[Stitcher]

PDTrials.org
Study of CERE-120 in People with Parkinson’s Disease
Official Study Title: Randomized, Double-Blind, Sham Surgery-Controlled Study of CERE-120 (Adeno-Associated Virus Serotype 2 [AAV2]-Neurturin [NTN]) to Assess the Efficacy and Safety of Bilateral Intraputaminal (IPu) Delivery in Subjects with Idiopathic Parkinson’s Disease
http://www.pdtrials.org/front/trial_...p?trial_id=128



ClinicalTrials.gov
Double-Blind, Multicenter, Sham Surgery Controlled Study of CERE-120 For Efficacy and Safety in Subjects With Idiopathic Parkinson's Disease
This study is currently recruiting patients.
Verified by Ceregene March 2007
http://clinicaltrials.gov/show/NCT00400634



Recombinate DNA Advisory Committee
Meeting Minutes
September 2006
http://www4.od.nih.gov/oba/RAC/minut...utes_09-06.pdf



PowerPoint Presentation
CERE-120 (AAV-Neurturin) for Parkinson’s Disease
NIH OBA Protocol # 0501-689
A Phase I, Open-Label Study of CERE-120 (Adeno-Associated Virus Serotype 2 [AAV2]-Neurturin [NTN] to Assess the Safety and Tolerability of Intrastriatal Delivery to Subjects with Idiopathic Parkinson's Disease
http://www.webconferences.com/nihoba...des16Mar05.pdf

[jeanb]

Carolyn,

Thanks for posting this - neurturin is a neurotrophic factor in the same family as GDNF. Since Amgen is withholding its synthetic GDNF from us, this treatment may be the next best thing for PWP.

Last week I talked to one of the scientists at the MJFOX Foundation at length about this trial. The scientist said that if the treatment is found to work in the Phase II trial, those patients who got the sham surgery could get the real surgery after that trial's end. The participants would not have to wait until after the Phase III trial - they could get the treatment after the Phase II trial is finished -- assuming the results show that neurturin works.

And thanks to all PWP who are considering joining this trial. Without trial participants, we will never have better treatments - or a cure.

[Stitcher]

Jean, I have put my name into the hat for the Ceregene trial. I hope to hear from them this coming week.

I understand that Duodopa Phase II trial should begin this summer sometime. Another option for every one to consider.

Solvay Pharmaceutisals...Duodopa
http://www.duodopa.com/faqs/faqsabou...990-2-0,00.htm - Q&A
http://www.solvaypharmaceuticals.com...641-2-0,00.htm
http://www.solvaypharmaceuticals.com...595-2-0,00.htm
http://www.hospitalmanagement.net/co...als/neopharma/

November 28, 2005: Solvay Pharmaceuticals completes market authorization in 28 European countries for Duodopa®
Solvay Pharmaceuticals has obtained market authorization for DUODOPA®, the product for treating advanced Parkinson’s disease in 28 European countries.
http://www.solvaypharmaceuticals.com...456-2-0,00.htm

e-Move 2004: http://www.mdvu.org/emove/article.asp?ID=722

[aftermathman]

if Duodopa is approved in Europe then why is it back at phase 2 in the US.

Its no wonder drugs take so long to get to market.

Have I misunderstood the situation ?

Neil.

[paula_w]

Actually it is a Phase III that is coming over to the US. A lady from Solvay was at the PAN forum and I spoke with her. They were supposed to start recruiting in January, but have pushed it back to summer.

She said she didn't understand the FDA...lol

After completing a very short registration to receive a user name and password, you can see the info about all the PD trials with history and pharmaceutical companies here.

http://tinyurl.com/3aol6k

paula

[chasmo]

Originally Posted by paula_w

Actually it is a Phase III that is coming over to the US. A lady from Solvay was at the PAN forum and I spoke with her. They were supposed to start recruiting in January, but have pushed it back to summer.

She said she didn't understand the FDA...lol

After completing a very short registration to receive a user name and password, you can see the info about all the PD trials with history and pharmaceutical companies here.

http://tinyurl.com/3aol6k

paula

Hi Paula;
IS their any word on complication rates??
Blockages, infections, etc.??

I remember seeing that video of the Scandinavian college professor at PAN 2002 or there abouts. It was very impressive, especially how the peak dose dyskinesias where controlled.

Charlie

[jeanb]

2007 will bring us some very hopeful treatments in clinical trials:

-- ceregene120 - phase II, 2007
-- duodopa - phase III, 2007
-- neurologix - phase II, latter half of 2007

[aftermathman]

Spheramine (phase IIb) due late 2007 / early 2008.

Neil.

