Category Archives: Stem Cell Research

A research report published earlier this week suggests a possible connection between the use of stem cells and a treatment for Huntingtons disease.

Su-Chun Zhang, a UW-Madison professor, is the senior author of the new study, which shows embryonic stem cells may be used to reinstate the neural circuit needed for motor control that is destroyed in Huntingtons patients.

There is no cure for Huntingtons, a terminal disease in which patients lose control of muscle coordination.

The key to the study is the use of GABA neurons, which are responsible for creating the network the brain uses to coordinate motor function, but are reduced in Huntingtons patients. According to the report, researchers found a way to manufacture large quantities of GABA neurons from embryonic stem cells, which they implanted in mice to test how well they would integrate in to the mouses brain.

The cells not only integrated in the mouses brain, but also projected to the corresponding target and reestablished the neural circuit, which restored muscle coordination.

This result suggests a possible treatment for a previously incurable disease, as well as discovering that the human brain may be more resilient than originally thought.

While the findings are optimistic, researchers said in a statement that the technology will not be ready for human trials until further research is completed.

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Stem-cell breakthrough could mean treatment for Huntington’s

Qatar Foundation for Education, Science and Community Development (QF) has signed a research collaboration agreement with Hamad Medical Corporation (HMC), driven by a shared desire for further advancement in stem cell research, with the ultimate goal of preventing cancer from evolving to a life- threatening stage. The world loses millions of people to cancer, which is why Qatar Foundation strives to combat this disease, Faisal Mohammed al-Suwaidi, president, research and development, QF, said, adding: The use of stem cells in researching new treatments has begun to prove its effectiveness, so we are undertaking more studies in this field. This will undoubtedly contribute to building a better future for mankind. The agreement was signed by al-Suwaidi and Edward Hillhouse, HMCs senior policy adviser for academic health systems and acting medical director. Abdelali Haoudi, QF vice-president of research and deputy director of Qatar Biomedical Research Institute (QBRI), commented: QF, through the QBRI, has established a new research collaboration agreement with HMC, which has the potential to deliver major advances in the field of stem cell therapy applied to cancer. Our joint efforts will not only work to discover treatments designed to destroy cancer cells, but revolutionise the scientific approach to fighting disease in the future. Research will be conducted at Al Amal Hospital in Doha, with efforts concentrated on enhancing its reputation across the globe, particularly for its studies on leukemia. Stem cell research is a growing innovative field and one of the priority research areas for both QF and HMC, said Dr Hanadi Raffi Elayoubi, head of the Bone Marrow Transplant Unit and chief of staff at Al Amal Hospital. This research programme does not only target leukemia, the top third cancer diagnosis in Qatar, but represents one of the first translational cancer research programmes in HMC. We have the setting and the clinical programme at Al Amal Hospital that will allow the translation to bed site to the patient. This is certainly a very important partnership initiated by QF to develop translational research that might impact patients care, he added. Scientists from both HMC and QBRI will work under the guidance of Dr Salem Chouaib, research director at the French National Institute for Health and Biomedical Research, and head of the cancer immunology division at Paris-based Institute Gustave Roussy. Labelled the European leader in cancer research and treatment, the institute will also share its expertise as part of this agreement. Through his work, Dr Chouaib seeks to transform theoretical concepts of cancer research into clinical application. As part of this agreement, he will work to foster a strong relationship between QF and other French scientists specialising in this area of study. QFs newly signed agreement with HMC will support QF on its mission to build human capacity in Qatar by ensuring that scientists have the necessary resources and means to explore alternative treatments for curing cancer. A proven cure for cancer continues to elude medical science. While chemotherapy and radiation treatment can kill malignant tumors, they do not cure every patient and, despite the billions of dollars invested globally on research, cancer remains one of the worlds major causes of death.

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QF signs research deal with HMC

By Matthew Dublin

Cycle Computing has named Victor Ruotti, a computational biologist at the Morgridge Institute for Research, as the 2011 CycleCloud BigScience Challenge.

