Biochemists everywhere are busy searching for better technologies to deliver therapeutic drugs to body cells that need them. Problem #1: Even the most advanced pharmaceutical can cause adverse side effects when it comes in contact with surrounding healthy tissue. Problem #2: getting medication from the bloodstream into the brain is a near impossibility because of the body’s protective blood-brain barrier (BBB).
At Israel’s Ben-Gurion University of the Negev, chemist Sarina Grinberg, biotech engineer Charles Linder and clinical biochemist Eli Heldman have licensed a system that increases the availability of medications at intended locations and reduces their side effects and toxicity elsewhere in the body.
The trademarked V-Smart technology holds particular promise in finding treatments for diseases that affect the central nervous system, including Parkinson's and Alzheimer's diseases and amyotrophic lateral sclerosis (ALS). Perhaps even more significantly, because its controlled-release mechanism allows the drug to pass through the blood brain barrier (BBB), the system could also help get chemotherapy to malignant brain tumors.
The revolutionary Israeli V-Smart system does the job by encapsulating the medication in vesicles -- highly stable, microscopic bubble-like structures. Delivered by mouth or injection, drugs sent on their way using this technology have so far proven quite effective in animal studies.
"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," says Heldman, a retired professor who has worked on this project for the past eight years.
He said that getting beyond the BBB was one of his team’s major incentives. ”Vesicles can also be used for delivering drugs to organs outside the brain, but our main challenge was to make them cross the BBB.”
Headed for commercialization
The university’s technology transfer company, BGN Technologies, signed a licensing agreement for V-Smart with New York-based Lauren Sciences, a privately held biotechnology company that will fund further development of the system at Ben-Gurion. As part of the agreement, Heldman was named chief scientific officer of Lauren Sciences.
“We’ve done proof-of-concept in mice and now we are developing V-Smart for specific diseases, mainly Parkinson’s, where we want to deliver a certain protein to the brain not only to alleviate symptoms but also to improve the condition by repairing some of the damaged cells,” he says. “This protein can be directly injected into the brain but it’s quite invasive, and delivering it through vesicles would be much better.”
Both the US company and the Israeli university received research grants from the Michael J. Fox Foundation to develop a V-Smart specifically for the delivery of proteins to treat Parkinson's disease; and from the Campbell Foundation for the delivery of the antiretroviral drug Tenofovir for treatment of neuro-HIV.
“The second disease we’re focusing on right now is neuro-HIV. HIV is the virus that causes AIDS. Today there is a ‘cocktail’ treatment of drugs to manage the virus in organs outside the brain. However, at least one of the very important components of the cocktail doesn’t penetrate the BBB, so the virus remains unharmed in the central nervous system,” he explains.
“We worked on the project for the past eight years and recently made significant progress. We’re dedicated to it,” says Heldman.