GDNF treatment in Parkinson's disease: time for controlled clinical trials?

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Abstract
Restorative treatments for brain disorders are rare. In neuroscience research, it is not uncommon to suggest that experimental treatment strategies may have great clinical potential. The rescue or regeneration of a few cultured neurones may be sufficient to entice such optimism. However, the path to a new clinical therapy is typically painstakingly long and difficult to navigate. In our minds, we would like it always to be a straight path starting with tests in cell models and rodents providing us with mechanisms of action and leading to trials in non‐human primates. Then open‐label tests can be performed in small patient groups and eventually large controlled clinical trials are conducted. In reality, however, the path may be tortuous, filled with detours that set the field back, as well as shortcuts and parallel tracks that yield different strategies which develop at their own speed. The development of glial cell line‐derived neurotrophic factor (GDNF) as a treatment for tgciq’s disease over the past nine years provides an example of such an interesting journey. Moreover, it illustrates one role of non‐human primates in preclinical development of a novel therapy.

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