The pharmaceutical industry gets a bad rap…
They are an easy scapegoat. Incredibly wealthy and powerful faceless entities that seem hell-bent on squeezing every penny they can out of desperate patients. While there are plenty of stories of unscrupulous practices from industry, they are not the bad guys that so many paint them to be.
I have visited dozens of pharmaceutical and biotech companies, often spending entire days touring their facilities and getting to know the people who work there. Most have the best of intentions and are sincerely trying to help individuals afflicted by the diseases they have devoted their lives to. They are constrained by market forces and investor interests, but they do what they can within those confines to bring therapies to market.
The important question is, what does it take to get something to market? What goal do they have in mind when they try to develop drugs that can alter the progression of Parkinson’s or Alzheimer’s or many other neurological conditions?
Well, it is actually pretty simple. Essentially they are trying to go from this.. 
To this…
The green line represents the pace of degeneration for the average person diagnosed with a brain disease. It typically starts from the time of diagnosis going to death or crippling disability. Pharma’s goal is to develop a profitable therapy that can extend that green line by at least 20%, the amount typically needed to be better than placebo. (exact figure varies) If they can demonstrate, in a phase 3 clinical trial, that their therapy can extend that line by the necessary amount, boom, success. The stock price of the company would jump, huge bonuses would be given, and champagne bottles would pop all through the night in company offices around the world.
While this would certainly be welcome news to all, the celebration coming from the patient community would be a little more subdued. Let me explain. (I’ll use Parkinson’s as my example because it’s the one I know best, but similar things can be said about most degenerate brain diseases.)
One myth often told in the Parkinson’s community is ‘you don’t die from Parkinson’s you die with Parkinson’s.’ This is bogus, especially for young-onset cases. A diagnosis of Parkinson’s disease is a prolonged death sentence; a slow, grueling, torturous, drawn out death sentence. And while we may be given 15-25 years to live after diagnosis (rough figure, stats vary), those aren’t exactly 15-25 pleasant years.
One way of putting this is using quality of life scales. Here is what a simplified QOL graph looks like for the average person diagnosed with Parkinson’s disease.
The first straight line represents the time from diagnosis to when one starts levodopa (the most effective symptomatic therapy we have). It is important to remember that each case is different, but it typically takes 1-3 years after diagnosis for a patient to start taking levodopa. After which there is a significant bump in quality of life. However, as the disease progresses, levodopa becomes less and less effective and has its own side effects that start to impact QOL. This period can be anywhere from 2-10 years. This is generally followed by the “last line of defense”, deep brain stimulation surgery for those who qualify. DBS typically results in another bump in QOL, but over time complications arise as the disease progresses, all sorts of motor and non-motor symptoms pop up that we do not have ways to properly manage, and eventually life itself becomes a daily struggle.
So, while all would welcome a 20% boost in the rate of decline, patients tend to have more ambitious goals in mind. For many of them success would look a little more like this…
Though many would also settle for this…
While some might say that we need to crawl before we can walk, we also need to ensure that we keep the end goal in mind. If the day ever comes when a drug does hit that 20% mark, there will be far more from champagne bottles being popped in industry offices around the world than in patient homes.
“The greater danger for most of us isn’t that our aim is too high and miss it, but that it is too low and we reach it.”
– Michelangelo
(Also important to note: the first successes are most likely to only be effective for a small subset of people with the disease.)