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FDA Reviews Groundbreaking Gene-Editing Cure for Sickle Cell Disease

FDA Reviews Groundbreaking Gene-Editing Cure for Sickle Cell Disease

A groundbreaking development in the quest to find a cure for sickle cell disease has emerged as the Federal Drug Administration (FDA) is currently reviewing a promising gene-editing treatment. This innovative approach has the potential to revolutionize the treatment of sickle cell disease, a condition that disproportionately affects African Americans in the United States. For decades, the primary treatment for this painful and life-threatening disease has been bone marrow transplantation. However, the gene-editing therapy currently under FDA review offers an alternative method, which may hold particular significance for the Black community in the United States.

The Impact of Sickle Cell Disease on African Americans

Sickle cell disease is a genetic disorder that primarily affects African Americans. It is characterized by the production of abnormal, crescent-shaped red blood cells that hinder the flow of oxygen throughout the body, causing excruciating pain, fatigue, and a range of serious health complications. The disease affects approximately 1 in 365 African American births, making it a pressing health concern for the community.

Historically, the Black community has been underserved when it comes to sickle cell disease. For many years, the medical community and federal government failed to adequately address this issue, leaving patients with limited treatment options. Unlike other conditions like diabetes and cancer, which have seen numerous therapeutic advances, sickle cell disease had few available treatments.

The Impact of Sickle Cell Disease on Detroiters

Approximately 3,000 residents of Michigan are currently affected by sickle cell disease, spanning both adults and children. Detroiters living with sickle cell disease frequently grapple with painful episodes known as pain crises, which can, on occasion, necessitate hospitalization. These episodes of excruciating pain are a challenging aspect of their ongoing battle with the disease, highlighting the need for improved care and support for those facing this condition. These painful episodes remain a constant threat, and newer treatments come with their own set of challenges. Some of the recently authorized treatments have serious side effects, while others are prohibitively expensive, making them inaccessible to many patients. Basic quality care for the disease remains elusive for numerous adult patients in Detroit.

One remarkable Detroit teenager is helping close the gap in patient care for Detroiters living with the disease.  MiCaringHeart, a foundation dedicated to ensuring that sickle cell patients reach their crucial medical appointments was founded by Reem Siddiqui, a compassionate high school student who was moved to make a difference while spending time at her mother’s workplace, the Children’s Hospital sickle cell clinic. It was there that she noticed that some patients faced a significant barrier to receiving the care they needed – the inability to make it to their medical appointments due to a lack of transportation.

Motivated by her observations, Reem Siddiqui launched her foundation last year and began raising funds to cover the roundtrip transportation expenses for these young patients. Through her dedicated efforts, she managed to collect a substantial sum of $40,000, which she used to provide invaluable support to these families.

This is the essence of the Detroit sickle cell community – unity and problem-solving. Many advocates within this community are either personally affected by the condition or have close connections to someone who is, making them ideal champions for the cause, both in the battle against the disease and in their ongoing commitment to finding solutions.

“We’re doing our best and building momentum, but we’re still dealing with a lot of health disparities, and we’re still being considered drug seekers,” said Clifton Kirkman II, a sickle cell patient and known advocate in the City of Detroit. “I may have sickle cell anemia, but I don’t live each day to make that an excuse.”

Dr. Wanda Whitten-Shurney, CEO and Director of Sickle Cell Disease Association of America, Michigan Chapter has been a compassionate and well-known presence for numerous families who have entrusted her with the outpatient care of their children throughout her three-decade career. Her unwavering dedication to assisting children and their families in coping with sickle cell has been marked by a strong focus on education and effective coping strategies, all aimed at enhancing the well-being and vitality of individuals.

“There are lots of people living well with Sickle Cell disease. There are people with gray hair living with sickle cell disease. We just want to dispel some of those myths and for people to be aware of the fact that this is a very challenging patient population that we’re trying to serve,” said Dr. Whitten-Shurney.

The Promise of Gene Editing

The gene-editing treatment under FDA review, known as “exa-cel,” is based on CRISPR technology, which was awarded the Nobel Prize in 2020 for its revolutionary impact on genetics and medicine. This technology has the potential to permanently alter the DNA of blood cells, effectively reprogramming the body to stop producing abnormal sickle-shaped cells.

For patients like Victoria Gray of Forest, Mississippi, the gene-editing technology has already shown remarkable success. She has been living without symptoms of sickle cell disease for nearly four years, thanks to this innovative treatment. Her story is a testament to the potential of gene editing to transform the lives of sickle cell patients.

The FDA’s Crucial Role

The FDA’s ongoing review of exa-cel represents a significant step forward in the fight against sickle cell disease. If approved, this gene-editing treatment could offer new hope to the approximately 100,000 sickle cell patients in the United States. The FDA’s advisory committee is currently reviewing the treatment, and their recommendations will play a pivotal role in the ultimate decision. While the recommendations are not legally binding, the FDA typically follows them.

Ethical Concerns

However, gene editing is not without its ethical concerns. Velvet Brown-Watts, the executive director of Supporters of Families with Sickle Cell Disease, recognizes the complexities of this technology. She left her job in the medical field to become a full-time advocate after her son was diagnosed with sickle cell disease. Brown-Watts raises questions about the long-term implications of permanently altering a person’s DNA through gene editing. Will the individual still carry the same bloodline and family DNA down the line? These questions highlight the importance of ethical discussions surrounding gene editing technologies, even as they offer hope for treating sickle cell disease.

Hope for the Future

The FDA’s impending decision on the approval of the gene-editing treatment for sickle cell disease is eagerly anticipated by the entire sickle cell community. With gene editing, there is new-found optimism for those affected by this debilitating condition. The potential to free patients from pain crises and hospitalizations, as demonstrated in clinical trials, offers a glimmer of hope for a community that has long been overlooked in the realm of medical advancements.

The FDA’s review of gene-editing treatment for sickle cell disease is not only a significant milestone in medical science but also a beacon of hope for the African American community. With the potential to revolutionize sickle cell treatment, this groundbreaking technology could offer relief to those who have long suffered from the disease’s debilitating effects. As the FDA’s decision draws near, the Black community and the entire nation hold their breath in anticipation of a brighter future for those battling sickle cell disease.

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