Federal science breakthrough delivers 'miracle' drug, extending life for the patient
In the mid-1990s, Melvin Mann, a 37-year-old Army major, was diagnosed with chronic myelogenous leukemia (CML). Before the advent of Gleevec, most people diagnosed with CML faced a grim prognosis, with many dying of the disease within five years.
Mann's white blood cell count began to rise, and he lost 30 pounds. Doctors told him that a bone marrow transplant was the only cure, but finding a match was difficult in 1995. Mann was among the first people to be treated with a tyrosine kinase-inhibiting drug in a clinical trial, which would eventually save his life.
The research that led to the development of Gleevec started in the 1950s and was funded by the federal government. Decades of federally supported studies identified the genetic mutation responsible for CML and the molecular mechanisms behind it, which directly enabled the creation of targeted drugs such as Gleevec.
Owen Witte, a UCLA professor, discovered in the mid-1980s that an abundance of tyrosine kinase caused CML symptoms. His NIH-funded work led to the development of molecules that could inhibit tyrosine kinase, forming the scientific foundation that pharmaceutical companies later used to develop Gleevec.
Today, about 9,000 Americans are diagnosed with CML each year, and many are taking tyrosine kinase inhibitors and leading relatively normal lives. This transformation in prognosis from fatal to manageable with long-term survival is a testament to the power of federally funded basic research.
Federally funded basic research has been instrumental in understanding and treating blood cancers, such as CML. Millions of people are benefiting from federal-funded research in the form of medications for various diseases. However, the administration's proposed spending bills for the coming year would cut funding for science agencies like the NIH and the National Science Foundation.
Pharmaceutical companies are keeping a lot of people employed and generating economic activity due to federal-funded research. If the government pulls back on funding basic research, progress for rare disease patients "would come to a standstill."
Mann and his wife regularly meet with elected leaders on issues such as strengthening Medicaid and Medicare for people without private insurance to access the care that saved his life. They are advocates for the importance of federally funded research in driving the development of targeted treatments like Gleevec, which has revolutionised the treatment of CML and many other rare diseases.
References:
- National Organization for Rare Disorders (NORD). (n.d.). About NORD. Retrieved from https://rarediseases.org/about-nord/
- National Institutes of Health (NIH). (n.d.). NIH History. Retrieved from https://www.nih.gov/about-nih/who-we-are/history
- National Cancer Institute (NCI). (2020). Chronic Myelogenous Leukemia Treatment (PDQ®) – Health Professional Version. Retrieved from https://www.cancer.gov/types/leukemia/hp/cml-treatment-pdq
- Cancer Research UK. (2020). Chronic myeloid leukaemia (CML). Retrieved from https://www.cancerresearchuk.org/about-cancer/blood-cancers/chronic-myeloid-leukaemia/causes
- National Institutes of Health (NIH). (n.d.). NIH Grants for Rare Diseases. Retrieved from https://www.nih.gov/research-training/medical-research-initiatives/rare-diseases-clinical-research-network/rare-diseases-clinical-research-network-grants
Science and medical advancements have significantly impacted the management of various health-and-wellness conditions, such as cancer, specifically chronic myelogenous leukemia (CML). The development of Gleevec, a tyrosine kinase-inhibiting drug, was made possible through decades of federally funded science, including clinical trials, and has transformed CML from a potentially fatal diagnosis to a manageable medical-condition for thousands. However, proposed spending cuts for science agencies like the NIH and National Science Foundation pose a risk to future medical breakthroughs and the well-being of those afflicted with rare diseases.
