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Archive for November, 2007

Gene Therapy for Advanced Parkinson’s disease

Tuesday, November 27th, 2007

A recent article about gene therapy was just released and I thought it important to post in its entirety.

New data from the first-ever clinical trial of a novel gene therapy for advanced Parkinson’s disease demonstrate the ability of Neurologix, Inc.’s (OTCBB:NRGX) gene transfer treatment to quiet the abnormal brain activity that is correlated with the motor deficits characterizing the disease. Details regarding the normalization of brain activity in treated patients, as measured through radiolabeled glucose uptake and positron emission tomography (PET), were published this week in the online edition of the Proceedings of the National Academy of Sciences.

“In recent years we have been able to identify highly specific patterns of abnormal metabolic activity in patients with Parkinson’s disease. These patterns can be quantified with high reliability using PET imaging,” said David Eidelberg, M.D., Director of the Center for Neurosciences at The Feinstein Institute for Medical Research, and senior author of the new publication. “In particular, the brain network associated with abnormal motor function demonstrates increasingly higher levels of activity as Parkinson’s disease progresses. Existing treatments for Parkinson’s symptoms including drug therapies and deep brain stimulation are known to suppress this abnormal brain network.”

“In this publication,” Dr. Eidelberg continued, “we report that the previously published safety and tolerability seen in the Phase 1 open label study of Neurologix’s Parkinson’s disease treatment was also associated with a significant reduction in abnormal network activity in patients receiving the experimental treatment. Moreover, the degree of reduction in this biomarker correlated with the clinical benefit that the treated patients experienced.”

In the Phase 1 study, the investigators injected an adeno-associated virus (AAV) vector carrying an inhibitory gene (glutamic acid decarboxylase or “GAD”) into one side of the subthalmic nucleus (STN) of the brain of 12 patients with advanced Parkinson’s disease. The GAD gene was intended to increase the synthesis of the major inhibitory neurotransmitter in the brain, gamma-aminobutyric acid, or “GABA”, and thus calm the overactive STN. Although all patients had symptoms on both sides of their bodies, the gene transfer procedure was performed on only one side of the brain, enabling the untreated side to serve as a study control. Improvements in both clinical symptoms and abnormal brain network activity were seen predominately on the treated side of the brain at six months following treatment. Both the clinical benefit and the metabolic improvement persisted through the 12 months of the study period. Moreover, the improvements in brain metabolism occurred in areas of the motor network different from those known to be produced by brain lesioning alone. At the same time, the activity of the cognition-related brain network did not change following the gene transfer procedure.

“We are very encouraged by these findings,” said John Mordock, Neurologix President and Chief Executive Officer. “They clearly support the previously reported results from this study, published earlier this year in The Lancet. Moreover, they also suggest a highly useful, measurable biomarker of efficacy for use in subsequent studies of our gene transfer approach to the treatment of Parkinson’s disease and potentially other neurodegenerative conditions. We expect to begin a Phase 2 study of our approach in Parkinson’s disease within the coming weeks, and believe that we are on track to begin a Phase 1 study in epilepsy.”

About Neurologix

Neurologix, Inc. (NRGX.OB) is a clinical-stage biotechnology company dedicated to the discovery, development, and commercialization of life-altering gene transfer therapies for serious disorders of the brain and Central Nervous System (CNS). Neurologix’s therapeutic approach is built upon the groundbreaking research of its scientific founders and advisors, whose accomplishments have formed the foundation of gene therapy for neurological illnesses. Current company programs address such conditions as Parkinson’s disease, epilepsy and Huntington’s chorea, all of which are large markets not adequately served by current therapeutic options.

Cautionary Statement Regarding Forward-looking Statements

This news release includes certain statements of the Company that may constitute “forward-looking statements” within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and which are made pursuant to the Private Securities Litigation Reform Act of 1995. These forward-looking statements and other information relating to the Company are based upon the beliefs of management and assumptions made by and information currently available to the Company. Forward-looking statements include statements concerning plans, objectives, goals, strategies, future events, or performance, as well as underlying assumptions and statements that are other than statements of historical fact. When used in this document, the words “expects,” “promises,” “anticipates,” “estimates,” “plans,” “intends,” “projects,” “predicts,” “believes,” “may” or “should,” and similar expressions, are intended to identify forward-looking statements. These statements reflect the current view of the Company’s management with respect to future events. Many factors could cause the actual results, performance or achievements of the Company to be materially different from any future results, performance or achievements that may be expressed or implied by such forward-looking statements, including, but not limited to, the following:

- The Company is still in the development stage and has not generated any revenues. From inception through September 30, 2007, it incurred net losses and negative cash flows from operating activities of approximately $26.2 million and $20.3 million, respectively. Management believes that the Company will continue to incur net losses and cash flow deficiencies from operating activities for the foreseeable future. Because it may take years to develop, test and obtain regulatory approval for a gene-based therapy product before it can be sold, the Company likely will continue to incur significant losses for the foreseeable future. Accordingly, it may never be profitable and, if it does become profitable, it may be unable to sustain profitability.

