{"id":9458,"date":"2025-04-11T00:53:13","date_gmt":"2025-04-11T00:53:13","guid":{"rendered":"https:\/\/www.europesays.com\/uk\/9458\/"},"modified":"2025-04-11T00:53:13","modified_gmt":"2025-04-11T00:53:13","slug":"rapamycin-improves-proms-in-patients-with-me-cfs-in-early-trial","status":"publish","type":"post","link":"https:\/\/www.europesays.com\/uk\/9458\/","title":{"rendered":"Rapamycin Improves PROMs in Patients With ME\/CFS in Early Trial"},"content":{"rendered":"<p class=\"pb-2\">Stephanie L. Grach, MD, MS<\/p>\n<p class=\"pb-2\">Credit: Mayo Clinic<\/p>\n<p><img decoding=\"async\" class=\"m-auto absolute inset-0 max-w-[0%] max-h-[0%] border-[3px] border-solid border-white shadow-[0px_0px_8px_rgba(0,0,0,0.3)] box-border transition ease-in-out duration-500\" src=\"https:\/\/www.europesays.com\/uk\/wp-content\/uploads\/2025\/04\/8ee50cedc9a4893658b46d50a167c07c3fef6268-201x251.jpg\"\/><\/p>\n<p class=\"pb-2\">Weekly rapamycin improved <a target=\"_blank\" href=\"https:\/\/www.hcplive.com\/view\/seeing-more-than-hiv-why-patient-reported-outcomes-matter\" rel=\"noopener\">patient-reported outcome measures <\/a>(PROMs) in patients with Myalgic Encephalomyelitis\/Chronic Fatigue Syndrome (ME\/CFS) after 3 months of use.1<\/p>\n<p class=\"pb-2\">These findings, from a phase 1 trial, were presented by Stephanie L. Grach, MD, MS, assistant professor of medicine at Mayo Clinic,\u00a0at the\u00a0<a target=\"_self\" href=\"https:\/\/www.hcplive.com\/conference\/acp\" rel=\"noopener\">American College of Physicians (ACP) Internal Medicine (IM) Meeting 2025<\/a>, held April 3-5, in New Orleans, Louisiana.<\/p>\n<p class=\"pb-2\">\u201cME\/CFS is a devastating infection-associated multisystem illness characterized by debilitating fatigue, autonomic dysfunction, and post exertional malaise (PEM), among other symptoms,\u201d Grach and colleagues wrote in their poster.1<\/p>\n<p class=\"pb-2\">The data reported were from the study\u2019s first 40 participants that had completed the 3-month follow-up period. Participants received 0.5 or 1 mg rapamycin weekly up-titrated weekly to 6mg weekly or as tolerated. Participants completed functional outcome questionnaires at baseline (pre-rapamycin), 1 month (T=1), 2 months (T=2), and 3 months (T=3) after beginning therapy. The questionnaires completed were Bell Activity Scale (BAS); Multidimensional Fatigue Inventory (MFl); RAND 36-Item Short-Form Health Survey (SF-36); and Fatigue, sleep, PEM, and orthostatic intolerance symptoms of the Symptom Severity Scale version 2.2 (SSS). Investigators also performed safety labs at each timepoint for monitoring.<\/p>\n<p class=\"pb-2\">\u201cRapamycin has been of increasing interest as a geroprotective drug, having potential longevity and systematic inflammation modulation effects via promoting autophagy through inhibition of mTOR. Accordingly, patients managed by a leading ME\/CFS clinician reported clinical improvements with use of rapamycin prompting further investigation utilizing PROMs and ATG13 biomarker,\u201d Grach and colleagues wrote.1<\/p>\n<p class=\"pb-2\">Participants had a mean age of 47.43 years (range, 24.80-76.38), weight of 171.35 lbs (range, 115.00-310.00), BMI of 26.64 (range,18.85-47.13), and a disease duration of 16.45 years (rang1<\/p>\n<p class=\"pb-2\" wp_automatic_readability=\"9\">The investigators found that PROMs statistically significantly improved from baseline to the 3-month time point, with BAS, MFI, SF-36 subscores, SSS fatigue, disrupted sleep. PEM, and orthostatic