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I have Parkinson's diseases and thought it would be nice to have a place where the contents of updated news is found in one place. That is why I began this blog.

I copy news articles pertaining to research, news and information for Parkinson's disease, Dementia, the Brain, Depression and Parkinson's with Dystonia. I also post about Fundraising for Parkinson's disease and events. I try to be up-to-date as possible.

I am not responsible for it's contents. I am just a copier of information searched on the computer. Please understand the copies are just that, copies and at times, I am unable to enlarge the wording or keep it uniformed as I wish.

This is for you to read and to always keep an open mind.

Please discuss this with your doctor, should you have any questions, or concerns.

Never do anything without talking to your doctor. I do not make any money from this website. I volunteer my time to help all of us to be informed. I will not accept any information about Herbal treatments curing Parkinson's, dementia and etc. It will go into Spam.

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Saturday, May 9, 2015

New stem cell may overcome hurdles for regenerative medicine

Last updated: 

Scientists at the Salk Institute have discovered a novel type of pluripotent stem cell--cells capable of developing into any type of tissue--whose identity is tied to their location in a developing embryo. This contrasts with stem cells traditionally used in scientific study, which are characterized by their time-related stage of development.
Novel Stem Cell

In this image, a novel type of human stem cells is shown in green integrating and developing into the surrounding cells of a nonviable mouse embryo. Red indicates cells of endoderm lineage. Endoderm cells can give rise to tissue that covers organs from the digestive and respiratory systems. The new stem cells, developed at the Salk Institute, holds promise for one day growing replacement functional cells and tissues.
Credit: Salk Institute

In the paper, published in Nature, the scientists report using these new stem cells to develop the first reliable method for integrating human stem cells into nonviable mouse embryos in a laboratory dish in such a way that the human cells began to differentiate into early-stage tissues.
"The region-specific cells we found could provide tremendous advantages in the laboratory to study development, evolution and disease, and may offer avenues for generating novel therapies," says Salk Professor Juan Carlos Izpisua Belmonte, senior author of the paper and holder of Salk's Roger Guillemin Chair.
The researchers dubbed this new class of cells "region-selective pluripotent stem cells," or rsPSCs for short. The rsPSCs were easier to grow in the laboratory than conventional human pluripotent stem cells and offered advantages for large-scale production and gene editing (altering a cell's DNA), both desirable features for cell replacement therapies.
To produce the cells, the Salk scientists developed a combination of chemical signals that directed human stem cells in a laboratory dish to become spatially oriented.
They then inserted the spatially oriented human stem cells (human rsPSCs) into specific regions of partially dissected mouse embryos and cultured them in a dish for 36 hours. Separately, they also inserted human stem cells cultured using conventional methods, so that they could compare existing techniques to their new technique.
While the human stem cells derived through conventional methods failed to integrate into the modified embryos, the human rsPSCs began to develop into early stage tissues. The cells in this region of an early embryo undergo dynamic changes to give rise to all cells, tissues and organs of the body. Indeed the human rsPSCs began the process of differentiating into the three major cell layers in early development, known as ectoderm, mesoderm and endoderm. The Salk researchers stopped the cells from differentiating further, but each germ layer was theoretically capable of giving rise to specific tissues and organs.
Collaborating with the labs of Salk Professors Joseph Ecker and Alan Saghatelian, the Izpisua Belmonte team performed extensive characterization of the new cells and found rsPSCs showed distinct molecular and metabolic characteristics as well as novel epigenetic signatures--that is, patterns of chemical modifications to DNA that control which genes are turned on or off without changing the DNA sequence.
"The region selective-state of these stem cells is entirely novel for laboratory-cultured stem cells and offers important insight into how human stem cells might be differentiated into derivatives that give rise to a wide range of tissues and organs," says Jun Wu, a postdoctoral researcher in Izpisua Belmonte's lab and first author of the new paper. "Not only do we need to consider the timing, but also the spatial characteristics of the stem cells. Understanding both aspects of a stem cell's identity could be crucial to generate functional and mature cell types for regenerative medicine."

