In the 1990s, Dr. Druker, who does research at Oregon Health & Science University, was part of a Novartis team that helped develop Gleevec, a “miracle drug” that soundly defeated a previously deadly type of blood cancer called chronic myeloid leukemia, or CML.
The team found the cancer’s cells contained a gene that produced an enzyme that allowed them to slip their genetic leash and run amok. The scientists used this information to engineer the Gleevec molecule, which jams up the enzyme and disables it.
Before Gleevec, “the only other hope was a bone-marrow transplant for younger patients,” Druker told the New York Times. “The problem there was that the death rate in the first year was 25 to 50 percent.” Scientists estimate that because of drugs like Gleevec, there could be 180,000 people living with CML by 2050.
“Druker jokes that he has achieved the perfect inversion of the goals of cancer medicine: his drug has increased the prevalence of cancer in the world,” writes oncologist Siddhartha Mukherjee in his Pulitzer Prize-winning book The Emperor of All Maladies: A biography of Cancer. (The book is also the basis for Ken Burns’ eponymous three-part cancer documentary, which started airing on PBS in the U.S. this week.)
Besides launching a revolution in cancer treatment, Gleevec was also one of the first “precision” drugs tailored to a cancer’s or a patient’s genetic makeup. These drugs aim for specific targets, rather than just attacking cells at random. Such “precision medicine” is now the focus of a new $215 million research initiative launched this year by U.S. President Barack Obama. “There is no question we can defeat cancer,” Dr. Druker writes on his website. “What it requires is knowledge. When we understand what is broken, we can fix it.“
The initiative includes representatives from government agencies like Francis Collins, director of the National Institutes of Health, but also from the private sector, including Sue Siegel, chief executive of GE Ventures and GE’s healthymagination campaign, Andrew Conrad, head of the life sciences team at Google X, and Susan Desmond-Hellmann, CEO of the Gates Foundation.
“Most medical treatments have been designed for the ‘average patient,’” the White House said in a press release. “As a result of this ‘one-size-fits-all’ approach, treatments can be very successful for some patients but not for others.”
The White House said that while precision medicine is “leading to a transformation in the way we can treat diseases such as cancer,” its potential for improving care and speeding up the development of new treatments “has only just begun to be tapped. Translating initial successes to a larger scale will require a coordinated and sustained national effort,” the White House said.
GE’s healthymagination, for example, has already supported Vanderbilt University researcher Jennifer Pietenpol, who has been trying to decode and kill an aggressive and chemotherapy-resistant type of cancer called triple-negative breast cancer. “Our task is to understand how these tumor cells grow, find their Achilles’ heel, and how we can hit it,” Dr. Pietenpol said.
Vanderbilt also teamed up with scientist Michael Gerdes and his team at GE Global Research, who have found a new way to study 60 different tumor markers at the same time and obtain a better idea of a cancer’s behavior. “With unprecedented views, we hope, will come unprecedented insights that will tell us more about how cancer forms, how it progresses, and most importantly, how to defeat it,” Gerdes says.
But precision medicine applies to many other diseases, not just cancer. Last year, GE Capital’s healthcare financial services arm invested in Assurex Health, a company developing DNA-based diagnostic tools that could allow doctors to study genes that may affect a patient’s response to antidepressant, antipsychotic, analgesic and attention deficit hyperactivity disorder (ADHD) medications.
There are many diseases that remain hard to treat. But deep inside them, they carry a secret message that spells out their biggest weakness. We are staring to decode that message and use it to fight back.