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1. Intracerebroventricular B7-H3-targeting CAR T cells for diffuse intrinsic pontine glioma: a phase 1 trial

Author

Vitanza, Nicholas A., Ronsley, Rebecca, Choe, Michelle, Seidel, Kristy, Huang, Wenjun, Rawlings-Rhea, Stephanie D., Beam, Madison, Steinmetzer, Leonel, Wilson, Ashley L., Brown, Christopher, Beebe, Adam, Lindgren, Catherine, Gustafson, Joshua A., Wein, Amy, Holtzclaw, Susan, Hoeppner, Corrine, Goldstein, Hannah E., Browd, Samuel R., Hauptman, Jason S., Lee, Amy, Ojemann, Jeffrey G., Crotty, Erin E., Leary, Sarah E. S., Perez, Francisco A., Wright, Jason N., Alonso, Marta M., Dun, Matthew D., Foster, Jessica B., Hurst, Diana, Kong, Ada, Thomsen, Alison, Orentas, Rimas J., Albert, Catherine M., Pinto, Navin, Annesley, Colleen, Gardner, Rebecca A., Ho, On, Pattabhi, Sowmya, Gust, Juliane, Wendler, Jason P., Park, Julie R., and Jensen, Michael C.

Published
2025
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2. STRIvE-02: A First-in-Human Phase I Study of Systemically Administered B7-H3 Chimeric Antigen Receptor T Cells for Patients With Relapsed/Refractory Solid Tumors

Author

Pinto, Navin, Albert, Catherine M., Taylor, Mallory R., Ullom, Heidi B., Wilson, Ashley L., Huang, Wenjun, Wendler, Jason, Pattabhi, Sowmya, Seidel, Kristy, Brown, Christopher, Gustafson, Joshua A., Rawlings-Rhea, Stephanie D., Cheeney, Safia H.E., Burleigh, Katelyn, Gustafson, Heather H., Orentas, Rimas J., Vitanza, Nicholas A., Gardner, Rebecca A., Jensen, Michael C., and Park, Julie R.

Published
2024
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4. A stem cell epigenome is associated with primary nonresponse to CD19 CAR T cells in pediatric acute lymphoblastic leukemia

Author

Masih, Katherine E., Gardner, Rebecca A., Chou, Hsien-Chao, Abdelmaksoud, Abdalla, Song, Young K., Mariani, Luca, Gangalapudi, Vineela, Gryder, Berkley E., Wilson, Ashley L., Adebola, Serifat O., Stanton, Benjamin Z., Wang, Chaoyu, Milewski, David, Kim, Yong Yean, Tian, Meijie, Cheuk, Adam Tai-Chi, Wen, Xinyu, Zhang, Yue, Altan-Bonnet, Grégoire, Kelly, Michael C., Wei, Jun S., Bulyk, Martha L., Jensen, Michael C., Orentas, Rimas J., and Khan, Javed

Published
2023
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6. Locoregional CAR T cells for children with CNS tumors: Clinical procedure and catheter safety

Author

Vitanza, Nicholas A., Ronsley, Rebecca, Choe, Michelle, Henson, Casey, Breedt, Mandy, Barrios-Anderson, Adriel, Wein, Amy, Brown, Christopher, Beebe, Adam, Kong, Ada, Kirkey, Danielle, Lee, Brittany M., Leary, Sarah E.S., Crotty, Erin E., Hoeppner, Corrine, Holtzclaw, Susan, Wilson, Ashley L., Gustafson, Joshua A., Foster, Jessica B., Iliff, Jeffrey J., Goldstein, Hannah E., Browd, Samuel R., Lee, Amy, Ojemann, Jeffrey G., Pinto, Navin, Gust, Juliane, Gardner, Rebecca A., Jensen, Michael C., Hauptman, Jason S., and Park, Julie R.

Published
2023
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7. "I was very scared when I found out my son had sickle cell": Caregiver knowledge and attitudes toward early intervention for young children with sickle cell disease: Implications for policy and practice from a multi‐site study.

Author

Hoyt, Catherine R., MacArthur, Erin, Leaver, Hayes, Wilson, Ashley L., Davis, Kandace, Harris, Kelly M., Damiano, Riley, Moore, Hunter, Housten, Ashley J., Britts, Rosemary, Hankins, Jane Silva, King, Allison A., and Heitzer, Andrew M.

Published
2024
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9. Locoregional infusion of HER2-specific CAR T cells in children and young adults with recurrent or refractory CNS tumors: an interim analysis

Author

Vitanza, Nicholas A., Johnson, Adam J., Wilson, Ashley L., Brown, Christopher, Yokoyama, Jason K., Künkele, Annette, and Chang, Cindy A.

