Kun Wei Song

All Publications

• Limited evidence of pseudoprogression following immune checkpoint inhibitor (ICI) therapy in glioblastoma. Neuro-oncology advances Rhee, J. Y., Ospina Botero, J. P., Nelson, T., Song, K. W., Parsons, M. W., Gerstner, E. R., Dietrich, J. 2026; 8 (1): vdaf232

  Abstract

  "Pseudoprogression" following immune checkpoint inhibitors (ICI) in glioblastoma is often considered in case of radiographic progression. To better characterize the frequency of this phenomenon in glioblastoma, we reviewed the imaging response characteristics of a total of 55 patients treated with ICI in the setting of recurrent (n = 45) or newly diagnosed (n = 10) disease. There was no evidence of pseudoprogression related to ICI-monotherapy in the entire cohort.

  View details for DOI 10.1093/noajnl/vdaf232

  View details for PubMedID 41613042

  View details for PubMedCentralID PMC12850524

• CAR T cells for pediatric CNS tumors: Clinical experience and reverse translational opportunities. Molecular therapy. Oncology Song, K. W., Kaur, A. P., Ramakrishna, S. 2026; 34 (2): 201196

  Abstract

  Central nervous system (CNS) tumors are one of the leading causes of cancer-related mortality in children and young adults. Despite decades of research, effective treatments for these tumors are still lacking and new therapeutic options are urgently needed. While chimeric antigen receptor (CAR) T cell therapies have revolutionized the treatment of hematologic malignancies, these cellular therapies are only beginning to show evidence of activity in CNS tumors. In this review, we will discuss current CAR T cell clinical trials with a focus on trials in the pediatric CNS tumor space. In addition, we will explore the power of reverse translation-defined by applying clinical research insights from bedside-to-bench to understand the mechanisms of CAR T cell success or failure in patients-for CNS tumor CAR T cells. Employing the full cycle of translational research, novel CAR T cell therapies are being tested in the clinic and reverse translational studies are being assessed at the bench. Combining these data, the CNS CAR T cell field is poised to iteratively improve CAR T cells for children and young adults with CNS tumors.

  View details for DOI 10.1016/j.omton.2026.201196

  View details for PubMedID 42022537

  View details for PubMedCentralID PMC13098344

• Peripheral Nerve Infiltration by CAR T-cells in a Case of Mononeuritis Multiplex after Ciltacabtagene Autoleucel. Blood cell therapy Hosoya, H., Song, K. W., Dyson, K., Dufort, G., Sahaf, B., Rosenberg, A., Sidana, S., Miklos, D., Vogel, H., Fernandez-Pol, S., Muppidi, S., Scott, B., Mikkilineni, L. 2026; 9 (2): 55-59

  Abstract

  Delayed neurological toxicity, distinct from acute immune effector cell-associated neurotoxicity (ICANS), is an emerging complication of ciltacabtagene autoleucel (cilta-cel), a B-cell maturation antigen (BCMA)-targeting CAR T-cell therapy. Peripheral nerve (PN) involvement has been described as cranial nerve palsies or Guillain-Barré-like syndromes, yet the underlying mechanisms remain poorly defined. We describe a case of asymmetric, immune-mediated multifocal PN involvement, clinically resembling autoimmune, T-cell-mediated mononeuritis multiplex (MNM), occurring after cilta-cel therapy. Several months post-infusion, the patient developed progressive asymmetric sensorimotor neuropathy preceded by bilateral facial nerve palsies. Electrodiagnostic studies confirmed multifocal axonal involvement, and nerve biopsy revealed Wallerian degeneration with CD3+ and CAR+ T-cell infiltration in the absence of BCMA expression on neural tissue. These findings suggest that delayed neurotoxicity may arise from immune-mediated, BCMA-independent mechanisms involving off-target tissue inflammation. Although CAR T-cells were detected within the affected nerve tissue, their pathogenic significance remains unclear and may reflect antigen-independent infiltration, immune activation or secondary bystander recruitment in the setting of immune dysregulation. The patient demonstrated marked neurological recovery following corticosteroids and cyclophosphamide, an immunosuppressive regimen modeled on MNM treatment. This case underscores the importance of comprehensive neurodiagnostic evaluation and early immunosuppression in managing delayed, non-ICANS CAR T-cell neurotoxicity.

