Wednesday, August 27, 2008
Tuesday, August 26, 2008
CALABASAS HILLS, Calif. (August 26, 2008) -- The ALS Association is funding a major clinical trial to determine if lithium can slow disease progression in patients in the early stages of ALS. The study builds on earlier promising results from preclinical research and a small, open-label investigation in ALS patients.
"Lithium has generated a lot of interest in the ALS community," according to Lucie Bruijn, Ph.D., senior vice president, research and development of The Association. "This trial is vital for testing the efficacy of lithium in a well-controlled way. It's the crucial next step for investigating the potential of this drug."
The trial is being supported through The Association's TREAT ALS (Translational Research Advancing Therapies for ALS) initiative, and will use the TREAT ALS/NEALS Clinical Trials Network. Lead investigators for the study will be Merit Cudkowicz, M.D., and Swati Aggarwal, M.D., of Massachusetts General Hospital, Lorne Zinman, M.D., from the University of Toronto, Petra Kaufman, M.D., from Columbia University, and Jeremy Shefner, M.D., of SUNY Upstate Medical University.
In addition to The Association, funding partners for the new study include the National Institute of Health/ National Institute of Neurologic Diseases and Stroke and ALS Society of Canada. Investigators worked together with the funding agencies to design the most appropriate study within the shortest possible time frame. "Collaboration among researchers and funding agencies makes this trial possible," Dr. Bruijn said. "All of us are working together to support this important research to find a new treatment for ALS."
Lithium is a simple chemical substance approved for use in humans and prescribed as a mood stabilizer. Lithium protects neurons in the brain in animal models of neurodegenerative diseases, including Alzheimer's disease and Parkinson's disease, and has been recently shown to do the same in a mouse model of ALS. In that study, lithium prolonged survival and protected cells in both the brain and spinal cord. While the exact mechanism of lithium's effect is unknown, researchers have proposed it promotes clearance of toxic protein accumulation.
A study in a small number of ALS patients compared lithium plus riluzole (an approved treatment for ALS) to riluzole alone. After 15 months, no deaths occurred in the 16 participants treated with lithium plus riluzole, while 29% of those taking riluzole alone had died. Patients on lithium also had a markedly lower decline in disease-related disability. However, the trial was limited by the small numbers of patients and the absence of a placebo treatment ? both the patient and the doctor knew whether the patient was taking lithium or not, which may have influenced the response to treatment or interpretation of disability. Due to diagnostic and prognostic variability in persons showing symptoms of ALS, the small numbers in this study were not sufficient to know whether lithium was indeed an effective treatment. Many apparently effective treatments in open trials (trials lacking a concurrent placebo group) fail in larger double-blind trials, in which neither doctor nor patient knows whether the patient is receiving the study drug.
The new trial will be a double-blind, placebo-controlled trial with 84 patients, who will be randomized to either lithium or placebo. A review of data will occur after the 84th person is enrolled, and then a decision will be made on whether to expand to 250 patients. Patients may be included if they are within three years of their diagnosis and are not already taking lithium. Treatment will be for up to one year. The disease course and safety assessments will be measured at regular intervals over that time. Further details including trial sites, enrollment criteria and start dates will follow shortly. "We formed collaboration with Canadian and U.S. sites to perform a study that will provide an answer quickly on whether lithium is effective in the treatment of ALS. The study is designed to answer this key question definitively and with appropriate monitoring of safety," commented Merit Cudkowicz, M.D., one of the lead investigators on the study.
Denise Figlewicz, Ph.D., director of research at the ALS Society of Canada said, "We welcome this opportunity to support the lithium clinical trial. The promising results from the Italian study data serve as the impetus for new research and new treatment strategies. This is very exciting news for the ALS community. We also welcome this opportunity to work together with our American colleagues. This collaborative approach between Canada and the United States will serve as a model for subsequent clinical trials."
ALS and The ALS Association Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease that affects nerve cells in the brain and the spinal cord. Motor neurons reach from the brain to the spinal cord and from the spinal cord to the muscles throughout the body. When the motor neurons die, the ability of the brain to initiate and control muscle movement is lost, leading to progressive paralysis.
The ALS Association is the only national, not-for-profit voluntary health organization devoted solely to fighting ALS through research, patient services, advocacy and public education and information. TREAT ALS is a drug discovery program and clinical trials process created by The Association that accelerates discovery and testing of clinical candidates.
