Developing drugs to treat disease is never easy. But recent research into a relatively unknown area of science provides us with a wealth of new opportunities for drug discovery in the areas of cancer, cardiovascular disease, diabetes, neurodegenerative disorders, and inflammation, among others.
Throughout most of history, new drugs have been discovered through a process of trial and error or simply through dumb luck. Think of Alexander Fleming who discovered penicillin in 1928 (actually he rediscovered it, but that's a story for another day) when some bacterial cultures he was working with became contaminated by a blue-green mold, and he noticed that colonies of bacteria adjacent to the mold were being dissolved.
Since neither trial and error nor reliance on luck make for a particularly strong business model, biotech and pharma companies have started to adopt a new method of drug development called rational drug design, wherein molecules and compounds are specifically designed or chosen to attack a particular target.
Today, the vast majority of molecular drug targets are proteins (and a small percentage are nucleic acids). According to biologist R.A. Sabbadini, writing in the British Journal of Cancer a few years ago:
“The focus on proteins was a natural consequence of our evolving understanding of biochemistry, which allowed researchers to identify potential protein targets involved in key metabolic and signaling pathways. Some of the first drugs developed by the rational drug design approach to the scientific method came after the discovery of key enzymes, receptors and ion channels [all proteins] as they emerged in the basic science literature. One can argue that target identification now is driven by the technological developments of proteomics and genomics, both of which reflect our persistent 'protein-centric' view of drug discovery.”
Long story short, it made sense for drug developers to target proteins, because proteins are known to regulate many normal cellular functions – like cell division, cell migration, and cell death – but over time they could become dysregulated and cause disease. So scientists focused on developing drugs that neutralized these disease-causing proteins.
While proteins will continue to play a big role in drug development for the foreseeable future, the emerging field of “lipidomics” provides both a wealth of new therapeutic targets and distinct advantages over protein-centric drug discovery.
Like the other “-omic” disciplines (i.e., genomics, proteomics, etc.), lipidomics refers to all the members of the lipid family of compounds. Scientists are trying to develop methods to analyze all the lipids of cells. This isn't easy, considering that lipids vary considerably in structure. Moreover, depending on whose estimate you use, the number of different molecular species could be in the tens of thousands to hundreds of thousands. In fact, of the four basic types of molecules that are the major players in biological systems (including the human body) – i.e., nucleic acids, carbohydrates, proteins, and lipids – lipids stand out for their sheer number of distinct molecular species.
The study of lipidomics is further complicated by the fact that, unlike genomics and proteomics, we can't predict the number of individual lipid molecules present in an organism. Thus, current technologies are still unable to map lipidomes. But scientists are making progress thanks to technological advancements, particularly those in mass spectrometry.
We used to think of lipids as just the building blocks of cell membranes and fuel-storage molecules for cellular maintenance. Through the study of lipidomics, however, scientists have discovered that lipids play key roles in all areas of cell biology as signaling and regulatory molecules – in other words, they can be what's known as bioactive molecules. The notion of “bioactive lipids,” which is broadly defined as changes in lipid levels that result in functional consequences, has started to gain traction over the past couple of decades; medical researchers now recognize that these bioactive lipids play key roles in many diseases and represent new targets for rational drug design.
For example, the bioactive lipid lysophosphatidic acid (LPA) plays a key role in regulating nerve injury and pain. If you can develop a drug that neutralizes LPA, you can block the signals to the brain and ease the pain of a patient who has been injured.
There are an estimated 1,000+ bioactive lipids that play key roles in all kinds of diseases, providing a gold mine of potential targets for drug makers. What's more, bioactive lipids are much smaller and simpler molecules than proteins and generally don't vary from species to species. Thus, the data from animal models tends to be much more predictive of success in the clinical setting than with protein-targeted drug development programs.
There are already well-known drugs that target lipid metabolic and signaling pathways, including the cholesterol-lowering statins. But so far, due to their extremely small size and the fact that they are not water soluble, drug companies have had a problem targeting bioactive lipids directly – until now. In a recent issue of Casey Extraordinary Technology, we recommended a micro-cap company that is poised to emerge as the leader in lipid-based therapeutics, thanks to its novel platform technology that can develop drugs to directly target any of these molecules. That's exciting enough, but to add icing on the cake, the company has already developed a pipeline of drugs in clinical trials. The firm's lead drug candidate (currently in Phase II trials) could generate annual peak sales many times that of its current market capitalization.
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