You can teach a dog (me) new tricks (insulin pump and CGM).

By Andrea Eisenman

Here I am at 54 years of age, learning new things that sort of overwhelm me but are a necessary evil to keeping my diabetes in check. Even though I was diagnosed with diabetes after my lung transplant in 2000, I only started using an insulin pump 5 years ago. The only reason I started using a pump was because my endocrinologist kept asking me to try it. I finally relented. Continue reading You can teach a dog (me) new tricks (insulin pump and CGM).

How to Pick the Perfect College

By Tabby Caldwell

I’ve been thinking about my future lately and realizing how soon its actually coming. I’m planning on going to graduate school next year, and deciding on which schools to apply to has been on my mind. This past week I thought to myself “I’ll be at this school for at least four years, so I should probably find a school with a good CF center nearby”. I realized that this thought never even occurred to me when I was choosing an undergraduate school three years ago. In hindsight, coming to Utah was not the wisest choice. The air is always polluted, the cold winters and the elevation make breathing even harder, and the CF center is not super close. Continue reading How to Pick the Perfect College

Apply for the Abbvie CF Scholarship

AbbVie, a global research and development-based biopharmaceutical company, announced that the AbbVie CF Scholarship program is accepting applications for the 2019-2020 academic school year. Undergraduate and graduate students are invited to apply for the scholarship until May 29, 2019 by visiting www.AbbVieCFScholarship.com. Now in its 27th year, the program has awarded over $3 million in scholarships to deserving students across the country.

“As a recipient of an AbbVie CF Scholarship, I hope that I can inspire other students living with CF to strive to be the best they can be,” said Vinny Holmquist, 2018 recipient of the Thriving Undergraduate Scholarship. “Living with CF presented many challenges for me and my family, but it also motivated me to be a hard worker. I’m proud to say that the AbbVie CF scholarship has enabled me to continue my education and has helped me pursue a degree in business administration.”

AbbVie will select 40 scholars to receive $3,000 for use during the 2019-2020 academic year based on their creativity, academic excellence, community involvement and the ability to serve as a positive role model for the CF community. These scholars will also be given the opportunity to compete for a total of $25,000 for use toward education-related expenses through one of two AbbVie CF Scholarship award categories: Thriving Undergraduate Student and Thriving Graduate Student.

“Students living with CF are able to lead meaningful, impactful lives and make valuable contributions in their schools and local communities,” said John Duffey, vice president, U.S. Specialty, AbbVie. “It’s a privilege to recognize the amazing accomplishments of our AbbVie CF scholarship recipients. We are proud to continue our commitment to these remarkable students.”

Students can apply online or by downloading an application on the scholarship website. For more information about the application criteria, contest rules and upcoming deadlines surrounding the application process, and to view creative submissions of past winners, visit www.AbbVieCFScholarship.com.

Cystic Fibrosis Research Study

A research study for adults 18 years of age and older, who have been diagnosed with Cystic Fibrosis is now enrolling. This study is being conducted to evaluate the safety and efficacy an experimental treatment for people with Cystic Fibrosis who also have a bacterial infection in their lungs.

You may qualify if you:

  • Are at least 18 years of age
  • Have been diagnosed with Cystic Fibrosis
  • Are currently being treated with inhaled antibiotics (for at least 3 months)

Continue reading Cystic Fibrosis Research Study

Holding Out for 3D-Printed Sinuses

By Sydna Marshall

I often suggest that I should model for the artist-rendering of worst-case scenario CF-sinuses. I’ve had countless sinus surgeries and my ENT (“Dr. E”) has tried everything along the way. Dr. E has opened up my cavities by cutting back some of the bone and membranes, he’s put a flap of sorts in the base of my sinus cavities to help the cheek cavities drain better. He’s fixed my septum and he’s removed a huge number of polyps in multiple surgeries, both under general anesthesia and at his office with local anesthetic. At Dr. E’s suggestion, I’ve also undergone the frontal obliteration. All of this, and my sinuses just plain suck. It’s the number one struggle I have as far as managing my CF.

