New Stem Cell Gene Therapy Gives Hope to Prevent Inherited Neurological Disease

Source: University of Manchester
Date: 25 July 2013

Summary:

Scientists from The University of Manchester have used stem cell gene therapy to treat a fatal genetic brain disease in mice for the first time.  The method was used to treat Sanfilippo – a fatal inherited condition which causes progressive dementia in children – but could also benefit several neurological, genetic diseases.  Researchers behind the study, published in the journal Molecular Therapy this month, are now hoping to bring a treatment to trial in patients within two years.

A New Weapon Against Stroke: Stem Cell Study Uncovers the Brain-protective Powers of Astrocytes

Source: University of California - Davis

Date: July 23, 2013

Summary:

(SACRAMENTO, Calif.) — One of regenerative medicine’s greatest goals is to develop new treatments for stroke. So far, stem cell research for the disease has focused on developing therapeutic neurons — the primary movers of electrical impulses in the brain — to repair tissue damaged when oxygen to the brain is limited by a blood clot or break in a vessel. New UC Davis research, however, shows that other cells may be better suited for the task.  Published today in the journal Nature Communications, the large, collaborative study found that astrocytes — neural cells that transport key nutrients and form the blood-brain barrier — can protect brain tissue and reduce disability due to stroke and other ischemic brain disorders.

Scientists Successfully Generate 'Artificial Bones' from Umbilical Cord Stem Cells

Source: University of Granada


Date: 18 July 2013

Summary:

 Granada-based researchers patent a new biomaterial based on an activated carbon cloth support that acts as scaffolding for the construction of cells capable of bone regeneration. Although their results were obtained ‘ex vivo’, in the future they could help manufacture medicines to treat neoplastic, traumatic or degenerative bone pathologies. After obtaining artificial bone in the laboratory, the next step is to implant the biomaterial in animals to see if it can regenerate bone in them.

Scientists in Granada, Spain, have patented a new biomaterial that facilitates generating bone tissue—artificial bones in other words—from umbilical cord stem cells . The material, consisting of an activated carbon cloth support for cells that differentiate giving rise to a product that can promote bone growth, has recently been presented at a press conference at the Biomedical Research Centre, Granada.

Although the method has not yet been applied with ‘in vivo’ models, laboratory results are highly promising. In the future, they could help manufacture medicines for the repair of bone or osteochondrial, tumour or traumatic lesions and to replace lost cartilage in limbs. After obtaining artificial bones in the laboratory, the researchers' next step is to implant this biomaterial in experimental animal models—like rats or rabbits—to see if it can regenerate bone in them.

HIV Positive Men Show No Signs of HIV after Bone Marrow Transplant and Discontinuation of Anti-Retroviral Therapy

Source: Brigham and Women's Hospital

Date: July 3, 2013

Summary:

Boston, MA - Two Brigham and Women's Hospital patients with longstanding HIV infections who underwent bone marrow transplants have stopped anti-retroviral therapy and have no detectable HIV in their blood cells.  One patient stopped anti-retroviral therapy 15 weeks ago, the other stopped 7 weeks ago.  These new findings will be presented on July 3, 2013 at the International AIDS Society Conference (IAS 2013) in Kuala Lumpur, Malaysia by Timothy Henrich, MD and Daniel Kuritzkes, MD, physician-researchers in the Division of Infectious Diseases at Brigham and Women's Hospital.

DeGette And Dent Introduce Bipartisan Stem Cell Legislation

Source: Office of Diana DeGette
Date: June 19, 2013

Summary:

WASHINGTON – Today, U.S. Reps. Diana DeGette (D-CO) and Charlie Dent (R-PA) introduced the bipartisan Stem Cell Research Advancement Act, to ensure a lasting framework for ethical embryonic stem cell research at the National Institutes for Health (NIH), and to bring certainty to the scientific community pursuing research that could produce life-saving cures and treatments.

The Stem Cell Research Advancement Act would support embryonic stem cell research, and codify the NIH’s guidelines for carrying out all human stem cell research, embryonic and adult. It also requires NIH to review its guidelines at least every three years and make periodic updates as scientifically warranted.

Stem Cell Injections Improve Spinal Injuries in Rats

Source: University of California - San Diego
Date: May 28, 2013

Summary:

An international team led by researchers at the University of California, San Diego School of Medicine reports that a single injection of human neural stem cells produced neuronal regeneration and improvement of function and mobility in rats impaired by an acute spinal cord injury (SCI).  The findings are published in the May 28, 2013 online issue of Stem Cell Research & Therapy.

