Molecules that curb errant proteins of AL amyloidosis point to new type of therapy
(Scripps Research Institute) Scientists at Scripps Research have identified a group of small molecules that prevent structural changes to proteins that are at the root of AL amyloidosis, a progressive and often fatal disease. The drug mechanism that the team identified is analogous to a different drug that also originated at Scripps Research: Tafamidis, which stabilizes the protein transthyretin to treat the most common systemic amyloid disease that affects the heart or other organs.
Blood Cancer Journal, Published online: 22 May 2020; doi:10.1038/s41408-020-0325-2The impact and importance of achieving a complete haematological response prior to renal transplantation in AL amyloidosis
Blood Cancer Journal, Published online: 11 May 2020; doi:10.1038/s41408-020-0321-6Venetoclax for the treatment of translocation (11;14) AL amyloidosis
Exploration of the personalized immune checkpoint atlas of plasma cell dyscrasias patients using high‑dimensional single‑cell analysis. Oncol Rep. 2020 Apr 21;: Authors: Tu C, Zheng Y, Zhang H, Wang J Abstract Immune checkpoint blockade endows patients with unparalleled success in conquering cancer. Unfortunately, inter‑individual heterogeneity causes failure in controlling tumors in many patients. Emerging mass cytometry technology is capable of revealing a multiscale onco‑immune landscape that improves the efficacy of cancer immunotherapy. We introduced mass cytometry to determine the persona...
CONCLUSION: In rare cases, as is the case in our observation, the serous degeneration of bone marrow can reveal underlying solid tumors or hemopathies, knowledge of it can help guide the investigations towards these etiologies. PMID: 32340756 [PubMed - as supplied by publisher]
Blood Cancer Journal, Published online: 14 April 2020; doi:10.1038/s41408-020-0306-5A validated composite organ and hematologic response model for early assessment of treatment outcomes in light chain amyloidosis
Heparanase - Discovery and Targets. Adv Exp Med Biol. 2020;1221:61-69 Authors: Lindahl U, Li JP Abstract Heparanase was discovered during a study of the heparin proteoglycan (serglycin) in mast cells. Newly synthesized polysaccharide chains, kDa 60-100 x 103, were rapidly degraded to fragments similar in size to commercially available heparin (averaging 15 x 103). Analysis of the degradation products identified reducing-terminal glucuronic acid residues, shown by studies of heparin biosynthesis to be of ßD-configuration in the intact polymer. Heparanase, thus identified as an endo-ßD-g...
We report a new class of natural-product-inspired covalent inhibitors of telomerase that target the catalytic active site. Age-Related Epigenetic Changes that Suppress Mitochondrial Function https://www.fightaging.org/archives/2020/03/age-related-epigenetic-changes-that-suppress-mitochondrial-function/ Today's open access research reports on two specific epigenetic changes observed in old individuals that act to reduce mitochondrial function. This joins an existing list of genes for which expression changes are known to impact mitochondrial function with age. A herd of hundreds of mitochondria are found ...
Blood Cancer Journal, Published online: 25 February 2020; doi:10.1038/s41408-020-0291-8Blood mass spectrometry detects residual disease better than standard techniques in light-chain amyloidosis
We report a case of localized small bowel amyloidosis studied by 18F-choline and 18F-FDG–iodinated PET/CT performed for staging an aggressive prostatic cancer.
Conditions: Cancer; Cardiovascular Diseases; Hereditary Cancer; Hereditary Cardiac Amyloidosis Interventions: Behavioral: e-Health (web-based) disclosure portal; Behavioral: Provider mediated disclosure Sponsors: University of Pennsylvania; Fox Chase Cancer Center Enrolling by invitation