A 26-year-old Downriver man seen at Children’s Hospital of Michigan since infancy for a rare bleeding disorder this week became the first person in Michigan to receive an FDA-approved, commercially available gene therapy for hemophilia B.

“It is a privilege to be part of this historic advancement, and I look forward to continuing our efforts to bring transformative therapies to our patients and families,” said Children’s Hospital of Michigan Division Chief of Hematology, Dr. Madhvi Rajpurkar, shortly after the successful, hour-long infusion on Tuesday, July 8.

The Wyandotte man’s treatment at Children’s Hospital of Michigan’s Hematology Center could help end his life-long dependency on medical infusions and avoid critically dangerous bleeding episodes.

“As a child, his family had to advocate for him and he had to learn from an early age to advocate for himself,” said Dr. Bulent Ozgonenel, who has been the patient’s hematologist for several years and was instrumental with Children’s Hospital of Michigan social worker Rachel Cain-Kellman in evaluating and preparing the patient for gene therapy. “If he fell or was playing when he was young, or in an activity that any one of us takes for granted, the resulting bleeding could cause him to not walk well, or it could be life-threatening. This advancement in medical science could be life-altering for him.”

Hemophilia patients’ blood lacks a protein – Factor IX, or Factor 9 – that helps with clotting. This genetic mutation, often passed down through families, puts them at risk for spontaneous bleeding, internally or externally, and they can suffer life-threatening bleeding if injured.

Until now, the only treatment for hemophilia has been regular infusions of clotting factor, sometimes multiple times a week, to prevent bleeding from injuries related to things like sports or other physical activity, bumps, bruises or cuts. The Children’s Hospital of Michigan patient had undergone infusions since he was an infant, and he was self-administering infusions three times a week before the July 8 procedure.

“He said he has wanted to get gene therapy since he was 15, but it was only available in trials,” said Children’s Hospital of Michigan Hemophilia Treatment Center Director of Nursing Charity Stadler, part of the team who helped start the Hemophilia gene therapy program at the Children’s Hospital of Michigan. “We’re very happy to be the first center to provide this treatment commercially in Michigan. This is definitely a revolutionary treatment. We’re hoping the very best for him.”

The gene therapy the man received on July 8, HEMGENIX produced by CSL Behring administered by Children’s Hospital of Michigan nurse coordinator Michelle Hyde, could end his infusions completely. The new genes introduced are designed to help the patient’s liver produce the blood clotting agent that was lacking: Factor 9. He will visit the Children’s Hospital of Michigan team weekly for testing to ensure his liver continues to function well, his Factor 9 level is rising and he is not experiencing symptoms of bleeding.

“Congratulations to Children’s Hospital of Michigan on becoming the first institution in the state to commercially administer HEMGENIX,” said Diego Sacristan, Senior Vice President, U.S., CSL Behring. “We’re proud to see this important innovation reaching patients and advancing care.”

Success is achieved when his Factor 9 level reaches 10% – 50% is a sufficient level for most people – and his blood has enough clotting factor to end the prophylactic infusions. After three months, he will be tested monthly, then annually if his first year of monitoring goes well. The only time he would potentially need an infusion to aid clotting is if he suffers an injury.

“This is a game-changer for many patients – young and old,” said Children’s Hospital of Michigan CEO Archie Drake. “From being able to enjoy everything from outdoor activities to cooking – where cuts could be a threat – we are so pleased this has the potential to offer freedom and a sense of normalcy to this patient and potentially many others with hemophilia. The Children’s Hospital of Michigan has a long history of offering life-altering treatments, and this is another wonderful opportunity for our patients.”

Dr. Rajpurkar said she is proud to be part of a long history of bleeding disorder care for patients at Children’s Hospital of Michigan. She said the Jeanne M. Lusher Center for Hemostasis and Thrombosis is named after one of her mentors, Dr. Jeanne M. Lusher, a researcher in the field of blood disorders and director of the Hemostasis Program at the Children’s Hospital of Michigan until she retired in 2013. Dr. Rajpurkar also credits Children’s Hospital of Michigan bleeding disorder pioneer Dr. Indira Warrier as a leader in the field.

“This milestone honors the legacies of Drs. Lusher and Warrier, who shaped my approach to hemophilia care with their unwavering commitment to excellence, compassion and patient-centered innovation,” Dr. Rajpurkar said. “I am grateful for the foundation they laid and proud to carry forward their work here at Children’s Hospital of Michigan — a place where generations of children with bleeding disorders have received world-class care.”

Dr. Meera Chitlur, director of the Hemophilia Treatment Center at Children’s Hospital of Michigan, agreed.

“Children’s Hospital has been a center of excellence in bleeding disorder care with stalwarts like Dr. Lusher and Dr. Warrier, who pioneered some of the early therapies and trials for treatment of Hemophilia,” Dr. Chitlur said. “We are truly in an era of revolutionary scientific advancement and it is humbling to be able to be continue in our mentors’ footsteps and offer this innovative treatment to patients at our center.”