Articles related to MEDICAL BREAKTHROUGHS

Discover how a groundbreaking trial using a new CRISPR gene-editing technique has successfully treated a patient with T-cell leukemia, a form of the disease resistant to conventional treatments. Learn how this innovative approach could revolutionize the treatment of various diseases affecting the blood, bone marrow, and immune systems.

Pioneering studies with clemastine, an over-the-counter antihistamine, are exhibiting groundbreaking findings in the repair of myelin, a protective coating on nerve fibers damaged in multiple sclerosis, based on ReBUILD clinical trial’s MRI data.

The invention of plant-based oral insulin, which provides a more secure and efficient substitute for insulin injections, marks a significant advancement in the management of diabetes. Investigate this ground-breaking strategy that could revolutionize accessibility to diabetes treatment worldwide.

Johns Hopkins researchers develop breakthrough urine test for liver cancer, increasing early detection rates by supplementing traditional AFP testing. A groundbreaking screening test for hepatocellular carcinoma (HCC), developed by Amy Kim, a hepatologist at Johns Hopkins, and her team, has demonstrated the potential to detect liver cancer at an earlier stage than the current standard-of-care. This novel diagnostic approach seeks to identify a subset of genetic biomarkers in urine associated with HCC, thereby supplementing the traditional combination of ultrasound and alpha-fetoprotein (AFP) blood testing. AFP tests have shown variable effectiveness with detection rates ranging between 40% and 90%, and a substantial number of liver cancer patients exhibit low AFP levels or misleadingly high levels due to liver disease, reducing the reliability of AFP as a singular biomarker. The study, conducted in collaboration with the Baruch S. Blumberg Institute and published in the British Journal of Cancer in 2022, analyzed samples from over 600 patients at high risk for liver disease across five medical centers. Kim’s team demonstrated that the combined use of urine circulating tumor DNA (ctDNA) testing and AFP increased detection sensitivity from 40% to 77% in early-stage liver cancer, and from 62% to 92% in the very early […]

More trials in larger groups of patients will be required to determine whether this drug, BIIB080 (/IONIS-MAPTRx), an antisense oligonucleotide (used to stop RNA from producing a protein), leads to clinical benefit, but the phase 1 results published in Nature Medicine – with results from 46 patients – are the first indication that this method has a biological effect. BIIB080 ‘silences’ the tau protein-coding gene, known as the microtubule-associated protein tau (MAPT) gene. This inhibits the gene from being dosed and reversibly translated into protein. It will also reduce the creation of that protein, changing the course of the condition.

Bispecific antibodies are molecules that are designed to engage or bind to two distinct targets, bringing two distinct cells together. The majority of those that have received regulatory approval are T-cell-engaging bispecific antibodies. Blinatumomab (Blincyto) for ALL, teclistamab (Tecvayli) for multiple myeloma, and mosunetuzumab-axgb (Lunsumio) for follicular lymphoma are some examples of FDA-approved treatments. Glofitamab, another bispecific antibody, is currently under FDA Priority Review for the treatment of R/R DLBCL. Another type of immunotherapy is chimeric antigen receptor (CAR) T-cell therapy, but bispecific antibodies provide the same spectrum of side effects with much less toxicity and are showing promise in solid tumors as well.