Thursday, September 13, 2012

Neuralstem Cells Induce Significant Functional Improvement In Permanent Rat Spinal Cord Injury, Cell Study Reports

Source: Neuralstem, Inc.
Date: September 13, 2012

Summary;

ROCKVILLE, Md. -- Neuralstem, Inc. announced that its neural stem cells were part of a study, "Long-Distance Growth and Connectivity of Neural Stem Cells After Severe Spinal Cord Injury: Cell-Intrinsic Mechanisms Overcome Spinal Inhibition," published online today in a leading scientific journal CELL. In the study, rats with surgically transected spinal cords, which rendered them permanently and completely paraplegic, were transplanted with Neuralstem's spinal cord stem cells (NSI-566). The study reports that the animals recovered significant locomotor function, regaining movement in all lower extremity joints, and that the transplanted neural stem cells turned into neurons which grew a "remarkable" number of axons that extended for "very long distances" over 17 spinal segments, making connections both above and below the point of severance. These axons reached up to the cervical region (C4) and down to the lumbar region (L1). They also appeared to make reciprocal synaptic connectivity with the host rat spinal cord neurons in the gray matter for several segments below the injury.

Further study showed that re-transecting the spinal cord immediately above the graft abolished the functional gain, indicating that the regeneration of host axons into the human stem cell graft was responsible for the functional recovery. The cells that Neuralstem contributed to the study, NSI-566, are the same cells used in the recently completed Phase 1 clinical trial for the treatment of amyotrophic lateral sclerosis (ALS or Lou Gehrig's disease). Neuralstem has also submitted an application to the FDA for a trial to treat chronic spinal cord injury with these cells.

Wednesday, September 12, 2012

Human embryonic stem cells could help to treat deafness

Source: University of Sheffield
Date: 12 September 2012

Summary:

In research funded by the Medical Research Council and leading UK research charity, Action on Hearing Loss, experts from the University of Sheffield’s Department of Biomedical Sciences developed a method to turn human embryonic stem cells into ear cells. They then transplanted them into deaf gerbils, obtaining a functional recovery that, on average, was of around 46 per cent. The improvement was evident about four weeks after administering the cells. As well as proving that stem cells can be used to repair damaged hearing, it is hoped the breakthrough – published in the journal Nature – will lead to new treatments and therapies in the future.

Tuesday, September 11, 2012

Stem Cell Researchers Use Gene Therapy to Restore Immune Systems in 'Bubble Boy' Disease

Source: University of California, Los Angeles (UCLA), Health Sciences
Date: September 11, 2012

Summary:

UCLA stem cell researchers have found that a gene therapy regimen can safely restore immune systems to children with so-called "Bubble Boy" disease, a life threatening condition that if left untreated can be fatal within one to two years.

In the 11-year study, researchers were able to test two therapy regimens for 10 children with ADA-deficient severe combined immunodeficiency (SCID). During the study, they refined their approach to include a light dose of chemotherapy to help remove many of the blood stem cells in the bone marrow that are not creating an enzyme called adenosine deaminase (ADA), which is critical for the production and survival of healthy white blood cells, said study senior Dr. Donald Kohn, a professor of pediatrics and of microbiology, immunology, and molecular genetics in Life Sciences and a member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA.

The refined gene therapy and chemotherapy regimen proved superior to the other method tested in the study, restoring immune function to three of the six children who received it, Kohn said. Going forward, an even further refined regimen using a different type of virus delivery system will be studied in the next phase of the study, which already has enrolled eight of the 10 patients needed.

The study appears Aug. 30 in the advance online issue of the peer-reviewed journal Blood.

Monday, September 10, 2012

New Genetic Mechanism for Controlling Blood Cell Development and Blood Vessel Integrity Found

Source: University of Wisconsin-Madison
Date: September 10, 2012

Summary:

The protein GATA2 is known as a "master regulator" of blood cell development. When a mutation occurs in the gene that makes GATA2, serious blood diseases such as acute myeloid leukemia can result. Zooming in on the GATA2 gene, University of Wisconsin-Madison researchers and their collaborators at the National Institutes of Health (NIH) have discovered unexpectedly that a small DNA sequence drives this powerful master regulator. The sequence plays an essential role in controlling GATA2 production and generating self-renewing blood stem cells responsible for the earliest steps in the development of blood cells of all kinds — red cells to transport oxygen and white cells to fight infection.

The researchers also found that the DNA sequence, which they call the +9.5 GATA2 switch site, ensures that blood vessels function properly to prevent hemorrhaging. Until now, GATA2 had not been implicated in blood vessel integrity. The study appears in The Journal of Clinical Investigation (online Sept. 10, 2012).

Monday, September 03, 2012

StemCells, Inc. Reports Positive Interim Data From Spinal Cord Injury Trial Cells and Procedure Well Tolerated; Gains in Sensory Function Confirmed

Source: StemCells, Inc.
Date: September 3, 2012

Summary:

NEWARK, Calif. -- StemCells, Inc. today announced that interim six-month data from the first patient cohort in the Company's Phase I/II clinical trial of its proprietary HuCNS-SC® product candidate (purified human neural stem cells) for chronic spinal cord injury continues to demonstrate a favorable safety profile, and shows considerable gains in sensory function in two of the three patients compared to pre-transplant baselines. The third patient remains stable. The data was presented by Armin Curt, M.D., principal investigator for the clinical trial, at the 51st Annual Scientific Meeting of the International Spinal Cord Society in London, England. The trial represents the first time that neural stem cells have been transplanted as a potential therapeutic agent for spinal cord injury.

Patients in the study's first cohort all suffered a complete injury to the thoracic (chest-level) spinal cord. In a complete injury, there is no neurological function below the level of injury. All three patients were transplanted four to nine months after injury with a dose of 20 million cells at the site of injury. The surgery, immunosuppression and the cell transplants have been well tolerated by all the patients. There were no abnormal clinical, electrophysiological or radiological responses to the cells, and all the patients were neurologically stable through the first six months following transplantation. Changes in sensitivity to touch, heat and electrical stimuli were observed in well-defined and consistent areas below the level of injury in two of the patients, while no changes were observed in the third patient. Importantly, tests of perception of different sensory stimuli as well as measures of electrical impulse transmission across the site of injury correlate with the clinical examination, providing independent and objective confirmation of the changes in sensory function.

Sunday, September 02, 2012

Scientists Discover 'Missing Link' Between Stem Cells and the Immune System

Source: University of California, Los Angeles (UCLA), Health Sciences
Date: September 2, 2012

Summary:

UCLA researchers have discovered a type of cell that is the "missing link" between bone marrow stem cells and all the cells of the human immune system, a finding that will lead to a greater understanding of how a healthy immune system is produced and how disease can lead to poor immune function. The studies were done using human bone marrow, which contains all the stem cells that produce blood during postnatal life.

Understanding the process of normal blood formation in human adults is a crucial step in shedding light on what goes wrong during the process that results in leukemias, or cancers of the blood. The study appears Sept. 2 in the early online edition of Nature Immunology.