CLINICAL TRIALS OF POTENTIAL DISEASE-MODIFYING TREATMENTS FOR MSA

These experimental therapies are being tested in MSA patients in approved clinical trials in order to evaluate whether they can alter the course of the disease. Scroll down and click on each therapy to learn more including links to opportunities to enroll in clinical trials in progress.

Please be sure to check back regularly to find out what’s new in the Multiple System Atrophy Treatment Pipeline and remember to Sign up for our newsletter to stay informed of the latest updates and clinical trial opportunities.

**NEW** October 19, 2021: Alterity Therapeutics Announces Expanded ATH434 Phase 2 Clinical Development Program

**NEW** September 29, 2021: Florey Institute of Neuroscience and Mental Health publishes research funded by The Multiple System Atrophy Coalition

September 27, 2021: Biohaven Provides Update On Phase 3 Trial And Multiple System Atrophy (MSA) Program

September 23, 2021: Inhibikase Therapeutics Receives Grant from U.S. National Institutes of Health to Evaluate IkT-148009 for the Treatment of Multiple System Atrophy

September 22, 2021: ICBII Announces Approval of Its 7th Patent on Blood-Brain Barrier Permeable Technology, Moving Closer to Clinical Trials on its drugs for Alzheimer’s, Parkinson’s, and Other Neuro-Degenerative Diseases

July 27, 2021: AC Immune Announces Strategic Acquisition of Industry-leading Parkinson’s Disease Vaccine Candidate

July 15, 2021: New Publication Demonstrates ATH434 is Neuroprotective

July 8, 2021: ATH434 Reduces α-Synuclein-Related Neurodegeneration in a Murine Model of Multiple System Atrophy

July 1, 2021: Alterity Therapeutics granted a new US patent targeting major neurodegenerative diseases including Alzheimer’s and Parkinson’s

June 23, 2021: European Union Regulatory Guidance for ATH434 Phase 2 Clinical Trial

June 11, 2021: Updated Q&A on Verdiperstat

May, 2021: Updates on Biohaven’s study to evaluate the efficacy and safety of BHV- 3241 (Verdiperstat)

April 21, 2021: ATH434 protects brain cells and improves motor function in Parkinsonian disorder

February 5, 2021: Biohaven Shares Verdiperstat’s Mechanism of Action

December 23, 2020: MODAG Initiates First-in-Patient Phase 1b Trial for Anle138b in Parkinson’s Disease

December 3, 2020: Inhibikase Advances Development Program for Multiple System Atrophy (MSA) Based on Demonstration of Key Role of c-Abl Kinase in MSA Neurodegeneration

November 16, 2020: Alterity Announces Approval of US Patent for Next Generation Compounds to Treat Neurodegenerative Diseases

November 6, 2020: M-STAR, an Ongoing Phase 3 Study in Participants with Multiple System Atrophy–Baseline Characteristics

October 27, 2020: Alterity commences enrolling Multiple System Atrophy patients in bioMUSE Study

October 16, 2020: IXICO and NYU Langone Health Sign Agreement to Develop Novel Imaging Markers in Multiple System Atrophy

September 24, 2020: Targeting α-synuclein by PD03 AFFITOPE® and Anle138b rescues neurodegenerative pathology in a model of multiple system atrophy: clinical relevance

September 3, 2020: A Phase 1 Randomized Trial of Specific Active α‐Synuclein Immunotherapies PD01A and PD03A in Multiple System Atrophy

August 5, 2020: MODAG Successfully Completes Phase 1 Study of their Lead Candidate Anle138b and Receives Additional USD 1.4 Million from Michael J. Fox Foundation

August 4, 2020: New data independently confirms and extends laboratory findings and expands safety profile of ATH434 (PBT-434)

July 21, 2020: Biohaven Completes Enrollment Ahead of Timelines in International Phase 3 Clinical Trial of Verdiperstat in Multiple System Atrophy

July 1, 2020: Alterity Therapeutics meeting with US FDA provides development pathway for ATH434 (PBT-434)

April 30, 2020: MSA Coalition Blog – Caution When it Comes to Stem Cells for MSA

April 16, 2020: MSA Coalition Blog – Q & A about Verdiperstat Clinical Trial

March 18, 2020: Biohaven’s Verdiperstat Receives Fast Track Designation for the Treatment of Multiple System Atrophy

January 14, 2020: European Commission approves Orphan Designation for Alterity’s lead drug candidate (PBT-434)

December 18, 2019: MODAG Initiates First-in-Human Phase 1 Clinical Trial for Anle138b

October 25, 2019: Research Speed Ahead – 1 Year Update from the Sirolimus MSA Trial

