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Monday, August 24, 2009

Earn $100 in 60 minutes for CF Research

Hey folks,

Market research opportunity YEY!

If you qualify, you will earn $100 for 60 minutes.

And, of course, I'm always candid. I get a referral bonus, yes. So if you would be so kind as to let them know that Amy from the NoExcuses blog sent you, I would be grateful. (I'm looking to pay off my stupid student loans).

From the company:

We need help from Cystic Fibrosis patients and/ or Caregivers of children with CF for our upcoming study on the Management and Treatment of Cystic Fibrosis. Please be assured that this study is for market research purposes ONLY and we are not involved in sales of any kind. Our client (whom we cannot mention) is trying to understand the techniques used to treat CF. Qualified individuals will be paid $100. for their participation in this 60 minute telephone interview. We will of course try to accommodate your busy schedule.

The study is scheduled to take place the week of August 24th so please call as soon as possible.

You can contact us by simply e-mailing lpittman@focuspointeglobal.com with your name and phone number or calling in to 1 800.220.3730. Please refer to the project topic and job #36994CY.

Sunday, August 23, 2009

It's time for YOU to participate

One of the biggest obstacles between us and new CF meds is clinical trial participation (often more of an issue than money!).

There are quite a number of cool studies going on out there, like another Fizzy NAC study , Denufosol which as already shown INCREDIBLE results, Vertex trials which may help G551D patients and those with DF508 , and even breathing in a type of sugar to help clear mucus.

Past studies with every single one of these drugs has shown a powerful impact on CF lung function and/or overall CF health. How could you not want to help get these drugs to the market?

This is EXCITING, FASCINATING stuff. Even just 1 of these drugs has the potential to make a CFer's life much better.

If you want to see what's in your area (and remember, you can participate in a clinical trial outside your main CF center), please go here:


Saturday, August 22, 2009

GERD & Lung Function

Gastric Acid Inhibition for Fat Malabsorption or Gastroesophageal Reflux Disease in Cystic Fibrosis: Longitudinal Effect on Bacterial Colonization and Pulmonary Function.

van der Doef HP, Arets HG, Froeling SP, Westers P, Houwen RH.

Department of Pediatric Gastroenterology (H.v.d.D., S.F., R.H.), Department of Pediatric Pulmonology (H.A.), and Julius Center for Health Sciences and Primary Care (P.W.), University Medical Center Utrecht, Utrecht, the Netherlands.

OBJECTIVES: To investigate bacterial colonization and pulmonary function longitudinally in patients with cystic fibrosis (CF) receiving drugs for gastric acid (GA) inhibition for fat malabsorption or for gastroesophageal reflux disease (GERD). STUDY DESIGN: A retrospective cohort study of 218 pediatric patients with CF was performed. Multilevel modeling was used to perform longitudinal analysis of forced expiratory volume in 1 second (FEV(1)), forced vital capacity (FVC), maximum expiratory flow at 50% of FVC (MEF(50)), and maximal mid-expiratory flow between 25% and 75% of FVC (MMEF(25-75)). Cox regression was used to calculate Pseudomonas aeruginosa- and Staphylococcus aureus-free survival. RESULTS: Patients with CF and GA inhibition had a significantly smaller yearly decline of MEF(50) and MMEF(25-75) compared with control subjects. Other pulmonary function parameters and P aeruginosa or S aureus acquisition or colonization were not different from that of control subjects. GERD was associated with a significantly reduced pulmonary function (FEV(1) and FVC) and an earlier acquisition of P aeruginosa and S aureus. CONCLUSIONS: GA inhibition did not affect pulmonary function or bacterial acquisition and therefore is not contraindicated in patients with CF. GA inhibition might improve pulmonary function with time, because the decline of MEF(50) and MMEF(25-75) was less pronounced. GERD was associated with a reduced pulmonary function and an earlier acquisition of P aeruginosa and S aureus. Therefore the diagnosis and treatment of GERD should be aggressively pursued in patients with CF.

PMID: 19683256 [PubMed - as supplied by publisher]

Monday, August 17, 2009

UM researcher aims to treat CF through nanoemulsion tech

POSTED: 8:00 p.m., Aug. 16, 2009
LAST MODIFIED: 8:20 p.m., Aug. 16, 2009

UM researcher aims to treat CF through nanoemulsion tech

University of Michigan
researcher Dr. James Baker, director of Michigan Nanotechnology Institute for Medicine and Biological Sciences, is working to develop a new use for his nanoemulsion technology — bacteria- and fungi-killing microscopic oil droplets — to treat cystic fibrosis.

