CIMIT Summer Education Series 2009: Frontiers of Inhalation Technologies in Biomedical Sciences and Clinical Medicine

7.14.2009 Treatment of Respiratory Diseases: Aerosolized Drugs in Medicine: Where are They? SPEAKER: Gerald C. Smaldone, MD, PhD: University of New York at Stony Brook MODERATOR: Dean Hess, PhD: HMS, MGH

Forum Summary

Aerosols are medically useful because they treat the lungs directly and because they can be used to get proteins into systemic circulation.  No major new aerosols have been successfully introduced into clinical practice in the last twenty years, however, and many pharmaceutical companies appear to have lost interest in aerosolized drugs.  For an aerosolized drug to succeed, the following three criteria must be met.  There must be a perceived need for the drug, the development team must possess expertise in both aerosol technology and medicine, and finally, there must be a sound business plan for marketing the drug.  A number of aerosols developed at Stony Brook demonstrate how difficult it can be to meet these three criteria.

Cyclosporine is a drug that suppresses the immune system in order to prevent the body from rejecting transplanted organs.  The drug has many side effects when delivered systemically, so in the 1980s, researchers decided to deliver cyclosporine in an aerosolized form to patients who had received lung transplants.  Aerosolized cyclosporine reduced mortality over four years, but because the researchers did not move quickly to protect their intellectual property rights and to develop a business plan, aerosolized cyclosporine has still not been brought to market.

Ventilator-associated pneumonia is a major problem in intensive care units around the world, and it is currently treated using powerful systemic antibiotics, which promote the evolution of resistant organisms.  Treating ventilator-associated pneumonia with aerosolized antibiotics seems to be more effective than treating the disease using systemic antibiotics, but a commercial system for delivering aerosolized antibiotics is still under development.  The researchers who control the intellectual property rights to the idea of treating ventilator-associated pneumonia with aerosolized drugs feel that they have no control over design of the commercial system being developed.

Pulmonary fibrosis is a chronic disease that affects five million people worldwide. Researchers hypothesize that aerosolized interferon-gamma could be a beneficial treatment for the disease, but this hypothesis is still being investigated.  Preliminary results are promising, but the researchers are having difficulty finding a pharmaceutical company willing to invest in their project, even though the potential market is estimated to be approximately five billion dollars.

---

Post a Comment