BARROW NEUROLOGICAL INSTITUTE
🇺🇸United States
Pridopidine Shows Promise for ALS Subgroups in Healey Platform Trial
• Pridopidine demonstrated safety and potential efficacy in secondary endpoints for ALS patients, particularly improving motor speech performance, despite not meeting the primary endpoint in the Healey Platform Trial.
• The drug showed greater impact in a prespecified subgroup of patients with definite ALS and shorter disease duration (≤18 months), suggesting targeted efficacy in rapidly progressing cases.
• The HEALEY platform trial approach reduces placebo assignments, cuts time and costs, and has evaluated seven drugs since 2020, with Pridopidine now advancing to Phase 3 based on promising signals.
NeuroSense to Present Promising Phase 2b Data for Novel ALS Drug PrimeC at AAN Meeting
• NeuroSense Therapeutics will showcase new data from PrimeC's Phase 2b trial in ALS at the American Academy of Neurology Meeting on April 8, 2025, featuring presentations on clinical outcomes and microRNA biomarker findings.
• PrimeC, a fixed-dose combination of ciprofloxacin and celecoxib, demonstrated safety, efficacy, and target engagement in an 18-month trial, showing marked reduction in ALS progression and significant changes in disease-related biomarkers.
• The oral therapy employs a multi-targeted approach addressing several ALS pathways including motor neuron degeneration, inflammation, iron accumulation, and RNA regulation, potentially offering a disease-modifying treatment for the fatal neurodegenerative disease.
Ivy Brain Tumor Center Advances Novel Therapies for Brain Cancer
• The Ivy Brain Tumor Center is focusing on early-phase trials using pharmacokinetic and pharmacodynamic strategies to identify effective brain cancer drugs.
• The center is expanding its focus to Phase 3 studies and exploring new drug classes like PROTACs and antibody-drug conjugates for neuro-oncology.
• A Phase 0/1 trial (NCT06072586) is underway, evaluating BDTX-1535, a fourth-generation EGFR inhibitor, for recurrent high-grade glioma with EGFR alterations.
• Biomarker testing, including gene sequencing and immunohistochemistry, aids in identifying targetable alterations, exemplified by vorasidenib for IDH mutations.
Brain-Penetrant EGFR Inhibitor BDTX-1535 Shows Promise in Recurrent High-Grade Glioma Trial
• The Ivy Brain Tumor Center is conducting a phase 0/1 trial of BDTX-1535, a fourth-generation EGFR inhibitor, in patients with recurrent high-grade glioma.
• BDTX-1535 is designed to penetrate the blood-brain barrier, addressing a limitation of previous EGFR inhibitors in neuro-oncology.
• The trial assesses drug accumulation in tumor tissue and explores resistance mechanisms through cerebrospinal fluid biopsies.
• Early data focuses on identifying patient populations that benefit most and adapting treatment strategies based on real-time tumor evolution.
Gliofocus Study Expands Globally to Evaluate Niraparib in Glioblastoma
• The Gliofocus Study, a Phase 3 trial, has expanded to new sites in Europe and the U.S. to assess niraparib versus temozolomide in newly diagnosed MGMT-unmethylated glioblastoma.
• The trial, supported by GSK, aims to enroll 450 participants across 100+ sites in 13 countries, offering potential access to cutting-edge research for patients.
• Niraparib showed promising results in earlier trials, achieving higher concentrations in tumor tissue and a median overall survival of 20.3 months compared to historical 14 months with standard care.
• The expansion includes sites like Groupe Hospitalier Pitié-Salpêtrière and UC San Diego Moores Cancer Center, increasing access to novel treatment options for glioblastoma patients.
Neuralink Implants Third Human with Brain-Computer Interface, Plans Expansion
• Neuralink has successfully implanted its brain-computer interface device in a third human patient, with all three implants reported to be functioning well.
• The company plans to expand its human trials significantly, aiming to implant the device in 20 to 30 more individuals in 2025.
• Upgrades to the Neuralink device include more electrodes, higher bandwidth, and longer battery life, enhancing its potential for restoring digital independence.
• A new clinical trial site will open at the University of Miami, focusing on patients with paralysis or ALS, to further research and development of the technology.
Anavex Life Sciences Announces EMA Acceptance of Blarcamesine MAA for Alzheimer's Treatment
• The European Medicines Agency (EMA) has accepted Anavex's Marketing Authorization Application (MAA) for blarcamesine to treat Alzheimer's disease.
