Aldoris Mbonya-Bate does not let sickle cell disease make her plans for her. She has built a career she loves, acted in film and television projects and traveled internationally, including to the Maldives and Thailand.
"I love traveling," says Mbonya-Bate. "With my sickle cell disease, that was a big fear for a long time."
Sickle cell disease (SCD) is a chronic inherited blood disorder in which abnormal hemoglobin causes red blood cells to become rigid and sickle-shaped. These cells can obstruct blood flow, causing anemia, severe pain, organ damage and other complications.
For Mbonya-Bate, pain crises have been an unpredictable presence throughout her life.
"I tell people to think of the pain like a lightning bolt," Mbonya-Bate says. "You never know when or where it's going to strike."
Today, Mboya-Bate receives care through the Johns Hopkins Sickle Cell Center for Adults, where her care team helps her manage the disease while pursuing the life she wants, including planning ahead to reduce risks when she travels. For the center's care team, supporting those goals is an important part of managing SCD.
"I never discourage a patient from wanting to pursue travel, work or other life goals," says Shannon Carroll, C.R.N.P., nurse practitioner manager and lead advanced practice provider at the center. "We work with the patient and make recommendations to help keep them as safe as possible. We think about what happens if a pain crisis occurs, where the nearest hospital is and what we can do to prevent it."
Research-driven advances have been part of Mboya-Bate's care since childhood, when she began taking hydroxyurea, the first FDA-approved drug for SCD. The drug increases fetal hemoglobin, helping prevent red blood cells from sickling and reducing complications. Johns Hopkins researcher George Dover, M.D., professor emeritus of pediatrics, conducted more than 20 years of NIH-funded research on SCD and fetal hemoglobin that helped lead to hydroxyurea's approval.
"That legacy extends beyond individual discoveries," says Lydia Pecker, M.D., director of research and advocacy at the Johns Hopkins Sickle Cell Center for Adults. "There's a really long tradition of Hopkins clinicians forming the relationships with their patients that lead to the kind of trust required for people to step out on faith and participate in critical research studies."
Mbonya-Bate knows that evolution firsthand. For years, she participated in questionnaires and other studies but was hesitant to participate in more robust research. But learning about the positive influences on mental health and how that research could help identify patients who might benefit from therapy or chronic pain behavioral support made the process feel more tangible for Mbonya-Bate.
"After that, I was like, 'You know what? I could do more things to help,'" Mbonya-Bate says.
Today, she continues to participate in SCD research, including clinical studies exploring new approaches to treatment, decades after she first benefited from advances in drug discovery as a child.
Continued progress in SCD depends not only on patients willing to participate, but also on researchers and clinicians working together across institutions.
"For patients with sickle cell disease, having a network of expert centers matters," Pecker says. "Trials may be open in one place or another, and patients may need care in different places. We all must work together."
Johns Hopkins Medicine is helping lead that collaborative approach through its participation in the National Alliance of Sickle Cell Centers, a nationwide effort to strengthen access to expert, comprehensive SCD care.
For Mbonya-Bate, participating in the research process has become an extension of her advocacy.
"I'm extremely excited about research," Mbonya-Bate says. "To be able to participate and test what we are doing for others like me is an opportunity I embrace."
Her hopes extend beyond whether a new treatment might work for her. She wants future advances to reach people with SCD regardless of their circumstances.
"I want everybody to have what I've gained from treatment advancements," Mbonya-Bate says. "Everybody should have a little bit of this magic."
When asked where the road will take her next, Mbonya-Bate shares that her dream trip is Tokyo.
"I still really try my best to live this life as a quote-unquote normal person does," Mbonya-Bate says. "Sickle cell never changed that."
