What’s the Next Frontier in Medicine?
These last couple of years, mRNA therapies have been a long-running great medical development, along with super-fast COVID-19 vaccines. These vaccines already demonstrated what mRNA can do, but infectious disease is not the only thing mRNA can treat. Now the challenge for researchers and biopharmaceutical companies is to make mRNA therapeutics for everything from cancer to genetic diseases to autoimmune diseases.
Here in this blog, I write about the science behind mRNA therapeutics, what they’re being used for, where they’re going, and what a bright future it will be for medicine.
What is mRNA?
mRNA: messenger RNA (mRNA) is a genetic code as a protein blueprint. It’s made in the nucleus and delivers DNA from the DNA into the ribosomes, the building blocks that make proteins in cells. mRNA is itself a kind of gatekeeper: it’s a transcription engine that takes DNA’s instructions to create proteins for other cells.
And treatments: we can program mRNA to encode single proteins to combat illness. Once put into the body, this mRNA instructs cells to create prophylactic proteins—one form of personal, individual medicine.
mRNA Vaccines: A Killer Insect New!
Vaccines are perhaps the most famous use of mRNA. Pfizer-BioNTech and Moderna COVID-19 vaccines—each uses mRNA to tell cells to manufacture the spike protein of the virus. SARS-CoV-2 is a novel new type of vaccine. Where conventional vaccines are made from dead viruses or virus proteins, mRNA vaccines release synthetic mRNA to trigger an immune response.
It’s mRNA vaccines for mRNA vaccines with the COVID-19 mRNA vaccines and mRNA vaccines forever. They’re designing mRNA vaccines for flu, Zika, HIV, and even cancer. This pace of mRNA vaccine production during COVID-19 revealed just how adaptable and scalable this technology is and how it can reshape the design and delivery of vaccines.
mRNA’s Future for Cancer Treatment?
mRNA treatment is the next frontier: cancer. And chemotherapy and radiation, the old-school cures for cancer, which have very serious side effects because they’re not targeted. Instead, mRNA treatments are highly specific drugs that prime the immune system to attack cancer cells.
The mRNA could be harnessed to make tumor antigens for cancer immunotherapy that tell the immune system to attack the cancer cells. Such a therapy could be personalized for each patient, using an mRNA vaccine against cancer cell mutations. Even early clinical trials are a success, as mRNA vaccines launch immune responses against cancer cells.
mRNA in Genetic Disorders
The second new use of mRNA is to cure genetic pathology. Deletion of copies of genes coding for the proteins we need explains genetic disorders like cystic fibrosis, muscular dystrophy, and hemophilia. mRNA therapy tries to implant living versions of these genes into the patient’s own cells.
The synthetic mRNA produced in these treatments replaces the missing or damaged protein, “resolving” the genetic disease “for a while.” If in traditional gene therapy you modified DNA in a patient’s cells, in mRNA therapy you just add artificial mRNA (a less lethal, less successful approach).
If in cystic fibrosis, for example, the CFTR gene was absent and a bad protein was made, mRNA could be translated into instructions to cells to make a functional CFTR protein. It would be a lifesaving enhancement for genetically damaged patients—and maybe the cure will not last.
mRNA-Based Therapeutics for Autoimmune Diseases
Autoimmune disorders result from the immune system attacking your own cells and tissues. Cure diseases such as rheumatoid arthritis, lupus, and multiple sclerosis are just a few diseases for which the immune system doesn’t make sense. It is the standard treatment for autoimmune conditions: suppression of the immune system, and infection is part of that process.
Instead, mRNA therapeutics could be used to develop drugs that tune the immune system increasingly less and less. The mRNA might be reprogrammed, for instance, to produce proteins to regulate the immune system or to repair damaged tissue. And they’re flirting with mRNA vaccines to introduce the antigen to the body and stave off autoimmune response.
The Overcoming of mRNA Delivery… Step By Step!
There are literally hundreds of possible mRNA therapies, but they still have a lot of work to do before they’re routine therapies. This last problem is the biggest, invading cells with mRNA.
mRNA molecules are big and thick, and they get rapidly eaten up by the immune system of the body or blood-borne enzymes. mRNA must fix it and produce it into the cells, and it does. That’s usually done through lipid nanoparticles (LNPs), tiny rafts that cover the mRNA and let it flow into cells.
LNPs are already working in mRNA vaccines, but it’s a conundrum whether they could also work in other mRNA therapies (for chronic diseases especially). You get the mRNA to the right cells, at the right levels, for the right duration, and then you’re good to go with the most efficacy and the least side effects.
The question now becomes how to make mRNA therapies actually effective, and lipid nanoparticles, viral vectors, and so on have the answer.
The Future of mRNA Therapeutics
There are never-ending mRNA treatments. Once the technology has a head start, there are likely to be many more potential treatments for intractable diseases. These COVID-19 vaccines already begun the democratization of cancer immunotherapy, genetic disease, and autoimmune disease.
In addition to vaccines and the cure of disease, mRNA might even be used in personalized medicine. If mRNA therapies could be customized to a patient’s genome, we might design very tailored treatments that are both more effective and less toxic than current therapies.
And just as there are mRNA therapies, so there are new therapies in regenerative medicine. Inducing cells to make therapeutic proteins, mRNA could even initiate tissue repair, wound healing, and organ repair.
Conclusion
mRNA therapies might also change medicine itself. Vaccines, cancer immunotherapy, gene therapy—there’s no end of areas of promise. There’s more to do, implementation and stability among them, but the successes of mRNA vaccines are not the end of the story.
As more are discovered and developed, mRNA therapies could be just the next frontier of medicine with their highly individualized, targeted, and potent treatments for any illness. It’s a technology that will rewrite medicine and rewrite the futures of millions of patients worldwide.
Below is a table that summarizes some of the key services and products offered by Creative Biolabs on our mRNA platform.
This table introduces some of the essential services and products available at Creative Biolabs for mRNA-based research and therapeutic development. We offer comprehensive solutions for various mRNA applications, including synthesis and modification services, as well as a range of mRNA products tailored to specific research needs.