University of Maryland School of Medicine scientists develop stem cell model for hereditary disease

Public release date: 15-Oct-2012 [ | E-mail | Share ]

Contact: Karen Robinson karobinson@som.umaryland.edu 410-706-7590 University of Maryland Medical Center

A new method of using adult stem cells as a model for the hereditary condition Gaucher disease could help accelerate the discovery of new, more effective therapies for this and other conditions such as Parkinson's, according to new research from the University of Maryland School of Medicine.

Scientists at the University of Maryland School of Medicine reprogrammed stem cells to develop into cells that are genetically similar to and react to drugs in a similar way as cells from patients with Gaucher disease. The stem cells will allow the scientists to test potential new therapies in a dish, accelerating the process toward drug discovery, according to the paper published online in the journal the Proceedings of the National Academy of Sciences (PNAS) on Oct. 15 (Panicker et.al.).

The study was funded with $1.7 million in grants from the Maryland Stem Cell Research Fund; researchers received a start-up grant for $200,000 in 2007 and a larger, five-year grant for $1.5 million in 2009.

"We have created a model for all three types of Gaucher disease, and used stem cell-based tests to evaluate the effectiveness of therapies," says senior author Ricardo Feldman, Ph.D., associate professor of microbiology and immunology at the University of Maryland School of Medicine, and a research scientist at the University of Maryland Center for Stem Cell Biology and Regenerative Medicine. "We are confident that this will allow us to test more drugs faster, more accurately and more safely, bringing us closer to new treatments for patients suffering from Gaucher disease. Our findings have potential to help patients with other neurodegenerative diseases as well. For example, about 10 percent of Parkinson's disease patients carry mutations in the recessive gene for Gaucher disease, making our research possibly significant for Parkinson's disease as well."

Gaucher disease is the most frequent lipid-storage disease. It affects 1 in 50,000 people in the general population. It is most common in Ashkenazi Jews, affecting 1 in 1,000 among that specific population. The disease occurs in three subtypes Type 1 is the mildest and most common form of the disease, causing symptoms such as enlarged livers and spleens, anemia and bone disease. Type 2 causes very serious brain abnormalities and is usually fatal before the age of two, while Type 3 affects children and adolescents.

The condition is a recessive genetic disorder, meaning that both parents must be carriers for a child to suffer from Gaucher. However, said Dr. Feldman, studies have found that people with only one copy of a mutated Gaucher gene those known as carriers are at an increased risk of developing Parkinson's disease.

"This science is a reflection of the mission of the University of Maryland School of Medicine to take new treatments from bench to bedside, from the laboratory to patients, as quickly as possible," says E. Albert Reece, M.D., Ph.D., M.B.A., vice president for medical affairs at the University of Maryland and John Z. and Akiko K. Bowers Distinguished Professor and dean of the University of Maryland School of Medicine. "We are excited to see where this research goes next, bringing new hope to Gaucher patients and their families."

Dr. Feldman and his colleagues used the new reprogramming technology developed by Shinja Yamanaka in Japan, who was recognized with this year's Nobel Prize for Medicine or Physiology. Scientists engineered cells taken from the skin of Gaucher patients, creating human induced pluripotent stem cells, known as hiPSC stem cells that are theoretically capable of forming any type of cell in the body. Scientists differentiated the cells to form white blood cells known as macrophages and neuronal cells.

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University of Maryland School of Medicine scientists develop stem cell model for hereditary disease

RBCC: Could Stem Cells Be Key to Promising Autism Therapy?

NOKOMIS, Fla.--(BUSINESS WIRE)--

Rainbow Coral Corp. (RBCC) subsidiary Rainbow BioSciences will keep a close eye on a new study that could potentially lead to stem cell therapies for children with autism.

Researchers have been given the go-ahead by the FDA to launch a small study evaluating the effectiveness of autism treatments using patients own umbilical cord blood. Thirty children, aged two to seven, will receive injections of their own stem cells from the cord blood banked by their parents at birth.

Scientists will evaluate whether the stem cell therapy helps improve language and behavior in the children. Although the cause of autism is unknown and there is no cure for the disorder, one theory suggests that autism occurs because cell in the brain, known as neurons, are not connecting normally. Its possible that stem cells may address this problem.

RBCC is working to capitalize on the rising demand for effective new stem cell treatments by bringing a potentially game-changing stem cell technology to market. The company is close to a deal with Regenetech to acquire a license to perform cell expansion using that companys Rotary Cell Culture SystemTM, a rotating-wall bioreactor originally developed by NASA.

