Clinical Trials Saving Kids

MIAMI In just three months, Joshua Pelling went from a healthy baby to an infant struggling to survive.

"He was breathing 100 times per minute," Joshua's mother Leticia told Ivanhoe.

Doctors told his parents their son didn't have an immune system. It's referred to as the "bubble boy" disease. Without treatment, a child usually won't survive past age 2.

"He actually almost died on us several times," Leticia said.

Desperate to save him, the Pellings turned to an experimental procedure. Joshua was given chemotherapy for eight days followed by a stem cell transplant from his mother.

"If you give chemotherapy you make it easier for the new cells to grow," Gary Kleiner, M.D., Ph.D., a pediatric immunologist at the University of Miami School of Medicine in Miami, Fla., explained to Ivanhoe.

His body responded, but after two weeks his liver failed. Blood was flowing in the wrong direction and he was at risk for a deadly clot.

"His liver took a hit from the transplant itself and he was very, very sick," Dr. Kleiner said.

Once again, the Pellings turned to a clinical trial. A drug not yet approved by the FDA restored blood flow. Today, Joshua is a healthy little boy.

"Joshua, I can tell you, is cured," Dr. Kleiner said.

Experimental medicine gave the Pellings a new appreciation for life. They're a family who took a chance and won.

The disease baby Joshua had is usually passed down through genes. Doctors can identify babies with the disorder before they're born by testing the placenta or amniotic fluid. Most are diagnosed in the first six months of life.


Omar Montejo, Director of Media Relations
University of Miami School of Medicine
(305) 243-5654


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