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  • This is a listing of the drugs currently being used - either as approved treatments, off label treatments, clinical trials or drugs in the pre-clinical stage for brain tumors.
  • This list was last updated: 04/29/2014
  • This area is always under development. Please let us know if we missed any drugs or have any errors.
  • To change the sort order, click on the column label.
  • There are general videos about most of these treatments in our video library. We also have videos or more details specific to each of the following treatmments:
Generic Name Product Name Company Status Mode of Action Comments Last Updated
ANG1005 AngioChem Phase 1 trials Transports drugs across BBB Interesting idea. Visit the website for details 07/24/2008
GliAtak Advantagene Phase 2 trials Advantagene core technology is based on Gene Mediated Cytotoxic Immunotherapy (GMCI™). The lead agent is an adenoviral vector that encodes the gene for Herpes Simplex thymidine kinase (AdV-tk). It is injected into the tumor and followed by an orally administered anti-herpetic prodrug. The combination of AdV-tk and the prodrug works through local and systemic mechanisms to attack local and distant tumor sites. The prodrugs are phosphorylated and converted into cytotoxic drugs that lead to termination of DNA synthesis during cell division and tumor growth, or during DNA repair, after radiation or chemotherapy damage. This kills the targeted cells, sorrounding tumor cells and fast growing tumor blood vessels (anti-angiogenesis). AdV-tk protein is a powerful antigen that in combination with surgery or radiation effectively stimulates a potent anti-tumor immune response: GMCI™. GMCI™ stimulates all three of the essential elements for a potent immune response, rather than only a subset of the required steps as used by previous only moderately effective immunotherapy attempts. GMCI™ includes the following: a) it exposes tumor-associated antigens (TAAs), (b) it promotes effective antigen presentation to the immune cells, and (c) it stimulates an immense amplification of the number of tumor-specific T-cells. The local cell death reduces primary tumor burden. This is good, but such an effect can also be achieved by cancer therapies currently in use, such as radiation and surgery. Although AdV-tk, as a radio-sensitizer, improves local efficacy without increased toxicity, the key aspect of GMCI™ is the complete induction of a systemic anti-tumor immune response, unlike many previous immunotherapy technologies, which address only a subset of the required immune-response components. The complete immunologic effect of GMCI™ using AdV-tk was discovered by Advantagene’s scientists and helps explain the potent cell-mediated antitumor response observed in animal and clinical trials. GMCI™ technology is the first to proactively combine all the components necessary to generate a potent immune response. Previous immunotherapy approaches with a subset of these components have shown little clinical significance due to short duration or limited potency. Independent presentation of tumor antigens generates immunity to the antigens but this has not translated to clinical improvement. Recruitment of APCs by GM-CSF recruitment or physical isolation have reported some clinical successes but can be very complicated and of limited in duration. Finally, T-cell expansion by cytokine stimulation, such as IL-2, or by physical expansion in the laboratory have also had enticing but limited success. Advantagenes GMCI™ technology combines all three of the components for a very potent response and can be easily and repeatedly administered. The potential to use an orally administered prodrug instead of the intravenous infusion is evident. This was a key advancement since it greatly decreased the morbidity of the study to the patients (a few pills a day, versus two or three one hour intra-venous infusions a day for fourteen days per course of treatment), and greatly reduced the cost per patient in the trial. With the advent of the oral prodrug, the company was able to design clinical gene therapy studies that involved only simple outpatient procedures. The first clinical study established a safe dose range for GliAtak™ and gave encouraging efficacy indications in terms of prolonged survival by several subjects. Since then, the company has focused its efforts on improving the clinical applicability of the AdV-tk approach by combining it with standard cancer treatments such as surgery and radiation therapy. Following on the experience from the Phase I study in brain tumors, and based on preclinical studies in animal brain tumor models, a Phase Ib/II clinical trial of GliAtak™ in conjunction with radiation, in newly diagnosed malignant gliomas has been developed. This is planned to be conducted at several premier academic centers including Massachusetts General Hospital and Ohio State University. Randomized clinical data from these products is expected within 36 months. 09/08/2009
3F8 MAb United Therapeutics Phase 2 trials Monoclonal antibody In December, 2007, United Therapeutics entered into an agreement with Memorial Sloan-Kettering Cancer Center to exclusively license certains right to this investigational antibody. This antibody is a mouse IgG3 MAb which is being tested for treatment of neuroblastoma, a rare cancer of the sympathetic nervous system mainly affecting children. It is the most common extracranial solid cancer in children and the most common cancer in infants. More than 400 patients have been treated with this antibody since 1986. There are fewer than 1,000 new cases of neuroblastoma diagnosed each year. 04/11/2009
8H9 MAb United Therapeutics Phase 1 trials Monoclonal antibody In December, 2007, United Therapeutics entered into an agreement with Memorial Sloan-Kettering Cancer Center to exclusively license certains right to this investigational antibody. This antibody is a mouse IgG1 MAb which is being tested for treatment of metastatic brain cancer. In the US, more than 100,000 cases of metastactic brain cancer are diagnosed each year. 04/11/2009
ABT-627 Atrasentan® Abbott Laboratories Phase 2 trials A pyrrolidine-3-carboxylic acid derivative with selectivity against the ETA receptor 9/13/05: FDA Advisory committee did NOT recommend approval. I suggest that we remove this item. 07/05/2007
AEE788 Novartis Pharmaceuticals Phase 1 trials 04/11/2009
AMG 102 Amgen Phase 2 trials Blocks the action of hepatocyte growth factor/scatter factor (HGF/SF). This is an investigational fully human monoclonal antibody that targets the action of anti-hepatocyte growth factor/scatter factor. HGF signaling through its receptor c-Met appears to play an important role in many types of human cancers. The HGF/SF:c-Met pathway mediates a large number of normal activities in cells of epithelial origin including proliferation, survival, migration, and invasion. The dysregulation of the HGF/SF:c-Met pathway often leads to tumorigenesis and metastasis. 04/16/2009
