Dr. Edward Donnell Ivy (left) is director of outreach and education at The Possibilities Clinic in Toronto, Ontario, Canada. He was a medical officer at the Health Resources and Services Administration and served the National Institutes of Health in Washington, D.C., where he worked on sickle cell clinical practice guidelines and on projects involving cardiovascular disease and diabetes. As someone living with sickle cell disease, Ivy has made it his life’s work to ensure patients have access to the resources they need. 

Dr. Crawford Strunk (center) is a pediatric hematologist with experience and expertise in sickle cell care. He serves on the Sickle Cell Disease Association of America Inc.’s Medical and Research Advisory Committee. Strunk is a staff physician and co-director of the Lifespan Sickle Cell Program at the Cleveland Clinic. He directed the pediatric sickle cell center and co-directed the adult sickle cell center at Toledo Children’s Hospital. He earned his doctorate at the State University of New York Upstate Medical University and graduated from Colby College. 

Adeyinka O. Ogunlegan (right) serves as vice president of government affairs and policy at EducationSuperHighway, a national nonprofit organization with the mission to close the broadband internet affordability gap. She served as chair of the Prince George’s Chamber of Commerce’s Legislative Action Committee as well as board president of The Arc Prince George’s County. Among her honors, Ogunlegan received The Maryland Daily Record’s VIP List and Leading Women awards and was recognized as a Prince George’s County Forty Under 40 honoree. She earned a law degree from Howard University and a bachelor’s degree from Georgetown University. 

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SCDAA Medical and Research Advisory Committee (MARAC) Statement: Pfizer’s Voxelotor (Oxbryta®) Withdrawal

9/27/24

What is the news?

Pfizer announced the withdrawal of voxelotor (Oxbryta®) from national and global markets on September 25, 2024.  Clinical research was also stopped. “Pfizer’s decision is based on the totality of clinical data that now indicate the overall benefit of OXBRYTA no longer outweighs the risk in the approved sickle cell patient population. The data suggest an imbalance in vaso-occlusive crises and fatal events, which require further assessment.” [1].

What is the reason?

The Pfizer statement mentions that worrisome new information came from three reports that link voxelotor with more pain and deaths:

1. A clinical research study of children with sickle cell disease and with higher risk of stroke (GBT440-032) had eight deaths in the voxelotor group compared to two deaths in the group without voxelotor. The study recruited 236 children (2y-15y) from Egypt, Ghana, Kenya, Nigeria, Oman, Saudi Arabia, USA and the United Kingdom.
2. Another clinical research study of adolescents and adults with sickle cell disease and leg ulcers (GBT440-042) had eight deaths on voxelotor. Eighty-eight patients at least 12 years of age were enrolled in Brazil, Kenya and Nigeria.
3. Monitoring reports from people taking voxelotor as a prescribed medication, not on a clinical research study. No numbers were listed.

Following standard rules when new risks are found, Pfizer voluntarily withdrew voxelotor from use worldwide, while further investigation is conducted. Pfizer also reports that they are halting manufacturing production and clinical research.

What if I have been doing well on voxelotor (Oxbryta®)?

The FDA has received questions about whether to allow some individuals living with sickle cell disease to continue voxelotor on a “compassionate use” basis. However, for now we cannot assume that “compassionate use” will be allowed.

Do I just throw away the voxelotor?

For people with sickle cell disease who have been on voxelotor and doing well, MARAC does not have evidence on what will happen when you stop taking the medication. There is a report that going from full-dose voxelotor to completely stopping taking the drug led to intense hemolysis (breakdown of red blood cells) and severe sickle cell problems within three days that injured the kidneys and other organs and required hospital care. Thus, many doctors are suggesting that people taper off voxelotor over a period of about two weeks. However, tapering is optional and not based on strong clinical evidence. We strongly urge individuals to discuss their options and next steps with their sickle cell health care professional.

What if I have unusual problems as I discontinue voxelotor?

Contact your sickle cell health care provider. Voxelotor acts by slowing down the breakage of red blood cells (hemolysis) and blocking the red cells from changing to the sickle shape. A possible problem is that if the hemolysis suddenly increases it may damage the kidneys. Your eyes will probably become more yellow (jaundiced), and your urine will be darker yellow-orange. Tell your doctor if you have brown urine (the color of cola). You will probably need to come in for lab tests and treatment. Immediately seek medical attention if you stop making urine even when you are drinking a lot of water. Comprehensive sickle cell centers and sickle cell health care providers are collecting reports from patients about any new problems experienced when they come off voxelotor.

What about my other sickle cell medicines?

This news is only about voxelotor.  Continue taking other medications as prescribed.

What are the alternatives to voxelotor?

In the USA, sickle cell disease severity can be reduced by three other medications approved by FDA:

• hydroxyurea (Droxia®, Siklos®),
• glutamine (Endari®), and
• crizanlizumab (Adakveo®)

Blood transfusions are used for selected problems in sickle cell disease, especially to prevent stroke.

