Lifeboat Foundation

Memorial Sloan Kettering Cancer Center made waves earlier this year when it announced the completion of a clinical trial that saw colorectal cancer in 18 patients disappear.

The patients received a drug that helped their immune system target and attack cancer cells, driving their cancer into remission and rendering it undetectable within six months.

Now, Lehigh Valley Health Network has joined a select group of networks and hospitals participating in the expanded clinical trial of the drug.

Macquarie University neuroscientists have developed a single-dose genetic medicine that has been proven to halt the progression of both motor neuron disease (MND) and frontotemporal dementia (FTD) in mice—and may even offer the potential to reverse some of the effects of the fatal diseases.

It may also hold opportunities for treating more common forms of dementia, such as Alzheimer’s disease, which is the second most common cause of death in Australia after heart disease.

The new treatment, dubbed CTx1000, targets pathological build-ups of the protein TDP-43 in cells in the brain and spinal cord.

Neurologic immune-related adverse events (nirAEs) following immune checkpoint inhibitor therapy for cancer are frequent and varied; a recent study identified risk factors for death after nirAEs.

Recent cohort studies have demonstrated that neurologic immune-related adverse events (nirAEs) following immune checkpoint inhibitor (ICI) therapy for cancer are frequent, varied, and associated with higher overall survival (NEJM JW Neurol Sep 29 2023 and Neurology 2023; 101:e2472). Researchers conducted a retrospective cohort study of consecutive patients referred to a tertiary center during a 5-year period to characterize the clinical features of nirAEs and identify predictors of ICI response and survival.

The researchers identified 64 patients with confirmed nirAEs, 81% involving the central nervous system (CNS). The vast majority of CNS nirAE patients had encephalopathy, of which 73% were neither seropositive for well-characterized neural autoantibodies, nor had a distinctive encephalitis syndrome, nor had evidence of CNS inflammatory changes. The most common peripheral nervous system (PNS) syndrome was myasthenia and myositis (with or without myocarditis) overlap syndrome. Only 17% of PNS nirAE patients were seropositive. Steroids were given to 91% of nirAE patients after a median of 90 days of symptoms, and 48% received additional immunotherapy. At 1-month follow-up, 72% of nirAE patients showed improvement, 9% had worsened, and 17% had died. Among the 53 patients who survived the first month, median follow-up was 6 months; during follow-up, 30% died, most commonly of cancer progression or cancer-related complications. Death was associated with lung cancer (hazard ratio, 2.

Continue reading “Risk Factors for Death After Neurologic Immune-Related Adverse Events” »

Talk by Ken Dickerson, CRISPR Group Leader, CRISPR QC, at the BACE Technology Showcase event in August 2022.

Chronic thromboembolic pulmonary hypertension (CTEPH) is a rare, potentially fatal condition caused when bloodclots in the lungs block blood vessels to the heart. Yale PCCSM’s Phillip Joseph, MD, and Yale Cardiac Surgery’s Prashanth Vallabhajosyula, MD, share how Yale’s highly specialized #

Clinicians diagnose and treat CTEPH.

Live Science spoke with Šikšnys about what it’s been like to see CRISPR enter clinical use and how he thinks the system might be applied and improved upon in the future.

Editor’s Note: This interview has been condensed and edited for clarity.

Related: Gene therapy: What is it and how does it work?

CHICAGO, Feb 19 (Reuters) — A study that analyzed the genetic code of a quarter of a million U.S. volunteers found more than 275 million entirely new variants that may help explain why some groups are more prone to disease than others, researchers reported on Monday.

The whole genome sequencing data from a wide range of Americans aims to address the historical lack of diversity in existing genomic datasets by focusing on previously under-represented groups. The U.S. National Institutes of Health-funded “All of Us” study turned up 1 billion genetic variants in total.

“Sequencing diverse populations can lead to new drug targets that are relevant to everyone,” said Dr. Josh Denny, a study author and its chief executive. “It can also help uncover disparities that lead to specific treatments for people that are experiencing higher burdens of disease or different disease.”

This is all good but I really like the telomeres results.

Liz Parrish presents the stunning progress of gene therapies and how to collaborate to cure aging in this clip.

Continue reading “Living Longer, Living Better: Gene Therapy’s Stunning Progress For Longevity” »

A new study led by Stanford Medicine scientists demonstrates a simple way of studying organ aging by analyzing distinct proteins, or sets of them, in blood, enabling the prediction of individuals’ risk for diseases.