The new application has brought additional funding to his company, NanoBio, which has already brought in more than $100 million in funding since 1996.

People with cystic fibrosis suffer from a genetic defect that often leads to severe bacterial infections in the lungs. But because the infections are treated with antibiotics, over time the bacteria can become resistant, leaving patients with no relief.

“Unfortunately there are many patients out there without any type of treatment available, and that is really the most common cause of death among CF patients,” he said.

But in February, his research demonstrated that the nanoemulsion can kill all the antibiotic-resistant bacteria in the lungs. He is currently testing the safety of nanoemulsions in the lungs.

Until now, the application for the nanoemulsions could only be delivered on infected areas on the surface, like cold sores. The oil droplets penetrate the skin and kill the viruses that cause cold sores, as well as bacteria and fungi on skin and mucus membranes.

If proven to be safe for lung tissue, the technology could be delivered through an aerosol inhaler.

“It could have a really important impact,” he said.

Saturday, August 15, 2009

Omega 3 Supplementation and CF

This study is currently recruiting participants.
Verified by Queen Fabiola Children's University Hospital, August 2009
First Received: August 13, 2009 No Changes Posted
Sponsored by: Queen Fabiola Children's University Hospital
Information provided by: Queen Fabiola Children's University Hospital
ClinicalTrials.gov Identifier: NCT00959010

Essential fatty acids (EFA) deficiency has been often reported in patients with cystic fibrosis (CF), particularly in those homozygous for the DF508 mutation. Clinical symptoms of CF may be influenced by correcting EFA deficiency. Nevertheless, the value of EFA supplementation in CF remains controversial. Within this multicentric and international randomized placebo-controlled trial it will be evaluated, according to recommendations of Cochrane analysis, beneficial effects of an oral supplementation with polyunsaturated fatty acids on selected biochemical and functional outcome parameters such as inflammatory biomarkers, incorporation into cell membrane phospholipids, lung function, exercise tolerance, clinical and nutritional status and properties of transepithelial ion transport. The study will be undertaken in a cohort of CF patients aged over 6 years old (60 patients), homozygous for the DF508 mutation and treated by Azithromycine. Supplementation will be performed with a triglyceride source at a daily dose of 60 mg/kg of omega-3 polyunsaturated fatty acids (Omega 3 Premiumâ, Laboratoires Ponroy, France). Before enrolled into the trial and during the study, patients will undergo nutritional assessment by evaluation of total and fat dietary intake and overall calorie intake using a 3-days diet records and a food frequency questionnaire. Plasma and erythrocyte membrane EFA profiles and inflammatory markers will be monitored in baseline conditions, at 3, at 6 and 12 months after starting the treatment. Lung function will be performed at each patient visit and an exercise test will be done before and at the end of the treatment. Properties of ion transport will be searched by sweat testing before and at the end of the treatment.

Condition Intervention Phase
Cystic Fibrosis
Dietary Supplement: omega-3 triglycerides
Dietary Supplement: Placebo
Phase III

Study Type: Interventional
Study Design: Treatment, Randomized, Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Placebo Control, Parallel Assignment, Safety/Efficacy Study
Official Title: Biochemical Effects of a Long-term Supplementation With Omega-3 Polyunsaturated Fatty Acids in Cystic Fibrosis

Resource links provided by NLM:

Further study details as provided by Queen Fabiola Children's University Hospital:

Primary Outcome Measures:
  • LTB4/LTB5 ratio from baseline to the end of treatment assessment. [ Time Frame: Assessment at 3-6-12 months ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • To explore the change in other inflammatory biomarkers such as TNF-alpha, IL-6, IL-8, IL-17 & alpha-1 anti-trypsin. [ Time Frame: Assessments at 3-6 and 12 months ] [ Designated as safety issue: No ]
  • To evaluate the incorporation into cell membrane phospholipids. [ Time Frame: Assessments at 3-6-12 months ] [ Designated as safety issue: No ]
  • To evaluate the effects on the pulmonary function (FEV1) and on the exercise tolerance (VO2 max). [ Time Frame: Assessment at 12 months ] [ Designated as safety issue: No ]
  • To evaluate the effects on the clinical status and the nutritional status. [ Time Frame: Assessments at 3-6-9 and 12 months ] [ Designated as safety issue: Yes ]
  • To investigate the properties of transepithelial ion transport (sweat test). [ Time Frame: Assessments at 12 months ] [ Designated as safety issue: No ]
  • To evaluate the long term overall safety and tolerability of Omega-3 EFA supplementation in CF patients. [ Time Frame: Assessment at 3-6-9 and 12 months ] [ Designated as safety issue: Yes ]

