SCDC Update, November 2019
FDA Approves Drug for Sickle Cell Disease
On Friday, November 15, Adakveo (crizanlizumab-tmca), a treatment to reduce the frequency of vaso-occlusive crisis was approved by the United States Food and Drug Administration (FDA). “Hope has never been higher for people living with sickle cell disease (SCD) and their families and supporters, with a pipeline of new treatments on the horizon, like the one being approved today, and several initiatives underway to better utilize current tools in the battle against the painful and deadly blood disorder,” said Acting FDA Commissioner Adm. Brett P. Giroir, MD. Adakveo, which was developed by Novartis, is only the third FDA approved drug for SCD. Click here to read the full press release.
CRISPR Treatment Has Started Working for Woman With SCD
Victoria Gray, who was born with sickle cell disease (SCD), is the first person with a genetic disease to be treated with CRISPR in the United States. CRISPR is a powerful gene-editing technique that easily alters gene sequences and modifies gene function.Gray’s edited cells are producing a crucial protein at levels that have already exceeded what doctors thought would be needed to alleviate the complications of SCD.”This preliminary data shows for the first time that gene editing has actually helped a patient with SCD. This is definitely a huge deal,” says Dr. Haydar Frangoul who is treating Gray. Click here to read the full story.
NIH and Gates Foundation to Contribute $100 Million Each to SCD and HIV
The National Institutes of Health plans to invest at least $100 million over the next four years toward developing affordable, gene-based cures for sickle cell disease (SCD) and HIV. The Bill & Melinda Gates Foundation will also invest $100 million toward this goal. The intention is for these cures to be made globally available, including in low-resource settings. Click here to learn more.
SCD Training and Mentoring Program (STAMP)
Many adults living with sickle cell disease (SCD) have challenges accessing care due to a national shortage of SCD-trained hematologists. To help address this gap, the Health Resources and Services Administration is collaborating with the U.S. Health and Human Services’ Office of Minority Health to deliver a new telehealth series for primary care providers called STAMP, the SCD Training and Mentoring Program. This series, taught by hematologists using a case study-based, tele-mentoring approach, will cover the basics of SCD care, such as pain management, hydroxyurea, and preventive services. Participating primary care providers will also be eligible to request on-demand consultative services from hematologists to further support patient care. The next trainings are scheduled for January 2020. Learn more here.
CDC’s Sickle Cell Data Collection Program Announcement
Sickle Cell Disease: Give Blood, Save a Life Podcast
This new Centers for Disease Control and Prevention (CDC) podcast highlights the importance of blood donations from African Americans to help people with sickle cell disease who might need one or more blood transfusions.
Importance of Raising Disease Awareness in SCD
Novartis has partnered with Dr. Alex Kumar, a global health physician and photographer, who has documented the challenging and inspiring stories of people with SCD from around the world. Visit www.NotAloneInSickleCell.com to hear the stories and gain information on how SCD and pain crises affect body, mind and overall life of patients with SCD.
Global SCD Quality of Life Survey (SWAY)
During the 61st American Society of Hematology (ASH) Annual Meeting & Exposition, a coalition of organizations and individuals will share compelling data from the Sickle Cell World Assessment Survey (SWAY), the largest ever-conducted international sickle cell survey. SWAY, which was sponsored by Sickle Cell Disease Coalition member Novartis, assessed the impact of sickle cell disease on the daily life of those living with the disease and uncovered critical insights into the real-world impact of the disease. Over 2000 individuals with SCD and 350 doctors from 16 countries contributed to the survey.The following abstracts, which detail the results of the survey will be presented during the ASH Annual Meeting:
The following abstracts, which detail the results of the survey will be presented during the ASH Annual Meeting:
- Management Strategies and Satisfaction Levels in Patients with Sickle Cell Disease: Interim Results from the International Sickle Cell World Assessment Survey (SWAY)
- Impact of Sickle Cell Disease Symptoms on Patients’ Daily Lives: Interim Results from the International Sickle Cell World Assessment Survey (SWAY)
New Global Sickle Cell Disease Alliance Formed
The Global Alliance of Sickle Cell Disease Organizations (GASCDO), a non-profit organization registered in Canada, which is an independent network of sickle cell disease (SCD) advocacy groups working together to advance patient care and help eradicate SCD, was recently formed. In March of 2019, 16 patient advocacy group representatives from five continents and 12 countries came together for the first time, for a face-to-face meeting in London, supported by Novartis, to discuss:
- Stigma in sickle cell disease (SCD) and patients experience across the world
- High need around education and level of awareness about SCD
- Access to optimal care and treatment
- Collaboration on issues where there is a shared need
Stay tuned for more information.
