new hope in the pipeline:
http://pharmalicensing.com/news/headlines/1014332716_3c757d2c0813b
Pharmlicensing 22 Feb. 2002
Titan Pharmaceuticals, Inc. has received a $2 million
milestone payment from Schering AG, Titan's corporate partner for
worldwide development, manufacture and commercialization of Spheramine,
Titan's novel cell therapy for the treatment of Parkinson's disease.
The milestone payment follows the recent successful
completion of Titan's Phase I/II clinical study of Spheramine, and the
decision to initiate larger, randomized clinical testing of Spheramine
for the treatment of late-stage Parkinson's disease. Data from the
recently completed 12-month study will be presented at medical
conferences later this year.
Spheramine is a novel cell therapy consisting of retinal
pigment epithelial (RPE) cells adhered to microscopic carriers. RPE
cells are normal, mature human cells that are implanted into the central
nervous system to produce dopamine, a neurotransmitter that is deficient
in the brains of Parkinson's disease patients. RPE cells are fully
developed and differentiated cells, thereby potentially eliminating
concerns associated with other approaches, such as use of embryonic
neuronal cells or stem cells that can migrate and change after
implantation. The elimination of the variables associated with
post-implantation migration and differentiation is an important
potential advantage for Spheramine.
"Successful completion of the Phase I/II clinical study is an
important milestone in the Spheramine product development program,"
stated Dr. Louis R. Bucalo, Chairman, President and CEO of Titan.
"We are pleased with the continued progress of this
development program," stated Dr. Joachim-Friedrich Kapp, Head of the
Strategic Business Unit Specialized Therapeutics of Schering AG.
"Preliminary results from the pilot clinical study are very encouraging,
and we now look forward to further clinical testing of Spheramine."
Titan Pharmaceuticals, Inc. is a biopharmaceutical company
focused on the development and commercialization of novel treatments for
central nervous system (CNS) disorders, cancer and other serious and
life-threatening diseases.
Stereotaxic Intrastriatal Implantation of Retinal Pigment Epithelial
Cells Attached to Microcarriers in Advanced Parkinson disease (PD)
Patients: Long Term Follow-Up
Ray L. Watts, Cathy D. Raiser, Natividad P. Stover, Michael L.
Cornfeldt, Alfred W. Schweikert, Richard C. Allen, Thyagarajan
Subramanian, Roy A. E. Bakay Atlanta, GA; Somerville, NJ; Cleveland, OH;
Chicago, IL
OBJECTIVE: Evaluation of the safety and efficacy of unilateral
stereotaxic implantation of cultured human retinal pigment epithelial
(RPE) cells attached to microcarriers (Spheramine) in patients with
advanced PD in an open label pilot study. BACKGROUND: Stereotaxic
intrastriatal implantation of RPE cells attached to crosslinked gelatin
microcarriers produces long term amelioration of parkinsonian motor
behavioral deficits in rodent and non-human primate models of PD, with
minimal host immune response. DESIGN/METHODS: Six patients with advanced
PD (3 males; 3 females; mean age 52.2 years; mean duration of PD 10.2
years; mean Hoehn and Yahr stage "off" 3.75) were assessed at baseline
and post-operatively using the modified CAPIT. Each patient underwent
MRI-guided stereotaxic surgery during which a total of 325,000 RPE cells
attached to microcarriers were implanted in 5 tracts, 5 mm apart in the
post-commissural putamen contralateral to the most affected side.
Immunosuppression was not used. RESULTS: All patients tolerated surgery
well and no major adverse events occurred. Post-op MRI scans revealed
accurate placement of tracts in all patients. Six patients have
undergone monthly safety assessments and post-op CAPIT evaluations at 1,
3, 6, and 9 months, three at 12 months, and one at 15 months. The UPDRS
Motor score in the practically defined "off" state (off all
anti-parkinsonian medication for at least 12 hours); UPDRS-M(off) is the
primary outcome measure. All patients have demonstrated some degree of
improvement. At 6 and 9 months post-op, the mean UPDRS-M (off) score
(n=6) improved to 35 (33%) and 30 (42%) respectively, from a pre-op
baseline mean of 52. At 12 months post-op, the mean UPDRS-M(off) score
(n=3) improved to 26 (48%) from a mean pre-op baseline value of 50. At
15 months post-op, the UPDRS-M (off) score (n=1) improved to 24 (44%)
from a pre-op baseline value of 43. Mean 9-month improvements (n=6) in
secondary outcome measures include the total UPDRS (40%), Timed Motor
Tests (on, 16%; off, 35%), PDQ39 QOL (38%), and Schwab and England
Physician Rated ADL (on, 11%; off, 34%). Bilateral improvement is
observed in motor symptoms, with the greater effect seen contralateral
to the implants. Moderate to marked reductions (37-53%) in time spent in
the "off" state are currently seen in half the patients. Three of six
patients currently have lower Dyskinesia Rating Scale scores than at
baseline, while the scores of the other three are unchanged from
baseline values. No "off-state" dyskinesias have been observed.
CONCLUSIONS: This new cell implantation therapy appears to be safe and
well tolerated out to 15 months post-implantation. Thus far efficacy
evaluations in this open-label pilot study show promise. Further
follow-up is ongoing. Supported By: Supported by a research grant from
titan Pharmaceuticals, Inc. and NINDS, NIH (4R44 NS39211-02).
Category: Movement Disorders
SubCategory: Surgical Intervention
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