Mycophenolate mofetil

United States National Library of Medicine

Accessed on 29 Jul 2013 from http://chem.sis.nlm.nih.gov/chemidplus/chemidheavy.jsp.

Target:

Purine synthesis

(1)

Roche/Food and Drug Administration

Accessed on 5 Jan 2012 from http://www.accessdata.fda.gov/drugsatfda_docs/label/2009/050722s021,050723s019,050758s019,050759s024lbl.pdf.

(3)

A one-year prospective, randomized, placebo-controlled, quadruple-blinded, phase II safety pilot trial of combination therapy with interferon beta-1a and mycophenolate mofetil in early relapsing-remitting multiple sclerosis (TIME MS).

Frohman EM, Brannon K, Racke MK, Hawker K

Clin Neuropharmacol. 2004 Mar-Apr; 27(2):80-3. PMID: 15252268. Abstract

Status for MS:

Phase II

(7)

Remington GM, Treadaway K, Frohman T, Salter A, Stüve O, Racke MK, Hawker K, Agosta F, Sormani M P, Filippi M, et al.

Ther Adv Neurol Disord. 2010 Jan; 3(1):3-13. PMID: 21180632. Abstract

(8)

Safety Study of Combination Therapy with Intramuscular Avonex and Oral CellCept in Patients with Multiple Sclerosis

ClinicalTrials.gov, 12 Feb 2009

Accessed on 6 Jan 2012 from http://clinicaltrials.gov/ct2/show/NCT00223301.

Administration:

Oral

(9)

Food and Drug Administration

Accessed on 6 Jan 2012 from http://www.accessdata.fda.gov/scripts/cder/drugsatfda/index.cfm?fuseaction=Search.Overview&DrugName=CELLCEPT.

Commercial:

Manufactured by Genentech

(1)

Roche/Food and Drug Administration

Accessed on 5 Jan 2012 from http://www.accessdata.fda.gov/drugsatfda_docs/label/2009/050722s021,050723s019,050758s019,050759s024lbl.pdf.

Names

Name:

Mycophenolate mofetil

Trade name:

CellCept

(1)

Roche/Food and Drug Administration

Accessed on 5 Jan 2012 from http://www.accessdata.fda.gov/drugsatfda_docs/label/2009/050722s021,050723s019,050758s019,050759s024lbl.pdf.

Synonyms:

CellCept

(2)

United States National Library of Medicine

Accessed on 29 Jul 2013 from http://chem.sis.nlm.nih.gov/chemidplus/chemidheavy.jsp.

Systematic name:

4-Hexenoic acid, 6-(1,3-dihydro-4-hydroxy-6-methoxy-7-methyl-3-oxo-5-isobenzofuranyl)-4-ethyl-, 2-(4-morpholinyl)ethyl ester, (E)

(2)

United States National Library of Medicine

Accessed on 29 Jul 2013 from http://chem.sis.nlm.nih.gov/chemidplus/chemidheavy.jsp.

Physiology

Class:

Immunosuppressor

(2)

United States National Library of Medicine

Accessed on 29 Jul 2013 from http://chem.sis.nlm.nih.gov/chemidplus/chemidheavy.jsp.

Target:

Purine synthesis

(1)

Roche/Food and Drug Administration

Accessed on 5 Jan 2012 from http://www.accessdata.fda.gov/drugsatfda_docs/label/2009/050722s021,050723s019,050758s019,050759s024lbl.pdf.

(3)

Treatment of neuromyelitis optica with mycophenolate mofetil: retrospective analysis of 24 patients.

Frohman EM, Brannon K, Racke MK, Hawker K

Clin Neuropharmacol. 2004 Mar-Apr; 27(2):80-3. PMID: 15252268. Abstract

Properties:

2-morpholinoethyl ester of mycophenolic acid

(5)

Jacob A, Matiello M, Weinshenker BG, Wingerchuk DM, Lucchinetti C, Shuster E, Carter J, Keegan MB, Kantarci OH, Pittock SJ

Arch Neurol. 2009 Sep; 66(9):1128-33. PMID: 19752302. Abstract

Mechanisms/Effects

Human:

Selectively inhibits inosine 5’-monophosphate dehydrogenase type II, an enzyme critical for synthesis of the nucleotide guanine, a pathway used for T and B cell proliferation

(3)

Treatment of neuromyelitis optica with mycophenolate mofetil: retrospective analysis of 24 patients.

Frohman EM, Brannon K, Racke MK, Hawker K

Clin Neuropharmacol. 2004 Mar-Apr; 27(2):80-3. PMID: 15252268. Abstract

(5)

Jacob A, Matiello M, Weinshenker BG, Wingerchuk DM, Lucchinetti C, Shuster E, Carter J, Keegan MB, Kantarci OH, Pittock SJ

Arch Neurol. 2009 Sep; 66(9):1128-33. PMID: 19752302. Abstract

Animal:

Impairs dendritic cell maturation and function in mice

(6)

Mycophenolic acid inhibits IL-2-dependent T cell proliferation, but not IL-2-dependent survival and sensitization to apoptosis.

Quéméneur L, Flacher M, Gerland L-M, Ffrench M, Revillard J-P, Bonnefoy-Berard N

J Immunol. 2002 Sep 1; 169(5):2747-55. PMID: 12193749. Abstract

Improves neurological function and reduces weight loss in a rat experimental autoimmune encephalomyelitis model

(16)

Mycophenolate mofetil improves neurological function and alters blood T-lymphocyte subsets in rats with experimental autoimmune encephalomyelitis.

