Significant gene therapy breakthrough

University of Iowa Health Care researchers played a key role in a landmark international study in which three patients with an inherited form of blindness reported improvement in vision following gene therapy transfer in the retina. It was the first report of successful gene therapy for an inherited eye disease in humans, in this case Leber congenital amaurosis (LCA).   The results were reported online in the New England Journal of Medicine . Although the patients have not achieved normal eyesight, the preliminary results set the stage for further studies of an innovative treatment for LCA and possibly other retinal diseases. "This is very exciting," said Edwin Stone, MD, PhD, the UI investigator who led the genetic testing portion of the study.

Stem cells may fight leukemia

University of Iowa Health Care researchers say leukemia patients may soon have another way to boost their immune systems -- embryonic stem cell-derived bleed cells. For the first time, scientists used embryonic stem cells in mice to develop functioning white blood cells that fight infection. The breakthrough may help doctors eventually use the cells as an alternative source for bone marrow transplants. The procedure has not been tested on humans, but researchers say they're getting closer to that goal. "Embryonic stem cell therapies are no longer distant," said Nicholas Zavazava, MD, the director of transplant research for University of Iowa Health Care and leader of the stem cell study at the Holden Comprehensive Cancer Center.

TRIALS OPEN TO PATIENTS

Smoking cessation

The University of Iowa is seeking volunteers for a study of smokers who would like to quit but are concerned about gaining weight or increasing blood pressure. The study is evaluating three programs designed to help manage weight and control blood pressure after quitting smoking. Qualified participants will be assigned, by chance, to one of three treatment groups. The study has two phases: the first to help smokers quit and the second to help them make and maintain healthy lifestyle changes.

UI LEAD INVESTIGATOR: Mark Vander Weg, PhD, UI associate professor of internal medicine

FOR MORE INFORMATION: Call Bill Barker, recruitment coordinator, at 319-384-5055 or visit PICstudies.org.

Enlarged prostate

Men age 45 and older with benign prostatic hyperplasia (prostate enlargement) are invited to participate in a University of Iowa research study comparing the safety and effectiveness of saw palmetto (a plant extract) to placebo (inactive substance) in reducing symptoms. Study medication(s) and all study tests will be provided at no cost. Compensation for parking is available.

UI LEAD INVESTIGATOR: Karl Kreder, MD, professor of urology

FOR MORE INFORMATION: Contact Diane Meyerholz at 866-236-1432 (toll free) or 319-384-5769 or email diane-meyerholz@uiowa.edu .

More Clinical Trials

FROM BENCH TO BEDSIDE

A gene and a related signaling pathway play a role in the development of glaucoma , according to a research team that included investigators from The University of Iowa. The study, which revealed that over-expression of the gene, sFRP1, elevates pressure in the eye, could help improve glaucoma diagnosis and lead to the development of sight-saving treatments. ? Patients with mild to moderate Parkinson's disease cope less well with the challenges of driving, making significantly more mistakes and driving less safely than neurologically normal elderly drivers, according to University of Iowa Health Care researchers. The researchers found that the driving problems experienced by patients with Parkinson's disease were associated more with cognitive and visual impairment than the severity of physical symptoms such as tremors. "Our findings suggest that people with Parkinson's disease should be particularly careful when they are driving in unfamiliar environments," said Ergun Uc, MD, associate professor of neurology. "They should carefully plan out their route so that the challenges of finding their way do not further degrade their driving safety." ? UI researchers have identified a molecular pathway in blood vessels that controls blood pressure and vascular function . The discovery may help explain why certain drugs for type 2 diabetes also appear to lower patients' blood pressure.