A year ago, I decided it was time to just see him every two weeks to keep things flushed out routinely and help cut down on hospital time and/or IV antibiotics. Pseudomonas has plagued me for ten years now and just like Bob in the movie, What About Bob, my colonization won’t leave. It’s a resilient little bugger! Because my sinuses are so swollen, we’ve had to think outside the box when it comes to numbing methods before they get washed out in-office. Years ago, three sprays of lidocaine did the trick. Now, I get a spray of lidocaine followed by two gauze pads soaked in lidocaine, which sit in my nostrils for thirty minutes or so. After that, I sometimes get the numbing gel and/or a shot of lidocaine directly in my sinus membranes. Just last week we tried tetracaine, which helped tremendously. It takes roughly 90 minutes for everything to work before we can start the process of suction, extraction, and flushing with huge syringes of water. My favorite part of this whole routine is the immense relief when he extracts something with the alligator tool. It’s the very definition of instant gratification and I’ve spent so much time in his office that my husband bought me my own alligator tool to hang as a Christmas ornament on our tree. One of these days I’m going to ask to decorate what is now my plastic bowl for the rinses. Sydna’s Snot Bowl has a nice ring to it!

Many years ago, I upgraded from the sinus rinse bottle to a SinuPulse machine (think Waterpik for your sinuses) as they really need the extra oomph to power through the mucus and crusting from the infection. I highly recommend it if you’re struggling to get relief from the regular sinus rinse bottle. Over the years, I’ve tried it all: silver sprays, essential oils in my rinse, nebulized antibiotics with a sinus nebulizer, and manuka honey. The latest attempt is a compounded drug consisting of two antibacterial meds, an antifungal, and a steroid, which gets mixed in my sinus rinse along with Alkalol and manuka honey. So far, this seems to help tremendously.

So, what does severe sinus disease look like? It looks like routine visits and trying new therapies, often with little change in outcome and/or comfort. It’s an ever-evolving process and I’ve had a long time to accept that with the therapies available now, my sinuses won’t really get better, as I previously thought they would. Back in 2007 when I first started seeing Dr. E, I mistakenly assumed that one sinus surgery would fix my sinus issues and I’d be on my way to relatively normal sinuses going forward. In hindsight, that was a poor expectation on my part as a routine adenoidectomy at 11 led to my CF diagnosis with the discovery of polyps in my sinuses.

Meanwhile, I’m holding out for 3d-printed sinuses!

Sydna lives in Austin, TX with her husband and fur baby. She loves to read, is a part-time practicing yogi, and enjoys cooking!

For Use in CF, New 2-in-1 Powder More Effective Against Resistant Bacteria

https://cysticfibrosisnewstoday.com/2018/12/18/new-powder-inhalation-more-effective-killing-antibiotic-resistant-bacteria/?utm_source=Cystic+Fibrosis&utm_campaign=c31e0676bf-RSS_MONDAY_EMAIL_CAMPAIGN&utm_medium=email&utm_term=0_b075749015-c31e0676bf-71418393

By Albert Molano

Originally published in Cystic Fibrosis News Today

12/18/2018

Purdue University researchers have invented a new way of delivering two antibiotics (colistin and ciprofloxacin) deep into the lungs of cystic fibrosis (CF) patients, enabling much more effective killing of antibiotic-resistant bacteria without exposing patients to high systemic doses of these therapies.

“We are providing a promising option to fight the global crisis of antimicrobial resistance,” Qi (Tony) Zhou, PhD, assistant professor at Purdue’s College of Pharmacy, who led the research team, said in a press release.

Respiratory infections caused by multidrug-resistant bacteria in CF patients can be deadly because they are resistant to most available antibiotics. In addition, therapies given intravenously (by injection into the vein) or orally have a hard time reaching the lungs, potentially requiring high doses for the therapy to be effective.

Last-resort antibiotics can still be effective, but these can be very toxic if they are given systemically. Colistin, for example, can damage the kidneys.

The existing alternative is to nebulize the antibiotics and deliver them directly into the lungs, which increases their local concentrations while reducing the risk of systemic toxicity. However, this requires expensive and complicated delivery devices, and prolonged administration times.

Now, Zhou’s team has succeeded in combining two antibiotics — colistin and ciprofloxacin — into a single particle that can be delivered as a dry powder.

According to the team, with this new formulation, more than 60 percent of the antibiotics reach the lungs, as opposed to only 10 percent with a jet nebulizer. In addition, the dry formulation offers improved chemical stability, and it is easier to use than conventional inhalation products.

“It has been a worldwide challenge to incorporate two antibiotics with different chemical properties into a single particle. Our novel formulation allows for a much more effective killing of drug-resistant bacteria in the deep lungs as two synergistic antibiotics can be simultaneously delivered to the same infection site,” Zhou said.

