Ex-mayor died after heart failed

9:50am Saturday 12th May 2012 in Local

A FORMER Westhoughton mayor died after his heart stopped as he was being taken to hospital by ambulance, an inquest heard.

Ronald Halliwell, who was a Liberal Democrat member of Westhoughton town council from 1999 to 2007, and Mayor in 2004 to 2005, died on February 22.

The 78-year-old was being moved from his home in Henry Street, Westhoughton, to an ambulance when his heart stopped, Bolton Coroner’s Court was told.

The father of two was pronounced dead at the Royal Bolton Hospital later that day.

The inquest heard that the former joiner, who had worked with asbestos, was diagnosed with incurable pulmonary fibrosis five months before his death. He suffered from breathlessness and was being treated with oxygen.

on the day he died, Mr Halliwell’s symptoms had worsened and his GP had requested an ambulance to take him to hospital.

at the inquest, Mr Halliwell’s wife, Florence, and his daughters, Karen Humphreys and Janet Halliwell, criticised paramedics for asking Mr Halliwell to slide down the stairs on his bottom, rather than carry him in a chair.

but deputy coroner Alan Walsh accepted it was up to the paramedics to determine the best way to move a patient.

He recorded a verdict of natural causes and said Mr Halliwell’s death was not caused by asbestos.

after the inquest, Mrs Humphreys said: “He put family first every time and gave a lot back to the community. It is a great loss, not just to us but to Westhoughton.”

Ex-mayor died after heart failed

9:50am Saturday 12th May 2012 in Local

A FORMER Westhoughton mayor died after his heart stopped as he was being taken to hospital by ambulance, an inquest heard.

Ronald Halliwell, who was a Liberal Democrat member of Westhoughton town council from 1999 to 2007, and Mayor in 2004 to 2005, died on February 22.

the 78-year-old was being moved from his home in Henry Street, Westhoughton, to an ambulance when his heart stopped, Bolton Coroner’s Court was told.

the father of two was pronounced dead at the Royal Bolton Hospital later that day.

the inquest heard that the former joiner, who had worked with asbestos, was diagnosed with incurable pulmonary fibrosis five months before his death. He suffered from breathlessness and was being treated with oxygen.

On the day he died, mr Halliwell’s symptoms had worsened and his GP had requested an ambulance to take him to hospital.

at the inquest, mr Halliwell’s wife, Florence, and his daughters, Karen Humphreys and Janet Halliwell, criticised paramedics for asking mr Halliwell to slide down the stairs on his bottom, rather than carry him in a chair.

But deputy coroner Alan Walsh accepted it was up to the paramedics to determine the best way to move a patient.

He recorded a verdict of natural causes and said mr Halliwell’s death was not caused by asbestos.

After the inquest, Mrs Humphreys said: “He put family first every time and gave a lot back to the community. it is a great loss, not just to us but to Westhoughton.”

23:45, April 2, 2012

Harutyun Sukiasyan is an active five year-old who wants to be a soldier when he grows up.

The boy also suffers from CF (Cystic fibrosis).  Most kids so diagnosed in Armenia don’t make it past the age of twenty.

It is a genetic disease affecting most critically the lungs, and also the pancreas, liver, and intestine. it is characterized by abnormal transport of chloride and sodium across the epithelium tissue, leading to thick, viscous secretions.

In 1959, the median age of survival of children with cystic fibrosis in the U.S. was six months. now, it is 37.4 years. In Canada, median survival increased from 24 years in 1982 to 47.7 in 2007.

Harutyun, his mother Svetlana and sister Srbuhie live with relatives in the #2 Section of Nork, Yerevan. Svetlana is divorced.

Eight people live in a damp one room apartment. Harutyun sleeps on the floor next to his mom.

The little boy’s coughing spells have gotten worse of late and his loss of sensitivity has increased.

Srbuhie is also sick. Svetlana has placed her in the care of a boarding school so that the girl can be properly fed and have a bed to sleep in. Srbuhie visits the family on the weekend.

Svetlana told me that Harutyun first started manifesting symptoms of the disease at the age of six months. at first, doctors couldn’t make a proper diagnosis.

It was only months later that doctors in Armenia diagnosed MS and told the mother that the disease was curable and wasn’t life threatening.

According to Wikipedia – while there are no cures for cystic fibrosis there are several treatment methods. the management of cystic fibrosis has improved significantly over the past 70 years. while infants born with cystic fibrosis 70 years ago would have been unlikely to live beyond their first year, infants today are likely to live well into adulthood. Recent advances in the treatment of cystic fibrosis have meant that an individual with cystic fibrosis can live a fuller life less encumbered by their condition.

Svetlana had to wait another three years before the health authorities registered the boy as “disabled”. (In Armenian – hashmandam. there are various degrees of disability in Armenia with each allowing for various pensions and treatment payments.)

She says that her son was never prescribed any medication for three years. now he receives Creon and some vitamins.

