| Roger D. Kornberg Joins Prize4Life's Scientific Advisory Board PR Web (press release) Prize4Life, a non-profit organization dedicated to accelerating the discovery of treatments and a cure for Amyotrophic Lateral Sclerosis (ALS), is pleased to announce the appointment of Roger D. Kornberg, PhD, to its Scientific Advisory Board. See all stories on this topic » |
| ALS Therapy Development Institute Announces Eighth Annual "The Cure Is Coming ... PR.com (press release) This year, event organizers hope for a record 700 walkers, runners and riders, all who will raise money and awareness of ALS TDI efforts to develop effective treatments for amyotrophic lateral sclerosis (ALS), better known as Lou Gehrig's disease. See all stories on this topic » |
Showing posts with label news. Show all posts
Showing posts with label news. Show all posts
Thursday, March 22, 2012
ALS MND Daily News Digest
Tuesday, March 20, 2012
Political Medicine
From The Hill Blog
There is new legislation, introduced by Senator Kay Hagen (D-N.C.), to expedite development and access to life-saving medicines by the U.S. Food and Drug Administration (FDA). The Transforming and Regulatory Environment to Accelerate Access to Treatments (TREAT) Act will help unleash and accelerate the biotech promise of developing new cures.
This legislation will enable the FDA to speed up the review and approval process and encourage more investment and innovation to attack diseases that are serious or life-threatening. While not all the diseases mentioned above would be covered by this legislation, the legislation provides a much needed jump start on a new approach at the FDA.
America landed a man on the moon in less than a decade and yet it takes 10-15 years to take a new drug from discovery to treating a patient. Given the pace of scientific change, America is held back and patients are suffering because of the long time lags. Investors and private funders are reluctant to invest with the regulatory barriers placed in their way.
According to James Greenwood, president of the Biotechnology Industry Organization, “Sixty-one percent of venture capitalists now cite FDA regulatory challenges as having the highest impact on their investment decisions, and 40% expect to decrease their investment in the development of new therapies.”
The approval process for many of these treatment efforts and drugs is simply unacceptably slow. The TREAT Act will speed it up and have a positive impact on these new breakthrough technologies. Congress needs to pass it this year.
Our company, Neuralstem, has seen encouraging signs of a treatment effect transplanting our neural stem cells in the spinal chord of ALS patients. Our trial has included 14 patients thus far. Others have implanted stem cells in the eyes and also seen encouraging early signs for treating various types of macular degeneration.
There are potentially important and specific changes championed by this legislation. Most notable among them is a new “standard” that the FDA may adopt to help speed along approval of new therapies for diseases like ALS. The FDA is encouraged to consider surrogate markers of efficacy; and to allow faster approval. For instance, if Neuralstem were to show that we can keep our patients’ breathing function strong enough to stay off of artificial breathing machines for a significant time, we might not have to wait until the patients actually die, to prove that they lived longer, to offer the therapy.
If a company such as Neuralstem demonstrates the benefit of a treatment according to the new FDA standards proposed in the TREAT Act, the therapy can be offered sooner and it can begin benefitting patients sooner. In a disease like ALS, where on average patients die around 3 years from diagnosis, time is clearly of the essence.
The FDA simply must keep pace with the safe and effective development of new, innovative, scientific breakthroughs that can literally provide hope for those with life-threatening diseases. The old, bureaucratic ways simply won’t work in the 21st century.
Garr is president and CEO of Neuralstem, a major biotechnology company doing stem cell research on some of America’s most devastating diseases
New hope for our toughest diseases
- 03/19/12 05:47 PM ET
As the saying goes, there is good news and there is bad news.
Here is the bad news: Right now, there are an estimated 5.4 million Americans suffering from Alzheimer’s disease, one million suffering from Parkinson’s, over five million who have a disease related to a traumatic brain injury, 265,000 with spinal cord injuries, 30,000 who have ALS (Lou Gehrig’s disease), and 129,000 who have brain cancer. Over 25 million Americans also suffer from diabetes – about 8.3% of the population – and the problem is growing.
After that, it’s hard to believe there is good news, but here it is: R & D breakthroughs are happening in science and biotechnology at a remarkable pace to deal with these diseases. Biotech companies are on the front lines to find cures for life-threatening diseases. There are currently 800 new treatments being developed for cancer by biotech companies, 300 for heart disease, 200 for diabetes and 100 for Alzheimer’s, according to the Biotechnology Industry Organization.
Some companies, such as the one I represent, Neuralstem, Inc., are using state-of-the-art stem cell research to attack diseases such as ALS, brain and spinal cord injuries, and down the road, Parkinson’s and Alzheimer’s.
