UCLA researchers in the department of Molecular, Cellular, and Developmental Biology have developed a treatment that could mitigate some mental issues related to various intellectual disabilities syndromes.
BACKGROUND: Approximately 6.5 million people in the United states have an intellectual disability syndrome, classified as an IQ of less than 70. These mental disability disorders, although not impossible to overcome, have no effective treatments to improve or regulate symptoms. This, unfortunately, requires many patients to require constant care costing around 14 to 57 billion dollars annually. Recently, researchers studying these syndromes have uncovered that many are linked to improper regulation of patient genes. If methods could be developed to help regulate these genes, a possible treatment could be developed that would help mitigate many of the issues related to intellectual disability. Therefore, a current unmet need exists for a treatment that can help address many of the phenotypic symptoms and improve the lives of millions of patients suffering from an intellectual disability.
INNOVATION: UCLA researchers in the Department of Molecular, Cellular, and Developmental Biology have developed a set of small molecules that may reduce some of the issues related to intellectual disabilities syndrome. Previously, these researchers discovered a unique cellular phenotype within the brains of patients diagnosed with intellectual disabilities. As such, they developed a set of small molecules targeting this particular phenotype, resulting in the cells reverting to a normal phenotype. This was further tested with specialized organoid cultures, which simulated tissues from patients with intellectual disabilities syndrome. By testing their molecules against these models, the inventors showed that their treatment reduced the phenotypic severity in diseased cells. This causes several of these cells to become normal compared to untreated samples. As no known cure or treatment has been discovered for any intellectual disabilities, this advancement could be the beginning of treatments to help patients with intellectual disabilities.
POTENTIAL APPLICATIONS:
• Intellectual Disabilities Treatment
• Stress Treatments
• Mental Aging Disorders
ADVANTAGES:
• Bypasses Body’s Natural Defenses such as Blood Brain Barrier
• Small Molecule Therapeutic
DEVELOPMENT-TO-DATE:
The inventors have discovered a set of small molecules. These molecules have been tested for efficacy in organoid cells and shown demonstratable improvements to the cell lines reducing phenotypic symptoms.
Related Papers:
Samarasinghe RA, Miranda OA, Mitchell S, et al. Identification of neural oscillations and epileptiform changes in human brain organoids. bioRxiv; 2019. DOI: 10.1101/820183.
Ohashi M, Korsakova E, Allen D, et al. Loss of MECP2 Leads to Activation of P53 and Neuronal Senescence. Stem Cell Reports. 2018;10(5):1453-1463. doi:10.1016/j.stemcr.2018.04.001