Kampen - The golden cell: gene therapy, stem cells and the quest for the next great medical breakthrough

FOR MY PARENTS,
DR. MURRAY AND LINDA MOFFAT

I was on a steep learning curve during the research and writing of this book, which would not have been possible without the time, energy, and attention of many individuals. For their scientific expertise, I would like to thank Dr. Michael Kaplitt, Dr. Martin Kaplitt, Lap-Chee Tsui, Molly Shoichet, John Davies, Michael Rudnicki and his team (namely Pearl Campbell), Jane Aubin and her team (specifically Shulin Zhang), James Till, Dr. Ernest McCulloch, Dr. Ron Worton, Jeffrey Karp, Kathryn Moore, Michael Sefton, Dr. Thomas Okarma, Dr. Roderick McInnes, Dr. Irving Weissman, Michael West, Ananda Mohan Chakrabarty, Richard Rozmahel, Dominique Stoppa-Lyonnet, Dr. Edward Diethrich, Dr. Nabil Dib, and Dr. Andres Metspalu. For their invaluable perspectives on regenerative medicine, I am grateful to Timothy Caulfield, Bartha Maria Knoppers, Halla Thorsteinsdottir, Dr. John Dossetor, Francoise Baylis, and Alan Milstein. For their insights into the books subjects, I thank Brian Shoichet, Max Cooper, and Miller Quarles. For their personal experiences, I thank Paul Gelsinger and Nathan Klein. For his understanding of the business of biotechnology, I thank Bob Mark. For their navigation of resources, I thank the staff of the University of Torontos Gerstein Science Information Centre and Bora Laskin Law Library, the Toronto Reference Library, and the Princess Margaret Hospital library. For his deft pen and incisive input, I thank my editor, Jim Gifford. I am indebted to my agents, Don Sedgwick and Shaun Bradley, whose confidence and enthusiasm made this project possible. I thank my brother, Grant Moffat, for his legal expertise and long, inspiring chats. I am grateful to my parents, Dr. Murray and Linda Moffat, for their encouragement, inspiration, and Muskoka writers retreat. I thank my husband, Dimitri van Kampen, for his meticulous eye for detail and unwavering support.

I n June 2002, I was sent by the National Post to cover ChaRM, the Challenges in Regenerative Medicine conference. Dozens of scientists and physicians had taken over a ballroom in Torontos Fairmont Royal York Hotel with their exhibits. It was like a science fair for adults. Colourful sheets of cardboard displayed successful experiments. But rather than a grade-school how-to on coaxing a paper-mch volcano to erupt, I found detailed instructions for building three-dimensional cellular bridges to repair damaged spines, and a step-by-step guide to create chemical channels using lasers to control nerve regeneration.

There were diagrams explaining how to metamorphose embryonic stem cells into other types of cells. These stem cells are content to remain as they are born, as blank slates free of motivation or foresight. Embryonic stem cells are magical in that they can be coaxed into becoming virtually all of the bodys cell types, which could one day be used to strengthen weakened hearts or ailing Parkinsons and Alzheimers brains.

As I navigated through the maze of exhibits, examining diagrams of hearts, brains, and spines, I realized that the body was a different landscape than I had envisioned. I have always been mystified by its complexities. How do our hearts, organs the size of a fist, manage to beat 100,000 times a day, pumping 10,000 litres of blood through our systems? How do our lungs know to exchange carbon dioxide for oxygen? How do our brains process everything that happens around us, guiding how we feel, what we dream, and what we remember?

The body has always been a familiar as well as an uncharted landscape. It is as if I am flying over the city in which I live. I can pinpoint landmarks, but everything in between is unrecognizable. I see the body as an aerial map of 100 trillion cells, a map that becomes dizzying when I try to take it in all at once. But if I focus on one section of land, zooming in to one city block, then one street, and finally one house, the picture moves into focus. Then, rather than scanning 100 trillion cells, I am looking at one single cell.

Life, it seems, can be simplified to the intricacies of the cell. Our genetic blueprint, our physical sketch, even our biological destinies are hiding within them. Each cell houses variations of genes that determine our hair and eye colour, the diseases we are born with, and the conditions that we may one day develop.

Regenerative medicine uses what is hiding within all of uswithin our own cellsto fight the diseases that may some day threaten our very lives. It replaces faulty genes and corrects renegade proteins. It uses the stem cells floating within our blood, bone, and muscle to mend broken limbs and repair weakened organs. For years, scientists studied the inner workings of the cell, stalking genes and proteins to record their every move. They learned what happens biologically within each of us during a lifetime, and specifically when certain diseases strike. The Golden Cell shows scientists posing a fundamental question: What makes a cell tick? Then there are the questions that follow. What are the mechanisms that convince a cell to grow, divide, or change? How does a cancerous cell come into being, multiplying by the millions and wreaking havoc on the body? How does the environment convince genes to mutate?

Like most people, I have seen how a genetic disease can crumple a healthy person, how a serious injury or accident can drain the life out of someone. Imagine if broken spines could be mended, tired organs restored, and addled minds awakened. As Molly Shoichet, co-chair of ChaRM, explained, these are the promises of regenerative medicine. As you will read, the next frontier in medicine can release the regenerative powers of the cell, unlocking new life within us all.