Yuan is working with the drug screening core at UCLA to screen over 90,000 chemical compounds for their effect on the RCS cell line, the model we use for drug screening to find treatments for achondroplasia.
Pavel and his collaborators have identified many new proteins involved in FGFR3 signaling using proteomics, a technique where the thousands of proteins in the cell are analyzed using mass spectrometry, a very sensitive way to analyze small samples. He is in the process of validating some of the hits from the proteomic studies. Understanding how FGFR3 signals is critical in identifying specific pathways to target.
We are working with 3 companies to acquire and test compounds they have made that are said to inhibit FGFRs. We will test these compounds in the lab to see how specific they are for FGFR3 and if their structures could be modified to make them useful for achondroplasia.
Cedars-Sinai will be participating in the BioMarin trials of the CNP analogue. Very shortly, we will start the measurement study in children 4.5 to 9 years of age. Younger children will be enrolled at a later date. At least 6 months of growth data is needed before a child would be considered for the drug study, once it starts. The study will require several trips to Los Angeles. If you have a child with achondroplasia and might be interested, contact Tara Funari at 310-423-4495 for more information.
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