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Patient ordeal reminds all of research needs by Holly Auer Post and Courier For months, Malia Heck complained of belly aches, of pain and itchiness on the left side of her tiny body. All night long, she screamed in pain each time she moved. At only 2-1/2 years old, she didn’t yet have the words to describe her agony.   All the while, a tumor grew inside Malia’s spinal cord, burrowed between her shoulder blades. By the time it was diagnosed, after a four-month shuffle among different types of doctors, the cancer was shaped like a pod, long and cylindrical. The surgery she would need to excise the tumor carried huge risks: The toddler could be left paralyzed or lose the ability to control her bladder and bowels.   Letitia Bolds, a first-year medical student, prepares culture slides in the Developmental Neuro-Oncology lab in MUSC’s Children’s Research Instititute in an effort to develop techniques for defeating cancer cells that resist most forms of treatment—on Sept. 11. She sees the potiential that the biomedical research there “will turn into possible treatments for patients.” photo by Wade Spees/The Post and Courier But she came out of the operation with only minor mobility issues—still the same little girl, rushing to slip her feet into a new pair of high-heeled Cinderella slippers. Then, less than a year later, the tumor crept back. Another surgery, and this time a round of chemotherapy. Radiation wasn’t an option because it would likely kill off the healthy spinal cord nerves Malia would need as she grew.   Dan and Kara Heck, Malia’s parents, knew the treatments might not banish the cancer forever, but they believed they were buying time. A treatment, they hoped, existed somewhere out there, in test tubes and potions destined to some day become lifesavers.   “We were putting her on this path hoping medical research would catch up,” Dan Heck said.   So the family started searching. They called the American Cancer Society. The National Institutes of Health. The Spinal Cord Tumor Association. And nothing. No one, it seemed, was working on a real cure for Malia’s illness.   The Hecks live in Hebron, Ky., near Cincinnati, but in the four years since their daughter’s cancer diagnosis, they’ve cast their net wide in pursuit of spinal cord tumor research. The foundation they set up, Malia’s C.O.R.D. Foundation, has raised some $400,000 so far, and they’re now funding work in the Medical University of South Carolina’s Children’s Research Institute and at the Johns Hopkins University in Baltimore, where Malia had surgery.   Scientists, doctors and students at MUSC aim to develop a treatment using hyaluronan—a “bio goo” now widely used for eye surgeries, treatment of arthritis and cosmetic purposes such as filling in wrinkles—to attack the most resilient and resistant cells inside spinal cord tumors. It’s thought that the treatment, when applied in the cavity left behind after surgery to “debulk” the tumor, will turn off the mechanisms that make those cells so hardy in the face of toxic chemotherapy drugs. It’s an ambitious and expensive project. Carrying the science all the way to market—where it would be widely available to patients across the nation—will require years of funding for basic science work in the laboratory, plus money for clinical trials if the researchers get promising results.   During the 2005 fiscal year, MUSC researchers pulled in more than $180 million in grants, which fund most of the work conducted in the university’s labs—staff salaries, equipment, supplies and animals. Nearly half the money came from the federal government, through the National Institutes of Health. But in the wake of budget cuts to the agency, the university lost $2 million in federal grants compared to the previous year.   The news is even worse so far this year, and researchers across the nation are feeling the pinch. Private foundations are increasingly being called upon to fund research projects, and basic scientists devote huge swaths of their time to writing grant proposals in hopes of securing money to test their ideas.   Researchers in the developmental neuro-oncology lab where the spinal cord tumor work is under way, for instance, have submitted more than 10 grants in recent months, some more than 100 pages long, said Jennie Gilg, Ph.D., who manages the lab. To attack all aspects of their work, labs generally must piece together several grants from different sources, from small foundations such as Malia’s C.O.R.D. Foundation to big government agencies such as the Department of Defense.   Pediatric neuro-oncologist Bernard Maria, M.D.,  serves as executive director of the CRI and oversees the work of the neuro-oncology researchers. He spends part of his week caring for sick kids and brings that perspective back to researchers in the lab, who say that the human reality of the microscopic work they’re doing is a powerful motivator.   That’s because central nervous system tumors—those found in the brain and spinal cord—are the leading cause of death from illness in children. Unlike childhood leukemia, for instance, which once killed most of its victims but now boasts an inspiring cure rate, doctors haven’t cracked the code on how to do battle with many central nervous system tumors.   When kids today present with those cancers, Maria said, he can look forward on the calendar and be almost sure they will die by a certain point. It’s maddening, and heartbreaking.   “To look into the eyes of a 7-year-old and know that it’s all over, that’s hard,” Maria said. “And that means we haven’t done squat.”   So on the fourth floor of the CRI, which opened last year and houses some of the university’s most spacious and newfangled labs, there is work to be done. Researchers in basic science laboratories are forever talking about whether or not their experiments are “working” —that is, rolling along according to the experiment’s hypothesis. Will it “work” and someday lead to a treatment in real patients, or will it wind up a confounding tangle of contradictory results?   