£11 million-a-year US pharmaceutical boss is accused of trying to ‘blackmail’ Theresa May into buying cystic fibrosis drug

By Stephen Adams and Glen Owen

The boss of a US pharmaceutical giant was last night accused of trying to ‘blackmail’ Theresa May into buying a groundbreaking drug at an eye-watering annual cost of £100,000 per patient.

Thousands of people with the deadly lung condition cystic fibrosis (CF) have been pleading with NHS bosses to strike a deal with Vertex over Orkambi, a new treatment that could extend their lives by years.

The talks collapsed acrimoniously ten days ago, after which Vertex boss Dr. Jeff Leiden wrote to the Prime Minister threatening to scale back the firm’s investment in the UK unless NHS England agreed to buy Orkambi on its terms.

In an extraordinary outburst, Dr. Leiden accused Britain of placing ‘a lower value on the life of a CF patient than other countries’ which have purchased Orkambi, and warned that British jobs relied on his benevolence.
The row comes amid growing anger among health chiefs about how the ‘profiteering’ of drugs companies is heaping pressure on the NHS budget.

The starting price for Orkambi negotiations is thought to be £104,000 per patient per year.

Tory MP Bob Stewart said: ‘Drug companies have every right to turn a profit to fund their vital research and development work, but it is completely immoral to overcharge for a life-saving drug. If the CEO is trying to blackmail the Prime Minister that is utterly disgraceful.’

In his letter to Mrs. May, Dr. Leiden, who received £11 million in salary and stock options last year, wrote: ‘Vertex is heavily committed to the UK with its international HQ in London, an R&D site near Oxford and runs many clinical trials in the NHS. Since 2006, Vertex has invested over £1.2 billion in the UK and now employs 250 people, of whom over 110 are scientists.’

But he added: ‘We are questioning this ecosystem as [the UK] is unable to value life-changing medicines for the patients that need them.’ Without major changes to how new drugs were assessed, he pointedly warned that ‘any future biotech investment in the UK is at significant risk’.

In 2014, Dr. Leiden, who owns a string of waterfront homes in Massachusetts and Florida, faced a shareholder revolt over his ‘exorbitant’ pay package of almost £35 million. Orkambi is the only current therapy that slows the inevitable progression of CF. It could benefit up to half of the 10,500 Britons with the condition.

CF causes lungs to produce too much mucus, reducing their effectiveness. Around half of sufferers die before the age of 47, but Orkambi has been shown to boost lung function by 42 percent.

Catherine Upstone, from Brackley, Northamptonshire, whose daughter Cerys, 14, is one of those who could benefit from Orkambi, said: ‘The two sides need to get back around the table.’

But NHS England boss Simon Stevens recently told MPs that Vertex must first drop its price.

‘Right now, the company is a very long way from acting responsibly,’ he said. ‘The implication of the kinds of prices that are being talked about would either be to deny patients those drugs or to rip off British taxpayers, and neither of those situations is acceptable.’

Vertex’s chief commercial officer Stuart Arbuckle said the firm had made NHS England ‘the best offer in the world, which provides all eligible patients immediate access to our current and future medicines. However, the NHS wants our existing medicines and those coming down the line in the next year for no extra money – this is essentially asking for our medicines for free.’

NHS England said: ‘If Vertex believes they are offering a reasonable deal, they should waive their confidentiality clause and let patients and taxpayers judge whether it is fair.’

Original article here.

Omega-3 Compound Reduces Inflammation in Cystic Fibrosis Patients in New Pilot Study

By Jennifer Prince

A marine omega-3 compound comprising a docosahexaenoic acid (DHA) sn1-monoacylglyceride (MAG-DHA) may act as an anti-inflammatory for subjects with cystic fibrosis, according to a new pilot study1 published in the journal Marine Drugs. In the study, MaxSimil (Neptune Wellness Solutions; Laval, QC, Canada) increased omega-3 red blood cell levels, helped moderate the ratio of arachidonic acid (AA) to docosahexaenoic acid, and reduced key inflammatory biomarkers in subjects with cystic fibrosis. Continue reading Omega-3 Compound Reduces Inflammation in Cystic Fibrosis Patients in New Pilot Study

Vertex Pharmaceuticals opens expanded San Diego research center with focus on cystic fibrosis

By Bradley J. Fikes

Vertex Pharmaceuticals opened its new San Diego research center Monday, starting a new chapter in a decades-long quest to not only treat but cure cystic fibrosis.

