Ohio State Cancer Research Played a Significant Role in FDA Approval of Important New CLL Drug

February 14, 2014

COLUMBUS, Ohio – On Feb. 12, 2014, the U.S. Food and Drug Administration expanded the approved use of the drug ibrutinib to chronic lymphocytic leukemia (CLL).

Much of the clinical and basic-science research that led to the approval of ibrutinib for CLL was performed by scientists at The Ohio State University Comprehensive Cancer Center – Arthur G. James Cancer Hospital and Richard J. Solove Research Institute (OSUCCC – James).

Ibrutinib (Imbruvica®) is the first drug designed to target Bruton’s tyrosine kinase (BTK), a protein essential for CLL-cell survival and proliferation. CLL, the most common form of leukemia, causes a gradual increase in white blood cells called B lymphocytes, or B cells. The National Cancer Institute estimates that 15,680 Americans were diagnosed with CLL and 4,580 died from the disease in 2013.

Much of the Ohio State work was led by John C. Byrd, MD, director, division of hematology, and professor of medicine, of medicinal chemistry and of veterinary biosciences at the OSUCCC – James. Byrd also directs the OSUCCC – James CLL Experimental Therapeutics Laboratory and co-directs the Leukemia Research Program. Other key team members include Amy J Johnson, PhD, Jason Dubovsky, PhD, Jeffrey Jones, MD, MPH, Joseph Flynn, DO, MPH, Jennifer Woyach, MD, Kami Maddocks, MD, and Kristie Blum, MD.

“Our clinical studies consistently suggested that ibrutinib is a highly active oral therapeutic that produces a high rate of durable remissions – the remissions last months on end – with acceptable toxicity in relapsed and refractory CLL,” says Byrd, holder of the D. Warren Brown Designated Chair in Leukemia Research.

“Patient responses can last for many months, in part because patients are willing to remain on the drug since the side effects are very tolerable,” Byrd says.

Studies of ibrutinib by OSUCCC – James researchers include:

  • 2011: A paper showing that BTK is a promising target for treatment of CLL, and that ibrutinib (then known as PCI-32765) effectively inhibits the molecule;
  • 2011: A study showing that ibrutinib selectively kills the malignant B lymphocytes that cause CLL, while sparing healthy T lymphocytes. This leaves part of the adaptive immune system intact. Other CLL treatments kill both malignant B cells and healthy T cells, leaving patients highly susceptible to life-threatening infections.
  • 2011: Interim phase I/IIb trial findings showing that ibrutinib is highly active and well tolerated in CLL patients, including those undergoing treatment for the first time and those who have relapsed and are resistant to other therapy;
  • 2012: Findings that ibrutinib shows great promise for the treatment of older CLL patients, those age 65 and older;
  • 2012: Updated findings showing that ibrutinib as highly active in CLL patients;
  • 2013: A study published in the New England Journal of Medicine showing that ibrutinib significantly prolongs survival in all classes of CLL patients, even those at high-risk of recurrence; similar results were shown in patients with mantle cell lymphoma;
  • 2013: A study showing that the molecule BTK is critical for the growth of CLL cells and the development of CLL, confirming that it is an important target for ibrutinib and for future CLL drugs;
  • 2014: How will B-cell receptor targeted therapies change future CLL therapy?;
  • 2014: A study showing Prolonged lymphocytosis during ibrutinib therapy is associated with distinct molecular characteristics and does not indicate a suboptimal response to therapy;
  • 2014: Impact of targeted therapy on outcome of chronic lymphocytic leukemia patients with relapsed Del(17p13.1) karyotype at a single center.

The FDA granted accelerated approval to ibrutinib for the treatment of mantle cell lymphoma in November 2013.

The Ohio State University Comprehensive Cancer Center – Arthur G. James Cancer Hospital and Richard J. Solove Research Institute 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 41 National Cancer Institute (NCI)-designated Comprehensive Cancer Centers and one of only four centers funded by the NCI to conduct both phase I and phase II clinical trials. The NCI recently rated Ohio State’s cancer program as “exceptional,” the highest rating given by NCI survey teams. As the cancer program’s 228-bed adult patient-care component, The James is a “Top Hospital” as named by the Leapfrog Group and one of the top cancer hospitals in the nation as ranked by U.S.News & World Report.


A high quality JPEG of John C. Byrd, MD, is available here.

Illustration of how ibrutinib works, Frontiers, winter 2013, The Ohio State University Comprehensive Cancer Center – James Cancer Hospital and Solove Research Institute

Written by Darrell E. Ward, Wexner Medical Center Public Affairs and Media Relations,614-293-3737, or Darrell.Ward@osumc.edu.

Media Contact: Amanda J. Harper, OSUCCC-James Director of Media Relations, 614-685-5420 (direct), 614-293-3737 (central line) or Amanda.Harper2@osumc.edu.

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