Clinical trial on sickle cell disease yields promising results

Sickle cell disease is when red blood cells, which are normally round, are instead crescent shaped. The disease can have numerous symptoms including abnormal breakdown of red blood cells, delayed development, inflamed fingers or toes, shortness of breath, jaundice and extreme pain.

More than 100,000 people suffer from the disease in the United States, with the majority being African American. Treatment options have been limited to blood  and bone marrow transplants.

Canaan Evans and his mom, Arenda, were all laughs outside University Hospitals Rainbow Babies and Children’s Hospital recently, but that hasn’t always been the case.

Dr. Jignesh Dalal, Canaan’s doctor and director of marrow and stem cell transplant at University Hospitals, is conducting a clinical trial for sickle cell disease.

“Those patients develop sudden onset of severe pain in various organs of body, starting from head to toe,” Dalal said.

He said their expected lifespans are 20 to 30 years less than that of a healthy individual.

Canaan is one of 10 people in the United States who recently completed the trial. Now, just three months later, his bookwork shows no trace of the disease.

“It was amazing,” Canaan said. “It’s like almost unbelievable”

Getting to this point wasn’t easy. Canaan has undergone blood transfusions since he was a young child, and the trial itself takes four to six months, including a round of chemotherapy.

“It wipes out his old, genetically abnormal stem cells and makes space and room for allowing this genetically modified infused own stem cells,” Dalal said.

These results essentially eliminate the need to find a donor that would be a match for a patient with sickle cell disease.

Despite the potential for a positive outcome, Canaan had his reservations. There were days he didn’t think he’d make it through the trial.

“To be honest, it was really tough,” he said, “because the chemo, you know, made me really sick.”

In spite of all the side effects, it was just three weeks before Dalal realized the treatment was working.

“It was very exciting, and even his pain, his mouth ulcers, everything started improving,” he said.

All the of patients involved in the trial have had similar results, and the Evans family is still processing the good news.

Dalal is optimistic about the future and said that, similar to other treatments of the disease, he hopes this one may be available for use shortly.