• Anavex presented Phase IIb/III ATTENTION-AD Open-Label-Extension (OLE) trial data at the J.P. Morgan 2025 Healthcare Conference.
• Preliminary Phase 2 clinical study results of ANAVEX3-71 for schizophrenia showed a dose-dependent effect on key EEG biomarkers.
• Anavex reported a strong cash position of $132.2 million, anticipating a cash runway of approximately four years.
HEALEY ALS Platform Trial: Streamlining Drug Development for Amyotrophic Lateral Sclerosis
• The HEALEY ALS Platform Trial is pioneering a new approach to accelerate the development of effective treatments for amyotrophic lateral sclerosis (ALS).
• By testing multiple drugs simultaneously using a shared infrastructure, the platform trial significantly reduces the time and cost associated with traditional clinical trials.
• Initial results from the platform have led to two drugs, CNM-Au8 and pridopidine, advancing to Phase 3 testing based on promising trends in secondary outcome measures and biomarker data.
• The collaborative effort unites patients, clinicians, scientists, and industry partners, fostering innovation and improving access to care across a network of over 70 sites.
Neuralink Receives Health Canada Approval for Brain-Computer Interface Trial
• Neuralink has received Health Canada approval to launch its first global trial, CAN-PRIME, evaluating its implantable brain-computer interface (BCI) device.
• The CAN-PRIME trial will assess the safety of Neuralink’s implant and surgical robot, as well as the preliminary functionality of the BCI in quadriplegic individuals.
• The trial will recruit Canadian residents with ALS or cervical spinal cord injury, focusing on those with limited or no hand use.
• University Health Network (UHN) hospital in Toronto will be the exclusive Canadian site for the wireless BCI implantation procedure.
Alzheimer's Blood Tests Poised for FDA Approval and Clinical Integration
• Plasma p-tau217 assays are demonstrating high accuracy, potentially serving as effective triage tests for Alzheimer's disease and meeting criteria for clinical use.
• Several vendors, including Fujirebio and Roche Diagnostics, have submitted applications to the FDA for approval of their Alzheimer's blood tests, with potential approval in 2025.
• Studies emphasize the need for real-world data and physician education to ensure appropriate use of these tests in diverse clinical settings and to avoid misdiagnosis.
• CMS reimbursement rates are under discussion, with concerns raised about proposed rates potentially hindering test implementation despite their clinical value.
Neuralink to Launch Trial Coupling Brain Implant with Robotic Arm for Quadriplegia
• Neuralink has received approval to launch a new feasibility trial pairing its brain implant with an experimental robotic arm, aiming to restore physical freedom.
• The trial will enroll participants from the ongoing PRIME study, which evaluates the safety and functionality of Neuralink's wireless brain-computer interface and surgical robot.
• The PRIME study assesses the initial impact of the implant on quadriplegic patients, enabling them to control external devices with their thoughts.
• Neuralink has also received approval to launch a trial of its device in Canada, recruiting six patients with paralysis, and has already implanted devices in two patients in the United States.
Neuralink Receives Approval for Brain Chip Trials in Canada
• Neuralink has received approval from Health Canada to begin clinical trials of its brain-implant device in Canada.
• The first trial site will be at Toronto Western Hospital, making it the first non-U.S. location for Neuralink's clinical research.
• Neuralink is recruiting patients with quadriplegia due to ALS or spinal cord injury for the initial Canadian trials.
• The company's device aims to enable patients to control external devices using their thoughts, with future applications targeting conditions like blindness.
Neuralink Receives Approval to Begin Brain Chip Trials in Canada
• Neuralink has received approval from Health Canada to launch its first clinical trial in Canada, marking the first trial site outside of the U.S.
• The trial will be conducted at Toronto Western Hospital, focusing on patients with quadriplegia due to ALS or spinal cord injury.
• The CAN-PRIME study will assess the functionality and safety of Neuralink's fully implanted wireless brain-computer interface (BCI) device.
• Neuralink is recruiting patients who can operate a computer or smartphone using only their thoughts, without physical movement or cables.
Neuralink Receives Approval to Launch Brain Chip Trial in Canada
• Neuralink has received approval from Health Canada to begin clinical trials of its brain-implant device, marking its first trial in the country.