Subjects
T cells -- Receptors, Antigen receptors, T cell -- Health aspects -- Testing, Nervous system tumors -- Diagnosis -- Development and progression -- Care and treatment, Immunotherapy -- Testing, Tumors in children -- Diagnosis -- Development and progression -- Care and treatment, Biological sciences, Health
Abstract

Locoregional delivery of chimeric antigen receptor (CAR) T cells has resulted in objective responses in adults with glioblastoma, but the feasibility and tolerability of this approach is yet to be evaluated for pediatric central nervous system (CNS) tumors. Here we show that engineering of a medium-length CAR spacer enhances the therapeutic efficacy of human erb-b2 receptor tyrosine kinase 2 (HER2)-specific CAR T cells in an orthotopic xenograft medulloblastoma model. We translated these findings into BrainChild-01 (NCT03500991), an ongoing phase 1 clinical trial at Seattle Children's evaluating repetitive locoregional dosing of these HER2-specific CAR T cells to children and young adults with recurrent/refractory CNS tumors, including diffuse midline glioma. Primary objectives are assessing feasibility, safety and tolerability; secondary objectives include assessing CAR T cell distribution and disease response. In the outpatient setting, patients receive infusions via CNS catheter into either the tumor cavity or the ventricular system. The initial three patients experienced no dose-limiting toxicity and exhibited clinical, as well as correlative laboratory, evidence of local CNS immune activation, including high concentrations of CXCL10 and CCL2 in the cerebrospinal fluid. This interim report supports the feasibility of generating HER2-specific CAR T cells for repeated dosing regimens and suggests that their repeated intra-CNS delivery might be well tolerated and activate a localized immune response in pediatric and young adult patients. In an interim analysis of a phase 1 trial, repeated intracranial infusions of HER2-specific CAR T cells were well tolerated with no observed dose-limiting toxicities in three young adult patients with CNS tumors., Author(s): Nicholas A. Vitanza [sup.1] [sup.2] , Adam J. Johnson [sup.1] [sup.3] , Ashley L. Wilson [sup.1] [sup.3] , Christopher Brown [sup.3] [sup.4] , Jason K. Yokoyama [sup.1] [sup.3] , [...]

Published
2021
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10. Locoregional CAR T Cells for the Treatment of CNS Tumors in Children: Investigational Drug Service Pharmacy Activities.

Author

Vitanza, Nicholas A., Choe, Michelle, Brown, Christopher, Beebe, Adam, Kong, Ada, Rogers, Lisa, Jacob, Susan, Mano, Elena, Abuan, Kimberly, Mgebroff, Stephanie, Lindgren, Catherine, Gustafson, Joshua A., Wilson, Ashley L., Noll, Alyssa, Ronsley, Rebecca, Crotty, Erin E., Leary, Sarah E. S., Foster, Jessica B., Pinto, Navin, and Gust, Juliane

Subjects
T cells, CHIMERIC antigen receptors, TUMORS in children, CENTRAL nervous system, BRAIN tumors
Abstract

BACKGROUND: A major obstacle in translating the therapeutic potential of chimeric antigen receptor (CAR) T cells to children with central nervous system (CNS) tumors is the blood–brain barrier. To overcome this limitation, preclinical and clinical studies have supported the use of repeated, locoregional intracranial CAR T-cell delivery. However, there is limited literature available describing the process for the involvement of an investigational drug service (IDS) pharmacy, particularly in the setting of a children’s hospital with outpatient dosing for CNS tumors. OBJECTIVES: To describe Seattle Children’s Hospital’s experience in clinically producing CAR T cells and the implementation of IDS pharmacy practices used to deliver more than 300 intracranial CAR T-cell doses to children, as well as to share how we refined the processing techniques from CAR T-cell generation to the thawing of fractionated doses for intracranial delivery. METHODS: Autologous CD4+ and CD8+ T cells were collected and transduced to express HER2, EGFR, or B7-H3–specific CAR T cells. Cryopreserved CAR T cells were thawed by the IDS pharmacy before intracranial delivery to patients with recurrent/refractory CNS tumors or with diffuse intrinsic pontine glioma/diffuse midline glioma. RESULTS: The use of a thaw-and-dilute procedure for cryopreserved individual CAR T-cell doses provides reliable viability and is more efficient than typical thaw-and-wash protocols. Cell viability with the thaw-and-dilute protocol was approximately 75% and was always within 10% of the viability assessed at cryopreservation. Cell viability was preserved through 6 hours after thawing, which exceeded the 1-hour time frame from thawing to infusion. CONCLUSION: As the field of adoptive immunotherapy grows and continues to bring hope to patients with fatal CNS malignancies, it is critical to focus on improving the preparatory steps for CAR T-cell delivery. [ABSTRACT FROM AUTHOR]

