So, yesterday I talked about how we need to shrink the unknown unknowns – and the known unknowns as much as possible.   And, that was in the context of how we’ve learned more and more about SARS-CoV-2 and living with the pandemic.

So, I’ve decided to go back to the beginning.  Of sorts.  After all, the savior of the pandemic is working out to be a most innovative vaccine concept- using messenger RNA (mRNA), instead of attenuated or dead viruses to help us develop immunity against the disease.

Well, consider this. It was 60 years ago when we first learned of mRNA- right about the time we were discovering the genetic code. Back in the Summer of 61.  (Yes, I was a fan of Frankie Avalon back then, too.)

Believe it or not, as important as mRNA is, no one was ever awarded the Nobel Prize for its discovery or describing its functionality.  (I could blame that on the rule of 3s- it’s considered to be undesirable to award the Nobel to more than 3 folks- and there were a ton of folks who taught us about mRNA.)

Yes, James Watson was involved (and the senior author of one of the three seminal papers describing mRNA).   Along with Drs. F Gros and H Hiatt (both from Institut Pasteur), plus W Gilbert, CG Kurland, and RW Risebrough (along with Watson, all from Harvard).  The second paper- Drs. S Brenner (Cavendish Labs, UK) , F Jacob (Institut Pasteur), and M. Meselson (CalTech). And, the third paper emanated from Drs. Jacob and J Monod (both Institut Pasteur).

An amazing group of 10 folks who provided the discovery and initial theory of how mRNA is critical for cellular activity and gene regulation- how the genetic information of DNA is used to produce cellular proteins. A process that took almost 20 years to get it right.  (Talk about having to change the story as we learn more…)

Then, there’s the outstanding work of Drs. Katalin Kariko and Drew Weissman at the University of Pennsylvania, who found a way to modify mRNA, so our immune system doesn’t destroy them- switching out uridine for pseudouridine.  This technology has been licensed by both BioNTech and Moderna.  (Karako is now- along with her spouse- the head of BioNTech.)

Given this advance, we are at the stage where we can use mRNA to create treatments for a slew of diseases and viruses.  After all, if viruses can commandeer the cell’s protein factories for nefarious purposes, why can we do the same for positive results.  Like using mRNA to inform our cells to produce proteins that recognize viruses- and attack them.  But more than that- why not go after cancer and genetic disorders?

Which is exactly what Moderna and BioNTech are doing.  BioNTech maintains its focus on cancers and the flu.  Moderna is aiming for autoimmune disorders and viral diseases (Epstein Barr, RSV [respiratory synclinal virus], metapneumovirus).  And, both big and small drug firms are entering this arena.

mRNA is going to be the next big tool for pharma.

 

 

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