Clinical Trial Drugs

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This page provides information on drugs in various stages of clinical trials. If you have any information that you would like to share with the community, please add your experiences below.

Androgen Blockade

Anti-IGF-IR (Insulin-Like Growth Factors)

CP-751,871 - study of anti-IGF-IR CP-751,871 in patients with solid tumors currently enrolling adult patients with rabdomyosarcoma and Ewing's family of tumors.

Anti-Angiogenesis Treatment


Etoposide, Cyclophosphamide, Thalidomide, Celecoxib, and Fenofibrate in Treating Young Patients With Relapsed or Progressive Cancer

Targeted therapy


Intravenous Isophosporamide Mustard (ZIO-201)

EKB-569 and CCI-779 - Temsirolimus

Ecteinascidin 743 - Trabectedin (ET-743, Yondelis®)

c-Kit VEGF PDGF Inhibitors

Imatinib Mesylate


VEGF=Vascular Endothelial Growth Factor

PDGF-R=Platelet Derived Growth Factor Receptor

C-KIT = c-Kit antibody (DSRCT tumors generally show c-Kit negative)

Enzyme Inhibitor Therapy

EKB-569 and CCI-779 - Temsirolimus

Histone Deacetylase Inhibitor

HSP90 (Heat Shock Protein 90) Inhibitors

IPI-504 - Safety Study of IPI-504 in Patients With Gastrointestinal Stromal Tumors (GIST) or Soft Tissue Sarcomas (STS)


mTOR Inhibitors

AP23573 Rapamycin Derivative

A Study of the mTOR Inhibitor Rapamycin (Rapamune, Sirolimus) in Combination With Abraxane in Advanced Solid Cancers

Protein Tyrosine Kinase Inhibitor (PTK)

Some of these drugs may be FDA approved and are available to patients to use 'off trial' if warranted. Newer drugs may also be obtained through a process called 'compassionate usage' and would skip clinical trial, again if warranted.

Sunitinib (Sutent)




EKB-569 and CCI-779 - Temsirolimus

PPAR Agonist

CS-7017 - Study of an Experimental New Drug, PPAR Agonist Taken by Mouth by Patients With Advanced or Metastatic Cancer

Radiolabeled Monoclonal Antibodies

Iodine I 131 Monoclonal Antibody 3F8

Inthrathecal Radioimmunotherapy Using I-8H9

A Phase I, Open-Label, Dose Finding Study Evaluating the Safety and Pharmacokinetics of AMG 479 in Subjects with Advanced Solid Tumors


anti-IGF-IR = Insulin-like growth factor Type 1

Combination Drugs

Rapamycin and CP-751871 -

Memphis researchers are in San Francisco this week to talk about a new strategy for tricking cancer cells into self-destructing.

The approach combines a drug already used to protect organ transplant patients with a synthetic antibody to combat sarcomas, a family of cancers targeting bone and soft tissue. Today St. Jude Children's Research Hospital investigators will present evidence the cocktail shrank such tumors in mice.

"This provides the first glimmer of a rational approach to treating" patients with advanced cases of Ewing's sarcoma, Dr. Peter Houghton explained. The combination appears to disrupt the biochemical pathways necessary for the continued growth that is a hallmark of cancer. As a result, in the laboratory and in mice, the cancer stops growing or dies.

Plans to test the combination in patients with Ewing's sarcoma are now being developed, Houghton said. He is chairman of the St. Jude Children's Research Hospital Department of Molecular Pharmacology and the senior author of the work being discussed this week.

The cancer is diagnosed in about 200 Americans annually, about half of whom are ages 10 to 20. About 65 percent of patients are now long-term survivors. It falls to about 30 percent for patients whose cancer has spread throughout their bodies, Houghton said.

The results build on nearly a decade of work by scientists in Houghton's laboratory. The study is one of the featured presentations at this week's American Association for Cancer Research's International Conference on Molecular Targets and Cancer Therapeutics.

Although the Memphis scientists have focused on Ewing's sarcoma, Houghton said the approach has potential for treating many other cancers. The drug combination disrupts a pathway associated with the p53 gene, which plays a crucial role in DNA repair. The p53 gene is damaged in more than half of human cancers.

The work involves the drug rapamycin and CP-751871, a protein engineered to target a specific cell type. Rapamycin is used to protect organ transplant patients and to battle kidney cancer.

The St. Jude scientists are apparently the first to combine the drug and protein against cancer.

Houghton said about six years ago scientists in his laboratory observed that in certain circumstances rapamycin triggered cell death.

But Ewing's and other sarcoma tumor cells produced proteins that functioned as growth factors and blocked cell death.

Turns out the antibody CP-751871 blocks the growth factor in Ewing's sarcoma. "The result is quite dramatic," Houghton said. The treatment specifically targets cancer cells, sparing healthy cells and reducing side effects, he added.

A handful of other antibodies are being studied that block the same biochemical pathway.

Both rapamycin and the antibody are also being tried in adults with advanced cases of breast, prostate, lung and several other cancers.

Other Treatment

Continuous Hyperthermic Peritoneal Perfusion

Stem Cell Transplantation

Clostridium novyi-NT (C. novyi–NT) spores -This study will include a one time intravenous (IV) infusion of Clostridium novyi-NT (C. novyi–NT) spores to treat solid tumors which have not responded to standard therapy.

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Disclaimer: The information of this page is not intended to be an endorsement for any clinical trial, drug, or treatment method. Please check with your health care professional about whether these clinical trials are beneficial in your own situation.

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