Published
2024

11. Differential protein-protein interactions underlie signaling mediated by the TCR and a 4-1BB domain–containing CAR

Author

Ritmeester-Loy, Samuel A., primary, Draper, Isabella H., additional, Bueter, Eric C., additional, Lautz, Jonathan D., additional, Zhang-Wong, Yue, additional, Gustafson, Joshua A., additional, Wilson, Ashley L., additional, Lin, Chenwei, additional, Gafken, Philip R., additional, Jensen, Michael C., additional, Orentas, Rimas, additional, and Smith, Stephen E. P., additional

Published
2024
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12. Abstract LB121: BrainChild-03: Intraventricular B7-H3 CAR T cells for recurrent/refractory central nervous system tumors and non-pontine diffuse midline glioma in children and young adults

Author

Vitanza, Nicholas A., primary, Gust, Juliane, additional, Ronsley, Rebecca, additional, Wilson, Ashley L., additional, Huang, Wenjun, additional, Seidel, Kristy, additional, Albert, Catherine M., additional, Pinto, Navin, additional, Orentas, Rimas J., additional, Gardner, Rebecca A., additional, Jensen, Michael C., additional, and Park, Julie R., additional

Published
2023
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13. Data from Modified Manufacturing Process Modulates CD19CAR T-cell Engraftment Fitness and Leukemia-Free Survival in Pediatric and Young Adult Subjects

Author

Ceppi, Francesco, primary, Wilson, Ashley L., primary, Annesley, Colleen, primary, Kimmerly, Gabriella R., primary, Summers, Corinne, primary, Brand, Adam, primary, Seidel, Kristy, primary, Wu, Qian Vicky, primary, Beebe, Adam, primary, Brown, Christopher, primary, Mgebroff, Stephanie, primary, Lindgren, Catherine, primary, Rawlings-Rhea, Stephanie D., primary, Huang, Wenjun, primary, Pulsipher, Michael A., primary, Wayne, Alan S., primary, Park, Julie R., primary, Jensen, Michael C., primary, and Gardner, Rebecca A., primary

Published
2023
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14. Supplementary Data from Modified Manufacturing Process Modulates CD19CAR T-cell Engraftment Fitness and Leukemia-Free Survival in Pediatric and Young Adult Subjects

Author

Ceppi, Francesco, primary, Wilson, Ashley L., primary, Annesley, Colleen, primary, Kimmerly, Gabriella R., primary, Summers, Corinne, primary, Brand, Adam, primary, Seidel, Kristy, primary, Wu, Qian Vicky, primary, Beebe, Adam, primary, Brown, Christopher, primary, Mgebroff, Stephanie, primary, Lindgren, Catherine, primary, Rawlings-Rhea, Stephanie D., primary, Huang, Wenjun, primary, Pulsipher, Michael A., primary, Wayne, Alan S., primary, Park, Julie R., primary, Jensen, Michael C., primary, and Gardner, Rebecca A., primary

Published
2023
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15. Data from Intraventricular B7-H3 CAR T Cells for Diffuse Intrinsic Pontine Glioma: Preliminary First-in-Human Bioactivity and Safety

Author

Vitanza, Nicholas A., primary, Wilson, Ashley L., primary, Huang, Wenjun, primary, Seidel, Kristy, primary, Brown, Christopher, primary, Gustafson, Joshua A., primary, Yokoyama, Jason K., primary, Johnson, Adam J., primary, Baxter, Blake A., primary, Koning, Ryan W., primary, Reid, Aquene N., primary, Meechan, Michael, primary, Biery, Matthew C., primary, Myers, Carrie, primary, Rawlings-Rhea, Stephanie D., primary, Albert, Catherine M., primary, Browd, Samuel R., primary, Hauptman, Jason S., primary, Lee, Amy, primary, Ojemann, Jeffrey G., primary, Berens, Michael E., primary, Dun, Matthew D., primary, Foster, Jessica B., primary, Crotty, Erin E., primary, Leary, Sarah E.S., primary, Cole, Bonnie L., primary, Perez, Francisco A., primary, Wright, Jason N., primary, Orentas, Rimas J., primary, Chour, Tony, primary, Newell, Evan W., primary, Whiteaker, Jeffrey R., primary, Zhao, Lei, primary, Paulovich, Amanda G., primary, Pinto, Navin, primary, Gust, Juliane, primary, Gardner, Rebecca A., primary, Jensen, Michael C., primary, and Park, Julie R., primary

