Blueprint Medicines announced that the FDA has approved AYVAKIT™ (avapritinib) for the treatment of adults with unresectable or metastatic gastrointestinal stromal tumor (GIST) harboring a platelet-derived growth factor receptor alpha (PDGFRA) exon 18 mutation, including PDGFRA D842V mutations.
Blueprint Medicines presented new preclinical data on its drug candidate, BLU-285, and its potential value in treatment-resistant GIST and on novel cancer drug targets at the AACR (American Association for Cancer Research) Annual Meeting [...]
The Gleevec for GIST story continues to remain one of the most successful stories in oncology. What could be better than a drug that works in 85 percent of patients? As great as this story is, there is room for improvement. Half of the patients on Gleevec for metastatic disease will progress in the first two years and 15 percent of patients are resistant to Gleevec right away or within the first six months of treatment. AROG Pharmaceuticals may have an option for a few of those patients.
UPDATE - January 6th, 2011 by Jerry Call, LRG Science Coordinator The first ever clinical trial specifically for the PDGFRA D842V mutation has been announced by Arog pharmaceuticals. The phase II trial is scheduled [...]
1. Agaram NP, Besmer P, Wong GC, Guo T, Socci ND, Maki RG, DeSantis D, Brennan MF, Singer S, DeMatteo RP, Antonescu CR: Pathologic and Molecular Heterogeneity in Imatinib-Stable or Imatinib-Responsive Gastrointestinal Stromal Tumors. [...]
This is part one of a two-part series on “KIT and PDGFRA kinase mutations in GIST: from A to Z”. In this newsletter, Dr. Heinrich will provide a background on the role of kinase mutations in GIST, focusing largely on the biological and clinical implications of these mutations. In part two (which will be featured in the July 2007 edition of the newsletter, following the special “Five-year anniversary” edition),