Justin Shopp hopes to raise $25K for Cystic Fibrosis research in 5K walk Saturday

Nine-year-old Justin Shopp of Hampden Twp. is hoping to raise a record $25,000 Saturday in the Cystic Fibrosis Foundation’s “Great Strides” walk in Friendship Park, Mechanicsburg.

Shopp, who was diagnosed cystic fibrosis two years ago, will be walking with his team, “Justin‘s Hope,” which raised $20,000 last year. The 5K walk gets under way at 10 a.m.

To contribute, click here.

Last year, the foundation raised $135 million, with $43 million coming from Great Strides walks across the U.S. in May, which is Cystic Fibrosis Awareness Month.

Justin’s treatment involves taking more than 40 pills a day, and a vest treatment to break up the mucus in his lungs, and two nebulizer breathing treatments twice daily.

Brad Shopp, Justin’s dad, said support from the foundation is key in supporting research into cures for cystic fibrosis. Since it afflicts only 30,000 children and young adults in the U.S. and 70,000 worldwide, Shopp said many pharmaceutical companies are not investing a lot of money into drug research. In Pennsylvania, there are 1,461 with this disease, most of them children, Shopp said.

“We realized early on we had a great partner in the Foundation, as their main goal is to find better treatments and ultimately a cure for this deadly disease,“ Brad Shopp said.

Pharmaceutical research has resulted in a promising drug in clinical trials that could help 85 percent of cystic fibrosis patients, Shopp said.

“Kids who were so sick that they were putting off plans to go to college because they were in the hospital or just too sick to leave the house, are now seeing as much as a 50 percent lung function improvement and it’s literally changing their lives – they’re back to playing sports, continuing their education and enjoying a much improved quality of life,” Shopp said.

“This is what keeps us going and pushes us to raise as much money as we can, knowing CFF is putting these funds towards research and we are so close,” he said, adding 90 cents of every dollar raised, goes directly to research and development.

The Cystic Fibrosis Foundation was established by families in the 1950s when most children with the disease were not living past their fifth birthday, Shopp said.

May 3 run raises money for ‘65 roses’

People are often perplexed by the symbolism of the rose at a cystic fibrosis event.



Holly Shaw and her son, IV, share a moment and a rose. Photo by C.T. Shaw.

The story began in 1965. Mary Weiss, a mother of three young boys with cystic fibrosis, and a passionate volunteer for the Cystic Fibrosis Foundation, was on the phone making calls. Her objective was to reach out to every civic club, service organization, and anyone that would listen to seek financial support for CF research.

After several phone calls, her4-year-old son Richard came into the room and said, “Mom, I know what you are working for.”

After her initial shock, she asked, “What am I working for?”

Young Richard replied, “You’re working for 65 Roses.”

With tears running down her face, Mary nodded “yes” and the term 65 Roses was born. And since that day in 1965, children with cystic fibrosis have used “65 Roses” to describe and pronounce their disease.

Though it may be easier for these children to say, it certainly has not made this disease any easier to live with.

As a CF dad living in Leland, I recently attended the Cystic Fibrosis Foundation’s Volunteer Leadership Conference and had the honor to meet Mary Weiss. What she and many others have accomplished over the past 50 years advocating for the foundation and those with cystic fibrosis has absolutely allowed my 2-year-old son a longer life while living with this disease.

The Cystic Fibrosis Foundation is the world’s leader in the search for a cure of cystic fibrosis. What they have done to change the face of this chronic disease is nothing short of amazing.

However, the backbone of this foundation is its volunteers. Volunteers just like Mary Weiss exist right here in the greater Wilmington area. People who simply will not quit fighting and fundraising for this disease until CF stands for Cure Found.

We invite you to be a part of our group!

Please, consider joining us at this year’s Great Strides Walk & 5K Run, which starts at 9 a.m. Saturday, May 3, on the Event Field at Mayfaire Town Center. Great Strides is our annual celebration of the incredible medical research and progress that has been made toward finding a cure for cystic fibrosis.

To register for this free event, go to Wilmington.GreatStridesNC.com.

To learn more about cystic fibrosis, go to www.cff.org.

To get involved locally, email us at 65RosesILM@gmail.com.

C.T. Shaw and his wife, Holly, are the parents of IV Shaw. IV (he’s the fourth C.T. Shaw) was diagnosed with cystic fibrosis when he was 6 months old. He’ll be 3 years old in July.