Published
2023
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16. Supplementary Figures 1-4 with figure legends from Differential Expression of Homing Receptor Ligands on Tumor-Associated Vasculature that Control CD8 Effector T-cell Entry

Author

Woods, Amber N., primary, Wilson, Ashley L., primary, Srivinisan, Nithya, primary, Zeng, Jianhao, primary, Dutta, Arun B., primary, Peske, J. David, primary, Tewalt, Eric F., primary, Gregg, Randal K., primary, Ferguson, Andrew R., primary, and Engelhard, Victor H., primary

Published
2023
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18. Pathogenetics of alveolar capillary dysplasia with misalignment of pulmonary veins

Author

Szafranski, Przemyslaw, Gambin, Tomasz, Dharmadhikari, Avinash V., Akdemir, Kadir Caner, Jhangiani, Shalini N., Schuette, Jennifer, Godiwala, Nihal, Yatsenko, Svetlana A., Sebastian, Jessica, Madan-Khetarpal, Suneeta, Surti, Urvashi, Abellar, Rosanna G., Bateman, David A., Wilson, Ashley L., Markham, Melinda H., Slamon, Jill, Santos-Simarro, Fernando, Palomares, María, Nevado, Julián, Lapunzina, Pablo, Chung, Brian Hon-Yin, Wong, Wai-Lap, Chu, Yoyo Wing Yiu, Mok, Gary Tsz Kin, Kerem, Eitan, Reiter, Joel, Ambalavanan, Namasivayam, Anderson, Scott A., Kelly, David R., Shieh, Joseph, Rosenthal, Taryn C., Scheible, Kristin, Steiner, Laurie, Iqbal, M. Anwar, McKinnon, Margaret L., Hamilton, Sara Jane, Schlade-Bartusiak, Kamilla, English, Dawn, Hendson, Glenda, Roeder, Elizabeth R., DeNapoli, Thomas S., Littlejohn, Rebecca Okashah, Wolff, Daynna J., Wagner, Carol L., Yeung, Alison, Francis, David, Fiorino, Elizabeth K., Edelman, Morris, Fox, Joyce, Hayes, Denise A., Janssens, Sandra, De Baere, Elfride, Menten, Björn, Loccufier, Anne, Vanwalleghem, Lieve, Moerman, Philippe, Sznajer, Yves, Lay, Amy S., Kussmann, Jennifer L., Chawla, Jasneek, Payton, Diane J., Phillips, Gael E., Brosens, Erwin, Tibboel, Dick, de Klein, Annelies, Maystadt, Isabelle, Fisher, Richard, Sebire, Neil, Male, Alison, Chopra, Maya, Pinner, Jason, Malcolm, Girvan, Peters, Gregory, Arbuckle, Susan, Lees, Melissa, Mead, Zoe, Quarrell, Oliver, Sayers, Richard, Owens, Martina, Shaw-Smith, Charles, Lioy, Janet, McKay, Eileen, de Leeuw, Nicole, Feenstra, Ilse, Spruijt, Liesbeth, Elmslie, Frances, Thiruchelvam, Timothy, Bacino, Carlos A., Langston, Claire, Lupski, James R., Sen, Partha, Popek, Edwina, and Stankiewicz, Paweł

Published
2016
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19. Intraventricular B7-H3 CAR T Cells for Diffuse Intrinsic Pontine Glioma: Preliminary First-in-Human Bioactivity and Safety

Author

Vitanza, Nicholas A., primary, Wilson, Ashley L., additional, Huang, Wenjun, additional, Seidel, Kristy, additional, Brown, Christopher, additional, Gustafson, Joshua A., additional, Yokoyama, Jason K., additional, Johnson, Adam J., additional, Baxter, Blake A., additional, Koning, Ryan W., additional, Reid, Aquene N., additional, Meechan, Michael, additional, Biery, Matthew C., additional, Myers, Carrie, additional, Rawlings-Rhea, Stephanie D., additional, Albert, Catherine M., additional, Browd, Samuel R., additional, Hauptman, Jason S., additional, Lee, Amy, additional, Ojemann, Jeffrey G., additional, Berens, Michael E., additional, Dun, Matthew D., additional, Foster, Jessica B., additional, Crotty, Erin E., additional, Leary, Sarah E.S., additional, Cole, Bonnie L., additional, Perez, Francisco A., additional, Wright, Jason N., additional, Orentas, Rimas J., additional, Chour, Tony, additional, Newell, Evan W., additional, Whiteaker, Jeffrey R., additional, Zhao, Lei, additional, Paulovich, Amanda G., additional, Pinto, Navin, additional, Gust, Juliane, additional, Gardner, Rebecca A., additional, Jensen, Michael C., additional, and Park, Julie R., additional

