Don't Deny Children The Chance Of Child-effective Treatments, Say Doctors And Parents

Main Category: Cystic Fibrosis
Also Included In: Genetics;  Pediatrics / Children's Health
Article Date: 25 May 2006 - 12:00 PDT

email to a friend   printer friendly   opinions  

New treatments are normally tested on adults before they are tested on children. A paper by doctors involved in gene therapy for cystic fibrosis argues that approach could be wrong. "We could be denying children life enhancing treatment on inadequate grounds" said lead author Dr Adam Jaffe.

There is widespread interest in gene therapy as a possible treatment for cystic fibrosis - a life-limiting condition, and the most common single gene disorder in white Caucasians. We are working towards the first ever trial to be undertaken in children with cystic fibrosis at Great Ormond Street Hospital for Children, helped made possible by funding from the Department of Health. We envisage using a new vector, which helps genes get into lung cells, developed by Dr Stephen Hart from the Institute of Child Health.

"There are scientific and medical reasons why the treatment might work better in children," said Dr Jaffe. "The main aim is to halt the decline in lung damage, which begins soon after birth so it makes sense to start treatment in early childhood. Children are not little adults; and this is particularly true in cystic fibrosis. "

"It is possible that if the gene therapy trials were carried out in adults we would not see a benefit as the mucus in the lungs of adults may act as a barrier and stop the gene from getting in. Furthermore, inflammation can be more serious in adults so they may suffer more severe side effects from some vectors which help to get the gene into the cell. There is evidence from other research that gene transfer may happen differently in growing and adult lungs."

In other words, Dr Jaffe Dr Hart and colleagues conclude "Tests in adults may not tell us how effective the treatment in children would be."

"We may be denying an effective treatment in children because of a misplaced sense that we ought to try the treatment in adults first."

The paper examines the scientific, medical, and ethical/ consent issues around a prospective trial. As part of this the team reports a survey of parents with CF. A very large proportion of families responding wanted to be offered the chance of taking part of clinical trials of gene therapy in their children.

About the GREAT ORMOND STREET HOSPITAL

GOSH is probably the UK's best known children's hospital, specialising in children with rare and complex conditions. It has the widest range of children's specialists of any UK hospital and is the biggest centre for research into childhood illness outside the USA.

GREAT ORMOND STREET HOSPITAL
3rd Floor, 26-27 Boswell Street

empeys@gosh.nhs.uk
http://www.gosh.nhs.uk

• Additional
• References
• Citations