World Sickle Cell Day Light Up

In recognition of World Sickle Cell Day on 19th June, Thalassaemia and Sickle Cell Australia has organised the 2nd annual light up of landmarks in Australia. 

 

Share your photo with #LightUpForSickleCell to raise awareness!

 

This year, the following buildings will light up:

 
June 17th
  • Trafalgar Bridge and Perth Council House, WA 
June 18th
  • Bolte Bridge, VIC
June 19th
  • Telstra Tower, ACT
  • Embassy of Greece in Canberra, ACT
  • The Bell Tower, Perth, WA 
  • Palmerston Water Tower and Frances Drive Light Pole, Palmerston NT 
  • Monash Park Tree and Mooroopna Water Towers, VIC
  • Launceston Town Hall,  TAS
  • Adelaide Town Hall Balcony, SA 
  • Story Bridge, Victoria Bridge and the Reddacliff Place sculptures, QLD 
  • Kurilpa Bridge, QLD 
  • Newcastle Clock Tower, NSW
 We want to thank all our light up partners that made this possible! 
  • Adelaide City Council
  • Brisbane City Council
  • City of Launceston
  • City of Newcastle
  • City of Palmerston
  • Embassy of Greece in Canberra
  • Greater Shepparton City Council
  • Telstra
  • The Bell Tower
  • Transurban
  • Queensland Department of Energy and Public Works

Bluebird Bio Announces Temporary Suspension on clinical trials of LentiGlobin Gene Therapy for Sickle Cell Disease

Bluebird Bio, developer of gene therapy Zynteglo, has suspended their clinical study of the treatment on sickle cell patients after adverse events were reported. Two patients developed Acute Myeloid Leukemia (AML), a type of blood and bone marrow cancer, after being treated with Zynteglo five years ago.

A patient was reported last week developing Myelodysplastic Syndrome (MDS), a blood cell production disorder, This follows a case of MDS in 2018 that was attributed to the chemotherapy received in preparation for gene therapy.

It is currently unknown as to whether the development of AML was caused by the viral vector used in the therapy. Further investigations are underway.

No cases of hematologic malignancy such as AML or MDS has been reported in the use of Zynteglo for β-thalassaemia so far. However, the marketing of Zynteglo in Europe has been suspended as a precautionary measure.

Zynteglo, previously known as Lentiglobin, is a gene therapy developed by US Biotechnology company Bluebird Bio for the treatment of sickle cell disease and transfusion-dependent β-thalassaemia. The therapy uses a viral vector to deliver genetic changes to patients with the hope of improving haemoglobin and red blood cell production.

European Medicines Agency Accepts GBT’s Marketing Authorization Application (MAA) for Oxbryta® (voxelotor) for the Treatment of Hemolytic Anemia in Sickle Cell Disease

Global Blood Therapeutics, Inc. (GBT)  announced that the European Medicines Agency (EMA) has completed the validation of GBT’s Marketing Authorization Application (MAA) for Oxbryta (voxelotor) tablets and started its standard review process.

A first-in-class oral, once-daily therapy, Oxbryta directly inhibits haemoglobin polymerization, the root cause of the sickling and destruction of red blood cells in SCD. The sickling process causes hemolytic anaemia (low haemoglobin due to red blood cell destruction) and blockages in capillaries and small blood vessels, which impede the flow of blood and oxygen throughout the body. The diminished oxygen delivery to tissues and organs can lead to life-threatening complications, including stroke and irreversible organ damage.

“Sickle cell disease has a devastating impact on the lives of patients and their families, including serious and life-threatening complications that can lead to organ damage and early death,” said Ted W. Love, M.D., president and chief executive officer of GBT. “Despite this overwhelming need, there are currently no approved therapies in Europe that have the potential to modify the course of the disease. We look forward to working with the EMA to meet our goal of bringing the first treatment for hemolytic anaemia in sickle cell disease to European patients as soon as possible.”

The marketing authorisation application is based on data from the Phase III HOPE study and the Phase II HOPE-KIDS 1 study, both of which enrolled patients at clinical sites in Europe. The HOPE study achieved its primary endpoint.

The analysis of the complete data from the HOPE study further demonstrated that Oxbryta, at a daily dose of 1,500mg, resulted in durable improvements in haemoglobin levels and markers of haemolysis over 72 weeks of treatment.

Oxbryta has already been approved in the US for the treatment of SCD in adults and children 12 years of age and older.

Read the Full GBT Press Release HERE