Even though this is about ALS, it is important to Parkinson's disease. The
governing bodies for diseases like ALS that do not have a pill therapy, are
much relaxed compared to PD. This means that gene therapy techniques are
more likely to be tried in humans with ALS first. If the therapies prove
positive, the technology transfer to GDNF and PD is relatively simple. So
what is happening here is of major importance to PD.
In my opinion fetal tissue transplantation is long gone.
Regards,
Alan Bonander ([log in to unmask])
--------- Press Release -------------
PROVIDENCE, R.I. March 23 /PRNewswire/ -- CytoTherapeutics, Inc.
(Nasdaq:CTII) today announced that the first human clinical trial for a
cell-containing implant to deliver a recombinant protein within the central
nervous system (CNS) is underway in Europe. The implants contain cells from
a non-human cell line genetically engineered to release ciliary neurotrophic
factor (CNTF), a growth factor under investigation to treat amyotrophic
lateral sclerosis (ALS). The cells are encapsulated in a membrane which
protects them from the patient's immune system, and offers the ability to
retrieve the cells, if this is desirable. The study is designed to test the
safety of the implants in up to 10 patients with ALS over a period of six
months. The trial is being conducted by Patrick Aebischer, M.D., Ph.D., a
founding scientist of CytoTherapeutics, at the Centre Hospitalier
Universitaire Vaudois (CHUV) in Lausanne, Switzerland where he is Director of
the Gene Therapy Center.
"This trial marks an important step in the Company's strategy," said Seth
A. Rudnick, M.D., CytoTherapeutics' Chairman and Chief Executive Officer.
"For the first time, it tests the ability of our proprietary
encapsulated-cell technology to deliver potent, genetically engineered growth
factors, such as CNTF, across the blood-brain barrier. Demonstrating safe
delivery of CNTF via our implants would validate the ability of our unique
cell and gene therapy approach to treat devastating neurodegerative
disorders."
CNTF is one several substances produced naturally by the body that is
thought to provide a protective effect on the nerve cells affected in ALS.
Human trials of CNTF conducted to date utilized systemic administration of
the factor. Those trials were halted reportedly due to side-effects and
unclear efficacy. Systemic administration generally requires that large
quantities of a substance be delivered to assure that a small amount may pass
across the blood-brain barrier. However, high systemic doses may be
associated with side-effects. By contrast, the implants used in this trial
are designed to deliver approximately 500 times less CNTF than that delivered
systemically in previous trials, and to deliver the factor directly to the
portion of the CNS where it is required.
Dr. Aebischer commented, "The selection of CNTF as the first of several
potential growth factors to deliver in humans was based on the profound unmet
need in ALS treatment, and our belief that our delivery approach may overcome
the difficulties CNTF has encountered when delivered by conventional
approaches."
The implants used in the trial contain CNTF-producing cells and measure
approximately 5 cm in length and less than half a millimeter in diameter.
The implants are surgically placed at the base of the spine in a common,
outpatient procedure similar to a spinal tap. The implant design and
surgical procedure are similar to those currently used in the Company's
CereCRIB(TM) clinical trials for the treatment of severe pain. After
implantation within the spinal fluid, the implants are designed to deliver
small quantities of biologically active CNTF, synthesized by the cells,
directly to the neuronal cell bodies located within the spinal cord and the
lower areas of the brain. This is the first trial to utilize a membrane
developed in conjunction with Akzo Nobel Faser AG in Wuppertal, Germany, the
Company's membrane partner.
CytoTherapeutics, Inc. is a leader in the development of cell-
containing, biocompatible implants designed to deliver therapeutic substances
for the treatment of central nervous system disorders. The Company is
currently developing products for the treatment of chronic pain, Parkinson's
disease, Alzheimer's disease and ALS, with research efforts directed to a
variety of other CNS disorders.
/CONTACT: Elizabeth Razee, Manager, Corporate Communications of
CytoTherapeutics, 401-272-3310, ext. 2132/
CO: CytoTherapeutics, Inc.
ST: Rhode Island
IN: MTC
SU:
Transmitted: 95-03-23 00:00:00 EST
