SAN FRANCISCO, July 20, 2016 – Scientists at Shasqi, Inc. and SUNY University at Albany have published research demonstrating a way to target drugs to specific areas of the body, enabling greater efficacy and fewer side-effects. The method, called local drug activation, uses activating agents in an injected polymer gel to concentrate and activate prodrug versions of a drug, such as a chemotherapeutic, directly at a desired location, limiting the drug’s action to that specific site.
The researchers recently published their findings online in ACS Central Science, the open-access flagship journal of the American Chemical Society.
Their research shows that the local drug activation technology can deliver the chemotherapeutic drug doxorubicin directly to cancerous sarcoma cells in mice, eliminating the tumors while reducing the undesirable side-effects commonly associated with chemotherapy. Moreover, while both traditionally delivered doxorubicin and the new delivery approach had an initial anti-cancer effect on the mice’s sarcoma tumors, only the site-activated drug kept the cancer from returning. Sarcoma is an aggressive form of cancer that’s responsible for up to 20 percent of childhood cancers. Surgery is often the primary treatment because traditional methods of chemotherapy are ineffective.
“During medical training it was frustrating to know that more than 97% of the medication that we give to patients never reaches its intended target. Whether the drug is ibuprofen or a chemotherapeutic, most of the medication is wasted, leading to side-effects,” said José M. Mejía Oneto, M.D., Ph.D., President and Chief Executive Officer of Shasqi, lead author of the new publication. “We believe that better methods of delivering drugs in the body will greatly improve the treatment of conditions where the desired site of action is well known by the physician and patient. This new research validates our approach in a challenging medical area of significant unmet need.”
In addition to the greater efficacy and lack of tumor recurrence, mice treated using the local activation strategy had fewer side-effects that can limit the use of combinations of cancer drugs. In particular, the researchers did not observe a decrease of new red blood cells, a marker of bone marrow suppression, which limits the tolerable dose of doxorubicin in patients. Other unpleasant side-effects, such as weight loss, were not observed compared to those mice treated conventionally. The researchers plan to investigate whether shorter courses of therapy using higher doses of the chemotherapeutic can be even more effective and to expand this approach to other drugs and types of tumors.
“This drug targeting method is independent of molecular markers and allows drugs to be activated at a specific location for multiple weeks after the initial preimplantation of the gel at the desired treatment site,” commented Dr. Mejía Oneto. “Because it protects healthy cells, including immune system cells, this approach may allow the concurrent use of multiple cytotoxic drugs or immunotherapies.”
