What’s holding us back to dream big and do things differently?
With degrees in molecular biology and medicine, I have been involved in drug development for over 20 years. It has always been science-led, personal, a pursuit of innovation and driven with a sense of urgency. These tenants are why I became a Rare Disease Renegade.
Within my lifetime, I have witnessed tremendous technological advancements. I was born in 1970, within a year of the first lunar landing, yet this incredible national achievement occurred only sixty-six years after the Wright Brothers conquered the air with their novel flight at Kitty Hawk, NC. The pace of this remarkable innovation leapfrogged within one lifetime, and we continue to witness this exponential progression of national advancement as it transitions to private sector opportunities for space tourism.
Throughout my career, I have witnessed the speed of innovation in oncology. In the past decades, the decoding of the human genome has allowed the creation of multiple innovative medicines in cancer, new ways to modulate the immune system, treating not only the symptoms of this devastating disease, but also the underlying causes. With that knowledge, urgency, renegade mindset, innovative trial design, private and public partnerships, deep translational science, groundbreaking novel treatments for both common and rare cancers have been developed and delivered to cancer patients. The pace of progress has been equally remarkable.
I have had the pleasure of working closely with Michelle Werner, and we have developed mutual admiration and friendship over the years working together and sharing our personal lives with each other. Her guidance has been instrumental for me as I navigate life and work. Michelle’s family received a devastating diagnosis of the scourge of a rare disease that has impacted her young family, and my devotion to her has been mandated to comfort, aid, and innovate with urgency to harness these innovated tools to combat rare diseases. These uncommon diseases, with underlying changes in genes, are devastating and often present in childhood with life-changing and life-shortening consequences. Up until now, the treatments have often aimed to target the symptoms, as opposed to the underlying genetic causes of these diseases which are often specific to each patient. What if we could apply the same urgency, same vision, same “moon shot” approach, as we have done in oncology and in space travel? What’s holding us back to dream big and do things differently? Do things faster, better?
