Is a cure imminent?

Short answer:  No.

Longer answer:  The popular press, even reputable institutions like NPR and the New York Times, periodically put out breathless pieces with a title like “Scientists at [some academic institution] have made a breakthrough that could lead to a cure for Parkinson’s disease”.  As I write this in October 2015, there have already been several such outbreaks this year; here are some of the actual titles (each referring to a different study):

“People with Parkinson’s walk again after promising drug trial”

“Harvard-Singapore team unveil potential Parkinson’s cure”

“Stem Cell Breakthrough May Be the Cure for Parkinson’s”

You could find many such articles in the year 2000, and of course none of those potential cures has panned out.  [I discuss the use of the word ‘cure’ with respect to Parkinson’s here.]

The press is well within its rights to write these hopeful articles, as long as they don’t cross the line into outright misrepresentation of the facts (which also happens).  I also don’t begrudge the scientists and clinicians involved the right to be optimistic about the future impact of their work; optimism is almost required to take on really difficult problems.  [Indeed, I sometimes know people involved in the studies being referenced and most are excellent scientists, including in the case of one of the titles listed above.]  There is indeed a great deal of promising work going on, and there is never anything wrong with hope.  As a patient myself, I very much want these headlines to come true.

That being said, this site is dedicated to the science behind Parkinson’s, and so I want to give some perspective on how to evaluate these claims, which range from hopeful extrapolations of real scientific progress, to complete nonsense.  I won’t focus on how to identify bunk (I’m looking at you, Dr. Oz); there are some excellent blogs (e.g., scibabe) which illustrate how to identify garbage ‘science’.  In the case of reports of actual scientific progress, the key first step is to contextualize the work in the long and perilous process from discovery to any therapeutic advance.  There are a number of nice overviews available, such as from the Phrma foundation and from the FDA.  The first distinction is whether the potential ‘cure’ has entered clinical trials, or is still under investigation in the laboratory.  Many of the stories on potential cures are actually in the latter category; needless to say, such discoveries, although possibly quite scientifically interesting, are almost always many years away from any potential impact on patients.

Once a potential drug has entered clinical trials, there are multiple stages of testing to ensure that the drug is safe, first and foremost, and that it has clinical benefit.  The FDA generally requires rather high standards of proof before they will consider approving a drug, and the vast majority of potential drugs fail at some stage.  A lot of ink has been spilled on why this is the case, but the most important reason is that it is really really hard to develop safe and effective drugs.  The first tests of safety are “Phase 1” clinical trials, often in healthy volunteers, but sometimes in patients as well.  [A little over 10% of drugs entering phase 1 studies are ultimately approved.]  Normally, you don’t even look at whether the drug works at this stage, just whether there are side effects, what the maximal tolerated dose is, etc.  But there is nothing to prevent companies from looking for a hint of efficacy in patients in even these very early clinical trials, which can then generate the attention-grabbing headlines.

Or in the case of Biogen, for example, newly reported phase 1 clinical trial results for an experimental Alzheimer’s therapy caused the market cap of the company to increase by >$3 billion.  Alzheimer’s is a huge unmet medical need and thus there is a huge potential windfall to any company that develops a drug that actually slows the disease, even modestly.  Four months later, additional data from the study looked less promising, and the stock gave back the gains.  All of this based on phase 1 results (although, in fairness, it was a relatively large and well-designed phase 1 study; I’ve seen breathless headlines based on much, much less).

When potential therapeutics start to generate promising results in phase 2 or phase 3, much larger studies aimed at demonstrating both clinical benefit and safety, then headlines about ‘potential new drug’ are more reasonable.  [I’m being generous; hardened veterans of drug discovery know that many promising results in phase 2 don’t confirm in the larger, definitive phase 3 studies.]

Again, I’m not advocating for headlines that read “Breakthrough discovery has <1% chance of generating a cure”, or “Twenty fascinating studies this year together have collectively moved the Parkinson’s field forward”.  But it would be nice if the press focused more on the truly exciting science going on, rather than meaningless claims about ‘potential’ cures.

[This phenomenon is by no means restricted to Parkinson’s disease; see this JAMA Oncology article for an analysis of news articles about cancer drug candidates.]