Public release date: 10-Oct-2012 [ | E-mail | Share ]

Contact: Jennifer O'Brien jennifer.obrien@ucsf.edu 415-502-6397 University of California - San Francisco

A Phase I clinical trial led by investigators from the University of California, San Francisco 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 the Oct. 10, 2012 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 Children's Hospital and a Howard Hughes Medical Institute Investigator.

"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 modest gains in neurological function, and while these can't 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."

In the trial, human neural stem cells developed by StemCells, 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).

In PMD, an inherited genetic defect prevents brain cells called oligodendrocytes from making myelin, a fatty material that insulates white matter which serves as a conduit for nervous impulses throughout the brain. Without myelin sheathing, white matter tracts short-circuit like bare electrical wires and are unable to correctly propagate nerve signals, resulting in neurological dysfunction and neurodegeneration. Patients with early-onset PMD cannot walk or talk, often have trouble breathing and undergo progressive neurological deterioration leading to death between ages 10 and 15.The disease usually occurs in males.

Multiple sclerosis and certain forms of cerebral palsy also involve damage to oligodendrocytes and subsequent demyelination.

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UCSF study shows evidence that transplanted neural stem cells produced myelin

Phase I Investigation Demonstrates Signs of Engraftment and Safety at One Year

Newswise A Phase I clinical trial led by investigators from the University of California, San Francisco 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 the Oct. 10, 2012 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.

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.

In the trial, human neural stem cells developed by StemCells, 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).

In PMD, an inherited genetic defect prevents brain cells called oligodendrocytes from making myelin, a fatty material that insulates white matter which serves as a conduit for nervous impulses throughout the brain. Without myelin sheathing, white matter tracts short-circuit like bare electrical wires and are unable to correctly propagate nerve signals, resulting in neurological dysfunction and neurodegeneration. Patients with early-onset PMD cannot walk or talk, often have trouble breathing and undergo progressive neurological deterioration leading to death between ages 10 and 15.The disease usually occurs in males.

Multiple sclerosis and certain forms of cerebral palsy also involve damage to oligodendrocytes and subsequent demyelination.

Before and after the transplant procedures in the children with PMD, which were conducted between 2010-2011, the patients were given standard neurological examinations and developmental assessments, and underwent magnetic resonance imaging (MRI). MRI is the most stringent non-invasive method we have of assessing myelin formation, said Rowitch. The investigators found evidence that the stem cells had successfully engrafted, receiving blood and nutrients from the surrounding tissue and integrating into the brain, a process that Rowitch likened to a plant taking root.

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Study Shows Evidence that Transplanted Neural Stem Cells Produced Myelin

BY LAURA OLENIACZ

loleniacz@heraldsun.com; 919-419-6636

DURHAM Stem cells from umbilical cord blood saved at 14-month-old Jase Howells birth are now being used in research to see if the cells can help his brain heal.

The research is looking into the use of the stem cells to treat brain damage from hydrocephalus, a condition characterized by the buildup of fluid in the skull.

His family traveled from Texas so he could receive an infusion on Tuesday at the Duke Childrens Hospital & Health Center of cord blood that was saved at his birth.

Mommys so proud of you, said LeaAnn Howell, to Jase, as he lay on a hospital bed, surrounded by medical personnel and family.

He periodically lifted his leg up and down to the beat of The Wheels on the Bus and other songs played by music therapist Tray Batson during the procedure.

Like I said, we were going to do anything humanly possible that we can do, Howell said in an interview prior to the procedure. Its a tough thing to fly, but once we (get here), I think the results are worth the wait, I guess.

The research into the use of cord blood stem cells to treat brain injury from hydrocephalus is being led by Dr. Joanne Kurtzberg, chief of the Division of Pediatric Blood and Marrow Transplantation at Duke.

The research is being done under a U.S. Food and Drug Administration Investigational New Drug application, Kurtzberg said.

Link:
Duke med school gets FDA approval for stem cell product

NEWARK, Calif., Oct. 10, 2012 (GLOBE NEWSWIRE) -- StemCells, Inc. (STEM) today announced that two papers reporting clinical and preclinical data demonstrating the therapeutic potential of the Company's proprietary HuCNS-SC(R) cells (purified human neural stem cells) for a range of myelination disorders were published in the Oct. 10 edition of Science Translational Medicine, the peer review journal of the American Association for the Advancement of Science (http://stm.sciencemag.org/).

The paper by Gupta, et al. describes the encouraging results of the Company's Phase I clinical trial in Pelizaeus-Merzbacher disease (PMD), a genetic myelination disorder that afflicts children. In the trial, which was completed in February 2012, four patients were transplanted with the Company's HuCNS-SC cells and all showed preliminary evidence of progressive and durable donor cell-derived myelination. Three of the four patients showed modest gains in their neurological function, which suggests a departure from the natural history of the disease; the fourth patient remained stable. Although clinical benefit cannot be confirmed in a trial without control patients, the small but measureable gains in function at one year may represent signals of a clinical effect to be further investigated in a controlled trial with more patients.

