Penn researchers are developing new ways to use mRNA technology to treat a range of diseases, including celiac disease and cancer.
mRNA research involves introducing molecules to human cells to instruct them on how to fight illness and treat medical conditions. It is a widely used technique, involved in treatments for various cancers, HIV, COVID-19, influenza, and more.
Research assistant professor of Medicine Jilian Melamed is focused on researching mRNA as a means of combating celiac disease, type one diabetes, multiple sclerosis, and other diseases.
If a celiac patient consumes gluten, an inflammatory response is triggered in the immune system and antibodies are released, which can lead to vomiting, diarrhea, and stomach pain.
“Our vision for how we would use mRNA therapeutics to treat these diseases is to develop antigen-specific, tolerizing therapies,” Melamed told The Daily Pennsylvanian.
Tolerizing refers to the process of training the human body to tolerate a substance it is reacting to or being exposed to. In the case of Melamed’s research, this substance is gluten.
In addition to celiac diasese, mRNA therapeutics are making new strides in cancer immunotherapy, and working towards the potential development of a cancer vaccine.
Bioengineering Professor Michael Mitchell is using lipid nanoparticles to facilitate the delivery of mRNA to directly target specific cells. As a result, these new mRNA treatments are uniquely able to adapt to combat different types of diseases.
“We could train the immune system from inside the body to now recognize tumors as foreign,” Mitchell said, adding that “not only can we melt away the tumors in mice, but when we rechallenge the mice with another tumor the immune system remembers, it has memory, and can then eradicate the tumor.”
Both Melamed and Mitchell believe mRNA has wide potential and implications for the medical and pharmaceutical industry.
“We’ve seen how effective mRNA lipid nanoparticles can be. We have systems in place to produce them at scale, and they’re actually less expensive to produce than protein therapeutics" Melamed said.
Previously, mRNA played a large role in innovating the COVID-19 vaccine, making the process of developing the vaccine much quicker and more efficient.
In an interview with Penn Medicine News, Roberts Family Professor in Vaccine Research Drew Weissman explained that especially due to the “plug-and-play” nature of the mRNA platform, researchers “could quickly edit the formula to teach the immune system to recognize different spike proteins in the evolving SARS-CoV-2 virus.”
Mitchell added that “what’s powerful about mRNA technology is all we have to do now is simply go in and alter the sequence of mRNA, and it can alter from a vaccine to a drug.”
He predicts that both personalized cancer vaccines, where a custom-designed vaccine is created based on individual sequencing of a patient’s tumor, as well as other types of immunotherapy for cancer, will emerge within the next five to ten years.
However, recently announced federal funding cuts are drawing concerns from researchers. In an email announcement to the Penn community, Interim President Larry Jameson mentioned “developing vaccines with mRNA technology” as a example of research funded by the National Institute of Health.
“We’re all worried about it. We don’t know what is actually going to happen in the future,” Melamed said.
In particular, she pointed to indirect costs, which are required to maintain core research facilities, equipment, and instruments as a primary concern.
“It’s the lack of funding that will be available to core facilities that’s most alarming about the reduction in indirect costs that go to the university,” Melamed explained. “That funding also supports administrative offices and grant offices.”
“There’s a lot going on in the world right now, but this type of science is really going to save lives in the future — not just for vaccines, but for other diseases as well,” Mitchell said.
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