IIT-H researchers find new molecule to treat ALS

There is a development worth mentioning at Indian Institute of Technology Hyderabad (IIT-H). The researchers there have found a new molecule ‘AIM4’ that can be potentially used to treat a rare and debilitating neurodegenerative disease called ‘Amyotrophic Lateral Sclerosis’ (ALS), also known as ‘Lou Gehrig’s disease.’

ALS is a neurodegenerative disorder that severely affects voluntary movement of muscles and can lead to paralysis and death. It occurs due to changes in specific genes which may be triggered by factors such as smoking, exposure to toxins, metals and pesticides.

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IIT-H

Sources from IIT-H say the research work was done by a team under the leadership of Dr. Basant Kumar Patel, Dr. Sandeep Singh and Dr. Rajakumara Eerappa, faculty from the Department of Biotechnology, IIT-H and Professor Ganesan Prabushankar, Department of Chemistry, IIT Hyderabad, has recently been published in the reputed peer-reviewed International Journal of Biological Macromolecules.

It is interesting to note that there are no drugs available at present to cure ALS and treatment options are limited to two drugs only for management of the condition.

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The ‘ice bucket challenge,’ which went viral in the U.S. and the U.K. in 2014, created awareness about this disease among people. The disease is relatively unknown in India although Indians seem to have an earlier onset of this malady compared to people of the West and show longer lasting symptoms and disease progression.

Research is ongoing throughout the world to find better drugs that can arrest this disorder and not merely manage the symptoms. Such research must first identify the causes for the onset of ALS.

Basant Kumar said One of the causes of ALS is the alterations in the genes that code for a critical protein called TDP-43. The gene alteration modifies the protein which results in its liquid-liquid phase separation. This phase separation in turn causes the proteins to be deposited on nerve cells, resulting in generalization.

‘We have shown that AIM4 prevents liquid-liquid phase separation of the modified protein, TDP-43-A315T. By this, AIM4 prevents aggregation of the protein and may potentially prevent the deposition on neurons.’

The research group compared the ability of AIM4 to prevent protein phase separation with that of other molecules such as Dimebon. Dimebon is an antihistamine that was studied for treatment of regenerative disorders by an American pharmaceutical company but failed clinical trials. The IIT Hyderabad Research team found that AIM4 was better than such earlier molecules in preventing protein phase separation.

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The research team has also discovered through computational studies that AIM4 has specific binding site on this protein and binding energy calculations have shown that the bond between AIM4 and the mutant protein is energetically favorable, which makes it a promising drug for potential treatment of ALS.

This work is an important step forward in the area of therapeutics of the regenerative disease ALS as current options for ALS are minimal or even non-existent, he said.