To address the need for effective treatment for rare diseases without using large population data, researchers from the Institute for Digital Medicine (WisDM) at the Yong Loo Lin School of Medicine, National University of Singapore (NUS Medicine) leveraged small amounts of data from a single patient with a rare disease to guide his treatment, with promising results. Co-led by Professor Dean Ho, Director of WisDM at NUS Medicine, the team conducted a clinical trial for the patient who was diagnosed with Waldenström macroglobulinemia—a rare blood disorder affecting approximately three in 1,000,000 people annually—by leveraging an artificial intelligence (AI) derived platform, CURATE.AI. Unlike traditional AI models that depend on large datasets, CURATE.AI utilises small data to dynamically adjust treatment dosages based on individual patient responses. Since the trial began in October 2021, substantial improvement in this patient’s red blood cell levels was observed, and the patient was able to avoid blood transfusions. Importantly, the patient did not suffer serious side effects from the treatment and hospital admissions were minimised.

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