Thursday, July 5, 2012

Scientists successfully treat autism, in mice

Autism spectrum disorders cover a wide array of brain malfunctions, that predominantly lead to impaired social skills, problems with communication, and, in severe cases, cognitive impairment. A proper treatment for autism does not exist, and patients often need special attention and care. Because scientists are constantly improving their knowledge of the brain, new treatment options have become available over the last couple of years. Some of them are currently under investigation, and a scientific group from the University of Cincinnati found a way to treat autism by reversing the cognitive impairment. So far, they have seen success in a mouse model.

The scientists used mice suffering from a condition that is similar to a form of cognitive impairment found in humans. Therefore, the mouse disease mimics the human condition and can be used as a model to test new treatments. Formally, the disorder is caused by something called creatine transporter deficiency (CTD), which basically means a certain protein in the brain stopped working correctly. This causes cognitive impairment, both in mouse and human.

In order to treat the condition, the scientists had to come up with a way to repair the deficiency, and they tried to do so by using a drug called CincY, which is an analogue of creatine. As said, the creatine transporter is impaired in patients with the aforementioned autism disorder, and because it is needed to allow creatine to do its job, supplying a medicinal analogue is a logical step.

A treatment schedule of about nine weeks was sufficient to revert the cognitive damage caused by CTD. Equally important, there were no observed adverse effects, indicating that the treatment is safe. One of the scientists that lead the conduct of the study said: "Treated mice exhibited a profound improvement in cognitive abilities, including recognition of novel objects, spatial learning and memory." This indicates that replacing creatine, generally needed for brain processes, helps restore brain function on a functional level.

Even though this study only focused on mice, CincY is already in the process of being approved for use in humans. If this treatment also works for human patients, it will certainly not cure all people suffering from disorders in the autism spectrum. At least it will help some of them, and it shows that brain deficiencies can be repaired, even though diseases such as autism were regarded as something psychological not long ago, as opposed to being fundamentally biological. Lately, we have found more biological components to be involved with causing autism, including various genetic factors.
There are many parts of the brain that can be affected by autism, meaning that one drug won't help all patients, and not all autism patients have the same underlying pathology.

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