MICHAEL R PINSKY MD LLC

INSPIRE Trials: Computerized Alerts Improve Antibiotic Selection in Hospitalized Patients by Up to 35%

a month ago

• NIH-funded clinical trials involving over 316,000 patients across 92 hospitals demonstrate that computerized alerts can improve appropriate antibiotic selection by 35% for abdominal infections and 28% for skin and soft tissue infections. • The INSPIRE trials used patient-specific data to identify individuals at low risk for antibiotic-resistant infections, prompting physicians to switch from broad-spectrum to standard antibiotics when appropriate. • Researchers from UC Irvine, Harvard Pilgrim Health Care Institute, and HCA Healthcare collaborated on the studies, which could significantly reduce antibiotic resistance and improve patient outcomes nationwide.

First Human Transplant of Sperm-Producing Stem Cells Offers Hope for Childhood Cancer Survivors

2 months ago

• A 26-year-old bone cancer survivor has received the first known transplant of sperm-producing stem cells preserved from his childhood, marking a significant advancement in fertility preservation for pediatric cancer patients. • Approximately one-third of childhood cancer survivors face infertility from treatments, with prepubertal patients having limited options as they cannot bank mature reproductive cells before therapy. • The experimental procedure, developed at the University of Pittsburgh Medical Center, follows successful animal studies and represents a potential breakthrough in oncofertility, though researchers caution results remain uncertain.

Breakthrough Bone Marrow Transplant Shows 88% Cure Rate for Sickle Cell Disease

3 months ago

• A novel reduced-intensity haploidentical bone marrow transplant procedure developed at Johns Hopkins demonstrates a 95% two-year survival rate and 88% cure rate for sickle cell disease patients. • The treatment accepts half-matched donors and costs significantly less than gene therapy at $467,747 versus $2-3 million, while requiring shorter hospital stays and fewer blood transfusions. • Clinical trial results from 42 patients show the procedure is safe and effective, with minimal serious side effects, making it a viable alternative to recently approved gene therapy treatments.

Datopotamab Deruxtecan's Role in HR+/HER2- Breast Cancer Treatment: Sequencing and Safety Considerations

4 months ago

• Datopotamab deruxtecan (Dato-DXd) is poised to become a standard treatment for metastatic hormone receptor-positive, HER2-negative breast cancer after prior systemic therapy. • Optimal sequencing of antibody-drug conjugates (ADCs) like Dato-DXd, sacituzumab govitecan, and trastuzumab deruxtecan remains uncertain, especially considering overlapping toxicities. • Real-world toxicity profiles suggest sacituzumab govitecan may cause more cytopenias and diarrhea, while Dato-DXd and trastuzumab deruxtecan are linked to interstitial lung disease. • The lack of overall survival difference in the TROPION-Breast01 trial raises questions about efficacy issues or the impact of crossover between treatment arms.

Novel Metabolic Signatures Show Promise for Earlier Pancreatic Cancer Detection and Prognosis

5 months ago

• Researchers have identified distinct metabolic signatures in pancreatic cancer patients using quantitative mass spectrometry, potentially offering new pathways for earlier diagnosis and survival prediction. • A metabolomic study revealed specific metabolite ratios as significant predictors of survival outcomes in pancreatic ductal adenocarcinoma (PDAC) patients, with some markers showing perfect accuracy in predicting survival beyond 36 months. • A separate study combining microbial-related metabolites with tumor marker CA19-9 achieved an impressive 0.86 AUC for predicting 2-year pancreatic cancer risk, surpassing traditional diagnostic methods.

Citius Pharma Reports Fiscal Year 2024 Results, Highlights LYMPHIR Approval and Mino-Lok Progress

5 months ago

• Citius Pharmaceuticals (Citius Pharma) achieved FDA approval for LYMPHIR for relapsed or refractory cutaneous T-cell lymphoma (CTCL). • The company's Phase 3 trial of Mino-Lok met primary and secondary endpoints, showing statistically significant improvement in catheter failure time. • Citius Pharma is preparing for the commercial launch of LYMPHIR in the first half of 2025 and plans to engage with the FDA on Mino-Lok and Halo-Lido. • The company reported a net loss of $39.4 million for fiscal year 2024, with R&D expenses decreasing and G&A expenses increasing.

Genprex's Reqorsa Advances in Phase 2 Trial for Small Cell Lung Cancer

5 months ago

• Genprex's Acclaim-3 trial, evaluating Reqorsa with Tecentriq for extensive stage small cell lung cancer (ES-SCLC), has advanced to Phase 2. • The Phase 1 dose escalation showed a favorable safety profile, leading to the selection of 0.12 mg/kg as the Recommended Phase 2 Dose (RP2D). • The Phase 2 expansion will enroll approximately 50 patients to determine the 18-week progression-free survival rate. • Reqorsa, a gene therapy expressing the TUSC2 protein, has received Fast Track and Orphan Drug Designations from the FDA for SCLC treatment.

