Author Archives: Emma Day

SCDAA Teams with MedicAlert Foundation


Sickle Cell Disease Association of America Teams with MedicAlert Foundation to Improve Emergency Outcomes During Sickle Cell Crises

Nonprofits partner to help people with sickle cell disease get faster, better emergency care.

The Sickle Cell Disease Association of America, the leading patient organization for people with sickle cell disease, announced a pilot program with MedicAlert Foundation to enhance the safety and well-being of people living with sickle cell disease.

More than 100,000 Americans live with sickle cell disease, a genetic blood disorder that affects red blood cells. Acute pain episodes known as sickle cell crises are one of the most common and debilitating symptoms of sickle cell disease. These crises can be unpredictable and extremely painful, lasting from a few hours to a few weeks. They’re the No. 1 reason people with sickle cell disease seek emergency treatment.

However, patients seeking treatment for a sickle cell crisis face hurdles to getting the care they need in the emergency department. Many emergency physicians are not well versed in sickle cell disease and treatment protocols. And with the rise of the opioid crisis, emergency department providers are cautious about providing the powerful pain medication needed to quell a sickle cell crisis. Sometimes sickle cell patients are unfairly labeled as “drug seekers” by emergency department personnel who don’t understand the disease.

The Sickle Cell Disease Association of America and MedicAlert pilot program aim to improve access to timely, effective emergency care for people experiencing a sickle cell crisis. MedicAlert Foundation is a leading nonprofit providing lifesaving medical identification and emergency response services for millions of people living with chronic health conditions.

“Delayed treatment in a sickle cell crisis can lead to long-term organ damage and other health complications — not to mention the unnecessary pain the person with sickle cell disease must endure,” said Regina Hartfield, president and CEO of the Sickle Cell Disease Association of America. “MedicAlert has decades of experience storing critical health information and making it available to emergency personnel. We want to leverage that to improve the experience for people seeking treatment for a sickle cell crisis.”

The pilot program will provide participants with a MedicAlert digital health profile to securely store their health information, treatment and pain plans, medications, physician information, emergency contacts and more. Each participant will also receive a customized Smart Medical ID Card, which provides easy access to their health information and physician-prescribed pain management plan via a QR code.

The goal is to decrease time to diagnosis and treatment, improving health outcomes from a sickle cell crisis. When seeking emergency treatment, participants can use the Smart ID Card to share their health history with emergency department personnel — confirming their sickle cell disease status and providing the critical details needed for personnel to provide care.

“Since 1956, MedicAlert has been globally trusted by both emergency medical personnel and people living with serious health conditions,” stated Karen Cassel, MedicAlert Foundation’s president and CEO. “Through this collaboration, we hope to equip and empower sickle cell patients with tools to help them quickly get the care they need during a pain crisis.”

The pilot program, which will run for one year, is set to launch in September 2023 during National Sickle Cell Awareness Month. Two-hundred-fifty individuals will be recruited for the first round.

“We believe this program has the potential to significantly improve outcomes for people experiencing sickle cell crises,” Hartfield said. “With positive results, we’ll seek additional funding to expand the program nationally.”

Cassel said, “Both MedicAlert Foundation and the Sickle Cell Disease Association of America express our deepest thanks to Insperity Inc. for funding this pilot through their grant program. We’re grateful for their support and commitment to improving quality of life in the communities they serve.” Insperity is a leading provider of scalable human resources solutions for small to mid-size companies.

More details about the pilot program, including eligibility guidelines and how to apply, will be announced in late June. Anyone living with sickle cell disease who is interested in participating in the pilot should contact either their local Sickle Cell Disease Association of America chapter or email MedicAlert at sicklecellpilot@medicalert.org.  

Afimmune joins the SCD C.A.R.E.S. Consortium

Dublin, Ireland, 19 June 2023: Afimmune, a clinical stage biopharmaceutical company developing novel rare disease therapeutics, today announced it has been invited to join the Sickle Cell Disease Association of America’s (SCDAA) Collaboration of Advocates for Research, Education and Science (C.A.R.E.S.) Consortium.

The mission of this initiative is to raise awareness about the importance of clinical trials and why it can help sickle cell patients to participate in them. The SCDAA along with other participating strategic pharma partners came together to help educate sickle cell patients on the potential treatment options available and to encourage more clinical trial participation.

Commenting on the update, Dr. Moayed Hamza, MD, Chief Medical Officer of Afimmune said,“With today being World Sickle Cell Day, it is timely to announce our membership to the SCDAA C.A.R.E.S. Consortium. I look forward to working with the other members to continue to drive improvement in the treatment landscape for patients suffering from sickle cell disease.”

SCDAA President and CEO, Ms. Regina Hartfield said, “We are thrilled that Afimmune has joined our expanding consortium and look forward to collaborating with their team to increase awareness within the sickle cell community.”

About Epeleuton

Epeleuton is 15-hydroxy eicosapentaenoic acid (15(S)-HEPE) ethyl ester, a novel synthetic fatty acid drug product. Epeleuton has been shown to have a unique dual mechanism of action for the treatment of SCD, targeting factors affecting severity, course of disease, and vaso-occlusive crisis risk.

