From Lab to Living Room: mRNA Therapeutics

In recent years, advancements in biotechnology have led to the development of innovative medicines such as messenger RNA (mRNA) therapeutics: a mysterious new field of science with tremendous applications in cancer, rare disease, and infectious disease. Yet, the complex science behind these novel biotechnologies can often be difficult for both healthcare providers (HCPs) and consumers to understand.

The only way for the life science industry to reach its full potential is to go well above and beyond MDs, PhDs, and Biology majors. It is mission critical for everyone, regardless of health literacy, medical specialty, and demographic profiles to understand the facts behind these therapeutics in order to make decisions that are right f0r them. After all, regardless of how powerful certain therapeutics are, if patients and HCPs do not understand the benefits, their potential will never be reached.

Fortunately, the consumerization of life sciences can help to overcome some of the challenges that healthcare professionals face in keeping up with the latest developments in new therapeutics. Consumerized content simplifies the science and technology behind biotech innovations into easily digestible formats. This content aims to break down complex scientific concepts into simple language and visuals, allowing consumers and doctors alike to better understand the science behind these new treatments.

The video above, narrated by Andrew Berg, MD, and I, outlines the role of mRNA within the cell and the basis for how mRNA therapeutics work.

Imagine every cell in your body is a medieval town, and every cell of your body has a set of jobs. mRNA is like a messenger that brings important instructions from our cells’ instruction book, called DNA, to the protein manufacturers in our cells. These instructions tell the protein manufacturers how to put together the building blocks of proteins, called amino acids, in a certain order. Once the protein is made, it can do important jobs like helping us see, move, and fight off disease. Without mRNA, our cells wouldn’t know how to make these important proteins.

mRNA therapeutics are like a recipe book that helps our cells make important proteins to fight diseases. Scientists can create mRNA instructions that tell our cells how to make a specific protein that our body needs, like a medicine. They then package these mRNA instructions into a special delivery system that helps them get into our cells. Once inside, our cells use the mRNA instructions to make the protein, which can help our body fight off the disease or illness. It’s important to note, that mRNA from these therapeutics can never enter the nucleus of the cell.

Although Moderna (NASDAQ
NDAQ
: MRNA) became a household name based on their work around infectious diseases such as COVID-19, a tremendous amount of their effort is being invested in validating medicines for oncology and rare disease.

“We are thrilled to be at the forefront of developing individualized mRNA therapies for oncology. Our technology allows us to create a unique treatment for each patient, based on the specific characteristics of their cancer,” says Arpa Garay, Chief Commercial Officer of Moderna. “By programming the patient’s own cells to recognize and attack their specific cancer cells, we are unlocking a new era in cancer treatment that has the potential to be a game-changer for cancer patients everywhere. We are proud to be partnering with Merck, a leader in oncology, to deliver this medicine to patients upon approval.”

Moderna’s personalized messenger RNA technology represents a groundbreaking approach to fighting diseases, as they are able to tailor the medicine to match the unique characteristics of each individual patient’s condition. This makes it possible to unleash the power of the body’s own immune system in a precise and effective way, offering new hope to patients and transforming the future of medicine.

So, first of all, let’s talk about what a tumor is. A tumor is basically a bunch of cells that are growing out of control in one part of the body. Sometimes, these cells can be cancerous, which means they can be really dangerous and can even spread to other parts of the body if we don’t stop them.

Now, when your body is trying to fight off cancer cells, it needs to be able to recognize them as something that doesn’t belong in your body. One way it does this is by looking for things called “epitopes” on the surface of the cancer cells.

Think of epitopes like little flags on the surface of the cancer cells that say “Hey, I’m not supposed to be here!” But here’s the thing: every tumor has different epitopes, so it can be tricky for our immune system to recognize all of them.

“Part of the remarkable power of mRNA is that in, for example rare metabolic diseases, as long as we know the protein that is involved in a disease pathway, the war is really over. The mRNA therapeutic enters the cell, is made into a protein exactly as your cell needs it to be made, and it is then shuttled exactly to the right place in the cell where it is needed. That would be extremely hard to do with a standard small or large molecule medicine. The same is true in cancer, we are able to knit up to 34 different mRNAs together, each one making a specific cancer antigen, that powerfully ignites and directs our immune system to recognize and kill the cancer cells. The fact that mRNA is synthesizable, scalable, specific and targetable makes it a tremendously powerful new therapeutic approach that can be brought to bear on so many diseases,” says Paul Burton, MD, PhD, Chief Medical Officer of Moderna.

Rare diseases, though each one by itself is quite unusual (by the FDA definition, affects fewer than 200,000 people in the US), as a group are not so rare at all. Nearly 25-30 million Americans have one of these 10,000 rare diseases, but for 95% of those, there are no therapeutic options available.

According to Pamela Davis, MD, PhD, a Professor of Case Western Reserve University who previously served as the Dean and SVP of Medical Affairs from 2007-2020, “Therapeutic use of mRNA offers great promise for diseases that occur due to a missing or dysfunctional protein. The half dozen diseases now being treated experimentally with mRNA should inform our strategy for other rare diseases and offer hope to many patients. For diseases that arise from a missing or dysfunctional protein, treatment with mRNA encoding the missing protein could direct cells to manufacture that protein, and use the cell’s own machinery to modify the protein appropriately after it is made.” Right now, treatments are being tested for over half a dozen such diseases, including methylmalonic acidemia, acute intermittent porphyria, Fabry disease. Glycogen Storage Disease Type 1a, Propionic Acidemia, Ornithine Transcarbamylase Deficiency, Phenylketonuria, and fatal infantile cardioencephalomyopathy. This strategy is potentially applicable to other disorders as well. What we learn from one will inform development of other mRNA therapeutics. Treating these diseases could save lives, or enormously improve the quality of life, for potentially millions of people, many of them children.

As R&D advances at an increasingly rapid pace for therapeutics options such as mRNA, it is difficult for healthcare professionals to stay up to date with the latest research and treatment options. Two reasons for this may include rapidly advancing research and limited time and resources. The field of medicine is constantly evolving, with new research and breakthroughs happening all the time. With so much new information to keep track of, it can be challenging for doctors to stay current on the latest therapeutic options. Furthermore, doctors are busy professionals who have many demands on their time. They may not have the time or resources to read the latest research articles or attend conferences to stay up to date on the newest therapeutic options. The growing shortage of HCPs is continuing to exacerbate these issues.

“In light of the growing time constraints faced by healthcare providers, it is crucial to deliver complex medical information to consumers in a digestible manner, as this will ultimately lead to more informed and engaged patients. Additionally, by enabling patients to better understand new therapies, we can ease the burden on physicians who would otherwise have to spend valuable time explaining treatment options to patients,” says Dr. Jeffrey Sachs from Sachs Policy Group. For consumers, staying

informed about their health can be much more challenging. Patients do not have the same level of medical training and access to resources as HCPs. They may struggle to understand medical terminology, navigate the healthcare system, and find reliable sources of health information. Additionally, medical information can be complex, ever-changing, and difficult to interpret, even for those with a high level of education.

“Life science manufacturers must recognize that investing heavily in research and development alone is not enough. Without effective methods of consumerization, even the most promising therapies will not reach their full potential. It is imperative that manufacturers prioritize developing streamlined pathways for delivering new treatments to both consumers and healthcare providers. Only then can we ensure that the significant investments made in R&D translate into meaningful advancements in patient care,” says Daniel Drexler, MD, Founder of Klick Group’s, btwelve.