top of page

Sickle Cycle White 2.png

Sickle Cycle

Advocacy and awareness for the sickle cell community

Understanding sickle cell starts here

Programs supporting patients and families

The story behind the mission

Our Mission and Vision

Sickle Cycle is a non-profit organization focused on advocacy, education, and public awareness to address the challenges faced by individuals and families affected by Sickle Cell Disease. We are dedicated to improve the quality of life for those impacted by this illness through partnerships and community engagement.

Beta Thalassemia is a type of Sickle Cell Disease. The Chinese biotech CorrectSequence Therapeutics, also known as Correctseq, reports good results from a Phase I study of its technology involving editing a person’s hematopoietic stem cells to treat beta thalassemia. The trial, published in Nature, included five patients with transfusion dependent beta thalassemia who were able to stop red blood cell transfusions, the standard treatment for the condition, after receiving the base-edited treatment CS-101. The participants continued to have good levels of hemoglobin with no serious side effects during follow-up. Beta thalassemia is a rare inherited condition affecting around one in 100,000 people in the U.S. Mutations in the beta‑globin gene HBB reduce or stop production of the beta chains of hemoglobin, leading to chronic anemia that varies in its severity. In this study, CS-101 was given to five patients with beta thalassemia, previously treated with blood transfusions. The process involves extracting their stem cells, reactivating fetal hemoglobin production using base editing, giving the patients chemotherapy to clear existing stem cells and make way for the newly edited population, and finally injecting the patients with the edited stem cells. There are already several therapies on the market for beta thalassemia. The most common treatment is still regular blood transfusions to treat the anemia, but recently the genetic therapies Zynteglo, a lentiviral gene therapy developed by Bluebird Bio, and Casgevy, a CRISPR edited therapy developed by Vertex Pharmaceuticals and CRISPR Therapeutics were approved by the FDA. #betathalassemia #sicklecell #sicklecellbetathalassemia

Beta Thalassemia is a type of Sickle Cell Disease. The Chinese biotech CorrectSequence Therapeutics, also known as Correctseq, reports good results from a Phase I study of its technology involving editing a person’s hematopoietic stem cells to treat beta thalassemia. The trial, published in Nature, included five patients with transfusion dependent beta thalassemia who were able to stop red blood cell transfusions, the standard treatment for the condition, after receiving the base-edited treatment CS-101. The participants continued to have good levels of hemoglobin with no serious side effects during follow-up. Beta thalassemia is a rare inherited condition affecting around one in 100,000 people in the U.S. Mutations in the beta‑globin gene HBB reduce or stop production of the beta chains of hemoglobin, leading to chronic anemia that varies in its severity. In this study, CS-101 was given to five patients with beta thalassemia, previously treated with blood transfusions. The process involves extracting their stem cells, reactivating fetal hemoglobin production using base editing, giving the patients chemotherapy to clear existing stem cells and make way for the newly edited population, and finally injecting the patients with the edited stem cells. There are already several therapies on the market for beta thalassemia. The most common treatment is still regular blood transfusions to treat the anemia, but recently the genetic therapies Zynteglo, a lentiviral gene therapy developed by Bluebird Bio, and Casgevy, a CRISPR edited therapy developed by Vertex Pharmaceuticals and CRISPR Therapeutics were approved by the FDA. #betathalassemia #sicklecell #sicklecellbetathalassemia

We have a new addition to the Sickle Cycle Family! The instrumental to this post is one of the songs that keeps me focused on living a purposeful life inspite of storms,challenges, and sickle cell complications that may appear to be roadblocks. The lyrics state: May your struggles keep you near the cross.
And may your troubles show that you need God.
And may your battles end the way they should.
And may your bad days prove that God is good.
And may your whole life prove that God is good.

Thankful to God for the pregnancy journey, delivery, and recovery especially as a sickle cell warrior. Sickle cell is complicated and pregnancy does not provide an exemption. Women with sickle cell disease are at higher risk of problems during pregnancy. Some sickle cell risks are linked with changes that occur in the body due to pregnancy, including: Raised metabolism, Higher hormone levels that raise the risk of blood clots, Serious anemia, Additional pain crises. If you are pregnant or planning for pregnancy, find a team of a healthcare providers who specialize in high-risk pregnancies and have experience with patients who have sickle cell disease. Meet with the team throughout your pregnancy to manage your risks. #sicklecellandpregnancy #sicklecell #godgetstheglory #frommystory

We have a new addition to the Sickle Cycle Family! The instrumental to this post is one of the songs that keeps me focused on living a purposeful life inspite of storms,challenges, and sickle cell complications that may appear to be roadblocks. The lyrics state: May your struggles keep you near the cross.
And may your troubles show that you need God.
And may your battles end the way they should.
And may your bad days prove that God is good.
And may your whole life prove that God is good.

Thankful to God for the pregnancy journey, delivery, and recovery especially as a sickle cell warrior. Sickle cell is complicated and pregnancy does not provide an exemption. Women with sickle cell disease are at higher risk of problems during pregnancy. Some sickle cell risks are linked with changes that occur in the body due to pregnancy, including: Raised metabolism, Higher hormone levels that raise the risk of blood clots, Serious anemia, Additional pain crises. If you are pregnant or planning for pregnancy, find a team of a healthcare providers who specialize in high-risk pregnancies and have experience with patients who have sickle cell disease. Meet with the team throughout your pregnancy to manage your risks. #sicklecellandpregnancy #sicklecell #godgetstheglory #frommystory

Closing out Sickle Cell Awareness Month with a community event hosted by @mscdaorg in Baltimore, MD with sickle cell warriors and their families. So many vendors and resources at the event! If you are in the DMV area, get involved with MSCDA Org to learn about the ways you can help warriors in the community. #sicklecell #baltimore #mscdaorg #sicklecelldisease #sicklecellevents

Closing out Sickle Cell Awareness Month with a community event hosted by @mscdaorg in Baltimore, MD with sickle cell warriors and their families. So many vendors and resources at the event! If you are in the DMV area, get involved with MSCDA Org to learn about the ways you can help warriors in the community. #sicklecell #baltimore #mscdaorg #sicklecelldisease #sicklecellevents

Happy Sickle Cell Awareness Month! Yesterday, @sicklequeent, founder of @sickle_red, hosted an amazing event filled with education, networking and awareness from Sickle Cell patients and providers regarding reproductive and maternal health care. Did you know the maternal mortality rate in deliveries among people with SCD was 26 times greater than in the non-Black control group and more than 10 times greater than among Black pregnant people without SCD? @sicklequeent thank you for being an example of turning your experience with a medical failure into service for the sickle cell community. Thank you for hosting an event including so many legends pictured and not pictured 🙃. #scred #sicklecellred #maternalhealth #reproductivecare #sicklecell

Happy Sickle Cell Awareness Month! Yesterday, @sicklequeent, founder of @sickle_red, hosted an amazing event filled with education, networking and awareness from Sickle Cell patients and providers regarding reproductive and maternal health care. Did you know the maternal mortality rate in deliveries among people with SCD was 26 times greater than in the non-Black control group and more than 10 times greater than among Black pregnant people without SCD? @sicklequeent thank you for being an example of turning your experience with a medical failure into service for the sickle cell community. Thank you for hosting an event including so many legends pictured and not pictured 🙃. #scred #sicklecellred #maternalhealth #reproductivecare #sicklecell

For D’Asia Jackson, every day is a guessing game over whether excruciating pain will upend her life.

