Pharmaceutical composition and application thereof in SMO mutant myeloblastoma

• Main Authors: YAO YUELIANG, MAO MIN, BIAN XIUWU, WANG YAN
• Format: Patent
• Language: Chinese; English
• Published: 19.11.2021
• Subjects: HUMAN NECESSITIES; HYGIENE; MEDICAL OR VETERINARY SCIENCE; PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES; SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS

The invention belongs to the technical field of medicine for medullary blastoma, and particularly relates to a pharmaceutical composition based on MK2206 and CX4945 and an application of the pharmaceutical composition in SMO mutant medullary blastoma/cytoma. The pharmaceutical composition contains MK2206 and CX4945. The expression of SMO and/or SUFU and/or PCTH1 and/or PTCH2 in the Hedgehog pathway can be inhibited by combined use of the MK2206 and the CX4945; or the signal conduction of the SMO/AKT/CK2 signal channel can be blocked; or the expression of PDK1 and/or LDHA protein in a glycolytic pathway is inhibited, so that the growth of drug-resistant medullary blastoma/cytoma is effectively inhibited. The pharmaceutical composition solves the problem that the SMO inhibitor generates drug-resistant medullary blastoma/cytoma, and can effectively act on SMO mutant drug-resistant medullary blastoma/cytoma. 本发明属于髓母细胞瘤用药技术领域，具体涉及一种基于MK2206和CX4945的药物组合物及其在SMO突变性髓母细胞/细胞瘤中的应用。所述药物组合物包括MK2206和CX4945。联合使用MK2206和CX4945