INDEPENDENT RESEARCH MONITOR

Emerging treatments

New therapeutic approaches and substantive development, safety or regulatory changes in relapsing and progressive MS. Inclusion is not a recommendation or proof of benefit.

Emerging treatments

New therapeutic approaches and substantive development, safety or regulatory changes in relapsing and progressive MS. Inclusion is not a recommendation or proof of benefit.

results

Emerging treatmentsMyelin repair
Registered study in peopleOngoing, but not accepting participantsNo evidence of benefit in people yet

Phase 3 study that can add or remove treatments

OCTOPUS

Metformin + alpha-lipoic acid

Ongoing • results from the first stage are being reviewed

Practical availability: Research only — not an established treatment

What kind of evidence exists?: Phase 3 study in people • the first-stage decision is still awaited

What to watch next: A decision on which initial treatment groups will continue is expected around October 2026

Emerging treatmentsMyelin repair
Registered study in peopleOngoing, but not accepting participantsNo evidence of benefit in people yet

Phase 2

MACSiMiSE-BRAIN

Metformin • progressive MS

Ongoing, but not accepting new participants

Practical availability: Research only — not an established treatment

What kind of evidence exists?: Phase 2 study in people • results are awaited

What to watch next: Completion of participant follow-up and publication of the results

Emerging treatmentsMyelin repair
Registered study in peopleLooking for participantsNo evidence of benefit in people yet

Phase 2

Metformin in aging MS

Metformin • people aged 55–75

Looking for participants • NCT06463743

Practical availability: Study in people — looking for participants does not guarantee access

What kind of evidence exists?: Phase 2 study in people • results are awaited

What to watch next: Results on safety and on changes measured with brain scans and specialised measurements of myelin

Emerging treatmentsMyelin repair
Registered study in peopleLooking for participantsNo evidence of benefit in people yet

Phase 2

ACT-1004-1239

Drug acting on the CXCR7/ACKR3 receptors • progressive MS

Looking for participants

Practical availability: Study in people — looking for participants does not guarantee access

What kind of evidence exists?: Phase 2 study in people • results are awaited

What to watch next: At 48 weeks, MRI, electrical nerve tests and biochemical tests will look for possible signs of myelin repair

Emerging treatmentsMyelin repair
Registered study in peopleLooking for participantsNo evidence of benefit in people yet

Phase 1/2

ReVIVE

Clemastine • long-term loss of myelin

Looking for participants

Practical availability: Study in people — looking for participants does not guarantee access

What kind of evidence exists?: Phase 1/2 study in people • results are awaited

What to watch next: MRI scans using several measurements will look for possible changes in myelin • estimated completion in 2027

Emerging treatmentsMyelin repair
Registered study in peopleLooking for participantsNo evidence of benefit in people yet

Phase 2

ReCOVER

Clemastine • sudden inflammation of the optic nerve

Looking for participants

Practical availability: Study in people — looking for participants does not guarantee access

What kind of evidence exists?: Phase 2 study in people • results are awaited

What to watch next: Electrical testing of the visual pathway and MRI will look for possible signs of myelin repair • estimated completion in 2028

Emerging treatmentsMyelin repair
Registered study in peopleNot accepting participants yetNo evidence of benefit in people yet

Phase 1/2

ReINFORCE

Clemastine • recent areas of myelin loss

Not yet accepting participants • scheduled start 15 September 2026

Practical availability: Research only — not an established treatment

What kind of evidence exists?: Phase 1/2 study in people • no results yet

What to watch next: The study will begin looking for participants and use MRI scans with several measurements to look for possible myelin repair

Emerging treatmentsMyelin repair
Registered study in peoplePausedNo evidence of benefit in people yet

Phase 1/2

MODIF-MS

Ifenprodil • possible myelin repair

Temporarily paused in the European CTIS registry

Practical availability: Research only — not an established treatment

What kind of evidence exists?: Phase 1/2 study in people • temporarily paused

What to watch next: Official confirmation that the study has restarted, or another update on its status

Emerging treatmentsMyelin repair
Registered study in peopleLooking for participantsNo evidence of benefit in people yet

