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Terminally ill patients getting access to experimental drugs

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Nathan Norman and his family travel to Duke Children's Hospital from Lynchburg, Virginia, to treat a persistent brain tumor that spread to the 7-year-old's spine. Nathan Norman and his family travel to Duke Children's Hospital from Lynchburg, Virginia, to treat a persistent brain tumor that spread to the 7-year-old's spine.
DURHAM, N.C. - Seven-year-old cancer survivor Josh Hardy's plight touched the world.

Hardy's family, who is from Fredericksburg, Virginia, wanted Triangle company Chimerix to provide an experimental drug through what is called "compassionate use."

"There's always hope. As long as your child or family member is still breathing, there's always hope," said Bill Burns, Josh's uncle.

That hope inspired a grassroots campaign to get Josh the drug that could save his life. They hoped the experimental drug Brincidofovir would be the cure for Josh's adenovirus infection.

Local company Chimerix makes the drug, which was still in clinical trials. After originally being denied the drug due to FDA regulations, Josh's family begged for an exception. In March, Chimerix and the FDA allowed Josh use the drug through a Compassionate Use clause. It is done on a case-by-case basis.

So far, the drug is working.

"He's got to go to basketball games. He's out of his ICU bed. He continues to get better. He continues to get stronger, and we hope in time he will be the loud Josh we know and love," Burns said.

For children with cancer, accessing experimental drugs can be the difference between life and death.

"Once you finish with surgery, chemo and radiation, we need other treatments to keep these children alive," said Dr. Sridharan Gururangan, clinical director for pediatric services at the Preston Robert Tisch Brain Tumor Center at Duke University.

Nathan Norman and his family travel to Duke Children's Hospital from Lynchburg, Virginia, to treat a persistent brain tumor that spread to the 7-year-old's spine.

Through it all, Nathan keeps fighting and the brave boy is inspiring other kids with pediatric cancer through his Hope For Tomorrow Foundation. Nathan's hope for a cure lies in a new drug called AZD6244, which is in the second phase of clinical trials.

At first, Nathan didn't qualify because his tumor is stage 2.

"We've had seven medicines that have failed him, and there's nothing out there other than the clinical trials available," Nathan's mom, Dawn Norman, said.

But Nathan's doctor pushed hard to get him in the trial.

"I think the minute he heard, we were probably the first people he called," Dawn Norman said.

"He's taken this medicine like a champ. It's important for these children to actually swallow the pills without crushing or chewing these pills because the drug companies don't allow us to do that and Nathan has been a champ swallowing these pills," Gururangan said.

"He's doing great. Dr. G says of all the patients that he's had on it, Nathan by far has had the least side effects," Dawn Norman said.

Nathan has been taking the drug since April 3. When asked if they are nervous about the unknowns, Nathan's mother responded, "Oh we are. We know God is in control of everything ... but it makes us anxious because we don't know what his will is."

But they know chemotherapy and radiation are not working. Gururangan says too much chemotherapy and radiation can sometimes harm rather than help children with cancer.

"We need newer therapies to treat children who have failed all these primary treatments, and I beg the pharmaceutical companies to help us," Gururangan said.

"We all believe this is something worth trying. If there's nothing else available, at least give them the chance to try," Dawn Norman said.

The Goldwater Institute, a libertarian think tank based in Phoenix, Arizona, is lobbying for Right-to-Try laws, where terminally ill patients can have access to any drug that passes phase 1 of clinical trials, the safety phase. The bills are pending in four states: Arizona, Colorado, Louisiana and Missouri.

The Oscar-winning movie "Dallas Buyer's Club" brought the issue to light in its story of an AIDS patient.

"This is about saving lives and empowering patients and their doctors. When you or a loved one is facing a terminal illness, the last thing you want or need at a time like that is to have a federal bureaucrat in the process creating potential delays," said Christina Corieri, health care policy analyst of the Goldwater Institute.

But doctors say the regulations are there for a reason. When asked if children should be able to access these drugs outside of clinical trials, Gururangan responded, "That is less optimal. Even though that one particular child gets access to the drug, the majority of children don't benefit from that approach."

Patients like Nathan are closely monitored in clinical trials. Drug company GlaxoSmithKline also said it needs to be up to the FDA and not the state.

No matter what, the families will keep fighting.

"Never quit. Never take no for an answer. Never quit," Burns said.

"What if it is their child and they have no other options for their child. Wouldn't they be willing to say, 'Let me sign off,'" Norman asked.

So far, there is no Right-to-Try law in North Carolina, but the Goldwater Institute said it looks forward to trying to work with North Carolina legislators about the issue.

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