At the Bedside: Nurse with sickle cell disease joins clinical drug trial and finds energy she’s never had before
The day that Dorcas Osho and her parents moved to the United States, just one day after her fifth birthday, she began screaming in pain. Her parents scrambled to help their daughter and rushed her to a children’s hospital, where she was diagnosed with sickle cell anemia.
“We, as a family, had to quickly learn how to deal with sickle cell anemia and what it was. We didn’t know anything about it. We experienced two major life changes that year,” Osho said.
Her parents were unaware of the genetic condition; however, people can be carriers of sickle cell and will not develop the disease, but may pass the risk to their children. Osho’s brother was also later diagnosed with the same disease.
Sickle cell anemia is an inherited blood disorder that causes red blood cells to become misshapen, or sickle-shaped. A red blood cell’s normal shape makes it flexible and allows it to easily move through blood vessels to deliver oxygen. When cells are sickle-shaped, they stick to vessel walls, preventing blood and oxygen flow, which causes severe pain called crises that can last up to a week.
Osho recalls her childhood as being limited, surrounded by her parents and teachers who would take extensive precautions to prevent triggers of pain and keep her safe. She regularly visited the hospital for countless blood transfusions and for treatment of infections that routinely impacted her.
“It was soul-crushing, because I couldn’t partake in sports or run as fast as the other kids. I was told not to do anything that involved physical activity,” Osho said.
Transitioning from children’s care to adult care was difficult for Osho while attending college. She made it her goal to find a doctor who was actively researching new ways to treat sickle cell disease in patients. While working as a nurse, she cared for a man who had the same condition and he recommended Modupe Idowu, MD, a hematologist at UTHealth Houston and medical director of the UT Physicians Adult Sickle Cell Center, which provides care for patients living with sickle cell disease.
“When I transferred to Dr. Idowu at UT Physicians, it was like I saw the light again,” Osho said.
Under Idowu’s care, Osho joined a Phase II clinical drug trial that is researching a hemoglobin S polymerization inhibitor drug called GBT021601. The next-generation inhibitor drug is intended to bind hemoglobin, an oxygen-transport protein, to help red blood cells hold on to more oxygen to keep their shape stable and prevent them from collapsing into a sickle form.
Researchers will evaluate GBT021601 in three sections. Part A will evaluate the safety, tolerability, and efficacy of GBT021601 in adult participants with sickle cell disease to determine an optimal dose. Part B will evaluate the efficacy of GBT021601 versus placebo in adult and pediatric participants for 48 weeks. Part C will evaluate the bodily absorption and safety of single and multiple doses of open-label single arm GBT021601 given to pediatric participants.
“I saw Dorcas’ frustration while dealing with exhaustion from managing her fatigue and pain, I recommended she joins this trial to help her. She’s had a major change in her quality of life,” Idowu said.
Since participating in the first arm of the trial, Osho said, she feels like a new person. The medication has helped her find more energy to regularly exercise with her husband and lose 10 pounds.
“Before, I would get winded after cleaning the house. Now, I can go to the gym to lift weights and do cardio. I’m ecstatic,” Osho said.
The multisite study, which aims to enroll a total of 480 people with sickle cell disease, is currently recruiting participants ages 6 months to 65 years old.
For more information, visit the UT Physicians Comprehensive Sickle Cell Center’s website or call 713-486-5660.
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