The last 10% of cystic fibrosis patients are still waiting for a breakthrough
cystic fibrosis
An inherited disease that causes thick mucus to build up in the lungs.
CFTR
A protein that helps balance salt and water in the body.
tolerability
How well a person’s body can handle a treatment.
What happened
Cystic fibrosis, or CF, is an inherited disease that fills the lungs with thick mucus. It can cause serious infections and breathing problems. About 40,000 people in the United States live with CF.
New medicines have transformed care for about 90% of patients. Vertex Pharmaceuticals, the drug company, makes Trikafta and the newer Alyftrek. These medicines improve the work of CFTR, a protein that helps balance salt and water in the body. That can make lung mucus thinner.
The remaining 10% often have mutations that do not produce working CFTR. The same medicines therefore cannot solve their main problem. Their disease did not disappear when treatment improved for most other patients.
Why the gap remains
Emily Kramer-Golinkoff founded Emily’s Entourage in 2011. The nonprofit supports research for patients who cannot benefit from current drugs. It has raised more than $22 million and funded 51 research projects.
The scientific challenge is not only finding a promising drug. Researchers must also deliver it to the right lung cells. Lungs are built to keep outside material away. Thick mucus in a person with CF creates another barrier. A treatment can look promising in a laboratory and still fail to reach its target in the body.
Recent setbacks
In May, Vertex and Moderna stopped an mRNA treatment trial for this group. The companies cited tolerability problems. In February, Boehringer Ingelheim ended a CF gene-therapy trial. These decisions do not prove that every approach will fail. They do show how difficult it is to make a treatment that the body can accept and the lungs can use.
The setbacks matter because the remaining patients have many different mutations. About 2,000 mutations can cause CF, and many are extremely rare. A single standard medicine may not fit everyone.
What has been confirmed
Research continues across several strategies. Emily’s Entourage has supported gene therapies, small-molecule drugs, and antisense approaches. It also supports phages, natural viruses that kill bacteria, and other medicines for drug-resistant infections. These infection treatments may buy patients time while researchers seek a deeper solution.
Spirovant Sciences has a gene therapy in an early-stage clinical trial. It could work regardless of a patient’s specific mutation. That possibility is important, but early testing cannot show whether the treatment will be safe and effective for the wider group.
What remains unknown
No reported result yet shows that one new treatment works for the whole remaining group. Researchers still need evidence about delivery, safety, biological activity, and lasting benefit. Work on custom-made medicines has also gained attention after the story of Baby KJ helped spur regulatory changes. It is not yet clear how quickly such medicines can reach many patients.
What to watch next
The clearest signals will come from clinical-trial updates. Watch for enrollment, safety findings, proof that a treatment reaches lung cells, and signs that it improves CF-related problems. Funding is another important signal. The 90% success story should not create the mistaken idea that CF has been solved for everyone.
At 41, Kramer-Golinkoff still uses antibiotics, round-the-clock oxygen, diabetes injections, and more than 30 pills. Her experience shows why the final 10% is not a footnote. It is a group still living with the disease while the next breakthrough is being tested.
Source: CNBC report
Why some cystic fibrosis patients still need a new treatment
📰 Full story: The last 10% of cystic fibrosis patients are still waiting for a breakthrough
New drugs help most people with cystic fibrosis. About 10% still need another answer.
cystic fibrosis
An inherited disease that makes thick mucus build up in the lungs.
CFTR
A protein that helps control salt and water in the body.
clinical trial
A study that tests a new treatment in people.
💡 The gist
- Most patients can now use helpful medicines.
- About 10% cannot benefit from those medicines.
- Researchers are testing several different approaches.
Cystic fibrosis is an inherited disease. It makes thick mucus build up in the lungs. That mucus can make breathing difficult. It can also lead to serious infections.
Vertex Pharmaceuticals is a drug company. It makes Trikafta and Alyftrek. These medicines help about 90% of patients. They improve CFTR, a protein that balances salt and water in the body. Better CFTR function can make lung mucus thinner.
The remaining 10% have different mutations. Their bodies may not make working CFTR. The current medicines cannot help enough when that protein is missing. This explains why medical progress has not reached everyone.
Emily Kramer-Golinkoff started Emily’s Entourage in 2011. The nonprofit supports research for these patients. It has raised more than $22 million. It has funded 51 research projects.
Researchers are trying gene therapy and other medicines. One treatment from Spirovant Sciences is in an early clinical trial. It might work across different mutations. However, researchers still need to test its safety and results.
Getting medicine into the lungs is also difficult. The lungs keep many outside materials away. Thick mucus creates an extra barrier. This can stop a treatment from reaching the cells it needs.
Two recent trials show the challenge. A Vertex and Moderna mRNA trial stopped in May. The companies cited tolerability problems. Boehringer Ingelheim ended a gene-therapy trial in February.
Those setbacks do not end the search. Other research targets drug-resistant infections. Phages are natural viruses that kill bacteria. Such treatments may buy time, but they are not the final cure.
Next, watch for trial safety results. Also watch whether treatments reach lung cells. Researchers must show real benefits for patients. The main lesson is simple. Helping 90% does not mean helping everyone.
Some people are still waiting for a medicine that works
📰 Full story: The last 10% of cystic fibrosis patients are still waiting for a breakthrough
New medicines helped many people. But the final 10% still need help.
cystic fibrosis
A sickness that makes sticky mucus fill the lungs.
Emily’s Entourage
A group that helps scientists look for new treatments.
Cystic fibrosis is a sickness that fills lungs with sticky mucus.
Sticky mucus can make breathing very hard.
Vertex Pharmaceuticals, a medicine company, makes Trikafta and Alyftrek.
These medicines help many people.
But they do not help the final 10%.
Emily Kramer-Golinkoff started Emily’s Entourage in 2011.
It is a group that helps scientists find new treatments.
Some tests stopped because people could not handle them.
Another gene treatment is still being tested.
No one knows yet if it works safely.
That is why the final 10% still need a treatment that works.