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November 28, 2004
Mithramycin and HD Brain Chemotherapy
Dr. Lavonne Goodman of HDDW sends along this commentary on the Mithramycin study. As is usual, this is a "must-read" for those in the HD community.
Mithramycin and HD Brain Chemotherapy
Drs’Robert Ferrante and Rajiz Ratan, with multiple collaborators, are lead authors of the recent article which details neuroprotective benefits of the drug mithramycin as found in “Chemotherapy for the Brain” (November 17 Journal of Neuroscience). Their elegant long story briefly summarized: Mithramycin extended survival of the HD R6/2 mouse longer than any other agent so far described. Likewise it improved motor performance more than any other agent. And most importantly, brain changes usually present in the R6/2 mouse were so much improved they were “essentially absent”. Though the authors use “comparatively large effect” as their careful language, these combined results are much better than for any other agent so far studied.
And, though mithramycin is not an easy drug to work with, it is already FDA approved. And accordingly the authors make the argument that mithramycin should be among the top priorities for testing in patients.
First: A Heartfelt Thank You
These researchers (and all those laid the groundwork to make this possible) are to be saluted for the long process that went first from scientific creativity, grant writing, the later through the long hours of laboratory work (that put this drug through a multitude of cell studies and more than a hundred HD mice) then through careful interpretation of results then finally to the report of this promising drug.
And this HD activist couldn’t agree more that it certainly makes good sense to promote this “best so far” drug for trials in HD people.
Next: A Heartfelt Ache
But, I’d go further: “Why hasn’t “best so far” gone farther than a thinking stage? The neuroprotective benefit of mithramycin (in combination with another drug gives even better survival numbers) in the R6/2 was known by these (and other HD research leaders) in early 2002. This data was not made public, but instead (presumably) the drug was taken back to the lab where attempts were made to elucidate molecular mechanism. The present article describes the many experiments done to show that it works (somewhere) far downstream in a broad neuroprotective way that remains to be defined.
From the research perspective, this is very good science. From the patient perspective it is downright exciting that researchers have found a drug that have virtually prevented nerve damage in the R6/2 mouse model. Of course a drug this good in the mouse model should be brought to clinical trial. The “heartache” part is that it was not brought to clinical trial closer to 2002. Is there an explanation that I have missed for why this drug is a better clinical trial candidate for HD people in 2005 than it was in 2002?
HD Needs “Sydney Farber” Docs
The Huntington’s community would benefit from docs like Sydney Farber who would do for HD what he did for childhood leukemia a generation ago. Sydney Farber wasn’t very popular with the traditional leukemia researchers of his day when he “bucked the system” and started small clinical trials in children with leukemia. But those same trials moved first successful treatments for childhood lymphocytic leukemia that began the success story of full treatment for this generation.
I bet an HD “Sydney Farber” type of doc would have taken a drug that looked as promising as mithromycin to a pilot clinical trial. But even if we had “Sydney Farber” docs (and I know we do), they don’t have a chance if information is withheld. Smart Docs can’t pick up the research ball and “run with it” if the researchers are doing “private “thinking” and don’t make promising results available to smart docs taking care of HD patients. And if the “Sydney Farber” docs don’t have a chance of picking up the treatment ball, then HD people have lost the game before the buzzer has sounded.
Maybe HD research is too good; maybe the researcher too powerful. It is the researcher, not the clinical docs, making (or not making) decisions on clinical trials for people. So it’s been back to the lab screening for the more perfect drug or homing in on molecular mechanism, etc. Good science for sure. But it is heartache for HD people that our disease has more drugs and fewer clinical trials than any other disease in the history of disease.
Thrilling research; suffering HD people. How good does a drug have to be before the powers (who are these people) that control HSG acts urgently? Though HD research may see HD “time” from the perspective of “large therapeutic window” HD people have a different perspective: The first year’s loss is devastating, the second year’s loss is devastating, and so on for the long, long duration of this illness.
Desperately Seeking . .
If a “Sydney Farber” doc would have had the chance to start closer to 2002, we wouldn’t still be at the stage of “thinking about thinking about” testing mithramycin in people: We would probably have had an answer. And maybe even a first treatment. More heartache: the same thing could be said for several other agents out there.
Posted by Dave at 11:39 PM | Comments (0) | TrackBack
November 27, 2004
NeurotrophinCell Update
Jean Miller has sent along the following on Living Cell Technologies promising treatment for Huntington's Disease. The timeframe seems overly optimistic to me, but the company says that if the current large-primate tests with Neurotrophincell go well they could be entering into Phase-I trials for humans next summer!
The details:
What did the trial involve?
The trial was conducted using a fairly standard rat model of Huntington's (The rats have quinolinic acid injected into the striatum (a part of the brain), which results in the rats displaying pathology and clinical presentation that mimics HD).
The rats were injected with either rat or piglet choroid plexus cell clusters (see attached abstract for details re: Choroid Plexus) encapsulated in alginate into the area immediately prior to inducing the lesions.
What were the results?
A remarkable prevention of the behavioural, pathological and clinical changes - around 80%. The animals recovered extremely well.
Where to from here?
The trials are being repeated in non-human primates first, using piglet choroid plexus - these should be completed by mid February 2005.
This is a necessary step to applying for regulatory approvals in order to begin Phase 1 human trials. How long these approvals take is uncertain. There are strong non-scientific prejudices against the use of animal cells, which may be difficult to overcome. In Australia we hope to hear from the regulatory body in December, as to whether or not they will be able to process an application.
Further research is also required to determine how long the treatment effect will last. The therapeutic cells are very hardy and last a long time (6 months) in artificial conditions (cell culture). The animal experiments that were conducted went for 6 weeks and the restorative effect remained for that time. It now needs to be determined if predictions can be made as to how long the treatment may last.
If the human trials are approved, what will they involve?
At present the trial would involve the injection of a small volume of encapsulated pig choroid plexus cells into the brain striatum of patients who are already experiencing severe symptoms of the disease. A small 'burr hole' would be made in the skull and a needle positioned by stereotactic means. An alternative might be to inject into the ventricles of the brain - this is yet to be determined.
What sort of time-frame are we looking at?
There are two stages. The company will know whether the treatment works or not in the larger (primate) animal model, hopefully by February 2005.
If it works, they will apply to do the human phase 1 study. The company is hopeful that phase 1 could be underway by the middle of 2005 - but this is in the hands of the regulators.
Posted by Dave at 07:42 AM | Comments (0) | TrackBack
London Medical Conference
This might be of interest to some of our European readers - a two-day medical conference on Neurodegenerative disorders to be held February in London. The details:
=====================================================
2nd Annual Neurodegenerative Disorders 2005
Dates: 16th - 17th February
Workshop: 15th February
Venue: Marriott Kensington, London
=====================================================
**** CONFERENCE BROCHURE AVAILABLE NOW - REQUEST YOURS TODAY ****
2nd Annual Neurodegenerative Disorders 2005 will explore the current unmet needs and highlight opportunities in the market by pinpointing potential therapeutic targets. In this conference, pioneering experts will share their concepts and current findings in novel therapeutic targets and treatments, to expose new pathways into drug developments. In addition, experts from pharmaceutical organization will demonstrate methods to maximize your R&D profit by strategic marketing and building cooperative platform to accelerate drug launches.
