Clinical trial saves Dalton boy`s life

Dec. 25--Cameron Watkins looks out of place as he leans back into the chair in Room 2 of the Pediatric Cancer and Blood Disorder Center at the Children's Hospital at Erlanger. Large plastic dinosaurs stand in some of the adjacent rooms, a giraffe towers over one wall and children toddle around a play area filled with plastic balls and a toy kitchen.

But Watkins, 23, is at home.

"I've spent more time here than I've spent in school," he said. "They've known me my whole life."

This is the 51st time he has made the trip from his home in Dalton, Ga., this year, once each week. He has been coming for 10 years, ever since he took part in a clinical trial for a new drug that saved his life.

On this Tuesday morning, his friend Triston Smith, who hopes to one day become a doctor, sprays a numbing anesthetic around the port inserted into Watkins's chest. Nurse Laura Gibson inserts the IV that will slowly drip a drug solution into his body over the next five hours. Then he and Triston settle down for a game of Connect 4, a version of tic-tac-toe on steroids.

"He's an amazing kid," said Dr. Manoo Bhakta, who has helped treat Watkins since he was 5. "People ask, do you believe in miracles -- he's simply a miracle."

When Watkins was a young boy his mother, Tressia, thought she saw problems she had not noticed with his older brother. Doctors initially brushed her off as an overprotective mom. She persisted, and found a doctor who realized something really was wrong.

The diagnosis was Hunter syndrome, an extremely rare condition caused by the lack of an enzyme in his body; a genetic defect. It only affects boys -- around one in 150,000.

The enzyme helps the body break down and clear out used material. When it is not present, the material builds up in the heart, the tendons and even the brain, causing myriad problems.

Bhakta remembers when he met Watkins.

"He had a massive spleen, and he had an ear infection that had started bleeding and couldn't be stopped," he said. Watkins also had a leaky heart valve and doctors were considering a risky transplant operation.

At the time, Hunter syndrome was a death sentence -- most boys diagnosed with it only lived until their midteens.

But Watkins got a lucky break. Bhakta put him in touch with doctors at Duke University to become part of the first clinical study of a new drug, idursulfase, sold by the Shire pharmaceutical company as elaprase. While the drug can't replace his missing enzyme, it does perform many of its functions, keeping the symptoms from becoming worse.

And then Watkins got really lucky. Since this was a test to determine if the drug worked, half of the boys in the test group got nothing, a placebo.

Cameron was one of the fortunate ones who got the drug, and it worked, almost immediately.

"His spleen shrank, his platelet count dropped," Bhakta said. "He would not be with us today if it weren't for this amazing drug."

Doctors decided his heart condition had improved to the point that he didn't need the valve transplant.

He still has problems with his hearing, and his heart can act up unexpectedly. He's had to fight to keep his body in shape to stay out of a wheelchair, but he is able to walk on his own or with a cane.

Sometimes he overreaches and pay the consequences. On a trip with Smith last year to Disney World, he walked on his own with a cane for five hours, but ended up back in his hotel room, exhausted.

"My heart went off," he said, racing out of control. The photo he posted to Facebook from that night shows him on a stretcher as paramedics raced to get him to a nearby hospital.

Given a lifelong history of medical problems, Cameron could be forgiven for feeling a little sorry for himself.

But what is most striking -- both in person and in the comments he posts on Facebook and on his blog, Cameron Lives 365 -- is his optimism.

"I've always lived with this condition," he said. "We all have to live with something, and this is it for me. I'm happy."

He graduated from high school with honors and took some college classes, although he has yet to graduate. He would like to write fiction for young adults, he said, or be a journalist.

A recent blog post seems to summarize his attitude about his life, a life he once thought he might miss out on.

"Maybe the trials in my life somehow helped people get their own perspective in life, and pushed them to reach for their goals," he wrote. "If in my life I achieve anything, I'd like it to be that my life helped someone else's life, and maybe made an impact."

Contact reporter Steve Johnson at sjohnson@timesfreepress.com, 423-757-6673, on Twitter @stevejohnsonTFP and on Facebook, facebook.com/noogahealth.

Copyright 2015 - Chattanooga Times Free Press, Tenn.