Re: THANK YOU DAVID!! WORTH MORGAN

[Guest guest]

Hi Barb;

This is not your fault, but I think the correct spelling

of " Remciade " should be " Remicade " [it was incorrectly spelled in

the article about the Worth Foundation!]. For the benefit of

others in the group who may not be familiar with it, Remicade is

a " Tumor Necrosis Factor Blocking Agent " , not without some possible

side-effects:

MMWR Morb Mortal Wkly Rep. 2004 Aug 6;53(30):683-6.

Tuberculosis associated with blocking agents against tumor necrosis

factor-alpha--California, 2002-2003.

Centers for Disease Control and Prevention (CDC).

The Food and Drug Administration (FDA) has determined that

tuberculosis (TB) disease is a potential adverse reaction from

treatment with the tumor necrosis factor-alpha (TNF-alpha)

antagonists infliximab (Remicade), etanercept (Enbrel), and

adalimumab (Humira); the three products are labeled accordingly.

These products work by blocking TNF-alpha, an inflammatory cytokine,

and are approved for treating rheumatoid arthritis and other

selected autoimmune diseases. TNF-alpha is associated with the

immunology and pathophysiology of certain infectious diseases,

notably TB; blocking TNF-alphacan allow TB disease to emerge from

latent Mycobacterium tuberculosis infection. In 2002, a California

county health department reported three cases of TB disease

occurring in association with infliximab therapy. This report

summarizes those cases and nine subsequently reported cases and

provides interim recommendations for TB prevention and management in

recipients of these blocking agents. Health-care providers should

take steps to prevent TB in immunocompromised patients and remain

vigilant for TB as a cause of unexplained febrile illness.

Publication Types:

Case Reports

PMID: 15295313

I've been trying to find out more about Dr. Gene Whitington; his

last published paper seems to have been in 1999:

Tenn Med. 1999 Nov;92(11):411-4.

In-situ split-liver transplantation.

Grewal H, Amiri H, Vera S, Stratta R, Whitington G, Lazar L, Black

D, S, Ammons J, Gaber AO.

Department of Transplant Surgery and Pediatric Gastroenterology,

University of Tennessee, Memphis 38163, USA.

PMID: 10548924

As far as I can tell he has not published on this experimental

therapy for PSC. The only published study on PSC and these types of

agents, is from Dr. Kaplan's group, and the results were somewhat

dissappointing:

Dig Dis Sci. 2004 Jan;49(1):1-4.

A pilot study of etanercept in the treatment of primary sclerosing

cholangitis.

Epstein MP, Kaplan MM.

Division of Gastroenterology, Tufts New England Medical Center,

Boston, Massachusetts 02111, USA.

There is no effective medical treatment for primary sclerosing

cholangitis (PSC), a chronic cholestatic liver disease that usually

progresses to cirrhosis and liver failure. The aim of this study was

to determine the safety and efficacy of etanercept, an inhibitor of

tumor necrosis factor, in the treatment of PSC. Ten patients with

clinically active PSC were studied. All had elevated serum alkaline

phosphatase levels, cholangiograms that were diagnostic of PSC, and

liver histology consistent with PSC. Five patients had elevated

serum bilirubin levels, five had pruritus, eight had failed to

respond to ursodiol and/or methotrexate, and six had rapidly

recurring dominant bile duct strictures. Patients were to receive

etanercept, 25 mg subcutaneously twice weekly, for 6 months if there

were no side effects and for 1 year if there was evidence of

efficacy after 6 months. Biochemical tests of liver function did not

improve in any patient. Mean serum bilirubin levels increased

significantly, from 2.0 to 3.6 mg/dl (P = 0.026). Two of the five

patients with pruritus had resolution of pruritus during treatment

with etanercept, recurrence when etanercept was stopped, and

resolution when it was restarted, although there was little change

in liver enzymes or bilirubin levels. There was no decrease in the

rate of stricture formation and there were no side effects.

