Saturday, November 8, 2014

WebMD Honors Michael J. Fox with First Health Heroes Hall of Fame Award

FoxFeed Blog

Posted by Christina Brdey, November 07, 2014

Last night Michael J. Fox was honored by WebMD in New York City with its first inaugural Health Heroes Hall of Fame award for his efforts in Parkinson’s disease research and advocacy. The WebMD Health Heroes Award recognizes inspiring individuals who have helped change the health care landscape by meeting a health challenge and giving back to others. Additional honorees were Carson Daly, Dr. Harold S. Koplewicz, Dr. Frank Papay, Zarin Ibnat Rahman and Martha Stewart.

Michael sat down with Robin Roberts, who hosted last night’s event, to discuss the honor and how he lives his life with Parkinson’s.

Accepting the award on Michael’s behalf, Claire Meunier, VP of research engagement, reiterated Michael’s sentiments on receiving this honor and that he shares the recognition with his fellow patients and all the researchers who are helping cure Parkinson’s disease in our lifetime.

Michael J. Fox's Crusade for a Parkinson's Cure

How the actor's Parkinson's diagnosis changed his life -- for the better, he says.

Michael J. Fox has always been a poster boy. With his youthful good looks and intelligent charm, he rose to fame playing a sassy Republican teenage son of ex-hippie parents in the TV sitcom Family Ties. In the blockbuster Back to the Future film trilogy, he was a time traveler with perfect comedic timing. And in a later sitcom, Spin City, he made us wish all politicians were as personable as his Deputy Mayor Mike Flaherty.

In 1998, Fox became a poster boy for another reason: He went public with the news he had Parkinson's disease, diagnosed 7 years earlier when he was 30. Parkinson's is marked by:

Trembling in the hands, arms, legs, jaw, and face

Stiffness of the body

Slow movements

Impaired balance and coordination.

The disease had become unmanageable for the actor, who until then was able to minimize his symptoms thanks to medication, surgery, and good timing. Eventually, the effort became too much.

"I needed every bit of those 7 years to say, 'I want to be out there,'" Fox says. "But at a certain point I woke up and said, 'What's the risk? That people will judge you? People are already judging you about whether you wear red shoes or blue shoes. So I talk funny or shake -- why should I restrict myself?'"

"You have to take your time and do what you need to do," he says. "But when you arrive at a place where you are no longer judging it, where there's no good or bad or right or wrong and it just is what it is, you accept it."

Much to his amazement, so did everyone else. While Fox feared becoming a sob story for the tabloids, he was met with huge support. Overnight, the actor beloved for his ability to make people laugh came to represent the face of an incurable illness that gets worse over time.

Laying the Foundation

Parkinson's disease develops due to the death of brain cells that make dopamine, a chemical crucial to balance, speech, and even memory. There's no cure, and the treatment -- generally a prescription for synthetic dopamine -- is far from perfect.

Regardless, the diagnosis turned out to be nothing short of a gift, Fox says. "Only when my body couldn't keep still was I able to find stillness in myself," he explains. "I think the key to it is the 12-step acceptance rule: 'My happiness grows in direct proportion to my acceptance, and in inverse proportion to my expectations.'"

Fox, now 53, turned the illness and his struggle with it into a gift for millions of others when he launched The Michael J. Fox Foundation for Parkinson's Research in 2000. Its mission is to fund and support research in the hope of discovering a cause, new treatments, and, ultimately, a cure. His celebrity has also helped raise awareness of Parkinson's, including a memorable appearance before Congress in 1999 when he spoke without using medication so people could see the ravages of the disease.

With a goal to move intelligently and quickly, Fox's foundation offers grants -- $450 million to date -- to researchers with remarkable speed. Angus Nairn, PhD, lead researcher for Yale's Michael Stern Parkinson's Research Foundation, says: "The NIH has cut back on research, but Parkinson's has been really fortunate, because The Michael J. Fox Foundation has been incredibly successful in doing things other people can't do on their scale. They have a different way of working, with a very fast turnaround funding research."

