BLADDER DISEASES AFFECT MILLIONS OF PATIENTS ALL OVER THE WORLD
Diseases of the bladder comprise a wide variety of conditions affecting millions of patients worldwide, including over 50 million in the United States alone. Major bladder diseases include bladder cancer, voiding dysfunction, urinary tract infections as well as renal and urinary stones. These diseases affect patients of all ages and have profound impact on their quality of life and overall health.
Bladder over-and under-activity affect nearly 40M Americans.
Voiding dysfunction includes disorders that disrupt the bladder’s ability to easily store and/or excrete urine. The most common dysfunction is overactive bladder (OAB), which affects over 33 million people in the United States, alone, and is usually associated with advancing age1. OAB, generally characterized by urinary urgency, frequency and incontinence, significantly limits patients’ daily activities. OAB can result from an underlying neurologic injury (neurogenic detrusor overactivity, or NDO) or from unclear etiology (idiopathic overactive bladder, or iOAB). In addition to significant loss of patient productivity, billions of dollars are spent annually to treat OAB patients with medications, surgical treatments and behavioral modifications2. There is a significant need for new therapeutics for patients whose symptoms are not sufficiently controlled by existing treatment options.
In addition to OAB, physicians and researchers are coming to recognize bladder underactivity (UAB) as a prevalent and markedly underserved condition. UAB patients often experience a loss of tone or coordination in the bladder wall, resulting in an inability to fully empty the bladder. This can lead to incontinence and urgency associated with overfilling, symptoms which are sometimes confused with OAB. Currently, the only effective management option for this condition is repeated and frequent self-catheterization.
Bladder cancer is the most expensive cancer to treat on a per-patient lifetime basis.
Bladder cancer affects roughly 2.7 million patients worldwide, including nearly 600,000 in the United States1. The National Cancer Institute estimates that there will be a total of 74,000 new cases and 16,000 deaths due to this disease in the year 20152. When measured as a cumulative per patient cost from diagnosis until death, the cost to treat bladder cancer exceeds all other forms of cancer3. The estimated U.S. national expenditure on bladder cancer was $4.3 billion4 in 2014.
There are two primary forms of bladder cancer, non-muscle invasive (NMIBC) and muscle invasive (MIBC) disease. NMIBC accounts for 70 to 80 percent of cases; patients with this form of the disease have tumors in the lining of the interior of the bladder. These patients require repeated instillations of chemotherapeutic or immunological agents, lifelong surveillance and repeated surgical management. Moreover, despite aggressive therapies, many of these tumors will recur and may even progress to the more dangerous muscle invasive form of disease, or MIBC, in which the tumor penetrates into the bladder muscle and potentially, beyond. The standard of care for treatment of MIBC includes radical cystectomy, or complete removal of the bladder, with or without neoadjuvant chemotherapy. Radical cystectomy is a major, life changing procedure, and many patients are medically unfit and/or unwilling to undergo the procedure.
Infections of the urinary tract affect millions of patients, and including tough-to-treat, chronic, recurrent urinary tract infections
Urinary tract infections impact roughly 6.5 million Americans per year. The majority of these patients are women1. These painful events range from relatively common bacterial infections managed by primary care physicians with systemic antibiotics, to infections of the kidneys that may damage these organs and may put patients’ lives at risk. Many patients experience frequent recurrences of these infections, requiring repeated medical visits and causing significant disruptions to quality of life.
A major driver of patient discomfort and healthcare resource utilization.
Renal and ureteral stones affect nearly 2 million Americans per year, cause substantial pain and discomfort, and represent a significant burden on the healthcare system. While many patients will only suffer one or two kidney stones in their lives, some patients form stones repeatedly, secondary to underlying infections (struvite stones) or genetic defects (cysteine stones).
A healthy and normal-functioning bladder is essential to a good quality of life. Any disruption to this system can lead to a rapid deterioration in daily activities at a minimum, and in some instances (e.g. bladder cancer) can cause significant morbidity and mortality. In most cases bladder diseases occur locally and, therefore, should be treated locally to maximize efficacy and minimize systemic side-effects. Though local instillations of drugs are used today for difficult-to-treat patients, their utility is markedly limited by short exposure time in the bladder before they are voided out (approximately 1 hour). Systemic therapies (e.g. oral medications) are more convenient, but often have bothersome side effects and limited efficacy. As a result, a substantial medical need exists for an approach that combines the benefits of local bladder treatment with prolonged drug exposure.