The medical problems that get the most attention are often the most dramatic and scary - a rare infection or a tragic, but uncommon life-threatening disease. But there are a slew of very common but less serious problems for which there are no good solutions; one treatment would make a world of difference for many millions of people.
A year ago, a group of researchers at Massachusetts Eye and Ear Infirmary discovered that an experimental drug could regenerate hair cells in the inner ear that sense sound and restore rudimentary hearing when administered to deaf mice.
Albert Edge, an associate professor at Harvard Medical School and Mass Eye and Ear who led the original work, founded Audion Therapeutics, a Dutch startup company, to help bring the treatment to patients while he continued more basic research in his lab. Audion announced this month a deal with the pharmaceutical giant Eli Lilly and Co. that gives the startup the rights to develop certain compounds that Lilly has in its portfolio for use in treating hearing loss. Edge said it may be possible to have the first, small clinical trial testing the safety of a drug within a few years.
Meanwhile, he and his group have continued to try to untangle the basic science behind the process of regenerating the delicate hair cells. For years, it had been known that the hair-like sensory cells were regenerated in birds and fish, but not in mammals. The report last year showed that the cells could be triggered to regenerate in mice, and on Thursday in the journal Stem Cell Reports, the team reported new insights into precisely which cells are becoming the new hair cells.
Using cellular markers, the researchers were able to trace the cells that turn into new hair cells. That insight will probably not be useful in the work Audion is doing to try to develop a first-generation drug that could be tested in people, because that drug will not be specifically targeted to one kind of cell. But the detailed knowledge of how regeneration occurs could help guide future efforts to create therapies.
'This is really basic work that I think would help more in other future treatments or therapies that we're working on,' said Edge, who is pursuing a range of strategies, from traditional drugs to gene therapy.
Carolyn Y. Johnson can be reached at cjohnson@globe.com. Follow her on Twitter @carolynyjohnson.
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