PerryC
10-10-2006, 03:15 PM
I have attended the ANA meeting in Chicago, where yesterday I spoke with Dr. Bartus, the COO of Ceregene. The data look promising for safety and efficacy for this neurothropic factor, but these are still very preliminary results, so only time and more tests will tell. One thing for sure is they have been aware of the controversies surrounding GDNF and want to avoid similar miss-communications as was found in that research. However, I am not aware of any patient input sought for their Phase II protocol which has already been given a green light by the RAC (advisory committee for gene therapy in humans) at NIH and the FDA. So time will tell on this element too.
Perry Cohen
Director, Parkinson Pipeline Project
Press Release Source: Ceregene, Inc.
http://biz.yahoo.com/prnews/061010/sftu019.html?.v=76
Ceregene Announces Promising Phase 1 Results From Gene Therapy Trial
for Parkinson's Disease
Tuesday October 10, 7:00 am ET
Michael J. Fox Foundation Awards $1.9 Million for Phase 2 Efficacy
Study
SAN DIEGO, Oct. 10 /PRNewswire/ -- Ceregene, Inc. announced today
that CERE-120, a gene therapy product in development for the
treatment of Parkinson's disease, was well tolerated and appeared to
reduce symptoms by approximately 40% (p<0.001), as measured by the
Unified Parkinson's Disease Rating Scale (UPDRS) motor "off" score,
in an open-label Phase 1 study in 12 patients with advanced disease.
Initial results of the study were presented by William J. Marks Jr.,
M.D., principal investigator of the study and associate professor of
Neurology at the University of California, San Francisco (UCSF) today
at the American Neurological Association annual meeting in Chicago.
The study was supported in part by a grant from The Michael J. Fox
Foundation for Parkinson's Research. Based on the initial results,
the Foundation today announced plans to partially fund a Phase 2
study with a $1.9 million grant
"We were encouraged by the results of the Phase 1 trial," said
Deborah W. Brooks, president and CEO of The Michael J. Fox
Foundation. "Based on these and on the intriguing efficacy
observations, we're eager to continue to support research in Phase 2
that will more definitively assess the potential of CERE-120 to treat
PD."
CERE-120 is comprised of an adeno-associated virus (AAV) vector
carrying the gene for neurturin (NTN), a naturally occurring protein,
whose role is to keep dopamine-secreting neurons alive and
functioning normally. All 12 patients enrolled in the study underwent
stereotactic neurosurgery to deposit CERE-120 into their putamen. The
putamen is a region of the brain that undergoes degeneration and
reduced dopamine production in Parkinson's disease patients and this
has been closely linked to the major motor deficits in these
patients. All patients entered in the trial were judged to have
inadequate control of their disease with standard levodopa therapy
and were otherwise potential candidates for additional treatment
interventions such as deep brain stimulation (DBS) surgery.
CERE-120 was delivered at 2 different doses, with patients receiving
the low dose demonstrating approximately 40% improvement in UPDRS
motor "off" scores by 9 months and patients receiving the 4-fold
higher dose showing a similar effect 3 months sooner. Patients also
demonstrated a 50% reduction in hours of "off" time (i.e., time when
normal Parkinson's medication was ineffective and symptoms were
troubling to the patient) and a doubling of good quality "on" time
without dyskinesias (i.e., time when a patient is functioning well)
according to self-reported diaries.
NTN (neurturin) is a member of the same protein family as glial cell-
derived neurotrophic factor (GDNF) and the two molecules have similar
pharmacological properties. GDNF has previously been tested in
Parkinson's disease patients. Ceregene owns exclusive technology and
product rights to CERE-120.
"Targeted delivery of the trophic factor neurturin is a compelling
approach to treating Parkinson's disease," said Dr. Marks. "The
safety data and preliminary efficacy data that we have seen in this
Phase 1 study are encouraging. Clearly, a larger-scale study is
warranted."
According to Dr. Marks, existing treatments for Parkinson's disease
treat symptoms only, and for only a limited period of time. "Patients
with Parkinson's disease urgently need therapeutic approaches that
not only improve symptoms and function, but also have the ability to
modify the underlying disease itself in a favorable manner," he said.
In addition to Dr. Marks, the study was authored by: Jill Ostrem,
M.D., UCSF neurologist; Philip Starr, M.D., Ph.D. and Paul Larson,
M.D., who conducted the neurosurgery at UCSF; neurologist Leo
Verhagen, M.D. with neurosurgeon Roy Bakay, M.D. at Rush University
Medical Center in Chicago; and Raymond T. Bartus, Ph.D., who led the
clinical and preclinical development of CERE-120 at Ceregene.
