REGENERATIVE MEDICINE: THERAPEUTIC CLONING SUCCESSFULLY TREATS PARKINSON’S DISEASE IN MICE
Research led by investigators at Memorial Sloan-Kettering Cancer Center has shown that therapeutic cloning can be used to treat Parkinson’s disease in mice. The study, published in the journal Nature Medicine, showed for the first time that therapeutic cloning (also known as somatic-cell nuclear transfer) has been successfully used to treat disease in the same subjects from whom the initial cells were derived. While this current work is in animals, it could have future implications as this method may be an effective way to reduce transplant rejection and enhance recovery in other diseases and in other organ systems, the researchers said. In therapeutic cloning, or SCNT, the nucleus of a somatic cell from a donor subject is inserted into an egg from which the nucleus has been removed. This cell then develops into a blastocyst from which embryonic stem cells can be harvested and differentiated for therapeutic purposes. The scientists used skin cells from the tail of the animal to generate customized or autologous dopamine neurons—the missing neurons in Parkinson’s disease. The mice that received neurons derived from individually matched stem cell lines exhibited neurological improvement. But when these neurons were grafted into mice that did not genetically match the transplanted cells, the cells did not survive well and the mice did not recover.

 

SUPERBUGS: ALLIGATOR BLOOD MAY BE SOURCE OF POWERFUL NEW ANTIBIOTICS
Scientists from Louisiana reported at the 235th national meeting of the American Chemical Society that proteins in alligator blood may provide a source of powerful new antibiotics to help fight infections associated with diabetic ulcers, severe burns, and “superbugs” that are resistant to conventional medication. The study, from researchers at McNeese State University in Lake Charles, Louisiana and Louisiana State University in Baton Rouge, Louisiana, is described as the first to explore the antimicrobial activity of alligator blood in detail. The researchers said they found a range of promising uses for the gator’s antibiotic proteins. This includes combating Candida albicans yeast infections, which are a serious problem in AIDS patients and transplant recipients, who have weakened immune systems, the scientists say. Unlike humans, alligators can fight microorganisms such as fungi, viruses, and bacteria without having prior exposure to them. In laboratory tests, tiny amounts of these protein extracts killed a wide range of bacteria, including MRSA (methicillin-resistant Staphylococcus aureus), the deadly bacteria that are moving out of healthcare settings and into the community. These “superbugs” are increasingly resistant to multiple antibiotics and cause thousands of deaths each year. The proteins also killed six out of eight different strains of Candida albicans, the researchers say. Their previous research also suggests that blood proteins may help fight HIV, the virus that causes AIDS.

 

GENETIC DISCRIMINATION: FEDERAL PROTECTION NEEDED
The American College of Physicians released a policy paper highlighting the need for federal protections against genetic discrimination in employment and insurance practices. The organization, the second largest physician group in the country, includes six points it believes should be included in Congressional legislation. This includes prohibitions on insurance providers using an individual’s genetic information to deny or limit health coverage or establish eligibility, enrollment, or premium contribution requirements. It also calls for prohibiting insurance providers from differential premiums based on an individual’s genetic information. Similarly, the group wants employers to be prohibited from using such information in making employment, promotion, or benefits decisions. The group also said both insurers and employers should be prevented from requiring people to undergo genetic tests or collecting and disclosing information from genetic tests without informed consent. The group said Congress should establish comprehensive and uniform federal protection that closes gaps in protection in various states.

 

DIAGNOSTICS: SPIT TEST MAY SOON REPLACE BLOOD TESTS
Researchers have taken a major step in harnessing the use of saliva as an alternative to blood for performing diagnostics in a less invasive way to detect cancer, heart disease, or diabetes. Researchers at the University of Rochester Medical Center and elsewhere have produced a catalogue of the proteins in human saliva, according to an article published in the Journal of Proteome Research. Replacing blood draws with saliva tests promises to make disease diagnosis, as well as the tracking of treatment efficacy, less invasive and costly. Recent, parallel efforts that mapped the blood and tear proteomes allows for useful comparisons of how proteins and potential disease markers are common or unique to different body fluids, the researchers said.

 

CANCER: LATE-STAGE TRIAL SUCCESSFUL UP TO HALF THE TIME
About one-fourth to one-half of new cancer treatments that reach assessment in phase 3 randomized clinical trials are eventually proven successful, according to a report in the Archives of Internal Medicine. Cancer remains the second leading cause of death in the United States, but continuous improvements have been made in survival and other outcomes. Researchers at H. Lee Moffitt Cancer Center and Research Institute at the University of South Florida, Tampa examined data from all completed phase 3 randomized clinical trials conducted by the National Cancer Institute cooperative groups since their inception in 1955. They analyzed a total of 624 trials involving 216,451 patients. Overall, 30 percent of the trials had statistically significant results. In 80 percent of those cases, new treatments were superior to established protocols. The original researchers reported that the risk-benefit ratio favored new treatments in 41 percent of comparisons, while standard treatments were favored in 59 percent of comparisons.

