Smarter clinical trials

Disappointing & intriguing

The results of the trial of the drug known as TCH346, conducted at 42 different clinics in Europe and North America, including centres in the UK, found that it did not delay disease progression in people with MND.

Describing these results of the chemical Creatine, Dr Rosenfeld (Carolinas Medical Center, USA) commented that “the patients felt better on creatine but it is hard to understand why”. Unfortunately he and his colleagues found no statistical differences in disease progression or muscle fatigue in this very preliminary analysis of the trial results. They are currently investigating whether creatine may be beneficial in a sub-group of ALS patients.

More hopeful trials

Dr Bob Miller (California Pacific Medical Centre, CA) and Dr Paul Gordon from Columbia University, New York presented more positive results on two preliminary trials of AEOL 10150 and Glatiramer Acetate respectively that have an anti-inflammatory action.

AEOL 10150 was found to be safe and well tolerated in the small number of patients receiving this novel drug. The results will allow the researchers to calculate the optimum drug concentration for larger trials in the future.

The aim of the Glatiramer Acetate (GA) study was to determine whether the drug was safe, and how frequently the drug should be given. Dr Gordon concluded that GA was safe and well tolerated given either daily or twice weekly and that the results supported plans for a larger study.

Smarter trials

One issue facing researchers in the search for more effective treatments is the time and numbers of patients needed to conduct trials – two commodities that are in short supply with MND. “A novel more efficient means of clinical drug screening is needed” commented Dr Gordon in his second presentation of the session. He suggested that this problem could be tackled by investigating the effects of many drugs simultaneously, going on to illustrate a novel trial design using a computer simulation. The following speaker Dr Dan Moore, from California Pacific Medical Center, San Francisco suggested another new approach, effectively turning the conventional drug design on its head. Instead of looking for new drugs that are better than the current treatment, this trial design eliminates new drugs that are worse than the existing treatments, known as ‘inferiority trials’. Analysing the data from a previous trial using this method he concluded “Inferiority trials result in substantial savings in numbers of people needed”. Subsequent longer and bigger studies can concentrate on drugs that are not eliminated in this way.