It took a massive social media campaign and an unorthodox solution to get Josh Hardy access to an experimental drug.
Now thanks to that drug, his family says, the seven-year-old’s health is indeed improving.
“Josh is doing well,” his uncle, William Burns, told me over the phone yesterday. “He’s got a long road ahead to recovery, but it appears that the medicine is working.”
In a recent post to the site CaringBridge.org, Josh’s mother Aimee said that after just two doses of brincidofovir, an oral antiviral drug, “the adenovirus is on its way to being suppressed” and that doctors at St. Jude Children’s Research Hospital in Memphis, Tennessee believe Josh is close to getting out of the ICU.
One major challenge still facing Josh include recovering his kidney function, which Aimee Hardy wrote “is the last major key for Josh’s full recovery.”
Josh, a cancer survivor who has recently had a bone marrow transplant, began treatment with brincidofovir for adenovirus last week. The common virus usually isn’t serious but for a patient with a compromised immune system like Josh, it can be deadly. Brincidofovir, made by the small North Carolina pharmaceutical start-up Chimerix, has yet to win FDA approval and is still in the trial stage.
Under what’s often called “compassionate use,” pharmaceutical companies can provide experimental drugs to patients facing life-threatening illnesses. But Chimerix officials initially rejected Josh’s doctors requests to provide its drug to the boy under compassionate use, saying the company had stopped honoring such requests for fear of endangering its efforts to get FDA approval and make the potentially life-saving drug widely available. (For more on the sizeable risks drug companies face when they provide compassionate use medicines to patients, see my earlier story on Josh Hardy here.)
That rejection prompted the #SaveJosh social media campaign on Facebook and Twitter, quickly attracting the attention of the national media. Days after the campaign began, Chimerix announced stunning news: together with the FDA, they found a way to provide Josh the drug without derailing efforts to get it approved: The FDA gave the greenlight for Josh and 19 other patients to receive brincidofovir in a pilot tiral.
Chimerix said that Josh Hardy’s story “helped accelerate a discussion between the FDA and Chimerix regarding the need for additional clinical development to assess brincidofovir’s potential in adenovirus infection.” In other words, Chimerix had already been working on starting a new brincidofovir study but the #SaveJosh campaign sped up the process.
But the unusually quick collaboration between a pharmaceutical company and the FDA has implications for patients beyond Josh and the 19 others in the new study. Colorado anethesiologist David Dragoo suggested that it could “completely change the game in terms of how drugs that are still in development can be provided to patients and yet still protect the integrity and safety that we have all come to expect in the prescriptions we take, and perhaps one day these experimental drugs may even be subsidized as part of our health insurance benefits in extenuating circumstances like this one.”
Bioethicist Dr. Arthur Caplan, of NYU Langone Medical Center, says there must be a systemic way to distribute compassionate use drugs so that it doesn’t take a social media campaign to get a child (or anyone else) lifesaving drugs.
“Where is the country’s national compassionate use fund to pay for experimental drugs for all — rich and poor, cute and not, young and old, media-savvy and not? Josh and those like him don’t need more than speeches, they need money. Where is compassionate use in the current debate about health reform? Nowhere,” Caplan wrote in an op-ed piece on NBCNews.com last week, before the new brincidofovir pilot trial was announced.
Caplan told me last week that the pilot trial that will include Josh Hardy is “a good compromise” but warned ” another child with adenovirus could appear (at another hospital) and the whole scenario will be recreated.”
The Hardy family, meanwhile, is grateful to all their supporters and to Chimerix for getting Josh the drug. They’re asking those with an interest in joining the battle against pediatric cancer to support the Coalition Against Childhood Cancer, Max Cure Foundation, St. Jude Children’s Research Hospital and Caringbridge.org.
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Photo courtesy of Josh Hardy family via SaveJosh.