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Saturday, October 31, 2015

Educational events connect local Parkinson's communities with resources and support


Thursday, October 29, 2015 1:00 am | Updated: 4:33 am, Sat Oct 31, 2015.


(BPT) - More than one year ago, The Michael J. Fox Foundation for Parkinson’s Research (MJFF) and AbbVie launched Partners in Parkinson’s, a strategic health initiative to provide resources to help the nearly 1 million Americans living with Parkinson’s disease (PD) optimize their care and live well with the disease. To date, nearly 8,500 people have attended Partners in Parkinson’s events across the country, and the free, day-long program is coming to one more location in 2015, and six additional U.S. cities on Saturdays in 2016.
Parkinson’s is a chronic, neurodegenerative disease with highly variable symptoms and progression. No two cases of PD are alike, making personalized care essential to better long-term outcomes. Yet, too few patients and their caregivers connect with resources that help them understand their care options and tailor a plan that evolves as the disease progresses. A 2014 Harris Poll survey found fewer than half of Parkinson’s patients and caregivers feel informed about where to turn for support.
Partners in Parkinson’s events take place on Saturdays and offer a comprehensive full-day program designed to help patients and families learn more about the disease, including what to expect as the disease progresses from a new diagnosis to more advanced stages. Event attendees have shared overwhelmingly positive experiences and report feeling more empowered after connecting with local Parkinson’s specialists and learning how they can get involved in their own PD community.
Pennsylvania resident Kate Harmon’s father, Bob Harmon, was diagnosed with PD in 2006 at age 58. Kate and Bob attended the Partners in Parkinson’s event in Philadelphia last year and were amazed by the support and resources available to benefit local patients.
“My dad is very active in his own disease care, but it hasn’t always been easy to find the right resources, and not everyone managing Parkinson’s disease has the support they need,” Kate says. “These events offer patients from early to advanced stages of the disease the chance to connect with a community of support, which is crucial with a complex disease like Parkinson's.”
The educational events are free of charge and feature panel discussions on the variability of PD, the latest updates in research toward improved treatments, and how to make the most of an appointment with a movement disorder specialist (a neurologist with specialized training in Parkinson’s disease). Breakout sessions cover how to build a health care team that addresses the many aspects of life with Parkinson’s and offer the opportunity for attendees to submit their own questions about the disease to a panel of experts. At the event’s resource fair, patients and caregivers can connect with local support and exercise groups, health care providers and advocacy organizations, all based in their own local community.
Debi Brooks, co-founder and executive vice chairman of The Michael J. Fox Foundation, says the events have fostered new connections within Parkinson’s communities.
“We’re excited to be connecting with patients and caregivers around the country — many of whom are new to us and have never had a chance to be at an event like this,” Brooks said. “Once folks come out, they’re amazed at the information and support that’s available to help them live well at every stage of Parkinson’s.”
To find an upcoming event near you, visit www.partnersinparkinsons.org.
http://www.michigansthumb.com/online_features/senior_living/article_e685ac66-3dea-53a0-bd9a-a710449a662e.html

'Living with Parkinson's disease means no games of Jenga or Buckaroo for us'





Brothers: Grant and Gary Jamieson have both been struck down with Parkinsons in their 20s

Wayne Walker reveals he has Parkinson's disease


October 30, 2015
Former All-Pro NFL linebacker and popular Bay Area sportscaster Wayne Walker has revealed he has been battling Parkinson's disease for the last five years.
In an interview with KPIX, where he was sports director from 1974 to 1994, Walker linked the disease to his 15 seasons with the Detroit Lions from 1958 to 1972.
The evidence suggests the cause is due to repetitive blows to the head.
By his count, Wayne had at least 20 concussions over the 200 games he played, and was knocked out cold twice. "I got knocked out totally and went back in the same game," said Wayne.
Parkinson's disease is a chronic disorder of the nervous system that affects motor skills and movement and worsens over time. Although it cannot be cured, its symptoms can be treated. 
The 79-year-old Walker, who lives in Idaho, told the station his medical expenses were being covered by the 88 Plan, a fund for players with dementia and related injuries named after former Baltimore Colts tight end John Mackey, who wore No. 88. Separately, the NFL has reached a billion-dollar settlement with retired players with brain injuries, which is being challenged by players who think it isn't enough. 
http://health.einnews.com/article/294391421/nQiBxwCRrwvo1vTX

Friday, October 30, 2015

Parkinson’s Researchers Uncover Key Molecular Connections


Finding of molecular links and targets make new therapies and earlier PD detection possibleUniversity of Dundeen scientists have uncovered the molecular mechanisms and targets under the effect of a specific gene mutation involved in Parkinson’s disease development and which could prove to be strong targets for new therapies and even early disease detection. The paper entitled “Phosphoproteomic screening identifies Rab GTPases as novel downstream targets of PINK1” was published in The EMBO Journal.

