Brain Druker
Translocation of the Philadelphia chromosome

Imatinib Turns Deadly Leukemia Into a Manageable Disease

The FDA approval of imatinib (Gleevec) for chronic myelogenous leukemia (CML) was not just the fastest in FDA history. It also sparked a new era of research focused on treatments that target a cancer cell’s specific molecular defects – and spare surrounding healthy cells.

Imatinib transformed the outlook for most CML patients, turning a disease with a three- to five-year life expectancy into a chronic, long-term condition, managed with a daily pill. Today, five-year survival rates have reached almost 90 percent.1

The story of imatinib actually began more than four decades earlier, in 1960, when researchers discover an abnormal chromosome – the so-called "Philadelphia chromosome” – is present in many patients with CML. Researchers later found this defect results when parts of two chromosomes switch places, in a phenomenon called translocation. In the late 1990s, Dr. Brian Druker and colleagues developed imatinib to target the abnormal protein produced by this genetic translocation.

Since 2001, research has shown that imatinib is also effective against other pediatric and adult leukemias driven by abnormalities in the Philadelphia chromosome, along with a specific form of gastrointestinal stromal tumors (GIST) that is driven by a mutation in the KIT gene.

Meanwhile, the number of FDA-approved targeted therapies has skyrocketed. More than 60 targeted drugs are available to treat patients with cancer today.2

Voter Comments

“Gleevec not only revolutionized CML, but cancer therapy in general. A targeted agent directed at a cancer-causing gene – it represents the dawn of targeted therapy for cancer.”

“This has transformed the direction of oncology research across the world and has the potential to not only convert cancer into a chronic disease but also cure with the real hope of discontinuing therapy.”

“The tyrosine kinase inhibitor (TKI) era begins and completely changes cancer treatment paradigms across all cancer types.”

“Development of imatinib provided validation of treatments targeting molecular pathways and created a high goal for all other targeted approaches to achieve.”

References

  1. Pray, L: Gleevec: The Breakthrough in Cancer Treatment. Nature Education. 2008;1: 37.
  2. National Cancer Institute, Fact Sheet – Targeted Cancer Therapies

Additional Information

Top Five - #3
Targeted Drug Transforms Treatment for Rare Leukemia, 2001; Dr. Peter Paul Yu, ASCO President