  View details for DOI 10.31547/bct-2025-027

  View details for PubMedID 42253352

  View details for PubMedCentralID PMC13237437

• Longitudinal single-cell atlas of GD2-CAR T cell therapy in H3K27M-mutant diffuse midline glioma identifies humoral and cellular anti-CAR immunity Chen, Y., Song, K., Desai, M. H., Huang, Y., Iswari, N., Ehlinger, Z. J., Daghagh, H., Koch, M. R., Reynolds, K., Mo, K. C., Tsui, K. C., Rietberg, S., Hamilton, M. P., Prabhu, S., Partap, S., Mahdi, J., Egeler, E., Feldman, S. A., Heitzeneder, S., Yamada-Hunter, S. A., Sotillo, E., Sahaf, B., Good, Z., Monje, M., Ramakrishna, S., Mackall, C. L. AMER ASSOC CANCER RESEARCH. 2026

  View details for DOI 10.1158/1538-7445.AM2026-6463

  View details for Web of Science ID 001734512300036

• Immunotherapies for Central Nervous System Tumors and their Neurologic Complications. Current neurology and neuroscience reports Song, K. W., Scott, B. J. 2026; 26 (1): 7

  Abstract

  To discuss the landscape of immunotherapy trials for central nervous system (CNS) tumors including immune checkpoint inhibitors, vaccine therapies, oncolytic viruses, and chimeric antigen receptor T-cell therapy as well as explore the corresponding spectrum of neurologic toxicities.As more clinical trials are underway in CNS tumors, we are starting to appreciate both the promise of immunotherapy and current limitations. While a subset of patients demonstrate benefit, immunotherapy trials have not transformed CNS tumor outcomes, which prompts the field to consider next generation therapies and combinatorial approaches. In addition, novel toxicities of CNS immunotherapy are being described such as tumor-inflammation associated neurotoxicity. Immunotherapy in CNS tumor is still in its nascent stages and shows early signs of promise. However, immunotherapeutic approaches in CNS tumors need to account for the unique physiology of the CNS and the corresponding neurologic toxicities that are associated with therapies targeting the CNS. Elucidating both will set the stage for advancement of safe and effective immunotherapy for CNS tumors.

  View details for DOI 10.1007/s11910-025-01477-9

  View details for PubMedID 41484655

  View details for PubMedCentralID 5637314

• SINGLE-CELL LANDSCAPE OF B7H3-CAR T THERAPY IN GLIOMA: MECHANISMS OF RESISTANCE AND SIGNATURES OF LONG-TERM RESPONSE Chen, Y., Song, K., Desai, M., Ehlinger, Z., Daghagh, H., Rietberg, S., Feeney, A., Tanner, K., Dyson, K., Stockdale, B., Dhapola, G., Lohman, C., Patil, S., Kuo, A., Good, Z., Prabhu, S., Kong, K., Egeler, E., Beebe, B., Carr, C., Mahdi, J., Bertrand, S., Iv, M., Threlkeld, Z., Charu, V., Tunuguntla, R., Therkelsen, K., Nagpal, S., Monje, M., Scott, B., Li, G., Lim, M., Sotillo, E., Sahaf, B., Ramakrishna, S., Thomas, R., Mackall, C. OXFORD UNIV PRESS INC. 2025: v13

  View details for DOI 10.1093/neuonc/noaf201.0044

  View details for Web of Science ID 001612039700001

• GD2 as an Immunotherapeutic Target in Oligodendroglioma: Preclinical Evidence Supporting CAR T Cell Therapy Song, K., Trivedi, V., Geraghty, A., Otubu, G., Kim, Y., Dyson, K., Mackall, C., Lopez, G., Monje, M. OXFORD UNIV PRESS INC. 2025: v372

