Category Archives: News

SCDAA Statement on Recent Natural Disasters

SCDAA stands in solidarity with the warriors, advocates and community-based organizations who have been recently affected by natural disasters, including the devasting wildfires in California and the many hurricanes which hit southern states in the fall. As we pray for a speedy recovery, we send our sincere gratitude to the emergency response teams who worked tirelessly to contain the fires and provide hurricane assistance.

If you would like to send support to the Los Angeles and/or those impacted by other natural disasters, we encourage you to explore the below resources. We additionally urge you to remember the importance of donating blood, especially for sickle cell warriors. An emergency of this magnitude can make an already short supply worse. No matter where you are in the country, giving blood as soon as you are able could help someone in need.

CALIFORNIA RESOURCES

OTHER NATURAL DISASTER RESOURCES

If you would like to donate to support recovery efforts, you can make contributions to both wildfire relief and hurricane recovery through SCDAA member organizations using the follow links:

In Memory of Frank Reddick

It is with great sadness that SCDAA shares the news of the passing of Frank Reddick on Dec. 23, 2024. Frank was the president and CEO of the Sickle Cell Disease Association of Florida, an SCDAA member organization headquartered in Tampa. He was a steadfast sickle cell advocate and was the head of the Florida state sickle cell chapters. As a former Tampa city councilman, Frank was a respected leader in his local community. SCDAA sends our sincerest condolences to his family, friends and loved ones. Click here to read more about Frank.

Sickle Cell Disease is Not a Joke

This weekend’s Saturday Night Live skit about the recent historic approvals of potentially curative gene therapies for sickle cell disease is distasteful at best and harmful at worst. Earlier this month, the Food and Drug Administration approved groundbreaking new treatments that could change the lives of thousands. SNL chose to cast a spotlight on this news with a tone-deaf skit depicting a workplace Yankee Swap event in which one of the gifts is the “cure” for sickle cell disease. It is given to an African American character, who quickly trades it for a “Boogie Woogie Santa Claus” toy. The rest of the skit consists of the white characters trying to convince their two Black co-workers to choose the cure over the other Yankee Swap gifts. Their attempts are unsuccessful.

We are disappointed that Saturday Night Live chose to trivialize this landmark moment in history during their program. More than 100,000 people in the United States and millions globally are impacted by this devastating disease, and yet it is one of the few debilitating conditions that you will find people joking about on television. Earlier this year, sickle cell disease was the subject of a lame and insensitive attempt at humor on the HBO Max show Velma and, shortly thereafter, as a quasi-joke-insult by comedian D.L. Hughley on The Daily Show. Some may argue that these references are “just jokes,” but for those impacted by this disease, it is no laughing matter.

Jokes like these undermine the seriousness of this condition. Sickle cell disease (SCD) is an inherited blood disorder and rare disease that affects red blood cells. When these red blood cells become sickle-shaped, or crescent-shaped, they block blood flow to the affected part of the body, causing irreversible organ and tissue damage. When this happens, individuals with sickle cell can suffer from intractable, crippling acute pain called a “crisis” and are at elevated risk for strokes, damage to affected tissue, and all too often, an early death. In fact, a recent study showed the median age of death of those suffering from chronic sickle disease complications was only 43 years.

SNL’s treatment of race in the Yankee Swap skit also misses the mark. Part of the “humor” revolves around the common myth that only Black people can have sickle cell disease. While it does disproportionately impact the Black community, sickle cell does not discriminate. People of all ethnic backgrounds can inherit the disease. In the United States, Hispanic and Latino populations have the second highest incidence, but Asian, Indian, Native American and – yes – White people, can all be born with the disease. On a global scale, sickle cell disease affects people from countries around the world, including Italy, India, the United Kingdom and Jamaica. One doesn’t develop sickle cell disease, nor can one “catch it.” Individuals are born with it, and there is no universal cure.

Why are we joking about a disease as serious as this one? Many people don’t understand the devastating reality of the condition. The onset of sickle cell pain is sudden and debilitating. A pain crisis is relentless and can last for hours or for days. It has been described as feeling like you are walking on hot coals or like shards of glass are traveling through your veins. Far too often, when individuals living with sickle cell disease, or “warriors” as they call themselves, are in crisis and seek medical care in some emergency departments, they face long waiting periods, are accused of exaggerating symptoms for attention, and far, far too often are characterized and treated as if they are drug seekers.