Low-dose natural antimicrobial exacerbates chronic lung infection in cystic fibrosis

Respiratory failure caused by chronic lung infection with Pseudomonas aeruginosa bacteria is a common cause of death in patients with cystic fibrosis (CF), a genetic disease that is common in individuals of European descent. A study published on April 24th in PLOS Pathogens demonstrates that an antimicrobial peptide produced by human immune cells can promote mutations in the bacterium that make it more lethal.

Daniel Wozniak, from The Ohio State University Wexner Medical Center, USA, and colleagues studied a process called “mucoid conversion”, which involves mutations in Pseudomonas that produce a sticky sugar coating of the bacteria which makes them more resistant to various treatments. The process is fairly well understood, and involves mutation of a particular Pseudomonas gene called mucA. Searching for factors of the human host that facilitate mucA mutation, the scientists found that specific immune system cells called polymorphonucleocytes (or neutrophils), which are present in large numbers in lung cells of patients with CF, can trigger Pseudomonas mucoid conversion, and that a specific antimicrobial factor produced by these cells called LL-37 plays a key role.

At high doses, LL-37 can kill bacteria by poking large holes into their cell walls. However, at lower concentrations (which seem to mimic the situation in the lungs of CF patients), the scientists found that some LL-37 molecules can enter the bacterial cells without killing them. Once inside, LL-37 appears to be able to directly interact with and alter the bacterial DNA, leading to mutation of the mucA gene. The resulting mucoid conversion makes the sugar-coated bacteria then resistant to higher doses of LL-37, including doses that would readily kill the “naked” Pseudomonas bacteria prior to mucoid conversion.

This shows the proposed model of LL-37 induced mutagenesis and mucoid conversion. Step Ia, at low doses LL-37 (*) interacts with Pseudomonas aeruginosa and enters bacterial cells, binding to DNA (Step Ib). LL-37/DNA interactions promote mucA mutagenesis and conversion to the mucoid phenotype (Step II). Mucoid P. aeruginosa biofilms are now more resistant to killing by lethal levels of LL-37 and are selected for in the CF pulmonary environment (Step III).

(Photo Credit:  Wozniak et. al)

The scientists went on to show that LL-37 can induce mutations besides those in mucA in both Pseudomonas and E. coli, showing that its function as a mutagen is neither restricted to a particular gene nor a particular pathogen.

Taken together, the results demonstrate that an antimicrobial substance can, at low dose, function as a mutagen that makes bacteria more dangerous. Given that antimicrobial peptides similar to LL-37 are being discussed as promising leads for the development of new antibiotics, the scientists say their data “reinforce how important it is to consider the impact of current and novel treatments and the host immune response on evolution of microbial communities during chronic infections.”

FDA Grants Orphan Drug Designation for Bayer’s Investigational Ciprofloxacin DPI (Dry Powder for Inhalation) for Treatment of Non-Cystic Fibrosis Bronchiectasis

WHIPPANY, N.J., April 22, 2014 /PRNewswire/ — Bayer HealthCare today announced that the U.S. Food and Drug Administration’s (FDA) Office of Orphan Products Development has granted orphan drug designation for its investigational Ciprofloxacin Dry Powder for Inhalation (Ciprofloxacin DPI) for the treatment of non-cystic fibrosis bronchiectasis (NCFB). Patients with NCFB suffer from frequent severe acute pulmonary bacterial exacerbations which lead to further inflammation, airway and lung parenchyma damage.

The Orphan Drug Designation program provides orphan status to drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases and disorders that affect fewer than 200,000 people in the U.S, or that affect more than 200,000 but are not expected to recover the costs of developing and marketing a treatment drug.

About Ciprofloxacin DPI Ciprofloxacin DPI is in development by Bayer HealthCare as chronic intermittent therapy for reducing the frequency of acute exacerbations in NCFB patients with bacterial respiratory pathogens. It comprises ciprofloxacin, a fluoroquinolone antibiotic, formulated into dry powder for inhalation using Novartis’ PulmoSphere™ technology and is administered with the T-326 Dry Powder inhaler.  Ciprofloxacin DPI therapy in NCFB is currently being investigated in a global Phase III clinical trial program (RESPIRE; NCT01764841).

