Ohio State Research Discovery Enters Phase I Clinical Testing

September 25, 2019
Robert Baiocchi

COLUMBUS, Ohio – A new targeted oral therapy – developed through research discoveries at The Ohio State University Comprehensive Cancer Center – Arthur G. James Cancer Hospital and Richard J. Solove Research Institute (OSUCCC – James) – is being offered as an experimental treatment option for patients with certain advanced cancers, including lymphoma.

Known only as PRT543 while in testing, this targeted treatment molecule is among the first in an emerging class of drugs called PRMT5 inhibitors.

Protein arginine methyltransferases (PRMTs) are a family of enzymes that regulate a wide variety of cellular functions. When dysregulated, PRMTs are associated with several aggressive human cancers, including diffuse large B-cell and mantle cell lymphoma, pancreatic cancer and glioblastoma. Research published by the OSUCCC – James and other institutions suggests that PRMT5, a member of this family, is a potential oncoprotein involved in transforming a normal cell into a cancer cell and could be an important target for new treatment strategies.

The OSUCCC – James is one of four cancer centers participating in this national trial, sponsored by Prelude Therapeutics. One hundred patients will be enrolled to the first-in-human clinical trial across the United States. Patients age 18 or older with metastatic or advanced solid tumors, advanced diffuse large B-cell lymphoma, advanced mantle cell lymphoma, relapsed myelodysplastic syndrome or relapsed myelofibrosis may qualify for this experimental therapy.

Led by Robert Baiocchi, MD, PhD, the OSUCCC – James research team was the first to discover PRMT5 as a cancer driver and the first to develop and then report a series of novel molecules to selectively inhibit PRMT5. The team has been evaluating PRMT5 inhibitors for the therapeutic treatment of various cancers as well as benign blood and autoimmune diseases. The targeted molecule currently in testing (PRT543) was developed by Prelude.

With support from the Drug Development Institute (DDI), a translational accelerator embedded within the OSUCCC – James, this entire portfolio of molecules was licensed to Prelude in 2016. The company is developing drugs that inhibit a specific enzyme (PRMT5) for cancer and other unmet medical needs. Prelude provides financial support for preclinical and clinical research conducted at the OSUCCC – James.

“PRMT5 is a compelling therapeutic target because it acts like a hub that coordinates diverse molecular activities for cancer cells. PRT543 selectively targets PRMT5. If proven effective in clinical testing, this could radically improve how we treat certain aggressive, recurrent cancers,” says Baiocchi.

The PRMT5 research conducted to date at the OSUCCC – James was supported by the DDI utilizing resources provided by Pelotonia, a Columbus-based cycling event that has raised more than $200 million to support cancer research at Ohio State as well as the Harry T. Mangurian foundation. The research also received support from the National Institutes of Health and the Leukemia and Lymphoma Society.

The DDI helps advance new cancer therapeutics through strategic partnerships within the global pharmaceutical and research/development industries. To learn more about the DDI, visit cancer.osu.edu/DDI. To learn more about clinical research conducted at the OSUCCC – James, visit cancer.osu.edu/clinicaltrials or call 1-800-293-5066.


About the OSUCCC – James
The OSUCCC – James strives to create a cancer-free world by integrating scientific research with excellence in education and patient-centered care, a strategy that leads to better methods of prevention, detection and treatment. Ohio State is one of only 51 National Cancer Institute (NCI)-designated Comprehensive Cancer Centers and one of only a few centers funded by the NCI to conduct both phase I and phase II clinical trials on novel anticancer drugs sponsored by the NCI. As the cancer program’s 356-bed adult patient-care component, The James is one of the top cancer hospitals in the nation as ranked by U.S. News & World Report and has achieved Magnet® designation, the highest honor an organization can receive for quality patient care and professional nursing practice. At 21 floors and with more than 1.1 million square feet, The James is a transformational facility that fosters collaboration and integration of cancer research and clinical cancer care. For more information, visit cancer.osu.edu.

Media Contact:
Amanda J. Harper
OSUCCC-James Media Relations

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