For physicians who are knowledgeable about sickle cell disease and experienced in caring for those living with it, their ability to prescribe the very drugs that will help their patients is hampered by current federal regulations put in place to address the opioid crisis thus limiting how these drugs can be used in cases such as sickle cell. Layer on issues of health care inequity, discrimination and limited access to consistent, comprehensive quality care and the word “crisis” takes on new meaning.

Community-based organizations, such as the 50-plus members of the Sickle Cell Disease Association of America, Inc., spanning 29 states, are on the ground and focused on providing support, resources, and services to serve more than 500,000 children and adults living with or impacted by sickle cell disease.

Sickle cell disease also puts a strain on caregivers and family members, who must fit trips to the emergency room, doctors’ appointments and sick days into the rigors of daily life. Parents of children with sickle cell may lose wages, promotion opportunities or jobs as they try to support their family while attending to pain crises and their child’s care. This pressure can cause personal and professional instability, compromise mental health and wellness, and, in too many cases we have seen, lead to homelessness.

It is for all of the above, and more, that the Sickle Cell Disease Association of America, Inc., condemns the use of sickle cell disease as a punchline. It demeans and ridicules a condition that people are born with and from which they will face devastating health challenges throughout their lifetimes.

Stereotypes and misinformation reinforced by thoughtless comedy have real-life consequences. Sickle cell patients struggle daily to be taken seriously—in school, at work and even playing sports. As we work to change the perception of sickle cell and increase education surrounding this condition, insensitive and inappropriate jokes like these demean, marginalize and disrespect those living with the disease. They work against progress and contribute to the spread of misinformation. As a society, we must do better and treat rare diseases and the people who live with them with the respect they deserve.

Sickle cell is not a joke.

Gene Therapy is Approved!

We are very excited to share that today, Dec. 8, the Food and Drug Administration approved two gene therapies to treat sickle cell disease! These potentially curative therapies are the first treatments of their kind available to individuals with SCD. We are heartened by this approval and are proud to support our community during this milestone moment. SCDAA will be releasing a full statement and additional information for patients and caregivers soon. To learn more about these approvals, click here.

 

 

SCDAA Statement About Gene Therapy Approval

The Food and Drug Administration (FDA) approved two cell-based gene therapies for sickle cell disease (SCD), Casgevy from CRISPR/Vertex and Lyfgenia from bluebird bio. These are the first treatments of their kind available to individuals with SCD in the United States. SCDAA welcomes the approval of these potentially curative therapies which mark major advances in the treatment of sickle cell disease; however, there are valid concerns about accessibility and the potential for adverse effects.

Dr. Lewis Hsu, chief medical officer of the Sickle Cell Disease Association of America Inc. said:
We at the Sickle Cell Disease Association of America Inc. celebrate that two gene therapies are approved for sickle cell disease by the FDA today, Dec. 8. This double milestone was a long time coming, and sickle cell disease now joins the ranks of other genetic diseases with gene therapy treatments. Sickle cell disease was called “the first molecular disease” about 70 years ago, and a gene therapy treatment was predicted for sickle cell disease in the 1950s when DNA was first described.

These two gene therapies mark a big step forward for sickle cell disease research and treatment. The results of the clinical trials are very impressive. The patients selected had a lot of pain (two or more vaso-occlusive crisis pain hospitalizations per year for two years). The data show that, after gene therapy was administered, nearly all patients were free of hospitalizations for vaso-occlusive crises for at least nine consecutive months. The patients’ health-related quality of life also improved in every way: physically, emotionally, socially and functionally with both gene therapies.

Regina Hartfield, president and CEO of the Sickle Cell Disease Association of America Inc. said:
Gene therapy is an exciting and potentially curative addition to the treatments available to sickle cell warriors. This is a historic milestone, but everyone may not be eligible for gene therapy. We must continue to move forward with research to ensure that there is a solution for every member of our community.

Is gene therapy a cure for sickle cell disease?
The Sickle Cell Disease Association of America Inc. recognizes gene therapy as a “potentially curative” therapy. The treatment is so new that more data is needed to understand its impact and patient prognosis. Additionally, the word “cure” suggests a simple solution that does not reflect the reality of these therapies. Even after completing treatment, the FDA recommends 15 years of patient monitoring for health issues.