Mutations in the PINK1 gene have been associated with the development of Parkinson’s disease (PD). The gene encodes PTEN-induced putative kinase 1, an enzyme that protects cells against stress caused by mitochondrial dysfunction. Researchers have linked PINK1 activity to neuronal protections, and its loss of function is associated with the onset of Parkinson’s symptoms. Until now, the hypothetical targets of PINK1 activity were still unknown.
Previous research identified the presence of Lewy bodies, hallmark structures in PD, in the brains of families with PINK1 mutations. Moreover, loss of PINK-1 genetic activity in mouse and C. elegans models led to excess of mutant α-synuclein, a protein also linked to PD pathology. Over-expression of Rab8A and related Rab GTPases proteins was also found to be involved in the rescue of α-synuclein-related neurodegeneration.
A molecular link between PINK1 and α-synuclein pathways has been suggested but never discovered. Through a series of state-of-the-art technology, scientists led by Drs. Miratul Muqit and Matthias Trost, have now uncovered a novel pathway controlled by PINK1, where this gene targets and alters Rab GTPases activity, which in turn regulates cell growth and survival, and protects against neurodegeneration in brain cells.
“Parkinson’s disease is at present incurable and it is vital we understand the molecular mechanisms in order to design the next generation of therapeutics against the disease. In previous work we had outlined a single pathway for PINK1, but the discovery of an entire family of Rabs as PINK1 targets indicates a more complex network of pathways that, if disrupted, renders brain cells vulnerable to stress and ultimately to the development of Parkinson’s,” explained Dr. Muqit in a university press release.
Based on their results, researchers hypothesize that Rabs could be strong therapeutic targets for PD therapy drug design, while monitoring modifications in these proteins can be used as a biomarker for early PD diagnosis.
http://parkinsonsnewstoday.com/2015/10/29/parkinsons-researchers-uncover-key-molecular-connections/

Voyager Therapeutics Sets Terms for IPO



Voyager Therapeutics has filed an S-1 form with the Securities and Exchange Commission (SEC) for its initial public offering (IPO). Roughly 4.69 million shares are expected, within the range of $15 to $17, with an overallotment option for an additional 703,125 shares. At the maximum price, the entire offering is valued up to $91.64 million. The company intends to list on the Nasdaq Global Market under the symbol VYGR.

The underwriters for the offering are Cowen, Piper Jaffray, Nomura and Wedbush PacGrow.
This clinical-stage gene therapy company is focused on developing life-changing treatments for patients suffering from severe diseases of the central nervous system (CNS). The company focuses on CNS diseases where it believes that an adeno-associated virus (AAV) gene therapy approach that either increases or decreases the production of a specific protein can slow or reduce the symptoms experienced by patients and therefore have a clinically meaningful impact.
The company has created a product engine that enables it to engineer, optimize, manufacture and deliver its AAV-based gene therapies that have the potential to provide durable efficacy following a single administration directly to the CNS. The product engine has rapidly generated programs for five CNS indications, including advanced Parkinson’s disease; a monogenic form of amyotrophic lateral sclerosis, or a form of the disease caused by a single gene mutation; Friedreich’s ataxia; Huntington’s disease; and spinal muscular atrophy.
Voyager’s most advanced clinical candidate, VY-AADC01, is being evaluated for the treatment of advanced Parkinson’s disease in an open-label, Phase 1b clinical trial with the goal of generating human proof-of-concept data in the second half of 2016.
The founders and members of the management team have extensive experience in drug discovery and development and have pioneered significant advances within the fields of AAV gene therapy and neuroscience.

Voyager intends to use the net proceeds from this offering to fund the costs of future clinical development and to fund the costs of additional preclinical development, manufacturing and clinical development, including Phase 1 and later-stage clinical trials. The remainder of the proceeds will be used to fund working capital and other general corporate purposes.

http://247wallst.com/healthcare-business/2015/10/30/voyager-therapeutics-sets-terms-for-ipo/

