Clinicians at the Loma Linda University Children’s Hospital successfully performed that institute’s first stem cell transplant, and report having “effectively cured” a girl with sickle cell disease (SCD).
“She is now disease free and can go back out into the world to do what an 11-year-old should be doing,” Akshat Jain, MD, a pediatric physician specialist in blood disorders, said in a news story written by Sheann Brandon.
The treatment was possible under a relatively new hospital program dedicated to patients with blood disorders, specifically sickle cell and hemophilia.
Doctors there developed the program in the hope it can ease access to better treatment for patients the Inland Empire region of Southern California that Loma Linda serves, the article reported.
A stem cell transplant (SCT) replaces the diseased blood-forming stem (precursor) cells of a patient using healthy stem cells from a donor. It is currently the only treatment available to people with severe sickle cell disease that offers the possibility of a cure.
The procedure involves replacing the patient’s bone marrow (the site within the bones where stem cells are produced) with stem cells from a healthy donor. If the transplant is successful, the sickle cell patient may well be cured.
This first transplant recently done at children’s hospital treated Valeria Vargas-Olmedo, who was born with sickle cell disease.
Her family started seeking advanced medical help last year, when damage caused by the disease left her unable to go on with her daily life, including attending school or walking.
She had chronic pain, bone loss, and bone necrosis (death of bone tissue) severe enough to be debilitating, the news article reported.
This transplant was also the first to be haploidentical, or genetically somewhat matched, at the Loma Linda hospital. The stem cells were donated by the girl’s father, and were a partial match in terms of genetic makeup to the girl’s own stem cells.
Her father’s cells were infused directly into the girl after conditioning chemotherapy given to eliminate her unhealthy stem cells.
“My daughter is much more animated now — she’s begun walking, she’s eating and gaining weight, she’s happy. Little by little she is living a normal life like before,” Clara Olmedo, the girl’s mother, said in the report.
The Inland Empire region, a vast metropolitan area east of Los Angeles County, is home to a large African-American and Hispanic population, both of which are at risk for sickle cell disease, Jain said. He added that the hospital program he and his team conduct treats roughly 250 to 300 patients.
More about the sickle cell disease and the program run at Loma Linda University Children’s Hospital is available on its disease-dedicated webpage.