Category Archives: Gene therapy

SAN DIEGO-- On day two of TEDMED , running between Oct. 27 and 30, three themes stood out: the difference between children and adults for therapies; the connection between animals, people and disease; and how genetics will shape health care.

Frances Jensen of Harvard University and Children’s Hospital Boston explained the dramatic differences between developing and adult brains. With faster synapses, teens learn faster than adults, for instance. But as a consequence, they also "get addicted faster, longer and stronger than adults do," she said. Because teens have more synaptic material to affect, they suffer greater brain damage from alcohol than in adults. Differences in developing brain mean should have "no more hand-me-down drugs" for youths, added Jensen.

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The psychologist Rollo May once described depression as “the inability to construct a future”. [More]

Despite the enormous efforts of clinicians and researchers, our limited insight into psychiatric disease (the worldwide-leading cause of years of life lost to death or disability) hinders the search for cures and contributes to stigmatization. Clearly, we need new answers in psychiatry. But as philosopher of science Karl Popper might have said, before we can find the answers, we need the power to ask new questions. In other words, we need new technology. [More]

Every day as a practicing psychiatrist, I confront my field’s limitations. Despite the noble efforts of clinicians and researchers, our limited insight into the roots of psychiatric disease hinders the search for cures and contributes to the stigmatization of this enormous problem, the leading cause worldwide of years lost to death or disability. Clearly, we need new answers in psychiatry. But as philosopher of science Karl Popper might have said, before we can find the answers, we need the power to ask new questions. In other words, we need new technology.

Developing appropriate techniques is difficult, however, because the mammalian brain is beyond compare in its complexity. It is an intricate system in which tens of billions of intertwined neurons--with multitudinous distinct characteristics and wiring patterns--exchange precisely timed, millisecond-scale electrical signals and a rich diversity of biochemical messengers. Because of that complexity, neuroscientists lack a deep grasp of what the brain is really doing--of how specific activity patterns within specific brain cells ultimately give rise to thoughts, memories, sensations and feelings. By extension, we also do not know how the brain’s physical failures produce distinct psychiatric disorders such as depression or schizophrenia. The ruling paradigm of psychiatric disorders--casting them in terms of chemical imbalances and altered levels of neurotransmitters--does not do justice to the brain’s high-speed electrical neural circuitry. Psychiatric treatments are thus essentially serendipitous: helpful for many but rarely illuminating.

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A decade ago biologists and nonbiologists alike gushed with optimism about the medical promise of the $3-billion Human Genome Project. In announcing the first rough draft of the human “book of life” at a White House ceremony in the summer of 2000, President Bill Clinton predicted that the genome project would “revolutionize the diagnosis, prevention and treatment of most, if not all, human diseases.”

A year earlier Francis S. Collins, then head of the National Human Genome Research Institute and perhaps the project’s most tireless enthusiast, painted a grand vision of the “personalized medicine” likely to emerge from the project by the year 2010: genetic tests indicating a person’s risk for heart disease, cancer and other common maladies would be available, soon to be followed by preventives and therapies tailored to the individual.

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When Jim Laidler’s oldest son, Benjamin, was diagnosed with autism, he and his wife started looking for help. “The neurologists were saying, ‘We don’t know what causes autism, and we don’t know what the outcome for your son will be,’” Laidler relates. “No one was saying, ‘Here’s what causes it; here’s what treats it.’”

But when the Laidlers, who live in Portland, Ore., searched the Web, they found dozens of “biomedical” treatments that promised to improve or even cure Benjamin’s inability to talk, interact socially or control his movements. So the parents tried them on their son. They began with vitamin B6 and magnesium, the nutritional supplements dimethylglycine and tri­methylglycine, vitamin A, gluten- and casein-free diets, the digestive hormone secretin, and chelation, a drug therapy de­signed to purge the body of lead and mercury. They applied the purported treatments to Benjamin’s little brother, David, who also was diagnosed with autism. Chelation did not seem to help much. Any effect from secretin was hard to tell. The diets showed promise; the Laidlers hauled special food with them everywhere. And Mom and Dad continued to feed the boys dozens of supplements, calibrating doses up and down with every change in behavior.

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