The rats received the pure stem cell grafts three days after injury (no other supporting materials were used) and were given drugs to suppress an immune response to the foreign stem cells. Marsala said grafting at any time after the injury appears likely to work in terms of blocking the formation of spinal injury cavities, but that more work would be required to determine how timing affects functional neurological benefit. The human stem cells, said the scientists, appeared to vigorously take root at the injury site.  Scientists observed the grafted stem cells appeared to be doing two things: stimulating host neuron regeneration and partially replacing the function of lost neurons.

The scientists used a line of human embryonic stem cells recently approved for Phase 1 human trials in patients with chronic traumatic spinal injuries. Marsala said the ultimate goal is to develop neural precursor cells (capable of becoming any of the three main cell types in the nervous system) from induced pluripotent stem cells derived from patients, which would likely eliminate the need for immunosuppression treatment.

Pending approval by UC San Diego’s Institutional Review Board, the next step is a small phase 1 trial to test safety and efficacy with patients who have suffered a thoracic spinal cord injury (between vertebrae T2-T12) one to two years earlier, and who have no motor or sensory function at or below the spinal injury site.

Human Skin Cells Converted Into Embryonic Stem Cells: First Time Human Stem Cells Have Been Produced Via Nuclear Transfer

Source: Oregon Health & Science University

Date: May 15, 2013

Summary:

Scientists at Oregon Health & Science University and the Oregon National Primate Research Center (ONPRC) have successfully reprogrammed human skin cells to become embryonic stem cells capable of transforming into any other cell type in the body. It is believed that stem cell therapies hold the promise of replacing cells damaged through injury or illness. Diseases or conditions that might be treated through stem cell therapy include Parkinson's disease, multiple sclerosis, cardiac disease and spinal cord injuries.

The research breakthrough, led by Shoukhrat Mitalipov, Ph.D., a senior scientist at ONPRC, follows previous success in transforming monkey skin cells into embryonic stem cells in 2007. This latest research will be published in the journal Cell online May 15 and in print June 6.

Sweet News for Stem Cell's 'Holy Grail'

Source: University of Manchester
Date: 26 February 2013

Summary:

Scientists have used sugar-coated scaffolding to move a step closer to the routine use of stem cells in the clinic and unlock their huge potential to cure diseases from Alzheimer's to diabetes.  Stem cells have the unique ability to turn into any type of human cell, opening up all sorts of therapeutic possibilities for some of the world's incurable diseases and conditions.  The problem facing scientists is how to encourage stem cells to turn into the particular type of cell required to treat a specific disease.

But researchers at the University of Manchester's School of Materials and Faculty of Life Sciences have developed a web-like scaffold, coated with long-sugar molecules, that enhances stem-cell cultures to do just this. The scaffold is formed by a process known as 'electrospinning', creating a mesh of fibres that mimic structures that occur naturally within the body.

The team's results – presented in the Journal of Biological Chemistry - are particularly promising, as the sugar molecules are presented on the surface of the fibres, retaining structural patterns important in their function. The sugars are also 'read' by the stem cells grown on the surface, stimulating and enhancing the formation of neuronal cell types.

Liver Stem Cells Grown in Culture, Transplanted With Demonstrated Therapeutic Benefit

Source: Oregon Health & Science University
Date: February 25, 2013

Summary:

For decades scientists around the world have attempted to regenerate primary liver cells known as hepatocytes because of their numerous biomedical applications, including hepatitis research, drug metabolism and toxicity studies, as well as transplantation for cirrhosis and other chronic liver conditions. But no lab in the world has been successful in identifying and growing liver stem cells in culture -- using any available technique -- until now.

In the journal Nature, physician-scientists in the Papé Family Pediatric Research Institute at Oregon Health & Science University Doernbecher Children's Hospital, Portland, Ore., along with investigators at the Hubrecht Institute for Developmental Biology and Stem Cell Research, Utrecht, Netherlands, describe a new method through which they were able to infinitely expand liver stem cells from a mouse in a dish.

In a previous Nature study, investigators at the Hubrecht Institute, led by Hans Clever, M.D, Ph.D., were the first to identify stem cells in the small intestine and colon by observing the expression of the adult stem cell marker Lgr5 and growth in response to a growth factor called Wnt. They also hypothesized that the unique expression pattern of Lgr5 could mark stem cells in other adult tissues, including the liver, an organ for which stem cell identification remained elusive.

In the current Nature study, Markus Grompe, M.D., study co-author, director of the Papé Family Pediatric Research Institute at OHSU Doernbecher Children's Hospital; and professor of pediatrics, and molecular and medical genetics in the OHSU School of Medicine.  Grompe and colleagues in the Papé Family Pediatric Research Institute at OHSU Doernbecher used a modified version of the Clever method and discovered that Wnt-induced Lgr5 expression not only marks stem cell production in the liver, but it also defines a class of stem cells that become active when the liver is damaged.

The scientists were able to grow these liver stem cells exponentially in a dish -- an accomplishment never before achieved -- and then transplant them in a specially designed mouse model of liver disease, where they continued to grow and show a modest therapeutic effect.