The ALS Association National Office
27001 Agoura Road, Suite 150 Calabasas Hills, CA 91301-5104 (818) 880-9007 FAX (818) 880-9006
Contact: The ALS Association Gary Wosk (818) 587-2241 firstname.lastname@example.org@alsa-
Friday, August 22, 2008
I ONLY MADE IT TO THE 3:17 min. mark. BEFORE I HAD TO SHUT IT OFF
IT MIRRORS MY LIFE. except for the cancer
Monday, August 18, 2008
I face a giant
It's not one you can see
Yet, it is not invisible
The giant insists on winning
It's hold reaches deep
I am trapped within its grasp
The giant holds me tight, yet
I am not captured.
We live as one
The giant feeds on motor neurons
Slowly, it disables my limbs
The giant wants all of me
Yet, not all is his to take
For my soul belongs to Jesus
I am not captured.
The giant has taken my voice
It has taken my throat too
Yet, my spirit remains free
What can be seen belies truth
There is more to me unseen
I am not captured.
There is strength
A deep well abides within
I live, even though I die
Yet, the giant demands more
With hands clasped, I pray
Thank you Lord for this day
The giant will not prevail
I am not captured.
I am alive
A wellspring of hope abides
Though my body slowly dies
My soul grows stronger still
Yet, the giant knows this not
No matter, God is on my side
The angels attend to me
I am not captured.
(© copyright, Jan 2008, Butterfly)
Saturday, August 16, 2008
ALS Association Research Grants Announced
New research grants by The ALS Association will support investigations of new disease models, pathogenesis of ALS, biomarkers of disease progression, and development of new treatments. The research portfolio supported by the Association is designed to speed therapy development by attacking ALS on all fronts, from basic research to clinical trials. Each year the Association funds the most promising research from hundreds of investigator-initiated proposals. In addition, it actively seeks researchers to undertake studies identified by the Association as critical for filling gaps in knowledge of ALS. The Association also gives the Milton Safenowitz post-doctoral fellowship award for ALS research to encourage young scientists to focus their research efforts on ALS.
Newly funded investigator-initiated research will study the following important questions:
Why do motor neurons die in ALS?
The cause of ALS is death of motor neurons, nerve cells that control muscle movements. A central question in ALS is why, and how, these neurons die. Nine new projects examine different aspects of that question.
Researchers have found that some motor neurons are more susceptible than others to the disease process. Now, they will examine how these different cells vary in the genes they express during the disease, looking for clues that influence vulnerability to ALS. Researchers will also investigate the details of the pathways by which motor neurons die, in order to improve prospects for interrupting those processes. [Aebischer, Tsunemi]
While it is motor neurons that die in ALS, evidence has accumulated to suggest that other cell types contribute to the disease. Researchers will use mice that express the disease causing SOD1 gene in multiple different cell types, to examine the role each plays. Researchers will also investigate changes at the junction between nerve and muscle to determine how muscle contributes to the disease. Finally, they will ask whether new compounds that aid the cell’s powerhouses can reduce the harmful effects of one type of cell. [Roos, Manuel, Cassina]
A newly discovered, pervasive form of gene regulation involves so-called “micro-RNAs,” which act at many different levels to control cell function. Recently researchers have leaned that micro-RNAs are altered in models of ALS, suggesting they may be a key to understanding the disease process. Researchers will extend these studies to further explore this phenomenon. [Moeller]
Researchers will further their investigations of a newly discovered cause of ALS, the protein TDP-43, to determine how the abnormal protein accumulates and why it causes disease. Understanding this may help explain how different causes can lead to the same disease, and whether there are common “downstream” targets in each form of ALS that would be appropriate therapeutic targets. [Petrucelli, Sanelli, Morton]
Why is mutant SOD1 toxic?
Mutations in the superoxide dismutase (SOD1) gene cause about 2% of all cases of ALS, but it is not yet clear how this mutation leads to the disease. Two projects examine that question, which should help in understanding not only SOD1-caused ALS, but possibly other forms as well.
