Unmet Need for Neuroprotective Disease-modifying DRUG Therapy for Parkinson’s Disease (PD)

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Incurable, Relentlessly Progressive & Debilitating;

  • NO prevention
  • NO early detection
  • NO drug cure
  • NO disease modifying drug treatment
  • Palliative drugs have debilitating severe dose-limiting side effects

 

 

  • 1 Million PD Patients in the USA
  • 60,000 new cases/year in US;
  • 1-2% of  People over 60  have Parkinson’s Disease
  • 10M sufferers worldwide.
  • Estimated care and productivity-loss cost in US ~$52B/year

NAC Exhibits  Neurorestorative Clinical Activity in PD Patients proven by Clinical study [1,2]

PROOF OF  NAC’s EFFICACY 

Clinical proof-of-concept with IV NAC showing neuro-restorative plus clinical efficacy: [1]

3-month weekly I.V. plus oral treatment improved UPDRS motor score and striatal synaptic dopamine transporter activity measured by DaTscan. [4]

PROBLEM with IV and Oral NAC administration:

IV delivery is not a viable treatment option for long term treatment.

High-dose IV NAC increases human brain GSH [3,4]

but high-dose Oral NAC alone [2,4,5] does not.

SOLUTION: INTRA-NASAL (IN) DELIVERY:

Intranasal (IN) NAC is 50 times more efficient in getting to the brain than IV delivery.

Proven by Neuronasal Human Pilot Study.

 

N2B NAC could provide a non-invasive efficient  neuro-protective treatment regimen for PD

 

Neuronasal: Nose to Brain (N2B) NAC Phase 1-ready with open US IND at FDA as proprietary drug-device combination.
  • Bypasses the blood-brain barrier-get drug to the brain.
  • Results in rapid delivery to the brain along both the olfactory and trigeminal nerves.

  • Reduces unwanted systemic exposure and side effects.

APPENDIX :

1.  Monti DA et al 2019. N-Acetyl Cysteine Is Associated With Dopaminergic  Improvement in Parkinson’s Disease. Clin Pharm Therapeut 104;4:884-890  2Shungu D ClinTrials.gov NCT01470027

2.  Shungu D ClinTrials.gov NCT01470027

3.  Holmay 2013. N-acetylcysteine Boosts Brain and Blood Glutathione in Gaucher and Parkinson’s Diseases, Clin Neuropharmacol. 36(4): 103–106

4.   Tuite P 2016. Magnetic resonance imaging as a potential biomarker for Parkinson’s disease. Translational Res 175:4-16

5 …Coles L 2018. Repeated-dose Oral N-acetylcysteine in Parkinson Disease: Pharmacokinetics and Effect on Brain Glutathione and Oxidative Stress. J Clin Pharmacol.58(2): 158–167