The foundation's approach comes from the founder himself. "Michael is the founder, but he is a patient first, and as a patient, he has a patient's sense of urgency," says Deborah W. Brooks, the foundation's co-founder and executive vice chairman.

Career View

Fox has always moved at lightning-fast speed. Raised along with three sisters and a brother by his mother and his father (a sergeant in the Canadian army), Fox discovered acting in high school. At 16, he won the lead in a Canadian series called Leo and Me. Enough work followed to give him the courage to quit high school his senior year and move to Los Angeles to seek acting work.

For several years, it looked like a bad decision, as he subsisted on fast food and residual checks from occasional parts. Then, in 1982, he won the role of Alex P. Keaton in Family Ties. By 1988, when he married actor Tracy Pollan (who he met on the set of Family Ties and who he calls "my bride, the one and only love of my life"), Fox was working nonstop in movies and television. He was making the Back to the Future trilogy, Teen Wolf, and Casualties of War while taping Family Ties.

Exhausted from his schedule and drinking heavily, Fox was on location in Florida filming Doc Hollywood in 1990 when his pinky began to twitch uncontrollably. A doctor linked it to an old injury Fox got while filming a stunt on Back to the Future. A year later, Pollan noticed that one side of her husband's body seemed rigid during a jog and insisted he see a neurologist. This time, there was no question: Fox was diagnosed with Parkinson's disease in September 1991.

The diagnosis made him among the approximately 10% of patients who have early onset Parkinson's -- the average age of those diagnosed is 60. As is the case with most people with Parkinson's, "by the time I had my first symptom, a twitching pinky, 80% of my dopamine-producing cells were already dead," Fox says. His doctors told him there was no cure. They could treat him with synthetic dopamine to replace the chemical deficiency caused by the disease, and he could expect to work for another decade. Fox pushed it as hard as he could, and a decade later, he took on the biggest role of his life: leading The Michael J. Fox Foundation.

"The first thing I wanted to do was put the pieces in place to move forward quickly and not keep good ideas on the shelf for too long," Fox explains of his mission. "As soon as ideas appeared, I wanted to give them the wings to fly."

While Fox has had a steep learning curve, he says he's had no problem being taken seriously by the research community. "They were like, 'Wow, grants? What line do we form in?' I think that the Parkinson's community is really excited to get the attention and have people interested in getting them to work."

Family Ties

The foundation is by no means the entirety of Fox's world. Most important to him, by his own account, is his family: He and Pollan have four children -- 19-year-old twins Aquinnah and Schuyler, who are in college; Esme, 13, a seventh-grader in New York City, where the couple live; and a son, Sam, 25, who lives nearby in Brooklyn.

"My family is the exception to the rule that 'what other people think of me is none of my business,'" Fox says. "I want them to be encouraged and emboldened by what I do and to see me as a refuge and resource." And yes, life in a large family can be chaotic, but the actor says it is within that tumult that he has learned to find a calm mind, much as he's found peace with the tremors of his disease. "Having a family means you don't always find moments of quiet, so you find quiet in the chaos.

Fox also continues to act. "I can play anybody as long as they have Parkinson's," he says with a laugh. In 2013, he starred in NBC's The Michael J. Fox Show, a comedy about a man with Parkinson's. "But it was more than I bargained for work-wise," he admits. The show ran for 19 episodes. Now, he enjoys roles like his guest-starring part on CBS's The Good Wife, playing an attorney with, yes, Parkinson's. And he's written two best-selling memoirs, Lucky Man and Always Looking Up: The Adventures of an Incurable Optimist.

"I've accomplished a lot already career-wise, so everything else is just gravy," Fox says. "What I really want is for our foundation to be so successful it goes out of business."

Pursuit of Happiness

Fox shares some of his hard-won wisdom about what it takes to be your best.

Accept what you can't change. "I think the key to my optimism was accepting my situation [as a person with Parkinson's]. When I saw it as just one of the things I was dealing with, then I could see the room around it."