Adapted by MNT from original media release

Hope is given to those suffering from Parkinson's disease


Karen Anderson of the American Parkinson Disease Association introduces speaker Dr. John M. Bertoni at the "Living with Parkinson's Disease" discussion Friday morning at Midland University.By Sam Pimper/Fremont Tribune
A strong message of hope was given to people suffering from Parkinson’s disease Friday morning at Midland University.
More than 50 people learned the intricacies of the debilitating disease affecting more than 1 million people in the United States from Dr. John M. Bertoni, specialist and director of Parkinson’s disease Program at the University of Nebraska Medical Center.
The American Parkinson’s Disease Association reports that more than 60,000 will be diagnosed with Parkinson’s this year — about one person every 9 minutes.
Parkinson’s is a progressive nerve disorder that affects peoples’ movement, resulting in tremors, stiffness and slow movement that can affect posture, balance and cause many other adverse symptoms.
With 44 years in the medical field, Bertoni has seen the disease manifest in nearly every form. Parkinson’s ranges from minor symptoms that are nearly unrecognizable to those that hinder basic life functions on a daily basis.
The cause of Parkinson’s is unknown and a cure is still being worked toward. People suspecting they have Parkinson’s go through a variety of different tests and procedures, but definitively targeting the true problem is impossible in the living.
Bertoni’s main focus, though, wasn’t dwelling on the disease, rather, it was on how to deal with it and carry on with life as normally as possible.
With the disease hindering peoples’ movement and muscle use, it can feel like a hopeless situation, he said. But improving quality of life is possible if people are willing to put in the effort.
“There are as many ways as there are people out there,” Bertoni said of treatment options.
When it has been determined that a patient does in fact have Parkinson’s, it is crucial that treatment start as soon as possible. With delayed reaction to the problem comes an entirely new set of problems.
With proper medication — often times Levodopa — balanced with a controlled workout regimen, the disease is manageable and symptoms can drastically improve.
This doesn’t mean diving head on into physical activity, he said. Bertoni recalled an instance of a patient trying to attempt a physically demanding lifting regimen — he tore both of his rotator cuffs. The goal shouldn’t be facilitating change overnight, but rather progressively over time.
Understanding limitations is important.
“We are talking about the long haul here,” he said. “It’s not like you have to go out there and become 20 years old again tomorrow.”
Working with a team of medical professionals — generally occupational, speech and physical therapists — people will gain a general understating of what their best course of action is.
Physical activity is one of the most important undertakings someone with Parkinson’s needs to focus on.
While Bertoni’s patient not following a controlled workout regime wound up injured, another on a controlled workout regimen found great success.
Bertoni has a friend — and former physician — suffering from Parkinson’s who was nearly fully immobilized. Undertaking a monitored program, his muscles strengthened, and soon, was riding his bike again. Later he even started competing in bike races.
Just getting started is huge, he said, but taking it at a slow pace is a must.
“If you start on a treadmill and fall, you are like a piece of wood, and the belt is the sander,” he said.
Caring Senior Service, the event sponsor, hosts a support group the third week of every month between 10 and 11 a.m., featuring a speaker each session. Meetings are held at 1455 N. Bell St. in Fremont.
Next month, Terry Nelson, a physical therapist from West Point, will be the key speaker. Nelson started an exercise group for Parkinson’s patients and is seeing results.
Fremont is making a push toward bringing in someone to start a similar group, said Mary Hilgenkamp, a registered home care consultant.
While medication and physical activity help, nothing gets better without a positive attitude and a burning sense of motivation, Bertoni said.In this situation you are like an athlete,” he said. “You are the only one who can run life’s race.

