Medical Bulletin 01/September/2023

Here are the top medical news of the day:

Promising evidence for sickle cell gene therapy found in new study

New research published in the New England Journal of Medicine indicates that stem cell gene therapy may offer a promising, curative treatment for the painful, inherited blood disorder sickle cell disease (SCD). The findings from a new clinical trial add to the body of evidence supporting gene therapy as a treatment for sickle cell disease, which primarily impacts people of color. Until recently, the only treatment options have been intensive bone marrow transplants from siblings or matched donors.

As part of the trial, researchers used CRISPR-Cas9 to edit specific genes in stem cells — the building blocks of blood cells — taken from each patient. The edits increased the cells’ production of fetal hemoglobin (HbF), a protein that can replace unhealthy, sickled hemoglobin in the blood and protect against the complications of sickle cell disease. The patients then received their own edited cells as therapeutic infusions.

Reference: CRISPR-Cas9 Editing of the HBG1 and HGB2 Promoters to Treat Sickle Cell Disease, New England Journal of Medicine, DOI 10.1056/NEJMoa2215643 

Timely and prompt cardiovascular risk factors control preserves brain metabolism

Results from recent study confirm the importance of controlling traditional cardiovascular risk factors, such as hypertension, cholesterol, diabetes, smoking, and a sedentary lifestyle, not only to preserve cardiovascular health but also to prevent Alzheimer’s disease and other dementias.

Published in The Lancet Healthy Longevity, the CNIC study shows that atherosclerosis—the accumulation of fatty deposits in the arteries—and its associated risk factors, in addition to being the main cause of cardiovascular disease, are also implicated in the cerebral alterations typically found in Alzheimer’s disease, the most frequent cause of dementia.

Reference: The Lancet Healthy Longevity, Article Title: Longitudinal interplay between subclinical atherosclerosis, cardiovascular risk factors, and cerebral glucose metabolism in midlife: results from the PESA prospective cohort study (DOI:https://doi.org/10.1016/S2666-7568(23)00134-4)

Experts warn against cannabis use during adolescence, pregnancy, and while driving

Experts recommend avoiding cannabis during adolescence and early adulthood, in people prone to or with mental health disorders, in pregnancy, and before and while driving, based on an in-depth evidence review published by The BMJ. However, they say cannabidiol (one active compound in cannabis) is effective in people with epilepsy, and cannabis-based medicines can help people with multiple sclerosis, chronic pain, inflammatory bowel disease, and in palliative care.

Their recommendations are based on an “umbrella review” of 101 meta-analyses on cannabis and health. Umbrella reviews synthesize previous meta-analyses and provide a high-level summary of evidence on a particular topic.

Reference: Balancing risks and benefits of cannabis use: umbrella review of meta-analyses of randomized controlled trials and observational studies, The BMJ, DOI 10.1136/bmj-2022-072348