Small pilot study of a medical device

SetPoint MS Pilot

Stimulation of the vagus nerve • relapsing-remitting MS

Looking for participants

Practical availability: Study in people — looking for participants does not guarantee access

What kind of evidence exists?: Pilot device study in people • early exploration of possible benefit

What to watch next: Safety and early signs of myelin repair will be assessed after 48 weeks

Emerging treatmentsMyelin repair
Registered study in peopleLooking for participantsNo evidence of benefit in people yet

Phase 2

TOTEM-RRMS

Testosterone • relapsing-remitting MS

Ongoing • looking for participants

Practical availability: Study in people — looking for participants does not guarantee access

What kind of evidence exists?: Phase 2 study in people • results are awaited

What to watch next: MRI scans will look for possible signs of myelin repair and protection of nerves

Emerging treatmentsMyelin repair
Safety and how the body processes the drugAnnounced or plannedPermission or regulatory step

Phase 1 • initially in healthy volunteers

PTD802

PTD802, a drug that acts on the GPR17 receptor

Permission has been given for the first study in people

Practical availability: Research only — not an established treatment

What kind of evidence exists?: Study in healthy volunteers • no evidence of benefit in people with MS

What to watch next: Researchers will first assess safety, side effects and how the body processes the drug

Emerging treatmentsMyelin repair
Planned study in peopleAnnounced or plannedPermission or regulatory step

Planned Phase 2 study

Lucid-MS / Lucid-21-302

Lucid-21-302, a drug that blocks PAD2 • progressive MS

The FDA allowed the research to move forward • no public study registration was found

Practical availability: Research only — not an established treatment

What kind of evidence exists?: Step toward a study in people • no evidence of benefit in people with MS

What to watch next: Public registration of the Phase 2 study, announcement of participating centres and the start of participant enrolment

Emerging treatmentsMyelin repair
Preparing for first human studiesAnnounced or plannedNo evidence of benefit in people yet

Preparing for the first study in people

Low-dose theophylline patch

Low-dose theophylline skin patch

Funding supports improvement of the patch and manufacture to pharmaceutical standards • a study in healthy volunteers is planned

Practical availability: Laboratory or animal research — not a treatment available to patients

What kind of evidence exists?: No evidence of benefit in people with MS • laboratory findings and patch-development work only

What to watch next: Testing the improved patch, manufacturing it to pharmaceutical standards and running the first safety study in people

Emerging treatmentsMyelin repair
Laboratory or animal researchLaboratory or animal programmePromising early finding

Laboratory and animal research

Bavisant / BRAVEinMS

Bavisant, a drug that acts on the histamine H3 receptor

Promising results in human cells and animal models • no registered study in people with MS

Practical availability: Laboratory or animal research — not a treatment available to patients

What kind of evidence exists?: Tests in human cells and animal models • no evidence of benefit in patients

What to watch next: Further drug development and formal registration of a study in people

Emerging treatmentsMyelin repair
Laboratory or animal researchLaboratory or animal programmePromising early finding

Laboratory and animal research aiming toward a future study in people

Petratos / DITPA research

DITPA, a drug related to thyroid hormone

Active Monash research through 2028 • it has not yet reached a study in people

Practical availability: Laboratory or animal research — not a treatment available to patients

What kind of evidence exists?: Laboratory and animal findings only • no proven benefit in people with MS

What to watch next: Confirmation in a paper reviewed by independent scientists, checks required by regulators and public registration of a study in people

Emerging treatmentsMyelin repair
Laboratory or animal researchLaboratory or animal programmePromising early finding

Improvement work before studies in people

CN045

CN045, a small molecule that helps immature cells become myelin-making cells

Promising results in human cells in the laboratory and in mice

Practical availability: Laboratory or animal research — not a treatment available to patients

What kind of evidence exists?: Laboratory and animal findings only • no proven benefit in people with MS

What to watch next: Improve the molecule, test how the body processes it and check for possible toxicity before selecting a final version for future study

Emerging treatmentsMyelin repair
Preparing for first human studiesLaboratory or animal programmeNo evidence of benefit in people yet

