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Until medicine proves otherwise, technology IS the cure…
That’s the mantra of the ALS Residence Initiative, a group spearheading the construction of a series of permanent residences specifically designed for individuals living with the fatal neurodegenerative disease, amyotrophic lateral sclerosis. The first such residence officially opened in Chelsea on August 13th, and it comes equipped with a technological payload that would make a nuclear submarine commander proud.
Each resident has a computer control panel mounted on his or her wheelchair. Controlling the computer mouse using whatever muscular movement they still possess (their head, eyes, or fingers,) the resident can issue commands that are beamed via infrared transmitters to a series of receivers scattered throughout the center. The commands are then bounced to a master computer that opens doors, turns lights on and off, draws the blinds, takes room service orders, and even operates toilets. The end result is a smart house that provides once unimaginable levels of independence to individuals suffering from neurodegenerative disease.
The center came to life through the work of Barry Berman, CEO of the Chelsea Jewish Foundation, and Steve Saling, a 41-year old former landscape architect who was diagnosed with ALS four years ago. The Chelsea Jewish Foundation operates the Leonard Florence Center for Living, a 100-bed nursing home in Chelsea. In addition to the ALS residence named after Saling, the Leonard Florence Center has also built a residence designed for multiple sclerosis patients.
Asked what he would do if he lost the ability to move his computer mouse using his eyes and head, Saling pointed to technology currently in development that would allow ALS patients to control computers with their brain waves. That technology would be the subject of the keynote speech at the 4th Annual ALS Roundtable that followed the grand opening of the residence.
Prize4Life partnered with the Massachusetts Chapter of the ALS Association to launch the Roundtables. Recognizing that there are many organizations across Massachusetts working to cure ALS, Prize4Life co-led the charge to bring these groups together so that they could share information and best practices and identify opportunities to work together towards a common cause. We are enormously proud of what the Roundtables have yielded, including the opportunity to learn from Dr. Leigh Hochberg the latest developments in the brain wave technology known as ‘BrainGate.’
BrainGate aspires to literally turn thoughts into action. A chip is placed in the brain and records signals that correspond to imagined limb movement. Decoder software and hardware then translate these signals into usable commands for an external device. In Saling’s case, the external device would be a computer mouse that would allow him to ‘think’ the cursor across the screen. But BrainGate’s imagination reaches much farther than that. Scientists hope that the technology can one day be used to control prosthetic limbs, or even coupled with electronic stimulation so that the mind can bypass damaged nerves and move once-paralyzed muscles.
But for the time being, ALS patients like Steve Saling must be content with the simpler joy of turning a light off when they are ready to sleep and merely dream of much more complex joys like walking and talking. Steve’s son, Finn, was born just one month before Steve was diagnosed. When interviewed for an article in the Boston Globe, Steve described his role as a father. “I’m right now watching my brother canoe to shore with Finn in his lap. I wish that could be me.”
While the Leonard Florence Center for Living Steve Saling Residence in Chelsea is a quantum leap forward in technology, it is, sadly, not a cure. It provides independence to be sure, but not the freedom to hold one’s child in one’s arms. To achieve that, we have no alternative but to bring together the minds, money, and breakthroughs needed to eradicate ALS.
That’s where Prize4Life comes in. Prizes have the ability to attract new ideas from new sources. And when those prizes are highly focused on results, are ours are, they can drive breakthroughs that will accelerate progress toward a cure. Awarding prizes in the biomedical field is a new, relatively unproven model. But the old ways simply haven’t gotten the job done.
Learn more about our prizes here.
For more information on the Leonard Florence Center for Living, you can visit www.leonardflorencecenter.org.
For more information on BrainGate, visit www.braingate2.org.
Tuesday, September 7, 2010
Monday, August 2, 2010
Drug Development for Neurodegenerative Diseases Part 4
This next installment of blog coverage from the Drug Development for Neurodegenerative Diseases conference organized by marcus evans focuses on the much buzzed about topic of regeneration.
Neurodegenerative diseases such as Alzheimers Disease, Parkinson’s Disease, Huntington’s Disease and Amyotrophic Lateral Sclerosis (ALS) are characterized by a significant and increasing loss of neurons. In the case of ALS, loss of the neurons that control voluntary muscle movement (motor neurons) underlies the progressively debilitating symptoms experienced by ALS patients. Even before patients start to notice problems with their motor function, changes have already begun to occur. It is estimated that 30-50% of a patient’s motor neurons have already degenerated by the time an ALS diagnosis is made.
As neurons begin to die, the remaining motor neurons can partially compensate for this loss (by increasing the strength and number of connections to the target muscle), but eventually the tipping point comes when too few neurons remain to properly control the muscle and clinical symptoms begin to manifest.
What can we do about this? Ah, regenerative medicine!
Speaker Judith Kelleher-Anderson, President and Chief Scientific Officer at Neuronascent, Inc., described to the audience that much of the focus in regenerative medicine has been placed on invasive therapies involving transplantation of healthy cells into the diseased area. These studies have led to a handful of clinical trials in which preparations of either human embryonic stem cells or human neural stem cells have been surgically transplanted in patients with Parkinson’s Disease, spinal cord injury (SCI) and more recently ALS.