(Creon contains digestive enzymes and is used to improve food digestion in people who cannot digest food properly)

“the last time Harutyun got the drug was in January. But the medication had already expired. I threw it in the garbage. In February, the polyclinic gave us the drug but it was the wrong dosage. I had to cut it up,” says the mother.

If he doesn’t take the drug before eating, he will throw up the food two or three minutes later.

Family physician Marina Gasparyan at the #14 Polyclinic in Yerevan says that the drug hadn’t expired, but does admit that the wrong dosage had been given afterwards.

Gasparyan says that they are doing all they can to assist, allocating drugs and vitamins.

The nebulizer Harutyun was using to take the drugs no longer works. the family can’t afford to purchase a new one.

All eight people survive on the 50,000 AMD ($129) pension of Svetlana’s mother.

Harutyun’s disability pension is only enough to buy some rice and macaroni. the boy has asked for some fruit for the past week, but Svetlana can’t afford any.

“I just don’t know how long my boy can live in these conditions. He’s not getting the nutrition he needs and the apartment makes matters worse. my priority is to get him out of here somehow and to provide sufficient meals,” said Svetlana.

She’s worked in stores and cafes as a clerk and waitress. But the jobs don’t last long.

When her employer finds out that she’s a single mom with a sick child, they fire her. they just don’t want the hassle.

As we were leaving, Harutyun turned and told us to visit him in the hospital.

“we don’t have a home of our own,” the little boy remarked.

Like other 14-year-olds, Austin Marine likes to play sports and make people laugh. He’s glued to his cell phone and plays the drums at school. he constantly makes funny faces and jokes with his two sisters and parents.

“I’ve got a girlfriend,” Austin said, smiling.

“What’s her name?” asked Renea, his mother, knowing the answer.

“I don’t know!” The whole family erupted into laughter.

The laughter and positive words are common in the Marine household. From the outside looking in, people would not know the family is coping with Austin’s terminal illness. The 14-year-old was diagnosed with Mucolipidosis when he was 2 years old.

“They kept telling us so many different things,” Jimmy, his father, said, adding that the family did not know what to do and were told to live their lives like normal. “They told us to enjoy him while we had him.”

The couple turned to the Internet and sought other families who have children with the same illness. They also attended conferences to meet these families so they could share stories and ideas. despite their positive attitudes, Austin’s illness seemed dire and he was not expected to live very long. he and his family went to Disney World when he was 3 years old through the Make-A-Wish Foundation because the family did not know how much time they had with him.

“It’s a blessing we have him,” Renea said.

ML is an I-Cell disease that prohibits the body from making certain enzymes. This results in cells storing unnecessary material, causing damage throughout the body. This damage affects a person’s heart, bones, joints, respiratory system and central nervous system. even though the disease is not apparent at birth, signs and symptoms develop with age as more cells become damaged by the accumulation of material. over time, Austin has developed severe issues with his bones, heart, lungs, airways, eyes and ears, and these problems will grow worse with age.

Austin’s parents held him back in pre-school when they were learning about the disease and developing a plan for the future. The 14-year-old is now a sixth-grade student at Woodlawn Middle School in Mebane and his parents have been very pleased with the school’s willingness to meet Austin’s needs. They are also grateful for the elementary school he attended.

“When I leave South Mebane, will it close,” Austin asked his parents, smiling. even though he was joking, his parents knew there was some truth to the question, saying many people at the school missed Austin after he graduated from the elementary school.

“He’s everybody’s hero,” Renea said.

AUSTIN’S BONES are very brittle and he has had two back surgeries and one hand surgery. on his most recent back operation, Austin was in the hospital for 21 days. His parents were desperate for him to recover and promised their son anything he wanted when he was released from the hospital. Like many teenagers, Austin wanted a dog. he is now happy to have Bailey, a Yorkie, running around the house.

The family cherishes every day they have together. From attending sporting events to playing catch in the front yard, this family knows how to work together. Their faith also gives them strength and the Marines appreciate the support they receive from the first Baptist Church of Mebane.

“at church they call me, ‘Austin’s dad,’” Jimmy said.

“at school I’m Austin’s sister,” Courtney, 11 years old, said.

“Austin’s the most popular guy in Mebane,” Renea added. Austin smiled and laughed, grateful to know he can make people happy just with his presence and his caring personality.

Austin’s two sisters are always willing to help their big brother and, like any younger sisters, love to tease him.

“Austin is very funny. We like to make him laugh,” Logan, 10 years old, said. She loves “everything” about her brother.

The Marine family is participating in the National MPS Society walk/run on May 19 at Bond Park in Cary. The Society works to find cures for mucopolysaccharidoses and related diseases, including ML. This nonprofit provides support for affected individuals and their families through awareness events, advocacy and research.

Since ML is a rare disease, limited research is being done to find a cure or treatments for its symptoms.

“It’s not like cystic fibrosis where there’s a lot of money going into it,” Jimmy said. “It’s almost sad you have to do something to make people donate.”

