The Faulty Gene Behind Chroni... Video Transcript

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JANET ROWLEY, MD: Normally, these signals are very intermittent, and well regulated, of course. Now what happens is that these signals are present more constantly and at a higher level, so the cell is receiving false signals to grow and divide in an inappropriate way.

ANNOUNCER: In the mid 1980s, the rouge enzyme was identified as a type called a tyrosine kinase. They were being studied in several labs around the world. One team of scientists sought ways to block their action in CML.

JOHN GOLDMAN, MD: It then became attractive to see whether you could develop basic molecules in the chemical laboratory that would block the action of these kinases. So again, in the early 1990s, a pharmaceutical company in Switzerland, Ciba-Geigy, and many other pharmaceutical companies, started programs of developing small molecules that would inhibit tyrosine kinases. Out of that program came STI-571, signal transduction inhibitor-571, now known as imatinib.

ANNOUNCER: In early tests and later, in clinical trials, imatinib proved very effective against chronic myeloid leukemia. Trials showed imatinib worked well in returning a CML patient's elevated white blood counts to normal, what's called a good hematologic response. What imatinib proved it did better than other drugs was reducing the number of white blood cells shown to contain the Philadelphia chromosome.

JOHN GOLDMAN, MD: The incidence of complete hematological control is probably better with imatinib, but what's really impressive is that the instance of complete chromosomal control, complete elimination of the Philadelphia chromosome in the bone marrow of patients, is now about 70% with imatinib, compared with 10% with interferon-alpha and cytarabine.

ANNOUNCER: Doctors think this good chromosomal control with imatinib, which is now known as Gleevec, may lead to longer survival in many patients. Peter Nowell, David Hungerford, and Janet Rowley, had no way of knowing where their pioneering research would lead. But they contributed tremendously to our understanding of the genetics of at least some types of cancer. And their work led to a drug that is helping many patients with chronic myeloid leukemia.

JANET ROWLEY, MD: I get calls now from patients relating my discovery to their response to Gleevec, the quality of life that they presently have. That's just a marvelous feeling.

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