UTHSCT physicians help lead clinical trial of new drug that offers hope for people with MAC, a rare but serious lung disease

Thursday, August 16, 2012

Two physicians at The University of Texas Health Science Center at Tyler are helping lead a clinical trial of a new drug that offers significant hope to people suffering from Mycobacterium avium complex (MAC), a rare but serious lung disease.

David Griffith, MD, and Richard Wallace, MD, are co-principal investigators in the study designed to determine if inhaled liposomal amikacin, (trade name ARIKACE) can help patients with MAC and other nontuberculous mycobacterial (NTM) lung diseases.

“The rate of MAC lung disease is increasing in the United States. And it is difficult to treat,” said Dr. Wallace, an infectious disease specialist who is nationally known for his success in treating people with MAC.

Dr. Griffith, a pulmonary physician at UTHSCT, agreed.

“While almost 98 percent of the people who contract tuberculosis are successfully treated, only 75 percent of people with MAC are cured. This drug offers the remaining 25 percent the hope of better treatment outcomes,” he said.

Dr. Griffith is the lead author of diagnosis and treatment guidelines for NTM approved by the American Thoracic Society and the Infectious Disease Society of America. MAC is the most common NTM to cause infections in people. Dr. Wallace recently was ranked in the top 1 percent of U.S. physicians who treat MAC by U.S. News and World Report magazine.

Principal investigator of the MAC clinical trial is Kenneth N. Olivier, MD, staff pulmonologist in the Laboratory of Clinical Infectious Diseases at the National Institute of Allergy and Infectious Diseases, part of the National Institutes of Health.

It’s estimated that seven or eight out of 100,000 people in the United States develop an NTM infection each year. The rate of infection is higher for people over 50 years of age and much higher for women older than 50, Dr. Griffith added.

MAC, while not contagious, is a serious disease, with symptoms including cough, production of sputum (secretions from the lungs and tubes that carry air to the lungs), fatigue, weight loss, blood in the sputum, night sweats, and fever. It is thought to be environmentally acquired, meaning that the germs get into the lungs or body via air, water, or soil.

Risk factors include having lung tissue that has been damaged by tuberculosis, heavy smoking, or bronchiectasis – a condition that causes destruction of the tubes that carry air in and out of the lungs.

This phase 2 clinical trial is funded by Insmed Inc., the company making the new drug. The study’s purpose is to evaluate the effectiveness of ARIKACE in treating people with NTM infections such as MAC and to identify ARIKACE’s common short-term side effects and risks.

If preliminary results indicate ARIKACE is safe and effective, it will be evaluated in larger phase 3 trials. These studies will gather additional information to see if the benefits of ARIKACE outweigh the risks and to give physicians the data they need to correctly prescribe it.

UTHSCT is one of about a dozen sites taking part in this clinical trial. These include the Mayo Clinic, National Jewish Health in Denver, and the National Institutes of Health.

Most of the individuals being enrolled here are UTHSCT patients, Dr. Griffith said. To participate in the study, individuals must be referred by their physicians and meet certain criteria.

Dr. Wallace is encouraged by the emergence of a new drug for MAC.

“There haven’t been many developments in the treatment of MAC in 20 years. This is the first glimmer of hope since the early 1990s,” he said.

For more than 60 years, The University of Texas Health Science Center at Tyler has provided excellent patient care and cutting-edge treatment, specializing in pulmonary disease, cancer, heart disease, primary care, and the disciplines that support them. UTHSCT’s annual operating budget of $125 million represents a major economic impact of over $287 million for the Northeast Texas region. Since 2002, scientists in the Center for Biomedical Research have been awarded more than $120 million in research dollars. As the academic health science center for Northeast Texas, its graduate medical education programs – with residencies in family medicine and occupational medicine – provide doctors for many communities throughout the region and beyond. UTHSCT is also the program sponsor of the residency program in internal medicine at Good Shepherd Medical Center in Longview.

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