The University of Chicago Medicine Comer Children’s Hospital will be among the first in the country to offer gene therapy for sickle cell disease in patients 12 years and older, after federal regulators approved two new treatments on December 8, 2023.
Thousands of patients with sickle cell disease experience vaso-occlusive crises (VOCs), which are often painful and frequently require hospitalization. The two new potentially curative treatments show promise for eliminating VOCs and offer an alternative to bone marrow transplants, which can be arduous and carry risk of rejection even if a matching donor is found.
People with sickle cell disease have a gene mutation that causes their blood cells to produce faulty hemoglobin — a protein that carries oxygen throughout the body — and function incorrectly. Gene therapy uses gene-editing technology to fix or compensate for this mutation.
Both of the newly approved gene therapies involve removing a patient’s own stem cells and modifying them before infusing them back into the patient.
One therapy adds a new copy of a gene to the patient’s stem cells so they produce a functional form of hemoglobin.
The other therapy is the first FDA-approved product to use CRISPR-Cas9 gene editing technology. This therapy changes existing DNA in the patient’s stem cells to “switch on” a gene that will make the cells revert to producing fetal hemoglobin instead of non-functional adult hemoglobin affected by the sickle cell mutations.
Comer Children’s is one of just nine authorized treatment centers immediately activated for this CRISPR-based therapy.
Comer Children’s Hospital participated in a clinical trial of a sickle cell gene therapy, giving UChicago physicians additional cell and gene therapy experience as well as specific experience administering it to sickle cell patients.
James LaBelle, MD, PhD
Associate Professor of Pediatrics
Director, Pediatric Stem Cell and Cellular Therapy Program
James Labelle, MD, PhD, provides care for children of all ages with cancer and blood diseases, including leukemia and lymphoma. In particular, Dr. LaBelle specializes in stem cell transplantation, replacing a patient’s diseased bone marrow and immune system with healthy cells.
For more information, visit https://uchicagomedicine.org/global.