Stem Cells Slow the Rate of Decline in Progressive Supranuclear Palsy

Stem Cells Slow the Rate of Decline in Progressive Supranuclear Palsy

Progressive supranuclear palsy, also known as PSP, is a disorder of the brain that gets worse over time (progressive neurodegenerative disorder). Many progressive supranuclear palsy symptoms are similar to Parkinson’s disease. These include rigidity, slowness of movement, cognitive (thinking) problems, difficulty speaking, and difficulty swallowing. While people with Parkinson’s disease can have an unsteady gait and “freeze,” these symptoms are much more prominent in people with progressive supranuclear palsy. Likewise, people with PSP have a particular eye problem called supranuclear gaze palsy, which causes PSP patients to have difficulty moving their eyes in certain directions.

Despite the similarities between PSP and Parkinson’s disease, there are no treatments for progressive supranuclear palsy as they are for Parkinson’s disease. Drugs like levodopa help reduce tremors and rigidity in people with Parkinson’s, but they have been largely ineffective in people with PSP. Some PSP patients may benefit from drugs like levodopa, but most experience severe visual hallucinations or other side effects, which causes them to stop the medication. Because there are so few treatments, patients with progressive supranuclear palsy rely on supportive care measures such as occupational and physical therapy, nutritional support, and palliative care.

To address this critical need, researchers are testing mesenchymal stem cells for their ability to treat progressive supranuclear palsy. Dr. Margherita Canesi and her colleagues selected five patients with progressive supranuclear palsy. Her research team used bone marrow from healthy volunteers to select healthy mesenchymal stem cells. The researchers then infused the mesenchymal stem cells into patients in a single infusion.

While patients with PSP normally deteriorate rapidly, the patients who received a single stem cell treatment remained stable for at least six months after treatment. Some patients still maintained function at the end of the study (12 months). All patients tolerated the treatment well, there were no major side effects. While the study was small, it suggests that stem cell treatment was able to change the natural course of the disease. Based on these encouraging results, the authors have set their sights on a randomized, placebo-controlled phase 2 study to further test mesenchymal stem cell treatments in patients with progressive supranuclear palsy. We look forward to their results with great enthusiasm.

 

 

Reference: Canesi, M. et al. (2016). Finding a new therapeutic approach for no-option Parkinsonisms: mesenchymal stromal cells for progressive supranuclear palsy. Journal of Translational Medicine. 14, Article number: 127 (2016).

Researchers Suggest A Way to Test the Safety of Stem Cell Therapy in Progressive Supranuclear Palsy

Researchers Suggest A Way to Test the Safety of Stem Cell Therapy in Progressive Supranuclear Palsy

A recent publication in the Journal of Translational Medicine has highlighted the potential for stem cells to help patients with progressive supranuclear palsy and has proposed measures for testing the safety of stem cell therapy. Like Parkinson’s disease, progressive supranuclear palsy is a neurodegenerative disease that is progressive and characterized by abnormal deposits of the protein tau in the brain. Also like Parkinson’s, there is not a reliable prevention or cure for the disease.

Previous studies have shown that stem cells can help improve Parkinson’s disease symptoms, including problematic functioning of the brain chemical dopamine. Though the etiology of progressive supranuclear palsy is unknown, because it has some physiological features that are similar to Parkinson’s, researchers have reasoned that stem cell therapies may also help the patients who suffer from this disease. However, given that Parkinson’s disease and progressive supranuclear palsy can be differentiated on a clinical basis, it was initially unclear what specific impact stem cells may have on progressive supranuclear palsy.

Nonetheless, consistent with the idea that stem cells may help those with progressive supranuclear palsy, some studies have provided promising data for the ability of stem cells to improve relevant symptoms. The aim of the recent Journal of Translational Medicine publication was to lay out a plan for testing the safety of stem cell therapy in progressive supranuclear palsy patients.

The researchers propose a randomized, double-blind phase I clinical trial that would also be placebo-controlled to assess the safety of stem cell therapy using mesenchymal stem cells in patients with progressive supranuclear palsy patients. In addition to providing evidence on the safety of the stem cell therapy, another value in executing the proposal from this publication is the ability to distinguish whether the effects of stem cells in progressive supranuclear palsy that have been previously observed are due to placebo. In other words, this placebo-controlled trial would allow researchers to determine whether patients improve because they believe they are going to improve or because the stem cell therapy has a direct impact on their disease.

Learn more about stem cell therapy for Parkinson’s disease.

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