Modern Drug Development
As we’ve entered the modern era, it seems that most medications these days are developed due to conscious attempts to target specific molecular patterns.
Gleevec, for instance, a major breakthrough in the treatment of a specific type of cancer, was made based off an understanding that the Bcr-Abl fusion product was playing a major role in the cancer. Researchers, to make it, tested compounds for activity against that product.
Such research is typical.
Understanding the target to choose is a key role in the successful development of a treatment. In some cases, it is a limiting factor.
In Alzheimer’s, for instance, no therapy has proven significantly successful. This is likely due to us not knowing what to target. If we had a specific protein that we could knock out or increase its activity, it is likely we would be able to develop better treatments.
It is somewhat underappreciated how release mechanisms of medications have improved in recent years. Yet these improvements have led to smoother release of substances.
This can provide significant benefits in multiple realms. With medications with a narrow therapeutic range of effect, for instance, smooth release can help avoid adverse events. Additionally, in psychiatric medications, smoother release can provide for better results.
Monoclonal antibody technology has developed significantly in the recent years. The advantage of using monoclonal antibody technology is the potential for increased specificity and possibly decreased dosing.
A monoclonal antibody treatment can be given as rarely as once every six months while still having significant clinical benefit.
Specific l or d form
A significant advance in some areas is the realization that mirror forms of a medication can play a role in its efficacy and side effects.
Molecules can have two forms: the l or d form. These forms are a lot like your left and right hands. They are mirror images of each other – exactly the same in composition, just opposite in arrangement.
Research indicates that with some medications, one form is more effective or has less side effects than the other. In the extreme, there have been cases of medications where one form is entirely ineffective.
Growing understanding of this has lead to improvements in some medications.
An increasing amount of interest is being paid to treatments that work as biologics.
Biologics are large molecules that are typically protein based that mimic natural substances in the body. The main interest in them started with the rise of Epogen, a complex treatment that stimulates the body to produce more red blood cells.
Biologics promise more sophisticated and more powerful treatment of many conditions. As such, interest in them has grown significantly.
Additionally, biologics, by their nature, are extremely hard to make. This means that producing generic versions can be very challenging. This, and their high price, makes them attractive to drug companies.