Clinical Research in the Practice

A vital aspect of this practice is the access to new research drugs that are under investigation. This is an essential component of my clinical approach to provide the best possible care for patients.

The background is that in 1976 the first studies on Tagamet [cimetidine] started. The success of the agent was measured by the healing of the stomach ulcer being treated. While not revolutionary, this set the standard of clinical studies in gastroenterology. The objective was always to cure. This remains the Holy Grail.

In order to market myself better in my primary area of interest, i.e. inflammatory bowel disease, it was clear that a drug-related study was essential on my CV. The problem was that the chances of a young investigator on the gangrenous tip of Africa joining a major international research project was minimal. At the time, the alternative practitioner groups believed that vitamin A deficiency was the cause of inflammatory bowel disease. The question was simple, so we started a self-funded, double-blind, randomized, placebo-controlled study to test the hypothesis. We randomized 80 patients, which was considered to be a vast number for a single practice, into the study. The statistics were beautiful and showed absolutely no benefit to the putative beneficial agent. The results were published in an article in Gastroenterology in 1980. This was the most important international journal at the time. With this boost to my CV, international studies followed, and the practice was established.

We have been fortunate to have a constant stream of new agents for the treatment of inflammatory bowel disease. We specialize in phase 2 and 3 studies. Phase 2 is for drugs that have already been shown to be safe in healthy volunteers but where the patient benefit is not yet clear. Phase 3 is where they are safe and effective in patients, but the optimal dose is not yet finalized.

We rode the first wave of anti-TNF agents now marketed as Revellex (Infliximab), Humira (Adalimumab) and Simponi (Golimumab) in both Crohn’s Disease and Ulcerative Colitis.

Many other molecules followed, but perhaps the most exciting was Tysabri (Natalizumab). This agent stopped the inflammatory cells accumulating in the bowel. It did not have the immunosuppressive qualities of the anti-TNF agents. This is of particular importance in South Africa, where the threat of tuberculosis is ever-present. The agent, unfortunately, was suddenly withdrawn as some patients had developed a very serious brain infection. In the end, the infection turned out to be nonspecific but clearly dangerous. The drug has now been licenced in the USA under strict controls. The proof of concept was, however, established, and molecules with similar benefits but more specific for the gastrointestinal tract were designed.

We are now on the verge of seeing these new agents being marketed. Many of our trial patients can bear testimony to their effectiveness and safety. It is this aspect that makes clinical research so rewarding and important in modern specialist clinical practice.

We are currently working with even more specific and powerful agents. Whereas all the drugs above need to be given by injection, we are now using some oral agents, which are just as exciting.

This collaboration between “big pharma”, research companies and clinical practices is leaving the romanticists, beetroot eaters and wishful thinkers alone with their symptoms and diseases.