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 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 13th 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 Libra "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.
and 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.
Investor Relations:
Angela Craig, 651-756-2191
acraig@sjm.com
or
Media Relations:
Guy Davis, 972-526-8227
gdavis@sjm.com
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."
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.
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.
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.
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.
