In this blog we analyse the current landscape of RNA therapeutics and postulate on where best digital health may support this innovative area of medicine.
What are RNA based therapeutics?
It may sound trite to claim that we’re entering a new era in therapeutics. Across industries, especially in the scientific space, almost every minor announcement is prefaced by a declaration of a significant turning point having been reached, which can dampen the impact of true leaps of innovation. However, though the small molecule still reigns supreme, we have witnessed some incredible advances in recent years in the study of other treatment modalities. This is critical at current as available small molecules and antibody therapies can only target around 0.05% of the human genome. A central issue with these existing therapies is that most of them are too large to enter our cells and thus they can only be applied to secreted or extracellular proteins. As a result, many diseases either have a below optimal therapy or no therapy at all due to the inaccessibility of key pathways and processes within our cells involved in their presentation.
Once believed to simply act as a genetic messenger, RNA has since been found to be involved in a plethora of processes within the cell; including activating or deactivating genes, transporting amino acids to their needed sites, and assisting in chemical reactions. Rather than addressing symptoms of an existing disease state or curing it post-occurrence by binding to a disease-related extracellular target, RNA therapies have the potential to alter our genetic and protein make-up at source, and edit out the causing factors that lead to that condition in the first instance. Indeed, the original discovery of the treatment potential of RNA, specifically of using RNA to silence certain genes through interference (“iRNA”), earned Andrew Fire and Craig Mello the Nobel Prize in 2006.
Since this breakthrough, multiple companies such as well-known industry behemoths Pfizer and Moderna, and more specialised players like Ionis, Arrowhead and Alnylam Pharmaceuticals have developed RNA based pipelines. Many formats of RNA are currently being examined for their potential in human health and disease.
What do these treatments look like in practice?
RNA therapies have received global attention recently due to the fact that some of the Covid-19 vaccines belong to this technology area. Pfizer/BioNTech and Moderna were the first companies to have mRNA vaccines reach the market for any indication worldwide. Beyond vaccines, there are only a handful of FDA approved RNA therapies in existence at present, with many of them currently in clinical trials. They are aimed at various disease states, including many rare diseases, such as Duchenne Muscular Dystrophy and Primary hyperoxaluria type 1 (PH1), and also more common disease states such as Cystic Fibrosis, Atherosclerotic Cardiovascular disease and Prostate Cancer. Two notable milestones were the FDA’s approval of Sarepta’s Exondys 51 to treat Duchenne Muscular Dystrophy (DMD) and of Ionis’ Spinal Muscular Atrophy (SMA) treatment Spinraza, both in 2016.
In terms of modalities, these therapies are delivered in a variety of ways, predominantly intravenously (IV) and subcutaneously (SQ). However, some are delivered through routes such as inhalation (such as Translate Bio’s Cystic Fibrosis treatment) and through implantation (Silenseed’s Pancreatic Cancer treatment). Depending on the treatment, patients may either need just one dose or, usually in the case of chronic conditions, a treatment course with multiple injections or a continuous infusion (for example for certain types of cancer or genetic disorders).
Where do digital health solutions fit in?
As digital health continues to search for a stable foothold in current healthcare markets, a new cohort of therapies potentially brings a fresh realm of possibilities. It is worth mentioning that due to the fact that RNA therapies are predominantly administered in a manner that requires a healthcare professional (e.g. via IV), it is unlikely that solutions such as patient adherence or motivation products will have broad applicability in this area. That being said, there may be a few RNA treatments that patients can self-administer; as mentioned, many of these products are in the pipeline and so their exact treatment regimes are yet to be fully realised. For those, medication tracking and adherence companions may be of great help, especially those in the injectables space such as smart disposal bins, injection site tracking or dosing aids. Otherwise, there is the potential for good traction by digital health for:
- Patient monitoring (both remote and on-site)
- Systems that allow for reporting or recording of the patient experience
- Day-to-day, patient-orientated diagnostic solutions
- Telehealth when it comes to RNA treatment
These areas of opportunity stem from the currently known side effects of some long term RNA based treatments. These include dyspnea, muscle spasms, upper respiratory tract infections, serum creatinine increases, fatigue, abdominal pain and arthralgia, among others. Depending on how ill the patient is at the commencement of treatment, these issues could negatively impact quality of life, or even pose serious risk to their well-being. Tools that aid in monitoring certain symptoms and communicating back with a patient's care team if any of them reach a worrying threshold while they are between doses would be extremely beneficial. Further, RNA therapies can affect liver function, leading to numerous issues such as deficits in certain vitamins. In order for patients to be able to stay well, it would be beneficial for them to be able to measure and track certain indicators from their blood and urine between doses. Finally, as there may be long intervals between dosing sessions with a specialist, tools such as telehealth that can connect key stakeholders in the patient’s journey to each other, and aid in information sharing, would go a long way in ensuring that the treatment journey is as smooth as possible for the patient.
Will We See RNA Focused Digital Health Products Soon?
RNA therapies are relatively new and not much research has been conducted on long term treatment regimes or the lasting effects of being on such technologies; it will be interesting to see how they continue to evolve over the coming years.
Further, though there are many prominent remote patient monitoring, at-home testing and telehealth solutions on the market; it was difficult at the time of writing to pinpoint any marketed specifically for RNA treatment, or having made a mention of it as a therapeutic area covered. We also could not locate any specific patient education or support resources specifically for those either on RNA therapies, or considering them. Therefore, there may be an opportunity toward developing awareness and education around RNA therapies, and toward considering what specific supports could be put in place for those on such regimes. For platforms that specialise in personalised remote patient monitoring, we will be interested to observe if RNA treatments become more commonly listed modalities covered by them in the near future.