• “top-off transfusions” or simple transfusions
• “exchange transfusions” or erythrocytapheresis

Hematopoietic stem cell transplant (also known as bone marrow transplant) is a cure that requires an:

• HLA-identical (fully-matched) sibling donor, or
• haploidentical (half-matched) related donor, or
• unrelated HLA-matched donor

Two gene therapy approaches can potentially cure sickle cell disease without having to find a donor.

• Casgevy^TM (CRISPR/Vertex) or
• Lyfgenia^TM (bluebird bio)

MARAC recommends making an appointment to discuss your sickle cell treatment plan with your sickle cell health care provider.

Is this the first time a medication has been withdrawn?

No. Other situations of medication withdrawal from the market have occurred.

• 2004: Merck withdrew rofecoxib (Vioxx) as an arthritis drug.
• 1997: FDA ordered Wyeth to remove the weight-loss medications fenfluramine (Pondimin) and a related drug, dexfenfluramine (Redux) from the market.
• 1961: Chemie withdrew thalidomide as an antinausea drug. 2006 thalidomide was approved for the treatment of plasma cell myeloma.

When will there be more updates?

We do not know. MARAC is following the news as it unfolds. The U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) regulations control some of the timing for release of information. SCDAA MARAC is continuing to monitor the situation and will update the community as soon as new information is released.

Additional Information — 9/30/24 Update

What details are publicly available about the deaths?
A document posted online by the European Medicines Agency has the most details that MARAC was able to find. “Notification to the CHMP/EMA Secretariat of a referral under Article 20 of Regulation (EC) 726/2004” electronically signed July 30, 2024, accessed Sept. 29, 2024. https://www.ema.europa.eu/en/documents/referral/oxbryta-article-20-procedure-notification_en.pdf The rules of EMA and FDA control release of information.

• Of the 8 deaths in the GBT440-032 randomized study of children with abnormal stroke risk: “Most of the fatal cases in the voxelotor group describe incidence of infection, including 3/8 who developed fatal malaria and 2/8 patients with sepsis.”
• Of the 8 deaths in the open-label GBT440-042 of voxelotor for leg ulcers: “In 4 cases, malaria was identified either the cause or contributing factor.”
• “The investigator and sponsor considered that none of the fatal cases were related to voxelotor in these studies.”

What information is still missing?
Quoting from the same EMA document from July 30, 2024:

• Some of the “case narratives” from GBT440-032 and GBT440-042 “are still not available, … and overall information provided to date is limited. However, given the fact that concerns due to possible immunosuppressive effects of voxelotor were raised at the time of the MA (with immunosuppressive effects observed in animal studies and decrease in WBC in clinical studies), and the study population in those studies partially overlap with the intended population based on the authorized indication, the findings from these emerging safety data need to be further reviewed, taking into account all available data, to determine whether there is an impact on the benefit-risk balance of Oxbryta in the authorized indication.”
• It is unclear how these deaths compare with the known vulnerability to infection and early death of sickle cell disease without treatment.

References

Pfizer statement, 9/25/24: https://www.pfizer.com/news/press-release/press-release-detail/pfizer-voluntarily-withdraws-all-lots-sickle-cell-disease

Pfizer letter to Health Care Providers, 9/26/24: https://webfiles.pfizer.com/dear-hcp-letter-oxbryta-us-final-092524?cmp=US-21234&campaign=US-21234&identitytype=account&tpn=1532773&LNK=GL_BD3-C2&mkt_tok=MTk1LVVMQy0yMDAAAAGV1F26wnPvgN6fKRyyOgU-kt_3Pa-xaL9I_wa6yP1ROToTo85YnZaSQ98g2cAj9j-cf9xvsubH4Rvs50AWKcAMrA4qw1nLMAsCEbu8-c6PeXuPhg

European Medicines Agency, July 2024: https://www.ema.europa.eu/en/medicines/human/referrals/oxbryta

FDA statement, 9/26/24: https://www.fda.gov/drugs/drug-safety-and-availability/fda-alerting-patients-and-health-care-professionals-about-voluntary-withdrawal-oxbryta-market-due

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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.

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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.

• The Democratizing Education for Sickle Cell Gene Therapy Project created materials with input from individuals with SCD and their families. Read their FAQs and visit their website.
• SCDAA has produced several videos on gene therapy. Watch the gene therapy masterclass and this webinar to learn more.
• OneSCDVoice hosts gene therapy resources on their Gene Therapy 101 page.
• The Dec. 8 FDA approval statement for Casgevy and Lyfgenia.

To learn more about Vertex’s Casgevy, visit casgevy.com. To learn more about bluebird bio’s Lyfgenia, visit my bluebird support.