October 5, 2019: A vaccine for Parkinson’s disease shows promising results (PD01A)

August 2, 2019: Green Tea Extract Shows No Significant Benefit for Multiple System Atrophy (EGCG)

July 31, 2019: Biohaven Enrolls First Patient in Phase 3 Clinical Trial of Verdiperstat, Oral Myeloperoxidase Inhibitor, for the Treatment of Multiple System Atrophy

July 29, 2019: Alterity Therapeutics Announces Successful Completion of Phase 1 Clinical Trial (PBT-434)

July 9, 2019: VIDEO: Alterity Therapeutics Limited CEO & Chairman, Geoffrey Kempler, discusses the company’s development pipeline and lead drug candidate PBT434 for use treating Multiple System Atrophy (MSA).

July 2, 2019: Safety and efficacy of epigallocatechin gallate in multiple system atrophy (PROMESA): a randomised, double-blind, placebo-controlled trial

June 27, 2019: Modag to Develop Treatments for Parkinsonian Disorders, including Multiple System Atrophy (Anle138b)

June 1, 2019: Biohaven Pharmaceuticals announces new Phase 3 Clinical Trial of Verdiperstat (BHV-3241) for Multiple System Atrophy patients. Learn more about the study.

CURRENT MSA TRIALS

The below experimental therapies are currently being tested in MSA Patients. Click on each experimental therapy to learn more details, including links to clinical trial opportunities.

VERDIPERSTAT (BHV-3241) - Phase 3 Clinical Trial - no longer recruiting ***

** NEW ** Preclinical work funded by the MSA Coalition

Grant: “Verdiperstat target engagement in a unique multiple system atrophy postmortem brain matched to iPSC”
Awardee: Vikram Khurana, MD PhD
MSA Coalition Grant #2021-09-006 – $50,000

A Phase 3 clinical trial in MSA patients is no longer accepting patients.

For Historical Phase 3 clinical trials details See: https://clinicaltrials.gov/ct2/show/NCT03952806 and https://www.msaresearchstudy.com/about-the-study

Verdiperstat is an oral myeloperoxidase (MPO) inhibitor. MPO is a key driver of oxidative and inflammatory processes and is significantly increased in a range of brain disorders. It is thought that inhibiting MPO activity may be a promising therapeutic strategy for neuroinflammatory and neurodegenerative conditions, including MSA.

A Phase 2 clinical trial of MSA patients was previously completed. For historical Phase 2 clinical trial details See: https://clinicaltrials.gov/ct2/show/NCT02388295

Related Videos:

May 2021: Biohaven Video: MSA MPO Inhibition

Related Posters:

May 2021: Updates on Biohaven’s study to evaluate the efficacy and safety of BHV- 3241 (Verdiperstat)

February 2021: Biohaven Shares Verdiperstat’s Mechanism of Action

November 2020: M-STAR, an Ongoing Phase 3 Study in Participants with Multiple System Atrophy–Baseline Characteristics (Verdiperstat)

Related Articles:

September 27, 2021: Biohaven Provides Update On Phase 3 Trial And Multiple System Atrophy (MSA) Program

September 15, 2021:Theravance Biopharma, Inc. announces top-line results from a Phase 3 study of Ampreloxetine in patients with symptomatic neurogenic orthostatic hypotension

June 2021: Updated Q&A on Verdiperstat

May 2021: Updates on Biohaven’s study to evaluate the efficacy and safety of BHV- 3241 (Verdiperstat)

April 2021: ATH434 protects brain cells and improves motor function in Parkinsonian disorder

February 2021: Biohaven Shares Verdiperstat’s Mechanism of Action

November 2020: M-STAR, an Ongoing Phase 3 Study in Participants with Multiple System Atrophy–Baseline Characteristics

July 2020: Biohaven Completes Enrollment Ahead of Timelines in International Phase 3 Clinical Trial of Verdiperstat in Multiple System Atrophy

April 2020: MSA Coalition Blog – Q & A about Verdiperstat Clinical Trial

March 2020: Biohaven’s Verdiperstat Receives Fast Track Designation for the Treatment of Multiple System Atrophy

July 2019: Biohaven Enrolls First Patient in Phase 3 Clinical Trial of Verdiperstat, Oral Myeloperoxidase Inhibitor, for the Treatment of Multiple System Atrophy

May 2019: Phase 3 clinical trial of Verdiperstat, an oral myeloperoxidase inhibitor, in multiple system atrophy will begin enrollment in third quarter of 2019

January 2019: Biohaven Receives FDA May Proceed Letter For Phase 3 Clinical Trial Of BHV-3241 For Multiple System Atrophy

September 2018: Biohaven Licenses Novel Myeloperoxidase Inhibitor From AstraZeneca: Potential First-In-Class Treatment For Multiple System Atrophy

July 2015: Failure of Neuroprotection Despite Microglial Suppression by Delayed-Start Myeloperoxidase Inhibition in a Model of Advanced Multiple System Atrophy: Clinical Implications

May 2012: Myeloperoxidase inhibition ameliorates multiple system atrophy-like degeneration in a transgenic mouse model.