Sandra Tye, a graduate student in the neuro-oncology lab, had some good results in her rats—the treatment worked just as the researchers had hoped—right out of the gate this year. But there are many steps left to go, and she knows that no matter how skillfully she operates on each tiny animal or how hard she works to keep them safe from infection, the experiment might not go as planned.   An animal could die before the protocol ends or she could find some sort of new and vexing cancer cell distribution in its spinal cord. Or the cells could continue growing, out of control, even after being injected with the treatment. Ultimately, the work is an uncertain mix of skill, luck, and perhaps, sometimes, a little bit of magic.   “Sometimes, you get lucky and something works the first time,” Tye said. “Sometimes, you really have to fiddle with it.”   Malia is 6 years old now, and she’s in remission. She’s the “queen of the family,” and she adores reading and doing crafts. Still, her family feels called to this cause, for all the kids who haven’t been as fortunate. It will be slow going, they know, but there is much to celebrate in even the tiniest steps toward a cure. This article ran in the Sept. 13 issue of the Post and Courier and is reprinted with permission. Demystifying medical research with animal models The following information was collected from the Foundation for Biomedical Research to describe preclinical research in relation to the transition from the laboratory to widespread patient use for medications and therapies.     Recent scientific breakthroughs have led researchers closer to discovering and implementing new treatments and cures for an abundance of diseases affecting humans and animals. The process by which these treatments and cures are developed is referred to as preclinical research, or research that takes place prior to human trials for a given medication or treatment. Preclinical research can be conducted in vitro in an artificial environment like a Petri dish; or in vivo, which requires the use of an animal model.     The natural progression of most medical research follows the testing of a hypothesis first in an in vitro model, then an animal model, and culminating in human clinical trials before a treatment or cure may be distributed to a large patient population. This process takes years, sometimes decades, to complete before a safe and effective conclusion is reached. Animal preclinical research plays a vital role in that process, because without the knowledge gained from in vivo trials, many treatments or cures would take much longer to reach humans or never make it to human testing at all. Scientific progress relies on animal research in developing new treatments, because one cannot predict that a medication is safe or effective in cultured cells or in a plastic dish. Despite numerous efforts on behalf of scientists everywhere to reduce the number of higher species used in research, to replace animals with other models where possible, and to refine tests to ensure the most humane conditions possible, sometimes it can be difficult to reconcile human love and appreciation for animals and the essential need for animal research. Part of that reconciliation involves knowing that research animals are treated responsibly, ethically, and humanely as possible.   From a researcher’s standpoint, treating the animal models inhumanely would be counterproductive. Research could be halted, hampered or discontinued if animals are not properly cared for, because mistreated animals could negatively influence study results and impair the development of valuable human treatments. For humane and scientific reasons, MUSC researchers, like most researchers, are deeply concerned about the condition of the animals they study. Poor care results in unreliable research data. It is widely recognized that animals are an indispensable component of scientific research, and researchers recognize the moral and professional duty to provide them with the best care and treatment possible.   Animal models provide invaluable and irreplaceable insight into human systems due to the striking similarities between the genetic and physiological systems of animals and humans. Medical advances often are supplemented by non-animal methods, but these methods alone cannot tell researchers what they need to know about their work before sending it on for human testing. As of yet, no complete alternative to animal research exists, and not even the most sophisticated technology models can mimic the complex cellular interactions that occur in a living system.   Federal regulations governing the care and use of animals in biomedical research are extensive, regarding every aspect of their care and treatment from housing, feeding and cleanliness to ventilation and medical needs. The Animal Welfare Act requires the use of anesthesia or analgesic drugs for potentially painful procedures and during post-operative care, and research institutions are required to establish Institutional Animal Care and Use Committees like the one at MUSC. Researchers must explain and justify the use of animals in their research to the committee, select the most appropriate species and use the fewest number of animals possible. Researchers must also complete training modules before conducting the work.     By using both in vitro and animal models to determine how medications, treatments or cures will interact with the human body, researchers are able to deliver discoveries that catapult the human condition closer to a future free of the diseases and conditions that now plague family members, children and friends.     Friday, Oct. 6, 2006 Catalyst Online is published weekly, updated as needed and improved from time to time by the MUSC Office of Public Relations for the faculty, employees and students of the Medical University of South Carolina. Catalyst Online editor, Kim Draughn, can be reached at 792-4107 or by email, catalyst@musc.edu. Editorial copy can be submitted to Catalyst Online and to The Catalyst in print by fax, 792-6723, or by email to catalyst@musc.edu. To place an ad in The Catalyst hardcopy, call Island Publications at 849-1778, ext. 201.