Estimated Enrollment: 60
Study Start Date: October 2008
Estimated Primary Completion Date: October 2010 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Omega 3 Premium: Experimental
capsules containing 300mg of omega-3 triglycerides with 100mg DHA and 150mg EPA
Dietary Supplement: omega-3 triglycerides
capsules containing 300mg of omega-3 triglycerides with 100mg DHA and 150mg EPA, 60mg/kg/day 3 times a day during 12 months.
Placebo: Placebo Comparator
capsules containing middle chain triglycerides
Dietary Supplement: Placebo
capsules containing middle chain triglycerides


Ages Eligible for Study: 6 Years to 60 Years
Genders Eligible for Study: Both
Accepts Healthy Volunteers: No

Inclusion Criteria:

  1. Male or female patient over 6 years of age at visit 1 with stable cystic fibrosis
  2. Documented Homozygous for DeltaF508 mutation
  3. Patient treated with stable dose of Azithromycine since at least 3 months
  4. Able to perform pulmonary function test and swallow capsules
  5. Female patient of childbearing potential must have a negative serum pregnancy test prior to enrolment and must agree to practice effective birth control during the study and 6 weeks thereafter
  6. Signed informed consent obtained from the patient, or in case the patient is minor,from patient's legally acceptable representative (parents or guardians) according to ICH & local regulations. Child assent will be nevertheless obtained

Exclusion Criteria:

  1. Ongoing acute illness including acute upper or lower respiratory infections within 2 weeks before baseline evaluation.
  2. Abnormalities on screening chest x-ray (or CT-scan) suggesting clinically active pulmonary disease other than CF, or new, significant abnormalities such as atelectasis or pleural effusion which may be indicative of clinically active pulmonary involvement secondary to CF.
  3. Any chronic (> 1 week daily) oral or intravenous inflammatory treatment, other than Azithromycine, given to the patient within 3 months before start of study treatment.
  4. Active bleeding or increased risk of bleeding (rate of platelets <>
  5. Patient has significant liver disease, defined as having elevated liver function tests with values 2-fold higher than the upper normal range of the investigational local lab or having abnormal ultrasound such as signs of cirrhosis.
  6. Hypercholesterolemia (>240mg%).
  7. Patient is pregnant or a breast-feeding mother
  8. Patient is participating or has participated in another investigational drug trial or is receiving or has received an investigational drug within the last 28 days before entry into this study.
  9. Patient is unlikely to comply with the visits scheduled in the protocol or enable to follow the study procedure.
Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00959010

Contact: Laurence Hanssens, MD +32/2/477.23.41 laurence.hanssens@huderf.be
Contact: Bernard Wenderickx +32/2/477.32.88 bernard.wenderickx@huderf.be

Hôpital Universitaire Des Enfants Reine Fabiola Recruiting
Brussels, Belgium, 1020
Contact: Daniel Cloquet +32/2/477.32.70 daniel.cloquet@huderf.be
Contact: Bernard Wenderickx +32/2/477.32.88 bernard.wenderickx@huderf.be
Principal Investigator: Laurence Hanssens, MD
Sponsors and Collaborators
Queen Fabiola Children's University Hospital
Principal Investigator: Laurence Hanssens, MD Queen Fabiola Children's University Hospital
More Information
No publications provided

Responsible Party: Hôpital Universitaire Des Enfants Reine Fabiola ( Thierry Schurmans )
Study ID Numbers: HU01/PNE/MUCO1, 2006-004155-38
Study First Received: August 13, 2009
Last Updated: August 13, 2009
ClinicalTrials.gov Identifier: NCT00959010 History of Changes
Health Authority: Belgium: Federal Agency for Medicinal Products and Health Products

Keywords provided by Queen Fabiola Children's University Hospital:
Omega 3
Cystic Fibrosis
Inflammatory markers

Study placed in the following topic categories:
Digestive System Diseases
Genetic Diseases, Inborn
Respiratory Tract Diseases
Cystic Fibrosis
Lung Diseases
Omega 3 Fatty Acid
Infant, Newborn, Diseases
Pancreatic Diseases

Additional relevant MeSH terms:
Pathologic Processes
Digestive System Diseases
Genetic Diseases, Inborn
Respiratory Tract Diseases
Lung Diseases
Infant, Newborn, Diseases
Pancreatic Diseases
Cystic Fibrosis

ClinicalTrials.gov processed this record on August 14, 2009

Friday, August 14, 2009

A great site full of inspiration

As many of you may know, I am a huge fan of cfvoice.com.

