Genetic therapy bears fruit for male diseases

I recently had the pleasure of hearing futurist Raymond Kurzweil speak at an SFU Public Square event in Vancouver. I have a big soft spot for him, ever since he invented the Kurzweil Reading Machine in the 1970s. It was a boon to the blind and people with poor eyesight, and opened the door for many other technologies.

One of the first machines was installed at the New York Public Library, and I was sent to interview its keeper. What do people bring to read on it? I asked. Mostly pornography, he replied. I thought I heard him wrong until he explained that there were readers for the blind who could tackle The History of the American Civil War,soamachine was being used for racier content. I use this story in my book Technocreep, to illustrate the unintended consequences of technology.

Now, Kurzweil is at it again, suggesting that if we make it through the next 10 or 15 years, some of us, at least, may become immortal. He predicts that, by then, we will be adding more than a year to the human lifespan each year, and not just for babies. He likens our bodies to computer software that has become out of date and needs upgrading. He foresees a day when we can reprogram our bodies to recognize and repel new pathogens, and even fix our existing ailments.

Kurzweil gives the example of the fat insulin receptor gene, which evolved in the distant past to help us store calories in case of famine. Im pretty sure the hunting season will be good next year at the supermarket, Kurzweil told the audience, and Im packing on a few pounds, so Id like to turn that gene off. He says that scientists at the Justin Diabetes Center have done just that, producing mice that eat to their hearts content and dont get fat.

Now, were starting to see the first fruits of genetic manipulation in a male-oriented condition.

Hemophilia, a disorder of blood clotting, affects men more than women because it is caused by a defect on the X chromosome. Since males are only dealt one of those in the genetic lottery, while females get two, our chance of getting the disease is much higher. There are two major varieties, each caused by lack of a key protein that helps blood to clot. Hemophilia A, where Factor VIII is impaired, occurs in about 1 in 5,000 male births. The more severe hemophilia B, involving Factor IX, shows up in about 1 in 30,000 male births.

Researchers at St. Jude Childrens Research Hospital, University College London recently announced success using gene therapy to treat hemophilia B in a number of men. In a press release, they report that years after receiving a single DNA treatment, the men continue to produce their own clotting factor with minimal side effects. Dr. Andrew Davidoff, chair of the St. Jude Department of Surgery adds that this study provides the first clear demonstration of the long-term safety and efficacy of gene therapy.

Gene therapy may also save health-care dollars. According to the researchers, overall spending on Factor IX replacement therapy for study participants is down more than $2.5 million (US). These scientists are now seeking to extend their work to hemophilia A and inborn errors of metabolism like phenylketonuria.

Experts believe the pace of gene therapy will increase, as genetic links to many conditions are discovered. In a new study from the University of Leeds, researchers found evidence of a gene mutation linked to autistic traits in mice. Lead researcher Stephen Clapcote said this is important because we are starting to build up a picture of the important role of genes involved in these synapse communications in better understanding autism.

Its good to know that, if Kurzweil is right about much longer lifespans, those added years may actually be great ones, free of diseases like hemophilia and perhaps even obesity.

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Genetic therapy bears fruit for male diseases