Allemani C, Matsuda T, Di Carlo V, Harewood R, Matz M, Niksic M, et al. Global surveillance of trends in cancer survival 2000-14 (CONCORD-3): analysis of individual records for 37 513 025 patients diagnosed with one of 18 cancers from 322 population-based registries in 71 countries. Lancet. 2018;391:1023–75.

Article  PubMed  PubMed Central  Google Scholar 

American Cancer Society: Survival Rates for Breast Cancer: American Cancer Society; [updated March 1, 2022. Available from https://www.cancer.org/cancer/breast-cancer/understanding-a-breast-cancer-diagnosis/breast-cancer-survival-rates.html.

Slamon D, Eiermann W, Robert N, Pienkowski T, Martin M, Press M, et al. Adjuvant trastuzumab in HER2-positive breast cancer. N. Engl J Med. 2011;365:1273–83.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Vu T, Claret FX. Trastuzumab: updated mechanisms of action and resistance in breast cancer. Front Oncol. 2012;2:62.

Article  PubMed  PubMed Central  Google Scholar 

Cho HS, Mason K, Ramyar KX, Stanley AM, Gabelli SB, Denney DW Jr, et al. Structure of the extracellular region of HER2 alone and in complex with the Herceptin Fab. Nature. 2003;421:756–60.

Article  ADS  CAS  PubMed  Google Scholar 

DeBusk K, Ike C, Lindegger N, Schwartz N, Surinach A, Liu Y, et al. Real-world outcomes among patients with HER2+ metastatic breast cancer with brain metastases. J Manag Care Spec Pharm. 2022;28:657–66.

PubMed  Google Scholar 

Barnholtz-Sloan JS, Sloan AE, Davis FG, Vigneau FD, Lai P, Sawaya RE. Incidence proportions of brain metastases in patients diagnosed (1973 to 2001) in the Metropolitan Detroit Cancer Surveillance System. J Clin Oncol. 2004;22:2865–72.

Article  PubMed  Google Scholar 

Pardridge WM. CSF, blood-brain barrier, and brain drug delivery. Expert Opin Drug Deliv. 2016;13:963–75.

Article  CAS  PubMed  Google Scholar 

Stemmler HJ, Schmitt M, Willems A, Bernhard H, Harbeck N, Heinemann V. Ratio of trastuzumab levels in serum and cerebrospinal fluid is altered in HER2-positive breast cancer patients with brain metastases and impairment of blood-brain barrier. Anticancer Drugs. 2007;18:23–8.

Article  CAS  PubMed  Google Scholar 

Mills MN, King W, Soyano A, Pina Y, Czerniecki BJ, Forsyth PA, et al. Evolving management of HER2+ breast cancer brain metastases and leptomeningeal disease. J Neurooncol. 2022;157:249–69.

Article  PubMed  Google Scholar 

Bousquet G, Darrouzain F, de Bazelaire C, Ternant D, Barranger E, Winterman S, et al. Intrathecal trastuzumab halts progression of CNS metastases in breast cancer. J Clin Oncol. 2016;34:e151–5.

Article  PubMed  Google Scholar 

Bonneau C, Paintaud G, Tredan O, Dubot C, Desvignes C, Dieras V, et al. Phase I feasibility study for intrathecal administration of trastuzumab in patients with HER2 positive breast carcinomatous meningitis. Eur J Cancer. 2018;95:75–84.

Article  CAS  PubMed  Google Scholar 

Delhaas EM, Huygen F. Complications associated with intrathecal drug delivery systems. BJA Educ. 2020;20:51–7.

Article  CAS  PubMed  Google Scholar 

Naso MF, Tomkowicz B, Perry WL 3rd, Strohl WR. Adeno-associated virus (AAV) as a vector for gene therapy. BioDrugs. 2017;31:317–34.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Hinderer C, Katz N, Dyer C, Goode T, Johansson J, Bell P, et al. Translational feasibility of lumbar puncture for intrathecal AAV administration. Mol Ther Methods Clin Dev. 2020;17:969–74.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Hordeaux J, Hinderer C, Goode T, Katz N, Buza EL, Bell P, et al. Toxicology study of intra-cisterna magna adeno-associated virus 9 expressing human alpha-L-iduronidase in Rhesus Macaques. Mol Ther Methods Clin Dev. 2018;10:79–88.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Hordeaux J, Jeffrey BA, Jian J, Choudhury GR, Michalson K, Mitchell TW, et al. Efficacy and safety of a krabbe disease gene therapy. Hum Gene Ther. 2022;33:499–517.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Kang L, Jin S, Wang J, Lv Z, Xin C, Tan C, et al. AAV vectors applied to the treatment of CNS disorders: Clinical status and challenges. J Control Release. 2023;355:458–73.