The team believes this technology can be applied to several antibiotic and compound combinations, potentially saving tens of thousands of lives from several deadly lung infections, including those affecting CF patients and people with ventilator-assisted pneumonia.

The Purdue Office of Technology Commercialization has filed a patent for the new technology, and researchers are looking for partners to continue its development.

The Hospital Comfort Kit Is Now Available!

The Hospital Comfort Kit Is Now Available!

When Rebecca Poole was admitted to the hospital in December 2014, she had no idea that she would not be discharged for 219 days. Her husband Ray focused daily on what he could do to make her more comfortable. Friends and family would ask what they could do to help and at the time he didn’t have an Continue reading The Hospital Comfort Kit Is Now Available!

A Drug Costs $272,000 a Year. Not So Fast, Says New York State.

From The New York Times:

A Drug Costs $272,000 a Year. Not So Fast, Says New York State.

New York’s Medicaid program says Orkambi, a new drug to treat cystic fibrosis, is not worth the price. The case is being closely watched around the country.

A wave of breakthrough drugs is transforming the medical world, offering hope for people with deadly diseases despite their dizzying price tags.

But what if it turns out that some of these expensive new drugs don’t work that well?

That’s the quandary over Orkambi, a drug that was approved in 2015 for cystic fibrosis and was only the second ever to address the underlying cause of the genetic disease. Orkambi, which is sold by Vertex Pharmaceuticals, costs $272,000 a year, but has been shown to only modestly help patients.

Now, in a case that is being closely watched around the country, New York state health officials have said Orkambi is not worth its price, and are demanding that Vertex give a steeper discount to the state’s Medicaid program. The case is the first test of a new law aimed at reining in skyrocketing drug costs in New York’s Medicaid program.

The high price of prescription drugs has ignited a populist furor, and in May, the Trump administration unveiled a set of proposals to address the issue. But while the ideas at the federal level are still mostly theoretical, some states have begun tackling the issue themselves. Earlier this year, Massachusetts asked the federal government for permission to limit its coverage of drugs in an effort to secure larger discounts from drug makers. Other states, like California and Vermont, have passed laws requiring drug companies to turn over certain financial details if they raise prices significantly.

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“There’s a number of states that are really trying to push forward and say, we need to be thinking very differently about how we’re paying for drugs,” said Matt Salo, the executive director of the National Association of Medicaid Directors. “We need the ability to say that there are some drugs that are just not priced in a rational way.”

Orkambi held great promise for people with cystic fibrosis when it was approved three years ago. A similar drug, Kalydeco, approved in 2012, was viewed as groundbreaking because it was the first to try to counteract the genetic defect that causes cystic fibrosis. The disease, which affects about 30,000 Americans, leads to a buildup of sticky mucus in the lungs and can lead to death by respiratory failure by the time many people are 40.

But while Kalydeco, also known as ivacaftor, was found to be effective, it was only approved for a sliver of patients with the disease — those who had certain genetic mutations. Orkambi, which combines ivacaftor and another drug, lumacaftor, was approved for mutations that covered nearly half of cystic fibrosis patients, but studies showed it was not as effective as Kalydeco.

Since Orkambi’s approval, several countries have balked at paying for it, including Britain, France and Canada.

In the United States, private insurers and Medicare plans have generally covered Orkambi. Medicaid programs, which cover health insurance for the poor, are required to cover all drugs.

Still, many insurers require patients to pay thousands of dollars out of pocket, and even though Vertex offers assistance, not everyone qualifies.

Lora Moser, 40, is covered by Medicare because she is disabled, and said she had to stop taking Orkambi in January because she could not afford the first month’s payment of more than $3,000 required by her insurer, Humana. A spokeswoman for Humana said that for high-cost drugs like Orkambi, the insurer helps patients identify outside assistance programs to cover out-of-pocket costs.

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Ms. Moser said a nonprofit group that had provided financial assistance declined to renew her grant because, she said she was told, her annual household income was too high.CreditTamir Kalifa for The New York Times

A nonprofit group that had provided assistance the previous year to Ms. Moser declined to renew her grant because, she said she was told, her annual household income was too high. She said her income is about $600 above their limit.

“I’ve never felt more destitute and hopeless as I do right now, from a medical standpoint,” Ms. Moser said.

A spokeswoman for Vertex, Heather Nichols, said more than 99 percent of cystic fibrosis patients who are eligible to take Orkambi in the United States have “broad access” to the drug.

“Vertex has a longstanding commitment to supporting access for all eligible patients, and we will continue to oppose any attempts to restrict patient access to these transformative medicines,” Ms. Nichols said.

