Plano Cancer Institute

Classic case

A Woman with Delusional Thinking and Paresthesia | NEJM Resident 360

New standard in the low risk group...

Avelumab plus Axitinib versus Sunitinib for Advanced Renal-Cell Carcinoma

We need to do better...

Sepsis Recovery: Recognition and Management of Long-term Sequelae This narrative review summarizes the epidemiology and pathophysiology of sepsis, management of post-sepsis sequelae in the months following hospital discharge, and directions for future research to reduce long-term sequelae and improve patient recovery.

Impressive...

Darolutamide in Nonmetastatic, Castration-Resistant Prostate Cancer

Immune dependent tumor...

On immunotherapy trial, long-term survivors of deadly skin cancer point to a hopeful future More than two-thirds of people with advanced Merkel cell carcinoma are on track to survive at least two years after starting an immunotherapy drug on a landmark trial.

No benefit in the absence of metastases...

New hope

Gene Therapy for Beta-Hemoglobinopathies: Milestones, New Therapies and Challenges

Inherited monogenic disorders such as beta-hemoglobinopathies (BH) are fitting candidates for treatment via gene therapy by gene transfer or gene editing. The reported safety and efficacy of lentiviral vectors in preclinical studies have led to the development of several clinical trials for the addition of a functional beta-globin gene. Across trials, dozens of transfusion-dependent patients with sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT) have been treated via gene therapy and have achieved reduced transfusion requirements. While overall results are encouraging, the outcomes appear to be strongly influenced by the level of lentiviral integration in transduced cells after engraftment, as well as the underlying genotype resulting in thalassemia. In addition, the method of procurement of hematopoietic stem cells can affect their quality and thus the outcome of gene therapy both in SCD and TDT. This suggests that new studies aimed at maximizing the number of corrected cells with long-term self-renewal potential are crucial to ensure successful treatment for every patient. Recent advancements in gene transfer and bone marrow transplantation have improved the success of this approach, and the results obtained by using these strategies demonstrated significant improvement of gene transfer outcome in patients. The advent of new gene-editing technologies has suggested additional therapeutic options. These are primarily focused on correcting the defective beta-globin gene or editing the expression of genes or genomic segments that regulate fetal hemoglobin synthesis.
Read more: https://link.springer.com/article/10.1007%2Fs40291-019-00383-4