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The Kidney Cancer Association and SCDAA launch KNOW & TELL

New campaign to raise awareness about connection between sickle cell trait and deadly form of kidney cancer.

The Kidney Cancer Association (KCA) and the Sickle Cell Disease Association of America, Inc. (SCDAA) are partnering to launch the “KNOW & TELL” initiative today to raise awareness about sickle cell trait (SCT) and its link to a rare, aggressive type of kidney cancer called renal medullary carcinoma (RMC).
The year-long initiative seeks to promote the early identification of RMC by encouraging people to know their SCT status and inform their family and health care providers about the connection between SCT and RMC. Being aware of both SCT status and the rare RMC symptoms could help in identifying RMC early, obtaining appropriate care and increasing positive outcomes.
RMC typically affects young Black men under age 30 and about half of those diagnosed do not survive beyond 13 months.1 RMC makes up less than 1% of all kidney cancers but is almost exclusively found in people who carry SCT, a usually benign condition that can occasionally cause symptoms similar to sickle cell anemia.
“One of my key platforms is bringing greater awareness to rare types of kidney cancer and supporting research into treatments for underrepresented populations,” said the KCA’s Medical Director Sallie McAdoo, MS, CGC. “We’re proud to partner with SCDAA to bring greater awareness to RMC and its association with SCT through KNOW & TELL in the hope of helping those who are at risk identify it sooner, increase research into the condition, and improve outcomes.”
The SCDAA is an advocacy organization focused on education and advancing research for sickle cell conditions.
“We are excited to be partnering on this vital strategic initiative to increase awareness about SCT and one of the many possible complications tied to the trait,” said Beverley Francis-Gibson, president and CEO of the Sickle Cell Disease Association of America. “This campaign shows that knowledge is power and can have a big impact on care.”
Newborns are routinely screened for SCT however, those who carry the trait may not show symptoms and are therefore often not told they have it. Low awareness about SCT status and the lack of understanding about the link between SCT and RMC contribute to late-stage RMC diagnoses and shortened life expectancy.
“The RMC community may be smaller but is no less important than that of other cancer types and they are passionate about advocating for greater awareness about the disease and access to clinical trials for the opportunity to better understand and treat this rare cancer,” said Gretchen E. Vaughan, KCA’s president and CEO. “Partnering with the SCDAA for this initiative is the first step toward bridging the knowledge gap about the link between RMC and SCT.”
KNOW & TELL launches on June 18, following World Kidney Cancer Day on June 17, and in advance of World Sickle Cell Day on June 19.  Learn more at www.kidneycancer.org/know-and-tell.

1. National Association for Rare Disorders

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About the Kidney Cancer Association
The Kidney Cancer Association is a global community dedicated to serving and empowering patients and caregivers, and leading change through advocacy, research, and education in order to be the universal leader in finding the cure for kidney cancer. Founded in 1990 by Eugene P. Schonfeld and a small group of patients and doctors in Chicago, Illinois, the KCA has grown into an international non-profit organization based in Houston, Texas. The KCA promotes scientific advances through two annual research symposiums and a robust grant program, participates in legislative advocacy, and seeks to be a source of education and resources for patients, caregivers, and anyone impacted by kidney cancer.
Contact: Radha Chitale, Director of Communications. 847.332.1051 ext. 113 | rchitale@kidneycancer.org
About the Sickle Cell Disease Association of America
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  

The Hibiscus Study is Enrolling Volunteers

Study Title:
An adaptive, randomized, placebo-controlled, double-blind, multi-center study of oral FT-4202, a pyruvate kinase activator, in patients with sickle cell disease (SCD).
Study Description:
The Hibiscus Study™ is evaluating the safety and effectiveness of an investigational medication compared with placebo (inactive drug) in people 12 to 65 years of age with SCD. The study is evaluating if the investigational medication can improve anemia and reduce the number of pain crises per year.
Eligibility Criteria:
Eligible participants must:

  • Be 12 to 65 years of age
  • Have a confirmed diagnosis of SCD
  • Have had at least 2 pain episodes (vaso-occlusive crises) within the past 12 months
  • Not have had more than 10 vaso-occlusive crises within the past 12 months
  • Be willing and able to follow all study requirements and study visit schedules

There are additional eligibility requirements, which the study doctor will explain to you. To learn more about study participation, and to see if you or someone you know may qualify, visit HibiscusSC.com or call 844-608-0808 to complete the prescreening questionnaire.  

