Today the results of a four year effort to test the potential effect of an herbal supplement on cystine stone burden were published in the Journal of Urological Research. The study, led by Dr. Stephen Erickson at the Mayo Clinic, evaluated the use of Cystone®, an herbal treatment that had been implicated in a case study in the reduction of stone formation. The results were reported to be negative, with neither a change in urinary composition nor a decrease in stone burden observed in patients receiving Cystone®. While the authors state that "A longer term study with more patients would be necessary to have power to definitively detect changes in stone events or enhanced stone passage", they conclude that "Nevertheless, this short-term trial failed to find evidence that Cystone® decreases kidney stone burden in recurrent cystine stone formers, which might dampen enthusiasm for further study."

The full article is available via Urological Research and as a part of the ICF Research Library.  

Your participation is essential to the improved understanding of this lifelong disease and in the development of new treatment options.

The Rare Kidney Stone Consortium has opened enrollment for the Cystinuria Registry at New York University.  In this registry, patients with cystinuria will be enrolled and information collected about their condition annually. The collected data in this registry will help provide a better understanding of the condition. The goal of this registry is to collect data about this rare disease, that may help to develop better treatment protocols in the future.

The privacy and confidentiality of all information collected in this registry is protected and will be used for research purposes only.

This registry is part of the Rare Kidney Stone Consortium, which in turn is part of the Rare Disease Clinical Research Network. This research is sponsored by the National Institute of Diabetes, Digestive and Kidney Diseases (NIDDK) and the Office of Rare Disease Research (ORDR). The registry was developed in conjunction with several academic institutions and leaders in kidney stone researchworldwide. At NYU, the registry is under the supervision of Dr David Goldfarb and his team.

If you are interested in joining the registry, please visit the official NYU registry page for further information. If you have any questions or concerns, please feel free to contact the team at This e-mail address is being protected from spambots. You need JavaScript enabled to view it .  

http://medicine.med.nyu.edu/nephrology/research/current/join+the+cystinuria+registry

On October 15th, 2010, new research on cystinuria was featured on the cover of one of the world's top academic research journals, Science. The publication, available online, describes the detailed study of cystine crystalization. This unprecedented understanding of the process by which cystine aggregates (partially illustrated in Science's online supplemental video material) allowed the researchers to rationally consider, identify, and test a chemical inhibitor of cystine crystal growth named cystine dimethyl ester (CDME). The group's findings are a significant leap forward in the effort to prevent cystine kidney stones.

The story has been well-covered in an article appearing in the American Chemical Society's Chemical and Engineering News and by a press release from the research group's institution, New York University.

The announcement demonstrating the use of CDME to inhibit cystine crystal growth is exciting both to the research, medical, and patient communities.  Indeed, this new research has shown that CDME is effective in a synthetic environment, making it a potential candidate for use in treating cystine stone formers. However, the road from lab science to pharmaceutical success is a long one, and some discussion of perspective is warranted.

At this stage, this research is essentially a proof of principle demonstrating the feasibility of chemically inhibiting cystine stone growth. A number of complex issues must be investigated before we can begin thinking of this chemical (CDME) or any chemical identified via this novel approach as yielding a viable treatment for cystinuria. Some interrelated key questions, common to all pharmaceutical development, will be focal points of continuing CDME research:

• Drug targeting: How much, if any, CDME would enter the urine, unchanged by human metabolism, where it can potentially inhibit cystine crystal growth?
• Drug efficacy: Are the cystine crystal growth properties observed in the synthetic system (in the “test tube”) applicable in real human being? If so, what range of urinary CDME concentration might be necessary for effective crystal inhibition?
• Toxicity and side effects: Would the prescribed doses necessary to deliver the effective amount of CDME to the urine be well tolerated by patients?

These questions need to be methodically addressed, and that process can require years of research. The good news is that researchers are actively working to get answers. Whether or not CDME has what it takes to be a viable candidate for pharmaceutical consideration is not yet known. However, the newly described method of identifying chemicals which have the ability, at least in the test tube, to inhibit cystine crystallization, opens a pipeline for the identification of additional candidates, increasing the chance of long-term success!