A dad-of-two is taking on a marathon challenge to raise money for charity.

Dominic Rees, of St Margaret’s Road, Pinkneys Green, has committed to run three miles, every day, for a year.

The IT services worker started his challenge on September 4 and will also run the London Marathon and cap the year off by running the Maidenhead Half Marathon.

Dominic is hoing to raise £3,000 for the Cystic Fibrosis Trust, which is close to the family’s heart as it affects their 15-month-old goddaughter and niece, Chloe.

He said: “It has got past the point where my joints ache, it’s more of a mental challenge now.

“I’ve had to get myself going to run over Christmas and New Year, but most of all running with a heavy cold, which was an absolute killer.

“Combining this with traditional marathon training is hard work, as I no longer have any rest days.

“I’m still required to complete long runs every Sunday, which makes life interesting on Monday.”

Dominic has racked up more than 600 miles so far and still has more than 200 days to go.

Visit uk.virginmoneygiving.com/DomRees to donate.

http://www.maidenhead-advertiser.co.uk/News/Areas/Maidenhead/Running-three-miles-every-day-for-a-year-in-aid-of-Cystic-Fibrosis-19022013.htm

Dr Judy Bradley has been appointed Professor of Physiotherapy at the University of Ulster.

The academic from Desertmartin has focused her research work in the areas of respiratory conditions with a special interest in cystic fibrosis, bronchiectasis, outcome measurement and rehabilitation.

Professor Bradley said: “I am delighted to receive this Professorship. I plan to continue my research and I am focused on improving the quality of life and patient care of people with respiratory conditions.”

The lecturer was presented the Award of Distinguished Research Fellowship by the University in 2004 in recognition of her contribution to research in the area of respiratory physiotherapy. She was subsequently promoted to a Reader at the University of Ulster/Belfast Health and Social Care Trust in 2005.

She has contributed nationally and internationally to guidelines and consensus statements focused on providing recommendations for care.

Professor Bradley took up her post of Lecturer Practitioner – a joint post by the University of Ulster and Belfast Health and Social Care Trust – in September 1998. A year later she obtained her PhD.

She is an active member of several national Research Advisory Committees including the Cystic Fibrosis Trust and was appointed Chair of European Cystic Fibrosis Training Committee of the European Cystic Fibrosis Society in 2012.

Professor Bradley plays an integral part of a Respiratory research team at BHSCT, which put forward an application to the HSC Public Health Agency Research and Development division to form a respiratory network.

In 2007, Professor Bradley accepted the role of co-lead of the Northern Ireland Clinical Research Network (Respiratory Health). This role involves managing a very successful respiratory clinical trial research portfolio, which includes a large number of both pharmacological and non-pharmacological clinical trials in cystic fibrosis, bronchiectasis and chronic obstructive pulmonary disease and asthma.

She provides leadership and direction to a multidisciplinary research team, which comprises of physiotherapists, nurses and a clinical physiologist a number of which have been trained to PhD level at the University of Ulster.

Professor Bradley has published widely in her areas of expertise and has over 50 publications to date, many of which have been published in high impact respiratory journals. She has obtained funding from a variety of prestigious external agencies/pharmaceutical companies to undertake research and this research has had an important impact on patient care and service delivery.

In the University, Professor Bradley is a senior member of the Centre for Health and Rehabilitation Technologies (CHaRT) and as such is working hard to increase the capacity of this research group. She is also involved in undergraduate teaching and in planning post-graduate modules.

http://news.ulster.ac.uk/releases/2013/6803.html

A new inhalable drug therapy developed by an Austin startup, Savara Inc., could improve the lives of thousands of cystic fibrosis patients. If it wins Food and Drug Administration approval, the antibiotic powder called AeroVanc might help an estimated one-fourth of those who have the incurable disease, which causes progressive lung damage. They have few options to rid their lungs of a drug-resistant bacterium.

Cystic fibrosis experts say that AeroVanc has promise because it is designed to take an antibiotic already shown to fight methicillin-resistant staphylococcus aureus (MRSA) and deliver it to the lungs more safely and effectively than other methods. AeoroVanc is set to begin its Phase II drug trial this year.