About Non-Cystic Fibrosis Bronchiectasis NCFB is a chronic respiratory disease of which up to 50 to 80 percent of cases may be idiopathic.1  A vicious cycle of recurrent microbial infections and persistent inflammation leads to further damage of airway walls and predisposes the lung to new infections.2,3  Bacterial colonization of the airways, most commonly with Haemophilus influenzae and Pseudomonas aeruginosa, is found in more than 60 percent of adult patients with stable bronchiectatic disease.2  In particular, presence of Pseudomonas aeruginosa has been associated with accelerated decline in lung function.1

About Bayer HealthCare Pharmaceuticals Inc. Bayer HealthCare Pharmaceuticals Inc. is the U.S.-based pharmaceuticals business of Bayer HealthCare LLC, a subsidiary of Bayer AG. Bayer HealthCare is one of the world’s leading, innovative companies in the healthcare and medical products industry, and combines the activities of the Animal Health, Consumer Care, Medical Care, and Pharmaceuticals divisions. As a specialty pharmaceutical company, Bayer HealthCare provides products for General Medicine, Hematology, Neurology, Oncology and Women’s Healthcare. The company’s aim is to discover and manufacture products that will improve human health worldwide by diagnosing, preventing and treating diseases.

Forward-Looking Statements This release may contain forward-looking statements based on current assumptions and forecasts made by Bayer Group or subgroup management. Various known and unknown risks, uncertainties and other factors could lead to material differences between the actual future results, financial situation, development or performance of the company and the estimates given here. These factors include those discussed in Bayer’s public reports which are available on the Bayer website at www.bayer.com. The company assumes no liability whatsoever to update these forward-looking statements or to conform them to future events or developments.


  1. O’Donnell AE, “Bronchiectasis,” Chest, no. 134, pp. 815-823, 2008.
  2. King PT, “The pathophysiology of bronchiectasis,” International Journal of COPD, no. 4, pp. 411-419, 2009.
  3. Angrill J, et al., “Bacterial colonization in patients with bronchiectasis: microbiological pattern and risk factors,” Thorax, no. 57, pp. 15-19, 2002.

Intended for U.S. media only


SOURCE  Bayer HealthCare

UVa creates virtual bacteria to shed light on cystic fibrosis infections

The two species of bacteria are genetically similar – both contagious, both drug resistant, both preying upon people with cystic fibrosis or weakened immune systems – yet they go about their sinister work very differently. There is much to be learned both from how they are alike and how they differ, and so researchers at the University of Virginia School of Medicine have recreated them in electronic form, building an elaborate computer model of each so that scientists can better understand them, better compare them and find new and better ways to stop them.

The new metabolic model lets scientists examine Burkholderia cenocepacia and Burkholderia multivorans like never before. Both are multi-drug resistant bacteria that pose a threat to patients with cystic fibrosis, setting up in their lungs and contributing to the rapid deterioration of the lungs and overall health. Once colonized, they are very difficult to eradicate and can contribute to other infections and lead to death.

“For these two particular bugs, there’s not a lot known and they have only recently been appreciated as important pathogens in the cystic fibrosis lung,” said UVA researcher Jason Papin, PhD, whose lab created the new metabolic simulations. “We want to be able to use the models to predict good drug targets, to try to understand why the pathogen behaves the way it does, to understand how it’s going to evolve under pressure of antibiotics. These bugs have a lot of natural antibiotic-resistance mechanisms – you give some antibiotics and the bug adapts and rewires its network in order to evade that drug, so you want to be able to come up with new targets.”

The new computer model will let scientists examine the bacteria’s growth capability, virulence, reactions to antibiotics and much more. The predictions of how the bacteria will behave then serve as a valuable indicator for what work needs to be done in the lab, suggesting productive lines of research and pointing to new drug targets.

“There are a lot of similarities between these bugs, but there are also some notable differences, and it’s not really understood why those differences exist. So we use these models to delineate the functional impact of some of these genetic differences,” said Papin, of the UVA Department of Biomedical Engineering. “Where one enzyme is present in one bug and absent in the other, what are the functional effects of that? Does it make one bug more capable of growing in a particular environment?”

