Heartbreak Hill
Why does it take so long to develop new treatments for diseases? And what can we do about it?
 In May 2010, stay-at-home mom Jill Wood learned that her two-year-old son Jonah had an extremely rare and fatal genetic disease called Sanfilippo Type C. For weeks, she was afraid to Google the illness because she’d been warned that would only upset her more. When she finally did, it confirmed her worst fears: a missing enzyme would allow toxic waste products to build up in Jonah’s brain, eventually robbing him of the ability to walk, talk, and see. There is no cure for Sanfilippo syndrome, and the only available treatment—cord blood transplant—carries a 20 percent mortality rate.
Devastated, Wood got into the shower and cried uncontrollably. Then, as she described it, she had an epiphany: she would start a foundation, she would find other options, she would save Jonah’s life.
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New UNC Center for Diabetes seeks to reduce health disparities
 The University of North Carolina at Chapel Hill has received a $3 million, 5-year grant from the National Institute of Diabetes and Digestive and Kidney Diseases to establish the UNC Center for Diabetes Translation Research to Reduce Health Disparities (CDTR). Its mission is to reduce diabetes-related disparities among poor and underserved populations by providing resources and support to foster translational research in North Carolina and beyond. The center’s research will examine and compare different techniques for bringing effective preventive and therapeutic interventions into practice.
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Right-sized Medicine
IPIT celebrates five years with scientific symposium
 DNA is often described as the genetic blueprint of life. Within the 3 billion A’s, T’s, C’s and G’s of the human genome lie clear specifications on eye color, bone structure, even intelligence. Those same instructions can also determine how an individual will respond to a particular drug therapy. This idea of tying genetic variation to drug response is the basis of pharmacogenomics, a field likely to revolutionize medicine.
Leading the effort to apply genetic knowledge to clinical practice is the UNC Institute of Pharmacogenomics and Individualized Therapy (IPIT), which celebrated its five-year anniversary with a symposium September 19 at the William and Ida Friday Center in Chapel Hill. The event featured presentations by the 2011 recipients of the IPIT Awards and concluded with a panel discussion on strategies for rationale therapeutics.
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UNC spin-off receives $3 million Small Business Innovation Research grant
G-Zero Therapeutics, an RTP company started in 2008 based on technologies from the University of North Carolina at Chapel Hill, has been awarded a $3 million Phase II Small Business Innovation Research Grant from the National Institute of Allergy and Infectious Diseases, part of the National Institutes of Health.
The grant will fund commercialization of technology developed by NC TraCS investigator and UNC professor of medicine and genetics, Norman Sharpless, MD, to protect patients from cellular damage caused by radiation or cancer chemotherapy.
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Evidence academy to address tobacco control and lung cancer screening
Tobacco use is the single most preventable cause of disease, disability and death in the United States. Every year, almost half a million people die prematurely from smoking or exposure to secondhand smoke. Over 90% of lung cancer deaths are caused by smoking. The good news, however, is that there are effective interventions to help people stay tobacco-free, as well as new procedures to detect lung cancer at an earlier stage.
The North Carolina Translational and Clinical Sciences (NC TraCS) Institute, UNC Lineberger Comprehensive Cancer Center and the South East Area Health Education Center will host day-long conferences during National Lung Cancer Awareness Month to share evidence-based approaches to addressing these important issues. The event, titled “Moving TALK (Tobacco And Lung Cancer Knowledge) into Action,” is open to health care providers, public health professionals and community members involved in tobacco prevention and control, and lung cancer screening and detection in North Carolina.
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NC TraCS and partners establish CDTR pilot and feasibility program
The UNC Center for Diabetes Translation Research to Reduce Disparities (CDTR) and the North Carolina Translational and Clinical Sciences (NC TraCS) Institute announces a program to facilitate the success of investigators in the field of diabetes translational research. The purpose of this program is to fund junior investigators as they enter the field of diabetes translational research, with a focus on becoming independently funded diabetes researchers, and to fund senior investigators as they change fields and enter the field of diabetes translational research.
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Kudos to Affiliates of NC TraCS
We congratulate those affiliated with the NC TraCS Institute about recent appointments and achievements. Be sure to let us know your news, or those of your colleagues, by emailing
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using the subject line: “Kudos.”
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