Josh Hardy turned 8 on March 31st.
A Fredericksburg, Va., family's fight for medicine to save their critically ill seven-year-old is over, but it may open the door to another fight for all children battling cancer.
After a struggle with Chimerix and the FDA, Josh Hardy received his first dose of a potentially life-saving antiviral medication Brincidofovir Wednesday night just before 9 p.m.
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According to St. Jude Children's Research doctors Josh is doing well, but it will take a few days to notice a difference with the new medication. They are monitoring his blood and kidney, watching for any side effects.
"He's tolerated this well to date and we're following his progress quite closely," Dr. Larry Kun, Clinical Director at St. Jude Children's Research Hospital said, "It will be some time before we know if this drug is really helpful and in the meantime he requires intensive medical support."
They add he was able to go outside to enjoy the weather, something doctors said the seven-year-old has been eager to do.
"He's a very lively and personable, if critically ill, young boy," said Kun.
Josh has battled kidney tumors off and on; he is currently fighting an adenovirus infection following a bone marrow transplant. St. Jude doctors say the hospital has used the IV trial version of the anti-viral, including on Josh, but they needed the oral version, not previously tested on children, to save his already damaged kidney.
"It had been shown to be both more potent in killing these double stranded DNA viruses as well as less toxic to the kidney," Dr. William Evans said.
"In Josh's case in particular we've actually been able to measure a reduction in the virus presence after each of the administrations of the intravenous drug but we were constrained in how much of that we could deliver because of its effects on the kidney," Dr. Kun added.
The FDA approved a compassionate care trial of the oral medicine for 20 cases, including Josh.
Josh's mother Aimee released a statement saying, "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. 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."
This one case may open up a whole new fight for pediatric cancer treatment.
"Even though the leading cause of death with disease in children is cancer, the number of trial cancer cases compared to adults is small, so there's not a great financial incentive for the pharmaceutical industry to pursue new anti-cancer drugs for children," Dr. Evans said, "In the last several years there have been no new anti-cancer drugs introduced for pediatric cancers in particular."
A family spokesperson said Thursday the Hardys are grateful to the FDA for jumping through hoops to get this compassionate use trial approved quickly.
Evans said trials for anti-cancer pediatric medicines has been a challenge and a problem that is a part of the current system.