zhliu0124 reviewed Breath from Salt by Bijal P. Trivedi
Review of 'Breath from Salt' on 'Goodreads'
5 stars
What does it take to develop a good drug?
It was a great read, much better than what I expected.
After reading this book, this horrible disease deeply saddened me and I feel so sorry for the kids and the families (I literally cried through the chapter when Joey died). The never-giving up families and the fund raisers also are quite moving. I was excited and inspired by all the scientific progresses made through the past 60 years. The history of fighting Cystic Fibrosis (CF) is such an outstanding example that we humans use modern science to conquer diseases.
To conquer CF, it takes a really long time.
- It was until 1938, when Dr. Dorothy Anderen noticed kids died from CF differed from normal pneumonia and found that this is a new type of disease.
- But it was until 15 years later, in 1953, that the first diagnosis method, the sweet test, was invented.
- During 1955 and 1978, over 20 years, the Cystic Fibrosis Foundation was slowly gaining its pace, securing funding and centralizing the information of CF patients. These efforts were the key elements which helped the drug discovery.
- The CF related research only took off after the foundation decided to focus on supporting research in 1981, and it was fruitful. In eight years, scientists found in CF patient lung airways that chloride levels are high in the mucus, which makes the mucus thick and difficult to be cleaned out.
- In 1989, the CF gene was cloned! This was the greatest milestone because it enables prenatal genetic testing, as well as the mechanistic studies which eventually led to the discovery of the drugs.
- Things were continuously getting better since then, from 1989 to 1997, two other drugs, the Pulmozyme, which cuts the DNAs in the mucus in the lung and makes the mucus more fluid, and the inhaled antibiotics TOBI. These drugs greatly reduced the chances of lung infections and improved the life expectancy of CF patients. Though there were some dark periods when the gene therapy failed and patients died during clinical trials.
- In 1998, the drug screening which finally found the panacea for CF started. It was a bold idea for the scientists and a bold decision for the foundation to fully support the endeavors.
- CF patients carry mutated versions of the CF gene, which encodes a chloride channel. This channel is either closed or degraded because of protein misfolding, depending on which types of the mutations the patients are carrying. The idea was to find the chemicals which can open the closed channels, and can help the mutated channels normally fold. No one knew if this strategy would work, and the project has been neglected for a long time. Indeed, it took them 13 years to get the first drug which opens the closed drug. The second drug which helps the folding of the mutated channel, was only approved by the FDA two years ago (2019).
As you can see, to develop good drugs for a disease, it relies on the scientific advances made through the years (for example, here the revolution of molecular biology and a cell system for high-throughput drug discovery), money, a lot of (millions of dollars were infused to the companies and institutions for CF related researches), perseverance of the patients (never give up!), a powerful and well-organized foundation (here the CFF), patient (science is really slow) and luck (the CF is still a lucky one. It happens in most of the patients that the CF proteins are non-functional but still exist, which makes designing drugs to improve their functions possible. In contrast, other diseases are not so lucky. For example, the Fragile X mental retardation, which also caused by the loss of function of a single gene, has no medicine yet. We even haven’t had a good idea what the gene does).
Support science, it is the future!
It was a great read, much better than what I expected.
After reading this book, this horrible disease deeply saddened me and I feel so sorry for the kids and the families (I literally cried through the chapter when Joey died). The never-giving up families and the fund raisers also are quite moving. I was excited and inspired by all the scientific progresses made through the past 60 years. The history of fighting Cystic Fibrosis (CF) is such an outstanding example that we humans use modern science to conquer diseases.
To conquer CF, it takes a really long time.
- It was until 1938, when Dr. Dorothy Anderen noticed kids died from CF differed from normal pneumonia and found that this is a new type of disease.
- But it was until 15 years later, in 1953, that the first diagnosis method, the sweet test, was invented.
- During 1955 and 1978, over 20 years, the Cystic Fibrosis Foundation was slowly gaining its pace, securing funding and centralizing the information of CF patients. These efforts were the key elements which helped the drug discovery.
- The CF related research only took off after the foundation decided to focus on supporting research in 1981, and it was fruitful. In eight years, scientists found in CF patient lung airways that chloride levels are high in the mucus, which makes the mucus thick and difficult to be cleaned out.
- In 1989, the CF gene was cloned! This was the greatest milestone because it enables prenatal genetic testing, as well as the mechanistic studies which eventually led to the discovery of the drugs.
- Things were continuously getting better since then, from 1989 to 1997, two other drugs, the Pulmozyme, which cuts the DNAs in the mucus in the lung and makes the mucus more fluid, and the inhaled antibiotics TOBI. These drugs greatly reduced the chances of lung infections and improved the life expectancy of CF patients. Though there were some dark periods when the gene therapy failed and patients died during clinical trials.
- In 1998, the drug screening which finally found the panacea for CF started. It was a bold idea for the scientists and a bold decision for the foundation to fully support the endeavors.
- CF patients carry mutated versions of the CF gene, which encodes a chloride channel. This channel is either closed or degraded because of protein misfolding, depending on which types of the mutations the patients are carrying. The idea was to find the chemicals which can open the closed channels, and can help the mutated channels normally fold. No one knew if this strategy would work, and the project has been neglected for a long time. Indeed, it took them 13 years to get the first drug which opens the closed drug. The second drug which helps the folding of the mutated channel, was only approved by the FDA two years ago (2019).
As you can see, to develop good drugs for a disease, it relies on the scientific advances made through the years (for example, here the revolution of molecular biology and a cell system for high-throughput drug discovery), money, a lot of (millions of dollars were infused to the companies and institutions for CF related researches), perseverance of the patients (never give up!), a powerful and well-organized foundation (here the CFF), patient (science is really slow) and luck (the CF is still a lucky one. It happens in most of the patients that the CF proteins are non-functional but still exist, which makes designing drugs to improve their functions possible. In contrast, other diseases are not so lucky. For example, the Fragile X mental retardation, which also caused by the loss of function of a single gene, has no medicine yet. We even haven’t had a good idea what the gene does).
Support science, it is the future!