Stem Cell Treatment for Polycystic Kidney Disease

Stem Cell Treatment for Polycystic Kidney Disease

Autosomal dominant polycystic kidney disease (ADPKD) is a genetic disease characterized by development of cysts in multiple organs, primarily the kidneys. It may progress to end-stage renal disease (ESRD).


Stem cell therapy is rapidly gaining momentum in the realm of regenerative medicine. The hope is that mesenchymal stromal cell therapy can reverse kidney damage and restore function with a faster recovery rate and minimal complications.


Currently, there is no cure for ADPKD.  Patients are usually given symptomatic treatments. Antihypertensive medications are usually given to treat high blood pressure, and analgesics are given for pain relief. Antibiotics may be administered to infection associated with the disease. In cases of ESRD, dialysis might be needed to maintain kidney function. The signs and symptoms are those associated with higher levels of blood urea and creatinine that include: fatigue, edema, troubled concentration, lack of appetite, and dry and itchy skin. The major ADPKD complications include: severe fluid retention, cardiovascular diseases, weak bones and increased frequency of fractures, impotence, central nervous system complications, electrolyte imbalance, especially potassium.


When ADPKD progresses, kidney vascular architecture disrupts because of emerging cysts. Vascular endothelial growth factor (VEGF) is an antiapoptotic molecule that can normalize this disrupted blood vessel network surrounding the cysts. VEGF can also normalize BUN, SCr, and macrophage infiltration. MSCs produce VEGF and when transplanted in the diseased kidney, have the potential to improve kidney vasculature and function.


Mesenchymal stem cells (MSCs) are harvested from bone marrow, adipose tissue, etc. and can be differentiated to osteoblasts, adipocytes, and chondroblasts. They have the ability to treat renal diseases by anti-apoptotic, anti-fibrotic, and anti-inflammatory capabilities. It has been reported that transplanted MSCs can improve kidney function and improve damaged vasculature in a PKD model.


MSCs can be reprogrammed by the introduction of transcription factors to generate nephrons. This facilitates artificial generation of organoids (mini-organs) that may help to increase our knowledge of how PKD occurs and how to best treat.


One preclinical study showed the beneficial effects of single intravenous infusion for improvements in systolic HTN and fibrosis using allogeneic MSCs in a PKD rat model. The infusion did not affect cyst size and number, but it significantly improved cortical and parenchymal vascular density, leading to better tubular function and improvements in creatinine clearance. They also reported the safety of the MSC infusion with major toxicities or mortality.


More research is accumulating that shows the MSC therapy for renal diseases is safe. There have been no reports of acute infusion toxicity, organ complications, infections, death or malignancies associated with the treatment. But there is not enough data on the efficacy of MSC therapy for ADPKD currently. This avenue of regenerative medicine continues to grow rapidly and holds a lot of promise for the future of chronic kidney disease and other conditions.

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