Hematopoietic Stem Cell Transplant (HCT) remains the only potentially curative option for patients with high-risk hematologic malignancies. However, many patients do not achieve long-term remission as approximately 40% of AML patients will relapse after HCT. The prognosis for these patients, particularly those with early relapse, is dismal due to the limited treatment options and the inability of these patients to tolerate the intensive or targeted chemotherapy that would be indicated in this setting.
Following HCT, patients are typically managed via watchful waiting, essentially waiting for the cancer to recur. This is because therapies are often highly toxic to the new bone marrow, which could result in aplasia and put the patient at significant risk. But what if potent targeted therapies could be used safely following HCT? Vor Bio is developing treatment-resistant hematopoietic stem cells (HSCs), potentially allowing the use of targeted modalities such as Antibody Drug Conjugates (ADCs) and Chimeric Antigen Receptor T (CAR-T) therapies in the post-transplant setting without concerns for graft failure or prolonged cytopenia, and could be used prophylactically or therapeutically following detection of marrow blasts.