Sickle cell anemia is a debilitating disease that affects 1 in 500 African Americans.
We first introduced you to the Means family in 2013. Then-25-year-old Julius along with his younger brother Desmond were babies when they were diagnosed with sickle cell anemia, an inherited defect of the red blood cells.
They spent their childhood in and out of the hospital as they struggled with the inherited blood disorder.
“Some of my first memories having sickle cell were playing one day and the next day not being able to even walk,” Julius Means said.
More rigid than normal round cells, the crescent or sickle-shaped cells get stuck in small vessels and block the delivery of oxygen to organs, tissues and joints. The result is chronic and intense pain.
“The majority of the pain would hit me in my legs and my back,” Julius Means said. “So being a young kid and not being able to play with the other kids was pretty awful.”
Dr. Damiano Rondelli is with University of Illinois Hematology.
“The kidney, the liver, the heart, the lung get worse and worse,” Rondellia said. “In fact many patients die before age 40 or 50.”
The best hope is a stem cell transplant. Doctors infuse healthy donor cells that gradually take over the patient’s bone marrow to produce normal red blood cells.
But, at the time, the procedure required high doses of chemotherapy to kill off a patient’s own cells and prep the body to accept the new ones, a practice deemed too dangerous for some sickle cell patients.
“It depends on how old they are and how long the disease has been working in their body,” Rondelli said.
Then a breakthrough.
Rondelli offered Julius and Desmond Means an experimental alternative using more tolerable immuno-suppressing drugs. Older brother Clifford served as their stem cell donor.
“It’s been awesome,” Julius Means said. “I’ve been able to really work, go out and have fun without always having to worry about getting sick.”
They have no hospitalizations since the treatment and are free from the debilitating pain.
“I can’t think of any other condition that we transplant that has such a big impact in so many patients,” Rondelli said.
Doctors at UIC have performed about 50 stem cell transplants without chemo with a 75 percent success rate. Some have involved non-compatible donors, which is now possible due to more advancements in research. And doctors are now working on a gene-editing procedure for sickle cell patients – another major breakthrough to help end the pain.