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October 31, 2004
Command-Post
As mentioned before. I'll be blogging the election for Command-Post.org. You can see my Colorado postings here.
I'll be one of 85 bloggers voluneering for this effort. Stay tuned!
Posted by Dave at 06:07 AM | Comments (0) | TrackBack
October 30, 2004
Trackback Spam
Imagine my surprise this morning (4am) when I noticed the spam hitting the website. I had gotten up early to do some client work but instead found myself trying to stop the spam. Turns out this version of the software doesn't have an easy way to stop it. I ended up 'breaking' the trackback system to stop the spam.
As you may know, I redid the coding on this website about a month ago to reduce the comment spam. I had to do this when I started to get well over 100 spam comments a night. I had to delete them one at a time.
It's now harder for automated programs to drop comment spam on this website and, so far, not one has succeeded.
Now there's a new type of spam...trackback spam. Blogs, such as this one, use 'Trackbacks' to notify readers of other blogs that have referenced an article. Now the spammers are using this feature to post their spam on blogs.
Before I broke the trackback feature, the website received 20 highly-pronographic (misspelling on purpose, unlike the other ones) messages in the span of 50 minutes.
Looks like I'll need to make more changes.
Posted by Dave at 11:09 PM | Comments (0) | TrackBack
October 29, 2004
Lost One
I posted an article here on the 29th on the election. Or at least I thought I did. It looks like it did not successfully post and I do not have a copy.
It wasn't one of my better ones, but here is the (very) condensed version of what I wrote regarding the election...
1. Sorry about not writing sooner, as promised, regarding President Bush's acceptance speech.
2. Bush did talk some about health care. Nothing on medical research or genetic discrimination.
3. For issues related to the Huntington's Disease community, neither candidate offers a significant advantage. Why? Increases in research spending that affects HD is not likely to be much different under either candidate. Neither candidate has taken a noticable stand on genetic discrimination. The Canadian's have just taken steps to limit the ability of the US to reimport drugs (they did this to preserve their prices). Various other positions on reducing medical costs (that would affect our community) are essentially a wash.
Now back to our regularly scheduled program...
Posted by Dave at 11:56 PM | Comments (0) | TrackBack
October 28, 2004
HD & Flu Shots
This came up on the Hunt-Dis email discussion group.
Naturally the question of whether HD patients are considered part of the "high risk" group that should get flu shots. Marsha Miller called the CDC and "they confirmed that there was no intention of excluding Huntington's Disease patients from the list of the chronically ill. They suggested that we visit our the websites for our state health departments and see what their guidelines are, and then call for clarification.
I think we should point out to the authorities that voluntary movement, swallowing and coughing to clear secretions are impaired in later stages of the disease and that 51 percent of people with HD die of pneumonia (I got that figure from the HOPES site)."
Thanks for doing that Marsha!
Posted by Dave at 08:59 PM | Comments (0) | TrackBack
October 27, 2004
James Gusella Honored
And who is James Gusella you ask?
He's the scientist who, building on and working with many other researchers, isolated the Huntington's gene a little over 10 years ago. His job was only slightly easier than 'finding a needle in a haystack' and he accomplished this in a fraction of the time it was expected to take.
Every year the Oregon Health & Science University gives an award to a neurological pioneer. This year it is Gusella and the Oregonian newspaper interviewed him:
How did you come to find the Huntington's gene?
Back in the early '80s, I began to approach Huntington's disease using a strategy that hadn't been applied to neurological disease, or really any disease, before. That was to find the chromosomal location of the disease just using inheritance patterns in the family and polymorphisms.
The first genetic marker we found correlated about 96 percent of the time with the disease. Then we found better ones, until we found a stretch that was inherited 100 percent of the time.
Because it was successful, the technique sort of set off a torrent of similar studies in other disorders.
Some people expected fast cures from this genetic medicine. Has it lived up to its promise?
I don't think the promise was misleading in terms of the ultimate impact. I just think it was misleading in terms of timing. I know, when we found a marker for Huntington's back in the 1980s, people predicted there would be a therapy within six months, and that was just crazy. The first thing that comes is (not treatments but) the ability for better diagnostics.
In Huntington's, we've had this gene now for 10 years. We understand what this defect is. We have some promising pathways to new therapies. But the disease is still invariably fatal.
Do you see similarities in discussions of stem-cell research?
I do think the concept that stem cells are going to be very useful in combating disease is true. But it's not going to be in the next year or two. I don't completely understand why, in trying to sell the promise of technologies, people have to over-promise the speed of technologies.
Posted by Dave at 11:23 PM | Comments (0) | TrackBack
October 26, 2004
Neural Stem Cell Research
Science Blog has an interesting article on the promise of stem cell treatments for various diseases, including Huntington's Disease. The excerpt below talks about the success of neural stem cells in Parkinson's and Huntington's Disease...
In other work, scientists at the University of Wisconsin at Madison have rescued the cells that are attacked by Parkinson's disease and Huntington's disease. Both diseases are movement disorders that specifically kill off neurons that use the neurotransmitter dopamine. ''Replacement of dopamine neurons using embryonic stem cells has long been the holy grail,'' says Clive N. Svendsen, PhD. ''But stem cell transplantation can introduce serious problems, including tumors and dyskinesia, or impaired, sporadic muscle movements.''
So instead of replacing the dopamine cells, she and her colleagues found a way to provide support to neurons under attack. Dopamine neurons require glial-derived neurotrophic factor (GDNF) to survive. So even if stem cells could be successfully introduced to an adult brain, chances are they would require GDNF. Yet in an earlier study, Berhstock's group showed that GDNF alone could restore function to the neurons affected by Parkinson's.
...
''We thought real cells might better deliver GDNF to the brain,'' Svendsen says. The group considered using embryonic stem cells, but realized they might lead to tumors and dyskinesia, so they tried neural stem cells. These cells don't have quite the enormous potential of embryonic stem cells, but they can become astrocytes, a type of glial cell found in the brain. Best of all, they do not induce tumors.
Posted by Dave at 05:53 PM | Comments (0) | TrackBack
October 25, 2004
At-Risk Hockey Player
The Wisconsin State Journal has a wonderful article on Jake Dowell, a UW-Madison Hockey player and draftee of the Chicago Blackhawks. This article does a great job of explaining Huntington's Disease to the masses.
Some of excerpts from the article:
That terrifying threat runs counter to Jake's dreams of someday playing in the National Hockey League -- he was chosen by Chicago in the 2004 draft -- and having a wife and family.
Those realities accompany Jake everywhere, but their voices were especially loud during those summertime trips back home. While part of him couldn't wait to see his father, a part of Jake cringed at the thought.
"I'm wondering if it's going to be a lot worse or if it's going to be a good visit with him," Jake said. "For the most part, the last few have (been good)."
The favorite activity for father and son was to head to a nearby golf course, sit down and catch up on the world while acquaintances come and go. Jake didn't mind carrying the conversational load.
"He doesn't say much now," Jake said of his father. "It's basically yes, no, one-word phrase answers. He can't elaborate on anything."
How does Jake know it's been a good visit with John?
"When I leave and he's smiling," the son said, "like he's been happy to see me."
Talking is therapeutic
You might wonder how Jake can do this. How can he talk so freely about something so ongoing and painful?
"It is hard," Jake said, "but at the same time I kind of look at it's a way for me to express myself and let people know about it. Kind of get things off my chest. It's kind of a way for me to vent when I talk to people about it, because otherwise I'd just hold it in."
His mother, a special education teacher, is the same way.
"(Jake) has his moments where he'll look at something and be down for a little bit, but he really does have my personality," Vicki said. "I'm not good at staying down. It's too much work."
Mother and son subsist on multiple daily telephone conversations, a wide-open dialogue and their faith in God.
"Jake's faith is strong," Vicki said. "Mine, I think, is stronger. I don't think I'd survive without it. I try to look at things and problem-solve. How can I make the most of this? What can I learn from this?"
...
"He's way more positive and upbeat than I would ever be, for sure," said UW-Madison junior winger Ryan MacMurchy, who shares a campus apartment with Dowell. "The things he has to deal with off the ice and he's still a happy person and performs so well on the ice is just amazing."
That focus was evident during the season-opening series against Mercyhurst. With his parents in attendance at the Kohl Center, Jake scored three goals and helped the Badgers to 3-2 and 8-0 wins.