Published
2022
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22. Modified Manufacturing Process Modulates CD19CAR T-cell Engraftment Fitness and Leukemia-Free Survival in Pediatric and Young Adult Subjects

Author

Ceppi, Francesco, primary, Wilson, Ashley L., additional, Annesley, Colleen, additional, Kimmerly, Gabriella R., additional, Summers, Corinne, additional, Brand, Adam, additional, Seidel, Kristy, additional, Wu, Qian Vicky, additional, Beebe, Adam, additional, Brown, Christopher, additional, Mgebroff, Stephanie, additional, Lindgren, Catherine, additional, Rawlings-Rhea, Stephanie D., additional, Huang, Wenjun, additional, Pulsipher, Michael A., additional, Wayne, Alan S., additional, Park, Julie R., additional, Jensen, Michael C., additional, and Gardner, Rebecca A., additional

Published
2022
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24. A Phase 2 Trial of SCRI-CAR19 Demonstrates Favorable Leukemia-Free Survival (LFS), with a Pilot Study of CD19-Expressing T-Cell Antigen Presenting Cells (CD19t T-APCs) Demonstrating Safety and Tolerability

Author

Annesley, Colleen, Seidel, Kristy, Wu, Qian Vicky, Summers, Corinne, Wayne, Alan S., Agrawal, Anurag K., Pulsipher, Michael A., Brown, Christopher, Mgebroff, Stephanie, Lindgren, Catherine, Wilson, Ashley L., Huang, Wenjun, Park, Julie R., Jensen, Michael C., and Gardner, Rebecca A.

Published
2023
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25. Supplemental Material, sj-pdf-3-jcn-10.1177_08830738211001209 - Availability of Services and Caregiver Burden: Supporting Individuals With Neurogenetic Conditions During the COVID-19 Pandemic

Author

Kowanda, Michelle, Cartner, Lindsey, Kentros, Catherine, Geltzeiler, Alexa R., Singer, Kaitlyn E., W. Curtis Weaver, Lehman, Christopher D., Smith, Simone, Smith, Rebecca Sheedy, Walsh, Lauren Kasparson, Diehl, Katharine, Nagpal, Natalie, Brooks, Elizabeth, Mebane, Caroline M., Wilson, Ashley L., Marvin, Alison R., L. Casey White, J. Kiely Law, Jensen, William, Daniels, Amy M., Tjernagel, Jennifer, LeeAnne Green Snyder, Taylor, Cora M., and Chung, Wendy K.

Subjects
FOS: Clinical medicine, 111403 Paediatrics, 110904 Neurology and Neuromuscular Diseases, Neuroscience
Abstract

Supplemental Material, sj-pdf-3-jcn-10.1177_08830738211001209 for Availability of Services and Caregiver Burden: Supporting Individuals With Neurogenetic Conditions During the COVID-19 Pandemic by Michelle Kowanda, Lindsey Cartner, Catherine Kentros, Alexa R. Geltzeiler, Kaitlyn E. Singer, W. Curtis Weaver, Christopher D. Lehman, Simone Smith, Rebecca Sheedy Smith, Lauren Kasparson Walsh, Katharine Diehl, Natalie Nagpal, Elizabeth Brooks, Caroline M. Mebane, Ashley L. Wilson, Alison R. Marvin, L. Casey White, J. Kiely Law, William Jensen, Amy M. Daniels, Jennifer Tjernagel, LeeAnne Green Snyder, Cora M. Taylor and Wendy K. Chung in Journal of Child Neurology

Published
2021
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26. Supplemental Material, sj-pdf-1-jcn-10.1177_08830738211001209 - Availability of Services and Caregiver Burden: Supporting Individuals With Neurogenetic Conditions During the COVID-19 Pandemic

Author

Kowanda, Michelle, Cartner, Lindsey, Kentros, Catherine, Geltzeiler, Alexa R., Singer, Kaitlyn E., W. Curtis Weaver, Lehman, Christopher D., Smith, Simone, Smith, Rebecca Sheedy, Walsh, Lauren Kasparson, Diehl, Katharine, Nagpal, Natalie, Brooks, Elizabeth, Mebane, Caroline M., Wilson, Ashley L., Marvin, Alison R., L. Casey White, J. Kiely Law, Jensen, William, Daniels, Amy M., Tjernagel, Jennifer, LeeAnne Green Snyder, Taylor, Cora M., and Chung, Wendy K.