Finalists' proposals were selected based on their benefit to humanity, originality, creativity and suitability. Entrants submitted projects that focus Parkinsons disease, diabetes, organic photovoltaics, genomic diversity mapping. The finalists were judged by Jason Stowe, CEO, Cycle Computing, as well as Matt Wood, technology evangelist for Amazon Web Services, and Peter Shenkin, vice president at Schrdinger.

Ruotti will be awarded $10,000 of time the equivalent of eight hours on a 30,000-core cluster on their cloud. In his submission for the contest, Ruotti proposed a knowledgebase indexing system for Human Embryonic Stem Cells and their derivatives, which usually requires hours of computational times.

The high throughput computing power of CycleCloud will enable the classification of currently uncharacterized cell types, including hES cells and iPS cells from our laboratory, says Ruotti. The transcript profiles from each cell type will be analyzed and compared by aligning billions of sequencing reads in combinatorial pair wise steps. By doing so, we will create the first read level index to yield classified cellular derivatives along with methods to produce these cell types in a laboratory setting which could become potential therapies of the future.

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Stem Cell Project Wins Cloud Computing Competition

NEWARK, Calif., March 13, 2012 (GLOBE NEWSWIRE) -- StemCells, Inc. (Nasdaq:STEM - News), a leading stem cell company developing and commercializing novel cell-based therapeutics and tools for use in stem cell-based research and drug discovery, today reported financial results for the fourth quarter and year ended December 31, 2011 and provided a business update.

"The StemCells team made significant progress in 2011 with regard to two critical goals for the Company, namely accelerating and broadening our HuCNS-SC neural stem cell clinical trial agenda for diseases of, and injuries to, the central nervous system, while at the same time reducing our operating cash burn. We are now uniquely positioned as the only stem cell company pursuing clinical trials for disorders of all three organs of the CNS, the brain, spinal cord and eye," said Martin McGlynn, President and CEO of StemCells, Inc. "We have strong preclinical data underlying all our clinical trials, much of which has already been published in peer-reviewed journals, but we realize that the true test of our proprietary cell-based technology will be in the clinic. In this regard, I am pleased to confirm StemCells remains on track to report safety and efficacy data from our recently completed Phase I Pelizeaus-Merzbacher disease trial at the European Leukodystrophy Association meeting to be held in Paris, March 31-April 1. We are confident that executing our clinical trial agenda, while controlling our cash burn, is the best way to build lasting shareholder value."

Fourth Quarter and Recent Business Highlights

Therapeutic Product Development

Tools and Technologies Programs

Other Business Activities

Fourth Quarter 2011 Financial Results

For the fourth quarter of 2011, the Company reported a net loss of $7,212,000, or $(0.47) per share, compared with a net loss of $8,957,000, or $(0.70) per share, for the fourth quarter of 2010. Loss from operations in the fourth quarter of 2011 was $7,313,000, which was 5% lower when compared to the $7,706,000 loss from operations in the fourth quarter of 2010. Included in net loss and loss from operations in the fourth quarter of 2011 is a charge of $655,000 for the write-off of an acquired intangible asset.

Total revenue during the fourth quarter of 2011 was $541,000, compared to $699,000 in the same period of 2010. The decrease of 23% from 2010 to 2011 was due to both lower product sales and lower licensing and grant revenues. Total revenues in the fourth quarter of 2010 were higher due to a particularly strong quarter in our SC Proven business as well as the receipt of a milestone payment under a licensing agreement of approximately $438,000 in 2010.

Total operating expenses in the fourth quarter of 2011 were $7,807,000, compared to $8,341,000 in the fourth quarter of 2010. Excluding the impairment of the intangible asset, which is included as an operating expense, total operating expenses in the fourth quarter of 2011 were $7,152,000, or 14% lower than the same period in 2010. In the fourth quarter of 2011, research and development expenses totaled $4,834,000, or 18% less than in the same period of 2010, while selling, general and administrative expenses totaled $2,290,000, or 8% lower. The significant reduction in operating expenses was primarily attributable to the Company's cost containment efforts, including the reduction in force effected in May 2011.