Here is the bad news: Right now, there are an estimated 5.4 million Americans suffering from Alzheimer’s disease, one million suffering from Parkinson’s, over five million who have a disease related to a traumatic brain injury, 265,000 with spinal cord injuries, 30,000 who have ALS (Lou Gehrig’s disease), and 129,000 who have brain cancer. Over 25 million Americans also suffer from diabetes – about 8.3% of the population – and the problem is growing.
After that, it’s hard to believe there is good news, but here it is: R & D breakthroughs are happening in science and biotechnology at a remarkable pace to deal with these diseases. Biotech companies are on the front lines to find cures for life-threatening diseases. There are currently 800 new treatments being developed for cancer by biotech companies, 300 for heart disease, 200 for diabetes and 100 for Alzheimer’s, according to the Biotechnology Industry Organization.
Some companies, such as the one I represent, Neuralstem, Inc., are using state-of-the-art stem cell research to attack diseases such as ALS, brain and spinal cord injuries, and down the road, Parkinson’s and Alzheimer’s.
What we need to do now is accelerate this research and speed up the development of new cures for those in desperate need.
This legislation will enable the FDA to speed up the review and approval process and encourage more investment and innovation to attack diseases that are serious or life-threatening. While not all the diseases mentioned above would be covered by this legislation, the legislation provides a much needed jump start on a new approach at the FDA.
America landed a man on the moon in less than a decade and yet it takes 10-15 years to take a new drug from discovery to treating a patient. Given the pace of scientific change, America is held back and patients are suffering because of the long time lags. Investors and private funders are reluctant to invest with the regulatory barriers placed in their way.
According to James Greenwood, president of the Biotechnology Industry Organization, “Sixty-one percent of venture capitalists now cite FDA regulatory challenges as having the highest impact on their investment decisions, and 40% expect to decrease their investment in the development of new therapies.”
The approval process for many of these treatment efforts and drugs is simply unacceptably slow. The TREAT Act will speed it up and have a positive impact on these new breakthrough technologies. Congress needs to pass it this year.
Our company, Neuralstem, has seen encouraging signs of a treatment effect transplanting our neural stem cells in the spinal chord of ALS patients. Our trial has included 14 patients thus far. Others have implanted stem cells in the eyes and also seen encouraging early signs for treating various types of macular degeneration.
There are potentially important and specific changes championed by this legislation. Most notable among them is a new “standard” that the FDA may adopt to help speed along approval of new therapies for diseases like ALS. The FDA is encouraged to consider surrogate markers of efficacy; and to allow faster approval. For instance, if Neuralstem were to show that we can keep our patients’ breathing function strong enough to stay off of artificial breathing machines for a significant time, we might not have to wait until the patients actually die, to prove that they lived longer, to offer the therapy.
If a company such as Neuralstem demonstrates the benefit of a treatment according to the new FDA standards proposed in the TREAT Act, the therapy can be offered sooner and it can begin benefitting patients sooner. In a disease like ALS, where on average patients die around 3 years from diagnosis, time is clearly of the essence.
The FDA simply must keep pace with the safe and effective development of new, innovative, scientific breakthroughs that can literally provide hope for those with life-threatening diseases. The old, bureaucratic ways simply won’t work in the 21st century.