Friday, May 8, 2015


ND0612H and ND0612L U.S. Clinical Development Cleared to Proceed

/EIN News/ -- REHOVOT, Israel, May 8, 2015 (GLOBE NEWSWIRE) -- NeuroDerm Ltd. (Nasdaq:NDRM), a clinical-stage pharmaceutical company developing drugs for central nervous system (CNS) diseases, today announced that the U.S. Food and Drug Administration (FDA) has lifted the clinical hold on U.S. clinical studies of ND0612H and ND0612L, the company's primary product candidates, that are based on proprietary, subcutaneously-delivered Levodopa/Carbidopa (LD/CD) liquid formulation, for the treatment of Parkinson's disease. The hold was lifted after the FDA reviewed additional information related to the product candidates' delivery devices. U.S. clinical development of these product candidates is therefore cleared to proceed in the second half of 2015.
"Having lifted the clinical hold in the first half of 2015 means that our U.S. clinical development program of ND0612H and ND0612L is proceeding on track," said Oded Lieberman, PhD, CEO of NeuroDerm. "Parkinson's patients have been hoping for a less invasive, non-surgical alternative that can deliver levodopa continuously. We remain committed to the execution of our plan to bring these product candidates to the market as soon as possible, and to make a significant impact on the lives of Parkinson's patients."
In June 2014, the FDA placed a hold on the U.S. clinical development of ND0612H and ND0612L, requesting additional information on the accuracy, safety, and compatibility of the devices used to deliver the drug. The company completed the required compatibility study and submitted the requested additional information to the FDA. Following the FDA's decision to lift the clinical hold, the company's U.S. clinical development program is now cleared to proceed, with several studies anticipated to commence in the second half of 2015.
About Parkinson's Disease
Parkinson's disease is a progressive neurodegenerative illness characterized by reduced dopamine in the brain, resulting in a debilitating decrease in the patient's motor and non-motor functions. Its symptoms, such as trembling in the extremities and face, slowness of movement and impaired balance and coordination, worsen over time and gravely impact the patient's quality of life. As the disease progresses, these symptoms become more severe, resulting in debilitating periods of decreased motor and non-motor functions, also referred to as "off" time. In addition, mainly as a result of excessive/intermittent oral doses of levodopa aimed at treating the "off" time, some patients experience involuntary movements, or dyskinesia. The "off" time and dyskinesia affect the majority of Parkinson's disease patients and interfere with day-to-day functions, causing patients to become severely disabled. Continuous administration of levodopa has been shown to effectively treat motor fluctuations in Parkinson's disease patients, however, a convenient route of continuous administration has not been introduced to date.
About Levodopa
Oral administration of LD/CD is regarded as the "gold standard" treatment for patients suffering from Parkinson's disease. Levodopa crosses into the brain and converts into dopamine to complement the reduced brain-dopamine levels. Virtually all patients diagnosed with Parkinson's disease will require levodopa at some point over the course of their treatment for the disease, and 70% to 80% of patients receive the drug at any given point in time. However, levodopa is limited by its short half-life. Approximately three to four hours after a single dose, almost none of the drug remains in the plasma and patients are required to take multiple LD/CD doses daily. This results in sharp fluctuations in levodopa levels which are associated with erratic "off" and "on" periods experienced by many patients. In addition, levodopa suffers from low absorption when administered orally, with only about 30% of the levodopa entering the blood stream. Continuous levodopa administration can overcome this limitation, but steady levodopa delivery can currently only be achieved after undergoing an invasive surgical procedure whereby a tube is permanently implanted into the duodenum, the upper part of the small intestine.
ND0612H, ND0612L
ND0612H and ND0612L are designed to significantly reduce motor complications in Parkinson's disease patients through continuous, subcutaneous delivery of LD/CD. Recently completed phase II trials demonstrated that ND0612L maintained steady, therapeutic levodopa plasma concentrations that were associated with major improvements in several clinical parameters including "off time" reductions when added to optimized oral standard of care. ND0612H, intended for severe Parkinson's disease patients, was shown to reach even higher levodopa steady plasma levels, indicating that it may provide an effective therapy alternative to current treatments requiring surgery such as deep brain stimulation and DuoDopa/Duopa®.
About NeuroDerm
NeuroDerm is a clinical-stage pharmaceutical company developing central nervous system (CNS) product candidates that are designed to overcome major deficiencies of current treatments and achieve enhanced clinical efficacy through continuous, controlled administration. In Parkinson's disease, the company has four product candidates in different stages of development which offer a solution for almost every Parkinson's disease patient from the moderate to the very severe stage of the disease. The company has developed a line of LD/CD product candidates administered through small belt pumps that deliver a continuous, controlled dose of LD/CD. The LD/CD line of product candidates includes: ND0612L and ND0612H, delivered subcutaneously, for moderate and for advanced Parkinson's disease patients, respectively, and ND0680 for a subset of severe Parkinson's disease patients whose symptoms have advanced to a highly advanced stage, requiring even higher doses of LD/CD. In addition, NeuroDerm is developing ND0701, a novel subcutaneously delivered apomorphine formulation for patients who suffer from severe Parkinson's disease and who do not respond well to LD/CD. NeuroDerm is headquartered in the Weizmann Science Park in Rehovot, Israel.
Forward-Looking Statements
This press release contains forward-looking statements, within the meaning of the safe harbor provisions of the Private Securities Litigation Reform Act of 1995, Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended that involve risks and uncertainties. Such forward-looking statements may include projections regarding our future performance and may be identified by words like "anticipate," "assume," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "future," "will," "seek" and similar terms or phrases. The forward-looking statements contained in this press release are based on management's current expectations and projections about future events. There are important factors that could cause our actual results, levels of activity, performance or achievements to differ materially from the results, levels of activity, performance or achievements expressed or implied by the forward-looking statements. In particular, you should consider the risks provided under "Risk Factors" in our annual report on Form 20-F for the year ended December 31, 2014 filed with the Securities and Exchange Commission. Any forward-looking statement made by us in this press release speaks only as of the date hereof. Factors or events that could cause our actual results to differ may emerge from time to time, and it is not possible for us to predict all of them. We undertake no obligation to publicly update any forward-looking statements, whether as a result of new information, future developments or otherwise.
CONTACT: NeuroDerm Contact:
                         Oded S. Lieberman, PhD, MBA, CEO
                         Tel.: +972-8-946 2729
                         Cell: +1-617-517 6077
                         U.S. Investor/Media  Contact:
                         David Carey
                         Lazar Partners Ltd.

