The Ethics of Medical Research on Children
Last Updated 2009-05-07 at 11:13:45
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Last Updated 2010-03-01 at 12:40:56
THe 2010 Request for Proposal is now available. Submitted proposals must be postmarked no later than 11:59PM , May 15, 2010. Emailed proposals will NOT be accepted. Inquiries may be directed to Lance W. Johnston, Executive Director, BDSRA, at 1-800-448-4570 or 740-927-4298. Email address: bdsra1@bdsra.org.
Read more in Microsoft Word(.doc)
Read more in Adobe Acrobat(.pdf)Social Security Adds 38 New Compassionate Allowance Conditions
Last Updated 2010-02-11 at 20:01:37
Michael J. Astrue, Commissioner of Social Security, today announced that the agency is adding 38 more conditions to its list of Compassionate Allowances. This is the first expansion since the original list of 50 conditions - 25 rare diseases and 25 cancers - was announced in October 2008. The new conditions range from adult brain disorders to rare diseases that primarily affect children. The complete list of the new Compassionate Allowance conditions is attached.
"The addition of these new conditions expands the scope of Compassionate Allowances to a broader subgroup of conditions like early-onset Alzheimer's disease," Commissioner Astrue said. "The expansion we are announcing today means tens of thousands of Americans with devastating disabilities will now get approved for benefits in a matter of days rather than months and years."
Compassionate Allowances are a way of quickly identifying diseases and other medical conditions that clearly qualify for Social Security and Supplemental Security Income disability benefits. It allows the agency to electronically target and make speedy decisions for the most obviously disabled individuals. In developing the expanded list of conditions, Social Security held public hearings and worked closely with the National Institutes of Health, the Alzheimer's Association, the National Organization for Rare Disorders, and other groups.
"The diagnosis of Alzheimer's indicates significant cognitive impairment that interferes with daily living activities, including the ability to work," said Harry Johns, President and CEO of the Alzheimer's Association. "Now, individuals who are dealing with the enormous challenges of Alzheimer's won't also have to endure the financial and emotional toll of a long disability decision process."
"This truly innovative program will provide invaluable assistance and support to patients and families coping with severely disabling rare diseases," said Peter L. Saltonstall, President and CEO of the National Organization for Rare Disorders (NORD). "On behalf of those patients and families, I want to thank Commissioner Astrue and his enthusiastic team for creating and now expanding a program that will have a direct impact on the quality of life of thousands of individuals."
"The initiative not only assists those whose applications are quickly processed, but also assists those whose applications need more time and attention from SSA adjudicators," said Marty Ford, Co-Chair, Social Security Task Force, Consortium for Citizens with Disabilities. "We are pleased to see today's expansion and look forward to working with Commissioner Astrue on further expansion of this decision-making tool and other ways to expedite determinations and decisions for disability claims."
"We will continue to hold hearings and look for other diseases and conditions that can be added to our list of Compassionate Allowances," Commissioner Astrue said. "There can be no higher priority than getting disability benefits quickly to those Americans with these severe and life-threatening conditions."
Social Security will begin electronically identifying these 38 new conditions March 1.
For more information about the agency's Compassionate Allowances initiative, go to www.socialsecurity.gov/compassionateallowances.
