The COVID-19 vaccines from Moderna and Pfizer leverage a unique delivery system that, until now, has not been successfully used on a large scale. Called messenger RNA, or simply mRNA, the technology can cause a body’s cells to create unique proteins that can be tailored to individual diseases and conditions such as different forms of cancer, heart failure, and cystic fibrosis.
Let’s look more at mRNA, how it may be used in the future, and what roadblocks to adoption exist.
mRNA and the COVID-19 Vaccines
Scientists can alter the sequence of mRNA to deliver different results, theoretically changing that sequence to encourage the body to perform different tasks. For the Moderna and Pfizer vaccines, the sequence created prompts the cells in a person’s body to make a SARS-CoV-2 protein that would train the body to recognize the virus and attack it. For a vaccine, the mRNA needs to be injected into a person for this positive effect to take place. Note: The Johnson & Johnson vaccine does not use mRNA.
mRNA is being studied in clinical trials to deliver vaccines against diseases such as rabies, Zika, cytomegalovirus, influenza, and more, but the COVID-19 vaccines are easily the largest implementation to date. A targeted local mRNA injection—into a specific muscle or a tumor—is also being studied in clinical trials.
The Potential for mRNA in Chronic Diseases
The early success of mRNA with the COVID-19 vaccines has excited the research community, who believes the delivery system could be a transformational technology in the treatment of diseases. Our body’s cells use mRNA to translate the genes of DNA into dynamic proteins that essentially affect everything our body does. Pharmaceutical companies currently make many of these proteins synthetically as drugs. mRNA would cause our bodies to create them naturally, turning our bodies into natural drug factories.
mRNA could theoretically be used to treat a host of other diseases, both as a vaccine or as a treatment for chronic conditions. For example, mRNA could help a person’s body create proteins that stimulate blood-vessel growth to battle heart disease, while another could encode a missing enzyme to reverse an inherited genetic disease. Researchers continue to look for potential use cases and how they can tweak both mRNA and lipid nanoparticles, the protective bubble commonly used to help ferry mRNA through the body.
Significant Challenges Ahead
While the concept of mRNA appears straightforward, especially with vaccines, the creation and application of new therapies still require significant study. “It’s not like you just put in another sequence and it will treat anything,” said Heleen Dewitte, a pharmaceutical scientist at Ghent University in Belgium.
Until the current pandemic, only a handful of mRNA treatments reached clinical trials—the last, and most important step of a medical treatment being approved for public use. Moderna and Pfizer were able to bypass most of this step due to the severity of the pandemic. The real-world viability of these vaccines has given researchers hope for future use, but each specific disease will require unique mRNA coding and, perhaps, a more targeted delivery system to a specific organ in the body.
There are also possible long-term challenges for chronic conditions. Vaccines, and some other preventative treatments, may only require one or two doses as the proteins created can outlast the disease. For chronic conditions, a person will likely need recurring treatments that could cause serious side effects, especially from a buildup of lipid nanoparticles over time. While a person may not mind feeling sick for a day after a one-time vaccine, those side effects may be difficult for someone who receives a treatment every few weeks.
Researchers today continue to find ways to make mRNA appear as natural as possible to the body while experimenting with different lipid nanoparticles that could be better for long-term use. While there is still a long road ahead, the successful development of more mRNA treatments could be transformative for medical treatment.
Treatments for diseases like cystic fibrosis and ornithine transcarbamylase deficiency (otherwise known as OTC and the result of a missing enzyme in the liver) are already underway, among others. Researchers believe it may be a decade until mRNA can be more widely used, but once fully proven, it could revolutionize the treatment of chronic conditions and cancer.
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