Five potential treatments for PKD in clinical trials

Since 1982, we have led the fight against PKD through vital research funding and patient education. Today, we are encouraged by the significant strides we are making to find treatments. We have gone from a single drug in clinical trials five years ago to five drugs in trials today. Four of these drugs are considered repurposed, which shaves off millions of dollars and several years to bring them to market if they are proven effective for the treatment of PKD. Read on to learn more about the five potential treatments for PKD currently in clinical trials.

Metformin

What it is
Metformin is a first-line, FDA-approved drug to treat type 2 diabetes.

Why it may be a potential treatment for ADPKD
Mouse models have shown that metformin blocks the aerobic glycosis pathway, which is linked to cell proliferation, the process that causes cysts to form. When treated with metformin, mouse models show inhibition of cyst growth. Metformin has an established safety profile because it has already been approved by the FDA for type 2 diabetes.

Watch our recorded webinar, PKD therapies and potential candidates, to learn more.

Clinical study status
Researchers at Tufts University in Boston and at the University of Maryland in Baltimore are currently recruiting patients for this study. Learn more and check if you’re eligible to participate in this study.

Niacinamide

What it is
Niacinamide is a form of vitamin B3 available over the counter. It is used for treating diabetes and certain skin conditions.

Why it may be a potential treatment for ADPKD
Niacinamide has been shown to inhibit a protein enzyme called sirtuin 1, which promotes cell growth and may promote cyst growth in PKD. Inhibition of sirtuin 1 may slow or stop cyst growth.

Niacinamide may be an appealing treatment option for PKD because of its low cost, safety profile and ease of access.  The European Nicotinamide Diabetes Intervention Trial (ENDIT) study showed that large doses of of niacinamide are safe in humans. Additionally, niacinamide is a dietary supplement, and therefore does not require approval by the FDA.

Clinical study status
Preliminary results from the ongoing Uncontrolled, Open Label, Pilot and Feasibility Study of Niacinamide in Polycystic Kidney Disease (NIAC-PKD1) show that niacinamide is safe and well tolerated in humans. The Randomized, Controlled Pilot Study of Niacinamide in Polycystic Kidney Disease (NIAC-PKD2), which is being conducted at the University of Kansas Medical Center, aims to observe the effects of niacinamide on markers of kidney injury, inflammation, kidney cyst growth and kidney function. This study is currently recruiting patients. Learn more and check if you are eligible to participate in this study.

Watch our recorded webinar, PKD therapies and potential candidates, to learn more.

Pioglitazone

What it is
Pioglitazone is an FDA-approved treatment for type 2 diabetes.

Why it may be a potential treatment for ADPKD
Pioglitazone has been shown to inhibit chloride secretion into cysts, which is a key factor in cyst growth. When chloride moves into the cyst, water follows, thus filling the cysts with fluid and causing them to grow. By inhibiting chloride from moving into cysts, pioglitazone may potentially slow or stop cyst growth. Pioglitazone may also inhibit cell proliferation, the process that causes cysts to form.

Watch our recorded webinar, A discussion about pioglitazone Actos as a potential therapy for PKD, to learn more.

Clinical study status
Researchers at Indiana University are recruiting patients for a pilot clinical trial to test the safety and efficacy of low doses of pioglitazone on ADPKD progression. Learn more and check if you are eligible to participate in this study.

Tesevatinib

What it is
Tesevatinib is a new drug that is a tyrosine kinase inhibitor designed to block key molecular drivers of tumor growth, metastases and drug resistance.

Why it may be a potential treatment for PKD
Early clinical studies have shown that tesevatinib inhibits epidermal growth factor (EGFR), which promotes cyst growth in ADPKD.

Clinical study status
A Phase 1 study has been completed, demonstrating that tesevatinib was generally well tolerated. Biopharmaceutical company, Kadmon Corporation, LLC, is currently recruiting patients for a Phase 1b/2a study to determine tesevatinib’s safety, pharmacokinetics and optimal dosage.

Tolvaptan

What it is
Tolvaptan is an FDA-approved short-term treatment used to increase low levels of sodium in the blood of patients who have congestive heart failure. Tolvaptan is a vasopressin V2 receptor antagonist that increases the amount of water released from the body as urine.

Why it may be a potential treatment for PKD
Tolvaptan slows cyst growth in ADPKD patients by preventing a hormone called vasopressin from binding to its receptor on renal tubule cells. Vasopressin is produced by the brain to signal the kidneys to conserve water; when this happens, a chemical called cyclic adenosine monophosphate (cAMP) is produced in the kidneys. This chemical has been shown to promote the growth of cysts by increasing cell division. Tolvaptan suppresses vasopressin action, which in turn reduces cAMP in kidney cyst cells, and thereby slows cyst growth.

Clinical study status
Tolvaptan is approved in the European Union, Canada and Japan for in the treatment of ADPKD. It was turned down by the FDA in 2013, and is currently in a phase 3b clinical trial. The current trial, REPRISE, closed recruitment in early 2016 and will be ongoing for about two years. Learn more about this clinical study.