Surprisingly, the goal isn’t for the model always to be right. “The model makes some predictions correctly and some predictions incorrectly,” Papin said. “What’s really neat about computer modeling of these biochemical networks and systems is it’s nice when the models are right but it’s really interesting when they’re wrong, because that helps point to aspects of the biology we don’t understand, which helps to generate new hypotheses, new ideas that can be tested.”

ThThe new model has been described in an article published online and in print by the American Society of Microbiology’s Journal of Bacteriology, appearing as the cover story of the print edition. The article was authored by the School of Medicine’s Jennifer A. Bartell and Phillip Yen, co-lead authors of the paper and builders of the models; John J. Varga and Joanna B. Goldberg of the Emory University School of Medicine in Atlanta; and Papin.

Camryn Koke continues her fight against Cystic fibrosis in Southold

In the basement of the Koke family’s home in Southold, 9-year-old Camryn is allowed to play with her dollhouse for one hour each morning before heading to school.

On a recent day, she held her favorite doll, Raquelle — a pretty fashionista who likes going to the spa and is featured on TV’s “Barbie: Life in the Dreamhouse.”

“She’s active and sassy — like me,” Camryn said with a smile as she smoothed the doll’s hair.

In Camryn’s other hand was a nebulizer. The fourth-grader also wore a vest that provides periodic electronic chest compressions.

Her mother, Jennifer, compared the motion to shaking a can of paint. When the vest is turned on, Camryn sounds like she’s talking through a fan.

Camryn has been wearing this type of vest since she was just 15 months old, and now uses it twice a day for an hour per session. She said she doesn’t mind, though, because it helps her breathe better.

Camryn is living with Cystic fibrosis.

The hereditary chronic disease, the result of a defective gene, causes the body to produce unusually thick, sticky mucus that clogs the lungs and leads to life-threatening lung infections, according to the Cystic Fibrosis Foundation. It also obstructs the pancreas and prevents natural enzymes from helping the body break down and absorb food. Life expectancy for people with CF is anywhere from 30 to 40 years.

Ms. Koke first found out Camryn had Cystic fibrosis during her pregnancy. Blood tests later revealed that she and her husband, Michael, both carry the gene that causes it.  The Kokes’ three younger children — Mackenzie, 8, Logan, 6, and Peyton, 4 — were conceived through in vitro fertilization.

Camryn has been fighting Cystic fibrosis her whole life, but she hasn’t been doing it alone.

The community has rallied support for her by participating in various fundraisers to benefit the Cystic Fibrosis Foundation, one of the few organizations that funds medical research to find a cure.

On Friday, April 25, Southold High School will host its fourth and final annual Cystic fibrosis fundraiser in Camryn’s honor. Unlike previous years, the event will feature more family-friendly activities, including carnival games, Zumba by Cutchogue fitness studio JABS and a magic/comedy show by Jester Jim.

Ms. Koke said she recently decided that this year’s fundraiser would be the biggest to date — and that in the future she will concentrate on other fundraising approaches.

Ms. Koke said her friend Jen Olsen has always played an integral part in organizing CF fundraising efforts — and introduced her to JABS owner Jill Schroeder.

In addition to organizing the school fundraisers, a group of supporters participates in the Cystic Fibrosis Foundation’s annual Great Strides benefit walk in Westhampton Beach. And Coastal Pipeline in Calverton — where Michael Koke is employed — created “Cure for Cammy” T-shirts and donates proceeds from sales to the foundation.

Last week, Camryn learned that her request to meet Buddy Valastro, star of TLC reality show “Cake Boss,” had been granted by the Make a Wish Foundation. She also found out that the Long Island Children’s Choir will perform a song composed specifically for her at the May 3 Long Island Ducks game at Bethpage Ballpark. The musical group works with songwriters nationwide to compose customized songs for children with serious illnesses, based on facts about their lives. Camryn’s song is expected to be available for download on iTunes.

Although there is no cure for CF, the Kokes continue to hope. Ms. Koke said Camryn is one of the foundation’s top fundraisers and has raised more than $100,000 overall.

“We wouldn’t be here if it wasn’t for the community,” Ms. Koke said. “I think they’ll always be behind her.”

For more information about the April 25 event, visit the 4th Annual Fundraiser for CFF Honoring Camryn Koke Facebook page.


Transplants give Garden Plain man a second chance at life

Chris Simon’s bucket list wasn’t anything fancy.

He simply wanted to be able to ride a bike again. Or take a swim. Or go camping with friends.