In 18 years, three drugs for the lung-ravaging disease have emerged from Vertex’s San Diego center and more are in the pipeline.

The first, Kalydeco, was approved in 2012. It is the first drug that treats the underlying cause of the disease. The second, Orkambi, was approved three years later. And the third, Symdeko, was approved in February.

These drugs can benefit about half of all patients with the incurable disease. In the next several years, Boston-based Vertex hopes its drugs can help nearly all patients live longer, healthier lives.

Cystic fibrosis is caused by a genetic defect that allows a buildup of thick mucus in the lungs, and other internal organs. This mucus clogs airways and promotes the growth of bacteria. The average lifespan of patients is 37 years, up from 20 years in 1980. Treatments include antibiotics to fight lung infections and mucus-thinning drugs.

The new 170,000 square-foot building on Torrey Pines Mesa more than doubles the company’s space. The center includes cell culturing equipment to grow lung cells from patients, to be used for drug screening. A 4,000 square-foot incubator suite will serve outside collaborators.

Asides from cystic fibrosis, the staff will work on other serious diseases.

Among the speakers Monday morning was a veteran in the fight against cystic fibrosis: Jennifer Ferguson, who has two children with the disease, Ashton and Lola. Both her children are taking Vertex drugs, and both were present with her at the event.

With these drugs and the promise of better therapies ahead, she says Ashton and Lola have a good chance of growing up and leading their own lives. She urged all Vertex employees to think of themselves as part of a team to cure the disease.

Ferguson, of San Diego, found out about the work from the Cystic Fibrosis Foundation. The foundation had invested $30 million in startup Aurora Biosciences to find therapies.

In 2001, Vertex purchased Aurora for $592 million in stock, the same year Ashton was diagnosed. The research went on under Vertex, and Ferguson became quite familiar with the research team.

“The Cystic Fibrosis Foundation asked me to come speak, to show them what it’s like to have a little child with CF,” she said. “So I came here about 17 years ago with him as a 6-month-old.”

At that time, many cystic fibrosis patients never reached adulthood.

“I had a hard time keeping it together,” Ferguson told the audience of that long-ago visit.

“But I looked in the staff’s faces — and some of you are still here — and I thought, I’m going to put my faith and trust in your hands, in your brains. And I was able to let go of my worry, because you were on the case.”

Ferguson started visiting every few years to check on what progress was being made, first with Ashton, and later including Lola. She also raises money for the Cystic Fibrosis Foundation.

Both her children have shown improvement since starting the Vertex drugs, Ferguson said. But they still need to go through a daily regimen of clearing out their lungs.

From medications, the research frontier has advanced to investigations into a cure. That means fixing the genetic defect, which can come in several variations, inside living patients.

That cure might come from the hot new gene editing technology called CRISR. In 2015, Vertex allied with startup CRISPR Therapeutics to develop curative therapies.

This post was originally published on The San Diego Union-Tribune

Broadway’s biggest stars come together to raise money for Cystic Fibrosis

What do you get when Broadway’s biggest stars, such as Javier Muñoz and Gideon Glick, come together in the recording studio? One heck of a song. And one heck of a message.

Joined by Broadway veterans Laura Osnes, Christy Altomare and actress Sarah Levy, Muñoz and Glick have banded together for a new campaign — called the “Anyway” campaign — for an original song to help raise money for Emily’s Entourage.

At the center of the Entourage is Emily Kramer-Golinkoff: a 33-year-old daughter, sister and granddaughter who was diagnosed with Cystic Fibrosis when she was just a few weeks old. Her parents, Liza and Michael, have done everything in their power to raise Emily as if she was a normal child.

As the fatal disease only affects 70,000 people worldwide, funding for a cure is limited. Emily and her family are even more restrained by her specific mutation, which means medical funding is even rarer. And with a life expectancy of only 35-37 years for her kind of CF, time’s ticking.

Six years after the Kramer-Golinkoffs decided to take matters into their own hands, they’ve raised $3 million to drive high-impact research and speed up breakthroughs to research not only Emily’s mutation, but many other diseases including muscular dystrophy, inherited blood disorders and certain cancers.

They’ve also built a network of family, friends, and strangers from around the world, all of whom have been welcomed into the Entourage. Each Entourage member has been more inspired by Emily’s story than the next.

