A multi-disciplinary group from Stanford University has proposed ten principles to guide the use of racial and ethnic categories in genetic research to minimize the misinterpretation and misuse of human genetic variation research. The principles include a declaration that the group does not believe that there is any scientific basis for hierarchically ordered categories of race or ethnicity, according to the report in open access journal Genome Biology. There is also a recognition that racial and ethnic categories are created and maintained by socio-political contexts and change over time. The group included members of the humanities, social and life sciences, law and medical schools at Stanford University. The group said it cautions against genetic explanations for group differences in complex traits, especially for human behavioral traits such as IQ, tendency to violence, or degrees of athleticism. The group said it believes the guiding principles constitute one step in an ongoing, open dialogue about these concerns and hope that they will encourage responsible practices.

 

BONE LOSS: AMGEN REORTS POSITIVE RESULTS FOR DENOSUMAB IN MEN WITH NON-METASTATIC PROSTATE CANCER

Thousand Oaks, California-based Amgen said its denosumab treatment for bone loss produced statistically significant greater increases in bone mineral density in a three-year Phase III trial involving men undergoing androgen deprivation therapy for non-metastatic prostate cancer. In this study of more than 1,400 men, denosumab resulted in statistically significant greater increases in bone mineral density at the lumbar spine (primary endpoint) and non-vertebral sites compared with placebo at multiple time points, Amgen said. The company said these improvements in bone mineral density were consistent with those seen in other denosumab studies evaluating the bone mineral density in women with breast cancer receiving aromatase inhibitor therapy, and in post-menopausal women with low bone mass. Denosumab is the first fully human monoclonal antibody in late stage clinical development that specifically targets RANK Ligand, the essential regulator of osteoclasts (the cells that break down bone). During the 36-month evaluation period, men receiving denosumab experienced less than half the incidence of new vertebral fractures (a secondary endpoint) compared with those receiving placebo, a statistically significant finding. Furthermore, in the denosumab arm there were fewer non-vertebral fractures over the 36-month period.

 

AVIAN FLU: INOVIO REPORTS DNA VACCINES SHOWS 100 PERCENT PROTECTION AGAINST BIRD FLU IN PRECLINICAL TEST
San Diego-based Inovio Biomedical said pre-clinical results from two proprietary plasmid DNA-based universal influenza vaccine candidates using the company’s proprietary delivery technology and a new intradermal device showed that 100 percent of the immunized mice given a lethal challenge of highly pathogenic H5N1 influenza virus survived and showed only minor weight loss. The DNA vaccine design was based on a different influenza strain (H1N1) than the influenza strain used in the challenge, providing evidence that a universal vaccine based on conserved genes common to multiple strains of seasonal influenza and even potential pandemic influenza may have the possibility to provide widespread protection against such viruses, the company said. Current strain-specific influenza vaccines are designed to stimulate an immune response specific to a single influenza strain.

 

ALZHEIMER’S: DIMEBON IMPROVES THINKING PROCESSES AND ABILITY TO FUNCTION IN PATIENTS WITH ALZHEIMER'S
San Francisco-based Medivation’s drug Dimebon, which had once been approved as an antihistamine in Russia, improved thinking processes and ability to function in patients with Alzheimer's disease, in a study conducted by researchers at Baylor College of Medicine in Houston. The researchers, who published their findings in the current issue of The Lancet, said Dimebon is the first drug for Alzheimer's disease that demonstrated continued improvement in patients over a 12 month period. Other approved drugs do not have this effect. Researchers believe the medication works by stabilizing mitochondria, the cellular components that produce energy, and possibly by inhibiting brain cell death. Researchers evaluated patients' thinking and memory ability, overall function, psychiatric and behavioral symptoms, and ability to perform daily activities. The researchers said that at this point in a drug’s development they would be happy to see improvement in one of the outcome measure, but they saw improvements in all five. Medivation funded the study and the lead author is a member of the company’s Scientific and Clincial Advisory board.

 

PANDEMIC FLU: VICAL SAYS PRELIMINARY HUMAN DATA ON DNA VACCINE SHOWS BREAKTHROUGH
San Diego-based Vical said it had a breakthrough with preliminary clinical trial data demonstrating that DNA vaccines can safely achieve significant immune responses against H5N1 pandemic influenza in humans. DNA vaccines are fundamentally different from conventional vaccines because they do not contain any part of the virus itself, and may offer compelling advantages in response to a pandemic outbreak because of significantly reduced development and manufacturing times. Specifically, preliminary human safety and immunogenicity data obtained in a 100-subject early-stage trial of the company’s Vaxfectin-formulated H5N1 pandemic influenza DNA vaccines demonstrated for the first time that DNA vaccines have achieved potentially protective levels of antibody responses in up to 67 percent of evaluable subjects in the higher dose cohorts. No significant safety issues were observed at any of the Vical vaccine doses tested.

