Two of Boston’s renowned teaching hospitals are taking part in separate clinical trials for a drug that some specialists believe is the best hope in quickly helping patients stricken by the novel coronavirus that is sweeping the globe.
As doctors and scientists the world over race to find treatments, Massachusetts General Hospital and Brigham and Women’s Hospital are conducting trials of the experimental drug remdesivir, originally developed by the California-based biopharmaceutical company Gilead Sciences to treat other viruses.
In a news conference on Thursday, President Trump touted the potential of remdesivir. After he stated that the drug was “approved or very close to approved" by the Food and Drug Administration, however, FDA commissioner Stephen Hahn clarified that “remdesivir is going through the normal process” of clinical trials.
As for whether the drug is being fast-tracked for FDA approval, Gilead Sciences provided the following statement to the Globe: “We are in discussions with regulatory agencies, including FDA, to determine the most appropriate pathway for approval of remdesivir in the event that the data are positive.”
MGH has joined what the National Institutes of Health describes as the country’s first clinical trial of an experimental treatment for COVID-19, sponsored by the National Institute of Allergy and Infectious Diseases, part of the NIH. MGH is currently the only hospital in New England participating in this trial, according to a list of sites shared by the hospital.
It’s a massive undertaking, with patients being enrolled at up to 50 sites across the country, taking remdesivir. Separate clinical trials of the drug are underway in China. The NIH trial, which can be adapted to evaluate other treatments, aims to determine if the drug relieves respiratory problems and other COVID-19 symptoms, helping patients leave the hospital sooner.
Remdesivir works not by attacking a specific virus such as COVID-19, but by targeting the way the virus replicates itself and spreads within an organism.
“This is one of the leading candidates” for treating COVID-19, said Dr. Libby Hohmann, MGH’s principal investigator for the NIH trial and an associate professor of medicine and infectious diseases at Harvard Medical School. “We're happy to have it here.”
The first patient joined the MGH trial on Sunday, and by Wednesday, three had already enrolled. But that doesn’t mean they’re all getting the drug. It’s a randomized, double-blind, placebo-controlled study. “If someone joins, they have a 50 percent chance of getting the drug and a 50 percent chance of getting a placebo,” explained Hohmann. Placebo-controlled trials help researchers determine if a drug delivers better results than no medicine.
Hohmann said the trial is for patients who have been diagnosed with COVID-19 and need to be hospitalized with significant lung problems. Patients will receive the drug intravenously, once daily. It’s a 10-day course of treatment, though if a patient recovers well enough to go home, the treatment would stop, according to Hohmann.
“I’m cautiously optimistic,” she said. “We absolutely appreciate the willingness of people to enroll. It’s not just helping them, should they get the drug and the drug is effective — that’s a lot of ifs — but helping us understand what’s worth pursuing and what’s worth giving the people who come after them.”
Meanwhile the Brigham is gearing up for two other remdesivir trials that Gilead recently launched itself to study the drug’s effectiveness and safety. The trials will involve patients with moderate and severe illness, encompassing five-day and 10-day courses of treatment.
Dr. Francisco Marty, principal investigator for the Brigham studies and associate professor of medicine at Harvard Medical School, said the hospital hoped to start recruiting patients on Thursday, joining multiple other sites worldwide in the Gilead-led trials.
“We feel the responsibility to do a good job, "Marty said. “It is key because if the drug works, [it] may be available for a lot more people.”
The Gilead trials, which are randomized by course of treatment but not placebo-controlled, will include 1,000 patients around the world. “At the rate of the epidemic we may be done in a month or two,” Marty said.
“In a way it’s scary that we may be able to enroll 1,000 patients very quickly,” he said. “On the other hand, if we do a good job, then in a month or two we should be able to know if this works and get it to the FDA for approval, and if it doesn’t work move to something else that may be more promising by then.”
Remdesivir is a broad-spectrum antiviral drug that has shown promise in test tube and animal studies on other coronaviruses such as SARS and MERS, but was disappointing in treating people with Ebola in a 2018-19 study published in December in the New England Journal of Medicine. It has not yet been approved for any use. Early data from three US patients who received remdesivir under an emergency compassionate use program have caused some to raise questions about whether the drug will ultimately be successful.
But even as numerous other treatments are being explored, hopes remain high. “There’s only one drug right now that we think may have real efficacy. And that’s remdesivir,” said Dr. Bruce Aylward, a World Health Organization official, at a press conference in China on Feb. 24.
“The speed with which remdesivir has moved into clinical development for this coronavirus reflects the pressing need for treatment options,” said Dr. Merdad Parsey, chief medical officer at Gilead, in a statement.
NIAID director Dr. Anthony S. Fauci has emphasized the importance of clinical trials in finding ways to address the pandemic. “We urgently need a safe and effective treatment for COVID-19. Although remdesivir has been administered to some patients with COVID-19, we do not have solid data to indicate it can improve clinical outcomes,” Fauci said in a statement from the NIH. “A randomized, placebo-controlled trial is the gold standard for determining if an experimental treatment can benefit patients.”