ANG-1005 AngioChem Phase 1 trials Studies have shown that ANG1005 gains entry into the brain compartment by targeting the low-density lipoprotein receptor-related protein (LRP) which is one of the most highly expressed receptors on the surface of the BBB. Once inside the brain, ANG1005 enters tumor cells using the same receptor-mediated pathway through LRP, which is upregulated in various cancer cells including malignant glioma and metastatic cancers in the brain. ANG1005 is a novel taxane derivative created from the Engineered Peptide Compound (EPiC) platform that is designed to cross the blood-brain barrier (BBB) by receptor-mediated transcytosis, overcoming the BBB’s normal function of blocking foreign substances which prevents more than 95 percent of all drugs from reaching the brain. ANG1005 is currently being evaluated in two separate Phase 1/2 multicenter, open-label, dose escalation studies to explore the maximum tolerated dose and obtain data on safety, tolerability and preliminary evidence of efficacy in patients with recurrent malignant glioma and in patients with advanced solid tumors and brain metastases. Studies demonstrate that ANG1005 has an excellent safety and tolerability profile, as evidenced by laboratory and neurocognitive data, and does not show evidence of eliciting an immune response in humans. Although the Phase 1/2 studies were designed for safety and tolerability, results to date have showed substantial reduction in the number and size of tumors in some patients. ANG1005 successfully and rapidly crosses the BBB into the brain tissues at a rate 100 times greater than paclitaxel and distributes homogeneously across all the brain regions. 04/16/2009
Antineoplaston Burzynski Clinic, Houston, Texas, Phase 2 trials Alternative medicine "Alternative medicine" Antineoplaston therapy is an alternative cancer treatment that involves using a group of synthetic chemicals called antineoplastons to protect the body from disease. Antineoplastons are made up mostly of peptides and amino acids originally taken from human blood and urine. 07/05/2007
AP23573 ARIAD Pharmaceuticals, Inc Phase 2 trials mTOR inhibitor On Aug. 18, the FDA approved orphan drug and fast-track status for a non-prodrug rapamycin analog (AP23573 injection, made by ARIAD Pharmaceuticals, Inc.) for the treatment of soft-tissue and bone sarcomas. The mTOR (mammalian target of rapamycin) is an kinase activated through the PI3K/Akt cascade. This blockade leads to cell arrest in G1. Rapamycin and its analogs are cyto-static against xenografts of glioblastoma, medulloblastoma, breast cancer, and prostate cancer. The mTOR inhibitor class of drugs include: sirolimus/rapamycin), temsirolimus/CCI-779, RAD-001 and AP23573. Rapamycin has long been used as an immunosupresor and off-lable as a cancer treatment. They are all in clinical development for cancer treatments. Among which temsirolimus is te most advanced. On May 30, 2007, the U. S. Food and Drug Administration granted approval for temsirolimus (Torisel™, made by Wyeth, Inc.) for the treatment of advanced renal cell carcinoma (RCC). Ref: Tsang et al. Targeting mammalian target of rapamycin (mTOR) for health and diseases. Drug Discovery Today, 12: 112-124, Feb 2007. 07/08/2007
Arsenic trioxide Trisonex Cell Therapeutics Approved, but used off label Not completely understood. It causes morphological changes and DNA fragmentation characteristic of apoptosis in leukemia cells in vitro. It may also cause damage or degradation of the fusion protein PML/ROR-alpha Approved for a type of leukemia. Just starting human trials. It has shown good results in glioblastoma tissue cultures. It entered PhaseI/II trials for glioblastoma (NCT00275067 & NCT00095771). 07/05/2007
AZD2171 Recentin (tentative), cediranib AstraZeneca Phase 3 trials Inhibitor of VEGFR tyrosine kinase AZD2171 (tentative trade name Recentin) is a potent inhibitor of vascular endothelial growth factor (VEGF) receptor tyrosine kinases. It is being developed by AstraZeneca as a possible chemotherapeutic agent for oral administration. Ref: Batchelor TT et al. AZD2171, a pan-VEGF receptor tyrosine kinase inhibitor, normalizes tumor vasculature and alleviates edema in glioblastoma patients. Cancer Cell. 2007 Jan;11(1):83-95. 07/12/2007
banoxantrone Transcept Pharmaceuticals, Inc. Phase 2 trials 04/17/2009
BCNU, Carmustine BiCNU® Bristol Myers Squib Approved for brain tumors Carmustine alkylates DNA and RNA and has been shown to inhibit several enzymes by carbamoylation of amino acids in proteins. 09/27/2005
berubicin hydrochloride (RTA 744) Reata Pharmaceuticals Phase 2 trials This drug is described as the hydrochloride salt of the anthracycline derivative berubicin possessing potential antineoplastic activity. Berubicin intercalates into DNA and interrupts topoisomerase II activity, resulting in the inhibition of DNA replication and repair, and RNA and protein synthesis. Unlike other anthracycline derivatives, this agent crosses the blood-brain barrier (BBB). It was reported that on April 15, 2008, researchers from MD Anderson would be presenting two posters on Reata's Phase 2 drug for brain tumors, berubicin hydrochloride. These posters were entitled "Berubicin, a Brain Tumor Targeting Anthracycline: Discovery and Clinical Evaluation" and "Metabolite Pharmacokinetics of Berubicinol (BRB-ol), the 13-hydroxy metabolite of RTA 744 (berubicin(BRB)), a Blood-Brain Barrier Penetrating Anthracycline Active Against High Grade Glioma, in Phase 1/II Clinical Trials." These posters describe the discovery and early development of this novel agent. Additionally, they include updated clinical data on the Phase I trial of berubicin in patients with primary brain tumors. The drug has shown significant activity in this study including multiple Objective Responses, which are rare in this patient population. The trial was to complete final data collection in December, 2008 and is now closed. Berubicin is currently undergoing Phase 2 clinical testing in women whose breast cancer has metastasized to the brain. 07/16/2009
Bevacizumab Avastin Genetech Approved, but used off label Anti-angiogenisis Currently in trials with CPT-11 for brain tumors. Results from a phase II clinical trail at Duke have been published: Vredenburgh JJ et al., Phase II trial of bevacizumab and irinotecan in recurrent malignant glioma. Clin Cancer Res. 2007 Feb 15;13(4):1253-9. 04/09/2009
BG00001 IDEC-201, Ad-hIFN-beta Biogen Idec Phase 1 trials Interferon Beta Gene Delivery The product is not listed on clinical development page of Biogen Idec website. Adenoviral vector encoding human interferon beta. Active Phase I trail as of 07/08/07: Gene Therapy for Pleural Malignancies (NCT00299962). Two Phase I trails closed. 07/08/2007
BIBW 2992 Tovok Boehringer Ingelheim Phase 2 trials BIBW is an orally bioavailable dual receptor tyrosine kinase (RTK) inhibitor with potential antineoplastic activity. EGFR/HER2 tyrosine kinase inhibitor BIBW 2992 irreversibly binds to and inhibits human epidermal growth factor receptors 1 and 2 (EGFR-1; HER2), which may result in the inhibition of tumor growth and angiogenesis. EGFR/HER2 are RTKs that belong to the EGFR superfamily; both play major roles in tumor cell proliferation and tumor vascularization and are overexpressed in many cancer cell types. Overexpression of EGFR and HER2 is associated with poor prognosis and advanced stage cancers. Once BIBW 2992 covalently binds to EGFR/HER2, the ATP site is permanently blocked, inhibiting the downstream signalling cascade. As of December, 2008, BIBW 2992 was undergoing a Phase III clinical trial for lung cancer and Phase II trials for breast, prostate, and head and neck cancers, including glioblastoma. 07/16/2009
BMS-844203 (CT-322) Adnectin Bristol-Myers Squibb Phase 2 trials Adnectins are a novel, proprietary class of targeted biologics derived from human fibronectin. The Adnectin, BMS-844203 (CT-322) is an anti-angiogenic antagonist of the VEGFR-2 pathway. 09/08/2009
BNCT (Boron Neutron Capture Therapy) Phase 2 trials Targets radiation to tumor cells only Available outside usa if you can't get into a usa trial 12/03/2005
