I found this and thought I would post it for anyone who hasn't
read about it. Looks really interesting for getting a cure.
Markus

First Drug Therapy for RP to Enter Human Safety Studies
The Foundation Fighting Blindness recently learned that the U.S. Food and
Drug Administration (FDA) approved an application from the biotechnology
company Neurotech, to begin a phase 1 human clinical trial to test the
safety of a delivery device containing a drug to treat patients with
end-stage RP.
Clinical testing is scheduled to begin in the fall of 2003.

This safety study represents the first clinical trial of a drug therapy for
RP.

One of the major challenges to treatment of retinal disease has been the
ability to deliver therapeutic drugs directly to the retina.
Neurotech, based in France and Rhode Island, developed Encapsulated Cell
Technology (ECT), which enables controlled, continuous, long-term delivery
of a drug called ciliary neurotrophic factor (CNTF) in patients with
end-stage RP.

The Foundation Fighting Blindness (FFB) was an early supporter of the use of
this technology for retinal diseases.

Dr. Gerald Chader, Chief Scientific Officer of FFB said, "Today's
announcement is one we've all been anxiously awaiting. CNTF and several
other drug therapies have shown promise in a wide variety of animal models
with RP.
However, none of these drugs can pass through the blood/retina barrier,
making traditional drug delivery with systemic injections or pills
ineffective.
Neurotech's implantable ECT device may have at last broken through this
previously formidable barrier."

The ECT device is a testament to The Foundation's perseverance in advancing
retinal disease therapies. The ECT device was originally developed for use
in treating Lou Gehrig's disease and cancer. It was during these studies
that FFB realized that the ECT might be adaptable to treat diseases of the
retina.
FFB encouraged Neurotech to test the device with CNTF in RP animal models.
Through FFB's innovative Medical Therapy Program, which reaches out to
industry to encourage and foster vision research, FFB provided funding to
support these important tests.

ECT consists of a very small capsule containing retinal pigment epithelial
cells (RPE) that have been genetically modified to produce CNTF. The
capsule has very small pores that allow oxygen and nutrients to diffuse in
to sustain the RPE cells and also allow CNTF to diffuse out. The tiny pores
prevent the modified RPE cells from escaping and protect the cells from the
body's immune system.

CNTF was chosen for its potent ability to delay the death of retinal
photoreceptor cells in animal studies.

Because CNTF has not been tested in patients before, the first phase of this
clinical trial will test the drug and the ECT device in 10 pre-selected
patients with end-stage RP. In this way, the safety of the treatment can be
evaluated without risk to existing vision. If all goes well in this phase I
safety study, a phase II trial would then test the ability of the treatment
to preserve vision in sighted RP patients. The timing of future trials is
not yet known, and at this time patients are not being selected for future
trials.

ECT and other emerging drug delivery devices could open the door for several
survival factors that, like CNTF, have shown promise in treating the entire
spectrum of retinal degenerative diseases. We hope to soon see other drug
therapies join CNTF in clinical trials. There's still more work ahead, but
The Foundation's efforts are clearly bearing fruit.

The Foundation Fighting Blindness' Mission FFB is dedicated to finding the
causes, preventions, treatments and cures for retinal degenerative diseases,
including retinitis pigmentosa (RP), macular degeneration, Usher syndrome
and others. FFB funds 155 research projects at
55 prominent institutions around the world and, as part of its mission,
works with companies and other research entities to help advance research.



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