Fox Focus I PPMI Special Edition 9
Therefore , most trials of potential disease modifying medications in early PD follow patients for about 6 months to 1 year .
“ The critical time of about one year from when the patient can be diagnosed with early PD ... until they truly require symptomatic therapy can be considered their ‘ Golden Year ’ for participation in disease modifying clinical trials .”
patients in a clinical trial of a potential disease modifying medication as long as possible to give us the best chance of identifying separation of rate of progression between those treated with active medication and those treated with placebo . If the time period over which we test the intervention is short , we reduce our ability to identify a difference between the intervention and placebo . If the time period over which we attempt to test the medication is too long , a substantial proportion of patients may require institution of symptomatic therapy and we lose our ability to monitor clinical disease progression during the observation period .
But patients with early PD who are available , and able and willing to enroll in a clinical trial , and whose PD symptoms are mild enough to go 6 months to 1 year before requiring symptomatic treatment are in short supply ! To test promising potential disease modifying therapies , patients with early PD must be identified as early as possible and referred to centers where these clinical trials are being conducted before they go on symptomatic PD medications . Unfortunately , this process fails all too often . Patients frequently wait to make an appointment to undergo evaluation until their tremor , slowness , or rigidity are close to the point where they want symptomatic treatment . This is understandable because why see a doctor when you don ’ t need treatment ? Nonetheless , at this point it is too late to participate in an early PD disease modifying trial .
The critical time of about one year from when the patient can be diagnosed with early PD based on mild classic motor features until they truly require symptomatic therapy can be considered their “ Golden Year ” for participation in disease modifying clinical trials . It is critical that care providers and patients don ’ t unknowingly waste this golden year .
Editor ’ s Note : This article originally appeared in Nature Partner Journals Parkinson ’ s Disease on September 25 , 2018 . It was shortened for publication . Dr . Hauser is an MJFF-funded researcher whose areas of expertise include clinical trials design and execution , and evaluation of emerging medical and surgical therapies for Parkinson ’ s .