After transplantation, these healthy cells face the formidable challenges of both maturing into the appropriate type of cell - a dopaminergic neuron for PD, an oligodendrocyte for SCI, or a motor neuron for ALS - and then rewiring themselves back into the existing cellular network. Functional recovery for patients depends on both of these steps happening.
The invasive nature of these regenerative therapies and additional safety and efficacy concerns (ie: potential to form tumors, uncertainty whether stem cells will integrate and restore function, and tissue graft rejection) have caused some researchers to pause and consider if there is another, less traumatic way to replace damaged cells in the CNS.
Kelleher-Anderson and her colleagues at Neuronascent are among the researchers considering an alternative approach in regenerative medicine. This line of research takes advantage of the revolutionary discovery made in the 1990s that, just as adults have the ability to generate new muscle, skin, and blood cells, adult brains have the ability to generate new neurons (you have to scroll down a half a page to get to the reference). This remarkable property opens up another avenue for therapeutic intervention for treating neurodegenerative disease. Rather than surgically adding back neurons, perhaps it is possible to deliver drugs that can stimulate the brain’s natural ability to generate more neurons.
Kelleher- Anderson presented some early results from her team at Neuronascent. They have identified a candidate small molecule drug that seems to enhance the ability of human neural stem cells to generate new neurons in culture. When they tested this candidate drug in a mouse model of aging, they saw that the drug could also enhance generation of neurons in the brains of treated mice and - even better - they saw that drug treatment could reverse the learning and memory deficits normally associated with aging.
Encouraged by these findings, they tested their drug in a rat model of neurodegeneration and found similar beneficial effects – new neurons were born and rats showed improvements in motor function. One exciting interpretation of these observations is that the newly generated neurons integrated into the existing neuronal circuitry to restore motor function in animals – but this awaits further studies.
The field of regenerative medicine is growing and fostering innovative ways to address the needs of patients with neurodegenerative diseases. From transplanting stem cells to harnessing the body’s own regenerative capacity, researchers are energetically working towards this goal. The road to safe and effective regenerative therapies has thus far been studded with both success and failure. Nevertheless, the road of regenerative medicine is one that many researchers and patients alike believe is worth traveling.
---Sheila Menzies
Neurodegenerative diseases such as Alzheimers Disease, Parkinson’s Disease, Huntington’s Disease and Amyotrophic Lateral Sclerosis (ALS) are characterized by a significant and increasing loss of neurons. In the case of ALS, loss of the neurons that control voluntary muscle movement (motor neurons) underlies the progressively debilitating symptoms experienced by ALS patients. Even before patients start to notice problems with their motor function, changes have already begun to occur. It is estimated that 30-50% of a patient’s motor neurons have already degenerated by the time an ALS diagnosis is made.
As neurons begin to die, the remaining motor neurons can partially compensate for this loss (by increasing the strength and number of connections to the target muscle), but eventually the tipping point comes when too few neurons remain to properly control the muscle and clinical symptoms begin to manifest.
What can we do about this? Ah, regenerative medicine!
Speaker Judith Kelleher-Anderson, President and Chief Scientific Officer at Neuronascent, Inc., described to the audience that much of the focus in regenerative medicine has been placed on invasive therapies involving transplantation of healthy cells into the diseased area. These studies have led to a handful of clinical trials in which preparations of either human embryonic stem cells or human neural stem cells have been surgically transplanted in patients with Parkinson’s Disease, spinal cord injury (SCI) and more recently ALS.
After transplantation, these healthy cells face the formidable challenges of both maturing into the appropriate type of cell - a dopaminergic neuron for PD, an oligodendrocyte for SCI, or a motor neuron for ALS - and then rewiring themselves back into the existing cellular network. Functional recovery for patients depends on both of these steps happening.
The invasive nature of these regenerative therapies and additional safety and efficacy concerns (ie: potential to form tumors, uncertainty whether stem cells will integrate and restore function, and tissue graft rejection) have caused some researchers to pause and consider if there is another, less traumatic way to replace damaged cells in the CNS.
Kelleher-Anderson and her colleagues at Neuronascent are among the researchers considering an alternative approach in regenerative medicine. This line of research takes advantage of the revolutionary discovery made in the 1990s that, just as adults have the ability to generate new muscle, skin, and blood cells, adult brains have the ability to generate new neurons (you have to scroll down a half a page to get to the reference). This remarkable property opens up another avenue for therapeutic intervention for treating neurodegenerative disease. Rather than surgically adding back neurons, perhaps it is possible to deliver drugs that can stimulate the brain’s natural ability to generate more neurons.
Kelleher- Anderson presented some early results from her team at Neuronascent. They have identified a candidate small molecule drug that seems to enhance the ability of human neural stem cells to generate new neurons in culture. When they tested this candidate drug in a mouse model of aging, they saw that the drug could also enhance generation of neurons in the brains of treated mice and - even better - they saw that drug treatment could reverse the learning and memory deficits normally associated with aging.