Zhu Z, You W, Xie Z, Wang P, Liu Z, Wang C, Bi J

J Int Med Res. 2014 Feb 4. PMID: 24496150. Abstract

Decreases the ratio of CD4+ to CD8+ T cells in blood in a rat experimental autoimmune encephalomyelitis model

(16)

Mycophenolate mofetil improves neurological function and alters blood T-lymphocyte subsets in rats with experimental autoimmune encephalomyelitis.

Zhu Z, You W, Xie Z, Wang P, Liu Z, Wang C, Bi J

J Int Med Res. 2014 Feb 4. PMID: 24496150. Abstract

Regulatory and Commercial Status

Status for MS:

Phase II

(7)

Remington GM, Treadaway K, Frohman T, Salter A, Stüve O, Racke MK, Hawker K, Agosta F, Sormani M P, Filippi M, et al.

Ther Adv Neurol Disord. 2010 Jan; 3(1):3-13. PMID: 21180632. Abstract

(8)

Safety Study of Combination Therapy with Intramuscular Avonex and Oral CellCept in Patients with Multiple Sclerosis

ClinicalTrials.gov, 12 Feb 2009

Accessed on 6 Jan 2012 from http://clinicaltrials.gov/ct2/show/NCT00223301.

Highest status achieved (for any condition):

Approved

(9)

Treatment of neuromyelitis optica with mycophenolate mofetil: retrospective analysis of 24 patients.

Food and Drug Administration

Accessed on 6 Jan 2012 from http://www.accessdata.fda.gov/scripts/cder/drugsatfda/index.cfm?fuseaction=Search.Overview&DrugName=CELLCEPT.

Other uses:

Autoimmune hepatitis

(5)

Jacob A, Matiello M, Weinshenker BG, Wingerchuk DM, Lucchinetti C, Shuster E, Carter J, Keegan MB, Kantarci OH, Pittock SJ

Arch Neurol. 2009 Sep; 66(9):1128-33. PMID: 19752302. Abstract

Autonomic ganglionopathy

(5)

Treatment of neuromyelitis optica with mycophenolate mofetil: retrospective analysis of 24 patients.

Jacob A, Matiello M, Weinshenker BG, Wingerchuk DM, Lucchinetti C, Shuster E, Carter J, Keegan MB, Kantarci OH, Pittock SJ

Arch Neurol. 2009 Sep; 66(9):1128-33. PMID: 19752302. Abstract

Blistering dermopathies

(5)

Treatment of neuromyelitis optica with mycophenolate mofetil: retrospective analysis of 24 patients.

Jacob A, Matiello M, Weinshenker BG, Wingerchuk DM, Lucchinetti C, Shuster E, Carter J, Keegan MB, Kantarci OH, Pittock SJ

Arch Neurol. 2009 Sep; 66(9):1128-33. PMID: 19752302. Abstract

May be useful for treating inflammatory bowel disease

(17)

Mycophenolate mofetil therapy in the management of inflammatory bowel disease - A retrospective case series and review.

Smith MR, Cooper SC

J Crohns Colitis. 2014 Feb 4. PMID: 24507162. Abstract

Chronic inflammatory demyelinating polyradiculoneuropathy

(5)

Treatment of neuromyelitis optica with mycophenolate mofetil: retrospective analysis of 24 patients.

Jacob A, Matiello M, Weinshenker BG, Wingerchuk DM, Lucchinetti C, Shuster E, Carter J, Keegan MB, Kantarci OH, Pittock SJ

Arch Neurol. 2009 Sep; 66(9):1128-33. PMID: 19752302. Abstract

Inflammatory myopathies

(5)

Treatment of neuromyelitis optica with mycophenolate mofetil: retrospective analysis of 24 patients.

Jacob A, Matiello M, Weinshenker BG, Wingerchuk DM, Lucchinetti C, Shuster E, Carter J, Keegan MB, Kantarci OH, Pittock SJ

Arch Neurol. 2009 Sep; 66(9):1128-33. PMID: 19752302. Abstract

Systemic lupus erythematosus

(5)

Treatment of neuromyelitis optica with mycophenolate mofetil: retrospective analysis of 24 patients.

Jacob A, Matiello M, Weinshenker BG, Wingerchuk DM, Lucchinetti C, Shuster E, Carter J, Keegan MB, Kantarci OH, Pittock SJ

Arch Neurol. 2009 Sep; 66(9):1128-33. PMID: 19752302. Abstract

In combination with rituximab, controlled lupus nephritis without the use of oral steroids in a prospective observational study (publ 2013)

(14)

Prospective observational single-centre cohort study to evaluate the effectiveness of treating lupus nephritis with rituximab and mycophenolate mofetil but no oral steroids.

Condon MB, Ashby D, Pepper RJ, Cook TH, Levy JB, Griffith M, Cairns TD, Lightstone L

Ann Rheum Dis. 2013 Aug; 72(8):1280-6. Epub 2013 Jun 05. PMID: 23740227. Abstract

Multifocal motor neuropathy

(5)

Treatment of neuromyelitis optica with mycophenolate mofetil: retrospective analysis of 24 patients.

Jacob A, Matiello M, Weinshenker BG, Wingerchuk DM, Lucchinetti C, Shuster E, Carter J, Keegan MB, Kantarci OH, Pittock SJ

Arch Neurol. 2009 Sep; 66(9):1128-33. PMID: 19752302. Abstract

Myasthenia gravis

(5)

Treatment of neuromyelitis optica with mycophenolate mofetil: retrospective analysis of 24 patients.

