Until recently, there has been no “biomarker” to establish a diagnosis of Parkinson’s, which is mainly clinical. Patients typically have tremor, slowness of movement, stiffness, and postural instability. New biomarkers are now available, including a DaTscan. It can differentiate Parkinson’s from essential tremor but not from other Parkinson’s-like diseases. A similar scan uses an isotope labeled fluorodopa.

There is more understanding of the genetic basis of Parkinson’s. Single-gene disorders account for less than 10% of all cases. New research has shown a growing number of “susceptibility genes,” which can increase the risk.

Intensive research is focused on neuroprotective or disease-modifying drugs for Parkinson’s, including isradipineinosine, which elevates urate and may act as an antioxidant; AZD-3241; RP103, which increases brain-derived neurotrophic factor; pioglitazone and similar medications used for diabetes and others. Other promising drugs have not proven helpful in research. They include a neurotrophic factor and neurturin. Further, a new gene delivery system using LentiVector gene technology shows so far only slight improvement and is considered highly experimental. Vaccines and immunotherapy with monoclonal antibodies targeting alpha-synuclein are being studied as well.

New treatment options include improvements in the delivery of levodopa using skin patches, pumps, inhalers. One is likely to be available commercially. It has been studied for 30 years, and it uses a levodopa-carbidopa intestinal gel infusion, but this requires a puncture through the abdomen into the small intestine. A new drug termed pimavanserin was found to be safe, effective, and well tolerated in research in treating levodopa-induced psychosis. This will probably be available as well.

In short, researchers are optimistic about the future of Parkinson’s treatment.

Dr. Jack Florin, MD

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