Brown University creating mini-brains for medical research


- Associated Press - Friday, October 30, 2015
PROVIDENCE, R.I. (AP) - Scientists at Brown University say they are creating 25-cent miniature brains the size of a pencil dot and using them for medical research.
Molly Boutin, the lead co-author of a recently released study on the so-called mini-brains, tells WLNE-TV (http://bit.ly/1ioznBK ) scientists take apart brain cells from rats and restructure them into tiny three-dimensional balls. The brains produce electrical signals and perform the basic functions of a real brain, but are not capable of thinking.
Mini-brains require about two weeks to become fully functional.
Researchers say the brains let them test the effects of different drugs and diseases, such as Parkinson’s disease. They hope the affordable process will one day allow for patients to receive their own personalized specimens.
http://www.washingtontimes.com/news/2015/oct/30/brown-university-creating-mini-brains-for-medical-/

Thursday, October 29, 2015

Super-strong, genetically-engineered dogs -- Could they cure Parkinson's disease?

do not agree with experimenting on animals but it is news, so I am sharing this with you.
Margie





Hercules and Tiangou were genetically engineered and have twice the muscle mass of normal beagles.
By Will Heilpern, for CNN

Updated 11:05 AM ET, Wed October 28, 2015 

(CNN)In a medical breakthrough that is as terrifying as it is extraordinary, scientists in China say they have created dogs that are twice as strong as they would be naturally, through genetic engineering.
The process used could help prevent human diseases, according to scientists who led the study, which was published by the Journal of Molecular Cell Biology in mid-October.
        
"There is certainly the potential for this model to help fight human diseases. The process we have been developing could help prevent muscular dystrophy and Parkinson's disease," Professor Xiang Gao told CNN. 
Gao, who led the project with Liangxue Lai, is a specialist in genetic engineering at Nanjing University in China.
The two modified beagles, named Hercules and Tiangou, had the myostatin gene deleted at the embryo stage. Myostatin inhibits muscle growth in animals, so the dogs were able to grow unnaturally bigger, more muscular, and stronger.
The process involved the introduction of gene-editing chemicals into around 60 dog embryos. The myostatin gene was successfully knocked out of only two of the dogs.

"The mutant dogs look much stronger than the others. They are overgrown in the thighs," Gao said. "We have not observed any negative side effects. The dogs are not in pain."
Their larger size and strength is expected to allow them to be better and stronger runners. 
"Their extra strength means that they may have uses in hunting, even the military," said Professor Gao. 
Elaine Ostrander, a scientist at the National Institutes of Health, told the MIT Technology Review that we should not rush to conclusions from the study. "The number of dogs is still small ... It will be interesting to see what types of variation come up as more dogs undergo the process," she said. 

Super-muscular humans?

Dogs were used because they have metabolic and neurological features that are similar to humans -- and the same mutation has occurred in humans before. 
In 2003, a baby boy in Berlin was determined to be born without myostatin. As a result, he was incredibly strong. 
According to the New England Journal of Medicine, "He appeared extraordinarily muscular, with protruding muscles in his thights and upper arms." 


The Journal report states that by the time the child was less than five years old, he had increased strength and bulk, and was able to, "hold two 3-kg dumbbells in horizontal suspension with his arms extended."
Hercules and Tiangou -- named after the "heaven dog" in Chinese myth -- will remain at the Guangzhong Pharmaceutical Research Institute, where they will be bred.
"The next step in our study is to see whether or not the dogs can pass on the mutation to future generations," Gao said. "This will be another huge breakthrough."
Prof. Gao told CNN it was possible that humans could be genetically modified, like the beagles, to make stronger athletes or better soldiers. 
"However, genetically modifying humans raises other issues," he added.

Ethical Issues

Advancement in genetic engineering is creating a wealth of new opportunities in medical science, but it also raises difficult ethical dilemmas. 
Penny Hawkins, head of Research Animals Department at the Royal Society for the Prevention of Cruelty to Animals, told CNN: "The creation of genetically engineered animals can involve painful, invasive procedures on animals; including the removal of eggs and hormone treatment.
"Genetic alteration is never predictable and can result in oversized embryos, resulting in painful births. It can leave the animals severely affected in a way which is impractical for life. The process also very wasteful." 
In reference to the dog study, Hawkins said, "The genetic alteration of animals simply to make them stronger, or to have greater running ability, is completely unacceptable. 
Yes, now there's a micro pig cafe


"If the purpose of the study was to help cure human diseases, then there is more justification. Yet, even so, we ought to look for alternatives to genetic engineering, because the effect on these animals can be so great."

A different Chinese institute, BGI, caused controversy earlier this year when they genetically engineered "micro-pigs" to sell as pets. However, Prof. Gao made clear that there are no plans for the extra-muscular dogs to end up on sale as pets.

http://health.einnews.com/article/293963491/Fln9g-XdCip6OIOe