Batten Disease — Research Summary
Printed from RareWays (rareways.com.au) on 5 April 2026
For general awareness only. Not medical advice. Discuss all care options with your healthcare team.
5 Most Recent Research Articles
- 1.
Oncogenic crosstalk between ErbB2, nucleolin and Ras.
Zysman Gal et al. — Cellular signalling (1 June 2026)
https://pubmed.ncbi.nlm.nih.gov/41672208/
- 2.
Towards understanding memory buffer based continual learning.
Zheng Guodong et al. — Neural networks : the official journal of the International Neural Network Society (1 May 2026)
https://pubmed.ncbi.nlm.nih.gov/41483569/
- 3.
The 1 -Cys peroxiredoxin, PRDX-6, suppresses an NHR-49-dependent pro-survival response, including the Flavin monooxygenase, FMO-2, that protects against fungal and bacterial infection.
Button Emma L et al. — Redox biology (1 April 2026)
https://pubmed.ncbi.nlm.nih.gov/41702017/
- 4.
Nucleolin (NCL) is essential for hair cell stereocilia maintenance and auditory function in mice.
Zong Wen et al. — Journal of cell science (23 March 2026)
https://pubmed.ncbi.nlm.nih.gov/41866947/
- 5.
Two thermostable and halotolerant recombinant β-glucosidases from Thermothelomyces thermophilus and their role in lignocellulosic biomass saccharification.
da Rocha Fortes Saraiva Ana Luiza et al. — International journal of biological macromolecules (19 March 2026)
https://pubmed.ncbi.nlm.nih.gov/41864383/
Clinical Trials — Currently Recruiting (Australia)
Ask your doctor whether you or your child may be eligible for any of these trials.
- 1.
A Global Phase III Study of Rilvegostomig or Pembrolizumab Plus Chemotherapy for First-Line Treatment of Metastatic Squamous Non-small Cell Lung Cancer (NSCLC)
Recruiting — Phase 3 — AstraZeneca
https://clinicaltrials.gov/study/NCT06692738
- 2.
A Study to Assess Adverse Events and Change in Disease Activity Comparing Oral Upadacitinib to Subcutaneous Dupilumab in Children From 2 to Less Than 12 Years of Age With Moderate to Severe Atopic Dermatitis
Recruiting — Phase 3 — AbbVie
https://clinicaltrials.gov/study/NCT06461897
- 3.
Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 in Clinical Trials
Recruiting — Phase 3 — Novartis Pharmaceuticals
https://clinicaltrials.gov/study/NCT05335876
- 4.
Implementation of Metformin theraPy to Ease Decline of Kidney Function in Polycystic Kidney Disease (IMPEDE-PKD)
Recruiting — Phase 3 — The University of Queensland
https://clinicaltrials.gov/study/NCT04939935
- 5.
Randomized, Embedded, Multifactorial Adaptive Platform Trial for Community- Acquired Pneumonia
Recruiting — Phase 3 — UMC Utrecht
https://clinicaltrials.gov/study/NCT02735707
- 6.
Brentuximab Vedotin in Early Stage Hodgkin Lymphoma
Recruiting — Phase 3 — University College, London
https://clinicaltrials.gov/study/NCT04685616
- 7.
FaR-RMS: An Overarching Study for Children and Adults With Frontline and Relapsed RhabdoMyoSarcoma
Recruiting — Phase 1 — University of Birmingham
https://clinicaltrials.gov/study/NCT04625907
- 8.
A Global Phase III Study of Rilvegostomig or Pembrolizumab Plus Chemotherapy for First-Line Treatment of Metastatic Non-squamous NSCLC
Recruiting — Phase 3 — AstraZeneca
https://clinicaltrials.gov/study/NCT06627647
- 9.
A Study of a Deuterated Psilocin Analog (CYB003) in Humans With Major Depressive Disorder
Recruiting — Phase 3 — Cybin IRL Limited
https://clinicaltrials.gov/study/NCT06793397
- 10.
