Trick or Treatment—The Undeniable Facts about Alternative Medicine (Electronic book text)

Despite its sheer simplicity and powerful ability to get to the truth, some alternative therapists argue that the clinical trial is a harsh test, which is somehow biased against their treatments. But that sort of attitude betrays a skewed understanding of the clinical trial, which merely seeks to establish the truth, regardless of the type of treatment being examined. In fact, the clinical trial provides a wholly unbiased and truly fair test of any medical treatment, either conventional or alternative. The unbiased nature of the clinical trial is demonstrated by the fact that the history of mainstream medicine is littered with apparently good ideas from conventional doctors that clinical trials proved to be useless or harmful.

For example, Bill Silverman, an American paediatrician who died in 2004, was a committed advocate of the clinical trial, even though he realized that it was a double-edged sword, capable of either validating or crushing any treatment. In 1949 he began working at the newly opened premature-infant station at the Babies Hospital in New York, and within a few weeks he was dealing with a premature baby suffering from a problem known as retinopathy of prematurity (ROP), which can result in permanent blindness. The baby was the child of the hospital’s biochemistry professor, whose wife had previously had six miscarriages. As this was the first time that the professor’s wife had successfully given birth, Silverman was particularly distressed at the prospect of the child becoming blind. Grasping at straws, he decided to administer a newly discovered hormone known as ACTH (adreno-corticotropic hormone), which had not previously been used to treat newborn infants. Although it was a fairly hit-and-miss approach, with Silverman varying the dosage according to the baby’s response, the end result was that she gained weight, her eyesight recovered and eventually she went home happy and healthy.

Inspired by this recovery, Silverman continued his ACTH treatment with subsequent cases of ROP. Furthermore, he compared his results with the recovery rates of babies with ROP at Lincoln Hospital, which was not offering ACTH treatment. The comparison was striking. Silverman gave ACTH to thirty-one babies suffering with ROP — twenty-five left with normal vision, two with near-normal vision, two with vision in just one eye and only two lost their sight completely. On the other hand, Lincoln Hospital had seven babies with ROP — they all lost their sight, except one.

For many doctors, the existing data — thirty-one babies treated with ACTH with a success rate of 80 per cent versus seven untreated babies with a recovery rate of only 14 per cent — would seem convincing enough. It would have been easy for Silverman to have continued with this therapy and recommended it to colleagues as a method for preventing blindness, but instead he had the humility and courage to question his own discovery. In particular, Silverman could see that his pilot study fell short of the rigour demanded by a high-quality clinical trial. For example, the babies were not randomly assigned to the treatment or non-treatment groups, so maybe the babies at Lincoln Hospital were suffering from particularly serious problems, hence their lower recovery rate. Or maybe Lincoln Hospital’s lack of success was a result of poorly trained staff or lack of equipment. Or maybe Lincoln Hospital was just unlucky — after all, the numbers involved were relatively small. To be confident about the efficacy of ACTH, Silverman decided to conduct a properly randomized controlled clinical trial.

Premature babies with ROP were randomly assigned to an ACTH treatment group or a no-treatment control group within the same hospital. Both groups were treated identically, apart from the use or not of ACTH. Within a few months the results emerged. An impressive 70 per cent of the infants treated with ACTH completely recovered their sight. Remarkably, the results in the control group were even more impressive, with an 80 per cent recovery rate. Babies in the untreated group had fared slightly better in terms of avoiding blindness, and moreover they suffered fewer fatalities compared to babies in the group treated with ACTH. It seemed that ACTH offered no benefit to babies and also had side-effects. A follow-up study confirmed the results of Silverman’s rigorous clinical trial.

The initial results from the Lincoln Hospital were abnormally poor, which had fooled Silverman into believing that he had discovered a powerful new treatment, but he had been wise enough not to be complacent and rest on his laurels. Instead, he re-tested his own hypothesis and disproved it. Had he had not been so critical of his own work, subsequent generations of paediatricians might have followed his example and administered ACTH, a useless, expensive and potentially harmful treatment.