Glioblastoma Research & Leading Companies

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give me the latest research in glioblastoma and the companies leading htis efforts

Recent glioblastoma (GBM) research is focused on overcoming challenges and developing new treatment strategies, given the aggressive nature of this brain cancer. Advancements include reprogramming cancer cells, personalized vaccines, drug repurposing, novel drug delivery techniques like skull-implantable ultrasound devices, and targeted therapies, all aimed at improving survival rates. Several companies and research institutions are leading these efforts, with promising developments emerging across various therapeutic approaches.

Key Facts:

  • UCLA scientists are investigating methods to reprogram glioblastoma cells and have identified BCL-XL as a key protein in evading cell death, with the drug ABBV-155 showing promise.
  • Northwestern Medicine scientists are using skull-implantable ultrasound devices to temporarily open the blood-brain barrier for chemotherapy drugs like doxorubicin.
  • Curtana Pharmaceuticals is developing CT-179, a small molecule inhibitor of OLIG2, which has received Fast Track and Orphan Disease designations from the FDA.
  • Bristol-Myers Squibb Co had the most products under development in the glioblastoma pipeline according to a 2022 report, alongside other key players like Novartis AG and Merck & Co Inc.
  • Mass General Cancer Center reported "dramatic and rapid" regression of glioblastoma in initial patients treated with a new CAR-T therapy in their INCIPIENT trial (NCT05660369).

Bristol-Myers Squibb Co

Bristol-Myers Squibb Co is a prominent pharmaceutical company actively involved in glioblastoma drug development. A 2022 report indicated that it had the most products under development in the glioblastoma pipeline.

Key Facts:

  • Bristol-Myers Squibb Co is a leading company in glioblastoma drug development.
  • A 2022 report highlighted them as having the most products in the glioblastoma pipeline.
  • They are a key player in advancing therapies for this aggressive brain cancer.

CC-115

CC-115 is a drug candidate in Bristol-Myers Squibb's pipeline that is in Phase II clinical development for glioblastoma multiforme (GBM). This orally administered drug targets DNA-dependent protein kinase (DNA-PK) and mammalian target of rapamycin (mTOR) 1 and 2.

Key Facts:

  • CC-115 is a drug candidate in Phase II clinical development.
  • It is being developed for glioblastoma (GBM).
  • CC-115 is administered orally.
  • It targets DNA-dependent protein kinase (DNA-PK).
  • It also targets mammalian target of rapamycin (mTOR) 1 and 2.

CheckMate -143

CheckMate -143 was a Phase 3 clinical trial conducted by Bristol-Myers Squibb, evaluating Opdivo in patients with first recurrence of glioblastoma multiforme (GBM). The trial did not meet its primary endpoint of improved overall survival compared to bevacizumab monotherapy.

Key Facts:

  • CheckMate -143 was a Phase 3 trial.
  • It evaluated Opdivo in patients with first recurrence of glioblastoma multiforme (GBM).
  • The trial's primary endpoint was improved overall survival.
  • It compared Opdivo to bevacizumab monotherapy.
  • The trial did not meet its primary endpoint.

CheckMate -498

CheckMate -498 was a Phase 3 trial by Bristol-Myers Squibb that assessed Opdivo in combination with radiation therapy against temozolomide and radiation in newly diagnosed O6-methylguanine-DNA methyltransferase (MGMT)-unmethylated glioblastoma. The study failed to meet its primary endpoint of overall survival.

Key Facts:

  • CheckMate -498 was a Phase 3 trial.
  • It assessed Opdivo in combination with radiation therapy.
  • The comparator arm was temozolomide and radiation.
  • The study population was newly diagnosed O6-methylguanine-DNA methyltransferase (MGMT)-unmethylated glioblastoma.
  • It failed to meet its primary endpoint of overall survival.

CheckMate -548

CheckMate -548 was a Phase 3 trial investigating the addition of Opdivo to the standard of care (temozolomide and radiation therapy) in newly diagnosed glioblastoma patients with MGMT promoter methylation. The study did not meet its primary endpoint of progression-free survival, and an update indicated it would also not meet its primary endpoint of overall survival.

Key Facts:

  • CheckMate -548 was a Phase 3 trial.
  • It investigated Opdivo added to standard of care (temozolomide and radiation therapy).
  • The study population included newly diagnosed glioblastoma patients with MGMT promoter methylation.
  • It did not meet its primary endpoint of progression-free survival (PFS).
  • An update in December 2020 indicated it would not meet its primary endpoint of overall survival.