Jacob A, Matiello M, Weinshenker BG, Wingerchuk DM, Lucchinetti C, Shuster E, Carter J, Keegan MB, Kantarci OH, Pittock SJ

Arch Neurol. 2009 Sep; 66(9):1128-33. PMID: 19752302. Abstract

Neuromyelitis optica

(5)

Treatment of neuromyelitis optica with mycophenolate mofetil: retrospective analysis of 24 patients.

Jacob A, Matiello M, Weinshenker BG, Wingerchuk DM, Lucchinetti C, Shuster E, Carter J, Keegan MB, Kantarci OH, Pittock SJ

Arch Neurol. 2009 Sep; 66(9):1128-33. PMID: 19752302. Abstract

Reduced the relapse rate in neuromyelitis optica by up to 87.4% (with a failure rate of 36%) in a retrospective, multicenter analysis

(18)

Comparison of Relapse and Treatment Failure Rates Among Patients With Neuromyelitis Optica: Multicenter Study of Treatment Efficacy.

Mealy MA, Wingerchuk DM, Palace J, Greenberg BM, Levy M

JAMA Neurol. 2014 Jan 20. PMID: 24445513. Abstract

Reduced the frequency of relapses (such that median annualized relapse rate declined from 1.5 to 0.0 after mycophenolate mofetil) and stabilized or improved disability in individuals with neuromyelitis optica spectrum disorder in a retrospective, observational study involving 59 individuals

(20)

Mycophenolate mofetil in the treatment of neuromyelitis optica spectrum disorder.

Huh S-Y, Kim S-H, Hyun J-W, Joung A-R, Park M S, Kim B-J, Kim H J

JAMA Neurol. 2014 Nov; 71(11):1372-8. PMID: 25199960. Abstract

Psoriasis

(5)

Treatment of neuromyelitis optica with mycophenolate mofetil: retrospective analysis of 24 patients.

Jacob A, Matiello M, Weinshenker BG, Wingerchuk DM, Lucchinetti C, Shuster E, Carter J, Keegan MB, Kantarci OH, Pittock SJ

Arch Neurol. 2009 Sep; 66(9):1128-33. PMID: 19752302. Abstract

Approved for prevention of transplant organ rejection

(1)

Treatment of neuromyelitis optica with mycophenolate mofetil: retrospective analysis of 24 patients.

Roche/Food and Drug Administration

Accessed on 5 Jan 2012 from http://www.accessdata.fda.gov/drugsatfda_docs/label/2009/050722s021,050723s019,050758s019,050759s024lbl.pdf.

Vasculitic neuropathies

(5)

Jacob A, Matiello M, Weinshenker BG, Wingerchuk DM, Lucchinetti C, Shuster E, Carter J, Keegan MB, Kantarci OH, Pittock SJ

Arch Neurol. 2009 Sep; 66(9):1128-33. PMID: 19752302. Abstract

Vasculitides

(5)

Treatment of neuromyelitis optica with mycophenolate mofetil: retrospective analysis of 24 patients.

Jacob A, Matiello M, Weinshenker BG, Wingerchuk DM, Lucchinetti C, Shuster E, Carter J, Keegan MB, Kantarci OH, Pittock SJ

Arch Neurol. 2009 Sep; 66(9):1128-33. PMID: 19752302. Abstract

Administration:

Oral

(9)

Food and Drug Administration

Accessed on 6 Jan 2012 from http://www.accessdata.fda.gov/scripts/cder/drugsatfda/index.cfm?fuseaction=Search.Overview&DrugName=CELLCEPT.

Negative effects:

Birth defects

(1)

Roche/Food and Drug Administration

Accessed on 5 Jan 2012 from http://www.accessdata.fda.gov/drugsatfda_docs/label/2009/050722s021,050723s019,050758s019,050759s024lbl.pdf.

Diarrhea

(1)

Roche/Food and Drug Administration

Accessed on 5 Jan 2012 from http://www.accessdata.fda.gov/drugsatfda_docs/label/2009/050722s021,050723s019,050758s019,050759s024lbl.pdf.

Fungal infections

(1)

Roche/Food and Drug Administration

Accessed on 5 Jan 2012 from http://www.accessdata.fda.gov/drugsatfda_docs/label/2009/050722s021,050723s019,050758s019,050759s024lbl.pdf.

High blood pressure

(1)

Roche/Food and Drug Administration

Accessed on 5 Jan 2012 from http://www.accessdata.fda.gov/drugsatfda_docs/label/2009/050722s021,050723s019,050758s019,050759s024lbl.pdf.

Miscarriage

(1)

Roche/Food and Drug Administration

Accessed on 5 Jan 2012 from http://www.accessdata.fda.gov/drugsatfda_docs/label/2009/050722s021,050723s019,050758s019,050759s024lbl.pdf.

Neutropenia

(1)

Roche/Food and Drug Administration

Accessed on 5 Jan 2012 from http://www.accessdata.fda.gov/drugsatfda_docs/label/2009/050722s021,050723s019,050758s019,050759s024lbl.pdf.

Pure red cell aplasia

(1)

Roche/Food and Drug Administration

Accessed on 5 Jan 2012 from http://www.accessdata.fda.gov/drugsatfda_docs/label/2009/050722s021,050723s019,050758s019,050759s024lbl.pdf.

Stomach pain

(1)

Roche/Food and Drug Administration

Accessed on 5 Jan 2012 from http://www.accessdata.fda.gov/drugsatfda_docs/label/2009/050722s021,050723s019,050758s019,050759s024lbl.pdf.

