We are used to, almost accepting of, the “spin” we experience from politicians. Today we see the biotech spin-doctors get to work after Aberdeen’s flag ship biotech TauRx reported that its drug LMTX “failed to meet its primary end-points” in an Alzheimer’s patient Phase III drug trial.
In layman’s terms this means that the drug fell at the last hurdle of its 15 year research journey as it unfortunately did not work in the way it was hoped or expected.
Just like in politics there are some who might take some pleasure at this failure but for patients this is a very sad day indeed. It is also a sad day for the entire biotech sector in Aberdeen as, unlike other industries, when one of our own suffers a body blow we all feel it and sympathise.
It is worth taking a step back for a moment to remember what TauRx are trying to achieve.
Their goal is a drug that, for the very first time, treats and maybe even reverses this terrible disease, and not one that simply helps alleviate some of its symptoms.
The target for their drug was a protein called Tau; a protein that was originally considered “undruggable” by the Alzheimer’s research community. When they started their endeavours, TauRx were seen as mavericks by some and trail-blazers by others. Today many of the world’s largest pharmaceutical companies have Tau in their sights as a potential therapy for Alzheimer’s.
And so was the trial a failure after all? Frustrating I know for non-scientists but the answer is probably “yes” and “no”.
We do trials to try and confirm both the efficacy and safely of new drugs. Ideally what we want is a drug that works for everyone.
However, as we understand more about the biology of complex diseases such as Alzheimer’s and begin to realise just how different we are as individuals, we have to conclude that this is probably not achievable for many conditions.
In TauRx’s trial there are an exciting sub-set of patients that were at an early stage in their disease (mild to moderate) that responded exceedingly well to LMTX as a drug on its own (monotherapy) showing all the signs of a slowing in disease progression, with improvements in cognitive function and the brains’ appearance when scanned .
However, these patients were only about 15% of the total number of patients treated so far and so not enough to draw any firm conclusions.
We now have to wait for the results of TauRx’s second phase III trial which is expected later this year and is focussing on monotherapy of 800 patients with mild disease.
I think it is fair to say that even if we are not in the “last-chance-saloon” just yet, it is probably visible on the horizon.
However, what is happening to LMTX has happened for many successful drugs in the past, with a re-focussing post initial trial results to a patient population that fully benefits from a particular drug’s therapeutic effects.
Andy Porter is a professor of medical biotechnology and director of the Scottish Biologics Facility, the University of Aberdeen