“Stuff I used to do all the time,” Simon said. “And all of a sudden it was taken away.”

Those once simple joys were stolen by cystic fibrosis, the same disease that claimed his younger sister Emily’s life in 2009.

The Simon family on Sunday will join millions of others around the world in celebrating Easter, a day symbolizing new beginnings. The holiday will have special significance for Chris, 26, who has another chapter dawning in his life after a double-lung/liver transplant several weeks ago.

“It’s been a long ride,” he said.

Cystic fibrosis is a genetic disease that attacks organs in the body – most commonly the lungs. Two of Ron and Sarah Simon’s three children were born with it.

Emily chose not to have the double-lung transplant she needed to extend her life. She died at the age of 19 in 2009.

After Chris was hospitalized twice in his senior year at Kansas State University, the disease seemed to go dormant for about a year. As medical tests showed his lungs were slowly failing, however, his doctor delivered painful news:

If he wanted to live, he would need a lung transplant.

A double blow

But that wasn’t all. The cystic fibrosis had attacked his liver as well.

“Liver disease as a result of cystic fibrosis is not unusual,” said Dr. Ramsey Hachem, medical director of the lung transplant program at Barnes-Jewish Hospital in St. Louis, who performed Chris’ transplant. “In a small number of patients, they can develop end-stage liver disease.”

In order to extend his life, Chris would need a double-lung and liver transplant – an operation performed only a handful of times a year in the United States.

He went on a waiting list in March 2013 and moved to St. Louis. He needed to be within an hour of Barnes-Jewish Hospital in case the call came.

His mother, Sarah, quit her job so she could be with him. Chris tried to keep life as normal as possible, including going to several baseball games when he was able to afford tickets.

His beloved Boston Red Sox even reached the World Series against the Cardinals. He yearned to get to a game when the series shifted to St. Louis.

His father has health insurance through his job, but the family was relying on money raised through donations and a variety of fundraisers put on by folks back in their hometown of Garden Plain to pay for rent, food and other living expenses.

But Chris couldn’t bring himself to spend several hundred dollars for a ticket to the series. Considering it would come from people helping out the family in its time of need, he said, “I couldn’t justify spending that kind of money.”

Instead, he and his younger brother Dustin watched the games on television and soaked up the electricity of the World Series.

His Red Sox won.

“That,” he said, “was really cool.”

The call comes

He became ill in early February and had to be hospitalized. At one point, he was placed on a ventilator. He would not be released for nearly two weeks.

“We spent the night at home,” his mother said. “We got the call the next day.”

They were told to return to the hospital for his transplant. The operation was performed the next day, on Feb. 19.

During a routine medical procedure the day after the transplant, an air bubble went to Chris’ brain.

“We get this call saying he may have had a stroke,” Sarah Simon said. “There’s a major change in his mental status. My husband went in there and he didn’t really know him or know of him.”

Chris was airlifted to another hospital in St. Louis and placed in a hyperbaric chamber for six hours.

“I only remember bits and pieces of it,” Chris said. “That was probably the scariest part of the ordeal. I didn’t understand what was going on.

“I assumed the worst.”

Aided by the hyperbaric chamber, Chris’ body absorbed the air bubble. His recovery resumed and accelerated.

“I remember my wife saying, ‘Everything is going so good. When is the bad going to happen?’ ” Ron Simon said.

The answer came within a matter of hours. Chris was about to be released from the hospital when he began complaining of shortness of breath.

A fresh round of X-rays led to a bronchoscopy, which revealed one of his new lungs was failing. He would need another transplant to survive.

“I was so upset” at the doctors, Sarah Simon said. “I said, ‘You just told me that he could go home!’ 

Looking back, family members now say, leaving the hospital would likely have cost Chris his life.

The second transplant

The second double-lung transplant, on March 8, was so hard on Chris’ body that he nearly died anyway.

One nurse later told Chris that, after seeing him the day after his second transplant, “I didn’t think you were going to make it.”

His mother wasn’t sure he’d survive, either.

“I went in after all this happened and I told him, ‘I love you so much. If you don‘t want to go through with this, I totally understand,’ ” she said.

It was her way of giving her son permission to let go.

But Chris didn’t want to die.

“I’m gonna fight,” he told himself on those dark days after the second transplant.

