TGen and Scottsdale Healthcare Begin Study of New Drug for Patients with Solid Tumors with a Focus on Brain Tumors and Gallbladder Bile Duct Cancer

Patients at the Virginia G. Piper Cancer Center Clinical Trials Will be the First to Receive AG-120


SCOTTSDALE, Ariz. — June 17, 2014 — The Virginia G. Piper Cancer Center at Scottsdale Healthcare and the Translational Genomics Research Institute (TGen) are studying the safety and effectiveness of a new drug, AG-120, for treatment of patients with solid tumors, especially those with brain tumors and gallbladder bile duct cancer.

“AG-120 is designed specifically for those patients who carry the IDH1 gene mutation,” said Dr. Daniel D. Von Hoff, Distinguished Professor and Physician-In-Chief at TGen, and Chief Scientific Officer for the Virginia G. Piper Cancer Center Clinical Trials at Scottsdale Healthcare, a partnership between Scottsdale Healthcare and TGen that delivers new treatments to cancer patients based on precision medicine.

The IDH1 gene mutation is most commonly found in gliomas, which make up the largest group of “primary” brain tumors, those that start in the brain. Gliomas include all tumors arising from the gluey or supportive tissue of the brain. They represent about 30 percent of all brain tumors, and about 80 percent of all malignant brain tumors.

As with all brain cancers, gliomas are difficult to treat and many grow back after surgery, radiation and standard of care chemotherapy. Many drugs cannot get to the brain because of a filtering mechanism in the body called the blood-brain barrier.

“There is a great need for more effective treatments for patients with gliomas and other solid tumors, such as gallbladder bile duct cancer,” said Dr. Von Hoff, who is the Principal Investigator for AG-120 clinical trial. This study will enroll as many as 50 patients. Researchers will use precision medicine to match treatments to patient’s specific genomic, or molecular, makeups.

Mutations in isocitrate dehydrogenase (IDH) 1 and 2, originally discovered in 2008, occur in the vast majority of low-grade gliomas and secondary high-grade gliomas. IDH mutations are oncogenic, meaning they have the potential to cause cancer. These mutations occur early in the formation of gliomas and in gallbladder bile duct cancer.

AG-120 is produced by Agios Pharmaceuticals Inc., based in Cambridge, Mass.

IDH1 is a metabolic enzyme identified by Agios as a protein that is mutated in a wide range of malignant tumors. Agios and its collaborators recently demonstrated that IDH1 mutations initiate and drive cancer growth by blocking differentiation, or maturation, of primitive cells. According to Agios, the inhibition of these mutated proteins may lead to clinical benefit for those cancer patients whose tumors carry them.

About the Virginia G. Piper Cancer Center at Scottsdale Healthcare
The Virginia G. Piper Cancer Center at Scottsdale Healthcare in Scottsdale, Ariz. offers comprehensive cancer treatment and research through clinical trials, diagnosis, treatment, prevention and support services in collaboration with leading scientific researchers and community oncologists. The Virginia G. Piper Cancer Center at Scottsdale Healthcare, Scottsdale Healthcare Research Institute, Scottsdale Healthcare Osborn Medical Center, Scottsdale Healthcare Shea Medical Center and Scottsdale Healthcare Thompson Peak Hospital and related entities are part of the non-profit Scottsdale Lincoln Health Network. For more information,

About Agios Pharmaceuticals, Inc.
Agios Pharmaceuticals is focused on discovering and developing novel drugs to treat cancer and inborn errors of metabolism, or IEMs, which are rare genetic metabolic diseases, through scientific leadership in the field of cellular metabolism. In addition to an active research and discovery pipeline across both therapeutic areas, Agios has multiple first-in-class lead product candidates in cancer metabolism and IEMs in clinical and/or preclinical development. All Agios programs focus on genetically identified patient populations, leveraging our knowledge of metabolism, biology and genomics. For more information, please visit our website at


What’s New at TGen

TGen Concussion Research Fund is first under ACF’s new Jerry Colangelo Center for Sports Philanthropy


PHOENIX, Ariz. — June 10, 2014 — The Arizona Community Foundation has created a new philanthropy center named for Arizona sports mogul Jerry Colangelo, and the Center’s first project is a fund to support concussion research led by theTranslational Genomics Research Institute (TGen), in partnership with Riddell, Barrow Neurological Institute and Arizona State University.

The TGen Concussion Research Fund is the first charitable endeavor under the Arizona Community Foundation’s newly established Jerry Colangelo Center for Sports Philanthropy. ACF has contributed $25,000 to the TGen fund in support of this important work.

