GRAND RAPIDS — The Van Andel Research Institute and Helen DeVos Children’s Hospital today announced participation in a national initiative, funded by computer giant Dell Inc. and based on technology developed by VARI and tested in collaboration with Spectrum Health.

The local impact comes on the heels of Dell announcing a major commitment of funding, employee engagement and cloud computing technology to support pediatric cancer research programs globally, including the world’s first FDA-approved personalized medicine trial for pediatric cancer conducted by the Neuroblastoma and Medulloblastoma Translational Research Consortium. The consortium is headquartered at the VARI and includes Helen DeVos Children’s Hospital as a member organization. It is also supported by the VARI affiliate, the Translational Genomics Research Institute.

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Neuroblastoma strikes one in 100,000 children annually, usually before the age of 5, and despite being rare, it is so lethal that it is responsible for one in seven pediatric cancer deaths.  It is the unique and aggressive nature of neuroblastoma that renders conventional approaches ineffective in many children. With little commercially or federally funded research underway because of the relatively small patient base, parents and pediatric oncologists have relied largely on “trial and error” approaches in their search for a treatment that will work from among the hundreds of  available cancer drug trials.

Personalized treatment underway

In May 2011, VARI officials announced the creation of the VARI Pediatric Cancer Translational Research Program, which included the key appointment of Giselle Sholler M.D., a pediatric oncologist and researcher specializing in the treatment of neuroblastoma, and chair of the NMTRC.

Also in May, the NMTRC, VARI and TGen teamed to launch the first FDA-approved personalized medicine trial for pediatric cancer. (www.nmtrc.org/participating-hospitals).

“The NMTRC clinical trial offers hope to those children facing what is among the worst of all pediatric cancers,” Sholler said. “We are confident the genomic-based personalized medicine approach is the right one, and Dell’s contribution will help remove barriers that currently exist in how rapidly and easily we can analyze and share information to benefit our patients.”

The trial, funded to this point primarily by parents of children with neuroblastoma and their family foundations, is based on West Michigan research stretching back at least five years including researchers, patients, and physicians from VARI and Spectrum Health.

“The generous support of Dell for the NMTRC’s ongoing pediatric neuroblastoma clinical trial is the next phase in a five-year process that began with a proof of concept trial that included 14 pediatric patients at Helen DeVos Children’s Hospital in 2006,” said VARI’s Head of the Laboratory of Translational Research Craig P. Webb, who co-directs VARI’s Pediatric Cancer Translational Research Program with Sholler.

The culmination of a five-year, multi-site process

In June 2006, a phase I protocol was launched by Spectrum Health and VARI to recruit up to 50 late stage adult and pediatric cancer patients under the research direction of Webb and the clinical direction of Timothy J. O’Rourke, M.D. and Deanna Mitchell, M.D.  Rourke is a Cancer and Hematology Centers of West Michigan physician and the Betz Family Endowed Chair for Cancer Research at Spectrum Health, and Mitchell is a Spectrum Health Medical Group pediatric hematologist and oncologist who practices at Helen DeVos Children’s Hospital. The results of the “proof of concept” trial were anecdotal but encouraging, and allowed the team to develop the processes required for real time personalized medicine trials.

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“The ability to analyze the gene expression of specific tumor cells and target our therapy as directly as possible to the malignant cells is one of the most exciting developments in oncologic molecular medicine,” said Dr. Mitchell, principle investigator for NMTRC at Helen DeVos Children’s Hospital. “Anecdotal evidence from the phase I trial identified therapeutic agents, which otherwise would not have been recognized as reasonable or feasible for specific patient’s cancers.”

Added Webb: “The protocol illustrated the true meaning of precision medicine, in which experts from multiple disciplines converge as a dedicated team to integrate clinical expertise with unprecedented advances in knowledge and technologies towards achieving our common goal of improving patient outcomes. We are now applying that same approach, on a larger scale, to targeted pediatric populations through this Dell-funded project.”

The project is also receiving in-kind support from Grand Rapids-based Intervention Insights, the Medical Mile’s first life science firm devoted to developing treatments based on Dr. Webb’s personalized medicine technology.

Dell expanding its Powering the Possible program

The expansion of Dell’s Powering the Possible program to focus on neuroblastoma and other pediatric cancers is due to the devastating nature of the disease and to address the void of new and innovative treatments available for children. Since the 1980s, the Food and Drug Administration (FDA) has approved only one new treatment for any type of childhood cancer, compared with 50 approved treatments for adult cancers in the same time period. Through Powering the Possible, Dell is making a multi-year, multi-million dollar commitment of funds and employee volunteerism to support innovative pediatric treatment programs globally.

Dell-donated cloud gives TGen and NMTRC power to do more for pediatric cancer

Dell’s donated cloud system will provide needed computing power to help increase TGen’s gene sequencing and analysis capacity by 1,200 percent and improve collaboration between the team of physicians, genetic researchers, pharmacists and computer scientists working on the trial.

Specifically, scientists and physicians will use the donated cloud to investigate new technologies that accelerate genetic analysis and identification of targeted treatments for each patient from months to days. The additional computing power will also improve the availability of critical information and allow researchers to develop a real-time knowledge repository of the latest findings on the most effective treatments for oncologists to use globally.

The researchers also intend to use the donated cloud to expand the program’s participation from a handful of children today to hundreds of children over the next three years, with the goal of establishing an information framework that, subject to regulatory approval, could one day help thousands of pediatric cancer patients. The new TGen cloud will also facilitate rapid transfer of information to international partners and lay the groundwork for expansion of the trial to additional types of childhood cancers in the future.

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More at www.nmtrc.org, www.vai.org, www.beatnb.org or www.helendevoschildrens.org.