Stem Cell Transplant
De Fabritiis P, Petti MC, Montefusco E, et al [ Universita “La Sapienza” Rome, Istituto Regina Elena, Rome; Lynx Therapeutics Inc, Hayward, Calif; et al ]
BCR-ABL Antisense Oligodeoxynucleotide in Vitro Purging and Autologous Bone Marrow Transplantation for Patients with Chronic Myelogenous Leukemia in Advanced Phase
Blood 91: 3156-3162, 1998
The Philaldephia chromosome [BCR-ABL complex] of patients with CML] has unveiled many of the pathogenic mechanisms. Its antisense oligodeoxynucleotides [ODN] have an antileukemia effect, documented so far in vitro studies. This study of 8 patients with CML autografted in an advanced disease stage were purged in vitro with junction-specific [J-sp] BCR-ABL antisense ODN at the time of transformation in accelerated phase or during second chronic phase. Two patients had a complete karyotypic response and other 6 had minimal or no response. This type of bone marrow purging and autograft mediated by ODN has low toxicity satisfactory haematopoietic reconstitution. The editor comments that the authors could bring about a meaningful clinical effect by the above study a clinical effect at stage of CML that is usually refractory to any standard therapy. Antisense strategies have also been developed in other malignant diseases for example, targeting mutated p53 genes.