Cystic Fibrosis
Cystic Fibrosis (CF) is a rare disease caused by a defected CFTR gene. It results in a buildup of mucus which impairs breathing and makes the lungs susceptible to bacterial infections. Unfortunately, there are not many treatments available and there is no cure for the disease. Additionally, the treatments which are on the market work for a very limited group of CF patients. This is because different patients have different types of mutations, and some of the patients don’t make the protein at all. This particular subset of patients equates to 10% of all CF diagnoses.
Currently, 70,000 people across the world are living with a CF diagnosis. 30,000 of these patients are in the United States.
New Findings
Martin D. Burke, a professor at the University of Illinois in Champaign, has recently conducted a new study which could lead to a new treatment option for this patient population. Supported by the NIH, this study investigated amphotericin in CF. Amphotericin is actually a very widely used drug already on the market. It’s currently prescribed as an antifungal therapy.
Dr. Burke’s study investigated amphotericin in animal models and human cells. Basically, amphotericin acts as a molecule surrogate, performing the function of the defective protein. This study is unique because it doesn’t try to correct the defective protein, and it isn’t a form of gene therapy (sadly, this has yet to be found effective in the lungs). Researchers are calling the therapy a molecular prosthetic.
Some of the perceived benefits of this form of therapy is that is has the potential to be effective for CF patients with all types of mutations. It even is believed that it could aid the 10% of patients who don’t have the protein at all. Additionally, because it can be delivered straight to the lungs, it will likely eliminate many of the common side effects of treatment.
Dr. Burke’s study ultimately found improved-
- Lung function
- pH levels
- Viscosity
- Antibacterial Activity
All of the above effects will help the lungs fight infections, which are so common in CF.
The findings from Dr. Burke’s study will be published in Nature.
Looking Forward
While human studies are still needed to investigate the full efficacy and safety of amphotericin, these initial findings are extremely promising. Researchers are hopeful that this therapy could become a new treatment option for this patient population with a high unmet need. It is especially exciting for the 10% of the CF community who do not have the protein at all, as they currently have a severe lack of therapeutic options.
Stay tuned to hear more about this investigation and amphotericin’s progress. In the meantime, you can read more about this latest study here.