NOTE: All of the above information is intended to assist MSA patients and their families in having a conversation with doctors, none of it should be considered medical advice or endorsements of any particular drugs or therapies. Always consult a licensed medical practitioner for expert care.

Tllsh2910 - Phase 3 Clinicial Trial in Progress ***

A Phase 3 clinical trial in MSA patients is in progress.

For Phase 3 clinical trial details See: https://www.clinicaltrials.gov/ct2/show/NCT03901638

Cerebellar NMDA (N-methyl-D-aspartate) receptors play a considerable role in motor learning and coordination, thus, may be a feasible target for treating ataxia. Tllsh2910, a NMDA modulator, has been found to attenuate the ataxic gait in the mouse model.

Related Articles:

NOTE: All of the above information is intended to assist MSA patients and their families in having a conversation with doctors, none of it should be considered medical advice or endorsements of any particular drugs or therapies. Always consult a licensed medical practitioner for expert care.

EXENDIN-4(EXENATIDE) - Phase 2 Clinical Trial in Progress **

** Preclinical work funded by the MSA Coalition **

Laboratory work to test Exendin-4 in MSA experimental mouse models was previously carried out at Bordeaux University, France. This project was funded by the MSA Coalition in 2015-2016 and showed positive results, paving the way for MSA patient trials.

Exendin-4 (Exenatide) is an FDA approved drug for treating Diabetes.

A Phase 2 pilot study to test Exendin-4 in MSA patients is in progress at the University College London. For more details see: https://clinicaltrials.gov/ct2/show/NCT04431713

Related Articles:

November 2019: Researchers begin trial of drug to slow progression of neurodegenerative condition Multiple System Atrophy

May 2017: Insulin resistance and exendin-4 treatment for multiple system atrophy

March 2017: Report on MSA Coalition funded research Exendin-4

June 2016: Glucagon-like peptide 1 analogues for treating multiple system atrophy: A translational study


NOTE: All of the above information is intended to assist MSA patients and their families in having a conversation with doctors, none of it should be considered medical advice or endorsements of any particular drugs or therapies. Always consult a licensed medical practitioner for expert care.

COQ10 (Coenzyme Q10) - Phase 2 Clinical Trial - no longer recruiting **

A Phase 2 clinical trial in MSA patients is currently underway.
For Phase 2 clinical trial details See: https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000036134

High dose CoQ10 is currently being evaluated in MSA patients in a trial in Japan.

Related Articles:

June 2017: Three-year follow-up of high-dose ubiquinol supplementation in a case of familial multiple system atrophy with compound heterozygous COQ2 mutations

Feb 2017: The efficacy and safety of coenzyme Q10 in Parkinson’s disease: a meta-analysis of randomized controlled trials.

May 2016: Decreased Coenzyme Q10 Levels in Multiple System Atrophy Cerebellum

May 2014: A randomized clinical trial of high-dosage coenzyme Q10 in early Parkinson disease: no evidence of benefit.

July 2013: Mutations in COQ2 in Familial and Sporadic Multiple-System Atrophy

NOTE: All of the above information is intended to assist MSA patients and their families in having a conversation with doctors, none of it should be considered medical advice or endorsements of any particular drugs or therapies. Always consult a licensed medical practitioner for expert care.

NBMI - Phase 2 Completed 2021

A Phase 2 clinical trial in MSA patients is complete.

For historical Phase 2 clinical trials details See: https://www.clinicaltrials.gov/ct2/show/NCT04184063

NBMI (N1, N3-Bis-(2-Mercaptoethyl) Isophthalamide) is a new metal chelator drug proposed as an alternative to the current chelators, and it is widely different; compared to the current chelators, consisting of two cysteamine molecules coupled to a single molecule of dicarboxybenzoate. It is used as a chelating agent and has the designation of an orphan drug, in the EU and USA; in the EU it is used for the treatment of mercury toxicity. It is freely soluble in solutions of dimethylformamide (DMF), dimethyl sulfoxide (DMSO) and sodium hydroxide diluted NaOH, slightly soluble in methanol and acetone, and insoluble in water. Pre-clinical data indicates low to no toxicity, and that it reduces the toxicity associated with acute exposure to Hg2+.

