Gene Therapy For Neurological Disorders: Parkinson’s Disease And Glioblastoma

Aghi, M., Vogelbaum, M. A., Kesari, S., Chen, C. C., Liau, L. M., Piccioni, D., Portnow, J., Chang, S., Robbins, J. M., Boyce, T., Huang, T. T., Pertschuk, D., Ostertag, D., & Cloughesy, T. F. (2014). AT-02 * INTRATUMORAL delivery of THE Retroviral replicating VECTOR (rrv) toca 511 in subjects with RECURRENT high GRADE Glioma: Interim report of Phase 1 Study (NCT 01156584). Neuro-Oncology, 16(suppl 5), v8–v8. https://doi.org/10.1093/neuonc/nou237.2

Bankiewicz, K. S., Eberling, J. L., Kohutnicka, M., Jagust, W., Pivirotto, P., Bringas, J., Cunningham, J., Budinger, T. F., & Harvey-White, J. (2000). Convection-enhanced delivery of AAV vector in parkinsonian monkeys; in vivo detection of gene expression and restoration of dopaminergic function using pro-drug approach. Experimental neurology, 164(1), 2–14. https://doi.org/10.1006/exnr.2000.7408

Bankiewicz, K. S., Forsayeth, J., Eberling, J. L., Sanchez-Pernaute, R., Pivirotto, P., Bringas, J., Herscovitch, P., Carson, R. E., Eckelman, W., Reutter, B., & Cunningham, J. (2006). Long-term clinical improvement in MPTP-lesioned primates after gene therapy with AAV-hAADC. Molecular therapy : the journal of the American Society of Gene Therapy, 14(4), 564–570. https://doi.org/10.1016/j.ymthe.2006.05.005

Caffery, B., Lee, J. S., & Alexander-Bryant, A. A. (2019). Vectors for Glioblastoma Gene Therapy: Viral & Non-Viral Delivery Strategies. Nanomaterials (Basel, Switzerland), 9(1), 105. https://doi.org/10.3390/nano9010105

Carlsson, S. K., Brothers, S. P., & Wahlestedt, C. (2014). Emerging treatment strategies for glioblastoma multiforme. EMBO molecular medicine, 6(11), 1359–1370. https://doi.org/10.15252/emmm.201302627

Chen, Y. H., Keiser, M. S., & Davidson, B. L. (2018). Viral Vectors for Gene Transfer. Current protocols in mouse biology, 8(4), e58. https://doi.org/10.1002/cpmo.58

Chiocca, E. A., Aguilar, L. K., Bell, S. D., Kaur, B., Hardcastle, J., Cavaliere, R., McGregor, J., Lo, S., Ray-Chaudhuri, A., Chakravarti, A., Grecula, J., Newton, H., Harris, K. S., Grossman, R. G., Trask, T. W., Baskin, D. S., Monterroso, C., Manzanera, A. G., Aguilar-Cordova, E., & New, P. Z. (2011). Phase IB study of gene-mediated cytotoxic immunotherapy adjuvant to up-front surgery and intensive timing radiation for malignant glioma. Journal of clinical oncology : official journal of the American Society of Clinical Oncology, 29(27), 3611–3619. https://doi.org/10.1200/JCO.2011.35.5222

Choong, C. J., Baba, K., & Mochizuki, H. (2016). Gene therapy for neurological disorders. Expert opinion on biological therapy, 16(2), 143–159. https://doi.org/10.1517/14712598.2016.1114096

Choudhury, S. R., Hudry, E., Maguire, C. A., Sena-Esteves, M., Breakefield, X. O., & Grandi, P. (2017). Viral vectors for therapy of neurologic diseases. Neuropharmacology, 120, 63–80. https://doi.org/10.1016/j.neuropharm.2016.02.013

Christine, C. W., Starr, P. A., Larson, P. S., Eberling, J. L., Jagust, W. J., Hawkins, R. A., VanBrocklin, H. F., Wright, J. F., Bankiewicz, K. S., & Aminoff, M. J. (2009). Safety and tolerability of putaminal AADC gene therapy for Parkinson disease. Neurology, 73(20), 1662–1669. https://doi.org/10.1212/WNL.0b013e3181c29356