Eaves said Jake is more at peace with his situation now than he was last year. "Last year it always seemed like there was a cloud there all the time," Eaves said. "Now I see him smiling and a lot more outgoing in his personality."
Eaves thinks Jake found peace through diligence. "Just working through it, facing this adverse situation with his mom and talking about it," Eaves said. "There's been a natural maturation process."
Harder times are ahead, but Jake is cultivating a sense of readiness. "I feel through this whole process that mentally I've become a lot stronger and learned how to deal with things," he said. "Each and every day is a gift. That's why they call it the present."
Posted by Dave at 11:00 PM | Comments (0) | TrackBack
October 24, 2004
Spreading The Word
Suppose you had a website devoted to covering Huntington's Disease and you had an opportunity to introduce it to 100's of thousands of people. Would you take the opportunity?
I did. Next week a popular blog/website will be covering the election and they've asked for volunteers to cover events in each of the 50 states. I've volunteered to cover Colorado (I probably won't be the only one) and in return they'll reference this blog.
The website is http://www.command-post.org and (I believe) they expect 1,000,000-2,000,000 page hits that day. It could be higher. I'll be posting something on this website explaining Huntington's Disease to those who've never heard of it.
I'll keep you posted!
Posted by Dave at 11:44 PM | Comments (0) | TrackBack
October 23, 2004
NeurotrophinCell Press Release
Here's Living Cell Technologies official press release on their announcement that NeurotrophinCell reduced cell damage 86% in their Huntington's Disease model.
The press release:
LCT treatment protects the brain from damage by Huntington's disease
22 October 2004, Australia:
Living Cell Technologies (ASX: LCT), has today released results of pre-clinical studies demonstrating that its specialised therapy protects nerve cells in the brain from damage caused by conditions similar to Huntington's disease. Animals receiving LCT's treatment, NeurotrophinCell, showed 86 per cent less damage to the brain and showed dramatically improved use of their limbs.
This is the first time that technology of this kind has been proven in a controlled pre-clinical setting to prevent the degeneration of the brain due to Huntington's disease-like conditions. The details and data are to be presented at the Society for Neuroscience annual conference in San Diego this weekend and published in NeuroReport in November.
Huntington's disease is a devastating and fatal neurodegenerative condition that can be diagnosed very early in life, before symptoms appear, but for which there is no cure or intervention strategy available.
"These findings have major implications for enabling treatment of human neurodegenerative diseases such as Huntington's and stroke," said Dr. Dwaine Emerich, VP of Research at LCT BioPharma Inc1 and co-author of the paper detailing the findings.
"What we have done is successfully implant new choroid plexus cells (the cells that produce cerebral spinal fluid and a number of factors important for the health and survival of the brain) thereby protecting specific areas of the brain from damage" continued Dr. Emerich.
In LCT's proprietary product, NeurotrophinCell, the choroid plexus cells are encapsulated in a clear capsule derived from algae. This encapsulation hides the cells from the patient's immune system yet allows the cells to receive nutrients and chemical signals necessary for functionality and survival."
"NeurotrophinCell has effectively shown the ability of LCT's technology, to protect brain tissue that would otherwise die, potentially forestalling or preventing the debilitating consequences of this disease," said Alfred Vasconcellos, LCT BioPharma's CEO.
Posted by Dave at 11:58 PM | Comments (0) | TrackBack
October 22, 2004
"A Dignified End"
We've all seen what the last few years of what Huntington's Disease is like.
A dear friend of mine has cared for her father for many years. He's not bedridden yet, but you know its coming. It's hard for her to see her father fade away. There isn't going to be a 'cure' for him.
What makes it more difficult for her is that she has also tested positive for the HD gene. When she see's her father, she see's her own future. She amazes me. You have to be a strong woman to care for someone as she has for her dad these last few years.
So when I read articles like this one, they make me think. The article is about a woman with Huntington's Disease who is campaigning for an assisted suicide law in England. She saw her grandmother die at age 64, her 56-year-old mother is symptomatic and she has the HD gene. She's 36 and isn't showing any signs of the disease.
I'm worried about her and others I know in similar situations. She believes that if she doesn't commit suicide, she's going to die from Huntington's Disease. I doubt that she will.
So why do I say that? First, she has no symptoms. She's healthy. Second, both her mother and grandmother had a late onset of the disease. While it is no guarantee, it's likely she won't see symptoms for many years. Third, researchers are finally having success against this disease.
A 'cure'/effective treatment is coming. Scientists are now doing it in the lab and work is being done to repeat it in clinical trials. In addition, we also have a number of treatments available which may slow/postpone the progression of the disease.
This woman, who has every reason to fear this disease, believes that her family would cope better with her death knowing that she went in a way she wanted to go.
But what if she goes as she wishes, while relatively healthy, and then a cure becomes reality? Would her family grieve more, not less, knowing they could have had her many more years? While this obviously very caring lady is working hard trying to help the HD community, is it possible that her actions are signaling others that HD is hopeless at a time when there is finally real hope?
As with all things, each one of us will make our own choices and for our own reasons.
It might be surprising for some, but for many in our community the idea that there is now 'hope' is disturbing. It can be easier to prepare for the future when you 'know' what is going to happen. (Truth is, we never really do 'know'). But when someone has prepared themselves for years for their eventual decline and are then suddenly faced with the possibility of planning for retirement...it makes their lives uncertain again. And uncertainty breeds stress & anxiety.
It's time we start rethinking Huntington's Disease in our community. What was good advice a few years ago may no longer be good advice today.
I believe it's time we start making a stronger effort to keeping people healthy so that they may benefit from the coming treatments. We need to become more proactive against this disease. It's time we start fighting back and not just assume that "it's inevitable".
So, yes, articles like this get me thinking. One resulting thought...thanks to the researchers, my dear friend may never face what her father is facing today. That puts a smile on my face.
Posted by Dave at 11:21 PM | Comments (1) | TrackBack
October 21, 2004
Prestwick CEO Honored
As mentioned earlier this week, Dr. Christopher O'Brien has given the Guthrie Family Humanitarian Award by HDSA.
Here's the press release from Prestwick Pharmaceuticals:
Christopher O'Brien, MD, Honored with Guthrie Family Humanitarian Award
HDSA Gala Recognizes Leadership in Research and Community Service
WASHINGTON - Oct. 21, 2004 - Prestwick Pharmaceuticals, Inc., a CNS specialty pharmaceutical company, announced today that Christopher O'Brien, MD, the company's Chief Medical Officer, has been selected by the Huntington's Disease Society of America (HDSA) to receive the Guthrie Family Humanitarian Award for compassion and dedication to the care and support of people with Huntington's Disease (HD) and their families. Being held this evening at the Waldorf Astoria in New York City, the Annual Guthrie Awards Dinner pays tribute to the memories of legendary folk singer Woody Guthrie and his wife, Marjorie, principal founder of the national drive to cure HD.
A leader in neurological clinical research for the past 15 years, Dr. O'Brien joined Prestwick Pharmaceuticals in December 2003. He maintains his role as a neurologist in the HDSA Center of Excellence for Huntington's Disease clinic at the University of California San Diego where he is a member of the voluntary faculty.
"Dr. Chris O'Brien has played a pivotal role in the care and cure of Huntington's Disease. He was chosen to receive the Guthrie Family Humanitarian Award by both the Guthrie Family and HDSA because of his dedication and commitment to helping people with HD and their loved ones. He is an inspiration to all healthcare professionals and is deserving of this recognition," stated Barbara Boyle, HDSA National Executive Director/CEO.
"It's an honor to be selected for this prestigious award," said Christopher O'Brien, MD. "The HDSA has been leading the search for a cure for nearly four decades, and it is because of their work that HD has gone from being a virtually unknown disorder to one that is widely recognized as a priority for research and funding. I am proud to be part of their team."
Dr. O'Brien has served as investigator or director for more than 100 clinical trials and has authored more than 75 publications in the neuroscience literature. He is Fellow of the American Academy of Neurology and has served on the boards of various national foundations supporting Huntington's Disease, Parkinson's Disease, Dystonia and Tourette's Syndrome. Dr. O'Brien received his MD from the University of Minnesota, his neurology training at Minnesota and fellowship training in movement disorders and neuropharmacology at the University of Rochester.
