Gene Therapy Trial Launched for Treatment of Painful Ascites in Advanced Ovarian Cancer Patients
Researchers at the University of Pennsylvania School of Medicine have opened a study to test a new drug’s ability to control accumulation of fluid in the abdomen – a condition called ascites -- of women with advanced ovarian cancer. Currently the only treatment available for ovarian cancer patients with ascites is to visit their doctor on a regular basis and have the fluid removed directly, through needle drainage. The drug is called DTA-H19 and researchers hypothesize that it will reduce the abdominal fluid accumulation, which is a painful and debilitating symptom that curtails many patients’ quality of life. If DTA-H19 helps control ascites, it would improve patients’ quality of life and may prolong their survival because ascites is a serious complication of ovarian cancer.
“The trial is among just a small handful of trials anywhere in the world for the palliation of ascites,” says George Coukos, MD, PhD, Director of the Penn Ovarian Cancer Research Center and lead investigator on the trial at Penn.
The phase 1/2a trial is open to patients with advanced ovarian cancer who have at least mild ascites. Eligible patients must also have experienced growth of their disease while on two or more previous chemotherapy regimens and have platinum resistant disease. All of the women enrolled in the trial will receive multiple doses of DTA-H19, which will be injected directly into their abdominal cavity where the fluid is accumulating.
DTA-H19 is a piece of DNA that encodes the diphtheria toxin protein under the control of a human cancer gene, called H19, which is only expressed in the tumor cells. When DTA-H19 is injected into a woman’s abdomen, her cells will absorb the DNA. Tumor cells that express the H19 gene will turn on the DTA-H19 construct and produce the diphtheria toxin protein. The diphtheria toxin then poisons and kills specifically the tumor cell. Healthy, non-cancerous cells are safe because they do not express the H19 gene and therefore are not expected to turn on the DTA-H19 construct.
The Ovarian Cancer Research Center at Penn comprises a multidisciplinary team of researchers and physicians dedicated at developing and implementing new detection and treatment strategies for ovarian cancer.
Researchers have already tested DTA-H19 in a phase 1/2a clinical trial in patients with advanced stage bladder cancer. The construct appears to be safe and has anti-tumor activity. The DTA-H19 ovarian cancer clinical trial is also open in Israel. The study is sponsored by Biocancell Ltd, a biotechnology company based in Israel. Patients interested in learning more about the DTA-H19 ovarian cancer trial should call 215-615-4939.