Targeting a specific gene.

Leukaemia is a cancer of the bone marrow and blood. It is marked by an uncontrolled build-up of blood cells that eventually stops the body from producing healthy blood. Every year, more than 250 000 people are diagnosed with leukaemia worldwide and approximately 66 000 people in Europe. Treatments against leukaemia have improved greatly since the disease was first identified in 1845. But even today, aggressive therapies can take a heavy toll on patients. A more benign treatment against chronic myelogenous leukaemia (CML) arrived in 1999 with a drug named Imatinib. It was developed by American oncologist Brian Druker and Swiss medicinal chemist Jürg Zimmermann. The drug's biggest advantage is that it specifically targets diseased cells while leaving healthy cells unharmed.

Swiss researcher Jürg Zimmermann was recently awarded “European Inventor of the Year” prize, along with his US colleague Brian Druker, for the discovery of a treatment against leukaemia. Working on the molecular structure of leukaemia cells, Jürg Zimmermann and his team created a potent and specific inhibitor of the enzyme that enables malignant cells to multiply. Pyrimidine analogue Researchers began looking for inhibitors that would stop BCR-ABL activity. In the mid-1980s, a team led by American oncologist Brian J. Druker at the Oregon Health and Science University developed a promising compound called STI571. In the early 1990s, Druker refined the compound together with Swiss medicinal chemist Jürg Zimmermann at pharmaceutical company Novartis. Finally, the two succeeded in creating a highly targeted inhibitor called Imatinib. Inside the body, Imatinib locates the site where BCR-ABL communicates with other cells. It inhibits signalling activity by sticking to the responsible receptors. Ultimately, this prevents BCR-ABL from stimulating overproduction of white blood cells. The first clinical trials for the drug, marketed by Novartis as "Glivec", began in 1999.

The results were astonishing: 98 percent of patients treated with Glivec experience complete remission. The cure rate in patients who later underwent a bone marrow transplant is at 60 to 80 percent. And the best news: Less than 5 percent of patients experience adverse side effects. By only targeting specific cells, Glivec is a landmark success in treating leukaemia without the adverse side effects of generalised chemotherapies Chronic myelogenous leukaemia (CML) was long-considered one of the deadliest forms of cancer, capable of striking at any time, causing extreme pain and worse still, affecting both adults and young children. Before the work of two pioneering medical researchers, a diagnosis of CML and subsequent attempts at treatment guaranteed prolonged pain and no certainty of remission. But now with Glivec, a cancer fighting drug with a 98 percent remission rate, CML has lost much of its former bite thanks to American oncologist Brian Druker and Swiss medicinal chemist Jürg Zimmermann.