Updated Dec. 14, 2023, 11:12 a.m. EST

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Alabama

• Sept. 5: Sickle Cell Awareness Month Blood Drive | Fairfield, AL
  Hosted by the Sickle Cell Disease Association of America, Central Alabama Chapter
• Sept. 12: Bessemer Recreation Center Sickle Cell Awareness Blood Drive | Bessemer, AL
  Hosted by the Sickle Cell Disease Association of America, Central Alabama Chapter
• Sept. 14: Strike for Sickle Cell Bowling Fundraiser | Mobile, AL
  Hosted by the Sickle Cell Disease Association of America, Mobile Chapter
• Sept. 17: Sixth Avenue Baptist Church Blood Drive | Birmingham, AL
  Hosted by the Sickle Cell Disease Association of America, Central Alabama Chapter
• Sept. 19: City of Birmingham Blood Drive, Sept. 19 | Birmingham, AL
  Supported by the Sickle Cell Disease Association of America, Central Alabama Chapter
• Sept. 25: Miles College Blood Drive | Sept. 25, Fairfield, AL
  Hosted by the Sickle Cell Disease Association of America, Central Alabama Chapter

California

• Sept. 13-16: 15th Annual SCD Educational Summit | Los Angeles, CA or virtual
  Hosted by the Cayenne Wellness Center

Connecticut

• Sept. 23: Walk Run Bike | New Haven, CT
  Hosted by the Sickle Cell Disease Association of America, Connecticut

Delaware

• Sept. 9: 12th Annual James L. Faucett III 3K Sickle Cell Awareness Run/Walk | Wilmington, DE
  Hosted by Tova Community Health

Florida

• Sept. 9: 42 Annual 5K Run | Tallahassee, FL
  Hosted by the Sickle Cell Foundation, Inc.

Georgia

• Sept. 9: Sickle Cell 5K Road Race/Walk, Concert and Vigil | Atlanta, GA
  Hosted by the Sickle Cell Foundation of Georgia
• Sept. 16: Sickle Cell Family Game Night | Atlanta, GA
  Hosted by the Sickle Cell Foundation of Georgia

Illinois

• Sept. 11: Swinging to Raise Sickle Cell Awareness | Chicago, IL
  Hosted by the Sickle Cell Disease Association of Illinois
• Sept. 23: Walk, Jog, Bike 5K | Chicago, IL
  Hosted by the Sickle Cell Disease Association of Illinois

Kansas

• Sept. 9: 13th Annual Fun Walk | Kansas City, KS
  Hosted by the Uriel Owens Sickle Cell Disease Association of the Midwest
• Sept. 22: Music Cantata | Location TBD
  Hosted by the Uriel Owens Sickle Cell Disease Association of the Midwest

Louisiana

• Sept. 23: Sickle Cell Fundraiser Radiothon | Shreveport, LA
  Hosted by the Sickle Cell Disease Association of America, Northwest Louisiana Chapter

Maryland

• Sept. 9: MSCDA Picnic | Baltimore, MD
  Hosted by the Maryland Sickle Cell Disease Association, Inc.

Michigan

• Sept. 9: The Annual Sickle Cell Matters Awareness Walk | Detroit, MI
  Hosted by the Sickle Cell Disease Association of America, Michigan Chapter

Minnesota

• Sept. 16: Sickle Cell-ebration of Hope | South Golden Valley, MN
  Hosted by the Sickle Cell Foundation of Minnesota

Missouri

• Sept. 9: Sickle Cell Stroll and 5k | Spanish Lake, MO
  Hosted by the Sickle Cell Association of St. Louis
• Sept. 18: Sickle Cell Roundtable | Virtual
  Hosted by the Sickle Cell Association of St. Louis
• Sept. 19 | Sickle Cell Documentary and Discussion | St. Louis, MO
  Hosted by the Sickle Cell Association of St. Louis

New Jersey

• Sept. 16: Sickle Cell Disease Awareness Walk | Atlantic City, NJ
  Hosted by the Sickle Cell Association of New Jersey
• Sept. 23: 4th Annual 2K Color Fun Walk | Trenton, NJ
  Hosted by the Sickle Cell Association of New Jersey
• Sept. 24: Shine the Light on Sickle Cell Bike Ride | Newark, NJ
  Hosted by the Sickle Cell Association of New Jersey

New York

• Sept. 16: 25th Anniversary Walk for Education and Awareness | New York, New York
  Hosted by the Sickle Cell Thalassemia Patients Network (SCTPN)

Ohio

• Sept. 16-17: Sickle Cell Sabbath Weekend | Ohio
  Hosted by the Ohio Sickle Cell and Health Association

Oklahoma

• Sept. 1-2: Women’s Wellness Retreat | Tulsa, OK
  Hosted by the Supporters of Families with Sickle Cell Disease, Inc.
• Sept. 23: 8th Annual 5K Walk/Run | Oklahoma City, OK
  Hosted by the Supporters of Families with Sickle Cell Disease, Inc. 

South Carolina

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

Tennessee

• Sept. 16: 15 Annual Mark Walden Memorial 5K | Memphis, TN
  Hosted by the Sickle Cell Foundation of Tennessee

Texas

• Sept. 2: Walk for Sickle Cell | Houston, TX
  Hosted by the Sickle Cell Association of Texas Marc Thomas Foundation
• Sept. 16: Sickle Cell Houston’s Amazing Race and 5K/1K | Virtual
  Hosted by the Sickle Cell Assocation of Houston

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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.

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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

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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.

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