STEM CELLS: CALIFORNIA AGENCY GRANTS LEVERAGED TO CREATE $1.1 BILLION IN RESEARCH FACILITY CONSTRUCTION
The California Institute for Regenerative Medicine, the state’s stem cell agency, voted today to distribute $271 million to 12 institutions to build stem cell research facilities throughout California. The institutions committed an additional $560 million from charitable donations and their internal reserves, bringing the total statewide investment in new research space to $831 million. This leverage of the state’s stem cell funds was further increased by additional institutional commitments for faculty recruitment packages and other related capital costs. In total, the state funding will have leveraged $1.1 billion in new resources to perform research that could lead to therapies for patients with chronic and debilitating disease and injury.

 

GENE THERAPY: STEM CELL PROVIDES SAFE APPROACH
Researchers at the University of California, Davis said they have evidence that using human bone marrow-derived stem cells to deliver gene therapy to cure diseases of the blood, bone marrow, and certain types of cancer does not cause the development of tumors or leukemia. The study, published in an online edition of the journal Molecular Therapy, shows that there is little risk of adverse events caused by gene transfer, and that adult human stem cells do not pose a cancer risk when implanted into different organs, the researchers said. The researchers tested the safety of gene transfer into bone marrow stem cells from human donors in more than 600 mice. None of the transplanted mice developed leukemia or solid tumors caused by the gene therapy treatment, during the evaluation period of up to 18 months.

 

CANCER: NANOWORMS CAN CRUISE THROUGH BLOODSTREAM LIKE MISSLE TO ITS TARGET
Scientists at the University of California, San Diego; the University of California, Santa Barbara; and MIT have developed so called “nanoworms” that can cruise through the bloodstream without significant interference from the body’s immune defense system and home in on tumors. The discovery, reported in the journal Advanced Materials, could allow doctors to eventually be able to target and reveal the location of developing tumors that are too small to detect by conventional methods. Carrying payloads targeted to specific features on tumors, these nanometer-sized vehicles could also one day provide the means to more effectively deliver toxic anti-cancer drugs to these tumors in high concentrations without negatively affecting other parts of the body. The scientists constructed their nanoworms from spherical iron oxide nanoparticles that join together, like segments of an earthworm, to produce tiny gummy worm-like structures about 30 nanometers long—or about 3 million times smaller than an earthworm. Their composition allows them to show up brightly in diagnostic devices, specifically the MRI, or magnetic resonance imaging, machines that are used to find tumors.

 

GASTROINTESTINAL DISEASE: UCSD MED SCHOOL SCORES $2.5 MILLION GRANT TO ESTABLISH DIGESTIVE DISEASE RESEARCH DEVELOPMENT CENTER
UC San Diego School of Medicine won a $2.5-million basic research core grant to establish a Digestive Diseases Research Development Center. The grant, funded by The National Institute of Diabetes and Digestive and Kidney Diseases of the National Institutes of Health, is the first-ever NIH Center award for the division, adding them to the ranks of only 19 other NIH-funded digestive disease centers in the United States. The UCSD Division of Gastroenterology researches and treats digestive diseases, ranging from stomach ulcers to colon cancer.

ALZHEIMER’S: RESEARCHERS IDENTIFY NEW TARGET
A study published in the Journal of Neuroscience, researchers at the Gladstone Institute of Neurological Disease in San Francisco have determined in mouse models that modulating the activity of enkephalin peptides in the brain might reduce the cognitive deficits seen in Alzheimer’s disease. Enkephalins are part of the endogenous opioid system, which modulates learning and memory and other brain functions. They are produced by several different cell types in the brain, particularly in areas affected by AD. Enkephalins are derived by enzymatic cleavage from a precursor protein, eproenkephalin, and stored in vesicles. Upon stimulation, enkephalins are released with neurotransmitters, such as glutamate. The researchers said the enkephalin pathway is an intriguing candidate because it is involved in many functions that are affected by Alzheimer’s and other neurodegenerative diseases. Although the research is early, it may lead the way to a new treatment for Alzheimer’s based on limiting enkephalin production of signaling, the researchers said.