• The trial will be conducted at Toronto Western Hospital, part of the University Health Network, making it the first non-US site for Neuralink's research.
• Neuralink is recruiting patients with quadriplegia due to ALS or spinal cord injury for the trial, which aims to enable control of external devices through thought.
• The company's initial focus is on assisting patients with motor impairments, with future aspirations including treatments for blindness and memory augmentation.
Real-World Leqembi Use Shows Comparable Safety to Clinical Trials
• Real-world data from the U.S. shows lecanemab's ARIA rates are consistent with clinical trials, despite a broader patient population with more comorbidities.
• In Japan, lecanemab use is growing rapidly, with ARIA rates half those observed in the U.S., mirroring findings from the Phase 3 Clarity trial.
• Excluding APOE4 homozygotes from lecanemab treatment may improve cognitive outcomes and reduce ARIA risk, influencing EMA's recommendation for approval.
• Monitoring protocols and multidisciplinary care are crucial for managing lecanemab treatment, ensuring appropriate MRI monitoring and patient safety.
Blarcamesine Shows Efficacy in Alzheimer's Patients with Specific SIGMAR1 Genotype
• Blarcamesine, a SIGMAR1 activator, slowed clinical progression in Alzheimer's patients without the SIGMAR1 rs1800866 gene variant in a Phase 2/3 trial.
• Patients with the SIGMAR1 wild-type gene demonstrated a 49.8% reduction in cognitive decline (ADAS-Cog13) and 33.7% less decline on the CDR-SB scale compared to placebo.
• The drug also significantly reduced brain atrophy and showed positive changes in plasma biomarkers related to amyloid and tau proteins.
• Anavex is on track for regulatory submission of blarcamesine in Europe (EMA) in the current quarter of 2024.
Lecanemab Shows Long-Term Benefits in Early Alzheimer's, Eisai Presents New Data
• Lecanemab demonstrates sustained cognitive and functional benefits over three years in patients with early Alzheimer's disease, particularly those with low amyloid or tau levels.
• New data suggests earlier initiation of lecanemab may positively impact disease progression, with a significant portion of patients showing improvement or no decline in cognitive measures.
• Eisai's research identifies a correlation between protofibrils in cerebrospinal fluid and neurodegenerative disease biomarkers, highlighting protofibrils' role in synaptic dysfunction.
• Analysis of real-world lecanemab use in the U.S. indicates expanding access, while long-term studies show sustained patient satisfaction and no new safety concerns.
Anavex's Blarcamesine Shows Efficacy in Phase IIb/III Alzheimer's Trial
• Anavex Life Sciences' blarcamesine (ANAVEX®2-73) demonstrated pre-specified clinical efficacy in a Phase IIb/III study for early Alzheimer's disease.
• The trial data confirmed blarcamesine's mechanism of action through SIGMAR1 gene activation, showing a significant reduction in clinical decline.
• Blarcamesine significantly slowed clinical progression by 36.3% in the primary endpoint ADAS-Cog13 (p=0.008) over 48 weeks in the ITT analysis.
• The company is preparing for regulatory submission in Europe (EMA) in the current quarter of 2024, with blarcamesine showing a good safety profile.
DOD Grant Funds Research into Neuropsychiatric Symptoms of Parkinson's Disease
• A collaborative research team received a $3 million grant from the Department of Defense to investigate the neuropsychiatric symptoms of Parkinson's disease.
• The research aims to understand how changes in serotonin-producing neurons contribute to cognitive, sleep, and psychiatric issues in Parkinson's patients.
• Researchers are exploring how L-DOPA, a common Parkinson's treatment, affects serotonin neurons and leads to uncontrolled dopamine release and side effects.
• The team has identified potential medications for repurposing to treat serotonin dysfunction in Parkinson's, with access to a large patient population for clinical trials.
Leading Neuro-Oncologist Emphasizes Importance of Proactive Mentorship in Brain Cancer Research
• Dr. Yoshie Umemura, Chief Medical Officer at Ivy Brain Tumor Center, advocates for aspiring neuro-oncologists to actively seek mentors beyond their institutions to advance in the field.
• The Barrow Neurological Institute division chief emphasizes that professionals should not limit themselves to local mentorship opportunities and encourages reaching out to potential mentors at conferences.
• Dr. Umemura stresses the importance of "paying it forward" through mentoring others, highlighting mentorship as a crucial element for advancing the brain cancer research field.
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