Congratulations to the American Public Health Association on a successful 2019 Annual Meeting and Expo focused on Creating the Healthiest Nation: For science. For action. For health. The conference convened over 13,000 public health professionals. The program included several sessions and abstracts that highlighted sickle cell disease (SCD), including the session entitled Pushing the Envelope of Public Health: A dialogue with Assistant Secretary for Health ADM Brett P. Giroir. ADM Giroir shared his vision and approaches on addressing the burden of SCD and other critical public health initiatives, and how a modernized U.S. Public Health Service will contribute to “changing the map.” Dr. Kimberly Smith-Whitley, from the Children’s Hospital of Philadelphia served on the panel and highlighted priorities from the SCD expert perspective.
Sickle Cell Disease: Progress in Treatment Options and Policies Briefing
On November 19, several members of the Sickle Cell Disease Coalition hosted a briefing on Capitol Hill to educate members of Congress and their staff on how policymakers can help support sickle cell disease (SCD) screening, treatment development, and access. The program featured Jennelle Stephenson, SCD gene therapy recipient, Matthew Porteus, MD, PhD, Stanford University, and Remy Brim, PhD, BGR Group. The briefing was co-hosted by the American Society of Gene & Cell Therapy, American Society of Hematology, American Society for Transplantation and Cellular Therapy, Sickle Cell Disease Association of America, and the Pediatric Hospital Sickle Cell Disease Collaborative. The event was also hosted in conjunction with Representatives Michael Burgess (R-TX) Danny Davis (D-IL) and Barbara Lee (D-CA). The program concluded with an overview of how policy makers could help, by supporting:
- National Institutes of Health research funding
- House Resolution 606, recognizing the importance of sickle cell trait awareness and the development of new treatments
- Novel payment models for approved gene therapies
- Dedicated funding for the Centers for Disease Control and Prevention Sickle Cell Data Collection program
ASH Annual Meeting
The 61st American Society of Hematology (ASH) Annual Meeting & Exposition, is scheduled for December 7-10, 2019, in Orlando, FL. The meeting provides an invaluable educational experience and an opportunity to review thousands of scientific abstracts highlighting updates in the hottest topics in hematology, including sickle cell disease (SCD). The following is a list of SCD specific programming.
Sickle Cell Disease Kiosk
This year’s SCD Kiosk will focus on care for individuals living with SCD. Visit the Kiosk to learn about ASH’s new SCD Guidelines and watch rapid fire 5-minute presentations on several guideline chapters. Are you an ASH member? Complete your Find a Hematologist profile so that individuals living with SCD can locate SCD providers in their area.
Friday Scientific Workshop
Friday, December 6, 2019, 1:00 pm - 6:00 pm, Room W314, Level 3 (Orange County Convention Center) Learn More. Novel Curative Options: Gene-Editing and Gene Therapy for Hemoglobinopathies with a Focus on Sickle Cell Disease
News Worthy SCD Abstracts
Three exciting sickle cell abstracts have been selected to be covered during ASH’s press briefing. ASH strictly limits attendance at the press briefings to registered media to ensure that the focus remains on communicating study details to media reporting on the meeting. However, the abstracts selected are available for those interested:
812.Chromatin Accessibility Mapping of Primary Erythroid Cell Populations Leads to Identification and Validation of Nuclear Factor I X (NFIX) As a Novel Fetal Hemoglobin (HbF) Repressor *(presenter Jeff Shearstone, Syros Pharmaceuticals)
613. Oral Arginine Therapy As a Novel Adjuvant in the Management of Acute Pain in Children with Sickle Cell Anemia in Nigeria: A Randomized Placebo-Controlled Trial(presenter Richard Onalo, FCPaed, University of Abuja, Nigeria)
4667. Fragmentation of Care for Young Adults with Sickle Cell Disease in California (presenter Anjlee Mahajan, MD, UC Davis Cancer Center)
Management of Sickle Cell Disease Complications Beyond Acute Chest
This session is offered twice:
Saturday, December 7, 2019: 7:30am-9:00am, Hall E2, Level 2 (Orange County Convention Center)
Monday, December 9, 2019: 2:45pm-4:15pm, Tangerine 1 (WF1), Level 2 (Orange County Convention Center) Learn more.
