Title: Cystis Fibrosis Not Just For Kids Anymore
Category: Child Care
Far more patients with cystic fibrosis are now living into adulthood. Because of better treatments, many internists and family doctors are now treating them. This is one new challenge which we are all happy to face since we didn't have this option a generation ago. Many CF patients are going on to have children of their own or adopt children since most males CF patients are sterile. Tulane University Medical Center is involved with much of the national and international research on this disease. Last August, researchers at Chapel Hill in North Carolina developed a gene-altered mouse with cystic fibrosis which should allow them to learn even more and hopefully one day provide a cure for this usually fatal disease.
Most cystic fibrosis patients begin to have trouble in infancy which becomes progressively worse throughout their childhood. The major cause of the disease is an abnormality in sodium transport which causes their exocrine gland secretions to be abnormal. This causes sticky mucous in the lungs which leads to infection and complications. It also causes destruction of the pancreas which leads to GI abnormalities and difficulty in absorbing certain proteins and fats. Patients with CF have a difficult time gaining weight and need huge amounts just to maintain their usually thin frame. Fortunately, with the tremendous increase in knowledge we have gained over the past 20 years along with constantly improving medicines, CF can be treated and controlled in most cases. Nevertheless, it is still a progressive disease with constant ongoing scarring in the lungs and other parts of the body.
One hope for the future is a treatment with DNAse. DNAse is a naturally occurring enzyme that breaks down DNA and seems to help cut down on the viscous mucous which leads to most of the CF complications. Research is ongoing with this drug and it may be available for all CF patients within the next few years.
Other research involves finding a way to introduce a gene which would correct CF. This type of gene therapy may one day be curative for cystic fibrosis as well as many other fatal diseases. The first step was finding that the abnormal gene was on chromosome number 7 in CF patients. This occurred just a few years ago and was a phenomenal research finding on its own. The future of gene research and therapy is extremely exciting. Patients with many hopeless diseases today may one day be offered potential cures with diseases from cystic fibrosis to cancer to many other hundreds and thousands of dread diseases we face in medicine.