A multi-institutional team has recently received a substantial grant exceeding $3.4 million, aimed at spearheading a pioneering clinical trial to combat acute myeloid leukemia (AML). This award, bestowed by the National Institutes of Health’s National Cancer Institute, brings together the expertise of the University of Cincinnati (UC) and the OSUCCC - James to focus their combined firepower on a dire medical frontier — the need for new AML treatments is urgent, given that the disease afflicts over 20,000 individuals annually in the U.S. alone, with a mortality rate tipping over half of that number each year. According to UC News, this infusion of resources not only funds research but also lays the groundwork for more efficient, patient-benefiting industry partnerships.
The journey of JBZ-001, the drug at the heart of the trial, is an unconventional one, having first taken shape in an undergraduate chemistry class at Hendrix College, Arkansas. Under the guidance of Professor Thomas Goodwin, the students set out to quickly develop small-molecule inhibitors targeting the dihydroorotate dehydrogenase (DHODH) pathway, which is essential for cancer cell growth—the Suzuki reaction, a less trodden pathway in drug synthesis.
Progress on what was then known as the HOSU series of compounds accelerated when Goodwin reached out to John C. Byrd, a notable Hendrix alumnus and board member, as well as a former student. Byrd undertook further development and rigorous preclinical testing alongside Erin Hertlein and Ola Elgamal at OSU before the project transitioned to UC in 2021. After many iterations, HOSU-53 emerged as a lead candidate, eventually attracting the interest of Jabez Biosciences, which licensed the drug for human therapy trials in 2024 under the name JBZ-001.
In an inspiring display of academic-industrial synergy, the Drug Development Institute at OSU played a crucial role in refining the initial compounds. Today, as patients and researchers alike face down the relentless threat posed by AML, the aspirations pinned on JBZ-001 carry the weight of a community's hope for a breakthrough. With the support of the NCI grant, the stage is set to potentially turn what began as a classroom project into a life-saving medical intervention.