Swelling of lower legs, angles and feet

(1)

Roche/Food and Drug Administration

Accessed on 5 Jan 2012 from http://www.accessdata.fda.gov/drugsatfda_docs/label/2009/050722s021,050723s019,050758s019,050759s024lbl.pdf.

Viral infections (such as progressive multifocal leukoencephalopathy and BK virus-associated nephropathy)

(1)

Roche/Food and Drug Administration

Accessed on 5 Jan 2012 from http://www.accessdata.fda.gov/drugsatfda_docs/label/2009/050722s021,050723s019,050758s019,050759s024lbl.pdf.

Vomiting

(1)

Roche/Food and Drug Administration

Accessed on 5 Jan 2012 from http://www.accessdata.fda.gov/drugsatfda_docs/label/2009/050722s021,050723s019,050758s019,050759s024lbl.pdf.

Commercial:

Manufactured by Genentech

(1)

Safety Study of Combination Therapy with Intramuscular Avonex and Oral CellCept in Patients with Multiple Sclerosis

Roche/Food and Drug Administration

Accessed on 5 Jan 2012 from http://www.accessdata.fda.gov/drugsatfda_docs/label/2009/050722s021,050723s019,050758s019,050759s024lbl.pdf.

Key Clinical Trials

Placebo-controlled Trials:

Trial name:

Safety Study of Combination Therapy with Intramuscular Avonex and Oral CellCept in Patients with Multiple Sclerosis (TIME-MS) (publ January 2010)

(7)

Remington GM, Treadaway K, Frohman T, Salter A, Stüve O, Racke MK, Hawker K, Agosta F, Sormani M P, Filippi M, et al.

Ther Adv Neurol Disord. 2010 Jan; 3(1):3-13. PMID: 21180632. Abstract

(8)

ClinicalTrials.gov, 12 Feb 2009

Accessed on 6 Jan 2012 from http://clinicaltrials.gov/ct2/show/NCT00223301.

Phase:

Phase II trial

(7)

Remington GM, Treadaway K, Frohman T, Salter A, Stüve O, Racke MK, Hawker K, Agosta F, Sormani M P, Filippi M, et al.

Ther Adv Neurol Disord. 2010 Jan; 3(1):3-13. PMID: 21180632. Abstract

(8)

Safety Study of Combination Therapy with Intramuscular Avonex and Oral CellCept in Patients with Multiple Sclerosis

ClinicalTrials.gov, 12 Feb 2009

Accessed on 6 Jan 2012 from http://clinicaltrials.gov/ct2/show/NCT00223301.

Study Design:

Company-sponsored, randomized, double-blind, placebo-controlled trial to characterize the adverse events (primary endpoint) associated with combining mycophenolate mofetil with interferon beta-1a

(7)

Remington GM, Treadaway K, Frohman T, Salter A, Stüve O, Racke MK, Hawker K, Agosta F, Sormani M P, Filippi M, et al.

Ther Adv Neurol Disord. 2010 Jan; 3(1):3-13. PMID: 21180632. Abstract

(8)

Safety Study of Combination Therapy with Intramuscular Avonex and Oral CellCept in Patients with Multiple Sclerosis

ClinicalTrials.gov, 12 Feb 2009

Accessed on 6 Jan 2012 from http://clinicaltrials.gov/ct2/show/NCT00223301.

Disease Stage:

RRMS

(7)

Remington GM, Treadaway K, Frohman T, Salter A, Stüve O, Racke MK, Hawker K, Agosta F, Sormani M P, Filippi M, et al.

Ther Adv Neurol Disord. 2010 Jan; 3(1):3-13. PMID: 21180632. Abstract

(8)

Safety Study of Combination Therapy with Intramuscular Avonex and Oral CellCept in Patients with Multiple Sclerosis

ClinicalTrials.gov, 12 Feb 2009

Accessed on 6 Jan 2012 from http://clinicaltrials.gov/ct2/show/NCT00223301.

Enrollment/Number of Patients:

24 treatment-naïve patients

(7)

Remington GM, Treadaway K, Frohman T, Salter A, Stüve O, Racke MK, Hawker K, Agosta F, Sormani M P, Filippi M, et al.

Ther Adv Neurol Disord. 2010 Jan; 3(1):3-13. PMID: 21180632. Abstract

(8)

Safety Study of Combination Therapy with Intramuscular Avonex and Oral CellCept in Patients with Multiple Sclerosis

ClinicalTrials.gov, 12 Feb 2009

Accessed on 6 Jan 2012 from http://clinicaltrials.gov/ct2/show/NCT00223301.

Duration:

1 year

(7)

Remington GM, Treadaway K, Frohman T, Salter A, Stüve O, Racke MK, Hawker K, Agosta F, Sormani M P, Filippi M, et al.

Ther Adv Neurol Disord. 2010 Jan; 3(1):3-13. PMID: 21180632. Abstract

(8)

Safety Study of Combination Therapy with Intramuscular Avonex and Oral CellCept in Patients with Multiple Sclerosis

ClinicalTrials.gov, 12 Feb 2009

Accessed on 6 Jan 2012 from http://clinicaltrials.gov/ct2/show/NCT00223301.

Status/Outcome:

No differences were observed between experimental and control groups in terms of patient-reported adverse events, MRI metrics, or laboratory abnormalities; there was a trend toward a therapeutic effect

(7)

Remington GM, Treadaway K, Frohman T, Salter A, Stüve O, Racke MK, Hawker K, Agosta F, Sormani M P, Filippi M, et al.