Practice patience. When Fox does have a down moment, "I just wait it out."

Embrace family ties. "My family makes me a better person because they take me out of myself."

Let go of judgment. "When there's no 'good' or 'bad,' 'right' or 'wrong,' it just is what it is."

Go for it. "Being small growing up, I had to make an extra effort in things I did, and it opened up so many possibilities for me. 'Nothing ventured, nothing gained' was obvious to me from an early age."

Say yes. From actor to author and head of a foundation, Fox is open-minded: "Being yourself and taking risks -- what's the downside?"

Live well. Fox doesn't drink, watches what he eats, and exercises. "I can't run marathons anymore, but I hike and have a dog who walks me."

http://www.webmd.com/parkinsons-disease/features/michael-j-fox-

Friday, November 7, 2014

Damage to the brain caused by Parkinson's disease can be 'healed' using stem cells

Scientists at Sweden's Lund University have hailed study a 'breakthrough'
Found stem cells can heal the damage caused by Parkinson's disease
Parkinson's UK said there remain many questions before human trials

By BEN SPENCER, SCIENCE REPORTER FOR THE DAILY MAIL

PUBLISHED: 08:18 EST, 7 November 2014 | UPDATED: 09:14 EST, 7 November 2014ments

Stem cells can be used to heal the damage in the brain caused by Parkinson's disease, according to scientists in Sweden.

They said their study on rats heralded a 'huge breakthrough' towards developing effective treatments.

There is no cure for the disease, but medication and brain stimulation can alleviate symptoms.

The disease is caused by the loss of nerve cells in the brain that produce the chemical dopamine, which helps to control mood and movement.

Comedian Billy Connolly revealed last year he had been diagnosed with Parkinson’s.

The Scottish actor, 71, was diagnosed by a doctor in a hotel lobby, who spotted that his gait suggested he was showing early signs of the illness.

In the new study, to simulate Parkinson's, Lund University researchers killed dopamine-producing neurons on one side of the rats' brains.

They then converted human embryonic stem cells into neurons that produced dopamine.

These were injected into the rats' brains, and the researchers found evidence that the damage was reversed similar method has been tried in a limited number of patients.

It involved taking brain tissue from multiple aborted foetuses to heal the brain.

Clinical trials were abandoned after mixed results, but about a third of the patients had foetal brain cells that functioned for 25 years.

Using embryonic stem cells may be preferable, as it is easier to get hold of the large numbers of cells needed for transplant by growing them in the laboratory.

But Parkinson's UK has said there are still questions that must be answered before human trials can proceed. The disease is caused by the loss of nerve cells in the brain that produce the chemical dopamine, which helps to control mood and movement

It also opens up the possibility of using less ethically charged sources of stem cells, such as those made from adult tissue.

The charity Parkinson's UK said the research 'could be a stride towards clinical trials in people with Parkinson's'.

Its director of research and development, Arthur Roach, said: 'This important research is a key step along the way in helping us to understand how stem cells might shape future Parkinson's treatments.

'There are important potential advantages of these cells over the foetal-derived cells used in past cell transplantation work.

'This study could be a stride towards clinical trials in people with Parkinson's but there are still many questions that need to be answered before this development can be tested in people with the condition.'

Parkinson's stem cell 'breakthrough'

6 November 2014 Last updated at 19:56 ET

The study is "a stepping stone towards clinical trials", according to researcher Malin Parmar.

They said their study on rats heralded a "huge breakthrough" towards developing effective treatments.

There is no cure for the disease, but medication and brain stimulation can alleviate symptoms.

Parkinson's UK said there were many questions still to be answered before human trials could proceed.

The disease is caused by the loss of nerve cells in the brain that produce the chemical dopamine ,which helps to control mood and movement.

To simulate Parkinson's, Lund University researchers killed dopamine-producing neurons on one side of the rats' brains.

They then converted human embryonic stem cells into neurons that produced dopamine.

