alternative and complementary therapies in childhood cancer

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EDITORIAL Alternative and Complementary Therapies in Childhood Cancer Ronald A. Anderson, MD Alternative and complementary therapies have greatly increased in popularity and usage in recent years, and many are discussed by J. Metz in this issue of Medical and Pediatric Oncology (pp. 20–26). Accompanying this is an increased interest in and implementation of these treatments by parents of children being treated for cancer [1]. Their impact and potential effectiveness, however, remain largely unstudied. As well, potential harmful ef- fects, whether from toxicity, interaction with ongoing therapy, or withdrawal of known effective treatments, are emerging as significant concerns. By contrast, the practice of evidence-based medicine is founded on the conviction that therapy must be based on persuasive data. Evidence for effectiveness is obtained by formulating a hypothesis that is subsequently tested. It has been through this approach that most children with cancer can now be cured [2]. Dramatic progress has oc- curred through individual institutional research and in- ternational collaborative efforts using hypothesis-driven, clinical, and laboratory-based investigations. The large multiinstitutional collaborative groups, consisting of the Pediatric Oncology Group, the Children’s Cancer Group, the National Wilms Tumor Study Group, the Interna- tional Rhabdomyosarcoma Study Group, and the Inter- national Society of Paediatric Oncology, provide the backbone for continued progress in an international, mul- tidisciplinary setting. Rigorously audited clinical practice and data analyses continue to be employed. Both funding of clinical trials and publication of results have been through a strict peer-review process. Society should be made more aware of the very dramatic progress that has occurred over the last 3 decades and the remarkable col- laborative efforts that have been responsible. It is in light of such concrete progress that the rapidly increasing and largely unquestioned usage of unproved alternative therapies is of such concern. Refusal of standard anticancer therapy and use of al- ternative therapy may be emerging as a new phenom- enon. We recently reported [3] an experience with two families who refused standard therapy for which cure rates of greater than 50% were expected. One of these children subsequently died; the other developed progres- sive disease and only then accepted therapy. It is with great frustration that pediatric oncologists face such situations. However, the dramatic promises made by certain alternative medicine practitioners can be very appealing even if the success of their therapies lacks any evidence. This was exemplified recently by the in- vestigation into the validity of the claims made by Dr. Luigi Di Bella of Modena, Italy. He was touted by many in the past year to be a hero who had a cancer cure that physicians and government were refusing to recognize. The government cancer and pharmaceutical commissions of Italy had refused permission to have the treatment recognized on several occasions. Finally, under public pressure, his treatment of somatostatin and vitamin A derivatives was studied in 385 patients and found to be a failure [4]. In the meantime though, this treatment had been promoted in the world media as a possible break- through in cancer treatment that was not being made available to all in need. Alternative therapies that show potential in prelimi- nary work deserve study. The investigation and applica- tion of these treatments must progress with the same rigorous scientific scrutiny that has led to the current cure rates in childhood cancer. Scientific investigation of al- ternative therapies is proceeding under the guidance of the U.S. National Institute of Health (NIH). Whether this will yield useful treatments remains to be seen. Under congressional mandate in 1992, an office of alternative medicine was established within the NIH. In 1993, a request for application was issued to allow for grants of up to $30,000 each. Unfortunately, the outcome of this initiative has been disappointing. From 30 research grants awarded, only 9 published papers have resulted, none of which reported a controlled clinical trial [5,6]. In October, 1996, the U.S. Congress established a National Center for Complementary and Alternative Medicine (NCCAM), with an appropriation of $50 million to fund its establishment. The goal is to provide a mechanism for objective scientific investigation of the potential uses of alternative therapies. Parents cannot be denied the option of using alterna- tive therapies when cure cannot be guaranteed or is no longer possible. Mothers and fathers only wish to do everything possible to help their children. However, in light of the progress that has been achieved in pediatric oncology over the past 40 years it is deplorable to see Medical and Pediatric Oncology 34:27–28 (2000) © 2000 Wiley-Liss, Inc.

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Page 1: Alternative and complementary therapies in childhood cancer

EDITORIAL

Alternative and Complementary Therapies in Childhood Cancer

Ronald A. Anderson, MD

Alternative and complementary therapies have greatlyincreased in popularity and usage in recent years, andmany are discussed by J. Metz in this issue ofMedicaland Pediatric Oncology(pp. 20–26). Accompanying thisis an increased interest in and implementation of thesetreatments by parents of children being treated for cancer[1]. Their impact and potential effectiveness, however,remain largely unstudied. As well, potential harmful ef-fects, whether from toxicity, interaction with ongoingtherapy, or withdrawal of known effective treatments, areemerging as significant concerns.