New Compassionate Allowance Conditions
1.Alstrom Syndrome
2.Amegakaryocytic Thrombocytopenia
3.Ataxia Spinocerebellar
4.Ataxia Telangiectasia
5.Batten Disease
6.Bilateral Retinoblastoma
7.Cri du Chat Syndrome
8.Degos Disease
9.Early-Onset Alzheimer's Disease
10.Edwards Syndrome
11.Fibrodysplasia Ossificans Progressiva
12.Fukuyama Congenital Muscular Dystrophy
13.Glutaric Acidemia Type II
14.Hemophagocytic Lymphohistiocytosis (HLH), Familial Type
15.Hurler Syndrome, Type IH
16.Hunter Syndrome, Type II
17.Idiopathic Pulmonary Fibrosis
18.Junctional Epidermolysis Bullosa, Lethal Type
19.Late Infantile Neuronal Ceroid Lipofuscinoses
20.Leigh's Disease
21.Maple Syrup Urine Disease
22.Merosin Deficient Congenital Muscular Dystrophy
23.Mixed Dementia
24.Mucosal Malignant Melanoma
25.Neonatal Adrenoleukodystrophy
26.Neuronal Ceroid Lipofuscinoses, Infantile Type
27.Niemann-Pick Type C
28.Patau Syndrome
29.Primary Progressive Aphasia
30.Progressive Multifocal Leukoencephalopathy
31.Sanfilippo Syndrome
32.Subacute Sclerosis Panencephalitis
33.Tay Sachs Disease
34.Thanatophoric Dysplasia, Type 1
35.Ullrich Congenital Muscular Dystrophy
36.Walker Warburg Syndrome
37.Wolman Disease
38.Zellweger Syndrome
SSA Press Office 440 Altmeyer Building 6401 Security Blvd. Baltimore, MD 21235 410-965-8904 FAX 410-966-9973
"The addition of these new conditions expands the scope of Compassionate Allowances to a broader subgroup of conditions like early-onset Alzheimer's disease," Commissioner Astrue said. "The expansion we are announcing today means tens of thousands of Americans with devastating disabilities will now get approved for benefits in a matter of days rather than months and years."
Compassionate Allowances are a way of quickly identifying diseases and other medical conditions that clearly qualify for Social Security and Supplemental Security Income disability benefits. It allows the agency to electronically target and make speedy decisions for the most obviously disabled individuals. In developing the expanded list of conditions, Social Security held public hearings and worked closely with the National Institutes of Health, the Alzheimer's Association, the National Organization for Rare Disorders, and other groups.
"The diagnosis of Alzheimer's indicates significant cognitive impairment that interferes with daily living activities, including the ability to work," said Harry Johns, President and CEO of the Alzheimer's Association. "Now, individuals who are dealing with the enormous challenges of Alzheimer's won't also have to endure the financial and emotional toll of a long disability decision process."
"This truly innovative program will provide invaluable assistance and support to patients and families coping with severely disabling rare diseases," said Peter L. Saltonstall, President and CEO of the National Organization for Rare Disorders (NORD). "On behalf of those patients and families, I want to thank Commissioner Astrue and his enthusiastic team for creating and now expanding a program that will have a direct impact on the quality of life of thousands of individuals."
"The initiative not only assists those whose applications are quickly processed, but also assists those whose applications need more time and attention from SSA adjudicators," said Marty Ford, Co-Chair, Social Security Task Force, Consortium for Citizens with Disabilities. "We are pleased to see today's expansion and look forward to working with Commissioner Astrue on further expansion of this decision-making tool and other ways to expedite determinations and decisions for disability claims."
"We will continue to hold hearings and look for other diseases and conditions that can be added to our list of Compassionate Allowances," Commissioner Astrue said. "There can be no higher priority than getting disability benefits quickly to those Americans with these severe and life-threatening conditions."
Social Security will begin electronically identifying these 38 new conditions March 1.
For more information about the agency's Compassionate Allowances initiative, go to www.socialsecurity.gov/compassionateallowances.
