Parkinson's Med

LRRK2 gene mutation affects Parkinson's disease risk

2010-02-01T07:42:00.001-08:00

A change in just one gene may affect the risk of Parkinson's disease. That could lead to genetic testing for Parkinson's, say scientists.

The gene in question is the LRRK2 gene. It's the topic of three studies in the Jan. 18 online edition of The Lancet.

A single mutation in the LRRK2 gene occurs in about 5% of inherited Parkinson's cases and 2% of isolated cases, experts report in The Lancet. The mutation was seen in familial Parkinson's disease as well as in patients without a family history of the disease.

The studies are from Italy, England, the U.S., Portugal, and Brazil. Two studies tracked the LRRK2 gene in patients with familial Parkinson's disease. For comparison, one of those projects also looked at the DNA of healthy people. The third study focused on people with Parkinson's disease without a family history of the disease.

All three studies suggest that the LRRK2 gene mutation affects Parkinson's disease risk.

"Our results suggest that a single LRRK2 mutation causes Parkinson's disease in 5% of individuals with family disease," say William Nichols, PhD, of Cincinnati Children's Hospital, and colleagues.

Likewise, another study linked the mutation to Parkinson's disease in four out of 61 families with the disease from Italy, Portugal, and Brazil. That's a little less than 7% of the families studied. The third study spotted the mutation in nearly 2% of isolated Parkinson's cases.

Tags: LRRK2

British All Party Parliamentary Group For Parkinson's Disease Announces Report & Parkinson's Disease Society Launches Fair Care For Parkinson's

2009-06-17T10:19:00.001-07:00

When: 8 July 2009 6:00pm - 8:00pm

What: Launch of the APPG's report into inequalities of access to services for people with Parkinson's and their carers.

In response to the report, the Parkinson's Disease Society will also launch Fair Care for Parkinson's, calling for the Government to ensure the APPG report recommendations are carried out.

Where: Macmillan Room, Portcullis House, Westminster

More information here.

St Jude Medical Completes Implants in U.S. Study of Deep Brain Stimulation for Parkinson's Disease

2009-06-17T08:40:00.001-07:00

St Jude Medical Inc. issued the following Press Release:

ST. PAUL, Minn.--(BUSINESS WIRE)--Jun. 8, 2009-- St. Jude Medical, Inc. (NYSE:STJ) today announced the completion of patient implants in its U.S. pivotal clinical study of deep brain stimulation (DBS) for the symptomatic treatment of Parkinson's disease, a neurological disorder affecting approximately 6.3 million people worldwide that progressively diminishes a person's control over his or her movements. The announcement was made at the Movement Disorder Society's 13^th International Congress of Parkinson's Disease and Movement Disorders in Paris.

"We are excited by the progress we've made in bringing the Libra^® deep brain stimulation systems to the market," said Chris Chavez, president of the St. Jude Medical Neuromodulation Division. "The completion of patient implants in this study and our recent European CE Mark approval represent significant steps towards our goal of providing physicians with an innovative deep brain stimulation system for treating Parkinson's disease."

Ongoing at 15 medical centers in the U.S., this randomized, controlled study is evaluating the St. Jude Medical Libraand LibraXP^™DBS systems, to determine the devices' safety and effectiveness in controlling many of the motor symptoms of advanced Parkinson's disease. The study is following 136 participants who have lived with the disease for more than five years and whose symptoms were insufficiently controlled with medication alone.

"Ultimately patients benefit from the development of new technologies," said Michele Tagliati, M.D., associate professor of neurology and division chief of movement disorders at Mount Sinai Medical Center in New York who enrolled the first patient in the study. "We are hopeful the Libra deep brain stimulation systems will prove effective at reducing the symptoms of Parkinson's disease and provide additional tools to better control this debilitating condition."

The Libra and LibraXP neurostimulators are constant current devices. The systems consist of a neurostimulator – a surgically implanted battery-operated device that generates mild electrical pulses – and leads, which carry the pulses to a targeted area in the brain.

