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New breakthrough in Dementia treatment! Academia Sinica develops adenosine-balancing drug to combat neurodegenerationJun 12, 2024

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Academia Sinica's Biomedical Translation Research Center, in collaboration with Professor Chun-Jung Lin from the National Taiwan University's Department of Pharmacy, has made a significant breakthrough in Alzheimer's disease treatment. Their newly developed drug, J4, has shown promising results in mouse models, potentially offering a new treatment option by regulating adenosine balance in the brain to address neural energy deficits and slow disease progression. 

Regulating brain adenosine balance to improve cognitive function and spatial memory in mice.

Alzheimer's disease (AD) is characterized by a variety of complex pathological mechanisms, with brain energy deficiency being a prominent feature. J4 is an equilibrative nucleoside transporter 1 (ENT1) inhibitor that helps regulate brain adenosine balance, thereby improving neural energy deficits and protecting neurons from degeneration and damage. Preclinical studies have confirmed that J4 exhibits excellent efficacy across various AD mouse models, improving cognitive function and spatial memory, reducing abnormal aggregates of amyloid-beta (Aβ) and tau proteins, alleviating oxidative stress and neuroinflammation, and enhancing mitochondrial and glucose metabolic activity. 

Three key highlights of J4: Unique mechanism, superior penetration, and convenient oral administration

Professor Lin's team highlights the following aspects of the novel Alzheimer's drug J4: 

  • Unique Mechanism: J4 is the first drug on the market that targets neural energy deficits by regulating brain adenosine balance, thus protecting neurons from degeneration and damage.
  • Superior Blood-Brain Barrier Penetration: J4 directly affects brain energy balance, offering more effective treatment for neurodegenerative diseases.
  • Convenient Oral Administration: As a small molecule drug that can be administered orally, J4 offers ease of use and low manufacturing costs.

Professor Lin emphasized that Alzheimer's disease has become a growing medical burden and challenge, with millions of patients worldwide due to an aging population. Current clinical drugs can only alleviate symptoms without significantly altering disease progression. The development of J4 represents a major breakthrough in Taiwan's efforts to treat neurodegenerative diseases, potentially improving patient outcomes and quality of life, while reducing long-term care pressures and healthcare costs for families. 

The research team is actively preparing to submit an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA). Looking ahead, they plan to collaborate with domestic and international pharmaceutical companies and institutions to secure funding or patent licensing, accelerating clinical trials to further validate J4's efficacy and safety.

Resource (mandarin):

《新創動態》 新發現失智症治療途徑!中研院研發腦中腺苷平衡藥物 改善腦神經退化缺損