David Rowitch, MD, PhD, professor and chief of neonatology, in the NICU.
A Phase I clinical trial led by investigators from the University of California, San Francisco (UCSF) and sponsored by Stem Cells Inc., showed that neural stem cells successfully engrafted into the brains of patients and appear to have produced myelin.
The study, published in Wednesday's issue of Science Translational Medicine, also demonstrated that the neural stem cells were safe in the patients brains one year post transplant.
The results of the investigation, designed to test safety and preliminary efficacy, are encouraging, said principal investigator David H. Rowitch, MD, PhD, a professor of pediatrics and neurological surgery at UCSF, chief of neonatology at UCSF Benioff Childrens Hospital and a Howard Hughes Medical Institute Investigator.
Nalin Gupta, MD, PhD
For the first time, we have evidence that transplanted neural stem cells are able to produce new myelin in patients with a severe myelination disease, said Nalin Gupta, MD, PhD, associate professor of neurological surgery and pediatrics and chief of pediatric neurological surgery at UCSF Benioff Children's Hospital, and co-principal investigator of the PMD clinical trial.
We also saw modestgains in neurological function, and while these cant necessarily be attributed to the intervention because this was an uncontrolled trial with a small number of patients,the findings represent an important first step that strongly supports further testing of this approach as a means to treat the fundamental pathology in the brain of these patients.
The study, one of the first neural stem cell trials ever conducted in the United States, is emblematic of UCSFs pioneering role in the stem cell field. In 1981, Gail Martin, PhD, professor of anatomy, co-discovered embryonic stem cells in mice. In 2001, Roger Pedersen, PhD, professor emeritus of obstetrics, gynecology and reproductive sciences, derived two of the first human embryonic stem cell lines. On Monday, Shinya Yamanaka, MD, PhD, of the UCSF-affiliated Gladstone Institutes and Kyoto University, received the Nobel Prize in Physiology or Medicine for his discovery that adult cells can be reprogrammed to behave like embryonic stem cells.
In the trial, human neural stem cells developed by Stem Cells, Inc., of Newark, California, were injected directly into the brains of four young children with an early-onset, fatal form of a condition known as Pelizaeus-Merzbacher disease (PMD).
This image illustrates direct injection of human neural stem cells into the brain's white matter, which is composed of bundles of nerve axons. There is lack of myelin, an insulating coating, in the severe pediatric condition Pelizaeus-Merzbacher disease (PMD). Over time, some stem cells become myelinating oligodendrocytes as reported in the papers from Uchida et al. and Gupta et al. Image by Kenneth Probst.
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Transplanted Neural Stem Cells Produced Myelin, UCSF Study Shows