Hong Kong Baptist UniversityCuring brittle bone disease

Curing brittle bone disease

Researchers from Hong Kong Baptist University (HKBU) have successfully developed a novel aptamer for the treatment of osteogenesis imperfecta (OI) with the aid of artificial intelligence (AI) technology. It is the first time a drug in Hong Kong has been granted orphan drug designation by the US Food and Drug Administration (FDA).

OI, which is also known as brittle bone disease, is a rare hereditary bone disease with no effective drugs for treatment. Genetic studies have demonstrated that inhibition of sclerostin, a protein that regulates bone formation, can improve the clinical presentation of OI.

Professor Zhang Ge and Professor Lyu Aiping of the School of Chinese Medicine led a research team to develop a new approach to treating the disease. The team strategically screened and optimised a new class of molecule-nucleic acid aptamers against the sclerostin protein, which significantly promotes bone formation in mice with OI. In addition, they found that it does not affect the cardiovascular protection function of sclerostin and hence will not increase cardiovascular risk, a major benefit over monoclonal antibody treatments.

Aptamers are single-stranded DNA or RNA molecules which are regarded as an alternative to antibodies. However, identifying the most optimal molecule for the treatment of OI is a time-consuming process.

Professor Zhang Ge

The HKBU team succeeded in substantially raising screening efficiency by using a microfluidic system designed by the researchers to screen the aptamers. After screening out tens of thousands of aptamers against sclerostin using the microfluidic system, the team then applied AI technology to further identify the optimal molecules.

“AI technology saves manpower, shortens screening time, and also reduces reagent consumption. It also avoids the loss of potentially promising candidates which happens frequently when using traditional analytical methods,” said Professor Zhang.

The research team expects that clinical trials can be conducted in three years’ time, at the earliest. “We will take receiving the FDA orphan drug designation as an opportunity to further strengthen our technological edge, and develop a more effective treatment strategy for OI in order to benefit patients and society,” said Professor Lyu.

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