Parkinson's is one of the commonest neurodegenerative diseases

These were injected into the rats' brains, and the researchers found that the damage was reversed.

There have been no human clinical trials of stem-cell-derived neurons, but the researchers said they could be ready for testing by 2017.

Malin Parmar, associate professor of developmental and regenerative neurobiology, said: "It's a huge breakthrough in the field [and] a stepping stone towards clinical trials."

A similar method has been tried in a limited number of patients.

It involved taking brain tissue from multiple aborted foetuses to heal the brain.

Clinical trials were abandoned after mixed results, but about a third of the patients had foetal brain cells that functioned for 25 years.

Using embryonic stem cells may be preferable, as it is easier to get hold of the large numbers of cells needed for transplant by growing them in the laboratory.

It also opens up the possibility of using less ethically charged sources of stem cells, such as those made from adult tissue.

The charity Parkinson's UK said the research "could be a stride towards clinical trials in people with Parkinson's".

Its director of research and development, Arthur Roach, said: "This important research is a key step along the way in helping us to understand how stem cells might shape future Parkinson's treatments.

"There are important potential advantages of these cells over the foetal-derived cells used in past cell transplantation work.

"This study could be a stride towards clinical trials in people with Parkinson's but there are still many questions that need to be answered before this development can be tested in people with the condition."

http://health.einnews.com/article/233320444

Study Finds that Mental Health Issues Lead People with Parkinson's to Leave the Workforce

- Nov 06 2014

Mental health may have greater influence than motor symptoms do on the decision of people with Parkinson’s disease to leave the workforce sooner than they would have preferred. The findings are published in the August 22 online edition of Parkinsonism and Related Disorders.

With an average age of diagnosis of 60 years, more than half of people with PD are of working age at the time of diagnosis. In the early stages of their disease, most people – 94 percent of respondents in one study – say they want to continue working. Yet, there is little data on the emotional, physical, financial and other factors that cause people to leave their jobs.

Researchers led by Melissa Armstrong, M.D., of the University of Maryland followed 419 people with Parkinson’s who received care at the university’s movement disorders center for nearly four years, and who were still employed. The researchers tracked who left the workforce during that time and why.

Results

Of 419 participants with PD followed in the study, 224 with an average age of 62 had left the workforce by their last follow-up appointment, slightly higher than the average age of 61 for the general US population.
Of the 224 participants who were no longer working,150 (67 percent) retired, 59 (26 percent) went on disability and 28 (13 percent) left their jobs.
In comparison with participants who remained employed, those who left the workforce were more likely to be female, older, have a lower income and have lived with PD longer. They also had greater depression, anxiety and overall psychiatric distress and worse mental health-related quality of life, which were present at their first medical visit.
The working and non-working groups showed no significant differences in race, education, marital status and PD-related medical conditions.

What Does It Mean?

Being able to work is a major concern among people with Parkinson’s disease and is often one of the first questions individuals ask their doctors when diagnosed. Continuing to work enhances the quality of life for people with Parkinson’s who are employed.

Previous studies have showed conflicting results regarding the role of motor symptoms in job performance: one study showed rigidity, tremor and bradykinesiacontributing to inability to work, and another found that tremor and slowness were major challenges at work, but a third study found that cardinal motor symptoms did not predict the need to leave the workforce.

The major finding of this study is that psychiatric features in PD, such as depression and anxiety, were most associated with participants leaving the workforce, while movement symptoms were not. Overall, there is growing awareness that people with PD often have great difficulty with non-motor symptoms of the disease. This emphasizes the importance of addressing mental health in PD.

Further research on how PD affects people's ability to work is needed in order to develop preventive strategies, make therapeutic recommendations, advise people with PD in employment decisions, adapt the workplace to accommodate employees with PD, and develop guidelines for the timing of disability benefits.