Bortezomib, PS-341 VELCADE™ Millennium Approved, but used off label Proteasome Inhibitor The ubiquitin/proteasome system is the main posttranscriptional degradation mechanism of proteins involved in cell cycle, DNA transcription and repair, apoptosis, angiogenesis, and cell growth. Target molecules of this system include p53, mdm2, p21, p27, Rb, cyclins A, B, and E, I IKB, NF-KB, Bcl-2, Bax, and caspase-3. The development of drugs that work on the ubiquitin/proteasome system is a potential anticancer strategy. The prototype drug of this class is PS-341 (Velcade), active as a single agent on murine and human xenograft models by enhancing apoptosis, specifically against transformed cells.[153] PS-341 arrests cells in G2-M phase, inducing apoptosis of these cells with simultaneous phosphorylation of Bcl-2. 09/27/2005
BSI-201 BiPar Sciences, Inc. Phase 1 trials BSI-201 targets PARP or poly (ADP-ribose) polymerase, a key enzyme involved in DNA repair and cell proliferation. By inhibiting the enzymatic activity of PARP, BSI-201 significantly enhances the anti-tumor effects of chemotherapy and has shown promising safety and efficacy results in patients with solid tumors. BSI-201 crosses the blood-brain barrier and has a mechanistic basis for synergizing with standard treatment of GBM. BSI-201 is being evaluated in a randomized Phase 2 clinical study of 120 patients with metastatic triple negative breast cancer, as well as in multiple Phase 2 clinical trials in ovarian, uterine, lung, and pancreatic cancers, as well as sarcoma and glioblastoma. BiPar Sciences plans to expand the development of BSI-201 in combination with various chemotherapies and other accepted treatment regimens for several types of cancer. BiPar Sciences is collaborating with the New Approaches to Brain Tumor Therapy (NABTT) consortium, a research organization sponsored by the National Cancer Institute, to evaluate BSI-201 in patients with GBM. The phase I portion of study is designed to determine the MTD (maximum tolerated dose) of BSI-201 with two clinically relevant dosing regimens of TMZ. Secondary objectives in the phase I trial include determining the PK of BSI-201 in malignant glioma patients and correlating BSI-201 PK with degree of PARP-1 inhibition. A safety run-in will confirm the safety of BSI-201 added to standard TMZ and radiation therapy and the phase II portion of the study will assess the efficacy and tolerability of the MTD dose of BSI-201 with daily TMZ and radiation therapy followed by adjuvant TMZ in patients with newly diagnosed GBM and assess overall survival as the primary outcome measure. This trial is recruiting. The drug is administered via IV infusion. Identifier: NCT00687765 04/17/2009
Carboplatin Paraplatin Bristol-Myers Squibb Approved, but used off label Produces DNA crosslinking - stopping cell divisions. 09/27/2005
CC-8490 Celgene Corporation Phase 2 trials CC-8490 is in the benzopyran family of drugs with potential antineoplastic activity. CC-8490 acts as a selective estrogen receptor modulator (SERM), inhibiting the proliferation of estrogen-sensitive breast cancer cells. This agent also inhibits growth and induces apoptosis of glioblastoma cells via a mechanism independent of estrogen receptor-related mechanisms. The phase I trial on this drug began in 2003 and it is listed on as being completed. There is no information on the company's webiste about the phase II trial but the contact number for Celgene Corporation is 908 673-9000. Celegine also makes Thalomid (thalidomide). 09/08/2009
CDX-110 Celldex Therapeutics Phase 3 trials CDX-110 is an immunotherapy that targets the tumor specific molecule called EGFRvIII, a functional variant of the epidermal growth factor receptor (EGFR), a protein that has been well validated as a target for cancer therapy. Unlike EGFR, EGFRvIII is not present in normal tissues, suggesting this target will enable the development of a tumor-specific therapy for cancer patients. Furthermore, EGFRvIII is a transforming oncogene that can directly contribute to the cancer cell growth. While originally discovered in glioblastoma multiforme (GBM), the expression of EGFRvIII has also been observed in various other cancers such as breast, ovarian, metastatic prostate, colorectal, and head & neck cancers. It is reported to be present in 25-40 percent of GBM tumors. The initial clinical development of EGFRvIII immunotherapy was led by collaborating investigators at the Brain Tumor Center at the Duke Comprehensive Cancer Center (Durham, NC) and at the M.D. Anderson Cancer Center (Houston, Texas). The investigators enrolled patients with newly diagnosed GBM into 2 studies: CDX-110 vaccine alone (the “ACTIVATE” study) or CDX-110 plus temozolomide (the “ACT II” study). The results of these studies have demonstrated a significant increase in the time to disease progression and overall survival relative to appropriately matched historical controls. The therapy has been well tolerated and significant immune responses to EGFRvIII were generated. The only frequently observed side effect was inflammation at the injection site. Importantly, 21/24 relapsed tumors that were biopsied or resected were found to no longer have significant EGFRvIII expression, suggesting a potent immune rejection of EGFRvIII expressing tumor cells. NEWS RELEASE: Pfizer and Celldex Therapeutics Present Update on CDX-110 (PF-04948568) Phase 2 Brain Cancer Studies at 45th Annual ASCO Meeting - Updated follow-up of time to progression and overall survival reported from Phase 2 clinical trials with CDX-110 (PF-04948568) and temozolomide - ORLANDO, Fla.--(BUSINESS WIRE)--May 30, 2009-- Pfizer (NYSE: PFE) and Celldex Therapeutics (NASDAQ: CLDX) today announced the presentation of updated data from two clinical trials of CDX-110 in newly-diagnosed glioblastoma multiforme (GBM) at the 45th Annual Meeting of the American Society of Clinical Oncology (ASCO). CDX-110, an investigational immunotherapeutic vaccine that targets the tumor-specific molecule, epidermal growth factor receptor variant III (EGFRvIII), was developed by Celldex Therapeutics and is now partnered with Pfizer. “Data from the 40 evaluable patients in ACTIVATE and ACT II continue to suggest that vaccination with CDX-110 may be able to improve time to tumor recurrence and overall survival when used in patients with newly-diagnosed GBM. These data also continue to suggest that tolerability and side effects associated with CDX-110 are minimal. These results are very encouraging and we look forward to the results from the ongoing multi-center ACT III Phase 2 study,” said John H. Sampson, MD, PhD, Associate Deputy Director of The Preston Robert Tisch Brain Tumor Center at Duke University Medical Center. ACTIVATE Results: In this single arm Phase 2 study, 18 patients with newly diagnosed and optimally resected EGFRvIII-positive GBM received CDX-110 as a monotherapy following completion of chemoradiation with concurrent temozolomide. Median overall survival (OS) was 26 months and median time to progression (TTP) was 14.2 months. Additionally, three patients remain without relapse more than 4 years from surgery and continue to receive the vaccine within the clinical trial. ACT II Results: In this single arm Phase 2 study, 22 patients with newly diagnosed and optimally resected EGFRvIII-positive GBM received CDX-110 in combination with maintenance temozolomide after having completed chemoradiation with concurrent temozolomide. Median time to progression (TTP) is 15.2 months and three patients continue without relapse after more than two years. Results to date from this ongoing study estimate median overall survival to be 23.6 months (data are not yet final). In