Walk with the Stars to support Sickle Cell Disease Association of America

Sickle Cell Disease Association of America will hold the eighth annual Walk with the Stars fundraiser, where participating teams and individuals can track their walk, run, dance or movement steps while raising funds to support the association in preventing the complications of sickle cell disease through awareness, education, advocacy and research.
Walk with the Stars will kick off May 22 at 10 a.m. with a Facebook Live event and culminate in a virtual Walk with the Stars celebration July 10 from 9 to 11 a.m., recognizing participants’ steps and success.
“Join family members, loved ones, friends and co-workers for Walk with the Stars to help support a universal cure for sickle cell disease,” said Beverley Francis-Gibson, president and CEO of Sickle Cell Disease Association of America. “Walking, running and moving for Walk with the Stars not only benefit participants but will change the lives of those affected by sickle cell disease. Together we can break the sickle cycle.”
Sickle Cell Disease Association of America has raised a total of over $500,000 through Walk with the Stars to support better treatments and improvements in the quality of health for individuals, families and communities affected by sickle cell disease.
To learn more and register for Walk with the Stars, visit bit.ly/SCDAAwalk2021. Sponsorships are available by emailing bfrancis-gibson@sicklecelldisease.org.  

MARAC Statement on Gene Therapy & Bone Marrow Therapies


Please note: A previous version of this advisory incorrectly stated that MARAC is aware of three cases of
leukemia and other cancer-like conditions in patients who have undergone gene therapy. MARAC is only aware
of two such cases. The below statement has been updated accordingly.
May 15, 2021 – The Sickle Cell Disease Association of America’s Medical and Research Advisory Committee is aware of two cases of leukemia or other cancer-like conditions in people who have undergone gene therapy for sickle cell disease. This is more than would be expected given the small number of individuals who have undergone gene therapy to date. It is unclear why cancer developed in these individuals. The mission of SCDAA is to promote the best interests of individuals living with SCD. Accordingly, SCDAA MARAC urges action in three directions:
1. Informing patients, families and others about the current understanding of cancer risk as it pertains to sickle cell disease in a clear and accurate way.

  • Educate individuals with SCD who are participating in gene therapy (gene editing as well as lentiviral gene transfer) and other stem cell transplants (bone marrow therapies). Ensure that the consent forms reflect the recent findings and potential risks of leukemia and MDS.
  • Educate the greater SCD community by providing cancer risk estimates in the general population of individuals with SCD treated with other types of therapies.
  • Enlist experts in cancer genetic risk communication (e.g., NCI) and SCD stakeholders to develop education about risks of cancer in SCD.

2. Funding for research to better understand and address cancer-related risks of gene therapy and stem cell transplant (bone marrow therapies) in sickle cell disease.

  • Understand whether sickle cell disease intrinsically involves an increased risk of cancer; and, if so, what causes the increased risk.
  • Identify people living with sickle cell disease who are at increased risk for the development of cancer as they undergo “bone marrow therapies.”
  • Identify components of “bone marrow therapies” that increase risk of cancer.
  • Bring together scientific experts from the fields of SCD, cancer, stem cell transplant and gene therapy to answer these questions.

3. Funding for and creation of a way to collect information about cancer-related risks from all studies that involve “bone marrow therapies” for sickle cell disease globally.
To view a full list of MARAC members and to download this statement, click here.
 

