Category Archives: Stem Cell Human Trials

Public release date: 28-Jun-2012 [ | E-mail | Share ] Contact: Diane Schrick diane.schrick@gladstone.ucsf.edu 415-734-2538 Gladstone Institutes SAN FRANCISCO, CAJune 28, 2012Scientists at the Gladstone Institutes and an international team of researchers have generated a human model of Huntington's diseasedirectly from the skin cells of patients with the disease. For years, scientists have studied Huntington's disease primarily in post-mortem brain tissue or laboratory animals modified to mimic the disease. Today, in Cell Stem Cell, the international team shows how they developed a human model of Huntington's disease, which causes a diverse range of neurological impairments. The new model should help scientists better understand the development of Huntington'sand provide better ways to identify and screen potential therapeutics for this devastating disease. This new model comes at a time of concentrated federal efforts to accelerate solutions for diseasesincluding a number of debilitating conditions that touch only small percentages of the population. Last year, the National Institutes of Health consolidated its efforts to attack rare diseases under the new National Center for Translational Sciences. Huntington's is such a rare disease, although it is the most common inherited neurodegenerative disorder. It afflicts approximately 30,000 people in the United Stateswith another 75,000 people carrying the … Continue reading →

Washington: Scientists in Japan claim to have developed a rudimentary human liver and a precursor of a human eyeball in the lab using stem cells, a feat they say could be a boon for the future organ replacement. At Yokohama City University in Japan, a team led by stem cell biologist Takanori Takebe grew a small, rudimentary liver using a recipe of just three types of cells. The trick was figuring out when to introduce each element into the mix of cells: "It took over a year and hundreds of trials," LiveScience quoted Takebe as telling journal Nature. First, the team placed genetically reprogrammed human skin cells, called "induced pluripotent stem cells", on growth plates in a specially designed chemical bath. After nine days, the cells began developing into hepatocytes, or liver cells. The scientists believe this could be a boon for the future organ transplant. At that point, the researchers added cells taken from an umbilical cord, which would develop into the lining of blood vessels, and cells from bone marrow that can differentiate into bone, cartilage or fat. Two days later, the cell assortment had self-organised to form a three-dimensional "liver bud" -- a 5mm-wide chunk of tissue … Continue reading →

NEWARK, Calif., June 21, 2012 (GLOBE NEWSWIRE) -- StemCells, Inc. (STEM) today announced initiation of a Phase I/II clinical trial of the Company's proprietary HuCNS-SC(R) product candidate (purified human neural stem cells) in dry age-related macular degeneration (AMD) referred to as Geographic Atrophy. There are no approved treatments for dry AMD. The trial is being conducted at the Retina Foundation of the Southwest's (RFSW) Anderson Vision Research Center in Dallas, Texas, one of the leading independent vision research centers in the United States. David G. Birch, Ph.D., Chief Scientific and Executive Officer of the RFSW and Director of the Rose-Silverthorne Retinal Degenerations Laboratory, is the principal investigator of the study. "Dry AMD is the most common form of macular degeneration, and has a very debilitating effect on quality of life," said Dr. Birch. "Transplanting neural stem cells to protect photoreceptors in patients diagnosed with AMD is an innovative, but logical, approach, well supported by the Company's recently published preclinical data. We are very excited to be conducting this trial at RFSW." A summary of the Company's preclinical data was featured in the February 2012 issue of the international peer-reviewed European Journal of Neuroscience (available online at http://onlinelibrary.wiley.com/doi/10.1111/j.1460-9568.2011.07970.x/abstract). The data demonstrated … Continue reading →