THEMES WILL INCLUDE:
**Profit from emerging trends in ND therapeutics **New approaches to improve clinical development for your organization **Learn from the best practice case studies such as: Aventis, Wyeth, Eli Lilly and Roche **Examine areas shaping the future of ND therapies: disease modification, proteomic, phamacogenetics and stem cell therapies **Pinpoint where opportunities lie: explore alliances and collaborations **Advancements in medical technologies **Streamline product launches by integrating business strategies in R&D
KEY SPEAKERS
**Dr. Steve Offord, Head, New Products CNS/Thrombosis, Global Marketing & Medical, Aventis **Dr. Menelas Pangalos, Vice President, Neuroscience Research, Wyeth **Dr. Christian Czech, Senior Research Scientist, CNS Research, Pharmaceutical Division, Roche **Dr. Michel Dib, Medical Director for CNS Department, Aventis **Dr. Michael O'Neill, Team & Project Leader, Discovery Neuroscience, Eli Lilly **Dr. Brian Dickie, Director, Research, Motor Neuron Disease Association **Dr. David Burn, Consultant Neurologist & Reader in Movement Disorder Neurology, Newcastle General Hospital **Dr. Ana Martinez, Director, R&D, Neuropharma **Dr. Jette Bisgaard Boll, Department of Molecular Disease Biology, Lundbeck **Dr. Karoly Nikolich, Founder & Chief Scientific Officer, AGY Therapeutics **Dr. Johan Häggblad, Head, Business Development, NeuroNova **Dr. Grant Krafft, Chairman & Chief Scientific Officer, Acumen Pharmaceuticals
------------------
WHO SHOULD ATTEND
------------------
**Director, CNS Clinical Discovery & Human Pharmacology **Director, R&D **Project Leader, Neurology Project Management Group **Vice President, Drug Development **Chief Scientific Officer **Chief Operating Officer **Clinical Pharmacologist **Managing Director **General Counsel **Medical Director, Internal Medicines **Medical Manager
---------------------
DELEGATE REGISTRATION
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Places at this event are strictly limited so BOOK YOUR PLACE NOW.
To make a booking on this event, please contact me via phone or email. Book early to secure a place.
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2 Day conference - ONLY GBP1299 plus VAT
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**** FULL CONFERENCE AGENDA BELOW ****
=======================================================================
PRE-CONFERENCE INTERACTIVE WORKSHOP Tuesday 15th February 2005 Strategies for Pricing and Market Access of New Neurodegenerative Drugs Led by: Ulf Munack, Director, Simon-Kucher & Partners =======================================================================
Workshop schedule
This workshop will give you the opportunity to engage in knowledge-sharing with fellow professionals, which can result in brainstorming and creating ideas which you can use back in your own organisation. You can gain a greater understanding of the pricing of neurodegenerative disorder drugs, have detailed discussions about market access and new ventures.
It is of most use to those who are willing and prepared to engage in meaningful discussions and network with others.
This 'facilitated open-space' provides you with ideas and time to explore possible new tideas and benchmark them against other approaches.
Main topics of discussion:
* P&R environment for NDs products in 2005
* Critical success factors for market access in neurodegenerative disorder
* Examples of recent learning from new ND drug launches
* What it means for R&D
====================================
Day One Wednesday 16th February 2005
====================================
Conference Chair Day One
Dr. Michel Dib
Medical Director, CNS
Aventis
09:00 Registration and coffee
09:30 Opening remarks from the chair
GLOBAL MARKET TRENDS IN NEURODEGENERATIVE DISORDERS
09:40 Focusing on Clinical NDs Drug Development
* What are the methodological issues in NDs clinical R&D?
- Human models, proof of concept & time of intervention
* Biomarkers in ND: from the diagnosis to the therapy
- What we can do? Methods to effectively utilise biomarkers
* What is the development plan for a new drug in the NDs?
* Can we speak about one neurodegenerative process in all
these diseases?
- Lessons from the past, consequences for the future
Dr. Michel Dib
Medical Director, CNS
Aventis
10:20 CASE STUDY: Glutamate Mechanisms - Promising Therapeutic Target?
* Glutamate receptor subtypes: Review of biology &
pharmacology - What have we learnt so far?
* How to use glutamate pathways to search for effective
neuroprotection?
* Examine efficaious results of in vitro neuroprotective studies
* How to modulate glutamate to develop novel therapies?
* Explore NMDA, AMPA and mGlu receptors: Examples of in
vivo neuroprotection
* Evaluate current clinical data of neuroprotective properties
Dr. Michael O'Neill
Team & Project Leader, Discovery Neuroscience Eli Lilly
11:00 Coffee
11:20 CASE STUDY: Novel Insights & Therapeutic Opportunities
* Human CNS disease - A global health concern
* Conceptual & technical approaches towards novel therapies
- Genomics and proteomics: how effective are they?
* Analyse AGY's approaches to CNS R&D development
- Focus on mechanistic pathways: Genomics & proteomics
* Explore novel mechanistic pathway involved in NDs
* Understand the underlying cause of the diseases
* Future outlook of NDs R&D: Pinpoint where opportunities lie
Dr. Karoly Nikolich
Founder & Chief Scientific Officer
AGY Therapeutics
12:00 CASE STUDY: Targeting Stem Cell Therapies
* Focus on the underlying cause of neurodegeneration
* How to develop compounds to target stem cells?
* "Therapeutic Neurogenesis" - analyse the mechanism of action
* Examine clinical evidence of efficacious results
* Explore potential candidates - possible collaboration?
* The future prospects of stem cell therapies' clinical application?
- Discover where opportunities lie within stem cell therapies
- How can the technology be implemented into your R&D?
Dr. Johan Häggblad
Head, Business Development
NeuroNova
12:40 Lunch
14:00 Integrating Business Strategies into NDs R&D
* Methods to develop common mechanism platforms
* Cross-functional team - Enhance communication between
departments i.e. marketing vs scientists
- Feasibility, regulatory risks, unmet needs and market value
* How to shorten time of profit vacuum?
- Create commercial strategies at early stage
- Optimize scientific creativity
* Understanding the value of NDs drugs
Dr. Steve Offord
Head, New Products CNS/Thrombosis, Global Marketing & Medical Aventis
ADVANCES IN THE TREATMENT OF PARKINSON'S DISEASE
14:40 Understanding PD Unmet Needs
* R&D advancements through the years:What have we learnt so far?
* Designing novel PD therapies - Multifactorial considerations
- Motor and non-motor complications
* Examine potential therapeutic targets
* Promising compounds in the pipeline
- Clinical evidence, trial data and safety assessment
* What do we hope for in the future
Dr. David Burn
Consultant Neurologist & Reader in Movement Disorder Neurology Newcastle General Hospital
15:20 Afternoon Tea
15:40 CASE STUDY: Collaborated Approach to Novel Therapy
* Identify current problematic issues faced by physicians
- Transplantation of embryonic nigral tissues
- Poor survival of dopaminergic neurones
* CEP-1347 - mixed-lineage-kinase (MLK) inhibitor: Discover
mechanism of action and efficacious results
* The future of the compound - A collaborated effort
* The pros and cons of collaborated approaches - What to watch
for and ideas to resolve problematic issues
Dr. Jette Bisgaard Boll
Department of Molecular Disease Biology
Lundbeck
16:20 CASE STUDY: Advancements in Medical Technologies
* Symptomatic relief: dyskinetic movements
* Implantation procedures and mechanism of action
* Current success stories in surgical interventions
- Activa and N'Vision Programmer
* Areas of improvement: Where partnerships can be formed
* Positive clinical results of mid-brain stimulation on other
symptoms:Pinpoint areas of combination therapies
Dr. Abed Hammoud
Procedure Solution Manager, Neurological Medtronic
NOVEL THERAPEUTIC TARGETS IN ALZHEIMER'S DISEASE
17:00 CASE STUDY: Anti-ADDL Therapeutics: Reversing Synaptic Degeneration and LTP Deficits
* ADDLs, NOT fibrils, cause AD and MCI
* Evidence: ADDLs attack specific synapses to compromise LTP
* ADDL intervention strategies - treatment and prevention
* Explore anti-ADDL antibody therapeutics - What can you learn?
- Discovery of ADDL assembly blockers and receptor antagonists
* What are the future outlook of Anti-ADDL therapeutics?