Take Elizabeth Phillipson-Weiner, from Emily’s hometown of Philadelphia, Pennsylvania and composer of “Anyway.” She took a simple journal entry from a songwriting retreat, turning lyrics like “when the going gets tough I ask questions” into a prolific melody.

Elizabeth explained to AOL Lifestyle. “The song wasn’t just cathartic for me, but could actually ring true for a lot of other people. I immediately thought of Emily.” Working with co-writers, producers and engineers, that melody was soon turned into a demo.

But as inspired as Elizabeth was by Emily’s story, the Entourage required “star power” to bring this project alive.

“I cold contacted agents and managers, I asked friends and friends of friends and friends of friends of friends,” said Elizabeth. “We did hear ‘no’ quite a bit, but whenever I became discouraged or frustrated I thought about who and what I was doing this for.”

This story was originally published on AOL.com

Surviving Home IVs As a Mom

By Janeil Whitworth

I’m no stranger to home IV antibiotics.

Actually, if I am being completely honest, I think we have gotten a little too friendly over the past decade or so. I prefer to do home IVs because I am one of those patients who goes a little nuts after being trapped in the hospital for too long. Seclusion and unlimited access to cable are not beneficial to my physical or mental well-being. I need the opportunity to be with people just as much as I need the comfort of my own bed to rest and heal completely. I’ve even gone as far as learning to access my own port-a-cath to create even further distance between the hospital and me. Plus, I enjoy the luxuries of home IVs such as creating my own schedule, eating my own food, using my own toilet paper, and coming and going as I please.

Releasing control

I’m positive I’m not the only one who struggles to release control over my disease while experiencing an exacerbation. Feeling the façade of freedom slipping away post-IV-talk, I involuntarily tighten my grip on my independence in a desperate attempt at normalcy.

In-patient admission? No way. Home IVs? Ok, I can do that. Thankfully, you can travel on home IVs, celebrate your bachelorette party on home IVs, and even graduate college on home IVs. But is it possible to successfully care for a 9-month-old baby on home IVs?

After the past few weeks of increased shortness of breath and unwavering fatigue, I agreed it was time for IVs. The reality of home IVs as a mom suddenly hit me as I exhaustedly said for the millionth time that day, “Please, don’t eat mommy’s IVs, buddy.”

I knew this was not going to be easy with a mobile, energetic baby crawling every which way, leaving a trail of alcohol pads in his path. (I think this might be the fatigue talking, but the crunchy hospital bed and one-ply toilet paper are starting to seem a little better right now.)

The last time I did home IVs, my son was 4 months old, so it made it a tad easier as he was just a chubby and adorable blob. I would set him down, hook myself up to the antibiotic-filled Eclipse ball, and he wouldn’t move. Those were the days.

There’s been very little rest this week, and admittedly, I tried my hardest to do it all even if I was feeling absolutely poisoned. In my defense, I felt my independence as a mom was slipping away once again, and the impulse to hold on tighter completely crept up on me. This way of thinking will not benefit me in the long run, neither as a mother, nor as a patient. A week has passed since I accessed my port to begin treatment, and I can see more clearly now that I am in desperate need of a different rhythm.

Finding a different rhythm

I am going to attempt to take my own advice for the remainder of treatment, including: 1) Ask for help; 2) Be easy on myself when it comes to everything else; 3) Remain grateful for the privilege of staying home. My normalcy in the coming weeks will look a little different. My parenting style will be altered as well. I need to accept that this is the price for staying home with my baby, while simultaneously being the nurse and patient. Everything needs to be taken in stride as I release my grasp and let the rest, antibiotics, and baby hugs heal me. I think this is the key to surviving home IVs with my health, sanity, and pride intact. I hope.

This blog was originally published on CF News Today.

Jerry Cahill’s CF Podcast: Stem Cell Research with Dr. Hans-Willem Snoeck

In this feature of The Path Forward with CF series, Dr. Hans-Willem Snoeck, Professor of Medicine (Microbiology and Immunology) at CUMC, sits down to discuss stem cell research as it relates to CF.

Because lung cells regenerate and repair themselves regularly, researchers believe that – some day – stem cell technology could be a one-time therapy to cure cystic fibrosis. Research is ongoing, but in the meantime, scientists can currently use human pluripotent stem cells to create lung organoids (tiny, 3-D structures that mimic features of a full-sized lung), introduce various mutations, and apply technologies to learn more about those mutations’ characteristics.

