Cysteamine and CF

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Mallika

New member
From the day I read about the research by Dundee university, I could not resist to look into this
Cysteamine. And very interestingly there are about half a dozen research papers out about Cysteamine and CF.


From the research papers I read, Cysteamine seems to be a mucolytic, something which melts the biofilm and acts as an antibiotic.. The so called Lynovex from Novabiotics is nothing again but the Cysteamine. They gave it a trade name called Lynovex(happy to be corrected). Suddendly most of the research off late is converging towards cysteamine.


Another point to note is that while Novabiotics are planing their trials on Cysteamine(Lynovex) for all mutations, the others seem to be targeting only DF508 mutation. confusing!




Most of them giving Cysteamine credit for restoring the Autophagy in CF lungs which is otherwise impaired. They say it works better than the vertex combo drug.
A patent(http://www.google.com/patents/EP2664326A1?cl=en) filed also stated that there are other agents that do the exact work of cysteamine which are lipoic acid, tiopronin, acetylcysteine, carboxymethylcystein, erdosteine, moguisteine, mesna and glutathione (GSH), as well as the ellagic acid, caffeic acid, cumaric acid, gallic acid, resveratrol and carotenoids such as luthein, astaxhantin and zheaxhantin .


Half of what the paper says went above me. Would need some help understanding it.


Since cysteamine(cystagon capsules given orally) is an already available drug(for treating cystinosis) and supposed to be safe(for cfers not sure).. Can I use it off label with the help of my doctor (ofcourse start it with low doses) Can some one tell me more about this cysteamine. Please please help.
 
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ladybird

New member
Hi Malika

CF Roundtable just published a blog on this at

http://www.cfroundtable.com/2014/11...drug-treatment-may-be-potential-game-changer/

Posted on November 22, 2014 by Andrea
Scientists from the University of Dundee have, as part of an international collaboration, made a potential breakthrough in the treatment of Cystic Fibrosis (CF).
The study of ten patients found that a combination of two drugs would offer hope to patients suffering from the debilitating and life-shortening inherited disease.
The research in Dundee has been supported by the Wellcome Trust since 1991, and initially by the UK Cystic Fibrosis Trust.
The candidate therapy involves an already licensed drug, Cysteamine, which is given alongside a second drug, epigallocatechin gallate (EGCG).
Researchers found that the two drugs given together reduced inflammation in nine out of ten of the patients’ airways and also dramatically reduced the levels of salt in their sweat, a characteristic feature of CF that can leave a baby in therapy for life.
The researchers are now hoping to set up a large-scale trial to prove the drug combination’s effectiveness across an international CF population, which could lead to a completely new approach to therapy for the commonest form of CF.
Dr Anil Mehta, a clinical reader at Dundee’s Medical Research Institute, has been working on CF for more than 20 years and described the findings as an extremely encouraging first step.
He said: “The results suggest that it might be possible to arrest the disease. Obviously, we are still at an early stage but if these results are replicated in a placebo controlled clinical trial, then I believe it could be a potential game-changer. However, we will need more support to make this happen.
“These drugs are already licensed, and being off-patent means the cost of developing a drug therapy should not be prohibitive.”
Cystic Fibrosis is the most common inherited disease in the UK, affecting more than 10,000 people.
As a result of screening in Scotland since 2003, it is known that one child in every 2345 is born with CF.
Dr Mehta’s colleagues at the Universities of Milan, Naples and Paris explored the possibility of using Cysteamine, used for decades to treat a rare children’s disease. They found that this single drug proved very effective in reversing the inflammatory aspects of CF.
He said: “I would like to thank all those who have volunteered in Dundee to donate their airway cells since 1991, they are the true, selfless folk who made this possible. The work shows the value of international cooperation in attempts to beat this very nasty disease.”
A spokesman for the Cystic Fibrosis Trust, which funded the research, said: “This is a very small sample size and talk of a breakthrough towards cure is premature.
“A much larger scale trial is now required before this can be declared to be an effective therapy for cystic fibrosis.
“The Cystic Fibrosis Trust funds a range of research and would welcome a funding application from Dr Mehta through any of our existing funding channels.”

This entry was posted in CF and Medications, CF and Optimism, clinical trials, new medical discoveries, Treatments for CF and tagged cf, cystic fibrosis, medical breakthroughs, medication. Bookmark the permalink.
 
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