Before we delve into the scientific origins of and the mechanics of the drug named Gleevec, let’s review the diagnosis of CML-Chronic Mylogenous Leukemia. CML is defined as a rare, slowly progressing blood cell cancer that begins in the Bone Marrow. A genetic change in the body’s Myeloid cells caused by a mutation that forms an abnormal gene called the BCR-ABL gene begins turning cells into CML cells. Most people diagnosed with CML have an abnormal chromosome in their DNA called the Philadelphia Chromosome. With CML, the patients Leukemic cells grow and divide rapidly, building up in the Bone Marrow and spilling over into the blood. These cells are very immature and are not suitable for fighting infection. Once the population of these cells become uncontrollable, they can spill into other organs and keep other cells in the body from working properly. Without treatment a patients immune system becomes overtly compromised and eventually enters the phase called a Blast Crisis from which recovery is rare and death inevitable. This cycle untreated can last anywhere from one to four years after diagnoses before succumbing to the Cancer. Past treatments included the drugs Hydroxyurea and Interferon, Chemotherapeutic treatment and eventually a Bone Marrow Transplant. Until the early 1990’s when the Bone Marrow Transplant began to be widely used, other treatments were not curative. Still, Bone Marrow Transplants were not a slam dunk. Patients had a 50/50 chance of making it through the transplant and then a 50/50 chance of it being a cure. Until Gleevec.
In 1996, the year that I was originally diagnosed with CML, a doctor named Brian Druker partnered with the drug company Novartis to test compounds that Novartis had been creating called “Tyrosine Kinase Inhibitors”. These compounds were created to stop the uninhibited growth of the proteins that trigger the CML cells to divide uncontrollably. The proliferation of these Tyrosine kinase proteins causes the overproduction and accumulation of immature white blood cells, the hallmark of CML. After 30 years of scientific research, Dr. Druker focused on one particular compound that would eventually become STI-571. In the first targeted FDA trial of STI-571 conducted in 1999, 31 patients were enrolled and 31 out of 31 experienced complete remission. This was virtually unheard of. Being well tolerated, the drug entered a second phase that showed the same results as Phase 1. I was luckily a recipient of STI-571 in the second phase. This would save my life. As I stated in my last blog, the drug would go on to be one of the fastest drugs ever approved for general use to the public. I entered the trial in October of 2000 and the drug was approved by the FDA in April of 2001.
As far as I am concerned, Dr. Brian Drucker is deserving the title of American Hero. His dedication for so many years to the search for a cure to this devastating cancer has saved thousands of lives and relieved so many, the hardships of the devastating treatments needed to survive CML. Now, the frontline treatment for treating the disease is compromised of simply digesting one or two pills a day, side effects withstanding. The discovery of Gleevec has subsequently led to the development of additional stronger Kinase Inhibitors that are used for patients not able to achieve a lasting remission on Gleevec.
Gleevec is indeed a miracle drug. It has made me a living miracle. I owe my life to Dr. Druker and his team as well as my other Doctors, especially Dr. Dave. To this day, I can email or call Dr Druker in Portland, Oregon and he will get back to me personally and quickly. Unbelievable.
Next up: The Philadelphia Chapter of the CML Club