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StemCells, Inc. Reports Fourth Quarter and Year End 2011 Financial Results and Provides Business Update

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International Stem Cell Corporation Completes $5 Million Financing and Elects Jim Berglund to the Board of Directors

LOS ANGELES Researchers at the UCLA stem cell center and the departments of chemistry and biochemistry and pathology and laboratory medicine have identified, for the first time, a generic way to correct mutations in human mitochondrial DNA by targeting corrective RNAs, a finding with implications for treating a host of mitochondrial diseases.

Mutations in the human mitochondrial genome are implicated in neuromuscular diseases, metabolic defects and aging. There currently are no methods to successfully repair or compensate for these mutations, said study co-senior author Dr. Michael Teitell, a professor of pathology and laboratory medicine and a researcher with the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA.

Between 1,000 and 4,000 children per year in the United States are born with a mitochondrial disease and up to one in 4,000 children in the U.S. will develop a mitochondrial disease by the age of 10, according to Mito Action, a nonprofit organization supporting research into mitochondrial diseases. In adults, many diseases of aging have been associated with defects of mitochondrial function, including diabetes, Parkinson's disease, heart disease, stroke, Alzheimer's disease and cancer.

"I think this is a finding that could change the field," Teitell said. "We've been looking to do this for a long time and we had a very reasoned approach, but some key steps were missing. Now we have developed this method and the next step is to show that what we can do in human cell lines with mutant mitochondria can translate into animal models and, ultimately, into humans."

The study appears today in the peer-reviewed journal Proceedings of the National Academy of Sciences.

The current study builds on previous work published in 2010 in the peer-reviewed journal Cell, in which Teitell, Carla Koehler, a professor of chemistry and biochemistry and a Broad stem cell research center scientist, and their team uncovered a role for an essential protein that acts to shuttle RNA into the mitochondria, the energy-producing "power plant" of a cell.

Mitochondria are described as cellular power plants because they generate most of the energy supply within a cell. In addition to supplying energy, mitochondria also are involved in a broad range of other cellular processes including signaling, differentiation, death, control of the cell cycle and growth.

The import of nucleus-encoded small RNAs into mitochondria is essential for the replication, transcription and translation of the mitochondrial genome, but the mechanisms that deliver RNA into mitochondria have remained poorly understood.

The study in Cell outlined a new role for a protein called polynucleotide phosphorylase (PNPASE) in regulating the import of RNA into mitochondria. Reducing the expression or output of PNPASE decreased RNA import, which impaired the processing of mitochondrial genome-encoded RNAs. Reduced RNA processing inhibited the translation of proteins required to maintain the mitochondrial electron transport chain that consumes oxygen during cell respiration to produce energy. With reduced PNPASE, unprocessed mitochondrial-encoded RNAs accumulated, protein translation was inhibited and energy production was compromised, leading to stalled cell growth.

The findings from the current study provide a form of gene therapy for mitochondria by compensating for mutations that cause a wide range of diseases, said study co-senior author Koehler.

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Repairing mutations in human mitochondria

LOS ANGELES UCLA stem cell researchers have shown that insulin and nutrition prevent blood stem cells from differentiating into mature blood cells in Drosophila, the common fruit fly, a finding that has implications for studying inflammatory response and blood development in response to dietary changes in humans.

Keeping blood stem cells, or progenitor cells, from differentiating into blood cells is important as blood stem cells are needed to create the blood supply for the adult fruit fly.

The study found that the blood stem cells are receiving systemic signals from insulin and nutritional factors, in this case essential amino acids, that helped them to maintain their "stemness," said study senior author Utpal Banerjee, the Irving and Jean Stone Professor and chairman of molecular, cell and developmental biology in the UCLA Division of Life Sciences and a researcher with the Eli and Edythe Broad Center of Regenerative Medicine at UCLA.

