When Mel Mann was diagnosed with chronic myeloid leukemia at 37, doctors told him he had about three years to live. It was January 1995. An Army major in Detroit, Mann had chalked up persistent back pain to working out until an MRI revealed trouble in his bone marrow. Facing mortality, he worried about his young daughter and volunteered for every clinical trial available. Some experimental drugs helped for a short time, but none changed the course of his disease — until he enrolled in a trial for a new drug in August 1998.
Within a year of starting that drug, imatinib (later sold as Gleevec), Mann had regained his strength and was running a marathon in Anchorage. His turnaround was dramatic, and his story became one of many that illustrated a profound shift in cancer treatment: the arrival of targeted therapies that aim at the molecular causes of cancer rather than broadly poisoning rapidly dividing cells.
The idea behind that shift was championed decades earlier by oncologist Brian Druker. In the 1970s and ’80s, Druker questioned the prevailing approach of using highly toxic chemotherapies with uncertain benefit. He believed a better path would be to understand and block the specific molecular drivers that make cancer cells grow uncontrollably.
Chronic myeloid leukemia (CML) presented a clear target. Many patients with CML carry the Philadelphia chromosome, a genetic abnormality that produces an abnormal enzyme keeping a growth switch stuck “on,” causing excessive white blood cell production. If that enzyme could be turned off, the disease might be controlled.
Druker moved to Oregon Health & Science University to pursue that idea. Soon after arriving he began testing compounds supplied by a company that would become Novartis. One compound, imatinib, discovered by biochemist Nicholas Lydon, inhibited the abnormal enzyme in the lab. That laboratory success raised hope but did not guarantee patient benefit.
Druker pushed for clinical trials designed specifically for patients with the Philadelphia chromosome–positive form of CML. Because the drug targeted the abnormal enzyme, the trials enrolled only patients with that molecular signature. Results were striking: within months, patients receiving adequate doses experienced dramatic responses and side effects were generally mild compared with traditional chemotherapy. Word spread quickly among patient communities, and demand for enrollment grew worldwide.
To get the medicine to patients more quickly, the drug company had to collect and present data to regulators. A young biostatistician, Insa Gathmann, joined the project at Novartis and became the statistical lead. Early survival analyses she ran showed survival curves that were unexpectedly strong. At first she assumed a programming error; the results were simply that good.
The Food and Drug Administration approved imatinib on May 10, 2001. The agency’s review was quick — 72 days — reflecting the clear-cut responses and tolerability profile seen in trials. For clinicians and researchers involved, approval felt like sending a promising child out into the world.
Gleevec’s approval signaled more than a new treatment for one leukemia. It ushered in an era of targeted cancer drugs, therapies designed around the specific molecular abnormalities driving particular tumors. Today there are well over a hundred targeted cancer therapies on the market, and many have meaningfully improved survival and quality of life for patients with cancers once considered uniformly fatal.
The Gleevec story also exposed tensions in the system that bring drugs to patients. At launch, Gleevec’s annual cost was about $26,000 — considered extremely expensive at the time — and prices rose over the years, prompting debates about affordability and access even as the drug delivered extraordinary clinical benefit. Since then, generic versions have become available and costs have dropped to a few hundred dollars a year for many patients, making the drug far more accessible.
Clinicians still prescribe Gleevec for patients with CML, and outcomes have been transformed. Some patients who began treatment as children have been able to stop the drug without recurrence and go on to lead full adult lives, raise families and reach milestones that would once have been unimaginable.
Insa Gathmann remained at Novartis and rose through the ranks; she reflects on Gleevec as a rare career moment. Druker has watched patients given a death sentence go on to experience weddings, graduations and grandchildren. Mel Mann, once told he had three years left, is now in his late 60s, finished another degree, and plans more marathons.
Gleevec’s 25th anniversary is a milestone that highlights both the scientific triumph of translating molecular understanding into an effective therapy and the broader lessons about how drugs are developed, priced and made available. It remains a powerful example of what can happen when research, clinical advocacy, industry capacity and patient communities converge to accelerate a game-changing medicine from the lab to the clinic.