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OHSU, Ionis Pharmaceuticals to develop drugs for ‘undruggable’ cancer targets

Collaboration aims to expedite discoveries for patients with leukemia
Prescription Medicine spilling onto a counter.
OHSU is pairing up with Ionis Pharmaceuticals, aiming to expedite drug development that targets “difficult to drug” molecular drivers of hematologic cancers. (Getty Images)

Oregon Health & Science University today announced the formation of a collaboration with Ionis Pharmaceuticals designed to discover and develop novel therapeutics in the area of hematologic malignancies, or cancers of the blood.

The collaboration will begin by focusing on several priority areas of research for the OHSU Knight Cancer Institute, including acute myeloid leukemia, or AML. AML is historically one of the most difficult forms of leukemia to treat, in part because of the nature of genes that are mutated in the blood-forming cells of people with the disease.

Jeff Tyner, Ph.D. (2018)
Jeff Tyner, Ph.D.

“I look forward to this work giving us the opportunity to more quickly validate novel targets through our preclinical efforts and to shed light on potential high-interest drug targets,” says Jeff Tyner, Ph.D., professor of medicine (hematology and medical oncology) in the OHSU School of Medicine and member of the OHSU Knight Cancer Institute. “Our goal is to speed up the process of bringing new drugs to cancer patients seeking treatment solutions. We have identified many promising targets from the national Beat AML initiative, and this breadth of knowledge combined with cutting-edge targeting methods from Ionis will help us take that work to the next step.”

The collaboration will utilize Ionis’ antisense technology to target specific previously “difficult to drug” molecular drivers of these diseases. Using cutting-edge genomic tools and other methods developed by the OHSU Knight Cancer Institute, the collaboration aims to employ Ionis-ASO technology for multiple important therapeutic targets in cancer, which will then undergo Phase I clinical trials at OHSU.

“We are excited to launch this collaboration that will combine the power and efficiency of Ionis’ antisense technology with OHSU’s exceptional insights and expertise in hematologic cancers to discover novel treatments for these devastating diseases,” said A. Robert MacLeod, Ph.D., vice president of Oncology Research and Development and head of the Oncology Franchise at Ionis. “This collaboration presents another opportunity for Ionis to pursue breakthrough discoveries that can deliver transformational medicines for patients who need them.”

“There are many genes that are essential to the biology of leukemia for which no drugs exist,” says Julia Maxson, Ph.D., assistant professor of medicine (hematology and medical oncology) in the OHSU School of Medicine and member of the OHSU Knight Cancer Institute. “This collaboration expands the realm of genes that can be targeted therapeutically and will fundamentally advance our mission of harnessing scientific discoveries to help patients.”

As the non-exclusive collaboration progresses, it is possible the work will expand to focus on a different leukemia, lymphoma or myeloma, or perhaps other cancers, depending on the target selected. OHSU and Ionis will mutually select targets and advance preclinical studies.

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