Unraveling the brain’s hardest puzzle
New hope is on the horizon for patients diagnosed with Parkinson's

What if you were handed a puzzle and told there was no solution? That you were welcome to try – that many of the best minds before you had – but it was almost certain that you’d never find the answer?

While there has been progress in alleviating symptoms, for decades, this has been a discouraging reality for the world’s Parkinson’s disease research community. And for years, there has been little progress against the devastating disease that attacks the brain, robs patients of mobility and leads to further disability, suffering and the possibility of early death.

But now it seems as if all that work may begin paying off – and science may finally be making headway on the brain’s toughest puzzle.

According to a 2014 Medicines in Development report by the Pharmaceutical Research and Manufacturers of America (PhRMA), new R&D in gene therapy and other avenues of treatment may begin bearing fruit in reducing the human cost of this debilitating disease as well as in easing its significant economic burden.

PhRMA notes that U.S. biopharmaceutical companies have 37 new medications for Parkinson’s, all of which are in clinical trials or are under review by the U.S. Food and Drug Administration. These potential medications are designed to treat Parkinson’s and related conditions in existing patients – but also help diagnose the disease in those who may not know they have it or who are prone to it due to genetics or other influencing factors.

“The nearly 40 medicines in development today offer great hope that together we can ease the tremendous burden of Parkinson’s on patients, public health and economies around the world,” says PhRMA President and CEO John J. Castellani in the release.

Among the advances being pursued are:

  • A new type of gene therapy that focuses on the movement-control center in the brain
  • New treatments that the body can absorb and use more effectively, such as an intranasal formulation and an intestinal gel
  • A medication designed to home in on a specific receptor found in the area of the brain where Parkinson’s-related damage and abnormalities are evident

Parkinson’s is a motor-system disorder that occurs when the brain loses dopamine-producing neurons. It can cause a range of symptoms, from tremors and balance problems to coordination deficits and speech impairments. According to the PhRMA Medicines in Development report, the disease affects an estimated 1.5 million Americans, with 60,000 new cases diagnosed every year.

Progress against Parkinson’s has been difficult for several reasons – including limited knowledge about why cells die in Parkinson’s patients, the need for more translational research and the lack of a reliable “biomarker” for determining disease progression and severity, the PhRMA report explains.

Although a cure for Parkinson’s has not yet been found, current treatments and therapeutic approaches continue to improve patients’ lives.

“The medical management of Parkinson’s has become more expert and more complex,” says Joyce Oberdorf, President and CEO of the National Parkinson Foundation. “Physicians today have a much better toolkit than they had 30 years ago.”

These new tools can stave off the most serious complications of the disease, buying patients an average of five to seven years in the moderate symptom range, she says.

In addition to slowing the disease’s progression, today’s medications also manage symptoms more effectively and target symptoms that weren’t a clinical priority in the past. This includes mood and cognitive problems that can turn life into a daily struggle for many, Oberdorf says.

Physicians are also augmenting treatment with lifestyle changes, including exercise. Research has found that regular activity not only improves symptoms, but may help protect the brain from damaging changes, according to the National Parkinson Foundation.

People living with Parkinson’s disease can play a role in progress by helping test new advances. There are currently 43 active clinical trials for Parkinson’s disease, many of which are still looking to recruit participants, according to the PhRMA Medicines in Development report.

Experts believe that ongoing research will dramatically change the lives of those living with the disease over the next decade.

“Things are on the verge of a dramatic change, and that means that there is hope in the Parkinson’s community that did not exist 10 years ago,” says Oberdorf.

“More collaboration throughout the biomedical ecosystem will help better meet the needs of people living with Parkinson’s, and allow us to continue to make strides against this global public health challenge,” PhRMA’s Castellani adds.