A Virginia family has apparently won its Internet campaign to persuade a pharmaceutical company to share its experimental, antiviral drug with them to save their 7-year-old son, who has cancer.
The campaign to get the drug, called brincidofovir, to treat an adenovirus developed by Josh Hardy after a bone marrow transplant had attracted nearly 24,000 Facebook followers.
The manufacturer, Chimerix of Durham, N.C., informed PEOPLE on Tuesday evening that it had worked out a deal with the FDA to initiate a new study of the effects of brincidofovir on the adenovirus – beginning Wednesday with Josh Hardy.
“Josh Hardy s story brought to public attention the often devastating impact of adenovirus infection, and helped accelerate a discussion between the FDA and Chimerix regarding the need for additional clinical development to assess brincidofovir’s potential in adenovirus infection,” Chimerix announced Tuesday afternoon. “This study is expected to begin with Josh Hardy as the first patient enrolled on Wednesday, March 12.”
It was unclear how many other patients would join the expanded study. In recent years, hundreds have expressed an interest, Chimerix says.
Josh’s mother, Aimee Hardy, was ecstatic.
“We are so relieved and grateful that government [the FDA], a corporation [Chimerix], the medical community [St. Jude’s] and individuals were able to come together to develop a solution that would give Josh a chance, while also providing the opportunity to many others to receive potentially lifesaving treatment,” Hardy told PEOPLE in a statement Tuesday. “This is such a wonderful example of the good that can happen when committed people come together to find a positive solution to an agonizing situation. We are very appreciative to everyone who put tremendous effort and thought into making this happen.”
According to Chimerix’s website, 86 percent of subjects in clinical trials were successfully treated with brincidofovir for the adenovirus; in two-thirds of the subjects, the virus became undetectable within the first week.
Debate Over ‘Compassionate Use’
But without being part of a clinical study such as the one Josh Hardy is about to join, his family was trying to get Chimerix to share the drug for compassionate use. And that’s an area that the pharmaceutical industry approaches with caution.
In an interview with Fox News in the days before Tuesday’s announcement, Chimerix CEO Ken Moch said the company “made the decision two years ago to stop the [compassionate use] program and focus resources on earning FDA approval.”
“Making an experimental drug, which currently has limited clinical evidence, available outside of controlled clinical trials has the potential to slow or derail our ability to make brincidofovir available as soon as possible to the thousands of patients each year who might benefit from it,” Moch explained in a statement to PEOPLE. “This is why we are focused on demonstrating the safety and efficacy of brincidofovir in our ongoing Phase 3 clinical trial.
Chimerix spokesman Tony Plohoros says that the company’s goal is to get brincidofovir to the market by 2016. He says that, when it comes to compassionate use, Chimerix has been following principles laid out by the Biotechnology Industry Organization, which says that “a patient’s right to treatment based on his or her autonomous decision-making ability does not supersede a company’s ethical responsibility to develop and market safe and effective products as fast as possible.”
He points to a recent article in The New York Times explaining that drug companies are becoming more careful about sharing unapproved drugs.
“If doctors simply let people take untested medicines without going through all the clinical trials, drug companies would most likely never get anyone to enroll in them, never get the data on safety and efficacy for FDA. Approval, and never pass the gateway to big sales,” says the report.