Empowered Patient Podcast Treating Rare Disease SAH Triggered by an Aneurysm with IV Infusion to Manage Blood Pressure with Jan D’Alvise Acasti Pharma
Jan D'Alvise is the President and CEO of Acasti Pharma, a late-stage specialty pharma company addressing rare and orphan diseases. The topic today is the treatment of the rare disease SAH, otherwise known as subarachnoid hemorrhage with GTX-104, a reformulation of the existing drug nimodipine. We also talk about reformulating and repurposing drugs for the rare neurodegenerative disorder ataxia-telangiectasia A-T that affects young children and for treating postherpetic neuralgia, the severe nerve pain that persists in some patients after they have a shingles infection.
Jan explains, "So an aneurysm in the brain is known as subarachnoid hemorrhage. Now you could have bleeding on the brain caused by other things, but if it's caused by a burst aneurysm, it's known as subarachnoid hemorrhage or SAH."
"So I mentioned that nimodipine is the standard of care today, and all patients who present with SAH are put on nimodipine post-surgery. The nimodipine is very potent, so you have to carefully monitor it. The problem today is that it's only available as an oral drug. Most of these patients are unconscious, so the nursing staff has to take a capsule, mix it with some liquid and actually give it to the patient through a nasogastric tube. So they literally put it through the nose, down the throat to deliver it into the stomach."
"What we've done is we have figured out a very clever way of solubilizing nimodipine, so providing it in a formulation that can be delivered via an intravenous infusion. These patients are very sick, they're in an intensive care unit, so they're all already on an IV. They're getting other drugs via IV, they're getting fluids, so it's very easy to hook our drug up to an IV."
"The key thing about it is because the nimodipine is then going directly into the bloodstream, you can get much tighter control of the amount of nimodipine that's actually going into the patient and being absorbed."
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