"We expect that this study will promote further investigation of possible direct signal sensing mechanisms by mammalian blood stem cells," Banerjee said. "Such studies will probably yield insights into chronic inflammation and the myeloid cell accumulation seen in patients with type II diabetes and other metabolic disorders."

The study appeared Sunday (March 11) in the peer-reviewed journal Nature Cell Biology.

In the flies, the insulin signaling came from the brain, which is an organ similar to the human pancreas, which produces insulin. That insulin was taken up by the blood stem cells, as were amino acids found in the fly blood, said Ji Won Shim, a postdoctoral fellow in Banerjee's lab and first author of the study.

Shim studied the flies while in the larval stage of development. To see what would happen to the blood stem cells, Shim placed the larvae into a jar with no food they usually eat yeast or cornmeal and left them for 24 hours. Afterward, she checked for the presence of blood stem cells using specific chemical markers that made them visible under a confocal microscope.

"Once the flies were starved and not receiving the insulin and nutritional signaling, all the blood stem cells were gone," Shim said. "All that were left were differentiated mature blood cells. This type of mechanism has not been identified in mammals or humans, and it will be intriguing to see if there are similar mechanisms at work there."

In the fruit fly, the only mature blood cells present are myeloid cells, Shim said. Diabetic patients have many activated myeloid cells that could be causing disease symptoms. It may be that abnormal activation of myeloid cells and abnormal metabolism play a major role in diabetes.

"Metabolic regulation and immune response are highly integrated in order to function properly dependent on each other. Type II diabetes and obesity, both metabolic diseases, are closely associated with chronic inflammation, which is induced by abnormal activation of blood cells," Shim said. "However, no systemic study on a connection between blood stem cells and metabolic alterations had been done. Our study highlights the potential linkage between myeloid-lineage blood stem cells and metabolic disruptions."

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Insulin, nutrition prevent blood stem cell differentiation

NEW YORK & PETACH TIKVAH, ISRAEL--(BUSINESS WIRE)--

BrainStorm Cell Therapeutics Inc. (OTCBB: BCLI.OB - News), a developer of adult stem cell technologies and CNS therapeutics, announces plans to initiate a preclinical study assessing the efficacy of its NurOwn stem cell technology in patients with Multiple Sclerosis (MS). Positive proof-of-concept results for MS have been confirmed in a set of in-vitro and in-vivo experiments, and the Company is working to advance MS into preclinical development in Q2 2012.

Based on initial promising pre-clinical data published by the Company's Chief Scientist, Prof. Daniel Offen of Tel Aviv University, BrainStorm has decided to explore MS as an additional indication for its NurOwn technology. The Company will draw plans to initiate pre-clinical safety trials, after which it will seek a leading medical center specializing in MS for clinical trials.

We have been focused on growing our pipeline of indications using our NurOwn stem-cell technology, commented Dr. Adrian Harel, Acting CEO of BrainStorm Cell Therapeutics. As we continue our ongoing trials to evaluate the safety, tolerability and therapeutic effects of NurOwn in ALS patients, we have determined through positive preliminary animal data that MS will be the next indication to pursue using our technology.

About NurOwn BrainStorms core technology, NurOwn, is based on the scientific achievements of Professor Eldad Melamed, former Head of Neurology, Rabin Medical Center, and Tel-Aviv University, and Professor Daniel Offen, Head of the Neuroscience Laboratory, Felsenstein Medical Research Center at the Tel-Aviv University.

The NurOwn technology processes adult human mesenchymal stem cells that are present in bone marrow and are capable of self-renewal as well as differentiation into many cell types. The research team is among the first to have successfully achieved the in-vitro differentiation of adult bone marrow cells (animal and human) into cells capable of releasing neurotrophic factors, such as glial-derived neurotrophic factor (GDNF), by means of a specific differentiation-inducing culture medium.

