R&D Support Program Types

Phase 1: Basic Research

The basic research can start from different starting points:

• 1.

  Researching a specific disease and trying to identify the chemical biological cycle responsible for it. A potential result is an understanding of the chemical cycle operating the disease with the different compounds and cellular organs involved in it. These offer potential targets for future drugs to either stop the process, repair it or prevent it;

• 2.

  Testing different compounds for stopping a certain process that is known regarding a disease, original compounds either natural or synthetic – originating from libraries or from traditional medicine, these are potential hits;

• 3.

  Accidental events happening in the lab – recall how penicillin was discovered?

Once either target or hit are suspected, the research will focus on two major aspects, to try and show that the target is valid, or that the hit has at least some preliminary effect (at least a specific minimum doze shows some impact); and a better understanding of the process in order to be able to further the work. Most of the basic research support programs / grants finish at this point their support. Safety is dealt with at any of the preliminary steps, as if the drug candidate is potentially harmful the rest of the work is normally useless.

Phase 2: Translational Research

Following the above some medical chemistry follows in which hits for the known targets are designed. Once that is done or if the hit was the starting point, the next step will be a better design of the hits, defined as improved efficacy by reducing the doze required for efficient proof of concept. In many cases this work can be done using cell and tissue cultures. It will also require some sort of anti-toxicity proof, unless the compounds are already known as nontoxic. During this stage, a small number of candidate molecules are selected (normally one or two) as the best candidates. This is done normally using several rounds of medicinal chemistry, developing “families” of molecules based on the most promising hits and testing them. The aim of this process as mentioned above is to arrive at the end with the most promising molecule to continue the process with. Once that is done the move to the next step is performed.

Phase 3: Pre-Clinical Research

The next step in this model process would be the preclinical trials, preferably performed on an animal model developed to simulate the researched disease. The development of such models can be complicated, and there is of course a preference for the proved and accepted models which require no development, but still require setting up and validating. In some cases, no accepted model exists, or most models are not considered good enough. One should remember that the model should simulate the disease in the animals to allow for the cure to be developed. One should also recall that this phase is a step towards clinical trials in humans. Pre-clinical trials will include anti-toxicity if it had not performed before, as well as several animal models (increasing in size if required), depending on the disease.