 

OBESITY: WEIGHT MAY KEEP SOME WOMEN FROM GETTING SCREENED FOR BREAST AND CERVICAL CANCER
A review of cancer screening studies shows that white women who are obese are less likely than healthy weight women to get the recommended screenings for breast and cervical cancer, according to researchers at the University of North Carolina at Chapel Hill’s School of Public Health. The researchers said that it is a disturbing trend that women who are at increased risk of cancer because of their body size are less likely to be receiving screening tests that can detect cancer early, when it is treatable. According to a study published in the New England Journal of Medicine in 2003, 20 percent of cancer deaths in the U.S. in 2000 were attributable to obesity. The study, published online in the journal Cancer, did not determine why larger women are not getting screened as frequently for these cancers.

 

HEART ATTACK: SIGNALING PROTEIN HELPS LIMIT DAMAGE
Scientists at the Center for Translational Medicine at Thomas Jefferson University in Philadelphia have shown that a specific signaling protein is crucial to protecting the heart and helping it to adapt during a heart attack. The protein Gi is known to have increased activity in the failing heart, but researchers had not known if it helped the heart adapt to damage or if it caused more heart cells to die. The researchers blocked the protein in the hearts of genetically engineered mice experiencing heart attacks. They found that the animals had greater heart damage than did similar mice with a working protein. The study, published in the journal Circulation, found the heart wants to activate Gi and to protect heart cells from dying.

 

HUNTINGTON’S: NEW DRUG TARGETS IDENTIFIED FOR NEURODEGENERATIVE DISEASE
Scientists at the University of Cambridge have identified a number of candidate drugs that may encourage cells to “eat” the malformed proteins that lead to Huntington's disease, a neurodegenerative disease. Huntington's disease is one of a number of degenerative diseases marked by build up of malformed proteins in brain cells, mainly in the basal ganglia and the cerebral cortex. Normally, cells dispose of or recycle their waste material, including unwanted or misfolded proteins, through a process known as autophagy, or “self-eating.” There are currently no treatments available that slow the neurodegeneration in people with Huntington's disease. A study published online in the journal Nature Chemical Biology shows FDA-approved drugs for treatments such as migraine and hypertension are able to stimulate autophagy in fruit flies and zebrafish through unexpected pathways.

HEALTHCARE ECONOMICS: PATIENTS WHO GET FREE SAMPLES HAVE HIGHER OUT-OF-POCKET COSTS FOR DRUGS
Patients who receive free drug samples have significantly higher out-of-pocket prescription costs than those who don't, according to researchers from the University of Chicago Medical Center in the first study to look at the out-of-pocket cost associated with free-sample use. In a study published in the journal Medical Care, researchers found that patients who never received samples had estimated out-of-pocket prescription costs of $178 over six months. Patients who received samples spent an estimated $166 for a six-month period prior to getting free samples, $244 for the six months in which they received samples and $212 for the six-month period following sample receipt. The researchers said that while patients may get short-term economic relief from free samples, often they end up continuing using the medicine begun as samples, even though older, less expensive alternatives may exist.

 

A protein known as REST blocks the expression of a microRNA that prevents embryonic stem cells from reproducing themselves and causes them to differentiate into specific cell types, researchers from The University of Texas M. D. Anderson Cancer Center report in the online edition of the journal Nature. Researchers showed RE1-silencing transcription factor, or REST, plays a dual role in embryonic stem cells. It maintains self-renewal, or the cell’s ability to make more and more cells of its own type, and it maintains pluripotency, meaning that the cells have the potential to become any type of cell in the body. In the laboratory, scientists have been able to induce embryonic stem cells to develop into heart muscle cells or insulin-producing cells of the pancreas. In studies using mouse embryonic stem cells, the researchers found that REST disarms a specific microRNA called microRNA-21, or miR-21. MicroRNAs are tiny pieces of RNA that control gene expression by binding to the gene’s messenger RNA. The team found that miR-21 suppresses embryonic stem cell self-renewal and is associated with a corresponding loss of expression of critical self-renewal regulators. REST counters this by suppressing miR-21 to preserve the cells’ self-renewal and pluripotency.

