Engineering Stem Cells to Withstand Targeted Therapies

Current treatment of acute myeloid leukemia generally consists of chemotherapy followed by a hematopoietic stem cell transplant to eliminate any remaining cancer cells from the bone marrow. Even though outcomes remain poor with around 40 percent of transplanted patients experiencing cancer relapse, and patients who do relapse having two-year survival rates of less than 20 percent, the approach has changed little in 40 years. One reason is that newly transplanted patients are unable to receive targeted cancer therapies since those therapies would be toxic to the fragile transplanted stem cells. Vor Bio’s solution is to edit the stem cells prior to transplanting them so they lack the receptors targeted therapies attack. We spoke to Robert Ang, president and CEO of Vor Bio, about the company’s platform technology, how it works, and why it has the potential to change outcomes for patients with AML and other hematologic malignancies.