GBT and SCDAA Kick Off “Lift Every Voice to Shine the Light on Sickle Cell”

May 04, 2021 at 8:00 AM EDT
SOUTH SAN FRANCISCO, Calif. and HANOVER, Md., May 04, 2021 (GLOBE NEWSWIRE) —In recognition of World Sickle Cell Day, which falls on June 19, 2021,Global Blood Therapeutics, Inc. (GBT) (NASDAQ: GBT) and the Sickle Cell Disease Association of America, Inc. (SCDAA) today launched “Lift Every Voice to Shine the Light on Sickle Cell” – a contest that will feature original spoken word pieces to raise awareness of sickle cell disease (SCD). The contest, which aims to elevate the voices and experiences of people living with SCD and their caregivers, is presented by Sickle Cell Speaks, GBT’s SCD education campaign that highlights authentic stories of those living with SCD to dispel misconceptions about the disease.
“People with sickle cell disease possess an incredibly rich and diverse array of creative abilities, often used to express the physical and emotional burden of living with this complex and devastating disease,” said Beverley Francis-Gibson, M.A., president and CEO of the SCDAA. “Celebrating these voices is critical as we work to overcome the legacy of stigma and misinformation that have a direct impact on health outcomes. We are proud to partner with GBT to shine the light on the challenges that SCD warriors face with strength and resilience.”
Spoken word poetry is an art form rooted in traditions of storytelling to convey compelling messages and personal experiences. People living with SCD and their caregivers are invited to sign up to submit videos of themselves performing original spoken word pieces about their experience with SCD. To learn more about the contest details and how to submit a video, please email patientevents@sicklecellspeaks.info or visit www.facebook.com/SickleCellSpeaks. Participants must sign up by May 24, 2021, and be U.S. residents. For each eligible submission received, GBT will donate $100 to the SCDAA, up to a total donation of$5,000. The contest winner will be featured in a GBT event at the SCDAA Annual Convention in October 2021. “Shine the Light on Sickle Cell” is a collaboration between SiNERGe and SCDAA.
“GBT is proud to partner with SCDAA to recognize the many inspiring voices within the sickle cell community who have rallied for progress in the face of tremendous health and societal challenges over the last year,” said Jung E. Choi, chief business and strategy officer, and head of patient advocacy and government affairs at GBT. “People with SCD suffer from a terrible, life-threatening disease that is made worse by being subjected to racial bias. We reiterate our commitment to shining a brighter light on the inequities these patients encounter and will continue working with all our partners to ensure access to the high-quality care patients deserve.”
Performances by the spoken word contest winner and finalists will be featured in a virtual event on Friday, June 18, 2021, at 4:00 p.m. PT and Saturday, June 19, 2021, at 12:00 p.m. PT on the Sickle Cell Speaks Facebook and Instagram pages. The event will be hosted and feature performances by three SCD advocates who are passionate about using spoken word to educate and inspire change:

  • Charly Richard, musician and writer
  • DeMitrious Wyant, musician and entrepreneur
  • Candis St. John, nurse and poet

About Sickle Cell Disease
Sickle cell disease (SCD) affects an estimated 100,000 people in the United States,1 an estimated 52,000 people inEurope,2 and millions of people throughout the world, particularly among those whose ancestors are from sub-Saharan Africa.1 It also affects people of Hispanic, South Asian, Southern European, and Middle Eastern ancestry.1 SCD is a lifelong inherited blood disorder that impacts hemoglobin, a protein carried by red blood cells that delivers oxygen to tissues and organs throughout the body.3 Due to a genetic mutation, people with SCD form abnormal hemoglobin known as sickle hemoglobin. Through a process called hemoglobin polymerization, red blood cells become sickled – deoxygenated, crescent-shaped, and rigid.3-5 The sickling process causes hemolytic anemia (low hemoglobin due to red blood cell destruction) and blockages in capillaries and small blood vessels, which impede the flow of blood and oxygen throughout the body. The diminished oxygen delivery to tissues and organs can lead to life-threatening complications, including stroke and irreversible organ damage.4-7
About SCDAA
Sickle Cell Disease Association of America(SCDAA) 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. Visitwww.sicklecelldisease.org.
About Global Blood Therapeutics
Global Blood Therapeutics(GBT) is a biopharmaceutical company dedicated to the discovery, development, and delivery of life-changing treatments that provide hope to underserved patient communities. Founded in 2011, GBT is delivering on its goal to transform the treatment and care of sickle cell disease (SCD), a lifelong, devastating inherited blood disorder. The company has introduced Oxbryta®(voxelotor), the first FDA-approved treatment that directly inhibits sickle hemoglobin polymerization, the root cause of red blood cell sickling in SCD. GBT is also advancing its pipeline program in SCD with inclacumab, a P-selectin inhibitor in development to address pain crises associated with the disease, and GBT021601 (GBT601), the company’s next-generation hemoglobin S polymerization inhibitor. In addition, GBT’s drug discovery teams are working on new targets to develop the next wave of treatments for SCD. To learn more, please visit www.gbt.com and follow the company on Twitter @GBT_news.
References