To boost Chris’ spirits, a nurse suggested the family bring Cooper, his corgi dog, into his room at the ICU.

“He was so down, he was in such pain,” Sarah said of her son.

They brought Cooper in perhaps three times, she said.

“It really made a big difference, I’m telling you,” Sarah Simon said. “They were calling him the miracle dog.”

Knowing he loved Kansas State, nurses at the hospital made him a K-State blanket and brought it to his room, Sarah Simon said. An X-ray tech who shares Chris’ love of comics brought him comic books to read.

‘She’d be proud of me’

Chris admitted he was surprised by how weak he was after the second transplant.

“I had to relearn to walk,” he said. “I had to relearn pretty much everything. I felt like a little baby, starting fresh.”

Chris said he thought about Emily often during his ordeal.

“I think she’d be proud of me,” he said.

Sarah Simon said her daughter initially decided not to have a transplant, but later changed her mind. By then, however, it was too late.

“I wasn’t even aware that she declined it until about a year ago,” Chris said. “I just couldn’t understand why she wouldn’t want to extend her life.

“After going through it, I completely understand why you wouldn’t want to. It’s not what I thought it was going to be.”

Chris began to stabilize and gain strength. But progress came slowly, leaving him frustrated.

“I had to remind him, ‘You were run over a truck, and then they backed over you again,’ ” Sarah Simon said, referring to the transplants.


Hachem said Chris’ bumpy road to recovery is no surprise.

“These were big operations that he had and it’s hard to recover from any one of those operations – never mind multiple,” Hachem said. “I think he’s coming along OK and slowly heading in the right direction, which is really what we all want.”

Small steps represent major milestones.

About a month after his second operation, Chris went for a walk in the hospital. It was his first walk without being attached to an oxygen canister in more than two years.

“Just getting rid of that feels amazing in itself,” he said. “It’s been a long while.”

He couldn’t go anywhere in recent years without a portable oxygen container tucked in a book bag that he slung over his back.

“Every time I went outside the house, I had to take that backpack with me,” he said. “It just feels so good to walk around without that on my back. It’s literally a weight off my back.”

Chris has to remain in St. Louis for three months of pulmonary rehabilitation before he can return to Garden Plain. But he’ll come home on the occasional weekend for special events – including watching Dustin graduate from Garden Plain High School next month.

Dustin talks of going into medicine.

Seeing his brother and sister battle cystic fibrosis and what doctors did to help them “inspired him,” Chris said.

Saying ‘thank you’

Chris and his family are grateful for the residents of Garden Plain who held special events to raise money for the family’s expenses.

Chris calls the support from folks back home “insane.”

“Friends I haven’t heard from in forever, people I don’t even know constantly asking how I’m doing,” he said. “People sending donations or food (to family) back home. People saying prayers or asking if there’s any way they can help.

“It’s been humbling.”

Saying “thank you” for all that support somehow doesn’t seem like enough, said Ron Simon, his father.

“I don’t know what to do to make them realize how much we do appreciate this,” Ron Simon said. “I don’t think there’s any way you could ever repay them.”

There is one particular item on Chris’ list of things to do that he already has his eyes on.

He’s always wanted to go to a game at Fenway Park, home of his beloved Red Sox. He has an aunt whose brother lives in Boston, he said, and she’s told him, “As soon as you get those lungs, we’re going out there.”

There’s no telling how long this bonus chapter of his life will last, but Chris said he intends to make the most of it.

Along with enjoying those simple pleasures taken from him by cystic fibrosis, there’s something else he’ll have to do from now on.

He’ll need to celebrate two birthdays each year, transplant nurses told him and his family: the day he was born – Oct. 17 – and the day he received his lung transplant – March 8.

“They call that his second birthday,” his father said.

It was the day he received new life.

Reach Stan Finger

Read more here: http://www.kansas.com/2014/04/19/3413112/transplants-give-garden-plain.html#storylink=cpy

Alcresta Announces Agreement with Cystic Fibrosis Foundation Therapeutics to Develop Point-of-Care Nutritional Products

NEWTON, Mass., Apr 17, 2014 (BUSINESS WIRE) — Alcresta®, a leading medical nutrition company developing       innovative enzyme-based products for individuals with unique nutritional needs battling acute conditions or chronic diseases, today announced the       company has signed an agreement with Cystic Fibrosis Foundation Therapeutics (CFFT) to accelerate the development of Alcresta’s       enzyme-based point-of-care products to support the nutritional status of people with cystic fibrosis (CF). CFFT is the nonprofit drug discovery  and development affiliate of the Cystic Fibrosis Foundation.