Dr. Grant Krafft
Chairman and Chief Scientific Officer
Acumen Pharmaceuticals
17:40 Question and Discussion
18:00 Summation of Day One from Chairman
===================================
Day Two Thursday 17th February 2005
===================================
Conference Chair Day Two
Dr. Menelas Pangalos
Vice President, Neuroscience Research
Wyeth
09:00 Registration and coffee
09:30 Opening remarks from the chair
09:40 CASE STUDY: Challenges in CNS Drug Discovery
* Why is CNS drug discovery so important?
* Anaylse NDs prevalence statistics and unmet needs
* Problematic issues with long-winding drug discovery process
* Wyeth's AD drug discovery programmes
- Explore novel therapeutic targets
- How to expand R&D pipeline?
* Focus on advancement in clinical approaches and trends in
collaboration - What can you implement?
Dr. Menelas Pangalos
Vice President, Neuroscience Research
Wyeth
10:20 CASE STUDY: New Disease Modifying Approaches in AD
* Roche's approach to AD R&D -Disease modification therapies
- Target amyloid and APP to delay disease progression
- What can you implement in your pipeline?
* Analyse current promising compounds in Roche's AD pipeline
- Analyse efficacious clinical results and drug trials
* Focus on cost effective therapies to optimize market needs
- Novel methods to penetrate the market
* Future outlook on AD's clinical studies - identifying global
market trends
Dr. Christian Czech
Senior Research Scientist, CNS Research Pharmaceutical Division Roche
11:00 Morning Coffee
11:20 CASE STUDY: Neuropharma's Neuroprotective Project
* Identify new neuroprotective agents and neuroprotective
activity detection methods
* Systematic search for neuronal antioxidants & calcium
channel antagonists -Explore other new targets & candidates
* Current pipeline analysis: NP031112 non atp-competitive
GSK- 3ß enzyme inhibitor - Positive neuroprotectors
* Neuropharma's future approaches in AD R&D and novel
ideas on neuroprotective strategies
Dr. Ana Martinez
Director, R&D
Neuropharma
EMERGING TRENDS IN MOTOR NEURON DISEASE THERAPIES
12:00 Targeting Clinical R&D of Motor Neurone Disease
* Overview of MNDs and the importance of treatments
* Current clinical drug trials & explore promising compounds
* Forecast of what will cause an impact on MNDs therapies
- Examine mutant genes & protein aggregation in MND
- Implement new therapeutic targets to penetrate MNDs market
* What is HUGO and how will it impact MNDs R&D discoveries?
* Identify current unmet needs & unmet needs in the market
* How to improve overall treatments?
Dr. Brian Dickie
Director, Research
Motor Neuron Disease Association
12:40 Lunch
14:00 CASE STUDY: Explore Emerging Options in Multiple Sclerosis Therapeutics
* Overview - Aetiology, pathophysiology and unmet needs
* The MS therapeutics pipeline - Multiple targeting strategies
* Immunopathological hypothesis - Targeting CNS inflammation
- Examine evidence of promising results
* Discover neuroprotection approaches - Targeting axonal loss
* BTG Ageing & Neuroscience's team focus on MS
- Novel concepts and therapeutic targets
* What is the future of MS therapies? Market trends and ways to
predict answers to global unmet needs
Dr. Russell Hagan
Head, Ageing and Neuroscience
BTG
14:40 CASE STUDY: Methods to Identify Biological Markers in CNS Development
* Calpain inhibitors as "magic bullets" in the treatment
of CNS disorders - Examine evidence of efficacious results
* Discover the activation of calpain in neurodegeneration
- Mechanism of action and therapeutic properties
* Therapeutic approaches using calpain inhibitors in the
treatment of DMD, MS, hearing loss and retinal degeneration
- Methods to utilise calpain inhibitors in each treatment
* Positive result on delaying degeneration of ALS
Dr. Alfred Stracher
Distinguished Professor & Chair / President & CEO State University of New York, CEPTOR Corp
15:20 Afternoon Tea
EXPLORE NEW SCIENCE IN NEURODEGENERATIVE DISEASES
15:40 CASE STUDY: Promising New Drug - Miraxion
* Etiology of Huntington's disease & global unmet needs
* Miraxion - Therapeutic target and mechanism of action
* Positive clinical trial results and recent multicentre studies data
- Near term focus and targeted developmental approach
* Estimated Miraxion global sales -How will it impact the market?
* Opportunities in terms of market space and development?
* What to expect in the coming years? Amarin's future
approach and ideas in Huntington's R&D
Rick Stewart
CEO
Amarin
16:20 CASE STUDY: Examine Possible Role of Chaperones in Protein Aggregation
* Overview of Prion disease: Global prevalence & unmet needs
- What have we learnt about Prion & aberrant proteins?
* Current therapeutic treatments and targets in Prion R&D
* Examine chaperone-facilitator: an RNA and non-nucleic acid
serum component - Mechanism of action and efficacy studies
* Possible therapeutic targets against Prion and other protein
misfolding disease? Current evidence to show promising results
Dr. Abraham Grossman
Founder and Chief Scientific Officer
Q-RNA
17:00 Questions and Discussion
17:20 Summation of Conference from chairman
17:30 Close of Conference
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Posted by Dave at 07:35 AM | Comments (0) | TrackBack
November 26, 2004
Stanford's "HOPES" Updates HD Information
For years, Stanford University has maintained one of the best websites for Huntington's Disease information and now they've added some great pages on promising treatments for HD.
They now have information on:
Trehalose - the sugar that has great potential to be a good, safe HD treatment.
RNAi - What I consider to be the most promising treatment on the horizon with the potential to be the effective treatment/cure we've been waiting for.
TUDCA - An FDA approved medication that has shown amazing results in the lab and could be highly effective in treating Huntington's Disease.
Cystamine - Another FDA-approved medication that might be highly effective in Huntington's Disease.
Posted by Dave at 07:22 AM | Comments (0) | TrackBack
November 25, 2004
Mithramycin - Read This
Thanks to HDLighthouse.org for this wonderful item!
Researchers have found that an already FDA-approved medication shows HUGE promise in treating Huntington's Disease. Dr. LaVonne Goodman's comments:
Mithramycin extended survival of the HD R6/2 mouse longer than any other agent so far described. Likewise it improved motor performance more than any other agent. And most importantly, brain changes usually present in the R6/2 mouse were so much improved they were “essentially absent”. Though the authors use “comparatively large effect” as their careful language, these combined results are much better than for any other agent so far studied.
Read the whole thing on HD Lighthouse's website.
Posted by Dave at 07:13 AM | Comments (0) | TrackBack
November 24, 2004
A Book Worth Buying
I don't believe I've ever recommending a book on this website. First time for everything...
And the book isn't about Huntington's Disease, it's about "Happiness". It's a book with an unlikely title from a (seemingly) unlikely source. The book is called "Happiness is a serious problem" and it is written by radio talk show host - Dennis Prager.
A friend of mine is reading this book now. Prager's items on "the missing tiles" has already changed her life and now she's now buying this book for all her friends.
This is an easy-to-read book which makes simple (and powerful) points. Here's a bit from one review:
Prager looks first at what happiness is, then what prevents it, and finally what produces it. This isn't a fluffy "feel good" read by any stretch. He says that true happiness is the result of sometimes difficult life decisions. Chapters include "Happiness is a Moral Obligation", "Accept Tension", "Develop Self-control", "Happiness is a By-product", "Life is tragic", "Comparing Ourselves with Others", and "Seeing Yourself as a Victim" among others.
Well, now you know why I don't write book reviews! Honestly, I wouldn't make the effort to get this book based what I just wrote. Nor would I based on the bland book cover or the author's occupation. However, I feel fortunate that I did come across this book.
Take a chance and get it. It's cheap, it sells for less than $10 on Amazon and it just might help you "live" your life with a lot more happiness.
Posted by Dave at 06:37 AM | Comments (0) | TrackBack
November 23, 2004
Denial
I don't know how HDAC.org does it, but they keep finding wonderful writers who write wonderful articles about the personal side of Huntington's Disease.
