The Philadelphia Chromosome: A Mutant Gene and the Quest to Cure Cancer at the Genetic Level
Just finished reading a fascinating book: It tells the story of 50 plus years of collaboration between researchers, academics, pharmaceutical giants and clinicians resulting in Gleevec, a drug that so dramatically improved on previous cancer treatments for Chronic Myeloid Leukemia (CML) that it set a record for the fastest approval ever received from the FDA!
This was the world’s first drug targeted against a SPECIFIC mutant protein that stopped cancer in its tracks and started a revolution in targeted therapy and personalized medicine!
Jessica Wapner’s narrative is gripping and seems very well researched!
A few learnings: Cancer is a genetic disease (Dr. Siddhartha Mukherjee’s book “The Emperor of All Maladies” discusses this in detail).
The Philadelphia chromosome results from a translocation of chromosomes 9 and 22, producing a shorter than normal chromosome 22 with this fused mutant gene called bcr/abl. This shorter chromosome 22 is named after the city of Philadelphia where the researchers at U Penn and Fox Chase Cancer Center first discovered it in CML patients.
This bcr/able mutant gene encodes the protein that signals the CML cells to grow and divide out of control resulting in cancer.
Gleevec targets this protein and specifically inhibits the signal pathway to prevent CML.
Loved reading it!