addition, and in line with preclinical data that suggested the combination with temozolomide could augment immune responses, patients show robust serological evidence of an immune response against EGFRvIII. Efficacy data from both ACTIVATE and ACT II compare favorably to data for a historical control group of 17 patients, matched for EGFRvIII expression, extent of resection and performance status (Median TTP: 6.3 months; Median OS: 15.0 months). In both studies, CDX-110 was generally well tolerated with local injection site reactions being the most commonly reported toxicity. Additional Results: In addition, preliminary data from a pilot study in a small number of patients with newly diagnosed GBM will be presented. In this study, CDX-110 was given in combination with daclizumab, an antibody that blocks suppressive T cells, to determine whether this combination could further augment immune responses. This data will be presented in a poster session on Sunday, May 31 from 8:00am – 12:00pm EDT. ACT III, a multicenter, single-arm Phase 2 clinical trial in GBM in which all patients will receive CDX-110 in combination with maintenance temozolomide, is ongoing. Total enrollment is expected to be approximately 60 patients. In addition, Pfizer and Celldex are working on the design of a randomized Phase 2 study in GBM to compare CDX-110 plus standard of care to standard of care alone. Contact: Celldex, Thomas Davis, Chief Medical Officer 908 454-7120 09/08/2009
Celecoxib Celebrex® Pfizer Approved, but used off label Cox-2 inhibitor Celecoxib is a nonsteroidal anti-inflammatory drug that inhibits prostaglandin synthesis by specifically inhibiting the cyclooxygenase-2 (COX-2) enzyme. This enzyme is overexpressed in gastric,[165] esophageal, and colorectal carcinomas,[166] and its expression may be important in carcinogenesis. COX-2 enzyme is also upregulated in high-grade human gliomas and is inversely related to patient survival by a mechanism that appears to be independent of p53 immunostaining, bcl-2 expression, loss of p16 or retinoblastoma protein expression, or high MIB-1 expression.[167] The inhibition of COX-2 enzyme activity reduced the proliferation and migration of human glioblastoma cell lines.[168] 09/22/2005
cintredekin besudotox NeoPharm Phase 3 trials Cintredekin besudotox is a tumor-targeting toxin being developed as a treatment for glioblastoma multiforme. It is a recombinant protein consisting of a single molecule composed of two parts: a tumor-targeting molecule and a cytotoxic agent. The targeting component consists of interleukin 13(IL-13), an immune regulatory cytokine. Malignant glioma cells, as compared to normal brain cells, express IL-13 receptors at a higher density. The cytotoxic agent is a potent bacterially derived toxin called PE38. Cintredekin besudotox is designed to detect and bind IL-13 receptors on the surface of malignant glioma cells and selectively deliver PE38 to destroy tumor cells. Cintredekin besudotox is administered by a technique known as convection-enhanced delivery, or CED, in which the drug is delivered through catheters inserted in brain tissue surrounding the tumor (peritumoral administration) or into the tumor (intratumoral administration) following surgical resection of the tumor. CED is designed to infuse Cintredekin besudotox directly to the tumor site and adjacent brain tissue with the goal of killing resident tumor cells and preventing recurrence of tumor cell growth. We hope to show that this method of delivery minimizes both damage to the surrounding cell and toxicity from systemic drug exposure. NeoPharm has exclusively licensed cintredekin besudotox from the NIH and the FDA, and have been developing this drug under a Cooperative Research and Development Agreement, or CRADA, with the FDA Center for Biologics Evaluation and Research, or CBER. Cintredekin besudotox has received orphan drug designation in the US and Europe and the FDA has designated it for the fast track drug development program. In addition, cintredekin besudotox has been selected to participate in the FDA's Continuous Marketing Application, CMA, Pilot 2 program. We also hold a non-exclusive license to utilize a patented process owned by the U.S. government relating to convection enhanced delivery, or CED, for use with drugs, including cintredekin besudotox, in the treatment of gliomas. In early 2007, NeoPharm completed its Phase III clinical trial for cintredekin besudotox. Upon review of the trial results in March 2007, the FDA concluded that an additional Phase III confirmatory trial would be required prior to acceptance of a potential BLA filing by NeoPharm. In June 2008, NeoPharm signed a letter of intent with a clinical research organization (CRO) with regard to the CRO's engagement to oversee a planned confirmatory Phase III trial for cintredekin besudotox. This initial Phase III confirmatory trial was to have the primary objective of overall survival, with a secondary objective of progression-free survival for patients with recurrent GBM against the standard of care. Subsequently, NeoPharm received notification from the Directorate General of Health Services - Office of Drugs Controller General (India) ("DCGI") that it will not at this time grant regulatory approval for NeoPharm to conduct its planned confirmatory IL-13 Phase III trial. In its previous correspondence, the DCGI requested concurrent enrollment of patients in the trial in the USA. In the fourth quarter of 2008, NeoPharm signed a Cooperative Research and Development Agreement ("CRADA") with the National Institute of Neurological Diseases and Stroke ("NINDS"), a part of the National Institutes of Health, for research on a therapeutic agent for untreatable brain diseases in humans. Under the terms of the CRADA, NINDS will deliver IL-13 in conjunction with a surrogate marker via NINDS' patented methodology of Convection Enhanced Delivery ("CED"), which was previously licensed to NeoPharm. NeoPharm will provide its proprietary drug and technical resources to study its effects in various brain cancers in humans. NINDS Institutional Review Board ("IRB") has approved the protocol and NeoPharm has granted NINDS authorization to cross-reference its prior IL-13 IND for the treatment of GBM. Contact: 847 887-0800 09/08/2009
Cisplatin Platinol-AQ Bristol-Myers Squibb Approved, but used off label May cause hearing loss - even with a single dose. Ask your doctor about ways to prevent it. 09/22/2005
CMT-3, COL-3, Collagenex Metastat® CollaGenex Pharmaceuticals Phase 2 trials Matrix Metalloproteinase Inhibitors 09/27/2005
Contusugene ladenovec Advexin Introgen Therapeutics Phase 1 trials p53 tumor suppressor therapy Advexin therapy combines the p53 tumor suppressor with a non-replicating, non-integrating adenoviral delivery system. The p53 gene is one of the most potent members of a group of naturally occuring tumor suppressors, which act to kill cancer ceels, arrest cancer cell growth, and protect cells from becoming cancerous. Advexin is currently in stage 1 trials for GBM, stage 3 trials for head and neck cancer, and stage 2 for breast and lung cancers. 04/11/2009
Dalteparin sodium Fragmin® Pharmacia Approved, but used off label Dalteparin may stop the growth of cancer by stopping blood flow to the tumor and by blocking the enzymes necessary for tumor cell growth. Radiation therapy uses high-energy x-rays to damage tumor cells. Combining dalteparin with radiation therapy may kill more tumor cells It is approved to prevent blood clots, but it is in trials for GBMs in combination with radiation 09/27/2005