[jeanb]

Spheramine as well!
thanks, Neil

[Stitcher]

Ceregene Presents Long Term Follow-Up Data From Phase 1 Trial of CERE-120 Demonstrating Improved Motor Function in Parkinson's Patients

http://www.ceregene.com/press.asp

Encouraging Results Prompted Phase 2 Study Now Underway at Nine Centers

SAN DIEGO, Calif., April 16 /PRNewswire/ -- Ceregene, Inc., a
biopharmaceutical company, today presented long term follow-up data from a
Phase 1 clinical trial of CERE-120, a gene therapy product in development
for the treatment of Parkinson's disease, demonstrating a 36 percent
(p<0.001) reduction in Parkinson's symptoms at 12 months after
administration, as measured by the Unified Parkinson's Disease Rating Scale
(UPDRS) motor "off" score ("motor off" meaning patients were off
Parkinson's medication at evaluation time). CERE-120 was also shown to be
well tolerated in this study. The 12-month results were presented today by
Philip Starr, M.D., Ph.D., associate professor of neurosurgery at the
University of California, San Francisco at the American Association of
Neurological Surgeons (AANS) annual meeting in Washington D.C. These data
were also highlighted by the AANS in a specific CERE-120 press release
issued by the Association.

The Phase 1 trial was an open-label study conducted in 12 patients with
advanced Parkinson's disease at two clinical trial sites -- University of
California, San Francisco and Rush University Medical Center in Chicago.
All 12 patients enrolled in the study underwent stereotactic neurosurgery
to deliver CERE-120 into their putamen -- a region of the brain affected by
the degeneration of neurons in Parkinson's disease.

CERE-120 was delivered at two different doses, and patients receiving
the low dose demonstrated approximately 36 percent improvement in UPDRS
motor "off" scores by nine months and patients receiving the four-fold
higher dose showed a similar effect three months sooner. This statistically
significant, 36 percent improvement was maintained in both groups at the
12-month mark, the final follow-up time point in the study. Patients also
demonstrated a 50 percent 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.

This Phase 1 clinical trial was partially supported by a grant from The
Michael J. Fox Foundation for Parkinson's Research. Based on the results of
the Phase 1 study, the Foundation has provided a $1.9 million grant, which
will provide partial funding for Ceregene's ongoing Phase 2 trial.

"We are pleased with the results of this early study which suggests
that the majority of patients treated with CERE-120 may have exhibited
significant and stable improvement for a full year after receiving a single
administration of CERE-120," stated Jeffrey M. Ostrove, Ph.D., president
and chief executive officer of Ceregene. "Each of our products, much like
CERE-120, is aimed at unmet neurodegenerative diseases including
Parkinson's disease, Alzheimer's disease and Amyotrophic Lateral Sclerosis
(AML or Lou Gehrig's disease) with the goal of not only improving the
symptoms but also preventing progression of the disease."

"Through stereotactic surgery, we are able to administer CERE-120 in a
highly targeted fashion to one area of the brain that Parkinson's disease
affects, and we were able to do this safely," stated Dr. Starr.

"Given the encouraging data from the Phase 1 trial of CERE-120 in
Parkinson's disease, we are conducting a follow-on Phase 2 clinical trial
that is currently enrolling patients at nine clinical trial sites in the
United States," said Raymond T. Bartus, Ph.D., Ceregene's senior vice
president of clinical and preclinical R&D and chief operating officer. "The
data from the Phase 1 trial are reflective of the impressive results we
gathered from preclinical studies, which demonstrated the ability of
CERE-120 to stimulate the survival and improve the function of key neuronal
cells affected by Parkinson's disease, as well as an excellent safety
profile over a wide range of CERE-120 dose levels."

About Phase 2 Trial of CERE-120 Currently Underway

A double-blind, controlled Phase 2 clinical trial is now enrolling 51
patients with advanced Parkinson's disease at nine medical centers in the
United States, with two thirds of patients being enrolled in the active
treatment group and one third in the control group. Patients are receiving
CERE-120 via stereotactic neurosurgery to deliver the drug into the putamen
region of the brain. Patients will be followed for 12 months for safety and
efficacy. Contacts at the medical centers involved in this study can be
found through a link on the Ceregene web site: http://www.ceregene.com.

About CERE-120

CERE-120 is composed of an adeno-associated virus (AAV) vector carrying
the gene for neurturin (NTN), a naturally occurring protein known to repair
damaged and dying dopamine-secreting neurons, keeping them alive and
functioning normally. NTN is a member of the same protein family as glial
cell-derived neurotrophic factor (GDNF). The two molecules have similar
pharmacological properties, and both have been shown to benefit the
midbrain dopamine neurons that degenerate in Parkinson's disease and are
responsible for the major motor impairments. CERE-120 is delivered by
stereotactic injection to the affected area of the brain, providing stable,
long-lasting expression of NTN in a highly targeted fashion. Ceregene owns
exclusive technology and product rights to CERE-120.

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), which is headquartered in
South San Francisco, CA. Ceregene's investors include Alta Partners, MPM
Capital, Investor Growth 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 more than $90 million in research to date, either
directly or through partnerships.