What is the treatment like?
The patient journey will be similar for both Casgevy and Lyfgenia. Gene therapy is administered during a one-time infusion; however, there are steps that patients must take to prepare for the treatment. First, the patient’s care team will collect stem cells, which are the progenitors of red blood cells, from their body. Then, those cells will be treated in a lab. The patient will undergo chemotherapy to remove the original, abnormal stem cells from the bone marrow. After this process is complete, the treated stem cells are injected back into the patient through an intravenous process like a transfusion (not surgery). The whole procedure takes about a year. It is similar to autologous bone marrow transplantation, as there is no need to find a donor of stem cells.

What is the difference between the two therapies?
The two gene therapy strategies are scientifically different. Casgevy is gene editing, the first of its kind, and Lyfgenia uses gene addition. Both gene therapy strategies have about the same patient journey and potential issues: access, cost, infertility, unknown possibility of organ damage and unknown long-term effects.

How effective is it?
Gene therapy provides a significant reduction in acute episodes of sickle cell pain within a few years of administration. More years of follow-up will be needed to determine whether it will also reduce the organ damage of sickle cell disease and if the stem cells treated with continue to produce non-sickling red blood cells for the rest of the person’s life, or if the stem cells die off over a certain number of years. Currently the treatment requires chemotherapy, which means there are also concerns about chemotherapy-associated complications, such as infertility or secondary cancer.

When will gene therapy be available for use?
Gene therapy will likely be available in early 2024.

Who is eligible?
Casgevy and Lyfgenia are approved for people ages 12 and up. Sickle cell disease SS and S-beta-zero-thalassemia are eligible. The FDA indicates that sickle cell disease SC is not included. Additionally, individuals may also not be able to receive gene therapy if they have:

  • A recurring viral infection
  • Significant organ damage

Where is gene therapy administered?
Individuals with SCD can receive gene therapy at existing bone marrow treatment facilities with sickle cell expertise, which may pose accessibility issues to patients. SCDAA encourages gene therapy centers to partner with sickle cell centers, such as in the National Alliance of Sickle Centers, so that there is expertise to monitor for sickle cell organ damage.

How much will it cost? Will insurance cover gene therapy?
Gene therapy treatments are produced through expensive, highly technical processes. Casgevy is estimated to cost $2.2 million, and Lyfgenia is estimated to cost $3.1 million. However, the high price should be worthwhile as the savings in lifelong care may exceed the one-time cost of gene therapy. FDA-approved high-cost medications come with insurance barriers and rules that are not evidence-based.

What do these approvals mean for people living with sickle cell?
These approvals are expected to be life-changing for many and usher in a new age of treatment for sickle cell disease. Until now, the only way to cure sickle cell disease was through a bone marrow transplant, which is not a widely accessible option because it requires a matched bone marrow donor. Gene therapy does not require a donor; therefore, it has the potential to be a more widely available treatment.

What does it mean for sickle cell treatment in the future?
Casgevy and Lyfgenia are the first gene therapy treatments approved by the FDA for sickle cell disease. They open the door for other gene therapies to gain approval and help advance research into other potentially curative treatments. At the same time, there are concerns that these approvals will create an increase in competition for health care resources that could make it difficult to access other forms of treatment outside of gene therapy. Many people will not be eligible to receive gene therapy. To provide the highest quality of care to these individuals, we need to continue research into a variety of treatment options beyond gene therapy.

What are the implications for the medical community at large?
More broadly, Casgevy is the first FDA-approved CRISPR gene editing therapy for a genetic disease. This could have wide-reaching impacts for individuals with other conditions like cystic fibrosis, Tay-Sachs disease and others.

Where can I find more information?
Research into Casgevy and Lyfgenia specifically is ongoing; however, there is a wide variety of information about gene therapy for sickle cell disease available as it relates to clinical trials.

Sickle Cell Awareness Month 2023 Events

All across the country, our member organizations are hosting exciting events for Sickle Cell Awareness Month this September. Check out the list below to find an event near you.

Alabama

California

Connecticut

Delaware

Florida

Georgia

Illinois

Kansas

Louisiana

Maryland

Michigan

Minnesota

Missouri

New Jersey

New York

Ohio

Oklahoma

South Carolina

  • Sept. 30: 5K Race | Columbia, South Carolina
    Hosted by the James R. Clark Memorial Sickle Cell Foundation

Tennessee

Texas

 

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