Ther Adv Neurol Disord. 2010 Jan; 3(1):3-13. PMID: 21180632. Abstract

(8)

Safety Study of Combination Therapy with Intramuscular Avonex and Oral CellCept in Patients with Multiple Sclerosis

ClinicalTrials.gov, 12 Feb 2009

Accessed on 6 Jan 2012 from http://clinicaltrials.gov/ct2/show/NCT00223301.

Head-to-Head Trials:

Trial name:

Frohman et al., Ther. Adv. Neurol. Disord., January 2010 study

(10)

A randomized, blinded, parallel-group, pilot trial of mycophenolate mofetil (CellCept) compared with interferon beta-1a (Avonex) in patients with relapsing-remitting multiple sclerosis.

Frohman EM, Cutter G, Remington G, Gao H, Rossman H, Weinstock-Guttman B, Durfee JE, Conger A, Carl E, Treadaway K, et al.

Ther Adv Neurol Disord. 2010 Jan; 3(1):15-28. PMID: 21180633. Abstract

Phase:

Pilot study

(10)

A randomized, blinded, parallel-group, pilot trial of mycophenolate mofetil (CellCept) compared with interferon beta-1a (Avonex) in patients with relapsing-remitting multiple sclerosis.

Frohman EM, Cutter G, Remington G, Gao H, Rossman H, Weinstock-Guttman B, Durfee JE, Conger A, Carl E, Treadaway K, et al.

Ther Adv Neurol Disord. 2010 Jan; 3(1):15-28. PMID: 21180633. Abstract

Study Design:

Randomized, blinded, parallel-group, multicenter pilot trial comparing the reduction of cumulative mean number of lesions (primary outcome measure) promoted by CellCept versus interferon beta-1a

(10)

A randomized, blinded, parallel-group, pilot trial of mycophenolate mofetil (CellCept) compared with interferon beta-1a (Avonex) in patients with relapsing-remitting multiple sclerosis.

Frohman EM, Cutter G, Remington G, Gao H, Rossman H, Weinstock-Guttman B, Durfee JE, Conger A, Carl E, Treadaway K, et al.

Ther Adv Neurol Disord. 2010 Jan; 3(1):15-28. PMID: 21180633. Abstract

Disease Stage:

RRMS

(10)

A randomized, blinded, parallel-group, pilot trial of mycophenolate mofetil (CellCept) compared with interferon beta-1a (Avonex) in patients with relapsing-remitting multiple sclerosis.

Frohman EM, Cutter G, Remington G, Gao H, Rossman H, Weinstock-Guttman B, Durfee JE, Conger A, Carl E, Treadaway K, et al.

Ther Adv Neurol Disord. 2010 Jan; 3(1):15-28. PMID: 21180633. Abstract

Enrollment/Number of Patients:

Enrolled 35 previously untreated patients; 31 completed the trial

(10)

A randomized, blinded, parallel-group, pilot trial of mycophenolate mofetil (CellCept) compared with interferon beta-1a (Avonex) in patients with relapsing-remitting multiple sclerosis.

Frohman EM, Cutter G, Remington G, Gao H, Rossman H, Weinstock-Guttman B, Durfee JE, Conger A, Carl E, Treadaway K, et al.

Ther Adv Neurol Disord. 2010 Jan; 3(1):15-28. PMID: 21180633. Abstract

Duration:

6 months

(10)

A randomized, blinded, parallel-group, pilot trial of mycophenolate mofetil (CellCept) compared with interferon beta-1a (Avonex) in patients with relapsing-remitting multiple sclerosis.

Frohman EM, Cutter G, Remington G, Gao H, Rossman H, Weinstock-Guttman B, Durfee JE, Conger A, Carl E, Treadaway K, et al.

Ther Adv Neurol Disord. 2010 Jan; 3(1):15-28. PMID: 21180633. Abstract

Status/Outcome:

Patients treated with CellCept showed a trend towards a lower accumulation of lesions compared to those treated with interferon beta-1a

(10)

A randomized, blinded, parallel-group, pilot trial of mycophenolate mofetil (CellCept) compared with interferon beta-1a (Avonex) in patients with relapsing-remitting multiple sclerosis.

Frohman EM, Cutter G, Remington G, Gao H, Rossman H, Weinstock-Guttman B, Durfee JE, Conger A, Carl E, Treadaway K, et al.

Ther Adv Neurol Disord. 2010 Jan; 3(1):15-28. PMID: 21180633. Abstract

Other Trials:

Trial name:

Pandit et al., Neurol. India, November-December 2014 study

(19)

Mycophenolate mofetil in the treatment of multiple sclerosis: A preliminary report.

Pandit L, Mustafa S, Malli C, D'Cunha A

Neurol India. 2014 Nov-Dec; 62(6):646-8. PMID: 25591678. Abstract

Phase:

Retrospective disease registry review

(19)

Mycophenolate mofetil in the treatment of multiple sclerosis: A preliminary report.

Pandit L, Mustafa S, Malli C, D'Cunha A

Neurol India. 2014 Nov-Dec; 62(6):646-8. PMID: 25591678. Abstract

Study Design:

Study to examine the effects of mycophenolate mofetil on the annualized relapse rate (ARR) and the Expanded Disability Status Scale (EDSS) score; ARRs were calculated for the year prior to beginning mycophenolate mofetil and the year after

(19)

Mycophenolate mofetil in the treatment of multiple sclerosis: A preliminary report.

