How RNA Is Making Personalized Medicine
a Reality


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From "Buck Rogers" to "Star Wars," the idea of targeted, personalized medicine has been a science fiction dream for decades.

The 2013 film “Elysium,” starring Matt Damon, took this concept a step further, featuring machines called Med-Bays that were capable of diagnosing, treating, and curing any disease or injury almost instantaneously based on the needs of each individual patient. In the “Star Trek” universe, throughout the long-running series’ various incarnations, high-tech medicine was the norm, with machines that could fix seemingly any medical problem that walked into the sick bay, automatically and precisely.

Sadly, this level of care remains solidly in the realm of sci-fi. We don’t yet have access to automated diagnosis machines or devices that can regrow broken bones, like those seen on TV and in the movies.

But personalized medicine, as a broader concept, is very much becoming a reality, thanks in large part to work being done in the field of genetics. The Human Genome Project, which fully mapped out the genetic code of human life and was completed in 2003, started this process by allowing researchers to, for the first time, identify and isolate disease risk factors on an individual basis, breaking down our DNA into segments that can be analyzed and studied.

It changed the scale of work as well. Before the genome was mapped, it would have cost as much as $5,000 and taken weeks for researchers to read a line of DNA a million letters long, which is the typical length of an individual DNA segment, in part because so much of the work was done manually at that point. With today’s advanced tools, though, that same work now can be done in minutes and cost just pennies.

RNA molecule. Image: Wikimedia Commons

The Role of RNA

But DNA is only one step on the path to personalized medicine. It’s RNA, our ribonucleic acid, that holds the true potential to create real, targeted treatments like those seen in sci-fi. That’s because, unlike DNA that is hardwired at our birth and doesn’t change much over our lifetimes, RNA is constantly in flux, reflecting our overall health on a minute-by-minute basis. The acid itself serves as a sort of messenger, carrying instructions from our DNA to control protein synthesis and other activities, effectively turning the DNA roadmap into reality.

“Genes are our blueprints, not our destiny,” says Dr. Sharad Paul, a New Zealand-based cancer surgeon and the author of The Genetics of Health, a survey of the role that our genes play in our day-to-day lives. “Knowing our genes, or relevant ones, helps us take charge of our health. If DNA are our blueprints, RNA are the things that help implement the plan. RNA are more changeable and not as stable as DNA.”

Researchers are using this fact to develop new treatments that, until recently, would have seemed all but impossible. With RNA, doctors will soon be able to target treatments to individual patients, drug developers will be able to create new classes of drugs that address specific genetic populations, and practitioners will even be able to track treatment responses in real-time.

That’s one potential application of personalized medicine that Dr. David Messina, COO of Cofactor Genomics, a biotechnology startup in St. Louis, MO, that is using RNA to diagnose disease, is particularly excited about. In the case of solid tumor cancers, such as lung cancer, the outlook for patients often is not great at the outset. Part of the challenge for treatment, then, is that front line therapies in these cases are often chosen based on how effective they are in most patients, most of the time. They cannot currently be targeted specifically to each patient. So, if the front line treatment works, that’s great. But there's a very high percentage of the time where the front line treatment doesn't work, and doctors are forced to move on to the next-most-common treatment and try again.

“In those cases you've spent a month or two on the wrong therapy and you've spent that money, because often these treatments are extremely expensive,” Dr. Messina said. “But then also that person has not gotten the right medicine for that length of time. In some cases they may not get another shot to try the next medicine that the doctor would try.”

By offering better insights into how the body is responding or not responding to a given treatment, Messina believes that doctors will soon be able to better determine whether or not the common front line treatment is the right one for a given patient, and even move directly to the second option is that’s a better choice. Eventually, he hopes, medical science will be able to actually predict which treatment will be most effective ahead of time, enabling drug developers to create targeted treatments to address genetically-similar populations in addition to individual patients, vastly expanding the potential for drug efficiency.

A Fast-Developing Field

Another growth area for RNA-based medicine involves RNA interference (RNAi), which is the process by which RNA stops or inhibits protein creation by the body’s genetic code. It’s a stop sign for our DNA, and it’s something that everyone on Earth has in their cells, regulating the amount of protein production and making sure the body does not overproduce anything that it needs.

Given this natural behavior, RNAi holds a lot of potential for drug developers, who are working to harness the “stopping power” of this process to target specific proteins and address individual genetic risks in patients.

“There are a number of diseases where certain proteins are produced that are not the right protein,” says Dr. Geert Cauwenberg, CEO of RXi Pharmaceuticals, a clinical-stage biotechnology company that is developing new therapies based on RNAi. “That, of course, is the case in cystic fibrosis and Lou Gehrig's disease, ALS, where there is a misfolded protein being produced, and so you don't want that protein there.”

RXi Pharmaceuticals, and others, are creating RNAi tools to stop these processes, creating biological triggers that mimic the RNAi sequence in the patient’s body. By creating synthetic RNAi, doctors will soon be able to take over the role of the biological RNAi in the body, in case the patient’s body isn’t doing the job properly, preventing these negative responses.

Cauwenberg sees great potential in this technology to deliver personalized treatments for cancer, effectively relegating today’s common treatments to medical history.

“I foresee that ten years from now, with the way progress has been made, chemotherapy is going to become the last resort, not the first resort,” he says. “So the survival rate in oncology in the next ten years is going to change dramatically.”

Tim Sprinkle is an independent writer.

Knowing our genes, or relevant ones, helps us take charge of our health. If DNA are our blueprints, RNA are the things that help implement the plan.

Dr. Sharad Paul, author of The Genetics of Health

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June 2017

by Tim Sprinkle, ASME.org