  1. Centers for Disease Control and Prevention website. Sickle Cell Disease (SCD). https://www.cdc.gov/ncbddd/sicklecell/data.html. Accessed June 3, 2019.
  2. European Medicines Agency.https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu3182125. Accessed June 12, 2020.
  3. National Heart, Lung, and Blood Institute website. Sickle Cell Disease. https://www.nhlbi.nih.gov/health-topics/sickle-cell-disease. Accessed August 5, 2019.
  4. Rees DC, et al. Lancet. 2010;376(9757):2018-2031.
  5. Kato GJ, et al. Nat Rev Dis Primers. 2018;4:18010.
  6. Kato GJ, et al. J Clin Invest. 2017;127(3):750-760.
  7. Caboot JB, et al. Paediatr Respir Rev. 2014;15(1):17-23.

GBT Contact Information:
Steven Immergut
650-410-3258
simmergut@gbt.com
SCDAA Contact Information:
Emma Day
eday@sicklecelldisease.org
Kyri Jacobs
kjacobs@sicklecelldisease.org

Download PDF | Visit the GBT Website  

SCDAA partners with Sickle Cell Community Consortium

Sickle Cell Disease Association of America partnered with the Sickle Cell Community Consortium to advocate for legislation benefiting people with sickle cell disease and their families. The partnership includes collaboration on the association’s annual advocacy day initiatives, meetings and trainings and in developing federal legislative priorities.
“Sickle Cell Disease Association of America and the Sickle Cell Community Consortium share the same mission of improving the lives of people with sickle cell disease,” said Beverley Francis-Gibson, president and CEO of the Sickle Cell Disease Association. “We’re excited to work with the consortium, which brings a range of organizations, advocates and advisers that will help us achieve our legislative goals together.”
A nonprofit formed in 2014, the Sickle Cell Community Consortium consists of sickle cell community organizations, patient and caregiver advocates, community partners and medical and research advisers working together to represent people with sickle cell disease. The consortium identifies and implements strategies and partnerships to address needs in the sickle cell community.
Sickle Cell Disease Association of America’s annual advocacy day, supported by the partnership between the association and consortium, generates public awareness of sickle cell disease and kickstarts momentum to push for legislative reform. The day provides training, resources and guidance to participants interested in advocacy work.  