About Multiple Sclerosis (MS) Multiple sclerosis (MS) is believed to be an autoimmune disorder that affects the central nervous system (CNS). Autoimmune means that the bodys immune system mistakenly attacks its own tissue, in this case, the tissues of the CNS. With MS, autoimmune damage to neurons disrupts the bodys ability to send and receive signals, thus causing MS-related symptoms. Symptoms may vary due to the location and extent of the damage. Worldwide, MS may affect more than 2 million individuals, including approximately 400,000 people in the United States.

About BrainStorm Cell Therapeutics Inc. BrainStorm Cell Therapeutics Inc. is a biotechnology company engaged in the development of adult stem cell therapeutic products derived from autologous bone marrow cells and intended for the treatment of neurodegenerative diseases. The Company holds the rights to develop and commercialize its NurOwn technology through an exclusive, worldwide licensing agreement with Ramot, the technology transfer company of Tel-Aviv University. For more information, visit the companys website at http://www.brainstorm-cell.com.

Safe Harbor Statement Statements in this announcement other than historical data and information constitute "forward-looking statements" and involve risks and uncertainties that could cause BrainStorm Cell Therapeutics Inc.'s actual results to differ materially from those stated or implied by such forward-looking statements. The potential risks and uncertainties include risks associated with BrainStorm's limited operating history, history of losses; minimal working capital, dependence on its license to Ramot's technology; ability to adequately protect the technology; dependence on key executives and on its scientific consultants; ability to obtain required regulatory approvals; and other factors detailed in BrainStorm's annual report on Form 10-K and quarterly reports on Form 10-Q available at http://www.sec.gov. The Company does not undertake any obligation to update forward-looking statements made by us.

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BrainStorm Cell Therapeutics Expands Pipeline with the Initiation of a Study for Multiple Sclerosis

CLEARWATER, FL--(Marketwire -03/12/12)- Biostem U.S., Corporation (OTCQB: BOSM.PK - News) (Pinksheets: BOSM.PK - News) (Biostem, the Company), a fully reporting public company in the stem cell regenerative medicine sciences sector, announced today the addition of cardiothoracic surgeon Thomas W. Prendergast, M.D. to its Scientific and Medical Board of Advisors (SAMBA).

Biostem CEO, Dwight Brunoehler stated, "The Company is now positioned for growth and international expansion. Adding a world class team of clinical, laboratory, and regulatory experts for our Scientific and Medical Board of Advisors to guide our pursuits is essential. Dr. Prendergast brings a wealth of experience not only in the scientific aspects of stem cell use in regenerative medicine, but also in forging research and international economic development opportunities."

Dr. Prendergast is a busy clinical cardiothoracic surgeon, who performs 200-250 open-heart operations and 5 to 15 heart transplants each year. He is deeply involved in numerous clinical and research activities associated with stem cells and heart repair. He is presently Director of Cardiac Transplantation at Robert Wood Johnson University Hospital in New Brunswick, New Jersey where he holds an Associate Professorship of Surgery at the University of Medicine and Dentistry of New Jersey. In addition to being an active participant in stem cell research program development and teaching medical students and residents, his other interests include medical research funding and humanitarian development of programs for Disabled American Veterans.

Dr. Prendergast received his undergraduate degrees in biophysics and Psychology, as well as his medical degree, at Pennsylvania State University. His general surgery residency was for five years at the University of Massachusetts Medical School. His cardiothoracic surgery training was at the University of Southern California School of Medicine, including the Los Angeles County Medical Center. Subsequent fellowship training included pediatric cardiac surgery at Children's Hospital of LA, along with thoracic transplant fellowships at University of Southern California in Los Angeles and at Temple University Hospital in Philadelphia. He spent three years at the University of Kansas establishing thoracic transplant programs until returning to Temple University Hospital as one of their staff heart and lung transplant surgeons. Subsequent to his time at Temple, he joined up with Newark Beth Israel/St. Barnabas Hospitals, where he assumed directorship as the Chief of Cardiac Transplantation and Mechanical Assistance.