intolerance all demonstrating improvements (P <\/p>\n<p class=\"pb-2\">Specifically, BAS scores improved from 34.84 (standard deviation [SD], 3.13) to 42.26 (SD, 3.87; P P P P .001)); and MFI aggregated scores improved from 79.63 (SD, 1.43) to 72.26 (SD, 2.28; P <\/p>\n<p class=\"pb-2\">\u201cIn line with suspected abnormalities of mTOR inhibition, the results of our study support the potential use of rapamycin in ME\/CFS,\u201d Grach and colleagues concluded.1<\/p>\n<p class=\"pb-2\">Other investigators on the study previously completed preclinical investigations of rapamycin in models of CE\/MFS that suggested that an impairment of autophagy following the release of ATG13 into serum could be a pathological signal in ME\/CFS.2<\/p>\n<p>REFERENCES<\/p>\n<ol class=\"my-2\">\n<li class=\"list-decimal ml-8\">Grach SL, Roy A, Kaufman DL, et al. Effect of Rapamycin on Functional Outcomes in Myalgic Encephalomyelitis\/Chronic Fatigue Syndrome.<\/li>\n<li class=\"list-decimal ml-8\">Gottschalk G, Peterson D, Knox K, et al. Elevated ATG13 in serum of patients with ME\/CFS stimulates oxidative stress response in microglial cells via activation of receptor for advanced glycation end products (RAGE). Mol. Cell. Neurosci. 2022; 120 (103731). doi: 10.1016\/j.mcn.2022.103731<\/li>\n<\/ol><\/p>\n","protected":false},"excerpt":{"rendered":"Stephanie L. Grach, MD, MS Credit: Mayo Clinic Weekly rapamycin improved patient-reported outcome measures (PROMs) in patients with&hellip;\n","protected":false},"author":2,"featured_media":9459,"comment_status":"","ping_status":"","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[4315],"tags":[849,6441,6435,6439,967,5205,535,6442,2460,6446,105,4348,6447,6449,6436,6443,466,4554,4326,3912,12,6440,5489,5389,6444,6437,3913,6438,6448,2343,6445,70,6434,16,15],"class_list":{"0":"post-9458","1":"post","2":"type-post","3":"status-publish","4":"format-standard","5":"has-post-thumbnail","7":"category-medication","8":"tag-allergy","9":"tag-cardiology","10":"tag-clinicians","11":"tag-dermatology","12":"tag-diabetes","13":"tag-doctors","14":"tag-drugs","15":"tag-endocrinology","16":"tag-fda","17":"tag-gastroenterology","18":"tag-health","19":"tag-healthcare","20":"tag-hematology","21":"tag-infectious-disease","22":"tag-insight","23":"tag-internal-medicine","24":"tag-interviews","25":"tag-medical","26":"tag-medication","27":"tag-medicine","28":"tag-news","29":"tag-ophthalmology","30":"tag-pharmaceuticals","31":"tag-pipeline","32":"tag-primary-care","33":"tag-providers","34":"tag-psychiatry","35":"tag-pulmonology","36":"tag-rare-disease","37":"tag-research","38":"tag-rheumatology","39":"tag-science","40":"tag-treatments","41":"tag-uk","42":"tag-united-kingdom"},"share_on_mastodon":{"url":"https:\/\/pubeurope.com\/@uk\/114316601168864108","error":""},"_links":{"self":[{"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/posts\/9458","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/users\/2"}],"replies":[{"embeddable":true,"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/comments?post=9458"}],"version-history":[{"count":0,"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/posts\/9458\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/media\/9459"}],"wp:attachment":[{"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/media?parent=9458"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/categories?post=9458"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/tags?post=9458"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}