HDSA
HDSA is a national, non-profit health organization dedicated to finding a cure for Huntington's Disease while providing support and services for those living with HD and their families. Founded in 1967 by Marjorie Guthrie, HDSA promotes and supports both basic and clinical HD research, aids families throughout the continuum of HD and educates families, the public and healthcare professionals about this devastating disease.
Huntington's Disease is an inherited, progressively degenerative brain disorder that results in a loss of both mental faculties and physical control. Symptoms usually appear in an individual between 30-50 years of age and progress over a 10-25 year period. HD affects the individual's ability to think, speak and walk. Presently, there is no effective treatment or cure. For more information about HD and HDSA please visit the national web site at www.hdsa.org or call (800) 345-HDSA.
Prestwick Pharmaceuticals
Prestwick Pharmaceuticals, Inc. is an emerging specialty pharmaceutical company that focuses on treatments for CNS disorders. The company has multiple product candidates in clinical development for Huntington's Disease, Parkinson's Disease and schizophrenia.
Prestwick recently announced positive Phase III results of the investigational drug tetrabenazine for chorea associated with Huntington's Disease. The company anticipates filing a New Drug Application (NDA) for tetrabenazine with the U.S. Food and Drug Administration (FDA) in the near future. Prestwick was granted fast track and orphan drug status by the FDA. Tetrabenazine is available in some European markets and Australia as XENAZINE(TM) and in Canada as NITOMAN(R) for the treatment of hyperkinetic movement disorders. For more information, visit http://www.prestwickpharma.com.
Posted by Dave at 11:35 PM | Comments (0) | TrackBack
October 20, 2004
Woo Hoo - HDSA & Sirna Pursue RNAi
Sirna Therapeutics has announced that it is going to start development of a RNAi treatment for Huntington's Disease. In a few months they'll select the compounds to use in preclinical trials.
They also announced that they are collaborating with HDSA and their 'network of clinical investigators'.
Though expected, this is great news. I'm very excited about the future of this treatment. It will take several years before it is fully developed and receives FDA approval, but I believe this one will become the effective treatment/'cure' that we've been waiting for.
(It's entirely foolish make statements like this about any treatment that isn't even in preclinical trials as normally only a tiny percentage of treatments will ultimately make it to FDA approval. Yet, I stand by my opinion on this one. The science is solid and impressive.)
Here's the press release:
Sirna Therapeutics Announces RNAi Therapeutic Development Program to Address Huntington's Disease
Sirna Partners With Huntington's Disease Society of America
Studies Ongoing With Collaborators at University of Iowa to Assess RNAi-Based Compounds for Neurodegeneration
BOULDER, Colo., Oct. 20 /PRNewswire-FirstCall/ -- Sirna Therapeutics, Inc. (Nasdaq: RNAI) today announced that the Company is moving forward with the development of an RNA interference (RNAI)-based treatment for Huntington's Disease. Huntington's Disease (HD) is a devastating, degenerative brain disorder for which there is, at present, no effective treatment or cure. HD affects more than 30,000 people in the United States, with another 200,000 at risk of inheriting the deadly gene. Early in 2005, Sirna expects to select investigational compounds for preclinical development based on the groundbreaking work of the Company's research collaborator, Dr. Beverly Davidson at the University of Iowa. Sirna also announced today its collaboration with the Huntington's Disease Society of America (HDSA) and is looking forward to working with the Society's network of clinical investigators.
Howard Robin, Sirna's President and Chief Executive Officer, commented, "We are extremely proud of our new relationship with the Huntington's Disease Society of America. The Society's efforts have been instrumentcl in bringing hope to patients and their families affected by this devastating disease. Sirna's researchers and scientists are dedicated to suppressing the expression of the gene that causes Huntington's Disease."
Dr. Roberto Guerciolini, Senior Vice President of Development and Chief Medical Officer at Sirna Therapeutics, commented, "For many neurodegenerative diseases, including Huntington's Disease, treatments are either non-existent or can do little to stop or reverse the progression of the disease. Together with our collaborator, Dr. Beverly Davidson, Sirna is leading the effort to develop an effective treatment for Huntington's Disease."
Sirna's collaborator, Dr. Beverly Davidson, recently demonstrated that a short interfering RNA (siRNA) efficiently inhibited gene expression in an animal model of a disease mimicking spinocerebellar ataxia 1 (SCA1), a member of a class of inherited human neurodegenerative diseases that includes Huntington's Disease. The study appeared in the August issue of Nature Medicine (Volume 10, pp 816, August 2004). The Company has exclusively licensed key patents from the University of Iowa Research Foundation for the use of RNAi technology in the field of neurological diseases, including those relating to SCA1, Huntington's, Parkinson's and Alzheimer's Diseases. These patents complement Sirna's intellectual property in Huntington's Disease and further strengthen the Company's broad intellectual property portfolio covering the seminal RNAi technology, chemical modifications, therapeutic targets, delivery and manufacturing of siRNAs.
About RNA Interference
Sirna Therapeutics is using its proprietary technology and expertise in nucleic acids to develop a new class of nucleic acid-based therapeutics involving RNA interference. RNAi is a mechanism used by cells to regulate the expression of genes and replication of viruses. The RNA interference mechanism uses short interfering RNA (siRNA) to induce the destruction of target RNA using naturally occurring cellular protein machinery. Harnessing the natural phenomenon of RNAi holds potential for the development of a new class of drugs with specificity towards a wide range of diseases that result from undesirable protein production or viral replication.
About the Huntington's Disease Society of America
The Huntington's Disease Society of America (HDSA) is a voluntary health agency with chapters, affiliates, support groups and Centers of Excellence throughout the United States. The Huntington's Disease Society of America (HDSA) funds and supports research to find a cure, helps people and families affected by the disease, and educates the public and healthcare professionals about this genetic disease.
The organization was founded by the late Marjorie Guthrie, widow of famous folk singer Woody Guthrie after he died of complications related to Huntington's Disease in 1967.
About Sirna Therapeutics
Sirna Therapeutics is a clinical-stage biotechnology company developing RNAi-based therapies for serious diseases and conditions, including age-related macular degeneration (AMD), hepatitis, oncology, and diseases of the central nervous system. Sirna has filed an IND for its most advanced compound, Sirna-027, a chemically modified siRNA targeting the clinically validated vascular endothelial growth factor pathway to treat AMD. The Company has strategic partnerships with Eli Lilly and Archemix and a leading intellectual property portfolio in RNAi. More information on Sirna Therapeutics is available on the Company's web site at http://www.sirna.com.
Statements in this press release which are not strictly historical are "forward-looking" statements which should be considered as subject to many risks and uncertainties. For example, Sirna's ability to select promising investigational compounds for preclinical development and its ability to achieve any progress in suppressing gene expression for Huntington's Disease, are subject to considerable scientific uncertainty. Other risks and uncertainties include Sirna's early stage of development and short operating history, whether Sirna can achieve and maintain profitability, whether Sirna can obtain and protect patents, the risk of third-party patent infringement claims, whether Sirna can engage collaborators and obtain regulatory approval for products, Sirna's concentration of stock ownership, and availability of materials for product manufacturing. These and additional risk factors are identified in Sirna's Securities and Exchange Commission filings, including the Forms 10-K and 10-Q and in other SEC filings. Sirna undertakes no obligation to revise or update any forward-looking statements in order to reflect events or circumstances that may arise after the date of this release.
Sirna Contact: Martin E. Schmieg, Senior Vice President and Chief Financial Officer, Sirna Therapeutics, Inc., 303-449-6500
Media Contact: Justin Jackson, jjackson@burnsmc.com; Investor Contact: Aline Schimmel, aschimmel@burnsmc.com, Burns McClellan, Inc., 212-213-0006
HDSA contact: Deb Lovecky, 212-242-1968 extension 28, Dlovecky@hdsa.org.
Posted by Dave at 06:49 AM | Comments (0) | TrackBack
October 19, 2004
Hit & Run Hits HD Family
An unknown driver took the life of Seth Elmore, a 42-year-old man who was helping care for his brother & sister who have Huntington's Disease. Seth himself was also starting to experience some of the symptoms of the disease.
From the article:
When Elmore's parents died, he moved back to Rockford to help care for his brother and sister who suffer from Huntington's, a degenerative brain disease that Elmore was also starting to battle.
"He was in the early stages of Huntington's and was very fiercely trying to maintain his independence," says Bauer.
Despite the disease, Elmore worked part time while helping to care for his brother and sister and lead a full and active life.
