First gene therapy for hemophilia in Indiana
INDIANAPOLIS (WISH) — A 42-year-old man has made history as the first person in Indiana to undergo gene therapy for hemophilia B.
Doctors say it’s a medical breakthrough in the state.
Hemophilia B is a rare genetic disorder that affects 1 in 20,000 males, preventing normal blood clotting. For Lyle Yoder, who has lived with it his entire life, the chronic bleeding disorder has dramatically affected his physical abilities and quality of life.
“I came down here feeling great, and I feel great this morning, and it’s been interesting. Usually, from a procedure, you usually find that you feel something. I don’t feel any different than I did before,” Yoder said.
The procedure happened at Innovative Hematology on the north side of Indianapolis.
“Looking back, I’ve realized that I was being careful even in my younger years. Before I was 10, I remember not doing some things because it hurt, and I didn’t realize why. Once I understood that I had hemophilia, it made sense,” Yoder said.
The gene therapy, that was approved by the U.S. Food and Drug Administration just two years ago, aims to address the root cause of hemophilia B by enabling the body to produce the missing clotting factor.
Dr. Anthony Betadal, adult hematologist-oncologist at Innovative Hematology, said, “The way that this gene therapy works is it’s a virus that we take the viral DNA out of and put in hemophilia protein, factor IX, and trick the body into making factor IX when it wasn’t really doing so before.”
Innovative Hematology spent nearly six months preparing for Yoder’s procedure, putting him through a battery of tests — including physical exams, blood work, liver scans, and psychosocial assessments — to ensure he was well-prepared for this one-time infusion.
The doctor said, “This is providing an option to get patients off of prophylaxis, especially for people like Lyle who have hemophilia B.”
“There isn’t a great option outside of using IV factor once a week or once every few days,” Betadal said.
After years of managing his condition with demanding weekly infusions of clotting factors, Yoder says he feels the gene therapy treatment could transform his life. “One thing we love to do is go to Florida. We couldn’t take the family to the beach because I was in pain. Even though I was on prophylaxis, I still had knees that were swollen tight and didn’t feel like walking. My normal activities … that’s what I’m looking forward to: to be able to work out and not hurt three weeks after.”
Yoder’s journey marks a new era for hemophilia treatment in Indiana. He said, “There’s a lot of risks when it comes to doing gene therapy … though I think the benefits are going to outweigh anything I will have to sacrifice or let go of for now.”