Public release date: 3-Oct-2012 [ | E-mail | Share ]

Contact: Marla Paul Marla-Paul@northwestern.edu 312-503-8928 Northwestern University

Researchers at Northwestern University Feinberg School of Medicine have developed the first animal model that duplicates the human response in rheumatoid arthritis (RA), an important step that may enable scientists to discover better medicines to treat the disease.

Corresponding and senior author Harris Perlman, associate professor of rheumatology at Feinberg, introduced his team's new prototype mouse model in a recent online issue of the Journal of Translational Medicine.

"This is the first time human stem cells have been transplanted into mice in order to find RA treatments," Perlman said. "We believe this will improve drug discovery because the reactions we observed were authentic human reactions."

Until now, scientists have relied on the common scientific method of using specially bred mice to find drugs to control RA. However, human and mouse immune systems differ dramatically, so studying RA in these mice does not give an accurate representation of how the disease functions in humans. In some cases, RA drugs that seemed promising based on results in mice failed in human clinical trials.

Mice implanted with human stem cells have been used before mainly to study infectious disease.

The Northwestern team injected day-old mice with human stem cells from umbilical cord blood, including white blood cells, which regulate immunity. Then, RA was introduced in the mice and suppressed with Enbrel, a common first-line drug for joint inflammation in humans. This offered evidence that their immune systems were indeed replicating human defenses.

Scientists seek mouse models that mimic RA in humans in order to learn how the complex disease operates. In the last decade, researchers and physicians have found many subtypes of RA that originate on the molecular level and are each produced by different pathways in the body.

A debilitating disease, rheumatoid arthritis is a chronic autoimmune disorder characterized by persistent inflammation around joint areas, predominantly in the wrist and fingers. The disease causes pain, swelling, stiffness and loss of function and can result in tissue destruction. Approximately 1.3 million people have the disease.

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'Humanized' mice advance study of rheumatoid arthritis

Pluristem Therapeutics Ltd. (Nasdaq:PSTI; DAX: PJT: PLTR) today announced that it has developed a portable instrument for thawing its placenta-based stems cell (PLX cells) on-site before they are administered to patients.

Pluristem will ship its off-the-shelf PLX cell therapy product candidates, which will be stored in multiple dose vials that require thawing prior to use. The vial will be placed into the proprietary thawing device and PLX cells will be ready for a convenient intramuscular injection. The company said that if the trials of the instrument are successful, it will be used as the final step in bringing high quality, clinical grade PLX cell products to patients around the globe.

"Pluristem understands the importance of providing a standardized product with every dose of these living drug delivery devices," said Pluristem chairman and CEO Zami Aberman. "If we are successful, we want our PLX cell products, once developed, to be an easy-to-use therapy. This thawing device will give us better control of several variables in our clinical trials and in treating patients after our products have been approved assuming we are successful. This instrument is an additional step in our vision to bring PLX cells as first-line therapies for a variety of indications and to think about the cell delivery process all the way from mass manufacturing to the patient's bedside."

Pluristem's share price rose 4.3% on the TASE today to NIS 16.05, after the announcement, after rising 4.4% on Nasdaq yesterday to $4.07, giving a market cap of $193 million.

Published by Globes [online], Israel business news - http://www.globes-online.com - on October 4, 2012

Copyright of Globes Publisher Itonut (1983) Ltd. 2012

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Pluristem unveils portable stem cells thawing device

REDWOOD CITY, Calif.--(BUSINESS WIRE)--

OncoMed Pharmaceuticals, Inc., a clinical stage, research and discovery company developing novel therapeutics that target cancer stem cells, today announced clinical progress with two of its Notch pathway product candidates, resulting in $8 million in milestone payments from the companys strategic collaborator GlaxoSmithKline (GSK).

Anti-Notch2/3 (OMP-59R5)

OncoMed has initiated a Phase 1b/2 clinical trial in its anti-Notch2/3 antibody (OMP-59R5) program. In the Phase 1b/2 ALPINE trial (Antibody therapy in first-Line Pancreatic cancer Investigating anti-Notch Efficacy and safety), Anti-Notch2/3 is being tested in combination with gemcitabine in first-line advanced pancreatic cancer patients. Following a Phase 1b safety run-in, a randomized Phase 2 clinical trial will proceed in these patients to compare the efficacy of standard-of-care gemcitabine either with Anti-Notch2/3 or with placebo. The two primary endpoints of the Phase 2 part of the trial will be progression-free survival (PFS) in the Anti-Notch2/3 arm compared to a placebo arm in all patients, as well as in patients who have a particular biomarker. Key secondary and exploratory endpoints include overall survival, response rate, and safety, and these endpoints will be assessed in all patients, as well as in the biomarker positive subset of patients.

Dr. Lon Smith, from the South Texas Accelerated Research Therapeutics (START) Center for Cancer Care, who treated the first patient dosed in the ALPINE study, noted, This is an exciting new clinical trial with a novel anti-cancer treatment that we hope will have a big impact for patients with pancreatic cancer. The fact that the trial also includes a predictive biomarker to potentially identify patients who might gain greater clinical benefit from Anti-Notch2/3 is also a new and exciting direction in the experimental treatment of patients with pancreatic cancer.

Anti-Notch1 (OMP-52M51)

An Investigational New Drug (IND) application filed by OncoMed has been accepted by the FDA, thereby allowing OncoMed to advance its anti-Notch1 antibody (OMP-52M51) to clinical testing. OncoMed plans to initiate a single-agent, dose escalation and expansion Phase 1 clinical trial in hematologic cancers in 2012 and plans to file an additional IND application later in 2012 in solid tumors. The clinical trials will assess safety, pharmacokinetics, pharmacodynamics, and initial evidence of efficacy via a biomarker-based patient selection approach.

