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Conquer Cancer Foundation Grants Lead to Breakthroughs in Treatment for Pediatric Cancers

When Yael Mossé, MD, was awarded a Conquer Cancer Foundation of ASCO Young Investigator Award (YIA) in 2003, she wanted to find a familial cancer gene in the rare, hereditary form of pediatric neuroblastoma. She imagined that finding such a gene would also be relevant in helping researchers understand the more common, sporadic form of neuroblastoma. It was only “a pie-in-the-sky dream” at that point, according to Dr. Mossé, that this gene would turn out to be a targetable oncogene and that her early research would lead to an extremely promising clinical trial on a new treatment for pediatric cancers. The dream is now reality. Dr. Mossé is presenting her work “Efficacy of crizotinib in children with relapsed/refractory ALK-driven tumors including anaplastic large cell lymphoma and neuroblastoma: A Children’s Oncology Group phase I consortium study” at the 2012 ASCO Annual Meeting in Chicago, and her research is being featured in the meeting’s press program. The results of the study are promising and have been reported on by NBC, The Wall Street Journal, Bloomberg, and HealthDay among others.

Initially, Dr. Mossé and her mentor, John Maris, MD (1996 YIA, 1997 CDA), believed from preliminary data on two families that this familial cancer gene was going to be a tumor suppressor gene on chromosome 16. She didn’t find the gene during her YIA grant period, but her progress and perseverance were compelling enough for the Conquer Cancer Foundation of ASCO to award her a 2004 Career Development Award (CDA)  entitled “High-throughput genomics to identify a hereditary neuroblast

oma predisposition gene” to continue this line of research.  Dr. Mossé noted that, “In between, I wrote my [NIH funded] K08 on something completely different, but pursued this high-risk project on the side.”  Dr. Mossé’s work and the Conquer Cancer Foundation’s support of the high-risk research paid off. With improving genomic technologies, she was able to do a genome-wide study in samples from 15 families with familial neuroblastoma. Although they didn’t find the gene they expected on chromosome 16, she and her colleagues identified mutations in the oncogene ALK on chromosome 2. This was the first oncogene found to be mutated in the germline of a childhood cancer, and the results were published in 2008 in the high-profile journal Nature, including a reference to the CDA funding. Dr. Mossé noted that along with the publication, she was now able to compete for an NIH R01 grant to start her own laboratory, on her first try.

“The support I got from the Conquer Cancer Foundation of ASCO has been directly responsible for all of the continued funding and high profile publications that I have had.”

Dr. Mossé credits her early YIA for helping shape her understanding of how important her work was to the mission of ASCO and the field of oncology. “It gave me a mission and allowed me to start to bridge the lab and the clinic….getting the support through a clinical organization helped me realize that I could do both.” She has continued to excel as a translational investigator. There is now an FDA-approved drug called crizotinib for treating ALK mutations in adult non-small cell lung cancer, and Dr. Mossé and her colleagues are paving the way for the use of this drug in pediatric cancer. Noting the importance of her new findings, Dr. Mossé says “now we have a major opportunity to personalize therapy upfront in not only kids with aggressive neuroblastoma but also those with anaplastic large cell lymphoma and inflammatory myelofibroblastic tumors.” Crizotinib is a relatively non-toxic treatment, a pill taken by-mouth twice-a-day, and the data that are being presented at the ASCO meeting show that a subset of children receiving the treatment are having complete responses and show no evidence of ALK transcripts in their blood. Because of the high-quality of her research, Dr. Mossé has been selected for the James B. Nachman ASCO Junior Faculty Award in Pediatric Oncology, which will be presented for the first time this year. This Special Merit Award is named in memory of Dr. James B. Nachman, Professor of Pediatrics at the University of Chicago and an internationally renowned pediatric cancer expert who died suddenly in 2011. The award recognizes a junior faculty member who submits the highest-ranked abstract in pediatric oncology to the ASCO Annual Meeting.

In Dr. Mossé’s study, different types of ALK alterations in the cancers are resulting in different response rates, so many research questions remain. But the preliminary trial results have been extremely promising and offer new hope to children with ALK-driven cancers. To illustrate the potent effects of this drug, Dr. Mossé tells the story of the first patient who enrolled in the trial and had a very large inflammatory myelofibroblastic tumor in his right shoulder. “He had previously received at least 10 cycles of intensive chemotherapy and not responded at all. The surgeon said the only way he could get it all out, which you have to do if you really want a cure, is to do a four-point amputation. Then this drug came along and the patient enrolled in the trial. There was a dramatic benefit for this patient, so it’s really proof of concept that when you know the abnormality that is causing the cancer and you have the right drug to turn it off, that may be all you need.” Dr. Mossé believes that for many of these patients with inflammatory myofibroblastic tumors, crizotinib could prevent the need for surgery.

Now that her high-risk project has paid off by leading to a breakthrough for the care of childhood cancer patients, Dr. Mossé remains devoted to bridging the transition between the laboratory and clinic. She describes her research philosophy as,

“Always, first and forefront, if there is not a direct relevance to how we care for patients in the experiments that we are doing, then we shouldn’t be doing the experiments.” 


To support groundbreaking researchers like Dr. Mossé who are working towards a world free from the fear of cancer, please visit www.conquercancerfoundation.org/donate.  

For more information on the following topics, please visit:

Conquer Cancer Foundation Young Investigator Award

Conquer Cancer Foundation Career Development Award

James B. Nachman Award