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(photo: Jeffrey Tyner, Ph.D, Oregon Health and


Science University)


A publication by LLS-funded researchers at the Knight Cancer Institute at Oregon Health and Science University (OHSU), and an accompanying editorial, were published this week in the New England Journal of Medicine describing exciting findings for patients with two rare forms of leukemia.


The authors of the study, “Oncogenic CSF3R Mutations in Chronic Neutrophilic Leukemia and Atypical CML” used genetic testing to identify a new target for therapy for patients with blood cancers called chronic neutrophilic leukemia (CNL) and atypical chronic myeloid leukemia (aCML). The researchers found mutations in a gene called colony stimulating factor 3 receptor (CSF3R)  and learned that the abnormal CSF3Rs  initiate a chain reaction with other genes(a pathway), subsequently driving cancer formation. 


This discovery is promising for patients because both of these diseases are very rare, difficult to diagnose and extremely difficult to treat successfully. The prognosis for most of these patients is very poor. But, this study shows that some of these patients may be able to be treated effectively with existing FDA-approved drugs, because these drugs appear to inhibit critical molecules in that CSF3R pathway and cause the cancer cells to die.


While the patient population impacted by these findings is small, this study is another example of LLS stepping in to address a clear unmet medical need for patients in desperate needs of new options,” said LLS Senior Vice President of Research Richard C. Winneker.


In the bigger picture, the study is further evidence of the power of using technology to identify cancer-driving gene mutations that can be targeted either with already approved drugs or new compounds yet to be developed.


In his supporting editorial, Jerald Radich, M.D., of Fred Hutchinson Cancer Research Center, notes: “This study shows the potential power of studying a small problem very craftily and is an example of what genetically informed treatment may look like in the near future. This is how we will beat cancer, one gene, one disease at a time.”


The main authors of the study are Julie Maxson, Ph.D., and Jeffrey W. Tyner, Ph.D., both of whom are members of an LLS Specialized Center of Research program grant led by OHSU’s Brian Druker, M.D., also listed as a co-author on this study.  LLS-grantee Michael Deininger, M.D. Ph.D, of University of Utah, also contributed to the study.


“What distinguished this research was our method for matching a voluminous amount of gene sequencing data with drug sensitivity data to quickly deduce which mutations were relevant for treatment so that we could make a difference for patients who don’t currently have good therapeutic options, “ Tyner said.


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