Davis M. E. (2016). Glioblastoma: Overview of Disease and Treatment. Clinical journal of oncology nursing, 20(5 Suppl), S2–S8. https://doi.org/10.1188/16.CJON.S1.2-8

Dorsey ER, Sherer T, Okun MS, Bloem BR. The Emerging Evidence of the Parkinson Pandemic. J Parkinsons Dis. 2018;8(s1):S3-S8. doi: 10.3233/JPD-181474. PMID: 30584159; PMCID: PMC6311367

Eberling, J. L., Jagust, W. J., Christine, C. W., Starr, P., Larson, P., Bankiewicz, K. S., & Aminoff, M. J. (2008). Results from a phase I safety trial of hAADC gene therapy for Parkinson disease. Neurology, 70(21), 1980–1983. https://doi.org/10.1212/01.wnl.0000312381.29287.ff

Emborg, M. E., Carbon, M., Holden, J. E., During, M. J., Ma, Y., Tang, C., Moirano, J., Fitzsimons, H., Roitberg, B. Z., Tuccar, E., Roberts, A., Kaplitt, M. G., & Eidelberg, D. (2007). Subthalamic glutamic acid decarboxylase gene therapy: changes in motor function and cortical metabolism. Journal of cerebral blood flow and metabolism : official journal of the International Society of Cerebral Blood Flow and Metabolism, 27(3), 501–509. https://doi.org/10.1038/sj.jcbfm.9600364

GBD 2016 Parkinson's Disease Collaborators (2018). Global, regional, and national burden of Parkinson's disease, 1990-2016: a systematic analysis for the Global Burden of Disease Study 2016. The Lancet. Neurology, 17(11), 939–953. https://doi.org/10.1016/S1474-4422(18)30295-3

Hadaczek, P., Eberling, J. L., Pivirotto, P., Bringas, J., Forsayeth, J., & Bankiewicz, K. S. (2010). Eight years of clinical improvement in MPTP-lesioned primates after gene therapy with AAV2-hAADC. Molecular therapy : the journal of the American Society of Gene Therapy, 18(8), 1458–1461. https://doi.org/10.1038/mt.2010.106

Huang, T. T., Hlavaty, J., Ostertag, D., Espinoza, F. L., Martin, B., Petznek, H., Rodriguez-Aguirre, M., Ibañez, C. E., Kasahara, N., Gunzburg, W., Gruber, H. E., Pertschuk, D., Jolly, D. J., & Robbins, J. M. (2013). Toca 511 gene transfer and 5-fluorocytosine in combination with temozolomide demonstrates synergistic therapeutic efficacy in a temozolomide-sensitive glioblastoma model. Cancer gene therapy, 20(10), 544–551. https://doi.org/10.1038/cgt.2013.51

Kaplitt, M. G., Feigin, A., Tang, C., Fitzsimons, H. L., Mattis, P., Lawlor, P. A., Bland, R. J., Young, D., Strybing, K., Eidelberg, D., & During, M. J. (2007). Safety and tolerability of gene therapy with an adeno-associated virus (AAV) borne GAD gene for Parkinson's disease: an open label, phase I trial. Lancet (London, England), 369(9579), 2097–2105. https://doi.org/10.1016/S0140-6736(07)60982-9

Kieran, M. W., Goumnerova, L., Manley, P., Chi, S. N., Marcus, K., Manzanera, A. G., Aguilar-Cordova, E., DiPatri, A. J., Tomita, T., Lulla, R., Aguilar, L. K., & Goldman, S. (2016). EPT-14PHASE I study of GENE MEDIATED Cytotoxic immunotherapy WITH adv-tk as adjuvant to surgery and radiation therapy for PEDIATRIC MALIGNANT glioma and Recurrent EPENDYMOMA. Neuro-Oncology, 18(suppl 3). https://doi.org/10.1093/neuonc/now069.13

Lang, F. F., Bruner, J. M., Fuller, G. N., Aldape, K., Prados, M. D., Chang, S., Berger, M. S., McDermott, M. W., Kunwar, S. M., Junck, L. R., Chandler, W., Zwiebel, J. A., Kaplan, R. S., & Yung, W. K. (2003). Phase I trial of adenovirus-mediated p53 gene therapy for recurrent glioma: biological and clinical results. Journal of clinical oncology : official journal of the American Society of Clinical Oncology, 21(13), 2508–2518. https://doi.org/10.1200/JCO.2003.21.13.2508

