An Indian researcher and her team at the University of Chicago are working to help the world understand degenerative neurological diseases such as Alzheimer’s better and help find a cure for them.

“Till date, every single trial for developing a potential cure for Alzheimer’s has failed. The reason probably is that we are considering Alzheimer’s as a single kind of disease. Which it may not be,” said Yamuna Krishnan, professor of chemistry at the University of Chicago.

Complex chemistry

Researchers led by Krishnan, who won the 2017 Infosys Prize for physical sciences, have perfected a technology which can help measure chemicals in what the scientists call organelles, which are sub-structures inside the cells which have very specific functions to perform.

“One of the organelles we are very interested in is lysosome, which is essentially the stomach of the cell. Lysosomes hold around 50 enzymes which are capable of breaking down different types biological materials such as proteins, lipids and carbohydrates,” Krishnan, who had earlier worked with the Bengaluru-based National Centre for Biological Sciences, said.

“When any of these enzymes is defective, there would be a storage disorder. That is, the system cannot break down products that are associated with that particular class of enzymes, leading to the accumulation of the material,” said the professor, who is on a visit to India.

There are nearly 50-60 kinds of lysosomal storage disorders. Collectively, they affect at least 1 in 5,000 new-born babies globally, leading to neurological disorders. Such children develop speech defects, balance problems or tremors. The mortality rate is almost 100 per cent.

What Krishnan and her colleagues did was to apply the technique they developed to one such disorder called Niemann-Pick disease, which scientists initially thought of as one disease but later uncovered three distinct sub-types. “We showed that with skin biopsy, we could accurately sub-classify the disease. In other words, we can identify different signatures associated with each sub-class of Niemann-Pick disease.

Their findings, which were reported in three publications in recent months, the latest being in February in the journal Nature Nanotechnology , had a profound impact and the US Food and Drugs Administration approached the team to be part of a trial being carried out on Niemann-Pick disease.

“We have been working on this technique (which uses short sequences of DNA) for a while and the technology has matured significantly. We can now apply it directly to the living cells that you can get from human beings – such as skin biopsies or circulating cells isolated from the blood,” Krishnan told BusinessLine.

Company launched

“If we can classify Alzheimer’s into different sub-types, then one can start looking at having specific drugs for specific sub-types. So, the first step is to sub-type the disease and this is what our company is trying to do,” she said.

Recently, Krishnan founded a company called Esya , which in Sanskrit means probing or examining something or someone. “We have raised a quarter million dollar already through family and friends round. We hope to raise $4 million,” Krishnan, who won India’s coveted science prize SS Bhatnagar Prize in 2013, said. ‘It is said that raising the first million is the most difficult for a new firm,” she remarked.

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