Non-Profit Save One Life Welcomes Chris Bombardier as New Executive Director
Chris Bombardier, who was the first person with hemophilia to scale Mount Everest and climb the Seven Summits, has been selected as the new executive director at Save One Life. The non-profit organization supports people with bleeding disorders in developing countries around the world. Chris raised funds for the organization as a part of his climbs and was on their board of directors. See full article at prweb.com.
Study Finds Hepatitis C a Major Predictor of Liver Cancer in Hemophilia Patients
A large study showed that past infection with the hepatitis C virus (HCV) is the major risk factor for the most common type of liver cancer. The study analyzed more than 18,000 US patients with hemophilia. HCV is a major risk factor for the general population, but the study found that those with hemophilia can be particularly vulnerable. See full article at hemophilianewstoday.com.
Phase 3b Study Shows Novoeight Safe and Effective Over Long Term
A study published recently in Haemophilia showed that long term treatment with the third-generation recombinant Novoeight (turoctocog alfa) is safe and effective at preventing bleeding episodes in patients with hemophilia A. The trial was for all ages and for patients who had already received prior treatment. See full article at hemophilianewstoday.com.
Sangamo Treats 1st Patient in Trial of In Vivo Genome Editing for Hemophilia B
Sangamo Therapeutics announced treatment of the first patient in their Phase 1/2 clinical trial evaluating SB-FIX, an investigational in vivo genome editing therapy for patient with hemophilia B. The trial is designed to assess safety, tolerability and preliminary efficacy of SB-FIX in adults. See full article at prnewswire.com.
Data on Eloctate and Alprolix Show Efficacy and Safety over Four Years
Bioverativ announced final results of their ASPIRE and B-YOND studies at the 60th Annual Meeting of the American Society of Hematology (ASH). The studies demonstrated long term prophylactic treatment with both Alprolix and Eloctate consistently improved annualized bleed rates across all populations studies and at extended dosing intervals. See full article at prnewswire.com.
Spark Announces Updated Data for Gene Therapy SPK-8011 for Hemophilia A
Spark Therapeutics shared updated preliminary data on the first 12 patients in their Phase 1/2 trial of their SPK-8011 for hemophilia A. The gene therapy showed dramatic reductions in bleeds and infusions needed. This is an ongoing study with a Phase 3 study to begin by the end of 2018. See full article at globenewswire.com.
Roache Shares Positive Data for Hemlibra at American Society of Hematology
Roache announced positive data from the primary analysis of their phase III HAVEN 2 study evaluating Hemlibra (emicizumab) prophylaxis in children younger than 12 years old with hemophilia A with inhibitors. This included longer follow up for once-weekly dosing and new data for less frequent dosing. See full article at roche.com.
BioMarin Sponsors "Hemophilia: The Musical" with Teens from Around the US
The BioMarin sponsored "Hemophilia: The Musical" was a show created by teens and performed after a 3 day workshop in New York in November. The teens, all who have a bleeding disorder, auditioned and were selected to take part in workshops and perform. The musical was streamed live on Facebook and can be viewed on the Bloodstream Media page. See full article at fiercepharma.com.
Local Carson McCabe Interviewed on 9News After Being Chosen for Musical
Carson McCabe, who was chosen to be a part of "Hemophilia: The Musical" a workshop and performance in New York City on November 12, 2018, was interviewed on Denver TV station 9News before he headed out. He shared how hemophilia impacts patients and what he will be doing in the musical. See full article at 9News.com.
Arvada Teen Performs in Musical About Bleeding Disorders in NYC
Local Coloradan Carson McCabe, one of our patients here at the HTC, was chosen to participate in "Hemophilia: The Musical" a musical theater workshop and performance in New York City. He will join 24 other teenagers who all have bleeding disorders to share their stories on the stage. See full article at arvadapress.com.
Mimicry Not Replacement: Molecular Engineering Transforms Outlook
The direct replacement of factor for patients with hemophilia has become a primary way to reduce the number and severity of bleeding episodes and prevent complications such as joint disease. However, new treatments aimed at mimicking the function of factor VIII instead of replacing it has found some success with emicizumab, known as Hemlibra. See full article at hematology.org.
AAV Gene Therapy Successfully Given Again to Dogs in Model of Hemophilia A
The re-administration of adeno-associated virus (AAV) based gene therapy for hemophilia A was shown to be safe and to achieve long-term improvement in a dog model of the disease, even after neutralizing antibodies were evident following initial treatment. See full article at hemophilianewstoday.com.
NHF Publishes Review After 70th Annual Bleeding Disorders Conference
The NHF has published a Highlights Edition of NHF Daily to share some of the varied events and new information shared at the 70th Annual Bleeding Disorders Conference that was held in Orlando, Florida in October. Articles included a review of the documentary Bombardier Blood, Mental Health, Medical Marijuana and more. See Highlights Edition of NHF Daily here.
FDA Approves Hemlibra for Hemophilia A Patients Without Inhibitors
The FDA has approved Hemlibra, a subcutaneous formulation of emicizumab, for hemophilia A patients who have not developed an inhibitor. The medication was already approved for those with inhibitors but this approval allows it to be used by more patients. See full article at bloomberg.com.