“If this medication works, it’s not going to be a complete game-changer, but it’s going to make a significant difference in the patients who have MRSA,” said Dr. Bennie McWilliams, an Austin pediatric pulmonologist and director of the pediatric cystic fibrosis program at Dell Children’s Medical Center of Texas. “That’s a huge area that we have an unmet need.”

Cystic fibrosis is a genetic disease that causes the body’s mucus membranes to produce unusually thick, sticky mucus that clogs the lungs, leading to chronic infections and severe lung damage, sometimes warranting transplants. The disease also affects digestive organs and interferes with absorption of nutrients.

Improved treatments in the last 30 years have lengthened the lifespan for many cystic fibrosis patients, though respiratory failure remains the most common cause of death. The condition afflicts an estimated 27,000 people in the United States and nearly 1,700 in Texas, according to the Cystic Fibrosis Foundation.

“Way back when I was in training, you were lucky to get out of your teenage years,” McWilliams said of the patients he treated in the 1980s. “Now the median survival is 37 years, and actually it’s getting to the point where it’s going to be a chronic disease.” The oldest patient in a database kept by the Cystic Fibrosis Foundation is 81, he said.

Patients are living longer partly due to improved treatment for lung infections. But those gains are diminished for the thousands affected by MRSA, which does not respond to commonly prescribed antibiotics. One recent study provides evidence that cystic fibrosis patients whose respiratory tracts contain the drug-resistant bacteria tend to die sooner than other patients. Patients with chronic MRSA infections were at a higher risk than those with short-term infections.

Despite MRSA’s prevalence among people with cystic fibrosis, little research specifically addresses it. Of 140 drug trials and studies the Cystic Fibrosis Foundation is tracking, only three involve MRSA infections.

Dr. Chris Oermann, medical director for the Respiratory Care Department at Texas Children’s Hospital in Houston, said the inhaled drug would target lung tissue more effectively than would an antibiotic taken by other methods and potentially be less toxic than those in use. The inhalable drug would require smaller doses while getting much higher concentrations of the drug into the infected airways.

Inhalable antibiotics that treat a more common bacteria called pseudomonas aeruginosa work very well, said Oermann, who also directs the Cystic Fibrosis Care Center at Texas Children’s.

“If we assume that staph behaves similarly to pseudomonas, then it’s a pretty compelling case to be made,” he said.

Jose Omar Jaime-Martinez, an 18-year-old patient, spends 45 minutes twice a day consuming an array of medications to help him breathe, digest food, manage allergies and fight off infection. A vibrating vest massages his lungs and helps expel mucus; an intravenous tube provides nutrition at night. Treatments including drugs to kill pseudomonas may keep him out of the hospital, except for the two-week “tune-ups” he undergoes once or twice per year to receive intravenous antibiotics and “knock down” the bacteria level in his lungs.

“Having CF is tough and challenging, and there’s a lot of steps through life that there are to manage having CF, but at the end I guess it’s just knowing how to live with it and how to handle it,” Martinez said, who is a senior at Hendrickson High School in Pflugerville, an Austin suburb. Martinez is fortunate that relatively safe and effective treatments for pseudomonas are available. That’s not the case with MRSA.

AeroVanc is a form of the antibiotic vancomycin hydrochloride, which is already used to treat MRSA. But it cannot be absorbed when taken by mouth, and intravenous delivery has been associated with such side effects as hearing loss and kidney damage.

With few good options to treat CF patients with MRSA infections, many practitioners have turned to Linezolid, which is potentially more effective than intravenous vancomycin but is costly and can have serious side effects, such as vision loss, said Elizabeth Hand, a pediatric infectious disease pharmacist at the Children’s Hospital of San Antonio.

“Having a drug that you can directly deliver to the lungs and overcome that poor lung penetration that everyone is concerned with could potentially be of benefit,” she said. While she did not see AeroVanc as a revolutionary breakthrough, she said, “I think it’s always, the more tools we have in our arsenal, the better it is.” Long Road to Approval

Savara, founded in 2007, specializes in pulmonary drug development. It began working on AeroVanc in 2009, said its chief executive, Rob Neville. Making the drug into an inhalable powder involved overcoming a lot of technical challenges, he said. Chief among these were making the medicine particles just the right size to penetrate deep into lung tissue and producing a product that did not require refrigeration.

