Earlier this month, Ian Lipkin, M.D., and his team at Columbia University submitted a paper for publication that suggests certain approved drugs could possibly be repurposed for a severe fatigue condition that currently has no approved treatment, while also highlighting prospective targets for new therapeutics.
About a week later, the National Institutes of Health (NIH) terminated grant funding for Columbia's Center for Solutions for ME/CFS, which stands for myalgic encephalomyelitis/chronic fatigue syndrome.
“The field of ME/CFS is ripe for clinical trials,” Lipkin, who directs the center, told Fierce Biotech in a March 19 interview. “These cuts have brought our work to a dead halt.”
The lost funds amount to about $1.2 million, plus a little more in indirect costs, according to a report from Stat. Columbia University as a whole has seen $400 million in federal funding cut off by the Trump administration, a move that is being challenged in court by the American Association of University Professors and the American Federation of Teachers.
Lipkin had been in talks with several biotechs, as well as the broader NIH-supported ME/CFS research network, to discuss working together on potential clinical trials, he said. With his funding gone, he now finds himself having to freeze hiring and lay off staff instead of accelerating clinical plans.
In 2017, the NIH announced grants to fund four centers focused on studying ME/CFS and, as Lipkin's research at the Columbia center shows, the disease's myriad potential causes.
The Columbia center's research focused on understanding the basic biology underlying ME/CFS and using that scientific understanding to suggest potential routes of action for therapeutics. An early part of Lipkin's work went toward proving that ME/CFS has a physical cause at all, he said; to this day, many physicians still incorrectly think that the disease is psychosomatic.
Guided by Lipkin, Columbia researchers have identified metabolic biomarkers and disturbances in the gut microbiome associated with ME/CFS. They have found that some cases of ME/CFS involve abnormal levels of inflammation-related molecules, while other cases don't. And, after the COVID-19 pandemic struck, Lipkin and other researchers found similarities between ME/CFS and long COVID—a disease now thought to have affected at least 400 million people worldwide so far.
Over the years, the team's understanding of ME/CFS grew and grew, Lipkin said. They found the disease is more prevalent in women than in men, especially older women, and that patients seem to have abnormalities in mitochondria and in responses to triggers of innate immunity, he said.
"As the science has matured, we've begun to see light at the end of the tunnel for these patients," Lipkin said. With the center's grant now terminated, "our ability to collaborate or participate in any sort of research right now at Columbia, it's just not there."
"We have a little bit of money to continue to write up the data we have already accumulated, but we're not getting any new data and we're not able to spend a lot of time analyzing what we have," Lipkin added. "Because, you know, we can't pay bills."
Columbia’s center is the “jewel in the crown” of the ME/CFS research network, Emily Taylor, the president and CEO of advocacy group Solve M.E., told Fierce Biotech in a March 26 interview.
The network also includes research centers at Cornell University in New York and the Sainte-Justine University Hospital Research Center in Canada, as well as a data management and coordination center housed at RTI International in North Carolina.
The funding cuts make little sense to Taylor, who says the government’s decision has left her shaking her head in disbelief. She compared it to investing millions of dollars into building a car, only to then hit the car with a sledgehammer just as it's ready to drive.
“It's just ugly that this vulnerable population and this stepping stone of progress has become a political football,” Taylor said.
When reached for comment by Fierce Biotech, a spokesperson for the Department of Health and Human Services (HHS), which oversees the NIH, did not provide a statement, but instead shared a link to the March 3 announcement that Columbia's federal grants and contracts were being reviewed for "potential violations of Title VI of the Civil Rights Act."
The HHS alleges that Columbia has failed to act against "relentless harassment of Jewish students," and thereby, the government is considering cutting $51.4 million in contracts between the university and the government, while also reviewing the more than $5 billion in federal grants to ensure the university is following federal regulations and civil rights responsibilities, according to the release.
