STEM CELLS: SOUTHERN CALIFRONIA RESEARCH INSTITUTIONS FORM COLLABORATION
Research institutions across southern California have joined forces to advance stem cell research by establishing the Southern California Stem Cell Scientific Collaboration. The institute, dubbed SC3, is a collaboration of the University of Southern California, Childrens Hospital Los Angeles, City of Hope, University of California at Santa Barbara, California Institute of Technology, and the House Ear Institute. The organizations said the agreement is a major step forward in supporting potentially significant stem cell findings by allowing members to share training programs and scientific core facilities and expertise, and to team up on a wide range of research programs. Each institution will appoint a faculty member to serve on a joint scientific advisory committee, which will serve as a forum to develop collaborative research ventures, facilitate access to scientific resources, and provide expertise across the collaboration. 

 

Menlo Park, California-based Geron said that it enrolled its first patient in a clinical trial of its telomerase inhibitor drug in patients with multiple myeloma, a malignancy of mature plasma cells. The trial is the fourth underway involving Geron’s novel inhibitor of telomerase, an enzyme that is expressed in all major types of cancer cells. The trial is being conducted to determine the safety and maximum tolerated dose of the drug known as GRN163L when administered intravenously in patients with multiple myeloma who have failed at least two courses of standard chemotherapy. In addition, researchers will perform correlative laboratory studies on primary patient samples to characterize the effects of GRN163L on myeloma cancer cells. Multiple myeloma usually arises in the bone marrow and is characterized by interference with production of blood cells, destructive lesions of bone that can cause debilitating fractures, and excessive production of specific antibody molecules. These abnormal molecules may interfere with the function of many tissues and organs in addition to bone marrow, including the kidneys. Multiple myeloma is the second most common hematological malignancy of adults, with about 20,000 new cases each year in the United States.

 

REGENERATIVE MEDICINE: STEM CELLS MAY OFFER ALTERNATIVE TO HEART TRANSPLANT
A study from University of California at Davis researchers report the first functional evidence that heart cells derived from human embryonic stem cells exhibit one of the most critical properties of mature adult heart cells, an important biological process called excitation-contraction coupling. The finding gives scientists hope that these cells can one day be coaxed into becoming functionally viable cells safe for transplantation into the damaged hearts of patients with end-stage disease, potentially avoiding the necessity of a heart transplant. Currently, there are nearly 3,000 people on heart transplant lists around the nation, including more than 300 in California. The study, published online in Stem Cell Express, is the first to find evidence of the functional calcium stores needed for fully functional pumping activity for heart cells derived from human embryonic stem cells.  

 

AGING: STANFORD RESEARCHERS PRODUCE SHORT-TERM REVERSAL OF PROCESS IN SKIN OF MICE
Researchers at the Stanford University School of Medicine have reversed the effects of aging on the skin of mice, at least for a short period, by blocking the action of a single critical protein. The work could one day be useful in helping older people heal from an injury as quickly as they did when they were younger, the researchers said. However, they warn that their findings, which will be published in the journal Genes and Development, will likely be useful in short-term therapies in older people, but not as a potential fountain of youth. The work backs up the theory that aging is the result of specific genetic changes rather than accumulated wear and tear, the researchers said. Those changes can be reversed even late in life. The study identified the protein NF-kappa-B and regulating age-related genes.

 

BioMarin Pharmaceutical said it has filed an investigational new drug application with the U.S. Food and Drug Administration for PEG-PAL, formerly known as Phynylase, for the treatment of phenylketonuria, or PKU. The Novato, California-based company expects to initiate a clinical study of PEG-PAL in PKU patients in the first quarter of 2008. PKU, a genetic disorder affecting approximately 50,000 diagnosed patients in the developed world, is caused by a deficiency of the enzyme phenylalanine hydroxylase, or PAH. PAH is required for the metabolism of phenylalanine, an essential amino acid found in most protein-containing foods. If the active enzyme is not present in sufficient quantities, phenylalanine accumulates to abnormally high levels in the blood and becomes toxic to the brain, resulting in a variety of complications, including severe mental retardation and brain damage, mental illness, seizures, tremors, and limited cognitive ability. 

