Working like an architect, Hagit Eldar-Finkelman, a professor in the department of human molecular genetics and biochemistry at Tel Aviv (Israel) University’s Sackler School of Medicine, is “building” a new drug — L803-MTS — to treat central nervous system (CNS) diseases such as Alzheimer’s. In preclinical studies, the compound also shows promise against Parkinson’s, Huntington’s, and diabetes. L803-MTS is based on the physical structure of the GSK3 protein, which plays a causative role in insulin resistance and type II diabetes. Working with chemists, biotechnologists, and 3-D modelists, Eldar-Finkelman and colleagues built — like engineers constructing a building — a drug that locks onto the GSK3 protein, rendering it harmless and unable to wreak havoc inside the body. Research findings on the L803-MTS drug have been published in the Journal of Molecular Biology and Biochimica et Biophysica Acta (BBA) – Proteins and Proteomics.
Since Eldar-Finkelman linked GSK3 to insulin resistance in diabetes more than 10 years ago, a race has been on among drug manufacturers to find a drug that can potentially turn off the harmful effects of the protein. Rather than build on existing drugs, Eldar-Finkelman and her colleagues worked from the ground up. “I decided to take a completely different approach from all the big drug companies rushing to find the ultimate drug,” Eldar-Finkelman says. “I designed my own.” Preclinical results have been positive, and the new drug does not exhibit dangerous toxic side effects — a problem with existing formulations. While L803-MTS cannot reverse the onset of a CNS disease, Eldar-Finkelman believes it can slow devastating effects such as memory impairment, depression, and insulin-resistance. With seed money from Ramot, the university’s TTO, she’s taken her basic research to the next step and is seeking a strategic partner to guide the research through the clinical process and eventual commercialization.
Source: Science Daily