By contrast, the practice of evidence-based medicineis founded on the conviction that therapy must be basedon persuasive data. Evidence for effectiveness is obtainedby formulating a hypothesis that is subsequently tested. Ithas been through this approach that most children withcancer can now be cured [2]. Dramatic progress has oc-curred through individual institutional research and in-ternational collaborative efforts using hypothesis-driven,clinical, and laboratory-based investigations. The largemultiinstitutional collaborative groups, consisting of thePediatric Oncology Group, the Children’s Cancer Group,the National Wilms Tumor Study Group, the Interna-tional Rhabdomyosarcoma Study Group, and the Inter-national Society of Paediatric Oncology, provide thebackbone for continued progress in an international, mul-tidisciplinary setting. Rigorously audited clinical practiceand data analyses continue to be employed. Both fundingof clinical trials and publication of results have beenthrough a strict peer-review process. Society should bemade more aware of the very dramatic progress that hasoccurred over the last 3 decades and the remarkable col-laborative efforts that have been responsible. It is in lightof such concrete progress that the rapidly increasing andlargely unquestioned usage of unproved alternativetherapies is of such concern.

Refusal of standard anticancer therapy and use of al-ternative therapy may be emerging as a new phenom-enon. We recently reported [3] an experience with twofamilies who refused standard therapy for which curerates of greater than 50% were expected. One of thesechildren subsequently died; the other developed progres-sive disease and only then accepted therapy.

It is with great frustration that pediatric oncologists

face such situations. However, the dramatic promisesmade by certain alternative medicine practitioners can bevery appealing even if the success of their therapies lacksany evidence. This was exemplified recently by the in-vestigation into the validity of the claims made by Dr.Luigi Di Bella of Modena, Italy. He was touted by manyin the past year to be a hero who had a cancer cure thatphysicians and government were refusing to recognize.The government cancer and pharmaceutical commissionsof Italy had refused permission to have the treatmentrecognized on several occasions. Finally, under publicpressure, his treatment of somatostatin and vitamin Aderivatives was studied in 385 patients and found to be afailure [4]. In the meantime though, this treatment hadbeen promoted in the world media as a possible break-through in cancer treatment that was not being madeavailable to all in need.

Alternative therapies that show potential in prelimi-nary work deserve study. The investigation and applica-tion of these treatments must progress with the samerigorous scientific scrutiny that has led to the current curerates in childhood cancer. Scientific investigation of al-ternative therapies is proceeding under the guidance ofthe U.S. National Institute of Health (NIH). Whether thiswill yield useful treatments remains to be seen. Undercongressional mandate in 1992, an office of alternativemedicine was established within the NIH. In 1993, arequest for application was issued to allow for grants ofup to $30,000 each. Unfortunately, the outcome of thisinitiative has been disappointing. From 30 researchgrants awarded, only 9 published papers have resulted,none of which reported a controlled clinical trial [5,6]. InOctober, 1996, the U.S. Congress established a NationalCenter for Complementary and Alternative Medicine(NCCAM), with an appropriation of $50 million to fundits establishment. The goal is to provide a mechanism forobjective scientific investigation of the potential uses ofalternative therapies.

Parents cannot be denied the option of using alterna-tive therapies when cure cannot be guaranteed or is nolonger possible. Mothers and fathers only wish to doeverything possible to help their children. However, inlight of the progress that has been achieved in pediatriconcology over the past 40 years it is deplorable to see

Medical and Pediatric Oncology 34:27–28 (2000)

© 2000 Wiley-Liss, Inc.

Page 2: Alternative and complementary therapies in childhood cancer

families or patients forfeit or decrease their chances forcure by pursuing phantoms at critical times in the courseof the illness.

The facts detailed by Dr. Metz are sobering. The lackof rationale and objective evidence of potential benefitare enough to give one pause. When the costs areweighed in addition, the arguments against the wantonuse of alternative therapies become compelling. Practic-ing oncologists will profit from reading the article by Dr.Metz.

Ronald A. Anderson,MD

Departments of Oncology and PediatricsFaculty of Medicine

University of CalgaryCalgary, Alberta, Canada

REFERENCES

1. Fernandez CV, Stutzer CA, MacWilliam L, and Fryer C. Alter-native and complementary therapy use in pediatric oncology pa-tients in British Columbia: prevalence and reasons for use andnonuse. J Clin Oncol 1996;16:1279–1286.

2. Murphy SB. The national impact of clinical cooperative grouptrials for pediatric cancer. Med Pediatr Oncol 1995;24:279–280.

3. Coppes MJ, Anderson RA, Egeler RM, Wolff JEA. Alternativetherapies for the treatment of childhood cancer. N Engl J Med1998;339:846–847.

4. Associated Press Report (Rome): Cancer-cure cocktail branded afailure. Calgary Herald. Nov. 14, 1998.

5. Angell M, Kassirer JP. Alternative medicine—the risks of un-tested and unregulated remedies. N Engl J Med 1998;339:839–841.

6. National Institute of Health, National Center for Complementaryand Alternative Medicine Web site. See: http://altmed.od.nih.gov/nccam/

28 Editorial