A generous philanthropist and one of the most influential leaders in business and sports in Arizona and across the nation, Colangelo has partnered with ACF to encourage athletes to support causes of personal importance and create a charitable legacy.  The Colangelo Center manages funds supporting sports-related causes and provides philanthropic giving vehicles and services for professional and retired athletes looking to make a positive impact in their communities.

“Sports have provided me most of the wonderful opportunities that I’ve enjoyed in my life, and like so many others who have benefited from this industry, we must finds ways to give back,” said Mr. Colangelo, the former owner of the Phoenix Suns and the Arizona Diamondbacks.

“Corporate and personal giving through ACF will allow for this, and I am proud to lend my name to their new Center for Sports Philanthropy,” said Colangelo, who now serves as CEO of USA Basketball.

The TGen Concussion Research Fund will support a TGen-led collaboration that began in 2013 with Riddell, Barrow Neurological Institute, and Arizona State University’s Sun Devil football team. This study is working to find a better way to diagnose concussions on the field. It compares football player head impact data with information uncovered through genetic testing of the players. The research could enable physicians to better identify when a player is concussed, and also when they might be expected to recover and get back on the field.

“By establishing the Center for Sports Philanthropy and the TGen Concussion Research Fund, Jerry Colangelo and the Arizona Community Foundation have generously created a permanent funding opportunity that will help find better ways to care for athletes now and in the future,” said Dr. Jeffrey Trent, TGen President and Research Director.

Dan Arment, President of Riddell, concurred:  “As a partner in this important study led by TGen, we at Riddell hope to answer a number of key questions that will lead to improved player protection, inform our continued development of new football helmet innovations, and further refine player monitoring technology. With this research, we are on the cusp of identifying a more definitive way to diagnose concussive injury, which will benefit football players and athletes in the broader sports universe.”

“Sun Devil Athletics is a community asset and we take great pride in collaborating with these three important local leaders in the Arizona Community Foundation, TGen and the Barrow Neurological Institute, as well as industry pioneer Riddell,” Vice President for Sun Devil Athletics Ray Anderson said. “We are always striving to advance and progress all aspects of collegiate athletics, and the health and well-being of our student-athletes is at the forefront of this goal.”

“This research is the ‘holy grail’ of concussion diagnosis,” said Dr. Javier Cardenas, M.D., Director of the Barrow Concussion Center. “The research combines the greatest expertise in the state, and is another great example of how Arizona is leading the nation in concussion care.”

The Arizona Community Foundation provides the charitable vehicle for supporters to make tax-deductible donations to a professionally managed fund dedicated exclusively to the project. “We are honored to be TGen’s partner in supporting this critical genomic research that we expect will lead to better medical diagnosis and treatment of sports-related concussions,” said Steve Seleznow, president & CEO of the Arizona Community Foundation. “As one of TGen’s founding funders, ACF takes great pride in supporting their ever-expanding research that is improving medicine and healthcare here and across the globe. This new fund and the work it supports are yet another clear example of TGen’s value and importance in developing breakthrough solutions to major health problems.”

“We are grateful to Jerry Colangelo and the Arizona Community Foundation for bringing their leadership to our fight against concussions,” said Michael Bassoff, President of the TGen Foundation. “Both have incredible legacies of helping the community and we are pleased that they have embraced this cause.”

Donors interested in making a charitable donation to the TGen Concussion Research Fund can give securely online with a credit card at, or by mailing a check to ACF, 2201 E. Camelback Road, Suite 405B, Phoenix, AZ 85016, with a notation of “TGENCO” in the memo line. Or, call 602-682-2042 for personal assistance.

About the Study
Too often, concussions and mild traumatic brain injury (mTBI) go undiagnosed. There is mounting evidence that repeatedly undiagnosed mTBI can lead to long-term health problems. Unfortunately, there currently are no tests available to diagnose this damage in real time.

In May 2013, TGen partnered with Riddell, the leader in football helmet technology and innovation, Barrow Neurological Institute and A. T. Still University to embark on a study to advance athlete concussion detection and treatment. Expanding upon TGen’s work in the area of head trauma, the investigative team led by Drs. Kendall Van Keuren-Jensen and Matt Huentleman is working to identify biomarkers associated with documented concussion and sub-clinical concussion.

In September 2013, ASU and its Sun Devil football program joined the study. Riddell equipped Sun Devil student-athletes with the company’s proprietary helmet sensors and head impact monitoring system to collect impact data. Biological information from a player’s genome is merged with real-time head impact data provided by Riddell’s Sideline Response System (SRS). SRS provides researchers, athletic staff and players with information about the frequency and severity of head impacts during games and practices. The Sun Devils’ medical team, consisting of athletic trainers and physicians, did not see the data or interpret any results until the end of the season, and the student-athletes wearing the Riddell SRS sensors in their helmets volunteered to partake in the study.