No other chelator has been reported to prevent acute mercury toxicity with only one exposure to the chelator. It has the ability to penetrate cell membranes and cross the blood-brain barrier and chelate Hg2+ in a complex that eliminates the availability of Hg2+ and essentially eliminates toxic effects. The antioxidant properties of NBMI could also reduce the toxicity levels of hydroxyl free radicals immediately, upon entering cells suffering from oxidative stress. It is possible that the combined chelation of Hg2+ and the elimination of hydroxyl free radicals contribute significantly to the protective effects observed with the NBMI.

Previous clinical studies conducted in subjects of the Phase I and Phase II a studies conducted, did not show significant adverse events in patients intoxicated with mercury, all patients who received the study medication have tolerated it well, with only mild or moderate adverse events reported; None of these were considered related to the pharmacological treatment of the study. In addition, there is no potential identified with safety problems in laboratory tests, or vital signs evaluations.

Related Articles:

NOTE: All of the above information is intended to assist MSA patients and their families in having a conversation with doctors, none of it should be considered medical advice or endorsements of any particular drugs or therapies. Always consult a licensed medical practitioner for expert care.

Rituximab - Phase 2 Clinical Trial in Progress **

An ongoing phase 2 study evaluating the efficacy of rituximab in MSA-C patients will shed light on the effect of B-cell inhibition in the context of oligodendroglial α-syn aggregation. For more details see: https://clinicaltrials.gov/ct2/show/NCT04004819

The CD20 antibody rituximab, which inhibits B-cell activation, is already approved for autoimmune diseases such as Multiple Scleroisis or rheumatoid arthritis. In a rodent spinal cord injury model rituximab reduced the expression of TNFα in neuronal and glial cells. However, it remains unclear whether rituximab affects the production of naturally occurring autoantibodies possibly playing a crucial role in blocking pathological proteins such as α-syn.

Related Articles:

NOTE: All of the above information is intended to assist MSA patients and their families in having a conversation with doctors, none of it should be considered medical advice or endorsements of any particular drugs or therapies. Always consult a licensed medical practitioner for expert care.

MSC (Mesenchymal Stem Cells) - Phase 1 Clinical Trial - no longer recruiting*

A Phase 1 clinical trial in MSA patients is in progress in the USA. See: https://www.clinicaltrials.gov/ct2/show/NCT02315027 and Mesenchymal Stem Cell Therapy in Multiple System Atrophy

US investigators are hoping to open a larger multi-center placebo-controlled Phase 2/3 clinical trial in the future.

A Phase 2 clinical trial of autologous mesenchymal stem cells – intra arterial infusion in MSA – was previously completed in Korea. For historical Phase 2 clinical trial details See: https://www.clinicaltrials.gov/ct2/show/NCT00911365

A Phase 1 clinical trial of CS10BR05 – injection in the carotid artery is underway in Korea. For more details see: https://clinicaltrials.gov/ct2/show/NCT03265444

Related Videos:

April 2017: VIDEO: Pilot stem cell trial for multiple system atrophy shows promising results

Related Articles:

May 2019: Intrathecal administration of autologous mesenchymal stem cells in multiple system atrophy.

June 2017: Intrathecal Administration of Autologous Mesenchymal Stem Cells in Multiple System Atrophy – A Phase I/II Dose-Escalation Trial

May 2017: Feasibility and Efficacy of Intra‐Arterial Administration of Mesenchymal Stem Cells in an Animal Model of Double Toxin‐Induced Multiple System Atrophy

July 2012: A randomized trial of mesenchymal stem cells in multiple system atrophy.

NOTE: All of the above information is intended to assist MSA patients and their families in having a conversation with doctors, none of it should be considered medical advice or endorsements of any particular drugs or therapies. Always consult a licensed medical practitioner for expert care.

ATH434/PBT434 - Phase 2 Clinical Trial in Progress **

A Phase 2 clinical trial is in progress. Link to enrolment information coming soon.

A Phase 1 clinical trial in healthy volunteers was previously completed.

For historical Phase 1 clinical trial details See: https://www.anzctr.org.au/Trial/Registration/TrialReview.aspx?id=374741

ATH434 (formerly called PBT434) is a small molecule designed to block the accumulation and aggregation of the protein alpha-synclein.

Related Video:

July 2019: VIDEO: Alterity Therapeutics Limited CEO & Chairman, Geoffrey Kempler, discusses the company’s development pipeline and lead drug candidate PBT434 for use treating Multiple System Atrophy (MSA).