  View details for DOI 10.1093/neuonc/noaf201.1469

  View details for Web of Science ID 001613222500002

• Clinical Impact of Intratumoral Cyst Decompression in Diffuse Intrinsic Pontine Glioma Patients: A Retrospective Analysis Guinle, M., Kim, L., Casazza, M., Ravi, K., Song, K., Mahdi, J., Nguyen, T., Grant, G., Monje, M., Prolo, L. OXFORD UNIV PRESS INC. 2025: v419

  View details for DOI 10.1093/neuonc/noaf201.1656

  View details for Web of Science ID 001613255100014

• EVALUATION OF B7-H3 IMMUNOHISTOCHEMISTRY IN HIGH-GRADE GLIOMAS FOR CHIMERIC ANTIGEN RECEPTOR T-CELL PREDICTIVE TESTING Dyson, K., Zuraski, C., Solomon, D., Li, G., Stocksdale, B., Song, K., Mahdi, J., Tanner, K., Bertrand, S., Iv, M., Threlkeld, Z., Ramakrishna, S., Sahaf, B., Egeler, E., Tunuguntla, R., Therkelsen, K., Nagpal, S., Lim, M., Monje, M., Scott, B., Mackall, C., Charu, V., Thomas, R. OXFORD UNIV PRESS INC. 2025: v49-v50

  View details for DOI 10.1093/neuonc/noaf201.0195

  View details for Web of Science ID 001613247800006

• HUMANIZED ANTI-CAR ANTIBODIES AFFECT DURABLE RESPONSE TO GD2-CAR T-CELLS IN DIFFUSE MIDLINE GLIOMA Chen, Y., Song, K., Huang, Y., Iswari, N., Desai, M., Ehlinger, Z., Daghagh, H., Reynolds, K., Mahdi, J., Majzner, R., Richards, B., Kamens, J., Barsan, V., Campen, C., Partap, S., Madhav, D., Moon, J., Baggott, C., Fujimoto, M., Li, A., Jariwala, S., Mavroukakis, S., Egeler, E., Jacobs, A., Erickson, C., Kaur, A., Soundaranayagi, S., Rietberg, S., Schultz, L., Davis, K., Feldman, S., Heitzeneder, S., Yamada-Hunter, S., Sotillo, E., Good, Z., Sahaf, B., Monje, M., Ramakrishna, S., Mackall, C. OXFORD UNIV PRESS INC. 2025: v114

  View details for DOI 10.1093/neuonc/noaf201.0462

  View details for Web of Science ID 001613237000002

• A phase 1 study of B7H3 CAR-T cells administered intracranially in recurrent glioblastoma. Li, G., Stocksdale, B. R., Song, K., Mahdi, J., Tanner, K., Bertrand, S., Iv, M., Threlkeld, Z., Ramakrishna, S., Sahaf, B., Egeler, E., Charu, V., Tunuguntla, R., Therkelsen, K., Nagpal, S., Lim, M., Monje, M., Scott, B., Mackall, C., Thomas, R. LIPPINCOTT WILLIAMS & WILKINS. 2025: 2018

  View details for DOI 10.1200/JCO.2025.43.16_suppl.2018

  View details for Web of Science ID 001509526200001

• Complex neural-immune interactions shape glioma immunotherapy. Immunity Song, K. W., Lim, M., Monje, M. 2025

  Abstract

  Rich neural-immune interactions in the central nervous system (CNS) shape its function and create a unique immunological microenvironment for immunotherapy in CNS malignancies. Far from the now-debunked concept of CNS "immune privilege," it is now understood that unique immunological niches and constant immune surveillance of the brain contribute in multifaceted ways to brain health and robustly influence immunotherapy approaches for CNS cancers. Challenges include immune-suppressive and neurotoxicity-promoting crosstalk between brain, immune, and tumor cells. Developing effective immunotherapies for cancers of the nervous system will require a deeper understanding of these neural-immune-malignant cell interactions. Here, we review progress and challenges in immunotherapy for gliomas of the brain and spinal cord in light of these unique neural-immune interactions and highlight future work needed to optimize promising immunotherapies for gliomas.

