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16 HEALTH • Spring 2015 There's new hope for patients, but keeping the cash flowing is tough \\ By Phyllis Hanlon F or the last 15 years, David R. Tabatadze, managing partner and co-founder of Zata Pharmaceuticals Inc. in Worcester, has continued the work of his mentor, the late Dr. Paul Zamecnik, who 50 years ago developed oligotherapeutics, a synthetic compound that restores mutated genes in patients with cystic fibrosis. In spite of recognition from the Massachusetts Biotechnology Council (MassBio) and pub- lication of several research papers, Zata has struggled to fund its research. A Small Business Innovation Research (SBIR) grant from the National Institutes of Health (NIH) and self- funding through equity loans have been the company's major sources of financing. CAN GENE THERAPY BE PROFITABLE? Cystic fibrosis, an inherited genetic disease, affects the lungs, pancreas, liver and intestines. Approximately 30,000 children and adults in the United States have cystic fibrosis (CF) and can expect to live an average of 37 years. There's no cure for CF. Treatments such as antibi- otics, medications to improve lung function, nutri- tional supplements and medical devices to clear mucus from the airways and ease breathing target the symptoms of the disease, but not the cause. "Restoration of mutated genes with oligothera- peutics will allow them to have full lives without restriction. How soon could this be done? Best sce- nario, five years," said Tabatadze. But that timeline relies on sufficient funding to keep the research engine going. "In 50 years, billions of dollars have been spent and nothing has been created," Tabatadze said. "I very badly want to see the concept my mentor devel- oped become commercial." Time- and cash-intensive research As scientists continue to unravel the mysteries of the human body, gene therapy research to cure seri- ous medical conditions has shifted into high gear. But taking a potentially viable therapy from lab to market is a time-and money-intensive process, according to Christian Mueller, assistant professor at the University of Massachusetts Medical School