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doi: 10.1097/01.COT.0000289222.08140.7b
Clinical Notes
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Orphan Drug Status for 131I-TM-601 for Glioma

The FDA has granted orphan drug status to the investigational new drug (IND) 131I-TM-601, made by TransMolecular, Inc. of Birmingham, AL, to treat glioma.

The drug is a radiopharmaceutical containing a synthetic version of a substance derived from chlorotoxin scorpions. The FDA approved the company's IND application to begin a Phase I/II clinical study, and the company will be starting a multicenter trial to test the safety and tolerability of a single dose of 131I-TM-601 in 18 patients, as well as overall response.

In pre-clinical studies, researchers from the company determined that the drug was able to extend survival in a mouse model that mimicked human brain tumors.

131I-TM-601 is based on chlorotoxin sequences that have evolved to precisely locate and bind to their receptor. The chlorotoxin sequences deliver 131I, and no toxicities have been observed in preclinical animal studies.

Orphan drug designation is designed to encourage research and testing for products treating a disease affecting fewer than 200,000 persons in the US.

The potential benefits include seven years of market exclusivity upon marketing approval, tax credits for related clinical research expenses, the availability of grant assistance, and clinical development assistance from the FDA.

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Better Detection

Two studies presented at the most recent annual meeting of the American Roentgen Ray Society suggest that new and better means of detecting ovarian cancer spread and breast cancer are on the horizon.

The first study, authored by Harpreet Pannu, MD, Assistant Professor of Radiology, and Elliott Fishman, MD, Professor of Radiology at Johns Hopkins University, suggested that multidetector computed tomography scans can detect ovarian cancer that has metastasized. It included patients previously diagnosed with the disease who underwent a multidetector CT scan of the abdomen and pelvis in which image slices as thin as 2.5 mm were reconstructed.

“These small slices appear to be a real advantage: We were able to see cancers that had spread that were as small as a few millimeters,” said Dr. Pannu. “In addition to the fact that we can see small lesions, the scan time has been dramatically reduced.”

A scan of the whole abdomen and pelvis takes about 30 to 35 seconds. She said that the scan may be used before surgery to guide the surgeon in removing the malignancy and afterwards to determine if there is any tumor left.

The second study was on Breast Cancer Radar (BCR) technology, a promising new test that uses radio wave signals, similar to those in cell phones and radar, to detect breast cancers that may be missed on mammography.

“Malignant tumors have a greater content of water than surrounding breast tissue,” said Allan Malmed, MD, Medical Director of Northwest Community Hospital Interdisciplinary Breast Center in Arlington Heights, IL.

“BCR technology is able to take advantage of this fact and create 3-D images that show the size and location of cancers.” He added that since the test is based on water content, breast density appears to have no effect on the image quality.

The procedure is a simple test that takes about five minutes. Thus far, it has been used to image 22 patients with known cancers or cysts, all of which were accurately depicted, according to Dr. Malmed. “Early indications are that this test could be an adjunct to mammography. It may benefit patients with dense breasts as well as others who have negative mammograms and yet, for some reason, such as a family history of breast cancer, we are concerned that additional tests are needed.”

Clinical trials comparing BCR as an adjunct to mammography to mammography alone are expected to begin very soon.

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2nd Dose of Leuprolide Gets FDA Approval for Palliative Treatment of Advanced Prostate Cancer

The FDA has approved the use of leuprolide (Eligard) at a dose of 22.5 mg for the palliative treatment of patients with advanced prostate cancer. The drug, trade name Eligard, has been approved at the 7.5 mg dose since the beginning of the year.

Approval of Eligard 22.5 mg (three-month sustained release product), comes following a less than 10-month approval process for the New Drug Application. The drug is made by Atrix Laboratories and will be marketed by the company's partner, Sanofi-Synthelabo, Inc.

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Darbepoetin Alfa Approved for Chemo-Related Anemia

The FDA has approved the use of darbepoetin alfa for the treatment of chemotherapy-induced anemia in patients with nonmyeloid malignancies. The drug, marketed under the trade name Aranesp by Amgen, is a recombinant erythropoietic protein and requires fewer injections than existing treatment (epoetin alfa) because it maintains its level in the blood approximately three times longer.

“Anemia can take a tremendous toll on patients undergoing chemotherapy, often leaving them too weak to perform routine activities, and in severe cases, anemia can force doctors to interrupt chemotherapy regimens,” Robert E. Smith, Jr., MD, President of South Carolina Oncology Associates PA and an Aranesp investigator, said in a news release from Amgen.

“Aranesp not only helps correct anemia and maintain hemoglobin levels during chemotherapy, but also helps chemotherapy patients and their physicians overcome barriers that can hinder the delivery of current anemia treatment, notably the need for frequent office visits.”

Aranesp was approved by the FDA in September 2001 for the treatment of anemia associated with chronic renal failure, for patients on dialysis and patients not on dialysis.

The drug is contraindicated in patients with uncontrolled hypertension. Erythropoietic therapies may increase the risk of thrombotic and other serious events, and dose reductions are recommended if the hemoglobin increase exceeds 1.0 g/dL in any two-week period.

The most commonly reported side effects in trials of Aranesp to date have been fatigue, edema, nausea, vomiting, diarrhea, fever, and dyspnea; no important differences were observed between Aranesp and Epoetin alfa.

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Clinical Trial Guide

A new patient reference guide on clinical trials is available from the American Association of Health Plans (AAHP) and ECRI (formerly the Emergency Care Research Institute), an independent, nonprofit health services research organization.

Titled “Should I Enter a Clinical Trial? A Patient Reference Guide for Adults With a Serious or Life-Threatening Illness,” the comprehensive online reference, designed for patients and physicians, answers questions about the risks, benefits, and implications of entering a clinical trial.

In addition to basic information such as the different types of trials, eligibility, and enrollment, the guide addresses ethical issues in clinical research and the potential impact on trial participants. There is a glossary, a list of resources for locating trials that are seeking patients, and step-by-step checklists for potential participants to work their way through during the decision-making process. Information on regulations, laws, and the principles governing trials is also covered.

The new guide is free and can be accessed on the Web sites of both organizations: and

© 2002 Lippincott Williams & Wilkins, Inc.
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