Encouraged by these findings, they tested their drug in a rat model of neurodegeneration and found similar beneficial effects – new neurons were born and rats showed improvements in motor function. One exciting interpretation of these observations is that the newly generated neurons integrated into the existing neuronal circuitry to restore motor function in animals – but this awaits further studies.
The field of regenerative medicine is growing and fostering innovative ways to address the needs of patients with neurodegenerative diseases. From transplanting stem cells to harnessing the body’s own regenerative capacity, researchers are energetically working towards this goal. The road to safe and effective regenerative therapies has thus far been studded with both success and failure. Nevertheless, the road of regenerative medicine is one that many researchers and patients alike believe is worth traveling.
---Sheila Menzies
Monday, July 26, 2010
Drug Development for Neurodegenerative Diseases Conference Part 3
After a bit of a hiatus (following the departure of Meghan Kallman, our fearless former Communications Manager), we now bring you Part III of our blog series covering the marcus evans 2nd Annual Drug Discovery For Neurodegeneration Conference (see Part I here and Part II here).
Switching tracks, the next speaker, Dr. Gregory Stewart, Director of CNS Drug Therapy R&D for Medtronic, offered a slightly different perspective on therapeutic development in his talk entitled: Targeted Drug Delivery for Neurodegenerative Disease: A New Hope.
Medtronic is the world’s largest medical technology company. One of their major markets is drug-infusion pumps. With current revenues over $1.4B annually, drug delivery is a good market to be in!
Dr. Stewart discussed many of the reasons why drug developers should be thinking carefully about drug delivery. Questions of safety, cost, speed, compliance, and intellectual property may all be factors influencing drug delivery. As one example, if a patient takes morphine orally for pain relief, they need 300 times (30,000%!!!) more drug then if the morphine were delivered locally (via a pump for example).
As another example, patients taking a drug orally can get effects (including side effects) anywhere in their bodies, as most organs of the body will be exposed to a given drug (whereas, as Stewart pointed out, using a pump limits exposure of a given drug to the organ of interest). This can be a good thing, as when you take an aspirin for a headache and then go on to stub your toe (the aspirin can affect the pain in both places) but can be less good when you take that same aspirin for your headache and it upsets your stomach.
Targeted delivery has the further benefit of bypassing the liver (which constantly breaks down and filters out substances in your body), which can lead to a reduction of drug dosage at the desired target. Dr. Stewart provided several other compelling benefits for use of targeted delivery for drugs, particularly when the brain is the target.
In Medtronic’s view, the key failure of biotechnology to date has been the problem of failing to understand the nuances of drug delivery. Now that we have a vastly improved understanding of delivery principles, the drug development community is poised to make great advances, particularly at the final frontier of crossing the blood-brain-barrier and getting drugs directly to the brain and spinal cord.
With the increasing number of big and bulky drugs like antibodies and cell-based therapeutics being developed for ALS, drug delivery issues will only continue to grow in importance. Dr. Stewart urged drug developers to think about drug delivery issues early in the process, as this will impact the ultimate efficacy of any candidate therapeutic.
Switching tracks, the next speaker, Dr. Gregory Stewart, Director of CNS Drug Therapy R&D for Medtronic, offered a slightly different perspective on therapeutic development in his talk entitled: Targeted Drug Delivery for Neurodegenerative Disease: A New Hope.
Medtronic is the world’s largest medical technology company. One of their major markets is drug-infusion pumps. With current revenues over $1.4B annually, drug delivery is a good market to be in!
Dr. Stewart discussed many of the reasons why drug developers should be thinking carefully about drug delivery. Questions of safety, cost, speed, compliance, and intellectual property may all be factors influencing drug delivery. As one example, if a patient takes morphine orally for pain relief, they need 300 times (30,000%!!!) more drug then if the morphine were delivered locally (via a pump for example).
As another example, patients taking a drug orally can get effects (including side effects) anywhere in their bodies, as most organs of the body will be exposed to a given drug (whereas, as Stewart pointed out, using a pump limits exposure of a given drug to the organ of interest). This can be a good thing, as when you take an aspirin for a headache and then go on to stub your toe (the aspirin can affect the pain in both places) but can be less good when you take that same aspirin for your headache and it upsets your stomach.
Targeted delivery has the further benefit of bypassing the liver (which constantly breaks down and filters out substances in your body), which can lead to a reduction of drug dosage at the desired target. Dr. Stewart provided several other compelling benefits for use of targeted delivery for drugs, particularly when the brain is the target.
In Medtronic’s view, the key failure of biotechnology to date has been the problem of failing to understand the nuances of drug delivery. Now that we have a vastly improved understanding of delivery principles, the drug development community is poised to make great advances, particularly at the final frontier of crossing the blood-brain-barrier and getting drugs directly to the brain and spinal cord.
