Meet the Drug Hunters: the scientists determined to cure rare diseases

Andy Martin explores the world of the resolute few who persistently pursue the cure for conditions which even doctors and pharmaceutical companies have given up on

Tim Guilliams (left) and David Brown, co-founders of Cambridge-based biotechnology company Healx (Onur Pinar Photography)

David Brown was running out of time and money. Having started his career at ICI Pharmaceuticals, he had been recruited as the head of the chemistry department for Pfizer and the drug he had come up with to treat angina (or coronary hypertension) was in clinical trials. The trials weren’t going well.

“Sildenafil citrate” (as they called it) had shown some signs of efficacy, but not enough. It was barely distinguishable from a placebo effect. The study in Aberystwyth, in the summer of 1993, was his last shot. After the closure of their coal mine, the local miners were willing to have a go at anything that paid, such as volunteering for drugs trials. They would come in one day, take their pills and stay overnight. Then the following day they had to fill out a questionnaire followed by an interview with the research associate, when she asked that crucial question: “Any other unusual effects to report?” Once one guy mentioned the erections everybody else chimed in. They’d all had them.

For Brown, a self-professed “drug hunter”, it was a eureka moment. Angina is the result of a restricted blood flow to the heart. Brown had been thinking about “vascular relaxation” and how to get more blood pumping through certain parts of the body. He had an “intuition” that he had found the solution – at least where other parts were concerned. When he heard about the result he chewed it over with his team, then rushed into the medical director’s office with his new idea but was promptly given the brush-off. They were out of time. The trials were obviously a failure. His budget was too tight. The director was calling it a day. A full eight years of hard work down the drain. Brown went to the door, pushed it shut, turned back to the director, and uttered a sentence that would not be out of place in a Tarantino movie: “I’m not leaving this office till you give me the money.”