The 28-year-old medical assistant was born with sickle cell disease, an inherited blood disorder. It can cause blood clots, organ damage and excruciating pain, which can worsen during the menstrual cycle. “Pain-wise, I always describe it as like being hit by a Mack truck,” Jackson said. “When I know my period is coming, I know pain is probably right behind it.” Over the last 10 years, Jackson has worked with her doctors to try a variety of contraception options to get her symptoms under control, from the Depo-Provera shot, a hormone injection given every three months, to an IUD to different forms of birth control pills. According to Jackson, none of them helped. Some made her periods even worse. At times, she’s ended up in the hospital needing iron infusions due to loss of blood. Doctors don’t know how to manage her symptoms. “The lack of education on sickle cell is very frustrating,” she said. “I go into hospital stays having to educate doctors and nurses.” Now, women like Jackson are worried that lack of knowledge, particularly about how contraception affects their conditions, could get worse. In April, a small team that compiled information into what some consider the country’s “contraception bible,” a set of guidelines used by physicians to assess which types of birth control are safe for their patients, was fired from the Centers for Disease Control and Prevention as part of mass layoffs from the agency. The eight-person team was responsible for evaluating research and recommending birth control methods for patients with various conditions, from sickle cell disease, kidney disease and lupus to those at risk for HIV. The guidelines, called the U.S. Medical Eligibility Criteria for Contraceptive Use, provided the latest research for doctors, including an app that recommended which contraceptive methods were safe. The updates included new recommendations for people with sickle cell disease, finding that combined hormonal contraception presents an “unacceptable health risk” due to patients’ risk of life-threatening blood clots. #sicklecell #mentralcycle

For D’Asia Jackson, every day is a guessing game over whether excruciating pain will upend her life.

The 28-year-old medical assistant was born with sickle cell disease, an inherited blood disorder. It can cause blood clots, organ damage and excruciating pain, which can worsen during the menstrual cycle. “Pain-wise, I always describe it as like being hit by a Mack truck,” Jackson said. “When I know my period is coming, I know pain is probably right behind it.” Over the last 10 years, Jackson has worked with her doctors to try a variety of contraception options to get her symptoms under control, from the Depo-Provera shot, a hormone injection given every three months, to an IUD to different forms of birth control pills. According to Jackson, none of them helped. Some made her periods even worse. At times, she’s ended up in the hospital needing iron infusions due to loss of blood. Doctors don’t know how to manage her symptoms. “The lack of education on sickle cell is very frustrating,” she said. “I go into hospital stays having to educate doctors and nurses.” Now, women like Jackson are worried that lack of knowledge, particularly about how contraception affects their conditions, could get worse. In April, a small team that compiled information into what some consider the country’s “contraception bible,” a set of guidelines used by physicians to assess which types of birth control are safe for their patients, was fired from the Centers for Disease Control and Prevention as part of mass layoffs from the agency. The eight-person team was responsible for evaluating research and recommending birth control methods for patients with various conditions, from sickle cell disease, kidney disease and lupus to those at risk for HIV. The guidelines, called the U.S. Medical Eligibility Criteria for Contraceptive Use, provided the latest research for doctors, including an app that recommended which contraceptive methods were safe. The updates included new recommendations for people with sickle cell disease, finding that combined hormonal contraception presents an “unacceptable health risk” due to patients’ risk of life-threatening blood clots. #sicklecell #mentralcycle

New York resident Sebastien Beauzile was treated with a pioneering new gene therapy approach called Lyfgenia, developed by biotech company Bluebird Bio. Mr Beauzile received the treatment on December 17th 2024 and has not had any symptoms of sickle cell anemia since, leading his doctors to believe that the treatment has likely cured the disease. Previously, people with sickle cell anemia have been mostly treated with therapies which can control, but not cure the disease. Some people have been cured by giving them bone marrow transplants from external donors, normally a close relative without sickle cell anemia, but the procedure does not always work, comes with a host of side effects and a risk of dying. According to the National Institutes of Health, about 1 in 20 children under 16 receiving bone marrow transplants for sickle cell anemia have died, and 1 in 10 of people 16 years or older. Lyfgenia works by extracting the patient’s own blood stem cells, genetically modifying them using a virus to paste copies of functional hemaglobin into the cells and then giving the patient chemotherapy to clear out the old, dysfunctional cells before infusing the new, modified ones back into the body. The red blood cells then produced from the transplanted, modified blood stem cells are then completely normal. Mr Beauzile is not the first person to be successfully treated with Lyfgenia in the U.S. Lyfgenia and another genetic-modification based cell therapy for sickle cell anemia, Casgevy (Vertex pharmaceuticals) were both FDA-approved in late 2023 for the treatment of sickle cell anemia in patients 12 and older, following impressive clinical trial results. In the Lyfgenia trial which led to the drug’s approval, 88% of 32 patients treated with the therapy ranging in age from 12 to 50 years old had complete resolution of their symptoms between 6-18 months after receiving the drug. Lyfgenia is priced at $3.1 million per treatment, whereas Casgevy costs $2.2 million. #sicklecellcure #newyork #lyfgenia #genetherapy #sicklecelldisease

New York resident Sebastien Beauzile was treated with a pioneering new gene therapy approach called Lyfgenia, developed by biotech company Bluebird Bio. Mr Beauzile received the treatment on December 17th 2024 and has not had any symptoms of sickle cell anemia since, leading his doctors to believe that the treatment has likely cured the disease. Previously, people with sickle cell anemia have been mostly treated with therapies which can control, but not cure the disease. Some people have been cured by giving them bone marrow transplants from external donors, normally a close relative without sickle cell anemia, but the procedure does not always work, comes with a host of side effects and a risk of dying. According to the National Institutes of Health, about 1 in 20 children under 16 receiving bone marrow transplants for sickle cell anemia have died, and 1 in 10 of people 16 years or older. Lyfgenia works by extracting the patient’s own blood stem cells, genetically modifying them using a virus to paste copies of functional hemaglobin into the cells and then giving the patient chemotherapy to clear out the old, dysfunctional cells before infusing the new, modified ones back into the body. The red blood cells then produced from the transplanted, modified blood stem cells are then completely normal. Mr Beauzile is not the first person to be successfully treated with Lyfgenia in the U.S. Lyfgenia and another genetic-modification based cell therapy for sickle cell anemia, Casgevy (Vertex pharmaceuticals) were both FDA-approved in late 2023 for the treatment of sickle cell anemia in patients 12 and older, following impressive clinical trial results. In the Lyfgenia trial which led to the drug’s approval, 88% of 32 patients treated with the therapy ranging in age from 12 to 50 years old had complete resolution of their symptoms between 6-18 months after receiving the drug. Lyfgenia is priced at $3.1 million per treatment, whereas Casgevy costs $2.2 million. #sicklecellcure #newyork #lyfgenia #genetherapy #sicklecelldisease

A three-year-old boy is cured today thanks to the special care he received at UPMC Children’s Hospital of Pittsburgh. Leon Johnson received a bone marrow transplant on May 16, 2024, after being diagnosed with sickle cell disease one week after being born. His mother, Stacy, delivered twins in 2021. Leon and his sister, Leigh-Marie, were born just two minutes apart.

“I was aware that I had this sickle cell trait, but their father was not aware that he had the trait. They do the newborn screenings now for all children, and that’s how we found out that he had sickle cell disease,” Stacy said. “He was in the NICU still. So, after he got released from the NICU, one of his first appointments was to go to hematology and to be put on medication and to start getting educated on what sickle cell disease is, what version of sickle cell he had, which was, SS, the most severe (version) for what they told us of sickle cell.”

Stacy said before Leon’s bone marrow transplant, he couldn’t do the same things most three-year-olds do, like playing outside, visiting the park, trick-or-treating, and spending quality time with his sister.

“We had to try to isolate him as much as possible to keep him from getting sick. There [are] certain instances or situations that can trigger a sickle cell crisis or a pain crisis, and that is changing from warm to extreme cold, like you can’t do the cold playing outside, when it’s hot without taking breaks to get fluids, if it’s too humid outside, too hot, the season’s changing,” Stacy said. Leon’s family got the good news about six months ago that Leon could get a bone marrow transplant.

“We found out about a month after blood work and such, that he’s officially cured of sickle cell, and after he got his three-month workup again, no sickle cell,” his mother said.

