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Odds & Ends
Abstracts by Tom Swartz and Bruce Shriver
In this issue:
The Keep Hope Forever Foundation The Keep Hope Forever Foundation is a group of young people who are helping to spread hope and awareness to children and teens who are fighting life threatening illnesses. It was established in honor of Mackenzie J. who is fighting Leukemia. Its mission is to share hope with young people and show them that just because of an illness, it does not make any child different from the next. They are there to share stories, spread awareness, and most of all hope to the children who are currently fighting. From digital graphics, to hand made bracelets the group engages in many projects and fundraisers to help all these amazing warriors. Each month they have three or four different projects going on along with banner offers for your child's caringbridge, care page, or myspace page. They also send out care packages to their Hoping Hearts each month to help bring smiles to young faces. By request they have done short presentations to daycares and schools about their activities.
Directory of Sarcoma Patient Advocacy Organizations and Foundations A copy of the current directory, which is being maintained by Arthur Beckert at the Sarcoma Alliance, can be downloaded by clicking here.
Ariad Initiates Phase III Trial of Oral Deforolimus Ariad Pharmaceuticals has initiated a Phase III clinical trial of oral deforolimus in patients with metastatic soft-tissue and bone sarcomas. This pivotal trial will assess progression-free survival as the primary endpoint and overall survival as a secondary endpoint in patients with metastatic sarcomas following a favorable response to chemotherapy. The sarcoma multi-center clinical evaluation of the efficacy of deforolimus (Succeed) trial is a randomized, double-blind, placebo-controlled Phase III study of oral deforolimus in metastatic sarcoma patients who have achieved a favorable response to chemotherapy. Continued treatment with traditional chemotherapeutic drugs has not been established to provide additional clinical benefit after such a response, according to Ariad. This trial is designed to evaluate approximately 650 patients who will be randomized (1:1) to oral deforolimus or placebo at approximately 125 sites. The trial is 90% powered to detect a 33% increase in median PFS (corresponding to a hazard ratio of 0.75) comparing the deforolimus arm with the placebo arm. Two interim efficacy analyses are included. Complete patient enrollment and the second interim analysis are expected to take place within approximately two years of the first patient being enrolled. George Demetri, director, the Ludwig Center at Dana-Farber Cancer Institute said: "The Succeed trial is one of the largest studies of patients with soft-tissue and bone sarcomas ever conducted - representing an expansive international collaboration of sarcoma experts to test a hypothesis that could shift the paradigm of how such patients are treated. In particular, this trial is designed to study a new treatment option vs. the current standard of 'watchful waiting' for disease progression. Thus, the trial will provide a unique opportunity to evaluate whether more active management of the disease prolongs progression-free survival over the traditional clinical approach." Harvey Berger, chairman and CEO of Ariad, added: "The start of the Succeed trial signals an important advance in our comprehensive global development of deforolimus in oncology. Deforolimus is a promising targeted oral treatment, and we look forward to obtaining further confirmation of its efficacy and safety in patients with metastatic sarcomas from this trial."
Biomira Initiates Phase 1 Trial Of PX-478 In Patients With Advanced Metastatic Cancers Biomira Inc. has enrolled its first patient in a Phase 1 clinical trial of PX-478 in patients with advanced metastatic cancer. PX-478 is a novel small molecule compound designed to inhibit the activity of hypoxia inducible factor (HIF)-1 alpha, a transcription factor that controls the expression of a number of genes important for growth and survival of cancer cells. Genes regulated by HIF-1 alpha contribute to diverse functions such as new blood vessel growth (angiogenesis), use of glucose for energy, and protection against apoptosis (programmed cell death). "HIF-1 alpha plays a critical role in the response of cancer cells to low oxygen levels, stimulating processes that increase oxygen concentrations and allow tumors to grow and thrive," said Dr. Lynn Kirkpatrick, Ph.D., Chief Scientific Officer of Biomira. "In our preclinical studies, PX-478 produced significant tumor regression and growth delay in a wide variety of models of human cancers, including lung, breast, prostate, colon, kidney, pancreas, and ovarian cancers. We believe this is the first small molecule agent to enter clinical trials that directly lowers levels of HIF-1 alpha protein in tumors." The Phase 1 trial is expected to enroll up to 36 patients with advanced solid tumors or lymphoma who have failed or are intolerant of standard therapy. PX-478 will be administered orally on days 1 through 5 of a 21-day cycle. Primary objectives of the study include establishment of a maximum tolerated dose, evaluation of safety, and analyses of pharmacodynamic and pharmacokinetic profiles of PX-478. Other objectives are to evaluate the effects of PX-478 on tumor blood flow and vascular permeability as measured by DCE-MRI, and to evaluate the anti-tumor activity of PX-478 in patients with advanced malignancies. "We are very excited about the potential for PX-478 as an anti-cancer compound with broad potential application," said Dr. Robert L. Kirkman, MD, President and Chief Executive Officer of Biomira.
