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Systemic scleroderma

GtoPdb Disease Summaries

This section gives an overview of the disease, and where available shows the following:

  • Synonyms: Shows known synonyms for the disease.
  • Description: Gives a basic description/definition of the disease.
  • Database Link: External links to the same disease at the Disease Ontology, OMIM or Orphanet sites.
  • Immunopharmacology comments: General comments about the target's role in immunopharmacology, provided by GtoImmuPdb curators.
  • Associated with: Counts are displayed for the total targets the disease is associated with in GtoPdb. The counts of targets and ligands of immunological relevance associated to the disease are also shown.

More information can be found in the help pages.

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Disease ID:1036
Name:Systemic scleroderma
Associated with:1 target
2 immuno-relevant ligands
Synonyms
Scleroderma, familial progressive | Systemic sclerosis
Description
A complex heterogeneous fibrosing autoimmune disorder characterised by extensive fibrosis, vasculopathy, and immune dysfunction.
Database Links
Disease Ontology: DOID:418
OMIM: 181750
Orphanet: ORPHA90291

Targets

GtoPdb Disease Summaries - Targets

Click on the target name to link to its detailed view page

Where available, information is display on the role of the target in the disease; drugs which target the disease and their therapeutic use and side-effects.

If there is mutation data curated in GtoPdb this is indicated, with a link back to the appropriate section on the target detailed view page

Immuno ligand interactions - If available, a table of immuno-relevant ligands is shown. These ligands have been curated as having an association to the disease and possess interaction data with the target in GtoPdb. The approval status of the ligand is shown, along with curator comments and an indication of whether the target is considered the primary target of the ligand.

More information can be found in the help pages.

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Key to terms and symbols
NLRP1

Ligands

GtoPdb Disease Summaries - Ligands

Click ligand name to view ligand summary page

  • Approved: If the ligand is an approved drug this is indicated, along with approval bodies.
  • Immuno: Immuno icon indicates the ligand is immuno-relevant

Click the arrow in the final column to expand comments

  • Immuno Disease Comments: Curatorial comments specifically added as part of GtoImmuPdb. They give more information on the association between the ligand and disease in the context of immunopharmacology.
  • Clinical Use: General clinical comments relating to the ligand and may not necessarily be specific to the disease in question. With hyperlink to more details on the ligand summary pages.
  • Bioactivty Comments: Curatorial comments specifically about the compounds biological activity - with hyperlink to more details on the ligand summary pages.

More information can be found in the help pages.

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Key to terms and symbols Click ligand name to view ligand summary Click column headers to sort
Ligand References Clinical and Disease comments
brentuximab vedotin
Immuno Disease Comments: Phase 1/2 clinical candidate for systemic sclerosis- see NCT03222492 and NCT03198689
Clinical Use: Brentuximab vedotin is an anti-neoplastic agent used in the treatment of Hodgkin's lymphoma [5] and systemic anaplastic large cell lymphoma. Several clinical trials are evaluating the combination of brentuximab vedotin with immune checkpoint inhibitors, to assess any synergistic effects as a result of dual targeting. Checkpoint inhibitors being investigated in this way include and (both anti-PD-1), and (anti-CTLA4). Brentuximab vedotin + pembrolizumab (NCT02684292) and brentuximab vedotin + nivolumab (NCT03138499) are both Phase 3 studies in patients with relapsed/refractory classical Hodgkin's lymphoma.

Expanded approvals:
In November 2017, the FDA approved brentuximab vedotin for the treatment of adult patients with primary cutaneous anaplastic large cell lymphoma (pcALCL) or CD30-expressing mycosis fungoides (MF) who have received prior systemic therapy, following review of results from the Phase 3 ALCANZA trial (NCT01578499) [2]. In March 2018, the FDA approved the use of brentuximab vedotin for the treatment of patients with previously untreated stage III or IV classical Hodgkin lymphoma (cHL) in combination with chemotherapy. This expansion was based on results from the ECHELON-1 clinical trial (NCT01712490) [1,6]. November 2018 saw further expansion of approval for the use of brentuximab vedotin plus chemotherapy for adult patients who are newly diagnosed with CD30-expressing peripheral T-cell lymphomas. | View clinical data
Bioactivity Comments: Detailed information contained in the covering patent [3] describes in vitro and in vivo activity of the invention, which supports its anti-CD30 action. However, no affinity data is provided for the interaction between the antibody and its molecular target. | View biological activity
lenabasum
Immuno Disease Comments: A Phase 3 multicenter trial to evaluate AJA's safety and efficacy in SSc has recently been initiated- see NCT03398837
Clinical Use: Ajulemic acid (research code JBT-101) is being evaluated as a treatment for systemic lupus erythematosus (SLE) in Phase 2 clinical trial NCT03093402.
It was also more effective in reducing chronic neuropathic pain than placebo in a Phase 2 clinical trial [4]. | View clinical data

References

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1. Connors JM, Jurczak W, Straus DJ, Ansell SM, Kim WS, Gallamini A, Younes A, Alekseev S, Illés Á, Picardi M et al.. (2018) Brentuximab Vedotin with Chemotherapy for Stage III or IV Hodgkin's Lymphoma. N Engl J Med, 378 (4): 331-344. [PMID:29224502]

2. FDA. FDA approves Brentuximab vedotin for the treatment of adult patients with primary cutaneous anaplastic large cell lymphoma. Accessed on 10/11/2017. Modified on 10/11/2017. fda.gov, https://www.fda.gov/Drugs/InformationOnDrugs/ApprovedDrugs/ucm584543.htm?utm_campaign=Oncology%2011%2F9&utm_medium=email&utm_source=Eloqua&elqTrackId=06d11c937bec44c79cd3d453daecb608&elq=4d1b00c5aad64538a4ce8e687b3f3786&elqaid=1289&elqat=1&elqCampaignId=770

3. Francisco JA, Risdon G, Wahl AF, Siegall C. (2006) immunotherapy; fusion proteins; antitumor agents; for treatment and prevention of hodgkin's disease. Patent number: US7090843. Assignee: Seattle Genetics, Inc. Priority date: 09/12/2014. Publication date: 15/08/2006.

4. Karst M, Salim K, Burstein S, Conrad I, Hoy L, Schneider U. (2003) Analgesic effect of the synthetic cannabinoid CT-3 on chronic neuropathic pain: a randomized controlled trial. JAMA, 290 (13): 1757-62. [PMID:14519710]

5. Moskowitz CH, Nademanee A, Masszi T, Agura E, Holowiecki J, Abidi MH, Chen AI, Stiff P, Gianni AM, Carella A et al.. (2015) Brentuximab vedotin as consolidation therapy after autologous stem-cell transplantation in patients with Hodgkin's lymphoma at risk of relapse or progression (AETHERA): a randomised, double-blind, placebo-controlled, phase 3 trial. Lancet, 385 (9980): 1853-62. [PMID:25796459]

6. Younes A, Connors JM, Park SI, Fanale M, O'Meara MM, Hunder NN, Huebner D, Ansell SM. (2013) Brentuximab vedotin combined with ABVD or AVD for patients with newly diagnosed Hodgkin's lymphoma: a phase 1, open-label, dose-escalation study. Lancet Oncol, 14 (13): 1348-56. [PMID:24239220]