When Kyle Planck got monkeypox, he was in the worst pain of his life. "I was at a seven or eight [out of 10] of the worst pain I could ever imagine, and it came and went throughout the day," he says, "especially because it was an internal sort of pain – it was very hard to deal with."

In early July, the infectious diseases graduate student in New York City had pustules in different stages on his body. He spent several days bedridden. Then Planck got access to tecovirimat – brand name TPOXX – a two-week course of antiviral pills. Within two days of taking them, he noticed improvements: "Some of the pustules were actually shrinking in size, and some of them just disappeared back into my skin. They didn't go through the normal progression that the lesions usually do," he says.

According to advocates in the LGBTQ community, Planck is one of the lucky few patients to get access to TPOXX. So far, around 215 patients have received TPOXX in New York City, according to a city health official on July 23.

It's not clear how many courses have been prescribed nationwide. The government's strategic national stockpile holds 1.7 million TPOXX courses. So far, 10,000 courses have been sent to states and cities on request – but "the number of [courses] deployed does not necessarily equate the number of [courses] administered," a spokesperson from the Department of Health and Human Services wrote in an email to NPR. HHS said the Centers for Disease Control and Prevention keeps track of courses administered, but the CDC referred NPR back to HHS, which suggested that NPR check with individual jurisdictions.

A drug for smallpox, tested against monkeypox

TPOXX is made by the New York-based pharmaceutical company SIGA. "The drug was developed in partnership with the U.S. government post-9/11, [to address] concerns about a [potential] smallpox attack," says SIGA CEO Phil Gomez. Smallpox, a much deadlier cousin to monkeypox, was eradicated as a human disease more than 40 years ago, but labs in the U.S. and Russia have samples, and it's considered a bioterror threat.

"Given the nature of how it was developed, we only sell to governments," Gomez says, "The intent has always been for this to be stockpiled because if there's a [smallpox] outbreak, it's too late to start ordering the drug."

SIGA proved that TPOXX worked against smallpox-like viruses by testing it on two animal models: monkeypox in monkeys, and rabbitpox in rabbits. And they proved that it was safe in humans by giving it to a few hundred healthy people in a safety trial and recording their side effects (headaches and nausea were most common). In 2018, the FDA approved the drug through the agency's "Animal Rule," which allows companies to use animal studies "to support an FDA approval when it is not feasible or ethical to conduct efficacy trials in humans."

The drug works across smallpox, monkeypox, and other related poxes because it blocks a protein these viruses need to reproduce. "It decreases virulence in the body tremendously, because even though some cells get infected they can't infect the next cells and spread it," says Rachel Roper, a professor of microbiology and immunology at East Carolina University who has worked on poxviruses for 30 years.

But even though TPOXX worked against monkeypox in monkeys, the FDA only approved its use for smallpox in humans. For monkeypox, the drug is classified as an "investigational new drug," allowed for compassionate use, which comes with requirements from the FDA and CDC. That has created barriers to access for doctors and patients in the current monkeypox outbreak.

Less paperwork, but some forms still required

In New York City, Dr. Robert Pitts, an infectious disease specialist at NYU Langone Health, has prescribed TPOXX to a dozen patients. "Every time I send a TPOXX prescription, I'm very cognizant that this is what the patient needs," he says. Patients felt better after taking the drug, and weren't too bothered with side effects. Still, the administrative paperwork was daunting. In early July, the protocol took up to three or four hours per patient to complete.

On July 22, the CDC updated its TPOXX protocol, streamlining the process to make the drug far easier to prescribe. Prescribers still go through their local health departments or the CDC to request the drug, but many of the forms are shorter, and some previously required documentation – including photos of lesions and patient diaries – are now optional. "Really, this has been substantially improved," says Dr. Melanie Thompson, a physician and HIV researcher in Atlanta, who prescribed TPOXX to a patient under the new requirements. "It took me about 15 minutes to do all of the TPOXX paperwork, and another 15 minutes for the informed consent for the patient."

The changes came after strong advocacy from clinicians and activists. "There were a lot of health care providers who spoke directly to CDC, advocates wrote letters and lobbied for these changes," Thompson says, "and CDC recognized that something had to be done."

Pitts agrees that the new process is a vast improvement, but says "it's still a significant amount of additional work to put patients on the treatment." He knows that the drug hasn't been used in a lot of people before, and that the rules are meant to protect patients. Still, he hears daily from patients who are struggling to find providers willing to prescribe the drug.

Seeing that lack of access has turned patients like Planck into advocates. From isolation, he wrote letters to his elected officials, pleading with them to make the drug more available. Now that he's healed, he's working with advocacy groups to push for broader access. TPOXX has the potential to do more than just help people feel better, he says; it could help stop the spread of the disease./agencies