They've done it again with an article by Kelly B. on "Denial". Here's one of the many good points:
...I try to take my symptoms in stride. To go with the HD rather than try to fight it saves so much energy. It does not mean that you give in, but instead that you "accept the things you can not change". And get back to the task of really living each day of your life.
Posted by Dave at 06:26 AM | Comments (0) | TrackBack
November 22, 2004
HD's Link To Other Diseases
I've said for some time that Huntington's Disease research, directly and indirectly, has advanced research for many other diseases. From an article on a recent study:
All of these diseases - Alzheimer's, Parkinson's, ALS, Huntington's - have the same unusual phenomena. Proteins - completely different proteins in each disease - assemble into ordered aggregates, amyloid fibrils, so that a vital organ, usually the brain, is crisscrossed by these structures,” Hall said. “This tells us that the problem has something to do with the general nature of proteins rather than with the specifics of the particular disease-associated proteins.”
Posted by Dave at 06:17 AM | Comments (0) | TrackBack
November 21, 2004
New Center Of Excellence
The University of South Florida is the latest HDSA Center of Excellence. From an article on this announcement:
"This is great news," said neuroscientist Juan Sanchez-Ramos, PhD, MD, who directs the HDSA Center of Excellence at USF. "The Center of Excellence will focus on offering services, comfort and hope to patients and their families while moving towards real solutions to the illness."
USF joins a select group of 21 HDSA Centers of Excellence across the country, including those at Johns Hopkins, Harvard/MGH, the University of Virginia, Emory and Baylor.
"The Huntington's Disease Society of America is committed to identifying and designating 25 HDSA Centers of Excellence by 2006," said Barbara Boyle, HDSA National Executive Director/CEO. "The addition of USF means that our HD families living throughout Florida will no longer have to travel to Atlanta, Georgia, or Birmingham, Alabama, to receive the exceptional quality of care offered by an HDSA Center of Excellence. We look forward to working with the staff at USF to make this an outstanding Center of Excellence, which we will formally dedicate in January 2005." ...
Dr. Sanchez-Ramos, who holds the Helen Ellis Endowed Chair in Neurology, is planning to study whether stem-like adult bone marrow cells administered intravenously may delay the onset of Huntington's disease in mice genetically engineered to have symptoms of the disease.
Posted by Dave at 06:04 AM | Comments (0) | TrackBack
November 20, 2004
Nancy Wexler & Testing
To those in the HD community listen carefully to what I have to say:
Not everybody in our community is like you. We are each individuals who are wired in our own unique ways. How one person handles Huntington's Disease may not work for another. Let me put it to you this way:
There are two types of HD people - those who deal better by gaining knowledge and those who deal better by not focusing on what they cannot change.
Neither path is wrong. I know of people who've been damaged by the knowledge of their HD status and I've known people who were helped. Ironically, some don't handle well the knowledge of being gene negative and others who lived miserably without knowing their status. I've seen the opposite as well.
Respect the decisions of others, even if it isn't the decision you would have made. They are doing what they believe is right for them - and it may be. Even if it isn't the right decision for you.
Why am I on the soap box and what does this have to do with Nancy Wexler? Well...in the wonderful CBS story on Huntington's Disease Nancy said:
"I know that with me, if I were to go to bed every night thinking I'm going to die of Hungtington's, you know, why should I bother getting up?" says Wexler.
And you know what? She (and her amazing family) have accomplished amazing things in the Huntington's Community. She's advanced HD research by years, if not decades. Her work was critical to the Human Genome project. The more I read about what she has accomplished, the more impressed I become with her drive and devotion.
She made the right choice for her! She leads a wonderfully productive and healthy life. How could it have been improved if she had taken the test for HD? I don't believe it would have.
So to those few who criticize her decision...Let her be, she made the right choice for her. Just as everyone in her position tries to do. And for those who feel that she was criticizing others read her sentence carefully. It starts "I know that with me..." She is talking about herself and nobody else. She is not passing judgement on anyone.
It was the right choice for her. That is what matters.
Posted by Dave at 05:25 AM | Comments (0) | TrackBack
November 19, 2004
Great Feature Story On HD By CBS News
This is the best job the national media has ever done on the story of Huntington's Disease.
I don't know how long this link will be available but you can view this Eye On America piece from the CBS Evening News here. A written article is here along with another link to this video.
Main subject of their feature was Jake Dowell, the UW-Madison hockey player & Chicago Blackhawk draftee. (Previous items on Jake Dowell.)
Posted by Dave at 05:22 AM | Comments (1) | TrackBack
The Pharmaceutical Industry
A regular reader here posted some excellent questions in response to the Tapestry article. One's that touch on a common question:
Will 'xyz' company go out of business before their Huntington's Disease treatment becomes available?
Answer: Maybe.
Here's what gets lost in the 'health care debate'...
Most pharmaceutical companies are not raking in the money. Sure, some of the biggest pharmaceutical companies make a pretty nice profit (for now) but there are a slew of young pharmaceutical companies with virtually no income and spending several million dollars a month.
These small companies start with 'seed capital' being put up by investors willing to gamble on a long shot. And it's little more than gambling at this point. The company then takes this seed capital and starts R&D on their chosen targets. From then on, and for the next several years, the young pharmaceutical company is working to show enough promise and results that more investors will provide them funding. If a key element of their research turns out to be a 'dud' then the company will not be able to raise new money and they go bankrupt. Anything of value will be liquidated to pay debtors.
So it's the norm and not the exception for companies like Tapestry & Amarin to have to constantly improvise in order to stay in business. And why would anyone want to invest in such a risky venture? Because of the lure of the big payoff. On average, pharmaceutical companies have to spend $800 million to get a drug successfully on the market.
And this is why the threat of price controls on drugs are so dangerous to our community. If there is no potential for a big payoff/large return then investors will deposit their money in safer places which offer same/better returns on their investment.
As for Tapestry, time will tell. They are now at a point where they have to focus on the research that is most likely to be successful and will generate positive cash flow the soonest. They're now focusing in on their cancer & Huntington's products. That's a good sign. If their research continues to be promising they WILL be able to raise additional funding when they need it.
As for timing, Tapestry's HD product hasn't made it to human testing yet. So, if the product 'works', it would still be 6-9 years before it became available.
Posted by Dave at 04:32 AM | Comments (0) | TrackBack
November 18, 2004
CBS News Featuring Huntington's Disease
I just saw this announcement today:
HDSA Bulletin
Huntington's Disease to be featured on CBS Evening News with Dan Rather tonight
CBS News has completed a story on genetic testing and will be airing part one and two today and tomorrow (November 17th and 18th) at 6:30 p.m. EDT.
Part one will feature experts in pre-implantation genetic diagnosis (PGD) which was reported in HDSA's Marker magazine in 2003.
Part two will focus on Huntington's Disease and includes an interview with Nancy Wexler, Ph.D., President of the Hereditary Disease Society (HDF) about her work to find the elusive gene that causes HD and her experiences of living with HD.
Part two will also feature an at risk hockey player from Wisconsin whose family is affected by HD.
Please consult your local listings for the CBS Evening News with Dan Rather for the time and station on which this two part story will air.
You have just received an HDSA BULLETIN. HDSA BULLETINS are swift way to communicate latest news in various areas that interest the extended HD community. As important news items are gathered, we aim to pass them on quickly to our leaders, committees, chapters, social workers and friends.
Please feel free to forward these bulletins to other interested parties in their unedited form. To add or remove yourself from the HDSA BULLETIN mailing list, send an e-mail to hdsainfo@hdsa.org and make sure that the subject line reads "HDSA BULLETIN." Be sure to include your name, e-mail address, and whether you want to be added, removed, or have a change of e-mail in the body of the message.
Posted by Dave at 08:10 AM | Comments (1) | TrackBack
November 17, 2004
Tapestry Hits Hard Times
Pharmaceutical research is a high-risk business. The only thing that keeps the big money in it is the hope of a big financial payoff. Far too often pharmaceutical companies run out of money before they are able to bring a profitable treatment to market.