With the increasing number of big and bulky drugs like antibodies and cell-based therapeutics being developed for ALS, drug delivery issues will only continue to grow in importance. Dr. Stewart urged drug developers to think about drug delivery issues early in the process, as this will impact the ultimate efficacy of any candidate therapeutic.
Friday, July 23, 2010
Drug Discovery for Neurodegenerative Diseases Part II
After a bit of a hiatus (following the departure of Meghan Kallman, our fearless former Communications Manager), we now bring you Part II of our blog series covering the marcus evans 2nd Annual Drug Discovery For Neurodegeneration Conference (see Part I here and Part III here).
We thought that there were some points worth mentioning from a presentation entitled: Translational AD Drug Discovery and Development: The Potential Role of In Vivo Multi-modal Imaging Techniques (yes, it is a mouthful), presented by Dr. Feng Luo of Abbott Laboratories.
Dr. Luo started off by reminding the audience of the recent dismal track record of developing treatments targeting neurodegenerative diseases. Over the past several years, there has been a 90% attrition rate in drugs for neurodegeneration. Clearly, this high failure rate suggests that the drug development community has a problem. The question is, how do we fix it?
Dr. Luo started off his presentation by telling the audience that he and his colleagues at Abbott Labs believe that one of the keys to more effective drug development is finding better biomarkers and creating more relevant “translatable” preclinical studies. They are specifically interested in answering the question “How could we incorporate biomarkers into early [i.e. preclinical] phases of drug development to increase R&D efficiency?” He went on to explain that the approach they are most interested in is the development of more reliable animal imaging tools. In pursuing this approach, they are adapting the most widely-used human imaging (PET, MRI) methods for mice. As a test of this, they sought to characterize one of the most widely-used mouse models of Alzheimer’s Disease (AD), the Tg2576 mouse.
It has long been observed that the human Alzheimer’s brain shows lowered energy usage (hypometabolism). Luo’s group was interested in exploring whether this hypometabolism could serve as a preclinical biomarker; however, no one had ever looked to see whether the Tg2576 mice display this feature of AD. Using three different imaging methods (FDG-PET, fMRI, and MRS) Luo and his group analyzed brain metabolism of the Tg2576 mouse over time. They were shocked to find that 7 month old Tg2576 mice, the age in which animals normally begin to display Alzheimer’s symptoms, not only failed to show decreased brain metabolism, but were instead hypermetabolic (meaning rather than using less energy, the brains of these mice were using more energy)!
As these studies showed, the Tg2576 mouse does not model all aspects of the disease in humans, suggesting that the Tg2576 mouse model may be of limited use in the development of imaging-based disease biomarkers for AD. As Dr. Luo noted in his presentation, animal models are often poorly characterized from a translational perspective. If we are to be able to assess the true utility of any potential therapy, it is critical to show that the therapy acts at its intended target early in the discovery and development process.
This finding in the AD model calls attention to the critical importance of careful characterization of animal models used to research other neurodegenerative diseases such as ALS. While it is generally accepted in the medical research community that there is no perfect animal model for any human disease (meaning disease in an animal will never perfectly mimic disease in a human), developing a detailed description and understanding of all relevant aspects of a given animal model enables researchers to understand the limitations inherent to any particular model so that results can be properly interpreted. The availability and use of multiple animal models for pre-clinical testing increases the probability of finding a drug that will translate to humans.
Although imperfect, animal models have long served as valuable drug development tools, leading to FDA approval of multiple important drugs now widely and safely used in humans. Fortunately for those interested in breakthroughs in ALS therapeutics, extensive characterization of the SOD1 mouse model by researchers at ALS TDI and many other institutions have enabled better and more interpretable studies using this valuable drug development tool. ALS TDI recently announced that extensive characterization of the new TDP-43 mouse model of ALS is also underway (http://quest.mda.org/news/als-tdi-full-speed-ahead).
We thought that there were some points worth mentioning from a presentation entitled: Translational AD Drug Discovery and Development: The Potential Role of In Vivo Multi-modal Imaging Techniques (yes, it is a mouthful), presented by Dr. Feng Luo of Abbott Laboratories.
Dr. Luo started off by reminding the audience of the recent dismal track record of developing treatments targeting neurodegenerative diseases. Over the past several years, there has been a 90% attrition rate in drugs for neurodegeneration. Clearly, this high failure rate suggests that the drug development community has a problem. The question is, how do we fix it?
Dr. Luo started off his presentation by telling the audience that he and his colleagues at Abbott Labs believe that one of the keys to more effective drug development is finding better biomarkers and creating more relevant “translatable” preclinical studies. They are specifically interested in answering the question “How could we incorporate biomarkers into early [i.e. preclinical] phases of drug development to increase R&D efficiency?” He went on to explain that the approach they are most interested in is the development of more reliable animal imaging tools. In pursuing this approach, they are adapting the most widely-used human imaging (PET, MRI) methods for mice. As a test of this, they sought to characterize one of the most widely-used mouse models of Alzheimer’s Disease (AD), the Tg2576 mouse.
