Release Brincidofovir for compassionate use to treat the adenovirus plaguing 7-year old Josh Hardy right now.
When Josh, my cousin's son, was 9 months old, he was diagnosed with a malignant rhabdoid tumor of the kidneys. It’s a highly aggressive, rare form of cancer that only 15 children are diagnosed with each year. In November 2013, he had a bone marrow biopsy and learned he had a bone marrow disorder, as a result of a treatment he had earlier in his life. On New Year’s Eve, Josh checked into St. Jude for inpatient care. He started 10 days of chemotherapy, and on Jan. 10, got the bone marrow transplant.
Though he had heart and kidney issues before, the transplant caused a lot of complications for Josh. On Jan. 14, Josh had to go to the ICU for heart failure issues and just five days later was put on a ventilator. As soon as thing started to improve Josh the adenovirus showed up because of Josh’s compromised immune system. The medicine Josh was given through an IV to treat the infection ended up damaging his kidneys.
His doctors recommended Brincidofovir, an antiviral drug manufactured by Chimerix that is still in trial form. They say that other children treated with the drug had their adenovirus clear up within two weeks. For complicated reasons, and because the drug is close to going to market, it isn’t available to Josh right now.
According to the American Cancer Society’s website, medical professionals use the term “compassionate use” to refer to the treatment of a seriously ill patient using a new, unapproved drug when no other treatments are available.
Drugs that are being tested but have not yet been approved by the U.S. Food and Drug Administration are called investigational drugs. These drugs are generally available only to people who are taking part in a clinical trial (a research study that is testing the drug).
Single-patient access can be granted if the patient’s doctor requests the drug from the company that makes it.
Josh’s doctor did.
If the company agrees, the patient’s doctor works with the drug company to ask the FDA to approve the drug for use by this one patient.
Kenneth Moch, the company’s president and CEO, said Friday that Chimerix officials are “acutely aware of the situation faced by this patient.”
However, he said they cannot help Josh.
Moch said that because the drug is in phase-three clinical development, they cannot allow it to be used by patients through the compassionate use program.
He said that five years ago, much earlier in the drug’s development, Chimerix began receiving requests for emergency use for many viral infections.
While they could fulfill a small number of requests, two years ago they had to stop fulfilling those.
“As we progressed to larger and more complex safety trials, we made the decision two years ago to stop the program and focus resources on earning FDA approval,” Moch said.
He said the drug could be widely available in 2016—if they are lucky.
“We understand this great need,” he said. “That’s what drives us to focus on getting it available. It creates these really difficult complex situations where the need to pursue the formal clinical development under the FDA process takes precedent over other needs,” he said.
Josh's family continues to keep their faith in God and pray for Chimerix to find a way to help or for the virus to go away on it's own! Please join us in asking Chimerix to provide compassionate use access to this drug for Josh!
Release a dose of Brincidofovir for compassionate use to save 7 year old Josh Hardy!
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