{"id":40207,"date":"2026-08-17T13:42:02","date_gmt":"2026-08-17T17:42:02","guid":{"rendered":"https:\/\/www.cancerresearch.org\/?post_type=cri_scientists&#038;p=40207"},"modified":"2026-08-17T13:42:56","modified_gmt":"2026-08-17T17:42:56","slug":"sabine-heitzeneder-md","status":"publish","type":"cri_scientists","link":"https:\/\/www.cancerresearch.org\/es\/cri-funded-scientists\/sabine-heitzeneder-md","title":{"rendered":"Sabine Heitzeneder, MD"},"content":{"rendered":"\n<p class=\"wp-block-paragraph\">Children and young adults with recurrent or refractory (R\/R) medulloblastoma are in dire need of novel targeted treatment strategies that increase cure rates and diminish treatment-related morbidity. The team at Stanford launched a phase-I clinical trial (NCT07087002) in August 2025 to evaluate the manufacturing feasibility and safety of autologous chimeric antigen receptor (CAR) T cells targeting the oncofetal protein Glypican-2 (GPC2), designed to treat pediatric and young adult patients with confirmed GPC2-positive R\/R medulloblastoma or other eligible Central Nervous System (CNS) Embryonal Tumors using intracerebroventricular (ICV) administration.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">This study administers sequential intracerebroventricular doses of GPC2-CAR T cells in up to eight cycles using a two-phased Bayesian statistical design employing intrapatient dose escalation. Subjects receive lymphodepleting chemotherapy prior to the initial dose of GPC2-CAR T cells, which can be repeated if the CD4 count recovers to &gt;200 cells\/uL and other eligibility criteria are met. To date, they have enrolled four patients and are actively recruiting.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">GPC2 is a compelling immune target, that is expressed in the developing brain, yet essentially absent from postnatal normal tissues. This trial builds upon preclinical work on engineering GPC2-CAR T cells tuned towards clinically relevant antigen densities in neuroblastoma (Heitzeneder et. al) and unpublished preclinical studies in orthotopic medulloblastoma xenograft models using locoregional CAR-T delivery. The key aspect of this proposal is to conduct extensive correlative studies and evaluate GPC2-CAR T cell mediated anti-tumor responses and predictors of response, resistance and toxicity with the goal to improve patient outcomes driven by insights gained from translational studies.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">Projects and Grants<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>GPC2-CAR T Cell Therapy for Relapsed or Refractory Medulloblastoma and other CNS Embryonal Tumors in Children and Young Adults<\/strong><\/p>\n","protected":false},"excerpt":{"rendered":"<p>Children and young adults with recurrent or refractory (R\/R) medulloblastoma are in dire need of novel targeted treatment strategies that increase cure rates and diminish treatment-related morbidity. The team at Stanford launched [&hellip;]<\/p>\n","protected":false},"featured_media":40188,"template":"","tax_cancer_type":[366],"tax_grant_type":[725],"tax_award_year":[881],"tax_institutions":[513],"tax_location_states":[501],"class_list":["post-40207","cri_scientists","type-cri_scientists","status-publish","has-post-thumbnail","hentry"],"acf":{"scientist_subhead":"CRI Clinical Innovator","quote":"","scientist_last_name":"Heitzeneder","show_on_landing":true,"scientist_publish_until":"20290831"},"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v28.1 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>Sabine Heitzeneder, MD - Cancer Research Institute<\/title>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"https:\/\/www.cancerresearch.org\/cri-funded-scientists\/sabine-heitzeneder-md\" \/>\n<meta property=\"og:locale\" content=\"es_ES\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"Sabine Heitzeneder, MD - Cancer Research Institute\" \/>\n<meta property=\"og:description\" content=\"Children and young adults with recurrent or refractory (R\/R) medulloblastoma are in dire need of novel targeted treatment strategies that increase cure rates and diminish treatment-related morbidity. 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