Current Clinical Research Studies in CF

Notes on a talk by Robert C. Bocian, M.D., Ph.D.

Fall 1994

"Research is moving quickly on new treatments for CF," said Dr. Robert Bocian, Assistant Professor of Pediatrics, Stanford University School of Medicine and Lucile Salter Packard Children's Hospital at Stanford. He gave quick updates on several promising developments.

Aerosolized Amiloride/UTP therapy

Amiloride is a sodium channel blocker. When people with CF inhale it into their lungs, it decreases excess sodium and water reabsorption, leaving mucus less dehydrated. The thinner mucus is easier to cough up and lung function improves. Studies are underway combining Amiloride with aerosolized Uridine Triphosphate (UTP). The combination is expected to be even more effective in preserving lung function ability.

Passive and active immunization against Pseudomonas aeruginosa

Pseudomonas aeruginosa (P. aeruginosa) is a bacterium that colonizes in the lungs of CF patients, creating an exuberant immuno-inflammatory response. The strong immune response can be stressful to the bodies of people with CF and still may not rid the lungs of the bacteria. A small passive immunization trial is currently underway with 20 CF patients who are colonized with P. aeruginosa. They report no adverse effects from the monthly infusions of antibodies against P. aeruginosa, and a larger study is planned for the near future. In another European study, patients are being actively immunized (vaccinated) against P. aeruginosa. Of the 30 non-colonized CF patients, no long-term adverse effects are reported. After three years, 19 patients remain without P. aeruginosa in their lungs; seven report occasional findings, and four have had repeated findings. The study primarily addresses the safety of the vaccine in the hope that controlled trials could be conducted in the near future.

Aerosolized antibiotic treatment of pulmonary infection

High-dose aerosolized tobramycin looks promising: it appears to kill 98% of the P. aeruginosa the laboratory isolates and does not seem to inordinately encourage the growth of tobramycin-resistant bacteria. Lung function increases in its users. It is less costly and less invasive than an I.V. in the hospital. The drawback to this new system is that it takes several hours of inhalation per day using conventional nebulizer equipment. Researchers are currently studying different nebulizer systems that will be more efficient.

Sinusitis in cystic fibrosis

Sinusitis frequently accompanies cystic fibrosis. Thicker sinus secretions prevent proper sinus drainage and ventilation, and passageways can become blocked. Endoscopic sinus surgery enlarges natural openings to facilitate drainage. It is followed by routine (i.e., every three to four weeks) antimicrobial lavage (a flushing with antibiotics), which is usually performed during a brief office visit. This procedure not only alleviates chronic sinus problems, but may help to curb lung infection flare-ups caused by the constant presence of infectious post-nasal drainage. Some physicians report that if the routine sinus flushings are not performed, the original sinus surgery may need to be repeated within a year.

Lung transplantation

This is obviously a treatment of last resort. However, not everyone qualifies for a transplant. Contraindications include poor kidney function, poor liver function, lack of social support and problems with compliance. Survival statistics are very encouraging. Two years after the transplant, 82% of patients with CF are alive. After five years, 63% of people with CF are alive. Interestingly, these survival rates are higher than for non-CF patients undergoing heart-lung or lung transplants. The most common long-term complication of lung transplant is obliterative bronchiolitis, or OB. If it is detected early and managed well, most patients either recover almost fully or learn to live with a low level of OB.

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