More than 90 percent of people with CF have a compromised pancreas and       need enzyme-based nutritional products to digest and absorb vitamins and  nutrients. To address these challenges, people with compromised  pancreatic or gastrointestinal function, including people with CF, consume nutritional drinks and use tube feedings to supplement their  diet. This agreement allows Alcresta to accelerate the development of  its nutrition technology and advance point-of-care products designed to address the specific needs of the CF population and others with unique nutritional needs by improving absorption of beneficial fatty acids and aiding in overall caloric intake.

According to the CF Foundation’s patient data report, a relationship has  been established in CF between good health outcomes and patients’ nutritional levels, reinforcing the need for individuals to maintain adequate weight gain (BMI). Specifically, malabsorption of fats may contribute to the inflammatory characteristics of CF, and negatively  affect a person’s ability to maintain or gain weight.

“This agreement continues to validate Alcresta’s technology and will allow us to speed up the development of our point-of-care portfolio of  products that we believe will improve the overall well-being of people  with CF and others with specific nutritional needs,” said Robert  Gallotto, co-founder and president of Alcresta. “We believe science-based nutrition can lead to improved care, and we look forward  to working with the CF Foundation to help make a meaningful difference  for people in need of improved nutrition.”

Alcresta’s lead program is an enzyme-based point-of-care product focused  on improving the digestion and enabling proper absorption of fats, including critically important “good fats” – in particular omega-3 fatty  acids like docosahexaenoic acid (DHA) and eicosapentaenoic acid (EPA).       The initial product offering is designed to mimic the function of pancreatic lipase by pre-hydrolyzing fats in liquid nutritional products and tube feeding formulas to fatty acids and mono-glycerides prior to consumption in individuals who require special nutritional care where there is a known relationship between proper nutrition, caloric intake and weight maintenance.

About the Cystic Fibrosis Foundation

The Cystic Fibrosis Foundation is the world’s leader in the search for a cure for cystic fibrosis. The Foundation funds more CF research than any other organization, and nearly every CF drug available today was made  possible because of Foundation support. We are a nonprofit donor-supported organization dedicated to attacking cystic fibrosis from every angle. Our focus is to support the development of new drugs to  fight the disease, improve the quality of life for those with CF, and ultimately to find a cure. Based in Bethesda, Md., the Foundation funds and accredits a national care center network that has been recognized by       the National Institutes of Health as a model of care for a chronic disease. For more information, visit         www.cff.org .

About Alcresta

Alcresta is a leading medical nutrition company developing and       commercializing innovative enzyme-based point-of-care products to       improve the digestion and absorption of key nutrients to support growth,       development and nutrition in people battling acute conditions or chronic       diseases. The demand for high-value nutritional products is increasing,       driving the growth of a $40 billion global market. While there have been       important recent advances in nutritional drinks and products, there       remains a significant need for enzyme-based nutritional products that       support healthier living, maximize nutrient and caloric absorption, and       complement conventional medical care. Leveraging its existing       proprietary platform of point-of-care nutritional products, Alcresta has       developed a lead product designed to improve the digestion and       absorption of fats, in particular long-chain polyunsaturated fatty acids       like omega-3 (including DHA, EPA). The importance of long-chain       polyunsaturated fatty acids like omega-3 is well documented across the       full spectrum of patient care from infants to adults and individuals       battling acute conditions or chronic diseases. The company’s platform is       supported by the Alcresta team’s extensive experience in pharmaceutical       and nutritional product development. Based in Newton, Mass., the company       is backed by top-tier venture investors Bessemer Venture Partners,       Frazier Healthcare and Third Rock Ventures. For more information, please       visit         www.alcresta.com .

SOURCE: Alcresta

Non-Cystic Fibrosis Bronchiectasis Patients Gain Mixed Results From Exercise, According To Study

Individuals with non-cystic fibrosis bronchiectasis are recommended to take on exercise training, however, the longer-term effects and benefits remain uncertain, according to researchers.  A new randomized controlled study was performed to determine the effects of exercise training and review of airway clearance therapy (ACT) on exercise capacity, quality of life and the incidence of acute exacerbations in individuals with non-cystic fibrosis (CF) bronchiectasis.

