Experimental Treatments for Parkinson’s Disease

Parkinson’s disease is a progressive condition of the nervous system that gradually affects movement. The symptoms of the disease are recognized in most people as tremors, limb stiffness, impaired balance, and slow movement, but also include non-motor symptoms such as mood changes, and speaking, swallowing, or chewing problems.

There is currently no cure for Parkinson’s disease but a number of experimental treatments are being tested in clinical trials.

Associations such as The Michael J. Fox Foundation or The Parkinson’s Disease Foundation provide information on approved therapies and experimental therapies under development. The experimental treatments are still in early stages and fall into two categories: symptomatic (drugs that aim to treat motor and non-motor symptoms) and neuroprotective (drugs that aim to slow or prevent the progression of Parkinson’s disease). Below are some examples of experimental treatments divided by clinical study phase.

Symptomatic experimental treatments

Treatments in Phase 1 and 2 clinical trial

In these phases, the drugs are being studied to evaluate their effectiveness and safety.

  • Tasigna (nilotinib) is being investigated to treat cognitive changes in Parkinson’s disease. The drug is usually administered when patients fail to improve with other medications and can target both motor and cognitive problems.
  • Mavoglurant (AFQ056) is being studied as an add-on therapy to improve dyskinesia associated with long-term use of levodopa (L-dopa) in Parkinosn’s disease patients.

Treatments in Phase 3 clinical trial

Most symptomatic drugs are in this phase of testing. Examples of drugs being developed to treat motor and non-motor symptoms of Parkinson’s disease are listed below.

  • CVT-301 is an “off” rescue drug to provide rapid relief of motor symptoms. A Phase 3 safety and effectiveness study (NCT02242487) is currently recruiting participants.
  • Two Phase 3 clinical studies (NCT01036139 and NCT01066442) have been completed for the investigational drug Pitolisant (BF2.649), to treat excessive sleepiness in Parkinson’s patients.

Neuroprotective experimental treatments

Treatments in preclinical phase

There are some drugs that target proteins in the brain produced by the alpha-synuclein and LRRK2 genes and drugs that reduce inflammation in the brain currently in preclinical studies.

Research is also underway in stem cells (stem cells are the precursor of many cell types in the body during early life and growth). A team from Lund University in Sweden used rat models in their experiments and showed that it is possible to make dopamine cells from human embryonic stem cells. The goal is to transplant these cells into the brain to replace the damaged cells. If successful, this type of therapy could improve symptoms in certain patients.

Treatments in Phase 1 and 2 clinical trials

  • MSDC-0160 was originally identified as a treatment for type 2 diabetes. It is thought that the drug has the potential to be the first Parkinson’s treatment with neuroprotective capability (protecting the nerve cells from becoming damaged or dying).
  • Affitope (PD01A) is a vaccine that targets the alpha-synuclein protein, found in toxic levels in Parkinson’s disease patients.

Treatments in Phase 3 clinical trials

DynaCirc (isradipine) is a calcium channel blocker already in use to treat high blood pressure. The drug may block damage caused by certain chemicals that flow through special channels in the brain cells that make dopamine. The drug’s effectiveness in early Parkinson’s disease is being evaluated in a Phase 3 study (NCT02168842).


Note: Parkinson’s News Today is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.

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