A team of scientists, including one of Indian origin, has designed a "nanovehicle" that could drive drugs straight to their destinations - the diseased tissues - thereby sparing other healthy organs from any unintended effects, with the side-effects of therapy being one of the major stumbling blocks in advancing treatment for deadly diseases.
Made of gold nanoparticles, the nanovehicle was found to be non-toxic when tested in cultured human cell lines, study co-author Arnab De of the Chicago-based AbbVie Bioresearch Centre said in an e-mail interview.
"Drugs should ideally act only on diseased tissues. However, it is difficult to deliver drugs only to diseased tissues without affecting other healthy tissues. The side effects of drugs are often seen on these healthy tissues," De, a PhD from Columbia University in the City of New York, explained.
"This is a proof-of-concept study that shows that it may be possible to selectively navigate a drug to certain tissues. However, extensive animal studies need to be done to determine non-specific targeting in animals," noted De, who collaborated for the research with a team of scientists from the University of Delhi.
The research was conducted at professor Subho Mozumdar's laboratory at the University of Delhi.
Other co-authors of the study were Tanusri Nandi and Seema Garg, a student in Mozumdar's lab who played an important role in synthesizing the nanoparticles.
"The beauty of the system is that it could in principle target drugs specifically to diseased tissues. While we have shown that we can target the liver, other tissues could potentially be targeted as well," De pointed out.
"At this point though, it is too early to talk in terms of specific diseases," he said.
To design this nanovehicle, Mozumdar, an associate professor of chemistry at the University of Delhi, said the researchers "simultaneously modified the surface of the nanovehicle with 'drugs' and 'tissue-specific molecules' to target the nanovehicle to the diseased tissue".
"We have successfully used the nanovehicle to deliver a model drug to the liver," Mozumdar said.
Appearing in the journal AAPS PharmSciTech, published by the American Association of Pharmaceutical Scientists, the study revealed that "the nanovehicle specifically targets the diseased tissue, thereby reducing the side effects of the drug". Another benefit of this is that the "therapeutic efficacy of the drug is substantially increased as the entire drug is delivered to the diseased tissue alone".
Highlighting the significance of the finding, Omid Farokhzad, professor at Harvard Medical School, said: "Targeting the drug molecule specifically to the diseased tissue without affecting the healthy tissues remains a formidable challenge. Targeted and responsive nanotechnology platforms such as those developed in this study can in principle achieve that goal in an elegant manner."
According to the study, "the estimated cost of synthesizing the nanovehicle is only about $1 per mg, a very cost-effective solution."
And the researchers believe that once the efficacy and safety of the technology are established in animal studies and human trials, the technology could be available in clinics within a decade.
"Mozumdar and I have plans to launch a startup company to advance such goals in the future," De said. "With opportunities opening up every day in India, I hope our dream will become a reality," De noted.