Not to be a downder, but I feel that some sites I've come across over the years just don't speak to real life as a CFer. Am I alone in that thinking?

But CF Voice has really resonated with me and I know when I've sent the link to other CFer's and their parents i have received tremendous feedback.

If you go to the 13-18 year old section, MentorU is one of my absolutely favorite sections.


Sunday, August 9, 2009

Airway Surface Liquid Contains Endogenous DNase Activity which Can be Activated by Exogenous Magnesium.

Airway Surface Liquid Contains Endogenous DNase Activity which Can be Activated by Exogenous Magnesium.

Rosenecker J, Naundorf S, Rudolph C.

Department of Pediatrics, University of Munich, Lindwurmstr. 2a, 80337 Munich, Germany. Joseph.Rosenecker@med.uni-muenchen.de.

Introduction: The removal of highly viscous mucus from the airways is an important task in the treatment of chronic lung disease like in cystic fibrosis. The inhalation of recombinant human DNase- I (rhDNase-I) is used to facilitate the removal of tenacious airway secretions in different lung diseases and especially in CF. Little is known about endogenous DNase activity in the airway surface liquid. Therefore, we analysed bronchoalveolar lavage fluid (BAL) and exhaled breath condensate (EBC) for the presence of endogenous DNase activity. -

Methods: The degradation of plasmid DNA by BAL from patients who had diagnostic bronchoscopy and bronchoalveolar lavage was analyzed. In a group of CF patients and healthy control volunteers the exhaled breath condensate was obtained and also analyzed for the ability to degrade plasmid DNA. In addition, the ability of magnesium to activate endogenous DNase activity in BAL and exhaled breath condensate was investigated. -

Results: The analyzed BAL samples degraded plasmid DNA only after preincubation with magnesium. When analyzing the exhaled breath condensate the samples obtained from the healthy volunteers showed no DNase activity even after preincubation with magnesium, whereas in one of the two samples obtained from CF patients we found a DNase activity after preincubation with magnesium. -

Conclusion: Increasing the magnesium concentration in the airway surface liquid by aerosolisation of magnesium solutions or oral magnesium supplements could improve the removal of highly viscous mucus in chronic lung disease by activating endogenous DNase activity.

PMID: 19661013 [PubMed - in process]

Modifier gene study of meconium ileus in cystic fibrosis

Modifier gene study of meconium ileus in cystic fibrosis: statistical considerations and gene mapping results.

Dorfman R, Li W, Sun L, Lin F, Wang Y, Sandford A, Paré PD, McKay K, Kayserova H, Piskackova T, Macek M, Czerska K, Sands D, Tiddens H, Margarit S, Repetto G, Sontag MK, Accurso FJ, Blackman S, Cutting GR, Tsui LC, Corey M, Durie P, Zielenski J, Strug LJ.

Program in Genetics and Genome Biology, Hospital for Sick Children, Toronto, ON, Canada, ruslan.dorfman@gmail.com.

Cystic fibrosis (CF) is a monogenic disease due to mutations in the CFTR gene. Yet, variability in CF disease presentation is presumed to be affected by modifier genes, such as those recently demonstrated for the pulmonary aspect.

Here, we conduct a modifier gene study for meconium ileus (MI), an intestinal obstruction that occurs in 16-20% of CF newborns, providing linkage and association results from large family and case-control samples. Linkage analysis of modifier traits is different than linkage analysis of primary traits on which a sample was ascertained.

Here, we articulate a source of confounding unique to modifier gene studies and provide an example of how one might overcome the confounding in the context of linkage studies.

Our linkage analysis provided evidence of a MI locus on chromosome 12p13.3, which was segregating in up to 80% of MI families with at least one affected offspring (HLOD = 2.9). Fine mapping of the 12p13.3 region in a large case-control sample of pancreatic insufficient Canadian CF patients with and without MI pointed to the involvement of ADIPOR2 in MI (p = 0.002). T

his marker was substantially out of Hardy-Weinberg equilibrium in the cases only, and provided evidence of a cohort effect. The association with rs9300298 in the ADIPOR2 gene at the 12p13.3 locus was replicated in an independent sample of CF families.

A protective locus, using the phenotype of no-MI, mapped to 4q13.3 (HLOD = 3.19), with substantial heterogeneity. A candidate gene in the region, SLC4A4, provided preliminary evidence of association (p = 0.002), warranting further follow-up studies. Our linkage approach was used to direct our fine-mapping studies, which uncovered two potential modifier genes worthy of follow-up.

