Allogeneic Hematopoietic Stem Cell Transplant (HCT) is the broader term used for transplants where there are donor cells involved. This includes bone marrow transplant and unrelated umbilical cord transplant.
Bone marrow transplant has improved to a level where it has become a more standard curative treatment for patients whose CGD has led to severe challenges. A study led by Dr. Jennifer Leiding showed that on average transplant has a high rate of success of 82% for CGD patients.
See a transplant success story from CGDAA community member Colton here!
However, patients and caregivers should be aware that there can be issues finding well matched donors, a risk of failure, risk of graft vs host disease, or other complications. Patients should also be aware that if a transplant is unsuccessful, it may cause you to be ineligible for clinical trials or other gene therapy treatments.
Gene therapy for chronic granulomatous disease is an experimental clinical treatment and is not yet a standard of care. However, it can offer a one-time therapy for CGD. A CGD patient’s blood stem cells are collected and a healthy version of the gp91- phox gene is inserted into the stem cells. The patient with CGD undergoes chemotherapy before the cells are intravenously infused. This is where stem cells undergo gene transfer or gene induction leading to lifelong daughter cells that produce the functional protein. After recovery from chemotherapy and the corrected cells have engrafted, the stem cells are potentially able to produce new white blood cells that can fight off infection.
This has been shown to be effective in CGD as a study published in Nature showed that 9 patients who did have durable engraftment with gene therapy for treatment of CGD.
Dr. Donald Kohn, a renowned expert in gene therapy recently shared his cutting edge insights with the CGDAA. Watch the Zoom recording on our Youtube page to see the latest in transformative therapy options for Chronic Granulomatous Disorder (CGD).
No course of action, whether CGD treatment/prophylaxis or curative option, is without risks. It is important to encourage patients with CGD and their families to educate themselves in order to select the path that is right for them
While each patient with CGD is different due to individual levels of NADPH-oxidase activity and form of CGD (as well as other unrelated health or environmental issues) it is recommended that patients be seen and, if necessary, managed proactively by a team of qualified specialists/physicians, who work in concert with a primary care physician. These can include (but are not limited to):
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