Wednesday, May 6, 2015

Kinect hacked to allow Parkinson's sufferers to walk the line


May 6, 2015
Amin Amini Maghsoud Bigy and Dr Konstantinos Banitsas with the Kinect modified to help Parkinson's patients overcome Freezing of Gait
Amin Amini Maghsoud Bigy and Dr Konstantinos Banitsas with the Kinect modified to help Parkinson's patients overcome Freezing of Gait
Most will be familiar with the telltale shaking of Parkinson's disease, but that isn't the only symptom sufferers must endure. They must also contend with what is known as Freezing of Gait (FOG), where the sufferer's muscles can freeze mid-stride, making them feel like their feet are glued to the ground or resulting in them falling over. Researchers at Brunel University London have hacked a Kinect sensor to overcome this.
Research has previously shown that a dot or lines projected onto the ground in front of Parkinson's sufferers can provide a visual cue that helps them "unfreeze" the muscles and devices have been developed to take advantage of this. However, these currently take the form of devices incorporate motion sensing technology that need to be worn.
Now Dr Konstantinos Banitsas and PhD candidate Amin Amini Maghsoud Bigy have taken a different approach. They have hacked Microsoft's Kinect motion sensor, teaming it with a ceiling-mounted laser to create a system that can be installed into a patient's home. When the system detects a FOG incident, the ceiling laser projects lines onto the floor to counter the muscle freezing. Additionally, if the person falls, the system will automatically trigger a video call for help.
"By mounting the laser guide marker on the ceiling it can provide the visual clues in any direction," says Dr Banitsas. "And it is only activated when a FOG incident occurs instead of having to be worn constantly."
Dr Banitsas adds that system has passed the proof of concept stage and he and Bigy are set to begin patient trials shortly.

MJFF, Prothena to accelerate new biomarkers discovery for Parkinson's disease

PBR Staff WriterPublished 05 May 2015
The Michael J. Fox Foundation for Parkinson's Research (MJFF) and Irish biotechnology firm Prothena have entered into an agreement to jointly accelerate the discovery of new biomarkers and development of assays to measure Parkinson's disease (PD) progression.