- The Company will need to conduct future clinical trials for treatment of Parkinson’s disease using the Company’s NLX technology. If the trials prove unsuccessful, future operations and the potential for profitability will be materially adversely affected and the business may not succeed.

- There is no assurance as to when, or if, the Company will be able to successfully receive approval from the FDA on its Investigational New Drug Application to commence a Phase I safety trial for the treatment of epilepsy.

Other factors and assumptions not identified above could also cause the actual results to differ materially from those set forth in the forward-looking statements. Additional information regarding factors that could cause results to differ materially from management’s expectations is found in the section entitled “Risk Factors” in the Company’s 2006 Annual Report on Form 10-KSB. Although the Company believes these assumptions are reasonable, no assurance can be given that they will prove correct. Accordingly, you should not rely upon forward-looking statements as a prediction of actual results. Further, the Company undertakes no obligation to update forward-looking statements after the date they are made or to conform the statements to actual results or changes in the Company’s expectations.

The Last Few Months in the World of Parkinson’s Disease

Monday, November 26th, 2007

It has been interesting to see how the world of Parkinson’s has developed just over the past year.  I recently read that Parkinson’s disease is projected to double in the top 15 developed countries.  Many of these countries are located in Asia and may not be ready for this growing threat, a very scary situation to think about.  The world in many ways has been focused on contagious diseases or diseases that have become epidemics, a very important thing to tackle, but it’s also important to keep in mind diseases like Parkinson’s disease that are affecting more and more people.

One thing that is relieving is the great research that has been taken place over the last year and even months.  It seems every day researchers are little bit closer to understanding Parkinson’s disease.  One recent finding submits that skin cells can be used as stem cells.  this type of procedure in some ways will put to rest some of the ethical issues from stem cell treatment, mainly with embryonic stem cell treatment which basically destroys a forming fetus so that stem cells can be applied.

Peptide Therapy to Prevent Progression of Parkinson’s Disease

Friday, November 23rd, 2007

MJFF-funded Scientists Find Peptide Therapy Can Prevent Progression Of Parkinson’s Disease

Researchers funded under The Michael J. Fox Foundation’s Community Fast Track 2003 initiative have successfully used a peptide to reverse biochemical, cellular and anatomical changes that occur in the brains of mouse models of Parkinson’s disease.

“This could be a new approach to halt disease progression in PD patients,” said study author Kali Pahan, PhD, professor of neurological sciences at Rush University Medical Center. Dr. Pahan and colleagues from Rush, along with researchers at the University of Nebraska Medical Center, Omaha, and Yale University, New Haven, published these findings in the Proceedings of the National Academy of Sciences, November 13, 2007.

The authors have shown that one protein, NF-kB, is increased in the midbrain of PD patients and mice with PD pathology, and the researchers used a novel peptide (small proteins) to block this protein in mice with PD-like symptoms.

Pahan explained that after intraperitoneal injection (injection into the abdomen of the mouse) this peptide enters into the brain and blocks protein NF-kB and other associated toxic molecules, and goes on to protects neurons, normalizes neurotransmitter levels, and improves motor functions in mice with PD. Peptides, proteins and certain drugs usually do not enter into the brain after crossing the blood-brain barrier. Therefore, at present, peptides, proteins or genes are injected into the brain which is risky and painful. “To overcome this problem, we have added a tag in front of that peptide that is helping the peptide enter into the brain. Therefore, there is no need to inject these peptides into the brain. This is an important discovery.

Understanding how the disease works is important to developing effective drugs that protect the brain and stop the progression of PD,” Pahan said. “Now we need to translate this finding to the clinic and test this peptide in patients with PD. If these results can be replicated in PD patients, it would be a remarkable advance in the treatment of this devastating neurodegenerative disease.”

Parkinson’s is a slowly progressive disease that affects a small area of cells within the mid-brain known as the substantia nigra. Gradual degeneration of these cells causes a reduction in a vital chemical neurotransmitter, dopamine. The decrease in dopamine results in one or more of the classic signs of Parkinson’s disease that includes: resting tremor on one side of the body; generalized slowness of movement; stiffness of limbs; and gait or balance problems. The cause of the disease is unknown. Both environmental and genetic causes of the disease have been postulated.

Parkinson’s disease affects about 1.2 million patients in the United States and Canada. Although 15 percent of patients are diagnosed before age 50, it is generally considered a disease that targets older adults, affecting one of every 100 persons over the age of 60. This disease appears to be slightly more common in men than women.