The Early Valve Replacement in Severe ASYmptomatic Aortic Stenosis Study
Recruiting — Na — University of Leicester
https://clinicaltrials.gov/study/NCT04204915
- 11.
A Master Protocol of Multiple Agents in Adults With Metabolic Dysfunction-Associated Steatotic Liver Disease (SYNERGY-Outcomes)
Recruiting — Phase 3 — Eli Lilly and Company
https://clinicaltrials.gov/study/NCT07165028
- 12.
An Open-label Study to Investigate ECUR-506 in Male Babies Less Than 9 Months of Age With Neonatal Onset OTC Deficiency
Recruiting — Phase 1 — iECURE, Inc.
https://clinicaltrials.gov/study/NCT06255782
- 13.
A Phase 3 Study to Evaluate Petosemtamab Plus Pembrolizumab vs Pembrolizumab in First-line Treatment of Head and Neck Cancer (LiGeR - HN1)
Recruiting — Phase 3 — Merus B.V.
https://clinicaltrials.gov/study/NCT06525220
- 14.
Study to Investigate Lifileucel Regimen Plus Pembrolizumab Compared With Pembrolizumab Alone in Participants With Untreated Advanced Melanoma.
Recruiting — Phase 3 — Iovance Biotherapeutics, Inc.
https://clinicaltrials.gov/study/NCT05727904
- 15.
Safety and Efficacy of BNT327, an Investigational Therapy in Combination With Chemotherapy for Patients With Untreated Small-cell Lung Cancer
Recruiting — Phase 3 — BioNTech SE
https://clinicaltrials.gov/study/NCT06712355
- 16.
A Study of Pasritamig With Docetaxel Versus Docetaxel in Participants With Metastatic Castration-Resistant Prostate Cancer
Recruiting — Phase 3 — Janssen Research & Development, LLC
https://clinicaltrials.gov/study/NCT07225946
- 17.
AZD0486 1L Therapy for Elderly or Unfit Participants With LBCL
Recruiting — Phase 3 — AstraZeneca
https://clinicaltrials.gov/study/NCT07215585
- 18.
ONC201 in H3 K27M-mutant Diffuse Glioma Following Radiotherapy (the ACTION Study)
Recruiting — Phase 3 — Jazz Pharmaceuticals
https://clinicaltrials.gov/study/NCT05580562
- 19.
Pragmatic Randomised Trial of High Or Standard PHosphAte Targets in End-stage Kidney Disease (PHOSPHATE)
Recruiting — Na — The University of Queensland
https://clinicaltrials.gov/study/NCT03573089
- 20.
A Clinical Study of Sotatercept (MK-7962) in People With Pulmonary Arterial Hypertension (MK-7962-038)
Recruiting — Phase 3 — Merck Sharp & Dohme LLC
https://clinicaltrials.gov/study/NCT07218029
- 21.
Bomedemstat vs Hydroxyurea for Essential Thrombocythemia (MK-3543-007)
Recruiting — Phase 3 — Merck Sharp & Dohme LLC
https://clinicaltrials.gov/study/NCT06456346
- 22.
A Phase III Study to Investigate the Efficacy and Safety of Anifrolumab in Adults With Chronic and/or Subacute Cutaneous Lupus Erythematosus
Recruiting — Phase 3 — AstraZeneca
https://clinicaltrials.gov/study/NCT06015737
- 23.
A Phase 3 Study to Evaluate Petosemtamab Compared With Investigator's Choice Monotherapy in Previously Treated Head and Neck Squamous Cell Carcinoma Patients
Recruiting — Phase 3 — Merus B.V.
https://clinicaltrials.gov/study/NCT06496178
- 24.
Maridebart Cafraglutide in Heart Failure With Preserved or Mildly Reduced Ejection Fraction and Obesity
Recruiting — Phase 3 — Amgen
https://clinicaltrials.gov/study/NCT07037459
- 25.
