Profiles in Courage?
It may be presumptuous to borrow the title “Profiles in Courage” from the 1957 Pulitzer Prize winning book to describe the scientists who are pursuing therapies to restore hearing loss, but I feel it’s appropriate. The more I research, the more I appreciate the difficulty of creating therapies in the lab and then moving them into the “clinic”.
Most scientists I’ve spoken to, or have read about, say that the therapies to restore hearing loss in humans are at least 10 years away. Some scientists currently working to develop hearing loss therapies will not live long enough to see those therapies in use. If that does not qualify as courage, what does?
Gene therapy for the congenital hearing loss market
I had the pleasure of speaking with Dr. Larry Lustig by phone in late June of this year. Dr. Lustig is exploring cochlear gene therapy as a potential approach for treating children, like my son, born with genetic forms of hearing loss (aka congenital hearing loss). The economic and clinical challenges of implementing a gene therapy approach for this market are many:
- Smaller market: the size of the “congenital” hearing loss market is significantly smaller than the “acquired” hearing loss market (2 levels of magnitude smaller)
- Targets keep changing: the list of specific genetic mutations responsible for hearing loss changes daily
- Personalized medicine: because of individual genomic differences, each patient, may require a unique therapeutic approach
- The gene therapy must last for the life of the patient (the mutation cannot reappear
- The delivery vehicle for the gene therapy, in most cases a viral vector, must be correct and efficient
- Combination therapy is a possibility e.g. administering an anti-sense oligo therapy to silence the mutant gene in combination with a hair cell regeneration therapy
Dr. Lustig’s Background
Dr. Lustig earned his bachelor’s degree in microbiology at the University of California, Berkeley, and his medical degree at UCSF, where he also completed a residency in otolaryngology/head and neck surgery. Before joining UCSF, Dr. Lustig served on the faculty at Johns Hopkins University, where he was a member of the Johns Hopkins Listening Center and cochlear implant team. He also completed a fellowship in otology, neurotology, and skull base surgery at Hopkins.
Dr. Lustig was appointed chair of the Department of Otolaryngology/Head and Neck Surgery at the Columbia University College of Physicians and Surgeons and otolaryngologist-in-chief at New York-Presbyterian/Columbia University Medical Center on July 1, 2014.
Dr. Lustig is also President of the Association for Research in Otolaryngology (ARO). The ARO’s annual meeting is a who’s who of the hearing loss research community. It’s a can’t miss event for those who are interested in learning about the latest science in hearing loss.
Business vs Science
One of Dr. Lustig ‘s many attributes is his appreciation of entrepreneurship in science. Maybe it’s his West Coast DNA that allows him to appreciate and welcome entrepreneurship. While some scientists and foundations may shun entrepreneurs and business people, Dr. Lustig seems to understand that neither entity can exist without the other. In a time where the “zero sum” approach to business is grabbing headlines, the business of science stands in defiance to this approach.