  View details for DOI 10.1016/j.immuni.2025.04.017

  View details for PubMedID 40324379

• CAR T-cell therapy for gliomas. Current opinion in neurology Song, K. W., Scott, B. J. 2024; 37 (6): 672-681

  Abstract

  To review the landscape of chimeric antigen receptor T-cell (CAR T) therapy for gliomas as seen in recently published trials and discuss on-going challenges with new cancer immunotherapy treatments.Given how CAR T therapy has revolutionized the treatment of several hematologic malignancies, there has been increasing interest in using immunotherapy, and particularly CAR T therapy for gliomas. Within the past decade, several first in human trials have published early patient experiences showing treatment is generally well tolerated but with limited efficacy, which may be improving with newer evolutions in CAR T design to overcome known resistance mechanisms in glioma treatment.CAR T therapy is a promising avenue of treatment for high-grade gliomas, which have a universally poor prognosis as well as limited therapeutics. There are a growing number of CAR T clinical trials for CNS tumors and thus, an understanding of their treatment strategies, toxicity management, and overcoming resistance mechanisms will be important for both clinical practice and to identify areas for future research.

  View details for DOI 10.1097/WCO.0000000000001318

  View details for PubMedID 39498846

• Author Correction: Intravenous and intracranial GD2-CAR T cells for H3K27M+ diffuse midline gliomas. Nature Monje, M., Mahdi, J., Majzner, R., Yeom, K. W., Schultz, L. M., Richards, R. M., Barsan, V., Song, K. W., Kamens, J., Baggott, C., Kunicki, M., Rietberg, S. P., Lim, A. S., Reschke, A., Mavroukakis, S., Egeler, E., Moon, J., Patel, S., Chinnasamy, H., Erickson, C., Jacobs, A., Duh, A. K., Tunuguntla, R., Klysz, D. D., Fowler, C., Green, S., Beebe, B., Carr, C., Fujimoto, M., Brown, A. K., Petersen, A. G., McIntyre, C., Siddiqui, A., Lepori-Bui, N., Villar, K., Pham, K., Bove, R., Musa, E., Reynolds, W. D., Kuo, A., Prabhu, S., Rasmussen, L., Cornell, T. T., Partap, S., Fisher, P. G., Campen, C. J., Grant, G., Prolo, L., Ye, X., Sahaf, B., Davis, K. L., Feldman, S. A., Ramakrishna, S., Mackall, C. 2024

  View details for DOI 10.1038/s41586-024-08452-3

  View details for PubMedID 39613972

• Intravenous and intracranial GD2-CAR T cells for H3K27M+ diffuse midline gliomas. Nature Monje, M., Mahdi, J., Majzner, R., Yeom, K. W., Schultz, L. M., Richards, R. M., Barsan, V., Song, K. W., Kamens, J., Baggott, C., Kunicki, M., Rietberg, S. P., Lim, A. S., Reschke, A., Mavroukakis, S., Egeler, E., Moon, J., Patel, S., Chinnasamy, H., Erickson, C., Jacobs, A., Duh, A. K., Tunuguntla, R., Klysz, D. D., Fowler, C., Green, S., Beebe, B., Carr, C., Fujimoto, M., Brown, A. K., Petersen, A. G., McIntyre, C., Siddiqui, A., Lepori-Bui, N., Villar, K., Pham, K., Bove, R., Musa, E., Reynolds, W. D., Kuo, A., Prabhu, S., Rasmussen, L., Cornell, T. T., Partap, S., Fisher, P. G., Campen, C. J., Grant, G., Prolo, L., Ye, X., Sahaf, B., Davis, K. L., Feldman, S. A., Ramakrishna, S., Mackall, C. 2024

