---

Human Fertilisation and Embryology Bill [HL]

My Lords, the noble Lord, Lord Winston, referred some time ago to the work that I did in the course of my professional career on muscular dystrophy. It is almost 60 years ago that I began to work on that topic in Newcastle-upon-Tyne. The most severe form of muscular dystrophy manifests itself only in young boys. It is due to an X-linked recessive gene, transmitted by female carriers which is manifest in half their sons. Indeed, half their daughters are themselves carriers of the gene. The late Professor Nattrass and I entitled the disease Duchenne muscular dystrophy because of the outstanding descriptions given in the 19th century of the disease by Duchenne of Boulogne. It causes difficulty in walking; boys begin to waddle as they walk, and by the time they are nine or 10 years of age, they are confined to a wheelchair because of widespread paralysis. In the old days, because of gross deformity, few of them survived beyond the age of 15 or 16. Now, as a result of much improved care, including ventilatory care, many of these boys live until their twenties and even later. Research has been vital in learning more about this disease and it is bringing with insight the prospect of potential treatment. In 1987, the gene responsible for this disease was discovered. As the noble Lord, Lord Winston, said, it was found to be a very large gene, with several different kinds of mutations within it. Broadly, the result of this abnormal gene is that it traduces in the muscle cells an absence of a protein called dystrophin, which is an important part of the membrane of the muscle fibre—it is rather like the skin of a sausage. Because of this, that membrane becomes inefficient and, as work in my department in Newcastle showed many years ago, it allows certain chemical substances to get in from the extra-cellular fluid which begin a process of digestion or breakdown of the muscle. This disease can now be diagnosed—and has been able to be diagnosed for many years—at birth, by means of a simple blood test. It is clear to all researchers that if you are going to produce a form of treatment that will be effective in controlling this disease, it has to be done very early in life—preferably in the young infant. There is a dystrophin deficient mouse, an X-linked recessive gene, which is a model of Duchenne muscular dystrophy. I will not go into detail, but in today’s Times, there is reference to yet another crucially important piece of research, which says: "““Embryonic stem cells have been used to cure mice with the equivalent of [Duchenne] muscular dystrophy … Scientists in the US have successfully coaxed mouse embryonic stem cells to develop into muscle tissue and then transplanted those cells into animals bred with the genetic mutation””—" similar to that which— "““causes Duchenne muscular dystrophy. When the cells were injected into the bloodstream of the mice they migrated to the muscles to replenish them with healthy tissue and improved their function.""The findings, from … the University of Texas Southwestern Medical Centre … ‘demonstrate the therapeutic potential of embryonic stem cells in muscular dystrophy’””." Who knows that this is not something that will emerge in human Duchenne dystrophy within the next few years? In the light of discussions that we have already had on this Bill, the prospect is that, with the appropriate consent as given by the parents and following the guidelines set out in this amendment, it should be possible to take a skin cell from an infant diagnosed at birth as suffering from Duchenne dystrophy to create a stem cell line from that skin cell, using the kind of admixed human embryo to produce a treatment which should be effective in controlling—and ultimately, we hope, in curing—that disease. This is a most exciting development that underlines the need to be able, with all the appropriate safeguards in the amendment, to pursue this kind of research involving children.