TASCA Newsletter (Autumn 2021)

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

Contents:

  • Our New Ambassador
  • Committee Updates
  • TIF Conference
  • Rare Disease Day
  • Lucas Garage Sale
  • Decathlon BBQ
  • International Women’s Day
  • TASCA’s Christmas Wrap Up
  • TASCA 4 Kids Page

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 Supports Rare Disease Day 2021

What is a rare disease?
  • The most widely accepted definition is that a rare disease is one that affects less than five in 10,000 people.
  • It is prominently cited that there are more than 7,000 different rare diseases.
  • While individual diseases may be rare, the total number of Australians living with a rare disease is not. Approximately eight per cent of Australians live with a rare disease. Extrapolated to an Australian population of over 25 million people, this equates to around two million Australians.
  • Approximately 80 per cent of rare diseases are of genetic origin.
  • While there is large variation among rare diseases, people living with a rare disease face common challenges including timely and accurate diagnosis, limited care and support options and lack of research.
National Strategic Action Plan for Rare Diseases

In February 2020, Australia’s Minister for Health launched the National Strategic Action Plan for Rare Diseases (the Action Plan), ahead of Rare Disease Day. Rare Voices Australia (RVA) led the collaborative development of the Action Plan, which involved extensive stakeholder consultation with all key stakeholders in the rare disease sector. 

The Action Plan has three interrelated Pillars with Priorities, Actions and Implementation areas that work towards the best possible outcomes for Australians living with a rare disease. 

The three Pillars are: 

1. Awareness and Education 

2. Care and Support 

3. Research and Data 

‘Developed by the rare disease sector, for the rare disease sector,’ the Action Plan can drive and future-proof much needed reform. 

Rare Disease Day 

First launched in 2008 by EURORDIS – Rare Diseases Europe, Rare Disease Day 2021 (28 February) will mark the thirteenth time that rare diseases have been recognised globally. It will be the twelfth year the event will be commemorated in Australia. 

The main objective of Rare Disease Day is to raise awareness among the general public and decisionmakers about rare diseases and their impact on people’s lives. In 2020, thousands of events were organised in over 100 countries and regions to mark the day. The rare disease community united across borders to show that Rare is many. Rare is strong. Rare is proud! In Australia, a number of events were held including the Action Plan launch at Parliament House in Canberra, the Rare Together Expo in Perth, Rare Star Day, the Rare Disease Day Fun Run and Walk in Cairns and more. A number of buildings around the country were also lit up to mark the day.

 Source: Rare Voices Australia

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.