“We are not here trying to make as much money as possible,” Neville said about Savara. “We’re here trying to make a difference in the lives of these patients, and the money will follow,” he said.

Neville said that AeroVanc could be on the market within five years but must clear a number of hurdles. Phase I trials conducted in Australia last year, involving both healthy subjects and those with cystic fibrosis, confirmed the drug’s safety. Next, AeroVanc will be given to people who have both cystic fibrosis and chronic MRSA lung infections. Medical facilities in Austin, Houston and Tyler are being considered to participate in the trial. Subsequent trials will depends on the results from this phase.

The FDA granted AeroVanc orphan drug status last year, a designation that gives certain benefits, such as a period of market exclusivity, to companies that develop drugs to fight rare diseases. But it’s no guarantee of approval. Since 1983, the FDA has granted 2,711 designations but approved just 410 orphan drugs for market, according to Sandy Walsh, press officer at the FDA.

And the drug development process is expensive, costing from $60 million to $100 million from drug start to finish, Neville said.

He said Savara has raised roughly $16 million in investments and grants, enough to get the company through the “valley of death.” That’s the period between initial discovery of  a drug or research on it and the point at which there’s enough evidence of potential success that a company can attract venture capital. Many startups don’t make it through that stage.

Current funding, which includes nearly $2 million from the Texas Emerging Technology Fund, will carry Savara through the next phase of testing, Neville said. If it’s successful, the company could find a buyer or new investors.

To win FDA approval, trials must show that AeroVanc makes a significant impact in at least one of three areas for patients, on top of eliminating the stubborn bacteria. “We’ve got to show, hopefully, that not only do we kill the bug – or reduce what’s called the CFUs, the colony forming units … but not only that, that they either feel better, function better, or survive better, “ he said.

http://reportingtexas.com/new-austin-based-inhalable-drug-could-bring-relief-to-cystic-fibrosis-patients/

The main video on the new worldwide cf day website we will be launching in a few days, thanks so much Ana and Isa!

Kalydeco wordt vanaf januari 2013 in Nederland als eerste in Europa vergoed voor mensen met Cystic Fibrosis en een G551D-mutatie. Kalydeco is het eerste middel dat daadwerkelijk de oorzaak van CF aanpakt. In Nederland is in de Nederlandse CF-registratie 1 patiënt met de G551D-mutatie bekend.

Het registratiedossier is volgens een versnelde procedure behandeld. De NCFS heeft hiervoor intensief samengewerkt met de behandelend artsen en Vertex.

Onderzoek in 2013

Eind 2012 werd al bekend dat dit jaar gestart wordt met een groot fase-3-onderzoek van Vertex-809 bij mensen met CF en een deltaF508-mutatie. Ook Nederlandse mensen met CF kunnen hieraan meedoen als zij 2 deltaF508-mutaties hebben en aan een aantal andere voorwaarden voldoen. (lees meer hierover in het artikel ‘Deelname Nederlandse CF-patiënten aan Vertex-809-onderzoek’).

In Frankrijk en België wordt in 2013 onderzoek gedaan naar het effect van Kalydeco bij mensen met CF en klasse-3-mutaties. Hieronder valt ook de S1251N-mutatie, die bij 33 mensen met CF in Nederland voorkomt. De resultaten van dit onderzoek worden begin 2014 verwacht.

De Europese Medicines Agency (EMA) gaf in juni 2012 een positief advies af voor de registratie van ®Kalydeco (stofnaam Ivacaftor) voor de behandeling van mensen met Cystic Fibrosis en een G551D-mutatie. Tijdens het onderzoek was het medicijn bekend onder de naam Vertex-770.

http://www.cfonderzoek.nl/news/show_article/29

People with cystic fibrosis (CF) will be able to access the latest research findings about their condition, volunteer for clinical trials and influence the direction of future scientific studies through a new website being launched later this week.

CF Unite.org is the brainchild of academics at The University of Nottingham who wanted to find a way of bringing patients together to discuss scientific and medical breakthroughs without risking the spread of infections that can be dangerous to those living with the condition.

