The Boomer Esiason Foundation

Category: CF News

Every day, researchers around the world come one step closer to discovering a cure for cystic fibrosis. In the meantime, the CF drug “pipeline” ensures therapies are moving from the laboratory to the marketplace. We keep an eye on medical news sources from around the world and report on these developments as they occur.

  • PTC Therapeutics Announces Results from Pivotal Phase 3 Clinical Trial of Ataluren

    SOURCE: PRESS RELEASE VIA PTS THERAPEUTICS

    PTC Therapeutics Announces Results from Pivotal Phase 3 Clinical Trial of Ataluren in Patients Living with Nonsense Mutation Cystic Fibrosis

     

    – ACT CF trial missed primary and secondary endpoints –

    – Company to host conference call today, March 2nd at 9:00 am ET –

     

    SOUTH PLAINFIELD, N.J., March 2, 2017 /PRNewswire/ — PTC Therapeutics, Inc. (NASDAQ: PTCT), today announced that the Ataluren Confirmatory Trial (ACT CF) in nonsense mutation cystic fibrosis (nmCF) did not achieve its primary or secondary endpoints. Ataluren was generally well tolerated and ACT CF confirmed a favorable safety profile for ataluren, which has now been used by more than 1,000 patients across multiple indications. PTC plans to discontinue current clinical development of ataluren in cystic fibrosis, close ongoing extension studies and withdraw its application for marketing authorization in cystic fibrosis in Europe.

     

    “We are disappointed with the outcome of this trial as there are no treatments that target the underlying cause of nonsense mutation cystic fibrosis, one of the most difficult forms to treat,” said Stuart W. Peltz, Ph.D., chief executive officer of PTC Therapeutics. “We are particularly grateful to patients and investigators who participated in our trials. We remain committed to patients receiving ataluren in other indications.”

     

    ACT CF was a double-blind, placebo-controlled, 48-week clinical trial comparing ataluren to placebo in nmCF patients six years of age or older not receiving chronic inhaled aminoglycosides. The Phase 3 study, conducted in 16 countries, enrolled 279 patients who were randomized to receive either ataluren or placebo. In the intent-to-treat population, the primary endpoint of lung function as measured by absolute change in percent-predicted FEV1 (forced expiratory volume in one second), over 48 weeks from baseline, there was a 0.6% difference in favor of ataluren versus placebo (-1.4% change on ataluren versus -2.0% change on placebo; p=0.534). For the secondary endpoint of rate of pulmonary exacerbations, there was a trend in favor of ataluren, with the rate in the ataluren group being 14% lower than the placebo group (p=0.401). The results were not statistically significant. 

     

    The safety profile of ataluren in the ACT CF study was consistent with previous studies and no new safety signals were identified.

     

    About Cystic Fibrosis

    Cystic fibrosis is among the most common life-threatening genetic disorders worldwide. It is caused by defects in a single gene known as the cystic fibrosis transmembrane conductance regulator, or CFTR. The CFTR gene encodes the CFTR protein, which is used by the body to transport chloride across cell membranes. Genetic mutations that result in the loss of function of the CFTR protein cause the body to produce abnormally thick and sticky mucus that clogs multiple organs, including the lungs, pancreas and liver. In particular, the absence or very low levels of CFTR leads to progressive loss of lung function, potentially life-threatening lung infections, permanent pancreatic damage and malnutrition because digestive enzymes from the pancreas do not reach the intestines to help break down and absorb food. The average age of death for CF patients is in their mid-thirties.

     

    About ataluren (Translarna™)

    Ataluren (brand name: Translarna™), discovered and developed by PTC Therapeutics, Inc., is a protein restoration therapy designed to enable the formation of a functioning protein in patients with genetic disorders caused by a nonsense mutation. A nonsense mutation is an alteration in the genetic code that prematurely halts the synthesis of an essential protein. The resulting disorder is determined by which protein cannot be expressed in its entirety and is no longer functional, such as dystrophin in Duchenne muscular dystrophy. Ataluren is licensed in the European Economic Area for the treatment of nonsense mutation Duchenne muscular dystrophy in ambulatory patients aged five years and older. Ataluren is an investigational new drug in the United States. The development of ataluren has been supported by grants from Cystic Fibrosis Foundation Therapeutics Inc. (the nonprofit affiliate of the Cystic Fibrosis Foundation); Muscular Dystrophy Association; FDA’s Office of Orphan Products Development; National Center for Research Resources; National Heart, Lung, and Blood Institute; and Parent Project Muscular Dystrophy.

