Polycystic kidney disease (PKD) is one of the most common inherited kidney conditions, affecting millions of people worldwide. But for the first time in years, researchers at MIT have discovered something genuinely exciting: a new drug candidate that can actually shrink the cysts that damage kidneys. This might sound technical, but the implications are profound.
What is polycystic kidney disease?
Polycystic kidney disease comes in different forms. The most common is autosomal dominant polycystic kidney disease (ADPKD), which means you inherit it from one parent. If you have ADPKD, fluid-filled cysts gradually develop in your kidneys, causing them to enlarge. Over time, usually by your 60s, these enlarged kidneys stop working properly, leading to kidney failure and the need for dialysis or transplant.
More than 12 million people worldwide live with ADPKD, and there are currently few treatment options.
Learn more about kidney disease management.
Why this matters for kidney patients
The MIT team discovered that a compound originally developed to fight cancer could be repurposed to treat ADPKD. Here’s the clever bit: kidney cyst cells and cancer cells actually share something in common – they’re both under “oxidative stress” (an imbalance of damaging molecules in the cell). The new drug pushes these cyst cells over their threshold, causing them to die, whilst leaving healthy kidney cells unharmed.
In animal studies, the drug dramatically shrank kidney cysts and improved kidney function. Even more encouraging, the researchers created a safer version called “11beta-dipropyl” that doesn’t carry the DNA-damaging properties of the original compound. Both versions worked equally well.
You can read the full original research from MIT News here: MIT Drug Candidate for Polycystic Kidney Disease.
As Bogdan Fedeles, lead researcher, noted: “We really believe this has potential to impact the field and provide a different treatment paradigm for this important disease.”
What this does – and does not – mean right now
This research is based on mouse models, not human trials yet. That’s crucial context. At MRIKPA, we always encourage cautious optimism. The research is genuinely promising, but moving from laboratory animals to safe, effective human treatment typically takes years.
The current FDA-approved drug for ADPKD, tolvaptan, slows cyst growth but requires daily dosing and can cause liver damage and frequent urination. The new approach could work differently: researchers suggest treatment once or twice a year might be effective, rather than daily medication. This would be transformative for quality of life.
If you have ADPKD or polycystic kidney disease, there’s no immediate change to your care. But stay informed. Your kidney team will be monitoring developments like this, and it’s worth asking them whether you might be eligible for any clinical trials in future.
Explore our park walks – a great way to stay active while managing your kidney health.
Have questions about your kidney condition? Contact MRIKPA at support@mrikpa.org.uk or call 07745 242 684.
Source: MIT News, January 2024 – read the original research
This article is provided for general information and awareness purposes only and was believed to be accurate at the time of publishing. It is not intended as medical advice – please always consult your doctor or renal team for guidance on your individual circumstances. Images used are for illustration purposes only and may not be medically or editorially accurate. While we take every care, errors can occur. If you spot an inaccuracy, please let us know at support@mrikpa.org.uk.