  Abstract

  H3K27M-mutant diffuse midline gliomas (DMGs) express high levels of the disialoganglioside GD2 (ref. 1). Chimeric antigen receptor-modified T cells targeting GD2 (GD2-CART) eradicated DMGs in preclinical models2. Arm A of Phase I trial no. NCT04196413 (ref. 3) administered one intravenous (IV) dose of autologous GD2-CART to patients with H3K27M-mutant pontine (DIPG) or spinal DMG (sDMG) at two dose levels (DL1, 1 × 106 kg-1; DL2, 3 × 106 kg-1) following lymphodepleting chemotherapy. Patients with clinical or imaging benefit were eligible for subsequent intracerebroventricular (ICV) intracranial infusions (10-30 × 106 GD2-CART). Primary objectives were manufacturing feasibility, tolerability and the identification of maximally tolerated IV dose. Secondary objectives included preliminary assessments of benefit. Thirteen patients enroled, with 11 receiving IV GD2-CART on study (n = 3 DL1 (3 DIPG); n = 8 DL2 (6 DIPG, 2 sDMG)). GD2-CART manufacture was successful for all patients. No dose-limiting toxicities occurred on DL1, but three patients experienced dose-limiting cytokine release syndrome on DL2, establishing DL1 as the maximally tolerated IV dose. Nine patients received ICV infusions, with no dose-limiting toxicities. All patients exhibited tumour inflammation-associated neurotoxicity, safely managed with intensive monitoring and care. Four patients demonstrated major volumetric tumour reductions (52, 54, 91 and 100%), with a further three patients exhibiting smaller reductions. One patient exhibited a complete response ongoing for over 30 months since enrolment. Nine patients demonstrated neurological benefit, as measured by a protocol-directed clinical improvement score. Sequential IV, followed by ICV GD2-CART, induced tumour regressions and neurological improvements in patients with DIPG and those with sDMG.

  View details for DOI 10.1038/s41586-024-08171-9

  View details for PubMedID 39537919

  View details for PubMedCentralID 5996391

• MYELOID POPULATIONS MODULATE GD2 CAR T CELL ACTIVITY IN DIFFUSE MIDLINE GLIOMA Ramakrishna, S., Geraghty, A., Good, Z., Desai, M., Mancusi, R., Mahdi, J., Song, K., Ehlinger, Z., Chen, Y., Hamilton, M., Rietberg, S., Majzner, R., Schultz, L., Richards, B., Kamens, J., Barsan, V., Campen, C., Partap, S., Moon, J., Baggott, C., Kunick, M., Fujimoto, M., Li, A., Jariwala, S., Mavroukakis, S., Egeler, E., Jacobs, A., Erickson, C., Prabhu, S., Davis, K., Feldman, S., Sahaf, B., Mackall, C., Monje, M. OXFORD UNIV PRESS INC. 2024

  View details for DOI 10.1093/neuonc/noae165.0652

  View details for Web of Science ID 001362581000011

• SEQUENTIAL INTRAVENOUS AND INTRACEREBROVENTRICULAR GD2-CAR T-CELL THERAPY FOR H3K27M-MUTATED DIFFUSE MIDLINE GLIOMAS Monje, M., Mahdi, J., Majzner, R. G., Yeom, K., Schultz, L. M., Richards, R., Barsan, V., Song, K., Kamens, J., Baggott, C., Kunicki, M., Lim, A., Reschke, A., Mavroukakis, S., Egeler, E., Moon, J., Patel, S., Chinnasamy, H., Erickson, C., Jacobs, A., Duh, A. K., Rietberg, S. P., Tunuguntla, R., Klysz, D., Fowler, C., Green, S., Beebe, B., Carr, C., Fujimoto, M., Brown, A., Peterson, A. G., Mcintyre, C., Siddiqui, A., Lepori-Bui, N., Villar, K., Pham, K., Bove, R., Musa, E., Reynolds, W., Kuo, A., Prabhu, S., Rasmussen, L., Cornell, T. T., Partap, S., Fisher, P. G., Campen, C. J., Grant, G., Prolo, L., Ye, X., Sahaf, B., Davis, K. L., Feldman, S. A., Ramakrishna, S., Mackall, C. L. OXFORD UNIV PRESS INC. 2024

