On the 17th October 2015, the NewScientist reported exciting results from a six month study of eleven patients, with advanced Parkinson’s disease; Parkinson’s disease with dementia; or Lewy body dementia, taking an expensive cancer drug called Nilotinib.  The article announced that scientists at the Georgetown University Medical Centre in Washington had clinically meaningful improvements in the volunteers who were all at an advanced stage of the disease. 

Although the trial was designed to test the safety of using this drug, within a matter of a few weeks the patients were commenting they felt as though they had greater fluency in their speech and a lot more energy - 'Nilotinib Effects in Parkinson’s disease and Dementia with Lewy bodies'

The results of this small open label study were met with scepticism from certain Parkinson's commentators, but The Cure Parkinson’s Trust (CPT) has already adopted a proactive and positive plan in the wake of the news of these results.  In 2013, Nilotinib was shortlisted as a potentially disease-modifying drug within its Linked Clinical Trials (LCT) programme.  Although the drug was not prioritised in that year, a number of similar cancer drugs such as Rucaparib and Axitinib are being considered for Parkinson’s trials within LCT for 2015/16.  The reaction from CPT’s LCT committee comprising a group of the most eminent Parkinson’s experts in the world was far from sceptical.  While cautious due to the lack of substantial data available and the need for more robust clinical evidence of the effect, the news from Georgetown prompted great activity amongst CPT’s scientific team. 

CPT is responding as quickly as it can to establish whether the use of Nilotinib in Parkinson’s can really change the course of the condition so dramatically as purported by those involved with the trial. 

For further information on outcomes of the trial, please refer to the online article in Oncology Times 17th November 2015.

“It’s easy to be cynical and negative about small-scale studies which don’t have a placebo controlled element”, said Tom Isaacs, President and Co-founder of The Cure Parkinson’s Trust, “but the nature of the improvements experienced by the people on the trial were so marked, it seems to me unlikely that these positive results could be attributed solely to a placebo effect.  As a patient-led organisation,  we prefer to take the standpoint that even the most anecdotal data should be followed up and scrutinised as closely as possible to ensure an opportunity for a breakthrough in the treatment of Parkinson’s is not being missed.  Nilotinib is a case in point.  When people with Parkinson’s volunteer for clinical trials and feel the type of marked improvement to their symptoms and wellbeing as those being reported in this study, we owe it to them, and indeed everyone, to follow the results up as quickly and as rigorously as possible.”