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Drugmaker reverses course, offers treatment to stricken 7-year-old

March 12, 2014|By Paresh Dave
  • Josh Hardy, a 7-year-old Virginia boy struggling with a serious infection, was scheduled to begin a potentially lifesaving drug treatment Wednesday after his family's awareness campaign went viral and spurred the FDA and a drug manufacturer to fast-track a study.
Josh Hardy, a 7-year-old Virginia boy struggling with a serious infection,… (Hardy family )

A 7-year-old Virginia boy struggling with a serious infection began a potentially lifesaving drug treatment Wednesday after his family's awareness campaign went viral and spurred the FDA and a pharmaceutical company to fast-track a new drug trial.

First-grader Josh Hardy contracted adenovirus during a bone marrow transplant, which he needed as part of his treatment for leukemia. Though the viral infection typically has mild effects, Josh's cancer-weakened immune system has been more susceptible. His doctors at a Tennessee hospital recommended that he receive the experimental drug brincidofovir.

But the manufacturer, Chimerix, a small drug manufacturer in Durham, N.C., said it was still in early testing of its only drug as a treatment for adenovirus patients with weakened immune systems. It refused to give Josh the drug through a "compassionate use" exception that would have ensured he didn't receive a placebo during the study.

Helping Josh, the company said, would have led other patients in drug trials to lodge similar emotional appeals in the media to avoid placebos. Deciding whom to help would have grown burdensome for the small company and could have marred attempts to get the FDA to approve it if notable patients, such as Josh, die despite its use.

Through an agreement with the FDA announced Tuesday, Josh becomes the first of 20 patients guaranteed to receive the medication in what the company described as a pilot test. It is expected to serve as a precursor to a typical, double-blind clinical trial.

"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," the company said in a statement.  

"Being unable to fulfill requests for compassionate use is excruciating, and not a decision any one of us ever wants to have to make," Chimerix Chief Executive Kenneth Moch said in the statement. "It is essential that each individual in a health crisis be treated with equal gravity and value."

St. Jude’s Children's Research Hospital called Josh's medical situation "critical and complex."

"We are grateful for the efforts of Chimerix, the FDA and many others who worked to achieve this outcome," the hospital said. "We ask that you continue to keep Josh and his family in your thoughts."

Josh’s mom celebrated the news.

“Glory to GOD! They are releasing the drug for Josh!!!!!!!!!,” Aimee Hardy said on Facebook.

Chimerix’s announcement came the night before Josh's supporters planned to travel to Durham to protest outside the company's office. Supporters had rallied on social media, and Josh's family had appeared on national television during the last few days. The company had said it would not reverse course.

But the growing interest in the case led to a conversation between the company and the FDA, which has generally granted compassionate use exceptions. The FDA said the pilot study agreement was in everybody's best interest and avoided the worries that the drugmaker had about making the drug available through the compassionate use provision. 

In a trial late last year, brincidofovir proved successful at warding off adenovirus immediately after it was detected in the blood. But Josh has been infected since mid-Feburary.

At 9 months old he developed an aggressive cancer in his kidneys, according to the family. Chemotherapy eviscerated the softball- and walnut-sized tumors, but only temporarily. He’s been in and out of hospitals his entire life, most recently suffering from leukemia.

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