Kael and Moci

Moci and Kael were adopted from an orphanage in China by their parents Mandy and Chris. Both have beta thalassemia major, a genetic blood disorder in which the body’s does not make enough hemoglobin, thereby reducing the amount of oxygen in the red blood cells, requiring regular blood transfusions and iron chelation therapy. Beta thalassemia can be quite common in China, affecting as many as one in every five people.

Since being adopted, Moci and Kael have had blood transfusion appointments at Nationwide Children's Hospital every three weeks, which last six to eight hours. The siblings also take daily medication to counter the side effects of frequent blood transfusions.

“We are so grateful to have access to health coverage and great doctors nearby to provide our kids with the treatment they need to live healthy, long lives. We are very thankful for the doctors and nurses from Nationwide Children’s who have supported us through our journey and will continue into the future,” says Mandy.

An incredible breakthrough was made for beta thalassemia patients earlier this year. The FDA approved a gene therapy cure for the disease. Beginning their therapy this coming summer, Moci and Kael will be some of the first few patients at Nationwide Children’s to undergo the treatment here at the Jerry R. Mendell Center for Gene Therapy at Nationwide Children’s.

Recently, Moci and Kael were in the hospital for the first step of the therapy: the collection of stem cells. Those cells are being edited to mutation to increase the production of oxygen-carrying fetal hemoglobin. Once ready, the edited cells will be transplanted back into Moci and Kael. These healthy stem cells will help their blood produce the increased amount of amount of oxygen-carrying fetal hemoglobin.

Both nerve-wracking and exciting, the siblings are looking forward to no longer missing school, time with friends, practices, recitals and competitions.

“I have been in awe at the ability of Nationwide Children’s to set up the infrastructure necessary to make this gene therapy happen,” Mandy shares. “I can’t say enough about Nationwide Children’s, specifically the nurses and doctors we get to work with.”

Moci and Kael have three other siblings, Indigo, Crew and Meilin. Meilin was also adopted from China and will begin her beta thalassemia therapy when she’s old enough.

“My hope for them is to be able to do whatever they want in life, that they are not restricted by their need for blood transfusions,” says Mandy. “I hope they can be whatever they want to be.”

When you give to Light Up a Lawn, Light Up a Life, you are supporting programs like Nationwide Children's cutting-edge gene therapy that can change the lives of patients like Moci, Kael, and soon Meilin.