The rotating-wall bioreactor is capable of multiplying functional, 3-D stem cells for use in a variety of research projects, said RBCC CEO Patrick Brown. Stem cells carry tremendous potential to help researchers develop new treatments and cures for devastating diseases from Parkinsons to Alzheimers and even autism, but much research must be done first. Consequently, were very optimistic about the market potential for this revolutionary bioreactor technology.

RBCC plans to offer the new technology to help kickstart billions of dollars worth of research in an industry currently dominated by Amgen, Inc. (AMGN), Celgene Corporation (CELG), Genzyme Corp. (NASDAQ:GENZ) and Gilead Sciences Inc. (GILD).

For more information on Rainbow BioSciences, please visit http://www.rainbowbiosciences.com/investors.html.

About Rainbow BioSciences

Rainbow BioSciences, LLC, is a wholly owned subsidiary of Rainbow Coral Corp. (OTCBB:RBCC). The company continually seeks out new partnerships with biotechnology developers to deliver profitable new medical technologies and innovations. For more information on our growth-oriented business initiatives, please visit our website at [http://www.RainbowBioSciences.com]. For investment information and performance data on the company, please visit http://www.RainbowBioSciences.com/investors.html.

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Realizing the potential of stem cell therapy

Public release date: 15-Oct-2012 [ | E-mail | Share ]

Contact: Kat Snodgrass 202-962-4090 Society for Neuroscience

NEW ORLEANS New animal studies provide additional support for investigating stem cell treatments for Parkinson's disease, head trauma, and dangerous heart problems that accompany spinal cord injury, according to research findings released today. The work, presented at Neuroscience 2012, the annual meeting of the Society for Neuroscience and the world's largest source of emerging news about brain science and health, shows scientists making progress toward using stem cell therapies to repair neurological damage.

The studies focused on using stem cells to produce neurons essential, message-carrying cells in the brain and spinal cord. The loss of neurons and the connections they make for controlling critical bodily functions are the chief hallmarks of brain and spinal cord injuries and of neurodegenerative afflictions such as Parkinson's disease and ALS (amyotrophic lateral sclerosis), also known as Lou Gehrig's disease.

Today's new findings show that:

Other recent findings discussed show that:

"As the fields of developmental and regenerative neuroscience mature, important progress is being made to begin to translate the promise of stem cell therapy into meaningful treatments for a range of well-defined neurological problems," said press conference moderator Jeffrey Macklis, MD, of Harvard University and the Harvard Stem Cell Institute, an expert on development and regeneration of the mammalian central nervous system. "Solid, rigorous, and well-defined pre-clinical work in animals can set the stage toward human clinical trials and effective future therapies."

###

This research was supported by national funding agencies such as the National Institutes of Health, as well as private and philanthropic organizations.

Todd Bentsen, (202) 962-4086

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Realizing the potential of stem cell therapy

Beauty salon ‘offers’ stem cell therapy

With all the publicity about the miraculous effects of stem cell therapy, the Department of Health (DOH) should prepare itself for the possibility that the new procedure would be performed by unqualified, and completely clueless, people.

I passed a beauty parlor recently and saw a huge poster on its door announcing the arrival of stem cell therapy. I was instantly reminded of botched breast enhancement and nose jobs performed by salon personnel who seemed to think it was as easy to learn complicated surgical procedures as it was to train to cut hair or do manicures and pedicures.

The DOH should start warning the public not to fall for these special offers just because they are available at giveaway rates.

Modern lifestyle problem

Experts have repeatedly talked about problems brought about by modern lifestyles. Changing diets and stress are two of the best known. Dr. Jaime G. Ignacio, section chief of gastroenterology at Veterans Hospital and head of the Digestive Malignancy Council of the Philippine Society of Gastroenterology, said constipation could be one of the consequences of the combination of these two factors.

Speaking at an event hosted by Boehringer Ingelheim, maker of Dulcolax (generic name Bisacodyl), a formulation for constipation relief, Ignacio, who, as a gastroenterologist is a specialist in digestive system disorders, defined the problem as having fewer than three bowel movements in a week (normal ranges from three times a week to three times a day).

He said constipation itself was not a disease but it could sometimes be a symptom of something serious, like colorectal cancer. But he said about 95 percent of cases were acuteoccurring suddenly and lasting for only a short periodresulting from some sudden lifestyle or hormonal changes, the taking of medication, lack of exercise, etc.

