at the spectre of cold bureau- |
to the surface but the chan- |
which was approved about |
Although the name ‘ cystic |
Even in the 1930s , when |
|
cratic hearts . |
nel not working effectively |
seven years ago now , and more |
fibrosis ’ was not coined until |
scientists were getting a bet- |
|
One exception was The |
or consistently — known as |
recently what has really set the |
the 1930s , where it was used to |
ter idea of what was happen- |
|
Guardian . |
“ gating mutations ”. |
world alight is Trikafta .” |
capture the association of exo- |
ing , life expectancy was barely |
|
|
“ Parents have been on a
roller-coaster ride in the last few years ,” it wrote in an
|
The first-generation CFTR modulators successfully addressed the gating mutations , |
He adds : “ Trikafta has in effect got three different types of CFTR modulators , |
crine pancreatic insufficiency and chronic lung disease , references to the doomed fate of |
more than five years . It has risen dramatically since then — partly because of antibiotic |
|
editorial . |
but Trikafta managed to |
which encompass two of the |
young babies who tasted salty |
treatment , airway clearance |
|
“ They have been taken to |
address the specific deficien- |
previous modulators , as well |
when kissed — a physical hall- |
techniques , and the process |
|
the giddy heights of hope by |
cies caused by delta F508 — |
as another modulator .” |
mark of the disease — go as far |
of increasing enzyme doses to |
|
the invention of drugs that for the first time work on the underlying causes of the illness |
where the problem lies with misfolding of the protein , preventing it from reaching |
proper folding of the protein to get it up to the surface ,” Profes- |
Increased longevity ?
Cystic fibrosis remains rel-
|
back as the Middle Ages . As the European medieval folklore goes : “ Woe to the child |
allow for a high-fat diet . The average life span for a person with cystic fibrosis is |
where previous treatment just |
the cell surface . |
sor Selvadurai explains . |
atively rare — about one in |
who tastes salty from a kiss on |
now about 47 years . |
alleviated symptoms . “ And they have been plunged to the depths when those |
“ What these new generations do is to facilitate the |
“ There was a drug called Symdeko [ tezacaftor / ivacaftor ] |
2500 babies are born with the condition . |
the brow , for he is cursed and soon will die .” |
But that median age does not include the impact of the |
new drugs have been denied |
|||||
them because of their cost .” |
|||||
But it added : “ It would be |
|||||
easy to paint NICE as the villain |
|||||
of the piece . But it is doing |
|||||
the job it was set up to do . |
|||||
There is a good reason why it |
|||||
can ’ t green light [ Trikafta ]. |
|||||
The price is astronomical . |
|||||
“ If the NHS chose to pay |
|||||
the price for these drugs , |
|||||
and had no extra cash , what |
|||||
would it have to cut ? How |
|||||
many hip replacements ? How |
|||||
much cancer care ?” |
|||||
|
Billions in sales
Vertex has reported more than
|
|||||
$ 31 billion in sales since it was |
|||||
approved by the US Food and |
|||||
Drug Administration in 2019 . |
|||||
It has always defended |
|||||
itself saying that novel drug |
|||||
development is not a cheap , |
|||||
risk-free business when outcomes |
|||||
are not guaranteed . |
|||||
Vertex told the BBC last |
|||||
year : “ The price of our medicines |
|||||
reflects their clinical |
|||||
value and benefits to |
|||||
patients , caregivers and |
|||||
healthcare systems .” |
|||||
It told AusDoc : “ The protection |
|||||
that patents provide |
|||||
enables us to continue |
|||||
to invest in our extensive |
|||||
research and development |
|||||
program into cystic fibrosis … |
|||||
We invest more than 70 % of |
|||||
our operating expenses into |
|||||
research and development , |
|||||
which is far higher than the |
|||||
industry average .” |
|||||
But what is the wonder of |
|||||
this drug ? How does it work ? |
|||||
It starts with the CFTR protein |
|||||
, one of a group of proteins |
|||||
that function as cellular transmembrane |
|||||
channels . |
|||||
One of its roles is the regulation |
|||||
of the movement of salt |
|||||
and water across cells . |
|||||
In cystic fibrosis , however , |
|||||
mutations of the CFTR gene |
|||||
mean this protein does not |
|||||
work effectively . |
|||||
Scientists have now identified |
|||||
2000 mutations , but the |
|||||
most common is known as |
|||||
delta F508 . |
|||||
Professor Selvadurai says |
|||||
Trikafta functions as a modulator |
|||||
and is one of a class of |
|||||
drugs engineered to fix a number |
|||||
of problems resulting from |
|||||
some of these mutations . |
|||||
For example , one problem |
|||||
could be insufficient protein |
|||||
getting to the surface of the |
|||||
cell , or the protein getting |