A Phase 3, Placebo-Controlled Study to Investigate LP352 in Children and Adults With Dravet Syndrome (DS)
Recruiting — Phase 3 — Longboard Pharmaceuticals
https://clinicaltrials.gov/study/NCT06660394
- 26.
ARDS in Children and ECMO Initiation Strategies Impact on Neurodevelopment (ASCEND)
Recruiting — University of Michigan
https://clinicaltrials.gov/study/NCT05388708
- 27.
A Study to Evaluate the Safety, Tolerability, Drug Levels, and Preliminary Efficacy of BMS-986507 Combinations in Adult Participants With Advanced Solid Tumors
Recruiting — Phase 1 — Bristol-Myers Squibb
https://clinicaltrials.gov/study/NCT06618287
- 28.
A Study Evaluating Efruxifermin in Subjects With Compensated Cirrhosis Due to NASH/MASH
Recruiting — Phase 3 — Akero Therapeutics, Inc
https://clinicaltrials.gov/study/NCT06528314
- 29.
A Study of Tucatinib With Trastuzumab and mFOLFOX6 Versus Standard of Care Treatment in First-line HER2+ Metastatic Colorectal Cancer
Recruiting — Phase 3 — Seagen, a wholly owned subsidiary of Pfizer
https://clinicaltrials.gov/study/NCT05253651
- 30.
A Study to Investigate the Effect of AZD6793 in Participants With Moderate to Very Severe Chronic Obstructive Pulmonary Disease
Recruiting — Phase 2 — AstraZeneca
https://clinicaltrials.gov/study/NCT07082738
- 31.
A Study to Investigate the Safety, Tolerability, and Efficacy of SAR446268, an Adeno-associated Viral Vector-mediated Gene Therapy in Participants Aged 10 to 50 Years of Age With Non-congenital Myotonic Dystrophy Type 1
Recruiting — Phase 1 — Sanofi
https://clinicaltrials.gov/study/NCT06844214
- 32.
A Study Evaluating Efruxifermin in Subjects With Non-Cirrhotic Nonalcoholic Steatohepatitis (NASH)/Metabolic Dysfunction-Associated Steatohepatitis (MASH) and Fibrosis
Recruiting — Phase 3 — Akero Therapeutics, Inc
https://clinicaltrials.gov/study/NCT06215716
- 33.
A Study to Compare the Efficacy and Safety of BMS-986393 Versus Standard Regimens in Adult Participants With Relapsed or Refractory and Lenalidomide-exposed Multiple Myeloma (QUINTESSENTIAL-2)
Recruiting — Phase 3 — Juno Therapeutics, Inc., a Bristol-Myers Squibb Company
https://clinicaltrials.gov/study/NCT06615479
- 34.
Active Surveillance, Bleomycin, Etoposide, Carboplatin or Cisplatin in Treating Pediatric and Adult Patients With Germ Cell Tumors
Recruiting — Phase 3 — Children's Oncology Group
https://clinicaltrials.gov/study/NCT03067181
- 35.
The Evaluation of PC14586 in Patients With Advanced Solid Tumors Harboring a TP53 Y220C Mutation (PYNNACLE)
Recruiting — Phase 1 — PMV Pharmaceuticals, Inc
https://clinicaltrials.gov/study/NCT04585750
- 36.
A Phase 2 Master Protocol Assessing Inebilizumab and Blinatumomab in Autoimmune Diseases
Recruiting — Phase 2 — Amgen
https://clinicaltrials.gov/study/NCT06570798
- 37.
Staphylococcus Aureus Network Adaptive Platform Trial
Recruiting — Phase 4 — University of Melbourne
https://clinicaltrials.gov/study/NCT05137119
- 38.
Study to Determine if BHV-7000 is Effective and Safe in Adults With Refractory Focal Onset Epilepsy
Recruiting — Phase 2 — Biohaven Therapeutics Ltd.
https://clinicaltrials.gov/study/NCT06309966
- 39.