Researchers know that mutant SOD1 protein forms insoluble clumps in ALS motor neurons, but it is possible that soluble forms of the mutant protein are actually more toxic. Now, researchers will study these soluble forms to understand how they affect motor neurons. [Marklund]
Researchers will also examine whether and how the cell environment affects the toxicity of SOD1, an important step in determining whether altering that environment might potentially be beneficial. [Carri]
What can we learn from new animal models of ALS?
Animal models are vital to understanding ALS and searching for treatments. A new model uses the fruit fly, whose advantages include its rapid generation and extensive understanding of its genetics. This model will be used to explore a new ALS gene to investigate how this gene causes the disease, and how other factors contribute to the disease. Another model will place a newly discovered ALS gene into mice to investigate how this gene causes the disease. [Tsuda, Dupuis]
Can imaging be used to track disease progression?
Researchers will build on previous studies to determine if magnetic resonance spectroscopy can be used to discover biomarkers of disease progression in ALS patients. This non-invasive technique is related to the more common MRI (magnetic resonance imaging), but provides additional information about chemical compounds in the body. Researchers hope that this technique will reveal one or more compounds that change in step with the disease, allowing it to be used to accurately track disease progression. [Benatar]
How can transplanted motor neurons be optimized for therapy?
One potential therapy for ALS would be to transplant healthy motor neurons into the spinal cord. However, functional motor neurons need long extensions, called axons, to reach their targets, and growing these axons in transplanted cells is an unmet challenge. Researchers will examine whether genetically modifying motor neurons can help them to better grow axons after transplantation. [Strittmatter]
Friday, August 15, 2008
Thursday, August 14, 2008
what's up man? Long time no talk. I haven't seen you in awhile, but read your blog daily and am truly inspired by your courage and fight. It's very humbling when I think about the many things I used to stress about at work and in my personal life and then compare it to the challenges you experience every day. Sadly I have a colleague who I've worked with for years who was recently diagnosed with brain and lung cancer. Just as I told her, God is just testing you to see how strong you are. Make sure you continue to fight and show him how serious you are about beating this sucker! Only the strong survive and I have no doubt you have the strength to keep on fighting and ultimately win. You are constantly in my thoughts and prayers. As evident by the comments on your blog, you have an amazing supporting cast and we all look forward to seeing you get through this.
As Jimmy Valvano one said "Cancer (ALS) can take away all my physical ability. It cannot touch my mind; it cannot touch my heart; and it cannot touch my soul. And those three things are going to carry on forever. Don't give up, don't ever give up".
All the best!-Chris
Wednesday, August 13, 2008
The Repair Stem Cell Institute would like to congratulate Beike for the excellent work they are doing in the field of stem cell treatments. Throughout history, experimental treatments have been performed in academic settings and huge advancements in knowledge have been the result that all of mankind has benefited from. Beike is following this trail blazing attitude and doing so while maintaining an extremely high level of quality and standard of care and placing the needs of the patient at the forefront of their focus. We would like to join the academic institutions, their government and their world-wide patient base in voicing our support of their efforts in innovative technologies in general and stem cells specifically. We hope their example can be matched by many other treatment centers; to ultimately provide great benefits to science, medicine and the patients who are without significant treatment options and may be helped by stem cell treatments today.Visit our website, click on "Treatment Centers" and see what the Repair Stem Cell Institute was saying about Beike even before their P.R. was released. Without a doubt, they are the World’s #1 stem cell treatment center.
Keep up the good work!
Repair Stem Cell Institute
Friday, August 8, 2008
By STEPHANIE NANO, Associated Press Writer1 hour, 3 minutes ago
Harvard scientists say they have created stems cells for 10 genetic disorders, which will allow researchers to watch the diseases develop in a lab dish.
This early step, using a new technique, could help speed up efforts to find treatments for some of the most confounding ailments, the scientists said.
The new work was reported online Thursday in the journal Cell, and the researchers said they plan to make the cell lines readily available to other scientists.
Dr. George Daley and his colleagues at the Harvard Stem Cell Institute used ordinary skin cells and bone marrow from people with a variety of diseases, including Parkinson's, Huntington's and Down syndrome to produce the stem cells.
The new cells will allow researchers to "watch the disease progress in a dish, that is, to watch what goes right or wrong," Doug Melton, co-director of the institute, said during a teleconference.
"I think we'll see in years ahead that this opens the door to a new way to treating degenerative diseases," he said.