TGen is measuring changes in RNA (DNA’s complimentary nucleic acid), associated with head impacts through samples of blood, saliva and urine. By using RNA as a sensitive indicator of changing conditions underlying brain injuries, this data could improve the ability to detect at-risk patients for future neurological, cognitive and behavioral complications.

Often, head injuries present no physical changes that can be viewed using conventional imaging techniques.

However, symptoms experienced by concussed athletes (headaches, loss of memory, temporary unconsciousness, confusion, drowsiness) indicate there are functional changes that can last several days. These symptoms possibly result from structural brain damage — only detectable at the molecular level — including torn axons and synaptic connections that prevent transmission of the brain’s electrical impulses.

As part of the study, TGen is working with the Barrow Neurological Institute, whose B.R.A.I.N.S. (Barrow Resource for Acquired Injury to the Nervous System) project treats patients age 15 and older who have sustained a traumatic brain or spinal cord injury. Joining with Barrow are athletic trainers from A.T. Still University and SAFE Football, which teaches alternative game-play techniques that reduce head impacts while increasing competitiveness.

About Arizona Community Foundation:
Established in 1978, the Arizona Community Foundation is a statewide family of charitable funds supported by thousands of Arizonans. With five regional offices serving communities across Arizona, ACF is among the top 30 community foundations in the nation with more than 
$650 million in trust and endowment assets, and is certified under the National Standards for U.S. Community Foundations. Last year, ACF and its affiliates awarded more than $40 million in grants and scholarship funding to some 3,000 nonprofit organizations, schools and government agencies. More information is available at

About Arizona State University:
Arizona State University enrolls more than 73,000 undergraduate, graduate, online and professional students on its four campuses configured across the Phoenix metropolitan area. Offering outstanding resources for research and academics, ASU has developed a new model for the American research university, creating an institution that is committed to excellence, access and impact. As a New American University, ASU pursues research that contributes to the public good and assumes major responsibility for the economic, social and cultural vitality of the communities that surround it.

Medical Heroes Appreciation 5K Run & Walk


Partnership with DIA to Honor People Who Give the Gift of Participation in Clinical Research

BOSTON, MA – March 1, 2014 — The Center for Information and Study on Clinical Research Participation (CISCRP), an independent non-profit, is excited to announce the launch of a new event — Medical Heroes Appreciation 5K Run & Walk in San Diego — to celebrate the volunteers who give the gift of participation in clinical research. The first annual Medical Heroes Appreciation 5K Run & Walk will coincide with the Drug Information Association (DIA) 2014 Annual Meeting. The event will take place on Monday, June 16, 2014 outside the San Diego Convention Center from 6:45-8:00am. Participants are encouraged to register early as space is limited. To register, visit

“Medical Heroes are the millions of people who help advance public health and medical knowledge by taking part in clinical trials each year,” said CISCRP’s founder, Ken Getz. “This special event recognizes their gift, raises public awareness and appreciation, and hopefully will become an annual tradition.”

Running and walking enthusiasts and all supporters of CISCRP’s mission are invited to participate or attend the event to show their support. Proceeds from the event will support education and outreach programs to patients and families interested in learning more about the clinical research process.

“We are honored the inaugural offering of the Medical Heroes Appreciation 5K will be held during the 50th Anniversary of DIA’s Annual Meeting.” said Lori Risboskin, DIA’s Associate Director Event Planning & Exhibits. “DIA members understand the importance of clinical research volunteers and appreciate their participation in clinical trials. We’re delighted to help CISCRP launch this special event.”

Registrants will have the opportunity to check-in early on Sunday June 15, 2014 from 8am – 9am, 12pm – 1pm, and 3pm – 6pm to receive event materials and giveaways. Regular check-in the day of the event begins at 6am; and the 5K Run & Walk will begin at 6:45am. For more information about the event, to volunteer, register or become a sponsor, visit Questions about event registration should go to Ellyn Getz (617) 725-2750, e-mail, or call toll free 1-877-MED-HERO.


CISCRP is a 501(c)(3) non-profit organization dedicated to engaging the public and patients as partners in the clinical research process. CISCRP provides free education and outreach to the general public and patient communities. Visit for more information or to support CISCRP.

