Since the defective CF gene was discovered in 1989,
the pace of CF research has greatly accelerated.
In 1990, scientists successfully made copies of the
normal gene, and added them to CF cells in laboratory
dishes, which corrected the defective cells.
The next major step was achieved in early 1993 when
the first experimental gene therapy treatment was
given to a patient with CF Researchers modified a
common cold virus to act as a delivery vehicle - carrying
the genes to the CF cells in the airways. Several
Foundation-supported studies are underway to test
new versions of the cold virus, and other cutting-edge
technology, such as fat capsules (liposomes) and synthetic
vectors. For more information on CF gene therapy,
see Gene Therapy and Cystic Fibrosis.
new drug therapy developed exclusively for CF in 30
years was approved by the Food and Drug Administration
(FDA) in 1993. In clinical trials, this mucus-thinning
drug called Pulmozyme®, reduced the number of respiratory
infections and improved lung function. In 1995,
a four-year Foundation-supported study showed that
the drug, ibuprofen, reduced the rate of lung inflmamation
in children with CF - under controlled conditions,
and in high doses.
In late 1997, the FDA approved the drug TOBI™ (tobramycin
solution for inhalation). In clinical trials,
this reformulated version of the common antibiotic
improved lung function in people with CF and reduced
the number of hospital stays. The benefits of
TOBI™ are that it can be delivered in a more concentrated
dose directly to the site of CF lung infections more
efficiently, and that it is preservative-free.
The development of TOBI should lead to a long line
of other aerosolized antibiotics for people with CF.
addition, other treatment strategies to correct the
protein product of the gene are currently being tested
in clinical trials. What makes these drugs unique
is that researchers appear to be treating the causes
of CF, not just the symptoms.