This research was supported by grants from The Michael J. Fox Foundation for Parkinson’s Research and National Institutes of Health.

Source: http://michaeljfox.org/newsEvents_parkinsonsInTheNews_article.cfm?ID=267

Skin Cells Transformed into Stem Cells

Tuesday, November 20th, 2007

A huge breakthrough was made in stem cell research–skin cells can be turned into any kind of human tissue, just as embryonic stem cells do. If this finding continues to stand correct it may wipe out the ethical issues of embryonic stem cell therapy.

The technique of transforming skin cells into stem cells takes place when 4 genes are injected in a prepared culture of skin cells and coaxes the skin cells to act like stem cells. Like embryonic stem cells these reprogrammed cells become pluripotent, meaning that they can turn themselves into any tissue in the body. The researchers that have proven this still say they are a few years away from actual human testing, because there is so much that is unknown. MSNBC reports that such findings could have the following effects on Parkinson’s disease, “If the recipe for stem cell-like behavior can be perfected, individualized pluripotent cells could be created to reflect a particular disease condition — for example, allowing researchers to test potential treatments for Parkinson’s disease on living human neurons created in a culture dish.

Eventually, your cells could be converted and grown into tissue suitable for transplantation back into your body, with no fear of immune rejection. In the process, no human embryos would be destroyed, and no human eggs would be used — easing the ethical concerns that have dogged stem cell research in the past.”

We live in exciting times when the world is coming closer and closer to finding better cures and treatments for diseases and illnesses that plague this world. My Parkinson’s Information plans to continue to follow such research and continue to let our subscribers and viewers stay up to date on the progressing research stem cells.

 

 

 

Cure in Five Years?

Tuesday, November 20th, 2007

I recently read through a few comments from patients with Parkinson’s disease and their frustration with the researchers in regards to finding a cure for Parkinson’s disease. One person stated that for 20 years researchers and other professionals have stated that they are about 5 years away from finding a cure. Just this year I can count on probably two hands how many times researchers have said that they are on the brink of finding a cure for Parkinson’s disease. Now don’t count me pessimist, but I’m not sure how fair it is to those suffering from Parkinson’s to project such statistics. Regardless, I hope with all of my heart and soul that a cure is found in 5 years or less.

Surviving Adversity: Living with Parkinson’s Disease

Friday, November 16th, 2007

Today I was reading through a few blogs and I came across Shake, Rattle and Roll–An insider’s view of Parkinson’s disease, the author, Kate Kelsall, mentioned a book that gives a breath of fresh air to the world of Parkinson’s.  The book is titled Survivng Adversity: Living with Parkinson’s Disease.  I haven’t read it, but Kate says that it is a book that “instills hope.”  Since I’ve begun blogging about Parkinson’s disease I’ve come across tons of gloomy material on Parkinson’s disease.  I was greatly surprised to see how much gloomy material came from people that, in a sense, had lost hope.  As painful and difficult as such diseases may be it’s important to keep ourselves focused on hope.

The above mentioned book focuses on the struggle that a professional golfer, neurologist, acclaimed lawyer, children’s author, nurse, legendary cyclist, former Attorney General, highly respected news anchor and other men and women have coped with Parkinson’s disease.  I learned a long time ago that if I wanted to become a successful businessman and family manI needed to surround myself with people that had already found success in business and familylife.  As I’ve surrounded myself with such people a hope has boiled up and I’ve felt that I can accomplish a great deal, despite my weaknesses and short-comings.  I hope that everyone strives to surround themselves with great, successful people as they continue to fight Parkinson’s disease.   

Taking Back Your Life Through Theracycle

Wednesday, November 14th, 2007

home_photo2.jpg We are all busy in some way or another and it’s easy to not make exercise a part of our lives. About a year ago I remember watching The Biggest Loser on TV and was impressed with one of the participants, a busy doctor, began neglecting his diet and exercise because he wanted to give more time to his family. He then realized that by not watching his diet or not exercising he could very well be giving his family the opposite. He then joined The Biggest Loser to take his life back. As I watched him change, both his figure and attitude, a desire swept over me, I wanted to be in full control of my life as well.

Each of us at some point will have a decision to take back our life. When Parkinson’s disease hits, many become a victim of the disease and they let it take over their life. One of the quickest ways to avoid this is to simply take the time to exercise. The Exercycle Company has produced an exercise cycle that is centered towards people with specific needs. It has a large cushioned seat, designed for balance, computerized settings to track time and speed so you can program your own workouts, secure pedal straps so that your feet are firmly secured, and lastly a rugged steel frame with wide legs to bring the user a low center of gravity for maximum stability and safety. This exercise cycle is just one way to ensure that Parkinson’s disease doesn’t control your life, there are many tools out there to help you. To learn more about the Theracycle click here.