NASHVILLE, Tenn. (Ivanhoe Newswire) — Sickle cell 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. But for the first time, even scientists say new therapies have the power to actually cure the disease.
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 recalled.
Danielle 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 another new therapy is also showing promise. Doctors at Vanderbilt are using a modified bone marrow transplant that uses a half-match donor.
“We collect stem cells, which is what we need, directly from the donor’s bone marrow. They have to be at least a 50 percent match,” said Adetola Kassim, MD, MS, FACP, professor of medicine, director of the Vanderbilt stem cell transplant and cellular therapy program, co-director of the Vanderbilt-Meharry Sickle Cell Center of Excellence at the Vanderbilt-Ingram Cancer Center at Vanderbilt University Medical Center.
Instead of drawing stem cells from the donor’s circulating blood, the traditional method, they collect stem cells directly from the bone marrow, which contains more immature stem cells that are less likely to attack the patient’s body.
“We want stem cells that are less likely to cause problems,” Dr. Kassim explained.
And the sickest patients are responding the best.
“Honestly, I think it’s exhilarating. They come back to you, able to sustain work, be with their families, go back to school,” Dr. Kassim told Ivanhoe.
And now patients like Danielle finally have a real chance at a cure.
Studies show a partial-match bone marrow transplant is rewriting the future for sickle cell patients, with survival rates climbing above 90%, even in adults once thought too sick to benefit. The treatment is now available at leading medical centers across the United States.
Contributors to this news report include: Marsha Lewis, Producer; Matt Goldschmidt, Videographer; Roque Correa, Editor.
Source:
https://www.nhlbi.nih.gov/health/sickle-cell-disease
* For More Information, Contact: Criag Boerner
Assistant Director, Media Director, & National News Director at Vanderbilt University Medical Center
(615) 322-4747
and
Adetola Kassim, MD, MS, FACP
Professor of Medicine, Director of the Vanderbilt Stem Cell Transplant and Cellular Therapy Program, Co-Director of the Vanderbilt-Meharry Sickle Cell Center of Excellence at the Vanderbilt-Ingram Cancer Center at Vanderbilt University Medical Center
(615) 936-8422
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