Children’s Hospital Los Angeles continues to ensure the highest level of access to breakthrough cell and gene therapies for an increasing range of debilitating and often life-threatening childhood diseases.
Recently, the hospital expanded its offerings to 12 FDA-approved therapies—more than any other pediatric center on the West Coast.
The 12 therapies target genetic drivers of disease across a wide range of conditions, including cancer, blood diseases, neurological disorders, and inherited retinal disease. CHLA is one of only a handful of hospitals across the country that offers access to many of these treatments.
“Not all pediatric centers have the infrastructure and multidisciplinary expertise to deliver this breadth of cell and gene therapies.”
Alan S. Wayne, MD, Pediatrician-in-Chief and Pasadena Guild Chair at Children's Hospital Los Angeles
“At CHLA, we’ve built the capabilities needed to provide these complex treatments safely and effectively.” says Alan S. Wayne, MD, Pediatrician-in-Chief and Pasadena Guild Chair at Children's Hospital Los Angeles
The therapies available at CHLA span multiple specialties across the hospital and treat more than a dozen conditions:
Elevidys (Duchenne muscular dystrophy)
Zolgensma (spinal muscular atrophy)
Casgevy (sickle cell disease and transfusion-dependent beta thalassemia)
Hemgenix (hemophilia B)
Lyfgenia (sickle cell disease)
Roctavian (hemophilia A)
Zynteglo (transfusion-dependent beta thalassemia)
Kymriah (B-cell acute lymphoblastic leukemia and diffuse large B-cell lymphoma)
Omisirge (provides an unrelated stem cell source for patients with blood cancers or blood system failures undergoing umbilical cord transplantation)
Ryoncil (acute graft-versus-host disease)
Luxturna (inherited retinal dystrophy due to RPE65 gene mutations)
Vyjuvek (dystrophic epidermolysis bulluosa)
One of the newest gene therapy approaches harnesses CRISPR/Cas9 technology, which enables direct editing of a person’s genome—versus using a viral vector to add a functional gene copy into affected cells. Both can correct the genetic abnormality, but through different mechanisms.
This year, CHLA will treat its first patient with Casgevy, the first FDA-approved therapy to use CRISPR/Cas9 gene editing.
CHLA is preparing to open the RESTORE1 study, which is a clinical trial of nula-cel, a sickle cell gene therapy product. This CRISPR-Cas9 therapy is designed to directly repair the mutation in the beta-globin gene that causes sickle cell disease, reducing or eliminating future complications of the disease as a result.
“In addition to safely providing FDA-approved treatments, we are actively partnering with pharmaceutical companies to give patients access to the most promising investigational therapies as well,” says Ashley N. Gray, MD, MS, Director of Gene Therapy for Hemoglobinopathies in the Cancer and Blood Disease Institute at CHLA.
“Many of these trials are available at only a few pediatric centers in the country and we are proud to provide our patients with access to the most leading-edge research.”
CHLA is also working to translate its own laboratory advances into patient care. A key part of that effort is the USC/CHLA cGMP Laboratory, a state-of-the-art facility that opened in 2023 and manufactures cell and gene therapies under the FDA’s current good manufacturing practice (cGMP) standards.
CHLA is preparing to launch its first clinical trial using that cGMP facility next year—a chimeric antigen receptor (CAR) T-cell therapy for pediatric solid tumors that was developed by CHLA investigators.
“We are in a transformational era for cell and gene therapies,” says Dr. Wayne. “Our goal is to ensure that children and young adults have access to the most advanced treatments available today, and to the innovations that will shape the future of care.”
Reference:
1) https://clinicaltrials.gov/study/NCT04819841
(Newswise/HG)