Related Slide Presentations:

June 2019: Alterity Therapeutics Investor Presentation

May 2019: Alterity Therapeutics AAN Presentation

Related Articles:

October 2021: Alterity Therapeutics Announces Expanded ATH434 Phase 2 Clinical Development Program

September 2021: Florey Institute of Neuroscience and Mental Health publishes research funded by The Multiple System Atrophy Coalition

July 2021: New Publication Demonstrates ATH434 is Neuroprotective

July 2021: ATH434 Reduces α-Synuclein-Related Neurodegeneration in a Murine Model of Multiple System Atrophy

July 2021: Alterity Therapeutics granted a new US patent targeting major neurodegenerative diseases including Alzheimer’s and Parkinson’s

June 2021: European Union Regulatory Guidance for ATH434 Phase 2 Clinical Trial

April 2021: ATH434 protects brain cells and improves motor function in Parkinsonian disorder

April 2021: ATH434 Preserves Dopaminergic Neurons, Reduces a-synuclein Oligomerization, and Improves Motor Function in a Transgenic Murine Multiple System Atrophy Model

November 2020: Alterity Announces Approval of US Patent for Next Generation Compounds to Treat Neurodegenerative Diseases (ATH434)

October 2020: Alterity commences enrolling Multiple System Atrophy patients in bioMUSE Study (ATH434)

August 2020: New data independently confirms and extends laboratory findings and expands safety profile of ATH434 (PBT-434)

July 2020: Alterity Therapeutics meeting with US FDA provides development pathway for ATH434 (PBT-434)

January 2020: European Commission approves Orphan Designation for Alterity’s lead drug candidate (PBT-434)

July 2019: Alterity Therapeutics Announces Successful Completion of Phase 1 Clinical Trial

June 2019: Alterity Therapeutics Presents to Finance News Network

May 2019: Initial data for Alterity Therapeutics Phase 1 clinical trial released at American Academy of Neurology Annual Meeting

January 2019: Prana Receives Orphan Drug Designation for PBT434 for the Treatment of Multiple System Atrophy

October 2018: PBT434 prevents the accumulation of glial cell inclusions and insoluble alpha-synuclein in a mouse model of Multiple System Atrophy

October 2018: Pre-clinical evidence demonstrates PBT434 as a potential treatment for MSA

NOTE: All of the above information is intended to assist MSA patients and their families in having a conversation with doctors, none of it should be considered medical advice or endorsements of any particular drugs or therapies. Always consult a licensed medical practitioner for expert care.

Anle138B - Phase 1 Completed 2020

A Phase 1 clinical trial to assess safety in healthy volunteers was recently completed. Details may be found at this link: https://www.clinicaltrials.gov/ct2/show/NCT04208152

Anle138B is a small molecule compound that specifically binds toxic oligomeric structures of alpha-synuclein, the core aggregating protein species in Parkinsonian disorders. Through the binding, it effectively dissolves the toxic oligomers and prevents new oligomer formation, addressing the disease at its core. Initial pre-clinical studies in Parkinson’s and MSA animal models have demonstrated the ability to halt disease progression and alleviate symptoms in vivo, effectively preventing the disease from causing further damage by stopping the accumulation of pathological protein aggregates in the brain.

See: ARTEMIS: Targeting Alpha-Synuclein for Treating Multiple System Atrophy

Related Articles:

September 2020: Targeting α-synuclein by PD03 AFFITOPE® and Anle138b rescues neurodegenerative pathology in a model of multiple system atrophy: clinical relevance

August 2020: MODAG Successfully Completes Phase 1 Study of their Lead Candidate Anle138b and Receives Additional USD 1.4 Million from Michael J. Fox Foundation

December 2019: MODAG Initiates First-in-Human Phase 1 Clinical Trial for Anle138b

June 2019: Modag to Develop Treatments for Parkinsonian Disorders, including Multiple System Atrophy

February 2019: Anle138b modulates α-synuclein oligomerization and prevents motor decline and neurodegeneration in a mouse model of multiple system atrophy.

March 2016: Anle138b Partly Ameliorates Motor Deficits Despite Failure of Neuroprotection in a Model of Advanced Multiple System Atrophy.

March 2014: The oligomer modulator anle138b inhibits disease progression in a Parkinson mouse model even with treatment started after disease onset

February 2014: New drug against Alzheimer’s and Parkinson’s on the way

June 2013: Anle138b: a novel oligomer modulator for disease-modifying therapy of neurodegenerative diseases such as prion and Parkinson’s disease

NOTE: All of the above information is intended to assist MSA patients and their families in having a conversation with doctors, none of it should be considered medical advice or endorsements of any particular drugs or therapies. Always consult a licensed medical practitioner for expert care.