Atezolizumab Fails to Improve Event-Free Survival in Triple-Negative Breast Cancer

5 months ago

• A phase 3 trial showed that adding perioperative atezolizumab to chemotherapy did not significantly improve event-free survival (EFS) in patients with triple-negative breast cancer (TNBC). • The NSABP B-59/GBG-96-GeparDouze trial revealed a non-statistically significant difference in 4-year EFS rates between the atezolizumab/chemotherapy and placebo/chemotherapy groups. • While the addition of atezolizumab boosted pathological complete response rates, it did not translate to an overall survival benefit at 4 years. • Researchers suggest further translational studies to identify TNBC patient subsets that may benefit from checkpoint inhibitors in neoadjuvant/adjuvant therapy.

FDA Approves Steqeyma and Yesintek: Expanding Biosimilar Options for Stelara

6 months ago

• The FDA has approved Steqeyma (ustekinumab-stba) and Yesintek (ustekinumab-kfce) as biosimilars to Stelara, offering new options for patients with autoimmune and inflammatory conditions. • Steqeyma and Yesintek are approved for plaque psoriasis, psoriatic arthritis, Crohn's disease, and ulcerative colitis, mirroring Stelara's indications. • These approvals are expected to increase market competition and reduce treatment costs, with launches anticipated in early 2025 following licensing agreements.

RenovoRx Advances TIGeR-PaC Trial for Pancreatic Cancer with Novel TAMP Therapy

6 months ago

• RenovoRx's TIGeR-PaC Phase III trial evaluates TAMP therapy for locally advanced pancreatic cancer, aiming to improve survival and reduce side effects. • SCRI Oncology Partners has initiated patient enrollment, expanding the trial's reach across a network of over 250 locations in the U.S. • Pharmacokinetic data presented at ASCO GI 2025 supports TAMP's potential to enhance local drug potency while minimizing systemic exposure to gemcitabine. • RenovoRx anticipates completing patient enrollment and the second interim analysis for the TIGeR-PaC trial by the first half of 2025.

Novel Blood Test Enhances Early Detection of Pancreatic Cancer

8 months ago

• A new blood test combining CA199.STRA and CA19-9 biomarkers significantly improves the detection rate of early-stage pancreatic cancer compared to the standard CA19-9 test alone. • The combination test demonstrated a 71% accuracy in identifying pancreatic cancer samples in the lab, a 27% improvement over the 44% accuracy of the CA19-9 test. • Incorporating a protein biomarker called LRG1 alongside CA199.STRA and CA19-9 further enhances the specificity of the test, reducing false positive results. • This multi-biomarker approach underscores the importance of combining different validated biomarkers for more effective and accurate pancreatic cancer detection.

Novel Strategies Emerge to Transform 'Cold' Tumors into Immunotherapy-Responsive 'Hot' Tumors

8 months ago

• Only 20% of cancers currently respond well to immunotherapy, with "hot" tumors like melanoma and lung cancer showing better outcomes than "cold" tumors such as pancreatic and brain cancers. • Innovative approaches including bispecific antibodies, engineered T-cells, and combination therapies are showing promise in converting immunologically "cold" tumors into "hot" ones, potentially expanding immunotherapy's reach. • Recent breakthroughs include tarlatamab's approval for small cell lung cancer with 50% response rates in some groups, and promising results from neo-antigen vaccines in pancreatic cancer treatment.

Text Messaging Shows Promise for Lung Cancer Screening Eligibility Assessment, But Equity Gaps Persist

9 months ago

• A new study reveals 90% of respondents use text messaging regularly, with 83% willing to report smoking status via text for lung cancer screening eligibility assessment. • Middle-aged, college-educated, and higher-income individuals showed greater willingness to participate in text-based screening, highlighting potential disparities in reach. • Researchers recommend a multimodal approach to ensure equitable identification of lung cancer screening candidates across all sociodemographic groups.

Mediar Therapeutics Advances Fibrosis Treatment with Phase 1 Trial of MTX-463

a year ago

Mediar Therapeutics has initiated a Phase 1 clinical trial for MTX-463, a first-in-class antibody targeting WISP-1-mediated fibrotic signaling, following FDA clearance. The trial aims to assess the safety, tolerability, and pharmacokinetics of MTX-463 in healthy participants. Additionally, Mediar has formed a Clinical Advisory Board of global fibrosis experts to guide the development of its fibrosis treatment portfolio, including a second antibody program, MTX-474, expected to enter Phase 1 trials in Q3 2024.

Safety and Efficacy of AADC Gene Therapy for Moderately Advanced Parkinson Disease: Three-Year Outcomes

3 years ago

The PD-1101 trial demonstrates the safety and potential efficacy of VY-AADC01, an AADC gene therapy, in patients with moderately advanced Parkinson disease, showing stable or improved motor function and quality of life over three years.