Monday 4 March 2024
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“Compassionate use” boost for children of families living with a rare disease

Families with children with a rare neurodegenerative disease, Ataxia Telangiectasia (A-T), received a major boost in June after news they could continue to receive a trial treatment thanks to the Nottingham University Hospitals Research & Innovation team.

The announcement that they could continue to receive Intra-Erytrhcyte Dexamethasone Sodium Phosphate – which they were receiving as participants in the ATTeST clinical trial – for compassionate usage is great news, and NUH is the only research centre in the world to have currently secured this agreement.

Members of staff in a number of teams combined forces to gain agreement to use the experimental drug on an ongoing basis and plan the logistics for its delivery to patients.

Helen Navarra, manager of the Children’s Research team at Research & Innovation, praised staff in departments including Clinical Contracts and Pharmacy who went beyond the call of duty and worked on beyond their usual hours until agreement to continue the treatment was confirmed.

She said: “This decision will have a huge impact on the families of the children involved. Everyone who has received the treatment at the QMC as part of the international ATTeST trial can now continue to receive it, which is very welcome news.”

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The EryDex system, for treating patients with AT, does not yet have full regulatory approval for widespread use, but preliminary results for the trial – conducted by paediatric researchers based at Nottingham Children’s Hospital and the NIHR Nottingham Clinical Research Facility (CRF) – have been encouraging.

Data showed that, especially in young children, the drug slowed the disease progression, compared to patients taking placebo.

A-T affects around 200 families in the UK, and it results in children losing their ability to walk through ataxia and dystonia. Children with the disease can become more susceptible to cancers including leukaemia and lung infections.

Many A-T patients will not survive beyond their twenties and there is presently no proven treatment.

Over 20 years ago, NUH set up the national children’s A-T clinic and has close links to many of the families and children with the disease from around the UK and abroad. Dr William Whitehouse has been pivotal to this service and the ATTeST trial.

Administration of the ATTeST trial treatment employs a complex system and relies on the experimental medicine skills of the Children’s Clinical Research Team.

Special trained nurses run the EryDex machine, which administers the trial drug into participants’ red blood cells through a transfusion process.

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