Other Aging News for 2017
May 13, 2017; A child in Europe has become the second individual ever to receive a commercial gene therapy, according to GlaxoSmithKline.
The treatment, called Strimvelis, can provide an outright cure for a rare inherited immune deficiency by revising a patient's genetic makeup (see "Gene Therapy's First Out-and-Out Cure Is Here").
Gene therapy has been widely explored in experimental medical studies, but its commercial potential is largely untested. Prior to now, only a single other individual, also from Europe, had ever accessed gene therapy to treat an inherited ailment outside of a clinical trial. That individual received a different drug, Glybera, in 2015.
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March 26, 2017; More pressure to include the aging processes as a disease from a number of important scientists. This time Sweden is the focus for the push to have aging included in their disease classification with an article in the press by biologist and activist Victor Bjoerk.
Now you may agree or disagree that the various processes of aging are diseases per se, however, from a research point of view classifying them within the international classification for diesease (ICD) at the World Health Organization will allow researchers to treat age-related diseases far more directly. So either way if you think we should find effective cures for cancer, Alzheimer’s, Parkinson’s and so on then the rest is largely a matter of pedantics. The important thing is making it easier for researchers to do what they need to do and find ways to deal with diseases that have plagued us for centuries.
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January 30, 2017; A growing body of research has traced the roots of Alzheimer’s disease to the deposition of a protein called tau throughout the brain. To date, however, targeting the rogue protein to treat dementia has produced mixed results, with a promising anti-tau compound recently failing in a late-stage clinical trial. Yet a new study hints that a twist on this established strategy could yield better results. The findings, reported yesterday in Science Translational Medicine, suggest compounds called antisense oligonucleotides, or ASOs, may not only reduce levels of existing tau but also prevent its formation.
ASOs are modified DNA-like molecules designed to recognize and cleave specific RNA sequences thereby preventing them from directing production of a certain protein. The class of drugs is showing promise in treating an array of conditions, including a number of brain disorders: one ASO was approved last year for treating spinal muscular atrophy, a devastating disorder of infancy; others are under investigation for ALS and Huntington’s disease.
The authors of the new study found ASOs prevented cognitive problems in a mouse model of tau brain disorders, or tauopathies, which include not only Alzheimer’s but also rarer brain disorders such as progressive supranuclear palsy and corticobasal ganglionic degeneration. The study also found treatment with ASOs decreased brain inflammation (thought to contribute to Alzheimer's), prevented neuronal death and even reduced tau aggregates already present in the brain. Advancing a step closer to relevance in humans, the researchers further demonstrated tau-targeted ASO treatment reduces tau protein levels in monkeys. "There are lots of tau-based treatments being looked at," says lead study author Sarah DeVos, then a graduate student at Washington University School of Medicine in Saint Louis. "But this is the first drug that we’re aware of that actually reduces the expression of the protein–that prevents it from even being made."
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