InDepth Pharmaceuticals, founded by Deepak Nihalani, Ph.D., the SmartState Endowed Chair in Renal Disease Biomarkers at the Medical University of South Carolina (MUSC), has taken a vital step toward commercializing an innovative diagnostic technique that could better predict kidney transplant success in patients with focal segmental glomerulosclerosis (FSGS), a kidney disease that often leads to end-stage renal failure. The company has executed an option agreement with the MUSC Foundation for Research Development (FRD), which gives it rights to evaluate the diagnostic technique further, with an eventual opportunity to license it for commercialization. For the evaluation, the company will use funding from a small business technology transfer grant it received from the National Institutes of Health in 2019.
With the option agreement executed, InDepth Pharmaceuticals can now continue to explore the feasibility of putting its novel diagnostic procedure into practice.
Nihalani has dedicated the past fifteen years to the study of kidney disease, with a particular emphasis on FSGS, which has multiple forms. One of those forms can recur within hours to weeks after transplantation, leading to the loss of the donated kidney.
Kidneys operate much like the water filters on top of water pitchers. The body goes through a multitude of processes, producing substances that one needs to live and those that can be toxic. The kidneys filter these toxins out of the blood while producing urine to rid them from the body. FSGS causes the filters within the kidneys, known as glomeruli, to scar and harden, making it difficult for them to eliminate waste products from the body. Eventually, this can lead to end-stage renal disease, leaving a patient with few options other than dialysis or a kidney transplant.
Unfortunately, transplantation will not work for all patients with FSGS. In as many as 30% of these patients, the transplanted kidney will eventually fail. Being able to diagnose which patients are within that 30% is vital.
"There is currently no way of predicting which FSGS patients who receive a kidney transplant will lose kidney function again," said Nihalani.
Not knowing whether their transplanted kidney will eventually fail can be very emotionally taxing for patients with FSGS. Those whose kidneys do fail also face a huge economic burden. These emotional and economic burdens could be avoided if it were possible to predict more conclusively which patients with FSGS would benefit from kidney transplant and which would not. Alternative treatment approaches, including dialysis or even immunotherapy, could then be pursued for patients with FSGS for whom a kidney transplant would have no chance of success.
Some years ago, Nihalani and his colleagues, including Milos N. Budisavljevic, M.D., a professor in the MUSC Division of Nephrology, brainstormed ways to predict transplant success in these patients. They were able to identify three different genes that, when put into stable cell lines, showed increased expression levels when interacting with plasma from patients with recurrent FSGS. Over the past two decades, Budisavljevic and his team had been collecting plasma samples from patients with recurrent FSGS, and these samples provided a perfect testing ground for their diagnostic concept. In a blinded study, they were able to test plasma from patients with FSGS and recurrent FSGS and found that they could diagnose recurrent FSGS with a sensitivity and specificity of over 80%. This procedure functions as a novel noninvasive diagnostic tool to detect recurrent FSGS.
"For diagnostic procedures, we must identify what is causing the disease," explained Nihalani. "This was a dire case where some patients were having two or three kidney transplants and not getting better. Thus, we owed it to them to think outside of the box and come up with a new approach."
To protect and begin to develop this novel technique, Nihalani was able to work with the MUSC FRD to patent the cell lines used for diagnosis. As a result, the startup company InDepth Pharmaceuticals was born. In addition to developing this diagnostic technique for recurrent FSGS, the company is working to come up with new therapeutics for other forms of kidney disease, as well as for liver fibrosis.
With the recent signing of the option agreement, InDepth Pharmaceuticals must now estimate the market value of the diagnostic procedure to acquire more funding. This includes determining the cost of the procedure as well as validating it in a larger patient population.
"There are approximately 1 million patients with FSGS worldwide, and 30% of them will have recurrent FSGS," said Nihalani. "However, we aim to serve all 1 million patients with FSGS, since any of them could potentially have recurrent FSGS."
Unlike water filters on pitchers that can be replaced without repercussions, patients with recurrent FSGS cannot safely replace their kidneys. InDepth Pharmaceuticals aims to get its innovation to the clinic both to enable more preventive treatments for patients with recurrent FSGS and to avoid the loss of precious kidneys.