"The progressive nature of Parkinson's disease often leads patients to a point where medication management alone can no longer adequately control the symptoms of the disease," said Bruno Gallo, M.D., assistant professor of neurology at the University of Miami in Florida and an investigator in the study. "Because these patients often become unable to care for themselves, we need to look for additional methods of treating this debilitating condition in order to help improve a patient's quality of life."

The National Parkinson Foundation (www.Parkinson.org) estimates that in the United States, more than 1.5 million people currently have the disease with 60,000 new cases diagnosed each year. According to the European Parkinson's Disease Association, as many as 6.3 million people are estimated to be affected by this disease worldwide.

For additional information about this study, visit www.PowerOverPD.com. St. Jude Medical is also currently developing new DBS applications to address a growing list of neurological disorders. Clinical studies are underway in the U.S. for depression and essential tremor. For more information about these studies, visit www.BROADENstudy.com and www.PowerOverET.com.

More than 45,000 patients in 35 countries have been implanted with St. Jude Medical neurostimulation systems. For more information about St. Jude Medical pain therapies, visit www.PowerOverYourPain.com.

About St. Jude Medical

St. Jude Medical develops medical technology and services that focus on putting more control into the hands of those who treat cardiac, neurological and chronic pain patients worldwide. The company is dedicated to advancing the practice of medicine by reducing risk wherever possible and contributing to successful outcomes for every patient. Headquartered in St. Paul, Minn., St. Jude Medical employs approximately 14,000 people worldwide and has four major focus areas that include: cardiac rhythm management, atrial fibrillation, cardiovascular and neuromodulation. For more information, please visit www.sjm.com.

Forward-Looking Statements

This news release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 that involve risks and uncertainties. Such forward-looking statements include the expectations, plans and prospects for the Company, including potential clinical successes, anticipated regulatory approvals and future product launches, and projected revenues, margins, earnings and market shares. The statements made by the Company are based upon management's current expectations and are subject to certain risks and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. These risks and uncertainties include market conditions and other factors beyond the Company's control and the risk factors and other cautionary statements described in the Company's filings with the SEC, including those described in the Risk Factors and Cautionary Statements sections of the Company's Quarterly Report on Form 10-Q for the fiscal quarter ended April 4, 2009. The Company does not intend to update these statements and undertakes no duty to any person to provide any such update under any circumstance.

Photos/Multimedia Gallery Available: http://www.businesswire.com/cgi-bin/mmg.cgi?eid=5980354&lang=en

Source: St. Jude Medical, Inc.

St. Jude Medical, Inc.
Investor Relations:
Angela Craig, 651-756-2191
acraig@sjm.com
or
Media Relations:
Guy Davis, 972-526-8227
gdavis@sjm.com

Interplay between cytosolic dopamine, calcium, and alpha-synuclein causes selective death of substantia nigra neurons.

2009-06-14T09:34:00.001-07:00

A team from Columbia University Medical Center, New York publishing in Neuron present research indicating "that elevated DA(cyt) and its metabolites are neurotoxic and that genetic and pharmacological interventions that decrease DA(cyt) provide neuroprotection. L-DOPA increased DA(cyt) in SN neurons to levels 2- to 3-fold higher than in VTA neurons, a response dependent on dihydropyridine-sensitive Ca2+ channels, resulting in greater susceptibility of SN neurons to L-DOPA-induced neurotoxicity. DA(cyt) was not altered by alpha-synuclein deletion, although dopaminergic neurons lacking alpha-synuclein were resistant to L-DOPA-induced cell death. Thus, an interaction between Ca2+, DA(cyt), and alpha-synuclein may underlie the susceptibility of SN neurons in PD, suggesting multiple therapeutic targets."

Novel Method to Stimulate Growth of New Neurons in Adult Brain

2009-06-12T09:51:00.001-07:00

University at Buffalo researchers have identified a new mechanism that plays a central role in adult brain stem cell development and prompts brain stem cells to differentiate into neurons.

Their discovery, known as Integrative FGFR1 Signaling (INFS), has fundamentally challenged the prevailing ideas of how signals are processed in cells during neuronal development.

The INFS mechanism is considered capable of repopulating degenerated brain areas, raising possibilities for new treatments for Parkinson's disease.

