New Hope

Pelotonia Dollars Support Innovative Clinical Trials

Clinical trials improve cancer care by demonstrating the safety and effectiveness of new treatments, examining treatment strategies and looking at problems associated with therapies so refinements can be made. Here are two examples of Pelotonia-supported clinical trials at the OSUCCC – James. For more information about these or other trials, call The James Line at 1-800-293-5066.

Researchers Detail Reasons for Ibrutinib Therapy Discontinuation in Some Patients With CLL

Kami MaddocksJennifer WoyachBasic and clinical research at Ohio State and elsewhere has shown the drug ibrutinib to be highly effective among certain patients with chronic lymphocytic leukemia (CLL), but a study at the OSUCCC – James has shown that about 10 percent of patients discontinued the drug because of disease progression.

CLL is the most common form of chronic leukemia among adults, with 14,600 new cases expected this year. The malignancy remains incurable, but advances in therapy have been made, notably the emergence of kinase inhibitors such as ibrutinib for patients whose disease has recurred or is resistant to other therapies.

Ibrutinib (marketed as Imbruvica®), is the first drug to target Bruton tyrosine kinase, a protein essential for CLL cell survival and proliferation. Work by OSUCCC – James researchers played a key role in gaining FDA approval of ibrutinib to treat certain patients with CLL or mantle cell lymphoma.

Clinical studies of this drug have continued, including a Pelotonia-supported study published in the Journal of the American Medical Association (JAMA) Oncology. The study by OSU hematologists Kami Maddocks, MD, Jennifer Woyach, MD, and colleagues described the outcomes of patients who discontinued ibrutinib therapy during four sequential clinical trials involving 308 patients at the OSUCCC – James.

With a midpoint follow-up of 20 months, the study showed that of the 308 patients:

  • 232 Remained on ibrutinib
  • 45 Stopped ibrutinib due to infection, other adverse events
  • 31 Stopped ibrutinib due to disease progression

The study concluded that this single-institution experience with ibrutinib “confirms it to be an effective therapy and identifies, for the first time, baseline factors associated with ibrutinib therapy discontinuation.” The study also showed poor prognosis for patients who discontinued therapy.

“These data enhance our understanding of how patients do on ibrutinib long-term and who is likely to relapse,” says Woyach, the study’s senior author and a member of the OSUCCC – James Leukemia Research Program.

“Many patients have durable remissions with ibrutinib, and understanding which patients are at higher risk helps us select who might benefit from clinical trials on other new agents and combination therapies rather than starting ibrutinib treatment by itself,” she adds. “We have confirmed that specific gene mutations are seen in patients who relapse, which gives us an idea of other drugs that might be effective in these circumstances.”

Clinical Trial Findings Could Improve Development of Targeted Therapies

Sameek Roychowdhury 2A phase II clinical trial at the OSUCCC – James is applying basic science knowledge and tools to help researchers develop better precision cancer drugs that molecularly target alterations observed in each patient’s cancer genes.

Specifically this trial, supported by Pelotonia funds, will determine how well a drug called ponatinib hydrochloride works in treating patients with cancer that has metastasized (spread) to other parts of the body, has not responded to previous therapy, and has one of several alterations, or mutations, in its DNA sequence.

“Ponatinib hydrochloride may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth,” says principal investigator (PI) Sameek Roychowdhury, MD, PhD, a specialist in translational genomics and member of the Translational Therapeutics Program at the OSUCCC – James. “It is not yet known whether a patient’s genetic alterations affect how well this drug works.”

Roychowdhury says there is a “seamless integration between this clinical trial and laboratory study to determine how cancers become resistant to therapy and why some patients respond to therapy and others do not, with a key connection of research tumor samples before and after treatment.”

The investigators will use a “team science” approach to 1) study gene and protein alterations by collaboration between the Ohio State basic science labs of Roychowdhury and co-investigator John Hays, MD, PhD, also of the Translational Therapeutics Program, and 2) complete the clinical trial via collaboration between Roychowdhury and scientists at the University of Michigan.

The trial is open to patients 18 and older with any metastatic cancer that has alterations in a protein called fibroblast growth factor receptor (FGFR). Roychowdhury describes this study as a “basket trial” because it involves patients with different cancer types that have common gene alterations.

Trial participants orally receive ponatinib hydrochloride once daily for 28 days, and then repeat this course every 28 days in the absence of disease progression or unacceptable toxicity (adverse effects).

The study’s primary objective is to evaluate overall patient response to this drug, which is an FGFR inhibitor.

“While we expect that patients with FGFR alterations will have disease regression, we also anticipate that patients will eventually acquire resistance to single-agent therapy with ponatinib and develop disease progression,” the researchers state in their study application. “Despite intensive study of FGFR in cancers, (the cause of) acquired resistance to FGFR inhibition is unknown.”

However, they hypothesize that acquired resistance is mediated through genomic or expressed alterations that provide a bypass for FGFR signaling. This study and its methods are designed to address their hypothesis.

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