Drug Development Program Paves the Way for FDA Approval of New Agents   
Pediatric New Agents Working Group Advances the Study of Novel Treatments in Young Patients   
DiaLog: Patients and Physicians: Partners in Health Care, by Shellie M. Scott, BS, Physician Assistant, Department of Urology   
House Call: Taking a More Active Role in Your Own Health Care   
As Population of Cancer Survivors Grows, Studies of Long-Term Health Effects Become More Critical, Researchers Sayhttps://openworks.mdanderson.org/oncolog/1107/thumbnail.jp

Chalaire, Dawn

O\u27Suilleabhain, Kate

Scott, Shellie M., BS

English

Drug Development Program Paves the Way for FDA Approval of New Agents Pediatric New Agents Working Group Advances the Study of Novel Treatments in Young Patients DiaLog: Patients and Physicians: Partners in Health Care, by Shellie M. Scott, BS, Physician Assistant, Department of Urology House Call: Taking a More Active Role in Your Own Health Care As Population of Cancer Survivors Grows, Studies of Long-Term Health Effects Become More Critical, Researchers Sayhttps://openworks.mdanderson.org/oncolog/1107/thumbnail.jp

OpenWorks @ MD Anderson

University of Texas MD Anderson Cancer Center OpenWorks @ MD Anderson OncoLog MD Anderson's Report to Physicians (All issues) OncoLog MD Anderson's Report to Physicians 6-2002 OncoLog Volume 47, Number 06, June 2002 Kate O'Suilleabhain Dawn Chalaire Shellie M. Scott BS Follow this and additional works at: https://openworks.mdanderson.org/oncolog  Part of the History of Science, Technology, and Medicine Commons, and the Oncology Commons                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                          Pediatric New Agents Working Group Advances the Study of Novel Treatments in Young Patients by Kate 6 Sullleabhaln A diagnosis of cancer in a child is particularly tragic and often devastating to the family. However, hope does exist for young patients: children tend to respond better to chemotherapeutic agents than do adults, and many of the drugs currently under development appear to be more effective in pediatric cancers than in adult malignancies. Seeking to improve the outlook for young patients at The University of Texac; M. D. Anderson Cancer Center is the Pediatric New Agents Working Group. Established in 1995, the group develops new anticancer therapies for patients younger than 18 years, who usually have less access to clinical trials than adults do. One reason for this difference is that many of the cancers that occur in pediatric patients are rare and biologically distinct from adult cancers. Thus, recruiting young patients in numbers sufficient for a trial can be challenging. "It depends on our referral base and the type of cancer," said John Kuttesch, M.D., Ph.D., the group's director and an associate professor in the Division of Pediatrics. "Patients are referred by outside oncologists, through inquiries of families and patients, and through information available on the [Division of] Pediatrics Web site or in publications such as OncoLog," he said. Another reason children may have less opportunity for inclusion in clinical trials is that researchers have traditionally tested drugs in adults before using them in children. However, the results of testing a drug exclusively in adults may be mislead­ing. "One problem with evaluating new drugs only in adults is that lack of efficacy might lead to abandonment of the drug," said Cynthia Herzog, M.D., a member of the group and an associate professor in the Division of Pediatrics. "So even though it might be very effective in pediatric patients, you never get a chance to test it [in children]," she said. mliat m&J & ellanglng. �@il� to Dr. Kuttesch, the U.S. Food and Drug Administration recently implemented a 4 I OncoLog policy designed to encourage the development of new agents for use in children and adolescents. "Whoever submits a plan to test a new agent in pediatric patients gains six months to one year of marketing and manufactur­ing exclusivity. This means the drug owner has exclusive control of the agent for up to a year to evaluate that drug in children," he said. While the Pharmaceutical Develop­ment Center at M. D. Anderson facilitates both preclinical and clinical testing of new agents, the Pediatric New Agents Working Group focuses on clinical development. (An exception is a study of liposomal interleukin-12 in a mouse model that is being led by Laura Worth, M.D., Ph.D., an assistant professor in the Division of Pediatrics, and Eugenie Kleinerman, M.D., profes­sor and chair of the Division of Pediat­rics. This preclinical study is being performed to obtain