Reference: Armstrong MJ, Gruber-Baldini AL, Reich SG, Fishman PS, Lachner C, Shulman LM (2014) Which features of Parkinson’s disease predict earlier exit from the workforce? Parkinsonism and Related Disorders. DOI: 10.1016/j.parkreldis.2014.08.005 http://dx.doi.org/10.1016/j.parkreldis.2014.08.005

Thursday, November 6, 2014

Transplant of stem-cell-derived dopamine neurons shows promise for Parkinson's disease

Date:

November 6, 2014

Summary:

Parkinson's disease is an incurable movement disorder that affects millions of people around the world, but current treatment options can cause severe side effects and lose effectiveness over time. In a new study, researchers showed that transplantation of neurons derived from human embryonic stem cells, hESCs, can restore motor function in a rat model of Parkinson's disease, paving the way for the use of cell replacement therapy in human clinical trials.Parkinson's disease is an incurable movement disorder that affects millions of people around the world, but current treatment options can cause severe side effects and lose effectiveness over time.

In a study published by Cell Press November 6th in Cell Stem Cell, researchers showed that transplantation of neurons derived from human embryonic stem cells (hESCs) can restore motor function in a rat model of Parkinson's disease, paving the way for the use of cell replacement therapy in human clinical trials.

"Our study represents an important milestone in the preclinical assessment of hESC-derived dopamine neurons and provides essential support for their usefulness in treating Parkinson's disease," says senior study author Malin Parmar of Lund University.

Parkinson's disease is caused, in part, by the death of neurons that release a brain chemical called dopamine, leading to the progressive loss of control over dexterity and the speed of movement. Currently available drug and surgical treatment options can lose effectiveness over time and cause serious side effects such as involuntary movements and psychiatric problems. Meanwhile, another approach involving the transplantation of human fetal cells has produced long-lasting clinical benefits; however, the positive effects were only seen in some individuals and can also cause involuntary movements driven by the graft itself. Moreover, the use of tissue from aborted human fetuses presents logistical issues such as the limited availability of cells, hampering the effective translation of fetal tissue transplantation as a realistic therapeutic option.

To rigorously assess an alternative hESC-based treatment approach, Parmar and lead study author Shane Grealish of Lund University transplanted hESC-derived dopamine neurons into brain regions that control movement in a rat model of Parkinson's disease. The transplanted cells survived the procedure, restored dopamine levels back to normal within five months, and established the correct pattern of long-distance connections in the brain. As a result, this therapy restored normal motor function in the animals. Importantly, the hESC-derived neurons show efficacy and potency similar to fetal neurons when transplanted in the rat model of Parkinson's disease, suggesting that the hESC-based approach may be a viable alternative to the approaches that have already been established with fetal cells in Parkinson's patients.

In a related Forum article published in the same issue, Roger Barker of Addenbrooke's Hospital and the University of Cambridge laid out the roadmap for taking stem-cell-derived dopamine neurons to the clinic for treating Parkinson's disease. "This involves understanding the history of the whole field of cell-based therapies for Parkinson's disease and some of the mistakes that have happened," he says. "It also requires a knowledge of what the final product should look like and the need to get there in a collaborative way without being tempted to take shortcuts, because a premature clinical trial could impact negatively on the whole field of regenerative medicine."

Story Source:

The above story is based on materials provided by Cell Press. Note: Materials may be edited for content and length.

Journal Reference:

Shane Grealish, Elsa Diguet, Agnete Kirkeby, Bengt Mattsson, Andreas Heuer, Yann Bramoulle, Nadja Van Camp, Anselme L. Perrier, Philippe Hantraye, Anders Björklund, Malin Parmar. Human ESC-Derived Dopamine Neurons Show Similar Preclinical Efficacy and Potency to Fetal Neurons when Grafted in a Rat Model of Parkinson’s Disease. Cell Stem Cell, 2014; 15 (5): 653 DOI: 10.1016/j.stem.2014.09.017

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Cell Press. "Transplant of stem-cell-derived dopamine neurons shows promise for Parkinson's disease." ScienceDaily. ScienceDaily, 6 November 2014. <www.sciencedaily.com/releases/2014/11/141106131845.htm>.

