RNA-based therapeutics is highlighted as a promising latest technology. This latest idea is you are introducing a Ribonucleic Acid (RNA) molecule into body as a treatment versus a protein otherwise a small chemical molecule. As there are two equivalent paths. More established one is based on a technology for which Nobel prizes were awarded a few years ago is known as RNA interference also has only been developed into late-stage clinical products in the last year.
This is done with molecules called short-inhibiting RNAs otherwise si-RNA, pieces of RNA which when they enter into a cell recognize also bind to a messenger RNA (mRNA) preventing production of a certain protein. It is the best technique to lower the amount of a protein being produced in a cell in a sequence-specific way.
There are many challenges naturally. Initially, how do you get these molecules into a certain type of cell also get them to bind the target protein? Elucidate many of these cells does it take to make a difference? Do these vary among diseases?
All of these issues have been subject of multiple academic also corporate efforts which are now beginning to bear fruit. However, what is emerging really in the last 3 years is an altogether dissimilar branch of RNA therapeutics.
As potential drugs researchers have now begun to develop full-length mRNAs. Researchers have figured out how to manufacture these full-length mRNAs also get them into cells in the body. As an alternative of inhibiting, the amount of a particular protein, you actually allow a cell to make just about any protein you desire.
As this type of protein could be a drug like an antibody Herceptin. It could also be utilize as a potential vaccine. Rather than use a dead virus otherwise a set of antigens to cause an immune response you would introduce the mRNA for an antigen which a cell in body produces also body recognizes it as foreign also uses the body's natural process to launch an immune response.
What conditions would this mRNA treatment target?
As the addition of a certain protein could help treat the condition for target any disease. This includes important diseases like cancer also cardiovascular disease as well as other rarer conditions.
Cancer can cause problems by increasing otherwise decreasing protein production. siRNA can deal with the overproduction also mRNAs those which decrease. As this approach allow us to target many proteins for the first time. Cholesterol management similarly has proteins where increasing otherwise decreasing them can be useful.
There are also rare disorders like Tay Sachs otherwise cystic fibrosis which lack an important protein otherwise its proper function - this approach offers a straightforward way to provide which missing protein otherwise function. This is very broad-based because it is a molecular technique which engages with important pathways also which is not disease-specific.
Is this a treatment which cures these conditions otherwise would it be used to manage them?
It would largely manage them; although the management of some of these diseases is the ultimate cure. We are seeing this with HIV also AIDS, many cardiovascular diseases, etc. There are precious few drugs which are cures for anything. Infectious diseases, some viral infections - these are things where you could get rid of the infection also get rid of the disease. In very few other things do you have which type of an effect? As it doesn't believe this modality is necessarily going to be any more otherwise less curative than the current methodologies; however it is too early to tell.
How would these treatments work in practice? Would a patient requires one treatment otherwise several?
Broadly speaking, it would say which RNA treatments will be used, depending on the disease, as a set of treatments which reduce a certain inflammation which has occurred otherwise any number of acute conditions. In some cases, it could be used in a chronic way, meaning every other week otherwise once a month otherwise once every three months. Yet again which part is disease-specific? It is not the molecule which dictates which; it is the intervention which you require also the biology of the disease which defines require.
It can give you an example of an acute case. A recent paper showed which, based on work in animal models, damage to the heart following a heart attack could be minimized with a single dose of the mRNA to ensure continued blood supply. In which case, it is an acute intervention; you would only have one shot also you are done.
In other cases, you might inject once a week otherwise once a month.
Also this treatment is administered through injections?
Individuals have used these intravenously. Others have shown which you can do this through intramuscular otherwise subcutaneous injection also even by inhalation. How viable these will be long term in oral formulations; however it is sure people will work on which as well.
How do you expect this treatment to develop over the next few years?
Expect the first generation of drugs which will be approved will be siRNAs. Four otherwise five years from now, we should begin to see an additional wave of mRNA therapeutics. Fully expect as it look out beyond 10 years, there will be several large drug categories which are going to have RNA-based treatments in them, which would have been unthinkable even five years ago.
Will this eventually become a fairly common treatment otherwise remains something for rare conditions?
Certainly, it will start treating some rare conditions; however it would say it will move pretty quickly into more common ones. This tends to be how new drug modalities get introduced into the therapeutic arsenal. The manufacturing advantage of mRNA where you can use the patient's own body to manufacture it will, however, provide a unique advantage for treating a vast range of chronic conditions. In cancer also in serious viral conditions, such as HIV also hepatitis, you may well see RNA-based approaches, However it is too early to predict with greater precision.
What kind of potential does this have to treat cancer?
It has vast potential for cancer; both on the RNA interference also on the mRNA side. Cancer is going to involve a multiplicity of approaches. It is a heterogeneous disease, however it would say which it is one of the areas where we expect to see a significant contribution from RNA therapeutics. What is exciting is which this approach gives an opportunity to go after a whole set of targets which previously could not be reached.