A New Mexico toddler is getting a second chance at life after being diagnosed with the ‘Bubble Boy Disease,’ a disease that meant getting sick could cost him his life.
That all changed after a doctor at UNM Hospital helped create a brand new treatment program.
Jayceon Golden was diagnosed with a genetic condition called X-Linked Severe Combined Immunodeficiency Disorder, also known as the Bubble Boy Disease.
“Babies born with an immune defect, a genetic defect due to the genetic mutation,” said Dr. Elif Dokmeci.
Dr. Elif Dokmeci is a Pediatric Immunologist who diagnosed the 2-year-old when he was only a few weeks old.
“They have difficulty fighting infections due to the immune defect they are born with,” said Dokmeci.
Patients with the Bubble Boy disease typically undergo a bone marrow transplant, but Dokmeci said that isn’t always a successful procedure.
“You might not correct immune defect 100 percent. You still might have defective b-cells or still have defective t-cells,” she said.
A new gene therapy procedure that Dokmeci helped create became available, so she sent the 2-year old to the University of California, San Francisco to undergo treatment.
“Gene therapy, you are basically using baby’s own cells. You are correcting the mutation and giving back to baby,” said Dokmeci.
The results were positive.
“We don’t want to use the word ‘cure,’ but you’re basically correcting the abnormality here and you’re curing the disease,” she said.
Doctors at UCSF said it only took a few months for the treatment to start working.
“Three to four months post the infusion not only was he able to go home and start to live a relatively normal life, we were able to take him off complete isolation and all prophylactic medications,” said Dr. Mort Cowan.
Now, Jayceon is back home in Las Cruces, living life like how a 2-year old should.
“He’s a healthy looking, and I will say, healthy toddler,” said Dokmeci.
Right now, there are eight babies who have undergone gene therapy.
Doctors said it’s too early to tell if this will be successful long term, but for now, it seems to be working in all patients.