News archive 2005

HFEA grants cloning licence to King's and Roslin Institute

08 Feb 2005, PR 12/05

Dolly-the-sheep creator, Professor Ian Wilmut and Dr. Paul de Sousa, who are both from the Roslin Institute, and King's College London researcher Professor Christopher Shaw have today announced that they have been granted a licence by the Human Fertilisation and Embryology Authority (HFEA) to generate stem cell lines using Cell Nuclear Replacement for the study of Motor Neurone Disease (MND).

‘This is only the second licence ever granted by the HFEA to use this technique, often referred to as therapeutic cloning. Our aim will be to generate stem cells purely for research purposes,' said Professor Wilmut. The researchers plan to generate stem cells that carry MND-causing gene defects. By turning these stem cells into motor neurones they will have a unique opportunity to discover what cause these cells to degenerate. ‘We will compare the behaviour and chemical profile of neurones with the gene defect to those without. This will tell us about the earliest events that ultimately lead to cell death,' said Professor Shaw.

The cultured neurones will also be used to discover drugs that can stop or reverse the disease process. Hundreds of thousands of drugs can be quickly screened in cultured cells within a year for around £100,000, whereas, it takes nearly two years and £20,000,000 to screen just one drug in MND patients. This technique could dramatically accelerate the discovery of new drugs that can effectively block the disease process.

‘This is potentially a big step forward for MND research,' said Professor Shaw. ‘We have spent twenty years looking for genes that cause MND and to date we have come up with just one gene. We believe that the use of Cell Nuclear Replacement will greatly advance our understanding of why motor neurones degenerate in this disease, without having to first hunt down the gene defect.'

To generate stem cells the researchers will first grow in the laboratory skin or blood cells from people who have an inherited form of MND whose genetic cause is unknown. They will then remove the genetic information (nucleus) from an unfertilised egg, and replace it with the nucleus of a cell from a familial MND patient. Eggs that have successfully received the nucleus containing the MND-causing gene defect will be encouraged to grow up to the 200 cell stage, (smaller than the head of a pin). Then embryonic stem cells will be removed, grown and directed to become motor neurones using a cocktail of growth factors.

‘This is not reproductive cloning in any way,' said Professor Wilmut. ‘The eggs we use will not be allowed to grow beyond 14 days. Once the stem cells are removed for cell culture the remaining cells will be destroyed. The embryonic stem cells that we derive in this way will only be used for research into motor neurone disease.'

Motor neurone disease is a relentlessly progressive muscle wasting disease that causes severe disability from the outset, and death usually within three to five years of onset. Every year, 1,200 people in the UK die of MND and at present there is no drug treatment that significantly improves survival.

‘We are delighted with the HFEA decision. Now that the licence has been granted the next task is to raise enough money to carry out this research, which is easier said than done' said Professor Shaw.

Notes to editors

About Motor Neurone Disease
Motor neurones in the brain and spinal cord normally work together using electrical impulses to activate the muscles of the body, precisely controlling all movements. In MND motor neurones degenerate and the muscles they control become weak and wasted. People affected by MND may progressively lose the ability to walk, talk, eat, feed or bathe themselves. Understandably this can engender feelings of helplessness and despair.

The strongest clue to the causes of this dreadful disease comes from the 5-10% of patients in whom there is a strong genetic basis. MND in these families is passed down through the generations due to an inherited gene defect but the vast majority of the defective genes are unknown. Gene hunting in families with other inherited conditions is usually done by collecting a large number of DNA samples from individuals with the disorder in a single family. The defective gene can be linked to a particular chromosome by finding out which genetic markers are shared by all of the affected individuals and therefore linked to the disease. These genetic linkage studies are extremely difficult in MND because in most families the affected individuals have long since died and their DNA is not available. Twenty years of genetic research in MND has yielded only one gene, called SOD1, which accounts for only 3-5% of all cases.

King's College London
King's is one of the oldest and largest colleges of the University of London with 13,800 undergraduate students and some 5,400 postgraduates in ten schools of study. The College had 24 of its subject-areas awarded the highest rating of 5* and 5 for research quality, demonstrating excellence at an international level. The recent Institutional Audit, carried out by the Quality Assurance Agency, received an excellent result.

King's is in the top group of universities for research earnings with income from grants and contracts of £100 million and has an annual turnover of £348 million. In 2004 the College was once again awarded a AA- credit rating from Standard & Poors. King's is a member of the Russell Group, a coalition of the UK's major research-based universities.

Roslin Institute
The Roslin Institute has over 340 staff, students and visiting scientists who work in a wide range of disciplines including molecular and cell biology, quantitative genetics, endocrinology, developmental biology, animal behaviour and nutrition. It also has programmes for the derivation of new human embryo stem cell lines and to control their differentiation into specific lineages. The Institute has excellent facilities.

The Institute collaborates with academic and commercial partners worldwide, and plays an important role in post-graduate education, mostly through the University of Edinburgh's Graduate School of Life Sciences.

Further information
Science Media Centre
smc@rigb.org
Tel: 020 7670 2980

King's College Public Relations Department
Email: pr@kcl.ac.uk
Tel: 020 7848 3202

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