'Support cells' in brain play important role in Down syndrome

Researchers from UC Davis School of Medicine and Shriners Hospitals for Children – Northern California have identified a group of cells in the brain that they say plays an important role in the abnormal neuron development in Down syndrome. After developing a new model for studying the syndrome using patient-derived stem cells, the scientists also found that applying an inexpensive antibiotic to the cells appears to correct many abnormalities in the interaction between the cells and developing neurons. “We have developed a human cellular model for studying brain development in Down syndrome that allows us to carry out detailed physiological studies and screen possible new therapies,” said Wenbin Deng, associate professor of biochemistry and molecular medicine and principal investigator of the study. “This model is more realistic than traditional animal models because it is derived from a patient’s own cells.”

Transparency Lacking in Clinical Trials, BU Study Finds

“Individuals who volunteer for clinical trials often do so out of a sense of altruism,” said Gill, a researcher with BU’s Center for Global Health and Development. ”I can imagine that many would be dismayed to learn that the results of a study that they participated in, that they took physical risks for, might never generate results known beyond the company that sponsored the trial. “Science learns from mistakes, as well as successes,” he said. “If we only learn about the scientific success stories, we are really only seeing part if the picture.”

Better use of electronic health records makes clinical trials less expensive

By studying these records, researchers are able to understand health patterns in relation to specific medications with potentially much larger and more diverse members of the public, and to understand which treatment offers the best results. The research is all conducted with minimal impact on the lives of the patients who, after offering their consent are not required to have any active involvement. Prof van Staa said: “The use of electronic health records in simplifying clinical trials means that we no longer need to remain uncertain about which medicine offers the best health benefits for patients. This study shows that scientists are able to conduct research which will highlight which treatment is best for patients.”

FDA sets out framework for rare childhood diseases

The FDA has laid out plans to encourage the development of new therapies for rare diseases in children, including a quicker approval route, greater use of biomarkers in clinical trials, and more engagement with the private sector. With the new strategic plan the US regulator wants to tackle the unique challenges faced when developing new drugs and medical devices for rare paediatric diseases, such as the low number of patients who can participate in studies. "Not only is there the potential for children to respond differently to products as they grow but there are also additional ethical concerns for this patient population," said Jill Hartzler Warner, FDA's associate commissioner for special medical programmes in a blog.

Single Dose Reverses Autism-like Symptoms in Mice

n a further test of a novel theory that suggests autism is the consequence of abnormal cell communication, researchers at the University of California, San Diego School of Medicine report that an almost century-old drug approved for treating sleeping sickness also restores normal cellular signaling in a mouse model of autism, reversing symptoms of the neurological disorder in animals that were the human biological age equivalent of 30 years old. Naviaux said the findings fit neatly with the idea that autism is caused by a multitude of interconnected factors: “Twenty percent of the known factors associated with autism are genetic, but most are not. It’s wrong to think of genes and the environment as separate and independent factors. Genes and environmental factors interact. The net result of this interaction is metabolism.”

Study finds cognitive performance can be improved in teens months, years after traumatic brain injury

Traumatic brain injuries from sports, recreational activities, falls or car accidents are the leading cause of death and disability in children and adolescents. While previously it was believed that the window for brain recovery was at most one year after injury, new research from the Center for BrainHealth at The University of Texas at Dallas shows cognitive performance can be improved to significant degrees months, and even years, after injury, given targeted brain training. "The after-effects of concussions and more severe brain injuries can be very different and more detrimental to a developing child or adolescent brain than an adult brain," said Dr. Lori Cook, study author and director of the Center for BrainHealth's pediatric brain injury programs. "While the brain undergoes spontaneous recovery in the immediate days, weeks, and months following a brain injury, cognitive deficits may continue to evolve months to years after the initial brain insult when the brain is called upon to perform higher-order reasoning and critical thinking tasks."

NIH Will No Longer Require Special Review for U.S. Gene Therapy Trials

Instead, the 40-year-old Recombinant DNA Advisory Committee (RAC) will review only a few trials that pose special risks. As the field has matured and gene therapy has been used to treat disorders such as inherited blindness and immune deficiencies, researchers have argued that the risks are no different than those of other experimental treatments.

Clinical Trials 101

Thinking of participating in a clinical trial? Check out our 101-course on what you need to know about clinical trials and the important questions you should be asking.

Medical devices for kids are often only tested on adults

Children are not simply 'small adults,' and a device found to be safe and effective in adults may have a very different safety and effectiveness profile when used in a pediatric population. Without this data, it is difficult for clinicians and parents to make informed treatment decisions that weigh the risks and benefits of a particular treatment.

NIH stem-cell programme closes

CRM will not continue in its current form. To that end, the NIH plans to hold a workshop in May to gather stem-cell researchers together and decide what to do with the programme and its remaining budget.