Even now, Elmore will continue to make the lives of other better. His family has donated his organs to help individuals with medical needs.
Posted by Dave at 06:57 PM | Comments (0) | TrackBack
October 18, 2004
Guthrie Awards Dinner
In case you missed it, here's the rundown on HDSA's upcoming Guthrie Awards Dinner:
The Annual Guthrie Awards Dinner, is a tribute to the memories of legendary folk singer Woody Guthrie and his wife, Marjorie, principal founder of the national drive to cure Huntington's Disease (HD). The Society is proud to have the support of Nora Guthrie and Arlo Guthrie, as well as the Woody Guthrie Foundation and Archives, in planning this annual awards dinner.
The Guthrie Award Dinner raises funds for the Woody and Marjorie Guthrie Research Fund, which directly supports the HDSA Coalition for the Cure.
The 2004 awards will take place at New York City's Waldorf Astoria Hotel, on Thursday, October 21, 2004. Cocktail Reception is at 6:00 pm and the Dinner will be held at 7:00 pm.
2004 Award Recipients:
Marjorie Guthrie Leadership Award -
Novartis International AG, Robert Pearson
Harold Leventhal Award -
JP Morgan Chase, C. Mack
Guthrie Family Humanitarian Award -
Christopher O'Brien, MD, Chief Medical Officer, Prestwick Pharmaceuticals
Woody Guthrie Award -
Peter, Paul and Mary
Posted by Dave at 09:43 PM | Comments (0) | TrackBack
October 17, 2004
HDSA Holiday Cards
HDSA has holiday cards available for sale. 40% of the price goes to Huntington's Disease research.
You're going to need them anyway...you might as well help the cause at the same time. Available here.
Posted by Dave at 11:36 PM | Comments (0) | TrackBack
October 16, 2004
Minocyline Study - Neuroprotective
There are a whole host of things that can cause a 14-person study to give bad results. As a result, such a small study can never be considered to offer conclusive evidence. Having said that...
This two year study followed 14 HD patients as they took Minocycline. The purpose of the study was to determine if the drug protected the brain cells from the effects of Huntington's Disease. The answer appears to be 'yes'. Testing showed that the patients stabilized and even showed improvement in psychiatric symptoms.
Here's the study abstract:
Neuroprotection in Huntington's disease: a 2-year study on minocycline.
Int Clin Psychopharmacol. 2004 Nov;19(6):337-42.
PMID: 15486519 [PubMed - in process]
Bonelli RM, Hodl AK, Hofmann P, Kapfhammer HP.
Department of Psychiatry, Graz Medical University, Graz, Austria.
Huntington's disease (HD), a relentlessly progressive neurodegenerative disorder, is characterized by a clinical triad of psychiatric, cognitive and motor disturbances. The antibiotic minocycline, a caspase inhibitor exhibiting antiapoptotic properties, has been shown to prolong survival in the transgenic mouse model of HD.
We administrated minocycline to 14 patients with genetically confirmed HD. The patients were psychiatrically, neurologically and neuropsychologically evaluated at baseline, and after 6 and 24 months of treatment, using the Unified HD Rating Scale and a neuropsychological test battery. After 12 months, three patients were lost to follow-up so that 11 patients were analysed at the endpoint.
Minocycline was well tolerated. Unlike the expected natural course of HD, patients exhibited stabilization in general motor and neuropsychological function at endpoint, after improving in the first 6 months. Moreover, we found a significant amelioration of psychiatric symptoms that was not apparent after the first 6 months.
In detail, the Mini-Mental State Examination, the Total Motor Score, the Total Functional Capacity Scale and the Independence Scale, as the most prominent scales in HD, were stabilized after 3 years of treatment. Our results confirm previous animal studies and indicate a neuroprotective effect of this agent in HD. A long-term, double-blind, placebo-controlled trial appears highly warranted for definitively establishing the value of minocycline in HD.
Posted by Dave at 11:39 PM | Comments (0) | TrackBack
October 15, 2004
Medical Research On The Campaign Trail
The important topic of medical research is finally reaching the presidential race.
For the most part, I'm glad it's happening. Voters are being exposed to the need in our society of medical research. Granted, a number of the 'facts' being thrown around are BS, but at least it is being discussed.
This week John Edwards gave a speech this week and he said "If we do the work that we can do in this country, the work that we will do when John Kerry is president, people like Christopher Reeve are going to walk, get up out of that wheelchair and walk again."
This statement created quite a bit of outrage, especially from paraplegic Charles Krauthammer who wrote "In my 25 years in Washington, I have never seen a more loathsome display of demagoguery. Hope is good. False hope is bad. Deliberately, for personal gain, raising false hope in the catastrophically afflicted is despicable."
Ouch. Well, yes, it's true about the demogoguery (in English - B.S.) but we have a candidate for vice-president saying that we have the ability to cure the serious medical ills of this society should we choose to fund it. That's a good thing.
It's about time we had this discussion. I just wish it wasn't limited to embryonic stem cells. There is a lot more research going on out there, such as RNAI, that offers great promise, especially for our community.
As far as the debate...Bush has greatly increased spending on medical research during his tenure. Under his watch, NIH has done a great job reorganizing in a way that will greatly help research Huntington's Diseae research. Now, Bush is recommending the increases in spending for NIH slow to around the rate of inflation. Kerry is promising more NIH spending (though his drug-reimportation plan would greatly hurt non-NIH spending on medical research).
I just wish the discussion extended beyond the limited field of embryonic stem cells. Oh well, something is better than nothing. Right?
Posted by Dave at 05:04 PM | Comments (0) | TrackBack
October 14, 2004
Another Press Release On The Neural Cell Findings
Here's another press release, this time from the Gladstone Institute of Neurological Disease, on the neural cell study:
Gladstone researchers resolve key Huntington's disease mystery in Nature cover story
Use innovative robotic microscopy to image molecular processes in neurons
A mystery long associated with Huntington's disease has been resolved by a team of researchers at the UCSF-affiliated Gladstone Institute of Neurological Disease, thanks to a specially designed microscope that allows researchers to track changes in cells, including those associated with neurodegeneration, over long lengths of time.
As reported in the cover story in the current issue of Nature (Oct. 14, 2004), the team determined that abnormal deposits of mutant huntingtin protein, which appear in the brains of all Huntington's disease patients, aren't the cause of neuronal death. Scientists know that mutant huntingtin protein is responsible for the disease, but they have not known in what form it wreaks its havoc. They haven't known, for instance, whether the abnormal deposits of the protein, known as "inclusion bodies," were, themselves, causative, protective or incidental to the disease. In the current study, the Gladstone team determined that inclusion bodies are a beneficial coping response, possibly sequestering mutant huntingtin protein, thereby reducing levels of the protein elsewhere in the neuron, and thus prolonging neurons' survival.
The finding suggests that mutant huntingtin protein inflicts its damage in some form other than as inclusion bodies, which are insoluble, or resistant to being dissolved in liquid. Investigators may now focus attention on the possibility that the real culprit is a more soluble form of mutant huntingtin spread throughout the neuron, or nerve cell, among other theories.
"We are very excited by these results," says lead investigator Steven Finkbeiner, MD, PhD, an assistant investigator at the Gladstone Institute of Neurological Disease and assistant professor of neurology and physiology at University of California, San Francisco (UCSF). "They will help us to better focus efforts to identify the mechanisms by which the huntingtin protein causes Huntington's and may add to the understanding of other neurodegenerative disorders."
Traditionally, scientists have tried to illuminate the role of the mutant protein within neurons by taking one-time snapshots of individual cells, a slow process that doesn't allow researchers to track changes in any given cell over time. Beyond slowness, a fundamental problem with this conventional approach is that the snapshots are not only taken at different times but also each image is of a completely different population of cells than the other. Scientists have tried to use these images to piece together theories of disease progression, but have had great difficulty interpreting their results because of the lack of continuity between images.
To address these issues, Finkbeiner developed an automated microscope that allows researchers to track changes in individual neurons over time, thus enabling them to identify factors that predict the fate of the cell.
"With this new technology, we can examine neurons well before they die, make measurements of whatever we wish, and then determine which factors have prognostic value, whether they predict survival or neurodegeneration, and how strong the prediction is. This is a powerful new way to guide our investigation into the underlying mechanisms of neurodegeneration," he explains.