Tapestry Pharmaceuticals is the latest to hit hard times. It's winding down its research in gene editing techniques, Sickle Cell, & diagnostics to focus on their HD & cancer products.
Here's what to take away from this announcement...they are going to continue working on their Huntington's Disease treatment. Tapestry is betting the future of the company on their Huntinton's Disease treatment just as Amarin has with Miraxion.
Folks, treatments are (slowly) becoming a reality. Even cash-strapped pharmaceuticals agree!
Oh..their HD product greatly extended the life of cells in early testing.
Tapestry Pharmaceuticals Announces Plan to Discontinue Gene Editing Operations
Wednesday November 17, 12:00 pm ET
Company to Focus on Lead Oncology Compounds
BOULDER, Colo., Nov. 17 - Tapestry Pharmaceuticals, Inc. announced today that it will discontinue development of its gene editing technologies, including programs in Sickle Cell disease and diagnostics. Tapestry will continue development of its Huntington's disease program, but will wind down all other activities of its Genomics Division by January 31, 2005.
Tapestry has engaged Rodman & Renshaw as its financial advisor to assist in the sale or licensing of the assets of its Genomics Division.
"Although we believe our gene editing technology has considerable long- term value, this current restructuring now focuses the majority of our assets on the development of our nearer-term oncology clinical drug candidate portfolio," commented Leonard P. Shaykin, Chairman and CEO. "We anticipate filing Investigational New Drug applications for TPI 287, our third generation taxane, and TPI 273, our novel quassinoid, before the end of this year, and thereafter, we plan to enter into Phase I clinical trials for these compounds."
Tapestry expects annualized savings from this restructuring of about $4 million per year, including personnel, facility and other research and operating costs. As part of the restructuring, Tapestry will close its facility in Newark, Delaware and terminate the employment of about 20 people, or approximately 25% of the Company's current workforce. After the restructuring of the Genomics Division, operations other than the Huntington's program, will be accounted for as discontinued operations.
The assets related to the discontinued operations, available for sale, include contracts, patents and other intellectual property of the Genomics Division as well as research equipment and other physical assets. The assets related to the Huntington's disease program will be maintained, if any.
Neither the timing nor the terms of a sale of any of the assets of the Genomics Division are certain at this time, and the Company can give no assurance that any sale or other transaction will occur. Tapestry, therefore, does not intend to comment further on the status of the sales effort prior to signing and announcing any definitive sales or licensing agreement for any or all of these assets.
Tapestry Oncology Drug Development Programs
* TPI 287: Third generation taxane. TPI 287, a proprietary taxane, is
designed to overcome acquired resistance to taxane-based therapies by
circumventing MDR-1, a pathway associated with taxane-resistance.
Preclinical studies have demonstrated that this compound is
substantially more potent than paclitaxel in paclitaxel-resistant
tumors and is as potent, or more potent, than paclitaxel in
treatment-naive tumors.
* TPI 273: Proprietary quassinoid. TPI 273, a proprietary quassinoid,
is a semi-synthetic analog of a naturally occurring molecule that
induces cancer cell death in a variety of tumors including those that
have elevated myc protein.
About Tapestry Pharmaceuticals, Inc.
Tapestry Pharmaceuticals, Inc. is a company focused on the development of proprietary therapies for the treatment of cancer and Huntington's disease.
Forward Looking Statements
The statements in this press release that are not historical facts are forward-looking statements that represent management's assumptions and beliefs as of the date of this 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 as to the long-term value of the Company's gene editing technology, the prospects for sale of the assets of the genomics division, the filing of INDs on two of the Company's cancer compounds and their entry into clinical trials, the expected savings from the restructuring and the amount of restructuring charges and their breakdown. Such statements involves risks and uncertainties including whether others view our gene editing technology as having value given its early stage of development and other factors; that the assets of the genomics division have sufficient perceived worth to attract potential buyers; whether the results of pre-clinical research of the Company's cancer compounds demonstrate sufficient efficacy and do not demonstrate safety concerns; that the FDA does not require the submission of additional information before permitting Phase I clinical trials to commence; the incurrence of costs in addition to those projected for the termination of employees, exiting facilities and disposing of assets; that additional costs incurred after the restructuring result in less savings; 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/A, dated February 11, 2004, Annual Report on Form 10-K/A for the year ended December 31, 2003 filed with the Securities and Exchange Commission on May 5, 2004, and Quarterly Report on Form 10-Q for the quarter ended September 29, 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 or additional 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.
Posted by Dave at 11:19 PM | Comments (1) | TrackBack
November 16, 2004
Fall Amaryllis Sale
HDSA is selling their Amaryllis gift boxes again. As they say...money goes for a good cause!
https://www.nyic.com/hdsa/fund/amaryllis.html
Posted by Dave at 11:15 PM | Comments (0) | TrackBack
November 15, 2004
A Sad Story
This happens to people without HD, but this one stings...
A woman with Huntington's Disease, with symptoms and down to only 90 pounds, leaves a bar at 1:30am on an evening and never returns home. The paper is careful on how they write-up the story but it sounds like she passed out and didn't survive the 20-degree temperatures that night.
She left behind a family that included two children and a husband.
Posted by Dave at 07:46 PM | Comments (0) | TrackBack
November 14, 2004
California's Prop 71 which successfully passed was supposedly setup to fund 'embryonic' stem cell research to the tune of $3 billion over the next 10 years. The proposition even used Huntington's Disease, among other diseases, as benefiting from research.
What has been missed in the resulting debate is that the proposition allows money to be spent on 'adult' stem cells as well as 'embryonic' stem cells. This is important as adult stem cells have a chance to help HD patients, something that is in real question with the embryonic stem cells.
Let's hope political correctness doesn't keep money from being spent on wonderful research results such as this report on successes in adult stem cell research:
In other recent work, scientists at Johns Hopkins Medical Institutes reported last month that they isolated heart stem cells from adult humans and other animals and were able to grow them in batches in a lab dish. The goal is to use them to replenish the damaged heart.
And Dr. Steven Goldman and his colleagues at University of Rochester Medical Center have made a discovery that he said could lead to a clinical trial in Huntington's patients within two years. There are no available treatments.
Like all stem cell scientists looking to treat brain disease, Goldman has aimed at reshaping the adult brain's store of stem cells. Less than a decade ago, scientists discovered that the adult brain has immature stem cells in two areas -- the ventricular zone and the olfactory system.
Goldman says these stem cells normally become support cells, or glia, that help neurons carry out their job. Scientists have characterized glia as the workhorses of the brain, and neurons as the generals. But his lab is taking the glia cells from the olfactory bulb of mice and turning them into neurons capable of restoring the damaged brain.
Researchers used a gene to pump brain-derived growth hormone -- considered important for stem cell growth -- into one of the stem cell zones. They found that when the stem cells of this region were bathed in a growth hormone called brain-derived neurotrophic factor, some of the glial cells developed into the kind of neurons that are lost in Parkinson's and Huntington's.
They then used another gene to block a protein that tells stem cells to mature into glia, and showed they could shut down production of glial cells and ramp up production of neurons in numbers that Goldman said could have clinical impact. The findings were presented last month at the Society for Neuroscience meeting in San Diego.
Mice in the experiment had a genetic mutation that causes Huntington's disease. Normally they would become very sick at 10 weeks, and those in the lab didn't get sick until they were 4 months old and lived almost twice as long as expected.
"The haze is really coming off the field," Goldman said. "We are beginning to understand where stem cells would work, and what the clinical opportunities will be."
Here is an important passage from California's Prop 17:
- Maximize the use of research funds by giving priority to stem cell research that has the greatest potential for therapies and cures...
In my book that includes the above study on adult stem cells for Huntington's disease. I hope the California readers of this blog will make sure that California does help fund further research in this area. They'll be needing the money when it comes time for clinical trials in a couple of years.