Subjects
FOS: Clinical medicine, 111403 Paediatrics, 110904 Neurology and Neuromuscular Diseases, Neuroscience
Abstract

Supplemental Material, sj-pdf-1-jcn-10.1177_08830738211001209 for Availability of Services and Caregiver Burden: Supporting Individuals With Neurogenetic Conditions During the COVID-19 Pandemic by Michelle Kowanda, Lindsey Cartner, Catherine Kentros, Alexa R. Geltzeiler, Kaitlyn E. Singer, W. Curtis Weaver, Christopher D. Lehman, Simone Smith, Rebecca Sheedy Smith, Lauren Kasparson Walsh, Katharine Diehl, Natalie Nagpal, Elizabeth Brooks, Caroline M. Mebane, Ashley L. Wilson, Alison R. Marvin, L. Casey White, J. Kiely Law, William Jensen, Amy M. Daniels, Jennifer Tjernagel, LeeAnne Green Snyder, Cora M. Taylor and Wendy K. Chung in Journal of Child Neurology

Published
2021
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27. Supplemental Material, sj-pdf-2-jcn-10.1177_08830738211001209 - Availability of Services and Caregiver Burden: Supporting Individuals With Neurogenetic Conditions During the COVID-19 Pandemic

Author

Kowanda, Michelle, Cartner, Lindsey, Kentros, Catherine, Geltzeiler, Alexa R., Singer, Kaitlyn E., W. Curtis Weaver, Lehman, Christopher D., Smith, Simone, Smith, Rebecca Sheedy, Walsh, Lauren Kasparson, Diehl, Katharine, Nagpal, Natalie, Brooks, Elizabeth, Mebane, Caroline M., Wilson, Ashley L., Marvin, Alison R., L. Casey White, J. Kiely Law, Jensen, William, Daniels, Amy M., Tjernagel, Jennifer, LeeAnne Green Snyder, Taylor, Cora M., and Chung, Wendy K.

Subjects
FOS: Clinical medicine, 111403 Paediatrics, 110904 Neurology and Neuromuscular Diseases, Neuroscience
Abstract

Supplemental Material, sj-pdf-2-jcn-10.1177_08830738211001209 for Availability of Services and Caregiver Burden: Supporting Individuals With Neurogenetic Conditions During the COVID-19 Pandemic by Michelle Kowanda, Lindsey Cartner, Catherine Kentros, Alexa R. Geltzeiler, Kaitlyn E. Singer, W. Curtis Weaver, Christopher D. Lehman, Simone Smith, Rebecca Sheedy Smith, Lauren Kasparson Walsh, Katharine Diehl, Natalie Nagpal, Elizabeth Brooks, Caroline M. Mebane, Ashley L. Wilson, Alison R. Marvin, L. Casey White, J. Kiely Law, William Jensen, Amy M. Daniels, Jennifer Tjernagel, LeeAnne Green Snyder, Cora M. Taylor and Wendy K. Chung in Journal of Child Neurology

Published
2021
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28. Availability of Services and Caregiver Burden: Supporting Individuals With Neurogenetic Conditions During the COVID-19 Pandemic

Author

Kowanda, Michelle, primary, Cartner, Lindsey, additional, Kentros, Catherine, additional, Geltzeiler, Alexa R., additional, Singer, Kaitlyn E., additional, Weaver, W. Curtis, additional, Lehman, Christopher D., additional, Smith, Simone, additional, Smith, Rebecca Sheedy, additional, Walsh, Lauren Kasparson, additional, Diehl, Katharine, additional, Nagpal, Natalie, additional, Brooks, Elizabeth, additional, Mebane, Caroline M., additional, Wilson, Ashley L., additional, Marvin, Alison R., additional, White, L. Casey, additional, Law, J. Kiely, additional, Jensen, William, additional, Daniels, Amy M., additional, Tjernagel, Jennifer, additional, Snyder, LeeAnne Green, additional, Taylor, Cora M., additional, and Chung, Wendy K., additional

Published
2021
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29. Abstract LB-141: Increased epigenetic plasticity and tumor heterogeneity predict resistance to CD19 CAR T cell therapy in pre-treatment pediatric acute lymphoblastic leukemia