Pandit L, Mustafa S, Malli C, D'Cunha A

Neurol India. 2014 Nov-Dec; 62(6):646-8. PMID: 25591678. Abstract

Disease Stage:

(19)

Mycophenolate mofetil in the treatment of multiple sclerosis: A preliminary report.

Pandit L, Mustafa S, Malli C, D'Cunha A

Neurol India. 2014 Nov-Dec; 62(6):646-8. PMID: 25591678. Abstract

Enrollment/Number of Patients:

(19)

Mycophenolate mofetil in the treatment of multiple sclerosis: A preliminary report.

Pandit L, Mustafa S, Malli C, D'Cunha A

Neurol India. 2014 Nov-Dec; 62(6):646-8. PMID: 25591678. Abstract

Duration:

24 months (mean duration of mycophenolate mofetil therapy)

(19)

Mycophenolate mofetil in the treatment of multiple sclerosis: A preliminary report.

Pandit L, Mustafa S, Malli C, D'Cunha A

Neurol India. 2014 Nov-Dec; 62(6):646-8. PMID: 25591678. Abstract

Status/Outcome:

Treatment was associated with a reduced mean ARR (0.11) as compared with the pretreatment mean ARR (0.95) and a reduced EDSS score (2.66) as compared with the pretreatment score (3.80)

(19)

Mycophenolate mofetil in the treatment of multiple sclerosis: A preliminary report.

Pandit L, Mustafa S, Malli C, D'Cunha A

Neurol India. 2014 Nov-Dec; 62(6):646-8. PMID: 25591678. Abstract

Trial name:

Xiao et al., Cochrane Database Syst. Rev., February 2014 study

(15)

Mycophenolate mofetil for relapsing-remitting multiple sclerosis.

Xiao Y, Huang J, Luo H, Wang J

Cochrane Database Syst Rev. 2014 Feb 7; 2:CD010242. PMID: 24505016. Abstract

Phase:

Retrospective clinical trial review

(15)

Mycophenolate mofetil for relapsing-remitting multiple sclerosis.

Xiao Y, Huang J, Luo H, Wang J

Cochrane Database Syst Rev. 2014 Feb 7; 2:CD010242. PMID: 24505016. Abstract

Study Design:

Analysis of randomized controlled trials that (i) compared mycophenolate mofetil (MMF), either alone or in combination with other therapies, to placebo or another drug and (ii) involved followup for a minimum of 12 months; the purpose was to examine the effectiveness of MMF in preventing disease activity

(15)

Mycophenolate mofetil for relapsing-remitting multiple sclerosis.

Xiao Y, Huang J, Luo H, Wang J

Cochrane Database Syst Rev. 2014 Feb 7; 2:CD010242. PMID: 24505016. Abstract

Disease Stage:

RRMS

(15)

Mycophenolate mofetil for relapsing-remitting multiple sclerosis.

Xiao Y, Huang J, Luo H, Wang J

Cochrane Database Syst Rev. 2014 Feb 7; 2:CD010242. PMID: 24505016. Abstract

Enrollment/Number of Patients:

(15)

Mycophenolate mofetil for relapsing-remitting multiple sclerosis.

Xiao Y, Huang J, Luo H, Wang J

Cochrane Database Syst Rev. 2014 Feb 7; 2:CD010242. PMID: 24505016. Abstract

Duration:

N/A

(15)

Mycophenolate mofetil for relapsing-remitting multiple sclerosis.

Xiao Y, Huang J, Luo H, Wang J

Cochrane Database Syst Rev. 2014 Feb 7; 2:CD010242. PMID: 24505016. Abstract

Status/Outcome:

Analysis concluded that one study that was included, which examined the effects of MMF versus placebo in participants treated with interferon beta-1a, had a high risk of bias and too few participants and too short a duration to determine the effects of MMF as an add-on therapy on RRMS

(15)

Mycophenolate mofetil for relapsing-remitting multiple sclerosis.

Xiao Y, Huang J, Luo H, Wang J

Cochrane Database Syst Rev. 2014 Feb 7; 2:CD010242. PMID: 24505016. Abstract

Trial name:

Michel et al., J. Neurol. Neurosurg. Psychiatry, May 2013 study

(13)

Mycophenolate mofetil in multiple sclerosis: a multicentre retrospective study on 344 patients.

Michel L, Vukusic S, de Seze J, Ducray F, Ongagna JC, Lefrère F, Jacq-Foucher M, Confavreux C, Wiertlewski S, Laplaud DA

J Neurol Neurosurg Psychiatry. 2013 May 23. PMID: 23704316. Abstract

; ECTRIMS abstract, 2011

(11)

Mycophenolate Mofetil as a Monotherapy in Multiple Sclerosis: a multicentre retrospective study on 361 patients

ECTRIMS 2011 abstract P480

Accessed on 6 Jan 2012 from http://www.posters2view.com/ECTRIMS2011/view.php?nu=393.

Phase:

Retrospective medical record review

(11)

Mycophenolate Mofetil as a Monotherapy in Multiple Sclerosis: a multicentre retrospective study on 361 patients

ECTRIMS 2011 abstract P480

Accessed on 6 Jan 2012 from http://www.posters2view.com/ECTRIMS2011/view.php?nu=393.

(13)

Mycophenolate mofetil in multiple sclerosis: a multicentre retrospective study on 344 patients.