Stacy said Leon is experiencing what they call “Leon’s firsts.” #bonemarrowtransplant #sicklecell

A three-year-old boy is cured today thanks to the special care he received at UPMC Children’s Hospital of Pittsburgh. Leon Johnson received a bone marrow transplant on May 16, 2024, after being diagnosed with sickle cell disease one week after being born. His mother, Stacy, delivered twins in 2021. Leon and his sister, Leigh-Marie, were born just two minutes apart.

“I was aware that I had this sickle cell trait, but their father was not aware that he had the trait. They do the newborn screenings now for all children, and that’s how we found out that he had sickle cell disease,” Stacy said. “He was in the NICU still. So, after he got released from the NICU, one of his first appointments was to go to hematology and to be put on medication and to start getting educated on what sickle cell disease is, what version of sickle cell he had, which was, SS, the most severe (version) for what they told us of sickle cell.”

Stacy said before Leon’s bone marrow transplant, he couldn’t do the same things most three-year-olds do, like playing outside, visiting the park, trick-or-treating, and spending quality time with his sister.

“We had to try to isolate him as much as possible to keep him from getting sick. There [are] certain instances or situations that can trigger a sickle cell crisis or a pain crisis, and that is changing from warm to extreme cold, like you can’t do the cold playing outside, when it’s hot without taking breaks to get fluids, if it’s too humid outside, too hot, the season’s changing,” Stacy said. Leon’s family got the good news about six months ago that Leon could get a bone marrow transplant.

“We found out about a month after blood work and such, that he’s officially cured of sickle cell, and after he got his three-month workup again, no sickle cell,” his mother said.

Stacy said Leon is experiencing what they call “Leon’s firsts.” #bonemarrowtransplant #sicklecell

Nearly one year ago, the Food and Drug Administration (FDA) approved two new gene therapies for the treatment of sickle cell disease. The Sickle Cell Disease Association of America Inc. (SCDAA) is pleased that the manufacturers of these two FDA-approved gene therapy treatments have entered into agreements with the Centers for Medicare & Medicaid Services (CMS) to participate in the Cell and Gene Therapy (CGT) Access Model. These cutting-edge treatments are poised to make a difference in the lives of many sickle cell warriors, but their high price tags are a barrier to access. The CGT Access Model is a promising effort to reduce cost for these potentially curative therapies for eligible individuals, allowing more patients to benefit from these significant advancements in treating diseases. According to an announcement from the Department of Health and Human Services, the model “will test outcomes-based agreements for cell and gene therapies, with the aim to improve health outcomes, increase access to cell and gene therapies, and lower health care costs.” We are also heartened to see that the model will provide fertility preservation for patients, marking an important acknowledgment of quality-of-life standards for our community.

The Model will launch in January 2025, and all 50 states may choose to begin participation anytime between January 2025 and January 2026. SCDAA looks forward to working with our community-based organizations and other stakeholders to advocate for state enrollment. The CGT Access Model will provide crucial support to patients where available, and we encourage full participation across the country.

Nearly one year ago, the Food and Drug Administration (FDA) approved two new gene therapies for the treatment of sickle cell disease. The Sickle Cell Disease Association of America Inc. (SCDAA) is pleased that the manufacturers of these two FDA-approved gene therapy treatments have entered into agreements with the Centers for Medicare & Medicaid Services (CMS) to participate in the Cell and Gene Therapy (CGT) Access Model. These cutting-edge treatments are poised to make a difference in the lives of many sickle cell warriors, but their high price tags are a barrier to access. The CGT Access Model is a promising effort to reduce cost for these potentially curative therapies for eligible individuals, allowing more patients to benefit from these significant advancements in treating diseases. According to an announcement from the Department of Health and Human Services, the model “will test outcomes-based agreements for cell and gene therapies, with the aim to improve health outcomes, increase access to cell and gene therapies, and lower health care costs.” We are also heartened to see that the model will provide fertility preservation for patients, marking an important acknowledgment of quality-of-life standards for our community.

The Model will launch in January 2025, and all 50 states may choose to begin participation anytime between January 2025 and January 2026. SCDAA looks forward to working with our community-based organizations and other stakeholders to advocate for state enrollment. The CGT Access Model will provide crucial support to patients where available, and we encourage full participation across the country.

Afrotech 2024 provided new information and there is one in particular that caught our eye, Sickle Sense. Sickle Sense does more than just provide generic health tips. Sickle Sense is a fleet of software services (distributed system, application, and data pipeline), all designed to provide a comprehensive solution for individuals to manage their sickle cell disease in the form of a web application. Every component is thoughtfully crafted to empower fellow warriors living with Sickle Cell to take control of their health and life. SickleSense leverages web crawling, semantic search, and retrieval augmented generation (rag) to deliver personalized recommendations and predict potential crises based on user-specific health data collected, all at the click of a button. This proactive approach empowers users to take charge of their health by anticipating issues before they arise, without the need for manual data entry like other platforms. Collecting a comprehensive health history improves personal care, enhances communication during medical consultations, aids the Sickle Sense A.I. services in making accurate suggestions, and ensures that in the event healthcare providers request patient data, they can have a complete view of the a users very own Sickel Cell history. #sicklecellAI #sicklesense #sicklecelldisease

Afrotech 2024 provided new information and there is one in particular that caught our eye, Sickle Sense. Sickle Sense does more than just provide generic health tips. Sickle Sense is a fleet of software services (distributed system, application, and data pipeline), all designed to provide a comprehensive solution for individuals to manage their sickle cell disease in the form of a web application. Every component is thoughtfully crafted to empower fellow warriors living with Sickle Cell to take control of their health and life. SickleSense leverages web crawling, semantic search, and retrieval augmented generation (rag) to deliver personalized recommendations and predict potential crises based on user-specific health data collected, all at the click of a button. This proactive approach empowers users to take charge of their health by anticipating issues before they arise, without the need for manual data entry like other platforms. Collecting a comprehensive health history improves personal care, enhances communication during medical consultations, aids the Sickle Sense A.I. services in making accurate suggestions, and ensures that in the event healthcare providers request patient data, they can have a complete view of the a users very own Sickel Cell history. #sicklecellAI #sicklesense #sicklecelldisease

The company announced it was voluntarily recalling Oxbryta and ending all active clinical trials and expanded access programs for its key ingredient, voxelotor. Pfizer explained that the decision was made “based on the totality of clinical data that now indicates the overall benefit of OXBRYTA no longer outweighs the risk in the approved sickle cell patient population.” The company said the data suggest the treatment can cause “an imbalance in vaso-occlusive crises and fatal events which require further assessment.” Pfizer noted it has advised regulators about the findings and recall.

Aida Habtezion, Chief Medical Officer and Head of Worldwide Medical and Safety at Pfizer, advised those taking Oxbryta to contact their doctors to discuss alternative medicines while the company continues to investigate.

The company announced it was voluntarily recalling Oxbryta and ending all active clinical trials and expanded access programs for its key ingredient, voxelotor. Pfizer explained that the decision was made “based on the totality of clinical data that now indicates the overall benefit of OXBRYTA no longer outweighs the risk in the approved sickle cell patient population.” The company said the data suggest the treatment can cause “an imbalance in vaso-occlusive crises and fatal events which require further assessment.” Pfizer noted it has advised regulators about the findings and recall.

Aida Habtezion, Chief Medical Officer and Head of Worldwide Medical and Safety at Pfizer, advised those taking Oxbryta to contact their doctors to discuss alternative medicines while the company continues to investigate.

@howard1867 Sickle Cell 5k walk/run is always a good time. Great people, vendors providing educational resources, and free testing for sickle cell disease!

@howard1867 Sickle Cell 5k walk/run is always a good time. Great people, vendors providing educational resources, and free testing for sickle cell disease!

