Novel N-acyl amino acid treatment for Alzheimer’s disease and other neurodegenerative diseases

Novel N-acyl amino acid treatment for Alzheimer's disease and other neurodegenerative diseases for techtransfer

Neurodegenerative diseases such as Alzheimer’s present a difficulty for current medicine. While there are treatments that temporarily mask their symptoms, there are no effective treatments available that can modify their progression and evolution. Currently, four treatments are approved by the regulatory agencies, the European Medicines Agency (EMA) and the Food and Drug Administration (FDA). However, all of them are symptomatic and do not block the progression of the disease. Recently, two additional treatments have been approved by the FDA, though not yet by the EMA, based on monoclonal antibodies that appear to reduce amyloid plaques. Additionally, current treatments are costly and can produce serious side effects, making their preventive use inadvisable. To address this, researchers at the Spanish National Research Council (CSIC) are exploring the potential of a N-acyl amino acid treatment that instructs microglia against amyloid plaques protecting from Alzheimer’s disease. Additionally, this treatment restores normal neuronal activity and may offer protection against various neurodegenerative diseases, including Parkinson’s, multiple sclerosis, and amyotrophic lateral sclerosis.

Researchers have identified a novel compound derived from the conjugation of fatty acids and amino acids that significantly reduces amyloid pathology and exhibits neuroprotective properties.  This compound has been shown to restore normal neuronal activity in two different models of the disease (APP/PS1 mice and GMC101 worms), as well as in various cellular models, including cell lines and primary cultures of neurons and microglia.

The molecule shows great potential for treating and preventing neurodegenerative diseases. It’s simple and cost-effective synthesis could enable the development of affordable, highly effective treatments for both clinical therapies and preventive dietary supplements.

The current Technology Readiness Level (TRL) of this technology is 4.

It has undergone in vitro and in vivo testing, including trials with primary cultures in worms and mice, yielding satisfactory results.

Figure. N-acyl amino acid (NAAA) treatment increases microglia (IBA1, red) association with amyloid plaques (MX04, blue). Cellular nuclei in green (DRAQ5).
Figure. N-acyl amino acid (NAAA) treatment increases microglia (IBA1, red) association with amyloid plaques (MX04, blue). Cellular nuclei in green (DRAQ5).

Benefits:

  • It has demonstrated effectiveness in vitro and in vivo against neuronal degeneration.
  • It offers greater efficacy and fewer side effects than existing chemical treatments.
  • The treatment is quite unique because of its ability to activate microglia to fight Alzheimer’s disease more efficiently.
  • Derived from biological sources, the molecule has shown no observed toxicity in cultures.
  • Its ease of synthesis makes it an affordable product, facilitating low-cost treatments.
  • It can be dissolved in water or milk, allowing for non-invasive and easier administration.

The represented institution is looking for a collaboration that leads to a commercial exploitation of the presented invention. The institution’s preferred scenario involves reaching an agreement for technology transfer through either sale or licensing (exclusive or non-exclusive) and collaborating in technology development for practical applications. However, the specific form, terms, and conditions of this collaboration can be openly discussed if the presented technology is of interest.

Institution: The Institute for Neurosciences (IN), a joint centre of the Spanish National Research Council (CSIC) and the Miguel Hernández University of Elche (UMH).

TRL: 4

Protection status: A European patent application has been submitted in 2024 to protect this technology.

Contact: Elisa Sáenz / tech@viromii.com