BOSTON – October 21, 2008 – Vladimir Torchilin, Ph.D., pro­fessor and chair of the depart­ment of phar­ma­ceu­tical sci­ences at North­eastern University’s Bouvé Col­lege of Health Sci­ences, was recently awarded a 5-​​year, $1.54 mil­lion grant from the National Insti­tutes of Health’s (NIH) National Cancer Insti­tute to inves­ti­gate ways to increase the effi­cacy of nanocarrier-​​based phar­ma­ceu­ti­cals for drug and gene therapy. Many phar­ma­ceu­tical agents, such as drugs or DNA, need to enter the cell to carry out their ther­a­peutic prop­er­ties, and this study will address the chal­lenges of get­ting these agents into the cell and reaching the target once inside.

(Image: Elec­tron micro­graph of lipo­somal nanocar­riers size from 100 to 500 nm)

Torchilin, the study’s prin­cipal inves­ti­gator, is the director of the Center for Phar­ma­ceu­tical Biotech­nology and Nanomed­i­cine (CPBN). His research exper­tise is in the area of drug delivery sys­tems using nan­otech­nology as a way to treat cancer and other diseases.

Nanomed­i­cine, the med­ical appli­ca­tion of nan­otech­nology, holds tremen­dous promise as a way to improve drug delivery sys­tems. Phar­ma­ceu­tical sci­en­tists are cre­ating tar­geted drug delivery sys­tems using nanopar­ti­cles that will allow the drugs or DNA to enter the cell and reach the intended target and exert their ther­a­peutic func­tion. The suc­cess of these agents once in the cell, how­ever, is com­pro­mised because of cel­lular activ­i­ties and func­tions, such as lyso­somal degra­da­tion, which can alter the drug or the DNA and can make them ineffective.

The main objec­tives of this research project include devel­oping the process of get­ting these phar­ma­ceu­tical agents into the cell, iden­ti­fying the target once inside the cell and pro­tecting the integrity and activity of the drug or the DNA. Devel­oping a plat­form for the cre­ation of drug delivery sys­tems capable of intra­cel­lular pen­e­tra­tion, bypassing the cell wall and mem­brane, and tar­geting spe­cific organelles, such as the mito­chon­dria or lyso­somes, will improve both the delivery of drugs and DNA and ther­a­peutic out­comes for dis­eases such as cancer, lyso­somal storage dis­eases, and dis­eases asso­ci­ated with genome dysfunctions.

The ulti­mate goal of this research is to solve the extremely impor­tant and chal­lenging problem of intra­cel­lular organelle-​​specific drug delivery, which will dra­mat­i­cally increase the effi­cacy of many ther­a­pies,” said Torchilin.

Tar­geted drug delivery sys­tems include gene therapy, inserting new genes into a cell to replace mutated genes, and anti­sense therapy, where a gene known to cause a dis­ease is “turned off.” For these sys­tems, the genes are specif­i­cally tar­geted to carry out their ther­a­peutic func­tion in the nucleus, also located inside the cell. In addi­tion, such delivery sys­tems are suit­able for anti­cancer pro-​​apoptotic drugs that target mito­chon­dria, and lyso­somal enzymes that need to reach the lyso­somal com­part­ment inside the cell.

The research team will pre­pare phar­ma­ceu­tical nanocar­riers, such as lipo­somes or micelles, that will enve­lope and pro­tect the drug. They will also attach lig­ands to the out­side of the nanocar­rier that will assist in crossing the cell mem­brane and then binding to the target inside the cell. They will examine the inter­ac­tions of these nanocar­riers inside normal and cancer cells to see if they reach their target, such as nuclei or other intra­cel­lular organelles, and if they func­tion cor­rectly and improve the effi­ciency of the drug.

We expect this research will lead to the devel­op­ment of a novel plat­form for tar­geted drug and gene delivery within cells and for pro­voking the effec­tive apop­tosis of cancer cells” added Torchilin. “Our hope is that this plat­form can be used to treat as many dis­eases as possible.”

For more infor­ma­tion about Pro­fessor Torchilin’s research, please visit http://​www​.pharmsci​.neu​.edu/​f​a​c​u​l​t​y​/​t​o​r​c​h​i​l​i​n​.​h​tml or con­tact Jenny Eriksen at (617) 373‑2802 or via email at j.​eriksen@​neu.​edu.

About North­eastern

Founded in 1898, North­eastern Uni­ver­sity is a pri­vate research uni­ver­sity located in the heart of Boston. North­eastern is a leader in inter­dis­ci­pli­nary research, urban engage­ment, and the inte­gra­tion of class­room learning with real-​​world expe­ri­ence. The university’s dis­tinc­tive coop­er­a­tive edu­ca­tion pro­gram, where stu­dents alter­nate semes­ters of full-​​time study with semes­ters of paid work in fields rel­e­vant to their pro­fes­sional inter­ests and major, is one of the largest and most inno­v­a­tive in the world. The Uni­ver­sity offers a com­pre­hen­sive range of under­grad­uate and grad­uate pro­grams leading to degrees through the doc­torate in six under­grad­uate col­leges, eight grad­uate schools, and two part-​​time divi­sions. For more infor­ma­tion, please visit www​.north​eastern​.edu.