Ben's Stem Cell News Name and Address Change: Stem Cell Sentinel

Ben's Stem Cell News will soon be changing its name to the Stem Cell Sentinel and will be able to be accessed at the website address below:

http://stemcellsentinel.blogspot.com

Please check this address for all the latest stem cell research and science news.

StemCells, Inc. Announces First Patient Cohort Completes Spinal Cord Injury Trial - Gains in Sensory Function Persist 12 Months After Stem Cell Transplant

Source: StemCells, Inc.
Date: February 12, 2013

Summary:

NEWARK, Calif. -- StemCells, Inc. today announced that the twelve-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 continued to demonstrate a favorable safety profile, and showed that the considerable gains in sensory function observed in two of the three patients at the six-month assessment have persisted. The third patient remains stable. A summary of the data was presented today by Martin McGlynn, President and CEO, at the 15th Annual BIO CEO & Investor Conference. By completing the twelve-month assessment, the first patient cohort has now completed the trial, and has entered into a separate follow-up study for long-term observation.

Neuralstem Receives FDA Approval To Commence Spinal Cord Injury Trial

Source: Neuralstem, Inc.
Date: January 14, 2013

Summary:

ROCKVILLE, Md. -- Neuralstem, Inc. announced that it received approval from the United States Food and Drug Administration (FDA) to commence a Phase I safety trial of its lead cell therapy candidate, NSI-566, in chronic spinal cord injury patients. This open-label, multi-site study, will enroll up to eight patients with thoracic spinal cord injuries (T2-T12), who have an American Spinal Injury Association (AIS) A level of impairment, between one and two years after injury. AIS A impairment refers to a patient with no motor or sensory function in the relevant segments at and below the injury, and is considered to be complete paralysis.

The primary objective of the study is to determine the safety and toxicity of human spinal stem cell transplantation for the treatment of paralysis and related symptoms due to chronic spinal cord injury (SCI). The secondary objectives of the study are to evaluate graft survival in the transplant site by MRI, as well as the effectiveness of transient immunosuppression.

BioTime Signs Definitive Agreement With Geron Regarding Stem Cell Assets

Source: BioTime Inc.
Date: January 7, 2013

Summary:

ALAMEDA, Calif.-- BioTime, Inc. and its recently formed subsidiary BioTime Acquisition Corporation (BAC) jointly announced today that they have entered into a definitive Asset Contribution Agreement with Geron Corporation (Nasdaq: GERN) to acquire the intellectual property, including patents and patent applications, and other assets related to Geron’s human embryonic stem (hES) cell programs consistent with the financial terms outlined in the letter of intent announced on November 15, 2012.

Ordinary Heart Cells Become 'Biological Pacemakers' With Injection of Single Gene

Source: Cedars-Sinai Medical Center

Date: December 16, 2012

Summary:

LOS ANGELES – Cedars-Sinai Heart Institute researchers have reprogrammed ordinary heart cells to become exact replicas of highly specialized pacemaker cells by injecting a single gene (Tbx18) – a major step forward in the decade-long search for a biological therapy to correct erratic and failing heartbeats.  The advance will be published in the Jan 8 issue of Nature Biotechnology and also will be available today on the journal’s website.

Cedars-Sinai researchers, employing a virus engineered to carry a single gene (Tbx18) that plays a key role in embryonic pacemaker cell development, directly reprogrammed heart muscle cells (cardiomyocytes) to specialized pacemaker cells. The new cells took on the distinctive features and function of native pacemaker cells, both in lab cell reprogramming and in guinea pig studies.

If subsequent research confirms and supports findings of the pacemaker cell studies, the researchers said they believe therapy might be administered by injecting Tbx18 into a patient’s heart or by creating pacemaker cells in the laboratory and transplanting them into the heart. But additional studies of safety and effectiveness must be conducted before human clinical trials could begin.

Salk Scientists Develop Faster, Safer Method for Producing Stem Cells

Source: Salk Institute for Biological Studies

Date: December 3, 2012

Summary:

LA JOLLA, CA—A new method for generating stem cells from mature cells promises to boost stem cell production in the laboratory, helping to remove a barrier to regenerative medicine therapies that would replace damaged or unhealthy body tissues.

The technique, developed by researchers at the Salk Institute for Biological Studies, allows for the unlimited production of stem cells and their derivatives as well as reduces production time by more than half, from nearly two months to two weeks.

They and their colleagues, including Fred H. Gage, professor in Salk's Laboratory of Genetics, have published a new method for converting cells in this week's Nature Methods.

Stem Cell-Derived Dopaminergic Neurons Rescue Motor Defects in Parkinsonian Monkeys

Source: Journal of Clinical Investigation

Date December 3, 2012

 Researchers have derived dopaminergic neurons from bone marrow stem cells in monkeys.