Michel L, Vukusic S, de Seze J, Ducray F, Ongagna JC, Lefrère F, Jacq-Foucher M, Confavreux C, Wiertlewski S, Laplaud DA

J Neurol Neurosurg Psychiatry. 2013 May 23. PMID: 23704316. Abstract

Study Design:

Multicenter, retrospective study of all MS patients treated with mycophenolate mofetil at three French MS centers, including patients who had previously received other immunosuppressive drugs and those who had not, to determine the drug’s effects as a monotherapy on the annualized relapse rate (ARR) and the Expanded Disability Status Scale (EDSS) score

(11)

Mycophenolate Mofetil as a Monotherapy in Multiple Sclerosis: a multicentre retrospective study on 361 patients

ECTRIMS 2011 abstract P480

Accessed on 6 Jan 2012 from http://www.posters2view.com/ECTRIMS2011/view.php?nu=393.

(13)

Mycophenolate mofetil in multiple sclerosis: a multicentre retrospective study on 344 patients.

Michel L, Vukusic S, de Seze J, Ducray F, Ongagna JC, Lefrère F, Jacq-Foucher M, Confavreux C, Wiertlewski S, Laplaud DA

J Neurol Neurosurg Psychiatry. 2013 May 23. PMID: 23704316. Abstract

Disease Stage:

(11)

Mycophenolate Mofetil as a Monotherapy in Multiple Sclerosis: a multicentre retrospective study on 361 patients

ECTRIMS 2011 abstract P480

Accessed on 6 Jan 2012 from http://www.posters2view.com/ECTRIMS2011/view.php?nu=393.

(13)

Mycophenolate mofetil in multiple sclerosis: a multicentre retrospective study on 344 patients.

Michel L, Vukusic S, de Seze J, Ducray F, Ongagna JC, Lefrère F, Jacq-Foucher M, Confavreux C, Wiertlewski S, Laplaud DA

J Neurol Neurosurg Psychiatry. 2013 May 23. PMID: 23704316. Abstract

Enrollment/Number of Patients:

344

(13)

Mycophenolate mofetil in multiple sclerosis: a multicentre retrospective study on 344 patients.

Michel L, Vukusic S, de Seze J, Ducray F, Ongagna JC, Lefrère F, Jacq-Foucher M, Confavreux C, Wiertlewski S, Laplaud DA

J Neurol Neurosurg Psychiatry. 2013 May 23. PMID: 23704316. Abstract

Duration:

2 years (1 year control period as compared with the 1 year after the onset of mycophenolate mofetil treatment)

(13)

Mycophenolate mofetil in multiple sclerosis: a multicentre retrospective study on 344 patients.

Michel L, Vukusic S, de Seze J, Ducray F, Ongagna JC, Lefrère F, Jacq-Foucher M, Confavreux C, Wiertlewski S, Laplaud DA

J Neurol Neurosurg Psychiatry. 2013 May 23. PMID: 23704316. Abstract

Status/Outcome:

Treatment with mycophenolate mofetil was associated with a reduction of ARR (from 1.11 in the control period to 0.35 in the treatment period) and stable EDSS scores; 11% of patients experienced adverse events

(13)

Mycophenolate mofetil in multiple sclerosis: a multicentre retrospective study on 344 patients.

Michel L, Vukusic S, de Seze J, Ducray F, Ongagna JC, Lefrère F, Jacq-Foucher M, Confavreux C, Wiertlewski S, Laplaud DA

J Neurol Neurosurg Psychiatry. 2013 May 23. PMID: 23704316. Abstract

Trial name:

Vermersch et al., Eur. J Neurol., January 2007 study

(12)

Combination of IFN beta-1a (Avonex) and mycophenolate mofetil (Cellcept) in multiple sclerosis.

Vermersch P, Waucquier N, Michelin E, Bourteel H, Stojkovic T, Ferriby D, de Seze J, G-SEP(Groupe septentrional d'études et de prise en charge de la sclérose en plaques)

Eur J Neurol. 2007 Jan; 14(1):85-9. PMID: 17222119. Abstract

Phase:

Pilot study

(12)

Combination of IFN beta-1a (Avonex) and mycophenolate mofetil (Cellcept) in multiple sclerosis.

Vermersch P, Waucquier N, Michelin E, Bourteel H, Stojkovic T, Ferriby D, de Seze J, G-SEP(Groupe septentrional d'études et de prise en charge de la sclérose en plaques)

Eur J Neurol. 2007 Jan; 14(1):85-9. PMID: 17222119. Abstract

Study Design:

Company-sponsored, open-label, single-center, non-randomized pilot study examining the effect on disease activity as measured by MRI (primary endpoint) and safety and tolerance of drug combined with interferon beta-1a

(12)

Combination of IFN beta-1a (Avonex) and mycophenolate mofetil (Cellcept) in multiple sclerosis.

Vermersch P, Waucquier N, Michelin E, Bourteel H, Stojkovic T, Ferriby D, de Seze J, G-SEP(Groupe septentrional d'études et de prise en charge de la sclérose en plaques)

Eur J Neurol. 2007 Jan; 14(1):85-9. PMID: 17222119. Abstract

Disease Stage:

RRMS

(12)

Combination of IFN beta-1a (Avonex) and mycophenolate mofetil (Cellcept) in multiple sclerosis.

Vermersch P, Waucquier N, Michelin E, Bourteel H, Stojkovic T, Ferriby D, de Seze J, G-SEP(Groupe septentrional d'études et de prise en charge de la sclérose en plaques)

Eur J Neurol. 2007 Jan; 14(1):85-9. PMID: 17222119. Abstract

Enrollment/Number of Patients:

30 enrolled, 28 completed study

(12)

Combination of IFN beta-1a (Avonex) and mycophenolate mofetil (Cellcept) in multiple sclerosis.

