January 9, 2017

News to look forward to

Last year, we had a bunch of early-stage Alzheimer’s trials in the news. I thought I’d look at what’s due out in the clinical trial world this year.

Perhaps most importantly, in March we should see the first real results on a new set of cholesterol-lowering drugs.  The ‘PCSK9’ inhibitors are one of the first drugs outside the cancer world to come from large-scale genetic studies without a particular hypothesis in mind. As the gene name ‘PCSK9’ indicates to those in the know, the gene was originally named just as the ninth in a series of genes that looked similar in structure.  It turned out that mutations in PCSK9 had big effects on LDL (‘bad’) cholesterol levels. Also, importantly, there is at least one person walking around alive and healthy with disabling mutations in both her copies of the gene — so there was a good chance that inhibiting the protein would be safe.  At least three companies have drugs (monoclonal antibodies) that target PCSK9 and reduce cholesterol by a lot; though the drugs need to be given by intravenous injection.

Although the drugs have been shown to reduce cholesterol, and have been approved for sale in the US for people with very high cholesterol not otherwise treatable, they haven’t been shown to prevent heart attacks (which is the point of lowering your cholesterol). The first trial looking at that sort of real outcome has finished, and there’s a good chance the results will be presented at the American College of Cardiology meeting in March.  For people in NZ the main interest isn’t in the new treatments — it’s hard to see them being cost-effective initially — but in the impact on understanding cholesterol.  If these drugs do prevent heart attacks, they will increase our confidence that LDL cholesterol really is a cause of disease; if they don’t, they will give aid and comfort to the people who think cholesterol is missing the whole point.

What else? There are some interesting migraine trials due out: both using a new approach to prevention and using a new approach to giving the current treatments.  The prevention approach is based on inhibiting something called CGRP in the brain, which appears to be a key trigger; the drug is injected, but only every few months.  The treatment approach is based on a new sort of skin patch to try to deliver the ‘triptan’ drugs, which they hope will be as fast as inhaling or injecting them and less unpleasant.

Also, there’s an earlier-stage New Zealand biotech product that will have results early in the year: using cells from specially bred pigs, coated so the immune system doesn’t notice them, to treat Parkinson’s Disease.



Thomas Lumley (@tslumley) is Professor of Biostatistics at the University of Auckland. His research interests include semiparametric models, survey sampling, statistical computing, foundations of statistics, and whatever methodological problems his medical collaborators come up with. He also blogs at Biased and Inefficient See all posts by Thomas Lumley »