See the full story at Machines Like Us. Or read an abstract of the publication which appeared Integrative Biology.

Ceregene Presents Additional Clinical Data from Phase 2 Trial of CERE-120 for Parkinson's Disease

2009-06-11T09:28:00.001-07:00

Press Releases

05/27/09

-- Longer term follow-up indicates modest efficacy in primary and related endpoints

San Diego, CA - May 27, 2009 – Ceregene, Inc. today reported additional clinical data from a double-blind, controlled Phase 2 trial of CERE-120 in 58 patients with advanced Parkinson's disease. CERE-120 uses AAV-based gene therapy to deliver the neurotrophic factor, neurturin, to Parkinson's disease patients in order to restore the function and protect degenerating nigrostriatal neurons. The company previously announced that the Phase 2 trial did not meet its primary endpoint of improvement in the Unified Parkinson's Disease Rating Scale (UPDRS) motor off score at 12 months of follow-up, although several secondary endpoints suggested a modest clinical benefit.

The additional, protocol-prescribed analyses reported today focused on further analyses of the data from the 30 subjects who continued to be evaluated under double-blind conditions for up to 18 months which indicate increasing effects of CERE-120 over time. A clinically modest but statistically significant treatment effect in the primary efficacy measure (UPDRS motor off; p=0.025), as well as similar effects on several more secondary motor measures (p<0.05), were seen at the 18 month endpoint. Not a single measure similarly favored sham surgery at either the 12 month or 18 month time points. Additionally, CERE-120 appears safe when administered to advanced Parkinson's disease patients, with no significant concerns related to the neurosurgical procedure, the gene therapy vector, or the expression of neurturin in the Parkinson's disease brain.

The company also reported the results of analyses of neurturin gene expression in the brains from two CERE-120 treated subjects who died of causes unrelated to treatment. These analyses revealed that CERE-120 produced clear evidence of neurturin expression in the targeted putamen but no evidence for transport of this protein to the cell bodies of the degenerating neurons, located in the substantia nigra. In addition to the known cell loss in Parkinson's disease, these findings suggest that deficient axonal transport in degenerating nigrostriatal neurons in advanced Parkinson's disease impaired transport of CERE-120 and/or neurturin from putaminal terminals to nigral cell bodies, reducing the bioactivity of CERE-120. The data were presented today at the American Society of Gene Therapy Meeting in San Diego, CA by Raymond T. Bartus, Ph.D., Ceregene's executive vice president and chief scientific officer.

"While we were disappointed that our initial analysis of the data from this trial did not demonstrate a benefit of CERE-120 in the primary endpoint at 12 months, we are greatly encouraged by both the results of these protocol-prescribed analyses in patients who remained blinded for up to 18 months, as well as by the insight we gained," stated Dr. Bartus. "Collectively, these data suggest that CERE-120 is indeed exerting a unique and potentially important biological effect on the degenerating dopamine neurons in moderately advanced Parkinson's disease patients but that the inability of these neurons to efficiently transport neurturin back to their cell bodies compromises and delays the neurotrophic effects of neurturin in a manner that had not been anticipated. Importantly, we believe that we can overcome the transport problems of these degenerating neurons by modifying the dosing paradigm to also directly target their cell bodies in the substantia nigra with CERE-120."

"We remain optimistic that CERE-120 has the potential to significantly improve the treatment of advanced Parkinson's disease patients," stated Jeffrey M. Ostrove, Ph.D., president and chief executive officer of Ceregene. "The information gained from this initial controlled Phase 2 trial in advanced Parkinson's disease patients has been invaluable, and we can now incorporate these insights in a follow-on clinical trial that we are planning to initiate later this year. Our goal remains to significantly improve the symptoms of Parkinson's patients and also to provide the opportunity to delay further disease progression."