The joint collaboration will focus on biomarkers that may facilitate therapeutic approaches targeting alpha-synuclein, a protein potentially involved in the onset and progression of PD.
MJFF CEO Todd Sherer said: "As more potential therapies come closer to and cross the line to clinical testing - and, in parallel, the number of people with Parkinson's grows as the population ages - the need for Parkinson's biomarkers grows more urgent."
Under the deal, the two parties will support scientific research studies directed toward biomarkers of disease progression and drug efficacy.
Additionally, they will work to identify opportunities where new endpoints or assays may be incorporated into ongoing or future studies.
Prothena president and chief executive officer Dale Schenk said: "We are committed to identifying new insights about the underlying cause and progression of Parkinson's disease and believe that biomarkers clearly defining disease progression may both enhance this understanding and enable more effective, efficient clinical development of disease-modifying therapeutics for patients and families impacted by Parkinson's disease.
"In addition, we believe this collaboration with MJFF will help inform the clinical development strategy for PRX002, a monoclonal antibody for the potential treatment of Parkinson's disease.
"We look forward to reporting results from our ongoing Phase I multiple ascending dose study of PRX002 in patients with Parkinson's disease during the first half of 2016."

Parkinson's and Neuropathic Pain Reviewed by NeuroPerspective

PR Newswire
CARDIFF, Calif.May 6, 2015 /PRNewswire/ -- NI Research (NIR) has released the May/June issue of NeuroPerspective, which features comprehensive reviews of two major areas: Parkinson's and Neuropathic Pain. 
Parkinson's is one of the major neurodegenerative disorders associated with aging, and while it does not approach the scale of Alzheimer's, approximately one million US patients suffer from PD. Current treatment options are purely symptomatic, and dopaminergic supplementation exacts a high long-term price in exchange for a few years of improved motor symptoms.
"It has become clear that PD is far more than just a disease of motor control areas: Cognitive deficits loom large as a major source of distress and disability. There are improvements on the symptom-relief theme that are late in clinical development and very likely to arrive; the same cannot be said of disease-modification, where many of the issues that have plagued Alzheimer's research are also impediments to disease-modification in PD--other than Parkinson's research has been less vulnerable to premature closure on the question of mechanism. But the questions of what therapeutic payload, delivered by which modality, to which anatomical locations, in what form and quantity, have yet to be definitively answered. But there has been a revival of neurotrophic research which, while facing all of these unknowns, may now be in a position to succeed after a history of frequent disappointments from 1995-2005. Novel targets, such as Gcase, are becoming more prominent and are receiving the funding they need to move ahead," said NeuroPerspective publisher Harry Tracy PhD.
Neuropathic pain is a highly heterogeneous disorder, which has numerous pharmacological treatment options available, none of which constitute anything more than a partial solution, providing incomplete relief to a subgroup of patients. The casualty rate in neuropathic programs over the past five years has been virtually identical to that in Alzheimer's: Many formerly promising novel mechanisms have turned into disappointments, but AT2 and Nav 1.7 antagonism have survived, and head the list of potential new avenues to neuropathic pain treatment.
The May/June issue also offers Company Reviews of MedGenesis Therapeutix and Cynapsus Therapeutics; discussion and commentary regarding recent events and topics, including NeuralStem's Phase II results in ALS; the limitations in Biogen's aducanumab results-thus-far; and more. Released as a 63 page pdf.
About NI Research
NI Research is the leading publisher of independent research on the neurotherapeutics industry, and has developed an unmatched information base regarding both publicly and privately held CNS companies. Since 1995, NeuroPerspective (formerly NeuroInvestment) has been the authoritative, independent, review of the neurotherapeutics area, providing critical analyses of therapeutics-in-development. 
A one-year (1-5 user) subscription to NeuroPerspective is $2450. The May/June issue is also being made available for single-issue purchase; $400.
NI Research is also preparing the 2015-16 edition of NeuroLicensing, which will comprehensively review current licensing trends in the CNS area, and evaluate large and midsize pharma companies in terms of their licensing agendas and performance. 
In addition to our publications, NI Research's Second Opinion service provides consultation to the pharma/biotech industry regarding licensing/development strategies.
Further information and online purchasing with immediate download are available at
Contact: NI Research, P.O. Box 1028, Cardiff CA 92007; 760.753.6376, Email
Harry Tracy

