Bone marrow mesenchymal stem cell-derived extracellular vesicle infusion for amyotrophic lateral sclerosis.

Background: In this pilot safety study, we hypothesized that a human bone marrow stem cell-derived extracellular vesicle (hBM-MSC EV) investigational product (IP) would be safe and exhibit potential efficacy in amyotrophic lateral sclerosis (ALS) patients.Methods: Ten ALS patients received two 10-ml intravenous infusions of the IP given 1 month apart and evaluated over 3 months.Results: There were no serious adverse events or adverse events related to the IP and 30% of subjects' ALS functional rating scale-revised (ALSFRS-R) scores did not decline.Conclusion: HBM-MSC EVs appear safe in ALS patients. This early investigation suggests a controlled study of EVs for the treatment of ALS is warranted.

Amyotrophic lateral sclerosis (ALS) is a nervous system disease that affects the brain and spinal cord, causing the loss of muscle control. Currently, there is no cure for ALS and the disease gets worse over time. A potential new treatment is being investigated using mesenchymal stem cell extracellular vesicles (MSC EVs). MSC EVs are small structures that contain useful molecules and proteins that can be transported to cells affected by the disease, helping to reduce inflammation and encouraging repair. This 3-month study looked at the safety of human bone marrow MSC-EVs (hBM-MSC EVs) given as treatment to ten ALS patients, as well as how well it worked at delaying worsening of the disease. They found that there were no serious side effects caused by the treatment and that hBM-MSC EVs may have the potential for delaying the progression of ALS. This indicates that more, larger studies need to be carried out to find out treatment specifics, such as dose (how much of the treatment to give) and frequency (how often to give the treatment), and how they could be related to patient outcomes.

Pertussis presenting as hoarseness in an adult.

Pertussis is a rare condition in adults. This report describes a 65-year-old man presenting to the otolaryngology service with hoarseness secondary to pertussis. He had a history of worsening cough and hoarseness occurring over several weeks. Laryngoscopy showed significant erythema at the medial edges of the vocal folds. Stroboscopy demonstrated bilateral decreased amplitude and decreased mucosal wave of the vocal folds. Polymerase chain reaction testing of the sputum revealed Bordetella pertussis. The patient was started on azithromycin, and he reported gradual resolution of the chest and laryngeal symptoms over the next few months. Pertussis, or whooping cough, is not common in the United States because of childhood vaccination protocols. Nevertheless, otolaryngologists need to be aware of this condition as a possible cause of hoarseness.