Dr Matthew Hurley, leading the CF Unite project in the University’s School of Clinical Sciences, said: “There are a number of charities and groups that support CF patients with their condition but until now there has been nowhere that they can go to directly engage with the scientists and researchers who are studying the disease.

“Equally, patients with CF have a wealth of untapped experience because they live with the condition every day. This unique knowledge could be extremely useful in designing new clinical trials and in guiding experts on the type of research that is going to have the greatest impact.”

Life-threatening disease

CF is the one of the UK’s commonest life-threatening inherited diseases. It is caused by a faulty gene that controls the movement of salt and water in and out of the cells in the body, which leads to the internal organs, especially the lungs and digestive system, becoming clogged with thick sticky mucus.

People with CF are affected by bacteria which grow in the lungs. These are harmless to healthy people but could be potentially dangerous to other people with CF. These infections can usually be eradicated or kept at bay with early antibiotic treatment but bugs often eventually become established or become antibiotic-resistant, leading to patients’ lungs becoming colonised.

The risk of cross-infection from such bacteria means that people with CF often have to avoid contact with others, which can lead to them becoming isolated.

The new website is a public engagement with science project which has been supported by a Wellcome Trust People Award. The site has been designed to work as a virtual ‘conference,’ where patients and their families will be able to access live webcasts of experts discussing the latest research findings and explaining the impact they could potentially have for people with CF. Patients will have the opportunity to ask the experts questions in real time.

Publicise and communicate

As the disease affects just 9,000 people in the UK researchers can often find it difficult to recruit enough patients on to clinical trials. CFUnite.org will offer academics and clinicians a shop window to publicise their future studies and to communicate with patients about how they can get involved.

The first virtual conference is set to take place on Saturday January 12 and the programme will include a discussion led by Dr David Sheppard from The University of Bristol on the new ‘wonder drug’ Ivacaftor, which is one of the most significant new treatments for some patients with CF in recent years.

The event will also include presentations on the results of clinical trials of Ivacaftor for two different types of CF mutations as well as a talk on the patient dimension by Ed Owen, CEO of the Cystic Fibrosis Trust.

CF patients and their families can access the site at www.cfunite.org, can follow on social media via Twitter via @CFUnite and Facebook/CFUnite

After reaching new highs earlier in the year, shares of Vertex Pharmaceuticals (NASDAQ: VRTX) struggled to hold on to their gains and have recently entered a very bearish reversal, signaling that the market is beginning to change its perspective on the $9.14 billion company.
Bullishness on VRTX stock reached its height back in May of this year when interim data from a phase II trial for their developing drug VX-809 showed significant efficacy when paired with already FDA-approved and marketed KALYDECO in the treatment of cystic fibrosis patients with the common F508del mutation. KALYDECO (also called ivacaftor and VX-770) was approved by the FDA on January 31, 2012 and is still the only approved drug for the treatment of the underlying causes of cystic fibrosis.

The big limitation to the drug and its potential is that it is only approved for patients that have at least one copy of the G551D mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, which represents a smaller portion of the aggregate pool of cystic fibrosis patients. Investors are excited about their other cystic fibrosis drug VX-809 because it targets a wider population of cystic fibrosis patients, specifically those who have two copies of a particular mutation that is referenced as “F508del.”

In the Phase II trials that studied VX-809 and KALYDECO/ivacaftor in a combination study with F508del patients, it was interesting to note that VX-809 did in fact perform better in combination with KALYDECO/ivacaftor and showed very weak results when used alone. It’s not clear why VX-809 did not perform when used alone, but it’s clear that the mixed results from the recent phase II trial caused some uncertainty in the market. Since October 12, 2012 when the detailed results of the ivacaftor and VX-809 combination study were released, Vertex stock dropped almost 26%!

Other factors have also been dragging down Vertex in recent trading, including a mediocre Q3 2012 earnings release from November 1st that showed weaker-than expected revenue (bringing up growth concerns from the analysts), and unexpectedly high increases in company expenses, especially in R&D which had a generous quarterly budget of $200.16 million.