Posted by Dave at 06:33 PM | Comments (0) | TrackBack
November 13, 2004
More On Charged Husband...
A PERTH man has been charged over the suspicious death of his wife, after he allegedly gave her an overdose of medication.
Police will argue that the man, 58, crushed up his partner's prescription medication and administered the fatal dose at their home in suburban Carlisle.
He then called an ambulance.
He was charged with committing an act intending to cause grievous bodily harm. ...
However, Sergeant Mike Gough said police were not treating it as assisted suicide.
"For it to be a euthanasia case there must be some evidence that she was involved and there is none at this stage," he said.
Posted by Dave at 06:24 PM | Comments (0) | TrackBack
November 12, 2004
Australian Man Accused Of Killing Wife With HD
A MAN has appeared in a Perth court accused of giving his terminally-ill partner a lethal dose of her prescription medication.
Terence Turton, 58, was charged with committing an act intending to cause grievous bodily harm after the body of his 44-year-old partner was found at the couple's home overnight.
...
Outside court, police said the man's partner had been suffering from Huntington's disease.
Posted by Dave at 06:20 PM | Comments (0) | TrackBack
November 11, 2004
DWMI - Driving While Medically Impaired
Thanks to Gerri on the Hunt-Dis email list for pointing out this article.
The NTSB is right. More should be done to help keep the medically impaired off the road. I know of too many stories where it took multiple accidents before a person with HD lost their driving privileges. I'm all for keeping people as independent as long as possible. However, I draw the line when others are put in danger - such as when a is child crossing a street.
The National Transportation Safety Board recommended that states adopt laws to guarantee legal immunity for doctors, family members and others who report dangerous drivers to state motor vehicle officials. Twenty states and the District of Columbia have no such laws.
Six states - Nevada, California, Delaware, New Jersey, Oregon and Pennsylvania - require doctors to report drivers with high-risk conditions.
Board members said police need to be trained to spot drivers with medical problems and doctors should be taught to talk to patients about whether they should be driving. The board also said federal agencies need to coordinate transportation possibilities for people who can no longer drive.
Posted by Dave at 05:33 AM | Comments (2) | TrackBack
HD Fundraiser In New Jersey
Research is Joan Hunters' only ray of hope.
She hopes Sunday's fund-raiser serves two purposes -- make people more aware of the disease and raise money for a cure. The family intends to make this an annual event.
Nicole Valente, a Sebastiano's employee and family friend, said she knows what's like to lose a child. She lost three of her four children when fire tore through their Alpha home in March.
Through her ordeal, Valente said the Hunter family did a lot to help her. Wanting to do the same for them, she helped organize the benefit.
"I wanted to give something back," Valente said. "They are so close to my heart."
Valente hopes other community members will follow the lead and give what they can.
"You're talking about somebody's whole family that is affected," she said. "We can't just sit back and do nothing while people die. We need to band together as a community."
Posted by Dave at 05:26 AM | Comments (0) | TrackBack
November 10, 2004
RNAi Takes Another Step Forward
They've now been able to lower cholesterol in animal tests with RNAi. Some tidbits from the latest article:
It ultimately could lead to a drug-like treatment for hereditary disorders such as Huntington's disease by injecting tailored snippets of RNA, DNA'S molecular cousin, to silence a specific gene. The approach also has promise for treating a range of diseases, including heart disease and cancer, that have genetic factors, specialists said. ...
The new study, published Thursday in the journal Nature, reports successful delivery of RNA pieces through the blood by chemically stabilizing them and attaching them to cholesterol molecules to help ease their entry into target cells. ...
RNA interference "has major potential for discovery and development of new medicines," said Nobel laureate Philip Sharp, a molecular biologist at the Massachusetts Institute of Technology and a co-founder of Alnylam. "It is the type of discovery that really only happens every decade or so."
Posted by Dave at 09:02 PM | Comments (0) | TrackBack
My Time Flies
The break was totally unplanned but welcome. More posts to come.
Posted by Dave at 06:46 PM | Comments (0) | TrackBack
November 09, 2004
Repligen, Uridine
Repligen mentions Huntington's and one of their drug candidates in this quarterly announcement. One question...when are they going to get serious about testing it for Huntington's Disease? The press release:
Repligen Reports Second Quarter 2005 Financial Results
Tuesday November 9, 4:10 pm ET
Conference Call on Thursday November 11th at 11:00 A.M. EST
WALTHAM, Mass., Nov. 9 -- Repligen Corporation (Nasdaq: RGEN - News) today reported results for the second quarter of fiscal year 2005 ended September 30, 2004. Total revenue for the quarter was $1,296,000 compared to total revenue of $1,419,000 for the second quarter of fiscal year 2004, a decrease of $123,000 or 9%. Gross profit for the second quarter of fiscal year 2005 was $593,000 (46%) compared to $678,000 (48%) for the second quarter of fiscal year 2004.
Operating expenses for the second quarter of fiscal year 2005 were $2,413,000 compared to $2,912,000 for the second quarter of fiscal year 2004. The net loss for the second quarter of fiscal year 2005 was $1,719,000 or $.06 per share, compared to $2,140,000 or $.07 per share for the second quarter of fiscal year 2004. Cash and investments as of September 30, 2004 were $24,004,000.
For the six-month period ended September 30, 2004 total revenue was $4,106,000 compared to $3,480,000 for the same period in fiscal 2004, an increase of $626,000 or 18%. Gross profit for the six-month period was $2,270,000 (55%) compared to $1,884,000 (54%) for the same period in fiscal year 2004. Operating expenses for the six-month period of fiscal year 2005 were $4,832,000 compared to $6,244,000 for the same period in fiscal year 2004. The net loss for the six-month period of fiscal year 2005 ended September 30, 2004 was $2,364,000 or $.08 per share, compared to $4,168,000 or $.14 per share for the same period of fiscal year 2004.
"Product sales for the first half of the year showed solid growth as demand increased for our Protein A products used in the production of monoclonal antibodies, consistent with the growth of the monoclonal antibody market," stated Walter C. Herlihy, President and CEO of Repligen. "We intend to continue to leverage the profits from our product sales, our intellectual property assets and internal capabilities to develop our emerging CNS pipeline."
Update on Product Development Programs
Secretin
* We have completed patient enrollment in a multi-dose, placebo-
controlled, Phase 2a clinical trial of secretin in schizophrenia. This
trial is designed to assess the impact of twice weekly dosing of two
different dose levels of secretin over two weeks to improve the
symptoms of schizophrenia when compared to placebo. We expect to
complete this trial by the end of the year.
* We initiated a Phase 1, open-label clinical trial of secretin in
Obsessive-Compulsive Disorder (OCD) in September. This open-label,
dose escalation study will enroll up to sixteen patients and will
evaluate the safety, tolerability and preliminary evidence of efficacy
of secretin on the symptoms of OCD including obsessive thoughts and
compulsive behaviors.
Uridine
We reported positive activity of uridine in validated animal models of anxiety at the annual meeting of the Society for Neuroscience in October. Published literature indicates that uridine is also active in animal models of neurodegenerative diseases including Parkinson's and Huntington's disease. We plan to submit an IND for a Phase 2a clinical trial of uridine in patients with bipolar disorder in 2005.
Intellectual Property
CTLA4-Ig
Bristol-Myers Squibb Corporation presented the results of two positive
Phase 3 clinical trials of its form of CTLA4-Ig at the annual meeting of
the American College of Rheumatology in October. Bristol-Myers Squibb
has previously stated that it intends to file a New Drug Application for
CTLA4-Ig by the end of the year. We own the rights to a United States
patent for the use of CTLA4-Ig for the treatment of rheumatoid arthritis,
multiple sclerosis and lupus and a patent in Europe for the use of CTLA4-
Ig for the treatment of autoimmune disease including rheumatoid
arthritis, as well as organ transplant. These patents will remain in
force until 2021 and 2013 respectively.