The family hopes the event raises awareness about Austin’s rare disease and money for research. Donations can be made online at mpsrunnc.com. The family asks donors to write that their gift is in honor of “Austin Marine for I-Cell Research” in the comments section.

Despite the uncertainty the family has about the future, the Marines remain positive and encourage Austin to achieve his dreams.

“We love you just the way you are,” Renea said to her son. “You can do whatever you want, no matter what.”

What would you do if you saw 1 in 88 children walking around with their hair on fire?

It would likely unnerve you, to say the least, and you’d probably be compelled to search for an explanation, asking everyone from doctors to scientists and even your next door neighbor: “What’s going on with our children?! why are so many of them walking around with their hair on fire? why haven’t we found a way to extinguish the flames yet?”

PHOTO COURTESY JO ASHLINE ADVERTISEMENT

For parents of children diagnosed with autism, the urgency we feel as the CDC announced today that 1 in 88 American children are now diagnosed with some form of an Autism Spectrum Disorder, is akin to a raging fire burning out of control with no water in sight.

The new data, which also shows that 1 in 54 boys are diagnosed with an ASD —  making them five times more likely to have the disorder than girls — was  based on a study conducted by the Autism Spectrum Disorder Monitoring Network , or ADDM.

The study, which gathered data on children living in 14 different areas within the United States and “comprised over eight percent of the United States population of 8-year-olds in 2008,”  focused on 8-year-olds with the presumption that most children that present with symptoms are identified and subsequently diagnosed with an ASD by that age.

The numbers, according to the CDC website:

2007: CDC’s ADDM Network reports that about 1 in 150 children have an ASD

2009: CDC’s ADDM Network reports that 1 in 110 children have an ASD

2012: CDC’s ADDM Network reports that 1 in 88 children have an ASD

Estimated prevalence of ASDs increased 23% during 2006 to 2008 and 78% during 2002 to 2008.

What causes autism? There are many theories, and I can only speak to my personal beliefs and experiences. in the case of our son Andrew, we know that many factors were at play, including a compromised immune system at birth due to undiagnosed Cystic Fibrosis and an onslaught of environmental triggers that created the perfect storm.

But what’s relevant today are the numbers: 1 in 88.

And while the spectrum is wide and varied, and not every child diagnosed with an ASD is non-verbal or severely cognitively delayed and physically impacted like my son, many are still struggling in other ways, in classrooms and on playgrounds across the country.

All of these children, regardless of the severity of their diagnosis, are both in need and deserving of therapies and interventions designed to provide them with the opportunities to succeed in life and, as is the case for Andrew, gain as much independence as possible through these therapies. with school districts and state-funded programs strapped for cash and insurance companies doing everything they can to make coverage for these services difficult to attain by parents for their children, I can’t help but wonder: What now?

My son’s classroom has 8 students, including him. There is one special education teacher (who is fabulous), and three aides. These kids are some of the most beautiful angels on earth, and just being in their presence gives me clarity and perspective; you’d count yourself lucky to be touched by their grace and unique outlook on life. But not once has there been a time that I’ve stopped by when I haven’t seen one of these amazing children whimper from pain, act out in defiance from frustration, or withdraw completely from being misunderstood.

There hasn’t been a day that I’ve gone into that room and not witnessed the side of autism that leaves parents desperate for an answer while they forsake everything in an attempt to help their child.

And while I can only speak to the challenges that my own child with autism deals with, such as not being able to talk or perceive danger of any kind, and pick at his hands until they bleed because it’s the only way to make the unruly world a little more tolerable, I can speak to the fear that many of us in the same boat feel as we watch more and more children suffer at the hands of a diagnosis that is spreading like wildfire, with no concrete cause or solution in sight.

1 in 88 folks. There’s a story behind every number.

See this post in its original form, and read more on OC Moms: The Mom Blog.

Two gene regions that affect a baby’s size at birth have been uncovered by a large international team of researchers, made up of scientists from several UK and international centres including Imperial. one of the regions is also associated with type 2 diabetes.

The research, published in Nature Genetics, is the first robust evidence that a well-known link between lower birth weight and susceptibility to type 2 diabetes has a genetic component.

The team analysed over 38,000 Europeans from 19 studies of pregnancy and birth. Two genetic variants showed strong associations with birth weight. one of the variants, in a gene called ADCY5, has recently been linked with susceptibility to type 2 diabetes. Individuals who inherit two risk copies of this variant are at a 25 per cent higher risk of diabetes in adulthood than those who inherit two non-risk copies.

Professor Marjo-Riitta Jarvelin, Chair in Lifecourse Epidemiology (Public Health), and one of the leaders of the research, said: “we have, for nearly two decades, tried to discover the factors which may explain why smaller fetal size associates with so many later life chronic conditions such as heart disease…Our own studies have demonstrated that a number of environmental factors influence fetal growth and that some fetuses are more vulnerable to those than the others. I believe that our genetic landmark discoveries will give us opportunity to answer these puzzling questions in the near future.”