     

    About PTC Therapeutics

    PTC is a global biopharmaceutical company focused on the discovery, development and commercialization of orally administered, proprietary small molecule drugs targeting an area of RNA biology we refer to as post-transcriptional control. Post-transcriptional control processes are the regulatory events that occur in cells during and after a messenger RNA, or mRNA, molecule is copied from DNA through the transcription process. PTC’s internally discovered pipeline addresses multiple therapeutic areas, including rare disorders and oncology. PTC has discovered all of its compounds currently under development using its proprietary technologies. PTC plans to continue to develop these compounds both on its own and through selective collaboration arrangements with leading pharmaceutical and biotechnology companies. For more information on the company, please visit our website www.ptcbio.com.

     

    SOURCE: PRESS RELEASE VIA PTS THERAPEUTICS

  • 2016-2017 Sacks for CF Scholarship Winners

    The Boomer Esiason Foundation has awarded prestigious “Sacks for CF” academic scholarships to 30 outstanding students with cystic fibrosis.

    Through the NFL season, longtime BEF partner Abbvie makes a donation to the BEF Sacks for CF scholarship program for every quarterback sack recorded during NFL Monday Night Football games. Then, during the Super Bowl, these funds are awarded to college students with cystic fibrosis based on academic achievement and adherence to daily CF therapy.

    The Sacks for CF scholarship winners for 2016-2017 are:

    Jacob Greene | Stanford University
    Benjamin James | Virginia Commonwealth University
    Julia Wright | Yale University
    Emily Hethcox | Auburn University
    Aleeya Young | Xavier University
    Joy Arnold | University of California – Berkeley
    Rosie Garvey | Clark University
    Thomas Brophy | University of Maryland
    Jared Cowan | Wabash University
    Luke McFadden | University of Arkansas

    Ambrose Bean | Ave Maria University
    Laura Cheevers | Providence College
    Sara Desrocher | College of St. Scholastica
    Alexandra Faircloth | Charleston Southern University
    Kevin Reis Missouri | University of Science and Technology
    Theresa Webber | UNC Chapel Hill
    Ryan Elitz | DeSales
    Mitchell Wekwert | Central Michigan University
    Beatrice Miner | University of Central Florida
    Charlie Kilgore | Brown

    Jake Reynolds | Springfield College
    Allison Johnson | Miami University
    Alexandria Naughton | University of South Alabama
    Benjamin Dodds | University of Pittsburgh
    Callie Deandra | Montreat College
    Corey Sarkis | Northeastern University
    Caroline Weber | South Dakota State University
    Brett Hatfield | University of Nebraska – Omaha
    Kelsey Wessels | Northern Kentucky University
    Mohammed Joudeh | Miami Dade College

    “Our ultimate goal and hope is that one day, a cure for cystic fibrosis is found. So while we wait – the Foundation has found ways to support the CF community every day in the here and now,” said Boomer Esiason. “Abbvie has been a proponent of that goal from day one and has changed the lives and futures of countless students who are battling CF.”

    For more information about the Sacks for CF program, please visit www.SacksForCF.com.

  • When you give, we give back!

    When you give, we give back!

    DONATE TODAY

    During the month of December 2016, the Boomer Esiason Foundation is giving back to our donors. Make a donation at one of the following levels, and we will send you a few special gifts! 