Bioprocessing Technology
As previously disclosed, Repligen and MIT filed suit against ImClone on
May 4th alleging that ImClone has infringed one of our U.S. patents in
its production of Erbitux(R), also known as C225. In August, the judge
set the preliminary schedule for the suit, and we currently anticipate
that exchange of documents and deposition of fact witnesses will be
completed by the middle of 2005.
Quarterly Conference Call
Repligen's President and Chief Executive Officer, Walter C. Herlihy, Ph.D., will host a conference call and webcast on Thursday November 11th at 11 a.m. EST, to provide a quarterly update of the Company's clinical development programs and Specialty Pharmaceuticals business. This call can be accessed via Repligen's website at http://www.repligen.com or you may listen to the live broadcast by calling (800) 299-7635 for domestic calls and (617) 786-2901 for international calls and entering the passcode: 90559203.
About Repligen Corporation
Repligen Corporation is a biopharmaceutical company committed to being the leader in the development of novel therapeutics for profound neuropsychiatric disorders and autoimmune disease with particular emphasis on applications for children. Repligen has a Specialty Pharmaceuticals business comprised of rProtein A(TM) and SecreFlo(TM), the profits from which will be used to partially support the development of our proprietary products. Repligen's corporate headquarters are located at 41 Seyon Street, Building #1, Suite 100, Waltham, MA 02453. Additional information may be obtained from http://www.repligen.com.
SELECTED FINANCIAL DATA
Operating Statement Data:
Three-months ended Six-months ended
September 30, September 30,
2004 2003 2004 2003
Revenue:
Product revenue $ 1,296,000 $ 1,383,000 $4,106,000 $3,426,000
Research revenue -- 36,000 -- 54,000
Total revenue 1,296,000 1,419,000 4,106,000 3,480,000
Cost of revenue 703,000 741,000 1,836,000 1,596,000
Gross profit 593,000 678,000 2,270,000 1,884,000
Operating expenses:
Research and
development 1,274,000 1,901,000 2,663,000 3,330,000
Selling, general
and administrative 1,139,000 1,011,000 2,169,000 2,914,000
Total operating
expenses 2,413,000 2,912,000 4,832,000 6,244,000
Loss from operations (1,820,000) (2,234,000) (2,562,000) (4,360,000)
Investment income 101,000 94,000 198,000 192,000
Net loss $(1,719,000) $(2,140,000)$(2,364,000)$(4,168,000)
Basic and diluted
net loss per share $(.06) $(.07) $(.08) $(.14)
Basic and diluted
weighted average
shares outstanding 30,058,000 28,860,000 30,051,000 29,423,000
Balance Sheet Data:
September 30, March 31,
2004 2004
Cash and investments $24,004,000 $24,863,000
Total assets 27,364,000 29,615,000
Stockholders' equity 24,878,000 27,164,000
This press release contains forward-looking statements which are made pursuant to the safe harbor provisions of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. The forward-looking statements in this release do not constitute guarantees of future performance. Investors are cautioned that statements in this press release which are not strictly historical statements, including, without limitation, statements regarding current or future financial performance and position, management's strategy, plans and objectives for future operations, plans and objectives for product development, plans and objectives for present and future clinical trials and results of such trials, plans and objectives for regulatory approval, litigation, intellectual property, product development, manufacturing plans and performance such as the anticipated growth in the monoclonal antibody market and our other target markets and projected growth in product sales, constitute forward-looking statements. Such forward-looking statements are subject to a number of risks and uncertainties that could cause actual results to differ materially from those anticipated, including, without limitation, risks associated with: the success of current and future collaborative relationships, the market acceptance of our products, our ability to compete with larger, better financed pharmaceutical and biotechnology companies, new approaches to the treatment of our targeted diseases, our expectation of incurring continued losses, our uncertainty of product revenues and profits, our ability to generate future revenues, our ability to raise additional capital to continue our drug development programs, the success of our clinical trials, our ability to develop and commercialize products, our ability to obtain required regulatory approvals, our compliance with all Food and Drug Administration regulations, our ability to obtain, maintain and protect intellectual property rights for our products, the risk of litigation regarding our intellectual property rights, our limited sales and manufacturing capabilities, our dependence on third-party manufacturers and value added resellers, our ability to hire and retain skilled personnel, our volatile stock price, and other risks detailed in Repligen's filings with the Securities and Exchange Commission. Repligen assumes no obligation to update any forward-looking information contained in this press release or with respect to the announcements described herein.
CONTACT:
Walter C. Herlihy, Ph.D.
President and Chief Executive Officer
(781) 250-0111, ext. 2000
Laura Whitehouse
Vice President, Market Development
(781) 250-0111, ext. 2306
Posted by Dave at 08:44 PM | Comments (0) | TrackBack
November 08, 2004
Eyesight & Huntington's Disease
Yes, Huntington's Disease can adversely affect vision. Researchers are finding the proof:
Fine-structural analysis and connexin expression in the retina of a transgenic model of Huntington's disease.
J Comp Neurol. 2004 Nov 8;479(2):181-97.
PMID: 15452853
Petrasch-Parwez E, Habbes HW, Weickert S, Lobbecke-Schumacher M, Striedinger K, Wieczorek S, Dermietzel R, Epplen JT. -Department of Neuroanatomy and Molecular Brain Research, Ruhr-University Bochum, D-44780 Bochum, Germany.
Elisabeth.Petrasch-Parwez@rub.de
Recent studies indicate that the visual system appears more frequently affected in polyglutamine diseases than expected previously. Here, we investigated retinal degenerations in adult transgenic R6/2 mice, a model for Huntington's disease (HD). Light microscopical analysis revealed retinal dystrophy all over the retina, with central areas showing major effects.
Electron microscopical analysis showed strong degenerations of outer and inner photoreceptor segments, shrinkage of photoreceptor cell somata, and signs of degeneration in photoreceptor terminals in the outer plexiform layer. Terminal deoxynucleotidyl transferase-mediated dUTP nick end labeling showed hints of apoptosis. Mutant huntingtin and ubiquitin were expressed in all classes of retinal neurons, the pigment epithelium, and to a minor extent in neuropil structures.
For investigating possible links to functional impairments in the rod-cone pathway, expression levels of three connexins (Cx) were compared in R6/2 and wildtype mice retinae. In R6/2 mice, expression of Cx36, the major neuronal connexin in the retina, was slightly reduced in the outer plexiform layer, indicating affected photoreceptor terminals as detected at the electron microscopical level. In contrast, Cx45, a putative neuronal connexin in the retina, was remarkably reduced in the inner plexiform layer of R6/2 mice.
This result corresponded to fainter signals of Cx45 mRNA as documented by in situ hybridization and to a lower level of mCx45 cDNA as obtained by polymerase chain reaction after reverse transcription, suggesting functional deficits in spatial processing of Cx45-mediated gap junction coupling due to transgene-induced retinal degenerations. Thus, it is important to clarify the meaning of visual involvement in HD.
2004 Wiley-Liss, Inc.
Posted by Dave at 08:35 PM | Comments (0) | TrackBack
November 07, 2004
Celastrol
Check Out HD Lighthouse's article on celastrols. An excerpt:
"Heat shock protein 70 has been found to arrest neurodegeneration in a fruitfly model. Heat shock proteins got their name because they are produced when cells are stressed by heat or toxins; however, they have a maintenance role in cells as well, helping newly made proteins to fold properly (their chaperone function) and carrying old ones off for degradation.
Celastrol was found to induce heat shock protein 70 as well as 40 and 27 in a variety of cell types, including neurons. It also was found to be cytoprotective, inhibiting apoptosis (cell death) under conditions of severe stress. Because of these exciting results, research with the Bates HD mice will be starting soon. This is clearly a compound to watch."