In their study, the scientists introduced fluorescently tagged versions of huntingtin protein into neurons. They then used the robotic microscope to monitor the accumulation of the abnormal protein into inclusion bodies, as well as to monitor the levels of intracellular huntingtin protein, and the length of survival of thousands of individual cells over time. Sophisticated statistical techniques for survival analysis were then used to determine whether a particular abnormality predicted early death and might be pathogenic, or predicted longer survival and might be beneficial.
The findings suggest, says Finkbeiner, that inclusion bodies lock up mutant huntingtin in other parts of a cell and keep it from interfering with the rest of the neuron in ways that can trigger cell death. These findings provide evidence that inclusion bodies in Huntington's disease, and possibly other neurodegenerative diseases, help neurons cope with toxic proteins and prevent neurodegeneration.
The approach developed by the Finkbeiner group -- combining the use of a robotic microscope with powerful techniques of statistical analysis -- could also be used in studies of other neurodegenerative diseases characterized by the accumulation of cellular proteins, including Alzheimer's disease, prion diseases, amyotrophic lateral sclerosis (Lou Gehrig's disease), Parkinson's disease, and a group of nine so-called polyglutamine diseases of which Huntington's is the most widely known.
Moreover, the approach could be used to measure the nature and magnitude of the relationship between any two biological events within a cell that are separated by time. With this tool, researchers can begin to answer such fundamental questions as:
Is there a relationship at all, or are the two events simply coincidental?
If there is a relationship, is one event possibly the cause or the effect of the other?
If it is a cause, is it a minor determinant or a major one?
These are questions that recur in all aspects of cell biology.
As Professor Harry T. Orr of the University of Minnesota explains in a companion Nature commentary, "In the long term, strength of this study lies in the approach itself. The capability to determine if a cellular feature of a disease is pathogenic, beneficial or merely incidental to a disease process will be of considerable advantage for understanding disease mechanisms. Will the results reported here end the debate on the pathogenic role of inclusion bodies in the polyglutamine diseases? If not, one wonders what would."
Huntington's disease is a hereditary, progressive neurodegenerative disorder characterized by the development of emotional, behavioral, and psychiatric abnormalities, loss of intellectual and cognitive functioning, and motor disturbances. Although symptoms typically become evident during the fourth or fifth decades of life, the age at onset is variable and ranges from early childhood to the 70s or 80s. It's named for the American physician who initially described the condition in 1872.
The paper, "Inclusion body formation reduces levels of mutant huntingtin and the risk of neuronal death," was co-authored by Finkbeiner and fellow GIND staff members Montserrat Arrasate and Siddhartha Mitra; Erik S. Schweitzer of the Brain Research Institute, UCLA; and Mark R. Segal of the Division of Biostatistics, UCSF. Primary support for this work was provided by the National Institute of Neurological Disorders and Stroke (NINDS), part of the National Institutes of Health (NIH). Additional support was provided by the National Institute of Aging within the NIH, and the J. David Gladstone Institutes.
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The Gladstone Institute of Neurological Disease is one of three research institutes of The J. David Gladstone Institutes, a private, nonprofit biomedical research institution affiliated with UCSF. For further information, visit www.gladstone.ucsf.edu/gind.
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October 13, 2004
NIH On The New HD & Neural Cell Findings
NIH Press Release on the new findings on HD & the inner workings of neural cells:
Study Using Robotic Microscope Shows How Mutant Huntington's Disease Protein Affects Neurons
Using a specially designed robotic microscope to study cultured cells, researchers have found evidence that abnormal protein clumps called inclusion bodies in neurons from people with Huntington's disease (HD) prevent cell death. The finding helps to resolve a longstanding debate about the role of these inclusion bodies in HD and other disorders and may help investigators find effective treatments for these diseases. The study was funded primarily by the NIH's National Institute of Neurological Disorders and Stroke (NINDS) and appears in the October 14, 2004, issue of Nature1.
Inclusion bodies are common to many neurodegenerative disorders, including HD, Parkinson's disease, Alzheimer's disease, and amyotrophic lateral sclerosis (ALS). The role of inclusion bodies in these diseases has long been controversial. Some studies suggest that they may be a critical part of the disease process, while others indicate that they may help protect the cells from toxic proteins or that they are merely bystanders in the disease process.
One problem in identifying how inclusion bodies influence disease is that researchers have been unable to track changes in individual neurons over time. "It was like viewing pictures of a football game and trying to imagine the score," says Steven Finkbeiner, M.D., Ph.D., of the Gladstone Institute of Neurological Disease and the University of California, San Francisco. "Much was happening that we couldn't see."
To overcome this problem, Dr. Finkbeiner and his colleagues wrote a computer program that allows a microscope to match images in a culture dish to images it has stored and to manipulate its controls to look at the same neurons over and over again — like time-lapse photography. This allowed the investigators to follow changes in a single neuron or a group of neurons over a period of days. They used this automated microscope to study neurons that contained a version of the huntingtin protein that causes HD. The huntingtin was fused to green fluorescent protein, a widely used marker that allows researchers to see where proteins accumulate.
Many neurons with the mutated HD gene died without forming inclusion bodies, the researchers found. The formation of inclusion bodies actually prolonged neurons' survival and lowered their overall risk of death. The rate of cell death was higher in neurons with larger gene mutations, but the death rate for each set of cells remained constant over time.
The researchers also examined the level of mutant huntingtin protein spread throughout the neurons, outside of inclusion bodies. They found that neurons with larger amounts of mutant huntingtin spread throughout the cell died more rapidly than cells with less of this protein. The amount of mutated protein decreased in other parts of the cell when inclusion bodies formed. Taken together, these findings suggest that inclusion bodies lock up mutant huntingtin and keep it from interfering with the rest of the neuron in ways that can trigger cell death.
These findings provide evidence that inclusion bodies in HD, and possibly other neurodegenerative diseases, help neurons cope with toxic proteins and avoid neurodegeneration. Many researchers have been working to develop ways of interfering with inclusion body formation as potential treatments for HD and other disorders. This study suggests that finding ways to remove mutant proteins diffused throughout the cell might be a more effective approach.
"This approach provides a way to connect cellular changes to fate," says Dr. Finkbeiner. The automated microscope system could be applied to sort out many important questions about how cellular changes or abnormalities affect disease, he adds. He and his colleagues are now planning studies to examine the role of proteasomes — enzyme-filled compartments that break down and recycle proteins — in HD.
The NINDS is a component of the National Institutes of Health within the Department of Health and Human Services and is the nation' primary supporter of biomedical research on the brain and nervous system.
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1 Arrasate M, Mitra S, Schweitzer ES, Segal MR, Finkbeiner S. "Inclusion body formation reduces levels of mutant huntingtin and the risk of neuronal death."Nature, October 13, 2004, Vol. 431, No. 7010, pp. 805-810.
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Rethinking Huntington's Disease
This study could have profound implications in the future of HD research. If this turns out to be true, this will help further focus research.
Read the article, but here's an excerpt:
He also points out that the aggregation of huntingtin might still play a detrimental role in the disease. But the extremely large protein clumps that form inclusion blobs appear to be a sign of cells fighting back, he says.
The result could change the development of HD therapy. Researchers are already looking for drugs that keep huntingtin from aggregating. But some of these drugs might prevent the formation of protective inclusion bodies while allowing the smaller – and possibly lethal - groups of huntingtin to form. "One prediction of ours is that some of these drugs could actually make the disease worse," he says.
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October 12, 2004
Argument Against Nationalized Research
Derek Lowe writes an interesting an provocative article on why private research speeds the drug development process:
So we race each other in the clinic, all the way through, trying to figure out what everyone else is up to, and then we fight it out in the market. And it's a mess! It's inefficient and it's wasteful! But the hellacious part is, it's the best way that anyone's found to do it. We chase the reward of a successful drug, and we fear failure - losing out to another company, or worse, losing out to hordes of swarming lawyers. What else would make us jump as high, what else would make us run as fast?
As they say..."Read the whole thing."
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October 11, 2004
More On NeurotrophinCell
"This is the first time that technology of this kind has been proven in a controlled pre-clinical setting to prevent the degeneration of the brain due to Huntington's disease-like conditions,"...
The technology protects the cells from attack by the body's immune system.
This allowing them to produce several factors important to the health and survival of the brain.
Alfred Vasconcellos, chief executive of Living Cell Technologies's US operation, LCT BioPharma, said the work had shown "the ability of LCT's technology to protect brain tissue that would otherwise die, potentially forestalling and preventing the debilitating consequences of this disease".
