The monkeypox drug paradox
U.S. says it has a huge stockpile of Tpoxx, but it’s hard to get
Joseph Cassara decided to head back to the emergency room when the pain made it unbearable to swallow.
“I tried to eat a banana for breakfast,” he said, “and it just felt like I was swallowing razor blades.”
Cassara, 33, was still waiting on the test results to confirm that he had monkeypox, but was suffering scorching fevers and gruesome lesions on his cheek and chin that felt as if they were on fire. The Fresno resident was taking ibuprofen and Tylenol, but the agony persisted; at one point, he said, he woke at 2 a.m. with his fists and jaw clenched, drenched in sweat, and screamed at the ceiling in pain.
He had been hospitalized and put on an IV, he said, when the doctor told him she was trying to get him Tpoxx, a drug not yet approved by the Food and Drug Administration to treat monkeypox. Cassara read through the paperwork and agreed.
Ten days into his illness, the drug arrived and the result seemed “miraculous,” Cassara said. Little lesions that had started to spread to his body seemed to freeze their onward march and then scabbed over, he said. The wounds on his face began to shrink. As Cassara began to recover, he wondered:
Why had it taken so long to get the medicine that finally eased his suffering?
Tecovirimat, commonly known as Tpoxx, is authorized in Europe and is an approved treatment in the U.S. for smallpox, but the FDA has not yet approved it to treat monkeypox. It can be provided either as oral capsules or injection vials for IV treatment.
Physicians can request it from the Centers for Disease Control and Prevention or state health departments for monkeypox patients under “compassionate use” — a pathway used to offer new drugs to patients with serious conditions that lack comparable alternatives. Doctors have reported that the process, which includes filling out paperwork, obtaining informed consent and reporting back to the CDC on how the patient is faring, can be daunting.
Federal officials say there are about 1.7 million courses of Tpoxx in a national stockpile — “more than enough to handle the need,” Health and Human Services Secretary Xavier Becerra told reporters when the Biden administration declared monkeypox a national public health emergency. Becerra said more than 15,000 of them had been deployed as of this week.
In California, health officials said nearly 1,900 courses of Tpoxx capsules and 182 intravenous doses had been distributed across the state as of Monday. Yet for many Californians suffering from monkeypox, an infectious disease that has taken a disproportionate toll on gay and bisexual men, getting the drug has not been simple.
In San Francisco, David Norman said his medical provider told him “we don’t have it.”
He was instead prescribed the pain medication gabapentin and sent home to isolate. The medicine “didn’t come anywhere close to touching the pain,” said Norman, 36. The painful bumps made it impossible to sit or stand up straight. He struggled to sleep.
It was through online networks that Norman found out that San Francisco City Clinic was providing Tpoxx to people suffering from the illness. Norman hobbled for blocks to the clinic and met with a nurse practitioner who walked him through the paperwork, then gave him the medicine.
It was “an absolute lifesaver,” said Norman, a human resources professional. “The very next day my bumps were all retreating.”
Thwarting the rash could also help people make a swifter exit from isolation, which can stretch for weeks as patients wait for their lesions to scab and heal over.
“This medication has been shown, so far, to accelerate the healing of the lesions,” said Michael Bosco, an infectious-disease pharmacist at NYU Langone Hospital–Long Island who has argued in favor of broader access to the treatment. If clinicians have this option, he said, “we’re doing a disservice if we’re not helping people get access to that.”
In Los Angeles County, the public health department recently said it had stocked more than 600 doses of Tpoxx at 30 “treatment hubs” across the county, including hospitals and community sites. It is also dispensing medication directly to patients’ homes, a service it said was open to any medical provider willing to follow the CDC protocol — including following up with their patient.
Yet early in August, the head of a nonprofit system of community clinics said they had not given Tpoxx to any monkeypox patients. Until recently, “we haven’t tried to access it because we haven’t even known that it was available,” Jim Mangia, president and chief executive of St. John’s Community Health, said in an interview last week.
At the time, Mangia said the clinics had encountered at least four cases of monkeypox and had told patients to isolate and take ibuprofen, which is “kind of like putting a Band-Aid on a huge open wound,” he said. The following day, Mangia reported that after reaching out to the county, the clinics had received 10 doses of Tpoxx for monkeypox patients.
