gene therapy

[Guest guest]

Hi all!

I just wanted to share what my family saw on the news the other

night. It was a Kansas City or Topeka station but they were reporting

on a medical procedure elsewhere (I think it was Chicago -- forgive

me if I'm incorrect). The point is, it was gene therapy on a 26 yr

old woman with cf. They put the genetic material in her via her nose

and it works for 21 days to clear the mucus etc. This is the first I

have heard or seen of gene therapy on or for CF patients specifically

so I was pretty excited (and so was my father! He's determined that

there has to be something medically worthwhile out there for his

grandson!)

Just wanted to share that info. Hope all are having good summers.

It's uncommonly HOT here in KS. ick!

Crystal

mom to Adam 17mo wcf, le 3yo nocf.

[Guest guest]

Hi Crystal!

Read on, It sounds as this is was what your family watched on tv:

Public release date: 29-Apr-2003

[ Print This Article | Close This Window ]

Contact: Eileen Korey

eric.sandstrom@...

University Hospitals of Cleveland

Cystic fibrosis gene therapy trial results encouraging

Cleveland, Ohio, April 29, 2003--Scientists and physicians in

Cleveland have announced encouraging results from the first-of-its-

kind gene therapy trial involving cystic fibrosis (CF) patients and a

new compacted DNA technology. The Phase I trial involving 12 patients

was launched one year ago by University Hospitals of Cleveland (UHC),

Case Western Reserve University (CWRU) School of Medicine, Children's

Hospital of Denver, and Cystic Fibrosis Foundation Therapeutics,

Inc., the nonprofit drug discovery and development affiliate of the

Cystic Fibrosis Foundation.

Cleveland-based biotechnology firm Copernicus Therapeutics Inc.

produced the non-viral gene transfer system used in the clinical

trial. Working together with UHC and CWRU researchers, Copernicus

formulated a way to " compact " or tightly bind strands of DNA so that

it is tiny enough to pass through a cell membrane and into the

nucleus. The ultimate goal is for the DNA to produce a protein needed

by people with CF to correct the basic defect in CF cells.

" The primary goal of this Phase I study was to determine if this gene

therapy method is safe and tolerable as administered in this trial.

All participants in this study completed the trial without

significant side effects and the treatment itself was well-

tolerated, " stated W. Konstan, M.D., Associate Professor of

Pediatrics at CWRU and Director of the LeRoy s Cystic Fibrosis

Center, Rainbow Babies & Children's Hospital of UHC. " The secondary

goals of the trial were to evaluate if the CF gene was successfully

transferred to airway cells and if it functioned normally, results

which would suggest that this therapy may be of benefit to people

with CF. Our data were very encouraging with indications that this

gene transfer may have occurred. "

" This gene therapy research has exciting potential as a new approach

to addressing the genetic root cause of CF, " said J. Beall,

Ph.D., president and chief executive officer of the Cystic Fibrosis

Foundation. " We are pleased that this novel method of gene delivery

has cleared the first hurdles of clinical research by proving both

safety and tolerability. We eagerly anticipate results of future

clinical studies utilizing this novel approach. " CFFT helped fund

this initial trial, along with Copernicus and the National Institutes

of Health through a Core Center Grant to the CF Center at UHC/CWRU

and through the General Clinical Research Center.

Copernicus Therapeutics recently received more than one million

dollars in state funding through the Technology Action Fund of the

Ohio Department of Development to continue development of compacted

DNA. The company is currently working on an aerosol version that

would enable people with CF to have the healthy gene delivered

directly into their lungs. During the recently completed study,

patients received the compacted DNA in a saline solution dripped into

the nasal passages. Future clinical trials will study the safety and

efficacy of the aerosol approach.

" Gene transfer technology is expected to revolutionize treatment of

genetic disease by using DNA as a novel therapeutic, " said Jeff

Wagener, M.D., Professor of Pediatrics at the Children's Hospital of

Denver, which enrolled three patients in the study. " Striking at the

root cause of CF should ultimately provide an even more effective

treatment for CF than those available today which are aimed at

managing the side effects of the disease. "

The underlying cause of CF, which affects approximately 30,000

Americans, is a defective gene that upsets a delicate salt/water

balance in the lungs. At the crux of the process is a protein,

produced by the CF gene, which controls the flow of salt and water in

and out of cells. In CF patients, this protein does not operate

normally in the cells that line the airways. In turn, the airways

accumulate thick and sticky mucus. Bacteria proliferate in the mucus

and cause chronic infections that permanently damage lungs.

