Articles related to MEDICAL BREAKTHROUGHS

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.

Swedish researchers have learned how to “grow” electrically conductive regions in tissue. The process involves injecting an enzyme-rich hydrogel into target tissues. The enzymes interact with molecules in the tissue and change the structure of the gel to make it conductive. The technique has potential application in areas as diverse as cardiac pacing to brain-computer interface development – all without the implanted electrodes that are used today and the physical limits they impose.

In this video, Dr. Robert Vonderheide, the Director of the Abramson Cancer Center at Penn Medicine, discusses cancer interception, an approach to cancer treatment that involves identifying and treating pre-cancerous cells before they develop into tumors. Dr. Vonderheide explains that cancer interception involves using genomic sequencing to identify cells that are at high risk of developing into cancer and then using drugs or other treatments to kill these cells before they become cancerous. He notes that cancer interception has the potential to be a highly effective form of cancer treatment, but that it requires a collaborative approach between researchers, clinicians, and patients. Dr. Vonderheide also discusses the role of immunotherapy in cancer interception and the challenges associated with developing effective interception strategies for different types of cancer.

A look into the results of a few patients that took part in a phase 1/2 trial at the Icahn School of Medicine at Mount Sinai in New York City: a woman who had breast cancer for 23 years and gone through 12 lines of therapy and a man with non-Hodgkin lymphoma (NHL) since 2017. Both showed complete response to the novel vaccine.

The development of effective new therapies has been challenging. This discovery of calcium signaling as a central abnormality in schizophrenia neurons is an exciting new finding, since available antipsychotics treat only positive symptoms and are not effective for all patients.