    $100
    5 Team Boomer bands, 5 YCF bands, YCF Children’s book, Tru Heroes drawstring bag, Team Boomer towel

    $250
    5 Team Boomer bands, 5 YCF bands, YCF Children’s book, Tru Heroes drawstring bag, Team Boomer towel, Boomer bobble head, Jump the Fear jump rope

    $500
    5 Team Boomer bands, 5 YCF bands, YCF Children’s book, Tru Heroes drawstring bag, Team Boomer towel, Boomer bobble head, Jump the Fear jump rope, Boomer Esiason autographed football

    $750
    5 Team Boomer bands, 5 YCF bands, YCF Children’s book, Tru Heroes drawstring bag, Team Boomer towel, Boomer bobble head, Jump the Fear jump rope, Boomer Esiason autographed football, Boomer Esiason autographed Maryland jersey

    $1000
    5 Team Boomer bands, 5 YCF bands, YCF Children’s book, Tru Heroes drawstring bag, Team Boomer towel, Boomer bobble head, Jump the Fear jump rope, Boomer Esiason autographed football, Autographed Boomer Esiason Bengal’s jersey, Personalized thank you from Boomer Esiason

  • Vertex Announces Planned Initiation of Phase 2 Studies Evaluating Next-Generation Correctors

    FULL PRESS RELEASE HERE

    Vertex Announces Planned Initiation of Phase 2 Studies Evaluating the Next-Generation Correctors VX-440 and VX-152 in Triple Combination Regimens to Treat the Underlying Cause of Cystic Fibrosis

    • VX-440 to be evaluated as part of 4-week triple combination dosing with tezacaftor (VX-661) and ivacaftor; VX-152 to be evaluated as part of 2-week triple combination dosing
    • Studies to enroll people with cystic fibrosis who have one copy of the F508del mutation and a minimal function mutation and also people with two copies of the F508del mutation
    • Additional next-generation correctors advancing into Phase 1 development; VX-659 Phase 1 clinical study expected to begin in 2016 and will enroll healthy volunteers and CF patients

    FULL PRESS RELEASE HERE

  • BEF to Participate in 2016 Cantor Fitzgerald Charity Day

    Boomer Esiason Foundation to Participate in 2016 Cantor Fitzgerald Charity Day

    Event to Honor the 15th Anniversary of the 9/11 World Trade Center Attacks

    The Boomer Esiason Foundation (BEF), a nonprofit organization dedicated to empowering the cystic fibrosis (CF) community through fundraising for research, programs, scholarships, transplant grants, and educational resources, today announced its participation in the 2016 Cantor Fitzgerald Charity Day. BEF and Cantor Fitzgerald have a long-standing history, dating back to 1995 when the foundation moved into Cantor Fitzgerald offices in the North Tower of the World Trade Center. The foundation has participated in numerous consecutive Charity Days, which were started to honor the 658 Cantor Fitzgerald friends and colleagues and 61 Eurobrokers employees lost in the 9/11 World Trade Center attacks. Among those lost was Tim O’Brien, Boomer Esiason’s close friend and an original member of BEF’s Board of Directors.

    Every year around the anniversary of the terrorist attacks, Cantor Fitzgerald and BGC Partners honor those who perished by distributing 100% of global revenues on Charity Day to The Cantor Fitzgerald Relief Fund (”Cantor Relief”). It is the mission of Cantor Relief to provide aid to victims of terrorism, natural disasters and other emergencies, as well as to a wide variety of other charitable organizations providing assistance to those in need. For this annual day, The Cantor Fitzgerald Relief Fund invites celebrity guests from the entertainment and sports industries to work with licensed brokers and their clients to raise money and awareness for a variety of causes.   

    “Cantor Relief’s Charity Day is an event that we hold very dear to our hearts at BEF, and on the 15th anniversary of the 9/11 World Trade Center attacks, this event gives us an opportunity to reflect. We lost many friends that day, so while Charity Day is bittersweet for me and all of us at the foundation, it also serves a reminder that positivity and generosity can grow out of tragic circumstances,” said Boomer Esiason, founder of BEF.  

    To date, The Cantor Fitzgerald Relief Fund has raised and distributed over $292 million, with Charity Day raising approximately $125 million globally.

