Promise – Summer 2004

Non-Profit Org. U.S. Postage PAID Permit No. 1112 Memphis, TN CHANGE SERVICE REQUESTED PromiseSummer 2004 332 N. Lauderdale St. Memphis, Tennessee 38105-2794 April Johnson Public Information: 1-866-2STJUDE, ext. 3306 Donations: 1-800-822-6344 One Patient, Visit our Web site at www.stjude.org. St. Jude Children’s Research Hospital, Three Lives American Lebanese Syrian Associated Charities and ALSAC are registered trademarks. Page 12 Promise is a quarterly publication of the St. Jude Children’s Research Hospital was founded by the late Department of Public Relations St. Jude Children’s Research Hospital 332 N. Lauderdale St. PromiseA publication of St. Jude Children’s Research Hospital Summer 2004 entertainer Danny Thomas. It opened February 4, 1962. The institution was created because Memphis, Tennessee 38105 St. Jude Children’s Research Hospital’s of a promise Danny made during the depression era to St. Jude Thaddeus, the patron saint mission is to find cures for children with Features catastrophic diseases through research and treatment. 4 Command Performance of the hopeless. The show must go on Hospital Director Arthur W. Nienhuis, MD 7 Service over Self ALSAC National Executive Director A continuing commitment “Show me my way in life,” Danny prayed. In return, Danny promised to build St. Jude Richard C. Shadyac ALSAC/St. Jude 8 Spa Kids Senior Vice President of Thaddeus a shrine. That shrine became a world-class research institution that treats Massage and humor Communications and Public Relations children regardless of race, color, creed or their ability to pay. This remarkable event also Jerry Chipman 12 One Patient, Three Lives Director of Public Relations April Johnson’s Faith and Miracle inspired the name of this magazine, Judith W. Black ALSAC Vice President of Communications 16 A Peek Under the Hood George Shadroui Scientists study molecular motors Promise. Publications Manager and Editor Elizabeth Jane Walker 18 On the Road to Peoria Art Director Running for a reason Jessica W. Anderson Photo Editor Jere Parobek 21 Parkinson’s Progress Research for both kids and adults Photographers Seth Dixon Karen Pulfer Focht Laura Hajar Ann-Margaret Hedges John Zacher Contributing Writers Highlights Tanuja Coletta Joe Hanna 2 Achievements and Events Laura Hajar Alicia H. Matthews Victoria Tilney McDonough Perspective Carrie L. Strehlau Guest Author 24 Amber Valletta Amber Valletta A Call to Action Editorial Advisory Board Lisa Baker Bonnie Cameron Leslie Davidson Pat Flynn, MD Mark Hendricks Marc Kusinitz, PhD Phil McCarty St. Jude Children’s Research Hospital is an equal-opportunity employer. For inquiries about stories Carlos Rodriguez-Galindo, MD in this publication, call the Public Relations department at (901) 495-2125 or e-mail Penny Tramontozzi [email protected]. Visit our Web site at www.stjude.org. Articles and photos may be St. Jude Children’s Research Hospital, Memphis, Tennessee David Tucker reprinted with permission. ©2004. Sally Wiard John Zacher On the cover: April Johnson with daughters Faith (on left) and Miracle (see article, page 12). Photo by Laura Hajar. Order from disorder A fresh look permit scientists either to restore complex St. Jude investigators have demon- St. Jude scientists have discovered that protein structures that are missing in cer- strated for the first time that—contrary a tumor-suppressor protein called Rb is tain cells or make multiple proteins that Highlights to the assumption that proteins must required for proper retinal development in act together as potent drugs against can- maintain a rigid structure in order to laboratory models. This is a major step cer and other diseases. perform an assigned task—many toward understanding why some children Dario Vignali, PhD, of St. Jude proteins exploit disorderliness in their develop the devastating eye cancer called Immunology is senior author of a report A missing link structure to perform various jobs. The retinoblastoma. The discovery should on this work, which appears in the May Researchers at St. Jude and Mayo research findings appeared in the eventually help scientists design a better 2004 issue of Nature Biotechnology. Clinic have discovered that inactivation of April 2004 Nature Structural and treatment for this disease. the CBP gene in certain immature white Molecular Biology. The St. Jude team showed that Rb blood cells causes lymphoma in lab- The St. Jude finding explains how limits growth of immature retinal cells Rising rates oratory models. The cancer is accompa- many proteins can adapt their structures so the retina develops to a normal size. The cure rate for pediatric acute nied by changes in the expression of spe- to the needs of the moment, binding to The Rb protein also prompts specific lymphoblastic leukemia (ALL) might cific genes associated with development different molecules depending on the job cells to develop into light-sensitive cells continue to rise with improved use of of the disease. at hand. called rods. conventional therapies. But even more “One finding that was particularly sur- “The