This enormous R&D budget is going towards a number of clinical trials that are related to the late-stage development of VX-809 (which may be combined with ivacaftor based on the results we saw in F508del patients), but there are also trials that are attempting to bring ivacaftor to cystic fibrosis patients with mutations other than G551D. For instance, the company is funding a phase III study for ivacaftor in the treatment of cystic fibrosis patients that have at least one copy of the R117H mutation, which represents ~3% of the US cystic fibrosis patient population. Another phase III trial is mentioned in company files for ivacaftor in the treatment of ~1% of the cystic fibrosis population with at least one “non-G551D CFTR gating mutation.” The takeaway is that increases in Vertex R&D expenses can have some very positive long-term effects on the company’s revenue, which will benefit from the increasing potential of ivacaftor and the upcoming VX-809 for a variety of cystic fibrosis subpopulations.

Investors who are interested in the long-term potential for Vertex in the cystic fibrosis drug market can find some comfort in the expected growth of ivacaftor, which only brought $49 million in the last quarter but is expected to gain considerable momentum upon approval for other portions of the cystic fibrosis patient population. There is also VX-809, which we mentioned as a potential combination therapy to ivacaftor for the common f508del patient population (a much larger target than currently targeted by ivacaftor in terms of potential revenue.)

VRTX stock is currently in a downtrend that isn’t seeing an increasing number of short positions (implying that there is a significantly amount of profit-taking from long-term investors). Since there are no major events in the near future there is no reason for interested buyers to rush into a long position, although Vertex becomes more favorable in fundamental value as market pessimism rises. A downgrade from Credit Suisse yesterday (November 14th) to “Neutral”, and a reduced price target on the stock from $56/share to $48/share suggests that bearishness might be getting too extreme.

Have you ever imagined what life would be like if every breath was, literally, a struggle? Before we continue, I challenge all the readers with healthy lungs to do the following: find a straw and try to breathe in and out through it for 30 seconds while plugging your nose. This is unfortunately what every breath feels like for most cystic fibrosis sufferers: shallow, difficult, and like they aren’t getting enough air inside their lungs.

Cystic fibrosis (CF) is a genetic lung disease affecting approximately 30 000 children and adults in the United States. There are many systems affected by CF, but this article will focus more on the impact it has on the lungs. A defective gene causes these individuals’ lungs to constantly fill up with thick, sticky mucus. Despite having a strong cough, most CF sufferers are unable to effectively expectorate this mucus without pharmaceutical and often physical assistance from a Physical Therapist (PT). This may come as a surprise, as our profession is primarily reputable for working with people who suffer from musculoskeletal/orthopedic issues.

Often times, older individuals with CF know their bodies so well that they can roughly estimate where the mucus is located in their lungs. Additionally, with time and teaching, they are able to self-treat, or receive assistance from a family member, when at home. In hospital, this knowledge can be very helpful, as it can assist in guiding the Physical Therapist’s treatment. Although we are armed with many tools for secretion clearance, some prove to be more effective than others for certain people. For this reason, it becomes very important to treat each patient on a case-by-case basis.

PT treatment for CF can be categorized as follows: breathing exercises such as active cycle of breathing technique (ACBT) and autogenic drainage; assistive devices, such as the Flutterâ and Acapellaâ; manual techniques, such as percussions and vibrations, and equipment, such as the high frequency chest wall oscillator (HFCWO), a large and expensive apparatus, which is not readily available in all hospital settings.

Physical Therapy treatments should be initiated soon after the diagnosis of CF has been posed, as it can help reduce the risk of infection and prevent lung damage caused by the over accumulation of mucus.

By Savara Pharmaceuticals

Savara Pharmaceuticals

Last modified: 2012-11-13T13:09:49Z

Published: Tuesday, Nov. 13, 2012 – 5:07 am

Copyright 2012 . All rights reserved. This material may not be published, broadcast, rewritten or redistributed.

AUSTIN, Texas, Nov. 13, 2012 — /PRNewswire/ – Savara Pharmaceuticals today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug status to AeroVanc™(vancomycin hydrochloride inhalation powder) for the treatment of pulmonary methicillin-resistant Staphylococcus aureus (MRSA) infection in cystic fibrosis (CF) patients. Orphan drug designation qualifies a company for several benefits, including the potential for market exclusivity, development grants, and for certain tax credits.