SCDAA Launches New Educational Materials to Support Children’s Blood Transfusion

Sickle Cell Disease Association of America, Inc. (SCDAA) and Hemanext Inc., a privately held medical technology company dedicated to improving the quality, safety, efficacy and cost of red blood cell (RBC) transfusion therapy, today announced the launch of new educational material to help SCDAA deliver on its mission and meet its goals. Hemanext has sponsored the creation of a set of educational materials, one for a child and one for a caregiver, to educate on blood transfusions. This collaboration is part of SCDAA and Hemanext’s partnership, which began in 2020.
Sickle cell disease (SCD) affects 100,000 individuals in the United States, disproportionately affecting African Americans. SCD occurs in about one in 365 Black or African American births.1 The genetic disease is associated with serious, life-threatening complications, including stroke and acute chest syndrome (ACS).2,3 As result, people with SCD often require chronic red blood cell transfusions,2 which for some patients is a life-saving therapy.3
Children and parents may experience anxiety because they are unaware of the process to receive a blood transfusion. The goal of these two educational materials is to inform and empower patients and their caregivers about this important therapy. SCDAA will make these the resources available to the sickle cell community.
“During a review of our currently available educational resources, we identified the opportunity to help young sickle cell warriors prepare for their transfusions,” said SCDAA President and CEO Beverley Francis-Gibson. “We appreciate that Hemanext has stepped up to help us fill this information need and make a difference breaking the sickle cycle.”
“It is a privilege to continue our partnership with Ms. Francis-Gibson and her dedicated team at SCDAA, the premier sickle cell organization,” said Hemanext President and CEO Martin Cannon. “We are committed to helping SCDAA achieve its mission and enhance the lives of members of the sickle cell community.”
“As we enter the second year of our alliance, we will continue to look for ways to support SCDAA, patients, and families during these difficult times,” said Alex Marichal, VP, Marketing, Hemanext. 
ABOUT SCDAA
SCDAA’s mission is to advocate for people affected by sickle cell conditions and empower community-based organizations to maximize quality of life and raise public consciousness while advancing the search for a universal cure. Visit www.sicklecelldisease.org.
ABOUT HEMANEXT
Hemanext’s mission is to help patients enjoy healthier lives through safer transfusions. Hemanext’s technology is a processing and storage system that is designed to remove the fuel for oxidative damage to red blood cells. Hemanext is focused on supporting clinicians and healthcare practitioners who prescribe life-saving RBC transfusions to their patients. Visit Hemanext.com to learn more.

  1. Data & Statistics on Sickle Cell Disease. Centers for Disease Control and Prevention. https://www.cdc.gov/ncbddd/sicklecell/data.html. Accessed March 19, 2021.
  2. Understanding Sickle Cell Disease. American Society of Hematology, 2019. Available at https://www.hematology.org/education/clinicians/guidelines-and-quality-care/clinical-practice-guidelines/sickle-cell-disease-guidelines. Accessed March 23, 2021.
  3. Chou ST, Fasano RM. Management of Patients with Sickle Cell Disease Using Transfusion Therapy: Guidelines and Complications. Hematology/oncology Clinics of North America. 2016 Jun;30(3):591-608. DOI: 10.1016/j.hoc.2016.01.011.

 

Temporary Suspension of Clinical Trials

 
March 1, 2021 – The Sickle Cell Disease Association of America’s Medical and Research Advisory Committee (MARAC) is aware of the announcement on February 16, regarding the temporary suspension of bluebird bio clinical trials of LentiGlobin Gene Therapy for Sickle Cell Disease and the pause of all commercial use of bluebird bio European gene therapy.
Additionally, on February 22, the National Heart, Lung, and Blood Institute (NHLBI) temporarily suspended their unrelated gene therapy trial — Pilot and Feasibility Study of Hematopoietic Stem Cell Gene Transfer for Sickle Cell Disease at Boston Children’s Hospital. The NHLBI stated that this temporary suspension was, “out of an abundance of caution” despite having no indications of harm.
On February 23, another gene therapy trial, Gene Transfer for Patients with Sickle Cell Disease, was also paused by the sponsor Aruvant.
MARAC has investigated the situation and met with bluebird bio to discuss the information available to the public. In the bluebird bio study, two patients developed blood cancer, and a third patient is under investigation for a related problem called myelodysplastic syndrome. The details of these patients are being examined by their doctors and the bluebird bio sponsors. Investigations are trying to determine whether the blood cancer can be linked to the gene therapy vector, the chemotherapy preparation for gene therapy, or damage of the host stem cell. No events occurred in the other clinical trials.
We value patient trust and patient concerns. SCDAA tries to express the voice of people living with sickle cell disease (SCD), and MARAC supports this mission with biomedical expertise. MARAC is monitoring developments and will continue to communicate findings to the SCD community. Nearly all the members of MARAC are involved in research to help those with SCD, and some who participated in developing this advisory statement are gene therapy investigators. The MARAC members with a potential conflict of interest due to their involvement in gene therapy clinical trials are in the full statement linked below.
MARAC acknowledges that there has been a history of clinical investigations that were unethical, including the infamous Tuskegee syphilis study, but this past week’s events highlight that clinical research is no longer in that era. The modern safeguards for clinical research are working. Preplanned “stopping rules” triggered a “pause” of enrollment by bluebird bio when unusual and concerning events occurred. The Data Safety and Monitoring Board for the NHLBI gene therapy study followed “out of an abundance of caution,” as did the Aruvant study. There were public announcements, and an intensive investigation is now underway to gather more information. The participants in the studies are being notified and are receiving appropriate medical care from the investigators.
Clinical research has been and continues to be the path for progress to improved SCD survival and quality of life. MARAC celebrates the decades of clinical research studies on which the progress in sickle cell care that we have today has been built — including penicillin, hydroxyurea, stroke screening and new medications.
SCDAA honors the SCD warriors who volunteer in clinical research. They have given their time so that others may benefit from new future treatments and cures. We pay tribute to all of those who have been lost to SCD, and we know many have died too young.

