• RETINOIC ACID (FORTE) TRIALS
  • Who is Alice Moore? Alice and her husband John went through Mary Burns' pulmonary rehab program many years ago, and have been very supportive of PERF, and all it stands for. But probably of more interest to all of you, Alice was the first person to go through the Retinoic Acid (Vitamin A) Trials. Did you know that it was because of the perseverance, and financial commitment of John Moore that these studies, so successful in the rat, were adapted to mankind? John feels that Alice's participation in this study prolonged her life.
  • The initial pilot program was set up by Alice's pulmonologist, Dr. Michael Roth at UCLA. As you know, NIH felt that the results were promising enough to warrant the investment of substantial funds by extending the program to other Centers around the United States. Admission of patients into this study will end in June.
  • The following letter from Dr. Roth was written for physicians but we feel it will be of interest to all of you.
  • "Until recently, it was believed that emphysema is an irreversible and progressive process. However, in 1997, an animal model demonstrated that all-trans retinoic acid (ATRA), a vitamin A derivative, could reverse the damage associated with emphysema and regrow new functioning alveoli (Nature Medicine, 3(6):675-7,). A pilot study was conducted and confirmed the feasibility of treating patients with this compound (Am J Respir Crit Care Med, 165:718-723, 2002). Expanding upon this exciting discovery, we helped the NIH/National Heart, Lung and Blood Institute develop a multi-center pilot study called the FORTE Study (Feasibility of Retinoid of Therapy for Emphysema). This is a randomized, double blind, placebo-controlled trial designed to measure the safety, tolerability and clinical/biological impact of ATRA (Vesanoid) and 13-cis retinoic acid (Accutane) on patients with emphysema. These medications are already FDA approved for other indications, though neither is approved for the treatment of emphysema. Outcome measures for this trial will include pulmonary function tests, CT scans, bronchoscopy with BAL for biomarker analysis, and quality of life questionnaires. This research examines an entirely new approach to treating emphysema.We are in the last 6-months of recruitment and are looking for patients, ages 45 and older, with physiologic (FEV1 between 25 to 80% predicted, DLCO < 80%) and radiographic evidence of emphysema (>10% lung involvement by CT). The study is designed with a crossover phase so that every patient who participates will be treated with one of the active medications. Active medical issues, such as unstable angina, renal failure, steroid-dependent lung disease, or ongoing smoking are exclusion criteria.
  • Other than the cost of the initial screening visit (which can be waived depending upon financial conditions), all medications, tests and visits are covered by the study and $500 in compensation will be provided to offset the subject's time and effort for participating. If you are interested, please contact one of us or our study coordinators, Grace Ibrahim and Francine Estrada, at these numbers.
  • (310) 206-7389
    (310) 267-2020 (FAX)
  • If you would like any further information on the rationale or design of the study, please feel free to contact me or visit our Website: http://www.lung.med.ucla.edu/Clinical%20Research/FORTE/
  • Many thanks for your help in advancing the study of this new treatment, Michael D. Roth M.D. Principal Investigator UCLA IRB#: 99-04-057-03C - notice approved Oct 11, 2001
  • Developed and funded through the National Heart, Lung and Blood Institute of the National Institutes of Health, the FORTE Study will enroll a total of 300 emphysema patients at 5 clinical centers across the country (60 patients at each site):
  • UCLA School of Medicine
  • UC San Diego School of Medicine
  • Boston University School of Medicine
  • Columbia University and Long Island Jewish Medical Center
  • University of Pittsburgh.
  • [Those of you outside of the UCLA area may call the above closest listed hospital for further information on joining this study.]
  • We've reviewed the paper that resulted from the first group of patients to receive retinoid acid, published in the American Journal of Respiratory and Critical Care Medicine just last month. In this small group, no beneficial effects on lung function were detected. However, we are all anxiously awaiting the results of the larger study!