Primary Sclerosing Cholangitis Research

[Guest guest]

Dear PSC Support Group Members:

I have heard from a number of you recently in regards to the ending

of the High Dose URSO trial conducted by Dr. Lindor and his colleagues

and your desire to use left over funds from this trial to fund other studies in

PSC. First, let me say that I appreciate you all for taking the time to

write to me and sharing your personal stories. I can only imagine how difficult

it is to either be a person affected by PSC or to have a close family member

who has PSC. I would also like to tell you all that I admire your courage

and your dedication in banding together to collaboratively seek to encourage

more research in PSC.

I am sure that you all are disappointed that Dr. Lindor's high dose

URSO study ended the way it did. We, at the National Institute of

Diabetes and Digestive and Kidney Diseases (NIDDK) are also disappointed.

However, I would like to point out that even " negative " studies have

some positive results and the study is continuing for at least another year,

though the treatment phase has ended. I have had the pleasure of working

with Dr. Lindor and his colleagues from the very beginning of the clinical trial.

Therefore, I can absolutely assure you that he and his co-investigators and the

entire research team did an excellent job of very carefully conducting this

clinical trial. They also had the forethought to collect a wealth of data

and biological specimens during the study that will be used to further the

understanding of PSC -- beyond just answering the question about whether high

dose URSO is beneficial to patients with PSC. While the answer to that

question wasn’t exactly what we hoped, those patients taking high dose

URSO can stop - why take an expensive drug that doesn't appear to be of

benefit? It also is an opportunity for clinicians and researchers to look

for new/different therapies other than URSO.

We at NIDDK share your desire to advance the science for PSC.

Unfortunately, we are limited by rules that govern how we distribute funds that

are appropriated to us by Congress. Decisions about redirecting funds

from one project to another are not a simple task and may not be permissible

because of federal regulations and current laws. But, please be assured

that we at NIDDK will do everything we can to work with Dr. Lindor to channel

the funds allocated for his present study into advancing the science of

PSC. In fact Dr. Lindor and colleagues are continuing to collect data on

all study participants and are very busy analyzing the data and planning

manuscripts and new studies.

One final point that I would like to make to all of you:

research is costly in terms of dollars, time and other resources. It really

requires a concerted effort on the part of researchers, investigators, patient

advocacy groups, industry, the public, people willing to participate in

clinical trials, the federal government, and others to achieve meaningful

results. It takes basic scientists and clinical researchers working

together to advance the science. And it takes people such as all of you

to let your voices be heard to make PSC research a national priority.

Sincerely yours,

R. Robuck, Ph.D., M.P.H.

Director, Clinical Trials Program

Division of Digestive Diseases and Nutrition, NIDDK