At least 280 million people worldwide live with rare genetic diseases. For many of them, the disease’s underlying cause is well defined, yet eludes definitive treatment. Stem cell and gene therapies hold enormous promise for curing these conditions by engineering cells to treat disease or alter a patient’s DNA to “fix” an abnormality.
To bring these new stem cell and gene therapies to patients, Stanford Medicine recently opened the Stanford Center for Definitive and Curative Medicine (CDCM), which
provides the organizational and physical infrastructure to support cutting-edge studies on stem cell and gene therapies, from initial discovery through completion of proof-of-concept clinical studies.
Renowned clinician and scientist Dr. Maria Grazia Roncarolo, chief of the Division of Pediatric Stem Cell Transplantation and Regenerative Medicine, will direct the new center.
“Stanford’s unique environment brings together scientific discovery, translational medicine and clinical treatment,” Roncarolo said.
From work performed by scientists over the past decade, the CDCM already has a queue of nearly 24 early-stage therapies in its pipeline.
The CDCM also benefits from Stanford’s new Laboratory for Cell and Gene Medicine (LCGM).
One of the first of its kind in the world, the LCGM can produce cell and gene therapies according to good manufacturing practice standards required for patient treatment.
Headed by David DiGiusto, the LCGM can produce diverse cellular products for patient use from genetically corrected bone marrow cells for sickle cell anemia, to genetically engineered skin grafts for children with the genetic disease epidermolysis bullosa, to genetically engineered lymphocytes that fight leukemia.
“The LCGM will bridge the gap between research and clinical investigation so we can reach the curative potential of these exciting cell and gene therapies,” DiGiusto said.