This technology describes the use of thiazolides, a class of FDA-approved antivirals that can cross the blood brain barrier, to treat tauopathic neurodegenerative diseases.
Unmet Need: Treatment to reduce accumulation of tau proteins in the brain
Tau proteins, whose main functionality is to stabilize microtubules, are found along the axons of the neurons in the central nervous system. However, the over-accumulation or hyperphosphorylation of tau proteins can lead to the formation of higher order structures of these proteins in the brain and is related to neurodegenerative diseases such as Alzheimer’s. While there are some small molecule inhibitors, antibodies and vaccines currently undergoing clinical trials, there is currently no approved treatment for tauopathies.
The Technology: FDA-approved antiviral that can decrease tau protein levels
This technology describes an alternative indication for thiazolides in the treatment of neurodegenerative diseases. Thiazolides are a class of small molecule drugs that have already been FDA-approved for use as anti-microbials. One of these, nitazoxanide (NTZ), has been correlated with decreased total tau protein levels in a mouse-embryonic stem cell-derived neuron model, and it has demonstrated efficacy for restoration of memory deficits in a transgenic mouse model for Alzheimer’s. The use of thiazolides as therapeutics could also be expanded to other neurodegenerative tauopathies.
This technology has been validated in mouse models of Alzheimer’s disease.
Applications:
- Therapeutic treatment for Alzheimer’s disease, dementia, progressive supranuclear palsy, and corticobasal degeneration
- Therapeutic for halting the progression of traumatic brain injury
- Research of mitochondrial uncoupling as it relates to tauopathies
Advantages:
- Already FDA-approved for a different indication
- No other currently available treatments for excessive tau proteins
- Mouse Alzheimer’s model tests show restoration of memory deficits
Lead Inventor:
Tae-Wan Kim, Ph.D.
Patent Information:
Patent Status
Related Publications:
Amireddy N, Puttapaka SN, Vinnakota RL, Ravuri HG, Thonda S, Kalivendi SV. “The unintended mitochondrial uncoupling effects of the FDA-approved anti-helminth drug nitazoxanide mitigates experimental parkinsonism in mice” J Biol Chem. 2017 Aug 10; 292(38): 15731-15743.
McIntire LBJ, Berman DE, Myaeng J, Staniszewski A, Arancio O, Di Paolo G, Kim TW. “Reduction of synaptojanin 1 ameliorates synaptic and behavioral impairments in a mouse model of Alzheimer’s disease” J Neurosci. 2012 Oct 31; 32(44): 15271-15276.
Oliveira TG, Chan RB, Tian H, Laredo M, Shui G, Staniszewski A, Zhang H, Wang L, Kim TW, Duff KE, Wenk MR, Arancio O, Di Paolo G “Phospholipase D2 ablation ameliorates Alzheimer’s disease-linked synaptic dysfunction and cognitive deficits” J Neurosci. 2010 Dec 8; 30(49): 16419-16428.
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