Boy Has Surprising Turnaround After Getting Unapproved Drug

April 16, 2014— -- After successfully pleading for a drug company to save her then-7-year-old son last month, Aimee Hardy announced that he has been discharged from the hospital.

“It’s just hectic right now,” she told ABCNews.com breathlessly, explaining that she no longer has the help of the hospital nurses to care for Josh during his recovery as an outpatient. “He has a ways to go, but we’re definitely on a good road to recovery.”

Read more about how families are using social media to get access to unapproved drugs.

Josh turned 8 last month, and got a pass from the hospital to see a Miami Heat basketball game the following week because he was doing so well, she said.

Josh has beaten cancer four times, his parents said, but they had feared that he would die last month from a virus that causes the common cold.

The Hardy family said their best hope for Josh’s survival was an unapproved antiviral drug called brincidofovir, but the company that makes it said it couldn’t give it to him. So the Hardys started several online petitions to change the company’s mind.

"Having survived four diagnoses of cancer, it would be an absolute travesty for him to meet his demise from a virus," mom Aimee Hardy told ABCNews.com in March. "Especially knowing there is a medicine in someone’s hands that can rid of this virus."

Josh 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. It 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 past 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.

Chimerix President and CEO Kenneth Moch said 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.

"We all have great compassion for this child," Moch told ABCNews.com March 11. "We spent our lives trying to develop new medications for patients just like Josh… We need to make sure to get this drug available as soon as possible to as many people as possible."

But the following day, 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.

But Moch resigned from his position last week, Chimerix announced.

“I did hear about Mr. Moch departing from Chimerix,” Aimee Hardy told ABCNews.com. “I felt terrible. I was hoping it wasn’t because it wasn’t what our intent was. All we wanted was to save our son. … I pray wherever he goes next, he’ll have tremendous success and I wish him well.”