Rapid Access to Intervention Development Program provides critical resources for drug development 

February 23, 2006 

WORCESTER, Mass.- Dario C. Altieri, MD, UMMS professor and chair of cancer biology and Director of the UMass Cancer Center, has received an exclusive Rapid Access to Intervention Development (RAID) Award from the National Cancer Institute for the clinical development of shepherdin, a novel molecular anti-cancer agent that he developed. Testament to the program's rigorous review process and the promise of shepherdin, Dr. Altieri's was the sole accepted proposal in the current project cycle. A promising new approach to cancer therapy, shepherdin selectively kills tumor cells while sparing normal cells nearby.

A unique and valuable resource that is open to academic institutions and other non-profit research organizations, the National Cancer Institute's (NCI) Rapid Access to Interventional Development program aims to support the rapid movement of novel molecules and concepts from the laboratory to the clinic for proof-of-principle clinical trials.  Simply defined, a RAID allows the NCI to perform all of the preclinical development tasks needed for an Investigational New Drug (IND) at no cost to the university.  Notably, RAID does not award direct funding to the principal investigator but rather assists those investigators who submit successful applications by providing any or all of the preclinical development steps that may be obstacles to clinical translation. Suitable agents for RAID will include small molecules, biologics, or vaccines and possible tasks may include production, bulk supply, good manufacturing process (GMP) manufacturing, formulation and toxicology.

"We believe that shepherdin is a very effective lead prodrug that can seek out and destroy only the malfunctioning, cancerous cells, leaving normal, healthy cells unaffected," said Altieri. "With this critical support from the National Cancer Institute, we can aggressively work to move shepherdin from the bench to the bedside for the benefit of cancer patients around the world."

In the initial stages of the shepherdin studies, RAID resources will be used to establish analytical assays, in vitro stability and formulation studies. Upon completion of this work, additional support will be given for dose, efficacy and safety studies.

In 1997, Altieri discovered the protein survivin, which is overabundant in cancer cells and protects the cells from death, allowing for unchecked proliferation and tumor growth. Based on his work with survivin and previous studies of another protein-heat shock protein 90 or Hsp90-which "chaperones" survivin to ensure its durability and its delivery into cells, Altieri sought a way to interfere with the function of these two particular proteins.  He engineered a compound called shepherdin, which binds to Hsp90 and blocks its ability to work with survivin.

In human cancer cell cultures and in mice harboring human cancer types, shepherdin binds to Hsp90 and impairs survival mechanisms of tumor cells, resulting in massive destruction of tumor cells and substantial inhibition of tumor growth in mice.  The anti-tumor activity of shepherdin is selective in that normal tissues and organs are not affected, and prolonged use of shepherdin in mice is well tolerated with no signs of toxicity.  In the studies published in 2005, shepherdin was active on a wide variety of types of cancers, regardless of their origin or genetic makeup.  It is because of these combined features that shepherdin makes an attractive lead drug for targeted therapy of many human tumors.

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Contact: Kelly Bishop, 508-856-2000