Federally funded research has played a key role in the development of new treatments for diseases, as shown by the story of Melvin Mann. At age 37, Mann was diagnosed with chronic myelogenous leukemia (CML), an aggressive blood cancer. His doctors told him that his only hope was a bone marrow transplant, which was difficult to obtain in 1995. Without it, he was given three years to live.
Mann joined several clinical trials over the next few years but did not see results. As his health declined and options ran out, he asked his doctor about any other possibilities. He learned of a drug still in the lab that targeted tyrosine kinase, a protein responsible for cell division that is overproduced in CML patients.
Mann became one of the first patients to try this tyrosine kinase inhibitor in a clinical trial. “And that was the drug that saved my life,” he said. After starting treatment, his condition improved significantly and within ten months he was able to run a marathon.
The drug, later known as Gleevec, showed strong results and received accelerated approval from the Food and Drug Administration (FDA). Before Gleevec’s approval, most people with CML died within five years of diagnosis; now, many can expect long lives with ongoing treatment.
“That drug was a miracle,” Mann said. He attributed his survival to decades of scientific work supported by federal funding at academic and government labs across the United States. Research on CML began in the 1950s at institutions like the University of Pennsylvania and continued through work funded by agencies such as the National Institutes of Health (NIH) and Atomic Energy Commission at universities including the University of Chicago and UCLA.
UCLA professor Owen Witte explained how federal investment in basic science led to breakthroughs: “This is how innovation has worked in this country: The federal government spends money on basic research. Basic research produces discoveries that can be exploited by the drug industry to make drugs that change the lives of patients,” Witte said.
Each year about 9,000 Americans are diagnosed with CML. While this number may seem large considering previous fatality rates associated with diagnosis, pharmaceutical companies historically avoided investing in rare diseases due to uncertain returns on investment without initial federal support.
Gleevec opened up a new class of pharmaceuticals—tyrosine kinase inhibitors—that are now used for multiple diseases beyond CML, including other cancers and autoimmune disorders. Millions benefit from these drugs today.
“Millions of people are taking these medicines and are walking around leading relatively normal lives, instead of getting sicker and dying. And those pharmaceutical companies are keeping a lot of people employed and generating a lot of economic activity,” Witte said. “I could not imagine a better use of federal funding than that.”
After recovering his health thanks to federally backed science, Mann became an advocate for cancer survivors’ rights and access to care—including visits to lawmakers regarding Medicaid and Medicare policies—and stressed the importance of maintaining support for research funding as Congress debates future budgets for agencies like NIH and NSF.
Mann highlighted that while current treatments allow many with CML to live well-managed lives through daily medication, there is still no cure: “I have to take a pill every day, but maybe in the future people won’t have to,” he said. “Eventually, if they keep at it, they will find a cure.”
He also pointed out that roughly 30 million Americans suffer from rare diseases—many still lacking effective treatment—underscoring how essential continued investment is for medical progress across many conditions (https://www.nih.gov/news-events/nih-research-matters/how-many-rare-diseases-are-there).
“If the government pulls back on funding basic research, progress for those families ‘would come to a standstill,’” Mann warned. “It would just be a big red light. It would just stop, and you’d have to just be satisfied with what you have now.”