Li, C., & Samulski, R. J. (2020). Engineering adeno-associated virus vectors for gene therapy. Nature reviews. Genetics, 21(4), 255–272. https://doi.org/10.1038/s41576-019-0205-4

Levy, R., Lang, A. E., Dostrovsky, J. O., Pahapill, P., Romas, J., Saint-Cyr, J., Hutchison, W. D., & Lozano, A. M. (2001). Lidocaine and muscimol microinjections in subthalamic nucleus reverse Parkinsonian symptoms. Brain : a journal of neurology, 124(Pt 10), 2105–2118. https://doi.org/10.1093/brain/124.10.2105

LeWitt, P. A., Rezai, A. R., Leehey, M. A., Ojemann, S. G., Flaherty, A. W., Eskandar, E. N., Kostyk, S. K., Thomas, K., Sarkar, A., Siddiqui, M. S., Tatter, S. B., Schwalb, J. M., Poston, K. L., Henderson, J. M., Kurlan, R. M., Richard, I. H., Van Meter, L., Sapan, C. V., During, M. J., Kaplitt, M. G., … Feigin, A. (2011). AAV2-GAD gene therapy for advanced Parkinson's disease: a double-blind, sham-surgery controlled, randomised trial. The Lancet. Neurology, 10(4), 309–319. https://doi.org/10.1016/S1474-4422(11)70039-4

Milone, M. C., & O'Doherty, U. (2018). Clinical use of lentiviral vectors. Leukemia, 32(7), 1529–1541. https://doi.org/10.1038/s41375-018-0106-0

Mittermeyer, G., Christine, C. W., Rosenbluth, K. H., Baker, S. L., Starr, P., Larson, P., Kaplan, P. L., Forsayeth, J., Aminoff, M. J., & Bankiewicz, K. S. (2012). Long-term evaluation of a phase 1 study of AADC gene therapy for Parkinson's disease. Human gene therapy, 23(4), 377–381. https://doi.org/10.1089/hum.2011.220

Naso, M. F., Tomkowicz, B., Perry, W. L., 3rd, & Strohl, W. R. (2017). Adeno-Associated Virus (AAV) as a Vector for Gene Therapy. BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy, 31(4), 317–334. https://doi.org/10.1007/s40259-017-0234-5

Oldfield, E. H., Ram, Z., Chiang, Y., & Blaese, R. M. (1995). Intrathecal gene therapy for the treatment of leptomeningeal carcinomatosis. GTI 0108. A phase I/II study. Human gene therapy, 6(1), 55–85. https://doi.org/10.1089/hum.1995.6.1-55

Oshiro, E. M., Viola, J. J., Oldfield, E. H., Walbridge, S., Bacher, J., Frank, J. A., Blaese, R. M., & Ram, Z. (1995). Toxicity studies and distribution dynamics of retroviral vectors following intrathecal administration of retroviral vector-producer cells. Cancer gene therapy, 2(2), 87–95.

Pardridge, W. M. (2008). Re-Engineering Biopharmaceuticals for delivery to brain with Molecular trojan horses. Bioconjugate Chemistry, 19(7), 1327–1338. https://doi.org/10.1021/bc800148t

Rainov N. G. (2000). A phase III clinical evaluation of herpes simplex virus type 1 thymidine kinase and ganciclovir gene therapy as an adjuvant to surgical resection and radiation in adults with previously untreated glioblastoma multiforme. Human gene therapy, 11(17), 2389–2401. https://doi.org/10.1089/104303400750038499

Ram, Z., Culver, K. W., Oshiro, E. M., Viola, J. J., DeVroom, H. L., Otto, E., Long, Z., Chiang, Y., McGarrity, G. J., Muul, L. M., Katz, D., Blaese, R. M., & Oldfield, E. H. (1997). Therapy of malignant brain tumors by intratumoral implantation of retroviral vector-producing cells. Nature medicine, 3(12), 1354–1361. https://doi.org/10.1038/nm1297-1354