Parkinson's disease is a degenerative disorder of the central nervous system that is characterized by tremors, rigidity, slowness of movement, and difficulty walking. It is caused by loss of the neurons that produce the neurotransmitter dopamine (known as dopaminergic neurons). One of the primary goals in Parkinson's disease research is to develop a replacement for dopaminergic neurons.

In a new study, researchers led by Takuya Hayashi at the RIKEN Center for Molecular Imaging Science in Kobe, Japan, derived dopaminergic neurons from bone marrow stem cells in monkeys. The cells were retrieved during a standard bone marrow aspiration and then treated with growth factors that directed the stem cells to become dopaminergic neurons. The monkeys that donated the stem cells were treated with a chemical to induce Parkinson's disease and then received a transplant of the new dopaminergic neurons that had been derived from their own bone marrow stem cells. Monkeys that received the transplant showed significant improvement in motor defects.

This study demonstrates that dopaminergic neurons derived from adult bone marrow stem cells can be safely used to improve motor function in Parkinson's disease in monkeys.

The research is published in the Journal of Clinical Investigation.

Neurons Made from Stem Cells Drive Brain Activity After Transplantation in Laboratory Model

Source: Sanford-Burnham Medical Research Institute

Date: November 15, 2012

Summary:

Researchers and patients look forward to the day when stem cells might be used to replace dying brain cells in Alzheimer's disease and other neurodegenerative conditions. Scientists are currently able to make neurons and other brain cells from stem cells, but getting these neurons to properly function when transplanted to the host has proven to be more difficult. Now, researchers at Sanford-Burnham Medical Research Institute have found a way to stimulate stem cell-derived neurons to direct cognitive function after transplantation to an existing neural network.

The study was published November 7 in the Journal of Neuroscience.

Researchers Develop Efficient, Protein-based Method For Creating iPS Cells

Source: Stanford University School of Medicine
Date: October 25, 2012

Summary:

Coaxing a humble skin cell to become a jack-of-all-trades pluripotent stem cell is feat so remarkable it was honored earlier this month with the Nobel Prize in Physiology or Medicine. Stem cell pioneer Shinya Yamanaka, MD, PhD, showed that using a virus to add just four genes to the skin cell allowed it to become pluripotent, or able to achieve many different developmental fates. But researchers and clinicians have been cautious about promoting potential therapeutic uses for these cells because the insertion of the genes could render the cells cancerous.

Now researchers at the Stanford University School of Medicine have devised an efficient and safer way to make these induced pluripotent stem cells, or iPS cells, by using just the proteins that the genes encode.

The research is published in the Oct. 26 issue of Cell.

Researchers at the Doorstep of Stem Cell Therapies for MS, Other Myelin Disorders

Source: University of Rochester Medical Center

Date: October 25, 2012

Summary:

When the era of regenerative medicine dawned more than three decades ago, the potential to replenish populations of cells destroyed by disease was seen by many as the next medical revolution. However, what followed turned out not to be a sprint to the clinic, but rather a long tedious slog carried out in labs across the globe required to master the complexity of stem cells and then pair their capabilities and attributes with specific diseases.

In a review article appearing today in the journal Science, University of Rochester Medical Center scientists Steve Goldman, M.D., Ph.D., ≈, and Martha Windrem, Ph.D., contend that researchers are now on the threshold of human application of stem cell therapies for a class of neurological diseases known as myelin disorders – a long list of diseases that include conditions such as multiple sclerosis, white matter stroke, cerebral palsy, certain dementias, and rare but fatal childhood disorders called pediatric leukodystrophies.

Scientists Pinpoint Key Player in Parkinson's disease neuron loss Stem cell study may help to unravel how a genetic mutation leads to Parkinson's Symptoms

Source: Salk Institute for Biological Studies
Date: October 19, 2012

LA JOLLA, CA—By reprogramming skin cells from Parkinson's disease patients with a known genetic mutation, researchers at the Salk Institute for Biological Studies have identified damage to neural stem cells as a powerful player in the disease. The findings, reported online October 17, 2012 in Nature, may lead to new ways to diagnose and treat the disease.

The scientists found that a common mutation to a gene that produce the enzyme LRRK2, which is responsible for both familial and sporadic cases of Parkinson's disease, deforms the membrane surrounding the nucleus of a neural stem cell. Damaging the nuclear architecture leads to destruction of these powerful cells, as well as their decreased ability to spawn functional neurons, such as the ones that respond to dopamine.

The Salk researchers found that a common genetic mutation involved in Parkinson's disease deforms the membranes (green) surrounding the nuclei (blue) of neural stem cells. The discovery may lead to new ways to diagnose and treat the disease.