PKD treatment pipeline

Since 1982, we have led the fight against PKD through the support of basic, translational and clinical scientists, 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 an approved drug (JYNARQUE™), and more drugs in the pipeline today than ever before.

Read on to learn more about the treatments for PKD currently being developed in the United States. In 1982, when the PKD Foundation was established, little was known about the molecular genetics or mechanisms of the disease. The following list shows how much is now known, much of it due to the research funding by the PKD Foundation. Each novel therapeutic addresses a unique pathway that is affected by the disease.

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 glycolysis pathway, which is linked to cell proliferation that causes cysts to form and grow. When treated with metformin, mice with PKD show inhibition of cyst growth. Metformin has a long established safety profile because it has been used for decades in the treatment of 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 regulatory protein 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 could be an appealing treatment option for PKD because of its low cost and benign safety profile.  The European Nicotinamide Diabetes Intervention Trial (ENDIT) study showed that large doses of niacinamide are safe in humans. Additionally, niacinamide is a dietary supplement, and therefore does not require approval by the FDA, although a pivotal trial would help to establish an optimal dose.

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, is designed to determine the effects of niacinamide on markers of kidney injury, inflammation, and kidney function and cyst growth. Analysis of the results is ongoing.

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 cyst cell division by causing terminal cell differentiation.

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 Purdue University in Indianapolis (IUPUI) are conducting a pilot clinical trial to test the safety and efficacy of low doses of pioglitazone on ADPKD progression. This study is active and ongoing but is no longer recruiting patients.

Curcumin

What it is
Curcumin is a dietary supplement that is produced by some plants and is found in the spice turmeric.

Why it may be a potential treatment for PKD
Curcumin activates transcription of key antioxidants, suppresses inflammation and reduces cell proliferation (growth). Because of these properties, it is thought that it could have positive effect in reducing cell growth as well as improve the health and function of arteries in ADPKD.

Clinical study status
The University of Colorado Anschutz Medical Campus is conducting a study to determine if curcumin can improve the function of blood vessels of children and young adults with ADPKD. The study is currently recruiting patients. Learn more about this clinical study.

Statin therapy

What it is
Statins comprise a class of drug that lowers the level of cholesterol in the blood.

Why it may be a potential treatment for PKD
In addition to their efficacy in lowering cholesterol, statins also have anti-proliferative, anti-inflammatory and antioxidant effects. Due to this, and their relative safety, it is thought they could potentially be used to slow the progression of ADPKD.

Clinical study status
The university of Colorado Anschutz Medical Campus is conducting a study to learn if pravastatin (a common statin) is helpful in slowing down the progression of ADPKD. This study is actively recruiting patients. Learn more about the study and see if you are eligible here.

Even more compounds in preclinical development

In addition to these compounds, other investigational drugs are in preclinical development and some of these could enter clinical trials in the coming years. At least 15 companies, if not more, are now actively pursuing the treatment of ARPKD and ADPKD, more than at any time previously. Much of the science of this growing pipeline can be traced to the millions of dollars invested by the PKD Foundation in promising research and in the scientists determined to discover the molecular pharmacology of PKD and the therapeutic approaches to treat it.