Caritas St. Elizabeth's Medical Center to Begin NIH Funded Gene Transfer Trial for Diabetic Neuropathy; Gene Transfer to Be Used for Patients with Painful Condition.

Business Editors/Health/Medical Writers

BOSTON--(BUSINESS WIRE)--Dec. 11, 2002

Caritas St. Elizabeth's Medical Center today announced that it has begun enrolling patients in a trial to evaluate the safety and impact of VEGF (Vascular Endothelial Growth Factor) gene transfer on patients with diabetic neuropathy. The trial is being funded as part of a $10.2 million dollar grant awarded to Caritas St. Elizabeth's by the National Institutes of Health (NIH) to establish a Center of Excellence in Gene Therapy.

Diabetic neuropathy is a potentially serious condition that is caused in part by blockage of small blood vessels within the nerves in the legs. The condition is treated with medications to reduce pain and numbness; but it is considered irreversible. However, data gleaned from an early stage clinical trial have suggested that diabetic neuropathy may be improved with gene therapy.

Allan Ropper, M.D., chief of Neurology and recipient of the Remondi chair in Neurology at Caritas St. Elizabeth's, is the principal investigator of the trial, and Douglas Losordo, M.D., chief of Cardiovascular Research, is the principal investigator of the Program Project Grant. Caritas St. Elizabeth's expects to enroll 200 patients for this Phase I/II trial over the next four years.

According to the Neuropathy Association, 20 million Americans suffer from various forms of neuropathy. Patients suffering from this ailment experience numbness, painful burning sensations, tingling, and weakness. Diabetes is the commonest cause of neuropathy in the U.S.

Last year, a preliminary study conducted at Caritas St. Elizabeth's, the results of which were published in Archives of Neurology, indicated that a related problem called ischemic neuropathy is reversed using the gene transfer of VEGF. Some of the patients in the trial had diabetic neuropathy and they too were improved. Work in the laboratory in an experimental type of pure diabetic neuropathy treated with gene therapy also showed a great deal of improvement in nerve function and in blood flow to the nerves.

"Our early findings suggest that ischemic neuropathy may be a reversible condition, even for diabetics, who tend to have the most severe cases of neuropathy," said Dr. Ropper. "We hope that as we continue our research, it may one day be possible to alleviate the suffering for the many patients who currently endure this sometimes painful condition."

In the preliminary study, VEGF was injected into a patient's critically ischemic limb. Seventeen patients, (19 limbs--two patients had both legs treated) completed the study. Six months following treatment, an improvement in neurological symptoms was seen in more than 50 percent of the patients. Sixty-three percent of treated limbs showed improvement in neurologic symptoms such as severe pain and loss of sensation, and 53 percent of treated limbs showed improved nerve responsiveness.

The $10.2 million dollar grant awarded to Caritas St. Elizabeth's by the NIH will also include a trial that will examine VEGF gene transfer to prevent coronary artery restenosis, the narrowing of coronary arteries due to plaque. The application of the gene is done in conjunction with angioplasty and the delivery of a stent for patients with narrowed coronary arteries. In addition, a trial conducted in cooperation with the Cleveland Clinic will evaluate the use of gene transfer to improve heart function for patients with congestive heart failure due to coronary artery disease, which occurs when the heart muscle has been damaged and is too weak to pump blood to the rest of the body.

Caritas St. Elizabeth's pioneered the use of gene transfer in 1994 when the world's first clinical trials were conducted by Jeffrey Isner, M.D., to treat patients with severe peripheral vascular disease (blockages in the leg arteries). History was made again in 1998 when the procedure was used to treat patients who had low blood flow to certain areas of the heart due to coronary artery disease.

For more information on the trials and the enrollment procedure, please phone the information line, 1-888-311-4363.

Caritas St. Elizabeth's Medical Center of Boston is a major academic medical center affiliated with Tufts University School of Medicine. Areas of medical excellence include cardiology and cardiovascular research, neurology, women's health, high-risk obstetrics, hematology/oncology, bone and joint health, pulmonary medicine and gastroenterology. Caritas St. Elizabeth's is a member of Caritas Christi Health Care, the second largest health care system in Massachusetts.