Written: April 2022
About five years ago I attended a Research Day program at Danbury Hospital in Connecticut. The guest speaker gave an enthusiastic view of the potential of mRNA (messenger ribonucleic acid). I would say I understood maybe 10% of what was presented. The person sitting next to me was a doctor in his seventies. He told me when he was in medical school he never heard of mRNA. Although it took years for mRNA to make into the limelight, scientists have been studying it for decades, since roughly 1960.
Over the past couple of years, mRNA technology has made waves with the success of the COVID-19 vaccines by Moderna and Pfizer-BioNTech. mRNA vaccines are just the start of what will be possible. First, a little bit about what mRNA is and what it does.
At the highest level, it is all about proteins, the naturally occurring, extremely complex, substance which consists of amino acid residues joined by peptide bonds. Proteins are present in all living organisms and include many essential biological compounds such as enzymes, hormones, and antibodies. We are made of proteins, and they are essential ingredients to our human lives. Our bodies contain trillions of cells, the basic units of life, and every cell contains proteins. The role of mRNA is to teach the body how to make specific proteins which can help our immune system to fight off certain diseases such as Covid-19. Proteins are the “workhorses” of our cells, and our body makes more than 100,000 different ones.
A common example of a protein which plays a key role is insulin, a protein which helps the body control sugar levels in the blood. The prevalence is significant. In 2019, 37.3 million Americans, 11.3% of the population, had diabetes.[i] Type 1 diabetes is thought to be caused by an autoimmune reaction where the body attacks itself by mistake and stops the body from making insulin. When the body doesn’t make the right amount or type of protein, it can cause serious medical conditions. The bottom line is proteins are essential for maintaining health and preventing disease.
Our cells are basically protein factories. The proteins are made in a process called protein synthesis, and that is where mRNA steps in with a key role. As the name implies mRNA is a messenger. The mRNA works with other components in our cells to make the proteins we need. Scientists have learned how to design mRNA which causes the body to in effect make its own medicine. Healthcare providers deliver the medicine through an infusion or an injection in the arm. After the medicine is absorbed, our body proceeds to make the protein following the mRNA’s instructions.
For Covid-19, scientists designed a specific mRNA for vaccines to carry instructions to make the spike protein which will trigger an immune response inside our bodies. Contrary to some misinformation, mRNA doesn’t stay in our body very long after completing its job. It does not cause any permanent changes.
The potential of mRNA goes far beyond vaccines. It can help grow new blood vessels after a heart attack or fight off an aggressive cancer. Scientists at the University of Pennsylvania have developed an experimental mRNA therapy which shows promise for treating heart disease.[ii]
The Penn team designed mRNAs that could turn regular T cells into therapeutic CAR-T cells which can target heart fibrosis. Patients who have cardiac disease can get a scarring which impairs heart function. In a mouse model of heart failure, an injection of the mRNA therapy removed scarring cells which in turn resulted in a significant reduction of fibrosis and improvement in heart function.
My reflections – 2022
The research at the University of Pennsylvania is impressive and shows a lot of potential for new forms of heart disease treatment. By combining mRNA and the successful blood cancer treatment called CAR-T, the researchers have been able to re-engineer heart cells to eliminate fibrosis.
Heart disease remains the #1 cause of death. mRNA research may result in a significant reduction in mortality. I believe we will be hearing much more about medical breakthroughs made possible by a deeper understanding of the magic of mRNA.
[i] “Statistics About Diabetes,” American Diabetes Association (2022), https://www.diabetes.org/about-us/statistics/about-diabetes
[ii] “Technology Used in Mrna Covid Vaccines Offers Hope for Treatment of Millions with Heart Disease, Study Suggests,” Penn Center for Innovation (2022), https://pci.upenn.edu/technology-used-in-mrna-covid-vaccines-offers-hope-for-treatment-of-millions-with-heart-disease-study-suggests/