Infinita, patient advocates and biotech companies met with FDA officials to request a guarantee that the agency would not act against them in Montana; they said they have not received a direct answer. An FDA spokesperson declined to comment.

The biotech industry has reason for optimism, according to the report: Health and Human Services Secretary Robert F. Kennedy Jr. has pledged to increase access to longevity treatments such as stem cell therapies, peptides and chelation, and last year said he had traveled to Antigua to receive a non-FDA-approved stem cell treatment for a neurological condition affecting his voice.

Under the FDA's standard pathway, approval typically requires three phases of clinical trials, including assessments of efficacy, dosage and adverse events. Its compassionate-use program grants requests for unapproved treatments for patients with severe or life-threatening diseases, and patients can also bypass the FDA under the federal right-to-try law enacted in Trump's first term. Few drugs have been used through that program, and companies are not allowed to make a profit under either program.

Kaeberlein said the existing federal right-to-try framework "while on paper, can work, the reality is many companies are hesitant to use it, and so it's not happening." He added: "Is this the perfect solution? Maybe. Probably not perfect. Is it a possible improvement? I think it is."

Montana's law does require some oversight: the board must publicly report treatment approvals and safety outcomes at least once a year, and the state health department will confirm that each experimental treatment center contracts with a review board complying with Montana's rules.

Infinita and the Alliance for Longevity Initiatives (搜索)搜索该词条 are now pushing for similar legislation in New Hampshire. Without assurance that the FDA will permit companies to participate in Montana's program, some patients say they may still have to seek experimental treatment abroad. Kris DeVault, a parent of a 3-year-old son diagnosed with an ultra-rare genetic disease (搜索)搜索该疾病 that has no treatment, wants to travel to Montana for an experimental treatment for his son, but the manufacturer is wary of a possible FDA shutdown. He is now exploring going to Próspera (搜索)搜索该词条, the autonomous private city in Honduras where Anzinger's Infinita helped create a biotech hub and where anti-aging startups can administer experimental treatments without government oversight or evidence of safety. Próspera, whose investors include Peter Thiel and Marc Andreessen, drew attention last year when longevity influencer Bryan Johnson was filmed receiving a $25,000 gene therapy treatment there in the Netflix documentary "Don't Die."