Ramamoorth, M., & Narvekar, A. (2015). Non viral vectors in gene therapy- an overview. Journal of clinical and diagnostic research : JCDR, 9(1), GE01–GE6. https://doi.org/10.7860/JCDR/2015/10443.5394

Rodrigues, G. A., Shalaev, E., Karami, T. K., Cunningham, J., Slater, N., & Rivers, H. M. (2018). Pharmaceutical Development of AAV-Based Gene Therapy Products for the Eye. Pharmaceutical research, 36(2), 29. https://doi.org/10.1007/s11095-018-2554-7

Sakthikumar, S., Roy, A., Haseeb, L., Pettersson, M. E., Sundström, E., Marinescu, V. D., Lindblad-Toh, K., & Forsberg-Nilsson, K. (2020). Whole-genome sequencing of glioblastoma reveals enrichment of non-coding constraint mutations in known and novel genes. Genome Biology, 21(1). https://doi.org/10.1186/s13059-020-02035-x

Smith, C., & Blomberg, P. (2017). Genterapi – från idé till verklighet - Ännu har få patienter behandlats och preparaten är ofta mycket dyra – men utvecklingen går fort nu [Gene therapy – from idea to reality]. Lakartidningen, 114, EWYL.

Sudhakar, V., & Richardson, R. M. (2019). Gene Therapy for Neurodegenerative Diseases. Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics, 16(1), 166–175. https://doi.org/10.1007/s13311-018-00694-0

Takahashi, M., Valdes, G., Hiraoka, K., Inagaki, A., Kamijima, S., Micewicz, E., Gruber, H. E., Robbins, J. M., Jolly, D. J., McBride, W. H., Iwamoto, K. S., & Kasahara, N. (2014). Radiosensitization of gliomas by intracellular generation of 5-fluorouracil potentiates prodrug activator gene therapy with a retroviral replicating vector. Cancer gene therapy, 21(10), 405–410. https://doi.org/10.1038/cgt.2014.38

Van Bulck, M., Sierra-Magro, A., Alarcon-Gil, J., Perez-Castillo, A., & Morales-Garcia, J. A. (2019). Novel Approaches for the Treatment of Alzheimer's and Parkinson's Disease. International journal of molecular sciences, 20(3), 719. https://doi.org/10.3390/ijms20030719

Wen, P. Y., Weller, M., Lee, E. Q., Alexander, B. M., Barnholtz-Sloan, J. S., Barthel, F. P., Batchelor, T. T., Bindra, R. S., Chang, S. M., Chiocca, E. A., Cloughesy, T. F., DeGroot, J. F., Galanis, E., Gilbert, M. R., Hegi, M. E., Horbinski, C., Huang, R. Y., Lassman, A. B., Le Rhun, E., Lim, M., … van den Bent, M. J. (2020). Glioblastoma in adults: a Society for Neuro-Oncology (SNO) and European Society of Neuro-Oncology (EANO) consensus review on current management and future directions. Neuro-oncology, 22(8), 1073–1113. https://doi.org/10.1093/neuonc/noaa106

Wheeler, L. A., Manzanera, A. G., Bell, S. D., Cavaliere, R., McGregor, J. M., Grecula, J. C., Newton, H. B., Lo, S. S., Badie, B., Portnow, J., Teh, B. S., Trask, T. W., Baskin, D. S., New, P. Z., Aguilar, L. K., Aguilar-Cordova, E., & Chiocca, E. A. (2016). Phase II multicenter study of gene-mediated cytotoxic immunotherapy as adjuvant to surgical resection for newly diagnosed malignant glioma. Neuro-oncology, 18(8), 1137–1145. https://doi.org/10.1093/neuonc/now002

Wong, A. D., Ye, M., Levy, A. F., Rothstein, J. D., Bergles, D. E., & Searson, P. C. (2013). The blood-brain barrier: An engineering perspective. Frontiers in Neuroengineering, 6. https://doi.org/10.3389/fneng.2013.00007

World Health Organization. (2020, December 9). The top 10 causes of death. World Health Organization: WHO; https://www.who.int/news-room/fact-sheets/detail/the-top-10-causes-of-death