85 trial participants (mean FEV1 74% predicted; median Modified Medical Research Council Dyspnea grade of 1 (IQR [1-3]) were randomly allocated to eight weeks of supervised training and review of ACT or control.  Baseline measurements included exercise capacity and HRQOL (Chronic respiratory disease questionnaire) and secondary outcomes of cough-related QOL (Leicester cough questionnaire), and psychological symptoms (Hospital anxiety and depression scale) as primary outcomes, following completion of the intervention period and at 6 and 12 months.  Exacerbation rate and time to first exacerbation were analyzed over 12 months as secondary outcomes.

The researchers report that exercise training increased the incremental shuttle walk distance (mean difference to control 62 m, 95% CI 24 to 101 m) and the 6-minute walking distance (mean difference to control 41 m, 95% CI 19 to 63 m). However, these improvements were not sustained at 6 or 12 months. Exercise training reduced dyspnea (p = 0.009) and fatigue (p = 0.01) but did not impact on cough-related QOL or mood.

Exercise training decreased the frequency of acute exacerbations (median 1[IQR 1-3]) compared to the control group (2[1-3]) over 12 months follow up (p = 0.012), with a longer time to first exacerbation with exercise training of 8 months (95% CI 7 to 9 months) compared to the control group (6 months [95% CI 5 to 7 months], p = 0.047).  The researchers conclude that exercise training in patients with non-cystic fibrosis (CF) bronchiectasis is linked with short-term improvement in exercise capacity, dyspnea and fatigue, and decreased exacerbations over twelve months. However, whether or not exercise is a long-term benefit to these patients remains in question.

AbbVie today announced that undergraduate and graduate students living with cystic fibrosis (CF) can now apply for the 2014 AbbVie CF Scholarship, which honors young adults with CF as they pursue higher education. Students can apply for the scholarship from now until Monday, June 9, at www.AbbVieCFScholarship.com .

NORTH CHICAGO, Ill., April 16, 2014 /PRNewswire/ — AbbVie today announced that undergraduate and graduate students living with cystic fibrosis (CF) can now apply for the 2014 AbbVie CF Scholarship, which honors young adults with CF as they pursue higher education. Students can apply for the scholarship from now until Monday, June 9, at         www.AbbVieCFScholarship.com .

The AbbVie CF Scholarship acknowledges students with CF who demonstrate creativity, academic excellence, community involvement and the ability to serve as a positive role model for the CF community. In honor of the 22nd year of the program, 40 AbbVie CF Scholarship recipients will have the opportunity to compete for public votes to win a Thriving Student Scholarship (based on level of study) for a total award of $22,000.

“AbbVie is honored to continue the legacy of recognizing the accomplishments of exceptional students living with CF,” said Jim Hynd, vice president, Endocrinology; Metabolic; GI Care and Institutional Accounts, AbbVie. “The scholars serve as ambassadors for the CF community, inspiring others to achieve their individual personal and academic goals.”

Cystic fibrosis is an inherited chronic disease that affects the lungs and digestive systems of approximately 30,000 children and adults in the United States. Today, more than 45 percent of people living with CF are aged 18 or older.

About the AbbVie CF Scholarship The AbbVie CF Scholarship was established 22 years ago in recognition of the financial burdens many families touched by CF face and to acknowledge the achievements of students with CF. The program has since awarded more than $2.4 million in scholarships. It is not necessary for scholarship applicants to have taken, currently take, or intend to take in the future, any medicine or product marketed by AbbVie, and this is not a consideration in the selection criteria. More information about the AbbVie CF Scholarship criteria and application can be found at         www.AbbVieCFScholarship.com .

About AbbVie AbbVie is a global, research-based biopharmaceutical company formed in 2013 following separation from Abbott Laboratories. The company’s mission is to use its expertise, dedicated people and unique approach to innovation to develop and market advanced therapies that address some of the world’s most complex and serious diseases. AbbVie employs approximately 25,000 people worldwide and markets medicines in more than 170 countries. For further information on the company and its people, portfolio and commitments, please visit         www.abbvie.com . Follow         @abbvie on Twitter or view careers on our         Facebook  or         LinkedIn page.