Karmanos Cancer Institute Becomes First Independent Cancer Center to Offer Hemophilia B Gene Therapy
Key Insights
The Barbara Ann Karmanos Cancer Institute has become the first and only independent cancer center in the U.S. to provide Etranacogene dezaparvovec-drlb (Hemegenix (search)®), a one-time gene therapy for adults with hemophilia B (search).
Five-year clinical study results demonstrate that 94% of patients discontinued routine factor IX prophylaxis after receiving the gene therapy treatment.
The therapy works by delivering the factor IX gene through a viral vector that travels to the liver, enabling patients to produce their own clotting protein.
The Barbara Ann Karmanos Cancer Institute has achieved a significant milestone by becoming the first and only independent cancer center in the United States to provide Etranacogene dezaparvovec-drlb, also known by its brand name Hemegenix (search)®. This FDA-approved one-time gene therapy represents a revolutionary treatment approach for adults with hemophilia B (search), a rare blood-clotting disorder.
Steve W. of Clinton Township became the first patient to receive the therapy at Karmanos Cancer Center in Detroit on October 28, 2025, marking him as the 52nd patient treated in the U.S. following clinical studies. "I'm just very blessed to be in the right spot at the right time with the right people," Steve expressed. "And it's working. I didn't have any problems the first few days… I did have some days where I was just tired, but about a week after the infusion, I was good."
Understanding Hemophilia B
Hemophilia B (search) is a rare blood-clotting disorder caused by a deficiency of factor IX, a clotting protein. Patients with this condition may experience excessive bleeding after injury or surgery, with severe deficiency potentially resulting in spontaneous bleeding, particularly to the joints. The condition can be life-threatening in cases of severe injury or surgery without proper therapy.
"Although most hemophilia is hereditary, sometimes patients can be diagnosed with this disease without a family history," explained Indryas Woldie, M.D., a hematologist and medical oncologist who specializes in benign hematological diseases. Dr. Woldie leads the Hemophilia and Other Bleeding Disorders Clinic and is a member of the Hematology Oncology Multidisciplinary Team at Karmanos.
The condition predominantly affects males, as they inherit the X chromosome from their mothers. "Males inherit the X chromosome from their mothers, and if it is hemophilic, they will have hemophilia," Dr. Woldie noted. While female carriers may not have severe disease manifestations, they can still experience bleeding, particularly during menstrual cycles, and require proper treatment during surgery and injuries.
Revolutionary Gene Therapy Mechanism
The gene therapy works through a sophisticated delivery system. "The gene coding for factor IX is placed into a vector containing an inactive viral capsid. We then infuse it into the patient intravenously, where the vector preferentially travels to the liver and helps it make the deficient protein, factor IX," said Dr. Woldie.
Patients are monitored during and after the infusion for at least three hours to ensure there are no infusion-related reactions. Potential symptoms during this monitoring period may include elevated levels of specific blood enzymes, fatigue, flu-like symptoms, headache, infusion-related reactions, liver enzyme elevations, and nausea.
"Patients will start making their own factor IX from the first few weeks of infusion up to six months and longer," Dr. Woldie explained. "There have been good long-term results of patients having a factor level that precludes the use of prophylaxis."
Impressive Clinical Results
The clinical data supporting this gene therapy is compelling. According to CSL (search), the company that manufactures Hemgenix, 94% of patients in the international clinical trial discontinued their routine factor IX prophylaxis. Remarkably, at the five-year mark after treatment, 94% of those patients maintained mild to normal factor IX levels.
This represents a dramatic shift from the current standard of care, which requires lifelong, routine infusions of factor IX replacement therapy (prophylaxis) to help reduce bleeding risk. Before receiving the gene therapy, Steve had to inject himself once a week with prophylaxis to increase his factor IX count in his blood. Now, after receiving the treatment, he doesn't require routine treatment unless he needs a procedure.
"I think it's exciting for him. It's a thing – the kitchen table on Saturday mornings, he'd rub it down with alcohol and spread out all his gear," shared Alison, Steve's wife. "Now, he doesn't have to do that unless he has to have surgery or was in an accident that causes trauma."
Rigorous Preparation and Training
The implementation of this gene therapy required extensive preparation. About six months before Karmanos became a treatment site, healthcare teams including pharmacists, physicians, and nurses completed rigorous training to ensure safe and effective infusion administration.
"My team is excited to be able to offer this gene therapy for patients," said Surender Kanaparthi, R.Ph., MBA, DPLA, FACHE, chief pharmacy officer at Karmanos Cancer Hospital. "Our mission has always been to expand access to the most advanced treatments available, and gene therapy is the future of hematology and oncology care."
The eligibility process involves comprehensive testing requirements to ensure patients are suitable candidates and that their liver is functioning properly. "There are many testing requirements before offering Hemgenix to ensure patients are eligible and that their liver is functioning properly. For the patient, once it is determined that they are eligible, administering it is relatively simple," explained Kanaparthi.
Patient Eligibility Criteria
Hemophilia B (search) patients eligible for this one-time therapy must meet specific criteria: they must currently use factor IX prophylaxis therapy and have current or historical life-threatening bleeding, or have experienced repeated, severe spontaneous bleeding episodes.
"When we see our patients, we talk to them about new treatments approved by the FDA, as well as available clinical trials. If they are interested in any of those options, we give them more information and spend time discussing the pros, cons and adverse effects that could occur, as well as eligibility criteria," said Dr. Woldie.
This milestone achievement by Karmanos Cancer Institute represents a significant advancement in hemophilia B (search) treatment, offering patients the potential for freedom from lifelong prophylactic treatments while maintaining effective factor IX levels for years following a single infusion.