LCT chief operating officer Roger Coats said the company had another 12 months or so of pre-clinical trials to complete before moving into clinical trials.
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October 10, 2004
NeurotrophinCell Success
Living Cell Technology released the results from their pre-clinical trial studies for NeurotrophinCell and the results were amazing. There was 86% less brain damage.
This animal study was not conducted against Huntington's Disease, but Living Cell hopes to get NeurotrophinCell approved as a treatment for HD. Based on the results of this study, it would be surprising if NeurotrophinCell wasn't a helpful treatment for Huntington's Disease.
From the article:
"These findings have major implications for enabling treatment of human neurodegenerative diseases such as Huntington's and stroke," LCT researcher Dwaine Emerich said.
Dr Emerich added that in the recent study, new choroid plexus cells, which produce cerebral spinal fluid, had been successfully implanted to protect specific areas of the brain from damage.
In NeurotrophinCell, the choroid plexus cells are covered in a clear capsule derived from algae, which hides the cells from the patient's immune system but allows the cells to receive nutrients and chemical signals needed to survive and function.
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October 09, 2004
Reducing/Increasing Research.
"...would you be willing to trade a 10% reduction in price for a 20% reduction in the growth rate of new drugs?"
Research is, ultimately, driven by money. It's expensive. We've seen what Amarin has gone through to survive and bring a promising Huntington's Disease drug to the market.
This article on the "Marginal Revolution" blog has an excellent discussion about the effects price controls have on reducing medical research. This topic is of vital importance to our community because, due to the relative rarity of Huntington's Disease, our research is more vulnerable to drops in available research money.
Acemoglu and Linn's paper is formally about a different issue; the effect of market size on innovation. What they find is that a 1 percent increase in the potential market size for a drug leads to an approximately 4 percent increase in the growth rate of new drugs in that category. In other words, if you are sick it is better to be sick with a common disease because the larger the potential market the more pharmaceutical firms will be willing to invest in research and development. Misery loves company.
...
We can expect, therefore, that a 1% reduction in price will reduce the growth rate of new drug entries by 4% and a 10% reduction in price will reduce new drug entries by 40%. That is a huge effect. I suspect that the authors have overestimated the effect but even if it were one-half the size would you be willing to trade a 10% reduction in price for a 20% reduction in the growth rate of new drugs? No one who understands what these numbers mean would think that is a good deal.
As i mentioned before, reductions in research would hit HD research harder than it would for other diseases with larger pools of potential buyers.
Posted by Dave at 12:52 PM | Comments (0) | TrackBack
October 08, 2004
Positively 'Gun Shy'
As we approach the election, any discussion remotely related to politics now has the potential of becoming a fight.
I'm not interested in fighting anything except Huntington's Disease. Because this fight necessarily involves public money and public policy decisions I've included related topics here on this website. Now, and for the next four weeks, I'll have some more items relating to the election. For example, I haven't yet written as I had promised on the Bush acceptance speech nor the current stands on the candidates on health care related issues.
I should have done it by now. Early voting has already started in some places and the climate is becoming increasingly polarized making polite discussions on policy more difficult.
So ya...I'm a bit 'gun shy' about writing on certain topics until after the election - like this next article on the effects of price regulation on medical research.
Let's fight Huntington's Disease & not each other this political season.
Posted by Dave at 11:56 PM | Comments (0) | TrackBack
October 07, 2004
Amarin Loads Up
Now it's becoming clear why the Miraxion/LAX-101 trial isn't starting until 2005...Amarin needed the time to arrange the finances to fund the trial. Now they're ready.
Today Amarin announced that they've raised another $12.75 million while enabling the company to remain as a traded stock on NASDAQ. It's important to note that Amarin's management is buying stock. That's an important vote of confidence in the future of Amarin.
Amarin is banking heavily on the success of a Miraxion Phase III trial. They've got good reason to expect success. It appears that Miraxion is very effective in treating/slowing the Huntington's Disease in patients with CAG counts under 46. (It may also help those with higher counts, but to a lesser degree.)
Here's the press release from Amarin:
$3 Million of Existing Debt Converted Into Ordinary Shares
LONDON, October 7 - Amarin Corporation plc (NASDAQSC: AMRN) today announced that it has completed a private placement of 13,448,546 ordinary shares to a group of new and existing accredited investors and management, raising gross proceeds to the Company of $12.75 million.
The purchase price of $0.947 per share was based on the average closing price of Amarin's American Depositary Shares on Nasdaq for the ten trading days ended October 6, 2004. Amarin intends to file a registration statement with the U.S. Securities and Exchange Commission within 60 days covering the ordinary shares sold to investors.
As previously announced, following Mr. Thomas Lynch's purchase of Elan Corporation plc's (Elan) entire debt and equity interest in Amarin, $3 million of the outstanding $5 million Loan Notes acquired were converted into ordinary shares immediately following the closing of this private placement. The debt was converted at a price of $1.104, a 16.6% premium to the private placement price. Amarin's management investing in the private placement also bought shares at this higher price to comply with Nasdaq rules. The remaining $2 million of Loan Notes can be converted into ordinary shares at the option of the holder at the offer price of any subsequent equity financing.
Rick Stewart, chief executive officer of Amarin, commented, "This successful financing allows Amarin to drive forward with its plans for the phase III clinical trials with MiraxionTM, our lead compound for Huntington's disease. Additionally, it will facilitate in-licensing discussions for new late-stage compounds and out-licensing discussions with prospective partners for indications outside neurology"
Amarin's future financing strategy will depend on the timing of clinical trial expenditure on Amarin's development pipeline, in particular the proposed phase III trials with MiraxionTM in Huntington's disease, plus the revenue generated from its licensing and partnering activities. As previously described, Amarin is seeking to partner the rights to its development pipeline for all indications outside neurology and for geographic markets outside the U.S. Amarin plans to directly commercialize its pipeline in the U.S. neurology market.
Amarin also announced today that the settlement agreement with Valeant Pharmaceuticals International (Valeant), announced on September 29, 2004, is now unconditional, as all conditions precedent to closing have been met.
Since September 29, 2004, Amarin has announced a number of transactions which add approximately $16 million to its shareholders' equity. The announcements are summarised as follows:
- On September 29, Amarin announced that it had reached a settlement agreement with Valeant whereby $6 million of the $8 million in contingent milestones due to Amarin from Valeant were waived. The remaining $2 million is now no longer contingent and is payable by Valeant to Amarin on November 30, 2004. After deducting $1 million due to Elan under the asset purchase agreement the net benefit to Amarin's shareholders' equity is $1 million; and
- On October 1, Amarin announced that its non-executive chairman, Mr. Thomas Lynch, had acquired Elan's entire debt and equity interest in Amarin, including the $5 million Loan Notes. Mr. Lynch has agreed to convert $3 million of the $5 million Loan Notes into ordinary shares. This debt conversion, which took place today, will improve Amarin's shareholders' equity by $3 million; and
- Today, Amarin announced the completion of a $12.75 million private placement, which will improve Amarin's shareholders' equity by approximately $12 million.
Rick Stewart continued, "Our original objective was to raise approximately $10 million, however significant investor interest increased that amount to $12.75 million. We limited the fund raising to $12.75 million in order to restrict shareholder dilution, even though we had demand in excess of this level."
Amarin had been previously informed by Nasdaq that it did not comply with the minimum shareholders' equity threshold of $2.5 million as set forth in Marketplace Rule 4310(c)(2)(B) ('the Rule'). Amarin believes that the improvement in Amarin shareholders' equity as outlined above restores Amarin's compliance with the Rule. Nasdaq will continue to monitor the Company's ongoing compliance with the Rule and, if at the time of its next relevant periodic report the Company does not evidence compliance, it may be subject to delisting.
The securities being offered have not been registered under the Securities Act of 1933, as amended and may not be offered or sold within the United States absent registration or an available exemption from such registration requirements. However, pursuant to the agreement with the investors, the Company is required to register the resale of the securities and the securities issuable upon exercise of the warrant under the Securities Act.
About Amarin Corporation
Amarin Corporation plc is a neuroscience company focused on the development and commercialisation of novel drugs for the treatment of central nervous system disorders. Miraxion is in phase III development for Huntington's disease and is in phase II development for treatment unresponsive depression.