Afimmune is developing Epeleuton for SCD due to its novel disease-modifying preclinical efficacy, first-in-class opportunity and a significantly reduced regulatory pathway. Epeleuton has received orphan drug designation for the treatment of SCD from the FDA and EMA

About Sickle Cell Disease
SCD is a group of inherited, progressive blood disorders carried by the β allele of the hemoglobin gene with an expected 30-year reduced life expectancy. The disease is characterized by abnormal polymerization of hemoglobin during oxygenation which results in the sickling of red blood cells. The disease is rare, with an estimated prevalence of only ~100,000 people affected in the US and ~52,000 people in the EU.

About Afimmune
Afimmune, headquartered in Dublin, Ireland, is a clinical stage drug discovery and development company working on new medicines to improve the quality of life for people with rare and inflammatory diseases.

About SCDAA

Sickle Cell Disease Association of America advocates for people affected by sickle cell conditions and empowers community-based organizations to maximize quality of life and raise public consciousness while advancing the search for a universal cure. The association and more than 50 member organizations support sickle cell research, public and professional health education and patient and community services. (www.sicklecelldisease.org)

Contact
investor.relations@afimmune.com  

CMO Speaks: Gene Therapy for SCD (Part 1)

CMO Speaks is a blog featuring the voices of SCDAA’s clinical leadership team. Part 1 of our Gene Therapy series was written by Dr. Lewis Hsu and Dr. Sri Lakshmi Jamalapur.

How can you cure an inherited disease like sickle cell disease? One way would be to develop some extremely effective medications that would alleviate all the symptoms. Another would be to transplant stem cells that produce red blood cells. But the ultimate way to cure genetic disease is with gene therapy.

Gene therapy is a relatively new treatment for sickle cell warriors. Researchers began conducting experimental studies (clinical research) in people about seven years ago. At first, the only approach to this treatment was a process called “gene addition” developed by the Boston-based pharmaceutical company bluebird bio. Soon, several other groups joined with other approaches for gene therapy. By early 2022, there were six competing organizations enrolling in gene therapy clinical research. They are listed in the table below.

There has been a lot of news recently regarding gene therapy. Earlier this year, The New York Times published this article about gene therapy and sickle cell disease, which highlighted the voices and experiences of advocates from around our community and brought renewed attention to this pioneering treatment. Two pharmaceutical groups say that they will bring their gene therapy clinical research results to the U.S. Food and Drug Administration (FDA) for possible approval as safe and effective in the first quarter of 2023 (highlighted green in the table).

At the same time, in late February, three other groups announced that they are closing their gene therapy clinical research studies (highlighted yellow in the table). Some people worry that seeing companies end their gene therapy studies is not good news for sickle cell disease. However, another way to look at these announcements is that the most fruitful studies are heading toward possible FDA approval. Less productive clinical research efforts are cutting their losses and being pruned away.

It’s like a gardener doing work in a little garden to keep the plants that are bearing fruit (the two groups that will bring their results to U.S. FDA) alive but removing those plants that have stopped growing (the groups that are closing their gene therapy clinical research). The wisdom of pruning down the less productive activity is recognized by many proverbs, including “if you run after two hares, you will catch neither,” or “have too many irons in the fire,” or “he who tries to sit on two chairs will end up sitting on neither,” or John 15:1 in the Bible. Drop-out studies are part of the healthy competitive process of research and investment.

Table 1: Current and upcoming studies of gene therapy in SCD. (adapted from Kanter and Falcon 2021; Hsu and Jamalapur 2022) N/A – not applicable; LVV – lentiviral vector; HbAT87Q – Hb with a single mutation conferring most of the antisickling effect of γ-globin; βAS3 – an antisickling β-globin containing 3 amino acid substitutions in the wild-type HBB; HbFG16D – modified HbF that increases affinity to α-globin to outcompete sickle mutated β-globin; AAV6 – Adeno-Associated Virus serotype 6.

Outside of gene therapy studies, promising sickle cell progress is everywhere. Researchers are developing innovative medications and studying stem cell transplants that could make a world of difference to warriors who are unable or unwilling to undergo gene therapy in the future. The clinical research process is fine-tuned to determine which treatments are safe and effective. Sickle cell warriors are encouraged to help push this research forward by participating in clinical trials.

Lewis Hsu, MD, PhD, is a pediatric hematologist who serves as director of the Sickle Cell Center and professor of pediatrics for the University of Illinois at Chicago. He has conducted sickle cell research, published over 50 peer-reviewed papers and co-authored “Hope and Destiny: The Patient and Parent’s Guide to Sickle Cell Disease and Sickle Cell Trait.” He currently serves as the SCDAA Chief Medical Officer.

Sri Lakshmi Jamalapur, MD, is a pediatric hematologist and the new leader for the Pediatric Sickle Cell Center at Children’s Hospital of Michigan in Detroit. She has collaborated with Dr. Hsu on health education and sickle cell advocacy since 2019.  

