‘Best Case’ Program Helps Agency Evaluate CAM Therapies

ARLINGTON, VA—To move into mainstream oncology, complementary and alternative medicine (CAM) must find convincing ways to prove efficacy in the treatment of cancer, said Jeffrey White, MD, director of the National Cancer Institute’s Office of Cancer Complementary and Alternative Medicine (OCCAM). This means moving beyond anecdotal evidence supporting CAM cancer therapies and finding appropriate standards for judging their effectiveness.

Through its Best Case Series program, OCCAM offers complementary and alternative medicine practitioners the opportunity to have successful cases reviewed by an expert panel of advisors to help develop the institute’s research priorities and allocate research support.

The Best Case program, Dr. White said at the Comprehensive Cancer Care 2000 conference, evaluates treatments used for patients with proven, quantifiable disease. Practitioners must document evidence of tumor reduction, although quality of life may be evaluated in selected cases.

Procedures call for submitting documentation for a minimum of four or five cases of remission, including a clinical history, the patient’s signs and symptoms, and a treatment history with any prior treatments listed. Pathology and medical imaging reports are required as well.

Once this material is reviewed, OCCAM will request actual pathologic and radiologic materials for further review by National Institutes of Health pathologists and radiologists.

If the diagnosis of cancer is confirmed and the case reports meet program standards, practitioners will be notified to appear before one of the twice-yearly meetings of the NCI’s Cancer Advisory Panel for Complementary and Alternative Medicine (CAPCAM). This panel will then decide if further research support is appropriate.

Under the Best Case program, NCI is now funding several CAM regimens: green tea compounds for precancerous lesions; shark cartilage for advanced cancers; stress and pain management for enhancing quality of life; a trial of a nutritional regimen for pancreatic cancer; and a homeopathic therapy for bronchiogenic carcinoma.

Gonzalez Diet

The pancreatic cancer trial by Nicholas J. Gonzalez, MD, a physician in private practice in New York, will test whether use of pancreatic enzymes from pigs, combined with dietary supplements, nutritional therapy, and coffee enemas, is effective in treating pancreatic cancer.

Dr. Gonzalez’ therapeutic ideas have a long history, dating back to anecdotal reports in medical journals in the early decades of the 20th century.

More significantly, he drew on the work of William Kelley, DDS, a dentist whom Dr. Gonzalez termed “eccentric and controversial” but who treated more than 10,000 patients by nutritional means and kept detailed records.

Review of Dr. Kelley’s Cases

Dr. Gonzalez evaluated 1,000 of Dr. Kelley’s cases of biopsy-proven disease that had poor prognosis or were considered terminal at the time of diagnosis. He narrowed those down to 50 cases in which Dr. Kelley’s therapy was the only treatment provided to the patient. He finished this retrospective study of Kelley’s patients in 1986 and began applying its principles to some of his own patients.

Dr. Gonzalez conducted a pilot study, supported by the Nestle Corporation, of 11 pancreatic cancer patients from 1994 to 1999. Patients on this regimen, he said, lived an average of 17.5 months, about three times the usual survival period for patients with advanced pancreatic cancer. Dr. Gonzalez presented this evidence to NCI, supported by a variety of medical and imaging tests.

Dr. Gonzalez designed a phase III trial to be conducted in cooperation with Columbia-Presbyterian Hospital, New York, and funded by the National Cancer Institute. The study was designed as a prospective, randomized, controlled trial of inoperable pancreatic cancer.

The control arm was to be standard treatment with gemcitabine (Gemzar). However, of 200 patients who inquired about the trial, 197 refused the 50% chance of randomization to the chemotherapy arm.

“Patients realized that the conventional therapy was a death sentence,” Dr. Gonzalez said, so the format was changed to a single-arm, nonrandomized, case-cohort study for the nutritional arm only. Cases will be compared with concurrently accrued patients on gemcitabine. Recruitment has begun for the 5-year, $1.4 million study.

“We need some flexibility on study design, especially in a disease where no one lives more than 19 months after diagnosis,” Dr. Gonzalez said. “A less aggressive cancer would call for a higher standard.”