Drug candidate improves protein recovery in Lowe syndrome models
A peer-reviewed study finds better protein recovery in cells and animals used to study Lowe syndrome, an inherited condition involving the kidneys and other organs. The candidate compound IB-MECA was evaluated in patient-derived cells, laboratory kidney models and mice. The results remain preclinical; a planned human study is under regulatory review.
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Kidney models recover more protein
A candidate medicine improved the recovery of filtered proteins in experimental models of Lowe syndrome, an inherited disease with effects on the kidneys, eyes and nervous system. The peer-reviewed research examines a kidney defect linked to disruption of cellular lipid distribution. It reports improvements in patient-derived cells, miniature laboratory kidney models and mice. None of these findings establishes treatment efficacy in people with the syndrome.[1]
IB-MECA targets a cellular signalling receptor
The compound IB-MECA acts through the adenosine A3 receptor, a signalling protein on the cell surface. The investigators connected its effects with improved movement of material between cellular membranes and increased protein uptake. Lowe syndrome involves loss of function of the OCRL gene, which disrupts this internal organization. The kidney organoids used in the study reproduce aspects of tissue behaviour while remaining simpler than a complete organ.[1]
Human testing remains under review
In the mouse comparison, a urinary protein marker was lower in treated animals than in untreated animals. The authors report that a planned phase II clinical study remains under review by health authorities. Previous human testing of IB-MECA for other conditions supplies no efficacy result for Lowe syndrome. A patent application relating to this use names several researchers as inventors. The work concerns kidney manifestations and does not demonstrate benefits for the disorder’s eye or neurological features.[1]