DCVax DCVax-L Northwest Biotherapeutics Phase 3 trials DCVax®-L is an experimental autologous cellular therapy designed to create a specific immune response against a patient’s cancer. DCVax®-L utilizes a patient’s own dendritic cells (DC), and an extract of the patient’s own tumor cells to achieve an immune response. The tumor from surgery is shipped to a manufacturing facility, as are blood cells, in order to prepare the DCVax®-L. DCVax®-L is then shipped frozen to the clinic for administration to the patient. DCVax®-L is usually manufactured in sufficient quantities for treatment of at least one year, and often for two or more years. Immunization starts following primary therapy, i.e., surgery, radiation therapy and concurrent chemotherapy. Immunizations are given at weeks 0, 2 and 4 and at months 2, 4, 8 and 12, 18, 24, and 30. DCVax® is a platform technology that can be applied to multiple cancers. It combines a patient’s own dendritic cells (DC) with cancer related proteins, or antigens, with the aim of inducing immune responses against a patient’s cancer cells. Early stage clinical trial data suggests that DCVax®-L may have the ability to significantly delay disease progression, in addition to possibly prolonging patient survival, while maintaining a good quality of life. The DCVax® platform is designed to naturally elicit an immune response against a patient’s cancer. by injection in the skin of dendritic cells that are "educated" to teach the immune system to recognize, and kill cancer cells. Clinical trials to date suggest that this immune response may be effective in delaying time to disease progression in brain cancer, and may significantly prolong survival in brain cancer while maintaining good quality of life. Development stage: A Phase III clinical trial (NCT00045968) to test the efficacy of DCVax®-L is currently enrolling patients. Data from two Phase I clinical trials carried out by Dr. Linda Liau at UCLA, support the overall safety of DCVax®-L, and suggest that DCVax®-L may induce an immune response. The clinical data suggest delayed time to disease recurrence and increased survival, especially in patients with stable disease at entry. Trial design: The Phase III DCVax®-L clinical trial will enroll approximately 300 patients with newly diagnosed GBM at 54 study locations in the USA and one in the UK. Indication: Patients with newly diagnosed Glioblastoma multiforme that require treatment with surgery, radiation and concurrent chemotherapy will be eligible for participation in this clinical trial. Other eligibility criteria include absence of measurable disease progression after completion of primary treatment. Endpoints: The primary endpoint for the Phase III clinical trial is survival free of disease progression. The first secondary endpoint is overall survival. Other endpoints include safety and the induction of immune responses. Historical control patients in the category of newly diagnosed GBM show a median time to progression of about 8-9 months with median survival of 15-16 months. It is expected that patient accrual will be completed over 9 months and that primary endpoint data will be completed at the 21 month time point. A biological license application is expected to be submitted shortly thereafter. Previous Phase I Clinical Trials Two Phase I clinical trials were carried out at UCLA by Dr. Linda Liau for patients with Glioblastoma multiforme (GBM). The second clinical trial is still ongoing, and focuses on newly diagnosed patients with GBM whose primary treatment included surgery, radiation and chemotherapy. Three injections of DCVax®-Brain were administered intradermally, or under the skin two weeks apart, followed for some patients with subsequent booster injections. Data from two Phase I clinical trials on DCVax®-Brain carried out at UCLA continue to mature: investigators continue to measure the delays in disease progression and the extensions of overall survival in DCVax®-Brain treated patients who survive. To date, the clinical data have shown a median survival of 33.8 months (and continuing) for DCVax®-Brain treated patients, with 9 of 19 patients still alive from 8-82 months after initial surgery. Most of these patients have no evidence of tumor recurrence. Four of these patients have survival times without progression or recurrence of their cancer that now extends beyond 45 months. 04/29/2014
Edotecarin Pfizer Phase 3 trials edotecarin is a topoisomerase-1 inhibitor this trial is closed. 09/27/2005
EM-1421 (tetra-O-methyl nordihydroguaiaretic acid) erimos Pharmaceuticals Phase 2 trials blocks cell cycle progression by inhibiting production of the cyclin dependent kinase cdc2 (p34, cdk-1), and to stimulate apoptosis by down-regulating the production and activation of survivin 03/28/2007
EMD 121974 Cilengitide Merck KGaA, Germany Phase 3 trials Angiogenesis Inhibitors - A potent selective inhibitor of the xß3 integrin receptor Cilengitide is a cyclic pentapeptide which has demonstrated inhibition of angiogenesis in a variety of models by a unique mechanism of action. Cilengitide targets anb3 and anb5 integrins that are crucial for endothelial cell proliferation. The vaccine was granted fast-track status in September 2004 by the FDA. 07/08/2007
Erlotinib Tarceva® Genentech Approved, but used off label Tarceva has been shown to target HER1/EGFR and inhibit Tyrosine Kinase (TK) phosphorylation. Tarceva's mechanism of clinical antitumor action has not been fully characterized. 09/27/2005
Etoposide VP-16 NOBEX Approved, but used off label A semisynthetic derivative of PODOPHYLLOTOXIN that exhibits antitumor activity. Etoposide inhibits DNA synthesis by forming a complex with topoisomerase II and DNA. This complex induces breaks in double stranded DNA and prevents repair by topoisomerase II binding. Accumulated breaks in DNA prevent entry into the mitotic phase of cell division, and lead to cell death. Etoposide acts primarily in the G2 and S phases of the cell cycle. Etoposide, although known to be active against several types of brain cancer, is not able to cross the blood-brain barrier. NOBEX has developed an etoposide that will cross the blood-brain barrier and have better solubility characteristics. 09/27/2005
G 207 MediGene AG Phase 1 trials MediGene AG commenced a phase I trial of the drug candidate G207 for the treatment of malignant glioma at the University of Alabama, Birmingham. This was reported several years ago. Any inquiry has been made of the company. 09/08/2009
Gefitinib (ZD1839) IRESSA ® AstraZeneca Approved, but used off label Epidermal growth factor receptor tyrosine kinase (EGFR-TK) inhibitor In phase 2 trials for GBMs. 09/27/2005
GRN163L Geron Corp. Phase 2 trials Telomerase Inhibitor Geron Corp: "We have designed and synthesized a special class of short-chain nucleic acid-like molecules, known as oligonucleotides, to target the template region, or active site, of telomerase. These oligonucleotides have demonstrated highly potent telomerase inhibitory activity at sub-nanomolar, or very low, concentrations in both biochemical assays and various cellular systems. Published research has shown that these template antagonists inhibit the growth of malignant human glioma cells in animals. In 2001, our development partner in Asia, Kyowa Hakko, selected our compound, GRN163, for development as a telomerase inhibitor for the treatment of cancer. GRN163 is a true enzyme inhibitor and can therefore be much smaller (lower molecular weight) than other oligonucleotide drug candidates. Also, it does not inhibit other critical nucleic acid-modifying enzymes, nor is it toxic to normal cells at concentrations needed to inhibit telomerase in tumor cells." 09/28/2005
