Gene Therapy: A New Possibility
for Parkinson’s Patients

A new gene therapy called ProSavin may help reduce motor symptoms of Parkinson’s disease, suggests a recent study published in The Lancet.

Although Parkinson’s cannot be cured, medications can improve symptoms. Typically, treatment includes dopamine-replacement therapy to replenish the main brain chemical that patients with Parkinson’s lose. This new gene therapy restores the patient’s dopamine production.

Researchers saw significant motor improvements in all study participants at six months and 12 months after treatment. The new study adds to the growing evidence that gene therapy for Parkinson’s can be safe and well-tolerated by patients.

Importantly, this was the first time researchers used a lentiviral vector, a tool used by molecular biologists to deliver genetic material into the cells, expanding the spectrum of potential gene delivery methods. While the results of this study are promising, the benefits need to be viewed with caution since this was an open-label (unblinded) trial that did not involve a placebo.

The Problem of Parkinson’s

Each year, approximately 60,000 Americans get a diagnosis of Parkinson’s disease, a chronic degenerative neurological condition that leads to tremors and difficulty with movement and coordination. Its four primary symptoms are:

  • Tremor–trembling in hands, arms, legs, jaw and face.
  • Rigidity–stiffness of the limbs and trunk.
  • Bradykinesia–slow movement.
  • Postural instability–impaired balance and coordination.

As Parkinson’s disease symptoms become more pronounced, patients may have difficulty walking, talking or completing other simple tasks. Other, less common movement disorders (called atypical Parkinsonian syndromes or Parkinson’s plus syndromes) have similar symptoms.

For help, request an appointment or call the Cushing Neuroscience Institute’s Movement Disorders Center at 516-325-7050.

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