Cancer drugs may hold key to treating Down syndrome and other brain disorders

Ann Arbor: A class of FDA-approved cancer drugs may be able to prevent problems with brain cell development associated with disorders including Down syndrome and Fragile X syndrome, researchers at the University of Michigan Life Sciences Institute have found. The researchers' proof-of-concept study using fruit fly models of brain dysfunction was published today in the journal eLife. They show that giving the leukemia drugs nilotinib or bafetinib to fly larvae with the equivalent of Fragile X prevented the wild overgrowth of neuron endings associated with the disorder. Meanwhile, the drugs—both tyrosine-kinase inhibitors—did not adversely affect the development or neuronal growth in healthy flies.

Down syndrome might help us understand Alzheimer’s disease better

TheConversation: Alzheimer’s disease is the most common form of dementia in older adults. At the moment there is no cure, but many clinicians feel that the earlier one is diagnosed, the better the possibilities are for treatment or slowing the disease. But developing treatments or prevention approaches for Alzheimer’s disease is difficult. There is no biomarker (for example a blood test) or definitive medical test for it and there is no set age where people develop memory impairments and dementia. Indeed, people can develop Alzheimer’s disease as young as 30 or well into old age. This is a real challenge for diagnosing Alzheimer’s in its early stages. But in people with Down syndrome there is an age-dependent progression of changes associated with Alzheimer’s disease. This makes it much easier to map when and what kinds of changes happen in the brain in the early stages of the disease.

Brains of people with Down syndrome age faster, UCLA study discovers

UCLA. US: A new UCLA study is the first to demonstrate that Down syndrome accelerates aging in different parts of the body. 

NIH launches tool to advance Down syndrome research

NIH. US: The National Institutes of Health has launched a subsite of DS-Connect: The Down Syndrome Registry for researchers, clinicians, and other professionals with a scientific interest in Down syndrome to access de-identified data from the registry. This Web portal will help approved professionals to plan clinical studies, recruit participants for clinical trials, and generate new research ideas using information gathered from the registry participants.