The second of the two papers, by Uchida, et al., summarizes extensive preclinical research which demonstrated that transplantation of the Company's neural stem cells in an animal model of severe myelin deficiency results in new myelin which enhanced the conductivity of nerve impulses. Myelin is the substance that insulates nerve axons, and without sufficient myelination, nerve impulses are not properly transmitted and neurological function is impaired. This preclinical data provided the rationale for the PMD clinical trial and supports the Company's cell therapy approach to other myelination disorders, such as transverse myelitis, certain forms of cerebral palsy, and multiple sclerosis.

"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," 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 modest gains in neurological function, and while these can't necessarily be attributed to the intervention because this was an uncontrolled trial with a small number of patients, it is an important first step which provides hope that HuCNS-SC transplantation may be able to address the fundamental pathology in the brain of PMD patients."

Patients with PMD have a defective gene which leads to insufficient myelin in the brain, which leads to a progressive loss of neurological function and death. In the clinical trial, four patients with connatal PMD, the most severe form of the disease, were enrolled and transplanted with HuCNS-SC cells. The patients were followed for twelve months after transplantation, during which time they underwent intensive neurological assessments and magnetic resonance (MR) imaging at regular intervals. The findings from the trial indicate a favorable safety profile for the HuCNS-SC cells and the transplantation procedure. Analysis of the MR imaging data showed changes consistent with increased myelination in the region of the transplantation, and which progressed over time and persisted after the withdrawal of immunosuppression at nine months. The results support the conclusion of durable cell engraftment and donor-derived myelin in the transplanted patients' brains. The development of new myelin signals is unprecedented in patients with connatal PMD. In addition, clinical assessment revealed small but measureable gains in motor and/or cognitive function in three of the four patients; the fourth patient remained clinically stable. While clinical benefit cannot be confirmed without a controlled study, these clinical outcomes suggest the HuCNS-SC cells may be having a beneficial effect on the patients.

The second paper, whose lead author is Nobuko Uchida, Vice President of Stem Cell Biology at StemCells, Inc., describes research which shows that when HuCNS-SC cells were transplanted into the shiverer mouse, a common model of severe central nervous system (CNS) dysmyelination, the cells formed new, functional myelin in the mice. Sophisticated analytical techniques were used to confirm that changes measured by MR images were in fact derived from new human myelin generated by the transplanted HuCNS-SC cells. MR imaging is routinely used in the diagnosis and clinical characterization of demyelinating diseases such as multiple sclerosis, and these results supported the use of similar techniques to detect and evaluate the degree of myelination in the Phase I PMD trial. Moreover, the new myelin was shown to be functional as conductivity of nerve impulses in the mice was enhanced.

"Demonstration of functional myelin formation in animals showing disease symptoms is significant and opens up the potential to treat patients with a range of severe myelin disorders," said Stephen A. Back, MD, PhD, professor of pediatrics and neurology at Oregon Health & Science University Doernbecher Children's Hospital, and senior author of the preclinical paper.

Stephen Huhn, MD, FACS, FAAP, Vice President and Head of the CNS Program at StemCells, Inc., added, "Having these two papers published concurrently illustrates the direct pathway of how we are translating groundbreaking scientific research to the clinical setting. The data in these papers make a powerful statement about the potential of our HuCNS-SC cells to address not only PMD, but a wide spectrum of myelination disorders. We are actively moving forward with our plans to conduct a controlled Phase II clinical study in PMD and evaluating our next steps with respect to other myelination disorders."

Conference Call

StemCells, Inc. will host a live webcast, today, October 10, at 4:30 p.m. Eastern Time (1:30 p.m. Pacific Time) to discuss the data reported in these papers. Interested parties are invited to view the webcast over the Internet via the link at http://www.stemcellsinc.com/News-Events/Events.htm. An archived version of the webcast will be available for replay on the Company's website approximately two hours following the conclusion of the live event and will be available for a period of 30 days.

Read more from the original source:
StemCells, Inc. Announces Simultaneous Publication of Preclinical and Clinical Results of Its Neural Stem Cells for ...

SAN FRANCISCO A Phase I clinical trial led by investigators from the University of California, San Francisco 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 today's (Oct. 10) 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, M.D., Ph.D., a professor of pediatrics and neurological surgery at UCSF, chief of neonatology at UCSF Benioff Children's Hospital and a Howard Hughes Medical Institute Investigator.

"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, M.D., Ph.D., 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 modest gains in neurological function, and while these can't 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."

In the trial, human neural stem cells developed by StemCells, Inc., of Newark, Calif., 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).

In PMD, an inherited genetic defect prevents brain cells called oligodendrocytes from making myelin, a fatty material that insulates white matter which serves as a conduit for nervous impulses throughout the brain. Without myelin sheathing, white matter tracts short-circuit like bare electrical wires and are unable to correctly propagate nerve signals, resulting in neurological dysfunction and neurodegeneration. Patients with early-onset PMD cannot walk or talk, often have trouble breathing and undergo progressive neurological deterioration leading to death between ages 10 and 15. The disease usually occurs in males.

Multiple sclerosis and certain forms of cerebral palsy also involve damage to oligodendrocytes and subsequent demyelination.

Before and after the transplant procedures in the children with PMD, which were conducted between 2010-11, the patients were given standard neurological examinations and developmental assessments, and underwent magnetic resonance imaging (MRI). "MRI is the most stringent non-invasive method we have of assessing myelin formation," said Rowitch.

Read more from the original source:
Trial: Transplanted neural stem cells produced myelin