Advanced laboratory development

Scripps / Lairson combination

A fixed-dose combination of two small molecules

A candidate combination was selected because the two molecules together increased myelin repair in experimental models

Practical availability: Laboratory or animal research — not a treatment available to patients

What kind of evidence exists?: Laboratory and animal findings only • no proven benefit in people with MS

What to watch next: Further development and safety checks before researchers can request permission for a study in people

Emerging treatmentsMyelin repair
Laboratory or animal researchLaboratory or animal programmeNo evidence of benefit in people yet

Laboratory and animal research • a development partner is being sought

Rutgers mGluR5 agonists

New small molecules that activate the mGluR5 receptor

Activity was seen in human myelin-making cells and animal models • a patent application is pending

Practical availability: Laboratory or animal research — not a treatment available to patients

What kind of evidence exists?: Laboratory and animal findings only • no proven benefit in people with MS

What to watch next: Select the best molecule and complete the checks needed before requesting permission for a study in people

Emerging treatmentsMyelin repair
Laboratory or animal researchLaboratory or animal programmePromising early finding

Cell therapy in animal models

Cambridge neural stem-cell research

Transplanting neural stem cells

Early evidence that the idea may work in models of long-term myelin loss

Practical availability: Laboratory or animal research — not a treatment available to patients

What kind of evidence exists?: Laboratory and animal findings only • no proven benefit in people with MS

What to watch next: Tests of delivery, dose and safety before a study in people can be considered

Emerging treatmentsMyelin repair
Laboratory or animal researchLaboratory or animal programmePromising early finding

Cell therapy in animal models

Gene-edited human myelin-making cells

Genetically modified human cells that can make myelin

The cells moved more effectively toward long-term injuries and increased myelin repair in rodents

Practical availability: Laboratory or animal research — not a treatment available to patients

What kind of evidence exists?: Laboratory and animal findings only • no proven benefit in people with MS

What to watch next: Develop a reliable manufacturing process and complete long-term safety checks before a study in people

Emerging treatmentsMyelin repair
Results from peopleCompletedPromising early finding

Phase 2a • completed

CCMR Two

Metformin + clemastine

Promising change in the brain's electrical response to vision

Practical availability: Research only — not an established treatment

What kind of evidence exists?: Results in people • a biological sign was seen, but no short-term improvement in disability

What comes next: A full paper reviewed by independent scientists and a larger study to confirm the result

Emerging treatmentsMyelin repair
Results from peopleCompletedNo clear effect found

Phase 2 • completed

ReWRAP

Bazedoxifene acetate

The study's main and secondary measures of myelin repair were not reached

Practical availability: Research only — not an established treatment

What kind of evidence exists?: Results in people • no clear benefit was found in the measures of myelin repair

What this means: The drug was generally well tolerated, but it did not show a statistically clear benefit

Emerging treatmentsMyelin repair
Results from peopleTerminated / stoppedDid not show the hoped-for effect

Phase 3 • stopped

RESTORE

Clemastine • an MS-related disorder of eye movement

The study stopped after an early review found it was unlikely to reach its goal

Practical availability: Research only — not an established treatment

What kind of evidence exists?: Phase 3 study in people • stopped after difficulty finding participants and an early review

What this means: The decision was linked to slow enrolment and the early review; by itself, it does not prove conclusively that clemastine is ineffective

Emerging treatmentsMyelin repair
Results from peopleCompletedDid not show the hoped-for effect

Phase 2 • completed

PIPE-307 / VISTA

PIPE-307, a drug that blocks the M1 receptor

The study's planned measures did not show the hoped-for benefit

Practical availability: Research only — not an established treatment

What kind of evidence exists?: Results in people • the measures of benefit did not show the expected effect

What this means: It remains uncertain whether the drug will continue to be developed for MS

Emerging treatmentsMyelin repair
Results from peopleCompletedNo evidence of benefit in people yet

Early Phase 1 • completed

Metformin PMS

Metformin • primary or secondary progressive MS

Completed • results have not yet been published

Practical availability: Research only — not an established treatment

What kind of evidence exists?: Early Phase 1 study in people • public results are awaited

What comes next: Public results on safety and possible benefit from study NCT05349474