University of Iowa
🇺🇸United States
- Country
- 🇺🇸United States
- Ownership
- Private
- Established
- 1847-02-25
- Employees
- 251
- Market Cap
- -
- Website
- http://www.uiowa.edu
Phase 3 Trial Shows Promising Results for Novel Encapsulated Cell Therapy in MacTel Treatment
• Phase 3 clinical trials of NT-501, an encapsulated cell therapy delivering CNTF, demonstrated significant reduction in disease progression for macular telangiectasia type 2 patients, with up to 52% reduction in ellipsoid zone loss.
• The innovative implantable device, developed by Neurotech, maintains long-term viability with CNTF production documented for up to 14.5 years, offering a potential alternative to frequent intravitreal injections.
• FDA review of the therapy is currently underway with a PDUFA date set for March 18, 2025, marking a potential breakthrough in MacTel treatment.
Beta-Blockers Offer New Hope for Huntington's Disease Patients
A recent study from the University of Iowa suggests that beta-blockers, commonly used for heart conditions, may slow the progression of Huntington's disease. This finding is significant as it offers hope for delaying symptom onset and slowing disease progression in affected individuals. The research team plans to conduct clinical trials specifically for Huntington's, leveraging the safety and affordability of these well-understood medications.
Community Engagement Boosts Recruitment in FM-TIPS Fibromyalgia Trial
• The FM-TIPS trial, evaluating TENS for fibromyalgia pain, implemented community engagement strategies to enhance recruitment.
• Activating 25 clinics across seven Midwestern states, the study team incorporated a Community Engagement Coordinator to boost enrollment.
• Community engagement led to increased study inquiries, steady screening rates, and higher enrollment, particularly in targeted clinics.
• Clinicians reported feeling more supported, and researchers gained deeper insights into participant experiences outside of therapy sessions.
Large Meta-Analysis Reveals Link Between High Fluoride Exposure and Reduced IQ in Children
• A comprehensive meta-analysis of 74 studies involving 20,932 children found that higher fluoride exposure was associated with lower IQ scores, with an average difference of 7 IQ points between high and low exposure groups.
• Studies measuring fluoride in urine showed a dose-response relationship, with each 1-mg/L increase in urinary fluoride corresponding to a 1.63-point decrease in IQ scores.
• While current U.S. recommended fluoride levels are 0.7 mg/L, the study revealed insufficient data to evaluate IQ effects at this concentration, prompting calls for reassessment of fluoride exposure guidelines.
Beta-Blockers Linked to Delayed Onset and Slower Progression of Huntington's Disease
• A study using the Enroll-HD database found that beta-blocker use is associated with delayed motor diagnosis in premanifest Huntington's disease (HD).
• In patients with early motor symptoms of HD, beta-blockers correlated with slower progression of motor symptoms and cognitive decline.
• Unlike ACE inhibitors, beta-blockers showed a specific positive association with HD, suggesting benefits beyond treating cardiovascular issues.
• The study highlights correlation, not causation, necessitating further clinical trials to confirm the effects of beta-blockers on HD.
High-Dose Vitamin C Doubles Survival in Pancreatic Cancer Patients in Phase 2 Trial
• A Phase 2 clinical trial reveals that high-dose intravenous vitamin C, when combined with chemotherapy, doubles the overall survival rate for patients with late-stage metastatic pancreatic cancer.
• The study demonstrated an extension in overall survival from eight to sixteen months and progression-free survival from four to six months with the addition of high-dose vitamin C.
• Researchers found that high doses of vitamin C generate hydrogen peroxide, which selectively kills cancer cells, and patients tolerated chemotherapy better with the addition of vitamin C.
• Experts emphasize the need for larger, randomized Phase 3 trials to validate these findings before widespread clinical adoption, while remaining cautiously optimistic about vitamin C's potential.
Detalimogene Voraplasmid and Gemcitabine Combinations Show Promise in Bladder Cancer Trials
• Detalimogene voraplasmid (EG-70) shows a 71% any-time complete response rate in BCG-unresponsive NMIBC patients with carcinoma in situ in the LEGEND trial.