SCDAA Announces FY24 Legislative Priorities

Did you know that the federal government is poised to make some of the biggest decisions in sickle cell history right now? Current pending legislation would direct an unprecedented amount of funds to sickle cell treatment, research and support. Our FY24 Legislative Priorities, developed in collaboration with Sick Cells, outline these exciting bills and requests. Read through the document to learn more about these initiatives and call your representatives to ask for their support!  

June 19, 2023, is Officially Sickle Cell Awareness Day in Maryland!

Maryland Governor Wes Moore has signed a proclamation to make June 19, 2023, Sickle Cell Awareness Day! This recognition goes a long way in raising awareness about sickle cell disease, combating prejudices and lifting up our community. Thank you for supporting our cause and helping us to “Shine the Light on Sickle Cell Disease!”  

Women’s History Month: Dr. Marilyn Hughes Gaston

This #WomensHistoryMonth, we are highlighting prominent women who have made lasting contributions to the SCD community. #WomenInMedicine #WomensHistoryMonth 

Meet Dr. Marilyn Hughes Gaston, an internationally recognized leader in health care equality and sickle cell disease advocacy. Since 1976, she has dedicated her career to improving medical care for poor and minority families and has contributed to significant changes in the management of sickle cell disease. Despite facing racial and economic barriers, Dr. Gaston made history by publishing a groundbreaking study on sickle cell disease that led to a nationwide screening program for newborns. This initiative resulted in a significant reduction of morbidity and mortality in young children with the disease around the world. Let us acknowledge Dr. Gaston’s invaluable contributions to public health and unwavering commitment to bridging the gap of health disparities for all Americans.  

SCDAA names new board members

The Sickle Cell Disease Association of America, a national nonprofit membership organization that advocates for people affected by sickle cell, named Melissa Creary and Monica Mitchell to the association’s board of directors and Chris Ruffin Jr. to the Corporate Advisory Council.

Melissa Creary is senior director for the Office of Public Health Initiatives at the American Thrombosis and Hemostasis Network and an assistant professor in the department of health management and policy at the University of Michigan’s School of Public Health. Over a nine-year career at the Centers for Disease Control and Prevention in the Division of Blood Disorders, she helped create and lead the first national program and data collection system for sickle cell disease at the agency. Creary received her doctorate in interdisciplinary studies focusing on health, history and culture, her Master of Public Health and her bachelor’s degree in biology at Emory University in Atlanta, Georgia.

Monica Mitchell is founder and president of MERAssociates, an award-winning, woman- and minority-owned education, research and evaluation consultancy based in the Washington, D.C., area. She has been the principal investigator or co-principal investigator of National Science Foundation-funded grants totaling over $2,500,000. Prior to launching MERA, Mitchell was program officer at the National Science Foundation and managed portfolios in the Division of Research on Learning in Formal and Informal Settings and the Division of Undergraduate Education. She earned her Doctor of Education and master’s degree in engineering at Columbia University and her bachelor’s degree in economics at the University of California, Los Angeles.

The Sickle Cell Disease Association of America’s Corporate Advisory Council advises the association’s board of directors with board recruitment, fundraising and general advisory information.

Chris Ruffin Jr. is a senior news producer for ABC24 TV in Memphis, Tennessee, and the author of “Succeeding with Sickle Cell.” Before moving to Memphis, he worked in TV news in Columbus, Georgia, and Winston-Salem and Charlotte, North Carolina, where he won an Emmy for Best Morning Newscast. Recently, he partnered with Aflac, Red Cross and Children’s of Alabama to spread awareness about sickle cell disease and mentor other sickle cell patients. Ruffin earned his bachelor’s degree in broadcast journalism from Stillman College in Tuscaloosa, Alabama.

Women’s History Month: Dr. Yvette Francis-McBarnette

This #WomensHistoryMonth, we are highlighting prominent women who have made lasting contributions to the SCD community. #WomenInMedicine #WomensHistoryMonth 

Meet Dr. Yvette Francis-McBarnette, a Jamaican-born physician who specialized in treating children with sickle cell anemia. As one of the first Black women to graduate from the Yale School of Medicine, Dr. Francis-McBarnette was credited with successfully using antibiotics to treat children with sickle cell anemia 15 years before the effectiveness of those drugs were confirmed. Dr. Francis-McBarnette was also part of the White House advisory committee which made recommendations that led to the 1972 National Sickle Cell Anemia Control Act. Her contributions continue to inspire and impact the sickle cell community today. 

Women’s History Month: Dr. Helen M. Ranney

This #WomensHistoryMonth, we are highlighting prominent women who have made lasting contributions to the SCD community. #WomenInMedicine #WomensHistoryMonth

Meet Dr. Helen M. Ranney, physician and hematologist. Born in 1920, Dr. Ranney dedicated her work to researching blood disorders. Her groundbreaking work on sickle cell anemia included the first description of abnormal blood cell structure and genetic factors, earning her the Dr. Martin Luther King Jr. Medical Achievement Award in 1972. Dr. Ranney was also the first woman president of the Association of American Physicians. Let’s take a moment to celebrate Dr. Ranney for the work she has done on behalf of the sickle cell community!