Thursday, October 10, 2013

A hopeful headline--and a spur to action

"Alzheimer’s treatment breakthrough: British scientists pave way for simple pill to cure disease"--that's the headline in The Independent (UK). 

Then, the fine print, as it were: "Although the prospect of a pill for Alzheimer's remains a long way off, the landmark British study provides a major new pathway for future drug treatments."  

And that should be our spur to action.

UPDATE: Jeremy Shane, a long-time medical observer living in Washington DC, adds these sage comments:

"First, it is critical for government funds to support ideas that seem out of the mainstream where mainstream ideas continue to disappoint.

"Second, figuring out a drug that "works" is only half the battle -- we need funds to figure out how to deliver drugs only to those places where they will be useful and not to places where they cause side-effects.  

"On the first point, Alzheimer's research for decades has focused on two competing causes of neuronal failure -- accumulation of beta-amyloid protein tangles or the malfunction of a neuronal protein called tau.  AD, they agreed, seems to happen when neurons stop "taking out their trash."  

"Then, along come a few out-of-the-mainstream scientists who look at the lack of real progress with either of these approaches, and say, you know, the way AD seems to spread in the brain sure looks like a prion disease, like Mad Cow's disease.  It's fair to say mainstream AD researchers laughed at them.  Certainly advocates of the prion idea got the Rodney Dangerfield treatment when it came to NIH grant applications.  But little by little, the "prion-like" advocates piled up studies until brain imaging studies were able to show that AD spreads from one brain region to the next pathogenically, much like a prion disease (even if the scientists couldn't point to an actual prion protein causing it).  

"So here is this UK article on an AD breakthrough and the two main theories of how AD is caused, theories that have garnered tens of billions of research dollars, aren't even mentioned.  What's more, it suggests to the reader that the prion thesis as a basis for AD research is pretty well established.  

"So, this is a good reminder that even science -- among scientists -- is political, and that consensus can -- must -- change when facts change.  Facts win.  So it is incumbent on funders, especially government funders, to create systems that allow unpopular ideas to get tested especially in areas like AD where time and again, consensus ideas yield paltry results.  (NIH does have a EUREKA grants program that is supposed to fund these out of the mainstream ideas, but its funding is quite small in the larger scheme of things).

"Second, discovering new drugs -- like discovering new explosives in the military -- is a necessary first step.  But they are of little value if you can't reliably deliver them on target without collateral damage.  This work on AD reinforces that challenge -- for researchers and funders.  This compound was delivered orally and was able to the brain.  Great, but it also messed up other parts of the body.  Drug delivery in AD -- finding new ways to deliver drugs directly into the brain and only the brain -- will be as important to a treatment's success as the active ingredient."