7-year-old cancer survivor still in intensive care after receiving experimental drug

10:39 AM, Mar 15, 2014   |    comments
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(CBS NEWS) Seven-year-old cancer survivor Josh Hardy began receiving an experimental drug Wednesday that his family had been pleading for as a potential last hope, doctors at St. Jude Children's Hospital in Memphis told reporters Thursday.

St. Jude's CEO Dr. William Evans said the boy tolerated the drug well to date, but still needed "intensive medical support and attention."

Since taking the drug, he's looked "relatively good" and even wanted to go outside.

Josh's story got national attention this week when his plight exploded on social and news media.

The boy from Fredericksburg, Va., had survived cancer four times before age 7, but his life was being threatened by an adenovirus, an infection that can cause severe illness in people with weakened immune systems.

 

Conventional treatments didn't help, so his doctors at St. Jude requested a "compassionate use" application to use brincidofovir, an experimental drug from a company called Chimerix which has yet to be approved by the Food and Drug Administration. The drug is being studied to treat 12 different viruses, including adenovirus.

When the company rejected Josh's application, a social media campaign ensued, with many tweeting Chimerix and sharing media reports of the story. Afterwards, the company got FDA approval for a new pilot study to treat Josh and several others, and his treatment began the next day.

Josh has been struggling with serious health problems for most of his young life, starting with a diagnosis of kidney cancer when he was just nine months old.

Last November, doctors found irregularities in Josh's blood and determined he needed a bone marrow transplant due to secondary myelodysplastic syndrome, a difficult-to-treat disease of the blood and bone marrow that occurs following chemotherapy or radiation. The transplant took place in January.

 

After the bone marrow transplant he required oxygen support and dialysis, and suffered gastrointestinal bleeding, Evans explained. His family told CBS affiliate WTVR in Richmond, Va., that Josh was in a coma for 16 days. Then he contracted the adenovirus.

The boy's doctors reached out to Chimerix because two other St. Jude patients had enrolled in an earlier study and tolerated the drug, so the hospital thought it might work for Josh. But the hospital noted his medical situation is complex.

"The family is grateful to the company for working to make this happen, as is St Jude," said Evans. "Overall pediatric drugs are hard to come by and they hope this will put attention on the fact that drugs for adults are more readily available."

"I think the most heartwarming thing about this is we were able to treat Josh Hardy, and we will be able to treat many more Joshes," Chimerix CEO Ken Moch told CBS News on Friday. "That was the issue."

Moch explained that other children had applied for compassionate use exemptions to take the experimental drug, but the company was initially worried about how it could ethically say yes to Josh and no to the "other Joshes."

Moch had said that prescribing the drug outside of its safety and efficacy trials could derail the drug approval process; the company has been working on this drug for 14 years, he pointed out.

"How could we choose for any compassionate basis for adenovirus or any virus, and at the same time we're trying to focus on approval to treat the broad approval?" he said.

 

When asked, the FDA could not think of an example of a drug that was not approved because of a compassionate use/expanded access program, a spokesperson told CBS News.

Chimerix said Friday evening that the company and the FDA worked jointly to develop a clinical trial for adenovirus infections in immunocompromised patients.

"Making a protocol available by Tuesday evening would not have been possible without the resources that both parties dedicated to this mission," he said.

 

There's no doubt social media played a role in the initiation of the new study with the FDA to include Josh in the most recent phase of testing, Moch said, and if he were in that situation he'd likely do the same as Josh's parents.

But the headlines that a drug company refused to give a sick child a life-saving drug don't tell the whole story, he said.

Said Moch, "This has been one of the most difficult heart-wrenching times for a company and the people of this company as we work to meet a set of conflicting obligations."

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