Nobel for microRNA discovery promising for new therapeutics, say Indian scientists

Beena Pillai, chief scientist at CSIR-Institute of Genomics and Integrative Biology in New Delhi, said the discovery of microRNA was purely a result of curiousity-driven research.

“This discovery was made in a tiny worm, and at the time, the impact it would have in years to come was not so obvious. Now we know that this is a wonderful way of tuning the expression of genes, in worms and humans alike,” Pillai said.

“When not expressed properly, it can lead to cancer, neurological diseases, and many other issues. There is also excitement around their utility as biomarkers for certain diseases,” Pillai said.

Two Americans Victor Ambros and Gary Ruvkun were awarded the Nobel Prize in Physiology and Medicine for their 1993 discovery of microRNA, a new form of RNA molecules that were found to control whether the characteristics of a gene in an organism were allowed to be expressed.

“MicroRNAs are tiny compared to other RNA molecules, so understanding their function when they were first found was a challenge. Ambros and Ruvkun were young researchers who pursued this problem independently but consulted each other and shared their findings. After their initial discovery of microRNAs it took almost a decade for the full impact to become clear when hundreds of other such RNAs were discovered. It shows us how important discoveries may not be immediately obvious. This particular one has opened new windows for therapeutics in future,” Samir Brahmachari, former DG, CSIR, told The Indian Express.

Sanjay Singh, CEO of Gennova Biopharmaceuticals, which has been at the forefront of developing India’s first mRNA vaccine for Covid-19, said this year’s Prize showed the growing recognition of the potential of gene-based health interventions.

“Last year, the Nobel Prize in medicine was given for the work that had made mRNA vaccines possible, including the ones for Covid-19. This year, the Nobel Prize is for microRNA. The understanding of microRNA functioning will open a new field of genetic regulation-based discoveries, primarily in healthcare and also in agriculture,” Singh said.

“In the future, microRNAs (miRNA) hold promise for personalised medicine, cancer therapy, regenerative medicine and neurological treatments. miRNA-based therapeutics and non-invasive diagnostics tools such as liquid biopsies are emerging. Challenges remain particularly with efficient delivery, off-target effects, and maintaining stability,” he said.

Former director of IISc G Padmanabhan said the Nobel Committee had made a very appropriate choice. “Medically, the discovery of microRNA is very important as gene-regulation and control is a fundamental process in the cell that will prevent certain proteins from being formed qualitatively and quantitatively. Its role in therapeutics is significant,” Padmanabhan said.

Noted scientist Gagandeep Kang said that diseases that occur because abnormal levels of proteins are important in all organ systems and all organisms.

“Being able to control gene regulation and therefore protein production is one critical approach of dealing with diseases that we derive from this Nobel winning discovery. In India there are scientists that work in this area but linking to industry R&D will be critical for making products with clinical utility. It is important to note that there is a long lag from proof of concept to actually having treatments and very careful clinical testing and evaluation for regulatory submission is essential,” Kang said.