Imagine the world’s most secure vault, protected by a seal impervious to methods used to break into other storage facilities. Beyond that, the vault is equipped with a self-destruct mechanism capable of destroying everything inside—or at least altering the contents to a point where they no longer function as they should; but we never know when or why the self-destruct will activate.
To scientists studying diseases of the brain, it can sometimes feel as if we are tasked with solving such a puzzle. Whereas in other areas of biology, blood samples and biopsies can illuminate how and why diseases develop and advance, brain chemistry is highly challenging to study in its live state. This has often left us unclear about where and what to target with drug candidates.
Diagnosis can be difficult as well, as some of the most common brain conditions, including Alzheimer’s and Parkinson’s disease, are only diagnosed through process of elimination. In other words, if a person shows symptoms of the disease, he or she must first fail several tests for other conditions before doctors make the final call. No single diagnostic test can provide a definitive answer.
It’s true we have made progress. For example, since our discovery of Parkinson’s disease more than 100 years ago, we have been able to develop treatments to help manage symptoms. But there still is no way to slow its progression, no known cure and no way to prevent it.
While the condition itself is not fatal, it is debilitating and can lead to complications that include tremors, stiffness, loss of balance and a host of mental side effects like apathy, depression and psychosis. According to the Centers for Disease Control and Prevention1, complications from the disease are the 14th cause of death in the United States, and the combined direct and indirect cost of Parkinson’s, including treatment, social security payments and lost income, is estimated to equal nearly $25 billion per year. With the aging of our population, the number of American’s impacted by Parkinson’s is expected to climb to 1.2 million by 20302.
While the stakes for the research community are high, I feel there is reason to be optimistic. Looking ahead, I believe we are on the cusp of a major leap forward in our understanding of Parkinson’s and how to treat it. Already, science has helped us find better ways to manage the breakdown of motor systems in patients; and today, we understand more about the mechanisms behind other disease-related symptoms, allowing us to develop treatments for those as well.
For decades we’ve been hard at work to understand Parkinson’s and develop better treatments, but a truly novel treatment has continued to evade us. However, our efforts have not been in vain. There are no “failures” in brain research; there is no such thing as wasted time. For every study that doesn’t work out, or every new drug that doesn’t meet its end points, we learn something that moves us forward. In recent years, we’ve greatly increased our understanding of biomarkers, genetics and the proteins that play a role in the progression of the disease. I have no doubt that soon, this understanding will lead to a discovery that will change the lives of patients for good.
Before joining the biopharmaceutical industry, I worked as a psychiatrist because I’ve always been fascinated by the brain. Every organ in the body has its own beauty and complexity, but the brain coordinates all of them. The brain houses our personalities, thoughts and our identities. This can be easy to take for granted until it breaks down, as is the case in Parkinson’s, Alzheimer’s or any number of other brain diseases like schizophrenia or bipolar disorder. I’ve dedicated my career to studying these diseases so we can find new treatments that improve the lives of people for whom these conditions are a daily reality. I will not stop until we do.
 Centers for Disease Control and Prevention, National Vital Statistics Reports, July 2018; Vol 67, No 5. Accessed 4/15/19
 Parkinson’s Foundation, “Statistics” Accessed 4/2/19
Image courtesy of NIH Image Library, Viviana Siless, PhD., Anastasia Yendiki, PhD., MGH/Harvard