About the DIA 2014 50th Annual Meeting

The DIA Annual Meeting is the premier event for professionals involved in the discovery, development and lifecycle management of pharmaceuticals, biotechnology, medical devices and related medical products. No other industry event rivals the depth and breadth of experience that this meeting delivers through 20 interest-area tracks, 260+ program offerings and 18 pre-conference tutorials. The presentations are geared to attendees of all disciplines and experience levels. Visit our website at and follow DIA at: LinkedIn, Twitter, YouTube, Facebook, Flickr, and Pinterest.



TGen News

New therapy for pancreatic cancer patients shows promising results

TGen-Scottsdale Healthcare lead international clinical trials in advance of FDA application for NAPOLI-1

SCOTTSDALE, Ariz. — June 6, 2014 — A clinical trial conducted by researchers at the Virginia G. Piper Cancer Center Clinical Trials, a partnership between Scottsdale Healthcare and the Translational Genomics Research Institute (TGen), showed that a new drug called MM-398, given in combination with 5-flourouracil (5FU) and leucovorin, produced a significant overall survival rate in patients with advanced, previously-treated pancreatic cancer.

The NAPOLI-1 (NAnoliPOsomaL Irinotecan) Phase 3 study — a final confirmation of a drug’s safety and effectiveness — was conducted among patients with metastatic pancreatic cancer who previously received gemcitibine, which has been the standard-of-care therapy for such patients.

The study, sponsored by Merrimack Pharmaceuticals, evaluated 417 patients enrolled at more than 100 sites in North America, South America, Europe, Asia and Australia, including patients at the Virginia G. Piper Cancer Center at Scottsdale Healthcare. Merrimack expects to submit a New Drug Application this year to the U.S. Food and Drug Administration (FDA) for the MM-398 combination regimen.

“This demonstration of a survival benefit from the MM-398 plus 5-FU and leucovorin combination is particularly important given that we have very few treatment options for patients in this tough clinical setting,” said Dr. Daniel D. Von Hoff, MD, FACP, global principal investigator of the NAPOLI-1 study, Chief Scientific Officer for Scottsdale Healthcare’s Virginia G. Piper Cancer Center Clinical Trials and Physician-In-Chief and Distinguished Professor at TGen. “The results of the NAPOLI-1 study are important because of the critical need to help patients with this devastating illness and move forward towards FDA approval.”

The combination of MM-398 with 5-fluorouracil (5-FU) and leucovorin achieved an overall survival of 6.1 months, a 1.9 month improvement over the 4.2 month survival demonstrated by the control arm of 5-FU and leucovorin alone.

Each year in the U.S., nearly 46,000 people are diagnosed with pancreatic cancer, and more than 39,000 patients die, making it the fourth leading cause of cancer death. Only about 1 in 4 patients survive more than one year after diagnosis, and only 6 percent survivor more than five years.

Symptoms of pancreatic cancer usually do not appear until the cancer is in its late stages, making it difficult to treat. Once the disease spreads to other parts of the body, most patients are not candidates for surgery and receive chemotherapy as their primary treatment.

This study will be presented at the European Society for Medical Oncology World Conference on Gastrointestinal Cancer being held June 25-28 in Barcelona, Spain.

Patients seeking information about research studies may contact the Virginia G. Piper Cancer Center Clinical Trials at Scottsdale Healthcare at 480-323-1339 or toll free at 1-877-273-3713 or e-mail:

About the Virginia G. Piper Cancer Center at Scottsdale Healthcare
The Virginia G. Piper Cancer Center at Scottsdale Healthcare in Scottsdale, Ariz. offers comprehensive cancer treatment and research through clinical trials, diagnosis, treatment, prevention and support services in collaboration with leading scientific researchers and community oncologists. The Virginia G. Piper Cancer Center at Scottsdale Healthcare, Scottsdale Healthcare Research Institute, Scottsdale Healthcare Osborn Medical Center, Scottsdale Healthcare Shea Medical Center and Scottsdale Healthcare Thompson Peak Hospital and related entities are affiliates of the non-profit Scottsdale Lincoln Health Network. For more information,

About TGen
Translational Genomics Research Institute (TGen) is a Phoenix, Arizona-based non-profit organization dedicated to conducting groundbreaking research with life changing results. TGen is focused on helping patients with cancer, neurological disorders and diabetes, through cutting edge translational research (the process of rapidly moving research towards patient benefit).  TGen physicians and scientists work to unravel the genetic components of both common and rare complex diseases in adults and children. Working with collaborators in the scientific and medical communities literally worldwide, TGen makes a substantial contribution to help our patients through efficiency and effectiveness of the translational process. For more information,