2018 was an impactful year for sickle cell disease (SCD). SCD is a group of disorders that cause red blood cells to become misshapen and break down, often causing severe, debilitating pain and blood vessel and organ damage.
The first drug in over 20 years was approved by the FDA on July 2017 for sickle cell patients , five years and older. January 2018, Emmaus Life Sciences and the Centers for Medicare and Medicaid Services came to an agreement and allowed coverage for use of Endari.
On September 20th , the American Society of Hematology (ASH) released the “State of Sickle Cell Disease Report Card”. This report card examined the current state of sickle cell, and the goals for the future for each of the following areas: access to care in the United States, provider training and education, research, clinical trials, and global issues. This report card included over 100 stakeholders in the SCD community . They were asked to rate the sate of SCD based on an established criterion set forth by ASH.
On December 3rd , Global Blood Therapeutics announced its intent to submit a new drug application to the Federal Drug Administration for the approval of their drug, , Voxelotor. If approved, this will mark the third drug made available for use with sickle cell patients.
Novartis Pharmaceuticals, the Sickle Cell Disease Association of America (SCDAA), and award-wining a singer/actress Jordin Sparks , teamed up to announce their project , Generation S, just days before the SCDAA’s 46th annual convention on October 10-13th. This campaign will allow sickle cell patients to tell their story, to share what life is like when living with sickle cell. Afterwards, five individuals will be selected to formally meet the singer and have their story told and captured by a film crew.
For this year’s Solutions to Empower Patients program, and continuing with Novartis’s commitment to sickle cell, the company announced in November that they will award up to $50,000 for original and creative ideas to help sickle cell disease patients and their families.
The company has also introduced an investigational therapy , crizanilzumab , that reduces the occurrences of disease complications and may lead to irreversible organ damage in SCD patients.
Lastly, on December 11th, the Senate passed bill S.2465, the “Sickle Cell Disease and Other Heritable Blood Disorders Research, Surveillance, Prevention, and Treatment Act of 2018”. It was signed into law on December 18th. This law means more research and clinical trials, and more funding for sickle cell patients. On that same day, the Bill and Melinda Gates Foundation dnated $1.4 million to Boston Children’s hospital for the development of gene therapy for sickle cell disease. The goal is to make drug therapy widely available on a global scale.
Overall, 2018 was more than impactful, more gains were made for sickle cell disease than ever before. The new year brings hope.
My name is Kay-Diene Robinson. I am 29 years old, and I was born with sickle cell anemia. For twenty-six years my entire existence consisted of pain and agony whenever I experienced a pain crisis; a pain I would not wish on anyone.
On September 11, 2015, I was blessed to undergo a bone marrow transplant that has changed the course of my existence. Now cured of the disease, I no longer deal with the unpredictable pain crises. Despite my cure, I still face my challenges with pain including recurring migraine headaches and the occasional joint pain from avescular necrosis.
September of this year, I was invited to join The Pain Community as a Board Member and Sickle Cell Disease Liaison. This opportunity has allowed me to meet and connect with individuals and discover that the common thread that connects us all, pain. Shedding light on ways to cope is paramount; therefore let us celebrate our differences and come together and share techniques and management skills when dealing with our common enemy…pain.