 

Carlsbad, California-based Ardea Biosciences said it has initiated a clinical development program directed toward the treatment of gout. Gout, also known as metabolic arthritis, is a painful and debilitating disease caused by abnormally elevated levels of uric acid in the blood stream. These elevated levels lead to the deposition of uric acid crystals in and around the connective tissue of the joints and in the kidneys, leading to inflammation, the formation of disfiguring nodules, intermittent attacks of severe pain, and kidney damage. Earlier this year, Ardea conducted a comprehensive early-stage clinical program in 98 healthy volunteers to evaluate the safety and pharmacokinetics of RDEA806, its lead non-nucleoside reverse transcriptase inhibitor for the potential treatment of HIV. Analysis of the data from the study showed a statistically significant, exposure-dependent reduction in serum uric acid. The magnitude of the uric acid reduction correlates with the individual's baseline uric acid level, such that the higher the baseline uric acid level, the larger the absolute uric acid reduction. Ardea is preparing to initiate a mid-stage, dose-ranging clinical efficacy study in gout patients with hyperuricemia in the first half of 2008. An estimated 3 million to 5 million people in the U.S. suffer from gout, which is the most common form of inflammatory arthritis in men over 40. The company said gout represents a major medical challenge, and, with no new treatments approved by the U.S. Food and Drug Administration for the underlying cause of gout in the past 40 years, there is a significant need for new therapeutic agents.

 

Synthetic Blood International of Costa Mesa, California said the U.S. Office of Naval Research has given a $1.3-million grant to Virginia Commonwealth University Reanimation Engineering Shock Center for the treatment and prevention of decompression sickness with Oxycyte. Oxycyte is Synthetic Blood’s proprietary perfluorocarbon therapeutic oxygen carrier and blood substitute. Synthetic Blood has begun the process of manufacturing and shipping Oxycyte to the center for use in these studies. Decompression sickness occurs when a diver or submarine ascends to the water’s surface too quickly, causing nitrogen gas that was dissolved in blood to form bubbles which then clog the blood vessels supplying oxygen to the heart and central nervous system. This can result in massive stroke, paralysis, and death. The company said Oxycyte has proven capability to carry significantly more nitrogen and oxygen than plasma. The researchers will look at the potential therapeutic benefits of Oxycyte for use in multiple states of critical illness and injury, including traumatic brain injury. 

 

The first study on progesterone and Alzheimer’s disease has found no clear preventive benefit for the widely prescribed hormone in an animal model. Progesterone is given with estrogen in hormone replacement therapy. Previous studies have suggested that estrogen offers women some protection against Alzheimer’s disease, but researchers at the University of Southern California, asked if the same could be true of progesterone. In a study highlighted in the Journal of Neuroscience, the researchers report that progesterone has only limited benefit for mice with Alzheimer’s symptoms when taken alone. When taken with estrogen, progesterone actually inhibits some of the other hormone’s beneficial effects, the study found.

 

DRUG DEVLEOPMENT: SINGULEX COLLABORATES WITH WYETH TO FIND BIOMARKERS IN BLOOD
Hayward, California-based Singulex said it is collaborating with researchers at Wyeth Research to develop immunoassays on the company's Erenna system. Singulex has installed the Erenna system at Wyeth and has begun optimizing assays that will allow Wyeth to accurately and precisely measure specific biomarkers in human blood. Biomarkers play an important role in the evaluation of compounds in both drug discovery and development. But many biomarkers are present in human blood at extremely low concentrations, making it difficult to accurately and precisely measure. Singulex has provided Wyeth with a new technology for detection of biomarkers at very low concentrations. Wyeth said it is now validating several assays that it expects will guide its clinical programs.

 

Carlsbad, California-based Isis Pharmaceuticals, Inc. and Encinitas, California-based Excaliard Pharmaceuticals said they will collaborate to discover and develop antisense drugs for the local treatment of fibrotic diseases, including scarring. Isis has granted Excaliard an exclusive worldwide license for the development and commercialization of certain antisense drugs. Excaliard made an upfront payment to Isis in the form of equity and paid Isis $1 million in cash for the licensing of a particular gene target. In addition, Isis is eligible to receive development milestones and royalties on antisense drugs developed by Excaliard. Antisense uses DNA or RNA strands to interfere with complimentary mRNA and to prevent the production of a protein underlying a given disease.

GENERICS: WATSON WINS APPROVAL FOR ITS COMBINATION PAIN DRUG
Corona, California-based Watson Pharmaceuticals said it received final approval from the U.S. Food and Drug Administration on its Abbreviated New Drug Application for Oxycodone Hydrochloride and Ibuprofen Tablets in the 5 mg/400 mg strength. Watson's Oxycodone Hydrochloride and Ibuprofen product is the generic equivalent to Forest Laboratories Inc.'s Combunox, which is indicated for the short-term (not more than seven days) management of acute, moderate to severe pain. For the 12-months ending September 2007, Combunox had total U.S. sales of approximately $4 million, according to IMS Health data. Watson intends to launch its Oxycodone Hydrochloride and Ibuprofen product immediately.