 

LIVER DISEASE: RAPTOR PHARMACEUTICAL ENTERS COLLABORATION WITH UCSD
Novato, California-based Raptor Pharmaceuticals said it entered into a collaboration agreement with the University of California, San Diego to include a mid-stage clinical trial to evaluate a delayed-release preparation of cysteamine bitartrate in adolescents diagnosed with Non-Alcoholic Steatohepatitis or NASH. NASH is a progressive form of liver disease that accounts for approximately 10 percent of newly diagnosed cases of chronic liver disease, and ranks as one of the leading causes of cirrhosis of the liver in the U.S. Under the terms of the Agreement, clinical researchers from UCSD will perform the study at the university’s General Clinical Research Center, and Raptor will provide funding and clinical supply of Cysteamine. In March 2008, Raptor acquired an exclusive, worldwide license to certain intellectual property and development rights from UCSD surrounding the use of Cysteamine as a potential treatment for NASH. A rapid-release form of Cysteamine is currently approved for sale by the U.S. Food and Drug Administration for the treatment of nephropathic cystinosis, a rare, genetic lysosomal storage disease. Raptor is currently developing DR Cysteamine, a proprietary, delayed-release formulation of Cysteamine, designed to improve bioavailability and potentially reduce side effects associated with the presently marketed form.

AUTISM: RESEARCHERS FIND FURTHER EVIDENCE OF GENETIC CAUSES
Some parents of children with autism evaluate facial expressions differently than the rest of us—and in a way that is strikingly similar to autistic patients themselves, according to researchers at the California Institute of Technology and the University of North Carolina at Chapel Hill. The researchers studied 42 parents of children with autism. Based on psychological testing, 15 of the parents were classified as being socially aloof. The subjects were shown images depicting facial expressions of emotion that were digitally filtered so that only certain regions of the face were discernible—the left eye, for example, or the mouth. The subjects were then asked to decide as quickly as possible if the emotion depicted was “happy” or “fear.” An analysis of the subjects’ correct responses revealed that “aloof” parents relied much more heavily on the mouth to recognize emotion than they did on the eyes, as compared to nonaloof parents and, to a greater extent, to a group of parents of children without autism. The researchers said the study, published in the online edition of Current Biology, indicates that certain aspects of autism do run in families. The researchers said their data strongly suggest that genetic factors make a substantial contribution to autism, but that does not mean that the entire cause of autism is genetic.

 

ENVIRONMENTAL POLLUTANTS: WOMEN EXPOSED TO HIGH LEVELS OF PCBs MORE LIKELY TO GIVE BIRTH TO GIRLS
Women exposed to high levels of PCBs (a group of banned environmental pollutants known as polychlorinated biphenyls) are less likely to give birth to male children, according to researchers at the University of California, Berkeley. The study, published in the open access journal Environmental Health found that among women from the San Francisco Bay Area, those exposed to higher levels of PCBs during the 1950s and 1960s, were significantly more likely to give birth to female children. PCBs are persistent organic pollutants identified worldwide as human blood and breast milk contaminants. They are associated with effects on immune, reproductive, nervous, and endocrine systems. Given the high quality measurements, this research provides the strongest evidence to date that PCBs affect sex ratio in human children. The women most exposed to PCBs were 33 percent less likely to give birth to male children than the women least exposed, the researchers said.

 

HEPATITIS C: UCSD DEVELOPED TISSUE CULTURE CAN MODEL HCV INFECTION TO TEST NEW THERAPIES
A University of California, San Diego School of Medicine researchers have developed the first tissue culture of normal, human liver cells that can model infection with the Hepatitis C virus and provide a realistic environment to evaluate possible treatments. The cell line, described in the journal PLoS ONE, will allow pharmaceutical companies to effectively test new drug candidates or possible vaccines for the HCV infection, which afflicts about 170 million people worldwide. Currently, there is no animal model that is effective for testing such therapies. New therapies are needed because currently, there is only a single treatment for HCV, PEG- interferon-α. The drug combination has an average response rate of about 50 percent in HCV cases, but it is much lower than that, closer to 20 percent, in individuals with liver cirrhosis. It can also cause severe flu-like side effects. Approximately 10,000 deaths due to cirrhosis of the liver and several thousand more from liver cancer are attributed to HCV infection in the United States each year.