New Compassionate Allowance Conditions
1.Alstrom Syndrome
2.Amegakaryocytic Thrombocytopenia
3.Ataxia Spinocerebellar
4.Ataxia Telangiectasia
5.Batten Disease
6.Bilateral Retinoblastoma
7.Cri du Chat Syndrome
8.Degos Disease
9.Early-Onset Alzheimer's Disease
10.Edwards Syndrome
11.Fibrodysplasia Ossificans Progressiva
12.Fukuyama Congenital Muscular Dystrophy
13.Glutaric Acidemia Type II
14.Hemophagocytic Lymphohistiocytosis (HLH), Familial Type
15.Hurler Syndrome, Type IH
16.Hunter Syndrome, Type II
17.Idiopathic Pulmonary Fibrosis
18.Junctional Epidermolysis Bullosa, Lethal Type
19.Late Infantile Neuronal Ceroid Lipofuscinoses
20.Leigh's Disease
21.Maple Syrup Urine Disease
22.Merosin Deficient Congenital Muscular Dystrophy
23.Mixed Dementia
24.Mucosal Malignant Melanoma
25.Neonatal Adrenoleukodystrophy
26.Neuronal Ceroid Lipofuscinoses, Infantile Type
27.Niemann-Pick Type C
28.Patau Syndrome
29.Primary Progressive Aphasia
30.Progressive Multifocal Leukoencephalopathy
31.Sanfilippo Syndrome
32.Subacute Sclerosis Panencephalitis
33.Tay Sachs Disease
34.Thanatophoric Dysplasia, Type 1
35.Ullrich Congenital Muscular Dystrophy
36.Walker Warburg Syndrome
37.Wolman Disease
38.Zellweger Syndrome
SSA Press Office 440 Altmeyer Building 6401 Security Blvd. Baltimore, MD 21235 410-965-8904 FAX 410-966-9973
Read more online (off-site link) Four New Members Appointed to National Neurological Disorders and Stroke Advisory Council
Last Updated 2010-02-04 at 12:54:49
The National Institute of Neurological Disorders and Stroke (NINDS) announced that four new members have joined its National Advisory Neurological Disorders and Stroke Council. The council serves as the principal advisory body to NINDS regarding the Institute’s research program planning and priorities.
"I am delighted to welcome these distinguished new appointees to the advisory council," said NINDS Director Story Landis, Ph.D. "They include a world-renowned stroke investigator and leader in thrombolytic therapy, a specialist in pediatric neurology and neonatal brain disorders, a neurologist and director of multi-disciplinary research in evidence-based treatment, and a long-time advocate for neurological research who helps families affected by serious diseases."
"I am delighted to welcome these distinguished new appointees to the advisory council," said NINDS Director Story Landis, Ph.D. "They include a world-renowned stroke investigator and leader in thrombolytic therapy, a specialist in pediatric neurology and neonatal brain disorders, a neurologist and director of multi-disciplinary research in evidence-based treatment, and a long-time advocate for neurological research who helps families affected by serious diseases."
BDSRA Approves Juvenile Drug Trial Funding
Last Updated 2009-12-10 at 08:32:07
The Board of Directors of the Batten Disease Support and Research Association is pleased to announce that it has approved a $400,000 award to the Batten Disease Diagnostic and Clinical Research Center at the University of Rochester for a clinical trial of a drug for Juvenile Batten Disease. The drug, Mycophenolate Mofetil, also known as CellCept, is an immunosuppressant used to prevent rejection of transplanted organs in children. In preliminary studies, mice affected by Batten Disease demonstrated improved motor skills when treated with the drug.
The total cost of the trial is $1.1 million. The research team, under the direction of Dr. Frederick Marshall and Dr. Jonathan Mink, is working to raise the additional $700,000 required to begin the study. BDSRA will provide updated information as it becomes available.
The total cost of the trial is $1.1 million. The research team, under the direction of Dr. Frederick Marshall and Dr. Jonathan Mink, is working to raise the additional $700,000 required to begin the study. BDSRA will provide updated information as it becomes available.
First Human Embryonic Stem Cell Lines Approved for Use Under New NIH Guidelines
Last Updated 2009-12-02 at 14:52:27
NIH Director Francis S. Collins, M.D., Ph.D., today announced the approval of the first 13 human embryonic stem cell (hESC) lines for use in NIH-funded research under the NIH Guidelines for Human Stem Cell Research adopted in July 2009.
