RE: Primary Sclerosing Cholangitis Research

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Arne

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Nice letter!

Penny T

From:

[mailto: ] On Behalf

Of Robuck, (NIH/NIDDK) [E]

Sent: Thursday, 3 July 2008 11:17 AM

To:

Subject: Primary Sclerosing Cholangitis Research

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