These compounds are still in the experimental laboratory study stage and have not yet moved from the lab to clinical trials in MSA patients but may hold promise for the future.

Click on each compound to learn more details about these experimental laboratory studies and what has been discovered so far.
PD01A & PD03A (alpha-synuclein vaccines) - Phase 1 Completed 2017
A Phase 1 clinical trial in MSA patients was previously completed.
For historical Phase 1 clinical trial details See: https://www.clinicaltrials.gov/ct2/show/NCT02270489
Two vaccines aimed at preventing the buildup of the alpha-synuclein protein are under development.
Related Videos:
October 2019: A vaccine for Parkinson’s disease shows promising results
Related Articles:
July 2021: AC Immune Announces Strategic Acquisition of Industry-leading Parkinson’s Disease Vaccine Candidate
September 2020: A Phase 1 Randomized Trial of Specific Active α‐Synuclein Immunotherapies PD01A and PD03A in Multiple System Atrophy
September 2016: Boost Vaccination Data Encourage Continued Development of AFFiRiS Therapeutic Parkinson’s Disease Vaccine Against Alpha-Synuclein
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.
KM-819 - Phase 1 Completed 2017
A Phase 1 clinical trial in healthy volunteers was previously completed. See: https://www.clinicaltrials.gov/ct2/show/NCT03022799
KM-819 is a new drug candidate targeting FAS-associated factor 1, which inhibits the death of dopamine-secreting cells, thereby stopping the progression or slowing the progress of the disease. KM-819 has been shown to inhibit the accumulation of alpha-synuclein.
Related Articles:
February 2019: Kainos Medicine starts developing treatment for multiple system atrophy
January 2019: Kainos Medicine Reaches Milestone in Bringing “KM-819” Treatment for Parkinson’s Disease to Market
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.
α-SYN SM (alpha-synuclein-SMART Molecule)
The drug company has filed a request with the FDA requesting orphan drug designation for Alpha-SYN SM in the treatment of MSA.
Related Articles:
April 2014: Brain Imaging Tracer for 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.
MPLA (monophosphoryl lipid A)
MPLA is a clinically approved vaccine adjuvant. It is being tested in MSA experimental animal models in Austria.
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.
BELNACASAN (VX-765)
Belnacasan is currently in Phase 2 clinical trials for Epilepsy and is also being tested in MSA experimental models in the lab by a collaborative research consortium in Austria, France and Germany.
See: ARTEMIS: Targeting Alpha-Synuclein for Treating Multiple System Atrophy
Related Articles:
August 2016: Reducing C-terminal truncation mitigates synucleinopathy and neurodegeneration in a transgenic model of 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.
YTX-7739
YTX-7739 has been shown in experimental lab studies to inhibit many of the key aspects of alpha-synuclein toxicity.
Related articles:
September 2018: Yumanity Therapeutics Announces First Clinical Candidate for the Potential Treatment of 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.
NaPB
NaPB provided GCI reduction, neuroprotection and motor improvement in experimental MSA mouse models when therapy started at the time of motor symptom onset.
Related Articles:
October 2016: Neuroprotection by Epigenetic Modulation in a Transgenic Model of 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.
BENZTROPINE
Benztropine provided remyelination, neuroprotection and motor improvement in experimental mouse models of MSA.
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.
A-443654
A-443654 is an inhibitor of the multiple functional kinase AKT. A-443654 and AKT inhibition may be a potential strategy for reducing SNCA expression and treating Parkinson’s disease pathology.
Related articles:
September 2021: The AKT modulator A-443654 reduces α-synuclein expression and normalizes ER stress and autophagy
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.
SBT-272
In
Related Articles:
November 2020: Stealth BioTherapeutics Reports Third Quarter 2020 Financial Results And Recent Business Highlights
May 2020: Stealth BioTherapeutics Reports First Quarter 2020 Financial Results and Recent Business Highlights
January 2020: Stealth BioTherapeutics Initiates First-in-Human Study of SBT-272
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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