Human Corticotropin-Releasing Factor Xerecept Neurobiological Technologies Phase 3 trials Reduces brain edema This does not treat the tumor - it treats swelling of the brain. It is being tested as a possible alternative to Decadron 12/04/2005
Human reovirus Reolysin® Oncolytics Biotech Inc. Phase 2 trials The virus infects and kills only tumor cells cells bearing an activated Ras pathway, and is harmless to normal cells. Because the reovirus kills cancer kills with an activated Ras pathway, REOLYSIN® is a potential therapeutic for up to two thirds of all human cancers, including, but not limited to, malignant glioma, pancreatic, colon and some lung cancers. 09/28/2005
ICT-107 ImmunoCellular Therapeutics Phase 1 trials Dendritic cells are critical facilitators of a T-cell response but are often not present in sufficient numbers and are often not aggressive enough against malignant tumors to permit an adequately potent immune response to fight cancer. Dendritic cell therapy generally involves harvesting dendritic cells from a patient, then culturing and processing them in a laboratory to produce more numerous and effective dendritic cells. In the laboratory, the dendritic cells are cultured with specific tumor antigens to enable the dendritic cells to recognize cancer cells as targets for attack. When the newly cultured dendritic cells are injected back into the patient, they seek out remaining tumor cells and signal the T cells to destroy them. IMUC recently completed a Phase I clinical trial of ICT-107, the company's dendritic cell-based vaccine. The trial enrolled 19 patients and was conducted at Cedars-Sinai Medical Center. It was reported in November 2008 that of the 19 patients enrolled, 17 patients were still alive with eight patients surviving at least one year after the surgery that preceded their vaccine treatment. Ten patients were evaluated for immune responses, and five of them had a significant immune response to at least one tumor-associated antigen. Patients demonstrating an immune response are exhibiting a trend toward longer overall survival. IMUC plans to present additional follow-up data on this trial at the 2009 American Society of Clinical Oncology (ASCO) Meeting. 09/08/2009
ICT-121 ImmunoCellular Therapeutics Pre-clinical ICT-121 is IMUC's cancer stem cell vaccine that consists of a peptide to stimulate a Cytotoxic T-Lymphocyte (CTL) response to CD-133, which is generally overexpressed on the cancer stem cells. It is designed as an off-the-shelf vaccine which may be applicable to multiple types of cancers overexpressing CD-133. IMUC will initially evaluate it in a Phase I clinical study for glioblastoma which the company expects to file an IND application for in the third quarter of 2009. The characterization of cancer stem cells from glioblastoma has provided an opportunity to study the etiology of this dreaded disease and to be engaged in the development of product candidates that would be able to target the cancer stem cells which are believed to be responsible for the initiation and maintenance of glioblastoma. 09/08/2009
ICT-121 ImmunoCellular Therapeutics Pre-clinical ICT-121 is IMUC's cancer stem cell vaccine that consists of a peptide to stimulate a Cytotoxic T-Lymphocyte (CTL) response to CD-133, which is generally overexpressed on the cancer stem cells. It is designed as an off-the-shelf vaccine which may be applicable to multiple types of cancers overexpressing CD-133. IMUC will initially evaluate it in a Phase I clinical study for glioblastoma which the company expects to file an IND application for in the third quarter of 2009. The characterization of cancer stem cells from glioblastoma has provided an opportunity to study the etiology of this dreaded disease and to be engaged in the development of product candidates that would be able to target the cancer stem cells which are believed to be responsible for the initiation and maintenance of glioblastoma. 09/08/2009
IL13-PE38QQR NeoPharm Inc. Unknown Check the home page of their website The Precise trial ended, and the results were just ok - but not good enough to continue development. The next generation of the drug will be in trials soon! 04/27/2008
Imatinib mesylate (STI571) Gleevec® Novartis Oncology Approved, but used off label This is the first tyrosine kinase inhibitor that is commercially available for clinical use. Gleevec selectively blocks cellular proliferation and induces apoptosis in cells harboring the Bcr-Abl tyrosine kinase. Gleevec also targets the tyrosine kinase activity of the receptors for platelet-derived growth factor (PDGF), stem cell factor (SCF), and Kit, and inhibits PDGF- and SCF-mediated cellular events. Gleevec as a treatment for brain tumors is in phase II clinical trials. 07/04/2007
INGN 201 ADVEXIN® gene therapy Introgen Therapeutics, Inc Phase 3 trials adenoviral p53 gene therapy SiBiono perform p53 gene therapy in China with goverment approval. Ref: Sue Pearson et al. China approves first gene therapy. Nature Biotechnology 22, 3 - 4 (2004). 07/08/2007
iodine-labelled metaiodobenzylguanidine Azedra Molecular Insight Pharmaceuticals Phase 2 trials Radiotherapeutic drug which binds selectively to molecular targets. It uses the tumor's norepinephrine uptake mechanism. Azedra is being tested in Phase 2 trials for the treatment of neuroendocrine tumors such as pheochromocytoma in adults and neuroblastoma in children. Azedra recognizes a unique molecular target expressed on the surface of certain neuroendocrine cancer cells, which enables the drug to be accumulated selectively by the tumor. Azedra, developed using Molecular Insight's proprietary Ultratrace technology, is designed to maximize the delivery of iobenguane I 131 molecules to targeted neuroendocrine tumors and to enable safe and effective treatment while minimizing the amount of non-radioactive iobenguane molecules administered to the patient. Azedra is based on iobenguane I 131, a radiotherapeutic currently marketed in Europe for treating several types of neuroendocrine cancers. Therapeutic iobenguane I 131 has not previously been approved for marketing in the United States. In 2006, Azedra was granted Fast Track designation and Orphan Drug status by FDA. Molecular Insight has completed a Phase 1 dosimetry study and a Phase 1 dose-finding study in patients with pheochromocytoma. A Phase 2a dose-ranging and efficacy clinical trial in children with high-risk neuroblastoma is underway and FDA's review of a second SPA (Special Protocol Assessment) for the pivotal trial supporting the pediatric neuroblastoma indication is in progress. 04/17/2009
Irinotecan CPT-11, CAMPTOSAR Pfizer Approved, but used off label Topoisomerase I inhibitor In trials now alone and in combination with Avastin for brain tumors. 03/29/2007
Irinotecan hydrochloride Camptosar® (CPT-11) Pfizer Approved, but used off label Topoisomerase I inhibitor Irinotecan, in conjunction with other chemo agents and/or radiation, is being used in phase II clinical trials. 09/28/2005
J-107088 Edotecarin Pharmacia Corporation Phase 3 trials J-107088 is a novel, non-camptothecin, DNA topoisomerase-1 inhibitor that belongs to a class of compounds known as indolocarbazole 09/28/2005
LAK Cell Phase 2 trials Immunotherapy 12/04/2005
Lomustine CeeNU, CCNU BMS Approved for brain tumors Alkylator and inhibitor of several enzymes Oral 09/28/2005
Mannitol B BRAUN Approved, but used off label Blood brain barrier disruption Not a treatment by itself- it is used to make other treatments work better 12/04/2005
