Positive News: A cure for two devastating illnesses?
Updated: Aug 20, 2022
by Vaibhav M. Varkhedkar
During the last couple of months, there has been news that delivers new hope and possibly cures for two fatal conditions:
1) Jemperli clinical trial results: Jemperli (dostarlimab), an anti-PD-1 monoclonal antibody, was reported to have a 100% clinical complete response rate among patients with mismatch repair-deficient locally advanced rectal cancer at the American Society of Clinical Oncology 2022 annual meeting. Colorectal Cancer (CRC) is the fourth most common type of cancer, and the specific condition outlined above affects about 70,000 patients worldwide, 5% of all CRC patients.
2) Gene Therapy: In August 2022, the FDA approved Zynteglo to treat severe beta-thalassemia, a genetic blood disorder, in kids and adults. It's the first gene therapy medication for this condition.
Beta-thalassemia major is a rare hereditary condition in which the body produces faulty hemoglobin, and these patients require constant blood transfusions and iron-chelation therapy, affecting life expectancy. Life expectancy has improved due to newer iron-chelation agents, but the quality of life is poor.
From 1996 - 2005, I was a drug discovery scientist, and I share the positive sentiment as a result of the above miracles, owing to the hard work and dedication of researchers.