Sickle Cell Disease in Young and Old: A Time for Re-Evaluation
This session is offered twice:
Saturday, December 7, 2019: 2:00pm-3:30pm, W304, Level 3 (Orange County Convention Center)
Sunday, December 8, 2019: 9:30am-11:00am, W304, Level 3 (Orange County Convention Center) Learn more.
Point-Counterpoint: Curative Therapies for SCD-Does it Make More Sense to Target the Root Cause Than All the Downstream Events
This is an Education Spotlight Session.
Sunday, December 8, 2019: 4:30pm-6:00pm, W312, Level 3 (Orange County Convention Center) Learn more.
Scientific Committee on Transplantation Biology and Cellular Therapies: Genome Editing for Transplantation and Cellular Therapies
This session is offered twice:
Saturday, December 7, 2019: 9:30am-11:00am, Hall D, Level 2 (Orange County Convention Center)
Sunday, December 8, 2019: 7:30am-9:00am, Hall D, Level 2 (Orange County Convention Center) Learn more.
Special-Interest Session on Sickle Cell Disease Centers
Saturday, December 7, 2019, 4:00 pm - 6:00 pm, Tangerine 3 (WF3-4), Level 2 (Orange County Convention Center) Learn more.
Special Education Session on ASH Clinical Practice Guidelines on Sickle Cell Disease
Sunday, December 8, 2019, 12:00 pm - 1:30 pm, Tangerine 2 (WF2), Level 2 (Orange County Convention Center) Learn more.
Global Sickle Cell Disease Network-Sponsored Meeting
Improving Outcomes for Sickle Cell Disease in Low-resource Settings: Bridging the Implementation Gaps
Sunday, December 8, 2019: 11:15am-12:30pm, Barrel Spring Room, Convention Space, Hyatt Regency Orlando Hotel
To RSVP for this meeting, email: email@example.com.
Consultative Hematology Course
Monday, December 9, 2019, 7:00 am - 11:30 am, Hyatt Regency Orlando, Regency Ballroom T-U
Molly Weidner Mandernach, MD, MPH will discuss: Sickle Cell Disease: Old and New Truths. Learn more.
ASH Research Collaborative Update
Monday, December 9, 2019, 6:15 pm - 7:45 pm, W315, Level 3 (Orange County Convention Center) Learn more.
The ASH Research Collaborative (ASH RC) is a non-profit organization that was established by ASH last year to foster collaborative partnerships to accelerate progress in hematology, with the goal of improving the lives of people affected by blood diseases. The foundation of the ASH RC is its Data Hub, a technology platform that facilitates the exchange of information by aggregating in one place, and making available for inquiry, research-grade data on hematologic diseases. The first research initiative of the ASH RC is a Sickle Cell Disease (SCD) Clinical Trials Network (CTN), which launched in 2019 with the goal of optimizing the conduct of clinical trials research in SCD. The Network will leverage the Data Hub to collect key information and identify gaps that will help advance SCD research and treatment.
The ASH Research Collaborative will provide updates on site selection for the SCD Clinical Trials Network and technology and data set developments for the Data Hub.
Sickle Cell Disease Coalition Members Exhibiting at ASH:
• American Society for Apheresis
• American Society for Clinical Pathology
• American Society of Pediatric Hematology/Oncology
• American Society for Blood & Bone Marrow Transplantation
• Bluebird bio
• College of American Pathologists
• Crispr Therapeutics
• Emmaus Medical, Inc.
• Foundation for Sickle Cell Disease Research
• Functional Fluidics
• Global Blood Therapeutics
• Medunik USA
• National Heart, Lung and Blood Institute
• National Marrow Donor Program/Be the Match
• Sanofi Genzyme
• Terumo BCT
Click here for information on exhibit locations.
Upcoming SCD Events
Is your organization a member of the Sickle Cell Disease Coalition? Send in your upcoming sickle cell events! Email Karina Ngaiza at firstname.lastname@example.org.