Melphalan Alkeran® GlaxoSmithKline Approved, but used off label Alkylating agent Oral. FDA approved for multiple myeloma and ovarian cancer 12/04/2005
MLN608 N/A Millennium Pre-clinical a potent inhibitor of the tyrosine kinases associated with the platelet-derived growth factor receptor (PDGFR), c-KIT and FLT-3 09/28/2005
MPC-6827 Azixa Myriad Pharmaceuticals Phase 2 trials Azixa has a dual mode of action; it acts as a cytotoxin and a vascular disrupting agent (VDA). The disruption of the tumor vasculature results in acute ischemia followed by massive tumor cell death. Mechanism of action studies have shown that, in vitro, Azixa binds to the same or nearby site on ß tubulin as colchicine, and inhibits microtubule assembly. This inhibition of microtubule formation interferes with the transition through the G2/M phase of the cell cycle and leads to mitotic arrest and cell death. In addition, as a VDA, Azixa kills tumor cells by reducing the blood supply to the tumor. The disruption of the tumor vasculature results in acute ischemia followed by massive tumor cell death. Azixa is believed to selectively disrupt tumor vasculature and not established vasculature in healthy tissue by inhibiting the formation of microtubules. Tumors rely on microtubules to maintain the cytoskeletal structure of their new vasculature, whereas mature vascular endothelium of healthy tissue uses actin filaments to provide the needed structure. In mice, the pharmacokinetics of Azixa indicated that the drug distributed rapidly into the brain. The concentration of Azixa in brain tissue was approximately 14 times higher than the plasma concentration and, and at that level, it easily exceeds the concentration required to activate caspase and induce the cancer cell killing apoptosis process in cancer cells [1,3]. Importantly, Azixa was cleared from the brain at a rate similar to that observed from blood [3]. This suggests a special opportunity to study anti-tumor activity in patients with primary brain tumors and brain metastases that currently cannot be treated effectively with chemotherapy due to ineffective drug penetration into the brain. Azixa (MPC-6827) is currently being evaluated in two Phase 2 human clinical trials, one in patients with primary brain cancer and one in melanoma that has spread to the brain. 06/15/2009
NovoTTF-100A NovoTTF-100A NovoCure Ltd. Phase 2 trials Electronic device Interesting idea. Click HERE for details. and also search our website for the trial 10/06/2006
O6-Benzylguanine (06-bg) Alkylade® Keryx Biopharmeceuticals Phase 3 trials Suppresses the ability to repair DNA damaged by BCNU 12/03/2005
Oxaliplatin ELOXATIN Sanofi-aventis Approved, but used off label Inhibit dna replication Approved for colon and rectal cancer 12/04/2005
pazopanib Armala GlaxoSmithKline Phase 2 trials Oral multikinase angiogenesis inhibitor This drug is in clinical trials for various cancers. Phase III trials for renal cell cancer and sarcoma demonstrated positive effects according to an October, 2008 press release. The drug is also in Phase III trials in combination with Tykerb (and Her2 and EGFR dual kinase inhibitor) for inflammatory breast cancer. Phase II trials are being conducted for non-small cell lung cancer, ovarian cancer, metastatic breast cancer, and reportedly, glioma of unspecified type, although the latter could not be verified. A Phase I trial is being conducted for colorectal cancer. 04/17/2009
PCV Procarbazine, CCNU and Vincristine Unknown A combination of 3 drugs Check for an article about pcv. 12/04/2005
PEG-Intron Peginterferon alfa-2b Schering Corp Approved, but used off label Immunotherapy Approved for hepatitis 12/04/2005
Photodynamic therapy Unknown Application of light to sensitized tumor cells 12/03/2005
Polifeprosan 20 with carmustine implant Gliadel Wafer MGI Pharma Approved for brain tumors Implant slowly releases chemotherapy directly to the tumor For more details, click HERE 03/28/2007
Poly ICLC Hiltonol™ Oncovir, Inc Phase 2 trials Biologic therapy Poly-ICLC is a relatively nontoxic product. see for details 09/28/2005
Procarbazine Matulane Sigma Tau Approved, but used off label inhibits DNA, RNA and protein synthesis. Oral 09/28/2005
PTK787/ZK-222584 N/A Novartis Oncology Phase 3 trials It is a potent and selective inhibitor of VEGF receptor-2, with a weaker blocking effect on PDGF tyrosine kinase. This is a newly synthesized compound belonging to the aminophthalazine class. The drug interferes with VEGF-and PDGF-mediated angiogenesis in in vitro and in vivo models. When PTK787 is combined with irradiation, tumor growth of radiation-resistant p53-dysfunctional xenografts is significantly delayed 09/28/2005
Radiation Unknown Damages DNA so that tumor cells can no longer multiply Radiation is one of the mainstays of malignant brain tumor treatment. There are many forms. Check our video library for details. 12/03/2005
Radiation therapy system Gliasite Approved for brain tumors Implantable device that delivers radiation directly to tumor 12/04/2005
Radiosurgery Gamma Knife Elekta Approved for brain tumors Stereotactic radiosurgery This is one of many tools that can be used for stereotatci radiosurgery 12/04/2005
Radiosurgery Varian Trilogy Varian Medical Systems Approved for brain tumors 12/04/2005
Rsr-13 (efaproxiral sodium) N/A Allos Therapeutics Phase 3 trials Enhances the diffusion of oxygen to hypoxic (oxygen deprived) tumor tissues from hemoglobin, the oxygen carrying protein contained within red blood cells. The presence of oxygen in tumors is an essential element for the effectiveness of radiation therapy and some chemotherapy agents in the treatment of cancer. Phase 2 trials for GBM recently ended, and phase 3 trial for brain mets is ongoing. 09/28/2005
Ruta-6 Ruta-6 Unknown This is a homeopathic treatment. For details, click HERE 03/28/2007
SCH66336 Lonafarnib Schering-plough Corp. Phase 2 trials Farnesyl Transferase Inhibitor Trial is for the combination of SCH66336 and Temodar. for GBMs 09/28/2005
Sirolimus Rapamune® Wyeth Approved, but used off label It is approved for use in organ transplants to prevent rejection. It is in clinical trials for GBMs. 09/28/2005
Stem cell transplant Phase 2 trials Allows a higher dose of chemotherapy to be used This is not a treatment for the tumor (yet). It is used as a rescue to correct bone marrow destruction from high dose chemotherapy 12/03/2005
Stereotactic Radiosurgery Approved for brain tumors Precise targetting of radiation There are now many tools used for this procedure. Check our video library for details. 12/03/2005
SU5416 (semaxanib) N/A Sugen Phase 2 trials A synthetic reversible inhibitor of VEGF receptor-2 and PDGF receptors. This drug is in phase I trials for brain cancer 09/28/2005
Talampanel Phase 2 trials Talampanel is a highly selective, orally active antagonist of the AMPA (alpha-amino-3-hydroxy-5-methyl-4-isoxazolepropionic acid) receptor. 09/28/2005
Tamoxifen Nolvadex® AstraZeneca Approved, but used off label PKC inhibitor at high doses. 09/28/2005
Temozolomide Temodar® Schering Corporation Approved for brain tumors Alkylation (methylation) occurs mainly at the O6 and N7 positions of guanine. Oral. Temozolomide is not directly active but undergoes rapid nonenzymatic conversion at physiologic pH to the reactive compound MTIC. 09/28/2005
Thalidomide Thalomid® Celgene Approved, but used off label Anti-angiogenesis Thalidomide, in conjunction with other chemo agents, is in phase II clinical trials for treatment of brain bumors 09/28/2005