The HHS did not respond to specific questions about Columbia's Center for Solutions for ME/CFS. The NIH did not respond to a request for comment from Fierce Biotech.
“Her own personal hell”
After Taylor’s mother came down with a virus in 2008, she never fully recovered. After four years of pain, sensitivity to light and touch, and trouble processing information, she finally received an ME/CFS diagnosis.
Patients with ME/CFS are often bed-bound, unable to seriously exert themselves lest they risk making their tiredness worse—a symptom called post-exertional malaise.
“My mom, she calls it the symptom of her own personal hell,” Taylor said. “If there is something that you love to do, this disease will punish you for it. If you like to read, you can't do that. If you like to dance, you can't do that.”
Though symptoms worsen after exertion, patients also experience an overall fatigue that doesn't improve even after sleep or relaxing. Some patients also deal with pain in their muscles or joints, or headaches.
Patients with ME/CFS often take medications off-label to manage their symptoms, Taylor said. But getting a medication off-label requires finding a doctor willing to prescribe it, and insurance companies often aren't willing to cover off-label drugs. If a drug was approved for use in ME/CFS, it would significantly improve the ability for patients to access treatment.
“You talk to any patient, and they will list 10 drugs that they have tried and three of which have maybe worked a little bit,” Taylor said. “That's a starting point of 10 drugs that we could start investigating in terms of clinical trials for efficacy in this disease space.”
Some such trials are already underway. For example, rapamycin, an immunosuppressive drug that is approved as Rapamune (sirolimus) to help transplant patients accept their new organs, is being trialed for the treatment of ME/CFS in a pilot study by Simmaron Research.
And an ongoing phase 2 study with about 160 participants is trying to nail down whether low doses of the addiction drug naltrexone can reduce fatigue in patients with the related disease long COVID.
“I truly could list at least a half dozen to a dozen different drugs that we should be exploring, and that's just the low-hanging fruit,” Taylor said. “We're not even talking about developing new therapeutics or new compounds, which also has incredible opportunity.”
But with the Columbia center’s grant canceled, most fruit may be left to rot on the vine.
“The withdrawal of funding from this Columbia center would do irreparable harm to the progress we've made in ME/CFS,” Taylor said.
A huge patient population left in limbo
The lack of approved drugs for ME/CFS is all the more surprising when you recognize how common the disease is.
“We're talking about 3 to 4 million people in the United States,” Columbia's Lipkin said, which means an approved treatment would have a large potential market. “You have people doing trials of medications for far fewer than 3 million people,” he added.
The loss of Columbia’s research center is a huge blow to the ME/CFS patient community, Taylor said, many of whom looked to Lipkin’s research as a beacon of hope. Lipkin himself recognizes the hope that research can provide to patients.
"Some of these patients, they've been living with this for many years," Lipkin said. "And they're despondent. We've lost patients not to illness, but to anguish and taking their own lives."
Taylor spoke to Fierce from Capitol Hill, where she was advocating for the restoration of funds to Columbia, along with other lost resources that can benefit ME/CFS researchers. This includes the Department of Defense’s Congressionally Directed Medical Research Programs (CDMRP), which has seen money that used to be available to researchers “zeroed out” under Trump, Taylor said.
ME/CFS has historically been included in CDMRP's Peer Reviewed Medical Research Program, which saw its funding drop from $370 million last year to $150 million this year.
Another NIH program that Solve M.E. has been watching closely, Researching COVID to Enhance Recovery or RECOVER, has also had several of its grants abruptly terminated, according to a report from Chemical & Engineering News.
With the pool of federal funds quickly drying up, Solve M.E. is doing what it can to fill the gap, Taylor said. But if money continues to evaporate, there’s no way the $2 million-a-year organization can generate enough funds to keep work going at the level the NIH used to.
“On the scale of research, there's only so much we can do,” Taylor said. “If we're looking to little organizations like us to be the salvation for this community, we have already failed.”