— Adapted from a news release issued by the Wellcome Trust

Read the full news story

Tags: Faculty of Medicine, Issue 218, News, Public Health Posted in College, Research | No Comments »

On the surface, Lindsay Shipp’s story may sound sad, but it’s a story of hope with a happy ending.

All her life, Shipp has been trying to keep up a positive attitude. she knew the disease from which she suffered – cystic fibrosis – could cut her life short, but she tried to play it off like she was fine.

“I wanted to be strong for my family,” said Shipp, 28, an inventory analyst for Apple who lives in San Diego with her husband, Charles. “If they saw fear in me, they’d crumble. But I was scared . . .I’m not going to die of natural causes, I am going to die from CF.”

Shipp – who was involved in a clinical trial for a drug that treats the root cause of CF — can finally start to relax. The drug, kalydeco (generic name ivacaftor and made by Vertex), was approved by the U.S. Food and Drug Administration at the end of January after demonstrating it improves the lung function in patients with a rare variant of the disease that affects 1,200 Americans.

“I can exercise, I can go out and run – I’ve never been able to do that my whole life,” Shipp said. “I’m training for a 5K – not because I enjoy running, but because I can do it.”

Affecting a total of 30,000 Americans, CF is a life-threatening disease that has no cure. it causes a sticky mucus build-up in the lungs and other organs as well as multiple infections and digestive problems.

The median lifespan of a CF patient is currently in the mid-30s, according to Dr. Mark Pian, director of the UCSD School of Medicine Rady Children’s Hospital CF Center in San Diego. Pian oversaw the clinical trial in which Lindsay took part, and he immediately noticed a difference.

“In the mildest situation, one may have a chronic cough, but no real impairment in day-to-day living,” Pian said. “However, this is not the case as the disease progresses. The most common symptoms are chronic, productive cough and mucus and nutritional impairment that gets progressively more severe with time.”

Such was the case for Shipp, who said she noticed her symptoms really intensified in high school. An avid dancer and singer, that’s when “things went downhill,” she said.

“I was having trouble breathing with dance, I was sick a lot and out of school – my peers were like, ‘Where are you?’ I’d have to explain I was being treated for CF,” Shipp said.

When Shipp was first diagnosed with the disease as a baby, doctors told her parents she’d be lucky if she made it to high school.

“They tried to give me the best life possible, and I kept outliving the life expectancy because it was changing,” she said. “As a kid, my goal was to graduate college. I didn’t have much plans for after that.”

Pian said more patients are expecting to lead full and productive lives, but with the knowledge that CF will shorten these lives. he expects there will one day be a cure for the disease, but in the meantime – there’s kalydeco.

“Lindsay described a difference to me almost immediately, and I could see this for myself in the first few weeks,” Pian said. “Of course, neither of us knew whether she was taking kalydeco or placebo. she was certain, and turned out to be correct, that she was taking (the drug).”

Kalydeco is a little blue pill that’s taken twice a day. Its most common side effects include upper respiratory tract infection, headache, stomachache, rash, diarrhea and dizziness.

But Shipp said she’s never felt better. her treatments prior to taking the drug were timely and annoying, filling up most of her day.

Shipp would often be treated with high-frequency chest wall compressions, or as CF patients refer to it – she would have to wear ‘the vest.’

According to Shipp, the vest has tubes attached to it connecting to a larger unit that puffs air – and the whole machine shakes your body. The treatment can take anywhere from 30 minutes to one hour and is done two or three times a day.

“It’s a full-time job on top of your job,” Shipp said, adding she also had to take nebulizers and inhaled antibiotics. and although Shipp said she has reduced the number of times she is using the vest, Pian said patients should still use it, along with other drug protocols prescribed by their doctors.

How the drug works

CF is caused by defective or missing CFTR proteins resulting from mutations in the CFTR gene, Pian explained. those proteins act as channels at the cell’s surface and affect the flow of salt and water across the surface of organs, such as lungs and the gastrointestinal tract. When the defective protein fails to work, that’s when the sticky mucus builds up.

The drug is approved for people age 6 and older who have at least one copy of the G551D mutation in the CFTR gene.

“Kalydeco is designed to keep CFTR protein channels at the cell surface open longer to improve the transport of salt and water across the cell membrane,” Pian said. “The desired effect is that the dry, thick CF mucus will become better hydrated to clear.”

Pian said a lot of work still needs to be done in the CF field – but kalydeco’s approval shows a cure can be possible.

Vertex said it anticipates major insurance companies to cover the cost of kalydeco, but since the drug’s approval is still so new, there’s not a lot of data available yet.

“We do have a comprehensive program in place to make sure eligible patients can get it,” said Megan Goulart, a spokeswoman for Vertex, which includes a free medicine program for people who do not have insurance, co-pay assistance for those who do have commercial insurance and referrals to third-party foundations for those with Medicare or Medicaid or who live in Massachusetts.

Visit Vertex’s website to learn more about these programs and the drug. 

Vertex worked with The Cystic Fibrosis Foundation, which provided data and contributed $75 million of funding toward the drug’s trial. 