It has long been observed that the human Alzheimer’s brain shows lowered energy usage (hypometabolism). Luo’s group was interested in exploring whether this hypometabolism could serve as a preclinical biomarker; however, no one had ever looked to see whether the Tg2576 mice display this feature of AD. Using three different imaging methods (FDG-PET, fMRI, and MRS) Luo and his group analyzed brain metabolism of the Tg2576 mouse over time. They were shocked to find that 7 month old Tg2576 mice, the age in which animals normally begin to display Alzheimer’s symptoms, not only failed to show decreased brain metabolism, but were instead hypermetabolic (meaning rather than using less energy, the brains of these mice were using more energy)!
As these studies showed, the Tg2576 mouse does not model all aspects of the disease in humans, suggesting that the Tg2576 mouse model may be of limited use in the development of imaging-based disease biomarkers for AD. As Dr. Luo noted in his presentation, animal models are often poorly characterized from a translational perspective. If we are to be able to assess the true utility of any potential therapy, it is critical to show that the therapy acts at its intended target early in the discovery and development process.
This finding in the AD model calls attention to the critical importance of careful characterization of animal models used to research other neurodegenerative diseases such as ALS. While it is generally accepted in the medical research community that there is no perfect animal model for any human disease (meaning disease in an animal will never perfectly mimic disease in a human), developing a detailed description and understanding of all relevant aspects of a given animal model enables researchers to understand the limitations inherent to any particular model so that results can be properly interpreted. The availability and use of multiple animal models for pre-clinical testing increases the probability of finding a drug that will translate to humans.
Although imperfect, animal models have long served as valuable drug development tools, leading to FDA approval of multiple important drugs now widely and safely used in humans. Fortunately for those interested in breakthroughs in ALS therapeutics, extensive characterization of the SOD1 mouse model by researchers at ALS TDI and many other institutions have enabled better and more interpretable studies using this valuable drug development tool. ALS TDI recently announced that extensive characterization of the new TDP-43 mouse model of ALS is also underway (http://quest.mda.org/news/als-tdi-full-speed-ahead).
Monday, June 28, 2010
ALS Drug Development Update
It can be difficult to keep abreast of all of the news that is relevant to research and treatment of ALS. Because of that, Prize4Life has designed tools to help keep researchers and the public up-to-date on what is happening in the research and drug development worlds (you can sign up for the free Weekly Digest and ALS Forum Newsletter if you want to get these alerts in your inbox). Below is a roundup of the most relevant ALS drug development news from the last six weeks. Check it out!
Invitrogen Contributes New Research for ALS
Invitrogen's stem cell business is participating in a new research program designed to find treatments for ALS. Life Technologies, Invitrogen's parent company, has partnered with the University of California San Diego and the Salk Institute for Biological Studies to research cell transplant therapies as a potential ALS treatment.
FoldRx Pharmaceuticals secures $29 million financing
FoldRx Pharmaceuticals has closed a $29 million financing deal to support a program in neurodegenerative disease. "With the addition of two top-tier venture firms to our already strong investor base, we believe we now have access to the resources necessary to commercialize tafamidis and build FoldRx into an independent, orphan-disease focused company," said Richard Labaudinière, Ph.D., President and CEO of FoldRx, in a FierceBiotech release. FoldRx Pharmaceuticals is focusing on disease-modifying small-molecule therapeutics to treat diseases of protein misfolding and aggregation, key pathological processes for many chronic neurodegenerative diseases, including ALS.
Aukera Therapeutics Named 2010 MIT Business Plan Contest Life Sciences Track Winner
Cambridge-based start-up biotech, Aukera Therapeutics, is developing Angiogenin, a novel protein therapy, to treat ALS. As the winner of MIT's Business Plan Contest, Aukera will receive seed funding and in-kind support from The Cambridge Innovation Center.
Stem Cell Therapeutics Wins US Patent
Stem Cell Therapeutics has received US patent rights for production of neural stem cells, a technology intensively investigated for its therapeutic use in ALS and other neurodegenerative diseases.
Amorfix Life Sciences and PREVENT Sign Licensing Agreement on ALS Vaccine
Amorfix has granted exclusive licensing rights to PREVENT for clinical development of its lead ALS vaccine for therapeutic purposes. Amorfix has retained the rights to develop the antibodies for diagnostic purposes.
Q Therapeutics’ Collaborators at Johns Hopkins Receive $1 Million to Study Glial Cells for Neurodegenerative Conditions
This infusion of funds is expected to accelerate characterization of Q Therapeutics’ proprietary glial cell-based therapeutic (Q-Cells®). This technology is being developed as a treatment for ALS and other neurodegenerative diseases.
ALS TDI: Changes in the Works
ALS TDI’s expertise using the SOD1 based model for target identification and drug discovery has made significant contributions to the ALS field. Recently, the non-profit ALS biotech has announced incorporation of a TDP-43 mouse model into its drug development program. This is a valuable effort that could bear dividends for the entire ALS research community.
