The UCLA ATRA Emphysema Trial
An Experimental Treatment for Emphysema
Has been completed. Analysis of the data is underway.
What is Emphysema?
Emphysema is a serious lung disease that affects over 2 million people in the United States. The most common cause of emphysema is cigarette smoking, although other causes such as an inherited genetic deficiency do exist (alpha-1 antitrypsin disease). In patients with emphysema, their normal lung tissue is gradually eaten away and replaced by large holes - reducing the ability of the lung to breath or take-up oxygen. Until recently, these changes were considered to be irreversible.
What standard treatments are available?
Current treatments for emphysema focus on relieving the symptoms of shortness of breath. They include inhaled medications to reduce spasm in the breathing passages (such as albuterol and atrovent), medications to reduce swelling and irritation in the airways (such as prednisone or steroid inhalers), antibiotics to treat episodes of bronchitis, and supplemental oxygen in people who have low oxygen values. None of these treatments actually reverse or improve the emphysematous damage. Patients interested in seeing a UCLA Lung Specialist about their emphysema or other lung disease should call: (310) 206-7858.
A new surgical treatment for emphysema is under investigation - Lung Volume Reduction Surgery. This experimental treatment involves surgery to remove part of the lung. This "lung reduction" is believed to allow the remaining lung to expand more normally and to work better. It is not yet clear what type of patients should have this surgery, or how long the effects will last. People interested in this should contact the "National Emphysema Treatment Trial (NETT Study)" which is going on at several institutions across the United States.
Vitamin A as a New Treatment for Emphysema
Until recently, the damage caused by emphysema was considered to be irreversible. However, landmark research performed at Georgetown University has provided some hope for a cure - a treatment that will regenerate lung tissue. In their work, Drs. Gloria and Donald Massaro tested a derivative of Vitamin A, called all-trans-retinoic acid (ATRA), on rats suffering from an experimental form of emphysema. Treatment with ATRA helped regenerate their lungs and restore their damaged lungs and lung function (pictures from publication by Massaro and Massaro, Nature Medicine, June, 1997).
The UCLA ATRA Emphysema Trial
Drs. Michael Roth and Jenny Mao, in the Division of Pulmonary and Critical Care Medicine at UCLA, have developed the first human study to test whether a pill containing ATRA will help reverse the effects of emphysema in patients. There is no way to predict whether people will respond to this medication in the same manner as rats do - that is what will be evaluated in this research study. UCLA is looking for patients with symptomatic emphysema who would like to participate in this research. Patients that pass a series of screening tests will be enrolled in a six-month program to test the effectiveness of ATRA. All testing will be performed at UCLA and patients will be evaluated for improvements in their breathing, lung function and lung CT-scans. This clinical trial has been approved as an investigational study by the Food and Drug Administration and by the UCLA Human Subject Protection Committee.
The UCLA ATRA Emphysema Trial has been completed. Analysis of the data is underway. This research continues as The FORTE Study, which stands for "Feasibility Of Retinoid Therapy for Emphysema". The FORTE Study is a multi-center clinical trial sponsored by the National Heart, Lung and Blood Institute of the National Institutes of Health.