AeroVanc is the first inhaled antibiotic being developed to address the growing population of MRSA-infected CF patients. Savara is currently preparing for its Phase IIa clinical study of AeroVanc’s efficacy, to be carried out in 20 CF centers in the United States. In Phase I studies of AeroVanc in healthy volunteers and CF patients, AeroVanc was well tolerated and demonstrated an excellent pharmacokinetic profile.

“AeroVanc is a much needed addition to the current treatment options for MRSA infected CF patients,” said Michael Konstan, M.D., Chairman, Department of Pediatrics, and Director of the Cystic Fibrosis Center at Rainbow Babies and Children’s Hospital and Case Western Reserve University. “In the absence of an FDA approved inhaled antibiotic therapy directed at MRSA, CF clinicians are increasingly prescribing off-label nebulization of the intravenous formulation of vancomycin. This therapy is generally well tolerated, has high antibacterial activity against MRSA, and patients have a good clinical response.”

“Orphan drug designation in combination with our intellectual property provides strong market exclusivity potential for AeroVanc,” said Robert Neville, Chief Executive Officer of Savara Pharmaceuticals. “We hope to provide similar benefits to MRSA infected patients that tobramycin provides for the treatment of Pseudomonas aeruginosa infections. The response from the cystic fibrosis community has been very encouraging, so much so that physicians like to refer to AeroVanc as the ‘TOBI for MRSA’.”

The U.S. Orphan Drugs Act aims to encourage the development of drugs involved in the diagnosis, prevention or treatment of a medical condition affecting fewer than 200,000 people in the United States. Orphan drug designation grants U.S. market exclusivity to a drug for a particular indication for a seven-year period if the sponsor complies with certain FDA specifications. Additional incentives for the sponsor include tax credits related to clinical trial expenses and a possible exemption from the FDA user fee, and the orphan status also allows the sponsor to apply for grants to support clinical trials. 

About AeroVanc™

AeroVanc™ (vancomycin hydrochloride inhalation powder) is a proprietary inhaled dry powder form of vancomycin in a capsule-based device designed for convenient self-administration. AeroVanc is being developed for the treatment of MRSA lung infection in cystic fibrosis patients. Vancomycin administered by IV is the antibiotic of choice for the treatment of MRSA-related bronchopneumonia, however, IV administration, poor penetration into the lungs and systemic toxicities limit its use in a chronic setting. By delivering vancomycin directly to the site of infection, AeroVanc is expected to improve clinical efficacy and reduce adverse effects due to systemic drug exposure.

AeroVanc has demonstrated positive safety and tolerability results in Phase I clinical studies conducted in healthy subjects and patients with cystic fibrosis, with a pharmacokinetic profile that supports its potential as a once- or twice-daily treatment for pulmonary MRSA infection.

About Cystic Fibrosis and MRSA

Cystic fibrosis is a life-shortening genetic disease characterized by thick, sticky mucus in the lungs and frequent lung infections, which result in loss of lung function. As the disease progresses, the lungs of CF patients are typically infected with bacteria that are difficult to eradicate. Such infections are usually treated with inhaled antibiotics. In recent years, infection by MRSA has become increasingly common, with a prevalence of almost 30 percent of the estimated 30,000 CF patients in the United States. Persistent MRSA infection is associated with faster decline in lung function and a significantly shortened life expectancy. Currently there is no approved inhaled therapy for MRSA infection for CF patients. 

About Savara Pharmaceuticals

Savara Pharmaceuticals is an emerging specialty pharmaceutical company developing innovative pulmonary drugs for the treatment of serious and life-threatening conditions.  The company’s lead product, AeroVanc™ (vancomycin hydrochloride inhalation powder), is the first dry powder inhaled antibiotic for the treatment of methicillin-resistant Staphylococcus aureus (MRSA) infection in patients with cystic fibrosis. For more information, please see Savara’s website at www.savarapharma.com.

SOURCE Savara Pharmaceuticals