potential importance of disorder “What we’re learning could eventually effective and less toxic therapies based prising was the specific effect the loss of in the function of some proteins has been help us to block the molecular signals on genetic and pharmacogenetic studies CBP had,” said article co-author Paul discussed by researchers for several that trigger retinoblastoma in children,” may one day push the success rate close Brindle, PhD, of St. Jude Biochemistry. years,” said Richard Kriwacki, PhD, of said Michael Dyer, PhD, of St. Jude to 100 percent, according to an article “It is commonly believed that CBP is St. Jude Structural Biology, the report’s Neurobiology, senior author of a published by Ching-Hon Pui, MD, involved in the control of many genes. senior author. “But until now no one February 2004 Nature Genetics article Leukemia/ Lymphoma division Yet the absence of CBP appears to pro- had actually demonstrated how such about this research. director; Mary Relling, PharmD, mote lymphoma by cooperating with a flexibility allows a protein to interact Pharmaceutical Sciences chair; and narrow set of dysregulated genes.” with different molecules. We’ve taken a James Downing, MD, Pathology chair. Another unexpected finding was the big step in understanding the subtle Tricks of the trade The article appeared in the April lack of association between the loss of details of a critical biochemical process A genetic trick that viruses use to 2004 edition of the New England CBP function and a gene called p53. CBP St. Jude patient David Moore and Debbie Crom, RN, PhD, of the After Completion of Therapy in the life of the cell.” replicate themselves has been adapted for Journal of Medicine. is known to help activate p53, but the loss (ACT) Clinic, discuss the importance of promoting strong bones after cancer therapy. Their dis- To view an online movie illustrating the laboratory to build complex protein The researchers based their pre- of CBP in white blood cells triggered can- cussion occurred at the Eighth Annual St. Jude Cancer Survivor’s Day conference, which also the movement of a protein, visit structures required by immune system diction on a review of leukemic cell cer even though p53 activity appeared marked the 20th anniversary of the ACT Clinic. Although Crom dressed as a skeleton to make www.stjuderesearch.org/data/ cells, according to St. Jude investigators. genetic abnormalities and host normal learning about bones fun, she experienced many poignant moments during the conference. “It normal. The findings were published in is very moving to have survivors say, ‘Do you remember me?’ and to be able to respond, ‘We not kriwackilab/p27.html. The approach could also be used to cell genetic characteristics. The genetic Cancer Cell, February 2004. only remember you; we hold you in our hearts and we are dedicated to you having as healthy a develop new gene approach to ALL treatment increases future as possible,” Crom says. therapy vectors in physicians’ ability to identify which cases when cells gene mutations are linked to responsive- The wizardry of Ozz must be modified to ness to anti-leukemic drugs. Clinical Research conducted by St. Jude scien- could alter the normal expression of Ozz tion and analyzed. The findings con- make high levels of trials are underway to test the safety and tists may help uncover the genetic cause and could cause the muscle problems of firmed that samples shipped from different proteins. A efficacy of drugs targeting a variety of of certain muscle diseases that occur for children with galactosialidosis. institutions in the United States and vector is a DNA gene mutations. unknown reasons in children. d’Azzo, who holds the Jewelers Australia can be analyzed at St. Jude for molecule used to St. Jude researchers are also conduct- A team led by Alessandra d’Azzo, Charity Fund Endowed Chair in Genetics genetic abnormalities rapidly enough to ferry specific genes ing Phase I clinical trials with investiga- PhD, of St. Jude Genetics and Tumor and Gene Therapy, was senior author of a provide physicians with information into cells in order to tional drugs to treat cases of ALL that Cell Biology found that a novel protein, report on the findings, which appeared in to guide their treatment decisions. give those cells the have resisted previous therapies. Ozz, directs the destruction of a the February 2004 Developmental Cell. The study also demonstrated that by ability to make par- structural protein in muscle that helps detecting the presence of a protein ticular proteins. organize and stabilize muscle growth. called ERBB2 in tumor samples, The achievement The researchers also discovered the Brain cancer progress doctors might be able to predict which gives researchers a These news items reflect only a Ozz gene overlaps another gene, which Completion of a pilot study brings children with medulloblastoma will way to study the codes for an enzyme called protective researchers closer to an international clini- require more intensive treatment.

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