Molecules that prevent protein misfolding point to new type of therapy for AL amyloidosis

(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.
Source: EurekAlert! - Cancer - Category: Cancer & Oncology Source Type: news

Related Links:

a Stoppacciaro Monoclonal Gammopathies of Renal Significance (MGRS) are a rather heterogeneous group of renal disorders caused by a circulating monoclonal (MC) immunoglobulin (Ig) component, often in the absence of multiple myeloma (MM) or another clinically relevant lymphoproliferative disorder. Nevertheless, substantial kidney damage could occur, despite the “benign” features of the bone-marrow biopsy. One example is renal amyloidosis, often linked to a small clone of plasma cells, without the invasive features of MM. However, patients with amyloidosis may present with a nephrotic syndrome and ren...
Source: Cancers - Category: Cancer & Oncology Authors: Tags: Review Source Type: research
RARITAN, NJ, June 13, 2020 – The Janssen Pharmaceutical Companies of Johnson &Johnson announced today results from the first randomized Phase 3 study investigating subcutaneous daratumumab[i] in the treatment of patients with newly diagnosed light chain (AL) amyloidosis, a rare and potentially fatal disease.[1],[2] The data demonstrated subcutaneous daratumumab in combination with cyclophosphamide, bortezomib, and dexamethasone (D-CyBorD) resulted in a higher hematologic complete response rate (CR), (53 percent vs. 18 percent [P
Source: Johnson and Johnson - Category: Pharmaceuticals Tags: Innovation Source Type: news
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
Source: Blood Cancer Journal - Category: Hematology Authors: Source Type: research
Conclusions: The p105-11-1F4 complex is a novel approach to developing a multi-amyloid targeting opsonizing reagent. The preformed peptope-mAb could be used as a single injection immunotherapeutic to effect resolution of diverse forms of tissue amyloid through cell-mediated uptake. Acknowledgment: This study was supported in part by the National Institute of Diabetes and Digestive and Kidney Diseases, DK110038.
Source: Journal of Nuclear Medicine - Category: Nuclear Medicine Authors: Tags: Preclinical Probes for Cardiovascular, Endocrine & amp; Other Source Type: research
Blood Cancer Journal, Published online: 11 May 2020; doi:10.1038/s41408-020-0321-6Venetoclax for the treatment of translocation (11;14) AL amyloidosis
Source: Blood Cancer Journal - Category: Hematology Authors: Source Type: research
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...
Source: Oncology Reports - Category: Cancer & Oncology Tags: Oncol Rep Source Type: research
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]
Source: Annales de Pathologie - Category: Pathology Authors: Tags: Ann Pathol Source Type: research
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
Source: Blood Cancer Journal - Category: Hematology Authors: Source Type: research
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...
Source: Advances in Experimental Medicine and Biology - Category: Research Tags: Adv Exp Med Biol Source Type: research
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 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 ...
Source: Fight Aging! - Category: Research Authors: Tags: Newsletters Source Type: blogs
More News: Amyloidosis | Cancer | Cancer & Oncology | Cardiology | Heart