In Vitro Testing of Three Novel USP7 Inhibitor Drug Therapies in Pediatric Neuroblastoma

Michelle working in vent hood

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Project Summary

This summer I was fortunate to conduct my PURM research in Dr. Maris’s translational pediatric neuroblastoma research laboratory, where I was able to become involved with and learn firsthand about the “life of a drug” from bench to clinic.

My specific project involved testing in vitro three novel drug compounds developed by a pharmaceutical company (name withheld for privacy reasons). The rationale behind these targets, which are USP7 inhibitors, is that they will damp down the highly expressed USP7 modulatory pathway seen in high-risk neuroblastoma patients. To assess drug potency and efficacy, I treated for 3 or 5 days and then utilized Cell-Titer Glo, a luminescent cell viability assay, to determine cell survival across 10 of our established neuroblastoma patient-derived cell lines. I also concurrently performed a Western blot to quantify protein expression of USP7. Coincidentally on my last day in the lab, we had a teleconference call with the company to present our findings and it was determined that we would be doing further experimentation with another batch of drugs and cells before going in vivo.

My overall research experience was a highly memorable one, as I was able to grow significantly over my 10 weeks here. Although I had conducted wet lab research before at Memorial Sloan-Kettering and UC Berkeley, nonetheless each lab also harbors a novel experience. On the technical side, I was able to refresh on my molecular biology techniques, including cell culture, protein assays, Westerns, etc. Interpersonally by experiencing the “full-time life of a research scientist” this summer, I learned to manage my daily project tasks with other duties of the lab and work collaboratively with other researchers especially when troubleshooting. Intrapersonally in terms of figuring out potential future plans, I had a recent mind-blowing experience shadowing Dr. Maris, who is also a pediatric oncologist, as it was the first time I was able to see the many targets studied or discussed in the lab administered to patients in different clinical trials. If that is not “science in practice” enough, I do not know what is.

Although it would be cliché to conclude that this summer thoroughly immersed me into the “deep, vast world that is scientific inquiry, exploration, and knowledge,” which can be said of many endeavors, the fact is that I was able to take away many invaluable understandings from my time in the lab, including learning more about myself as a student researcher. As I am sure how I learn, think about, and work with science continues to evolve, for now I just know that I would be eager to return in the fall and keep on learning.