Researchers Discover Potential Treatment For Polycystic Kidney Disease

Healthy kidneys are essential for everyone. If your kidneys don’t function properly, you’ll be at risk for a variety of other health problems that can include nerve damage, heart disease, bone disease, and infertility.

However, for most people, a precise and early diagnosis can often reduce and sometimes even reverse the consequences of kidney disease.

In other cases, early diagnosis and proper treatment can help patients manage their kidney disease so that it does not progress to end-stage kidney failure.

For every kidney disease patient, an early and accurate diagnosis is imperative. A doctor’s failure to diagnose kidney disease may harm a patient’s health and may constitute medical malpractice.

If you are a kidney disease sufferer and you are concerned about malpractice or believe that you may be a victim – or if you’ve been harmed or injured while seeking kidney disease treatment – you should discuss your concerns and circumstances with a trustworthy medical malpractice attorney.

Approximately 600,000 people in the United States are afflicted with polycystic kidney disease (PKD), a genetic disorder that causes abnormal cysts to develop and grow in the kidneys.

According to the National Institute of Diabetes and Digestive and Kidney Diseases, the cysts typically cause back pain, high blood pressure, problems with blood vessels in the heart and brain, and eventually lead to kidney failure.

Polycystic kidney disease is a general term for these two types of kidney disease: autosomal dominant polycystic kidney disease (ADPKD) and autosomal recessive polycystic kidney disease (ARPKD).

PKD is the world’s most frequently inherited kidney disease, affecting about 12.5 million persons globally. It is responsible for nearly ten percent of all cases of end-stage renal disease.

Fifty percent of PKD patients suffer complete kidney failure by age 60. PKD affects men, women, and children of all ages and races equally.

Polycystic kidney disease causes abnormal kidney growth due to the proliferation of fluid-filled cysts.

These cysts continue to expand and grow until the kidney no longer functions properly and loses its capacity to filter blood and remove bodily waste.


PKD has been considered incurable, but researchers at the University of Texas Southwestern Medical Center have found what may be an effective new treatment.

Their research was published in Nature Communications in February.

Assistant professor Vishal Patel, who was the senior author of the study, says the drug that may cure PKD is called RGLS4326, which is currently in the clinical testing stage.

The researchers tell us that polycystic kidney disease patients may unknowingly have the disease for many years until a symptom – like blood in the urine, for example – shows up.

Until now, no drug has been available to treat polycystic kidney disease.

When the disease reaches the point of complete kidney failure, the only options until now have been kidney dialysis and kidney transplants.

Dr. Patel’s research led him, back in 2009, to investigate microRNAs (called “MiRs”) that might offer researchers a better understanding of PKD. MiRs are extremely tiny strands of RNA – ribonucleic acid – that can interfere with normal genes.

Kidney researchers became interested in MiRs in the 1990s, reasoning that if MiRs are responsible for PKD, then creating an “anti-MiR” with the exact opposite chemical sequence should neutralize the effect of MiRs on kidneys.

In a 2013 research study, scientists discovered a microRNA sequence that they believe promotes the growth of kidney cysts, the “17~92” sequence.

In the current study led by Dr. Patel, researchers inhibited the 17~92 microRNA sequence in mice and found that the deletion of the 17~92 sequence not only slowed cyst growth but extended – in fact, almost doubled – the lifespan of the mice.

“In support of this conclusion,” Dr. Patel’s researchers write, “we show that genetic deletion of miR-17∼92 attenuates disease progression in ADPKD mouse models irrespective of the mutated gene, the type of mutation (null or hypomorphic) or the dynamics of cyst growth (rapidly fatal, aggressive but long-lived or slowly progressing).”


The University of Texas Southwestern Medical Center’s research follows up on earlier PKD research conducted by the University College London Institute of Child Health.

The London-based researchers noted that tiny blood vessels surrounding the cysts are altered very early in cyst development.

They targeted the blood and lymphatic vessels inside the kidneys of mice with a growth protein called VEGFC.

The blood vessel patterns quickly normalized and the function of the kidneys improved. The researchers in London published their results in 2015 in the Journal of the American Society of Nephrology.

“If we could target these blood vessels early in the development of the condition it could potentially lead to much better outcomes for patients,” according to Adrian Woolf, who is a professor of pediatric science and was the co-author of the study.

Because polycystic kidney disease is caused by an inherited gene, it is not preventable. If one parent carries the gene, a child has a fifty percent chance of developing PKD.

Inevitably, if someone carries the PKD gene, he or she will develop the disease at some point in his or her life. Although there is no cure at the present time, RGLS4326 and VEGFC offer real hope for the immediate future.


While there is no PKD cure for now, treatments and surgeries can help those who struggle with PKD manage the pain and deal with the infections associated with the condition.

However, the misdiagnosis of PKD can lead to a swift deterioration of a patient’s medical condition, and such a misdiagnosis may constitute medical malpractice.

When you receive a routine medical examination, urine tests and blood tests are standard. Those test results should indicate to your doctor if you are in the early stages of kidney disease.

If you are a PKD patient and you have been misdiagnosed or otherwise victimized by medical negligence, speak at once with a qualified medical malpractice attorney.

Kidney disease patients are entitled to full compensation for any injuries they suffer as a result of medical malpractice.

A medical malpractice lawsuit is often the only way to hold a healthcare professional accountable for carelessness or negligence.

The importance of an early and accurate diagnosis of kidney disease cannot be overstated.

Some people don’t even realize they have kidney trouble until that trouble becomes severe, but with the proper medical approach, PKD can be diagnosed accurately and managed effectively.

However, when a doctor fails to diagnose any kidney disease accurately or fails to treat kidney disease appropriately, it may constitute the kind of medical malpractice that puts a patient’s life at risk.

If you are the victim of medical malpractice involving PKD or any other type of kidney disease, don’t wait – discuss your situation promptly with a medical malpractice attorney.

The symptoms of kidney disease include blood in the urine, foamy urine, or discolored urine; high blood pressure; and/or the swelling of the hands and ankles.

If you are over age 60, if you suffer from diabetes, high blood pressure, or heart disease, or if there is a history of kidney disease in your family, get tested for kidney disease, even if you feel healthy.

In its earliest stages, kidney disease manifests no definitive signs or symptoms.

Without a precise early diagnosis, a patient’s kidney disease may become more difficult and burdensome to manage or treat.

If you suffer from kidney disease, and if medical professionals did not accurately diagnose or treat your condition from the start, speak at once with an experienced medical malpractice attorney.

New treatments and breakthroughs are providing plenty of hope for the victims of polycystic kidney disease, but even when these treatments become widely available, PKD patients will still need to seek an accurate early diagnosis and receive the proper treatment.

By: Jed Kurzban

Medical malpractice attorney Jed Kurzban graduated from the University of Alabama in 1992 and earned his Juris Doctor from the University of Miami School of Law in 1995. He is a member of the Dade County Bar Association, the Florida Bar Association, the American Association for Justice, the Academy of Florida Trial Lawyers, and the American Bar Association. Mr. Kurzban is happily married and the father of two.