  View details for DOI 10.1093/neuonc/noae064.100

  View details for Web of Science ID 001252720000169

• Neurotoxicity of Cancer Immunotherapies Including CAR T Cell Therapy. Current neurology and neuroscience reports Song, K. W., Scott, B. J., Lee, E. Q. 2023

  Abstract

  To outline the spectrum of neurotoxicity seen with approved immunotherapies and in pivotal clinical trials including immune checkpoint inhibitors, chimeric antigen receptor T-cell therapy, vaccine therapy, and oncolytic viruses.There has been an exponential growth in new immunotherapies, which has transformed the landscape of oncology treatment. With more widespread use of cancer immunotherapies, there have also been advances in characterization of its associated neurotoxicity, research into potential underlying mechanisms, and development of management guidelines. Increasingly, there is also mounting interest in long-term neurologic sequelae. Neurologic complications of immunotherapy can impact every aspect of the central and peripheral nervous system. Early recognition and treatment are critical. Expanding indications for immunotherapy to solid and CNS tumors has led to new challenges, such as how to reliably distinguish neurotoxicity from disease progression. Our evolving understanding of immunotherapy neurotoxicity highlights important areas for future research and the need for novel immunomodulatory therapeutics.

  View details for DOI 10.1007/s11910-023-01315-w

  View details for PubMedID 37938472

  View details for PubMedCentralID 8291304

• Novel trial designs in neuro-oncology. Current opinion in neurology Song, K., Wen, P. Y. 2023

  Abstract

  PURPOSE OF REVIEW: An important factor contributing to the low rate of success in identifying effective therapies for brain tumor patients is the slow, inefficient, and expensive process of drug development, as well as small patient numbers, low patient participation in clinical trials, and reluctance of patients to enroll in ineffective control arms. In recent years, a number of novel trial designs have been developed to try to address some of these issues.RECENT FINDINGS: Surgical 'window-of-opportunity' trials that evaluate tumor drug concentrations and pharmacodynamic effects provide invaluable early data early guiding the development of novel therapies. Basket and bucket trials facilitate the development of therapies that target specific biomarkers subsets. Platform trials utilizing Bayesian adaptive randomization and shared control arms such as the INSIGhT and GBM-AGILE trials increase the efficiency and cost-effectiveness of developing novel therapies. There is also growing interest in leveraging external control arms with patient level data to evaluate efficacy in single arm trials, and facilitate interim analysis and potentially reduce the number of control patients in randomized trials.SUMMARY: These novel designs will hopefully reduce the inefficiencies of developing novel therapies in neuro-oncology and facilitate the identification of more effective therapies for brain tumor patients.

  View details for DOI 10.1097/WCO.0000000000001210

  View details for PubMedID 37865854

• Immune checkpoint inhibitor (ICI) therapy in glioblastoma: Institutional experience at the Massachusetts General Hospital (MGH) Rhee, J., Song, K., Arrillaga-Romany, I., Forst, D., Jordan, J. T., Ly, I., Plotkin, S., Wang, N., Parsons, M., Gerstner, E. R., Dietrich, J. LIPPINCOTT WILLIAMS & WILKINS. 2023

  View details for Web of Science ID 001053772000388

• GFAP astrocytopathy presenting after COVID-19 infection with meningoencephalomyelitis and good recovery with immunotherapy Song, K., Oetjen, L., Harrold, G., Dietrich, J., Ly, K., Venna, N. LIPPINCOTT WILLIAMS & WILKINS. 2023

  View details for DOI 10.1212/WNL.0000000000202122

  View details for Web of Science ID 001053672102155