Lexington girl set to become one of first kids to get gene therapy for rare disease

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KANSAS CITY, Mo. -- Gene therapy holds huge potential for curing many diseases including cancers and blood diseases. A Lexington girl is ready to become one of the first kids in the world to get gene therapy for an inherited blood disease called beta thalassemia major.

Faith Rockhold was six when she was adopted from Vietnam. The adoption happened quickly.

"The country could no longer provide the resources to give Faith blood," said her mother, Bambi Rockhold.

Now 14, Faith gets two units of blood every two weeks at Children's Mercy Hospital. She must get the transfusions because her body cannot make enough of the protein in blood that carries oxygen throughout the body.

"And if I don't get blood, I'll die," said Faith.

Her mother said, "We worry about her organs. She could have organ failure and die. We worry about medications we have to buy. Just medication that keeps her alive costs $7,000 a month."

So Faith and her family are taking a leap of faith. If she passes medical tests in a few weeks at Chicago Children's Hospital, Faith will become one of the first adolescents to receive gene therapy for the disease.

"The early results are very encouraging for patients," said Dr. Ram Kalpatthi, a hematologist at Children's Mercy.

Dr. Kalpatthi says Faith's own stem cells will be removed. In a lab, a functional gene -- what she's lacking -- will be inserted into them. The gene is carried by a virus. The doctor says of five young adults who've received the therapy, "a couple of patients are free from chronic blood transfusions."

He adds that much longer follow-up is needed to see if they are cured.

Risks include infection since chemotherapy is needed. But Faith and her family are willing to take risks.

"'Cause I don't want to have this anymore. I just want to get over with it," she said.

Faith hopes to be free of transfusions and free of disease.

Faith and her mom will have many expenses in going to Chicago for the therapy. They will need to stay there at least several months. For more information, go to www.gofundme.com/u6hf32a or www.facebook.com/Savingfaithrockhold?fref=ts