Scientists at the Scintillon Institute, University of California, San Diego School of Medicine, and Sanford Burnham Prebys Medical Discovery Institute are combining their expertise to test the effects of a new autism drug, NitroSynapsin. Spearheaded by Dr. Stuart Lipton, NitroSynapsin, an aminoadamantane nitrate compound, intends to “restore an electrical signaling imbalance in the brain found in virtually all forms of autism spectrum disorder (ASD).”
In 1993, Lipton and his team discovered MEF2C, a gene found in the brain that, when disrupted, could potentially lead to autism-like tendencies. Using mice as their experimental group, Lipton and his colleagues intentionally provoked the MEF2C portion of their brain to see if their findings were conclusive. As suspected, all cases of MEF2C disturbances created what has now been coined MEF2C Haploinsufficiency Syndrome (MHS). While MHS is only found in a small portion of autism disorders, recent studies suggest “mutations underlying various autism disorders frequently involve genes whose activity is switched on by MEF2C.” Since Dr. Lipton was able to draw parallels between MHS and ASD, the present NitroSynapsin studies were conducted on mice with MHS.
Much like Lipton’s preliminary research, the NitroSynapsin research used mice that were specifically engineered to have MEF2C deficiencies. As a result, the mice displayed behavioral impairments including abnormal anxiety, abnormal repetitive movements, memory gaps and other symptoms similar to human MHS tendencies. Moreover, the mice exhibited an excitatory/inhibitory (E/I) imbalance, which is common among ASD. When the researchers were satisfied with the cognitive and behavioral state of the mice, NitroSynapsin was put to the test.
For three months, the MHS mice were given doses of NitroSynapsin. Researchers found that the treatment reduced E/I imbalance, lessened abnormalities, and improved the cognitive performance of the mice. In fact, some cases restored “performance essentially to normal.” While these findings show promise, Lipton’s team must first perform research on mice with other autism disorders before graduating to clinical trials.
Lipton’s studies have gained positive traction from parents of children with MHS. Among his most fond admirers is Michelle Dunlavy, mother to a boy with MHS, who states that “we are all hanging on to the hope that one day our children will be able to speak, to understand and to live more independent lives.” In an effort to show her gratitude, Michelle, along with other parents, have created a Facebook group in support of Lipton and his research. Their goal is to bring awareness to the cause and facilitate his efforts in moving forward.
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