Stanford Center Hopes to Take Stem Cell and Gene Therapies to a New Level

Stanford Center Hopes to Take Stem Cell and Gene Therapies to a New Level
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Genetic diseases center

The new Stanford Center for Definitive and Curative Medicine will foster the development of stem cell and gene therapies for genetic diseases, including sickle cell anemia.

More than 280 million people around the world have diseases with genetic causes, experts estimate. While research has identified the underlying causes of several, scientists have developed few therapies that can address the causes or cure the diseases.

Treatments have been developed that significantly improve patients’ health, however. They include public health initiatives, targeted therapies and surgery.

Scientists believe stem cell and gene therapy can cure some genetic diseases. They would likely do this either by rewiring cells to fight a disease more efficiently or by correcting a genetic error in a patient’s DNA.

Stanford not only does excellent research in disease mechanisms, cell and stem cell biology, but also promotes collaboration between its medical schools and hospitals.

The initiative is a joint venture of the Stanford University School of MedicineStanford Health Care and Stanford Children’s Health.

Dean Predicts Center Will Be Major Force in the Precision-health Revolution

“The Center for Definitive and Curative Medicine is going to be a major force in the precision-health revolution,” Dr. Lloyd Minor, dean of the School of Medicine, said in a press release. “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,” said Christopher Dawes, the president and CEO of Stanford Children’s Health. “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.”

“The work of the center is not being done anywhere else in the country — only at Stanford,” said 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.”

Dr. Maria Grazia Roncarolo will direct the center, which will be in the Department of Pediatrics. The renowned medical doctor and scientist is the George D. Smith Professor of Stem Cell and Regenerative 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.

Main Mission Will Be to Turn Scientific Discoveries Into Treatments

“Stanford Medicine’s unique environment brings together scientific discovery, translational medicine and clinical treatment,” Roncarolo added. “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’s main mission will be to turn scientific discoveries into treatments. A world-class interdisciplinary team of scientists should help it deliver on that promise.

Leaders of the team will include Dr. Matthew Porteus, an associate professor of pediatrics, and Dr. Anthony Oro, the Eugene and Gloria Bauer Professor of dermatology. Dr. Sandeep Soni will direct the center’s stem cell clinical trial office.

“The center will provide novel therapies that can prevent irreversible damage in children, and allow them to live normal, healthy lives,” said Dr. Mary Leonard, chair of pediatrics 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.”

A unique feature of the center will be a close association with the Stanford Laboratory for Cell and Gene Medicine, which is working on new cell and gene therapies.

The lab has already developed genetically corrected bone marrow cells as a treatment for sickle cell anemia. Other genetically modified cells it has created include skin grafts for children with the genetic disease epidermolysis bullosa and lymphocytes for children with leukemia.

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