Vermersch P, Waucquier N, Michelin E, Bourteel H, Stojkovic T, Ferriby D, de Seze J, G-SEP(Groupe septentrional d'études et de prise en charge de la sclérose en plaques)

Eur J Neurol. 2007 Jan; 14(1):85-9. PMID: 17222119. Abstract

Duration:

6 months

(12)

Combination of IFN beta-1a (Avonex) and mycophenolate mofetil (Cellcept) in multiple sclerosis.

Vermersch P, Waucquier N, Michelin E, Bourteel H, Stojkovic T, Ferriby D, de Seze J, G-SEP(Groupe septentrional d'études et de prise en charge de la sclérose en plaques)

Eur J Neurol. 2007 Jan; 14(1):85-9. PMID: 17222119. Abstract

Status/Outcome:

No significant adverse events; drug caused a decrease in Gadolinium-enhanced lesions, annualized relapse rate, and disability score

(12)

Combination of IFN beta-1a (Avonex) and mycophenolate mofetil (Cellcept) in multiple sclerosis.

Vermersch P, Waucquier N, Michelin E, Bourteel H, Stojkovic T, Ferriby D, de Seze J, G-SEP(Groupe septentrional d'études et de prise en charge de la sclérose en plaques)

Eur J Neurol. 2007 Jan; 14(1):85-9. PMID: 17222119. Abstract

Trial name:

Frohman et al., Clin. Neuropharm., March-April 2004 study

(3)

Frohman EM, Brannon K, Racke MK, Hawker K

Clin Neuropharmacol. 2004 Mar-Apr; 27(2):80-3. PMID: 15252268. Abstract

Phase:

Retrospective medical record review

(3)

Frohman EM, Brannon K, Racke MK, Hawker K

Clin Neuropharmacol. 2004 Mar-Apr; 27(2):80-3. PMID: 15252268. Abstract

Study Design:

Retrospective surveillance study

(3)

Frohman EM, Brannon K, Racke MK, Hawker K

Clin Neuropharmacol. 2004 Mar-Apr; 27(2):80-3. PMID: 15252268. Abstract

Disease Stage:

61 patients with SPMS, 14 with RRMS, 4 with PPMS

(3)

Frohman EM, Brannon K, Racke MK, Hawker K

Clin Neuropharmacol. 2004 Mar-Apr; 27(2):80-3. PMID: 15252268. Abstract

Enrollment/Number of Patients:

Followed 79 patients (44 already on interferon beta, 20 already on glatiramer acetate, 15 on monotherapy); 22 stopped treatment due to side effects, continued disease progression, or lack of insurance coverage

(3)

Frohman EM, Brannon K, Racke MK, Hawker K

Clin Neuropharmacol. 2004 Mar-Apr; 27(2):80-3. PMID: 15252268. Abstract

Duration:

1 year

(3)

Frohman EM, Brannon K, Racke MK, Hawker K

Clin Neuropharmacol. 2004 Mar-Apr; 27(2):80-3. PMID: 15252268. Abstract

Status/Outcome:

Treatment appeared to be well-tolerated and several patients seemed to stabilize or improve

(3)

Frohman EM, Brannon K, Racke MK, Hawker K

Clin Neuropharmacol. 2004 Mar-Apr; 27(2):80-3. PMID: 15252268. Abstract

References

Roche/Food and Drug Administration

Accessed on 5 Jan 2012 from http://www.accessdata.fda.gov/drugsatfda_docs/label/2009/050722s021,050723s019,050758s019,050759s024lbl.pdf.

United States National Library of Medicine

Accessed on 29 Jul 2013 from http://chem.sis.nlm.nih.gov/chemidplus/chemidheavy.jsp.

Note: Compound name must be entered under "Substance Identification" and then "Names and Synonyms" selected to view synonyms.

USPTO list of patent term extensions

Frohman EM, Brannon K, Racke MK, Hawker K

Clin Neuropharmacol. 2004 Mar-Apr; 27(2):80-3. PMID: 15252268. Abstract

USPTO

Accessed on 6 Jan 2012 from http://www.uspto.gov/patents/resources/terms/156.jsp.

Treatment of neuromyelitis optica with mycophenolate mofetil: retrospective analysis of 24 patients.

Jacob A, Matiello M, Weinshenker BG, Wingerchuk DM, Lucchinetti C, Shuster E, Carter J, Keegan MB, Kantarci OH, Pittock SJ

Arch Neurol. 2009 Sep; 66(9):1128-33. PMID: 19752302. Abstract

Mycophenolic acid inhibits IL-2-dependent T cell proliferation, but not IL-2-dependent survival and sensitization to apoptosis.

Quéméneur L, Flacher M, Gerland L-M, Ffrench M, Revillard J-P, Bonnefoy-Berard N

J Immunol. 2002 Sep 1; 169(5):2747-55. PMID: 12193749. Abstract

Remington GM, Treadaway K, Frohman T, Salter A, Stüve O, Racke MK, Hawker K, Agosta F, Sormani M P, Filippi M, et al.

Ther Adv Neurol Disord. 2010 Jan; 3(1):3-13. PMID: 21180632. Abstract

Safety Study of Combination Therapy with Intramuscular Avonex and Oral CellCept in Patients with Multiple Sclerosis

ClinicalTrials.gov, 12 Feb 2009

Accessed on 6 Jan 2012 from http://clinicaltrials.gov/ct2/show/NCT00223301.