This video was originally published on JerryCahill.com

Vertex Employees Donate $1M to CF and Other Communities via Matching Gift Program

By Carolina Henriques

Vertex Pharmaceuticals employees have raised more million $1 million  using  the Vertex Foundation‘s matching gift program in a show of commitment to causes that include the cystic fibrosis (CF) community, a company press release states.

The dollar-for-dollar matching gift program is being run through the nonprofit Vertex Foundation, established by the company in November 2017 as part of it’s charitable giving goal of donating $500 million to qualified nonprofits and other causes worldwide over 10 years.

To date, more than 500 Vertex employees have used the program to support 753 charities around the globe working to advance work in areas that include healthcare, human services, education, and disaster relief.

Vertex’s charitable commitment has four primary goals: supporting CF patients and caregivers worldwide, including enabling access to Vertex’s medicines; helping underserved students and young women with STEAM (science, technology, engineering, arts and math) education; supporting young doctors and scientists; and strengthening and fostering innovation in local communities through health and wellness programs.

“Giving back is in our DNA at Vertex, and our employees have a long history of going the extra mile to improve the lives of patients, students and their neighbors,” Jeffrey Leiden, president, chairman and chief executive officer of Vertex, said in the release. “I’m proud that The Vertex Foundation is able to help extend the impact of our employees’ giving and look forward to seeing the reach of these investments in the causes they care about most.”

Also as part of its 10-year commitment, Vertex awarded $400,000 in scholarships to eligible CF patients and their family members in May as part of its second “All in for CF” scholarship program. In total, 80 scholarships worth $5,000 each were awarded for the upcoming academic year.

Vertex, which specializes in cystic fibrosis, has three approved CF therapies: Kalydeco (ivacaftor), Orkambi (lumacaftor/ivacaftor), and Symdeko (tezacaftor/ivacaftor).

The company is also testing potential triple combination treatments for CF.

For the rest of this article, click here.

How to Beat Brain Fog

By Wendy Caroline

As I sat down to write my column for this week, a massive wave of writer’s block hit me. This has been plaguing me for a while. Great timing, right?!

I started five different column pieces, each of which I would get a solid start on, and then nothing. I stopped understanding where the piece was going and didn’t like any of the words that I was writing. A few years ago, I started developing chronic fatigue. It started slowly, then quickly snowballed. In the past year, it has developed into brain fog. Very frustrating. I love using my brain for writing, reading, problem-solving, and critical thinking. It increasingly feels like cystic fibrosis is taking all these parts of my personality away from me. So here I am today, compiling a list of some tips and tricks that I use to combat brain fog and feel a little more like myself.

1. Don’t force it.

I can’t tell you how many times I have blocked my own brain by trying to force my way through brain fog. “Just do it.” How many times have we heard that? Unfortunately, at least for me, if I “just do it” and try to force myself to work through the brain fog, it just puts up more blocks. Then I get frustrated, and all I can think about is how I can’t think. It just spirals from there.

Cool, so how do you get past brain fog and still make your deadlines (because if you’re forcing it, that’s often why)? Be ahead of the game. I am guilty of this not being a natural instinct. In fact, I’m still learning how to do this. I used to be a procrastinator. I would thrive under the stress. My brain fog does not. Now I start writing and drafting at least a week before.

2. Get inspired.

One of the best ways to get inspired is to look to others. CF News Today columnist Brad Dell’s latest piece about brain fog directly inspired this post. It got me thinking about my own experience with brain fog, and how I have been insanely frustrated by its hold on my life. Find a piece of other people’s lives or the world around you that makes you think about your own. Capture it and go. I find that making a note of these inspirations in a note-taking app on my phone really helps because brain fog causes forgetfulness.

2. Create the mood.

Ditch the phone and whatever other fancy gizmos that cause a distraction. Find the type of environment that makes you feel inspired and ready to work. For me, that’s open space with lots of natural light and structured seating.

Next, put on some music — music that is going to make you focus and productive. My go-to is this Classical Essentials playlist on shuffle. This helps me drown out enough thoughts that I won’t get distracted without drowning out the thoughts that I need to work. Whatever you listen to, just make sure it doesn’t have words.

It’s also important to get your brain in the right mood. My brain fixates on chores and tasks that need doing, so I always make sure to get those done before I sit down to write. Also yoga and meditation, that’s some great stuff.