Thank you @crescentfoundationscd for kicking off sickle cell awareness month by hosting an event shining the light on sickle cell warriors living well with the inherited blood disorder. Living well means that we have learned lessons to help us navigate our life better (as much as possible) with doctor appointments, sickness, and treatments. As a panelist, I’m grateful for the opportunity to learn from other warriors. We are stronger than we think and powerful beyond measure. #sicklecelldisease #panelist #sicklecelleducation #learningandgrowing #sicklecellawarenessmonth

Thank you @crescentfoundationscd for kicking off sickle cell awareness month by hosting an event shining the light on sickle cell warriors living well with the inherited blood disorder. Living well means that we have learned lessons to help us navigate our life better (as much as possible) with doctor appointments, sickness, and treatments. As a panelist, I’m grateful for the opportunity to learn from other warriors. We are stronger than we think and powerful beyond measure. #sicklecelldisease #panelist #sicklecelleducation #learningandgrowing #sicklecellawarenessmonth

Sickle cell disease affects more than 20 million people worldwide and can be a devastating condition. The inherited blood disorder affects the hemoglobin that carries oxygen through the body.

The genetic link between sickle cell disease and malaria is a story of how our genome adapts to the environment. Humans evolved in Africa 300,000 years ago. And at one point the Sahara desert was a big glacier. But when it melted, Central Africa became much warmer, creating an ideal habitat for mosquitoes. About 50,000 years ago, those mosquitoes, which initially infected primates, began to infect humans.

From time to time, humans have spontaneous mutations in our genes. And some 20,000 years ago, one of those mutations—the mutation for sickle cell disease—happened to be protective against malaria.

If you have one copy of that sickle cell mutation, hemoglobin-S, you are a carrier. You will not become sick from sickle cell disease, and you‘ll be more resistant to malaria. But if you have a double copy, one from each parent, you have sickle cell disease.

As Africa’s population evolved, those without the single mutation would often die of malaria, and those who had two copies of the gene would die of sickle cell disease. That’s why the single mutation became extremely common in Africa as populations settled, became more agriculturalist, and expanded. #genome #genotype #scdhistory #sicklecelldisease #malaria #scdmalaria

Sickle cell disease affects more than 20 million people worldwide and can be a devastating condition. The inherited blood disorder affects the hemoglobin that carries oxygen through the body.

The genetic link between sickle cell disease and malaria is a story of how our genome adapts to the environment. Humans evolved in Africa 300,000 years ago. And at one point the Sahara desert was a big glacier. But when it melted, Central Africa became much warmer, creating an ideal habitat for mosquitoes. About 50,000 years ago, those mosquitoes, which initially infected primates, began to infect humans.

From time to time, humans have spontaneous mutations in our genes. And some 20,000 years ago, one of those mutations—the mutation for sickle cell disease—happened to be protective against malaria.

If you have one copy of that sickle cell mutation, hemoglobin-S, you are a carrier. You will not become sick from sickle cell disease, and you‘ll be more resistant to malaria. But if you have a double copy, one from each parent, you have sickle cell disease.

As Africa’s population evolved, those without the single mutation would often die of malaria, and those who had two copies of the gene would die of sickle cell disease. That’s why the single mutation became extremely common in Africa as populations settled, became more agriculturalist, and expanded. #genome #genotype #scdhistory #sicklecelldisease #malaria #scdmalaria

Happy Sickle Cell Awareness Day! Over 2 million people across the 🌎 have this inherited disease. More awareness 🟰 more funding, research, and quality care for all. #sicklecelldisease #sicklecellanemia #sicklecellawareness #sicklecell #sicklecycle

Happy Sickle Cell Awareness Day! Over 2 million people across the 🌎 have this inherited disease. More awareness 🟰 more funding, research, and quality care for all. #sicklecelldisease #sicklecellanemia #sicklecellawareness #sicklecell #sicklecycle

On Wednesday, Kendric Cromer, a 12-year-old boy from a suburb of Washington, became the first person in the world with sickle cell disease to begin a commercially approved gene therapy that may cure the condition (Bluebird Bio, a Somerville, Mass., company). For the estimated 20,000 people with sickle cell in the United States who qualify for the treatment, the start of Kendric’s monthslong medical journey may offer hope. “Sickle cell always steals my dreams and interrupts all the things I want to do,” he said. Now he feels as if he has a chance for a normal life. Bluebird is charging $3.1 million for its gene therapy, called Lyfgenia. It’s one of the highest prices ever for a treatment.

Kendric — whose family’s health insurance agreed to cover the procedure — began his treatment at Children’s National Hospital in Washington. Wednesday’s treatment was only the first step. Doctors removed his bone marrow stem cells, which Bluebird will then genetically modify in a specialized lab for his treatment. The whole process is so involved and time-consuming that Bluebird estimates it can treat the cells of only 85 to 105 patients each year.

“We always prayed this day would come,” Deborah (his mom) said. But, she added, “We’re nervous reading through the consents and what he will have to go through.”

Kendric, though, is looking forward to the future. He wants to be a geneticist.

And, he said, “I want to play basketball.”
#sicklecell #genetherapy #cromerfamily #insurance #medicalcost #bluebird #futurebasketballplayer #futuregeneticist

On Wednesday, Kendric Cromer, a 12-year-old boy from a suburb of Washington, became the first person in the world with sickle cell disease to begin a commercially approved gene therapy that may cure the condition (Bluebird Bio, a Somerville, Mass., company). For the estimated 20,000 people with sickle cell in the United States who qualify for the treatment, the start of Kendric’s monthslong medical journey may offer hope. “Sickle cell always steals my dreams and interrupts all the things I want to do,” he said. Now he feels as if he has a chance for a normal life. Bluebird is charging $3.1 million for its gene therapy, called Lyfgenia. It’s one of the highest prices ever for a treatment.

Kendric — whose family’s health insurance agreed to cover the procedure — began his treatment at Children’s National Hospital in Washington. Wednesday’s treatment was only the first step. Doctors removed his bone marrow stem cells, which Bluebird will then genetically modify in a specialized lab for his treatment. The whole process is so involved and time-consuming that Bluebird estimates it can treat the cells of only 85 to 105 patients each year.

“We always prayed this day would come,” Deborah (his mom) said. But, she added, “We’re nervous reading through the consents and what he will have to go through.”

Kendric, though, is looking forward to the future. He wants to be a geneticist.

And, he said, “I want to play basketball.”
#sicklecell #genetherapy #cromerfamily #insurance #medicalcost #bluebird #futurebasketballplayer #futuregeneticist

On Dec. 8, 2023, 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.

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.

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. Gene therapy will likely be available in early 2024. More information can be found at the SCDAA website. #geneediting #geneaddition #sicklecell #sicklecelldisease

On Dec. 8, 2023, 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.

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.

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. Gene therapy will likely be available in early 2024. More information can be found at the SCDAA website. #geneediting #geneaddition #sicklecell #sicklecelldisease

Loma Linda University Health was awarded a significant $2.2 million research grant from the Health Resources and Services Administration (HRSA) to further advance efforts in improving the treatment and quality of life of youth and young adults with sickle cell disease (SCD). The grant comes as from HRSA to help more systematically address maternal health disparities. Lisa Roberts, DrPH, MSN, FNP-BC, FAANP, FAAN, research director at Loma Linda University School of Nursing, is leading the initiatives to implement the $2.2 million into LLUH’s system.

SCD affects one in 12 people of African American origin in the United States, according to Akshat Jain, MD, MPH, medical director of the Pediatric Sickle Cell Center of Excellence at Children’s Hospital. The CDC reported that San Bernardino County houses the second most people living with SCD in California. The TDP grant is dedicated to addressing the unique challenges faced by individuals living with SCD by increasing access to comprehensive, coordinated, and quality care.

The grant allows Roberts and her interdisciplinary team made up of investigators from Behavioral Health and Medicine to focus on enhancing the quality of care for SCD patients aged 12 to 25 and address the lack of reproductive health education resources for this age group. She says this is critically important as patients with SCD face some of the highest maternal health risks. #reproductivehealth #sicklecell #maternalhealth #familyplanning

Loma Linda University Health was awarded a significant $2.2 million research grant from the Health Resources and Services Administration (HRSA) to further advance efforts in improving the treatment and quality of life of youth and young adults with sickle cell disease (SCD). The grant comes as from HRSA to help more systematically address maternal health disparities. Lisa Roberts, DrPH, MSN, FNP-BC, FAANP, FAAN, research director at Loma Linda University School of Nursing, is leading the initiatives to implement the $2.2 million into LLUH’s system.