The OMP-59R5 Phase 1b/2 clinical program is our first Phase 2 trial and represents a significant advancement in the companys pipeline of anti-cancer stem cell therapies, said Paul Hastings, President and Chief Executive Officer of OncoMed Pharmaceuticals. In addition, the acceptance from the FDA to begin clinical testing for OMP-52M51 represents the fifth product candidate from our R&D platform to be cleared to enter clinical testing. For both programs, we have developed comprehensive biomarker strategies to facilitate efficient development in patients we believe will be most likely to benefit from targeted Notch pathway signaling blockade, added Hastings.

OMP-59R5 and OMP-52M51 are part of OncoMeds collaboration with GlaxoSmithKline. In December 2007, OncoMed and GSK entered into a broad strategic alliance to discover and develop novel product candidates targeting cancer stem cells via Notch pathway signaling modulation. GSK retains an option through the end of certain Phase 2 clinical trials to obtain an exclusive license to OMP-59R5. GSK also retains an option through the end of certain Phase 1 or certain Phase 2 clinical trials to obtain an exclusive license to OMP-52M51.

About Cancer Stem Cells

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OncoMed Pharmaceuticals Advances Two Notch Pathway Product Candidates in Clinical Development

NEWARK, Calif., Oct. 4, 2012 (GLOBE NEWSWIRE) -- StemCells, Inc. (STEM) today announced that the first patient in its Phase I/II clinical trial in dry age-related macular degeneration (AMD) has been enrolled and transplanted. The trial is designed to evaluate the safety and preliminary efficacy of the Company's proprietary HuCNS-SC(R) product candidate (purified human neural stem cells) as a treatment for dry AMD, and the patient was transplanted with the cells yesterday at the Retina Foundation of the Southwest (RFSW) in Dallas, Texas, one of the leading independent vision research centers in the United States. AMD afflicts approximately 30 million people worldwide and is the leading cause of vision loss and blindness in people over 55 years of age.

"This trial signifies an exciting extension of our on-going clinical research with neural stem cells from disorders of the brain and spinal cord to now include the eye," said Stephen Huhn, MD, FACS, FAAP, Vice President and Head of the CNS Program at StemCells, Inc. "Studies in the relevant animal model demonstrate that the Company's neural stem cells preserve vision in animals that would otherwise go blind and support the therapeutic potential of the cells to halt retinal degeneration. Unlike others in the field, we are looking to intervene early in the course of the disease with the goal of preserving visual function before it is lost."

David G. Birch, Ph.D., Chief Scientific and Executive Officer of the RFSW and Director of the Rose-Silverthorne Retinal Degenerations Laboratory and principal investigator of the study, added, "We are excited to be working with StemCells on this ground breaking clinical trial. There currently are no effective treatments for dry AMD, which is the most common form of the disease, and there is a clear need to explore novel therapeutic approaches."

In February 2012, the Company published preclinical data that demonstrated HuCNS-SC cells protect host photoreceptors and preserve vision in the Royal College of Surgeons (RCS) rat, a well-established animal model of retinal disease which has been used extensively to evaluate potential cell therapies. Moreover, the number of cone photoreceptors, which are responsible for central vision, remained constant over an extended period, consistent with the sustained visual acuity and light sensitivity observed in the study. In humans, degeneration of the cone photoreceptors accounts for the unique pattern of vision loss in dry AMD. The data was published in the international peer-reviewed European Journal of Neuroscience.

About Age-Related Macular Degeneration

Age-related macular degeneration refers to a loss of photoreceptors (rods and cones) from the macula, the central part of the retina. AMD is a degenerative retinal disease that typically strikes adults in their 50s or early 60s, and progresses painlessly, gradually destroying central vision. According to the RFSW website, there are approximately 1.75 million Americans age 40 years and older with some form of age-related macular degeneration, and the disease continues to be the number one cause of irreversible vision loss among senior citizens in the United States with more than seven million at risk of developing AMD.

About the Trial

The Phase I/II trial will evaluate the safety and preliminary efficacy of HuCNS-SC cells as a treatment for dry AMD. The trial will be an open-label, dose-escalation study, and is expected to enroll a total of 16 patients. The HuCNS-SC cells will be administered by a single injection into the space beneath the retina in the most affected eye. Patients' vision will be evaluated using both conventional and advanced state-of-the-art methods of ophthalmological assessment. Evaluations will be performed at predetermined intervals over a one-year period to assess safety and signs of visual benefit. Patients will then be followed for an additional four years in a separate observational study. Patients interested in participating in the clinical trial should contact the site at (214) 363-3911.

About HuCNS-SC Cells

StemCells' proprietary product candidate, HuCNS-SC cells, is a highly purified composition of human neural stem cells that are expanded and stored as banks of cells. The Company's preclinical research has shown that HuCNS-SC cells can be directly transplanted in the central nervous system (CNS) with no sign of tumor formation or adverse effects. Because the transplanted HuCNS-SC cells have been shown to engraft and survive long-term, there is the possibility of a durable clinical effect following a single transplantation. StemCells believes that HuCNS-SC cells may have broad therapeutic application for many diseases and disorders of the CNS, and to date has demonstrated human safety data from completed and ongoing clinical studies.

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StemCells, Inc. Announces First Transplant of Neural Stem Cells Into Patient in Clinical Trial for Dry Age-Related ...