Empowered Patient Podcast Treating Liver Disease by Tackling Membrane Protein Dysfunction with Positive Functional Modulators with Dr. Pol Boudes Rectify Pharmaceutical
Dr. Pol Boudes, CMO of Rectify Pharmaceutical, highlights the importance of membrane proteins, specifically the role transporters play in how cells interact with their environment. Rectify is working on developing positive functional modulators (PFM) to address diseases related to dysfunctional transporters the first being primary sclerosing cholangitis, a rare liver disease with no current treatment options. The PFM can potentially restore the normal function of transporters involved in bile composition and secretion, which are key mechanisms in PSC.
Pol explains, "So you have different types of membrane proteins, but what we're working with are called transporters, so they’re proteins that are based on the membrane but channel components out and in the cells. So basically, it's a way for the cell to interact with its environment, and you have multiple types of transporter proteins. We're specifically working or started to work with one group of family of proteins that are called the ABC transporters. They're a very important function, and they can either be disrupted because there is a mutation in the protein, so it's a genetic disease, or they are potentially disrupted because there is just a functional deficit of this transporter. The protein is normal, but it's not functioning properly. So it's a functional deficit of what we call the wildtype protein."
"So with the PFMs, what we're doing is to use small molecules that you can administer orally, and they bind to the transporter, and by doing so, they can modify the three-dimensional structure of the transporter and consequently the way this protein behaves within the cellular environment. So we correct the function of the transporter by changing the size, if you want, of the shape of the transporter. So we started to focus on this type of proteins and you have many potential diseases due to transporter deficit."
"We're focusing on one disease, liver disease. That's our lead PFM for a disease called primary sclerosing cholangitis, which is a disease of the liver. This very severe liver disease is also a disease that has no treatment available, which is a little bit frustrating because the progression of this disease leads to liver cirrhosis. And the only thing you can do at this stage is liver transplantation. As you might know, liver transplantation is problematic because it's a very complex process, it's also very expensive, and unfortunately, there is a shortage of transplants. So we're trying to address this problem."
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