About Phase 2 Trial of CERE-120
Ceregene's Phase 2 trial was a double-blind, controlled clinical trial that completed enrollment of 58 patients with advanced Parkinson's disease in October 2007. This study was launched after successful execution of an extensive nonclinical program and preliminary evidence of safety and efficacy in advanced Parkinson's patients via an open-label Phase 1 trial in 12 patients. Patients in the Phase 2 trial were enrolled across nine leading academic medical centers in the United States, with two thirds of patients receiving CERE-120 and one third enrolled into a control group. Patients received a single administration of CERE-120 via stereotactic neurosurgery to deliver the drug into the putamen region of the brain and were followed for a minimum of 12 months for safety and efficacy, with over half the subjects followed for 15 to 18 months under blinded conditions, allowing longer-term analyses of the therapeutic effects of CERE-120. Ceregene gratefully acknowledges the financial support received from the Michael J. Fox Foundation for Parkinson's Research to help defray some of the costs of the CERE-120 Phase 1 and Phase 2 clinical trials.

About CERE-120 and its Application to Treating Parkinson's Disease
CERE-120 is composed of an adeno-associated virus (AAV) vector carrying the gene for neurturin, a naturally occurring protein known to repair damaged and dying dopamine-secreting neurons, keeping them alive and restoring normal function. Neurturin is a member of the same protein family as glial cell-derived neurotrophic factor (GDNF). The two molecules have similar pharmacological properties, and both have been shown to benefit the midbrain dopamine neurons that degenerate in Parkinson's disease. Degeneration of these neurons is responsible for the major motor impairments of Parkinson's disease. CERE-120 has been delivered by stereotactic injection to the terminal fields (i.e., the ends of the degenerating neurons), located in an area of the brain called the putamen. The cell bodies for these same neurons are located in a different area of the brain, called the substantia nigra. Once CERE-120 is delivered to the brain, it provides stable, long-lasting expression of neurturin in a highly targeted fashion. Genzyme Corporation (Nasdaq: GENZ) has licensed the ex-North American rights for the development and commercialization of CERE-120 from Ceregene, an agreement that was announced in June 2007.

About Parkinson's Disease
Parkinson's disease is a progressive movement disorder that affects a million people in the United States. Its main symptoms, stiffness, tremors and slowed movements and gait, are caused by a loss of dopamine-containing nerve cells in the substantia nigra, which project their axons to the putamen. Dopamine is a neurotransmitter involved in controlling movement and coordination, so Parkinson's patients exhibit a progressive inability to initiate and control physical movements. There is currently no treatment that can reverse the degeneration of these neurons, let alone cure Parkinson's disease.

About Ceregene
Ceregene, Inc. is a San Diego-based biotechnology company focused on the delivery of nervous system growth (neurotrophic) factors for the treatment of neurodegenerative and retinal disorders using gene delivery. Ceregene's clinical programs include CERE-110, an AAV2 based vector expressing nerve growth factor that is currently in a multi-center, controlled Phase 2 study for the treatment of Alzheimer's disease, and CERE-120 (AAV2-Neurturin) for Parkinson's disease. CERE-135 and CERE-140 are in preclinical development for ALS (Lou Gehrig's disease) and ocular disorders, respectively. Ceregene was launched in January 2001. The company's investors include Alta Partners, MPM Capital, Investor Growth Capital and Cell Genesys, Inc. (Nasdaq: CEGE) as well as Hamilton BioVentures and California Technology Partners.

Stem cell - gene therapy may cure human genetic diseases

2009-06-11T09:12:00.001-07:00

A study led by researchers at the Salk Institute for Biological Studies, has catapulted the field of regenerative medicine significantly forward, proving in principle that a human genetic disease can be cured using a combination of gene therapy and induced pluripotent stem (iPS) cell technology.

A team for the Salk Institute in the USA and colleague at the CMRB, and the CIEMAT in Madrid, Spain, decided to focus on Fanconi anemia (FA), a genetic disorder responsible for a series of hematological abnormalities that impair the body's ability to fight infection, deliver oxygen, and clot blood. Caused by mutations in one of 13 Fanconi anemia (FA) genes, the disease often leads to bone marrow failure, leukemia, and other cancers.

After taking hair or skin cells from patients with Fanconi anemia, the investigators corrected the defective gene in the patients' cells using gene therapy techniques pioneered in Verma's laboratory. They then successfully reprogrammed the repaired cells into induced pluripotent stem (iPS) cells

In an interview reported by Health News Track one of the leading members of the team stressed that research at this stage was limited to the laboratory, stating "We haven't cured a human being, but we have cured a cell,". Definitive proof of the efficacy of this approach must await transplanting the cells into patients.

