Turn discoveries into stem cell and gene therapies

(Stem Cells News image)

Stanford announces new Center for Definitive and Curative Medicine

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.

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 Health Care and Stanford 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.” (…)

“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 (…)

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.” (…)

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.” (…)

read more:med.stanford.edu/news/all-news/2017/07/stanford-announces-new-center-for-definitive-and-curative.html

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