Opdivo

Opdivo (nivolumab) is a programmed death-1 (PD-1) immune checkpoint inhibitor that has been a central component of Bristol-Myers Squibb's glioblastoma research. Despite extensive trials, Opdivo did not meet primary endpoints in several Phase 3 trials for glioblastoma.

Key Facts:

  • Opdivo is a programmed death-1 (PD-1) immune checkpoint inhibitor.
  • It was evaluated in glioblastoma research by Bristol-Myers Squibb.
  • CheckMate -143, a Phase 3 trial, evaluated Opdivo in patients with first recurrence of GBM.
  • CheckMate -498 assessed Opdivo in combination with radiation therapy against temozolomide and radiation in newly diagnosed O6-methylguanine-DNA methyltransferase (MGMT)-unmethylated GBM.
  • CheckMate -548 investigated the addition of Opdivo to standard of care (temozolomide and radiation therapy) in newly diagnosed GBM patients with MGMT promoter methylation.

Relatlimab

Relatlimab (BMS-986016) is an investigational compound from Bristol-Myers Squibb that is currently in Phase I clinical development for recurrent glioblastoma. This drug represents a part of BMS's ongoing efforts to explore novel immunotherapies for challenging cancers.

Key Facts:

  • Relatlimab is also known as BMS-986016.
  • It is in Phase I clinical development.
  • The target indication is recurrent glioblastoma.
  • It is part of Bristol-Myers Squibb's pipeline.
  • It is an investigational immunotherapy.

XL184

XL184 (BMS-907351) is an oral inhibitor that showed activity in patients with previously treated glioblastoma multiforme (GBM) in Phase 2 data reported in 2009. Bristol-Myers Squibb collaborated with Exelixis on this compound, which targets MET, VEGFR2, and RET.

Key Facts:

  • XL184 is also known as BMS-907351.
  • It is an oral inhibitor of MET, VEGFR2, and RET.
  • Phase 2 data in 2009 showed activity in previously treated glioblastoma (GBM).
  • Bristol-Myers Squibb collaborated with Exelixis on this drug.
  • It is a targeted therapy approach for GBM.

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Curtana Pharmaceuticals

Curtana Pharmaceuticals is developing highly targeted therapies for glioblastoma, including their drug candidate CT-179. CT-179 is a small molecule inhibitor of OLIG2, critical for tumor initiation and growth, and has received Fast Track and Orphan Disease designations from the FDA.

Key Facts:

  • Curtana Pharmaceuticals is developing targeted therapies for glioblastoma.
  • Their drug candidate, CT-179, is a small molecule inhibitor of OLIG2.
  • CT-179 targets OLIG2, which is critical for tumor initiation and growth.
  • Curtana Pharmaceuticals received Fast Track and Orphan Disease designations from the FDA for CT-179.
  • They plan to initiate Phase 1 clinical trials for CT-179.

Cancer Prevention and Research Institute of Texas

Curtana Pharmaceuticals received a significant $7.6 million grant from the Cancer Prevention and Research Institute of Texas (CPRIT) in 2014. This funding contributed to the early development of Curtana's research and drug candidates.

Key Facts:

  • Curtana Pharmaceuticals received a $7.6 million grant from CPRIT.
  • The grant was awarded in 2014.
  • CPRIT stands for the Cancer Prevention and Research Institute of Texas.
  • This grant represents a significant funding round for Curtana Pharmaceuticals.
  • It supported the company's early research efforts.

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Catalent

Curtana Pharmaceuticals has partnered with Catalent for the tablet formulation and manufacturing of CT-179. This collaboration is crucial for supporting Curtana's clinical trials by ensuring the production of the drug candidate.

Key Facts:

  • Curtana Pharmaceuticals partnered with Catalent.
  • Catalent is responsible for the tablet formulation of CT-179.
  • Catalent also handles the manufacturing of CT-179.
  • This partnership supports Curtana's clinical trials.
  • It is a key collaboration for the production of the drug candidate.

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CT-179

CT-179 is Curtana Pharmaceuticals' lead drug candidate, a first-in-class, small molecule inhibitor of OLIG2, designed to treat aggressive brain cancers like glioblastoma and medulloblastoma. It works by disrupting OLIG2 homodimerization, thereby blocking tumor growth and recurrence by preventing cancer stem cells from transitioning to proliferative states.

