Sometimes more is not necessarily better. This may especially be true of Alzheimer’s disease clinical studies, too many of which have been tried and failed, leading top scientists to question their design and execution. Better planning in the application of more promising approaches might make more sense — and improve a trial’s chance of success.
This concern was the topic of a recent symposium in Barcelona, Spain, where an international group of experts from academia and industry, as well as the editors from The Lancet journal, asked: Is there simply too much wasted effort in current Alzheimer’s clinical trials? The symposium, part of the 8th International Conference on Clinical Trials for Alzheimer’s Disease (CTAD), took place on Nov. 6.
Lon S. Schneider, MD, professor of psychiatry, neurology, and gerontology at the University of Southern California, moderated the discussion on disappointing Alzheimer clinical trial results. “There has been considerable concern about the lack of success in clinical trials in Alzheimer’s disease and the performing of very, very large trials in an effort to detect small signals; or moving from phase 2 to phase 3 trials absent a proof of concept,” said Dr. Schneider.
There are many points at which a clinical trial can go wrong, from an idea’s inception and the start of basic research through to its clinical study phases. Rustam Al-Shahi Salman, Professor of Clinical Neurology at the University of Edinburgh, noted in a press release: “Waste arises from questions being overlooked or unnecessarily addressed, research being underpowered or done too slowly, and research being too costly. Ultimately, these problems are a threat to public health — they cost people their lives through a failure to identify and introduce effective treatments and prevent harmful treatments from continuing.”
Dr. Schneider also pointed out that failed clinical trials risk doing harm to the researchers conducting them, the groups or companies funding them, and the patients participating in them. He and others noted that improving a trial’s efficiency and value should lead to more transparent and optimal outcomes, helping ensure that participants benefit from a trial’s outcome.
Other notable experts who shared their insights included Professor Malcolm Macleod, from the University of Edinburgh; Elisabetta Vaudano, of the Innovative Medicines Initiative (IMI); Rachel J. Schindler, MD, Pfizer; José Luis Molinuevo, MD, from the Hospital Clinic in Barcelona, Spain; and two Lancet editors, Sabine Kleinert and Elena Becker-Barroso.
About 5.3 million people in the U.S. have AD and an estimated 473,000 individuals will develop the disease in 2015. Five FDA approved drugs are available to treat the disease, however, these medications only marginally delay symptoms and can neither cure the disease nor stop its progression. The development of novel therapies is therefore critical. Hopefully, this symposium will encourage new perspectives regarding Alzheimer clinical trials.