UNIVERSITY OF SHEFFIELD

Novel Treatment Regimen Extends Myeloma Remission by Seven Months in UK Clinical Trial

8 days ago

• A phase 3 clinical trial led by the University of Leeds demonstrated that a second stem cell transplant followed by ixazomib maintenance therapy extends myeloma remission by seven months compared to standard care. • The ACCoRd trial treatment protocol combines autologous stem cell transplantation with targeted drugs thalidomide, dexamethasone, and ixazomib, offering patients improved quality of life compared to chemotherapy or long-term steroid use. • Ixazomib works by preventing protein breakdown in myeloma cells, causing toxic buildup while allowing healthy cells to grow, presenting a promising alternative for patients eligible for a second bone marrow transplant.

FDA Clears BlackfinBio's Novel Gene Therapy Trial for Rare Childhood Neurological Disease SPG47

24 days ago

• The FDA has cleared BlackfinBio's IND application to begin a Phase 1/2 clinical trial of BFB-101, a gene therapy designed to treat Hereditary Spastic Paraplegia Type 47 (SPG47), a rare neurological disorder with no existing treatments. • BFB-101 aims to deliver a functional copy of the AP4B1 gene via a single injection into the cerebrospinal fluid, potentially halting or reversing disease progression in affected children who experience progressive lower-limb spasticity and developmental delays. • The trial will be conducted at Boston Children's Hospital with recruitment expected to begin by late 2025, evaluating both safety and efficacy in up to five children with the rare genetic condition that has received orphan drug and rare pediatric disease designations.

J&J Seeks FDA Approval for TAR-200 in BCG-Unresponsive Non-Muscle Invasive Bladder Cancer

4 months ago

• Johnson & Johnson has initiated a New Drug Application (NDA) submission to the FDA for TAR-200. • TAR-200 is intended for patients with Bacillus Calmette-Guérin (BCG)-unresponsive high-risk non-muscle-invasive bladder cancer (HR-NMIBC). • Phase 2b SunRISe-1 study data showed an 83.5% complete response rate with TAR-200 monotherapy. • The FDA is reviewing the application under the Real-Time Oncology Review (RTOR) program to expedite potential approval.

Durvalumab Immunotherapy Shows Promise in Muscle-Invasive Bladder Cancer Treatment

4 months ago

• A Phase 3 clinical trial reveals that durvalumab, combined with chemotherapy and surgery, significantly reduces cancer recurrence in bladder cancer patients. • The study, involving 1,063 patients, found a 32% reduction in disease progression or recurrence with the addition of durvalumab to standard treatment. • Two-year overall survival rates were notably higher in the durvalumab group (82.2%) compared to the chemotherapy and surgery alone group (75.2%). • Experts hope durvalumab will soon become the new standard of care for muscle-invasive bladder cancer, pending regulatory approval.

Stroke Trial Tests Innovative Device to Combat Arm Weakness

4 months ago

A pioneering trial led by the University of Sheffield and Sheffield Teaching Hospitals NHS Foundation Trust is testing a portable, pacemaker-like device aimed at helping stroke survivors regain arm strength. The device, which delivers electrical pulses to damaged brain areas, could significantly improve hand and arm function, offering a new hope for those affected by stroke-induced arm weakness.

Novel Nanoparticle Immunotherapy Shows Promise in Delaying Prostate Cancer Treatment Resistance

10 months ago

• University of Sheffield researchers have developed a new nanoparticle-based immunotherapy that significantly delays resistance to androgen deprivation therapy (ADT) in prostate cancer patients. • The innovative approach targets macrophages around blood vessels in prostate tumors, causing them to release interferon-beta which activates T cells to attack cancer cells. • This breakthrough could potentially extend the effectiveness of hormone therapy for thousands of men, addressing a critical gap as traditional immunotherapies have historically shown limited success in prostate cancer.

Alzheimer's Researchers Urge Trial Participation Following Fiona Phillips' Diagnosis

2 years ago

• Researchers are advocating for increased participation in Alzheimer's drug trials, highlighting potential benefits for participants regardless of treatment efficacy. • Fiona Phillips, diagnosed with early-onset Alzheimer's, is participating in a trial for miridesap, a novel drug targeting SAP to break down amyloid plaques. • Experts emphasize the need for diversified research approaches, increased funding, and improved diagnostic tools to advance Alzheimer's treatment. • Early diagnosis and intervention are crucial for stabilizing the disease and reducing its severity, prompting calls for greater awareness and access to diagnostic resources.

Immunotherapy Significantly Improves Survival Rates for Operable Bladder Cancer Patients

2 years ago

A major clinical trial reveals that adding the immunotherapy drug durvalumab to standard chemotherapy and surgery significantly improves survival rates for patients with operable bladder cancer, marking a breakthrough in treatment.

Low-Dose Interleukin-2 Shows Promise in Slowing Motor Neurone Disease Progression in Landmark MIROCALS Trial

3 years ago

• The MIROCALS clinical trial demonstrated that low-dose interleukin-2 (IL2LD) is safe and reduced the risk of death by over 40% in approximately 80% of motor neurone disease patients with lower levels of a specific biomarker. • The groundbreaking study provides compelling evidence that modifying the immune system could be an effective strategy for altering MND/ALS progression, potentially adding to the disease-modifying effects of riluzole. • While not yet licensed for MND treatment, IL2LD increases regulatory T cells to reduce inflammation, and ILTOO Pharma has secured exclusive licensing rights to pursue regulatory approval for this novel therapeutic approach.