 

Black History Month: Dr. Roland Scott

For our final #SCDHistoryHighlight of Black History month, we are excited to reflect on the life and work of Dr. Roland Scott. Dr. Scott has been celebrated as the “father of sickle cell disease” in the United States, and completed groundbreaking research as he advocated for his patients during a time of intense discrimination.
Dr. Scott was born in 1909 and graduated with his medical degree from Howard University in 1934. He spent the majority of his career in the pediatrics department of Howard University. During this time, he began to notice the high number of African American children in the emergency room experiencing sickle cell symptoms and complications. Dr. Scott was a trained allergist, but he switched his focus to help improve treatment for this misunderstood disease. A compassionate pediatrician, Dr. Scott held office hours in the evenings during which he would see African American children and families who were discriminated against and denied access to medical treatment.
Dr. Scott would go on to publish hundreds of articles on sickle cell disease during his time at Howard University. Although he did not specialize in hematology, in 1948 Dr. Scott published a paper on the sickling of red blood cells in newborns. This paper paved the way to a better understanding of sickle cell disease and laid the groundwork for newborn screening as we know it today.
In addition to being a dedicated researcher, Dr. Scott was a fierce advocate for sickle cell disease. His advocacy played a large role in the federal government’s passing of the Sickle Cell Anemia Control Act of 1971, which ensured nationwide funding for the research and treatment of sickle cell disease. In 1972, Dr. Scott founded the Howard University Center for Sickle Cell Disease, which continues to conduct research and provide care to sickle cell patients today. Dr. Scott’s work and advocacy changed the landscape for sickle cell disease, and we owe so much of our capability to treat and understand sickle cell to his efforts.

 

Black History Month 2021: Dr. Angella Dorothea Ferguson

We’re excited to continue our celebration of Black history this week by highlighting the life and work of Dr. Angella Dorothea Ferguson, a pediatrician and sickle cell pioneer. Dr. Ferguson’s research changed the landscape for sickle cell diagnosis in children and made a huge impact on how we identify and treat SCD to this day.
Dr. Ferguson was born in 1925 and received her bachelor’s and medical degrees from Howard University. After graduation, she began work as a medical researcher at Howard University’s School of Medicine, where she aimed to gather data correlating the height and weight of children with age. While completing this research, she discovered that a large number of African American children suffered from sickle cell disease, which, at the time, was a fairly unknown condition. She changed gears and committed her time to understanding how the disease presents itself in children. In doing so, she became one of the first researchers to dedicate her studies to sickle cell.
Dr. Ferguson’s research had lasting impacts on how sickle cell is diagnosed and treated. She developed a blood test to diagnose the disease in infants, and her test is the standard in most states to this day. Thanks to her research, we better understand which symptoms to look for in children and can start treating sickle cell earlier. Thanks to Dr. Ferguson for the work she has done on behalf of our community!