SCD affects one in 12 people of African American origin in the United States, according to Akshat Jain, MD, MPH, medical director of the Pediatric Sickle Cell Center of Excellence at Children’s Hospital. The CDC reported that San Bernardino County houses the second most people living with SCD in California. The TDP grant is dedicated to addressing the unique challenges faced by individuals living with SCD by increasing access to comprehensive, coordinated, and quality care.

The grant allows Roberts and her interdisciplinary team made up of investigators from Behavioral Health and Medicine to focus on enhancing the quality of care for SCD patients aged 12 to 25 and address the lack of reproductive health education resources for this age group. She says this is critically important as patients with SCD face some of the highest maternal health risks. #reproductivehealth #sicklecell #maternalhealth #familyplanning

Dr. Benjamin was a trailblazing physician who made many outstanding contributions to the sickle cell community. She was one of the first to establish a “day hospital” as an alternative to the emergency room for pain management – an approach that is today recognized as a best practice in care.

Prior to her retirement, Dr. Benjamin led the Montefiore Sickle Cell Center for Adults in the Bronx, New York. She was an SCDAA board member emeritus and served on the SCDAA Medical and Research Advisory Committee.

Dr. Benjamin was also a dedicated advocate and mentor. She made a global impact and raised sickle cell awareness in West Africa, Brazil and beyond. “She was always looking out for others, patients and peers alike,” recalls Dr. Lewis Hsu, SCDAA chief medical officer. “She was full of warm advice and expert guidance.”

Dr. Benjamin passed away in Houston, Texas on Oct. 20 at the age of 82. Please keep her family in your thoughts and prayers #sicklecelldisease #houston

Dr. Benjamin was a trailblazing physician who made many outstanding contributions to the sickle cell community. She was one of the first to establish a “day hospital” as an alternative to the emergency room for pain management – an approach that is today recognized as a best practice in care.

Prior to her retirement, Dr. Benjamin led the Montefiore Sickle Cell Center for Adults in the Bronx, New York. She was an SCDAA board member emeritus and served on the SCDAA Medical and Research Advisory Committee.

Dr. Benjamin was also a dedicated advocate and mentor. She made a global impact and raised sickle cell awareness in West Africa, Brazil and beyond. “She was always looking out for others, patients and peers alike,” recalls Dr. Lewis Hsu, SCDAA chief medical officer. “She was full of warm advice and expert guidance.”

Dr. Benjamin passed away in Houston, Texas on Oct. 20 at the age of 82. Please keep her family in your thoughts and prayers #sicklecelldisease #houston

Howard Sickle Cell 5k! #fundraiser #awareness #sicklecelldisease #curesicklecellnow #hu #howarduniversity

Howard Sickle Cell 5k! #fundraiser #awareness #sicklecelldisease #curesicklecellnow #hu #howarduniversity

I just had the most incredible experience. I’m pictured with Dr. Adrienne Scott and Dr. Morton Goldberg. When I had bleeding in my eye due to sickle cell disease, my hematologist recommended for me to see Dr. Scott and I have been her patient ever since. Dr. Morton is the first physician to discover the connection with sickle cell disease and the eye. It’s because of his discovery, training, and curiosity why so many patients are able to receive treatment options for their eye without it leading to blindness (me included) 🤯. On top of that, he paid for a historian to discover the name of the first person described with having sickle cell disease and because of him we know it was Dr. Walter Clement, dentist from Grenada studying in the US. Unsung heroes!!! Grateful and in awe of the full circle moment. #sicklecelldisease #retina #optomologist

I just had the most incredible experience. I’m pictured with Dr. Adrienne Scott and Dr. Morton Goldberg. When I had bleeding in my eye due to sickle cell disease, my hematologist recommended for me to see Dr. Scott and I have been her patient ever since. Dr. Morton is the first physician to discover the connection with sickle cell disease and the eye. It’s because of his discovery, training, and curiosity why so many patients are able to receive treatment options for their eye without it leading to blindness (me included) 🤯. On top of that, he paid for a historian to discover the name of the first person described with having sickle cell disease and because of him we know it was Dr. Walter Clement, dentist from Grenada studying in the US. Unsung heroes!!! Grateful and in awe of the full circle moment. #sicklecelldisease #retina #optomologist

There is so much work needed to help those impacted by this global inherited blood disorder. I believe as warriors, it is our obligation to educate, inform, and provide awareness so that change is not dictated by others but is lead by us. Thank you @bluebird_bio for featuring my perspective. Happy sickle cell awareness month! #sicklecellawareness #sicklecellwarrior #sicklecelldisease

There is so much work needed to help those impacted by this global inherited blood disorder. I believe as warriors, it is our obligation to educate, inform, and provide awareness so that change is not dictated by others but is lead by us. Thank you @bluebird_bio for featuring my perspective. Happy sickle cell awareness month! #sicklecellawareness #sicklecellwarrior #sicklecelldisease

When NFL running back Tevin Coleman and his wife Akilah Coleman were growing up, they knew that they both carried the sickle cell gene. But the reality of what that meant hit home in 2017 when one of their twins, Nazaneen, was diagnosed with sickle cell disease (SCD) when she was just an infant. “We really just wanted to come forward and start that conversation and normalize the conversation with our daughter,” Coleman said. “It’s about normalizing it for her so that she is not scared to talk to us about what she’s experiencing.”

Coleman made the remarks during the recent annual National Association of Black Journalists convention in Birmingham, Alabama during a panel about sickle cell care and the health inequities Black women face. Coleman was joined at the event by Teonna Woolford, who has SCD and is the co-founder of Sickle Cell Reproductive Health Education Directive (SC RED), and Dr. Kim Smith-Whitley, an advisor of Scientific and Clinical Affairs at Pfizer who has dedicated much of her career to sickle cell research. The panel was moderated by MSNBC’s Symone Sanders-Townsend.

Woolford, who received a bone marrow transplant, shared her desire to be a mother to six children. She communicated that desire with her healthcare provider. Research has shown that women with SCD have unique risk factors that may impact their ability to conceive.

“I just don’t want to be infertile,” she told them. “I’ll risk everything for a cure, but I don’t want to be infertile.”While she did reject more treatment, she still found herself living with sickle cell and infertility. After researching and seeking resources, Woolford felt defeated as she found little to no information about the intersection of fertility and SCD. With the help of Dr. Smith-Whitley and Dr. Lydia Pecker, the three women founded SC RED. #voicesofsicklecell #sicklecelldisease #sicklecell #infertility #advocates #paintopassion

When NFL running back Tevin Coleman and his wife Akilah Coleman were growing up, they knew that they both carried the sickle cell gene. But the reality of what that meant hit home in 2017 when one of their twins, Nazaneen, was diagnosed with sickle cell disease (SCD) when she was just an infant. “We really just wanted to come forward and start that conversation and normalize the conversation with our daughter,” Coleman said. “It’s about normalizing it for her so that she is not scared to talk to us about what she’s experiencing.”

Coleman made the remarks during the recent annual National Association of Black Journalists convention in Birmingham, Alabama during a panel about sickle cell care and the health inequities Black women face. Coleman was joined at the event by Teonna Woolford, who has SCD and is the co-founder of Sickle Cell Reproductive Health Education Directive (SC RED), and Dr. Kim Smith-Whitley, an advisor of Scientific and Clinical Affairs at Pfizer who has dedicated much of her career to sickle cell research. The panel was moderated by MSNBC’s Symone Sanders-Townsend.

Woolford, who received a bone marrow transplant, shared her desire to be a mother to six children. She communicated that desire with her healthcare provider. Research has shown that women with SCD have unique risk factors that may impact their ability to conceive.