Japanese scientists have used induced stem cells to create a liver-like tissue in a dish. Although they have yet to publish their results and much work remains to be done, the achievement could have big clinical implications. If the results bear out, they would also constitute a significant advance in the ability to coax stem cells to self-organize into organs. Induced pluripotent stem cells could be a useful source of human organs such as livers. STEVE GSCHMEISSNER/SCIENCE PHOTO LIBRARY The work was presented by Takanori Takebe, a stem-cell biologist at Yokohama City University in Japan, at the annual meeting of the International Society for Stem Cell Research in Yokohama last week. It blew my mind, said George Daley, director of the stem-cell transplantation programme at the Boston Childrens Hospital in Massachusetts, who chaired the session. It sounds like a genuine advance, says Stuart Forbes, who studies liver regeneration at the University of Edinburgh, UK. Forbes, who also works as a consultant for Scotlands liver-transplantation unit, says that the advance could one day help to avoid the bleak outcome currently experienced by the many patients who dont survive long enough to get a new liver. But the liver described by Takebe … Continue reading →

SEATTLE, June 20, 2012 /PRNewswire/ -- Cell Therapeutics, Inc. ("CTI") (NASDAQ and MTA: CTIC), a company focused on translating science into novel cancer therapies, today announced that it is re-aligning its resources and re-prioritizing its product development pipeline to focus on the launch of Pixuvri in the EU and accelerate the advancement of pacritinib, CTI's recently-acquired, highly-selective JAK 2 inhibitor, into phase III clinical trials. As a result, CTI's operating burn rate will be reduced from an average of $6.5 million per month to an average of $4.5 million per month. "In-market research across the five largest EU markets confirms significant physician interest in product adoption in the target patient population. With the potential to generate meaningful Pixuvri product sales, coupled with the recent acquisition of pacritinib and investigator interest in participating in its pivotal studies, we believe that focusing our resources on the Pixuvri launch and accelerating the start of pacritinib phase III studies is the best deployment of our limited resources to build near-term shareholder value," said James A. Bianco, M.D., Chief Executive Officer of CTI. "We are re-evaluating the tostedostat phase III clinical trial design and are considering both studies in a refractory setting and in front … Continue reading →

Image: NASA, Johnson Space Center From Nature magazine Japanese scientists have used induced stem cells to create a liver-like tissue in a dish. Although they have yet to publish their results and much work remains to be done, the achievement could have big clinical implications. If the results bear out, they would also constitute a significant advance in the ability to coax stem cells to self-organize into organs. The work was presented by Takanori Takebe, a stem-cell biologist at Yokohama City University in Japan, at the annual meeting of the International Society for Stem Cell Research in Yokohama last week. It blew my mind, said George Daley, director of the stem-cell transplantation programme at the Boston Childrens Hospital in Massachusetts, who chaired the session. It sounds like a genuine advance, says Stuart Forbes, who studies liver regeneration at the University of Edinburgh, UK. Forbes, who also works as a consultant for Scotlands liver-transplantation unit, says that the advance could one day help to avoid the bleak outcome currently experienced by the many patients who dont survive long enough to get a new liver. But the liver described by Takebe has a long way to go before that. Takebe told how … Continue reading →

By Jason Napodano, CFA Neuralstem, Inc. (NYSE MKT: CUR ) has developed a technology that allows large-scale expansion of human neural stem cells ("hNSC") from all areas of the developing human brain and spinal cord. The company owns of has exclusive license to 25 patients and 29 patent applications pending worldwide in the field of regenerative medicine and cell therapy. Management is currently focusing the company's efforts on replacing damaged, malfunctioning, or dead neural cells with fully functional ones that may be useful in treating many central nervous system diseases and neurodegenerative disorders. Neuralstem's lead development program is for Amyotrophic Lateral Sclerosis ("ALS"), also known as Lou Gehrig 's disease, named after the famous New York Yankee first baseman who was diagnosed with the disease in 1939, and passed in 1941 at the age of only 37. ALS Background ALS is a rapidly progressive neurodegenerative disease characterized by weakness, muscle atrophy and twitching, spasticity, dysarthria (difficulty speaking), dysphagia (difficulty swallowing), and respiratory compromise. The disease is almost always fatal, typically due to respiratory compromise or pneumonia, in two to four years. Initial symptoms of ALS include weakness and/or stiffness followed by muscle atrophy in the arms and legs. This is … Continue reading →

