{"id":21,"date":"2025-12-24T22:37:26","date_gmt":"2025-12-24T22:37:26","guid":{"rendered":"https:\/\/indaccess.org\/?page_id=21"},"modified":"2025-12-26T05:09:21","modified_gmt":"2025-12-26T05:09:21","slug":"brain-tumors-and-inds","status":"publish","type":"page","link":"https:\/\/indaccess.org\/?page_id=21","title":{"rendered":"Information on Cancers and Brain Tumors including current INDs (draft)"},"content":{"rendered":"\n<p>Disclaimer: Not an advise. Informational and Educational use only.<\/p>\n\n\n\n<p>Standard of care: ACNS0423 Study<br>Introduction: https:\/\/pmc.ncbi.nlm.nih.gov\/articles\/PMC5035517\/<br>Reports outcomes (EFS\/OS) and toxicity profile, including the central conclusion that this regimen had <strong>acceptable but meaningful hematologic toxicity<\/strong> and only <strong>modest improvement<\/strong> vs historical expectations. <a href=\"https:\/\/pmc.ncbi.nlm.nih.gov\/articles\/PMC5035517\/?utm_source=chatgpt.com\" target=\"_blank\" rel=\"noreferrer noopener\"><\/a><br><br>Disclaimer: Not advocating for any specific drugs. Informational and educational purposes only for may be used to apply for SPIND only. May add more drugs. <\/p>\n\n\n\n<p>Brain Tumor IND: ACT001 : How it works<br>Mechanism in glioblastoma models: <strong>targets STAT3 \u2192 lowers PD-L1 transcription \/ immunosuppression<\/strong> (preclinical + mechanistic). <a href=\"https:\/\/pmc.ncbi.nlm.nih.gov\/articles\/PMC7254983\/?utm_source=chatgpt.com\" target=\"_blank\" rel=\"noreferrer noopener\">PMC<\/a><br>Additional glioma mechanism work: <strong>targets glioma stem-like cells<\/strong> and anti-tumor effects in GBM models (mechanistic preclinical). <a href=\"https:\/\/pmc.ncbi.nlm.nih.gov\/articles\/PMC7738851\/?utm_source=chatgpt.com\" target=\"_blank\" rel=\"noreferrer noopener\">PMC<\/a><\/p>\n\n\n\n<p>Brain Tumor IND: ACT001 : <strong>Safety:<\/strong><\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>First-in-human Phase I (advanced glioma)<\/strong>: reported <strong>safe \/ well tolerated<\/strong> with PK and tolerability focus (ASCO abstract \/ JCO supplement). <a href=\"https:\/\/ascopubs.org\/doi\/10.1200\/JCO.2021.39.15_suppl.2044?utm_source=chatgpt.com\" target=\"_blank\" rel=\"noreferrer noopener\">ASCOPubs+1<\/a><\/li>\n\n\n\n<li><strong>Pediatric DMG\/DIPG Phase 1 dose escalation<\/strong>: reported <strong>well tolerated<\/strong> with preliminary anti-tumor activity at higher doses (conference abstract on Neuro-Oncology \/ OUP). <a href=\"https:\/\/academic.oup.com\/neuro-oncology\/article\/26\/Supplement_4\/0\/7694624?utm_source=chatgpt.com\" target=\"_blank\" rel=\"noreferrer noopener\">OUP Academic+1<\/a><\/li>\n<\/ul>\n\n\n\n<p>Brain Tumor IND: ACT001: <strong>Efficacy (signals, early):<\/strong><\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Phase I glioma: <strong>clinical responses noted in a subset<\/strong> (pathologic response mentioned in abstract). <a href=\"https:\/\/ascopubs.org\/doi\/10.1200\/JCO.2021.39.15_suppl.2044?utm_source=chatgpt.com\" target=\"_blank\" rel=\"noreferrer noopener\">ASCOPubs+1<\/a><\/li>\n\n\n\n<li>Pediatric DMG\/DIPG Phase 1: preliminary evidence of anti-tumor activity at highest dose levels (early signal, not definitive). <a href=\"https:\/\/pmc.ncbi.nlm.nih.gov\/articles\/PMC11183933\/?utm_source=chatgpt.com\" target=\"_blank\" rel=\"noreferrer noopener\">PMC+1<\/a><\/li>\n\n\n\n<li>Recurrent GBM combo program (ACT001 + anti-PD-1): designed to evaluate both <strong>safety and efficacy<\/strong> in surgically accessible recurrent GBM. <a href=\"https:\/\/www.clinicaltrials.gov\/study\/NCT05053880?utm_source=chatgpt.com\" target=\"_blank\" rel=\"noreferrer noopener\">ClinicalTrials<\/a><\/li>\n<\/ul>\n\n\n\n<p>Brain Tumor IND: ACT001: <strong>Existing Trials:<\/strong><br><strong>NCT05053880<\/strong> \u2014 ACT001 + <strong>anti-PD-1<\/strong> in <strong>surgically accessible recurrent GBM<\/strong>. <br><strong>NCT06838676<\/strong> \u2014 Phase II ACT001 in <strong>children\/adolescents with DIPG and H3K27-altered HGG<\/strong> (CONNECT2110). <a href=\"https:\/\/www.clinicaltrials.gov\/study\/NCT06838676?utm_source=chatgpt.com\" target=\"_blank\" rel=\"noreferrer noopener\">ClinicalTrials+1<\/a><br><\/p>\n\n\n\n<p>Other existing IND trials and hospital informations:<\/p>\n\n\n\n<p><a href=\"https:\/\/connectconsortium.org\/clinical-trials\/\">https:\/\/connectconsortium.org\/clinical-trials\/<\/a><\/p>\n\n\n\n<p><a href=\"https:\/\/clinicaltrials.gov\/search?cond=gbm\" title=\"https:\/\/clinicaltrials.gov\/search?cond=gbm\">https:\/\/clinicaltrials.gov\/search?cond=gbm<\/a><br><\/p>\n","protected":false},"excerpt":{"rendered":"<p>Disclaimer: Not an advise. Informational and Educational use only. Standard of care: ACNS0423 StudyIntroduction: https:\/\/pmc.ncbi.nlm.nih.gov\/articles\/PMC5035517\/Reports outcomes (EFS\/OS) and toxicity profile, including the central conclusion that this regimen had acceptable but meaningful hematologic toxicity and only modest improvement vs historical expectations. Disclaimer: Not advocating for any specific drugs. Informational and educational purposes only for may be &#8230; <a title=\"Information on Cancers and Brain Tumors including current INDs (draft)\" class=\"read-more\" href=\"https:\/\/indaccess.org\/?page_id=21\" aria-label=\"Read more about Information on Cancers and Brain Tumors including current INDs (draft)\">Read more<\/a><\/p>\n","protected":false},"author":1,"featured_media":0,"parent":0,"menu_order":0,"comment_status":"closed","ping_status":"closed","template":"","meta":{"slim_seo":{"title":"Information on Cancers and Brain Tumors including current INDs (draft) - FYI: IND Access Information","description":"Disclaimer: Not an advise. Informational and Educational use only. Standard of care: ACNS0423 Study Introduction: https:\/\/pmc.ncbi.nlm.nih.gov\/articles\/PMC50355"},"footnotes":""},"class_list":["post-21","page","type-page","status-publish"],"_links":{"self":[{"href":"https:\/\/indaccess.org\/index.php?rest_route=\/wp\/v2\/pages\/21","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/indaccess.org\/index.php?rest_route=\/wp\/v2\/pages"}],"about":[{"href":"https:\/\/indaccess.org\/index.php?rest_route=\/wp\/v2\/types\/page"}],"author":[{"embeddable":true,"href":"https:\/\/indaccess.org\/index.php?rest_route=\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/indaccess.org\/index.php?rest_route=%2Fwp%2Fv2%2Fcomments&post=21"}],"version-history":[{"count":7,"href":"https:\/\/indaccess.org\/index.php?rest_route=\/wp\/v2\/pages\/21\/revisions"}],"predecessor-version":[{"id":44,"href":"https:\/\/indaccess.org\/index.php?rest_route=\/wp\/v2\/pages\/21\/revisions\/44"}],"wp:attachment":[{"href":"https:\/\/indaccess.org\/index.php?rest_route=%2Fwp%2Fv2%2Fmedia&parent=21"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}