When the final Phase 3 data came out last November showing the mRNA vaccines made by Pfizer/BioNTech and Moderna were more than 90% effective, Dr. Anthony Fauci had no words. He texted smiley face emojis to a journalist seeking his reaction.
This astonishing efficacy has held up in real-world studies in the US, Israel and elsewhere. The mRNA technology – developed for its speed and flexibility as opposed to expectations it would provide strong protection against an infectious disease – has pleased and astonished even those who already advocated for it.
The messenger RNA, or mRNA, platform may be new to the global public, but it’s a technology that researchers had been betting on for decades. Now those bets are paying off, and not just by turning back a pandemic that killed millions in just a year.
This approach that led to remarkably safe and effective vaccines against a new virus is also showing promise against old enemies such as HIV, and infections that threaten babies and young children, such as respiratory syncytial virus (RSV) and metapneumovirus. It’s being tested as a treatment for cancers, including melanoma and brain tumors. It might offer a new way to treat autoimmune diseases. And it’s also being checked out as a possible alternative to gene therapy for intractable conditions such as sickle cell disease.
Vaccines
The story of mRNA vaccines dates back to the early 1990s, when Hungarian-born researcher Katalin Kariko of the University of Pennsylvania started testing mRNA technology as a form of gene therapy. The idea is similar whether scientists want to use the mRNA molecule to cure disease or prevent it; send instructions to the cells of the body to make something specific.