Garr is president and CEO of Neuralstem, a major biotechnology company doing stem cell research on some of America’s most devastating diseases
Monday, March 19, 2012
ALS MND Daily News Digest
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Tuesday, March 13, 2012
ALS MND Daily News Digest
| Fresh hope for motor neuron disease PhysOrg.com (press release) Motor neuron disease, also called amyotrophic lateral sclerosis, is a neurodegenerative disease which causes the death of motor nerve cells within the spinal cord and brain that are responsible for controlling muscle movement. See all stories on this topic » |
Thursday, March 8, 2012
ALS MND Daily Digest
| Fourteenth Patient Dosed in Neuralstem ALS Stem Cell Trial Citybizlist (press release) ... the second patient to receive stem cells in the cervical (upper back) region of the spine was dosed on February 29th in the ongoing Phase I trial of its spinal cord neural stem cells in amyotrophic lateral sclerosis (ALS or Lou Gehrig's disease). See all stories on this topic » |
Wednesday, March 7, 2012
ALS MND Daily News Digest
| EMA grants CK-2017357 orphan medicinal product designation Pharmaceutical Business Review Cytokinetics' fast skeletal muscle troponin activator CK-2017357 has been granted orphan medicinal product designation by European Medicines Agency (EMA), for the treatment of amyotrophic lateral sclerosis (ALS), also commonly known as Lou Gehrig's ... See all stories on this topic » |
| Fourteenth Patient Dosed in Neuralstem ALS Stem Cell Trial Sacramento Bee Neuralstem is in an FDA-approved Phase I safety clinical trial for amyotrophic lateral sclerosis (ALS), often referred to as Lou Gehrig's disease, and has been awarded orphan status designation by the FDA. In addition to ALS, the company is also ... See all stories on this topic » |
Tuesday, March 6, 2012
ALS MND Daily News Digest
| Cytokinetics Announces That the European Medicines Agency Has Granted CK ... MarketWatch (press release) Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease that afflicts 20000 to 30000 people in the United States and fewer than 1 in 10000 persons in the EU. Approximately 5600 new cases of ALS are diagnosed each year in the US, ... See all stories on this topic » | ||
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Friday, March 2, 2012
ALS MND Daily News Digest
| Toxins Afloat in Shark Fin Soup? Health.com THURSDAY, March 1 (HealthDay News) — Shark fins contain high levels of a neurotoxin called BMAA, which is linked to neurodegenerative diseases in humans such as Alzheimer's and Lou Gehrig's disease (amyotrophic lateral sclerosis), according to a new ... See all stories on this topic » |
| A Passion For The Rainforest Leads Botanist To Fight Human Diseases Livermore Independent ... is helping to make inroads into one of the most difficult problems in medical research: understanding and treating the neurological diseases Alzheimers, Parkinsons and amyotrophic lateral sclerosis, or ALS, better known as Lou Gehrig's disease. See all stories on this topic » |
Wednesday, February 29, 2012
ALS MND Daily News Digest
| Observations refute widely held view on causal mechanism in ALS EurekAlert (press release) IMAGE: This is a microscopic image of two nerve-muscle synapses in a mouse model of a human motor neuron disease (amyotrophic lateral sclerosis, ALS) in which nerve-muscle contacts break. Energy-producing organelles... "The methods – both in terms of ... See all stories on this topic » |
Monday, February 27, 2012
ALS MND Daily News Digest
| Nature: BrainStorm's NurOwn™ Stem Cell Technology Offers Hope for Treating ... EON: Enhanced Online News (press release) BrainStorm is currently conducting a Phase I/II Human Clinical Trial for Amyotrophic Lateral Sclerosis (ALS) also known as Lou Gehrig's disease at the Hadassah Medical center. Initial results have shown that Brainstorm's NurOwn™ therapy is safe, ... See all stories on this topic » |
| (Graphic: Business Wire) TheStreet.com (press release) SYN is developing, or has partnered the development of, product candidates to treat pulmonary arterial hypertension (PAH), relapses in multiple sclerosis, cognitive dysfunction in multiple sclerosis, fibromyalgia and amyotrophic lateral sclerosis (ALS) ... See all stories on this topic » |
| 'Zumba junkies' raise money for ALS research Helena Independent Record Leah Bernhardt has seen how amyotrophic lateral sclerosis, or ALS, slowly kills. The nerve-cell disease is genetic and runs in her family. Her grandmother died from complications of ALS, which is also known as Lou Gehrig's disease, and her aunt is ... See all stories on this topic » |
Friday, February 24, 2012
ALS MND Daily News Digest
| BrainStorm Featured on CNBC MarketWatch (press release) ... a developer of innovative stem cell technologies for neurodegenerative disorders, announced that NurOwn(TM), its autologous stem cell therapy for amyotrophic lateral sclerosis (ALS), or Lou Gehrig's Disease, was profiled yesterday on CNBC. See all stories on this topic » |
Wednesday, February 22, 2012
ALS MND Daily News Digest
| ALS Thanks Havertown Woman For Volunteering Patch.com The ALS Association Greater Philadelphia Chapter The ALS (Amyotrophic Lateral Sclerosis) Association Greater Philadelphia Chapter shares its praise of Kelly Slipakoff and her selfless work for the association with the Haverford-Havertown Patch. See all stories on this topic » |
| NV students dance to aid teacher Toledo Blade THE BLADE/AMY E. VOIGT Enlarge | Photo Reprints Northview High School students filled the gymnasium recently to dance and celebrate their mathematics teacher, Melissa Belcher, who learned in late 2010 that she has amyotrophic lateral sclerosis, ... See all stories on this topic » |
Wednesday, February 15, 2012
ALD MND Daily News Digest
| Without key protein, synapses break up Futurity: Research News (Credit: Fallon Lab, Brown University) The research may be relevant to efforts to treat motor neuron diseases, such as amyotrophic lateral sclerosis (ALS, Lou Gehrig's Disease) and spinal muscular atrophy. "What neuromuscular junctions do ... See all stories on this topic » |
Medical Update
from mdanews.orgALS TDI to Test Multiple
Sclerosis Drug in ALS
The MDA-supported ALS Therapy Development Institute (ALS-TDI) plans to launch a phase 2 clinical trial of TDI 132, a compound that modulates the immune system
Article Highlights:
The nonprofit biotech ALS Therapy Development Institute plans to launch a phase 2 trial to determine the safety and tolerability of TDI 132 — also known as fingolimod — in ALS.