Although it is not FDAapproved for monkeypox, Tpoxx has been approved for treating smallpox, thanks in part to its success in quashing monkeypox in primates. Because researchers could not ethically test whether it stopped smallpox in humans, they instead tested whether it could treat similar viruses in macaques and rabbits.
“This drug was approved for smallpox for an emergency use with a disease with a mortality of 30[%] to 50%,” based on animal testing, but had not been tested in people with disease, FDA Commissioner Dr. Robert Califf told reporters last week. “We think it’s going to be effective in monkeypox, but we have no proof of that yet.”
Some studies have been promising. In Massachusetts, physicians reported that when three patients were given Tpoxx, their lesions improved and their illness did not become severe, without any serious side effects.
Local physicians said they have seen encouraging results. “It’s very effective at curbing the pox outbreak almost immediately,” said Dr. Anthony Mills, chief executive and founder of Men’s Health Foundation in L.A. County, who said he had six patients taking the drug for monkeypox.
Dr. Graham Walker, an emergency physician in San Francisco who has treated monkeypox patients, said that “if I had monkeypox or if my husband did, I would absolutely be doing everything in my power to get Tpoxx.”
“We’ve already screwed up this monkeypox pandemic,” Walker said. “Why are we not using this?”
Califf told reporters asking about Tpoxx: “Make no mistake — our goal is to make it available without excess bureaucracy and paperwork. But it’s critical that we collect data because we don’t know about the risk of the drug or ... how effective it will be.” The FDA commissioner said a clinical trial was being planned to pave the way for its full approval.
The California Department of Public Health did not immediately provide a list of locations providing Tpoxx. Some California patients have been frustrated by obstacles to getting the treatment: Another San Francisco resident, who asked not to be identified to protect the privacy of his partner, said that when his partner went to Zuckerberg San Francisco General Hospital, he was repeatedly told it had no Tpoxx and “there was nothing they could do to help him.”
After he and his partner persisted, the ill man was eventually admitted and given other medications but was told that a particular doctor would have to assess him for Tpoxx because obtaining the drug “would take two hours of paperwork,” the San Francisco resident said. His partner ultimately went to San Francisco City Clinic, where he was quickly provided the medication, the man said.
The San Francisco resident said he feared that the free clinic would be inundated with insured patients turned away from other providers. Both Zuckerberg San Francisco General and San Francisco City Clinic are operated under the San Francisco Department of Public Health, which said it could not comment on the experience of an individual patient, but “currently, the number of providers in San Francisco prescribing Tpoxx is limited due to the burdensome federal regulatory process required for clearance and approval.”
Among the sites now prescribing it is Zuckerberg, the department said.
In Los Angeles, another patient said that when he went to the emergency room at Cedars-Sinai, a nurse told him after hours of waiting that “I just want you to know that you’re not going to get Tpoxx.”
The next day, after a test came back positive for monkeypox, he got a phone call that his primary care provider at Cedars was seeking Tpoxx for him, the man said. But his fear is that other patients who turn up at the ER in pain — and don’t have a doctor for primary care — will never get the medication.
In L.A., “nobody knows what facility is offering it,” said the man, who did not want to be named because of concerns about how his employer might respond. (The L.A. County Department of Public Health said that a list of its Tpoxx sites was “not publicly available,” but that patients and providers could get help accessing the medication through its call center.)
Cedars-Sinai spokesperson Marni Usheroff said the medical center could not comment on the experience of any individual patient for privacy reasons, but emphasized that the CDC requires patients who receive the investigational drug to get follow-up visits, and “emergency departments are not set up to offer this type of follow-up care.”
Usheroff said that Cedars-Sinai has given Tpoxx to “a handful of patients” and is working to make it as easy as possible for eligible patients to receive it.
Like many of the Californians who have been hit by the virus, Cassara is gay, and when others argue that “we don’t need to worry about monkeypox,” he said, “that just makes me feel like gay men are not part of the ‘we.’ ” He believes he got the virus from kissing, because his blisters clustered above the neck, and worries about whether his face will bear lasting scars.
If he had gotten Tpoxx earlier, he said, maybe his pain and anxiety could have been avoided. Vaccines for monkeypox are in short supply, “but we do have the Tpoxx,” Cassara said. “So why can’t they start giving this to people?”