“I just don’t want to be infertile,” she told them. “I’ll risk everything for a cure, but I don’t want to be infertile.”While she did reject more treatment, she still found herself living with sickle cell and infertility. After researching and seeking resources, Woolford felt defeated as she found little to no information about the intersection of fertility and SCD. With the help of Dr. Smith-Whitley and Dr. Lydia Pecker, the three women founded SC RED. #voicesofsicklecell #sicklecelldisease #sicklecell #infertility #advocates #paintopassion

I’m so excited to share that my doctor, Dr. Adrienne Scott has been named Best Consulting Physician at Johns Hopkins Medicine! Notably, Dr. Scott is the first ophthalmologist at Johns Hopkins to receive this award. The Office of Johns Hopkins Physicians launched the Johns Hopkins Medicine Clinical Awards for Physicians and Care Teams in 2015 to honor the physicians and care teams who embody the best in clinical excellence.

The Best Consulting Physician award is given to the Johns Hopkins physician who provides superior consulting or specialized services. As a retina specialist, Dr. Scott is a crucial partner across the institution in the care of patients with sickle cell disease. Her research and work to understand sickle cell retinopathy has changed the landscape for diagnosis and treatment of the condition. But perhaps just as importantly, her expert care, kind encouragement, and close follow-up are motivating factors for patients to continue to pursue their goals and ambitions even in the face of chronic illness.

The Johns Hopkins sickle cell team provides care for more than 600 adults, and nearly all are referred to Dr. Scott for ophthalmic care. She also provides care for children with the condition, and often follows them through adulthood. Here at Wilmer, she is appreciated for her responsiveness and willingness to provide a consult for any patient who needs her care. Among trainees, Dr. Scott, who formerly directed our retina fellowship, is a respected mentor who is always available to provide guidance to young physicians as they navigate challenging surgical cases. To learn more about Dr. Scott and her work, check out episode 7 of the sickle cycle podcast. #careteam #retinaspecialist #sicklecell #thesicklecyclepodcast #optomologist #optomologistdmv

I’m so excited to share that my doctor, Dr. Adrienne Scott has been named Best Consulting Physician at Johns Hopkins Medicine! Notably, Dr. Scott is the first ophthalmologist at Johns Hopkins to receive this award. The Office of Johns Hopkins Physicians launched the Johns Hopkins Medicine Clinical Awards for Physicians and Care Teams in 2015 to honor the physicians and care teams who embody the best in clinical excellence.

The Best Consulting Physician award is given to the Johns Hopkins physician who provides superior consulting or specialized services. As a retina specialist, Dr. Scott is a crucial partner across the institution in the care of patients with sickle cell disease. Her research and work to understand sickle cell retinopathy has changed the landscape for diagnosis and treatment of the condition. But perhaps just as importantly, her expert care, kind encouragement, and close follow-up are motivating factors for patients to continue to pursue their goals and ambitions even in the face of chronic illness.

The Johns Hopkins sickle cell team provides care for more than 600 adults, and nearly all are referred to Dr. Scott for ophthalmic care. She also provides care for children with the condition, and often follows them through adulthood. Here at Wilmer, she is appreciated for her responsiveness and willingness to provide a consult for any patient who needs her care. Among trainees, Dr. Scott, who formerly directed our retina fellowship, is a respected mentor who is always available to provide guidance to young physicians as they navigate challenging surgical cases. To learn more about Dr. Scott and her work, check out episode 7 of the sickle cycle podcast. #careteam #retinaspecialist #sicklecell #thesicklecyclepodcast #optomologist #optomologistdmv

Sickle cell disease was the first human disorder understood on a molecular level, its underpinnings explained in a landmark 1949 paper by a future two-time Nobel laureate.

Yet the inherited blood disease ― which causes unpredictable bouts of crushing pain, damages organs, and cuts lives short ― has eluded transformative treatments longer than a number of other genetic diseases, including far rarer ones. Two rival gene-based treatments developed by three biotech firms with headquarters, or most of their operations, in Massachusetts could be approved late this year or early in 2024.

The medicines ― a gene therapy from Bluebird Bio and a gene-editing treatment by a partnership between Vertex Pharmaceuticals and CRISPR Therapeutics ― have generated extraordinarily promising results in clinical trials. Some scientists even call them “potentially curative,” although others say that conclusion is premature. “They’re both amazing,” said Dr. Julie Kanter, director of the adult sickle cell clinic at the University of Alabama at Birmingham and one of the principal investigators for Bluebird’s trial. Dvaizea Moore, who at 5 years old suffered a stroke brought on by sickle cell disease, reported similar results about the Bluebird gene therapy he received in February at the University of Alabama at the suggestion of his doctor, Kanter.

“I haven’t had any pain since the transplant, and it’s never been like that in my life,” said Moore, 29, who lives near Birmingham and recently began working as a car mechanic.

Despite such remarkable outcomes, several sickle cell specialists worry about the cost of the treatments ― and the resulting impact on federal and private insurance plans ― if they win approval from the Food and Drug Administration. Each medicine is expected to cost at least $1 million to $2 million, not including the expense of administering them and other aspects of the treatment. It also remains to be seen how durable the benefits of the therapies are. Researchers plan to follow trial volunteers for 15 years after treatment.

Sickle cell disease was the first human disorder understood on a molecular level, its underpinnings explained in a landmark 1949 paper by a future two-time Nobel laureate.

Yet the inherited blood disease ― which causes unpredictable bouts of crushing pain, damages organs, and cuts lives short ― has eluded transformative treatments longer than a number of other genetic diseases, including far rarer ones. Two rival gene-based treatments developed by three biotech firms with headquarters, or most of their operations, in Massachusetts could be approved late this year or early in 2024.

The medicines ― a gene therapy from Bluebird Bio and a gene-editing treatment by a partnership between Vertex Pharmaceuticals and CRISPR Therapeutics ― have generated extraordinarily promising results in clinical trials. Some scientists even call them “potentially curative,” although others say that conclusion is premature. “They’re both amazing,” said Dr. Julie Kanter, director of the adult sickle cell clinic at the University of Alabama at Birmingham and one of the principal investigators for Bluebird’s trial. Dvaizea Moore, who at 5 years old suffered a stroke brought on by sickle cell disease, reported similar results about the Bluebird gene therapy he received in February at the University of Alabama at the suggestion of his doctor, Kanter.

“I haven’t had any pain since the transplant, and it’s never been like that in my life,” said Moore, 29, who lives near Birmingham and recently began working as a car mechanic.

Despite such remarkable outcomes, several sickle cell specialists worry about the cost of the treatments ― and the resulting impact on federal and private insurance plans ― if they win approval from the Food and Drug Administration. Each medicine is expected to cost at least $1 million to $2 million, not including the expense of administering them and other aspects of the treatment. It also remains to be seen how durable the benefits of the therapies are. Researchers plan to follow trial volunteers for 15 years after treatment.

The ranks of ESPN analysts are filled with former players, many of who bring up specific circumstances from their careers in relation to current sports issues. A particularly unusual example of that came from ESPN NFL analyst Ryan Clark on First Take Wednesday.

At one point on that show, Clark, JJ Redick, Stephen A. Smith, and host Molly Qerim were discussing the Denver altitude (5,276 feet above sea level). That came out of how Los Angeles Lakers’ star LeBron James is set to play his first playoff game there against the Nuggets, and how he described that challenge as “real.” And that led to Clark bringing up his own experience with playing in Denver. Notably, that saw him develop splenic infarction in a 2007 game there thanks to his sickle cell trait, which led to him having to have his spleen and gallbladder removed and miss the rest of the season, and which led to him never playing in Denver again. Clark says “I’m not sure who else on that team has sickle cell trait, though. But yeah, I mean, I played there, and I lost my spleen, my gall bladder, a decent part of my liver. Clark says “I can agree with LeBron that Denver sucks. It’s like the worst place to play.” An advantage of former players as analysts is their ability to work in their own experiences and personal knowledge when relevant. #sicklecelltrait #complications #highaltitude #highaltitudeexperience #sicklecelltraiteducation

The ranks of ESPN analysts are filled with former players, many of who bring up specific circumstances from their careers in relation to current sports issues. A particularly unusual example of that came from ESPN NFL analyst Ryan Clark on First Take Wednesday.