Under the direction of Dr. Konstan, the research teams for this

recently completed Phase I study delivered the healthy gene into

twelve adult CF patients in a saline solution dripped slowly into

their nasal passages. Investigators monitored salt transport in the

nose, called the " nasal potential difference, " as a barometer of the

procedure's success. " CF patients have a markedly abnormal nasal

potential difference, " Dr. Konstan said. Through biopsies of nasal

tissue, researchers determined whether the healthy gene was " taken

up " by the cells and produced enough protein to affect the transport

of salt and water in and out of the cells. They found that two-thirds

of patients treated had a meaningful increase in the transport of

chloride ion in the nose.

The researchers and physicians involved in this study will present

their findings at the American Society of Gene Therapy meeting in

Washington, D.C. in early June, and at the Cystic Fibrosis Foundation

burg meeting at the end of May.

###

University Hospitals Health System (UHHS) is the region's premier

healthcare delivery system, serving patients at more than 150

locations throughout northern Ohio. The System's 947-bed, tertiary

medical center, University Hospitals of Cleveland (UHC), is the

primary affiliate of Case Western Reserve University (CWRU).

Together, they form the largest center for biomedical research in the

State of Ohio. The System provides the major clinical base for

translational researchers at the Case Research Institute, a

partnership between UHC and CWRU School of Medicine, as well as a

broad and well-characterized patient population for clinical trials

involving the most advanced treatments. Included in UHC are Rainbow

Babies & Children's Hospital, among the nation's best children's

hospitals; Ireland Cancer Center, northern Ohio's only National

Cancer Institute-designated Comprehensive Cancer Center (the nation's

highest designation); and Mac Women's Hospital, Ohio's only

hospital for women.

Founded in 1843, the Case Western Reserve University School of

Medicine is the largest medical research institution in Ohio and the

14th largest among the nation's medical schools for research funding

from the National Institutes of Health. Seven Nobel Laureates have

been affiliated with the school. The School of Medicine is recognized

throughout the international medical community for outstanding

achievements in research, teaching and service. Annually, the School

of Medicine trains more than 600 M.D. and M.D./Ph.D. students.

Founded in 1908, The Children's Hospital of Denver is a private, not-

for-profit pediatric health care network. It is consistently ranked

one of the best children's hospitals in America by U.S. News & World

Report and other publications. With more than 1,116 pediatric

specialists and 2,000 full-time employees, Children's is home to a

number of nationally and internationally recognized medical programs.

Children's provides care at its main campus and through a network

that includes four community-based urgent care sites, seven specialty-

care centers and more than 400 outreach clinics in three states each

year.

The Cystic Fibrosis Foundation was created in 1955 to assure the

development of the means to cure and control CF and to improve the

quality of life for people with the disease. CFFT is the nonprofit

drug development affiliate of the CF Foundation that operates drug

discovery, development and evaluation efforts. Total support of CFFT

is provided by the CF Foundation.

Additional contacts:

Tobin

Director of Media Relations

Cystic Fibrosis Foundation

C. Moen, M.D.

President and CEO

Copernicus Therapeutics, Inc.

Media Relations

The Children's Hospital of Denver

School of Medicine

For more information:

Eileen Korey

Director of News Services

University Hospitals of Cleveland

Stamatis

Director of Public Affairs

Case Western Reserve University

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[ Print This

> Hi all!

>

> I just wanted to share what my family saw on the news the other

> night. It was a Kansas City or Topeka station but they were

reporting

> on a medical procedure elsewhere (I think it was Chicago -- forgive

> me if I'm incorrect). The point is, it was gene therapy on a 26 yr

> old woman with cf. They put the genetic material in her via her

nose

> and it works for 21 days to clear the mucus etc. This is the first

I

> have heard or seen of gene therapy on or for CF patients

specifically

> so I was pretty excited (and so was my father! He's determined that

> there has to be something medically worthwhile out there for his

> grandson!)

>

> Just wanted to share that info. Hope all are having good summers.

> It's uncommonly HOT here in KS. ick!

>

> Crystal

> mom to Adam 17mo wcf, le 3yo nocf.