Key Facts:

  • CT-179 is Curtana Pharmaceutical's lead drug candidate.
  • It is a first-in-class, small molecule inhibitor of OLIG2.
  • CT-179 is orally bioavailable and readily crosses the blood-brain barrier.
  • Preclinical studies show it significantly prolongs event-free survival and overall survival in animal models of medulloblastoma and other brain cancers.
  • It has potential for combination therapy, enhancing efficacy of radiotherapy and combining with palbociclib.

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Fast Track Designation

Curtana Pharmaceuticals has also secured Fast Track Designation from the FDA for CT-179. This designation is intended to expedite the development and review of drugs that treat serious conditions and fulfill unmet medical needs, signaling the FDA's recognition of CT-179's potential.

Key Facts:

  • Curtana Pharmaceuticals received Fast Track Designation for CT-179.
  • This designation is aimed at expediting the development and review of drugs.
  • It applies to drugs that treat serious conditions and fulfill unmet medical needs.
  • Fast Track Designation can lead to earlier drug approval.
  • It reflects the FDA's view of CT-179's potential significance.

OLIG2

OLIG2 is a transcription factor critical for normal early brain development, but its re-expression is crucial for tumor initiation, growth, invasiveness, and resistance to therapy in various brain cancers, including glioblastoma. CT-179 targets OLIG2 to disrupt tumor growth.

Key Facts:

  • OLIG2 is a transcription factor crucial for normal early brain development.
  • It is re-expressed and critical for tumor initiation, growth, invasiveness, and resistance to therapy in various brain cancers.
  • CT-179 is designed to disrupt OLIG2 homodimerization.
  • Blocking OLIG2 prevents cancer stem cells from transitioning to proliferative states.
  • Research published in Nature Communications highlighted CT-179's potential in treating Sonic Hedgehog (SHH) subgroup medulloblastoma by inhibiting OLIG2.

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Orphan Drug Designation

Curtana Pharmaceuticals received Orphan Drug Designation from the FDA for CT-179 for the treatment of malignant gliomas, including glioblastoma, in August 2017. This designation provides incentives such as tax credits for clinical trials, fee waivers, and potential market exclusivity upon approval.

Key Facts:

  • Curtana Pharmaceuticals received Orphan Drug Designation for CT-179 in August 2017.
  • The designation is for the treatment of malignant gliomas, including glioblastoma.
  • Orphan Drug Designation provides incentives like tax credits for clinical trials and fee waivers.
  • It also offers potential market exclusivity upon approval.
  • This is one of several FDA designations received by Curtana for CT-179.

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Rare Pediatric Disease Designation

Curtana Pharmaceuticals obtained Rare Pediatric Disease Designation from the FDA for CT-179 for the treatment of medulloblastoma in September 2020. This designation recognizes the drug's potential to address serious conditions in pediatric populations.

Key Facts:

  • Curtana Pharmaceuticals received Rare Pediatric Disease Designation for CT-179 in September 2020.
  • The designation is for the treatment of medulloblastoma.
  • This is one of the important FDA designations for CT-179.
  • The designation aims to expedite development for serious conditions in children.
  • It highlights CT-179's potential impact on pediatric brain cancers.

Kazia Therapeutics

Kazia Therapeutics is a company actively involved in glioblastoma research and drug development. They are recognized for their contributions to the pipeline of therapies aimed at this aggressive brain cancer.

Key Facts:

  • Kazia Therapeutics is an active participant in glioblastoma research.
  • They are involved in drug development efforts for glioblastoma.
  • Kazia Therapeutics is listed among key players in the glioblastoma pipeline.

Evotec SE

Evotec SE is the company from which Kazia Therapeutics licensed EVT801, a small-molecule inhibitor of VEGFR3, in April 2021. This licensing agreement brought an additional promising oncology asset into Kazia's development pipeline, diversifying its therapeutic focus.

Key Facts:

  • Kazia Therapeutics licensed EVT801 from Evotec SE in April 2021.
  • EVT801 is a small-molecule inhibitor of VEGFR3.
  • Evotec SE is a company involved in drug discovery and development.
  • The licensing agreement expanded Kazia's oncology pipeline beyond brain cancer.
  • EVT801 is currently in Phase 1 trials for advanced solid tumors.

EVT801

EVT801 is a small-molecule inhibitor of VEGFR3, in-licensed by Kazia Therapeutics from Evotec SE. It is currently in Phase 1 trials for advanced solid tumors and has demonstrated preclinical activity across various tumor types, including synergy with immuno-oncology agents.