“(This) marks an important milestone in our journey to find a cure for cystic fibrosis,” said Robert J. Beall, president and CEO of the foundation. “Kalydeco addresses the underlying cause of cystic fibrosis, and the science behind the drug has opened new doors that may eventually lead to a cure for all people living with this disease.”

A recent newlywed, Shipp never imagined she would be able to have children naturally, a dream she is beginning to think may come true thanks to the clinical trial.

“With this pill, it’s possible that I can have kids and lead a full life to raise them,” she said. “I don’t have to worry about dying before they graduate.”

Click here to learn more about Lindsay by reading her blog.

Portfolio news 2012 Synairgen plc – Preliminary statement of results for the six months ended 31 December 2011 01 Feb 2012

Synairgen plc (LSE: SNG), the respiratory drug discovery and development company with a particular focus on viral defence of the lungs, today announces its audited results for the six months ended 31 December 2011 following the change of year-end to that date.

Operational highlights Significant progress on core development programmes:

• Phase II trial of inhaled interferon beta ('IFN-beta') in asthma on schedule: last subjects were dosed in December 2011, with results anticipated in March 2012
• Positive results from pre-clinical study completed in November 2011 showing that aerosolised IFN-beta reduced virus-induced pneumonia, suggesting that inhaled IFN-beta may have potential in two further areas:
  • as a broad spectrum antiviral for use in patients admitted to hospital with suspected viral lung infections; and
  • as a post-exposure prophylactic defence against a lethal virus threat to the lungs
• Business development activity for out-licensing of IFN-beta programme being coordinated to coincide with the availability of key clinical trial data 

Financial highlights

• Research and development expenditure for the period: ?1.8 million (year ended 30 June 2011: ?2.9 million)
• Post-tax loss for the period: ?2.0 million (year ended 30 June 2011: ?3.2 million)
• Cash at 31 December 2011: ?3.4 million (30 June 2011: ?4.9 million)

Commenting on the results, Simon Shaw, Chairman of Synairgen, said: "Positive data from the Phase II asthma trial in March 2012 will be a pivotal requirement for us to pursue the further development of inhaled IFN-beta with a licensing partner. such a partnership would be a significant commercial milestone for the Company and we have an ongoing dialogue with several potential development and commercialisation partners."

For further information, please contact:

Synairgen plc Tel: + 44 (0) 23 8051 2800 Richard Marsden, Chief Executive Officer John Ward, Finance Director FinnCap    Tel: + 44 (0) 20 7220 0500 Geoff Nash, Christopher Raggett (Corporate Finance) Stephen Norcross, Simon Starr (Corporate Broking) Newgate Threadneedle  Tel: + 44 (0) 20 7653 9850 Graham Herring Josh Royston

Following the change of year-end, this report covers the six month period to December 2011, during which time the Company has made significant progress with its Phase II study in asthma and its pre-clinical study of viral pneumonia.

in December 2011 Synairgen completed recruitment for its Phase II proof of concept trial of inhaled interferon beta ('IFN-beta') for the prevention of virus-induced asthma exacerbations (acute and prolonged worsening of asthma symptoms). during January and February 2012, the data will be audited and entered into the database. The results are expected in March 2012. 

We announced in November 2011 that, independent of the asthma development programme, the Company had generated strongpre-clinical data, which opens up the potential for two additional indications related to viral infection in the overall population. these are: 1.   Hospitalised patients with a suspected severe viral lung infection; and 2.   Post-exposure prophylaxis for: a.   Highly pathogenic viruses (for example SARS-like viruses) b.   Common respiratory viruses (for example swine flu) in vulnerable populations

these are attractive potential markets, for which there is today little or no effective therapy. Indeed the area of defence against highly pathogenic viruses has become a focus for a number of governments around the world, which are prepared to commit resource to appropriate programmes. We will be investigating this area during 2012.

Asthma and COPD

Phase II clinical trial – Proof of concept in asthma Common respiratory viruses are the major cause of asthma exacerbations. many different viruses, including RSV, parainfluenza virus, coronavirus, over 200 different types of rhinovirus, and many others, are loosely termed 'common cold' or 'flu' viruses.  Influenza infections tend to be more severe and yet not all suspected influenza infections turn out to be influenza at all; many of the suspected influenza infections will be caused by one of the common cold viruses, and vice versa.

in asthmatic patients, and those with other respiratory diseases such as COPD (chronic obstructive pulmonary disease) or cystic fibrosis, the lungs are unable to defend themselves adequately against these common viruses and an exacerbation can ensue. The poor defence appears to be due to compromised IFN-beta-driven immunity. This can be corrected by introducing a small amount of IFN-beta to help cells defend themselves against viruses. This has been demonstrated in Synairgen's in vitro models, using cells from volunteers with asthma (Synairgen's Biobank). it is very important to note that these vulnerable patients do not become severely ill on account of cold symptoms such as a really bad sore throat or very blocked nose. these patients become ill because of the viral infection in their lungs and the way this interacts with their pre-existing lung disease. Synairgen is developing inhaled IFN-beta for delivery directly to the lungs in the form of an aerosol and is not trying to modulate the incidence or severity of symptoms in the nose and throat.