• Gemcitabine and docetaxel combination demonstrates a 92% complete response rate at 12 months in BCG-naive, high-risk NMIBC patients.
• Gemcitabine-BCG combination shows a 94% complete response rate at 6 months in BCG-exposed NMIBC, suggesting enhanced efficacy and safety.
• Studies highlight the potential of novel intravesical therapies to improve outcomes and reduce the need for radical cystectomy in bladder cancer.
Talquetamab and Teclistamab Combination Shows Promise in Relapsed/Refractory Multiple Myeloma
• A Phase Ib/II trial of talquetamab plus teclistamab shows promising antitumor activity and durable responses in relapsed/refractory multiple myeloma patients.
• The combination therapy demonstrated an 80% response rate in patients administered the recommended Phase II dose, including those with extramedullary disease.
• The study also reported a higher incidence of grade 3 or 4 infections compared to either drug as monotherapy, warranting further investigation.
• These results suggest the dual-targeting combination therapy may be a valuable option for heavily pretreated multiple myeloma patients.
Enhancing Glycolysis via PGK1 Shows Promise in Parkinson's Disease Treatment
• Researchers identify phosphoglycerate kinase 1 (PGK1) as a rate-limiting enzyme in glycolysis, crucial for neuronal ATP production.
• Overexpression of PGK1 or treatment with terazosin rescued synaptic function in neurons with Parkinson's-linked mutations.
• Study validates PGK1 as a therapeutic target, suggesting interventions that enhance its activity could benefit Parkinson's patients.
• Findings support the theory that Parkinson's, despite varied causes, involves neuronal energetic deficits, guiding new treatment designs.
GE HealthCare's SIGNA MAGNUS Head-Only MRI System Receives FDA Clearance
• GE HealthCare's SIGNA MAGNUS, a 3.0T head-only MRI, has received FDA clearance, offering advanced neuroimaging capabilities for clinical and research use.
• The SIGNA MAGNUS features an innovative asymmetric gradient coil design, enhancing gradient performance and enabling high-resolution imaging with shorter scan times.
• This new MRI technology aims to improve the detection of neurological, oncological, and psychiatric conditions through advanced imaging and biomarker research.
• The system's HyperG gradient technology achieves high performance levels, improving spatial resolution and image clarity for more accurate diagnoses.
PMV Pharma's Rezatapopt Shows Promise in TP53-Mutated Solid Tumors and AML/MDS
• PMV Pharmaceuticals' PYNNACLE trial advances with Rezatapopt monotherapy for TP53 Y220C mutated solid tumors, showing over 75% site activation across multiple regions.
• A Phase 1b study will assess Rezatapopt with azacitidine for relapsed/refractory AML/MDS with TP53 Y220C mutation, beginning enrollment in early 2025.
• Rezatapopt's exposure levels improved when taken with food, reducing gastrointestinal adverse events, supporting the 2000 mg QD dose in the PYNNACLE Phase 2 study.
Wave Life Sciences Achieves First Human RNA Editing, Advances Pipeline
• Wave Life Sciences reported the first-ever RNA editing in humans with WVE-006 for alpha-1 antitrypsin deficiency (AATD), showing increased wild-type AAT protein levels.
• Clinical trial for WVE-007, a GalNAc-siRNA targeting INHBE for obesity treatment, is on track for Q1 2025, with potential for monotherapy or combination with GLP-1s.
• FDA provides supportive initial feedback on WVE-003 for Huntington’s disease, open to caudate atrophy as a biomarker for accelerated approval; IND submission expected in 2H 2025.
• Positive interim data from FORWARD-53 study of WVE-N531 for Duchenne muscular dystrophy (DMD) shows dystrophin expression; complete data and regulatory feedback expected in Q1 2025.
Trodelvy's Bladder Cancer Approval Withdrawn After TROPiCS-04 Fails to Meet Overall Survival Endpoint
• Gilead Sciences voluntarily withdrew FDA approval for Trodelvy in advanced urothelial cancer after the TROPiCS-04 trial failed to meet its primary endpoint of overall survival.
• The accelerated approval, granted in 2021, was contingent on confirmatory data, which TROPICS-04 did not provide, impacting treatment options for bladder cancer patients.
