Empowered Patient Podcast Developing Small Molecule Compound to Manage Rare Disease Classic Congenital Adrenal Hyperplasia with Javier Szwarcberg Spruce Biosciences
Javier Szwarcberg, CEO of Spruce Biosciences, is developing a compound to address the needs of patients with classic congenital adrenal hyperplasia, CAH, a rare disease first diagnosed at birth. Only recently have companies started to explore innovative therapies to improve the standard of care discovered in the early 1960s. Currently, patients manage their conditions with glucocorticoids but often suffer from steroid-related complications.
Javier explains, "What we're trying to do with our drug is to treat the disease. The compound is a small molecule that binds to a receptor in the pituitary and blocks the release of a hormone called ACTH or adrenal corticotropin hormone. By blocking that hormone, you reduce the amount, the stimulation on the adrenals, and the buildup of precursors that are going to lead to a hyperandrogenic state. So our drug treats the disease. Patients, of course, will still need to get steroids daily to supplement the missing cortisol. But the CAH piece of the disease will be well managed. That's the hope."
"The whole pharmaceutical industry and academic community are starting to pay more attention to rare diseases. And back when I started my career in industry, it was all about addressing the totality of a population with, say, hypertension or diabetes or cancer, for that matter. Now there's a realization that companies ought to be more thoughtful, and research ought to be more thoughtful and address the needs of a subset of patients with rare diseases with more kinds of patient-targeted therapies."
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