|
Getting your Trinity Audio player ready...
|
I just read a post on the PSP Blog of Dr. Lawrence Golbe, one of the world’s leading PSP neurologists, based at Rutgers Robert Wood Johnson Medical School, who has been writing this blog for clinicians, patients and families since 2014. In his latest post he pointed readers to a review article he co-authored, just published in Alzheimer’s & Dementia: Translational Research & Clinical Interventions.
The paper is principally about improving the trial measures used in PSP research, specifically how to reduce the number of patients needed to run a valid clinical trial, which is a chronic problem given how rare PSP is. Technical and important work. But buried within it, almost as a side remark, is a statistic that stopped me in my tracks.
PSP’s average survival of around seven years makes it only slightly more aggressive than Parkinson’s disease was before levodopa, when mean PD survival was just 9.2 years. Yes, 9.2 is still worse than PSP’s seven — but look at what levodopa did to that number. Today, people with PD routinely live for decades. That is the transformation one drug achieved.
I had to read that twice.
Parkinson’s disease today is rightly seen as a serious, life-altering condition, but also as one that is, to a meaningful degree, manageable. People live well with PD for decades. What changed everything was one drug: levodopa. Before it existed, PD and PSP were not so far apart in what they did to people.
When I spent my first four years believing I had PD, I was among the many who complained bitterly about levodopa, the wearing-off, the on-off fluctuations, the side effects. I had no appreciation of what I was holding in my hand. It is, whatever its imperfections, a wonder drug. Not a cure, but a transformation of what the disease means for the person living with it.
PSP has no levodopa. It responds at best modestly and briefly to dopaminergic medication, and nothing else has yet proven effective. PSP is more closely linked scientifically to Alzheimer’s disease than to PD, both involve the accumulation of misfolded tau protein, which is why PSP research is watched closely by the Alzheimer’s community. But the blunt human reality is this: one disease got its levodopa moment. The other is still waiting.
That gap, between a disease with a drug and a disease without one, is not just a medical statistic. It is measured in years of life, in falls prevented, in conversations still possible, in families less shattered.
I hope everyone working on PSP, the researchers, the trial designers, the pharmaceutical sponsors weighing whether the economics justify the effort, feels the full weight of that gap. One molecule changed what Parkinson’s meant. We are waiting for ours.
