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Spotlight on Research

Marina E. Emborg, M.D., Ph.D.

Marina E. Emborg,
M.D., Ph.D.

Imagine if your doctor was able to treat your Parkinson’s disease (PD) with a therapy made from your very own skin cells! It may sound futuristic, but it’s exactly the type of treatment, called ‘personalized medicine,’...

Learn more about Dr. Emborg's research

Find a Clinical Trial


Results: Center Grants Program

The centerpiece of PDF’s research program is our Center Grants Program. The concept behind funding select, specialized PD research groups is that “the whole is greater than the sum of its parts.” In 2008, PDF was able to modestly increase our financial support of Parkinson’s research centers at Columbia and Cornell Universities in New York, and Rush University in Chicago.

All three of these programs are of the highest scientific quality, and continue to be peer-reviewed periodically by an independent group of renowned Parkinson's disease scientists. 

Columbia University Medical Center Team

PDF's largest research grant — $2.7 million in 2008 — supports our ongoing partnership with the Center for Parkinson's Disease and other Movement Disorders at Columbia University . The center is comprised of six main laboratories and a variety of clinical study and training programs. The following are examples of some recent accomplishments of the Columbia University team.

Dr. David Sulzer continues to make great strides in his research to understand why dopamine neurons die in Parkinson's. In 2004, he showed how synuclein, a protein that mutates in certain cases of Parkinson's, is normally broken down and why this does not occur in mutant forms that are found in PD. This blockade, and subsequent protein accumulation, is believed to lead to the death of neurons. He has now discovered that when non-mutated synuclein is exposed to dopamine, it also disrupts this same protein breakdown. This finding may provide, for the first time, an explanation for why dopamine neurons are the most vulnerable in PD.

Dr. Robert Burke has found a way to better utilize the potential of neurotrophic factors — naturally occurring substances that can protect the brain cells that die in Parkinson's. Researchers believe neurotrophic factors show great promise in treating PD but in the past, had experienced challenges in effectively administering the treatment. Dr. Burke has shown in animal studies that a gene therapy approach using enzyme, AKT, delivers the treatment more efficiently and both increases neuron size and offers complete neuron protection.

Dr. Lorraine Clark has led a study implicating a specific gene mutation in the development of PD. She has demonstrated that people who carry the mutation, which is in the GBA gene, have an increased risk of developing PD, especially among those younger than age 50 and among Jews of Eastern European.

Rush University Medical Center

Since 1999, PDF has supported the Rush University Medical Center Movement Disorders program in Chicago, IL. For 2007, PDF’s grant of $275,000 was matched dollar-for-dollar by Rush’s own private fundraising, for a total of $550,000 in funding for PD research.

Dr. Jeffrey Kordower, a Rush neuroscientist, reported on the promising Phase I trial of a gene therapy technique in which neuturin, a “growth factor,” is used in an attempt to protect and perhaps restore dopamine function. The trial confirmed the safety of the technique and suggested that it may be effective, paving the way for a Phase II trial, which completed enrollment in November 2007.

A second area with particularly important research outcomes has been a series of studies on placebo-related influences on PD. A group of international investigators, led by Dr. Christopher Goetz, analyzed placebo-related improvements in several clinical trials of new treatments for Parkinson’s disease. Neuroimaging studies have suggested that this effect is biochemically based and linked to increased dopamine release in the brain area known as the “ventral striatum.” Increased motivation, anticipation of change and involvement in novel experiences are thought to activate this dopaminergic pathway and may explain the marked improvement seen during placebo treatment in some patients.

Weill Cornell Medical University

In 2007, PDF’s grant of $100,000 to Weill Cornell Medical Center supported the work of several preeminent researchers.

This includes Dr. M. Flint Beal, whose work on neuroprotection shows great potential for developing treatments that could slow, or even halt the progression of PD, particularly for those who are in its early stages. Following a successful Phase II trial demonstrating the efficacy of Coenzyme Q10 (CoQ10), a substance which occurs naturally in the body, Dr. Beal is currently leading a Phase III trail that commenced in early 2008.



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