Ignacio said acute was easy to treat, with products like Dulcolax to solve the problem. But, if left unattended, acute constipation could lead to a chronic or long-term condition, which was the more worrisome, and would need medical attention.

He said constipation should be treated as soon as the problem had lasted for four or more days.

Constipation is part of modern living. [Like other diseases] prevention is the key. Safe and effective treatment is available [if needed], Ignacio stressed.

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Beauty salon ‘offers’ stem cell therapy

Chinese bid to save girl's sight

Chloe Wilson, from Forrester, Edinburgh, was born with septo-optic dysplasia a rare condition which means her pituitary gland has not developed properly.

As a result, the 19-month-old's optic nerve is underdeveloped, leaving her with hormone deficiencies and very little vision.

Her parents, Kayleigh and Scott Wilson, both 22, started a campaign in April to raise 15,000 to take her to the Chinese city of Guangzhou to undergo a controversial stem-cell treatment not available in the UK.

But thanks to the efforts of many other fundraisers and donors, some of whom have never even met the family, the total raised now stands at nearly 25,000.

The total means the young family can afford far more stem- cells, vastly increasing the chance of success.

Mrs Wilson, a nursery assistant, said: "I thought we were looking at a year-and-a-half before we hit our target. Now we've gone nearly ten grand over it in less than half that time.

"We just want to thank everyone who has helped give us this chance. People have been amazing,. Their generosity will stay with us forever, and we'll make sure Chloe knows all about it when she is older."

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Chinese bid to save girl's sight

Stem cell model for hereditary disease developed

ScienceDaily (Oct. 15, 2012) A new method of using adult stem cells as a model for the hereditary condition Gaucher disease could help accelerate the discovery of new, more effective therapies for this and other conditions such as Parkinson's, according to new research from the University of Maryland School of Medicine.

Scientists at the University of Maryland School of Medicine reprogrammed stem cells to develop into cells that are genetically similar to and react to drugs in a similar way as cells from patients with Gaucher disease. The stem cells will allow the scientists to test potential new therapies in a dish, accelerating the process toward drug discovery, according to the paper published online in the journal the Proceedings of the National Academy of Sciences (PNAS) on Oct. 15.

"We have created a model for all three types of Gaucher disease, and used stem cell-based tests to evaluate the effectiveness of therapies," says senior author Ricardo Feldman, Ph.D., associate professor of microbiology and immunology at the University of Maryland School of Medicine, and a research scientist at the University of Maryland Center for Stem Cell Biology and Regenerative Medicine. "We are confident that this will allow us to test more drugs faster, more accurately and more safely, bringing us closer to new treatments for patients suffering from Gaucher disease. Our findings have potential to help patients with other neurodegenerative diseases as well. For example, about 10 percent of Parkinson's disease patients carry mutations in the recessive gene for Gaucher disease, making our research possibly significant for Parkinson's disease as well."

Gaucher disease is the most frequent lipid-storage disease. It affects 1 in 50,000 people in the general population. It is most common in Ashkenazi Jews, affecting 1 in 1,000 among that specific population. The disease occurs in three subtypes -- Type 1 is the mildest and most common form of the disease, causing symptoms such as enlarged livers and spleens, anemia and bone disease. Type 2 causes very serious brain abnormalities and is usually fatal before the age of two, while Type 3 affects children and adolescents.

The condition is a recessive genetic disorder, meaning that both parents must be carriers for a child to suffer from Gaucher. However, said Dr. Feldman, studies have found that people with only one copy of a mutated Gaucher gene -- those known as carriers -- are at an increased risk of developing Parkinson's disease.

"This science is a reflection of the mission of the University of Maryland School of Medicine -- to take new treatments from bench to bedside, from the laboratory to patients, as quickly as possible," says E. Albert Reece, M.D., Ph.D., M.B.A., vice president for medical affairs at the University of Maryland and John Z. and Akiko K. Bowers Distinguished Professor and dean of the University of Maryland School of Medicine. "We are excited to see where this research goes next, bringing new hope to Gaucher patients and their families."

Dr. Feldman and his colleagues used the new reprogramming technology developed by Shinja Yamanaka in Japan, who was recognized with this year's Nobel Prize for Medicine or Physiology. Scientists engineered cells taken from the skin of Gaucher patients, creating human induced pluripotent stem cells, known as hiPSC -- stem cells that are theoretically capable of forming any type of cell in the body. Scientists differentiated the cells to form white blood cells known as macrophages and neuronal cells.