Article  CAS  PubMed  Google Scholar 

Keeler AM, Flotte TR. Recombinant adeno-associated virus gene therapy in light of luxturna (and Zolgensma and Glybera): where are we, and how did we get here? Annu Rev Virol. 2019;6:601–21.

Article  CAS  PubMed  PubMed Central  Google Scholar 

George LA, Monahan PE, Eyster ME, Sullivan SK, Ragni MV, Croteau SE, et al. Multiyear factor VIII expression after AAV gene transfer for hemophilia A. N. Engl J Med. 2021;385:1961–73.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Kuzmin DA, Shutova MV, Johnston NR, Smith OP, Fedorin VV, Kukushkin YS, et al. The clinical landscape for AAV gene therapies. Nat Rev Drug Discov. 2021;20:173–4.

Article  CAS  PubMed  Google Scholar 

Rothwell WT, Bell P, Richman LK, Limberis MP, Tretiakova AP, Li M, et al. Intrathecal viral vector delivery of trastuzumab prevents or inhibits tumor growth of human HER2-Positive Xenografts in Mice. Cancer Res. 2018;78:6171–82.

Article  CAS  PubMed  Google Scholar 

Lock M, Alvira M, Vandenberghe LH, Samanta A, Toelen J, Debyser Z, et al. Rapid, simple, and versatile manufacturing of recombinant adeno-associated viral vectors at scale. Hum Gene Ther. 2010;21:1259–71.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Katz N, Goode T, Hinderer C, Hordeaux J, Wilson JM. Standardized method for intra-cisterna magna delivery under fluoroscopic guidance in nonhuman primates. Hum Gene Ther Methods. 2018;29:212–9.

Article  CAS  PubMed  Google Scholar 

Lal S, Lacroix M, Tofilon P, Fuller GN, Sawaya R, Lang FF. An implantable guide-screw system for brain tumor studies in small animals. J Neurosurg. 2000;92:326–33.

Article  CAS  PubMed  Google Scholar 

Donoghue JF, Bogler O, Johns TG. A simple guide screw method for intracranial xenograft studies in mice. J Vis Exp. 2011;55:3157.

Maple L, Lathrop R, Bozich S, Harman W, Tacey R, Kelley M, et al. Development and validation of ELISA for herceptin detection in human serum. J Immunol Methods. 2004;295:169–82.

Article  CAS  PubMed  Google Scholar 

Bouwer NI, Steenbruggen TG, van Rosmalen J, Rier HN, Kitzen J, van Bekkum ML, et al. Cardiotoxicity during long-term trastuzumab use in patients with HER2-positive metastatic breast cancer: who needs cardiac monitoring? Breast Cancer Res Treat. 2021;186:851–62.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Boutin S, Monteilhet V, Veron P, Leborgne C, Benveniste O, Montus MF, et al. Prevalence of serum IgG and neutralizing factors against adeno-associated virus (AAV) types 1, 2, 5, 6, 8, and 9 in the healthy population: implications for gene therapy using AAV vectors. Hum Gene Ther. 2010;21:704–12.

Article  CAS  PubMed  Google Scholar 

Horiuchi M, Hinderer CJ, Greig JA, Dyer C, Buza EL, Bell P, et al. Intravenous immunoglobulin prevents peripheral liver transduction of intrathecally delivered AAV vectors. Mol Ther Methods Clin Dev. 2022;27:272–80.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Verdera HC, Kuranda K, Mingozzi F. AAV vector immunogenicity in humans: a long journey to successful gene transfer. Mol Ther. 2020;28:723–46.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Gopinath C, Nathar TJ, Ghosh A, Hickstein DD, Nelson EJR. Contemporary animal models for human gene therapy applications. Curr Gene Ther. 2015;15:531–40.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Hinderer C, Bell P, Vite CH, Louboutin JP, Grant R, Bote E, et al. Widespread gene transfer in the central nervous system of cynomolgus macaques following delivery of AAV9 into the cisterna magna. Mol Ther Methods Clin Dev. 2014;1:14051.

Article  PubMed  PubMed Central  Google Scholar 

Kyeong S, Cha YJ, Ahn SG, Suh SH, Son EJ, Ahn SJ. Subtypes of breast cancer show different spatial distributions of brain metastases. PLoS One. 2017;12:e0188542.

Article  PubMed  PubMed Central  Google Scholar 

Pestalozzi BC, Zahrieh D, Price KN, Holmberg SB, Lindtner J, Collins J, et al. Identifying breast cancer patients at risk for Central Nervous System (CNS) metastases in trials of the International Breast Cancer Study Group (IBCSG). Ann Oncol. 2006;17:935–44.

Article  CAS  PubMed  Google Scholar 

Scott BJ, Kesari S. Leptomeningeal metastases in breast cancer. Am J Cancer Res. 2013;3:117–26.

CAS  PubMed  PubMed Central