Encouraging early data from trial of cancer drug on people with Parkinson’s

Largepill packetsOn the 17th October, the New Scientist reported exciting results from a six month study of twelve patients with Parkinson’s or Lewy Body Dementia taking an expensive cancer drug called nilotinib.  The article announced that scientists at Georgetown University Medical Centre in Washington had seen ‘fast improvement’ in the volunteers who were all at an advanced stage of illness.

Although the trial was designed to test the safety of using this drug, within a matter of a few weeks the patients commented they felt as though they had greater fluency in their speech and a lot more energy.

The results of this small open label study were met with considerable scepticism from certain Parkinson’s experts and commentators, but The Cure Parkinson’s Trust 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 are being considered for Parkinson’s trials within LCT for 2015/16.  The reaction from the charity’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 a flurry of activity over the weekend within 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.

‘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.’

Read the New Scientist article

RELATED ITEMS

  • At our patient advocate Rallying to the Challenge Meeting in 2014 it was unanimously agreed in the value of developing a clinical trials charter to significantly improve communication between those taking part in trials and those running clinical trials. The Charter was developed by people living with Parkinson's and clinicians…
  • The quiet revolution I think we all accept the notion that clinical trials are central to the development of new medicines and new treatments for Parkinson's. And it's also true that the majority of these will be conducted by the pharmaceutical industry or academic units. Both have a strong vested…
  • Parkinson’s Movement: Clinical Trial Town Hall Session June 2016 Atlanta;  July 2016 Saint Joseph University, West Hartford Connecticut; September 2016, Grand Rapids What are Parkinson’s Clinical Trial Town Hall Sessions? The “Clinical Trials Town Hall” sessions aim to increase the visibility and build awareness of Parkinson’s research and clinical trials…
  • Rallying to the challenge 2014 focused on how people with Parkinson’s (PwP) can strongly influence the effectiveness of clinical trials. Clinical trials are essential in assessing how effective or safe treatments are as well as advancing a greater understanding about a condition. The process of medicine development from test-tube to…
  • GRAND RAPIDS, Mich. (February 22, 2016)—A medication approved to treat various respiratory diseases and that has demonstrated neuroprotective effects in preclinical studies is the focus of a new clinical trial for Parkinson’s disease. The trial is the latest to be launched as part of the Linked Clinical Trials (LCT) initiative,…