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Durham company under pressure to give experimental drug to ailing 7-year-old

  • http://media.charlotteobserver.com/smedia/2014/03/10/22/13/14-ivA0I.Em.156.jpeg|219
    Peter Cihelka - The Free Lance-Star
    Seven-year-old Josh Hardy, shown in May 2013 photo, was treated for malignant rhabdoid tumor at St. Jude's when he was nine months old. He's been cancer free for more than two years and has been raising money for St. Jude's with his kindergarten class at Hugh Mercer Elementary.
  • http://media.charlotteobserver.com/smedia/2014/03/10/22/13/319-NS5j5.Em.156.jpeg|421
    - Chimerix
    Kenneth Moch, Chief Operating Officer, Chimerix Inc.

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An obscure Durham drug company is facing a barrage of unwanted national attention for refusing to donate potentially lifesaving drugs to a 7-year-old boy who is gravely ill.

Chimerix, a company with no products on the market, has spent the past 13 years developing a virus-killing drug that’s now desperately sought by the family of Josh Hardy, an ailing first-grader from Fredericksburg, Va. Josh, a four-time cancer survivor, lies hospitalized with a potentially fatal adenovirus that colonized in his 46-pound body after a bone-marrow transplant.

“They keep giving him medicine, and he keeps getting worse,” said Todd Hardy, the boy’s father.

Chimerix’s experimental drug is so promising that the federal government’s Biomedical Advanced Research and Development Authority is backing the company’s work to develop the drug as a weapon against bioterrorism. Chimerix raised $118 million in April in a stock offering on the drug’s promise. The drug – called Brincidofovir (CMX001) – is not expected to win regulatory approval for public use until late 2016 at the earliest, and Chimerix has not said how much it might cost per dose.

The Hardy family’s hope stems from Chimerix’s history of distributing the medication to 430 patients between 2009 and 2012. Chimerix cut off its “compassionate care” program as the drug entered advanced stages of development and testing.

Since then Chimerix has turned away hundreds of requests from ill patients, said Chimerix CEO Kenneth Moch. He said that testing drugs on patients requires some patients to take placebos, and it would be difficult to attract patients to a drug study in which seriously ill people might end up taking a useless sugar pill when they could get the drug they need in a competing program.

Getting the drug approved – to where it would be widely available and covered by insurance – must be a drug developer’s priority, even if it means turning down requests from people who need the drug, Moch said.

“This is an ethical dilemma of substance,” Moch said. “We are heartstricken about this child.”

In medicine’s endgame of dwindling treatment options, requests like the Hardys’ are not unusual, but until recently such negotiations between doctors and drug developers played out in private. Today, desperate families are seizing control and occasionally turning to social media to apply intense pressure when startup drug companies reject a request that doesn’t meet ethics guidelines or medical standards.

Todd Hardy said he made a personal appeal to Moch by phone but said his legions of supporters will go over the CEO’s head.

“Basically, he reads off a card,” Hardy said. “We’re going after the board of directors.”

Protest planned

The Hardy family – which has been featured in national TV interviews on Fox and CNN and was one of Monday’s top trending topics on Twitter – is organizing a caravan to roll into North Carolina on Thursday. The family intends to stage a televised protest at the headquarters of the 54-employee company with the goal of breaking the drug maker’s corporate will.

“This is the first time people have tried to use this kind of pressure,” Moch said. “If we were to make this drug available for Josh Hardy, then others could make the same request. Could we say ‘no’ to them? Wouldn’t they start a similar campaign?”

In a similar campaign, Texas legal aid attorney Andrea Sloan launched a social media blitz last year to obtain experimental drugs to treat her ovarian cancer. Her online petition included support from Wynonna Judd, Rosie O’Donnell and Mia Farrow. The drugmaker, California’s BioMarin, did not offer the medication but Sloan succeeded in getting unproven drugs from another drugmaker. She did not survive her illness.

The Hardy family is requesting Chimerix’s anti-viral under an established “compassionate care” practice in which pharmaceutical companies distribute medications that are still under development to seriously ill patients who have run out of treatment options. The program carries the risk that unproven medicines may prove ineffective or cause dangerous side effects.

The U.S. Food and Drug Administration can grant such an emergency approval in less than a day in urgent cases and approved 974 “compassionate care” requests of 977 submitted in the 2013 fiscal year. To qualify a patient has to have a serious or life-threatening disease with no treatment alternatives.

Drugmakers are not required to offer “compassionate care” medications, but those that do typically establish guidelines to make sure the program meets biomedical ethics standards. For Chimerix, that means that the free drugs have to be offered to everyone who qualifies, Moch said.

Multinational companies have strict rules about when and how they dispense drugs before they’re approved by regulators.

“Compassionate use involves unidentified risk and may create a false sense of hope in patients,” according to standards set by the Eli Lilly pharmaceutical firm. “Compassionate use also can negatively impact the operations of new or ongoing clinical studies ... and subsequently delay the approval of a new medication.”

Chimerix said that Brincidofovir is available under limited circumstances and only a handful of patients are using the drug. Qualifying patients are those who have previously tested the drug in clinical trails, are infected with pox viruses, or are newborns who suffer from a herpes simplex virus.

Effectiveness unclear

The Hardy family’s request was submitted to Chimerix last week by the St. Jude Children’s Research Hospital in Memphis, Tenn., which tested the antiviral drug on 48 patients two years ago and is familiar with its attributes. In the request, Victor Santana, St. Jude’s vice president, told Chimerix that the boy is fighting for his life and “it is likely that after having fought off against childhood cancer for so long, he may succumb to this infection.”

Josh Hardy was diagnosed with Stage 5 renal cancer at 9 months of age and has been in and out of hospitals when he wasn’t healthy and playing on baseball, basketball and lacrosse teams, his father said. He’s had cancer of the lungs and of the thymus and most recently was stricken with leukemia. He is being treated for renal failure; because his immune system is compromised, Josh was infected with adenovirus about three weeks ago.

The infection itself raises another complication with Brincidofovir because the antiviral was designed to prevent infections, not to treat them. For that reason it’s not clear if the drug would help Josh, Moch said.

The St. Jude’s clinical trial was conducted on patients with early infections of adenovirus, and the results were not statistically significant, but they showed no side effects, clearing the way for further study. The current trial, using 450 patients, began in September and will continue into 2015, but it’s designed to measure the prevention of a similar virus, not adenovirus.

Arthur Caplan, head of the medical ethics division of the NYU Langone Medical Center, said companies like Chimerix are in an impossible position. Yielding to public appeals could undermine the development of experimental drugs and skew a health care system that already favors some over others.

“Josh has savvy parents. Josh is kind of cute. Josh has a puppy. Josh has a media campaign,” Caplan said. “But the next Josh may not have any of this.”

Murawski: 919-829-8932
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