The new technique reprograms cells, giving them the chameleon-like qualities of embryonic stem cells, which can morph into all kinds of tissue, such as heart, nerve and brain. As with embryonic stem cells, the hope is to speed medical research.
Research teams in Wisconsin and Japan were the first to report last November that they had reprogrammed skin cells, and that the cells had behaved like stem cells in a series of lab tests. Just last week, another Harvard team of scientists said they reprogrammed skin cells from two elderly patients with ALS, or Lou Gehrig's disease, and grew them into nerve cells.
Melton said the new disease-specific cell lines "represent a collection of degenerative diseases for which there are no good treatments and, more importantly, no good animal models for the most part in studying them."
A new laboratory has been created to serve as a repository for the cells, and to distribute them to other scientists researching the diseases, Melton said.
"The hope is that this will accelerate research and it will create a climate of openness," said Daley.
He expects stem cell lines to be developed for many more diseases, noting, "this is just the first wave of diseases." Other diseases for which they created stem cells are Type 1, or juvenile, diabetes; two types of muscular dystrophy, Gaucher disease and a rare genetic disorder known as the "bubble boy disease."
Daley stressed that the reprogrammed cells won't eliminate the need or value of studying embryonic stem cells.
"At least for the foreseeable future, and I would argue forever, they are going to be extremely valuable tools," he said.
The reprogramming work was funded by the National Institutes of Health and private contributions to the Harvard Stem Cell Institute.
On the Net:
Harvard Stem Cell Institute: http://www.hsci.harvard.edu
Thursday, August 7, 2008
By Maggie Fox, Health and Science EditorThu Aug 7, 12:01 PM ET
U.S. stem cell experts have produced a library of the powerful cells using ordinary skin and bone marrow cells from patients, and said on Thursday they would share them freely with other researchers.
They used a new method to re-program ordinary cells so they look and act like embryonic stem cells -- the master cells of the body with the ability to produce any type of tissue or blood cell.
The new cells come from patients with 10 incurable genetic diseases and conditions, including Parkinson's, the paralyzing disease amyotrophic lateral sclerosis, or ALS, juvenile diabetes and Down's Syndrome.
Writing in the journal Cell, the team at Harvard Medical School and Children's Hospital in Boston said the point is not yet to treat anyone, but to get as many researchers as possible experimenting with these cells in lab dishes to better understand the diseases.
"This is just the first wave of diseases," said Dr. George Daley, who is also a Howard Hughes Medical Institute investigator.
The new cells are called induced pluripotent stem cells, or iPS cells, and are made using four genes that reprogram an ordinary adult cell into a primitive stage resembling the first days of a human embryo -- a method pioneered by Shinya Yamanaka of Kyoto University in Japan.
WHAT GOES WRONG
"They allow researchers ... to watch the disease progress in a dish, to watch what goes right or wrong," said Harvard's Dr. Doug Melton, who will head up the distribution of the cells. "I think we'll see in the years ahead that this opens the door to a new way of treating degenerative diseases."
Every cell in the human body contains the same genetic instructions, and in people with inherited genetic diseases, every cell carries the same mistakes, Daley and Melton said.
Stem cells -- and the new iPS cells -- will grow virtually immortal in the lab, and given the right conditions, can be made to form any desired tissue, from heart muscle to brain cells.
One day these might be used as tailor-made patches to fix diseased or damaged organs, but right now Melton said it is important to simply understand the diseases.
"We don't even know when a patient gets diabetes if each patient gets it the same way," Melton said. "There could be 50 different ways."
One thing his lab will do is try to make pancreatic beta cells -- the cells that make insulin and are destroyed in type 1 diabetes -- and study them to see what may be different among patients with the disease.
While the cells are an alternative to the more controversial embryonic stem cells, taken from a human embryo, Daley and Melton are adamant that they do not replace them.
For one thing, viruses are used to carry the transformative genes to make the iPS cells. Daley says cloning technology is still superior. "The egg does it faster and better," he said.
The federal government severely restricts embryonic stem cell research because of moral objections, but the Harvard lab works with private funding.
Melton said researchers can get batches of cells from the lab to grow on their own. Other cell types they have made to date include samples from people with Huntington's disease, a form of combined immunodeficiency commonly known as "bubble boy's disease," Lesch-Nyhan syndrome, Gaucher's disease, and two forms of muscular dystrophy.