"I am happy to say that we now have human embryonic stem cell lines eligible for use by our research community under our new stem cell policy," Dr. Collins said. "In accordance with the guidelines, these stem cell lines were derived from embryos that were donated under ethically sound informed consent processes. More lines are under review now, and we anticipate continuing to expand this list of responsibly derived lines eligible for NIH funding."
"I am happy to say that we now have human embryonic stem cell lines eligible for use by our research community under our new stem cell policy," Dr. Collins said. "In accordance with the guidelines, these stem cell lines were derived from embryos that were donated under ethically sound informed consent processes. More lines are under review now, and we anticipate continuing to expand this list of responsibly derived lines eligible for NIH funding."
StemCells, Inc. Provides Update on NCL Program
Last Updated 2009-11-18 at 08:23:22
StemCells, Inc. (NASDAQ: STEM) today provided an update on the ongoing clinical development program of its proprietary HuCNS-SC® product candidate (purified human neural stem cells) for neuronal ceroid lipofuscinosis (NCL), often referred to as Batten disease.
NIH Announces Expansion of Rare Diseases Clinical Research Network
Last Updated 2009-11-03 at 13:07:33
The National Institutes of Health announced today a second phase of the Rare Diseases Clinical Research Network (RDCRN) including funds for 19 research consortia. The Rare Diseases Clinical Research Consortia and a Data Management Coordinating Center (DMCC) will be awarded a total of just over $117 million over the next five years. The research conducted with the new funding will explore the natural history, epidemiology, diagnosis, and treatment of more than 95 rare diseases.Molecular correlates of axonal and synaptic pathology in mouse models of Batten disease
Last Updated 2009-11-03 at 13:05:51
The paper shows that distinct compartments of neurons (specifically the long fibre-like processes called axons and points of connection known as synapses) are particularly vulnerable in the NCLs. However, our understanding of how and why these parts of neurons break down during the early stage of the disease remains poor. We have shown that there are distinct protein changes which occur during the early stages of axonal and synaptic breakdown, providing a protein "fingerprint" of the process. We have also identified two specific proteins which may have the potential for use as biomarker proteins, reporting on the underlying status of axon and synapse pathology by examining their levels in easily-accessible tissues such as muscle. This study highlights the need for further research investigating ways of targeting and protecting axons and synapses in the NCLs and also provides a new approach to study and monitor axon and synapse pathology in living individuals without having to take samples of the nervous system.UI scientists use blood brain barrier as therapy delivery system
Last Updated 2009-09-22 at 14:54:49
The blood brain barrier is generally considered an obstacle to delivering therapies from the bloodstream to the brain. However, University of Iowa researchers have discovered a way to turn the blood vessels surrounding brain cells into a production and delivery system for getting therapeutic molecules directly into brain cells. Please click on the files below to read the entire press release from the University of Iowa and the scientific research paper describing the scientific advancement from Dr. Beverly Davidson.
FDA Sets New Rules on Experimental Drugs
Last Updated 2009-09-15 at 06:55:41
The U.S. Food and Drug Administration finalized new regulations Wednesday designed to provide broader access to experimental drugs for seriously ill people who have exhausted all other commercially available treatments. The new rules, which were posted on the FDA's Web site Wednesday, mostly clarify regulations explaining how patients can receive drugs in development outside of a clinical trial, and set standards for when drug companies or researchers can charge for the treatments.
StemCells, Inc. Demonstrates Protection Of Neurological Function In Batten Mouse
Last Updated 2009-09-03 at 06:56:54
StemCells, Inc. (NASDAQ: STEM) today announced the publication of preclinical data demonstrating for the first time that transplantation of its proprietary, purified human neural stem cells delays the loss of motor function in a mouse model of infantile neuronal ceroid lipofuscinosis (NCL).