PMID: 19662435 [PubMed - as supplied by publisher]

Tuesday, August 4, 2009

Cystic fibrosis: defining a disease under-diagnosed in Pakistan

Trop Med Int Health. 2009 May;14(5):542-5.

Cystic fibrosis: defining a disease under-diagnosed in Pakistan.Shah U, Frossard P, Moatter T.Harvard Medical School Dubai Center, Dubai, UAE.Objective

Cystic fibrosis is frequently missed in the Pakistani population due to lack of appropriate diagnostic tools. Thus our aim was to define unknown disease-causing mutations to help create suitable diagnostic tests and improve understanding of what appears to be an aggressive and under-diagnosed disease in this population.

Methods Patients with elevated sweat chloride values and clinically suspected CF were recruited from Aga Khan University, Pakistan. Mutations DF508, S549R, S549N, Y569D, 296 + 12(T>C), G553X, G551D and G551X were screened for by allele specific polymerase chain reactions. CFTR exons 10, 11 and 12 were sequenced by direct DNA sequencing.

Results Of 150 patients tested by PCR, 26 (17.3%) were positive for DeltaF508. One patient was a F508/S549N compound heterozygote. Eighty-three of 87 patients sequenced for mutations in exon 10 were normal; 42/43 for exon 11 and 29 for exon 12 were normal.

Conclusion This first step in defining mutations involved in Pakistani CF suggests that DeltaF508 is uncommon and S549 was the only additional mutation identified in CFTR exons 10, 11 and 12. Identification of the remaining mutations and their frequency is required to design appropriate tests and improve understanding and management of the disease.

PMID: 19645745 [PubMed - in process]

Saturday, August 1, 2009

Long-term effects of a partially supervised conditioning program in cystic fibrosis

Long-term effects of a partially supervised conditioning program in cystic fibrosis.

Hebestreit H, Kieser S, Junge S, Ballmann M, Hebestreit A, Schindler C, Schenk T, Posselt HG, Kriemler S.

Julius-Maximilians-Universität Würzburg, Germany.

Little is known about the long-term persistence of positive effects induced by a physical conditioning program. Therefore, this study determined the effects of a 6-months conditioning program on peak oxygen uptake (primary outcome), and other markers of fitness, physical activity, anthropometry, lung function, and quality of life (secondary outcomes) 18 and 24 months after the program was initiated.

Patients with CF aged 12-40 years were randomly assigned to an intervention (n=23) and a control (n=15) group. The intervention group consented to add three hours of sports per week for at least six months to their previous activities. Controls were asked to maintain their level of activity for 12 months. Patients were seen at baseline and after 3, 6, 12, 18, and 24 months.

There was no significant difference between groups at baseline.

The intervention induced positive effects on peak oxygen uptake (difference in changes from baseline to the 18- and 24-months assessments between groups: +3.72+/-1.23 ml.min(-1).kg(-1), p<0.01),>

A home-based partially supervised physical conditioning program can improve physical fitness, lung function and perceived health long after the intervention has ended.

PMID: 19643946 [PubMed - as supplied by publisher]


Infection control in cystic fibrosis: barriers to implementation and ideas for improvement

Infection control in cystic fibrosis: barriers to implementation and ideas for improvement.

Saiman L, Garber E.

aDivision of Infectious Diseases, Department of Pediatrics, Columbia University and Department of Epidemiology, NewYork-Presbyterian Hospital, USA bDivision of Infectious Diseases, Department of Pediatrics, Columbia University, New York, New York, USA.

PURPOSE OF REVIEW: This review will focus on recent research documenting baseline adherence to infection control recommendations and barriers to their implementation as experienced by multidisciplinary cystic fibrosis (CF) care providers. In addition, controversies regarding optimal infection control will be discussed. Finally, suggestions to improve infection control in CF will be proposed.

RECENT FINDINGS: Compliance with recent guidelines was assessed for clinical microbiology laboratories and for infection control policies at CF care centers in the United States. Unlike earlier reports, the vast majority of laboratories used selective media for Burkholderia cepacia complex and identified all species of nonlactose fermenting Gram-negative bacilli. Fewer used selective media for Staphylococcus aureus or used agar-based susceptibility testing assays for Pseudomonas aeruginosa. Only 103 (65%) of 158 CF care centers provided written infection control policies for review and these were more likely to address inpatient than outpatient settings. Surveys of healthcare professionals showed that access to a copy of the CF infection control guidelines reduced barriers to adherence to selected infection control practices.

SUMMARY: These data suggest that access to national infection control guidelines and written local policies are critically important to improving infection control for CF.

PMID: 19644375 [PubMed - as supplied by publisher]