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

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Kampen The golden cell: gene therapy, stem cells and the quest for the next great medical breakthrough
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The promise of a regenerative medicine -- the regrowth of lost limbs and organs, new hope for patients with Alzheimers or multiple sclerosis, the cellular fountain of youth--Sounds like science fiction, but its real and on the cutting edge of medicine. Veteran medical journalist Karen van Kamped looks at this new technology through the stories of the scientists who are creating it and the ethical and legal questions with which they must deal. Many companies and universities in Canada are at the forefront of cellular research, and this fascinating book shows that the next frontier of medicine is unfolding around us.

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FOR MY PARENTS DR MURRAY AND LINDA MOFFAT I was on a steep learning curve - photo 1

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.

Chapter One
Taxicabs and Roadblocks
Navigating Genes through the Highways of the Body

F or five excruciating hours he lay here, pinned to an operating table at New York Weill Cornell Medical Center. He willed every sensation, every impulse, to leave his body. The pins and needles crawling up his legs; the suppressed cough scratching his sandpa-pery throat. Everything he felt had to be ignored. Even the wild beating of his heart. For five hours, his mind had to forget his body. His thoughts had to shut down, while he had to remain awake. If he surrendered to sleep, the operation would also be surrendered. The months of hope and anticipation would have been for naught.

With the turn of a few screws he had been clamped in place, anchored by bits of steel. He had become as solid and immobile as the operating table. It was as if he had been poured into a mould and set in this fixed position. His concentration, though, was being taunted. There was the slow whoo-sh forward and quick s-nap back of the swinging door; the monotone be-eep, be-eep, be-eep of machines encircling him; and the clink of scissors and scalpels and other disinfected tools as they were lined up on a tray, ready to be used, gasp, on him. Just stare at the blank walls, or the blank ceiling, and disappear into this nothingness. The room was a clean slate, and suddenly, so was his life.

It was a muggy August day in 2003, but the operating room was impervious to weather or time. The scene, if caught on film, would reproduce as a blur of movement and colour, streaks of green scrubs staining a white background. It was a maze of bodies darting from one place to another, each following a prescribed course, all in their own world. Physicians, nurses, and technicians, gowned and gloved, popped in and out of view as he lay on his back, his spine kissing the cold stainless steel table. He too was concentrating on what he had to do. He could not be put under general anesthetic because this would lull his brain to sleep, making the operation impossible to perform. It was his job to stay awake for the entire surgery, and not move. No shifting. No twitching. He couldnt even think about coughing or yawning or giving a good stretch of those achingly sedentary legs. One sneeze rattling his body could prove fatal.

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