On September 25th, ZIOPHARM Oncology, Inc. announced positive interim data from an ongoing phase II trial of ZIO-201 (isophosphoramide mustard- IPM) to treat refractory sarcoma patients. The data were presented by Rashmi Chugh, MD, Principal Investigator from the University of Michigan at the 14th Annual European Cancer Conference (ECCO) meeting held in Barcelona, Spain. The trial has now enrolled 51 patients with soft tissue sarcoma or osteosarcoma and the reported interim dataset included the first 31 evaluable patients. These patients were refractory to treatment, having undergone a median of four prior regimens. Key findings for these 31 evaluable patients at the time of analysis include: In 14 of the 31 patients (45%), there was a clinically beneficial response including one partial response (PR), four minor responses (MR), and nine with stable disease (SD). In patients with leiomyosarcoma, five of seven patients (71%), had stable disease or better. Of the 31 patients, 80% had previously been treated with ifosfamide (IFOS) while 20% were IFOS naive; of the IFOS naive, six of seven (86%) have stable disease or better. ZIO-201 was well tolerated with adverse events primarily mild to moderate and gastrointestinal or renal related, while no significant bone marrow suppression, alopecia (hair loss), or neurotoxicity were reported. Principal investigator, Rashmi Chugh, M.D, stated, "These positive interim results are encouraging with ZIO-201 clearly demonstrating clinical activity without the bone marrow suppression and neurotoxicities associated with IFOS treatment. These findings also suggest that ZIO-201 is a promising candidate to combine with other treatments to enhance activity without overlapping toxicities." Robert G. Maki, MD, PhD., co-leader of the Adult Sarcoma Disease Management Team at Memorial Sloan Kettering Cancer Center and not directly participating in this study, commented, "These interim data from a difficult-to-treat population demonstrate ZIO-201 to be a very promising drug candidate. We look forward to the maturation of data for the phase II trial with the expectation of an ensuing pivotal trial, as ZIO-201 could be of significant clinical benefit in the treatment of this disease." The Company expects to report final data at upcoming medical meetings, including the Connective Tissue Oncology Society Annual Meeting in November 2007.
NCCN Updates Dermatofibrosarcoma Protuberans Guidelines The National Comprehensive Cancer Network (NCCN) has announced important updates to the NCCN Dermatofibrosarcoma Protuberans (DFSP) Guidelines. DFSP is a rare, low-grade sarcoma characterized by a t(17;22) chromosomal translocation resulting in the over-expression of platelet-derived growth factor receptor-beta (PDGFRB). This abnormal proliferation of cells can quickly grow into a malignant tumor. In a phase II open-label trial--Study B2225, the largest study involving patients with DFSP)--imatinib mesylate, an inhibitor of PDGFRB, was found to be safe and effective in the treatment of patients with locally advanced and metastatic DFSP tumors containing t(17;22) translocation. Based on the results of this study and five other published case reports, the FDA approved imatinib mesylate for the treatment of adult patients with unresectable, recurrent and/or metastatic DFSP. In response to the favorable results and the FDA approval, the NCCN DFSP Guidelines now include imatinib mesylate (Gleevec®, Novartis Pharmaceuticals) as an option for patients with DFSP with the following indications: (i) positive surgical margins following re-resection, if further resection is not feasible; (ii) recurrent disease, if additional resection would lead to unacceptable functional or cosmetic outcomes; (iii) metastatic disease. Study B2225 also showed that patients with DFSP lacking t(17;22) may not respond to imatinib. As a result, the guidelines suggest that molecular analysis of a tumor using cytogenetics may be useful prior to the institution of imatinib therapy. The NCCN Clinical Practice Guidelines in Oncology(TM) are widely recognized and applied as the standard of care in oncology in the United States in both the community and the academic practice settings. These guidelines are developed and continually updated through an evidence-based process with explicit review of the scientific evidence by multidisciplinary panels of expert physicians from NCCN Member Institutions. The most recent version of this and all the guidelines are available free of charge at www.nccn.org.