Key Facts:

  • EVT801 is a small-molecule inhibitor of VEGFR3.
  • Kazia Therapeutics licensed EVT801 from Evotec SE in April 2021.
  • It is currently in Phase 1 trials for the treatment of advanced solid tumors.
  • Preclinical studies have shown EVT801's activity across various tumor types.
  • EVT801 has also demonstrated synergy with immuno-oncology agents in preclinical models.

GBM AGILE

GBM AGILE is an adaptive Phase 3 study evaluating paxalisib for potential registration in newly diagnosed and relapsed glioblastoma patients. Topline results from this study in July 2024 indicated a clinically meaningful improvement in overall survival for a specific subgroup.

Key Facts:

  • GBM AGILE is an adaptive Phase 3 study for glioblastoma.
  • It is evaluating paxalisib in patients with newly diagnosed glioblastoma (unmethylated MGMT promoter) and relapsed glioblastoma.
  • Topline results in July 2024 showed a 3.8-month overall survival improvement in newly diagnosed unmethylated glioblastoma patients treated with paxalisib.
  • Kazia Therapeutics has aligned with the FDA on key aspects for a proposed registrational Phase 3 study for paxalisib in NDU glioblastoma, following GBM AGILE.
  • The study design aims to efficiently assess multiple therapies in parallel for glioblastoma.

NDL2

NDL2 is the lead compound from a first-in-class PD-L1 degrader program, which Kazia Therapeutics in-licensed through an exclusive collaboration agreement with QIMR Berghofer. This program represents Kazia's expansion into novel immuno-oncology strategies beyond its primary focus on brain cancer.

Key Facts:

  • NDL2 is the lead compound in a first-in-class PD-L1 degrader program.
  • Kazia Therapeutics announced an exclusive collaboration and in-licensing agreement for this program with QIMR Berghofer in October 2025.
  • The program focuses on PD-L1 degradation, representing a novel immuno-oncology approach.
  • NDL2's development signifies Kazia's diversification into other oncology and research areas.
  • The collaboration highlights Kazia's strategy of in-licensing innovative assets.

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paxalisib

Paxalisib, formerly known as GDC-0084, is Kazia Therapeutics' lead drug candidate, a brain-penetrant inhibitor of the PI3K/Akt/mTOR pathway. This drug is primarily recognized for its development in glioblastoma and its ability to cross the blood-brain barrier, addressing a significant challenge in CNS drug delivery.

Key Facts:

  • Paxalisib is a brain-penetrant inhibitor of the PI3K/Akt/mTOR pathway, which is activated in over 85% of glioblastoma patients.
  • It is the subject of ten clinical trials for brain cancer, including an adaptive Phase 3 study called GBM AGILE.
  • Topline results from the GBM AGILE study in July 2024 showed a 3.8-month overall survival improvement in newly diagnosed unmethylated glioblastoma patients.
  • Paxalisib has received Orphan Drug Designation and Fast Track Designation for glioblastoma from the FDA.
  • It is also being investigated in other brain cancers and metastases, having received Rare Pediatric Disease Designation and Orphan Drug Designation for DIPG and AT/RT.

PNOC 022

PNOC 022 is a study investigating paxalisib in pediatric brain cancer, from which encouraging interim data has been reported. This study contributes to Kazia Therapeutics' broader efforts in addressing various forms of brain cancer, including those affecting children.

Key Facts:

  • PNOC 022 is a study investigating paxalisib in pediatric brain cancer.
  • Encouraging interim data has been reported from the PNOC 022 study.
  • This study is part of Kazia's efforts to investigate paxalisib in other forms of adult and pediatric brain cancer.
  • Paxalisib has received Rare Pediatric Disease Designation for DIPG and AT/RT, which are pediatric brain cancers.
  • The Pediatric Neuro-Oncology Consortium (PNOC) is known for conducting studies in pediatric brain tumors.

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QIMR Berghofer

QIMR Berghofer is a research institution that entered into an exclusive collaboration and in-licensing agreement with Kazia Therapeutics for a first-in-class PD-L1 degrader program, featuring the lead compound NDL2. This partnership underpins Kazia's expansion into new oncology therapeutic areas.

Key Facts:

  • Kazia Therapeutics announced an exclusive collaboration and in-licensing agreement with QIMR Berghofer in October 2025.
  • The agreement is for a first-in-class PD-L1 degrader program.
  • The lead compound from this program is NDL2.
  • QIMR Berghofer is a research institution contributing to Kazia's oncology pipeline.
  • This collaboration diversifies Kazia's focus beyond primary brain cancers.

