Full Text:"We could completely inhibit the growth of the tumor if we used a big enough dose," said study co-author Bharat B. Aggarwal, chief of the Cytokine Research Section in the Department of Experimental Therapeutics at the University of Texas M.D. Anderson Cancer Center in Houston. His report is set to appear in the Aug. 15 issue of Cancer.

LINKThe eight founding members of the Brain Tumor Funders’ Collaborative (BTFC) are: the American Brain Tumor Association, Brain Tumour Foundation of Canada, Brain Tumor Society, Children’s Brain Tumor Foundation, the Goldhirsh Foundation, the James S. McDonnell Foundation, the National Brain Tumor Foundation and the Sontag Foundation. BTFC’s goal is bridging the “translational gap” that prevents promising laboratory findings from yielding new medical treatments.

For individuals diagnosed with aggressive brain tumors called “gliomas” the dedicated and heroic efforts of researchers, clinicians, patients, and families has been unable to alter the devastating outcome. For most patients the tumors will be lethal. “The aggressive nature of gliomas, the logistical difficulties in delivering drugs across the blood-brain barrier, and the functional uniqueness of the human brain all contribute to the lack of progress against these deadly tumors” explains Rob Tufel of the National Brain Tumor Foundation.

Other hurdles are also contributing to the lack of progress. The Brain Tumor Society’s Neal Levitan points out that “the preclinical pipeline for the identification and testing of potential new therapies is fragmented among different populations of research scientists and clinicians. The current system is dated and fails to take optimal advantage of the biomedical community’s emerging capacity for discovering, testing, and developing novel therapies.” The disconnection between the experimental systems used at the laboratory bench and the clinical realities of patients often means that compounds showing promise in early phases of pre-clinical research fail in later stages of clinical investigation. “It is time for the new tools of genomics, proteomics, complex systems biology, and informatics to be brought to bear on the quest for new brain tumor therapeutics,” observes Susan Fitzpatrick of the James S. McDonnell Foundation.

The new funding initiative, developed after two years of workshops identifying opportunities and barriers to progress, is aimed at addressing the frustrations of scientists and clinicians working in the brain tumor field. “BTFC decided to target its funding towards helping researchers develop new approaches, new ways to assemble information, and enabling them to form new colleague connections to make this happen,” stated American Brain Tumor Association’s Naomi Berkowitz. Susan Marshall, with the Brain Tumour Foundation of Canada continues “With BTFC support, newly formed teams will be able to design novel, innovative, approaches to the problem of moving promising results from basic and pre-clinical research to drug development and clinical applications.”

“Progress against this horrible disease requires new collaborations assembling skills and expertise not traditionally associated with brain tumor research,” according to Goldhirsh Foundation’s Rita Berkson Noting that it is not only the scientists who need to collaborate, Kay Verble of the Sontag Foundation added “We thought it only fair that if we were asking researchers to broaden their collaborations then so should the funders!” Long-time brain tumor research advocate Susan L. Weiner, of Children’s Brain Tumor Foundation is overjoyed with the collaborative nature of new funding initiative. “BTFC represents a dream come true. Together, we can do what no one organization could do alone.”

Further information about the BTFC and its new funding initiative can be found at www.braintumorfunders.org . Brain tumor facts are available at http://www.nci.nih.gov/cancertopics/types/brain/
BTFC member organizations and primary contacts:
• American Brain Tumor Association - Naomi Berkowitz (847) 827-9910
• Brain Tumour Foundation of Canada – Susan Marshall (519) 642-7755 x 22
• Brain Tumor Society – Neal Levitan (800) 770-8287
• Children’s Brain Tumor Foundation – Susan L. Weiner (718)788-1503
• Goldhirsh Foundation – Rita Berkson (203) 488-2697
• James S. McDonnell Foundation – Susan Fitzpatrick (314) 721-1532
• National Brain Tumor Foundation – Rob Tufel (800) 934-CURE (ext. 107)
• Sontag Foundation- Kay Verble – ( 904) 273- 8755

link TransMolecular, Inc., a biotechnology company focused on cancer drug research, today announced the initiation of a phase II open-label, multiple-dose study of intracavitary administered 131I-TM-601 in adult patients with recurrent high-grade glioma. The company plans to have more than 12 sites participating in the study. Sites currently participating include City of Hope National Medical Center, Duarte, CA; Florida Hospital, Orlando, FL; Northwestern University, Evanston, IL; Saint Louis University, St. Louis, MO; Saint Mary's Health Care, Grand Rapids, MI; and University of Alabama at Birmingham, Birmingham, AL.

"On the strength of the results of our phase I/II data that showed good safety and tolerability, we are very excited to advance this program into phase II development. We are proud to be working on a product that could fill a need for safe, more effective treatment for those who suffer from this devastating disease," said Lyle A. Hohnke, Ph.D., chairman of the board and acting CEO of TransMolecular and a partner at Tullis-Dickerson & Co., Inc., an investor in the company.

131I-TM-601 is a radiopharmaceutical containing a synthetic version of chlorotoxin, a substance derived from scorpion venom. Chlorotoxin, or TM-601, specifically seeks out and binds to a receptor expressed on tumor cells, but not on normal cells. TM-601 acts as the guidance system that very effectively delivers a radioactive payload to its target, precisely killing the tumor cells and minimizing collateral damage to normal cells. 131I-TM-601 has received Orphan Drug and Fast Track Development Program status from the FDA.

The Phase II study will be conducted in two parts, both involving adult patients with recurrent high-grade glioma. The first sequence is an open- label dose escalation, multi-dose study. Four cohorts of patients will be treated postoperatively at escalating dose levels until the Maximum Practical Dose (MPD) is reached or until determination of the Maximum Tolerated Dose (MTD). After the MPD or MTD is reached, this dose will be expanded in the second trial sequence.

The second trial sequence is an open-label, randomized study in a larger group of patients. Patients will receive either a three- or six-dose treatment cycle at the previously determined MPD or MTD to evaluate the safety, time to disease progression and survival rates after treatment.

The company anticipates that in the first sequence of the study, three patients will be enrolled in each dose cohort. An additional group of three patients may be enrolled at a given dose based on safety. In the second sequence, a total of 54 patients will be randomized in two equal groups treated with either one cycle of three or one cycle of six repeat doses of intracavitary 131I-TM-601.

ABOUT GLIOMA

Glioma is highly invasive, sending cancerous cells throughout the brain and spinal cord. Surgical techniques fail to eradicate the tumor and other adjuvant therapies are inadequate. Brain cancers are among the most difficult and expensive cancers to treat. About 36,000 primary brain tumors are reported in the U.S. each year; of these, more than 17,000 are diagnosed with high- grade gliomas. About half of these patients die within the first year, according to the American Cancer Society.