How Many FSGS
Patients are in the U.S.?
Dr. Norman Siegel, former Yale University nephrologist who chaired the steering committee for the five-year, nationwide Clinical Trial for FSGS patients, knew a good deal about FSGS. But in one meeting of his peers, he was thrown a question that even he could not answer.
Dr. Siegel had once told a group of more than 100 experts that the Clinical Trial needs 20 patients enrolled each month in the 150 participating centers throughout the country. At question time, a respected fellow researcher strode to the microphone.
“How many new FSGS patients are there in the U.S. a month?” the expert asked.
“I don’t have the answer to that question,” said Dr. Siegel.
Nor, it seems, does anyone. Getting a handle on the incidence of FSGS in this country has been confounding and frustrating the brightest minds in the field of glomerular injury research. Fighting the condition that injures, scars and destroys the glomerulus (the kidney filtering component) has some researchers shaking their heads.
“Why is it so difficult to get long term studies in glomerular disease?” asked one expert as he presented to 100 or so of his peers at a recent meeting convened by the National Institute of Diabetes and Digestive and Disease of the Kidney (NIDDK) in Bethesda, MD.
The same physician continued his lament in a smaller workshop, asking: “Why is it that we are so far behind? We’ve been a cottage industry, working with different kinds of diseases, using some orphan drugs that have public health concerns, and it has been very disorganized. The result is we have very few drugs for our patients – and one of them is toxic to the kidney.”
There are signs that the lack of organization – and mystery regarding the incidence of FSGS – may eventually be resolved through greater communication and cooperation between the scientists who have in the past been scrutinizing the disease in relative isolation.
At the recent meeting, the experts heard of the experiences of the 19-year-old Glomerular Disease Collaborative Network, which has spread to seven states from its base at the University of North Carolina at Chapel Hill. Susan Hogan, Ph.D., a UNC assistant professor helping lead the project, said it helps connect 300 nephrologists from more than 100 private practices.
The project, which includes patient registries, DNA and RNA studies treatment programs and trials, also may provide at least a small clue toward the answer to that baffling question: How much FSGS in U.S.?
Dr. Hogan explained that the biopsies and data on 10,264 kidney patients are on file with the network. But only 2545 of those patients have given consent to have their specimens further studies.
Of those 2545 patients, 629 have FSGS, she reported. That’s just about 25 percent of all kidney patients who have given consent. One of her colleagues at the network has suggested that the 25 percent figure would probably hold true for all 10,264 patients.
That would be 2566 patients with FSGS in those seven states. However, other experts point out that the network, as good as it is, probably misses many thousands of cases. So the 2566 number for seven southern states remains a tantalizing numerical appetizer.
The good news, says the experts, is that more
and bigger collaborative networks dealing
with glomerular diseases such as FSGS probably
are on the way. To prime that pump,
the recent workshop brought in an expert from
the Southwest Oncology Group, one of the nation’s
largest cooperative cancer groups, to explain
how the organization has attracted participation
by 283 institutions and 4,000 members.
Southwest Oncology Group has 10 disease committees and has enrolled more than 150,000 cancer patients in clinical trials.
Observing such success has fueled the enthusiasm of some gomerular disease experts. One expert reported that some 15 institutions are ready to join a glomerular trials network that would enroll patients, compile detailed data on their conditions, and operate clinical trials to test drugs on the patients.
While the initial effort to obtain NIDDK funding failed, the group (and others like it) are expected to make more attempts at getting off the ground.
One factor working against the formation of such groups are what were described as “hard times at the NIH” (National Institutes of Health) by Josephine Briggs, M.D., director of the NIDDK division that deals with kidney issues.
Dr. Briggs said the federal agency was asked to put together budget requested that ranged from a two percent increase to a two percent decrease.