Merck & Co Inc

Merck & Co Inc is involved in glioblastoma research and has expanded its efforts through acquisitions. They are a key pharmaceutical company contributing to new therapies for this aggressive brain cancer.

Key Facts:

  • Merck & Co Inc is a key player in glioblastoma research and drug development.
  • They have advanced therapies, including through acquisitions like Modifi Biosciences.
  • Merck is listed among other major pharmaceutical companies in the glioblastoma field.

KEYTRUDA

KEYTRUDA is Merck's brand name for the immunotherapy drug Pembrolizumab, which is under active investigation for various brain cancers, including glioblastoma. It is currently undergoing Phase II clinical development for recurrent glioblastoma multiforme and metastatic brain tumors, and has been part of collaborative studies, such as with DNAtrix.

Key Facts:

  • KEYTRUDA is the brand name for Merck's immunotherapy drug, Pembrolizumab.
  • It is currently in Phase II clinical development for recurrent glioblastoma multiforme.
  • KEYTRUDA is also being investigated for metastatic brain tumors.
  • In 2015, Merck collaborated with DNAtrix to study KEYTRUDA in combination with DNX-2401.
  • This collaboration focused on recurrent glioblastoma in a Phase II study.

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M3554

M3554 is a first-in-class anti-GD2 Antibody-Drug Conjugate (ADC) within Merck's oncology pipeline, slated to enter first-in-human studies in 2024 and 2025. This investigational compound is intended to be evaluated in solid tumors, including glioblastoma, as part of Merck's broader research into DNA Damage Response (DDR) inhibitors and ADCs.

Key Facts:

  • M3554 is described as a first-in-class anti-GD2 Antibody-Drug Conjugate (ADC).
  • It is part of Merck's broader oncology pipeline.
  • M3554 is scheduled to enter first-in-human studies in 2024 and 2025.
  • It will be evaluated in solid tumors, specifically mentioning glioblastoma.
  • This compound aligns with Merck's research in DNA Damage Response (DDR) inhibitors and ADCs.

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MOD-246

MOD-246, also referred to as KL-50, is a novel preclinical DNA-damaging agent developed by Modifi Biosciences and subsequently acquired by Merck & Co. Inc. It is designed to selectively target the DNA of malignant glioblastoma cells, overcoming resistance to standard chemotherapy like temozolomide, and represents a 'precision chemotherapy' approach.

Key Facts:

  • MOD-246 (KL-50) is a novel DNA-damaging agent.
  • It was developed by Modifi Biosciences and acquired by Merck & Co. Inc.
  • MOD-246 is intended to overcome resistance to temozolomide (TMZ) in glioblastoma.
  • It selectively targets malignant cell DNA while sparing healthy cells.
  • Merck aims for clinical trials of MOD-246 by late 2025 after IND-enabling studies.

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Modifi Biosciences

Modifi Biosciences is a company that was acquired by Merck & Co. Inc. in October 2024 for an upfront payment of $30 million. This acquisition brought their preclinical asset, MOD-246 (KL-50), into Merck's pipeline, which is a novel DNA-damaging agent designed to overcome temozolomide resistance in glioblastoma.

Key Facts:

  • Merck & Co. Inc. acquired Modifi Biosciences in October 2024.
  • The acquisition involved an upfront payment of $30 million and potential milestone payments up to $1.3 billion.
  • Modifi Biosciences developed MOD-246 (KL-50), a preclinical DNA-damaging asset.
  • MOD-246 is designed to overcome resistance to temozolomide (TMZ) in glioblastoma.
  • Merck plans to initiate IND-enabling studies for MOD-246 with clinical trials aimed for late 2025.

Pembrolizumab

Pembrolizumab, marketed as KEYTRUDA by Merck, is an immunotherapy drug actively being investigated for its application in brain cancer, including glioblastoma. It is currently in Phase II clinical development for recurrent glioblastoma multiforme and metastatic brain tumors, and has been explored in combination with other oncolytic immunotherapies.

Key Facts:

  • Pembrolizumab is an immunotherapy drug marketed as KEYTRUDA by Merck.
  • It is being investigated for brain cancer, including glioblastoma.
  • Pembrolizumab is in Phase II clinical development for recurrent glioblastoma multiforme and metastatic brain tumors.
  • Merck collaborated with DNAtrix in 2015 to evaluate KEYTRUDA with DNX-2401 for recurrent glioblastoma.
  • KEYTRUDA is part of Merck's broader oncology pipeline.