     

    About the Boomer Esiason Foundation 
    Founded in 1993 by Boomer Esiason and his wife, Cheryl, after their son was diagnosed with cystic fibrosis, the Boomer Esiason Foundation (BEF) works to ensure that people living with CF and their loved ones are encouraged, empowered and educated to take an active role in treating the disease. BEF aids the CF community through fundraising, programs, scholarships, transplant grants and educational resources. The passionate work of a committed core of volunteers alongside leaders in the medical and business communities allows BEF to raise awareness, money and quality of life for those affected by this deadly disease.
    For more information, visit to www.esiason.org.

     

    About The Cantor Fitzgerald Relief Fund
    Founded in the wake of the September 11, 2001 terrorist attacks on the World Trade Center, The Cantor Fitzgerald Relief Fund has raised and distributed approximately $180 million to over 800 families and 932 children of the victims of that tragedy. Since 9/11, The Cantor Fitzgerald Relief Fund expanded its scope to not only include victims of terrorism, but to also assist victims of natural disasters and emergencies, including victims of Superstorm Sandy, the tornado in Moore, Oklahoma, Hurricane Katrina, the tsunami of 2004 and the Haiti earthquake.
    The Cantor Fitzgerald Relief Fund also works to assist wounded members of the U.S. military and a wide variety of other charitable organizations. Since its inception, The Cantor Fitzgerald Relief Fund has distributed over $292 million.

    The Cantor Fitzgerald Relief Fund is unique in that it often provides direct assistance directly to the people affected by these tragedies without intermediary or secondary organizational involvement. Cantor Fitzgerald Chairman and CEO Howard Lutnick and the partners of Cantor Fitzgerald underwrite all the expenses of the Fund so that 100% of every dollar raised goes directly to those in need.

    For more information, please visit www.cantorrelief.org.

     

    About Cantor Fitzgerald, L.P.
    Cantor Fitzgerald, L.P. is a financial services firm with operating units that are involved in a variety of market-based business initiatives. From its position as a leading financial intermediary in both the equity and fixed income markets, Cantor has expanded the scope of its business to include portfolio trading, investment banking, financial spread betting, market advisory, energy brokerage, CO2 emissions trading and electronic trading technology. Cantor brings its experience and expertise in the financial markets and its legacy of innovation in technology to benefit institutional investors and traders around the world. Whether creating new products and services or enabling its clients to trade more efficiently and effectively through cutting edge technological solutions, Cantor is reinventing markets every day.

  • Senators Markey and Grassley Pass Bipartisan Resolution Designating May 2016 CF Awareness Month

    SOURCE

    Washington (May 26, 2016)- Senators Edward J Markey (D-Mass.) and Chuck Grassley (R-Iowa) today applauded the passage of a bipartisan resolution to designate May 2016 as Cystic Fibrosis Awareness Month. The resolution strives to bring attention to this tragic disease, acknowledges the tremendous investments and scientific achievements made to improve lives of those with Cystic Fibrosis (CF), and celebrates the community of individuals with CF and who care for those with CF. Last month, Senators Markey and Grassley founded the Cystic Fibrosis Caucus in the U.S. Senate and will hold its inaugural event on Thursday, June 23, when both senators will host a discussion with teen advocates.

    “I am proud to work with my colleague across the aisle, Senator Grassley, to strive towards making the CF of Cystic Fibrosis stand for Cured Forever,”said Senator Markey, who co-founded the Congressional Cystic Fibrosis Caucus in the House of Representatives in 2006. “With increased awareness of this rare disease, and with additional resources, we can help those who battle daily with the realities of living with cystic fibrosis. Breakthrough therapies and innovative care delivery systems can serve as both a model and an inspiration for other rare diseases that afflict countless Americans. The CF community continues to work tirelessly for their cause and we will continue to partner with them as we move forward.”

    “It’s inspiring to work with families who advocate for people with cystic fibrosis,” said Senator Grassley, who spoke at a “Great Strides” walk for cystic fibrosis in West Des Moines last Saturday. “The parents who want the best for their children raise awareness about living with cystic fibrosis and work on research toward treatment and a cure.  Picking up the thread in the Senate with the caucus is meant to advance the cause these families have embraced.”

    A copy of the resolution, S.Res. 476, can be found HERE. 

