MIAMI (Ivanhoe Newswire) -- More than a million people are currently
enrolled in clinical trials across the country, all hoping experimental
medicine will cure them. For a baby battling a deadly disease, turning to a
trial is scary, but his family says taking the risk saved his life.
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
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
FOR MORE INFORMATION, PLEASE CONTACT:
Omar Montejo, Director of Media Relations
University of Miami School of Medicine