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Temodar

Temodar, generically known as temozolomide (TMZ), is a primary chemotherapy treatment for glioblastoma previously developed by Merck. Despite its approval in 1999 and its status as a standard treatment, nearly half of glioblastoma patient tumors develop resistance to it, highlighting the need for new therapies like MOD-246.

Key Facts:

  • Temodar (temozolomide) was previously developed by Merck.
  • It was approved in 1999 as a primary treatment option for glioblastoma.
  • A significant challenge is that nearly half of glioblastoma tumors develop resistance to Temodar.
  • The development of new therapies like MOD-246 is driven by Temodar resistance.
  • Temodar is considered a standard chemotherapy for glioblastoma.

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Northwestern Medicine

Northwestern Medicine scientists have made breakthroughs in novel drug delivery techniques for glioblastoma, particularly in circumventing the blood-brain barrier (BBB). They are utilizing skull-implantable ultrasound devices to temporarily open the BBB, enhancing drug penetration.

Key Facts:

  • Northwestern Medicine scientists are focused on novel drug delivery for glioblastoma.
  • They use skull-implantable ultrasound devices to temporarily open the blood-brain barrier (BBB).
  • This method allows chemotherapy drugs like doxorubicin and immune checkpoint blockade antibodies to penetrate the brain.
  • The approach has been shown to boost the immune system's recognition of cancer cells.

Adam Sonabend

Adam Sonabend is a scientist at Northwestern Medicine, working alongside Roger Stupp, who has played a key role in the first-in-human clinical trials of a skull-implantable ultrasound device for glioblastoma. This device facilitates the temporary opening of the blood-brain barrier to enhance the penetration of therapeutic drugs.

Key Facts:

  • Adam Sonabend is a scientist at Northwestern Medicine.
  • He has been instrumental in the first-in-human clinical trials of a novel skull-implantable ultrasound device.
  • This device is used to temporarily open the blood-brain barrier to enhance drug delivery for glioblastoma.

Carthera

Carthera is a French company that developed the Sonocloud-9, a skull-implantable ultrasound device used by Northwestern Medicine scientists in their glioblastoma research. This device is crucial for temporarily opening the blood-brain barrier to improve drug delivery to the brain.

Key Facts:

  • Carthera is a French company.
  • Carthera developed the Sonocloud-9, a skull-implantable ultrasound device.
  • The Sonocloud-9 device is utilized by Northwestern Medicine in glioblastoma clinical trials.

Lou and Jean Malnati Brain Tumor Institute

The Lou and Jean Malnati Brain Tumor Institute is housed within Northwestern Medicine, emphasizing a collaborative approach to brain tumor treatment and research, including glioblastoma. This institute likely serves as a central hub for various research initiatives and patient care related to brain tumors.

Key Facts:

  • The Lou and Jean Malnati Brain Tumor Institute is part of Northwestern Medicine.
  • It focuses on brain tumor treatment and research.
  • The institute emphasizes a collaborative approach to combating aggressive cancers like glioblastoma.

Roger Stupp

Roger Stupp is a scientist at Northwestern Medicine who has been instrumental in the first-in-human clinical trials of a novel skull-implantable ultrasound device for glioblastoma treatment. His work focuses on using this device to temporarily open the blood-brain barrier (BBB) to enhance drug delivery.

Key Facts:

  • Roger Stupp is a scientist at Northwestern Medicine.
  • He was instrumental in the first-in-human clinical trials of a skull-implantable ultrasound device.
  • The device is designed to temporarily open the blood-brain barrier for improved drug delivery.

Sonocloud-9

The Sonocloud-9 is a novel skull-implantable ultrasound device developed by the French company Carthera. Northwestern Medicine scientists utilize this device in clinical trials to temporarily and reversibly open the blood-brain barrier, significantly enhancing the delivery of chemotherapy and immunotherapy drugs for glioblastoma.

Key Facts:

  • Sonocloud-9 is a skull-implantable ultrasound device.
  • It was developed by the French company Carthera.
  • The device consists of a grid of nine ultrasound emitters.
  • It is used by Northwestern Medicine to temporarily open the blood-brain barrier for drug delivery in glioblastoma.

Novartis AG

Novartis AG is a significant player in glioblastoma research and drug development. They are recognized among the key companies actively involved in advancing treatment strategies for this brain cancer.

Key Facts:

  • Novartis AG is a key player in glioblastoma research and drug development.
  • They are actively involved in advancing treatment strategies for glioblastoma.
  • Novartis is listed among other major pharmaceutical companies contributing to the field.