Author

Masih, Katherine E., primary, Abdelmaksoud, Abdalla, additional, Milewski, David, additional, Gryder, Berkley E., additional, Gardner, Rebecca, additional, Wilson, Ashley L., additional, Stanton, Benjamin Z., additional, Song, Young K., additional, Wang, Chaoyu, additional, Wen, Xinyu, additional, Adebola, Serifat, additional, Rae, Zachary, additional, Cheuk, Adam, additional, Lack, Justin, additional, Altan-Bonnet, Gregoire, additional, Kelly, Michael, additional, Wei, Jun, additional, Jensen, Michael, additional, and Orentas, Rimas J., additional

Published
2020
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30. Impact of patient education videos on genetic counseling outcomes after exome sequencing

Author

Hernan, Rebecca, primary, Cho, Megan T., additional, Wilson, Ashley L., additional, Ahimaz, Priyanka, additional, Au, Catherine, additional, Berger, Sara M., additional, Guzman, Edwin, additional, Primiano, Michelle, additional, Shaw, Jessica E., additional, Ross, Meredith, additional, Tabanfar, Leyla, additional, Chilton, Ilana, additional, Griffin, Emily, additional, Ratner, Chana, additional, Anyane-Yeboa, Kwame, additional, Iglesias, Alejandro, additional, Pisani, Laura, additional, Roohi, Jasmin, additional, Duong, Jimmy, additional, Martinez, Josue, additional, Appelbaum, Paul, additional, Klitzman, Robert, additional, Ottman, Ruth, additional, Chung, Wendy K., additional, and Wynn, Julia, additional

Published
2020
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33. Bohring–Opitz syndrome (BOS) with a new ASXL1 pathogenic variant: Review of the most prevalent molecular and phenotypic features of the syndrome

Author

Dangiolo, Silvana Beatriz, Wilson, Ashley L., Jobanputra, Vaidehi, and Anyane-Yeboa, Kwame

Subjects
Pancreatitis, FOS: Biological sciences, Medical genetics, Developmental biology, Genetics, Genetic disorders--Diagnosis, Cystic fibrosis--Patients, Medical sciences
Abstract

Bohring–Opitz syndrome (BOS) was first described by Bohring et al. [1999]. The authors reported four cases which had several features in common, including a prominent metopic suture, hypertelorism, exophthalmos, cleft lip and palate, limb anomalies, as well as difficulty feeding with severe developmental delays. In almost 50% of cases that meet the clinical criteria for BOS, de novo frameshift and nonsense mutations in the ASXL1 gene have been detected, suggesting that loss of function of this gene is a major cause. We report on the clinical characterization of one young female patient who was evaluated because of severe developmental delays, failure to thrive, and multiple minor anomalies and was clinically diagnosed with BOS. Whole exome sequencing analysis detected one novel disruptive frameshift mutation in the ASXL1 gene and we were also able to confirm the presence of two CFTR mutations associated with her chronic pancreatitis with acute severe breakthrough attacks requiring multiple ICU admissions. This latter complication of pancreatitis further contributed to the complexity of the clinical presentation and represents an independent genetic finding. Our case report emphasizes the importance of highly specific phenotypic characterization of patients with complex phenotypes before proceeding with molecular studies. That approach will lead to more accurate molecular data interpretation and better clinical genetic diagnosis, particularly for those patients with rare, difficult-to-diagnose disorders.

Published
2015
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36. De novo mutations in PURA are associated with hypotonia and developmental delay

Author

Tanaka, Akemi J., primary, Bai, Renkui, additional, Cho, Megan T., additional, Anyane-Yeboa, Kwame, additional, Ahimaz, Priyanka, additional, Wilson, Ashley L., additional, Kendall, Fran, additional, Hay, Beverly, additional, Moss, Timothy, additional, Nardini, Monica, additional, Bauer, Mislen, additional, Retterer, Kyle, additional, Juusola, Jane, additional, and Chung, Wendy K., additional

Published
2015
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37. Breast cancer antiestrogen resistance 3 (BCAR3) promotes cell motility by regulating actin cytoskeletal and adhesion remodeling in invasive breast cancer cells.

Author

Wilson, Ashley L, Schrecengost, Randy S, Guerrero, Michael S, Thomas, Keena S, Bouton, Amy H, Wilson, Ashley L, Schrecengost, Randy S, Guerrero, Michael S, Thomas, Keena S, and Bouton, Amy H

