Popular Science has an article describing medical treatments for four diseases that could be available to the general public in a few years. One is an effort to reverse autism:
While studying mice, he learned that the disease allows a neuron’s mGluR5 receptor to send out a flurry of signals telling the cell to produce protein. The protein overload causes a neuron to form many more connections to other neurons than normal, creating chaos by spreading nerve instructions to too many cells. Bear’s drug, called STX107, inhibits the receptors to pare back the overproduction of proteins associated with Fragile X to a normal range. His company, Seaside Therapeutics, plans to test STX107 in patients this fall. If it works as well as it did in mice, Bear says, it could be a first step to treating other causes of autism.[...]
Fragile X neurons lack the ability to mute messages from the mGluR5 receptor, leading to an overproduction of protein. STX107 binds to the receptor, dampens its productivity, and slows protein production to a normal rate.
The other innovative treatments are for patients of Leber’s congenital amaurosis, persistent vegetative state, and brain tumors.
http://www.popsci.com/science/article/2010-02/radical-cures | Image: John MacNeill