Cytokinetics Announces Opening of a Phase IIa "Evidence of Effect" Clinical Trial of CK-2017357
Cytokinetics will begin a Phase II study of its fast skeletal muscle troponin activator, CK-2017357, for patients with peripheral artery disease and claudication. This announcement follows an ongoing Phase IIa trial in ALS patients. Cytokinetics believes this second study will further inform their understanding of possible therapeutic applications for this novel drug candidate for ALS and other skeletal muscle-associated diseases.
Neuralstem Updates Clinical Trial Progress
The first FDA approved stem cell clinical trial for ALS is reported to be on track. Neuralstem has completed safety evaluations of its first cohort of ALS patients and plans to move forward with the second cohort using a more extensive treatment regime.
Pfizer, Washington University announce collaboration
Pharma giant Pfizer grants Washington University unprecedented access to proprietary information regarding 500+ drug candidates. The collaboration is expected to uncover new uses for existing compounds.
Teva Reports Disappointing ALS Phase II Results for Talampanel
Teva’s talampanel is a no-go for ALS after discouraging Phase II data are reported. Despite demonstrating safety, talampanel did not reach its primary endpoint and will not be advanced as an ALS therapeutic.
New Virtual ALS-focused Company Aukera Therapeutics Wins MIT Award
Aukera, a finalist in the MIT $100K Business Plan Contest, wins the audience award for its development of a novel ALS therapeutic. The technology is based out of research from Harvard Medical School.
Repligen Files IND for Neurodegenerative Disease Treatment
Repligen has filed an investigational new drug application with the FDA for a Phase I study of its selective HDAC3 inhibitor, RG2833. While Repligen's initial drug development focus will be Friedreich's ataxia, RG2833 may have a broader therapeutic use for the treatment of ALS and other neurodegenerative diseases.
Invitrogen Contributes New Research for ALS
Invitrogen's stem cell business is participating in a new research program designed to find treatments for ALS. Life Technologies, Invitrogen's parent company, has partnered with the University of California San Diego and the Salk Institute for Biological Studies to research cell transplant therapies as a potential ALS treatment.
FoldRx Pharmaceuticals secures $29 million financing
FoldRx Pharmaceuticals has closed a $29 million financing deal to support a program in neurodegenerative disease. "With the addition of two top-tier venture firms to our already strong investor base, we believe we now have access to the resources necessary to commercialize tafamidis and build FoldRx into an independent, orphan-disease focused company," said Richard Labaudinière, Ph.D., President and CEO of FoldRx, in a FierceBiotech release. FoldRx Pharmaceuticals is focusing on disease-modifying small-molecule therapeutics to treat diseases of protein misfolding and aggregation, key pathological processes for many chronic neurodegenerative diseases, including ALS.
Aukera Therapeutics Named 2010 MIT Business Plan Contest Life Sciences Track Winner
Cambridge-based start-up biotech, Aukera Therapeutics, is developing Angiogenin, a novel protein therapy, to treat ALS. As the winner of MIT's Business Plan Contest, Aukera will receive seed funding and in-kind support from The Cambridge Innovation Center.
Stem Cell Therapeutics Wins US Patent
Stem Cell Therapeutics has received US patent rights for production of neural stem cells, a technology intensively investigated for its therapeutic use in ALS and other neurodegenerative diseases.
Amorfix Life Sciences and PREVENT Sign Licensing Agreement on ALS Vaccine
Amorfix has granted exclusive licensing rights to PREVENT for clinical development of its lead ALS vaccine for therapeutic purposes. Amorfix has retained the rights to develop the antibodies for diagnostic purposes.
Q Therapeutics’ Collaborators at Johns Hopkins Receive $1 Million to Study Glial Cells for Neurodegenerative Conditions
This infusion of funds is expected to accelerate characterization of Q Therapeutics’ proprietary glial cell-based therapeutic (Q-Cells®). This technology is being developed as a treatment for ALS and other neurodegenerative diseases.
ALS TDI: Changes in the Works
ALS TDI’s expertise using the SOD1 based model for target identification and drug discovery has made significant contributions to the ALS field. Recently, the non-profit ALS biotech has announced incorporation of a TDP-43 mouse model into its drug development program. This is a valuable effort that could bear dividends for the entire ALS research community.
Cytokinetics Announces Opening of a Phase IIa "Evidence of Effect" Clinical Trial of CK-2017357
Cytokinetics will begin a Phase II study of its fast skeletal muscle troponin activator, CK-2017357, for patients with peripheral artery disease and claudication. This announcement follows an ongoing Phase IIa trial in ALS patients. Cytokinetics believes this second study will further inform their understanding of possible therapeutic applications for this novel drug candidate for ALS and other skeletal muscle-associated diseases.
Neuralstem Updates Clinical Trial Progress
The first FDA approved stem cell clinical trial for ALS is reported to be on track. Neuralstem has completed safety evaluations of its first cohort of ALS patients and plans to move forward with the second cohort using a more extensive treatment regime.
Pfizer, Washington University announce collaboration
Pharma giant Pfizer grants Washington University unprecedented access to proprietary information regarding 500+ drug candidates. The collaboration is expected to uncover new uses for existing compounds.
