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Clinical Trial News

UCLA Gene Therapy Achieves 100% Cure Rate for Bubble Baby Disease in Clinical Trials

FDA Approval
  • UCLA researchers led by Dr. Donald Kohn have successfully cured all 18 children with ADA-deficient SCID using a novel stem cell gene therapy approach over two clinical trials since 2009.
  • The breakthrough treatment uses patients' own genetically modified blood stem cells to restore immune function, eliminating the need for lifelong enzyme injections or risky bone marrow transplants.
  • All treated patients, ranging from 3 months to 4 years old, developed fully functioning immune systems without side effects and can now live normal lives.
  • The team is seeking FDA approval for the therapy and plans to extend the approach to treat sickle cell disease in clinical trials beginning in 2015.

Naltrexone Shows Promise in Reducing Fatigue in Primary Sjögren’s Syndrome

  • A prospective, open-label study suggests low-dose naltrexone (LDN) may alleviate fatigue in patients with primary Sjögren’s syndrome (pSS).
  • The study observed a statistically significant reduction in fatigue scores, as measured by the Fatigue Severity Scale (FSS), following LDN treatment.
  • LDN was well-tolerated, with no serious adverse events reported, indicating a favorable safety profile for pSS patients.
  • These findings support further investigation of LDN as a potential therapeutic option for managing fatigue, a common and debilitating symptom of pSS.

Landiolol Effectively Manages Post-Operative Supraventricular Tachycardia After Lung Resection

  • Landiolol, an ultra-short-acting β1-blocker, significantly reduced heart rate in patients experiencing post-operative supraventricular tachycardia after pulmonary resection.
  • The study demonstrated that continuous intravenous infusion of landiolol led to a reduction in heart rate from 135±24 bpm to 85±19 bpm (P<0.0001).
  • Conversion to normal sinus rhythm was observed in 56% of patients treated with landiolol, highlighting its potential in managing post-operative atrial fibrillation or flutter.
  • Landiolol was found to be safe and effective, with no detectable side effects on the circulatory or respiratory systems in the studied patient population.

Psychedelics Show Promise in Treating Anxiety and Depression in Cancer Patients

  • A review of five randomized, double-blind, placebo-controlled trials suggests psychedelics may offer a novel approach for treating anxiety and depression in cancer patients.
  • Studies showed significant reductions in anxiety and depression scores using various scales, with benefits potentially lasting for 6 to 12 months after only a few sessions.
  • The unique mechanism of action of psychedelics may provide relief for patients who do not respond well to traditional antidepressants, addressing a significant unmet need.
  • Despite promising results, the studies were small and had methodological limitations, including observed increases in blood pressure and heart rate.

Abemaciclib Shows Promise in ER+/HER2- Advanced Breast Cancer Treatment

• Abemaciclib, a CDK 4/6 inhibitor, presents a valuable therapeutic avenue for patients with ER+/HER2- advanced metastatic breast cancer, offering a continuous dosing schedule unlike other CDK 4/6 inhibitors. • Clinical trials demonstrate that abemaciclib provides similar clinical benefits to palbociclib and ribociclib in first- and second-line treatments, but with a distinct toxicity profile. • The ongoing MONARCH-E trial exploring abemaciclib in the adjuvant setting for high-risk, node-positive patients could significantly broaden its impact in early breast cancer treatment. • Biomarker research from studies like neoMONARCH may help identify tumors most responsive to abemaciclib, optimizing its use in personalized treatment strategies.

Ropeginterferon Alfa-2b Evaluated as Second-Line Therapy for High-Risk Essential Thrombocythemia in SURPASS-ET Trial

  • The SURPASS-ET trial is evaluating ropeginterferon alfa-2b as a second-line treatment for high-risk essential thrombocythemia (ET).
  • This Phase III trial compares ropeginterferon alfa-2b to anagrelide in patients resistant to or intolerant of hydroxyurea.
  • Ropeginterferon alfa-2b, a long-acting interferon, has shown a good safety profile in polycythemia vera and may offer a new option for ET patients.
  • The study assesses the safety, efficacy, tolerability, and pharmacokinetics of ropeginterferon alfa-2b, addressing an unmet need for effective ET therapies.

Ropeginterferon Alfa-2b (P1101) vs. Anagrelide in Essential Thrombocythemia Patients With Hydroxyurea Resistance or Intolerance

NCT04285086Active, Not RecruitingPhase 3
PharmaEssentia
Posted 8/25/2020

Topotecan Shows Significant Activity in Second-Line Treatment of Small-Cell Lung Cancer

  • Topotecan demonstrates notable efficacy in treating small-cell lung cancer (SCLC) patients who have previously undergone chemotherapy.
  • The study reveals a higher response rate among patients sensitive to prior chemotherapy, with an overall response rate of 37.8%.
  • Hematologic toxicity, particularly neutropenia, was the primary adverse effect, but it was generally manageable and short-lived.
  • The findings suggest that topotecan could be a valuable component in future combination chemotherapy regimens for SCLC.

Pre-Transplant MRD Positivity Linked to Poorer Survival in Acute Leukemia Patients

  • A study of 114 acute leukemia patients undergoing allogeneic hematopoietic stem cell transplantation (allo-HSCT) reveals that pre-transplant minimal residual disease (MRD) positivity is associated with worse outcomes.
  • Patients with MRD+ status before transplantation had significantly lower one-year overall survival (OS) and progression-free survival (PFS) rates compared to MRD- patients.
  • Multivariate analysis identified MRD positivity as an independent prognostic factor for overall survival, highlighting the need for improved pre-transplant MRD management strategies.
  • The research suggests focusing on more effective chemo regimens with targeted agents to achieve MRD- status before transplantation and better management of GvHD prophylaxis.

PLEASE Trial Assesses Prolonged Antibiotic Treatment for Persistent Lyme Symptoms

  • The Persistent Lyme Empiric Antibiotic Study Europe (PLEASE) investigates optimal treatment for borreliosis-attributed persistent symptoms.
  • The study is a randomized, double-blind, placebo-controlled trial comparing short-term versus long-term antibiotic regimens.
  • Researchers aim to determine if prolonged antibiotic treatment improves patient outcomes compared to standard short-term therapy.
  • The trial includes patients with persistent pain, fatigue, and cognitive issues following Lyme disease, evaluating physical and mental health.

Phase III LEAP-012 Study Shows Promising Results for Intermediate-Stage HCC Treatment

The LEAP-012 phase III trial demonstrates that combining lenvatinib, pembrolizumab, and TACE significantly improves progression-free survival in patients with intermediate-stage hepatocellular carcinoma, compared to placebo plus TACE.

Safety and Efficacy of Lenvatinib (E7080/MK-7902) With Pembrolizumab (MK-3475) in Combination With Transarterial Chemoembolization (TACE) in Participants With Incurable/Non-metastatic Hepatocellular Carcinoma (MK-7902-012/E7080-G000-318/LEAP-012)

NCT04246177Active, Not RecruitingPhase 3
Merck Sharp & Dohme LLC
Posted 5/22/2020

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