"The planned Phase 2 trial will be a randomized controlled trial
involving approximately 50 patients, and is designed to test if the
efficacy we have seen in our initial Phase 1 trial will hold up in a
controlled study," stated Jeffrey M. Ostrove, Ph.D., president and
CEO of Ceregene.
Eight medical centers will participate in the Phase 2 study: Baylor
College of Medicine, Duke University, Oregon Health Sciences
University, University of Alabama at Birmingham, University of
Pennsylvania and Mount Sinai College of Medicine. UCSF and Rush will
also be participating.
"The Phase 1 data reported today affirms that the functioning of CERE-
120 closely resembled its performance in preclinical studies both in
terms of its overall safety as well as its possible efficacy," noted
Raymond T. Bartus, Ph.D., Ceregene's chief operating officer. "The
development of growth factors as a treatment for neurodegenerative
diseases has been hampered by the difficulty of delivering them
specifically to the targeted areas that need their neuroprotective
properties. We believe our programs increasingly demonstrate that
gene transfer may represent a safe and effective means of solving
this age-old problem," said Raymond Bartus.
About Ceregene
Ceregene, Inc. is a San Diego-based biotechnology company focused on
the development of gene therapies for neurodegenerative disorders.
Ceregene is in the clinic with CERE-110, an AAV2 based vector
expressing nerve growth factor that is being tested as a treatment
for Alzheimer's disease, and with CERE-120 for Parkinson's disease.
CERE-130 is in late preclinical development for ALS. Ceregene was
launched in January 2001 and is a former subsidiary of Cell Genesys,
Inc. (NASDAQ: CEGE - News), which is headquartered in South San
Francisco, CA. Ceregene's investors include Alta Partners, MPM
Capital and Cell Genesys, as well as Hamilton BioVentures and
California Technology Partners.
About The Michael J. Fox Foundation
Founded in 2000, The Michael J. Fox Foundation for Parkinson's
Research is dedicated to ensuring the development of a cure for
Parkinson's disease within this decade through an aggressively funded
research agenda. The Foundation has funded approximately $80 million
in research to date, either directly or through partnerships.
Media Contacts: Ceregene; UCSF
Jeffrey M. Ostrove; Ceregene, Inc. Carol Hyman; UCSF
858-458-8808 415-476-2557
jostrove@ceregene.com chyman@pubaff.ucsf.edu
----------------------------------------------------------
----------
Source: Ceregene, Inc.
Perry Cohen
Director, Parkinson Pipeline Project
Press Release Source: Ceregene, Inc.
http://biz.yahoo.com/prnews/061010/sftu019.html?.v=76
Ceregene Announces Promising Phase 1 Results From Gene Therapy Trial
for Parkinson's Disease
Tuesday October 10, 7:00 am ET
Michael J. Fox Foundation Awards $1.9 Million for Phase 2 Efficacy
Study
SAN DIEGO, Oct. 10 /PRNewswire/ -- Ceregene, Inc. announced today
that CERE-120, a gene therapy product in development for the
treatment of Parkinson's disease, was well tolerated and appeared to
reduce symptoms by approximately 40% (p<0.001), as measured by the
Unified Parkinson's Disease Rating Scale (UPDRS) motor "off" score,
in an open-label Phase 1 study in 12 patients with advanced disease.
Initial results of the study were presented by William J. Marks Jr.,
M.D., principal investigator of the study and associate professor of
Neurology at the University of California, San Francisco (UCSF) today
at the American Neurological Association annual meeting in Chicago.
The study was supported in part by a grant from The Michael J. Fox
Foundation for Parkinson's Research. Based on the initial results,
the Foundation today announced plans to partially fund a Phase 2
study with a $1.9 million grant
"We were encouraged by the results of the Phase 1 trial," said
Deborah W. Brooks, president and CEO of The Michael J. Fox
Foundation. "Based on these and on the intriguing efficacy
observations, we're eager to continue to support research in Phase 2
that will more definitively assess the potential of CERE-120 to treat
PD."
CERE-120 is comprised of an adeno-associated virus (AAV) vector
carrying the gene for neurturin (NTN), a naturally occurring protein,
whose role is to keep dopamine-secreting neurons alive and
functioning normally. All 12 patients enrolled in the study underwent
stereotactic neurosurgery to deposit CERE-120 into their putamen. The
putamen is a region of the brain that undergoes degeneration and
reduced dopamine production in Parkinson's disease patients and this
has been closely linked to the major motor deficits in these
patients. All patients entered in the trial were judged to have
inadequate control of their disease with standard levodopa therapy
and were otherwise potential candidates for additional treatment
interventions such as deep brain stimulation (DBS) surgery.
