I had written about Rapamycin recently as I had visited Easter Island
[1]. Rapamycin is a macrolide, which is produced by the bacterium Streptomyces
hygroscopicus; samples of this bacterium were taken from Easter Island. The
name Rapamycin commemorates Easter Island as Rapa Nui (Great Rapa) is the
Polynesian name of Easter Island.
I was surprised that I’ve found three abstracts on Rapamycin at the 2017
ACR Annual Meeting in San Diego.
Olivier Benveniste and colleagues [2] presented a study called [Abstract
5L]: “Rapamycin Vs. Placebo for the Treatment of Inclusion Body Myositis:
Improvement of the 6 Min Walking Distance, a Functional Scale, the FVC and
Muscle Quantitative MRI”. Conclusion: “Even if the primary endpoint was not
reached, these first results showed coherent data in favor of rapamycin. …”
A Japanese group of scientists around Yo Ueda looked at Rapamycin and
found, that arthritis in SKG mice can be ameliorated [3].
Earl Sands and colleagues yet looked at another interesting field [4]:
“Initial Phase 2 Clinical Data of SEL-212 in Symptomatic Gout Patients: Monthly
Dosing of a Pegylated Uricase (Pegsiticase) with Svp-Rapamycin Enables
Sustained Reduction of Serum Uric Acid Levels By Mitigating Formation of
Anti-Drug Antibodies”. Conclusion: “SEL-212 has been well-tolerated, and,
unlike pegylated uricases alone, has mitigated immunogenicity, reduced flare
rate and enabled repeated monthly dosing with sustained control of sUA levels
in gout patients.”
The abstract of Thomas Winans and colleagues looked at [5]: “mTORC1
Blockade with Rapamycin and N-Acetylcysteine Reduces Anti-Phospholipid Antibody
Levels in Controlled Clinical Trials of Patients with SLE”. Conclusion: “These
ancillary studies suggest that rapamycin and NAC limit aPL production which
should be included as an efficacy outcome in clinical trials of mTORC1 blockade
in patients with SLE and APS.”
I thought that Rapamycin had its place in transplantation medicine, but
now I see that Rapamycin will be useful in Rheumatology, too.
Links and References:
[2] Benveniste O, Hogrel JY, Annoussamy M, Bachasson D, Rigolet A,
Servais L, Salem JE, Hervier B, Landon Cardinal O, Mariampillai K, hulot JS,
Carlier P, Allenbach Y. Rapamycin Vs. Placebo for the Treatment of Inclusion
Body Myositis: Improvement of the 6 Min Walking Distance, a Functional Scale,
the FVC and Muscle Quantitative MRI [abstract]. Arthritis Rheumatol. 2017; 69 (suppl 10). http://acrabstracts.org/abstract/rapamycin-vs-placebo-for-the-treatment-of-inclusion-body-myositis-improvement-of-the-6-min-walking-distance-a-functional-scale-the-fvc-and-muscle-quantitative-mri/.
Accessed December 7, 2017.
[3] Ueda Y, Okano T, Yamada H, Ichise Y, Naka I, Takahashi S, Sendo S,
Akashi K, Onishi A, Saegusa J, Morinobu A. Inhibition of the Mechanistic Target
of Rapamycin Pathway and Glutaminolysis Facilitates the Expansion of
Myeloid-Derived Suppressor Cells and Synergistically Ameliorates Arthritis in
SKG Mice [abstract]. Arthritis Rheumatol.
2017; 69 (suppl 10). http://acrabstracts.org/abstract/inhibition-of-the-mechanistic-target-of-rapamycin-pathway-and-glutaminolysis-facilitates-the-expansion-of-myeloid-derived-suppressor-cells-and-synergistically-ameliorates-arthritis-in-skg-mice/.
Accessed December 7, 2017.
[4] Sands E, Kivitz AJ, DeHaan Ph.D. W, Johnston L, Kishimoto TK. Initial
Phase 2 Clinical Data of SEL-212 in Symptomatic Gout Patients: Monthly Dosing
of a Pegylated Uricase (Pegsiticase) with Svp-Rapamycin Enables Sustained
Reduction of Serum Uric Acid Levels By Mitigating Formation of Anti-Drug
Antibodies [abstract]. Arthritis Rheumatol. 2017; 69 (suppl 10). http://acrabstracts.org/abstract/initial-phase-2-clinical-data-of-sel-212-in-symptomatic-gout-patients-monthly-dosing-of-a-pegylated-uricase-pegsiticase-with-svp-rapamycin-enables-sustained-reduction-of-serum-uric-acid-levels-by-m/.
Accessed December 7, 2017.
[5] Winans T, Kelly R, Lai ZW, Faraone S, Phillips PE, Banki K, Perl A.
mTORC1 Blockade with Rapamycin and N-Acetylcysteine Reduces Anti-Phospholipid
Antibody Levels in Controlled Clinical Trials of Patients with SLE [abstract]. Arthritis Rheumatol. 2017; 69 (suppl
10). http://acrabstracts.org/abstract/mtorc1-blockade-with-rapamycin-and-n-acetylcysteine-reduces-anti-phospholipid-antibody-levels-in-controlled-clinical-trials-of-patients-with-sle/.
Accessed December 7, 2017.
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