AAA601

AAA601 is a radioligand therapy under investigation by Novartis for glioblastoma, with two distinct formulations (Lutathera® and 177Lu-DOTA-TATE) in different phases of clinical trials.

Key Facts:

  • AAA601 (Lutathera®) is a radioligand therapy targeting SSTR and is in Phase 2 clinical trials for glioblastoma.
  • AAA601 (177Lu-DOTA-TATE) is another radioligand therapy in a Phase 1 dose-finding study for newly diagnosed glioblastoma in combination with standard care.
  • The Phase 1 study for 177Lu-DOTA-TATE is also investigating it as a single agent for recurrent glioblastoma.
  • The estimated completion date for the 177Lu-DOTA-TATE Phase 1 trial is February 2026.

AAA603 (177Lu-NeoB)

AAA603 (177Lu-NeoB) is an investigational radiopharmaceutical solution from Novartis, undergoing a Phase 1 dose-finding study for both newly diagnosed and recurrent glioblastoma.

Key Facts:

  • AAA603 (177Lu-NeoB) is an investigational radiopharmaceutical solution.
  • It is being studied in a Phase 1 dose-finding study for newly diagnosed and recurrent glioblastoma.
  • The study aims to determine the recommended dose of 177Lu-NeoB in combination with temozolomide (TMZ) and radiotherapy (RT) for newly diagnosed glioblastoma.
  • For recurrent glioblastoma, AAA603 (177Lu-NeoB) is being studied as a single agent.
  • The trial is currently recruiting participants and is expected to be completed by August 2031.

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Dabrafenib

Dabrafenib (Finlee) is an oral drug developed by Novartis, used in combination with Trametinib (Spexotras) for treating a specific subgroup of pediatric low-grade gliomas with a BRAF V600E mutation.

Key Facts:

  • Dabrafenib (Finlee) is an oral drug from Novartis.
  • It is used in combination with Trametinib (Spexotras).
  • The combination treats pediatric low-grade gliomas with a BRAF V600E mutation.
  • Clinical trials showed the combination could halt tumor growth for over two years, three times longer than existing treatments.
  • The National Institute for Health and Care Excellence (NICE) has recommended this treatment for eligible children and young people in England and Wales.

Ribociclib succinate

Ribociclib succinate is a drug from Novartis that is currently in Phase 2 clinical development for Glioblastoma Multiforme (GBM) and various other cancer indications, showcasing its potential as a broad-spectrum oncology agent.

Key Facts:

  • Ribociclib succinate is a drug developed by Novartis.
  • It is currently in Phase 2 clinical development.
  • The drug is being investigated for Glioblastoma Multiforme (GBM) and other cancers.

Sabatolimab

Sabatolimab is an anti-TIM-3 monoclonal antibody developed by Novartis, currently in Phase 1 clinical development for Glioblastoma Multiforme (GBM) and other solid tumors, representing an immunotherapy approach.

Key Facts:

  • Sabatolimab is an anti-TIM-3 monoclonal antibody.
  • It is under clinical development by Novartis.
  • Sabatolimab is currently in Phase 1 trials for Glioblastoma Multiforme (GBM) and other solid tumors.
  • The drug targets T cell immunoglobulin mucin-3 (TIM-3).

Trametinib

Trametinib (Spexotras) is an oral drug from Novartis, specifically used in combination with Dabrafenib (Finlee) to treat pediatric low-grade gliomas that carry the BRAF V600E mutation.

Key Facts:

  • Trametinib (Spexotras) is an oral drug developed by Novartis.
  • It is combined with Dabrafenib (Finlee) to treat pediatric low-grade gliomas.
  • The target for this combination therapy is the BRAF V600E mutation in pediatric patients.
  • The combined treatment has demonstrated the ability to inhibit tumor growth for more than two years in clinical trials.
  • NICE has recommended this therapy for specific children and young people in England and Wales.

UCLA Health

UCLA Health is actively involved in glioblastoma research, focusing on understanding and overcoming resistance mechanisms in cancer cells. Their scientists are investigating methods to reprogram cancer cells to halt tumor growth and analyze blood vessel-tumor interactions.

Key Facts:

  • UCLA scientists are investigating methods to reprogram cancer cells to halt tumor growth.
  • They are gaining insights into how blood vessel-tumor interactions fuel glioblastoma.
  • UCLA combines genetic and functional profiling to predict treatment response.
  • They identified BCL-XL proteins as key players in evading cell death.