Posted by Dave at 08:22 PM | Comments (0) | TrackBack
November 06, 2004
Autophagy
Researchers learning more every day:
A cell undergoing autophagy assembles tiny capsules called vesicles that surround and chew up parts of the cellular machinery from within. Autophagic vesicles have been seen in cells undergoing programmed cell death, but the evidence is not clear yet whether they're trying to protect the cell from apoptosis, or hastening its demise.
"Autophagy is the only way to get rid of damaged parts of the cell without trashing the whole thing. So in a nerve cell, for example, you'd want autophagy to correct problems without destroying the cell."
High levels of autophagic vesicles also have been noted in some forms of degenerative muscle disease, and in degenerative nervous system diseases like Huntington's, Parkinson's, Alzheimer's and ALS, (Lou Gehrig's disease). But it's not clear why the vesicles are accumulating. They may be building up because they aren't being used, or it may be that the distressed cells are producing more vesicles.
Posted by Dave at 08:16 PM | Comments (0) | TrackBack
November 05, 2004
Cannabinoids
There are so many "cautions" on this article, but I'll leave that for another time. Here's a piece on cannabinoids as a potential treatment for a variety of disease.
Posted by Dave at 08:07 PM | Comments (0) | TrackBack
November 04, 2004
Genetic Tests Under Pressure?
1-in-15 doesn't sounds too bad on the surface, but this article brings up some good points on family pressures & genetic testing.
Posted by Dave at 07:53 PM | Comments (0) | TrackBack
November 03, 2004
Thank You
Some may find this surprising but I'll go months without checking my my "traffic" - how many people visit this website.
I took a look tonight to see how much of a bump this website got from Command-Post.org. It wasn't much of a bump, but...
I was shocked at how much traffic continues to steadily grow. October was the busiest month ever.
Thank you for your support!
Posted by Dave at 09:34 PM | Comments (0) | TrackBack
HD Research Benefits Alzheimers & Parkinson's
Huntington's Disease research involves many fundamental processes that affect other diseases. The latest:
Research leads to one cause of Alzheimer's-like diseases
A team of scientists led by biology Prof. Richard Morimoto, former dean of Northwestern's Graduate School, recently made a key discovery in understanding the molecular processes of diseases such as Alzheimer's, Parkinson's and Huntington's. Such neurodegenerative diseases break down the body's nervous tissue.
While studying toxic proteins involved in Huntington's disease, the team discovered that a disease-causing protein partially inhibits proteasome, which normally eliminates damaged proteins from cells. These harmful proteins allow damaged ones to reside in the cell, where they can form toxic clumps that destroy cell function.
Posted by Dave at 09:25 PM | Comments (0) | TrackBack
November 02, 2004
Fast-Growing Market for RNAi
A press-release from a market research firm:
Fast-Growing Market for RNAi Products Shows No Signs of Slowing
ARLINGTON, Va., - The promise of RNA interference (RNAi) therapeutics to combat human illnesses such as Huntington's disease, Lou Gehrig's disease and hepatitis C has vastly increased scientific interest in the field. In fact, according to a recent survey, 65% of RNAi researchers only began using these techniques in the past 12 months. Increased activity by these current practitioners as well as an influx of new users will fuel rapid growth in the market for RNAi research products over the next year.
These findings were recently published by BioInformatics, LLC (http://www.gene2drug.com) in the report, "The Market for RNA Interference Products: Challenges and Opportunities," which is designed to help life science suppliers understand and respond to the needs of scientists using RNAi technology. Based on a 35-question survey of over 500 scientists who currently use small interfering RNA (siRNA), the report details RNAi experimental design with a special focus on customers' experiences with pre- designed, validated or library siRNA duplexes, chemically synthesized custom siRNAs and transfection reagents. Additionally, another 360 scientists who plan to use siRNA within the next year help shed light on the future direction of the market.
"By better understanding the preferences, needs and expectations of researchers, RNAi product suppliers can make refinements to existing tools as well as design new tools that support this technology," said Bill Kelly, President of BioInformatics, LLC.
This fast-growing market offers numerous opportunities for life science suppliers and has engendered interesting dynamics among competitors. According to the scientists surveyed, Ambion, Dharmacon (a subsidiary of Fisher Scientific International), Invitrogen (Nasdaq: IVGN) and Qiagen (Nasdaq: QGENF) dominate the market for RNAi products, however, their positions vary across market segments and geographic regions. For instance, while Ambion, Invitrogen and Qiagen are used equally in academia and industry, Dharmacon was cited as a supplier far more frequently by industrial researchers than by academic researchers. Results also indicate that Invitrogen has a consistent share of the market worldwide, while Ambion and Dharmacon are more frequently used in North America than other regions and Qiagen has a stronger market presence in Europe.
Market leaders also vary by specific product categories with some competitors specializing in one type of product, while others compete by offering a full line of RNAi products. For example, Dharmacon is the leading supplier of both pre-designed, validated or library siRNA duplexes as well as chemically synthesized custom siRNA duplexes, but the company has not established itself as a supplier of transfection reagents. On the other hand, Invitrogen is the top supplier of transfection reagents and has also become a major supplier of siRNA products following last year's acquisition of Sequitur and the Stealth(TM) RNAi technology.
To understand the factors that influence scientists to choose one supplier over another, the report analyzes the relationship between the importance, expectation and performance of various supplier attributes. Several attributes such as quality control and technical service/support for scientific questions were found to be important to scientists, however, suppliers aren't meeting their customers' expectations -- indicating key areas where suppliers need to focus. The report also evaluates the performance of leading suppliers with regards to less tangible, but critically important, elements of customer value such as customer loyalty and retention -- finding that some companies stand out above, and others below, the overall market average. "Our use of attitudinal and behavioral measurements in this survey provides management with a powerful tool to segment customers by their degree of loyalty, evaluate the effectiveness of initiatives to meet customer expectations and predict future corporate performance," said Kelly.
For a complimentary Executive Summary of this report, please visit: http://gene2drug.com/report/81/
ABOUT BIOINFORMATICS, LLC
BioInformatics, LLC is a market research firm located in Arlington, Virginia. BioInformatics supports marketing, sales and R&D executives in the life science, medical device and pharmaceutical industries through published research reports, custom research and consulting. BioInformatics sponsors the world's largest market research panel of scientific customers -- The Science Advisory Board (http://www.scienceboard.net) -- which consists of more than 22,000 scientists, physicians and other life science and medical professionals from 62 countries who participate in surveys that address emerging technologies, test customer reactions to new product concepts, measure brand awareness and assess advertising effectiveness.
For more information, please contact:
Alyssa Martin BioInformatics, LLC
2111 Wilson Blvd., Suite 250
Arlington, VA 22201
703.778.3080 x12 (phone) 703.778.3081 (fax)
a.martin@gene2drug.com
Posted by Dave at 09:12 PM | Comments (0) | TrackBack
November 01, 2004
Welcome Command-Post Visitors
If you've clicked through to here from the Command-Post website you may have noticed we are not a current affairs/political blog. The HD Blog is one of the first blogs devoted to a disease - Huntington's Disease (HD).
HD is a (currently) incurable disease that generally strikes after the age of 35. It's been described as a combination of Alzheimers, Parkinson's, & Manic Depression all rolled into one disease. It's a hereditary disease where offspring have a 50% chance of getting the disease.
HD was one of the first diseases to have its root genetic cause found. The gene was identified in 1993. Data from HD research was used in the Human Genome project (identified markers).
Today there is no cure for the always-fatal Huntington's Disease and too little money put into support and research. Despite that, treatments and potential cures are now finding real success in the lab and its believed that they will reach patients in the next 6-10 years. It appears that HD may be one of the first diseases to be successfully treated through RNAi research.
If you wish to help please take the time to learn more about the disease. Urge your politicians to support the ban on genetic discrimination which hits our community hard. Too many of our community members must hide this disease in order to maintain their jobs and insurance.
Thank you for visiting and please come back!
Posted by Dave at 11:07 PM | Comments (0) | TrackBack