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Pediatric Cancer Clinical Trial Launching at 16 U.S. Centers and Internationally Gives Hope to Children and Families

Non-profit Cookies for Kids' Cancer funded critical preclinical work necessary to begin the phase 1 trial of a promising treatment. 

Aug 3, 2017

Califon, N.J. - Thanks to one doctor, her diligent research team and the generous supporters of Cookies for Kids’ Cancer, a phase 1 clinical trial rolling out at 16 research centers for a potentially groundbreaking pediatric cancer treatment, will give hope to children battling this deadly disease. Cookies for Kids’ Cancer, a national 501(c)3 non-profit dedicated to raising funds and awareness for childhood cancer research, awarded $400,000 in grants to Dr. Yael Mossé from Children's Hospital of Philadelphia over the past two years, enabling her to conduct the preclinical work needed to further her research and fast-track this treatment to the clinical trial phase.

This exciting development began with Dr. Mossé and her research team which is focused on finding new therapies for a particularly cruel form of childhood cancer called neuroblastoma. Her work led to a grant submission to Cookies for Kids’ Cancer in 2015, and approval by its Medical Advisory Board. It was the first grant Mossé received for this stage of preclinical work on the promising new drug, called Lorlatinib.

“Cookies’ (for Kids’ Cancer) support has made an immeasurable difference,” said Mossé. “The organization’s commitment to the preclinical studies is vital. This stage of research is often overlooked by foundations and thought to be not as exciting to really support.”

Six months after receiving the first grant from Cookies for Kids’ Cancer, Dr. Mossé’s results were moving ahead so rapidly that the NANT [New Approaches to Neuroblastoma Therapy] Consortium and the drug’s manufacturer, Pfizer, all began to merge interests to move toward the clinical trial phase. Alongside these developments came another grant from Cookies for Kids’ Cancer to ensure the testing and data were of the highest quality in hopes of benefitting children with neuroblastoma. The work has led to the launch of the trial’s enrollment process at several sites including Children’s Hospital of Philadelphia, and will become available at all 16 centers over the next several weeks.

“The ability to fund Dr. Mossé’s preclinical work is the exact mission of the Cookies for Kids’ Cancer organization,” said founder Gretchen Holt Witt. “Phase 1 clinical trials represent hope to children, another chance at bat, and the majority of children battling relapsed cancer, like my son Liam did, are part of a phase 1 trial at some point during their treatment. I know that phase 1 trials ultimately allowed him to be here with us longer. These cutting-edge trials are critically important to children and the promise this one holds is incredibly encouraging." 

 The phase 1 study is enrolling patients at the following 16 centers:

  • UCSF School of Medicine                     Children’s Hospital Los Angeles
  • Cincinnati Children’s Hospital               Lucile Packard Children’s Hospital
  • Children’s Hospital Colorado                Children’s Hospital of Philadelphia
  • C.S. Mott Children’s Hospital                Cook Children’s Healthcare System
  • Hospital for Sick Children,                   Seattle Children’s Hospital  
  • Toronto, Canada                                  Memorial Sloan-Kettering Cancer Center
  • Children’s Healthcare of Atlanta            University of Chicago, Comer Children’s Hospital     
  • Royal Marsden Hospital, London,           Institut Curie, Paris, France
  • England                                         
  • Children’s Hospital Boston, Dana-Farber Cancer Institute

 Today, cancer is the number one disease killer of children in the U.S. yet receives less than four percent of the National Cancer Institute's budget. More than 40,000 children are battling cancer in the U.S. but unlike many adult cancer diagnoses, children's cancers are defects of normal human development, and not the result of lifestyle choices and environmental exposures. Cookies for Kids' Cancer is committed to raising funds for research to develop new, improved and less toxic treatments that will allow children to not only survive, but thrive. The organization will continue funding this type of research and working alongside leading researchers like Dr. Mossé and respected research institutes like Children’s Hospital of Philadelphia, to develop new treatments for children who are fighting for their lives.

 If you would like to learn more and support Cookies for Kids’ Cancer, please visit http://www.cookiesforkidscancer.org/

 About Cookies for Kids’ Cancer

Cookies for Kids' Cancer, a national 501(c)3 non-profit, is committed to raising funds for research to develop new, improved treatments for pediatric cancer, the #1 disease killer of children in the U.S.  The organization provides the support & resources for individuals & businesses to host grassroots fundraising events, as well as other simple ways to get involved. Founded in 2008 by parents inspired by their young son’s courageous battle, Cookies for Kids’ Cancer has granted $12.5 million to childhood cancer research, funding 91 research grants, resulting in 36 new treatments now in clinical trial and available to children today. Learn more at cookiesforkidscancer.org. Follow the organization on Facebook and Twitter.






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