Clinically known as Neuronal Ceroid-Lipofuscinoses (NCL), Batten disease, is a rare neuron killing disease and one of the lysosomal storage disorders (LSDs). It is associated with a mutation or lack of palmitoyl-protein thioesterase-1 (PPT1) gene. It manifests very early in a child's life causing absence of brain activity as early as 4 years of age.
Acute lymphoblastic leukemia (ALL) is the most common cancer in children with approximately 3,250 new cases occurring per year in the United States. About 20% of cases are refractory to current treatment protocols and there is a desperate need for targeted therapies that do not result in adverse side effects such as cognitive impairment.
This technology from the NCI Molecular Imaging Program relates to a Zirconium-89 (89Zr)-oxine complex for cell labeling, tracking of labeled cells by whole-body positron emission tomography/computed tomography (PET/CT) imaging, and associated methods. A long half-life of 89Zr (78.4 hours), high sensitivity of PET, and absence of background signal in the recipient enable tracking cells over a week using low levels of labeling radioactivity without causing cellular toxicity.
Cytidine analogs remain an area of active drug discovery and development, with five FDA approved drugs for the treatment of acute myeloid leukemia (AML). Two of these drugs, azacitidine (Vidaza®) and decitabine (Dacogen®), which were approved for myelodysplastic syndromes in 2004 and 2006, respectively, inhibit the DNA maintenance methyltransferase DNMT1. Because of the general toxicity of azacitidines, other nucleoside analogs are favored as therapeutics.
Summary:
The National Institute on Aging (NIA) seeks research co-development partners and/or licensees for the pre-clinical and clinical development of the compounds as anti-inflammatory therapeutics for systemic degenerative and neurodegenerative disorders.
Description of Technology:
This technology includes an alpha-galactosidase-A knockout mouse model that can be used to study Fabry disease, an X-linked lysosomal storage disorder. Alpha-galactosidase-A is a crucial enzyme responsible for the breakdown of glycolipids, particularly globotriaosylceramide (Gb3), within lysosomes. In Fabry disease, a rare and inherited lysosomal storage disorder, mutations in the GLA gene lead to deficient or non-functional alpha-galactosidase-A enzyme activity.