There is enormous progress among C.F. researchers in understanding the disease, developing new therapies, and dealing with the disease from different angles. Below, we will detail the latest steps to achieve improved healing and find a cure for the disease.
When cystic fibrosis was first diagnosed in 1938, it was thought that almost all children with the disease would not live longer than the first six months of their lives. By 1950, the average life expectancy of C.F. patients was only five years (i.e., only half of C.F. patients reached the age of 5 years or older). Until 1966 the average life expectancy was eleven years.
Following improved awareness, early diagnosis, and effective treatment methods, there has been a marked improvement in life expectancy and quality of life. Today, more than half of them reach the age of 30 and over. Many patients even reach the age of 40 and 50, and some reach the age of 60 and up. Continuous improvement in early diagnosis, careful treatment, and follow-up prolongs C.F patients’ quality and life expectancy every year.
It is still not possible to predict the chances of a particular patient because the severity of the disease in each person can be completely different in the purpose of the change. However, combining new treatment methods with existing methods will allow C.F. patients to expect a more optimistic future.
There has been enormous progress among C.F. researchers in understanding the disease, developing new therapies, and dealing with the disease from different angles. Below, we will detail the latest steps to achieve improved healing and find a cure for the disease.
Correction of the genetic defect in C.F.
In 1989, C.F. researchers discovered the gene responsible for the disease. They prepared normal gene transcripts in laboratories and attached them to the abnormal cells within a year. The average gene acted and corrected the function of the damaged cells. meaning
The discovery is that curing C.F is no longer just an imaginary idea but is advancing towards realization in reality.
The average gene was inserted into the cells and later into a laboratory animal in two ways
A. As part of DNA within a virus that has been altered and weakened by genetic engineering.
B. Inside a particle with a fatty coating called a liposome. (LIPOSOME).
Success has brightened the face of research! Therapy by gene repair is underway.
About one hundred CF patients (1998) are involved in these trials. The study aims to determine the safety of the process, and subsequently, its effectiveness. Several issues have been identified so far:
Lack of uniformity in the introduction of normal genes into airway cells.
Development of an immune response in the patient following the introduction of the virus that prevents treatment a second time.
In the first stage, the researchers inserted the modified gene into the nasal mucosa cells of the C.F. In some patients, an attempt has been made to insert the repaired gene into the cells lining the bronchi. The results were encouraging but not identical in all cases.
Thus for the first time, a fundamental defect was corrected within the cells of cystic fibrosis patients. At this early stage of the study, the experience is too small to conclude the clinical impact on patients. However, research continues at an increased rate.
Improved treatment methods
The latest news from the clinical front is particularly noteworthy. The first new C.F. drug in 30 years, the Pulmozyme, has been available worldwide since 1994. Inhalation (Pulmozyme DNase) can be done directly into the airway of the C.F. patient, where the drug breaks down the thick mucus into liquid.
Extensive studies conducted at many cystic fibrosis centers have demonstrated the drug’s efficacy, which reduces inflammatory processes and improves lung function in many C.F. patients.
Today, about a dozen other C.F. drugs are being tested in experimental laboratories at hundreds of C.F. centers in the U.S. and worldwide. News on chest physiotherapy as well as contraceptives for health inflammatory processes. Other researchers hope to shed light on ways to improve nutritional status.
Transplants of heart-lung or of both lungs
In recent years there has been the possibility of performing heart-lung transplants, or of both lungs combined or even lung transplantation from living donors in patients with severe lung disease. Although this is a complicated and serious process, the success rate has made it a possible choice for C.F. patients.
C.F is a severe disease but far from incurable.
Early diagnosis, improved healing methods, and close monitoring have improved the prognosis in recent years. Proper care allows most patients to reach adulthood and lead productive and happy lives. It is essential to know that we are learning more and more about the disease almost every day. So there is good reason to be optimistic about the future.