September 2007
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Children's latest research
Immunology Novel drugs relieve swelling attacks in hereditary angioedema
Endocrinology How diet and exercise help us live longer
Stem Cell Program Stem cell finding explained on YouTube
Treatments emerge for a life-threatening disease
Patients with hereditary angioedema (HAE) never know when their next attack of swelling will come. Often starting in their teens or 20s, the swelling typically strikes the arms, legs, feet and hands. But there can also be disfiguring facial swelling, excruciating gastrointestinal swelling, or, worst of all, life-threatening throat swelling. Children’Äôs Hospital Boston has been part of two recent clinical trials that may finally provide an emergency treatment.Immunology’Äôs Lynda Schneider, MD, program director of Allergy, with colleagues at 25 other institutions, reports in the August Journal of Allergy and Clinical Immunology that 73 percent of patients given a single dose of the drug ecallantide (also known as DX-88) got significant relief from HAE attacks within four hours. Now, Schneider is enrolling children in a multicenter trial that is testing a second promising HAE drug called C1 inhibitor.
These trials cap decades of research into a rare, difficult and commonly misdiagnosed disease. ’ÄúThe pathways involved in HAE have only recently been understood,’Äù says Schneider.
Much of this understanding was provided by the late Fred Rosen, MD, former chief of Immunology at Children’Äôs. HAE begins with a gene defect that depletes or damages a regulatory protein called C1 inhibitor. The resulting biochemical imbalance leads to excess production of bradykinin, a protein that makes blood vessels leaky, causing tissues to swell. The two new drugs work in somewhat different ways: ecallantide blocks generation of the enzyme that causes release of bradykinin, while C1 inhibitor replaces the protein that’Äôs lacking in the body.
Pamela Johnson, now 31, suffers from HAE and would love to get either drug, since she’Äôs been able to access them only through studies at Children’Äôs. Her hands once swelled so badly that she had to type a final college exam with her thumbs, and, out of desperation, her father took to sleeping on the floor next to her bed to monitor her breathing. She ultimately resorted to chronic androgen therapy, despite its masculinizing side effects. ’ÄúI was too sick to continue going from trial to trial,’Äù she explains.
Reducing insulin in the brain may increase lifespan
You hear it all the time: diet and exercise help you live longer. But why? The answer is in your brain and insulin’Äôs action in it, say Children’Äôs Hospital Boston endocrinology researchers Morris White, PhD, associate scientist in Medicine, Akiko Taguchi, PhD, and Lynn Wartschow.Insulin is vital to metabolism’Äîa deficiency can lead to diabetes. But White suspected that too much insulin may have effects in the brain that promote aging. His team created mice whose brains were partially shielded from insulin’Äôs effects, and found they lived 18 percent longer than normal mice. Further work, reported in the July 20 issue of Science, suggested that this longevity owed to higher levels of superoxide dismutase, an enzyme that protects cell damage linked to diseases of aging’Äîsuch as cancer, atherosclerosis and Alzheimer’Äôs disease.
As we age, obesity and sedentary lifestyles tune down our sensitivity to insulin, forcing the body to make more. Diet and exercise tune this sensitivity back up, says White, allowing the body to run on less insulin’Äîand protecting our brains from too much exposure.
Stem cell researchers clarify Korean discovery
In 2004, Korean researchers got worldwide acclaim for creating the world’Äôs first human embryonic stem cells made through cloning. In 2006, their data were found to be falsified, and their glory unraveled. But last month, George Daley, MD, PhD, associate director of Children’Äôs Stem Cell Program, and Kitai Kim, PhD, also of the Stem Cell Program, set the record straight, showing that the Koreans created something else that’Äôs been hotly pursued by scientists¬‚’Äîthe world’Äôs first human embryonic stem cells derived from a woman’Äôs egg alone. As Daley explains on YouTube (www.youtube.com/watch?v=oVdV5-3Dfag), the Korean cells had a distinct genetic signature reflecting their unique biological origins. Daley’Äôs lab is keenly interested in egg-derived stem cells because they’Äôre relatively easy to make and offer a practical way of creating cells to treat disease that are compatible with a patient’Äôs own immune system.
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