Researchers have conducted the first-ever analysis of clinical trials for Alzheimer’s disease, revealing an urgent need to increase the number of agents entering the AD drug development pipeline and progressing successfully towards new therapy treatments.
A comprehensive look at all clinical trials underway shows:
- There are relatively few drugs in development for Alzheimer’s disease.
- The failure rate for AD drug development is 99.6 percent for the decade 2002-2012.
- The number of drugs has been declining since 2009.
“Our goal was to examine historical trends to help understand why Alzheimer’s disease treatment development efforts so often fail,” said Jeffrey L. Cummings, M.D., ScD, Director of the Cleveland Clinic Lou Ruvo Center for Brain Health. “With an estimated 44 million people living worldwide with the condition, the study shows that the Alzheimer’s disease drug development ecosystem needs more support given the magnitude of the problem.”read more
A study led by Alfonso Valencia, Vice-Director of Basic Research at the Spanish National Cancer Research Centre (CNIO) and head of the Structural Computational Biology Group, and Michael Tress, researcher at the Group, updates the number of human genes -those that can generate proteins- to 19,000; 1,700 fewer than the genes in the most recent annotation, and well below the initial estimations of 100,000 genes. The work, published in the journal Human Molecular Genetics, concludes that almost all of these genes have ancestors prior to the appearance of primates 50 million years ago.read more
Now, research led by investigators at Beth Israel Deaconess Medical Center (BIDMC) adds another piece to the puzzle, demonstrating that the transcription factor IRF4 (interferon regulatory factor 4) plays a key role in brown fat’s thermogenic process, regulating energy expenditure and cold tolerance. The findings appear in the July 3 issue of the journal Cell.
“The discovery several years ago that brown fat plays an active role in metabolism suggested that if we could manipulate the number or activity of these fat cells, we could force our bodies to burn extra calories,” explains the study’s senior author Evan Rosen, MD, PhD, an investigator in the Division of Endocrinology, Diabetes and Metabolism at BIDMC and Associate Professor of Medicine at Harvard Medical School. “Now that we have identified a major factor driving this process, we can look for new approaches to exploit this for therapeutic benefit.”read more
Scientists have successfully mapped the whole genome sequence of the Atlantic salmon, a breakthrough that should accelerate selective breeding programs for farmed salmon and reduce the aquaculture industry’s impact on wild salmon stocks.
“This has the potential to improve the sustainability of aquaculture, to reduce feed demand and increase feed efficiency and it might lead to reduced susceptibility to disease in fish farms,” said Brian Riddell, president and CEO of the Pacific Salmon Foundation, a non-governmental conservation organization. “If we can improve the performance of aquaculture it will translate into less risk for the Pacific salmon.”read more
Understanding the basis of psychiatric disorders has been extremely challenging because there are many genetic variants that may increase risk but are insufficient to cause disease. Now investigators describe a strategy that may help reveal how such ‘subthreshold’ genetic risks interact with other risk factors or environmental exposures to affect the development of the nervous system. Their research pinpoints a genetic variant that may predispose individuals to schizophrenia.
The work takes advantage of a recently developed technology that allows skin cells from patients to be reprogrammed into induced pluripotent stem cells (iPSCs) that can then generate any cell type in the body. Through this technology, scientists obtained stem cells from individuals with a genetic abnormality that confers increased susceptibility to schizophrenia, and they observed deficits during nerve development that could be traced to a specific gene called CYFIP1, which helps maintain a nerve cell’s structure. The team then blocked the expression of this gene in developing mouse embryos and noticed defects in the formation of the cerebral cortex, a brain region that plays an important role in consciousness.read more