Scientists create Parkinson's disease in a dish

Date:

November 6, 2014

Source:

New York Stem Cell Foundation

Summary:

A team of scientists created a human stem cell disease model of Parkinson's disease in a dish. Studying a pair of identical twins, one affected and one unaffected with Parkinson's disease, another unrelated Parkinson's patient, and four healthy control subjects, the scientists were able to observe key features of the disease in the laboratory, specifically differences in the patients' neurons' ability to produce dopamine, the molecule that is deficient in Parkinson's disease.

A team of scientists led by The New York Stem Cell Foundation (NYSCF) Research Institute successfully created a human stem cell disease model of Parkinson's disease in a dish. Studying a pair of identical (monozygotic) twins, one affected and one unaffected with Parkinson's disease, another unrelated Parkinson's patient, and four healthy control subjects, the scientists were able to observe key features of the disease in the laboratory, specifically differences in the patients' neurons' ability to produce dopamine, the molecule that is deficient in Parkinson's disease. In addition, the scientists also identified a potential strategy for developing novel therapies for Parkinson's disease.

Attributed to a combination of genetic and nongenetic factors, Parkinson's disease has no completely effective therapy or cure. Parkinson's disease is moderately heritable, but the mechanisms of this inheritance are not well understood. While genetic forms of the disease exist, sporadic forms are far more common.

"The unique scenario of identical twins, one with this disease and one without, allowed our scientists an unprecedented look into the mechanisms of Parkinson's disease," said Susan L. Solomon, NYSCF Chief Executive Officer. "Advanced stem cell research techniques allow us to push the boundaries of science and see what actually goes wrong at the cellular level, step by step during the disease process."

DNA mutations resulting in the production of a specific enzyme called glucocerebrosidase (GBA) have been linked to a five-fold greater risk of developing Parkinson's disease; however, only 30% of individuals with this mutation have been shown to develop Parkinson's disease by the age of 80. This discordance suggests that multiple factors contribute to the development of Parkinson's disease, including both genetic and non-genetic factors. To date, there has been no appropriate model to identify and test multiple triggers leading to the onset of the disease.

In this study, published in Cell Reports, a set of identical twins, both with a GBA mutation, provided a unique opportunity to evaluate and dissect the genetic and non-genetic contributions to the development of Parkinson's disease in one twin, and the lack of disease in the other. The scientists made induced pluripotent stem (iPS) cells from skin samples from both twins to generate a cellular model of Parkinson's in a dish, recapitulating key features of the disease, specifically the accumulation of α-synuclein and dopamine deficiency.

Upon analyzing the cell models, the scientists found that the dopamine-producing neurons from both twins had reduced GBA enzymatic activity, elevated α-synuclein protein levels, and a reduced capacity to synthesize and release dopamine. In comparison to his unaffected brother, the neurons generated from the affected twin produced less dopamine, had higher levels of an enzyme called monoamine oxidase B (MAO-B), and poor ability to connect with each other. Treating the neurons with molecules that lowered the activity of MAO-B together with overexpressed GBA normalized α -synuclein and dopamine levels in the cell models. This suggests that a combination therapy for the affected twin may be possible by simultaneously targeting these two enzymes.

"The subject of Parkinson's disease discordant twins gave us an incredible opportunity to utilize stem cell models of disease in a dish to unlock some of the biological mechanisms of disease," said Dr. Scott Noggle, NYSCF Vice President, Stem Cell Research and The NYSCF -- Charles Evans Senior Research Fellow for Alzheimer's Disease. "Working with these various different groups and scientists added to the depth and value of the research and we hope our findings will be applicable to other Parkinson's disease patients and other neurodegenerative disorders."

In this particular scenario, genetic and stem cell analysis identified an avenue for a potentially useful combination therapy for the twin affected by Parkinson's disease and may be applicable more broadly to other Parkinson's patients. While this case study is unique, this type of research and cellular analysis could yield further clues to all cases of genetic and sporadic Parkinson's disease and other related neurological disorders.