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gene therapy to reverse bronchiectasis?
Hi community,
Early this year the FDA granted the human clinical trial of ER-100. ER-100 is an investigational gene therapy designed to reverse vision loss by restoring aged and damaged ocular cells to a younger, healthier state.
We bronchiectasis patients should push this idea for reversing bronchiectasis. To me the damage to our lungs is similar to the damage of vision caused by glaucoma. But glaucoma is a well-known disease, while bronchiectasis is not. How can we have our voice heard and push the research going towards this direction sooner? Most people who have bronchiectasis are older, so we may not be able to see this happen; who knows? But the young people certainly have the chance.
I raise the idea here because I truly believe that gene therapy can restore our damaged lungs. So what do you think and what should we do?
Ling
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HugInterested in more discussions like this? Go to the MAC & Bronchiectasis Support Group.
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Once the lung is damaged it cannot be repaired. Gene therapy however can be used to alter the underlying cause and can slow down or halt the progression of the disease. The damaged part of the lungs is essentially non functional. You cannot bring it back from the dead so to speak.
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2 Reactions@sandpiper49 Yes irreversible,the airways are widened,but early phase 1 trail results in humans is promising using there own lungs cells (P63 basal stem cells),
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2 Reactions@sandpiper49, why couldn't it be repaired?? Once the senescent cells are removed, why couldn't there be some regeneration process? Just becasuse it is not currently done doesn't mean it will never be possible. I firmly believe we'll get there.
best wishes of great health to all of us!
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1 Reaction@ling @gigiv @brody89 @sandpiper49 and all who have an interest in this subject (me too)
Let's talk about emerging therapies and research, @ling you said "We bronchiectasis patients should push this idea for reversing bronchiectasis. " When we are immersed in living with a condition or disease, and reading about it every day on Mayo Connect and other forums, it seems hard to believe, but Bronchiectasis is still considered rare compared to many other diseases, and NTM is very rare. Yet there is a lot of research ongoing on these therapies for bronchiectasis and a host of other diseases. You can use Google Scholar (scholar.google.com) to read about it.
Why not more? High-tech, targeted therapies cost BILLIONS of dollars to develop, and each one is disease specific, sometimes even disease-variant or individual patient specific. What does that mean for us? Diseases that affect more people or that are rapidly fatal will naturally be at the top of the list. So bronchiectasis (about 1/2 to 1 million) will not get the same attention as COPD (15M) or Asthma (28M), and NTM (about 100,000) is less researched than MRSA (2.5 million).
But the good news is that as each targeted therapy is perfected, other scientists can use that knowledge to streamline their research and development of treatments for other conditions.
What can we do? We can encourage our elected officials to fully fund scientific and medical research. If we have the financial ability, we can contribute to specific research efforts by qualified organizations.
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4 Reactions@sueinmn, Thanks Sue for joining in the conversation. I think the reason they started with glaucoma might be: the disease is so specificand it is easier to target. Bronchiectasis is the same way, it is narrow and specific. If it is as common as glaucoma disease, they might be interested in investigating it. I even have a naive idea to write to Dr. David Sinclare's team, but of course if we do something as a community it is more powerful. Dr. Sinclair mentioned once the glaucoma drug is on the market, they will try to target the heart, kidney and liver. Lung was not even mentioned, let alone bronchiectasis. Not sure if the lungs could reverse to its healthy and younger state, does that mean bronchiectasis will be healed from that?
Ling
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2 Reactions@ling, this might be of interest to you: https://newsnetwork.mayoclinic.org/discussion/new-discovery-may-unlock-regenerative-therapies-for-lung-disease/
although you probably already read it. Anyway, it is quite hopeful for our future.
@sandpiper49 and @brody89, the above link might broaden your horizon.
We will get there, yes, we will.
Best wishes of great health to all of us!
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1 Reaction@gigiv It isn't just that the airways are widened. They lose their elasticity which means that their elastic recoil is diminished and the airways become floppy. The pathway through which air travels is then grossly decreased. Trying to cough up mucus causes the floppiness to further block the airway. Some airways become completely blocked with mucus and without oxygen they become stagnant and non functional. Removing the damage, as you suggest, can mean removing large branch-like areas of the lung. It isn't just a few cells. It isn't as simple as removing the damage and replacing it with healthy cells.
@gigiv , thanks for the link. The future looks promising. We just don't know when it happens to us.
Like Sue mentioned, once there is a breakthrough, the rest comes faster ....
Ling
@sandpiper49 , well some senolytics are senomorphic, those molecules are quite powerful indeed. There is plenty of hope.
Best wishes of great health to all of us!
@sueinmn It’s great to see that despite bronchiectasis being relatively unknown, there appears to be more interest in the lung disease in the last 5-10 years.
If cilia (tiny hair like structures) in the small airways could somehow be repaired or regenerated, that would be a game changer for many. We can hope.