Two weeks ago, 15,000 people from around the world gathered at the annual BIO International Convention in San Diego. BIO was a four-day affair that included hundreds of panel discussion, tens of thousands of business partnering meetings, an exhibition hall with hundreds of companies, state and national exhibits, pavilions and not a few receptions. With the whirlwind of activity, it can be difficult to discern what should be the central focus of such a convention – the patient.
Not so in the Massachusetts Pavilion. Annually developed by MassBio in concert with leading sponsors like the Massachusetts Life Sciences Center, the thematic focus in the Massachusetts Pavilion is the patient. Front and center are the vivid photos of patients and the stories of how their lives have been positively impacted by products and services developed in Massachusetts. For all of the hundreds of panel discussions that took place at BIO that week, none was more compelling than one featured in the Massachusetts Pavilion on the very last day of the convention. It featured four men, each a leader of a biopharmaceutical organization and each a dad with a child suffering the ravages of an “orphan disease.” Orphan diseases are those which affect a small population, by definition less than 200,000 in the U.S., but often much smaller groups.
Daniel Fisher is the founder and CEO of Intellimedix, which is working on neurological disorders including Davets Syndrome, a catastrophic pediatric epilepsy affecting between 7,500 and 15,000 children in the U.S. Ilan Ganot is the founder and CEO of Solid Ventures, focused on acquiring and developing therapies for Duchenne Muscular Dystrophy, which affects one in 3,600 boys. Benjamin Seckler heads Charley’s Fund, also focused on Duchennes. The moderator of the panel was Bob Coughlin, who is the CEO of MassBio and the father of a child with cystic fibrosis, a genetic disease affecting the lungs, with a patient population of 30,000 children and adults in the U.S.
Beyond These Four Walls
An hour with these four dads was time well spent in better understanding just how important the research being conducted in the life sciences from Worcester to Woburn to Kendall Square is. For each, it’s a race against time to find cures for his child. Each father has altered his life and profession to focus not on making money for their organization, but on raising money to fund research toward cures. For each, making the business of life sciences more efficient isn’t about making more profit; it’s about getting life-extending and life-saving therapies to the bedside faster.
For them, making the Food and Drug Administration work better isn’t about approving yet another erectile dysfunction drug; it is about fast-tracking clinical trials for investigational drugs aimed at serving the smallest, rather than the largest, of patient populations.
These dads spoke to the biggest problems facing the drug development industry and the provision of health care in a way not often heard. There were a few barnyard expressions thrown about, to be sure, but what was revealing and rewarding was hearing the profound optimism each had for the future, for their children and the pursuit of the new therapies that may someday cure them. The concepts of business risk and uncertainty are not hurdles when one is focused on patient populations that can’t wait for someone else to find a cure. They’ve taken on the jobs themselves, come what may, and are not accepting “no” as an answer.
Massachusetts is home to several leaders in the development of therapies for orphan diseases, including Genzyme, Biogen Idec and Vertex. The reputation of this cluster in orphan diseases has encouraged the entry of new companies to the scene. Daniel Fisher moved Intellimedix from Florida to Cambridge. Ilan Ganot located Solid Ventures here despite a background in Israel and New York. Benjamin Seckler’s Charley’s Fund provided support for Cambridge’s Sarepta Therapeutics, an arrival from Seattle.
Our region’s strength in orphan research is one in which we can be proud, but not yet satisfied. It’s why we are grateful for the efforts of the dads of Charley, Eytani, Natasha and Bobby. In business and in policy, when we focus on the patient first, the reward is progress – and eventual breakthrough cures.
Peter Abair is director of economic development and global affairs at the Massachusetts Biotechnology Council (MassBio).
