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.
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.
“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.
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.
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.
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.
“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.
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.
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.
Source : Bellevision , 27th October 2013
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.
“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.
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.
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.
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.
“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.
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.
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.
Source : Bellevision , 27th October 2013
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