For press releases and other corporate information, visit our website at http://www.amarincorp.com.
Statements in this press release that are not historical facts are forward-looking statements that involve risks and uncertainties which may cause the Company's actual results in future periods to be materially different from any performance suggested herein. Such risks and uncertainties include, without limitation, the uncertainty of entering into and consummating a definitive agreement on terms acceptable to the parties, the inherent uncertainty of pharmaceutical research, product development and commercialization, the impact of competitive products and patents, as well as other risks and uncertainties detailed from time to time in periodic reports. For more information, please refer to Amarin Corporation's Annual Report for 2003 on Form 20-F and its Form 6-Ks as filed with the U.S. Securities and Exchange Commission. The company assumes no obligation to update information on its expectations. There can be no assurance that the Company has regained compliance with Market Place Rule 4310(c)(2)(B) until the Company has received notification from Nasdaq.
Posted by Dave at 10:50 AM | Comments (0) | TrackBack
October 06, 2004
Comments Are Working Again
We're slowly getting the new site fully functional. You can now post comments to messages.
The process has changed slightly as comments are now authenticated by Typekey. Hopefully this will stop the spammers who use automated programs to dump 100's of ads on this site.
Posted by Dave at 09:53 PM | Comments (2) | TrackBack
DNA & 'Cures"
Remember the discussions after the HD gene was found in 1993? There was a lot of enthusiasm and many believed "that a cure is right around the corner."
That was 11 years ago. What researchers have since found was...how much they didn't know. I'm amazed at just how much has been learned in just the last three years alone. Each discovery is quickly building on each other.
Derek Lowe has another excellent article on his blog called "DNA to Drug?" which gives an insiders view to the difficulties researchers are facing.
His article reflects the frustion that the pharmaceutical industry has faced in turning DNA knowledge into treatments. In a nutshell - it ain't easy folks.
We're fortunate in that Huntington's Disease is single-gene disease & that we've had a few researchers, such as Dr. Nancy Wexler, who've done unbelievable work.
In our corner of the DNA/drug world, I've very excited and I believe the RNAi research coming out of the University of Iowa will ultimately lead to a very effective treatment for Huntington's Disease. But that is still 6-9 years away from reality (i.e. FDA approval) and, should it happen, HD would be one of the earliest diseases 'cured' in such a manner. That's 20 years after the discovery of the gene.
Derek's right, converting DNA knowledge into drugs is tough work. But the effort will be worth it. Twenty, thirty, fifty years from now historians will look back with amazement at the technologies that were developed and the diseases that were 'cured'. And that will be because of the hard work done by Derek and thousands of other researchers.
Posted by Dave at 08:08 PM | Comments (1) | TrackBack
October 05, 2004
Stem Cell Treatment For HD
ReNeuron, a British stem cell research and development company, has an HD stem cell therapy they intend to enter into Phase I trials.
The therapy, ReN005, is derived from the same stem cell line as their stroke therapy 'ReN001'. They would be surgically implanted into the brain and, hopefuly, produce dopamine. This potential treatment is not a 'cure', but it has the potential of possibly slowing the progression of the disease.
It will take many years for ReN001 to come to market (assuming it is successful in doing so).
No thanks to the press and politicians, there is confusion on the subject of stem cells. This therapy uses 'adult' stem cells that were taken from fetuses and grown into this line. These are not 'embryonic' stem cells which have the problems of forming tumors.
Here's the press release with more information:
ReNeuron Business Update and Funding Plans
Tuesday October 5, 7:03 am ET
TORONTO, Oct. 5 - ReNeuron, the UK-based stem cell research and development company, is pleased to give an update on recent progress and future plans regarding its stem cell programmes. The Company is today separately announcing groundbreaking pre-clinical efficacy data with its ReN001 cell therapy programme for stroke. Based on this progress, the Company also announces its intention to seek further funding in order to progress its lead stem cell therapy programmes to the clinic.
Technology platform
ReNeuron's focus is on translating the enormous potential of stem cells into real and practical therapies capable of serving significant unmet or poorly met medical needs. Based around its proprietary c-mycER(TAM) cell immortalisation technology, ReNeuron has now developed a fully controllable system for generating somatic, or lineage-restricted, human stem cell lines. These cell lines have stable, normal genomes and are non-tumour-forming, both pre-requisites for future clinical development. They are also capable of immediate scale-up for clinical use and therefore form the basis of a commercially viable stem cell therapy product.
Stroke programme
Over the last year, ReNeuron has developed a rigorous screening and selection process to identify its most promising neural (brain) stem cell lines and has taken these cell lines forward into pre-clinical efficacy testing in various central nervous system disease models. From these studies, the Company has achieved important and unique positive efficacy results in a stroke model using ReN001, one of its lead stem cell lines. These results are being presented today at the American Neurological Association Annual Meeting in Toronto, Canada.
ReNeuron is now taking its ReN001 stem cell therapy for stroke forward into manufacturing scale-up and late pre-clinical testing, and hopes to have approval to commence clinical trials by the end of 2005. The Company believes that ReN001 could be the first such clinical-stage neural stem cell programme addressing a major disease condition.
Other therapeutic programmes
ReNeuron has broadened its stem cell therapy pipeline by initiating programmes addressing a number of other significant and currently incurable diseases.
The Company is deriving pancreatic and retinal stem cell lines and has established academic and commercial partnerships to use these cell lines to develop stem cell therapy treatments addressing Type 1 diabetes (ReN002) and diseases of the retina such as age-related macular degeneration and retinitis pigmentosa (ReN003). Together with ReN001 for stroke, these are ReNeuron's lead stem cell therapy programmes, with pre-clinical efficacy testing scheduled to commence shortly with ReN002 and ReN003.
ReNeuron has also established and presented a method for generating dopamine-producing stem cells at concentrations not achieved elsewhere to the Company's knowledge. This has made possible the initiation of a stem cell therapy programme, ReN004, addressing Parkinson's disease.
Finally, the Company has a programme, ReN005, addressing Huntington's disease. Progress with this programme is in part dependent upon late pre- clinical success with the ReN001 stroke programme, as the same cell line would be used in both indications.
ReNeuron continues to strengthen its patent position in stem cell therapy, having filed a number of new patent applications in the last year.
Non-therapeutic programmes - ReNcell
ReNeuron has generated and presented data showing that its lead ReNcell lines exhibit the electrophysiological and other properties necessary for their use in non-therapeutic applications such as drug discovery. These cell lines are currently under evaluation by a number of commercial and academic organisations. A second generation ReNcell hepatocyte (liver) cell line is under development for toxicology screening applications.
Funding
In order to build on its recent successes and progress the above programmes, ReNeuron is seeking between 10 million and 15 million pounds sterling in further private equity funding. The Company will deploy these funds over the next two to three years, taking its ReN001 programme through initial Phase I/II clinical trials, as well as taking its diabetes and retinal programmes through pre-clinical development.
Michael Hunt, Chief Operating Officer and Finance Director of ReNeuron commented:
"ReNeuron has come a long way in the last year, culminating in the breakthrough efficacy data in stroke that we are announcing today. The Company is now poised to take a leading competitive position in the stem cell therapy field.
"The funding we are seeking will take ReNeuron into the next phase of its development over the next couple of years or so. By that time, we hope to have achieved the next benchmark position -- first clinical data in our stroke programme and our other lead cell therapy programmes well on their way to the clinic."
NOTE TO EDITORS:
ReNeuron is a privately held, UK bio-pharmaceutical company and a pioneer in stem cell research and development. The Company has leading edge, proprietary stem cell technologies from which it is developing groundbreaking cell therapy products. ReNeuron's focus is on cell therapy treatments designed to reverse the effects of major diseases such as stroke, diabetes and diseases of the retina.
ReNeuron has also leveraged its stem cell technologies into non-therapeutic areas such as drug discovery -- its ReNcell product.
More information on ReNeuron and its programmes can be found on the Company's website at http://www.reneuron.com.
Posted by Dave at 11:27 PM | Comments (0) | TrackBack
October 04, 2004
Cyberchondria
This Welsh newspaper has an article on 'cyberchondria'. This is a term that cropped up a few years ago in the press. The word relates to people who find diseases on the internet and then go to the doctor believing they have this disease. In other words, a variation of hypochondria.
Doctors blame the wealth of web-based information for a rise in "cyberchondria" and claim scores of patients are heeding poor or mistaken advice. They claim many patients make their own incorrect self-diagnosis after reading about conditions and then seek treatment they do not need.