(Reporting by Maggie Fox; editing by Julie Steenhuysen and Stacey Joyce)
Monday, August 4, 2008
Friday, August 01, 2008 9:53 PM
Recently, Shenzhen’s Beike Biotechnology held the first annual 2008 China Stem Cell Stem Cell Technology Forum, a gathering that was attended by over 300 of the world’s most renowned stem cell biologist and researchers. The Mandarin-language conference included many participants from China , while others attended from the United States , Canada , India , Australia , and Malaysia . The focus of the conference was on advanced induced pluripotent stem (iPS) cell research and therapeutic uses of stem cells. The event was held at the China Medical City complex in Taizhou , China and chaired by Dr. Sean Hu, who also serves as Chairman of Beike Biotechnology Co., Ltd. Beike is a controversial company because it has already begun treating patients with injections of stem cells, usually a combination of umbilical cord cells and stem cells derived from each patient. Beike claims to treat over 250 patients per month, a total of more than 3,000 so far. Approximately 70% to 80% of the patients report themselves satisfied with their treatments, and the treatments have the ability to produce at least some therapeutic effect in 86% of its patients, according to statistics provided by Beike. Beike has not submitted its techniques to the rigors of a double-blind clinical test. Nor has it done the otherwise necessary animal testing. Nevertheless, the company points to its success with the majority of its patients as sufficient proof that its stem cell therapies are a valuable addition to the medical arsenal. Beike charges its patients between $20,000 and $30,000 for several rounds of stem cells injections, which it says can help cure diseases such as Alzheimer’s, atoxia, multiple sclerosis, optic nerve hypoplasia, and spinal muscular atrophy . Although most patients come from China , its therapies draw significant numbers of patients from the West. The former Lord Mayor of Manchester , Audrey Jones, for example, flew to China for a round of stem cell injections to treat her cerebellar atrophy and came back to England proclaiming herself much improved. Before the Beike therapy, she was confined to a wheelchair. Now she plans to walk into Manchester City Hall without aid. Beike’s web pages are replete with similar success stories for a wide variety of diseases (www.stemcellschina. com). Stem cells are commonly used for bone marrow transplants in the West, but experts say that bone marrow, which constantly undergoes renewal, is a better candidate for basic stem cell injections than Beike’ specialties of nerve/heart/ spinal cord problems. Stem cells will not necessarily find the correct nerve to repair without coaxing, according to the experts. They believe a set of simple stem cell injections is unlikely to correct septo-optic dysplasia, for example. On the other hand, Dr. Hu does not claim to know the mechanism behind Beike’s treatments, but he points to the results. Adding to Beike’s credibility is the high quality of its academic affiliations. Beike was founded with capital from Beijing University , Hong Kong University of Science and Technology and Shenzhen City Hall . It is also supported with funds from the China State National Fund. Its research and clinical work benefits from collaborations with Beijing University , Hong Kong University of Science and Technology, No. 3 Army Medical University, Zhongshan Medical University , Guiyang Medical College and Zhengzhou University . Beike has over 60 PhD researchers focusing on stem cell research in its labs. In November 2007, Beike Biotechnology teamed up with the Szenzhen Graduate School of Tsinghua University to establish a laboratory that will research cell reprogramming and gene engineering. The lab will conduct research on stem cells, the nuclear transfer and reprogramming of cells, and monoclonal antibodies. The new lab was honored with the “China Key Laboratory” designation and given a grant worth $4 million for equipment from the Shenzhen Municipal Government. The grant was part of $12 million given out in 2007 by Shenzhen to encourage innovative technologies. In June of this year, Beike announce it began outfitting a 21,500 square foot comprehensive medical stem cell storage and processing facility in Jiangsu province. When completed, the Jiangsu Stem Cell Storage Facility will boast the technology to freeze, store, and process a broad range of human stem cell samples for clinical application. Initially, the facility will provide stem cell banking for human umbilical cord blood stem cells, placenta stem cells, amniotic membrane stem cells, bone marrow stem cells, and later it will also be able to house induced pluripotent stem cells (iPS). The facility is expected to begin operations later this year, adding another dimension to Beike’s manifold stem cell-related activities
http://www.istockan alyst.com/ article/viewarti cle+articleid_ 2466354&title=Beike_ Uses_Research. html