One of the biggest challenges in treating disorders of the brain is something which otherwise constitutes its greatest protection: the blood-brain barrier. A thin membrane which normally ensures that the body’s most complex organ stays protected from stray chemicals, infections, and other harmful substances in the blood, the barrier also stops medicines from getting to the cells where they are so badly needed – presenting a significant obstacle to the treatment of neurodegenerative diseases such as Alzheimer’s, Parkinson’s, and multiple sclerosis.

But Israeli scientists have found a way around the problem. Research by Prof. Daphne Atlas, professor of neurochemistry at the Hebrew University of Jerusalem, working with Dr. Daniel Offen from Tel Aviv University and Prof. Eldad Melamed, chairman of the department of neurology at the Rabin Medical Center in Petah Tikva, has led to the development of a molecule small enough to pass through the brain’s defenses – and it could make the treatment of brain-based diseases as simple as popping a pill.

“In our aging society there is a growing need for safe and effective drugs for age-related diseases,” Professor Atlas said. “AD4, which overcomes the blood-brain barrier, is an excellent candidate for both the prevention and treatment of various neurodegenerative disorders.”

The molecule, N-acetylcysteine amide (AD4), is an antioxidant that works by preventing and reversing the effects of oxidative stress, which is induced by harmful free radicals. Oxidative stress plays an important role in the progression of neurodegenerative and age-related diseases, causing damage to proteins, DNA, proteins and fat molecules in the cells.

Increasing evidence suggests, for example, that Parkinson’s Disease is connected to the accumulation of oxidative damage in specific neurons in the brain.

Pre-clinical data showed the ability of AD4, which can be administered orally, to protect cells in culture from oxidative damage, while in rodent trials, it was shown to protect neuronal cells from damage by both Parkinson’s disease and multiple sclerosis. The molecule has low toxicity and gets right to the brain cells where it is needed, the researchers found.

Thanks to the efforts of the scientists, Yissum – the Hebrew University’s technology transfer arm – last week signed a worldwide licensing agreement with Australian pharmaceutical company Eucalyptus to produce and market the molecule commercially.

Under the terms of the agreement, Eucalptus has acquired exclusive worldwide rights to develop and commercialize the molecule.

“This invention… is a breakthrough in the treatment of oxidative stress, which plays a major role in CNS disorders,” said Nava Swersky Sofer, CEO of Yissum. “We are delighted… to take our invention into the clinic for the benefit of patients.”

Professor Ashley Bush, the chief scientific officer of Eucalyptus and a world expert on Alzheimer’s disease, said he was confident that the collaboration would prove therapeutically and commercially successful in the near future. “I expect this to be a rapid development project,” he said.

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