Autologous bone marrow transplantation with alkyl-lysophospholipid-purged marrow

Autologous bone marrow transplantation (ABMT) appears to offer clinical benefit to leukemia patients, but the major reason for failure is relapse. This may be related to the presence of residual leukemic cells in the harvested marrow. To circumvent this problem, various procedures have been developed to purge ex vivo residual leukemic cells from the marrow. The alkyl-lysophospholipids are a new group of anticancer drugs that target membranes as their major site of action. They are unique in that they are relatively selectively toxic to neoplastic cells and spare normal marrow stem cells and progenitor cells. The most active compound is edelfosine. Twenty-nine patients with acute leukemia in second or subsequent remission or early relapse or in first remission, either with a history of treated extramedullary relapse, or requiring more than one induction program to achieve remission, underwent ablative therapy followed by infusion of autologous marrow which had been purged by a 4-hour exposure to edelfosine prior to cryopreservation. Thirty-one percent of the patients are alive and free of leukemia for a median of 630 days (range 185-1,613). These results in this high-risk group of patients warrant further investigation.