At one point on that show, Clark, JJ Redick, Stephen A. Smith, and host Molly Qerim were discussing the Denver altitude (5,276 feet above sea level). That came out of how Los Angeles Lakers’ star LeBron James is set to play his first playoff game there against the Nuggets, and how he described that challenge as “real.” And that led to Clark bringing up his own experience with playing in Denver. Notably, that saw him develop splenic infarction in a 2007 game there thanks to his sickle cell trait, which led to him having to have his spleen and gallbladder removed and miss the rest of the season, and which led to him never playing in Denver again. Clark says “I’m not sure who else on that team has sickle cell trait, though. But yeah, I mean, I played there, and I lost my spleen, my gall bladder, a decent part of my liver. Clark says “I can agree with LeBron that Denver sucks. It’s like the worst place to play.” An advantage of former players as analysts is their ability to work in their own experiences and personal knowledge when relevant. #sicklecelltrait #complications #highaltitude #highaltitudeexperience #sicklecelltraiteducation

For as long as he can remember, Jimi Olaghere felt he was destined to be a father. “It’s so true in my soul,” he told his wife, Amanda, when they struggled to get pregnant. But when they were finally expecting a baby boy in 2019, joy was tinged with despair.
For 34 years, sickle cell disease had been hammering Jimi’s body and stealthily shredding his ambitions. Then, midway through Amanda’s pregnancy, the couple read an article about Victoria Gray, a woman whose genes had been experimentally edited to treat her sickle cell disease. It was still too soon to know exactly how well it worked, but Jimi wanted in.There are risks and unknowns with any new technology; one doctor told Jimi the magnitude of the challenge was comparable with landing on the moon. In November 2019, Jimi and Amanda flew to Nashville to meet with Haydar Frangoul, the pediatric hematologist leading a trial of a CRISPR gene therapy for sickle cell disease at Sarah Cannon Research Institute. They learned shortly after Christmas that Jimi qualified for the trial. Their son, Sebastian, had just been born. It felt like a gift. From start to finish, Jimi’s treatment would take the better part of a year. First, his stem cells needed to be collected from his blood. This required long car trips to Nashville and being hooked up to a machine for hours at a time. Once the researchers collected enough stem cells, they edited the cells to disable the BCL11A switch. Then the cells needed to be carefully checked for quality. Jimi also needed chemotherapy to kill off existing cells in his bone marrow so that his edited stem cells would have room to engraft and grow. His hair fell out and he developed painful sores in his mouth.
Amanda, Jimi and baby Sebastian lived in the hospital for weeks. Jimi is one of 31 participants whose results have been made public in the sickle cell trial run by Vertex Pharmaceuticals and CRISPR Therapeutics. None have had pain crises since their treatment, according to data through February 2022, though at that time, only 11 patients had been followed for at least a year. #treatment #genediting #sicklecell #crisprsicklecell #crispr

For as long as he can remember, Jimi Olaghere felt he was destined to be a father. “It’s so true in my soul,” he told his wife, Amanda, when they struggled to get pregnant. But when they were finally expecting a baby boy in 2019, joy was tinged with despair.
For 34 years, sickle cell disease had been hammering Jimi’s body and stealthily shredding his ambitions. Then, midway through Amanda’s pregnancy, the couple read an article about Victoria Gray, a woman whose genes had been experimentally edited to treat her sickle cell disease. It was still too soon to know exactly how well it worked, but Jimi wanted in.There are risks and unknowns with any new technology; one doctor told Jimi the magnitude of the challenge was comparable with landing on the moon. In November 2019, Jimi and Amanda flew to Nashville to meet with Haydar Frangoul, the pediatric hematologist leading a trial of a CRISPR gene therapy for sickle cell disease at Sarah Cannon Research Institute. They learned shortly after Christmas that Jimi qualified for the trial. Their son, Sebastian, had just been born. It felt like a gift. From start to finish, Jimi’s treatment would take the better part of a year. First, his stem cells needed to be collected from his blood. This required long car trips to Nashville and being hooked up to a machine for hours at a time. Once the researchers collected enough stem cells, they edited the cells to disable the BCL11A switch. Then the cells needed to be carefully checked for quality. Jimi also needed chemotherapy to kill off existing cells in his bone marrow so that his edited stem cells would have room to engraft and grow. His hair fell out and he developed painful sores in his mouth.
Amanda, Jimi and baby Sebastian lived in the hospital for weeks. Jimi is one of 31 participants whose results have been made public in the sickle cell trial run by Vertex Pharmaceuticals and CRISPR Therapeutics. None have had pain crises since their treatment, according to data through February 2022, though at that time, only 11 patients had been followed for at least a year. #treatment #genediting #sicklecell #crisprsicklecell #crispr

More than four years ago, American Victoria Gray received a life-changing phone call. It was her hematologist, offering her entry into a clinical trial with an experimental drug. Gray, 37, has sickle cell anemia and was given seven years to live at birth. In July 2019, Gray became the first patient to receive a new therapy for her disease based on gene editing with CRISPR. This revolutionary technology invented in 2012 makes it possible to correct errors in the instruction book of 3 billion letters of DNA that make up the genome of a human being.

The new treatment involved extracting blood stem cells from Gray’s bone marrow, isolating them in the lab and using CRISPR’s molecular scissors to cut her genome right at the position of the BCL11A gene. Nearly four years later, Gray feels like she has a new life ahead of her. “I’m no longer in pain and haven’t had to be admitted to the hospital, when I used to have to go every few months.” Dozens of people in several countries have participated in clinical trials with this therapy, developed by Vertex Pharmaceuticals and CRISPR Therapeutics. These treatments will be among the most expensive in the world. They will cost around three million dollars (about 2.8 million euros), to which one must add the cost of several months of hospitalization, transfusions and chemotherapy. #geneediting #crispr

More than four years ago, American Victoria Gray received a life-changing phone call. It was her hematologist, offering her entry into a clinical trial with an experimental drug. Gray, 37, has sickle cell anemia and was given seven years to live at birth. In July 2019, Gray became the first patient to receive a new therapy for her disease based on gene editing with CRISPR. This revolutionary technology invented in 2012 makes it possible to correct errors in the instruction book of 3 billion letters of DNA that make up the genome of a human being.

The new treatment involved extracting blood stem cells from Gray’s bone marrow, isolating them in the lab and using CRISPR’s molecular scissors to cut her genome right at the position of the BCL11A gene. Nearly four years later, Gray feels like she has a new life ahead of her. “I’m no longer in pain and haven’t had to be admitted to the hospital, when I used to have to go every few months.” Dozens of people in several countries have participated in clinical trials with this therapy, developed by Vertex Pharmaceuticals and CRISPR Therapeutics. These treatments will be among the most expensive in the world. They will cost around three million dollars (about 2.8 million euros), to which one must add the cost of several months of hospitalization, transfusions and chemotherapy. #geneediting #crispr

Newly approved medications are rarely used for sickle cell disease (SCD), according to a study published online March 8 in Blood Advances.

Robert Cronin, M.D., from The Ohio State University in Columbus, and colleagues examined the use of U.S. Food and Drug Administration-approved medications for SCD by age, sex, and geographic location using data from MarketScan from 2016 to 2020. Use of hydroxyurea, l-glutamine, voxelotor, and crizanlizumab, alone or concurrently, was examined as the primary outcome in a cohort of 7,957 individuals aged 18 years or older with SCD. The researchers found that hydroxyurea was used by 24.6 percent of all individuals with SCD, while l-glutamine, voxelotor, and crizanlizumab were used by 2.0, 1.9, and 1.4 percent, respectively. For every 10-year increase in age, the odds of using hydroxyurea, l-glutamine, and crizanlizumab were reduced. The odds of hydroxyurea, but not other medication, use were increased for women (odds ratio, 1.27). “I think it is important that we educate both providers and patients about the options that exist to treat their condition," Cronin said in a statement. "From the patient standpoint, providing patient-friendly material that allows them to be knowledgeable about their disease and advocate for themselves can help improve care for this population." #sicklecell #medication #hydroxyurea #l-glutamine #crizanlizumab

Newly approved medications are rarely used for sickle cell disease (SCD), according to a study published online March 8 in Blood Advances.

