Why Drug Company Chimerix Is Denying Meds To A Dying Seven-Year-Old

Josh Hardy is a survivor. At just seven years old, he has already beaten cancer four times and survived a bone marrow transplant — but now, his life hangs in the balance because of a virus that causes the common cold. The one drug that Josh's family and doctors believe could help him, brincidofovir, is still unapproved, and the drug company that manufactures it, Chimerix, says it can't give it to him.

Brincidofovir is made by Chimerix, a small North Carolina drug company. This antiviral has shown in clinical trials that it can clear up adenovirus infections like Josh's in as little as two weeks. Josh has had the infection for two months now, and his family has started a petition to get Chimerix to make the drug available for compassionate use.

Josh was first diagnosed with cancer, with aggressive rhabdoid tumors in both kidneys, when he was just nine months old. He went through surgery, chemotherapy and radiation therapy, but over the following years he developed cancer in his thymus gland and his lung.

In November 2013, nearly four years after being declared cancer-free, Josh was diagnosed with a precancerous bone marrow disorder that can be brought on by cancer treatments. He needed a bone marrow transplant.

Following the bone marrow transplant and chemotherapy in January 2014, Josh developed graft versus host disease, a condition where the new, implanted cells start to attack the body. To stave this off, Josh's doctors suppressed his immune system with drugs, which is what left the little boy wide open to contracting adenovirus.

His doctors at St. Jude's Children's Research Hospital in Tennessee first attempted to fight the virus with an intravenous form of brincidofovir, but the drug was toxic to Josh's kidneys and didn't help to stave off the infection. Josh's doctors, who had been part of the trials of the oral form of the drug, thought that the alternative form of the drug was Josh's best chance of survival. They asked Chimerix to make the drug available to Josh.

The reasons why Chimerix, which has received $72 million in federal funding to help develop brincidofovir, won't just give the Hardys the drug are much more complex than just being a cold, Big Pharma corporation unsympathetic to Josh's plight. In fact, Chimerix President and CEO Kenneth Moch, a father himself, has said he is heartbroken by Josh's situation, but that this is about more than saving just one boy.

Moch says that if Chimerix makes the drug available to Josh for compassionate use then it would have to make it available for all of the hundreds of other patients hoping to get their hands on it. Not only would it cost the drug company $50,000 per patient, as insurance companies rarely pay for experimental drugs, it would also mean diverting valuable resources away from getting brincidofovir to market, where it could ultimately help the most people.

Up until two years ago, Chimerix did make brincidofovir available under a broader compassionate use program, but now it only gives the drug out to those who meet very strict criteria - which Josh does not.

It's also often not in a drug company's interest to give out unapproved drugs on compassionate grounds, because it can make it harder for the drug win approval from the Food and Drug Administration. Those looking for access to a drug on compassionate grounds are usualy very sick patients, who might not necessarily do well on the drug. Any poor outcomes have to be reported by the company to the FDA in its application to market the drug, which Chimerix is hoping will be available to all by the end of 2016.

But whatever the reason, Josh's family and his supporters are refusing to take "no" for an answer. Along with the online petition - which has already received more than 11,000 signatures - a Twitter campaign with the hashtag #savejosh has been launched. Thousands of people have also called, emailed and tweeted Chimerix to try to convince them to change their mind.

UPDATE: Chimerix has agreed to give Josh the medication.