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Novel Gene Therapy Treats T Cell Leukemia

Scientists at the University College London (UCL) have developed a novel therapy that helps treat patients with T cell acute lymphoblastic leukemia (T-ALL). This form of therapy used genome editing tools to modify immune cells and boost immune system response. T-ALL is a rare and aggressive blood cancer that affects specialized immune cells, known as T cells. This immune subset is responsible for identifying and targeting foreign pathogens. Unfortunately, in T-ALL, genetic mutations prevent T cells from maturing and properly functioning.

The world’s first gene therapy (BE-CAR7) uses base-editing, which can specifically change a single base in a cell’s DNA. BE-CAR7 was the first therapy to treat T-ALL in both children and adults. In 2022, a 13-year old girl was given BE-CAR7 followed by another eight children and two adults undergoing the same treatment. The following results from these patients were published in the New England Journal of Medicine (NEJM), by Dr. Waseem Qasim and others. Qasim is a Professor of Cell and Gene Therapy in the Department of Infection, Immunity, and Inflammation at the UCL. His work focuses on pediatric oncology with a focus in gene therapy. Qasim’s work has long involved treatment of T-ALL and improving therapies for children with leukemia.

Qasim and his team reported that 82% of patients receiving BE-CAR7 achieved remission, which allowed them to undergo stem cell transplant without disease. Treatment was accompanied by tolerable side effects, including low blood counts, cytokine release syndrome, and rashes. Additionally, 64% of patients remained disease-free even after three years. These remarkable results indicate the strong impact gene therapy has on T-ALL.

Single-dose oral treatment for gonorrhea effectively combats drug-resistant infections, clinical trial finds

A single-dose oral medication called zoliflodacin shows promise as a new treatment for antibiotic-resistant gonorrhea, according to a Phase III trial published in The Lancet.

The study found that one dose of zoliflodacin was as effective as the current standard treatment, which combines two antibiotics: an injection of ceftriaxone followed by an oral dose of azithromycin.

Gonorrhea is one of the most common sexually transmitted infections, affecting over 82 million people globally each year. However, it is increasingly difficult to treat as the bacteria that cause infection develop resistance to current antibiotics. This new medication has the potential to help slow the spread of antibiotic-resistant bacteria and make gonorrhea treatment more accessible worldwide.

Ultra‐refractory epilepsy: The newly described entity

“Ultra-refractory epilepsy (URE) is a newly described form of extremely difficult-to-treat epilepsy. In our study of 62 patients, all had already failed at least six different treatment attempts, including medications, brain surgery, or nerve stimulation. Most patients continued to have frequent seizures despite all available therapies, and only a small minority achieved long-term seizure freedom. These findings show that URE is a severe condition with a major unmet medical need, highlighting the importance of developing new treatment strategies.”

Read this original article from Epilepsia Open Journal at doi.org/10.1002/epi4.70196.

Objective Drug-resistant epilepsy (DRE) is defined as the failure of two antiseizure medications (ASMs) to achieve complete seizure control, affecting approximately 30% of epilepsy patients. In some cases, additional ASMs and surgical approaches are also unsuccessful. Ultra-Refractory Epilepsy (URE) is a newly described entity, characterized by the failure of six distinct epilepsy treatment strategies, including ASMs, surgical resection, and neurostimulation techniques. This study aimed to analyze demographic and clinical data of URE patients managed at the Brno Epilepsy Center, a member of the European Reference Network (ERN)—EpiCARE.

Revitalizing liver function in mice with liver failure through transplantation of 3D-bioprinted liver with expanded primary hepatocytes

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3D-bioprinting livers based on expandable primary hepatocytes and liver-specific bioink could save mouse liver failure.

The complexity of the relationship between thyroid disease and body weight

Hypothyroidism and hyperthyroidism are associated with gain and loss of body weight, respectively. This Review discusses the epidemiological evidence for weight changes in thyroid dysfunction, the role of thyroid hormone in weight regulation, the effect of treatment and the implications for population health.

Role of ubiquilin-2 liquid droplets in α-synuclein aggregation

Parkinson’s disease (PD) is an age-related, progressive neurodegenerative disorder. The hallmark of PD pathogenesis is the Lewy bodies (LBs) that accumulate in neurons in the substantia nigra region of the brain, damaging these neurons and leading to the motor symptoms of the disease. α-synuclein (α-syn), a misfolded protein, aggregates and forms fibrils, which leads to the formation of LBs. The exact molecular mechanism behind this aggregation process is yet to be uncovered. With an increasing number of elderly patients suffering from Parkinson’s and other neurodegenerative diseases worldwide, it is important to understand the aggregation process, find potential therapeutic targets to mitigate or inhibit the aggregation, and slow down the disease progression.

Liquid-liquid phase separation (LLPS), a process where a uniform mixture spontaneously divides into two liquid phases with differing component concentrations, is often considered the reason behind α-syn aggregation. Even though LLPS of α-syn was previously reported, the question remains: are they involved in catalyzing the early stage of aggregation? Ubiquilin-2 (UBQLN2) protein, mainly involved in maintaining protein homeostasis, also undergoes LLPS under certain physiological conditions. Interestingly, it is known to be associated with several neurodegenerative diseases.

Are liquid droplets formed by UBQLN2 catalyzing the α-syn protein aggregation? A team of researchers decided to unravel the involvement of UBQLN2 in α-syn aggregation and fibril formation. “By uncovering the mechanisms that trigger the aggregation process, we hope to find new ways to prevent it and ultimately contribute to the development of disease-modifying treatments,” mentioned the senior author of the study. The study was published in The EMBO Journal.

Identification of molecular markers and exploration of the oncogenic role of exomeres in hepatocellular carcinoma

This study isolates extracellular vesicles and particles (EVPs) from hepatocellular carcinoma (HCC) cells. Biophysical and proteomic analyses demonstrate that sEVs and exomeres are distinctive entities. GALNS and MAN2B1 are identified as potential EM markers. HCC-derived EMs promote oncogenesis via several mechanisms, including PI3K/AKT/mTOR activation, cell cycle progression, and lipidomic dysregulation.

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