Articles related to GENE THERAPY

PIK3CA is the most mutated oncogene across many of the common solid tumor types. Consequently, targeting PIK3CA has been of major clinical interest. Initial efforts at have not been promising, however. The limiting factors have primarily been lack of isoform specificity of inhibitors and dose-limiting toxicities. This study evaluates copanlisib, an α and δ isoform–specific phosphoinositide 3-kinase (PI3K) inhibitor, in patients with PIK3CA mutations.

A systematic review identified 204 eligible publications from 2000 through 2017, with much of the evidence consisting of systematic reviews of observational data, consensus guidelines, case series, and case reports. Due to the paucity of high-quality evidence on management of immune-related adverse events, recommendations are based on expert consensus.

The U.S. Food and Drug Administration (FDA) held a two-day public meeting to discuss managing the potential side effects of gene therapies following the death of a boy with a rare neuromuscular disease. High doses of gene therapies are known to cause liver damage, and recent studies have also found a potential long-term cancer risk in mice and dogs treated with gene therapies.

Messenger RNA (mRNA) is in the spotlight right now as the basis of the Moderna and Pfizer/BioNTech vaccines for COVID-19. Thanks to this newfound prominence, its profile has been raised as a powerful tool in cancer research as well. As a courier of genetic information and protein assembler, mRNA can tell scientists which genes are active within a cell and produce the proteins that alert the immune system to the presence of cancer.

Letermovir is a recently approved antiviral shown to decrease clinically significant cytomegalovirus (CMV) infection after hematopoietic cell transplantation (HCT) in a phase 3, randomized, placebo-controlled trial. Appearing in Blood, the authors make two key points: 1) Letermovir prophylaxis may be associated with decreased polyfunctional CMV-specific T-cell immunity after HCT, and 2) COMPASS is an analytical tool that can effectively measure polyfunctional CMV-specific T-cell immune responses.

The FDA announced a trial hold after two participants with sickle cell disease (SCD) in the LentiGlobin gene therapy trials developed AML and MDS. According to bluebird bio, the manufacturer of LentiGlobin products, the company was unable to find any cancer cells in the patient’s bone marrow and has asked the FDA for permission to resume clinical trials.