- Findings Show Cystic Fibrosis Patients Cough More Than 600 Times Per Day
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SOUTH PLAINFIELD, N.J., May 21 /PRNewswire/ -- PTC Therapeutics, Inc.
(PTC) today announced new data suggesting that the quantification of cough
frequency may offer a clinically meaningful outcome measure in cystic
fibrosis (CF). Cough is one of the most prominent and burdensome
disease-related symptoms in CF. According to data presented today at the
2008 International Conference of the American Thoracic Society, patients
with CF cough a remarkable 324 to 1,569 times per day, with an average of
643 coughs per day. In comparison, healthy individuals generally cough
fewer than 16 times in an entire day, according to the European Respiratory
Journal (Hsu 1994).
(Logo: http://www.newscom.com/cgi-bin/prnh/20010919/PTCLOGO )
"Cough is one of the major symptomatic manifestations of the underlying
disease process in CF," stated Eitan Kerem, M.D., head of the Department of
Pediatrics and Cystic Fibrosis Center, Hadassah University Hospital.
"Chronic excessive coughing is a burden on CF patients and a source of
anxiety for caregivers and loved ones. Frequent and intense coughing has a
profound effect on the overall quality of life of the patient --
compromising work, school, sleep and social interactions."
CF is among the most common life-threatening genetic disorders
worldwide and affects nearly 70,000 adults and children. Patients with CF
lack the cystic fibrosis transmembrane conductance regulator (CFTR)
protein, a chloride ion channel that maintains proper hydration of
epithelial cells in the lungs, pancreas, and liver. Loss of the CFTR
protein leads to chronic airway inflammation in association with
obstruction and mucopurulent secretions, all of which provoke persistent
coughing. While there are approved palliative treatments for CF, clinical
development efforts would benefit from reliable quantitative endpoints that
are clinically meaningful because they directly measure changes in how a
patient feels and functions.
"The ability to quantify cough represents a significant step forward in
our understanding of one of the most prominent symptoms of CF," added
Preston Campbell, III, M.D., Executive Vice President of Medical Affairs at
the Cystic Fibrosis Foundation. "A significant reduction in cough frequency
would be of real value to patients."
The study was designed to assess cough frequency as a measure of
clinical benefit. Quantitative measurement was achieved using a new
technology, the VivoMetrics(R) LifeShirt(R), which integrates cough signals
from chest wall motion transducers and a throat microphone and stores the
data for computer analysis. FEV1 (Forced Expiratory Volume in the first
second) and FVC (forced vital capacity) -- key measures of lung function --
were assessed by spirometry, and patients completed a symptom survey.
Patients included 19 adults not in CF exacerbation. Although there was
occasional neck pressure due to the throat microphone, compliance was
excellent, with cough data collected for a median of 24 hours. Cough
frequency averaged 643 coughs per day (ranging from 324 to 1,569 coughs per
day) and tended to increase with lower FEV1 and greater age.
"Anecdotal information concerning cough frequency in CF patients led us
to assess cough quantitatively using the LifeShirt(R)," said Langdon
Miller, M.D., Chief Medical Officer of PTC. "The cough frequency data
announced today highlights the potential for cough assessment as an outcome
measure that may be used in the development of new therapies for CF. We
look forward to presenting data on the effect of PTC124 on cough frequency
at the 31st annual European Cystic Fibrosis Society Conference in June, and
to initiating longer-term trials later this year to further evaluate the
clinical efficacy of PTC124 in patients with CF."
About Cystic Fibrosis
Cystic fibrosis (CF) is among the most common life-threatening genetic
disorders worldwide. According to the Cystic Fibrosis Foundation, CF
affects approximately 30,000 adults and children in the United States and,
according to the European Cystic Fibrosis Foundation, it affects a similar
number of patients in Europe. CF occurs in approximately one of every 3,500
live births, with approximately 1,000 new cases diagnosed each year in the
United States. There is a commercially available genetic test to determine
if a patient's CF is caused by a nonsense mutation, and it is estimated
that nonsense mutations are the cause of CF in approximately 10% of
patients in the United States. There is currently no available therapy to
correct defective CFTR production and function. Instead, available
treatments for CF are designed to alleviate the symptoms of the disease.
These treatments include chest physical therapy to clear the thick mucus
from the lungs, antibiotics to treat lung infections and a mucus-thinning
drug designed to reduce the number of lung infections and improve lung
function. In addition, the majority of cystic fibrosis patients take
pancreatic enzyme supplements to assist with food absorption in digestion.
There is a significant unmet medical need for treatments for the underlying
cause of CF. More information regarding CF is available through the Cystic
Fibrosis Foundation (http://www.cff.org).
About PTC124
PTC124 is an orally delivered investigational new drug in Phase 2
clinical development for the treatment of genetic disorders due to nonsense
mutations. Nonsense mutations are single-point alterations in the genetic
code that prematurely stop the translation process, producing a shortened,
non- functional protein. PTC124 has restored production of full-length,
functional proteins in preclinical genetic disease models harboring
nonsense mutations. In Phase 1 clinical trials, PTC124 was generally well
tolerated, achieved target plasma concentrations that have been associated
with activity in preclinical models and did not induce ribosomal read
through of normal stop codons. PTC124 has demonstrated pharmacodynamic
proof of concept in Phase 2a clinical trials in nonsense-mutation-mediated
Duchenne muscular dystrophy (DMD) and cystic fibrosis (CF).
About PTC Therapeutics Inc.
PTC is a biopharmaceutical company focused on the discovery,
development and commercialization of orally administered, proprietary,
small-molecule drugs that target post-transcriptional control processes.
Post-transcriptional control processes regulate the rate and timing of
protein production and are of central importance to proper cellular
function. PTC's internally-discovered pipeline addresses multiple
therapeutic areas, including genetic disorders, oncology and infectious
diseases. In addition, PTC has developed proprietary technologies and
extensive knowledge of post-transcriptional control processes that it
applies in its drug discovery and development activities, including the
Gene Expression Modulation by Small-molecules (GEMS) technology platform,
which has been the basis for collaborations with leading pharmaceutical and
biotechnology companies such as Pfizer, Celgene, CV Therapeutics and
Schering-Plough. For more information, visit the company's website,
http://www.ptcbio.com.
SOURCE PTC Therapeutics, Inc.
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Related links:
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Photo Notes:
NewsCom: http://www.newscom.com/cgi-bin/prnh/20010919/PTCLOGO
CONTACT:
Jane Baj, PTC Therapeutics, Inc.,
+1-908-912-9167, jbaj@ptcbio.com; Sheryl Seapy, Pure
Communications, +1-949-608-0841,
sheryl@purecommunicationsinc.com, for PTC Therapeutics, Inc.
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