BIIB 101 - Phase 1 Clinical Trial In Progress *

A Phase 1 clinical trial in MSA patients is currently underway in Europe.

Details may be found at this link: https://www.clinicaltrials.gov/ct2/show/NCT04165486

BIIB101 (ION464/IONIS-BIIB6Rx ) is an antisense drug targeting alpha-synuclein (SNCA) messenger ribonucleic acid (mRNA). BIIB101 is designed to prevent the production of alpha-synuclein protein and is being developed as a potential therapy for Parkinson’s disease (PD), Multiple System Atrophy (MSA) and related synucleinopathies. Alpha-synuclein protein aberrantly accumulates in the brains of PD and MSA patients and is thought to be one of the key drivers of pathogenesis. It is hypothesized that reduction of SNCA mRNA and, subsequently, reduced synthesis of alpha-synuclein protein will ameliorate the toxic effects of gain-of-function mutations as well as the primary pathology in PD and MSA patients without SNCA mutations.

Related Articles:

August 2020: Ionis reports second quarter 2020 financial results and recent business achievements

April 2018: Biogen and Ionis Expand Strategic Collaboration to Develop Drug Candidates for a Broad Range of Neurological Diseases

July 2017: Ionis Earns $10 Million Milestone Payment from Biogen for Advancing a New Program in its Neurology Collaboration

NOTE: All of the above information is intended to assist MSA patients and their families in having a conversation with doctors, none of it should be considered medical advice or endorsements of any particular drugs or therapies. Always consult a licensed medical practitioner for expert care.

AAV2-GDNF Gene Therapy - Phase 1 Clinicial Trial in Progress *

A Phase 1 clinical trial in MSA patients is in progress.

For Phase 1 clinical trial details See: https://www.clinicaltrials.gov/ct2/show/NCT04680065

Related Articles:

NOTE: All of the above information is intended to assist MSA patients and their families in having a conversation with doctors, none of it should be considered medical advice or endorsements of any particular drugs or therapies. Always consult a licensed medical practitioner for expert care.

HISTORICAL MSA TRIALS

The below experimental therapies were tested in multiple system atrophy patients in past years. No disease modifying effects have been proven. Click on each experimental therapy to learn more details.

SIROLIMUS (rapamycin) - Phase 2 Terminated 2020

A Phase 2 clinical trial in MSA patients was previously completed.

For historical Phase 2 clinical trial details See: https://www.clinicaltrials.gov/ct2/show/NCT03589976

Sirolimus is an FDA approved immunosuppressant drug (also known as rapamycin).

Related Articles:

October 2020: IXICO and NYU Langone Health Sign Agreement to Develop Novel Imaging Markers in Multiple System Atrophy (Sirolimus)

October 2019: Research Speeds Ahead – 1 Year Update from the Sirolimus MSA Trial

December 2018: New study testing a potential drug to slow the progression of MSA opens at NYU

October 2018: Rapamycin for treating MSA: A preclinical proof of concept study

NOTE: All of the above information is intended to assist MSA patients and their families in having a conversation with doctors, none of it should be considered medical advice or endorsements of any particular drugs or therapies. Always consult a licensed medical practitioner for expert care.

EGCG (Epigallocatechin gallate) - Phase 3 Completed 2016

A Phase 3 clinical trial to test green tea extract (EGCG) in MSA patients was previously completed.

For historical Phase 3 clinical trial details See: https://www.clinicaltrials.gov/ct2/show/NCT02008721

EGCG, a polyphenol found in green tea, has shown to inhibit the formation of toxic α-synuclein oligomers in vitro and has shown to transform α-synuclein-oligomers in non-toxic oligomer species. There is also evidence for a neuroprotective effect in MPTP-mouse models of PD and is an antioxidant and iron chelator. There are currently 63 clinical studies (http://clinicaltrial.gov) in which EGCG was applied for various indications, such as Multiple Sclerosis, various forms of cancer and Huntington’s disease. All of which have shown good tolerability and safety with the applied doses of EGCG of up to 1200 mg per day, demonstrating the safety of the drug under controlled clinical conditions. These data provide a solid rationale for testing in a clinical trial if supplementation of EGCG can interfere with the core disease mechanism in MSA and consequently retard the clinical progression of the MSA-related disability.

