5 things to know about the power of mRNA technology

Hopkins scientists build on mRNA research to unlock a potential new way to treat cancer and other diseases.

Major scientific discoveries never happen overnight. In fact, they’re often the result of many years of research from around the world. This is the case with messenger RNA, or mRNA, technology.

Known for its use in producing a COVID-19 vaccine that helped the world manage a global outbreak, researchers have found that it can help the body fight other serious diseases. More recently, Johns Hopkins scientists discovered a way to use mRNA to potentially treat rare genetic diseases, including cancers, immune diseases, and neurodegenerative diseases like Alzheimer’s.

“As a technology, it’s become one of the most powerful in our medical arsenal that we are just beginning to unravel and discover what we can do with it,” said Jeff Coller, Bloomberg distinguished professor of RNA biology and therapeutics at Johns Hopkins University and one of the scientists involved in the research.

This discovery underscores the potential of mRNA technology to go beyond infectious diseases and into therapies that can save lives and prevent life-threatening conditions. Here are five things you should know about this groundbreaking health technology.

1. mRNA is not new 

mRNA is a genetic messenger that carries ready-made instructions telling your cells to produce specific proteins that can help your body fight infection and disease. mRNA technology is a result of research from scientists around the world. Since mRNA’s discovery in the 1960s, researchers have developed nanotechnology that delivers mRNA via lipids to cells, created specific mRNA sequences that kick your body into action against sickness, and leveraged years of vaccine use to get this groundbreaking health solution to the public.

Quick facts on the evolution of mRNA technology

• 1960s: mRNA was discovered
• 1970s: Research determined how to deliver mRNA into cells
• 1990s: Researchers explored using mRNA to get the immune system to respond to cancer cells
• 2000s: Scientists started exploring mRNA’s potential for treating infectious diseases like Zika and the flu
• 2013: The first mRNA vaccine for rabies was tested in humans
• 2020: mRNA vaccines were used to protect against COVID-19

2. Initial mRNA research focused on cancer treatment, not infectious diseases

Though mRNA technology got its big break in powering safe and effective protection against COVID-19, original research around mRNA was centered on cancer treatment.

This focus has continued in today’s mRNA research, including in a recent discovery by Hopkins scientists. By creating a genetic “tail” that attaches to mRNA molecules that churn out specific proteins, they’ve developed a potential new way to treat a variety of rare genetic diseases, including some cancers.

In these types of diseases, proteins are often missing or have a mistake. That genetic tail can extend the lifespan of mRNA to boost production of the remaining proteins.

“The benefit of mRNA in treating various genetic disorders is that it’s using the body’s natural systems,” Coller said. “By using [mRNA] to achieve a therapeutic benefit, we’re taking advantage of what normally exists.”

Read the research: Scientists Design Experimental Protein Booster for Rare Genetic Diseases

These discoveries are game-changing, and the world is starting to notice how important mRNA technology is for global health. Andy Geall, co-founder and chief development officer of Replicate BioSciences and board chair of Alliance for mRNA Medicines, has seen this enthusiasm in South America.

“They’re investing in RNA technology,” he said during a discussion about mRNA research at the Hopkins Bloomberg Center. “They have unmet needs like dengue, chikungunya, Zika, yellow fever, rabies. They need solutions, and they believe that the RNA technology will provide…a safe and effective strategy. And they’re not waiting, they’re not hesitating.”

3. mRNA technology allows for faster, more personalized treatment—and potentially disease prevention

The unique potential of mRNA tech stems from the fact that mRNA naturally exists in every cell. Delivering human-made mRNA into targeted cells that changes what they do unlocks a well of opportunity for telling your body how to fight, adapt to, and potentially prevent diseases.

Develop treatments faster. mRNA tech is easy to make and can be quickly deployed or adjusted to meet the changing health needs of an individual patient or a large population.

“This is a game-changer because in the past we were not nimble,” said Elizabeth Jaffee, deputy director of the Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins.

Treatment development that typically takes months or years can now be done in weeks with mRNA technology, she added. This can make all the difference with cancer patients in particular.

Get personalized treatment. Not only is this technology safe, effective, and efficient, but it’s also more customizable to the patient compared to other types of treatment.

In the case of cancer, mRNA research allows medical professionals to spot specific proteins in a patient’s tumor, quickly stitch them into an mRNA, and give it to them to instruct their immune system to target and fight those cancer cells.

Potentially prevent diseases. Aside from treating existing health issues, mRNA tech has the potential to prevent cancer in much the same way as hepatitis B and HPV shots prevent cancers. Jaffee said she can foresee mRNA preventing cancer progression from the earliest stages by using cancer cell genes.

“We have the ability to really change the face of cancer treatment over the next five to 10 years,” she said.

4. mRNA research largely relies on federal funding

These kinds of discoveries depend on federal funding, particularly from the National Institutes of Health. This agency is the world’s largest single public funder of biomedical and behavioral research, and most of its budget goes toward supporting these kinds of scientific breakthroughs that help tackle our biggest public health issues.

Other federal investments over 10 years ago paved the way for mRNA technology, too. The Defense Advanced Research Projects Agency (DARPA) funded Geall’s mRNA research in 2011 and 2012, which pushed the tech forward and brought in investments from pharmaceutical companies and banks. This helped mRNA technology mature so that by the time the COVID pandemic happened, it was ready to be used to save lives around the world.

“Federal funding is really a social contract between the American public and scientists, such as myself,” Coller said. “Research really does save lives, and it saves lives based on the American taxpayer and their investment into us.”

5. Two mRNA studies in clinical trials for cancer treatment look promising

As research continues to fuel advances and possibilities for this exciting technology, there are two studies already putting it to real-life use against cancer. Currently in clinical trials, they’re using mRNA to treat melanoma, which causes a large majority of skin cancer deaths in the U.S., and pancreatic cancer, which affects 1 in 56 in men and about 1 in 60 in women, according to the American Cancer Society. Both have a high chance of coming back. So far, the studies show promising results in potentially keeping these cancers from returning in patients.