Illustrator’s Art To Be Auctioned For Sarcoma Research Chris Gall is designing a snowflake that will fight cancer. The illustrator’s “Dinoflake,” a five-inch wooden flake, will hang in a gallery and museum in Massachusetts before being auctioned in November to fund research of sarcoma. The only parameters for the charity project, titled “Robert’s Snow: for Cancer’s Cure” was that each piece of contributed art be handcrafted or painted on a wooden snowflake. “I was trying to think of something that was completely unexpected,” Gall said. “I was thinking the easiest would be a Christmas theme or something snowy, so I got off way on another tangent.” The project, which will benefit the Dana-Farber Cancer Institute in Boston, began after a children’s book illustrator’s husband was diagnosed with sarcoma. During treatment, Grace Lin told her husband a story about a mouse that couldn’t go outside and play in the snow. The story grew into the children’s book, “Robert’s Snow.” When Lin’s husband had a relapse, her colleagues began creating snowflakes. Gall shared an editor with Lin through the publishing company Little, Brown and Company and was asked to participate. He has drawn illustrations for the pages of Time, People, The New York Times and the Washington Post, not to mention ads for Pepsi, Ford and Radio City Music Hall. Most recently, he wrote and illustrated “Dear Fish,” which was honored last year as an “Original Voices” selection at Borders bookstores nationwide. Appropriately enough for a guy who created a book that speculates about what would happen if fish visited land, Gall decided to create his representation of crystallized ice in the likeness of a dinosaur. He said the theme relates to a book of his that is slated for publication in the spring of 2009. He said he couldn’t give details about the book. “My dinosaur has a Santa hat,” he said. “That’s my nod to Christmas.” About 200 children’s book illustrators are participating in the charity project. Other notable participating illustrators include Mo Willems of the Knuffle Bunny Series and Kevin Hawkes of “The Library Lion.” Three online auctions will take place Nov. 19-23, Nov. 26 through 30 and Dec. 3-7. To preview Gall’s snowflake or place a bid, visit www.robertssnow.com.
Pediatric Caregiver Telephone Support Network Caring for a child with a devastating illness is never an easy task. To provide support to people caring for young cancer patients, the Anderson Network at The University of Texas M. D. Anderson Cancer Center has developed a new component to its existing telephone support program. The purpose of the Pediatric Caregiver Telephone Support Network is to connect pediatric cancer caregivers and patients in hopes that sharing similar experiences will provide information and support. As the program grows, the Anderson Network hopes to build its volunteer base by asking current and former pediatric cancer caregivers, as well as survivors of childhood cancers, to sign up to become telephone support volunteers. Survivors and caregivers can sign up for the program regardless of where they or their children received treatment. For more information, contact the Anderson Network at (713) 792-2553, at 1-800-345-6324 or at andersonnetwork@mdanderson.org. To sign up online, visit the Anderson Network site and click on “Membership Profile.”
Uterine LMS & Aromatase Inhibitors (AIs) by Sharon Anderson, MSW, LMS Research Advocate & Survivor Almost half of the ACOR LMS list members had LMS that was thought to be a uterine fibroid. Here are some suggestions for ULMS we have discussed on the list:
GIST in the UK — Realities Bite Hard Read this informative article by Roger Wilson of Sarcoma UK in the October 2007 (Vol. 8, No. 10) issue of the Life Raft Group Newsletter.
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