Related Articles:

July 2019: Safety and efficacy of epigallocatechin gallate in multiple system atrophy (PROMESA): a randomised, double-blind, placebo-controlled trial

October 2018: PROMESA: A randomised, double-blind, placebo-controlled trial to evaluate the progression rate of MSA under EGCG supplementation as anti-aggregation-approach

June 2017: PROMESA: Progression Rate of MSA under EGCG Supplementation as anti-Aggregation-Approach – evaluation of serious adverse events

April 2016: The PROMESA-protocol: progression rate of multiple system atrophy under EGCG supplementation as anti-aggregation-approach

ALZForum

NOTE: All of the above information is intended to assist MSA patients and their families in having a conversation with doctors, none of it should be considered medical advice or endorsements of any particular drugs or therapies. Always consult a licensed medical practitioner for expert care.

IVIG (intravenous immunoglobulin) - Phase 2 Completed 2015

A Phase 2 clinical trial in MSA patients was previously completed.

For historical Phase 2 clinical trial details See: https://www.clinicaltrials.gov/ct2/show/NCT00750867

Related Articles:

November 2012: Treatment of multiple system atrophy using intravenous immunoglobulin

NOTE: All of the above information is intended to assist MSA patients and their families in having a conversation with doctors, none of it should be considered medical advice or endorsements of any particular drugs or therapies. Always consult a licensed medical practitioner for expert care.

RIFAMPICIN - Phase 3 Terminated 2013

A Phase 3 clinical trial in MSA patients was previously completed.

For historical Phase 3 clinical trial details See: https://www.clinicaltrials.gov/ct2/show/NCT01287221

Rifampicin, because of its ability to inhibit the formation of α-synuclein fibrils and disaggregate fibrils already formed, was hoped to delay progression or reverse neurologic and autonomic functions and symptoms in MSA. In an experimental model of MSA, it was hypothesized that Rifampicin would improve behavioral abnormalities of MSA and halt or reverse the pathological change.

The Data Safety Monitoring Board (DSMB) recommended stopping the study after an interim analysis of the primary endpoint revealed that futility criteria were met.

Related Articles:

March 2014: Efficacy and safety of rifampicin for multiple system atrophy: a randomised, double-blind, placebo-controlled trial.

NOTE: All of the above information is intended to assist MSA patients and their families in having a conversation with doctors, none of it should be considered medical advice or endorsements of any particular drugs or therapies. Always consult a licensed medical practitioner for expert care.

INTRANASAL INSULIN - Phase 2 Completed 2012

A Phase 2 clinical trial in Parkinson and MSA patients was previously completed.

For historical Phase 2 clinical trial details See: https://clinicaltrials.gov/ct2/show/NCT02064166

Related Articles:

May 2019: Treatment with Intranasal Insulin May Improve Verbal Fluency and Motor Function, Early Study Shows

April 2019: Safety and preliminary efficacy of intranasal insulin for cognitive impairment in Parkinson disease and multiple system atrophy: A double-blinded placebo-controlled pilot study

ALZForum

NOTE: All of the above information is intended to assist MSA patients and their families in having a conversation with doctors, none of it should be considered medical advice or endorsements of any particular drugs or therapies. Always consult a licensed medical practitioner for expert care.

LITHIUM - Phase 2 Terminated 2011

A Phase 2 clinical trial of Lithium in MSA patients was terminated in 2011.

See: https://clinicaltrials.gov/ct2/show/NCT00997672

Related Articles:

NOTE: All of the above information is intended to assist MSA patients and their families in having a conversation with doctors, none of it should be considered medical advice or endorsements of any particular drugs or therapies. Always consult a licensed medical practitioner for expert care.

FLUOXETINE(Prozac) - Phase 2 Completed 2011

A Phase 2 clinical trial in MSA patients was previously completed.

For historical Phase 2 clinical trial details See: https://clinicaltrials.gov/ct2/show/NCT01146548

Related Articles:

NOTE: All of the above information is intended to assist MSA patients and their families in having a conversation with doctors, none of it should be considered medical advice or endorsements of any particular drugs or therapies. Always consult a licensed medical practitioner for expert care.

RASAGILINE - Phase 2 Completed 2011

A Phase 2 clinical trial in MSA patients was previously completed.

For historical Phase 2 clinical trial details See: https://clinicaltrials.gov/ct2/show/NCT00977665

Related Articles:

NOTE: All of the above information is intended to assist MSA patients and their families in having a conversation with doctors, none of it should be considered medical advice or endorsements of any particular drugs or therapies. Always consult a licensed medical practitioner for expert care.

MINOCYCLINE - Phase 3 Completed 2005

A Phase3 clinical trial in MSA patients was previously completed

For historical Phase 3 clinical trial details See: https://clinicaltrials.gov/ct2/show/NCT00146809

NOTE: All of the above information is intended to assist MSA patients and their families in having a conversation with doctors, none of it should be considered medical advice or endorsements of any particular drugs or therapies. Always consult a licensed medical practitioner for expert care.

