Global Blood Therapeutics, Inc. (GBT) announced that the U.S. Food and Drug Administration (FDA) has informed GBT through discussions and written correspondence that the agency agrees with the Company’s proposal relating to use of an accelerated approval pathway for voxelotor for the treatment of sickle cell disease (SCD). GBT plans to submit an NDA for voxelotor for the treatment of SCD under this pathway. GBT proposed that by raising hemoglobin, voxelotor is reasonably likely to reduce strokes in SCD patients. As part of these discussions, the FDA agreed that transcranial doppler (TCD) flow velocity would be an acceptable primary endpoint in a post-approval confirmatory study to demonstrate stroke risk reduction.

  After over a decade of preclinical research and development, a new gene therapy treatment for sickle cell anemia (SCA) is reversing disease symptoms in two adults and showing early potential for transportability to resource-challenged parts of the world where SCA is most common. Preliminary data from a pilot Phase 1-2 clinical trial testing the gene-addition therapy were presented on Dec. 3 at the American Society of Hematology's (ASH) annual meeting in San Diego by principal investigator Punam Malik, MD. She is a physician-scientist at the Cincinnati Children's Cancer and Blood Diseases Institute and director of its Comprehensive Sickle Cell Center. Malik called the data promising. One year after treatment of our first patient, and six months after treatment of our second patient, both have seen a remarkable improvement in the quality of life due to remarkable reduction in disease symptoms.  

An inexpensive, easy-to-use diagnostic tool for sickle cell disease showed 100% accuracy among a large cohort of children from Uganda, according to findings presented during the late-breaking abstract session of ASH Annual Meeting and Exposition.

The test — HemoTypeSC (Silver Lake Research Corporation) — only requires a drop of blood and produces results in about 10 minutes.

Diagnosis, prophylaxis, counseling and treatment have dramatically reduced sickle cell disease incidence and mortality in the developing world, but screening for the disease has not been widely implemented in those regions, particularly in sub-Saharan Africa, according to Erik Serrao, PhD, director of business and product development at Silver Lake Research Corp.

“Although this disease is acknowledged as a public health problem by the U.N. and WHO, African governments and ministries of health still largely neglect this disease,” he said during a press conference. “Individuals with this disease are commonly identified only after they die or when they present at the hospital with life-threatening symptoms.”

  Nigerians are paying tribute to popular radio presenter and entertainer Tosyn Bucknor, who has died aged 37. Bucknor died from complications due to sickle cell, her sister and renowned event planner, Funke Bucknor Obruthe said in an Instagram post. "My darling sister and besto Tosyn passed away last night due to complications from sickle cell," she wrote. Bucknor was found unconscious by her husband, a French national, Aurélien Boyer when he returned home from work on Monday night, according to local media reports. Known as the 'Voice of Lagos,' her early morning shows aired on two local radio stations and attracted a strong fanbase among young Nigerians. She was vocal about her health condition and founded the 'These Genes Project' in 2007 to raise awareness about sickle cell.  

Brunel Etienne says one his childhood dreams has come true thanks to New England Patriots free safety Devin McCourty. "I got two Super Bowl tickets from Devin McCourty. I was completely awestruck," Brunel said.

The 26-year-old was born with sickle cell anemia, an inherited life-threatening blood disease common among African Americans. Brunel actually suffered a severe stroke when he was young. "One big stroke where my whole left side was paralyzed," he said. "Now my left side is weaker than my right side."

The McCourty twins have made it their mission to help tackle sickle cell. Some of their relatives also have the disease. Recently Devin invited Brunel to Gillette Stadium and surprised him with two tickets to Super Bowl LIII. "I always wanted to go to the Super Bowl but never thought it would come so soon," Brunel said.

 

Twin brothers Jason and Devin McCourty shared on social media Sunday that their aunt, Winifred, has died following a difficult battle with sickle cell disease. “She fought tirelessly with unwavering faith to live a full life even when doctors told her she wouldn’t live to see 35,” they wrote. “Her life is a true testament of defying the odds.” Winifred was the inspiration behind the “Tackle Sickle Cell” campaign — an initiative the McCourty brothers started at Rutgers to raise awareness and money to benefit those affected.  

One in 2,400 are born with sickle cell disease (SCD) in the U.S. As one of the most common inherited blood disorders, it is widely misunderstood. Last month, the Minnesota Sickle Cell Coalition — comprised of patients, doctors, healthcare providers, and advocates — co-sponsored a Sickle Cell Disease Stakeholder Forum at Sanctuary Covenant Church in North Minneapolis. Approximately 60 healthcare professionals and others attended the Dec. 10 event to not only help improve care for those afflicted by the disease but also to increase awareness of the disease, screening and healthcare disparities. SCD is a group of inherited red blood cell disorders including Sickle Cell Anemia (Hgb SS Disease), Hemoglobin SC Disease, and Hemoglobin Sickle Beta Thalassemia. People with sickle cell disease produce abnormal hemoglobin (Hgb S), which can interrupt blood flow and prevent oxygen from getting to important parts of the body. This can cause many potential health complications, including extreme pain, risk of stroke, and pulmonary hypertension.  

Camp New Hope is a seven-day; six-night residential camp for children ages 7-17 with sickle cell disease. Our mission is to provide an enriching experience that fosters independence, self-esteem, and friendships.

Camp New Hope offers campers the excitement and fun of a traditional summer camp in addition to the unique advantages of an environment and staff responsive to their unique health concerns. Campers enjoy a week of adventure while learning self-help, sickle cell disease management skills, and developing a more positive self-image. Campers are encouraged to explore a variety of interests including swimming, canoeing, tennis, climbing, and arts and crafts.

Date:
July 17, 2022 – July 23, 2022

 

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Elliott Vichinsky estimates that at least 30% of his adult patients with sickle-cell disease die from preventable causes. Red blood cells are supposed to be shaped like concave discs, but in people with sickle-cell disease, a mutation in a single gene collapses them into a crescent shape. The pointy sickles catch on each other and clog blood vessels. They cut off oxygen to limbs. They cause kidney failure, hypertension, lung problems and strokes — along with bouts of excruciating pain. These are common and treatable complications, so why the high death rate? Vichinsky attributes it to a lack of infrastructure, such as care centres, to properly monitor adults with sickle-cell disease. This is partly because the disease mainly affects low-income minorities and people in developing countries. “If they were tracked before,” says Vichinsky, “they would not be dead.”  

The Chicago Sickle Cell Summit convenes stakeholders and providers from health system leadership, primary and specialty care, and the sickle cell community for updates in sickle cell care. Our goal is to create synergy to improve care for sickle cell patients across Chicago. Lunch will be served. Hourly-rate parking is available in the UI Health parking facility on Wood and Fillmore Streets. Street parking is available on both Wood and Washburne Streets, immediately surrounding the Mile Square Health Center building. Date and Time: Thu, February 28, 2019 12:00 PM – 5:00 PM CST Location: Mile Square Health Center 1220 South Wood Street 3rd floor Chicago, Illinois, United States   This event has ended.