Clinical trials are essential to improving the care of PAH patients. These clinical trials allow us to determine if new medicines are safe and effective. Clinical trials also allow us to make improvements to existing treatments. For example, we are eagerly awaiting the approval of the implanted pump for intravenous Remodulin.
Another example of a company that is trying to improve the delivery system for an already approved PAH drug is Liquidia. They have just started a new clinical trial that looks to improve the way that inhaled treprostinil (Tyvaso) is delivered. The current delivery system is not very portable and requires 5-10 minutes of time to do each treatment. The active medicine is structurally identical to Tyvaso.
Liquidia uses a proprietary technology to attach treprostinil molecules to small carrier molecules. The linked molecules are then placed inside a small capsule. The capsule is then placed inside a small inhaler device. Just before use, the device pokes 2 small holes in the capsule. The patient then inhales through a mouthpiece. The tiny linked carrier-treprostinil molecules are inhaled into the airway where they are deposited. The active treprostinil is then separated from the carrier and is absorbed into the blood stream.
If approved by the Food and Drug Agency, I am optimistic that this delivery system will allow patients much greater flexibility and reduce treatment times dramatically.
If you are interested in participating in a clinical trial, contact your PAH Center of Excellence. Stay tuned for more updates on exciting clinical research trials for pulmonary arterial hypertension patients.