Letter from the Dean
Aside from the bruises, scrapes and occasional broken bones that accompany childhood, most children — thankfully — are quite healthy. They see the inside of a doctor’s office for annual wellness checkups and vaccines, but are otherwise unfamiliar with treatment in a hospital.
They don’t know what it feels like to have an I.V. inserted, and they haven’t experienced the disappointment of missing yet another season of soccer because they’re too sick to run up and down the field. But for those children who are not so healthy, the consequences can be devastating.
Childhood diseases are crippling for the children and families they affect. Here at Stanford Medicine, we are exploring the tremendous potential of stem cells and gene therapy to find definitive cures to rare childhood diseases for which there are currently no therapeutic options. We are seeking to cure the incurable disease.
The work of Maria Grazia Roncarolo, a pediatric immunologist and the co-director of Stanford’s Institute for Stem Cell Biology and Regenerative Medicine, presents some especially inspiring examples. Under her leadership, Lucile Packard Children’s Hospital Stanford performs blood stem cell transplants to cure children with genetic diseases and cancer. And she expects to begin testing several experimental stem cell gene therapies in patients next year. It has never been more exciting to see Stanford’s strengths in basic research translated to new medical therapies.
There is other hopeful news for stem cell and gene therapy on the horizon. A treatment for severe combined immune deficiency that Dr. Roncarolo developed while the director of the Telethon Institute for Gene Therapy and scientific director of the San Raffaele Scientific Institute in Italy is currently under review for marketing authorization at the European Medicines Agency — Europe’s equivalent of our Food and Drug Administration. If approved, the treatment for the condition, more widely known as “bubble boy disease,” would be the first stem cell gene therapy to be authorized by a major medical regulatory agency. (A second stem cell gene therapy for a lethal metabolic disease, also developed by Dr. Roncarolo’s team, will soon be submitted to the FDA for registration and marketing authorization.) Approval would mean the treatments are available like any other treatment and covered by insurance.
Treatments like these hold enormous promise and could usher in an era in which genetically modified stem cells are used to treat or cure a wide range of human diseases, presenting real hope for children whose lives had been devoid of it.
Now, Roncarolo and her team are busy researching cures for other congenital immune disorders and developing methods that could lead to stem cell treatments for a variety of diseases. And although we have a long bridge to cross between research and application, we eagerly look forward to a future where scraped knees are all that interrupt the boundless energy and hope of childhood.
Lloyd Minor, MD
Carl and Elizabeth Naumann Dean of the School of Medicine
Professor of Otolaryngology-Head & Neck Surgery