SOURCE NI Research

Tuesday, May 5, 2015

Dancing proves a good move for people with Parkinson’s disease

Parkinson’s disease is a neurological condition that causes tremor, rigidity and slowness of movement, among other things. It affects around 127,000 people in the UK, and there is no cure. But while it is progressive and debilitating, getting the right medication and support after diagnosis can make all the difference to quality of life. One of the most difficult, yet crucial recommendations is to keep active – because mood, flexibility and balance can all be improved by activity.
Alison Underwood, who is in her 60s, was diagnosed with Parkinson’s 10 years ago and now runs a support group in the Llandudno area in Wales for others with the condition. “I set up a monthly dance group, that many of us find physically helpful and enjoyable,” she explains. “We do a lot of ‘free’, expressive dancing, and there is considerable emphasis on the fact that it doesn’t matter if you’re sitting or standing. The positive effect that dancing has on us is quite magical.”
Only 1% of people with Parkinson’s in the UK live within reach of a dance practitioner specifically trained to work with them. It was this statistic that inspired People Dancing, a community dance organisation, to join the US-based Dance for Parkinson’s group in putting together an online training programme to help dance teachers better meet their needs.

The online programme enables dance teachers to learn more about the condition, and helps them to adapt their lessons to make them more appropriate – and beneficial – for those with Parkinson’s. Anna Leatherdale, producer at People Dancing, is a dance practitioner and a member of the Dance for Parkinson’s Network UK. “The more that people with Parkinson’s can engage with dancing, the more benefits they’ll feel,” she explains. “So if an hour’s dancing means an hour’s benefit, and you’re only able to get to a dance group once a month because there are so few teachers, that means you’ll only be feeling an hour’s benefit a month. It’s not much.”
 Dance teachers have been working with people with Parkinson’s for more than a decade in the US, thanks to pioneering work at the Brooklyn Parkinson Group and Mark Morris Dance Group in New York. “However, interest has been growing in the UK in the past few years, and we’re now seeing more of a demand for dance classes of this type,” says Leatherdale.

The Dance for Parkinson’s Network UK supports experienced teachers in providing classes – including freelance dance and music artists, as well as artists from English National Ballet and Pavilion Dance South West. Teachers work in a range of dance styles, often teaming up with local support groups.
 Underwood was inspired to set up her own group after attending a conference at which Dr Peter Lovatt, a dance psychologist also known as “Dr Dance”, explained how dancing benefits people with Parkinson’s. “We all seem to move more fluently, without bumping into others, which is difficult to do when you have Parkinson’s,” says Underwood.
She continues: “The opportunity for intentional physical contact in dance is important, too. We often work closely with a partner. When you have Parkinson’s, you can withdraw emotionally – I suppose you don’t feel as comfortable in your own body any more. Physically touching each other, while dancing, helps us to overcome inhibitions and to relax. This is useful because stress and tension exacerbate symptoms dramatically. Dancing leaves us all smiling, and we return home re-energised and ready to deal with the realities of life with Parkinson’s.”
Because the social element is so beneficial, the online training programme emphasises the importance of working with friends and carers, as well as with people with Parkinson’s. According to Parkinson’s UK: “Some people find gentle exercise, including dance, can help them to move with greater ease and gain some relief from symptoms, as well as improving mood. The social benefits of being active and doing something enjoyable with others can be just as important.”
Leatherdale has seen these benefits first hand. “By giving people with Parkinson’s an opportunity to dance, you give them confidence in their own bodies again. I’ve met people who have thought: ‘I’ve got a movement disorder – I can’t move any more.’ But by concentrating on a clear beat, rather than the physical act of moving, shuffling can, temporarily, turn back to walking. Then people can take what they’ve experienced into their day-to-day lives, for example by wearing headphones and listening to music with a beat while they’re out shopping.”
Imagery used in dancing can really help too. “Instead of saying to someone, put your arms out and move them around in a circle, we might say, pretend your hands are tracing ripples on water,” says Leatherdale. “By focusing on the image rather than the action, it becomes a lot easier. With Parkinson’s, you can lose a lot of facial expressions, but one of the best things is seeing the joy that emerges when people with Parkinson’s rediscover the delight that dancing with other people can bring.”
Joyce Plaice has been attending lessons in Plymouth for the past two years: “Dancing is the opposite of life with Parkinson’s, which I find is a slow, depressing slog – like walking through treacle. The tight muscles remind me of new knicker elastic with little give. But when I hear the music, the muscle tension eases and my mood lifts. I am doing what normal people do: socialising, making physical contact, using my brain to learn new steps. And my body gets a healthy boost with a much wider range of movements than it would get during the course of my day.”