Tipifarnib (R115777, FTI) Zarnestra® Johnson & Johnson Phase 2 trials farnesyl transferase inhibitors Zarnestra is 1 of a group of drugs known as farnesyl transferase inhibitors (FTIs). FTIs inhibit an enzyme known as farnesyl transferase, which is needed for the activation of a growth-promoting gene called ras. FTIs inhibit the growth of various cancer cells. It is also a radiosensitizer, inducing postmitotic necrotic cell death of radioresistant glioma cell lines in vitro. 09/28/2005
TNT-Tumor necrosis therapy Cotara® Peregrine Pharmaceuticals Phase 3 trials monoclonal antibody to DNA from necrotic cells, which carries the radioactive isotope Iodine-131 09/28/2005
Topotecan HYCAMTIN Glaxo Approved, but used off label Topoisomerase I inhibitor Approved for lung and ovarian cancer 12/04/2005
Torisel Temsirolimus, CCI-779 Wyeth Approved, but used off label mTOR inhibitor Inhibits a kinase known as mTOR (mammalian target of rapamycin), an enzyme activated through the PI3K/Akt cascade. This blockade leads to cell arrest in G1. Rapamycin and its analogs are cyto-static against xenografts of glioblastoma, medulloblastoma, breast cancer, and prostate cancer. The mTOR inhibitor class of drugs include: sirolimus/rapamycin), temsirolimus/CCI-779, RAD-001 and AP23573. Rapamycin has long been used as an immunosupresor and off-lable as a cancer treatment. They are all in clinical development for cancer treatments. Among which temsirolimus is te most advanced. On May 30, 2007, the U. S. Food and Drug Administration granted approval for temsirolimus (Torisel™, made by Wyeth, Inc.) for the treatment of advanced renal cell carcinoma (RCC). 07/08/2007
TP-38 Toxin Phase 2 trials Immunotherapy TP-38 is a recombinant chimeric protein composed of the epidermal growth factor receptor (EGFR) binding ligand (TGF-á) and a genetically engineered form of the Pseudomonas exotoxin PE-38. It is administered directly to the tumor site by convection enhanced delivery. 12/04/2005
Trabedersen (AP 12009) Antisense Pharma GmbH Phase 3 trials Trabedersen is is a substance that prevents the production of a protein known as transforming growth factor beta 2 (TGF-ß2). The cells of advanced high-grade brain tumors produce an excessive amount of TGF-ß2, which acts as a shield, protecting the tumor from the body’s defense system (the immune system). This effectively weakens the immune system, resulting in accelerated growth and spreading of the tumor. Trabedersen helps to prevent this from happening by blocking the production of TGF-ß2, thereby breaking down the protective shield and allowing the immune system to attack and destroy the cancer cells. 08/25/2011
Transmid Xenova Group plc Phase 3 trials TransMIDTM is a modified diphtheria toxin conjugated to transferrin. Transferrin receptors are particularly prevalent on rapidly dividing cells, and the high level of transferrin receptor expression on glioma cells makes it an ideal target for brain cancer. The diphtheria toxin gains entry to the tumour cell when the transferrin to which it is attached binds to transferrin receptors on the surface of the tumour cell. Once inside a cell the diphtheria toxin interferes with protein synthesis which ultimately kills the cancer cell. 12/04/2005
tumor necrosis therapy Cotara Peregrine Pharmaceuticals Phase 1 trials Tumor Necrosis Therapy (TNT) uses monoclonal antibodies attracted to the dead and dying cells found at the core of tumors. When these antibodies are conjugated (attached) to therapeutic agents such as radioisotopes, they carry the anti-cancer agent into the tumors to kill them from the inside out. Cotara® is the company’s lead TNT compound. Peregrine is developing Cotara as a potential treatment for patients with high grade brain cancer. Tumor Necrosis Therapy (TNT) acts by binding to dead and dying cells found primarily at the necrotic core of a tumor. Most solid tumors develop a core of dead or dying (necrotic) cells in the center of the tumor mass as it grows. The outer membranes of necrotic cancer cell become leaky, which exposes the DNA making it an abundant but selective target. The DNA target may also be more stable and reliable than conventional targets, since it is not believed to modulate as is commonly seen with tumor-specific cell surface antigens that are used with other antibody-based therapeutic modalities. Because it is attracted to necrotic regions throughout the tumor, TNT can deliver a toxic payload to neighboring viable cancer cells, resulting in their death. TNT antibodies are potentially capable of carrying a variety of therapeutics agents into the interior of solid tumors including radioisotopes and chemotherapeutic agents. Peregrine’s first TNT-based product, Cotara, is an antibody conjugated to Iodine 131, a therapeutic radioisotope. The radioisotope kills adjacent cancer cells and has an additional advantage over other therapeutics, since it can be tracked by scanning devices to be sure it is entering the tumor and not dispersing in healthy tissues. Cotara’s conjugated isotope has an additional advantage over other therapeutics. As demonstrated by SPECT analysis of the cranium of a patient suffering from glioblastoma multiforme, Cotara can be easily tracked by scanning devices to be sure it is entering the tumor and is not dispersing in healthy tissues. Another potential advantage is that each successive treatment with TNT potentially kills more cancer cells, expanding the necrotic area of the tumor, thus becoming more effective upon subsequent doses, contrary to conventional chemotherapy, which may lose its therapeutic effect due to increased drug resistance. The TNT targeting mechanism could be the basis for an entire class of new products effective across a wide-range of solid tumor types, including brain, lung, colon, breast, liver, prostate and pancreatic cancers. Cotara® is an experimental new treatment that links a radioactive isotope (iodine 131) to a targeted monoclonal antibody. This monoclonal antibody is designed to bind tumor cells and deliver radiation directly to the center of the tumor mass while minimizing effects on normal tissues. Cotara® thus destroys the tumor "from the inside out." This may be an effective treatment for glioblastoma multiforme. A Phase I clinical trial designed to further evaluate the safety and radiation distribution of Cotara in patients with recurrent glioblastoma multiforme (GBM) is currently recruiting. Locations: Arizona Barrow Neurological Institute Phoenix, Arizona, 85013 Contact: Andrea Ralph, RN OCN 602-406-6262 Principal Investigator: William R Shapiro, MD Ohio University Hospitals Case Medical Center Cleveland, Ohio, 44106 Contact: Dawn M Diorio, R.N., B.S.N., B.A. 216-983-5167 Principal Investigator: Andrew E Sloan, MD, FACS Pennsylvania University of Pennsylvania Philadelphia, Pennsylvania, United States, 19104 Contact: Joanna Lopinto, BSN, RN 215-615-4590 Principal Investigator: Kevin Judy, MD South Carolina Medical University of South Carolina Charleston, South Carolina, United States, 29425 Contact: Bonnie Muntz-Pope 843-792-8967 Principal Investigator: Sunil J Patel, MD Principal Investigator: Kenneth Spicer, MD A phase II clinical trial evaluating the safety and efficacy of Cotara in patients with GBM at first relapse is currently recruiting in India. 09/08/2009
Tumor Treating Fields NovoTTF-100A NovoCure Phase 3 trials Noninvasive Tumor Treating Field Click HERE to view a video of what this treatment is and how it works. 03/28/2007
Vincristine Oncovin, Vincasar Lilly Approved, but used off label Inhibits cell division 09/28/2005
Whole Brain Radiation Unknown Stops cells from dividing Used to be standard treatment for gbms. Now mostly used only when there are numerous or large tumors 12/04/2005

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