many therapeutic approaches to tackling viral respiratory infections have failed due to the great diversity and adaptability of these very contagious viruses. Tamiflu? and Relenza? are successful examples of the 'one drug one bug' approach, the bug being influenza. however influenza only accounts for approximately 5 – 10% of all respiratory infections and there remains a concern about progressive resistance; Synairgen's inhaled IFN-beta has the advantage of boosting the body's powerful 'virus agnostic' defences in the lung. it has demonstrated activity against all of the common respiratory viruses and some highly pathogenic viruses such as H5N1 'Bird Flu' and the coronavirus SARS. thus Synairgen's inhaled IFN-beta is increasingly proving itself as an inhaled broad spectrum antiviral defence. Furthermore it is considered that resistance to the activity of IFN-beta is highly unlikely to emerge. Synairgen has been fortunate in that the development of inhaled IFN-beta is a re-profiling exercise.  IFN-beta has been administered by injection to thousands of patients with multiple sclerosis over the years, since its first approval in 1993. This significantly de-risks the inhaled programme as any drug which passes through the lungs to the blood system is unlikely to cause any unexpected adverse events. Furthermore, through its exclusive supply and licence arrangement with the Rentschler Group (a manufacturer of IFN-beta), Synairgen has access to a pH neutral formulation suitable for inhalation.

in the Phase I safety and pharmacodynamic clinical trial, conducted in 2008/09, inhaled IFN-beta was well tolerated in moderate asthmatics. there was also convincing biomarker evidence that even in the absence of a virus inhaled IFN-beta 'switched on' the antiviral defences. This provided the Company with 'proof of mechanism', i.e. that the drug could switch on antiviral defences in the lungs of patients with an IFN-beta deficiency.

The Phase II study reporting results in March 2012 will hopefully confirm that there is benefit in boosting/restoring the IFN-beta-driven antiviral defences in asthmatics. in this blinded clinical trial approximately 150 exacerbation-prone asthmatics have been treated with inhaled IFN-beta or placebo at the onset of cold or flu symptoms. The objective is to boost the lungs' immunity so that, as the virus infection builds in the nose and throat, the lungs are able to defend themselves adequately. The primary endpoint is the sACQ (shortened Asthma Control Questionnaire), which is a measure of asthma symptoms. there are a number of secondary endpoints which may also be very informative for signalling the future development strategy for the drug. these are the AI (the Asthma Index – an alternative measure of asthma symptoms), lung function, virus load, markers of pulmonary inflammation and drug safety.

Phase II clinical trial – Proof of concept in COPD Synairgen has developed a Phase II clinical trial protocol for the COPD population. This study has been approved by the relevant authorities, although it will not commence until a partner has been found through the business development licensing activity.

Business development for the asthma and COPD programmes Synairgen has regularly discussed its IFN-beta programme with major pharmaceutical companies and input from these companies has contributed to the design of the overall development programme. Synairgen has, over the years, generated a package of material necessary for the out-licensing of these programmes for asthma and COPD. This package includes safety data, in vitro efficacy data, granted patents in the US, EU and Japan and biomarker data demonstrating proof of mechanism. The most important pieces of information yet to come will be the analysed outcomes of the Phase II trial in asthma, which are anticipated in March 2012. it is Synairgen's intention to out-license the asthma and COPD programmes on the back of the Phase II results and our continuous interactions with potential business development partners have been designed to expedite this process.

Use of inhaled IFN-beta outside of asthma and COPD

during 2011, Synairgen has completed a series of experiments that have demonstrated reduced lung viral load and reduced cell damage in an advanced viral pneumonia pre-clinical model. The drug was given either prior to infection or post infection. these data open up two new significant applications:

1.   Hospitalised patients with suspected severe viral lung infections Inhaled IFN-beta could be used to treat patients as a therapy (prior to the identification of the virus) upon admission to hospital with breathing difficulties caused by a viral lung infection. The broad spectrum antiviral properties of the drug would be advantageous as it takes some time to identify the causative pathogen. As this is an area of great unmet clinical need, Synairgen believes that, subject to an acceptable safety profile from the Phase II asthma trial, it could be in the interest of a government to support this work either through a grant or an award.

2.   Post-exposure prophylaxis against high pathogenic respiratory viruses The threat posed by emerging respiratory viruses such as SARS or terrorist-manufactured virus threats such as aerosolised Ebola or a variant of H5N1 'bird flu' remains one of significant concern to governments. We understand that governments may be interested in funding the development of a broad spectrum inhaled antiviral that can be stockpiled in sufficient quantities to be issued  to people in the event of exposure to lethal viruses, such as key workers or people placed in quarantine following contact with an infected person (for example on a flight). Synairgen will engage with governmental organisations over the coming months to gauge interest and map out a potential development programme.