Extension Study for the Port Delivery System With Ranibizumab (Portal)
Recruiting — Phase 3 — Hoffmann-La Roche
https://clinicaltrials.gov/study/NCT03683251
- 40.
Study of Subcutaneous Epcoritamab in Combination With Intravenous Rituximab and Oral Lenalidomide (R2) to Assess Adverse Events and Change in Disease Activity in Adult Participants With Previously Untreated Follicular Lymphoma
Recruiting — Phase 3 — Genmab
https://clinicaltrials.gov/study/NCT06191744
- 41.
MEDI5752 in Combination With Carboplatin Plus Pemetrexed in Unresectable Pleural Mesothelioma
Recruiting — Phase 3 — AstraZeneca
https://clinicaltrials.gov/study/NCT06097728
- 42.
Saruparib (AZD5305) vs Placebo in Men With Metastatic Castration-Sensitive Prostate Cancer Receiving Physician's Choice New Hormonal Agents
Recruiting — Phase 3 — AstraZeneca
https://clinicaltrials.gov/study/NCT06120491
- 43.
A Study of Subcutaneously Injected Epcoritamab Plus Oral Lenalidomide Tablets Compared to Intravenously (IV) Infused Rituximab Plus IV Infused Gemcitabine and IV Infused Oxaliplatin in Adult Participants With Relapsed or Refractory Diffuse Large B-Cell Lymphoma
Recruiting — Phase 3 — Genmab
https://clinicaltrials.gov/study/NCT06508658
- 44.
A Study to Evaluate Mezigdomide, Bortezomib and Dexamethasone (MEZIVd) Versus Pomalidomide, Bortezomib and Dexamethasone (PVd) in Participants With Relapsed or Refractory Multiple Myeloma (RRMM)
Recruiting — Phase 3 — Celgene
https://clinicaltrials.gov/study/NCT05519085
- 45.
A Clinical Trial of Study Medicine (Marstacimab) in Pediatric Patients With Hemophilia A or Hemophilia B
Recruiting — Phase 3 — Pfizer
https://clinicaltrials.gov/study/NCT05611801
- 46.
Enroll -HD: A Prospective Registry Study in a Global Huntington's Disease Cohort
Recruiting — CHDI Foundation, Inc.
https://clinicaltrials.gov/study/NCT01574053
- 47.
Substudy 01A: Zilovertamab Vedotin in Pediatric and Young Adult Participants With Hematologic Malignancies or Solid Tumors (MK-9999-01A/LIGHTBEAM-U01)
Recruiting — Phase 1 — Merck Sharp & Dohme LLC
https://clinicaltrials.gov/study/NCT06395103
- 48.
ARTEMIS - A Research Study to Look at How Ziltivekimab Works Compared to Placebo in People With a Heart Attack
Recruiting — Phase 3 — Novo Nordisk A/S
https://clinicaltrials.gov/study/NCT06118281
- 49.
EASi-PROTKT™ - A Study to Test Vicadrostat (BI 690517) Taken Together With Empagliflozin in People With Type 2 Diabetes, High Blood Pressure, and Cardiovascular Disease
Recruiting — Phase 3 — Boehringer Ingelheim
https://clinicaltrials.gov/study/NCT07064473
- 50.
A Study to Investigate Progression-Free Survival With Sonrotoclax Plus Obinutuzumab Or Sonrotoclax Plus Rituximab Compared With Venetoclax Plus Rituximab Treatment In Patients With Relapsed and/or Refractory Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma (CELESTIAL-RRCLL)
Recruiting — Phase 3 — BeOne Medicines
https://clinicaltrials.gov/study/NCT06943872
- 51.
A Study to Evaluate the Safety and Efficacy of Pumitamig in Combination With Chemotherapy Versus Bevacizumab in Combination With Chemotherapy in Participants With Previously Untreated, Unresectable, or Metastatic Colorectal Cancer
Recruiting — Phase 2 — Bristol-Myers Squibb
https://clinicaltrials.gov/study/NCT07221357
- 52.
Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-101 in Participants With Myotonic Dystrophy Type 1
Recruiting — Phase 1 — Dyne Therapeutics
https://clinicaltrials.gov/study/NCT05481879
- 53.
A Study to Learn About Study Medicine ALTA2618 in Adults With AKT1 E17K-Mutant Solid Tumors
Recruiting — Phase 1 — Alterome Therapeutics, Inc.
https://clinicaltrials.gov/study/NCT06533059
- 54.
A Study of BMS-986504 in Participants With Pre-treated Advanced or Metastatic Non-small Cell Lung Cancer (NSCLC) With Homozygous MTAP Deletion (MountainTAP-9)
Recruiting — Phase 2 — Bristol-Myers Squibb
https://clinicaltrials.gov/study/NCT06855771
- 55.
A Study of Surovatamig (AZD0486) Plus Rituximab in Previously Untreated Follicular Lymphoma Patients
Recruiting — Phase 3 — AstraZeneca
https://clinicaltrials.gov/study/NCT06549595
- 56.
A Study to Find Out How EMPAgliflozin is Tolerated and if it Helps Children and Adolescents With Chronic KIDNEY Disease (EMPA-KIDNEY® Kids)
Recruiting — Phase 3 — Boehringer Ingelheim
https://clinicaltrials.gov/study/NCT07107945
- 57.
A Study to Assess Disease Activity and Safety of Lutikizumab in Adult and Adolescent Participants With Moderate to Severe Hidradenitis Suppurativa
Recruiting — Phase 3 — AbbVie
https://clinicaltrials.gov/study/NCT06468228
- 58.
DAY101 vs. Standard of Care Chemotherapy in Pediatric Participants With Low-Grade Glioma Requiring First-Line Systemic Therapy (LOGGIC/FIREFLY-2)
Recruiting — Phase 3 — Day One Biopharmaceuticals, Inc.
https://clinicaltrials.gov/study/NCT05566795
- 59.
A Phase I/II Study of AZD4512 Monotherapy or in Combination With Anticancer Agents in Participants With Relapsed/Refractory B-cell Non-Hodgkin Lymphoma
Recruiting — Phase 1 — AstraZeneca
https://clinicaltrials.gov/study/NCT07123454
- 60.
A Study to Investigate LP352 in Children and Adults With Developmental and Epileptic Encephalopathies (DEE)
Recruiting — Phase 3 — Longboard Pharmaceuticals
https://clinicaltrials.gov/study/NCT06719141
- 61.
Study of AZD9574 as Monotherapy and in Combination With Anti-cancer Agents in Participants With Advanced Solid Malignancies
Recruiting — Phase 1 — AstraZeneca
https://clinicaltrials.gov/study/NCT05417594
- 62.
A Long-term Extension Study of JNJ-81201887 (AAVCAGsCD59) Parent Studies in Participants With Geographic Atrophy (GA) Secondary to Age-related Macular Degeneration (AMD)
Recruiting — Phase 2 — Janssen Research & Development, LLC
https://clinicaltrials.gov/study/NCT06635148
- 63.
A Study Evaluating the Safety, Pharmacokinetics, and Efficacy of Alectinib in Pediatric Participants With ALK Fusion-Positive Solid or CNS Tumors
Recruiting — Phase 1 — Hoffmann-La Roche
https://clinicaltrials.gov/study/NCT04774718
- 64.
A Rollover Extension Program (REP) to Evaluate the Long-term Safety and Tolerability of Open Label Iptacopan/LNP023 in Participants With Primary IgA Nephropathy
Recruiting — Phase 3 — Novartis Pharmaceuticals
https://clinicaltrials.gov/study/NCT04557462
- 65.
