
At one time, patients with blood cancers were treated with harsh drugs, like interferon or hydroxyurea, yet only two to three percent would ever achieve any sort of remission. Many would suffer such extreme side effects from these drugs they would stop taking the medication early, decreasing even further their potential odds for survival.
The fate of these patients is changing. And the proof is in print -- in today's issue of the
New England Journal of Medicine.
It all began with the study of a highly targeted molecular therapy called STI571 -- designed to block the genetic aberration that gives rise to chronic myeloid leukemia (CML), a disease that affects about 6,000 Americans every year. A clinical trial followed, and a compound marketed by the drug company Novartis emerged. Today, this compound is know as Gleevec.
In the clinical trial of Gleevec, 1,106 CML patients were randomly chosen to receive either Gleevec or Interferon. Early results were so encouraging that all but three percent of the participants using Interferon switched to Gleevec. Five-year survival rates were 89 percent. And 93 percent of patients saw no progression to the acute phase of the disease. Many patients witnessed their blood counts return to normal, and a large number experienced a reverse in the gene mutation that causes CML. Virtually no one reported side effects while using the drug.
Despite a rare reaction that can cause heart failure, Gleevec has now been approved by the FDA for the treatment of six other rare, life-threatening disorders. And other drugs similar in nature to Gleevec are hitting the scene. Some believe long-term suppression of CML will come from a cocktail of these types of drugs.
For now, Gleevec -- on its own -- is a success story.