University of Cambridge

Gary Gibson hypothesizes that thiamine deficiency alters glucose metabolism in brain cells, leading to Alzheimer’s. His research culminated in the Benfoteam clinical trial evaluating benfotiamine, a thiamine precursor, for slowing Alzheimer’s progression. The trial aims to determine the highest safe dose and its efficacy in improving cognition and global function.

Stem cell-based treatments show promise for conditions like cancer, diabetes, epilepsy, heart failure, eye diseases, and Parkinson’s, with brain-related conditions being more responsive. Clinical trials for multiple sclerosis also yielded positive results. However, caution is advised against fraudulent stem cell treatments.

Gene therapy lenadogene nolparvovec for LHON due to MT-ND4 gene variant showed sustained BCVA improvement in both eyes up to 5 years post-treatment, with 66.1% of patients improving at least -0.3 logMAR (+3 lines) in at least one eye. Vision benefits persisted over 3 years, with no permanent complications and manageable inflammation. The therapy aims to correct the genetic defect causing LHON, a rare inherited optic nerve disease affecting one in 50,000 people.

Andrew Cassy, diagnosed with Parkinson's in 2010, volunteered for a 2024 trial where human embryonic stem cell-derived neurons were placed in his brain to replace damaged tissue. Over 100 clinical trials explore stem cell potential for diseases like cancer, diabetes, epilepsy, heart failure, and eye diseases. Challenges include defining suitable cells and immunosuppressant use. Stem cell therapies for conditions like Parkinson's and diabetes show promise, with some trials reporting safety and efficacy.

Dr. Gary Gibson hypothesizes that thiamine deficiency alters glucose metabolism in brain cells, leading to Alzheimer's. His research led to a nationwide clinical trial evaluating benfotiamine, an oral drug boosting thiamine levels, as a potential treatment.

Top 2024 stories highlight gene therapy for deafness, deep brain stimulation for epilepsy, benralizumab for asthma/COPD, new schizophrenia drug, mRNA-4359 cancer vaccine, pig immune system insights, type-1 diabetes link to bacterial proteins, NK cell cancer therapy, Alzheimer's research, liver cholesterol regulation, lemur rhythm insights, fish resilience research, zebrafish kidney disease model, and NAMs for chemical testing.

African populations are underrepresented in genomic research, hindering precision medicine development. Despite challenges like funding and infrastructure, progress is being made in countries like South Africa and Uganda. Initiatives like H3Africa aim to improve genomic diversity and capacity in Africa, but significant barriers remain, including brain drain and lack of government support. Efforts to build infrastructure and knowledge are ongoing, with the hope of expanding access to precision medicine across the continent.

MIT and Cambridge researchers unveiled AI-powered ChemAI to accelerate cancer drug discovery, using machine learning on millions of data points to predict drug interactions, potentially shortening development timelines and revolutionizing cancer therapy.

Prostate cancer clinical trials need innovative approaches to better capture patient experience and improve treatment options, focusing on progression-free and disease-free survival as primary endpoints.

Molecular imaging using hyperpolarized carbon-13 MRI can differentiate ovarian cancer types and predict chemotherapy response within 48 hours, aiding personalized treatment selection.