Ok, sounds reasonable. The Internet allows for the freeflow of information. Incorrect, as well as correct information is easily disseminated. There certainly are scam artists that use the Internet to peddle fake remedies.
Also in the article:
The study examined sites dedicated to helping sufferers and carers of people with skin disorders, HIV and Aids, diabetes, cancer and Huntington's Disease. It found that most sites connected to societies, charities or professional bodies offered sound advice, but that those set up by individuals could include glaring inaccuracies.
Hmmm, HD Blog is a website setup by an individual and you know what? It is entirely possible that there could be a "glaring inaccuracy". And there have been. For example, one time I typed an abbreviation for "grams" instead of milligrams. A kind reader pointed that out.
Finally the article finishes with:
The team has urged health professionals to offer information and guidance to any website with mistakes or untruths. It also praised the internet as "a great source of comfort" that provides a vital support network to millions of people when used correctly.
I couldn't agree more. But I'll add one comment. Doctors are not perfect either. If you are not comfortable with something your doctor has said or done, talk to them about it. If you don't like the answer...see a different doctor. Not every doctor is the right one for you. The better informed patients will tend to get better care.
Posted by Dave at 09:23 AM | Comments (2) | TrackBack
October 03, 2004
Tapestry
Tapesty Pharmaceuticals (formaly NaPro) has a paper published in the Journal of Molecular Neuroscience where they discuss their proprietary
oligonucleotides that appear to prevent the mutant huntingtin protein from 'clumping'. This could end up, one day, being an effective treatment for Huntington's disease.
Tapestry has also announced that they are starting a second mouse study (probably in preparation for an initial human study) to measure various dosing levels.
Technology Detailed in September Issue of Journal of Molecular Neuroscience
BOULDER, Colo., Sept. 30 - Tapestry Pharmaceuticals, Inc. announced today that the Company is initiating a second in vivo study to establish the dose response for its proprietary oligonucleotides in a transgenic mouse model of Huntington's disease (HD). The new in vivo study is designed to further validate the results of previous studies as to the efficacy of Tapestry's proprietary oligonucleotides. These oligonucleotides may potentially inhibit the aggregation of the huntingtin protein, which is thought to be a leading cause of neurotoxicity and neuronal cell death in HD. To date there is no effective treatment for the disease, which afflicts approximately 35,000 people in the United States.
The Company also announced that Dr. Eric Kmiec, Professor of Biological Sciences at the University of Delaware and Senior Scientific Advisor to Tapestry, has published a paper describing the discovery of these proprietary oligonucleotides. The report, in the September issue of the Journal of Molecular Neuroscience, disclosed that the oligonucleotides were found to retard inclusion body formation in a model neuronal cell line. The in vitro assay was used in conjunction with a standardized biochemical assay to identify molecules that could disrupt the process of aggregate formation. This publication served as the basis for selection of the Tapestry oligonucleotides that are being evaluated for in vivo efficacy.
"The in vitro and preliminary in vivo data that we have gathered are important first steps and are quite encouraging. This second preclinical study will expand our experience with these molecules and provide us additional insight into the potential therapeutic benefit of our proprietary oligonucleotides in patients who are suffering with Huntington's disease," stated Anne L. Bailey, Vice President and General Manager, Genomics.
Data on earlier studies of Tapestry's proprietary oligonucleotides were presented by Dr. Kmiec at an oral session of the 2003 annual scientific meeting of the American Society of Gene Therapy. These studies described the single-stranded oligonucleotides' ability to extend the life of relevant neuronal cells by at least 40 percent in a validated cell culture model.
Huntington's disease is characterized by an expansion of a tract of CAG codons in the gene encoding the huntingtin protein. In contrast to normal, soluble huntingtin protein, mutant huntingtin protein with its expanded polyglutamine tract, aggregates with other proteins in microscopically visible intracellular inclusions. Tapestry's oligonucleotides are designed to block or retard this protein aggregation, reducing the number of inclusions, and consequently may have a potential therapeutic effect on the progression of the disease.
Citation
Parekh-Olmedo H., Wang J., Gusella J.F., Kmiec E.B. (2004) "Modified Single-Stranded Oligonucleotides Inhibit Aggregate Formation and Toxicity Induced by Expanded Polyglutamine." Journal of Molecular Neuroscience, Vol 24, pages 281-291.
About Huntington's Disease
Huntington's disease is a hereditary neurodegenerative disorder that is characterized by aggregate formation and cell death in most areas of the brain. The disease usually occurs in mid-life, and is characterized by involuntary physical movements, severe emotional disturbance and increasing cognitive decline. In the United States, the prevalence of the disease is about 10 cases per 100,000 people -- about 35,000 people in all -- with another 175,000 people genetically at risk.
About Tapestry Pharmaceuticals, Inc.
Tapestry Pharmaceuticals, Inc. is a company focused on the development of proprietary therapies for the treatment of cancer and hereditary disease.
For more information about Tapestry and its technologies, visit Tapestry's web site at http://www.tapestrypharma.com.
The statements in this press release that are not historical facts are forward-looking statements that represent management's beliefs and assumptions as of the date of this press release, based on currently available information. Forward-looking statements can be identified by the use of words such as "believes," "intends," "estimates," "may," "will," "should," "anticipates," "expected" or comparable terminology or by discussions of strategy, and include statements regarding the therapeutic potential for Tapestry's proprietary oligonucleotides, their ability to block or retard protein aggregation associated with Huntington's disease and whether the study being announced will provide sufficient insight into the potential therapeutic benefit of Tapestry's proprietary oligonucleotides in the treatment of Huntington's disease. Such statements involve risks and uncertainties, including whether Tapestry's proprietary oligonucleotides will show sufficient prospect in the treatment of disease to warrant further development, the costs of continuing development of any or all of the Company's development programs and whether the Company will have sufficient resources to pursue and complete development of the molecules, as well as other factors identified under the captions "Risk Factors," "Special Note Regarding Forward Looking Statements" or "Cautionary Note Regarding Forward Looking Statements" in the Company's documents filed from time to time with the SEC, including the Company's Current Report on Form 8-K, as amended, filed February 11, 2004, Annual Report on Form 10-K/A for the year ending December 31, 2003 filed on May 5, 2004 and Quarterly Report on Form 10-Q for the quarter ending June 30, 2004. Should one or more of these risks materialize (or the consequences of such a development worsen), or should the underlying assumptions prove incorrect, actual results could differ materially from those forecasted or expected. The Company disclaims any intention or obligation to update publicly or revise such statements whether as a result of new information, future events or otherwise.
For further information, please contact L. Robert Cohen, Vice President, Investor Relations of Tapestry Pharmaceuticals, Inc. at 212-218-8715.
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October 02, 2004
Getting Closer
It's been a challenge, but the rebuild of HD Blog is almost done. Keep checking back!
Posted by Dave at 03:43 PM | Comments (0) | TrackBack
"Spectacular" Alzheimer's Breakthrough
I can't say if this will ever benefit HD, but the potential is there.
Thanks to Jean Miller for pointing this article out. An excerpt:
The researchers, led by Paivi Liesi, at the University of Helsinki, isolated a combination of amino acids known as tripeptide lysine-aspartic acid isoleucine. When tested in rats, the combination prevented neurotoxins from destroying neurons in the rats' brains.
The therapy could slow or even reverse the effects of Alzheimer's disease, the researchers said. It could be a boon for the millions of people dealing with the condition because there are few medications available for those in the later stages of the disease.
The combination of amino acids isolated by Liesi and her team may hold promise for people with spinal cord injuries. Tests showed it regenerated nerve impulses in laboratory rats with damaged spinal cords. Those rats, whose spinal cords had been severed, regained limited body movement after the drug was administered.
``This is one of the most spectacular results I've seen in the last five years,'' said Huntington Potter, the Byrd center's chief executive officer. ``The promise is very, very great.''
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October 01, 2004
HDAC Website A "Must Visit"
If you haven't been by the HDAC (Huntington's Disease Advocacy Center) website lately, be sure to check it out.
In the last few months they've updated their format and are very actively posting updates on Huntington's Disease. While you're there be sure peak around their website. You'll find many resources that you won't find (easily) anywhere else. I particularly like their "first-person" stories of coping with Huntington's Disease.
"Check it out!"
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