<p>Scientists have found out a new drug target against one of the commonest types of autism, opening up a window for developing a medicine for a disorder, incurable at the moment.<br /><br /></p>.<p>The target is specific to Fragile X Syndrome – the commonest inherited form of mental retardation and autism – was discovered in a laboratory experiment and required to be taken up by pharmaceutical industry before any medicine is available commercially.<br /><br />“But the new drug, as and when it will be available, would not have any side-effects unlike two other molecules, which are currently being tested by two pharmaceutical majors,” said Sumantra Chattarji, a senior scientist at National Centre for Biological Sciences, Bangalore, and one of the team members. India has an estimated 10 million autism patients and the numbers are on the rise.<br /><br />Chattarji and his colleagues at the Institute for Stem Cell Biology and Regenerative Medicine, Bangalore and several universities in the US studied the Fragile-X syndrome at a deep fundamental level to come out with the new drug target.<br /><br />As the disease depends on the interplay of two crucial proteins inside the brain, scientists manipulated secretion of the two proteins to check if the disease can be cured in mice.<br /><br />Fragile X Syndrome is caused by the absence of a regulator protein, named FMRP, leading to uncontrolled release of other proteins which adversely impact the communication between two nerve cells. As a counter-strategy, the scientists knocked out another regulator gene named CPEB1 that produces those proteins in the first place.<br /><br />“It’s akin to set a thief to catch a thief,” Chattarji told Deccan Herald. In the absence of the CPEB1, those proteins causing adverse effects were not generated, leading to restoration of biochemical balance in brain cells. As a result, physiological functions improve. The proof of the concept was reported on October 20 issue of Nature Medicine.<br /><br />For drug development, scientists will now have to design a molecule to block the functions of CPEB1 as Fragile X patients lack FMRP gene leading to uncontrolled release of harmful proteins.<br /><br />This, Chattarji said, would be a “paradigm shift” in treatment strategies. In the absence of a cure, doctors currently manage autistic patients with symptomatic treatment. But two major pharmaceutical companies are testing a drug, which is now in the clinical trial phase.</p>
<p>Scientists have found out a new drug target against one of the commonest types of autism, opening up a window for developing a medicine for a disorder, incurable at the moment.<br /><br /></p>.<p>The target is specific to Fragile X Syndrome – the commonest inherited form of mental retardation and autism – was discovered in a laboratory experiment and required to be taken up by pharmaceutical industry before any medicine is available commercially.<br /><br />“But the new drug, as and when it will be available, would not have any side-effects unlike two other molecules, which are currently being tested by two pharmaceutical majors,” said Sumantra Chattarji, a senior scientist at National Centre for Biological Sciences, Bangalore, and one of the team members. India has an estimated 10 million autism patients and the numbers are on the rise.<br /><br />Chattarji and his colleagues at the Institute for Stem Cell Biology and Regenerative Medicine, Bangalore and several universities in the US studied the Fragile-X syndrome at a deep fundamental level to come out with the new drug target.<br /><br />As the disease depends on the interplay of two crucial proteins inside the brain, scientists manipulated secretion of the two proteins to check if the disease can be cured in mice.<br /><br />Fragile X Syndrome is caused by the absence of a regulator protein, named FMRP, leading to uncontrolled release of other proteins which adversely impact the communication between two nerve cells. As a counter-strategy, the scientists knocked out another regulator gene named CPEB1 that produces those proteins in the first place.<br /><br />“It’s akin to set a thief to catch a thief,” Chattarji told Deccan Herald. In the absence of the CPEB1, those proteins causing adverse effects were not generated, leading to restoration of biochemical balance in brain cells. As a result, physiological functions improve. The proof of the concept was reported on October 20 issue of Nature Medicine.<br /><br />For drug development, scientists will now have to design a molecule to block the functions of CPEB1 as Fragile X patients lack FMRP gene leading to uncontrolled release of harmful proteins.<br /><br />This, Chattarji said, would be a “paradigm shift” in treatment strategies. In the absence of a cure, doctors currently manage autistic patients with symptomatic treatment. But two major pharmaceutical companies are testing a drug, which is now in the clinical trial phase.</p>