 

REGULATION: FDA DEADLINES, BY RUSHING APPROVAL, MAY COMPROMISE SAFETY
Many medications are approved by the U.S. Food and Drug Administration on the brink of Congressionally mandated deadlines, and those drugs are more likely to face later regulatory intervention than those approved with greater deliberation, researchers at Harvard University report. Drugs fast-tracked by the FDA are more likely to eventually be withdrawn from global markets for safety reasons, undergo manufacturing revisions, or face labeling changes, according to the study published in the New England Journal of Medicine. The researchers said their findings suggest that drug safety might improve under an FDA approval protocol that is more flexible and less driven by deadline pressures and more by stable growth in FDA resources. The deadlines imposed on the FDA’s drug-approval process were first enacted as part of the Prescription Drug User Fee Act of 1992, which mandated that the FDA must act on 90 percent of all drug candidates within 12 months of submission or face funding cuts. The timeline was tightened to 10 months as part of the 1997 Food and Drug Administration Modernization Act, a timeline renewed by Congress in 2002 as part of bioterrorism legislation and renewed again in 2007. The researchers said they are not arguing that these deadlines should be abandoned, but said their research indicates that mechanisms other than strict deadlines may better balance the need for expeditious yet rigorous drug approval.

 

GENETICS: STUDY ANALYZES CELLULAR FUNCTION OF HOMINOID-ONLY GENE
Scientists at Washington University School of Medicine in St. Louis have produced the first detailed analysis of the cellular functions of a hominoids -only gene, TBC1D3. They affirmed earlier evidence linking the gene to cancer, showing that TBC1D3's protein can keep cellular growth factors active and helps turn on RAS, a protein that is active in a third of all human cancers. Among the approximately 23,000 genes found in human DNA, scientists currently estimate that there may be as few as 50 to 100 that have no counterparts in other species, and several hundred genes that are unique to the primate family known as hominoids.

 

ANTIDPRESSANTS: LINK FOUND BETWEEN SSRIs AND TYPE 2 DIABETES
People with a history of depression had a 30 percent increased risk of Type 2 diabetes, according to researchers at the University of Alberta’s School of Public Health. The researchers studied the medical history of 2,400 people who were diagnosed with depression and were taking antidepressants to determine whether there was a clear correlation between that disease and Type 2 diabetes. The study, published in Diabetes Research & Clinical Practice, foundthe risk of diabetes almost doubled for the patients who were using two types of therapies at the same time, tricyclic antidepressants, or TCAs, and selective serotonin reuptake inhibitors, or SSRIs. The researchers said their findings, as well as previous studies that found an increased risk of Type 2 diabetes in people with depression, emphasize the need for regular screening for Type 2 diabetes in people with depression, particularly those taking more than one antidepressant.

 

CANCER: NEW FORM OF INHERITED RISK IDENTIFIED
A multi-institution group led by researchers at Weill Cornell Medical College, has identified parts of the genome that are strikingly similar among people from a particular population group who have the same type of cancer. The researchers said this so-called autozygosity (identical copies of DNA inherited from both parents) might serve not only as a way to predict susceptibility to cancer in some people, but may lead researchers to novel cancer-causing genes. More broadly, the work suggests a new type of genetic signpost that clinicians might follow for a range of cancers, in many population groups.

The study was published online in the journal Cancer Research.

 

SCHIZOPHRENIA: RARE MUTATIONS AS MUCH AS FOUR TIMES HIGHER IN PEOPLE WITH THE DISORDER
People with schizophrenia have high rates of rare genetic deletions and duplications that likely disrupt the developing brain, according to teams of researchers led by the National Institute of Mental Health, Cold Spring Harbor Laboratory, and the University of Washington. These tiny anomalies were found in 15 percent of adult onset schizophrenia patients and 20 percent of child and adolescent onset patients, compared with only 5 percent of healthy participants. Collectively, the mutations carried by patients were significantly more likely than those in healthy participants to disrupt genes involved in brain development—potentially implicating hundreds of genes in the illness, which affects about 1 percent of adults. The researchers, who published their findings online in Science Express, said identifying genes prone to harboring these mutations in brain development pathways holds promise for treatment and prevention of schizophrenia, as well as a wide range of other neurodevelopmental brain disorders. 

 

Most “sandwich caregivers”—the parents or guardians of children under 21 who also care for an aging parent, other relative, or friend with Alzheimer’s disease—say their children are assisting with caregiving responsibilities that range from attending doctors’ appointments to feeding and dressing their loved ones. Survey results found that about three in five caregivers say their children aged 8 to 21 are involved in caring for a loved one with Alzheimer’s disease. Of the caregivers who feel they do a good job balancing the care of their loved ones with Alzheimer’s disease and children under 21, more than one-third (36%) specifically cited support from children as a contributor to their success.

Among children, ages 8 to 21, who are involved in caregiving, many are reported as taking on significant tasks. It is estimated that 5.7 million Americans caring for aging relatives and loved ones also have children whom they care for. With the United States population aging rapidly, the need for family caregivers will markedly increase in the years ahead.