A Phase 3 Study Evaluating Efficacy and Safety of Lanifibranor Followed by an Active Treatment Extension in Adult Patients With (NASH) and Fibrosis Stages F2 and F3 ( NATiV3 )
Recruiting — Phase 3 — Inventiva Pharma
https://clinicaltrials.gov/study/NCT04849728
- 66.
INBRX-106 in Combination With Pembrolizumab in First-line PD-L1 CPS≥20 HNSCC
Recruiting — Phase 2 — Inhibrx Biosciences, Inc
https://clinicaltrials.gov/study/NCT06295731
- 67.
Real-World Study to Assess Subcutaneous Epcoritamab in Adult Participants With Diffuse Large B-Cell and Follicular Lymphoma
Recruiting — AbbVie
https://clinicaltrials.gov/study/NCT06830759
- 68.
A Long-term Extension Study of PCI-32765 (Ibrutinib)
Recruiting — Phase 3 — Janssen Research & Development, LLC
https://clinicaltrials.gov/study/NCT01804686
- 69.
An Early Access Study of Ivosidenib in Patients With a Pretreated Locally Advanced or Metastatic Cholangiocarcinoma
Recruiting — Phase 3 — Servier Affaires Médicales
https://clinicaltrials.gov/study/NCT05876754
- 70.
A Study of Patritumab Deruxtecan in Pediatric Participants With Relapsed or Refractory Solid Tumors (MK-9999-01C/LIGHTBEAM-U01)
Recruiting — Phase 1 — Merck Sharp & Dohme LLC
https://clinicaltrials.gov/study/NCT06941272
- 71.
Outpatient Treatment With Anti-Coronavirus Immunoglobulin
Recruiting — Phase 3 — University of Minnesota
https://clinicaltrials.gov/study/NCT04910269
- 72.
Product Surveillance Registry
Recruiting — Medtronic
https://clinicaltrials.gov/study/NCT01524276
- 73.
Long-term Outcomes of Lidocaine Infusions for Post-Operative Pain (LOLIPOP) Trial
Recruiting — Phase 3 — Monash University
https://clinicaltrials.gov/study/NCT05072314
- 74.
A Study to Assess the Efficacy and Safety of Induction and Maintenance Therapy With Afimkibart (RO7790121) in Participants With Moderately to Severely Active Crohn's Disease
Recruiting — Phase 3 — Hoffmann-La Roche
https://clinicaltrials.gov/study/NCT06819878
- 75.
A Clinical Study to Test if an Investigational Treatment Called BNT326 is Safe and Potentially Beneficial When Used Alone or in Combination With Other Investigational Treatments Such as BNT327, for People With Advanced Malignant Tumors
Recruiting — Phase 1 — BioNTech SE
https://clinicaltrials.gov/study/NCT07070232
- 76.
A Study to Investigate Multiple Sclerosis Relapse Prevention With mRNA-1195 Compared With Placebo in Participants Aged 18 to ≤55 Years
Recruiting — Phase 2 — ModernaTX, Inc.
https://clinicaltrials.gov/study/NCT06735248
- 77.
A Study to Evaluate Tovorafenib in Pediatric and Young Adult Participants With Relapsed or Progressive Low-Grade Glioma and Advance Solid Tumors
Recruiting — Phase 2 — Day One Biopharmaceuticals, Inc.
https://clinicaltrials.gov/study/NCT04775485
- 78.
A Study to Investigate the Biological Effects of Saruparib (AZD5305), Darolutamide, and in Combination in Men With Newly Diagnosed Prostate Cancer.
Recruiting — Phase 1 — AstraZeneca
https://clinicaltrials.gov/study/NCT05938270
- 79.
A Study to Assess Efficacy and Safety of Pembrolizumab With or Without Sacituzumab Tirumotecan (MK- 2870) in Adult Participants With Resectable Non Small Cell Lung Cancer (NSCLC) Not Achieving Pathological Complete Response (pCR) (MK-2870-019)
Recruiting — Phase 3 — Merck Sharp & Dohme LLC
https://clinicaltrials.gov/study/NCT06312137
- 80.