• against common respiratory viruses in vulnerable populations

Vulnerable patients, for example transplant patients, pregnant women, the elderly, and the very young, can suffer lung complications due to common viruses migrating from the nose and throat to the lungs. Inhaled IFN-beta could be given to these patients as and when they believe they have been exposed to a viral infection. such exposure could be from a work colleague or somebody they live with. Synairgen will consider the future development of inhaled IFN-beta for this indication following the results of the Phase II asthma trial.

Change of Accounting Reference Date The Group has brought forward its financial year-end from 30 June to 31 December for administrative reasons to expedite the production of its annual report and accounts. As a result the audited financial statements cover the six months ended 31 December 2011 with comparative financial information being given for the year ended 30 June 2011.

Statement of Comprehensive Income The loss from operations for the six months ended 31 December 2011 was ?2.24 million (year ended 30 June 2011: ?3.70 million). Research and development expenditure for the period amounted to ?1.82 million (year ended 30 June 2011: ?2.91 million). The main areas of expenditure have been on the asthma Phase II study (SG005) and the pre-clinical study in viral pneumonia. The last subject was recruited into SG005 in December 2011 and the results are anticipated in March 2012. The significant majority of costs on the pre-clinical study were also incurred prior to 31 December 2011 and therefore expenditure on both these projects will reduce significantly during the first half of 2012.

other administrative costs for the period amounted to ?0.42 million (year ended 30 June 2011: ?0.90 million). The research and development tax credit for the period was ?0.25 million (year ended 30 June 2011: ?0.43 million). The loss after tax for the period was ?1.97 million (year ended 30 June 2011: ?3.23 million) and the loss per share was 2.83p (year ended 30 June 2011: loss of 5.37p).

Statement of Financial Position and cash flows At 31 December 2011, net assets amounted to ?3.12 million (30 June 2011: ?4.99 million), including net funds of ?3.35 million (30 June 2011: ?4.89 million). The principal elements of the ?1.54 million decrease over the six months ended 31 December 2011 (year ended 30 June 2011: ?0.12 million decrease) in net funds were:

• Cash used in operations of ?1.93 million (year ended 30 June 2011: ?3.02 million outflow);
• Research and development tax credits received of ?0.40 million (year ended 30 June 2011: ?0.38 million); and

Share issue proceeds (net of costs) ?nil (year ended 30 June 2011: ?2.50 million).

Positive data from the Phase II asthma trial in March 2012 will be a pivotal requirement for us to pursue the further development of inhaled IFN-beta with a licensing partner. such a partnership would be a significant commercial milestone for the Company and we have an ongoing dialogue with several potential development and commercialisation partners.

Independent of an asthma and COPD licensing transaction, we have a programme to investigate the potential development of inhaled IFN-beta as a broad spectrum antiviral for use in patients admitted to hospital with suspected viral lung infections, and also as a post-exposure prophylactic defence against a lethal virus threat to the lungs.

DRACUT — Mark Miller will be spending this Christmas being thankful to a stranger who gave him one of life’s greatest gifts — life itself.

Miller, 43, received a liver transplant in February. all he knows about the person whose liver saved his life is that he was a 38-year-old man who died of a brain aneurysm at Beth Israel Hospital in Boston. But Miller said he thinks about him every day.

“It’s really the ultimate gift you can give,” said Miller, sitting in the kitchen of his Dracut home, with presents arranged under a beautifully decorated Christmas tree in the next room. “It’s Christmastime. what better gift could I have gotten this year?”

Last Christmas, Miller’s health suddenly turned for the worse. He had just been diagnosed in November with liver disease after a quick onset of symptoms. the day after Christmas, he was admitted to the hospital, not knowing what was ahead of him.

After a series of tests, it was determined Miller was a candidate for a liver transplant.

He said his brothers, friends and other members of his family said they were willing to be a live donor, but because of the severity of Miller’s condition, live donation wasn’t an option.

Liver transplant candidates are given a score up to 40 based on three laboratory values that determine where they are listed on the recipient list. a score of 40 means there is about a 90 to 95 percent chance that the patient will die in the next three months, according to Dr. Elizabeth Pomfret, chairwoman of the Department of Transplantation at Lahey Clinic in Burlington.

Miller said his score was 40.

He waited three weeks for a match.

Miller said he would love to meet his donor’s family, but understands that it may be difficult for them to meet him.

“I just want to express my gratitude,” he said. “I think of them often.”

The new England Donor Bank ensures that privacy is kept between the donor family and recipients.

Laura Dempsey, a spokeswoman for the donor bank, said sometimes a recipient will meet their donor’s family, but it’s not common.

“The recipient is so thankful and so happy that they’ve had this second chance at life, sometimes they are eager to thank the donor family,” she said. “But you have to keep in mind the donor family is grieving.”

Giving life-saving gifts

A Westford family, whose 14-year-old daughter’s organs were donated after she died in a car accident, will be honoring their daughter on Jan. 2 in Pasedena, Calif., with other organ donors’ families from around the world.