RILUZOLE - Phase 3 Terminated 2004

A Phase 3 clinical trial in Progressive Supranuclear Palsy and MSA patients was previously terminated.

For historical Phase 3 clinical trial details See: https://clinicaltrials.gov/ct2/show/NCT00211224

Related Articles:

June 2009: Riluzole Does Not Prolong Survival in Progressive Supranuclear Palsy and Multisystem Atrophy

 

NOTE: All of the above information is intended to assist MSA patients and their families in having a conversation with doctors, none of it should be considered medical advice or endorsements of any particular drugs or therapies. Always consult a licensed medical practitioner for expert care.

PARKINSON DISEASE TRIALS

The below experimental therapies are being tested in Parkinson’s Disease Patients and there is hope they may eventually be tested in MSA Patients. Click on each experimental therapy to learn more details, including links to clinical trial opportunities.

Prasinezumab/PRX002/RO7046015(Phase 2 for Parkinson's Disease)

“Passive Immunization”

A Phase 1 clinical trial in Parkinson’s patients was previously completed. For historical Phase 1 clinical trial details see: https://clinicaltrials.gov/ct2/show/NCT02157714

PRX002 is a humanized IgG1 monoclonal antibody directed against aggregated α-synuclein. Genetic and pathological evidence suggest that this protein plays a central role in the pathogenesis of Parkinson’s disease (PD) and other α-synculeinopathies such as dementia with Lewy bodies (DLB).

Related Articles:

ALZForum

NOTE: All of the above information is intended to assist MSA patients and their families in having a conversation with doctors, none of it should be considered medical advice or endorsements of any particular drugs or therapies. Always consult a licensed medical practitioner for expert care.

BIIB054 (Phase 2 for Parkinson's Disease)

“Passive Immunization”

BIIB054 is an anti-alpha-synuclein antibody being tested in people with Parkinson’s in a Phase II clinical trial.

For clinical trial information see: https://clinicaltrials.gov/ct2/show/NCT03318523

Related Articles:

January 2018: First patient dosed in the Phase 2 SPARK study of BIIB054 (anti-alpha-synuclein antibody) in Parkinson’s disease

ALZForum

NOTE: All of the above information is intended to assist MSA patients and their families in having a conversation with doctors, none of it should be considered medical advice or endorsements of any particular drugs or therapies. Always consult a licensed medical practitioner for expert care.

ABBV-0805(Phase 1 for Parkinson's Disease)

ABBV-0805 is an alpha-synuclein antibody that works by binding to toxic clumps of alpha-synuclein and helping remove them. Pre-clinical studies showed that it did remove the majority of these clumps, which led to slower disease progression and reduced motor symptoms in Parkinson’s disease models.

Related Articles:

NOTE: All of the above information is intended to assist MSA patients and their families in having a conversation with doctors, none of it should be considered medical advice or endorsements of any particular drugs or therapies. Always consult a licensed medical practitioner for expert care.

MEDI-1341 (Phase 1 for Parkinson's Disease)

“Passive Immunization”

Phase I Clinical trial of the alpha-synuclein antibody MEDI-1341 is currently underway in healthy volunteers. See: https://clinicaltrials.gov/ct2/show/NCT03272165

Related Articles:

August 2017: AstraZeneca and Takeda establish collaboration to develop and commercialise MEDI1341 for Parkinson’s disease

ALZForum

 

NOTE: All of the above information is intended to assist MSA patients and their families in having a conversation with doctors, none of it should be considered medical advice or endorsements of any particular drugs or therapies. Always consult a licensed medical practitioner for expert care.

 

Lu AF82422 (Phase 1 for Parkinson's Disease)

Lu AF82422 is a monoclonal antibody targeting alpha-synuclein. Misfolding, aggregation and extracellular spreading of alpha-synuclein is believed to play a major role in disease pathology and progression in Multiple System Atrophy (MSA), Parkinson’s disease and other neurodegenerative disorders.

A Phase I Clinical trial of the alpha-synuclein antibody Lu AF82422 in Healthy Non-Japanese and Japanese Subjects and in Patients With Parkinson’s Disease is currently underway. See: https://www.clinicaltrials.gov/ct2/show/NCT03611569

Related Articles:

August 2018: Lundbeck Brings Potential New Biologic Treatment of Parkinson’s Disease into Clinical Development

Alzforum entry

NOTE: All of the above information is intended to assist MSA patients and their families in having a conversation with doctors, none of it should be considered medical advice or endorsements of any particular drugs or therapies. Always consult a licensed medical practitioner for expert care.

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