Teva Reports Disappointing ALS Phase II Results for Talampanel
Teva’s talampanel is a no-go for ALS after discouraging Phase II data are reported. Despite demonstrating safety, talampanel did not reach its primary endpoint and will not be advanced as an ALS therapeutic.
New Virtual ALS-focused Company Aukera Therapeutics Wins MIT Award
Aukera, a finalist in the MIT $100K Business Plan Contest, wins the audience award for its development of a novel ALS therapeutic. The technology is based out of research from Harvard Medical School.
Repligen Files IND for Neurodegenerative Disease Treatment
Repligen has filed an investigational new drug application with the FDA for a Phase I study of its selective HDAC3 inhibitor, RG2833. While Repligen's initial drug development focus will be Friedreich's ataxia, RG2833 may have a broader therapeutic use for the treatment of ALS and other neurodegenerative diseases.
Monday, June 21, 2010
Global Day for ALS
As a member of the International Alliance of ALS/MND Associations, Prize4Life is pleased to participate in Global Day for ALS. Since 1997, the International Alliance has celebrated 21 June as the global day of recognition for ALS– a disease that affects people in every country of the globe.
June 21st is the summer solstice—a turning point—and every year the ALS community undertakes a range of activities to express its hope that this day will be another turning point in the search for cause, treatment and cure of this disease.
Members of the International Alliance, and those who have been touched by ALS worldwide, do a number of things to call attention to the plight of ALS patients. Some organize meetings of patients and caregivers; they put on social events, create working groups, hold fundraisers, or simply come together to reflect on being part of the global fight against ALS.
The following is a reprint of the annual letter from Gudjon Sigurdsson, the Chairman of the International Alliance of ALS/MND Associations:
Global day 2010
The fight continues for a world free of ALS/MND. This year’s global day is no different from others. We will do our best to spread the word about the damn thing we all fight against. Soon we will celebrate this as the day we remember as the day we won the fight and got rid of ALS/MND.
Until then we all must do our best to enjoy every moment of each day. As well as we possibly can. “A glass full of darkness and sorrow is always by our hand. A bit further is a glass filled with light and opptimism. Reach for it.” These words of an Icelandic poet who died from ALS/MND, are worth keeping in mind.
My wife and I are celebrating our 25 years anniversary of marriage this month. I am very lucky she has put up with me all this time. We have agreed that this is only possible if both do their best to make it last and be enjoyable. Friendship and connections with others does not happen by itself. No, we need to work on it. Communicate and tell people we love, that we do.
In those times of crisis we need to defend our rights, find new ways of doing things and do not forget we are here for all the PALS around the world. Making it possible for them to enjoy life, support research and inprove the law and regulations in our home country.
BEST OF LUCK
Gudjon Sigurdsson
Chairman, International Alliance of ALS/MND Associations
Global Day is a day of commemoration and strength-building. It, like ALS Awareness Month here in the United States, is an opportunity to assert the resilience of the human spirit over the ravages of ALS.
Prize4Life believes deeply in this resilience, and makes change our business. We know that it is a priority to assist those in need right now, and our colleagues in patient service programs are doing a spectacular job of that. Other organizations, such as the ALS Association and the MDA, fund researchers upfront, and their support has done incredible things for the progression of ALS science.
Prize4Life’s programs are intended to speed up this work by providing resources and incentives that may not otherwise be available to researchers and to the ALS community. Our prizes are intended to foster creativity and new approaches (see the range of talented teams who are currently competing here). This blog, for example, is designed to bring different stakeholders and opinions together to discuss various elements of the ALS, R&D, and philanthropy spaces. Our ALS Forum is a free news source for patients and researchers alike, a cutting-edge resource that gathers the most up-to-date ALS research information in one place.
We are proud of these contributions, and we are always on the lookout for other ways to bring innovative parties and stakeholders together.
What will you do to help win this battle? For a list of ideas, click here. Or email us!
June 21st is the summer solstice—a turning point—and every year the ALS community undertakes a range of activities to express its hope that this day will be another turning point in the search for cause, treatment and cure of this disease.
Members of the International Alliance, and those who have been touched by ALS worldwide, do a number of things to call attention to the plight of ALS patients. Some organize meetings of patients and caregivers; they put on social events, create working groups, hold fundraisers, or simply come together to reflect on being part of the global fight against ALS.
The following is a reprint of the annual letter from Gudjon Sigurdsson, the Chairman of the International Alliance of ALS/MND Associations:
Global day 2010
The fight continues for a world free of ALS/MND. This year’s global day is no different from others. We will do our best to spread the word about the damn thing we all fight against. Soon we will celebrate this as the day we remember as the day we won the fight and got rid of ALS/MND.
Until then we all must do our best to enjoy every moment of each day. As well as we possibly can. “A glass full of darkness and sorrow is always by our hand. A bit further is a glass filled with light and opptimism. Reach for it.” These words of an Icelandic poet who died from ALS/MND, are worth keeping in mind.
