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ASH 2024 | Briquilimab-based conditioning for patients with Fanconi anemia receiving alloSCT
Agnieszka Czechowicz, MD, PhD, Stanford University, Stanford, CA, comments on the use of briquilimab antibody-based conditioning in patients with Fanconi anemia receiving allogeneic stem cell transplantation (alloSCT). Dr Czechowicz notes that traditional conditioning regimens, including total body irradiation (TBI) and busulfan, have high toxicities. She highlights the promising results of a clinical trial using briquilimab to replace genotoxic TBI and busulfan, achieving rapid engraftment and resolution of bone marrow failure without treatment-related adverse events. This interview took place at the 66th ASH Annual Meeting and Exposition, held in San Diego, CA.
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Transcript (AI-generated)
So Fanconi anemia patients are a unique patient population that have DNA repair deficiencies that lead them to develop bone marrow failure and myeloid malignancies in particular. These patients commonly are treated with allogeneic stem cell transplants today which resolve the bone marrow failure, although unfortunately the way that these transplants are performed still have high toxicities. And in particular for patients who don’t have matched sibling donors, we still use total body radiation and busulfan in the conditioning regimen of these patients...
So Fanconi anemia patients are a unique patient population that have DNA repair deficiencies that lead them to develop bone marrow failure and myeloid malignancies in particular. These patients commonly are treated with allogeneic stem cell transplants today which resolve the bone marrow failure, although unfortunately the way that these transplants are performed still have high toxicities. And in particular for patients who don’t have matched sibling donors, we still use total body radiation and busulfan in the conditioning regimen of these patients. While these types of transplants can have excellent outcomes with resolution of the bone marrow failure, the conditioning agents do lead to toxicities, both short-term and long-term toxicities, which are exaggerated in these patients that have DNA repair deficiencies and have a hard time recovering from the use of genotoxic agents. So together with colleagues, we initiated clinical trials several years ago that is attempting to replace the use of total body radiation and busulfan in the conditioning regimen of these patients, instead using a briquilimab agent, which is an anti-CD117 monoclonal antibody agent, in place of that genotoxic TBI and busulfan. The idea behind this conditioning agent is that it depletes host hematopoietic stem cells, creating space in the bone marrow for the donor stem cells to engraft. And in combination with using that antibody with an immunosuppression backbone that consists of ATG, fludarabine, cyclophosphamide, rituximab. We’ve launched this clinical trial to be able to treat patients with haploidentical grafts using alpha-beta T-cell depletion, specifically Fanconi anemia patients who are in bone marrow failure. And we’ve really had excellent outcomes showing rapid engraftment of these patients with resolution of their bone marrow failure without any treatment-related SAEs related to the briquilimab antibody. And in our poster presentation here, we are specifically interrogating the effects of the antibody and the transplant on these patients through extensive corollary studies which are presented.
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Disclosures
Magenta Therapeutics: Divested equity in a private or publicly-traded company in the past 24 months, Patents & Royalties; Rocket Pharma: Research Funding; Prime Medicine: Consultancy, Current equity holder in publicly-traded company, Membership on an entity’s Board of Directors or advisory committees; Spotlight Therapeutics: Consultancy, Current holder of stock options in a privately-held company; Teiko Bio: Current holder of stock options in a privately-held company; Global Blood Therapeutics: Divested equity in a private or publicly-traded company in the past 24 months; Jasper Therapeutics: Patents & Royalties, Research Funding; Editas Medicine: Current equity holder in publicly-traded company, Patents & Royalties: Patent, no royalties; Decibel Therapeutics: Divested equity in a private or publicly-traded company in the past 24 months, Patents & Royalties: Patent, no royalties; Beam Therapeutics: Current equity holder in publicly-traded company; Inograft Therapeutics: Consultancy, Current holder of stock options in a privately-held company, Membership on an entity’s Board of Directors or advisory committees, Patents & Royalties; STRM.Bio: Research Funding.
