CLEVELAND — Danielle Lee is a thrill-seeker and a self-described adrenaline junkie.
“I’m still trying to go skydiving," she said.
The Garfield Heights native refuses to let her sickle cell disease stop her.
Currently, there are few approved therapies for the genetic blood disorder and on average, patients live until their mid 40s.
“Definitely in rebellion. It’s like me proving to myself I don’t have these limitations or I can do stuff. I just have to make certain adjustments," said Lee.
Lee is a writer and aspiring actress and director. The 33-year-old also wants to do her own stunts.
“Dream job as an actor, I want to do the movie based off of this book. It’s called Children of Blood and Bone.”
She moved to Los Angeles to chase her dreams more than a decade ago.
“I was in a movie that came out last year called Workout Partners that’s currently on Tubi. I did an episode of one of my best friend’s TV shows called Partners and Rhyme," she said.
Lee has been back in Ohio for the last year to participate in a study called the RUBY Trial that Cleveland Clinic Children’s Hospital is involved in.
The experimental gene therapy modifies a patient’s own stem cells to correct the defect causing sickle cell disease.
Currently, a blood or marrow transplant can be a cure, but it requires a donor and can be high-risk for complications.
Dr. Rabi Hanna is the director of the pediatric blood and bone marrow transplant program at Cleveland Clinic Children’s.
He explained that with this gene therapy, every patient can be their own donor.
“This technique enables us to precisely target the gene that we want, be able to cut it like a scissor exactly where we want and replace it with the desired gene that we will produce natural fetal hemoglobin. This hemoglobin f, or fetal hemoglobin, is very protective from the effects of sickle cell disease," said Hanna.
To make room for the repaired cells, patients undergo chemotherapy to destroy their remaining bone marrow after stem cells are collected for gene editing.
Hanna said early results are promising that this may be a functional cure for sickle cell disease that provides relief to patients.
Lee is one of four people part of the trial so far and the goal is to enroll 40.
“It’s definitely something that could possibly change the entire trajectory of my life," said Lee.
Prior to the trial, Lee needed two surgeries and was hospitalized for debilitating pain in her joints multiple times a year.
For her, gene therapy has been life-changing.
“I only get pain in my joints from where I was affected because the trial doesn’t get rid of damage that I’ve already had, but it’s something that I’m able to take Motrin now. And like I haven’t been able to take just Motrin in years, like I probably was in middle school or high school when Motrin worked for me,” said Lee.
Lee is hopeful about the future of care for sickle cell patients and is excited to continue to pursue her passions with less pain.
“Hoping that I can be an example to other people with sickle cell that yeah, we have this disease, but it doesn’t have to stop everything and all our hopes and dreams. Like we can still hope. We can still dream. We can still do things,” said Lee.