ANTIBIOTICS: NEKTAR GETS PATENT FOR INHALABLE ANTIBIOTICS
San Carlos, California-based Nektar Therapeutics said that the U.S. Patent and Trademark Office has issued a patent that covers compositions and methods for creating and delivering Tobramycin Inhalation Powder and other aminoglycoside antibiotics delivered via Nektar’s proprietary Dry Powder Inhaled Technology. This patent extends through the year 2022. Nektar is co-developing Tobramycin Inhalation Powder with Novartis and is currently in a late-stage development for the prevention and treatment of Pseudomonas aeruginosa pneumonia. Cystic fibrosis patients are susceptible to chronic infection by P. aeruginosa, which is responsible for high rates of illness and death in this patient population. Nektar’s Dry Powder Inhaled Technology has the capability to administer high local lung concentrations of Tobramycin Inhalation Powder that may provide significant benefits to patients suffering from this debilitating disease, the company said.

 

OBESITY: AMYLIN INITIATES MID-STAGE TRIAL OF COMBINATION THERAPY
San Diego-based Amylin Pharmaceuticals said it initiated a mid-stage clinical trial to  evaluate various dosing combinations of pramlintide, an analog of the natural hormone amylin, and recombinant human leptin for the treatment of obesity. Leptin is a critical hormone in energy metabolism and plays a unique role in inducing fat-specific weight loss. The objective of this dose-ranging study is to support dose selection for a late-stage clinical trial, and to inform the ongoing development of a convenient delivery system for this combination regimen. The six-month, randomized, double-blind, placebo-controlled multi-center study will enroll approximately 600 overweight and obese subjects and is expected to complete in mid-2009.

 

INFLAMMATORY DISEASE: REGULUS LICENSES INTELLECTUAL PROPERTY FROM STANFORD
Carlsbad, California-based Regulus Therapeutics, a joint venture between Alnylam Pharmaceuticals and Isis Pharmaceuticals, said it has obtained exclusive rights from Stanford University to worldwide patent applications covering methods and compositions for antagonizing miR-181a to regulate immune responses. Changes in miR-181a levels have been shown to modify the response of immune cells such as T lymphocytes to specific stimuli, and its antagonism could lead to a new way to treat inflammatory diseases. In 2007, scientists at Stanford University and Alnylam reported data in the journal Cell that demonstrated that modulation of miR-181a levels in an immune cell modified the sensitivity of the cell to specific stimuli. Researchers found that by increasing expression of miR-181a, an increase in the immune cell’s response to an inflammatory stimulus occurred. Conversely, decreasing levels of miR-181a in the immune cell led to a diminution in the cell’s response to an inflammatory stimulus, thereby de-sensitizing the cell to the stimulus.

PROSTATE CANCER: IMAGING DEVICE THAT PINPOINTS CANCER GETS FDA CLEARANCE
Grass Valley, California-based Eigen said its imaging device known as ei•Nav/Artemis has cleared the U.S. Food and Drug Administration. The device offers urologists technology that the company said will significantly help in the fight against prostate cancer. Artemis will be introduced at the American Urological Association’s annual meeting May 17 through May 22 in Orlando, Florida. Using proprietary next-generation 3D/4D imaging, Artemis provides technology not available today by enhancing urologists’ existing ultrasound machines, the vast majority of which are only 2D. Now, Artemis allows urologists to virtually see inside the prostate in real time during biopsy, guides them with 4D needle navigation during the delicate procedure, maps biopsy locations, and generates an image of 3D biopsy coordinates for future reference.

OSTEOPOROSIS: FDA ACCPETS OSTEOLOGIXO APPLICATION TO BEGIN CLINICAL TRIALS ON LEAD CANDIDATE
San Francisco-based Osteologix said that the U. S. Food and Drug Administration has accepted its investigational new drug application for its lead development candidate NB S101 (strontium malonate), for the treatment and prevention of osteoporosis. NB S101, a dual acting bone agent, has a demonstrated ability to decrease resorption of bone while maintaining formation of new bone. The drug has been formulated as a once-daily tablet. In September 2007, the company presented data from an early-stage study at the American Society for Bone and Mineral Research, demonstrating that NB S101 has superior bioavailability as compared to Protelos (strontium ranelate), a strontium-based compound approved for sale in Europe and other territories outside the United States. In November 2007, Osteologix announced significant results from its mid-stage study, demonstrating that NB S101 met its primary endpoint. At all doses tested, NB S101 demonstrated statistically significant decreases in CTX-1, a well-validated biomarker that measures bone resorption activity.