Regarding his appointment to the Biostem U.S. Scientific and Medical Board of Advisors, Dr. Prendergast said, "I am looking forward with excitement to working again with Dwight at Biostem. The expansion plan is sound, well paced, and will afford improved quality of life opportunities to many people around the world."

About Biostem U.S., Corporation

Biostem U.S., Corporation (OTCQB: BOSM.PK - News) (Pinksheets: BOSM.PK - News) is a fully reporting Nevada corporation with offices in Clearwater, Florida. Biostem is a technology licensing company with proprietary technology centered around providing hair re-growth using human stem cells. The company also intends to train and license selected physicians to provide Regenerative Cellular Therapy treatments to assist the body's natural approach to healing tendons, ligaments, joints and muscle injuries by using the patient's own stem cells. Biostem U.S. is seeking to expand its operations worldwide through licensing of its proprietary technology and acquisition of existing stem cell related facilities. The company's goal is to operate in the international biotech market, focusing on the rapidly growing regenerative medicine field, using ethically sourced adult stem cells to improve the quality and longevity of life for all mankind.

More information on Biostem U.S., Corporation can be obtained through http://www.biostemus.com, or by calling Kerry D'Amato, Marketing Director at 727-446-5000.

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Biostem U.S., Corporation Appoints Heart Surgeon, Thomas W. Prendergast, M.D. to Its Scientific and Medical Board of ...

Public release date: 12-Mar-2012 [ | E-mail | Share ]

Contact: Kim Irwin kirwin@mednet.ucla.edu 310-206-2805 University of California - Los Angeles Health Sciences

Researchers at the UCLA stem cell center and the departments of chemistry and biochemistry and pathology and laboratory medicine have identified, for the first time, a generic way to correct mutations in human mitochondrial DNA by targeting corrective RNAs, a finding with implications for treating a host of mitochondrial diseases.

Mutations in the human mitochondrial genome are implicated in neuromuscular diseases, metabolic defects and aging. There currently are no methods to successfully repair or compensate for these mutations, said study co-senior author Dr. Michael Teitell, a professor of pathology and laboratory medicine and a researcher with the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA.

Between 1,000 and 4,000 children per year in the United States are born with a mitochondrial disease and up to one in 4,000 children in the U.S. will develop a mitochondrial disease by the age of 10, according to Mito Action, a nonprofit organization supporting research into mitochondrial diseases. In adults, many diseases of aging have been associated with defects of mitochondrial function, including diabetes, Parkinson's disease, heart disease, stroke, Alzheimer's disease and cancer.

"I think this is a finding that could change the field," Teitell said. "We've been looking to do this for a long time and we had a very reasoned approach, but some key steps were missing. Now we have developed this method and the next step is to show that what we can do in human cell lines with mutant mitochondria can translate into animal models and, ultimately, into humans."

The study appears March 12, 2012 in the peer-reviewed journal Proceedings of the National Academy of Sciences.

The current study builds on previous work published in 2010 in the peer-reviewed journal Cell, in which Teitell, Carla Koehler, a professor of chemistry and biochemistry and a Broad Stem Cell Research Center scientist, and their team uncovered a role for an essential protein that acts to shuttle RNA into the mitochondria, the energy-producing "power plant" of a cell.

Mitochondria are described as cellular power plants because they generate most of the energy supply within a cell. In addition to supplying energy, mitochondria also are involved in a broad range of other cellular processes including signaling, differentiation, death, control of the cell cycle and growth.

The import of nucleus-encoded small RNAs into mitochondria is essential for the replication, transcription and translation of the mitochondrial genome, but the mechanisms that deliver RNA into mitochondria have remained poorly understood.

Link:
Correcting human mitochondrial mutations