Drug hope for killer lung disease

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Drug hope for killer lung disease

A cheap antibiotic can overcome one of the genetic flaws that can lead to

cystic fibrosis, say experts.

However, it will not help the majority of UK patients diagnosed with the

killer lung condition.

In a small percentage of cases, a key lung cell protein is left

half-completed because a mutated gene halts the production process.

However, the drug gentamycin helps the cell " skip " this gene and finish

making the protein, alleviating the condition.

Pharmaceutical companies are scrambling to make new versions of the drug

which might be able to make an impact in other conditions caused by a similar

fault.

Some cases of muscular dystrophy could be checked, they believe, if their

work is successful.

Cheap and effective

Gentamycin is already used by some cystic fibrosis clinics as an ingredient

in a nebulised inhaler.

It appears to work on conditions where there is a fault called a " premature

stop mutation " on an important gene.

In the case of cystic fibrosis, the CFTR gene is important because without

it, an excess of thick mucus builds up in the lungs, leading to a cycle of

infection and scarring that contributes to long-term damage.

Normally, these stop signals are placed towards the end of a gene, and tell

the cell's protein-making machinery when the protein they are constructing is

complete.

However, in cystic fibrosis, the stop signal has arisen earlier in the gene's

makeup, and the resulting protein is not complete, and cannot perform its

function.

However, a component of gentamicin binds to part of this stop signal, and

allows the machinery to carry on past, making the complete protein.

Nostril cure

A study published in the New England Journal of Medicine last week revealed

how gentamicin applied to the nostrils of people with cystic fibrosis led to

normal CTFR production from those tissues.

New Scientist magazine reports that drug company PTC Therapeutics is trying

to develop a new form of gentamicin which is both more potent and carries fewer

side-effects.

However, a UK cystic fibrosis expert said that relatively few people

worldwide carry " premature stop " gene faults - their disease is caused by other

types

of fault.

Dr Iolo Doull, from the University of Wales College of Medicine, told BBC

News Online: " This research was carried out in Israel, which is unusual because

a

far higher proportion of Jewish CF patients carry these mutations.

" In the UK, it is probably fewer than 10 per cent. "

However, he said that while efforts to produce other forms of gene therapy to

help cystic fibrosis patients were continuing, there were expectations that

drugs would emerge which could tackle other types of gene mutations.

He said: " You might be able to give customised treatments depending on the

genetic makeup of the patient. "

Professor Duncan Geddes, from the Royal Brompton Hospital in London, said

that setting up a clinical trial of gentamicin for cystic fibrosis was

impractical due to the small number of patients involved.

However, he added: " Any new drug coming through for CF is welcome - it boosts

the morale of the CF community. "

Story from BBC NEWS:

http://news.bbc.co.uk/go/pr/fr/-/1/hi/health/3194928.stm

Published: 2003/10/15 23:52:37 GMT

© BBC MMIII

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