  • Vertex Launches All in for CF

    2,000 dedicated Vertex employees released an open letter to the CF community annoucning a brand new initiative. It reads as follows: 

     

    Each May, the cystic fibrosis (CF) community recognizes CF Awareness Month to raise awareness of the disease and to underscore that, despite progress made in recent years, there is still more important work to be done. Together with the CF community – people living with CF, their families and loved ones, healthcare providers and advocates – we have made tremendous progress. Today, approximately one-third of people living with CF are eligible for treatment to address the underlying cause of the disease, but as you know… that is not enough. And that is why we all continue to work tirelessly toward the goal of one day developing medicines for all people living with CF.

    If the CF community had a window into our labs and offices, they would see that at the heart of Vertex, it is the community that motivates us each and every day. You all motivate us to dig deeper, to do more, to explore the “what if,” and to push the boundaries of what we know.

    Today, we launched the Vertex All in for CF initiative to provide that window for the CF community. To give everyone an inside look at our people and the approacheswe’re taking to pursue our goal of developing medicines for all people with CF. The site also shines a spotlight on members of the CF community and highlights the amazing work they are doing. This month we have highlighted Emily Schaller of the Rock CF Foundation and Lauren & Lee Bombardier. We will update the spotlight section each month and feature new stories from the CF Community. You inspire us all, and the work we do!

    You can visit the All in for CF site by clicking here or by going to www.vrtxallincf.com. Please share this initiative with others in the community and  use the hash tag #AllInForCF when you do! Members of the CF community can share their own stories on the website by visiting the “Voices of CF” section of the site. As always, we welcome any feedback you may have.

    We have come a long way, but have more work to do. We know you’re All in for CF. At Vertex, we are, too.

  • OneRepublic Features Teen with Cystic Fibrosis in New Music Video via CFF.org

    SOURCE

    September 25, 2014

    Bryan Warnecke, who has cystic fibrosis, is featured in a new music video from the American pop band OneRepublic. Their new hit song, “I Lived,” focuses on living life to the fullest.

    The American pop band OneRepublic, known for chart-topping hits such as “Apologize” and “Counting Stars” joined the search for a cure today with the release of the music video for their hit song, “I Lived.”

    The video features 15-year-old Bryan Warnecke of Colorado, who has cystic fibrosis. Despite his rigorous treatment regimen, which includes 40-50 pills and twice per day breathing treatments, Bryan is a double black diamond skier, avid skateboarder, long distance cyclist and one of the best hockey goalies in the state.

    Drawing from the words in the song, Bryan says, “I believe in living every second to its fullest, taking the jump without fear of the fall and taking the pain while giving it my all.” He adds, “My dream is to become the first person with CF to compete in the Olympics as the goalie for the USA hockey team. The CF Foundation and OneRepublic are helping to keep that dream alive.”

    “We are thrilled that OneRepublic has chosen to feature Bryan and support the 30,000 Americans living with cystic fibrosis through this inspiring song,” said C. Richard Mattingly, executive vice president and chief operating officer of the CF Foundation. “The funds and awareness it raises will help us achieve our mission to find a cure for all people with the disease.”

  • The CF Foundation Provided New Resources on Infection Prevention and Control via CFF.org

    SOURCE: CFF.org

    The Cystic Fibrosis Foundation has released new health education materials to reduce the risk of people with cystic fibrosis getting and spreading dangerous germs that can lead to serious lung infections. The materials are part of the Foundation’s expanded effort to help people with CF and their families make informed decisions while managing the daily challenges of living with the disease.

    Explore all of the CFF resources here.

  • Application Now Open for Students with Cystic Fibrosis for the 2014 AbbVie CF Scholarship

    PRESS RELEASE

    ·         Scholarship recognizes 40 exceptional students with cystic fibrosis (CF) who serve as role models for the community through academic excellence, creativity and community service

    ·         Students selected as 2014 recipients will compete for Thriving Student Scholarship awards of $22,000 each

    ·         Application available at http://www.AbbVieCFScholarship.com

    NORTH CHICAGO, Ill., April 16, 2014 – 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 http://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 http://www.abbvie.com Follo.w @abbvie on Twitter or view careers on our Facebook or LinkedIn page.

    Media:

    Libby Holman, +1 847-935-9361