Important Research from İTÜ Could Contribute to Alzheimer's Treatment

Research by Prof. Dr. Yusuf Yağcı, Faculty Member at Istanbul Technical University's Department of Chemistry, and Dr. Antonino Puglisi, postdoctoral researcher, into Niemann-Pick Type C (NP-C) disease, known as "Childhood Alzheimer," is being closely followed by the scientific community. The two scientists' new work, which will also contribute to the treatment of diseases such as Alzheimer's, Parkinson's and Huntington's, was featured in an article published in Advanced Science News. Niemann-Pick Type C (NP-C) disease, known as "Childhood Alzheimer," is recognized as a genetic disorder that occurs in 1 in 100,000 people in the EU according to research. The disease impairs the function of a protein that regulates cholesterol trafficking, enabling its recycling or reuse. This causes cholesterol accumulation in the brain, liver and spleen. The accumulated cholesterol leads to brain damage and inevitably causes a reduction in cognitive abilities and mobility. Typically diagnosed during childhood, NPC progresses rapidly to life-threatening levels in the second or third decade of life. The scientific community is searching for solutions to treat the disease. At this point, an important contribution to the scientific world came from Istanbul Technical University. Istanbul Technical University Faculty Member Prof. Dr. Yusuf Yağcı from the Department of Chemistry and Dr. Antonino Puglisi, postdoctoral researcher, drew attention to the ability of Cyclodextrins (CDs), which are naturally occurring sugars, to remove cholesterol and examined the potential of the disease's treatment. Scientists at Istanbul Technical University believe that the problem can be solved by embedding CDs into larger macromolecular structures.

Research Work Continues

Prof. Dr. Yusuf Yağcı, Faculty Member at the Department of Chemistry, and Dr. Antonino Puglisi, regarding their research, stated: "Although promising, current NPC treatment based on chemical modification of ß-CD suffers from poor pharmacokinetics and bioavailability in the brain. CDs do not appear to effectively cross the blood-brain barrier (BBB). Furthermore, to achieve sufficient therapeutic effect, high doses of CDs are required, as CDs administered systemically have a short half-life due to rapid renal elimination in blood circulation. Overcoming some of these limitations is possible by embedding the CD macrocycle into a larger macromolecular structure.

Impaired Cholesterol Metabolism

Polymers containing cyclodextrin have three main advantages: the higher molecular weight of the polymer can delay rapid renal elimination, thereby extending the half-life of the system in blood, and when combined into nanoparticles, CDs can be delivered to the brain via systemic administration. This strategy is an emerging tool in NPC treatment and may also be beneficial in treating other neurodegenerative diseases characterized by impaired cholesterol metabolism, including Alzheimer's, Parkinson's and Huntington's diseases. If this research addresses successfully an important question that urgently needs to be resolved, it is expected to make a significant contribution to the treatment of this neglected disease."