Targeted Pills: Scientists Bypass Drug Side-Effects With Smart Delivery System

Despite great advances in therapeutic drugs, unwanted side effects remain a serious obstacle to disease treatment. Most adverse side effects result from the drug reaching parts of the patient’s body that are not relevant to the drug’s medicinal action.

A team of scientists at Ben Gurion University developed an innovative method of addressing this: a delivery system that increases the availability of medications at target locations and reduces their side effects and toxicity. The technology, named V-Smart, is of particular interest in developing treatments of diseases that affect the central nervous system, as well as brain malignancies.

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V-Smart employs bubble-like microscopic structures known as vesicles, which are useful in carrying proteins and other molecules across cell membranes.

The scientists demonstrated in animal studies that V-Smart vesicles which are injected or consumed orally can deliver encapsulated materials into the brain and achieve localized results. This means that drugs can be administered to the brain and have their desired treating/pain-killing effect.

Reducing side effects 

The V-Smart technology is based on nano-sized vesicles that are highly stable and provide a controlled release mechanism which allows the drug to pass through biological barriers, including the blood brain barrier (BBB).

There are some ways to traverse the BBB, such as by injecting directly into the brain with a needle, but even then the challenge remains of reaching the exact part of the brain that needs treatment. V-Smart’s technology pinpoints where the drug will be released in the brain, increasing efficiency and reducing adverse effects.

“Our goal is to transport encapsulated small molecules, peptides, proteins and nucleic acids, across the BBB and release them in the brain where their activity is needed,” explains Professor Eliahu Heldman, a researcher from the Department of Clinical Biochemistry.

BGN Technologies, the technology transfer company for BGU, signed a licensing agreement for V-Smart with Lauren Sciences LLC, a privately held biotechnology company in New York. Lauren Sciences is developing the technology as a platform for treating a variety of diseases that affect the central nervous system. Such diseases include Parkinson’s, Alzheimer’s, Amyotrophic Lateral Sclerosis (ALS), as well as brain malignancies.

Lauren Sciences and BGU were recently awarded two prestigious research grants. One grant is from the Michael J. Fox Foundation and is meant to fund  the development of a V-Smart for the delivery of proteins to treat Parkinson’s disease. The other grant is from the Campbell Foundation and is for the delivery of the antiretroviral drug Tenofovir to treat neuro-HIV.

The BGU team behind the research includes Dr. Sarina Grinberg of the Department of Chemistry, Dr. Charles Linder of the Avram and Stella Goldstein-Goren Department of Biotechnology Engineering, and Professor Emeritus Eliahu Heldman of the Department of Clinical Biochemistry.

“We hope that the success of these projects will improve these patients’ lives,” concludes Professor Heldman, who also serves as Chief Scientific Officer of Lauren Sciences.