Etanercept, at the dosage used, was well tolerated but not effective

in the treatment of PSC. It may be helpful in treating pruritus due

to cholestasis.

Publication Types:

Clinical Trial

PMID: 14992426

Best regards,

Dave

(father of (19); PSC 07/03; UC 08/03)

PRIMARY SCLEROSING CHOLANGITIS LITERATURE

http://home.insightbb.com/~rhodesdavid/

[Guest guest]

Dave , I found this article as well with contact numbers. At this point

is my concern that I never heard of this hospital. Does anyone in the

group know about it?

Lee

Le Bonheur Announces Foundation

For the Study of Rare Liver Disease Primary Sclerosing Cholangitis (PSC)

MEMPHIS, Tenn., Jan. 21 /PRNewswire/ -- Le Bonheur Children's Medical

Center is now the home of the newly formed Musette & , Jr.

Foundation for the Study of Primary Sclerosing Cholangitis. Primary

Sclerosing Cholangitis, or PSC, is a rare liver disease in children

that causes the bile ducts inside and outside the liver to become

inflamed, scarred and blocked. Often the treatment for this

potentially fatal disease is liver transplantation. While waiting for

a liver transplant, children can suffer from severe abdominal pain,

jaundice, weight loss and a profound lack of energy.

The family made a $200,000 donation to launch the foundation and

address their concerns about the lack of information known nationally

about PSC, the lack of research and the lack of consistency in

treatment.

In 1997, pediatric gastroenterologist Dr. Gene Whitington diagnosed the

's 10-year-old son, Worth, with PSC and began treatment of his

disease. Now at age 16, Worth is a six-foot-tall competitive tennis

player and a wide receiver for Memphis University School and has no

symptoms of PSC.

Dr. Whitington, the director of the Foundation and Dr. Dennis Black,

co- director of The Foundation and director of the Children's

Foundation Research Center, are working together on the organization of

the Foundation.

The Foundation is currently working to develop a Web site to provide

information to parents and physicians and has plans to recruit four to

five full-time researchers to study PSC, as well as to fund basic

research and coordinate multi-center clinical studies with other

researchers around the country. For more information about the

Foundation, please contact Dr. Dennis Black at the Children's

Foundation Research Center at Le Bonheur Children's Medical Center at

or email at dblack@... .

" The whole point of research and the Foundation is to bring more

education on this disease and to have physicians come together to help

other children, " said B. , Jr.

" We are grateful to the s for this grant which has allowed us to

set up the foundation for the study of PSC, " said Dr. Gene Whittington.

" We want to look at the basic cause of PSC as well as aggressive new

therapies. We know that 60 percent of patients with PSC will need a

liver transplant in less than 20 years and our goal is to change that.

The 's generosity will give us the opportunities we need to make

strong advances with this disease. "

" With the strong support of the Foundation, we will be able to

target both basic and clinical research to this devastating disease in

a manner that will accelerate the development of new and effective

treatments. This represents a major step forward for a disease that

has, until now, received too little attention and inadequate

resources, " said Dr. Dennis Black, co-director of the Foundation

and Professor of Pediatrics and Physiology at the University of

Tennessee Health Science Center.

Football star Walter Peyton died from PSC. The disease usually begins

between ages 30 and 60, but the disease can also arise during

childhood. PSC is more common in men than women and progresses slowly,

so a person can have the disease for years before symptoms develop.

Founded in 1952, Le Bonheur Children's Medical Center is the

Mid-South's first and only comprehensive pediatric medical facility,

with 225 beds and a medical staff of 500. More than 130,000 children

throughout the region are treated annually on an inpatient or

outpatient basis. Children are referred to Le Bonheur from all over the

United States and throughout the world. For more information, please

call or visit http://www.lebonheur.org/ .

CONTACT:

Anne Glankler

glanklea@...

LBCMC

CONTACT: Anne Glankler of LBCMC, +1-, or

glanklea@... Web site: http://www.lebonheur.org/

Copyright 2004 PRNewswire

Issued: 01/21/2004 02:00 PM GMT