Robert Cronin, M.D., from The Ohio State University in Columbus, and colleagues examined the use of U.S. Food and Drug Administration-approved medications for SCD by age, sex, and geographic location using data from MarketScan from 2016 to 2020. Use of hydroxyurea, l-glutamine, voxelotor, and crizanlizumab, alone or concurrently, was examined as the primary outcome in a cohort of 7,957 individuals aged 18 years or older with SCD. The researchers found that hydroxyurea was used by 24.6 percent of all individuals with SCD, while l-glutamine, voxelotor, and crizanlizumab were used by 2.0, 1.9, and 1.4 percent, respectively. For every 10-year increase in age, the odds of using hydroxyurea, l-glutamine, and crizanlizumab were reduced. The odds of hydroxyurea, but not other medication, use were increased for women (odds ratio, 1.27). “I think it is important that we educate both providers and patients about the options that exist to treat their condition," Cronin said in a statement. "From the patient standpoint, providing patient-friendly material that allows them to be knowledgeable about their disease and advocate for themselves can help improve care for this population." #sicklecell #medication #hydroxyurea #l-glutamine #crizanlizumab

Mary tells her story @maryshaniqua: I recently returned home to the U.K. after a three-week trip to Jamaica. Not only was it my first long-haul flight, but it was also my first time using a portable oxygen concentrator.

Not all sickle cell patients require additional oxygen when traveling abroad, but I developed the need a few years ago. In December 2018, I suffered a pulmonary embolism, essentially a blood clot in my lung. Perhaps I’ll share that experience in greater detail in a future column. For now, just know that it means I must take blood-thinning medication for life and am more susceptible to blood clots going forward.

Then, in May 2019, I took a short flight from London to Glasgow, Scotland, and suffered one of my worst sickle cell crises to date. Doctors determined that the reduced oxygen at the higher altitude had triggered my crisis. Both of these experiences mean I now need additional oxygen when flying. When a patient requires this, there may be two options, depending on the airline: oxygen cylinders or a portable oxygen concentrator (POC). I haven’t traveled much since the aforementioned incidents, but I’ve had the chance to use both options in flight. Cylinders are great in that they provide a constant flow of oxygen and tend to be very user-friendly. Patients also have the choice between an oxygen mask, which goes over the mouth and nose, or a nasal cannula. A POC filters and concentrates the surrounding air to generate medical-grade oxygen. The biggest advantage of using a POC is that the amount of oxygen available to the patient is not finite or limited to what can fit into a compressed tank. Also, as a single device, it’s not as bulky to transport. Unfortunately, both cylinders and the POC draw attention. This isn’t ideal if, like me, you don’t like being the center of attention due to anything medical-related. Apart from that, I’m grateful to have these options so that I don’t have to miss out on travel opportunities because of sickle cell complications. #poc #supplmentaloxygen

Mary tells her story @maryshaniqua: I recently returned home to the U.K. after a three-week trip to Jamaica. Not only was it my first long-haul flight, but it was also my first time using a portable oxygen concentrator.

Not all sickle cell patients require additional oxygen when traveling abroad, but I developed the need a few years ago. In December 2018, I suffered a pulmonary embolism, essentially a blood clot in my lung. Perhaps I’ll share that experience in greater detail in a future column. For now, just know that it means I must take blood-thinning medication for life and am more susceptible to blood clots going forward.

Then, in May 2019, I took a short flight from London to Glasgow, Scotland, and suffered one of my worst sickle cell crises to date. Doctors determined that the reduced oxygen at the higher altitude had triggered my crisis. Both of these experiences mean I now need additional oxygen when flying. When a patient requires this, there may be two options, depending on the airline: oxygen cylinders or a portable oxygen concentrator (POC). I haven’t traveled much since the aforementioned incidents, but I’ve had the chance to use both options in flight. Cylinders are great in that they provide a constant flow of oxygen and tend to be very user-friendly. Patients also have the choice between an oxygen mask, which goes over the mouth and nose, or a nasal cannula. A POC filters and concentrates the surrounding air to generate medical-grade oxygen. The biggest advantage of using a POC is that the amount of oxygen available to the patient is not finite or limited to what can fit into a compressed tank. Also, as a single device, it’s not as bulky to transport. Unfortunately, both cylinders and the POC draw attention. This isn’t ideal if, like me, you don’t like being the center of attention due to anything medical-related. Apart from that, I’m grateful to have these options so that I don’t have to miss out on travel opportunities because of sickle cell complications. #poc #supplmentaloxygen

The Sickle Cell Trait Awareness Campaign (STAC) was launched in January after 5 years of efforts from the Cayenne Wellness Center, which supports individuals living with sickle cell disease in California. The initiative is aiming to reach adolescents of all races, because despite sickle cell disease (SCD) being predominant in individuals of Black and Hispanic descent, sickle cell trait affects everyone, and it's not asymptpomatic as was once thought. "It behooves us to target the athletes and the coaches at the schools, so that they can become aware," Carolyn Rowley, PhD, Executive Director of Cayenne Wellness Center said in an interview with HCPLive. The awareness only means that you make accommodations for them to be able to hydrate, that you make accommodations for when they say 'I need to sit this one out for a moment', that they listened to that." Individuals with sickle cell trait not only need to know their status to make informed family planning decisions, but also so they can watch out for the possible symptoms.

"There's a rare form of cancer that can actually occur, that we didn't know. And when you over exert, that can be fatal, if it's not attended to," she continued. Research has shed light on a lot of areas related to sickle cell trait that hadn't been observed before. In addition to that, the societal perspective of chronic illness has also shifted.

"If you're feeling with it, though, back in the day, it was 'push, push, push, push, pain, pain, pain is great,' and they would be without water - we can't allow that to occur," Rowley said. "We don't want that to occur. So, we want people, we want the coaches and the athletes to be aware, it is okay. As a matter of fact, everyone should hydrate. #sicklecelltrait

The Sickle Cell Trait Awareness Campaign (STAC) was launched in January after 5 years of efforts from the Cayenne Wellness Center, which supports individuals living with sickle cell disease in California. The initiative is aiming to reach adolescents of all races, because despite sickle cell disease (SCD) being predominant in individuals of Black and Hispanic descent, sickle cell trait affects everyone, and it's not asymptpomatic as was once thought. "It behooves us to target the athletes and the coaches at the schools, so that they can become aware," Carolyn Rowley, PhD, Executive Director of Cayenne Wellness Center said in an interview with HCPLive. The awareness only means that you make accommodations for them to be able to hydrate, that you make accommodations for when they say 'I need to sit this one out for a moment', that they listened to that." Individuals with sickle cell trait not only need to know their status to make informed family planning decisions, but also so they can watch out for the possible symptoms.

"There's a rare form of cancer that can actually occur, that we didn't know. And when you over exert, that can be fatal, if it's not attended to," she continued. Research has shed light on a lot of areas related to sickle cell trait that hadn't been observed before. In addition to that, the societal perspective of chronic illness has also shifted.

"If you're feeling with it, though, back in the day, it was 'push, push, push, push, pain, pain, pain is great,' and they would be without water - we can't allow that to occur," Rowley said. "We don't want that to occur. So, we want people, we want the coaches and the athletes to be aware, it is okay. As a matter of fact, everyone should hydrate. #sicklecelltrait

.pro-gallery-wix-wrapper {display: block !important;} .pro-gallery-wix-wrapper .gallery-item-container {opacity: 1 !important; display: block !important;}

Follow us on Instagram

@thesicklecycle

"Instead of viewing the disease as a burden, I choose to focus on using my voice as a platform to enhance awareness."

Charlotte Curtis

Founder and President of Sickle Cycle

bottom of page