As of now, management is planning to conduct the pivotal program on its own, mostly likely seeking funding through grants with the ALS Association and U.S. National Institutes of Health. However, management is also in discussion with potential pharmaceutical partners on the pivotal program. ALS is a highly attractive area for Big Pharma. Depending on the strength of the phase 1 / 2 data, Neuralstem may be able to strike a commercialization partnership in 2014 to help defer the costs of the planned pivotal trial. We expect that any deal with a larger pharmaceutical company would include a substantial upfront payment that Neuralstem would then use to fund expansion of the development platform into new indications, such as spinal cord injury (IND filed) or stroke. Market Opportunity In February 2011, the U.S. FDA granted Neuralstem an Orphan Drug designation for its human spinal cord stem cells (HSSC) for the treatment of ALS. As noted above, there are approximately 30,000 patients in the U.S. living with ALS. We estimate that approximately half of these patients are characterized with an FVC > 60% and may be eligible for treatment with Neuralstems hNSCs. Given the Orphan Drug designation, the limited patient population, and … Continue reading →

SAN CARLOS, Calif.--(BUSINESS WIRE)-- Cellerant Therapeutics Inc., a biotechnology company developing novel hematopoietic stem cell-based cellular and antibody therapies for blood disorders and cancer, announced today that Cellerants President and CEO, Ram Mandalam, Ph.D., will be presenting at the International Society for Stem Cell Research (ISSCR) 10th Annual Meeting held June 13 16 in Yokohama, Japan. Dr. Mandalam will be co-chairing a session Stem Cells and Cancer and will be giving a presentation, titled Antibodies Targeting Cancer Stem Cells in Hematological Malignancies, on Saturday, June 16 at 1:30 p.m. JST (Japan time). Dr. Mandalam will provide an overview of Cellerants cancer stem cell discovery approach and new data on its therapeutic antibodies demonstrating activity against acute myelogenous leukemia (AML) cancer stem cells. Cellerant scientist, Anna Sedello, Ph.D. will also be presenting Poster #1144 titled Ex vivo-Generated Mouse Myeloid Progenitor Cells Mitigate Gastrointestinal Acute Radiation Syndrome when Administered up to 5 Days after Irradiation. These results demonstrate that cryopreserved, allogeneic myeloid progenitor cells from mice prevent death from lethal radiation doses known to cause death through hematopoietic and gastrointestinal injury. These results also show that myeloid progenitor cells are one of the most promising radiation countermeasures among all therapeutics currently under … Continue reading →

SAN CARLOS, Calif.--(BUSINESS WIRE)-- Cellerant Therapeutics Inc., a biotechnology company developing novel hematopoietic stem cell-based cellular and antibody therapies for blood disorders and cancer, announced today the appointment of Gisela Schwab, M.D. to its Board of Directors and the appointment of Lowell Sears as Chairman of the Board. Richard Rathmann, Cellerants former Chairman of the Board, will remain a director on the Board. Dr. Schwab joins Cellerants Board with more than 20 years of experience in the development of oncology therapeutics. She currently serves as Executive Vice President and Chief Medical Officer of Exelixis. Previously, she held the position of Senior Vice President and Chief Medical Officer at Abgenix, Inc., a human antibody-based drug development company. Prior to Abgenix, Dr. Schwab held positions of increasing responsibility at Amgen Inc., most recently as Director of Clinical Research and Hematology/Oncology Therapeutic Area Team Leader. Dr. Schwab also serves as a member of the board of directors of Topotarget A/S, a publicly-held biopharmaceutical company. She received her Doctor of Medicine degree from the University of Heidelberg, trained at the University of Erlangen-Nuremberg and the National Cancer Institute and is board certified in internal medicine and hematology and oncology. Dr. Schwab is an accomplished … Continue reading →