• Trodelvy remains approved for certain breast cancer indications, highlighting the importance of cancer-specific clinical trials to determine efficacy.
• Patients currently on Trodelvy for urothelial cancer should consult their healthcare providers to discuss alternative treatment strategies.
New Alzheimer's Treatments Slow Disease Progression, Offer Hope for Early-Stage Patients
• Lecanemab and donanemab have received full FDA approval, demonstrating both amyloid removal and clinical benefit in slowing Alzheimer's progression.
• These disease-modifying treatments are most effective in the early stages of Alzheimer's, specifically mild cognitive impairment or mild dementia.
• Anti-amyloid therapies carry risks, including amyloid-related imaging abnormalities (ARIA), necessitating careful monitoring and MRI examinations.
• Early recognition and triaging of appropriate patients are crucial for the successful implementation of these treatments.
Fabhalta Demonstrates Sustained Efficacy in C3 Glomerulopathy Patients at 12 Months
• Novartis presented 12-month data from the Phase III APPEAR-C3G study, showing Fabhalta (iptacopan) significantly reduced proteinuria in C3 glomerulopathy (C3G) patients.
• The study demonstrated that Fabhalta, an oral Factor B inhibitor, led to sustained proteinuria reduction as early as 14 days, with continued improvement over 12 months.
• Fabhalta also improved estimated glomerular filtration rate (eGFR) slope compared to patients' historical decline, with a favorable safety profile observed during the study.
• Regulatory submissions for Fabhalta in C3G are completed in the EU, China, and Japan, with submissions expected in the US by the end of the year.
Fabhalta Demonstrates Sustained Efficacy in Phase III APPEAR-C3G Trial for C3 Glomerulopathy
• Fabhalta (iptacopan) showed sustained proteinuria reduction at 12 months in patients with C3 glomerulopathy (C3G), according to new data from the APPEAR-C3G trial.
• The study observed improvement in estimated glomerular filtration rate (eGFR) slope upon Fabhalta initiation, indicating a positive impact on kidney function.
• Fabhalta maintained a favorable safety profile throughout the study, with no new safety signals identified.
• Regulatory submissions for Fabhalta in C3G are completed in the EU, China, and Japan, with submission expected in the US by year-end.
Pegcetacoplan Shows Promise in C3 Glomerulopathy and IC-MPGN: VALIANT Trial Results
• Pegcetacoplan demonstrated a statistically significant reduction in proteinuria compared to placebo in patients with C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN).
• The VALIANT trial showed that pegcetacoplan led to a significant reduction in C3c staining on renal biopsy, indicating a potential impact on disease pathology.
• Treatment with pegcetacoplan resulted in stabilization of eGFR, suggesting a potential benefit in preserving kidney function in this patient population.
• The safety profile of pegcetacoplan was generally favorable, with the frequency and severity of adverse events being similar between the treatment and placebo groups.
Fabhalta Demonstrates Sustained Efficacy in C3 Glomerulopathy Patients at One Year
• Fabhalta (iptacopan) maintained significant proteinuria reduction in C3G patients after 12 months, according to new data from the Phase III APPEAR-C3G study.
• The study showed improvement in estimated glomerular filtration rate (eGFR) slope upon Fabhalta initiation, indicating a positive impact on kidney function decline.
• Fabhalta, an oral alternative complement pathway inhibitor, exhibited a favorable safety profile with no new safety concerns identified during the study.
• Regulatory submissions for Fabhalta in C3G are completed in the EU, China, and Japan, with US submission expected by the end of the year.
ASN Kidney Week: Promising Clinical Trial Results Highlight Advances in Kidney Disease Treatment
• A trial showed targeted correction of hyponatremia in hospitalized patients did not improve mortality and rehospitalization rates.
• Pegcetacoplan significantly reduced proteinuria in patients with glomerulopathy in the VALIANT trial, showing favorable safety.
• Semaglutide reduced major kidney outcomes in type 2 diabetes patients with chronic kidney disease, irrespective of CKD severity.
• Acellular tissue-engineered vessels showed higher success rates in maintaining vascular access for hemodialysis, especially in high-risk patients.
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