Breakthrough therapies offer hope for sickle cell disease cure

INDIANAPOLIS (WISH) — For the first time, scientists say new therapies have the power to cure sickle cell disease.

The disease is a devastating, inherited blood disorder that affects an estimated eight million people worldwide and roughly 100,000 here in the United States, most of them African American.

It causes misshapen red blood cells that block blood flow, triggering excruciating pain, chronic anemia, and damage to the heart, lungs, kidneys and brain. Despite decades of research, the average life expectancy for an American with sickle cell remains about 20 years shorter than the general population.

Danielle Lee is all smiles today, but for years she hid her pain.

“I went from like screaming, crying, ‘please don’t let me die’ to not caring if I died, I just wanted the pain to be over,” she said.

Lee has lived her entire life with sickle cell disease.

“My blood cells are like sickle shape instead of like normal circle shape. And they are sticky and they can leak together anywhere in your body. So, when that happens, it causes a pain crisis,” she explained.

Doctors at Cleveland Clinic used a one-time gene editing cell therapy to modify Danielle’s own blood-forming stem cells to correct the mutation responsible for sickle cell disease. She is now pain-free! And ano...