Kings College London Batten Disease Lab now on Twitter and Facebook
Last Updated 2009-08-04 at 10:40:08
You can now follow Dr. Jon Cooper and his laboratory (Batten Disease/Pediatric Storage Disorders) on Twitter and Facebook. This is a great way to stay informed about what is happening in the lab and the latest research that Dr. Cooper is undertaking.To follow PSDL on Facebook, go to:
PSDL Facebook Page
To follow PSDL on Twitter, please go to:
Twitter.com/Batten_PSDL
2009 NCL Congress Meeting
Last Updated 2009-06-12 at 10:45:08
The 12th International Congress on Neuronal Ceroid Lipofuscinoses (NCL) was held in Hamburg, Germany from June 3-6, 2009. The International Congress only meets once every 2 years. This meeting gives researchers an opportunity to gather and share their research findings on NCL with their colleagues and peers.The Abstract Summary Book is available for download. We have included it here in PDF format.
StemCells, Inc. Announces Positive Phase I Batten Trial Results
Last Updated 2009-06-11 at 11:10:55
PALO ALTO, Calif., June 8, 2009--
StemCells, Inc. (NASDAQ: STEM) announced today positive results from the first Phase I clinical trial of its proprietary HuCNS-SC product candidate (purified human neural stem cells), including demonstration of a favorable safety profile along with evidence of engraftment and long-term survival of the HuCNS-SC cells.
StemCells, Inc. (NASDAQ: STEM) announced today positive results from the first Phase I clinical trial of its proprietary HuCNS-SC product candidate (purified human neural stem cells), including demonstration of a favorable safety profile along with evidence of engraftment and long-term survival of the HuCNS-SC cells.
The stem cell transplant Phase I Safety Study in Oregon
Last Updated 2009-06-11 at 10:54:53
Starting in 2006, doctors at Oregon Health & Science University opened the brains of six severely ill children and injected special stem cells derived from human fetuses, the first such surgery known.No dangerous side effects from stem cell treatment for brain disorder
Last Updated 2009-06-09 at 11:01:19
An experimental stem-cell treatment developed by StemCells of Palo Alto has shown no dangerous side effects after being injected into six children with a rare and as-yet always fatal brain disorder, the company said Monday.
The 2009 Request For Proposals are posted
Last Updated 2009-05-12 at 11:16:31
IMPORTANT:
THE DEADLINE FOR RFPs HAS BEEN EXTENDED TO MAY 23, 2009 AT 11:59PM EST.
There is a PDF version and a Microsoft Word version. BDSRA is committed to reviewing and helping fund advancements in the treatment and prevention of Batten disease.
THE DEADLINE FOR RFPs HAS BEEN EXTENDED TO MAY 23, 2009 AT 11:59PM EST.
There is a PDF version and a Microsoft Word version. BDSRA is committed to reviewing and helping fund advancements in the treatment and prevention of Batten disease.
Drug Trial for Juvenile Batten Disease
Last Updated 2009-05-09 at 11:16:15
Finally, after 180 years, we may be able to do something about Juvenile Batten disease. Please, read on . . .
Rochester Receives $1.2 Million to Battle Rare Childhood Disease
Last Updated 2009-05-08 at 11:14:15
A $1.2 million grant from the National Institutes of Health will bolster University of Rochester experts' research and care efforts for children suffering Batten disease, a rare neurological disorder that erupts without warning, stealing kids' sight, crippling their cognitive and motor capacities, and ultimately, taking their lives.The Ethics of Medical Research on Children
Last Updated 2009-05-07 at 11:13:45
The Ethics of Medical Research on Children : NPR's "All Things Considered" , October 31, 2006. Sometime in the next few weeks, a surgeon in Portland , Ore., will drill a series of small holes in a young child's brain and inject neural stem cells. The child has a rare brain disease that is usually fatal before puberty. The hope is that the cells will halt the progression of the disease.
National Public Radio (NPR) News Story on Batten Disease
Last Updated 2009-05-06 at 11:11:50
Hear the audio version w/interviews or read the Health & Science article which aired on Tuesday, June 20, 2006. In what may be an unprecedented collaboration, a rare and as yet incurable illness has brought together two unlikely communities: parents of children and owners of dogs. The two groups are linked by the fatal illness known as Batten disease.