Patient Health Goals Are The Primary Consideration In Developing A Treatment Plan For Parkinson’s
For most Parkinson’s disease patients, controlling motor symptoms is one of the primary goals of any treatment. The symptoms are disruptive, and depending on their severity can significantly impair quality of life and a patient’s ability to engage in the essential activities of daily living — activities that many of us take for granted and perform on autopilot. These motor fluctuations and dyskinesias can affect everything, including walking, writing, sleeping, eating, and family relationships. The challenges to such basic experiences can come with a heavy emotional toll.
It’s widely accepted in the Parkinson’s community that the holy grail of Parkinson’s research is a drug that does not just treat symptoms but is a disease modifier for both motor and non-motor symptoms. The good news is that we clinicians have a growing pharmacopeia in our disposal to address Parkinson’s motor (and non-motor) symptoms, including several in the research pipeline that are significantly improving longer-term outcomes.
And as with all Parkinson’s treatments — as exciting as they may be — it’s our responsibility to actively listen to our patients, understand their lifestyles and goals, and respect their decisions about the approaches we suggest for treating and managing their Parkinson’s. Ultimately, it is THEIR experience, and we clinicians are here to bring our voice and expertise in a supporting role to help our patients live the life they want to as people with Parkinson’s.
Hope in the research pipeline: disease modification is a near-term prospect
While today’s standard treatments — like levodopa — can help manage Parkinson’s symptoms, they don’t stop disease progression. Researchers are continually working to fully understand the biology of Parkinson’s to support the development of new therapies that address the condition holistically, not just target or control the mechanisms of particular symptoms. One of the most significant challenges for any Parkinson’s treatment is addressing motor fluctuations. We are continually seeking ways to reduce “off” time and improve “on” time in which the medication is working; with patients feeling maximally mobile, functional and independent, with minimized dyskinesias. There are currently three treatments under investigation showing great promise for motor symptom control: prasinezumab, bemdaneprocel, and ambroxol.
Prasinezumab is a monoclonal antibody designed to bind to alpha-synuclein, a protein that clumps together and spreads through the brain in Parkinson’s disease. The drug targets the protein buildup system and binds to misfolded alpha-synuclein to prevent it from spreading between brain cells. Prasinezumab may offer incremental benefits but is not a cure. Phase III of the drug’s clinical trials was initiated in June 2025.
Potential benefits, limitations and side effects:
◎ Some studies suggest it may slow motor symptom progression, particularly over longer follow-up periods. Continued research shows signals of reduced disease progression over time, though results vary.
◎ Major trials were not consistent or statistically definitive; effects appear modest and may depend on early use; the drug is given by IV infusion, which can be time-consuming for patients.
Bemdaneprocel is a stem cell–based therapy designed to replace the dopamine-producing neurons lost in Parkinson’s disease. The drug targets the dopamine system using stem-cell-derived neuron precursors implanted into the brain. These cells develop into mature dopamine-producing neurons after transplantation. The treatment essentially replaces what Parkinson’s destroys. Phase three clinical trials are currently enrolling patients, with data expected in 2027.
Potential benefits, limitations and side effects:
◎ Early trials show improvements in motor symptoms and daily function. Data suggests reduced “off” periods and long-lasting benefits, especially at higher doses.
◎ May reduce “off” periods when medications stop working.
◎ Requires brain surgery and patients typically need temporary immunosuppression to help transplanted cells survive.
◎ Long-term effectiveness is still being studied in clinical trials and not widely available.
Ambroxol is a cough medication used in Europe that is now being studied as a potential Parkinson’s treatment. This medication is considered to be low-risk, though it’s still considered experimental for the use of treating Parkinson’s disease. Ambroxol targets the brain’s waste-disposal system and boosts activity of an enzyme called glucocerebrosidase (GCase). It also helps protect dopamine-producing neurons by clearing out harmful proteins like alpha-synuclein.
Potential benefits, limitations and side effects:
◎ Shown to be safe and well tolerated in early clinical trials.
◎ May stabilize cognitive and psychiatric symptoms in some patients.
◎ Clinical trials have not yet shown clear disease-modifying effects in primary outcomes, with benefits appearing modest and inconsistent and the drug is not approved for Parkinson’s disease in the U.S.
Patients and family goals are paramount
With so many emerging options — each with different risks and benefits — treatment decisions for Parkinson’s disease are highly personal. Ultimately, a patient’s health goals — not the latest drug research findings — should be the primary guidance for treatment approach. Patients whose symptoms are mild or who feel that their day-to-day life has not been significantly affected, may decide to delay treatment. Some patients may decide against taking medication suggested for prevention or see how their symptoms respond. These moments can feel like pinch points in the clinician-patient relationship. But the truth is that our patients aren’t necessarily on our clinical timeline and may simply not be “there” yet with us in a treatment suggestion: They may not want to deal with side effects, be tied to a medication schedule, or simply may have not fully processed their PD and need more time to come to understand their diagnosis. It’s critical that we physicians consider patients’ lifestyle, symptom priorities, risk tolerance and long-term expectations in our treatment conversations. Our job is not to evangelize or treat our patients simply as people with a disease that needs curing at some point in the future, it’s to inform them of treatment options, and manage their Parkinson’s symptoms on a daily basis, so they can live well as they want to and thrive in the present.
Pravin Khemani, MD, FAAN is the Medical Director of the Movement Disorders Program at the Providence Swedish Neuroscience Institute in Seattle. Dr. Khemani is also a Clinical Professor University of Washington in Seattle.
Ideas and opinions expressed in this post reflect that of the author(s) solely. They do not necessarily reflect the opinions or positions of the World Parkinson Coalition®