CLEVELAND — The Food and Drug Administration approved two new gene editing treatments this month that offer hope to the roughly 100,000 Americans living with sickle cell disease following clinical trials conducted in part at the Cleveland Clinic.
The gene editing cell therapy that modifies a patient’s own blood-forming stem cells to correct the mutation responsible for sickle cell disease. The patient then undergoes chemotherapy to destroy the remaining bone marrow to make room for the repaired cells which are then later infused back into the body.
"This is really exciting time for patients with sickle cell disease," said Dr. Rabi Hanna, the Cleveland Clinic's Chairman of Pediatric Hemotology and Oncology who was the principal investigator of the trial. "This therapy is basically offering them the chance for potentially functional cure."
Sickle cell disease is an inherited disorder that affects red blood cells which are normally round and can move through small blood vessels and deliver oxygen. In people with the sickle cell mutation the shape of the red blood cell is more of a sickle. This can block blood flow, preventing oxygen from getting to the vital organs and tissues throughout your body.
"That's why the median life for patients with sickle cell is approximately 43 years," Hanna told News 5.
In looking for potential participants in the trial one of his patients, 32-year-old Danielle Lee of Garfield Heights stood out.
"Starting at 4-years-old I was in the hospital three, four times a year," said Lee. "It got a little bit better some years and some years not so much so I think the longest I ever went not being in the hospital is almost a year, I was shy by like three weeks."
In middle and high school, her teachers were able to work with her through the hospitalizations, college was tougher.
"Once I got to college it was very difficult, it took me nine years to graduate," she said.
She knew to avoid triggers that could land her in the hospital one of the ones that was hard to avoid living in Northeast Ohio was simply being cold. Another is too much physical activity. Swimming for example at times combined both.
"Swimming has sent me to the hospital multiple times because I can get to cold," she said.
When Hanna first approached her about the clinical trial she wasn't immediately on board.
"I thought about it and you know I wasn't excited to do it like a lot of people with sickle cell would because I was just like I know what sickle cell is, I know how to handle that. What if I do this and get worse?"
Lee is an actress though and said she found herself one day watching "The Gray Man" on Netflix and yearned for the opportunity to one day take on a role that required the physical action she was watching play out on the screen but realized how in her current state she would have been likely to end up back in the hospital.
"So I was like if I want to do movies like this I should probably go do this," she said telling Hanna that she was in.
The process, the chemo, was not easy on her but now on the other end of things she says it was worth it.
"So far so good knock on wood," she said. "Certain things I've done haven't sent me to the hospital."
She still has residual pain because it doesn't undue the damage that's been done to her bones.
"But I'm able to take Motrin now which I haven't been able to treat pain with Motrin since middle school or high school."