The abstract of the Nature publication is available here.

MJFF Announces Launch of PD Online Research

2009-06-11T08:48:00.001-07:00

The Michael J. Fox Foundation for Parkinson's Research announced the launch of PD Online Research (pdonlineresearch.org), a resource-rich virtual workplace that MJFF hopes will become a vibrant Web-based community of PD researchers and funders collaborating and conversing across the globe. MJFF's goal in developing the site is simple: quicker knowledge turns and therefore faster progress in developing transformative new therapies for people with Parkinson's disease.

Read the full press announcement here.

Incorporate Gene Therapy To Make Stem Cell Treatment Safer, More Effective

2009-06-11T08:43:00.001-07:00

"Stem cell therapy offers enormous potential to treat and even cure serious diseases. But wayward stem cells can turn into a runaway train without a conductor," said Ronald G. Crystal, MD, chief of the Division of Pulmonary and Critical Care Medicine at New York Presbyterian Hospital/Weill Cornell Medical Center. "This is an issue that can be dealt with and we have the technology to do that in the form of gene therapy."

Full article from Medical News Today here.

Most Common Brain Cancer May Originate In Neural Stem Cells

2009-06-11T08:37:00.001-07:00

A team from the University of Michigan have published finding in the June 2 edition of Cancer Cell which indicate that
Glioblastoma, the type of cancer that afflicts U.S. Sen. Edward Kennedy and is diagnosed in about 10,000 Americans each year, may originate in neural stem cells located in a brain region known as the subventricular zone, or SVZ. In mice, neural stem cells that normally live in this niche give rise to more specialized nerve cells that migrate out of the niche. Cancer could begin with a single genetic mutation in the p53 gene, which makes stem cells migrate out of the niche like their specialized progenies.

For an article summarising the findings click here.

The article includes the following quote "The link between neural stem cells and this aggressive type of cancer is a warning sign for scientists to proceed carefully with new treatments for neurodegenerative diseases such as Parkinson's disease, where the hope is to use neural stem cells to help regenerate lost nerve function"

Transgenic Monkeys May Provide New Models of Diseases

2009-06-11T08:21:00.001-07:00

New experiments reignite old ethics controversies. Scientists have created a line of monkeys carrying a gene that encodes a green fluorescent protein integrated into their DNA and passed on to their offspring. The research, published this week by a group of Japanese and American scientists in the British journal Nature, marks the first such accomplishment in primates and could lead to new models of human diseases including Parkinson's disease. Genes thought to predispose patients to developing Parkinson's disease could be transferred to the germ cells (eggs or sperm) or monkeys giving rise to offspring would carry the genes and enable deeper study of their contribution to disease and how the disease could be treated.

To read the abstract from the Nature publication click here.

Mice With Parkinson's Disease Gene May Point the Way to New Treatments

2009-06-11T08:10:00.001-07:00

NEW YORK (June 7, 2009) — Scientists at Weill Cornell Medical College have developed a new mouse model of Parkinson's disease (PD) that successfully reproduces the impairments of movement and the degenerative brain changes that occur in the human disease. Their research, performed in collaboration with investigators at Columbia University Medical Center, appears in the June 7 issue of the journal Nature Neuroscience. For more information click here.

Canadian researchers turn skin tissues into stem cells

2009-04-05T04:12:00.000-07:00

Stem Cell Research Blog carries an important summary of work recently published in Nature.

In a breakthrough in medical sciences, Canadian researchers have pioneered a new method to turn skin tissues into stem cells.

Though American and Japanese scientists have recently turned human skin cells into cells that act like embryonic stem cells, they were forced to use a virus to help re-programme skin cells to develop into a state similar to stem cells. This risked damaging the DNA of the skin cells by the virus, raising the possibility of cancers.

