A World’s Worth Of Change in A Week

Three events in early June spotlighted the pace of change in how new treatments are being created.  The biggest winners from new approaches to drug development will be patients, who hold all the keys to how new treatments are found and tested.  The clearest losers will be policymakers, who will face more worries about meeting patients’ demands for the best and latest without bankrupting public programs.  And, in between – some will win, some will lose – will be scientists and drug makers, now able to target therapies in amazing ways, but the very same targeting for some will mean therapies don’t work for others.  (That’s probably true of some existing therapies too, we just didn’t/don’t really know who they really work for best.)

The first slew of news came from ASCO, the annual gathering of top cancer specialists.  Pfizer reported on an early stage drug that worked well on advanced lung cancers, though only for the approximately 4% of patients with a rare genetic mutation.  In melanoma, one of the deadliest (and hardest-to-treat cancers), scientists provided a glimmer of hope, reporting on the first treatment to meaningfully increase survival time.  New drugs for chronic myeloid leukemia (from BMS and Novartis) showed promise versus Gleevec, a wonder drug when it hit the market just a decade ago.  What links these great results is a growing sense that new cancer treatments may have less to do with where a cancer occurs than the kind of cancer it is.  One researcher compared the proliferation of information promising targeted therapies to the shift from four-channel network TV to 500-channel cable and satellite.

The second piece of news came from a large-scale study genetic study of those with autism spectrum disorders.  In this most confounding of brain disorders, where no clear genetic cause or combination of causes have emerged, researches decided to take an unconventional tack and try to see how wide the range of genetic mutations could be.  Underwritten by the NIH, Autism Speaks, and the Simons Foundation among others, the study generated provocative avenues of future research.  Surprisingly, the study suggested that a relatively high number of rare mutations associate with autism are not inherited.  Rare mutations involving deletion of genetic information seemed to be more highly correlated with autism than mutations that added extra genetic information.  Aggregating the data, researchers were able to map even rare mutations against genes known to impact how neurons form and grow, and connect to each other.

The third piece of news, from the world of Alzheimer’s research is a landmark success born of abject failure.  For years, drug companies large and small have watched seemingly promising anti-Alzheimer’s drugs fail in clinical trials.  So drugmakers have agreed to pool their “failures,” open-sourcing in effect their search for what went wrong in the trials – was it a common thread or threads among the patients or something awry in the basic medical theory underlying the way the drugs were supposed to work.

So what does this all mean?  With more refined knowledge of the way tumors grow and die, or neurons for that matter, we can build silver bullets, but probably not silver bombs.  It’s just not the way tough tumors and hard neurological diseases are.  Second, for policymakers, the way we price drugs will have to change.  Over time, drug prices will be less of a cost-plus activity, and more based on the incremental value of the discovery not just in patient outcomes like longevity, but the pathways opened to new research.  Drug pricing may come, as well, to reflect how long a patient has to take a treatment, in conjunction with programs that work to ensure compliance.  The front-end of drug discovery will get more efficient as well as “in silico” research replaces in vitro spade work.

All of this is cool.  But none of this is possible without patient involvement, or as we at HealthCentral think of patients (i.e. us) – real people.  Un fortunately this is not always the case in the medical establishment.  One of the industry leaders who assembled the new Alzheimer’s clinical trial alliance said innovation is being made possible by “scientists in the biopharma industry, patient advocates, academia and government.”  Yes, this is true, but the most important contributor is patients (and not just their advocates).  Tens of thousands of willing patients and their relatives offer the raw genetic information without which genome-wide studies would not exist.  Engaged patients are seeking information from all comers to understand how they can be well, rather than just feeling better.  And educated patients, literally dying to know if they have the right kind of condition to qualify for a silver bullet – even at the risk of much worse news – will force a much closer connection between the tests that will give definitive guidance, and the treatments designed for their situation.  The news of this week, like the larger trends of genetic and proteomic research herald the end of passive patients.  And, while this is mightily uncomfortable for the established order (government included), patient-driven demand for therapies that work offers the brightest possible future for researchers, manufacturers, and policymakers’ elected masters.  And yes, of course, for tomorrow’s patients too.