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

TASCA Newsletter (Summer 2020)

The latest TASCA newsletter is out now. Have a read and get updated on all of TASCA’s activities and advocacy work! 

Contents:

  • Chair’s address
  • TASCA 2020 AGM
  • Christmas Activities and Self Care
  • Thalia App
  • Patient Pathway Program
  • Interview with Donna, Social Worker
  • TASCA Store Launch
  • Around Australia
  • TASCA 4 Kids 

Want a physical copy? Let us know by sending a request through email.

You can also download a PDF here.

View previous newsletters here.

TASCA’s Elves at Work!

TASCA has been hard at work in Melbourne’s treatment centres to make them extra festive for the holidays!

Thank you to the team at Royal Melbourne Hospital (5 West Day Medical Centre) and Monash Health (Medical Infusion Unit) for letting our elves bring a little Christmas cheer to your space!

Be sure to take a photo when you are in for treatment and take a candy cane from the TASCA Christmas Tree!

 
 

Peter on 3XY Radio Hellas

Peter, TASCA’s Chair, will be a guest on John Paganis’ radio segment live at 5pm next Friday on 3XY Radio.

Peter will be speaking about his own experience living with thalassaemia.

Tune in to AM 1422 or stream it online.

TASCA Guests on 3XY Radio Hellas

Dr John and Mary Tassigiannakis will be guests on John Paganis’ radio segment live at 5pm tomorrow on 3XY Radio Hellas.

They will be speaking about haemoglobin disorders and the shifting landscape of the conditions in Australia.

Tune in to AM 1422 or stream it online.

TASCA AGM 2020 Notice

Thalassaemia and Sickle Cell Australia

Dear Member     

You are invited to attend TASCA’s Annual General Meeting to be held on 11th November 2020, online via ZOOM. (See link details in the agenda) 

TASCA is formally calling for nominations to the Committee of Management for the period 2021-2022. The Committee consists of 9 positions, 8 of which are currently filled.   

The Committee of Management positions are as follows:  

Executive Committee 

  • Chair – Peter Verveniotis 
  • Deputy Chair – Robbin Vissakodeti 
  • Treasurer – Joanne Mucciaciaro 
  • Secretary – Pat Bollard 

General Committee 

  • Yannis Androulakis 
  • Ella Luong 
  • Karen Parr 
  • Dr John Malios 

Therefore, one General Member position will be open to nominations from current financial members. The term of the position will be two years as per the Rules of Association.      

The following documents are attached: 

Please read all documents carefully and note that if you wish to nominate either yourself or another person to Committee positions, current financial membership of TASCA is necessary. 

The Committee and I look forward to seeing you there. 

Please RSVP through Eventbrite using the link below

Kind regards 

Peter Verveniotis 

Chair 

TASCA Newsletter (Spring 2020)

The latest TASCA newsletter is out now. Have a read and get updated on all of TASCA’s activities and advocacy work! 

Contents:

  • Chair’s address
  • TASCA 2020 AGM
  • TASCA Update
  • Upcoming Events
  • Research Developments
  • Who’s Inspiring Us
  • TASCA Online
  • Around Australia
  • TASCA 4 Kids (Including fun activities and competition for kids!)

Want a physical copy? Let us know by sending a request through email.

You can also download a PDF here.

View previous newsletters here.

TASCA Newsletter (Winter 2020)

The latest TASCA newsletter is out now. Have a read and get updated on all of TASCA’s activities and advocacy work!

Contents:

  • Mental Health Support for Patients
  • Chair’s Report
  • Australia Lit Up Red
  • National Blood Donor Week
  • Medical Infusion Unit Opening
  • Gina’s Birthday Fundraiser
  • Smart Apps and Anemia
  • TASCA Staff Changes
  • Online Chat Room for Teens
  • Genetics Workshops
  • Social Media Challenges
  • Upcoming Events

Want a physical copy? Let us know by sending a request through email.

You can also download a PDF copy.

View previous newsletters here.

[Media Release] AUSTRALIA LIGHTS UP RED FOR WORLD SICKLE CELL DAY

FOR IMMEDIATE RELEASE

DATE: 17/06/2020

AUSTRALIA LIGHTS UP RED FOR WORLD SICKLE CELL DAY

Melbourne, Australia: LANDMARKS across Australia are set to light up red for World Sickle Cell Day on 19 June to shine a light on the rare genetic blood condition unknown to many around the country

For the first time, structures including the Melbourne Star, Brisbane’s Story Bridge, Perth’s Trafalgar Bridge and Canberra’s Telstra Tower will light up red in solidarity for patients affected across Australia by sickle cell anaemia. More than 270,000 infants are born with sickle cell anaemia each year across the globe, and cases are increasing in Australia.

Thalassaemia and Sickle Cell Australia (TASCA), the nation’s peak patient advocacy body, has coordinated this event to raise awareness on the condition. TASCA is a not-for-profit organisation that has been serving Australians living with genetic haemoglobin disorders, and their families, since 1976.

Sickle cell anaemia affects the body’s ability to produce healthy red blood cells that provide oxygen around the body. Sufferers battle frequent, painful blood clotting episodes caused by the sickle-shaped blood cells and require lifelong blood exchanges.

Robbin Vissakodeti, TASCA’s Deputy Chair, whose wife and daughter both have a form of genetic blood disorders said, “For us, sickle cell affects our everyday lives. My wife had her spleen removed because of sickle cell complications and we have to take every precaution to prevent infections as her immune system is compromised.

“When we started thinking about having kids, I knew the risks and we were prepared. World Sickle Cell Day this year is about getting people to have that conversation with their doctors, being informed about the risk and making the right decision when it comes to having children.

“Hospital visits definitely take a toll on the family, but we are grateful that we live in a country with a supportive healthcare system. Treatment seems to be constantly improving and we are looking forward to what the future will bring.”

ENDS

For media enquires contact:

Samuel Lam, Thalassaemia and Sickle Cell Australia

E: hpo@tasca.org.au, T: 03 7015 5637

Notes to editors:

  • For more information on TASCA, sickle cell anaemia and World Sickle Cell Day, visit tasca.org.au.
  • Full list of sites that will participate in the light up include:
    • Melbourne Star (VIC)
    • AAMI Park (VIC)
    • Kingston Clock Tower (VIC)
    • Story Bridge (QLD)
    • Victoria Park (QLD)
    • Trafalgar Bridge (WA)
    • Council House (WA)
    • Canberra Telstra Tower (ACT)
    • Elizabeth Mall (TAS)
    • Kennedy Lane (TAS)
    • Railway Roundabout (TAS)
    • Franklin Square (TAS)
  • Thalassaemia and Sickle Cell Australia was established in 1976 and provides support and advocacy to those affected by genetic blood disorders.
  • Sickle cell anaemia is an inherited blood condition causing misshapen red blood cells called sickle cells. This leads to a reduction of useful red blood cells and anaemia. Sickle cells also result in painful and hospitalisable blood clots in many patients.
  • A simple blood test will show if couples are at risk of having a child with sickle cell anaemia.
  • Treatment for sickle cell anaemia include medication and blood exchanges.
  • Sickle cell anaemia is most common in people of sub-Saharan African, Indian, Middle Eastern and Mediterranean background. However, migration has spread this condition globally.