Andreas Nagy of Toronto’s Mount Sinai Hospital, who led the research, used a novel method to introduce four re-programming genes into skin cells, restoring them to an embryonic-like state. Nagy and his team used a jumping gene or piggyback - which is a mobile piece of DNA - which (in species like moths and corn) hops from chromosome to chromosome, inserting itself randomly into the genome. Jumping genes create genetic variability in species, helping them to adapt to changing conditions. After triggering the restoration of stem cell like properties the modified gene was removed from the cells.

Full story here.

Nurr1 protects against neurotoxins that spur inflammation and Parkinson's disease

2009-04-05T04:02:00.000-07:00

A new study in the April 3rd issue of the journal Cell, a Cell Press publication, helps to explain why people who carry mutations in a gene known as Nurr1 develop a rare, inherited form of Parkinson’s disease, the most prevalent movement disorder in people over the age of 65.

They have found evidence that the gene normally acts to suppress an inflammatory response and, in turn, the production of neurotoxins in the brain. Those neurotoxins can otherwise spawn the damage to dopaminergic neurons that is characteristic of Parkinson’s disease. The findings not only offer new insight into the causes of the disease, but also may point to new avenues for therapy, according to the researchers.

Click for full story.

Experimental Parkinson's therapy may have robust weight-loss effect

2009-04-05T03:56:00.000-07:00

A growth factor used in clinical experiments to rescue dying brain cells in Parkinson patients may cause unwanted weight loss if delivered to specific areas of the brain, according to University of Florida researchers in the March online edition of Molecular Therapy. Full details here.

Google Founder Helps Patients with Cost of Gene Analysis

2009-04-03T05:25:00.000-07:00

Google founder Sergey Brin who recently discovered he carries a gene increasing his risk of developing Parkinson's disease has agreed to fund most of the cost of genetic analysis for 10,000 patients already diagnosed with the disease. Analyses will be performed by 23andMe, a genetics company part led by Brin's wife. As a result of Brin's offer the cost of genomic analysis will be reduced from $400 to only $25 (plus sample shipping charges). The offer is open on a first-come, first-served basis to the next 10,000 Parkinson's patients wishing to participate in the study and agreeing to provide necessary information about their disease. Wired blog carried the story and a link is available here. Another article can be found here.

Naturally Produced Estrogen May Protect Women From Parkinson's Disease

2009-04-02T07:09:00.000-07:00

Women who have more years of fertility (the time from first menstruation to menopause) have a lower risk of developing Parkinson's disease than women with fewer years, according to a large, new study by researchers at Albert Einstein College of Medicine of Yeshiva University.

"These findings, involving nearly 75,000 women, suggest that longer exposure to the body's own, or endogenous, hormones, including estrogen, may help protect the brain cells that are affected by Parkinson's disease. However the study showed no reduction in Parkinson's disease frequency in patients taking hormone therapy. More details here.

Video Lecture on DBS from Vanderbilt University Medical Center

2009-04-02T06:35:00.000-07:00

The link below is to a one hour video lecture covering DBS surgery for Parkinson's Disease at Vanderbilt University Medical Center, TN, USA. The lecture includes extensive video of the surgery. It shows the use of a smaller alternative to the traditional stereotaxic frame still used in many centers. Click here to view the video.

Interesting Lecture on Nerve Growth Factor

2009-03-21T10:44:00.001-07:00

We have included below a link to a lecture titled "Therapy for Alzheimer's Disease and Spinal Cord Injury" presented by Mark Tuszynski, M.D., Ph.D. Professor of Neurosciences & Director for the Center of Neural Repair

This laboratory studies anatomical, electrophysiological and functional plasticity in the intact and injured adult central nervous system. The staff focus in particular on the functional role of growth factors in modulating plasticity. Models studied in the lab include: 1) mechanisms of learning and memory in the intact adult brain, 2) plasticity and cell degeneration in models of aging and Alzheimer's disease, and 3) axonal plasticity and regeneration after spinal cord injury. In rodent and primate models of spinal cord injury, the staff examine the influences of growth factors and extracellular matrix molecules in modulating axonal responses to injury and the ability of these substances to promote axonal regeneration. In models of basal forebrain and cortical degeneration in rodents and primates, the ability of neurotrophic factors delivered by gene therapy to modulate cellular plasticity and survival. These studies are relevant to the understanding of aging and neuronal loss in Alzheimer's disease and also Parkinson's Disease.