UNC Team uses cellular bubbles to deliver Parkinson's meds directly to brain

Researchers from the University of North Carolina at Chapel Hill show that exosomes loaded with catalase (shown in red) efficiently interact with neurons (shown in black) to protect them from the effects of Parkinson's disease.

Pharmaceutical researchers at UNC are the first to use exosomes -- lipid-and-protein spheres produced by cells -- as vehicles to deliver a potent large-molecule drug to the brain

May 4,2015
Researchers at the University of North Carolina at Chapel Hill have used exosomes -- tiny bubbles of protein and fat produced naturally by cells -- to bypass the body's defenses and deliver a potent antioxidant directly to the brain to treat Parkinson's disease.
And what's the best way of getting her drug-packed exosomes to the brain? It looks like a simple nasal spray will do the trick, say Elena Batrakova and her colleagues at the UNC Eshelman School of Pharmacy's Center for Nanotechnology in Drug Delivery.
Batrakova and her colleagues extracted exosomes from immune cells and successfully loaded them with the enzyme catalase, a potent antioxidant that counters the neuron-killing inflammation responsible for Parkinson's and other degenerative neurological disorders. Their work was published in the Journal of Controlled Release.
This is the first time a large therapeutic protein like catalase has been delivered to the brain using exosomes. Getting drugs into the brain is extremely difficult in general because it is protected and isolated from the rest of the body by the blood-brain barrier, which is extremely selective about what is allowed to pass through.
Batrakova and her team at the pharmacy school harvested exosomes from macrophages, white blood cells that are responsible for clearing foreign material from the body. Exosomes are tiny spheres produced by cells to carry chemical messages. They are made of the same material that makes up cell membranes. Diseases like cancer and AIDS propagate throughout the body by hijacking exosomes.
"Exosomes are engineered by nature to be the perfect delivery vehicles for proteins and genetic material," Batrakova says. "Catalase is a huge protein, and it is almost impossible to deliver across the blood-brain barrier alone. We use exosomes from white blood cells, which are invisible to the immune system and easily interact and fuse with the blood-brain barrier to deliver their cargo across it."
Catalase counteracts the effects of free radicals, destructive molecules that are byproducts of cellular activity and especially prevalent in areas of chronic inflammation.
"Catalase is one of the most potent antioxidants in nature," Batrakova says. "One molecule of catalase can deactivate about one million free radicals per second, and it never stops because the enzyme is not consumed in the reaction. No small molecule drug even comes close to matching it in speed or efficiency."
Traditional drugs -- from cold medicine to chemotherapy -- are composed of small molecules of a few dozen atoms, typically. Biopharmaceuticals, or biologics, are proteins produced by living cells. Proteins such as catalase are tens of thousands of times larger than the small molecules that make up traditional drugs.

Batrakova's goal is to develop personalized treatments by loading proteins into exosomes that have been extracted from a patient's own white blood cells. These packages of medicine will be ignored by the patient's immune system, which works against unknown proteins as well as many synthetic delivery vehicles.