Maria Grazia Roncarolo will direct the Stanford Center for Definitive and Curative Medicine.
Norbert von der Groeben
The new Stanford Center for Definitive and Curative Medicine will work to turn discoveries into stem cell and gene therapies to aid the millions of people who have genetic diseases.
For Release: July 24, 2017
At least 280 million people worldwide are living with a rare genetic disease. For many of these millions, the underlying cause of disease is known and well-defined, and yet eludes definitive treatment. At times, surgical interventions, public health measures, biological and small-molecule therapies can transform the health of these populations; often, however, the currently available treatment modalities result in mere palliative, rather than curative, medicine.
Stem cell and gene therapy hold enormous promise to cure conditions with well-defined genetic causes by engineering cells to treat disease or altering a patient’s personal DNA to “fix” an abnormality. To bring these new stem cell and gene therapies to their patients, Stanford Medicine has announced the opening of the Stanford Center for Definitive and Curative Medicine, a joint initiative of the Stanford University School of Medicine, Stanford Medicine Health Care Stanford Medicine Children’s Health.
The center provides the organizational and physical infrastructure to support investigator-initiated clinical translational studies on stem cell and gene therapy from initial discovery through completion of clinical proof-of-concept studies. Stanford Medicine is in a unique position to develop the CDCM because of its outstanding expertise in disease pathophysiology, cell and stem cell biology, and an optimal and collaborative environment between the medical school and the hospitals.
“The Center for Definitive and Curative Medicine is going to be a major force in the precision health revolution,” said Lloyd Minor, MD, dean of the School of Medicine. “Our hope is that stem cell and gene-based therapeutics will enable Stanford Medicine to not just manage illness but cure it decisively and keep people healthy over a lifetime.”
Stanford Medicine’s clinical enterprise provides an exemplary clinical environment in which to deploy cures. The center will support the development of life-changing and curative treatments for patients who come to Stanford to receive the highest level of care.
“We are entering a new era in medicine, one in which we will put healthy genes into stem cells and transplant them into patients. And with the Stanford Center for Definitive and Curative Medicine, we will be able to bring these therapies to patients more quickly than ever before,” said Christopher Dawes, president and CEO of Stanford Children’s Health.
“The work of the center is not being done anywhere else in the country — only at Stanford,” added David Entwistle, president and CEO of Stanford Health Care. “We have a pipeline of clinical translational therapies that the center is now driving forward, enabling us to translate basic science discoveries into state-of-the-art therapies for diseases which up until now have been considered incurable.”
Housed within the Department of Pediatrics, the new center will be directed by renowned clinician and scientist Maria Grazia Roncarolo, MD, the George D. Smith Professor in Stem Cell and Regenerative Medicine, and professor of pediatrics and of medicine.
“It is a privilege to lead the center and to leverage my previous experience to build Stanford’s preeminence in stem cell and gene therapies,” said Roncarolo, who is also chief of pediatric stem cell transplantation and regenerative medicine, co-director of the Bass Center for Childhood Cancer and Blood Diseases and co-director of the Stanford Institute for Stem Cell Biology and Regenerative Medicine. “Stanford Medicine’s unique environment brings together scientific discovery, translational medicine and clinical treatment. We will accelerate Stanford’s fundamental discoveries toward novel stem cell and gene therapies to transform the field and to bring cures to hundreds of diseases affecting millions of children worldwide.”
The center consists of several innovative pieces designed to allow the rapid development of early scientific discoveries into the clinic that in the past have languished. This includes an interdisciplinary team of basic and clinical scientists to shepherd nascent therapies developed at Stanford. The team will be headed by associate directors Matthew Porteus, PhD, associate professor of pediatrics, and Anthony Oro, MD, the Eugene and Gloria Bauer Professor and professor of dermatology.
To help with clinical development, the center boasts a dedicated stem cell clinical trial office with Sandeep Soni, MD, clinical associate professor of pediatrics, as medical director. In addition, the center has dedicated clinical trial hospital beds in the Bass Center for Childhood Cancer and Blood Diseases located on the top floor of the soon-to-open Lucile Packard Children’s Hospital. From work performed by scientists over the past decade, the center already has a backlog of nearly two dozen early stage therapies whose development the center will accelerate.
“The center will provide novel therapies that can prevent irreversible damage in children, and allow them to live normal, healthy lives,” said Mary Leonard, MD, professor and chair of pediatrics and physician-in-chief at Stanford Children’s Health. “The stem cell and gene therapy efforts within the center are aligned with the strategic vision of the Department of Pediatrics and Stanford’s precision health vision, where we go beyond simply providing treatment for children to instead cure them definitively for their entire lives.”
One of the unique features of the center is its close association with the recently opened $35 million Stanford Laboratory for Cell and Gene Medicine, a 23,000-square-foot manufacturing facility located on California Avenue in Palo Alto. One of the first of its kind in the world, the laboratory has the ability to produce newly developed cell and gene therapy therapies according to the Good Manufacturing Practice standards as required for patient treatment.
Headed by executive director David DiGiusto, PhD, the lab can produce diverse cellular products for patient use, such as genetically corrected bone marrow cells for sickle cell anemia, genetically-engineered skin grafts for children with the genetic disease epidermolysis bullosa or genetically-engineered lymphocytes to fight rejection and leukemia.
“We are fortunate that Stanford researchers have created such a strong portfolio of innovative candidate therapeutics to develop,” said DiGusto. “The capabilities of the laboratory will bridge the gap between research and clinical investigation so that the curative potential of these exciting cell and gene therapies can be realized.”
Stanford Medicine Children’s Health, with Lucile Packard Children’s Hospital Stanford at its center, is the Bay Area’s largest health care system exclusively dedicated to children and expectant mothers. Our network of care includes more than 65 locations across Northern California and more than 85 locations in the U.S. Western region. Along with Stanford Health Care and the Stanford School of Medicine, we are part of Stanford Medicine, an ecosystem harnessing the potential of biomedicine through collaborative research, education, and clinical care to improve health outcomes around the world. We are a nonprofit organization committed to supporting the community through meaningful outreach programs and services and providing necessary medical care to families, regardless of their ability to pay. Discover more at stanfordchildrens.org.