Dying Boy to Get Unapproved Drug After Family's Plea

Drug company answers family's plea for unapproved drug for sick son.

ByABC News
March 12, 2014, 9:00 AM

March 12, 2014— -- A dying 7-year-old boy will get the unapproved drug his family has been pleading for.

Josh Hardy has survived cancer four times, his parents said, but he’s been in the intensive care unit since January fighting a virus that causes the common cold. They say their best hope for Josh’s survival is an unapproved anti-viral drug called brincidofovir, but the company that makes it, Chimerix, has said it can’t give it to him.

The Hardys launched a campaign, including several online petitions to change the company’s mind. And it worked.

"The one thing I would want to share with America is thank you so much for the love and support," Josh’s mother, Aimee Hardy, told Good Morning America.

Chimerix announced that Josh’s story accelerated talks with the Food and Drug Administration, allowing it to launch a 20-patient open-label clinical trial today. Josh will be the first patient and is expected to receive the drug within 48 hours.

The boy received his first cancer diagnosis when he was just a baby: Aggressive rhabdoid tumors in both kidneys, Hardy said. He went through surgery, chemotherapy and radiation, but the cancer returned in his thalamus gland and then his lung.

Then, after nearly four years of being cancer-free, Josh received bad news in November 2013, Hardy said. He had myelodysplastic syndrome –- the same precancerous bone marrow disorder that ABC News anchor Robin Roberts was diagnosed with in 2012. This can be caused by cancer treatments.

Josh underwent a bone marrow transplant to remedy the disorder, but he developed graft versus host disease -- meaning the new cells started to attack his body, Hardy said."He was in complete heart failure and kidney failure and went into the ICU on January 14," Hardy said.

To stop it, Josh’s doctors at St. Jude Children’s Hospital in Tennessee suppressed his immune system with drugs, allowing his heart and kidneys to start healing.But with a weakened immune system, Josh came down with adenovirus, a common virus that causes colds but can also be much more serious, Hardy said.

In Josh’s case, reawakening his immune system to fight the virus could bring back his graft versus host disease, so doctors gave him an antiviral medicine to get rid of the adenovirus. But the drug was toxic to his kidneys and wasn’t working, Hardy said.

"He’s at a physical standstill," Hardy said.

That was when Josh’s doctor at St. Jude suggested brincidofovir, a drug that researchers at Chimerix, a small North Carolina drug company, have been developing for the last 14 years.

St. Jude had been involved in a clinical trial of brincidofovir in which children who had undergone bone marrow transplants and had early adenovirus infections took the drug and were able to decrease the amount of virus in their bodies.

But Dr. Hervé Momméja-Marin, Chimerix vice president of clinical research, said the drug has not been proven to do this in more advanced adenovirus cases – like Josh’s. Josh has now had his infection for two months.

Chimerex President and CEO Kenneth Moch said on Monday -- before the creation of the 20-patient trial -- that giving the drug to Josh would mean they would have to give the drug to the hundreds of other patients hoping to get it under the FDA's compassionate use rules -– which allow patients to get drugs even if they aren’t enrolled in clinical trials.