My wife and I are celebrating our 25 years anniversary of marriage this month. I am very lucky she has put up with me all this time. We have agreed that this is only possible if both do their best to make it last and be enjoyable. Friendship and connections with others does not happen by itself. No, we need to work on it. Communicate and tell people we love, that we do.
In those times of crisis we need to defend our rights, find new ways of doing things and do not forget we are here for all the PALS around the world. Making it possible for them to enjoy life, support research and inprove the law and regulations in our home country.
BEST OF LUCK
Gudjon Sigurdsson
Chairman, International Alliance of ALS/MND Associations
Global Day is a day of commemoration and strength-building. It, like ALS Awareness Month here in the United States, is an opportunity to assert the resilience of the human spirit over the ravages of ALS.
Prize4Life believes deeply in this resilience, and makes change our business. We know that it is a priority to assist those in need right now, and our colleagues in patient service programs are doing a spectacular job of that. Other organizations, such as the ALS Association and the MDA, fund researchers upfront, and their support has done incredible things for the progression of ALS science.
Prize4Life’s programs are intended to speed up this work by providing resources and incentives that may not otherwise be available to researchers and to the ALS community. Our prizes are intended to foster creativity and new approaches (see the range of talented teams who are currently competing here). This blog, for example, is designed to bring different stakeholders and opinions together to discuss various elements of the ALS, R&D, and philanthropy spaces. Our ALS Forum is a free news source for patients and researchers alike, a cutting-edge resource that gathers the most up-to-date ALS research information in one place.
We are proud of these contributions, and we are always on the lookout for other ways to bring innovative parties and stakeholders together.
What will you do to help win this battle? For a list of ideas, click here. Or email us!
Thursday, June 10, 2010
Prize4Life holds Art, Life, Spring Gala and Art auction to raise funds for ALS
On Thursday, May 27, 2010, Prize4Life held its inaugural A.L.S. Gala and Art Auction at the Roger Smith Hotel in New York City. Sponsored by Bloomberg LP, Vertex Pharmaceuticals, Biogen IDEC, and Bristol-Myers Squibb, the event raised over $110,000 towards Prize4Life’s innovative efforts to find a cure for this devastating neurodegenerative disease (for more on our programs, click here).
The evening included dinner and an auction, led by Sotheby’s auctioneer Jamie Niven (son of David Niven, the famed actor who passed away from ALS). Mayor Bloomberg presented the evening’s opening remarks, and attendees included Nobel laureate Dr. Torsten Wiesel, Asaf Shariv, the Consul General of Israel, and renowned sports painter Bill Lopa.
A companion cocktail party and art showing, held at Sotheby’s on May 20th, also drew a large crowd, including guest of honor Gabriella Shalev, the Israeli Ambassador to the United Nations, who graciously gave an introduction to the evening. Guests sipped champagne, watched a short video on Prize4Life's co-founder and CEO Avi Kremer, and enjoyed the art that was hung for display.
Prior to the auction, the Huffington Post ran an emotive op-ed by artist Miriam Cabessa on her work's physical qualities, and its relationship with ALS. See that piece here. Photographs from both events are available here.
Works auctioned on May 27th included Gold Landscape, by Miriam Cabessa, At the Beach by Natan Elkanovich, Hanna & Shemu'el, by Boaz Vaadia, Ruth and Gehrig by Bill Lopa, The Unveiling by MuKha, Window Box, 2002 by Tim Prentice, Untitled #100 by Leora Laor, and Boy's Head, by Itmar Jobani.
Thanks to all w
ho attended and helped make both events such a great success!
The evening included dinner and an auction, led by Sotheby’s auctioneer Jamie Niven (son of David Niven, the famed actor who passed away from ALS). Mayor Bloomberg presented the evening’s opening remarks, and attendees included Nobel laureate Dr. Torsten Wiesel, Asaf Shariv, the Consul General of Israel, and renowned sports painter Bill Lopa.
Mayor Bloomberg presented the evening's opening remarks
Sotheby's Jamie Niven led the A.L.S. Art auction
A companion cocktail party and art showing, held at Sotheby’s on May 20th, also drew a large crowd, including guest of honor Gabriella Shalev, the Israeli Ambassador to the United Nations, who graciously gave an introduction to the evening. Guests sipped champagne, watched a short video on Prize4Life's co-founder and CEO Avi Kremer, and enjoyed the art that was hung for display.
Prior to the auction, the Huffington Post ran an emotive op-ed by artist Miriam Cabessa on her work's physical qualities, and its relationship with ALS. See that piece here. Photographs from both events are available here.Works auctioned on May 27th included Gold Landscape, by Miriam Cabessa, At the Beach by Natan Elkanovich, Hanna & Shemu'el, by Boaz Vaadia, Ruth and Gehrig by Bill Lopa, The Unveiling by MuKha, Window Box, 2002 by Tim Prentice, Untitled #100 by Leora Laor, and Boy's Head, by Itmar Jobani.
Thanks to all w
ho attended and helped make both events such a great success!
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