Abstract

Metastatic breast cancer is incurable. In order to improve patient survival, it is critical to develop a better understanding of the molecular mechanisms that regulate metastasis and the underlying process of cell motility. Here, we focus on the role of the adaptor molecule Breast Cancer Antiestrogen Resistance 3 (BCAR3) in cellular processes that contribute to cell motility, including protrusion, adhesion remodeling, and contractility. Previous work from our group showed that elevated BCAR3 protein levels enhance cell migration, while depletion of BCAR3 reduces the migratory and invasive capacities of breast cancer cells. In the current study, we show that BCAR3 is necessary for membrane protrusiveness, Rac1 activity, and adhesion disassembly in invasive breast cancer cells. We further demonstrate that, in the absence of BCAR3, RhoA-dependent signaling pathways appear to predominate, as evidenced by an increase in RhoA activity, ROCK-mediated phosphorylation of myosin light chain II, and large ROCK/mDia1-dependent focal adhesions. Taken together, these data establish that BCAR3 functions as a positive regulator of cytoskeletal remodeling and adhesion turnover in invasive breast cancer cells through its ability to influence the balance between Rac1 and RhoA signaling. Considering that BCAR3 protein levels are elevated in advanced breast cancer cell lines and enhance breast cancer cell motility, we propose that BCAR3 functions in the transition to advanced disease by triggering intracellular signaling events that are essential to the metastatic process.

Published
2013

44. Intraventricular B7-H3 CAR T Cells for Diffuse Intrinsic Pontine Glioma: Preliminary First-in-Human Bioactivity and Safety.

Author

Vitanza NA, Wilson AL, Huang W, Seidel K, Brown C, Gustafson JA, Yokoyama JK, Johnson AJ, Baxter BA, Koning RW, Reid AN, Meechan M, Biery MC, Myers C, Rawlings-Rhea SD, Albert CM, Browd SR, Hauptman JS, Lee A, Ojemann JG, Berens ME, Dun MD, Foster JB, Crotty EE, Leary SES, Cole BL, Perez FA, Wright JN, Orentas RJ, Chour T, Newell EW, Whiteaker JR, Zhao L, Paulovich AG, Pinto N, Gust J, Gardner RA, Jensen MC, and Park JR

Subjects
Humans, B7 Antigens, T-Lymphocytes, Diffuse Intrinsic Pontine Glioma, Brain Stem Neoplasms therapy
Abstract

Diffuse intrinsic pontine glioma (DIPG) remains a fatal brainstem tumor demanding innovative therapies. As B7-H3 (CD276) is expressed on central nervous system (CNS) tumors, we designed B7-H3-specific chimeric antigen receptor (CAR) T cells, confirmed their preclinical efficacy, and opened BrainChild-03 (NCT04185038), a first-in-human phase I trial administering repeated locoregional B7-H3 CAR T cells to children with recurrent/refractory CNS tumors and DIPG. Here, we report the results of the first three evaluable patients with DIPG (including two who enrolled after progression), who received 40 infusions with no dose-limiting toxicities. One patient had sustained clinical and radiographic improvement through 12 months on study. Patients exhibited correlative evidence of local immune activation and persistent cerebrospinal fluid (CSF) B7-H3 CAR T cells. Targeted mass spectrometry of CSF biospecimens revealed modulation of B7-H3 and critical immune analytes (CD14, CD163, CSF-1, CXCL13, and VCAM-1). Our data suggest the feasibility of repeated intracranial B7-H3 CAR T-cell dosing and that intracranial delivery may induce local immune activation., Significance: This is the first report of repeatedly dosed intracranial B7-H3 CAR T cells for patients with DIPG and includes preliminary tolerability, the detection of CAR T cells in the CSF, CSF cytokine elevations supporting locoregional immune activation, and the feasibility of serial mass spectrometry from both serum and CSF. This article is highlighted in the In This Issue feature, p. 1., (©2022 The Authors; Published by the American Association for Cancer Research.)

Published
2023
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45. De novo mutations in PURA are associated with hypotonia and developmental delay.

Author

Tanaka AJ, Bai R, Cho MT, Anyane-Yeboa K, Ahimaz P, Wilson AL, Kendall F, Hay B, Moss T, Nardini M, Bauer M, Retterer K, Juusola J, and Chung WK

Abstract

PURA is the leading candidate gene responsible for the developmental phenotype in the 5q31.3 microdeletion syndrome. De novo mutations in PURA were recently reported in 15 individuals with developmental features similar to the 5q31.3 microdeletion syndrome. Here we describe six unrelated children who were identified by clinical whole-exome sequencing (WES) to have novel de novo variants in PURA with a similar phenotype of hypotonia and developmental delay and frequently associated with seizures. The protein Purα (encoded by PURA) is involved in neuronal proliferation, dendrite maturation, and the transport of mRNA to translation sites during neuronal development. Mutations in PURA may alter normal brain development and impair neuronal function, leading to developmental delay and the seizures observed in patients with mutations in PURA.

Published
2015
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