4. Let it flow.

A friend of mine gave me this very useful tip. Just let the words flow out of you, even if you don’t like them. When I use this technique, I have to write everything my heart desires and then walk away. Don’t reread it right then. You will get frustrated. Instead, write all you can and come back to it after you’ve had a solid break. For me, this is usually the next day. Your rough work can have a really good base but just needs to be re-worked.

I know that these tips and tricks are focused on writing, but I use the general ideas in all aspects of my life. Whether it is work, family, friends, or hobbies, brain fog can affect every aspect of your life. It’s one of the reasons that I haven’t been very active on any of my social media platforms — Instagram, YouTube, Facebook, and even my blog. As my brain fog developed into almost a daily issue, I took a step back and prioritized what I needed to focus on first. I truly believe that as these steps of beating brain fog become a habit, I will be able to handle more. Hopefully, you’ll see me around the internet again real soon.

» Find me on the internet at The Living, Breathing Wendy «

This article was originally published on CF News Today.

This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition.

Jerry Cahill’s CF Podcast: The Pre-Transplant Process with Dr. Emily DiMango

The latest video in The Path Forward with Cystic Fibrosis series, Dr. Emily DiMango, Director of the Gunnar Esiason Adult CF Program at Columbia University Medical Center, discusses the lung transplant process through the lens of a CF doctor.

First, she reviews the importance of CF patients participating in drug trials in order to start life-changing medications sooner. She then answers the following questions:

· What does pre-transplant management look like for a CF patient?
· When is the right time to be referred to the list?
· What is the referral process like?

Finally, she reiterates the importance of well-rounded treatment that includes physical health, nutritional health, and emotional health.

This video was originally posted on JerryCahill.com

Antioxidant Supplement Helps Cystic Fibrosis Patients, Study Finds

By Carolina Henriques

A special formulation of an antioxidant-enriched multivitamin helped decrease the frequency of pulmonary exacerbations in patients with cystic fibrosis (CF), according to a new study.

The findings, by researchers at Children’s Hospital Colorado and the University of Colorado School of Medicine, were published in the American Journal of Respiratory and Critical Care Medicine under the title “Effects of an Antioxidant-enriched Multivitamin in Cystic Fibrosis: Randomized, Controlled, Multicenter Trial.”

Inflammation is an important contributor to lung damage and to progressive lung function decline in CF. In the study, researchers looked at the effects of a “cocktail” of multiple antioxidants on inflammation and health outcomes in CF patients.

“Single oral antioxidant formulations have been tested previously in CF with mixed results. However, there had not been a well-designed, randomized controlled trial of an antioxidant ‘cocktail’ that included multiple antioxidants in a single formulation,” Scott Sagel, MD, PhD, said in a University of Colorado news story. Sagel is the study’s first author and a pediatric pulmonologist at Children’s Colorado.

The 16-week study (NCT01859390) was conducted from September 2013 to October 2015 at 15 U.S. CF centers affiliated with the CF Foundation’s Therapeutics Development Network.

The study included 73 pancreatic-insufficient CF patients, 10 years and older (average age 22), who could not adequately absorb important dietary antioxidants such as beta-carotene, coenzyme Q10 (CoQ10), tocopherols (vitamin E), and selenium, which help neutralize inflammation in the body.

Participants received either capsules of antioxidant-enriched multivitamins, or control multivitamins without antioxidant enrichment. The capsules were designed specifically for people with difficulties in absorbing fats and proteins, like CF patients.

The investigational antioxidant-enriched multivitamin supplement, called AquADEKs-2, contains standard amounts of fat-soluble vitamins (A, D, E, K) plus several antioxidants, including beta-carotene, mixed tocopherols, CoQ10, mixed carotenoids (lutein, lycopene and zeaxanthin), and the minerals zinc and selenium.

Sagel and his team found that the antioxidant-enriched multivitamin supplement increased the concentration of antioxidants in the bloodstream, and temporarily reduced inflammation at four weeks, even though these results were not sustained through the end of the 16 weeks of the study.

In addition, antioxidant supplementation was found safe and well-tolerated by study participants.

Researchers also observed that antioxidant treatment appeared to prolong the time to the first pulmonary exacerbation requiring antibiotics, and also to reduce the frequency of pulmonary exacerbations altogether.

For the full article, please visit CF News Today.