A therapeutic revolution: What will the treatments of the future look like?

With messenger RNA vaccines, treatments with CRISPR scissors and other gene therapies, the drug therapy of the future looks like something out of a science fiction movie. For genetic researchers, human DNA and RNA are turning out to be real gold mines that will allow them to design a whole new generation of treatments. We had a taste of it during the COVID-19 pandemic with the first generation of messenger RNA vaccines, but researchers predict that RNA, combined with new technologies under development, could completely revolutionize medicine. They envision how new vaccines could be designed to act against a wide range of viruses and pathologies that cannot yet be cured. Éric Lécuyer, director of the RNA biology research unit at the Montreal Clinical Research Institute, explains that unlike traditional drug therapy approaches, the use of genetics, and particularly RNA, enables us to target defective cells directly. “The emergence of RNA-based therapies will make it possible to tackle a whole new panoply of diseases,” he says. "It opens the way to treat many diseases that were not treatable by conventional drugs." For Jean-Pierre Perreault, holder of the University of Sherbrooke research chair in RNA structure and genomics, using RNA will make it possible to carry out precision medicine by having the sequencing of each patient, therefore have more effective drugs. One of the most promising aspects of these new therapeutic approaches is that they can bring deviant cellular functions back to normality. It would thus be possible to treat many pathologies that are currently difficult to treat, such as nucleotide repeat diseases such as myotonic dystrophy, also known as Steinert's disease (a musculo-degenerative disease particularly present in Quebec), as well as neurodegenerative diseases such as amyotrophic lateral sclerosis. Treatments using RNA also have a very broad scope. “All viruses have surface proteins. We could therefore target any virus whose surface protein sequence is known. This is also one of the objectives of the pharmaceutical company Moderna: to produce a whole series of vaccines against infectious agents," Perreault says, noting that the same approach could be used to target proteins associated with potential cancers. Read: Integrating safe and effective e-prescribing However, there are some limitations on the side of RNA technologies. Indeed, the transport and delivery of therapeutic agents can be a problem “especially when it comes to hard-to-reach organs like the brain,” says Marc-André Legault, a postdoctoral fellow at McGill University who works on the intersection between machine learning and healthcare, with a focus on genomics. “The specificity of agents can also cause problems, as it is essential that only the target of interest is affected by the therapy in order to minimize the risk of unexpected and adverse effects.” Researchers hope to meet these challenges, in particular through the development of new technologies such as artificial intelligence. “Artificial intelligence will perhaps help us identify targets on a certain number of proteins or even which sequences should be targeted rather than others,” says Perreault. Artificial intelligence technology, he says, would also make treatments usable by a larger population and help reduce the cost of research. “There are several levels of application and these are sectors that are very well intertwined in terms of multidisciplinary research,” adds Lécuyer. Curing by modifying the genome Gene therapies that modify a patient's genome represent another new and promising tool for the treatment of diseases. “Certain therapies aimed at editing the host genome are also in development. For example, the Vertex company is in the process of developing a gene-editing therapy aimed at rendering the PCSK9 enzyme inactive in a very long-lasting manner,” explains Legault. This type of therapy would reduce low density lipoprotein cholesterol and therefore the risk of developing atherosclerotic cardiovascular disease. Lécuyer adds that chimeric antigen receptor technologies that allow cells to be modified and reintroduced into a patient hold great promise in the treatment of cancer. He anticipates that gene therapies, such as treatments based on CRISPR technologies—where DNA is cut at a specific location in the genome to replace one segment with another—will come with their share of ethical questions. “From an ethical point of view, but also from an evolutionary point of view, we very possibly do not want to modify the genetic heritage,” continues Perreault. He adds that targeting RNA is a technology that could achieve promising results, but without modifying the genetic heritage like CRISPR technology. Great clinical potential Despite the obstacles and debates that genetics will inevitably raise, researchers are optimistic about the therapeutic results that these new therapies suggest. “The therapeutic arsenal available to clinicians will undoubtedly be increased,” says Legault, who hopes that understanding the genome will one day help to treat rare diseases and diseases for which there is no curative treatment such as Parkinson's or Alzheimer's. For his part, Perreault is convinced that a therapeutic revolution is upon us. “There's a lot of excitement, it's like we've just touched the tip of the iceberg. We have seen the performance of messenger RNA vaccines in the context of the pandemic, but it is only a precursor to many innovations in the RNA sector in the years to come,” says Lécuyer. This article has been translated from its original French and first appeared on our sister site Profession Santé.

A therapeutic revolution: What will the treatments of the future look like?

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