Donate Life sponsors a float at the Rose Bowl Parade. this year, Katie Enos’ image will be part of the float, along with 71 donors who will be honored from all over the U.S., Canada, Japan and Taiwan.

The image was decorated with earthen materials in a ceremony at Westford Academy earlier this month that included Katie’s family and friends.

Katie was killed in June 2010 when she was hit by a car while crossing Carlisle Street in Westford after leaving a soccer camp.

Six months earlier, after watching a television show about organ donation, Katie had told her parents that she wanted her organs to be donated if she died.

That decision has since not only affected the six people who received Katie’s organs, including a 17-year-old boy who has cystic fibrosis and a woman in her 50s, but Edward Enos said his daughter’s decision has impacted her friends.

He said he’s sure some of Katie’s friends have told their parents that they want to be organ donors, a conversation that many of them probably wouldn’t have had.

“It’s really about awareness, that you can impact a lot of lives when you donate your organs,” Enos said.

Even as a young teenager, Katie knew how important it is to become an organ donor.

“This is something that Katie cared about a lot that when you’re gone there is something more that you can give,” Enos said.

According to the United Network for Organ Sharing, the private, nonprofit organization that manages organ transplants across the country, there are more than 112,000 people in the country awaiting transplants.

On average, 18 people in the U.S. die each day while awaiting a life-saving transplant, according to the network.

In new England, 4,500 people are waiting for an organ transplant, according to the new England Organ Bank.

Last year in new England, there were 817 transplants from 273 donors, according to the new England Organ Bank.

Thirty-six percent of registered drivers in Massachusetts are registered organ donors, according to organ bank.

Pomfret said education and awareness about organ donation is required so that people have conversations with their loved ones and make their wishes known.

Miller said he doesn’t know if his donor was a registered organ donor or his family made that decision for him, but he said he’ll be forever grateful for the gift he received.

“I honestly feel like the luckiest person in the world,” he said.

Intense endurance exercise — such as running a marathon — may induce cardiac damage confined to the right ventricle, a small study showed. Highly trained endurance athletes had reductions in right ventricular function immediately after a race, although it mostly returned to normal about a week later, according to André La Gerche, MBBS, PhD, of the University of Melbourne in Australia, and colleagues. however, a handful of the athletes had signs of subclinical myocardial scarring on cardiac MRI, “suggesting that repetitive ultra-endurance exercise may lead to more extensive right ventricular change and possible myocardial fibrosis,” the researchers reported online in the European Heart Journal. Action Points  

• This study of 40 endurance athletes found evidence of right ventricular effects after an endurance event that largely resolved by six to 11 days later.
• Also, no concomitant left ventricular effects were observed.

There were no changes in left ventricular function, which “provides further circumstantial evidence for the emerging concept that the right ventricle may be more susceptible to exercise-induced injury [than the left],” they wrote.

The study included 40 athletes (mean age 37) who were participating in a marathon, an endurance triathlon, an alpine cycling race, or an ultra triathlon. all trained for more than 10 hours a week and had finished in the top quarter of a recent endurance race. none had cardiac symptoms or risk factors.

The researchers evaluated the athletes two to three weeks before the race, immediately after the race, and six to 11 days after the race.

Compared with baseline, right ventricular volumes increased, and all measures of right ventricular function worsened immediately post race. Left ventricular function was unaffected.

Levels of two biomarkers of myocardial injury — cardiac troponin I and B-type natriuretic peptide — significantly increased following the race (P≤0.003 for both). the changes were associated with reductions in right ventricular ejection fraction (P≤0.002 for both), but were unrelated to left ventricular ejection fraction.

Lower right ventricular ejection fraction was significantly associated with longer race duration and increasing peak oxygen uptake (P≤0.011 for both).

In the 39 athletes who underwent cardiac MRI, five had delayed gadolinium enhancement confined to the interventricular septum, indicative of subclinical myocardial fibrosis. these athletes had been competing in endurance sports longer and had lower right ventricular ejection fractions compared with those with normal MRI findings.

Because the study was not powered to assess clinical outcomes, the significance of the MRI findings requires further study, according to the authors.

The study “begs the hypothetical question whether repetitive longstanding bouts of arduous exercise result in the development of an acquired form of arrhythmogenic right ventricular cardiomyopathy,” Sanjay Sharma, MD, and Abbas Zaidi, MBBS, of St. George’s University of London, wrote in an accompanying editorial.

“The results provide food for thought and the data should be embraced to galvanize more detailed and longitudinal assessment of large groups of endurance athletes,” they wrote. “The potential for such projects is enormous considering the colossal increase in participation rates in endurance events such as the marathon.”

La Gerche is supported by a postgraduate scholarship from the National Health and Medical Research Council in Australia. the project was financed, in part, by a Cardiovascular Lipid Grant from Pfizer Australia.

The authors reported that they had no conflicts of interest.

The editorialists reported that they had no conflicts of interest.