Despite its lukewarm reception, Orkambi has been a boon for Vertex. In 2017, the drug was its top-selling product, bringing in about $1.3 billion in sales, a considerable sum for a product that is only approved to treat about 28,000 people worldwide.

Dr. Steven D. Pearson, the president of the Institute for Clinical and Economic Review, which evaluates the cost-effectiveness of drugs, said the problem is that in the United States, drug companies control the prices, especially in the case of newly approved drugs like Orkambi.

“Our system is set up not to distinguish very well between those drugs that are fairly priced and those that are not,” he said. Dr. Pearson’s institute concluded that Vertex’s cystic-fibrosis drugs should be discounted by as much as 77 percent. “That gives the incentive to the company to overreach, and that’s part of why our system is so out of whack,” he said.

In April, Orkambi became the test case for the New York law when a state board ruled that the drug was not worth its cost, recommending that it be discounted from the list price by roughly 70 percent — an amount that was influenced by work done by Dr. Pearson’s institute. New York’s law, passed in 2017, allows the state to ask manufacturers for a deeper discount if the state’s Medicaid drug budget exceeds a certain amount.

Under federal law, state Medicaid programs get a rebate of at least 23 percent. New York officials said that they identified 30 drugs this year that were priced too high, and that those products’ manufacturers agreed to deeper discounts, resulting in about $60 million in annual savings. Vertex, which is based in Boston, was the only company that refused, the state said. New York officials did not identify the manufacturers that agreed to steeper discounts.

For now, at least, Vertex appears to have the upper hand because federal law requires the state to cover Orkambi, although the state can limit its use. Under its new law, New York could also demand that Vertex disclose details about how it sets its price, including how much goes toward research and development or to other areas, like marketing. But even if Vertex complied, that information would not be made public because it is considered proprietary.

Ms. Nichols, the Vertex spokeswoman, said the company had no plans to agree to a discount below the 23 percent required by law.

And Donna Frescatore, the director of New York’s Medicaid program, said she was reluctant to limit the use of Orkambi for those who need it. “It’s certainly a balance with our ability to get fair pricing for this medication,” she said.

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Ms. Moser prepares to administer medication through a nebulizer at her home in Leander, Tex. CreditTamir Kalifa for The New York Times

But despite the impasse, Mr. Salo said big states like New York are major buyers of prescription drugs, and companies may see an interest in taking those states seriously. “I see this as being of very, very widespread interest,” he said. “A lot of other states are kind of watching and saying, ‘How is that going to work?’”

The debate over Orkambi may soon become moot — earlier this year, the Food and Drug Administration approved a new cystic fibrosis drug, also made by Vertex. Symdeko, as the drug is called, treats a similar population as Orkambi, but has been proven to be more effective. It carries a list price of $292,000 a year, and some analysts, including Geoffrey Porges, of Leerink, say they believe Symdeko will eventually replace Orkambi.

Given the arrival of Symdeko, some analysts said New York would be smart to negotiate a package deal for all three of Vertex’s cystic fibrosis drugs, similar to a deal recently made with Ireland. Ms. Frescatore said that’s an approach that she would consider.

“You don’t want a patient being forced to take Orkambi because it’s cheaper,” Mr. Porges said. “You want the right patient to get the right medicine.”

Katie Thomas covers the business of health care, with a focus on the drug industry. She started at The Times in 2008 as a sports reporter. @katie_thomas

TEDx talk: The Case for Realistic Optimism

The Case for Realistic Optimism

Have you ever struggled to stay positive when dealing with a sick loved one? When Ray’s wife Rebecca went into respiratory failure from end stage cystic fibrosis he was faced with this challenge. What he learned was that choosing to be realistically optimistic helped him to remain strong for Rebecca during Continue reading TEDx talk: The Case for Realistic Optimism

New drug for the treatment of exocrine pancreatic insufficiency

http://markets.businessinsider.com/news/stocks/AzurRx-BioPharma-and-Mayoly-Spindler-Announce-MS1819-SD-Investigational-Medicinal-Product-Dossier-IMPD-Submission-1005030890

AzurRx BioPharma and Mayoly Spindler Announce MS1819-SD Investigational Medicinal Product Dossier (IMPD) Submission

AzurRx BioPharma Inc. (NASDAQ:AZRX) (“AzurRx” or the Continue reading New drug for the treatment of exocrine pancreatic insufficiency