Genes, Medicine and Parent Power: Lorenzo’s Oil
I learned about the movie, Lorenzo’s Oil, from my friend this summer, but only watched it recently. It is based on a true story of two parents’ relentless and ultimately successful quest to find an intervention–if not a cure–for their son’s rare genetic “error of metabolism” that was causing his brain to degenerate.
The thrust of the movie centers around the interplay between individual parents, organized groups of exceptionally motivated lay people (parents of involved and dying children) and the necessarily methodical, underfunded and coldly-objective scientific community as they all sought a cure, in this case for adrenoleucodystropy–an “orphaned disorder” too rare to be of financial interest to the pharmaceutical companies.
My old friend mentioned this movie and suggested I watch it to better understand the work he is involved with for the past three decades since he and I left Auburn. The June day I flew out of The City from my visit and our biking and kayaking excursions, he accepted the position as director of a prominent NYC developmental disabilities research facility in addition to continuing to head an associated genetic neurological disease clinic.
The work his group does centers on genetic disorders common in one ethnic group, but the discoveries they have made and will make solve problems over a wider biology of disease, and will give back the lives of countless children–and their parents.
The work this team of scientists is doing in collaboration with a well-organized and generous group of parents has some unique strengths, and could become a model for sufferer-scientist synergies that will work in powerful and effective ways into the coming decades.
I understood that from our long conversations in June. Now, I understand it much better. Good work, my friend.