CERE-120 was delivered at 2 different doses, with patients receiving
the low dose demonstrating approximately 40% improvement in UPDRS
motor "off" scores by 9 months and patients receiving the 4-fold
higher dose showing a similar effect 3 months sooner. Patients also
demonstrated a 50% reduction in hours of "off" time (i.e., time when
normal Parkinson's medication was ineffective and symptoms were
troubling to the patient) and a doubling of good quality "on" time
without dyskinesias (i.e., time when a patient is functioning well)
according to self-reported diaries.
NTN (neurturin) is a member of the same protein family as glial cell-
derived neurotrophic factor (GDNF) and the two molecules have similar
pharmacological properties. GDNF has previously been tested in
Parkinson's disease patients. Ceregene owns exclusive technology and
product rights to CERE-120.
"Targeted delivery of the trophic factor neurturin is a compelling
approach to treating Parkinson's disease," said Dr. Marks. "The
safety data and preliminary efficacy data that we have seen in this
Phase 1 study are encouraging. Clearly, a larger-scale study is
warranted."
According to Dr. Marks, existing treatments for Parkinson's disease
treat symptoms only, and for only a limited period of time. "Patients
with Parkinson's disease urgently need therapeutic approaches that
not only improve symptoms and function, but also have the ability to
modify the underlying disease itself in a favorable manner," he said.
In addition to Dr. Marks, the study was authored by: Jill Ostrem,
M.D., UCSF neurologist; Philip Starr, M.D., Ph.D. and Paul Larson,
M.D., who conducted the neurosurgery at UCSF; neurologist Leo
Verhagen, M.D. with neurosurgeon Roy Bakay, M.D. at Rush University
Medical Center in Chicago; and Raymond T. Bartus, Ph.D., who led the
clinical and preclinical development of CERE-120 at Ceregene.
"The planned Phase 2 trial will be a randomized controlled trial
involving approximately 50 patients, and is designed to test if the
efficacy we have seen in our initial Phase 1 trial will hold up in a
controlled study," stated Jeffrey M. Ostrove, Ph.D., president and
CEO of Ceregene.
Eight medical centers will participate in the Phase 2 study: Baylor
College of Medicine, Duke University, Oregon Health Sciences
University, University of Alabama at Birmingham, University of
Pennsylvania and Mount Sinai College of Medicine. UCSF and Rush will
also be participating.
"The Phase 1 data reported today affirms that the functioning of CERE-
120 closely resembled its performance in preclinical studies both in
terms of its overall safety as well as its possible efficacy," noted
Raymond T. Bartus, Ph.D., Ceregene's chief operating officer. "The
development of growth factors as a treatment for neurodegenerative
diseases has been hampered by the difficulty of delivering them
specifically to the targeted areas that need their neuroprotective
properties. We believe our programs increasingly demonstrate that
gene transfer may represent a safe and effective means of solving
this age-old problem," said Raymond Bartus.
About Ceregene
Ceregene, Inc. is a San Diego-based biotechnology company focused on
the development of gene therapies for neurodegenerative disorders.
Ceregene is in the clinic with CERE-110, an AAV2 based vector
expressing nerve growth factor that is being tested as a treatment
for Alzheimer's disease, and with CERE-120 for Parkinson's disease.
CERE-130 is in late preclinical development for ALS. Ceregene was
launched in January 2001 and is a former subsidiary of Cell Genesys,
Inc. (NASDAQ: CEGE - News), which is headquartered in South San
Francisco, CA. Ceregene's investors include Alta Partners, MPM
Capital and Cell Genesys, as well as Hamilton BioVentures and
California Technology Partners.
About The Michael J. Fox Foundation
Founded in 2000, The Michael J. Fox Foundation for Parkinson's
Research is dedicated to ensuring the development of a cure for
Parkinson's disease within this decade through an aggressively funded
research agenda. The Foundation has funded approximately $80 million
in research to date, either directly or through partnerships.
Media Contacts: Ceregene; UCSF
Jeffrey M. Ostrove; Ceregene, Inc. Carol Hyman; UCSF
858-458-8808 415-476-2557
jostrove@ceregene.com chyman@pubaff.ucsf.edu
----------------------------------------------------------
----------
Source: Ceregene, Inc.