BCL-XL Proteins

BCL-XL proteins have been identified by UCLA researchers as a significant factor in helping cancer cells avoid death, forming a key area of study in understanding glioblastoma treatment resistance. Targeting BCL-XL with drugs like ABBV-155, in combination with standard treatments, has shown promise in shrinking tumors in lab models.

Key Facts:

  • BCL-XL proteins are a significant factor in helping glioblastoma cells avoid death.
  • UCLA researchers have identified BCL-XL's role in cancer cell survival.
  • Targeting BCL-XL with drugs like ABBV-155 is being investigated.
  • Combined with standard treatments, ABBV-155 significantly shrank tumors in lab models.
  • This research contributes to understanding how glioblastoma cells evade cell death.

Cancer Cell Reprogramming

UCLA scientists are exploring methods to reprogram glioblastoma cells to halt tumor growth, a key area of focus in their glioblastoma research. This involves pushing adaptable glioblastoma stem cells toward neuron-like or microglia-like states to stop uncontrolled division and tumor regeneration.

Key Facts:

  • UCLA scientists are exploring methods to reprogram glioblastoma cells to halt tumor growth.
  • Dr. Frank Pajonk and his team are combining radiation therapy with forskolin for cell reprogramming.
  • The goal is to push adaptable glioblastoma stem cells toward neuron-like or microglia-like states.
  • Reprogramming aims to stop uncontrolled division and tumor regeneration.
  • This research contributes to understanding treatment resistance and predicting response in glioblastoma.

DCVax-L

DCVax-L is a patient-specific brain tumor vaccine pioneered by Dr. Linda Liau at UCLA Health, currently undergoing a phase-3 clinical trial. This vaccine aims to stimulate the body's immune system to recognize and attack glioblastoma cancer cells.

Key Facts:

  • DCVax-L is a patient-specific brain tumor vaccine.
  • It was pioneered by Dr. Linda Liau at UCLA Health.
  • DCVax-L is currently in a phase-3 clinical trial.
  • The vaccine aims to stimulate the body's immune system to attack cancer cells.
  • UCLA is also launching clinical trials for a personalized cancer vaccine targeting H3 G34-mutant diffuse hemispheric glioma.

Dr. Frank Pajonk

Dr. Frank Pajonk and his team at UCLA are leading efforts in cancer cell reprogramming, specifically combining radiation therapy with a plant-derived compound called forskolin. Their work focuses on pushing adaptable glioblastoma stem cells toward neuron-like or microglia-like states to halt tumor growth and regeneration.

Key Facts:

  • Dr. Frank Pajonk leads a team investigating cancer cell reprogramming at UCLA.
  • His team combines radiation therapy with forskolin for glioblastoma treatment.
  • The research aims to induce glioblastoma stem cells into neuron-like or microglia-like states.
  • This approach is designed to stop uncontrolled cell division and tumor regeneration.
  • Dr. Pajonk's work is a key contribution to UCLA Health's glioblastoma research focus areas.

Dr. Linda Liau

Dr. Linda Liau is a prominent figure at UCLA Health, having pioneered the development of the DCVax-L vaccine, a patient-specific brain tumor vaccine now in a phase-3 clinical trial. Her work focuses on utilizing the body's immune system to combat glioblastoma.

Key Facts:

  • Dr. Linda Liau pioneered the DCVax-L vaccine.
  • DCVax-L is a patient-specific brain tumor vaccine for glioblastoma.
  • The vaccine is currently in a phase-3 clinical trial.
  • Her research focuses on stimulating the immune system to attack cancer cells.
  • Dr. Liau's work is a significant contribution to personalized treatment strategies at UCLA Health.

UCLA Health Jonsson Comprehensive Cancer Center

The UCLA Health Jonsson Comprehensive Cancer Center (JCCC) is the primary concentration point for UCLA Health's glioblastoma efforts and is recognized as one of the top cancer centers in the nation. It is an NCI-designated comprehensive cancer center, indicating adherence to rigorous standards for transdisciplinary research and patient care.

Key Facts:

  • UCLA Health's glioblastoma research efforts are primarily concentrated within the UCLA Health Jonsson Comprehensive Cancer Center (JCCC).
  • JCCC is recognized as one of the top cancer centers in the nation.
  • JCCC is a National Cancer Institute (NCI)-designated comprehensive cancer center.
  • The JCCC upholds rigorous standards for transdisciplinary research and patient care.
  • The Neuro-Oncology Program at UCLA, a leading brain tumor program in Southern California, is part of the JCCC's efforts.