A key function of macrophages in the body is to ingest and eliminate damaged or aged red blood cells. In Gaucher disease, the macrophages are unable to do so -- they can't digest a lipid present in the red blood cell membrane. The macrophages become engorged with lipid and cannot completely clear the ingested red blood cells. This results in blockage of membrane transport pathways in the macrophages lodged in the bone marrow, spleen and liver. The macrophages that the scientists created from the reprogrammed stem cells exhibited this characteristic hallmark of the macrophages taken from Gaucher patients.

To further test the stem cells, the scientists administered a recombinant enzyme that is effective in treating Gaucher patients with Type 1 disease. When the cells were treated with the enzyme, the function of the macrophages was restored -- they completely cleared the red blood cells.

"The creation of these stem cell lines is a lovely piece of stem cell research," said Curt Civin, M.D., professor of pediatrics and physiology, associate dean for research and founding director of the Center for Stem Cell Biology & Regenerative Medicine at the University of Maryland School of Medicine. "Dr. Feldman is already using these Gaucher patient-derived macrophages to better understand the disease fundamentals and to find novel medicines for Gaucher disease treatment. A major goal of our Center for Stem Cell Biology & Regenerative Medicine is to translate our fundamental discoveries into innovative and practical clinical applications that will enhance the understanding, diagnosis, treatment, and prevention of many human diseases. Clinical applications include not only transplantation of stem cells, but also the use of stem cells for drug discovery as Dr. Feldman's studies so beautifully illustrate."

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Stem cell model for hereditary disease developed

StemCells, Inc. Launches Four New Human Neural Stem Cell Kits Under SC Proven(R) Brand

NEWARK, Calif., Oct. 16, 2012 (GLOBE NEWSWIRE) -- StemCells, Inc. (STEM) announced today the launch of four new SC Proven human neural stem cell (NSC) kits for use in neuroscience research. Each kit will contain high purity, multipotent NSCs derived from a different area of the human central nervous system (CNS), and will provide researchers with a reproducible and scalable serum-free platform with which to perform a broad range of assays. With these kits, researchers will now have the ability to compare and contrast the biological, functional and neural differentiation properties of human NSCs isolated from specific CNS regions, as well as to screen for the effects of different compounds on such cells.1,2

"These kits represent the first in a new family of human cell-centric products we are adding to the SC Proven portfolio to provide researchers with a unique set of tools to realize the promise of regenerative medicine," said Stewart Craig, Ph.D., Senior Vice President, Development and Operations at StemCells, Inc. "Stem cell research is flourishing and these kits will enable investigators to derive and characterize human neural lineage cells using published methods, or the ability to customize their own assay formats up to and including scale-up for non-commercial screening applications."

Kits containing multipotent human NSCs derived from Hindbrain (HNS-HIN-001), Cortex (HNS-COR-001), Spinal Cord (HNS-SPI-001), and Mid-forebrain (HNS-MIF-001), and RHB-A(R), StemCells Inc.'s proprietary serum-free cell culture medium, are now available. For a limited time a special discount of 20% can be obtained when placing an online order with the Discount Code JAV66.

References

1 Hook L, et al., Non-immortalized human neural stem cells as a scalable platform for cellular assays. Neurochem Int. 2011 59(3): 432-44.

2 McLaren D, et al., Automated large-scale culture and medium-throughput chemical screen for modulators of proliferation and viability of human Induced Pluripotent Stem Cell-derived Neuroepithelial-like Stem Cells. J Biomol Screen. Oct 4: 2012 doi:10.1177/1087057112461446.

About SC Proven Products

The SC Proven product portfolio comprises a range of products for the detection, isolation, expansion, differentiation, and characterization of a variety of different human and animal cell types. The entire SC Proven product catalog and online ordering can be found at http://www.scproven.com.

About StemCells, Inc.

StemCells, Inc. is engaged in the research, development, and commercialization of cell-based therapeutics and tools for use in stem cell-based research and drug discovery. The Company's lead therapeutic product candidate, HuCNS-SC(R) cells (purified human neural stem cells), is currently in development as a potential treatment for a broad range of central nervous system disorders, including Pelizaeus-Merzbacher disease (PMD), a fatal myelination disorder, chronic spinal cord injury, and dry age-related macular degeneration (AMD). StemCells also markets a range of stem cell research products under the SC Proven(R) brand (www.scproven.com), and offers contract cell process development and production services (cellservices@stemcellsinc.com). Further information is available at http://www.stemcellsinc.com.

