Twelve years ago, when I was learning how to be a pediatric oncologist, I met a young man I'll call Brad whose white blood cell count was 50 times what it should have been. He had not been feeling well for two weeks; his mother thought it was a viral thing. Brad was 13, so it could easily have been mono causing his fatigue and his sallow, grayish tone.
But the blood test was unequivocal: Brad had leukemia, and one of the worst kinds: Philadelphia chromosome leukemia, which, at the time, killed 80 percent of the people who had it. The Philadelphia chromosome is a genetic abnormality that makes leukemia especially aggressive. We fought for Brad's life. We gave him state-of-the art care, including a bone marrow transplant from his sister, who was desperate to help him.
But Brad's leukemia returned with a vengeance not six months later. The disease had survived at least a dozen chemotherapy drugs. He would, without a miracle, be dead within days. We were at the No. 1 children's hospital in the world; yet we could not save him.
At precisely that time, it just so happened that a new medication was about to become available for people with Brad's type of leukemia. A group of scientists at Oregon Health & Science University had developed a unique drug. Gleevec, as it was later named, was the first molecularly "targeted" agent specifically designed to block the cancer-causing protein of the Philadelphia chromosome. It was like drone air strikes compared to carpet bombing, with the promise of killing cancer cells with much less collateral damage than "regular" chemotherapy. In a world of blunt instruments, it was a scalpel. It offered hope.
However, Gleevec wasn't commercially available. Fortunately, through our desperate pleas and the manufacturer's goodwill, we were able to get Gleevec on a compassionate-need basis. None of us knew whether it would work or how bad its side effects would be. But we knew Brad would die within days if we did nothing. Both he and his parents were on board. What was the harm in trying it?
Within a week, Brad's blood counts were back to normal and he was feeling well again. We were astonished that a single drug could do what so many powerful chemotherapy drugs could not. Little did I realize then that I was witnessing the start of a revolution in the fight against cancer, the beginning of targeted therapy.
Brad enjoyed a great quality of life for the next four months, but he eventually relapsed. Toward the end, I visited him at his parents' request. We sat together on his couch watching a Jets playoff game. Brad was having a hard time breathing because his lungs were filling up with fluid, and I felt helpless to save him. He died two days later. I still miss him 12 years later. Brad's ordeal taught me the promise, the hope and the limitations of science.
I have been practicing now as a pediatric oncologist for over 10 years. Last month, in the clinic where I work, I saw a college student — let's call him Cory — with the same leukemia that Brad had. Cory is 20 and studying to be an engineer. He is a wonderful young man with a wry sense of humor and a zest for life; he plays the acoustic guitar and rides a motorcycle. He has been in complete remission for five years, having gone through chemotherapy and using Gleevec in a different way: very early on and in combination with conventional drugs.
Cory is alive because of scientific discovery and clinical research into targeted drugs like Gleevec. Philadelphia chromosome leukemia had a 20 percent survival rate a decade ago. Now it might be as high as 70 percent or more, and it's all due to critical advances made together by hard-working biomedical research scientists, clinicians and brave patients.
But future advances are in real danger because of the erosion of support for clinical and scientific research in our nation for at least a decade. Effective research requires well-trained scientists and physicians with creative ideas and an unwavering desire to help patients in need. Effective research requires money, lots of it, and this reality has been a huge problem in our troubled economic times. Though some private foundations fund important science, the majority of biomedical research is financed by the U.S. government through the National Institutes of Health whose budget has remained flat for almost 10 years, victim to a fractured political environment, sluggish economy and other governmental priorities.
The NIH funds research using revenues collected almost exclusively through taxation. According to Dr. Harold Varmus, the director of the National Cancer Institute, the largest and oldest disease center of the NIH, the flat-lining of the NIH budget since 2003 represents a 20 percent decrease in research buying power over the past decade because of the increased cost of doing research today. "We're unable to fund all the people who have good ideas," Varmus told the National Press Club in September.
I am one of the lucky ones. I was awarded an NIH grant a few years ago, and as a result, my lab and I are urgently working to make further inroads in the battle against cancer, in our case melanoma.
But federal grants are exceptionally difficult to get these days. Young people who would otherwise be called to biomedical research because of their talent and curiosity are increasingly opting for other career paths to avoid the enormous stresses associated with competing for federal grants in times of fiscal stringency. Relevant biomedical research is retreating to fewer and fewer medical schools and research institutes. With every scientist lost, we lose fresh ideas and opportunities for future miracle discoveries.
The average life span for an American today is about 80, roughly 30 years longer than it was a century ago. Our quality of life as we age is vastly better as well, heavily influenced by new vaccines and better drugs.
The biomedical research industry not only improves the health of our citizens, it fuels the economy and advances American interests and reputation across the globe. America is the undisputed leader in biomedical research. NIH funding directly supports hundreds of thousands of American jobs in the scientific industry, including biotech and small businesses. As we move into the next four years, federally funded research should be viewed by lawmakers, Democrats and Republicans, along with the public, as a critical investment with both immediate and long-term societal benefits. We forsake this investment at our peril. None of us is immune to disease. Only biomedical research offers us any realistic hope of the next miracle drug like Gleevec. Although I hope it is never the case, one day your life, my life or the life of someone we love might depend on it.
John D'Orazio is practicing pediatric oncologist and physician scientist at the University of Kentucky College of Medicine.John D'Orazio is practicing pediatric oncologist and physician scientist at the University of Kentucky College of Medicine.John D'Orazio is practicing pediatric oncologist and physician scientist at the University of Kentucky College of Medicine.