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Recruiting — Sanford Health
https://clinicaltrials.gov/study/NCT01793168
- 81.
EXtubation Related Complications - the EXTUBE Study (EXTUBE)
Recruiting — University Health Network, Toronto
https://clinicaltrials.gov/study/NCT06442930
- 82.
A Multi-Arm, Platform Trial For Relapsed Neuroblastoma
Recruiting — Phase 1 — University of Birmingham
https://clinicaltrials.gov/study/NCT07334301
- 83.
Clinical Trial Evaluating the Safety, Tolerability and Preliminary Efficacy of BNT116 Alone and in Combinations in Patients With Advanced Non-small Cell Lung Cancer
Recruiting — Phase 1 — BioNTech SE
https://clinicaltrials.gov/study/NCT05142189
- 84.
Efficacy and Safety Study of Frexalimab (SAR441344) in Adults With Nonrelapsing Secondary Progressive Multiple Sclerosis
Recruiting — Phase 3 — Sanofi
https://clinicaltrials.gov/study/NCT06141486
- 85.
A Dose-Escalation and Expansion Study of BGB-16673 in Participants With B-Cell Malignancies
Recruiting — Phase 1 — BeOne Medicines
https://clinicaltrials.gov/study/NCT05006716
- 86.
A Study of Tirzepatide in Adolescents With Obesity and Weight-Related Comorbidities (SURMOUNT-ADOLESCENTS-2)
Recruiting — Phase 3 — Eli Lilly and Company
https://clinicaltrials.gov/study/NCT06439277
- 87.
Physiology-guided vs Angiography-guided Non-culprit Lesion Complete Revascularization for Acute MI & Multivessel Disease
Recruiting — Na — Population Health Research Institute
https://clinicaltrials.gov/study/NCT05701358
Source: RareWays research directory. Data from PubMed, Europe PMC, OpenAlex, ClinicalTrials.gov.
Always verify information with your healthcare team before making any decisions about your care.
Batten Disease
Batten disease (neuronal ceroid lipofuscinosis) is a group of rare and fatal genetic neurological disorders that cause progressive deterioration of vision, seizures, and cognitive and motor decline, often beginning in childhood. The first approved enzyme replacement therapy (cerliponase alfa) targets CLN2 disease and has slowed progression.
Most Recent Research
Overexpression of ErbB receptors, their signaling components and expression of mutant Ras are major contributors to cancer development. Mutant Ras can affect ErbB receptors activation, and the nucleolar protein nucleolin (NCL) can bind and activate ErbB receptors. Previously it was also demonstrated that activated Ras could interact and enhance nucleolin and ErbB1 receptors interaction. Moreover, it was shown that this interaction enhances tumor cell growth. Since ErbB receptors are major oncogenes and ErbB2 is often overexpressed in many cancer types, in the present study we explored the link between ErbB2, Ras and nucleolin oncogenes. Using cell biology and biochemistry methods, we have demonstrated that NCL interaction with ErbB2 is enhanced by H-Ras (12 V) protein. The three proteins colocalize mainly at the plasma membrane, and in other intracellular sites. Using proximity ligation assay, we show that H-Ras (12 V) enhances the formation of ErbB2/NCL complexes, which reside mainly at the plasma membrane but may also be in the cytosol and nucleus. The interaction of the three oncogenes is associated with enhanced ErbB2 phosphorylation as well as ErbB2 mediated downstream signaling to Erk and Akt. Moreover, expression of the three proteins enhances colony formation, anchorage independent growth and cell migration. Thus, activated Ras can enhance the oncogenic effect of ErbB2 and NCL. These findings can be used to develop new ways to treat tumors that overexpress ErbB2/nucleolin and mutant Ras.
This information is for general awareness only.
For guidance specific to your situation, please speak with your healthcare team.