Developing a drug for a disease is more complicated and arduous than you may think. It all starts with basic research on how the body works and uncovering the functionality of specific cellular pathways. Without that initial framework, it is difficult to see what problems the disease causes, and how to prevent them.
What we call a drug can be any number of things, such as molecules that block binding of receptors, or those that activate genes to overcome or bypass the problem, or a drug can be an artificial supply of a missing or inactive enzyme. Once a solution has been identified, it must be tested to prove effectiveness in the laboratory, and then in an animal model, and eventually in humans. There is generally a step in this process in which the expertise transitions from the scientist familiar with the disease and the corresponding drug, to a team that understands how to get that drug into the hands of those who need it. This commercialization step is really where the ACC Bioscience Incubator gets involved. It is extremely expensive and complicated, so generally one really has to have a strong motivation to go down this path. For Alice McConnell, founder of Speragen, that motivation is her two children, both born with the same rare metabolic disorder. Finding a way to get a known drug out of the research lab, so that her kids and those in her close network of affected families can use it, has become Alice’s mission since leaving her profession and founding Speragen several years ago. Listen as she shares what that journey has involved, from understanding the science, researching regulations, writing grants, and the process of getting the disorder added to the newborn screening panel, all while advocating for fellow families and being a mother to two kids with extensive health issues. What you will hear is someone who approaches difficult tasks with tenacity and focus and, most importantly, a positive attitude with a great amount of dedication and love.
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