Fingolimod (brand name Gilenya) is approved by the U.S. Food and Drug Administration for the treatment of multiple sclerosis. In humans, Gilenya inhibits certain immune system cells from entering the central nervous system, where they can result in damage to motor neurons.
ALS TDI reports that mice treated with TDI 132 showed positive outcomes on several disease measures.
To learn more, read the full ALS News Online article.
Sclerosis Drug in ALS
The MDA-supported ALS Therapy Development Institute (ALS-TDI) plans to launch a phase 2 clinical trial of TDI 132, a compound that modulates the immune system
Article Highlights:
To learn more, read the full ALS News Online article.
als.mda.org
Scott Wiebe
National Director — Outreach Services & ALS Division
World Leader in ALS Research and Services
Scott Wiebe
National Director — Outreach Services & ALS Division
World Leader in ALS Research and Services
Tuesday, February 14, 2012
ALS MND Daily News Digest
| The ALS Therapy Development Institute to Launch Phase II Clinical Trial of TDI ... MarketWatch (press release) About ALS Therapy Development Institute ( www.als.net ) The mission of the ALS Therapy Development Institute (ALS TDI) is to develop effective therapeutics that slow or stop amyotrophic lateral sclerosis (ALS, Lou Gehrig's disease) as soon as possible ... See all stories on this topic » |
| Concussion In Contact Sports (VIDEO) Huffington Post At the age of 34, he is also living with a debilitating disease, amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's Disease. And he's not alone. A recent story in Scientific American profiles Kevin Turner, called "the collision expert," ... See all stories on this topic » |
Friday, February 10, 2012
ALS MND Daily News Digest
| Tony Proudfoot's presence lives on Montreal Gazette The steps have been small for Vicki Proudfoot, somewhat painstakingly taken, in the nearly 14 months since the death of her husband, Tony, to amyotrophic lateral sclerosis (ALS) - the degenerative disease that paralyzes muscles of the body. See all stories on this topic » |
Tuesday, February 7, 2012
ALS MND Daily News Digest
| Kubly back in action at the Capitol Granite Falls Advocate Tribune The message was reported to have choked up colleagues on both sides of the aisle, in light of the 68-year-old senator's battle with amyotrophic lateral sclerosis (ALS), or Lou Gehrig disease. "I would like to thank all the members and the staff who ... See all stories on this topic » |
| Father Leaves Lasting Legacy to Families Affected by ALS Patch.com VCCF Greg Merfield passed away in January at the age of 51 from amyotrophic lateral sclerosis, also known as ALS or Lou Gehrig's disease. But before he died, the Agoura Hills dad wanted to leave behind a lasting legacy. Diagnosed at the age of 49, ... See all stories on this topic » |
Sunday, February 5, 2012
ALS MND News Digest
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Friday, February 3, 2012
ALS/MND News Digest
| Remembering Tony Judt New York Times Mr. Judt famously wrote the autobiographical fragments collected in "The Memory Chalet" after he was stricken with amyotrophic lateral sclerosis and had become effectively quadriplegic. In the Times Book Review, Geoff Dyer said the book was most deeply ... See all stories on this topic » |
| Students walk for muscular dystrophy The Baylor Lariat Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease, is a form of muscular dystrophy. Muscular dystrophy diseases can affect a person of any age, Wheeler said. John Morris, assistant athletic director for broadcasting at Baylor, ... See all stories on this topic » |
Tuesday, January 31, 2012
ALS-MND News Digest
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| The inspirational Colm Murray Mayo News IT was hard not to be moved by the excellent documentary on RTÉ's Colm Murray and his battle with Motor Neuron disease, which was aired on Monday night last. The 58-year-old is one of the most popular news or sports broadcasters on Irish television and ... See all stories on this topic » | ||
| Biogen profit up; 2012 forecast falls short Reuters On Tuesday, Weston, Massachusetts-based biotech issued a 2012 earnings forecast that was lower than Wall Street expected after several clinical trials, including one for its drug to treat amyotrophic lateral sclerosis, or ALS, enrolled faster than ... See all stories on this topic » |
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