Beth was only 47 years old when routine bloodwork revealed she had a blood cancer— polycythemia vera. Polycythemia vera causes the bone marrow to make too many red blood cells in particular and this often causes serious problems. Further, it can progress to myelofibrosis or acute leukemia. For Beth, it meant an abnormally enlarged spleen that was attempting to clear a greater number of blood cells than normal. This uncomfortable condition is called splenomegaly. Beth was able to treat her condition locally with phlebotomies, then a drug called Hydrea, and finally Jakafi. Over time, her diagnosis progressed from polycythemia vera to myelofibrosis. In 2018, Beth’s hematologist told her it was time for greater intervention and recommended a visit to see doctors at UAB. After she learned that a stem cell transplant wouldn’t be possible because of how enlarged her spleen had become, she was guided toward a potential clinical trial for a drug that showed hopeful promise.
Beth soon connected with Pankit Vachhani, MD – Leukemia physician, assistant professor of medicine in the UAB Division of Hematology & Oncology and associate scientist at the O'Neal Comprehensive Cancer Center at UAB – who had a phase two clinical trial underway for a drug called KRT-232 (navtemadlin) for those with relapsed/refractory myelofibrosis. The drug inhibits a protein called MDM2 which in itself negatively regulates the activity of a tumor suppressor gene called p53, also known as the “guardian of the genome”. Beth thought very carefully about her participation in the trial, but she soon knew what she wanted to do. “I wanted life. I had two young grandchildren that I wanted to see, and a wonderful family I wanted to be here for.” Beth enrolled in the trial in 2019 and was met with incredible support from friends and family, especially her husband Tom and sisters Ruth and Becky. It was Beth’s daughter Betsy who encouraged her mom to begin sharing her diagnosis and journey publicly so that others could also rally around her to help her get through the days ahead.
Beth and her husband began driving to Birmingham for testing and treatments, which were all administered in the CCTS Clinical Research Unit (CRU), a dedicated outpatient area for research in UAB Hospital’s Jefferson Tower. The unit’s trained nursing staff and patient rooms support a wide range of clinical research. “I cannot say enough about the hospital and the Clinical Research Unit team. The nurses…they truly care. They must not enjoy the unpleasant things they sometimes see, but they must love their jobs to do such amazing work.” Beth worked hard to maintain a healthy diet, exercise, and to follow all of Dr. Vachhani’s instruction. She experienced days in her trial where she was met with setbacks, but she kept showing up.
Beth began feeling noticeably better, experiencing major improvement to her quality of life. Her spleen and symptoms decreased significantly on treatment compared to baseline. In September of 2020, after carefully weighing the benefits and some adverse events from the drug, Dr. Vachhani halted Beth’s treatment. And then the big news. In February of 2021, Dr. Vachhani was able to tell Beth she had achieved a very significant clinical improvement, including the reduction in her enlarged spleen. She remains in excellent control to this day, still yielding the incredible grandmother energy that was part of her original goal for clinical trial participation. “Now I can help my daughter and son-in-law with my grandchildren. I can be there for my family. Dr. Vachhani and this clinical trial have changed my life.”
Learn more about the CRU
The CCTS Clinical Research Unit (CRU) provides investigative teams a research environment and broad range of services guided by good clinical practice. This contributes to the conduct of excellence in clinical and translational research. The unit consists of 12 rooms and equips investigators with essential tools and critical resources, while providing a highly efficient and flexible infrastructure that is sustainable through a comprehensive cost-recovery system. If you are interested in leveraging the capacity of the CRU, contact: CRU Nurse Manager: