After a baby became the world's first CRISPR gene editing therapy patient, CHOP wants to expand treatment to others
One year ago, a baby from Delaware County, Pennsylvania, became the first person in the world to receive a new revolutionary therapy at Children's Hospital of Philadelphia.
"We didn't know that we were gonna reach his first birthday, if I'm being honest," Nicole Muldoon, KJ's mother, said. "Now we're seeing him crawl. Now he's walking."
A year ago, KJ, who was born with a rare, life-threatening genetic disorder, became the first person in the world to receive a personalized gene editing therapy at CHOP.
"This is cutting edge at the forefront of something that no one has done before," Dr. Rebecca Ahrens-Nicklas, of CHOP, said.
The CHOP team used something called CRISPR, which is able to identify and edit the mutated gene.
"In doing what we did for KJ, it opened the door to potentially helping hundreds, thousands, eventually millions of kids and adults around the world," Dr. Kiran Musunuru, of CHOP, said.
Hoping to expand the treatment opportunities, the Muldoon family and CHOP doctors were recently on Capitol Hill to share KJ's story with lawmakers.
"We really feel now that we have a responsibility to the world," Musunuru said.
Doctors said growing the technology depends on more funding for pediatric research and policies that expand access to personalized gene therapies.
"We have made it our mission to figure out ways of being able to scale this so that more rare disease patients have access to this transformational technology," Ahrens-Nicklas said.
KJ's parents are thrilled to see their little boy thriving and want others to have the same opportunity to get the life-saving therapy.
"When it comes to getting funding, this is a bipartisan issue," Kyle Muldoon, KJ's father, said. "There are no politics in saving your kid's life."
KJ isn't considered cured, but doctors said he's doing very well and will continue to be monitored closely to track long-term outcomes of the CRISPR technology.