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StemCells, Inc. Launches Four New Human Neural Stem Cell Kits Under SC Proven(R) Brand

Stem Longevity Research Links with BioProtein Technology – Renowned Partnership to Elevate Brand’s Innovative …

BioProtein Technology, a company that manufactures therapeutic proteins in the form of growth factors, has announced an alliance with Stem Longevity Research, a company partnered by Dr. Joseph Purita and CNC Reid Eckert.

(PRWEB) October 15, 2012

Stem Longevity Research has expertise in the medical applications of stem cells. Dr. Purita and Eckert will offer their high level of credibility to increase the exposure of BioProtein Technology's products on the part of physicians across the United States.

We are thrilled to welcome Dr. Purita and Reid Eckert to our team, as they are among the world's most respected stem cell researchers, said Paul Morave, National Sales Director for BioProtein Technology. With their endorsement of our products, we immediately gain a tremendous level of credibility in the eyes of doctors and medical professionals. Were very excited to begin our work with Stem Longevity Research and are looking forward to leveraging the knowledge they bring to our company.

Dr. Joseph Purita is one of the pioneers of PRP and stem cell injection research. His passion for stem cell research has helped him create the Institute of Regenerative and Molecular Orthopedics, where the standard is set for orthopedic stem cell treatments. Doctors from all over the world train in the use of PRP and stem cell treatment with Dr. Purita at the Institute.

Eckert specializes in the treatment of diseases and disorders for people of all ages. He is a certified nutritional counselor who is dedicated to bringing nutritional education and quality of life to those suffering from health challenges. Eckert also currently serves as a medical advisor for InHealth Media, a media and marketing firm that works with brands in the nutraceutical and sports nutrition industries.

BioProtein Technology offers a family of natural treatment therapies that combine the effective concepts of traditional medicine with modern technological breakthroughs. The company differentiates itself by dedicating unparalleled time and energy into a single raw material, giving its team complete top-down control of the manufacturing process.

The BioProtein Technology product line includes Velvet Antler Extract, which promotes anti-aging and naturally supports healthy cell regeneration and repair. This product balances hormones, modulates endocrine and immune systems and improves memory and cognition, among many other benefits. Other products include natural hormone and immune modulator Sub-L Tropin, topical anti-inflammatory solution Derma-T Tropin and Derma-T Tropin, an anti-aging skin care solution.

Its an honor to join the team of BioProtein Technology, a company that is doing groundbreaking work in creating top-quality alternative treatment products, said Eckert. Our goal is to significantly increase physicians awareness of this brand and the numerous benefits these products offer to individuals. Were very pleased to take part in what BioProtein Technology is doing for both doctors and their patients.

Stem Longevity Researchs work with BioProtein Technology will begin effective immediately. For more information, visit http://www.bioproteintech.com.

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Stem Longevity Research Links with BioProtein Technology - Renowned Partnership to Elevate Brand’s Innovative ...

Spinal Cord Injury with Residual Quadriparesis Improved After Stem Cell Treatment – Video

09-10-2012 05:40 I met with an accident in 1997.I suffered a spinal cord injury in that accident.3 months go I took stem cell therapy at these 3 months I feel I have improved a lot more than before. My legs feel loose. My back also feels free. While walking my legs used to crossover each other, that has now reduced. All the muscles of my legs were very tight before now it has reduced.Climbing up and down the stairs is also easier than earlier. I can also move my toes know. Earlier while walking my legs used to cross over each other, now that has reduced and I can walk much better. From lying down I can get up much easily and I can do it on my own now.Earlier I used to do bed rolling as an exercise which is much easier now.I get up from bed very easily now, back seems less tight and I can do the movements freely.I can roll, turn and crawl in the bed freely, than before.I can now shift forward and backward in the bed easily and on my own. Earlier while standing and walking the heel of my foot used to lift off the ground, now it touches the ground. I can balance myself on both legs now. I feel I have a good grip while walking , bearing more weight on both the legs. While climbing stairs I can lift my leg up on my own and climb. I can now stand on my own without any support. Earlier both these muscles were very tight Now they have become loose helping me Walk better Earlier I could not do this exercise at all But now I am able to lift my back easily Improvement Adductor spasticity ...

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Spinal Cord Injury with Residual Quadriparesis Improved After Stem Cell Treatment - Video