Because of the relevence to Parkinson's disease and the clarity of the lecture a link is provided below.

Watch the YouTube video here

Spinal cord treatment offers hope to Parkinson's patients

2009-03-21T10:21:00.000-07:00

Spinal cord treatment offers hope to Parkinson's patients - Reported in the Daily Telegraph. Parkinson's disease patients are being offered fresh hope with a device that stimulates the spinal cord. Early research on mice and rats using the approach improved the slow, stiff movements associated with the condition, and experts believe that if the technique works in humans it could offer the possibility of a more normal lifestyle for Parkinson's patients. Reported in the journal Science (Nicolelis M. Vol. 323(5921) pp. 1578 - 1582. Abstract).

Clinical Research Study Now Enrolling People with

2009-01-26T06:50:00.000-08:00

While Parkinson's disease is recognized for its profound effects on movement such as tremors, less known is that 1 in 3 Parkinson's disease patients will develop changes that include hallucinations, illusions and delusions. According to Arcadia Pharmaceuticals "As Parkinson's disease progresses, up to 40 percent of patients can experience what is called 'Parkinson's Psychosis.' A condition in which patients experience changes in thought, behavior and judgment that can have devastating effects. In more advanced stages these symptoms include hallucinations, illusions and delusions where patients see things that aren't really there, believe things that are untrue, create false ideas and falsely accuse those closest to them." Arcadia, with sponsorship from the Michael J Fox Foundation is sponsoring a study aimed at improving the quality of life for people with Parkinson's disease and its behavioral complications. More details of the study and how to enrol are available at www.ParkinsonsMindStudy.com

Neurologix Commences its Phase 2 Clinical Trial of Novel Gene Transfer Approach for Treatment of Parkinson's Disease

2009-01-26T06:37:00.000-08:00

The following news-release is copied from Neurologix web site:

FORT LEE, N.J.--(BUSINESS WIRE)--Jan. 21, 2009--Neurologix, Inc. (OTCBB:NRGX), a biotechnology company engaged in the development of innovative gene therapies for the brain and central nervous system, announced today that it has initiated its Phase 2 clinical trial for the treatment of advanced Parkinson's disease. The first trial participants have undergone surgery at multiple institutions and additional subjects are currently being enrolled.

The purpose of the trial is to validate the safety and efficacy of Neurologix's gene transfer therapy, a novel non-dopaminergic approach to restore motor function in Parkinson's patients who are sub-optimally responsive to available drug therapy. Neurologix's approach is to reestablish the production of GABA (gamma-aminobutyric acid), the major brain inhibitory neurotransmitter that helps "quiet" excessive neuronal firing and has been determined to be deficient in patients in the advanced stages of Parkinson's disease.

John E. Mordock, President and Chief Executive Officer of Neurologix, stated, "Initiating this Phase 2 clinical trial represents a significant milestone. We expect to enroll 40 subjects across six to eight leading U.S. academic research centers, with completion of enrollment expected during the second half of 2009."

In Parkinson's disease there is degeneration of many cells in the central nervous system including those that produce dopamine, which leads to a downstream deficiency in GABA signaling in areas of the brain that regulate movement. Most current therapies and research approaches target dopamine. Mr. Mordock commented, "In contrast, our preclinical and clinical research suggests that directly targeting GABA production rather than dopamine replacement may be a more effective way of improving brain function in late-stage Parkinson's disease while also avoiding the known therapeutic limitations and complications associated with the over-production of dopamine."

The Co-Chairmen of the trial Steering Committee are Dr. Andrew Feigin, Director of the Neuroscience Experimental Therapeutics Research Program at the Feinstein Institute of Medical Research of the North Shore-Long Island Jewish Health System, and Dr. Peter LeWitt, a neurologist who directs the Parkinson's Disease and Movement Disorders Program at Henry Ford Hospital in Southfield, Michigan.