data sufficient to warrant study of this immune stimulant in pediatric patients.) Drug; currently under development target a diverse array of pediatric cancers, and most of the patients enrolled in the trials have recurrent malignancies or disease refractory to standard therapy. "Ideally, we select drugs for testing based on whether we think they will show some benefit in a particular (pediatric] cancer. Our Dr. Cynthia Herzog, an associate professor in theDivision of Pediatrics andmember of the Pediatric New Agents Working Group, examines -·selection of drugs for clinical trials is bac;ed on results of previous lab testing, in vitro results in cell cultures, and previous testing in adults," Dr. Kuttesch said. He is currently testing liposomal vincristine, a drug that was first studied in adults at M. D. Anderson, for the treatment of relapsed Ewing's sarcoma and other solid tumors in children. Another drug, clofarabine, is being tested in children with leukemia who are enrolled in a phase I study. Clinical trials involving pediatric patients also raise important ethical issues. Dr. Kuttesch relies on three criteria for including young patients in clinical trials. "First, there must be a rationale for testing a given drug in a certain type of malignancy," he said. "The drug must show a mechanism of action specific for that given tumor." Second, the pharmacologic data must show margins of safety for µitient protection. Third, because a child cannot provide consent, the parents or guardians must do so. "Surrogate [informed] consent requires comprehension by the family about what we know and don't know about a drug," said Dr. Kuttesch. Physicians and families interested in clinical trials of agents for patients with pediatric cancers at M. D. Anderson can contact the Pediatric New Agents Working Group at (713) 792-6620. • As Population of Cancer Survivors Grows, Studies of Long--Term Health Effects Become More Critical, Researchers Say by Dawn Chalaire s treatments im, prove, more and more patients are battling cancer­and winning. According to the National Cancer Institute, 8. 4 million Americans are living with a history of cancer. But what is cause for celebration is also a source of concern for forward, thinking researchers because very little is known about the overall health of these lang,term survivors. Most of the more serious long-term effects of cancer and cancer treatments are well documented: the surgical defects caused by the removal of a tumor, for ex;1.mple, or the late effects of radiation therapy to certain areas of the body. But what about less serious, chronic condi­tions that could be caused or aggravated by past cancer therapies? "There is no documentation of what people who were treated 10 years ago look like-virtually nothing about their medical health," said Rena Sellin, M.D., a professor in the Departmentof Endocrine Neoplasia and HormonalDisorders and director of the Life AfterCancer Care clinic at The University ofTexas M. D. Anderson Cancer Center.While a good deal of information exists about survivors of childhood cancers, studies of adult cancer survivors have tended to focus only on quality of life and social adjustment. The few studies of survivors of adult cancers that do address medical questions are of discrete groups o f  cancer survivors or types of disease rather than the population as a whole. According to Dr. Sellin, this paucity of information was the primary motiva-Dr. Rena Sellin (right), a professor in clte Deparcmeni of Endocrine Neoplasia and Hormonal Disorders and dir ector of ihe Life After Cancer Care clinic, and Marth Beck, an advanced practice nurse, review medical recards wich pacienc tion for a survey of adult cancer survivors she began in 2000, along with Pam Schultz, Ph.D., program director for the Department of Endocrine Neoplasia and Hormonal Disorders. "Usually, research involved in cancer means a better treatment, a new discovery, a better way of doing something to make the patient's out­come better, and we don't think beyond that," Dr. Schultz said. "I think this opens up the idea of a way of looking at patients differently. Once you have cancer, you may never have cancer again, but you must live with the consequences of having had it." The five-page survey was mailed to 9,512 cancer survivors identified in the M. D. Anderson database in  September 2000 as having completed cancer treatment at M. D. Anderson (Continued on page 6) OncoLog / 5 

OncoLog, Volume 47, Number 06, June 2002

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OncoLog Volume 47, Number 06, June 2002

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