St. Jude officials: 7-year-old ‘relatively stable’ after receiving Chimerix drug

Mar. 13, 2014 @ 08:27 PM

Seven-year-old Josh Hardy received his first dose of Durham-based Chimerix’s antiviral drug before 9 p.m. Wednesday, and was tolerating it well, officials from St. Jude Children’s Research Hospital said in an interview with reporters Thursday.

The experimental drug has not yet been approved by the U.S. Food and Drug Administration, but was shipped by the Durham company to the hospital in Memphis after people around the country used social media to join the boy’s family in calling for the company to release it.
While their request was for the drug to be given through the FDA’s “compassionate use” track that allows patients with serious or life-threatening diseases to access investigational drugs outside of clinical trials after they’ve exhausted all available treatment options, the company was able to come to an agreement with the FDA for the launch of a pilot study instead. The pilot is expected to be part of a larger Phase 3 clinical trial that would allow the company to make the experimental drug to just Josh as well as other patients.
“He is a very sick little boy, and this is a marathon, not a sprint, providing his treatment going forward,” said William Evans, St. Jude’s director and CEO, in the call on Thursday. Josh received Chimerix’s antiviral medication brincidofovir to help fight what St. Jude clinical director Dr. Larry Kun said is a widespread adenovirus infection. Josh contracted the virus after receiving a bone marrow transplant several weeks ago, which was part of his treatment for cancer.
Doctors believed the experimental drug would be effective in fighting the virus, and would also spare his kidneys. There is already an antiviral drug on the market virus that’s delivered intravenously, Kun said, but Chimerix’s oral antiviral drug brincidofovir was expected to do less harm to the kidneys. Kun said Josh’s initial cancer involved his kidneys.
Josh been “relatively stable” over the past 24 hours, Kun said, and he’s tolerated the medication “well to-date.” He also said that medical staff is following his progress closely and it would be “some time” before it’s known if the drug is really helpful.
In the meantime, he said Josh will require intensive medical support. Thursday afternoon, Josh was also possibly going to go outside -- briefly under secure conditions -- to enjoy a 65-degree and sunny Memphis day.
“Our top priority every day is to give every child the very best treatment possible for their disease; that’s why we’re pleased that Chimerix and the FDA found a way to make this medication available via a new protocol for Josh, and other patients like Josh, around the country,” Evans said.
And while St. Jude staff praised Chimerix for finding a way to provide the drug in this case, they also pointed to challenges they face in accessing experimental drugs for children in general. Pointing to a difference in the number of cases in children compared to the number in adults, he said there’s not a great financial incentive for pharmaceutical companies to pursue new anti-cancer drugs for children.
“It’s a challenge, it’s a problem, and it’s just part of the current system,” he said.
Sandy Walsh, a spokesman for the FDA, said in an email that the FDA has been working actively for many years on getting older as well as newer drugs studied for pediatric use.
“Because there is not a large financial incentive for companies to do these clinical studies in children, we offer incentives to companies to go on and test products in children,” he said in an email.