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Pulmonary reactions to chemotherapeutic agents: the ‘chemotherapy lung’
Published in Philippe Camus, Edward C Rosenow, Drug-induced and Iatrogenic Respiratory Disease, 2010
Fabien Maldonado, Andrew H Limper
Busulfan is an alkylating chemotherapeutic agent essentially used in the treatment of myeloproliferative disorders. This agent was discovered in 1961 and was the first chemotherapeutic agent found to be responsible for significant lung toxicity, as initially reported by Oliner et al. that same year.31 The incidence of busulfan-induced pulmonary toxicity is estimated at about 6 per cent overall, with a wide range of 2.5–43 per cent.32 The reported mortality is extremely high, approximating 80 per cent in some series.17 There is no known effective treatment for busulfan lung toxicity.
Genome Editing and Gene Therapies: Complex and Expensive Drugs
Published in Peter Grunwald, Pharmaceutical Biocatalysis, 2020
Recent results of a phase 1,2 safety and efficiency study about a lentiviral gene therapy combined with low-dose busulfan in eight infants with an average age of 3.5 months and a SCID-X1 diagnosis raises hope of cure; for none of these patients a matched sibling donor was available so that a bone marrow transplantation was excluded. Mamcarz et al. (2019) and colleagues from the University of San Francisco and the St. Jude’s Children’s Research Hospital in Memphis, Tennessee employed a second generation codon-optimized SIN-LV (C12044-EF1α-hycOPT) vector (De Ravin et al., 2016) for transferring the IL2RG DNA to harvested bone marrow; to gamma-retroviral (yRV) gene a self-inactivating element (SIN) was added in order to avoid that nearby genes become activated and convert the cells to be treated to tumor cells. Prior to infusion of the transduced cells, busulfan was administered in an individualized dose. Non-myeloablative Busulfan conditioning was included due to other findings according to which only a small part of patients showed reconstitution of functional B cells after hematopoietic stem-cell transplantation; about non-myeloablative (NMA) regimens in connection with allogeneic hematopoietic cell transplantation (HCT) in the treatment of hematologic malignancies, see Storb and Sandmaier (2016) or Blaise et al. (2010). Busulfan (FDa-approved in 1999) is used in combination with other cytostatic drugs as a conditioning agent in connection with bone marrow transplantation for the treatment of myeloproliferative diseases such as myelogenous leukemia (CML); nowadays it is often substituted by the significantly more expensive tyrosine kinase inhibitor Imatinib that is on the World Health Organization’s List of Essential Medicines.
Overview of biological mechanisms of human carcinogens
Published in Journal of Toxicology and Environmental Health, Part B, 2019
Nicholas Birkett, Mustafa Al-Zoughool, Michael Bird, Robert A. Baan, Jan Zielinski, Daniel Krewski
Busulfan (1,4-butanediol dimethane sulfonate) is a direct-acting bifunctional alkylating agent which has been widely used for the treatment of chronic myeloid leukemia prior to the introduction of Imatinib. Busulfan produces acute myeloid leukemia.