£2.5 Million for gene therapy research into cystic fibrosis

Main Category: Cystic Fibrosis
Article Date: 06 Jan 2005 - 0:00 PDT

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UK Health Minister, Lord Warner today announced the winning bids for £2.5 million to support further research into gene therapy for cystic fibrosis. The funding commitment was made in the Genetics White Paper, 'Our Inheritance, Our Future: Realising the potential of genetics in the NHS' and will help to ensure that those with cystic fibrosis benefit as soon as possible from the latest advances in genetic knowledge and technologies.

Health Minister Lord Warner said:

"The Genetics White Paper set out our vision that the NHS should take maximum advantage of new genetic knowledge and technologies. Supporting this innovative research will help make sure that cystic fibrosis patients benefit as quickly as possible from gene therapy advances. This work represents a major step in our endeavour to relieve the suffering and ultimately find a cure for this terrible disease".

Cystic fibrosis is the most common inheritable disorder in this country, and affects over 7,500 children and adults in the U.K and the life expectancy for children born with this disease is just 30 years. It is a distressing condition in which mucus continually builds up in the lungs causing breathing difficulties and respiratory infections. There is currently no cure although regular physiotherapy, antibiotics and enzyme supplements can slow the progression of the disease.

Following an open call for proposals two contracts, one to the Institute of Child Health and the other to the UK Cystic Fibrosis Gene Therapy Consortium, have now been awarded to support further essential gene therapy research in this area. The research teams will work on a different but complementary strategies in their proposals and have agreed to work together to maximise the likely benefits.

Rosie Barnes from the Cystic Fibrosis Trust said

"Gene Therapy holds a future cure for the thousands of people living with Cystic Fibrosis in the UK. We are delighted that Lord Warner has recognised the importance of this ground-breaking research and hope that the Department of Health will continue to support the work of the Cystic Fibrosis Trust and Gene Therapy Consortium".

Dr Adam Jaffe, Respiratory Consultant at Great Ormond Street added:

"We are delighted that the DoH are supporting the research into gene therapy for children with cystic fibrosis at Great Ormond Street Hospital and the Institute for Child Health. We believe, that gene therapy will be most effective in children with the disease as the aim is to prevent deterioration of lung disease. Our families are very keen to see effective new therapies developed. This support will bring the first ever gene therapy trial in children with CF one step closer".

Related links

http://www.advisorybodies.doh.gov.uk/genetics/gtac

Notes to editor

1. Gene therapy is the deliberate introduction of genetic material into human somatic cells for therapeutic, prophylactic or diagnostic purposes. In the case of single gene disorders, gene therapy offers the potential to cure or ameliorate the induced disease by introducing working copies or modifications of defective genes into cells to correct the inherited defect.

2. "Gene therapy in the UK is regulated by the Gene Therapy Advisory Committee. Website on the link provided:

2. The research contracts were awarded to the UK Cystic Fibrosis Trust and Gene Therapy Consortium for 'Vector testing, selection of clinical trial material and clinical trials on gene therapy for cystic fibrosis' and to Drs Adam Jaffe and Steven Hart at the Institute of Child Health for 'Gene therapy for cystic fibrosis in children nebulised delivery of a synthetic vector formulation to the nose, comparisons of vector formulations'.

3. The complementary research programmes will be conducted in three phases. The first phase will involve the acquisition of the evidence needed to identify which of the vectors available should be used for the subsequent clinical assessments. The second phase will involve the GMP manufacture and any associated toxicological assessments of the chosen vector(s). The final phase will involve clinical trials, the nature of which will depend on the vector(s) chosen.This will require approval from the regulatory authorities, will involve early stage safety clinical trials, the nature of which will depend on the vector chosen at the end of phase one.

4. The Cystic Fibrosis Trust is the UK's only national charity dedicated to all aspects of Cystic Fibrosis. It funds medical research aimed at understanding, treating and curing Cystic Fibrosis. It also aims to ensure that people with CF receive the best possible care and support in all aspects of their lives.

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