"Based on the encouraging functional and imaging results seen in the Phase 1 study of this innovative approach to improving Parkinson's disease, we are extremely excited to be part of this study," said Dr. Feigin.

Dr. LeWitt added, "The start of this clinical trial provides hope to the patient population which has had a longstanding need for new treatment options."

The scientific underpinnings of Neurologix's approach have undergone rigorous peer review resulting in highly cited articles in Nature Genetics and Science by the company's co-founders, Drs. Matthew During and Michael Kaplitt. Moreover, the current trial follows a successful Phase 1 study, as published in the Lancet and Proceedings of the National Academy of Sciences, USA, in which 12 subjects completed the study showing no related serious adverse events and significant functional benefit with supportive imaging data.

Phase 2 Clinical Trial Design

As previously announced, 20 participants will receive an infusion of the gene-based treatment bilaterally via a catheter temporarily placed by stereotactic surgery in each participant's subthalamic nucleus (STN), a deep brain structure that is the main target of surgery to treat Parkinson's disease. The other 20 participants will receive sterile saline solution into a partial thickness burr hole made into the skull, with no brain infusion. Trial participants will be assessed for treatment effects by standardized Parkinson's disease ratings at multiple time points post-procedure. The primary endpoint for the trial will be a clinical assessment of motor function at 6 months using the Unified Parkinson's Disease Rating Scale (UPDRS). All participants in the trial will also be monitored for safety for 12 months following the gene transfer procedure. If the primary endpoint is met following the analysis of 6 month data, then the sham-control participants will be offered the opportunity to crossover into an open label study of the Neurologix gene transfer therapy if they continue to meet all entry, medical and surgical criteria.

For details about participating in the clinical trial, please visit the following link: http://clinicaltrials.gov/ct2/show/NCT00643890?term=neurologix&rank=1. Details about trial participation are also available at http://www.pdtrials.org/en/browse/all/view/241.

About the Neurologix Gene Transfer Approach to Parkinson's Disease

In Parkinson's disease, patients lose dopamine-producing brain cells, resulting in substantial reductions in the activity and amount of GABA (gamma-aminobutyric acid). This reduction in GABA causes a dysfunction in brain circuitry responsible for coordinating movement. GABA is made by a gene called glutamic acid decarboxylase, or GAD.

Neurologix's gene transfer approach to Parkinson's disease seeks to restore GABA -- and thus improve the patient's motor control -- by inserting the GAD gene back into an area of the brain called the subthalamic nucleus, a key regulatory center for movement.

Toxicity Mechanism Identified For Parkinson's Disease

2009-01-05T01:15:00.000-08:00

(January 2, 2009) -- Alpha-synuclein is the main component of Lewy bodies, the clumps of aggregated proteins that form in the brains of Parkinson's disease patients. The alpha-synuclein gene is mutated or triplicated in some cases of inherited Parkinson's. A process called chaperone-mediated autophagy (CMA) plays an important role in recycling of specific proteins in brain cells. Alpha-synuclein disrupts a key survival circuit in brain cells by interfering with CMA and the recycling of the protein MEF2D. ... > full story

Carbon nanotubules may have future role in DBS

2008-12-24T06:16:00.000-08:00

New research is published showing that carbon nanotubules which are highly electrically conductive form extremely tight contacts with neuronal cell membranes. Carbon nano-electrodes could also be used to replace metal parts in clinical applications such as deep brain stimulation for the treatment of Parkinson's disease or severe depression. And they show promise as a whole new class of "smart" materials for use in a wide range of potential neuroprosthetic applications. Read more.

Fox Foundation launching “PD Online Reseach”

2008-12-24T06:06:00.000-08:00

The Michael J Fox Foundation has announced plans to launch “PD Online Research”. The Foundation describes the site as “the future home of a new web community of scientists, industry professionals, grant makers and financial investors dedicated to advancing the treatment, prevention, and cure of Parkinson's disease” and promises that the “PD Online Research community will work to build a sophisticated and continuously updated knowledge base of Parkinson's disease, and to actively guide research funding and investment through interaction with funders and financial investors”.
The site is currently in development and due to launch in the spring 2009 but is already seeking applications for membership. Click to here apply.