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Trial of Magnetic Resonance-Guided Putaminal Gene Therapy for Advanced Parkinson's Disease

Trial of Magnetic Resonance-Guided Putaminal Gene Therapy for Advanced Parkinson's Disease

John D Heiss, Codrin Lungu, Dima A Hammoud, Peter Herscovitch, Debra J Ehrlich, Davis P Argersinger, Sanhita Sinharay, Gretchen Scott, Tianxia Wu, Howard J Federoff, Kareem A Zaghloul, Mark Hallett, Russell R Lonser, etc.

Mov Disord. 2019 Jul;34(7):1073-1078.

PMID: 31145831

Abstract:

Objective:
To investigate the safety and tolerability of convection-enhanced delivery of an adeno-associated virus, serotype-2 vector carrying glial cell line-derived neurotrophic factor into the bilateral putamina of PD patients.
Methods:
Thirteen adult patients with advanced PD underwent adeno-associated virus, serotype-2 vector carrying glial cell line-derived neurotrophic factor and gadoteridol (surrogate MRI tracer) coinfusion (450 μL/hemisphere) at escalating doses: 9 × 1010 vg (n = 6); 3 × 1011 vg (n = 6); and 9 × 1011 vg (n = 1). Intraoperative MRI monitored infusion distribution. Patients underwent UPDRS assessment and [18 F]FDOPA-PET scanning preoperatively and 6 and 18 months postoperatively.
Results:
Adeno-associated virus, serotype-2 vector carrying glial cell line-derived neurotrophic factor was tolerated without clinical or radiographic toxicity. Average putaminal coverage was 26%. UPDRS scores remained stable. Ten of thirteen and 12 of 13 patients had increased [18 F]FDOPA Kis at 6 and 18 months postinfusion (increase range: 5-274% and 8-130%; median, 36% and 54%), respectively. Ki differences between baseline and 6- and 18-month follow-up were statistically significant (P < 0.0002).
Conclusion:
Adeno-associated virus, serotype-2 vector carrying glial cell line-derived neurotrophic factor infusion was safe and well tolerated. Increased [18 F]FDOPA uptake suggests a neurotrophic effect on dopaminergic neurons. © 2019 International Parkinson and Movement Disorder Society.

Chemicals Related in the Paper:

Catalog Number	Product Name	Structure	CAS Number	Price
AP120066548	Gadoteridol		120066-54-8	Price

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