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Clinical Organ Target - The Human Kidney
The first organ that Bioquark Inc. is focusing on in its clinical development program for BQ-A,
based on a combination of substantial unmet medical need and minimal competitive landscape, is
the human kidney.
While humans can live normally with just one kidney, when the amount of functioning kidney
tissue is greatly diminished by disease or damage, chronic kidney disease will develop leading
towards a progressive loss in function. This loss of kidney function leads to a downward spiral
throughout the body including negative effects on the cardiovascular system, nervous systems,
and endocrine function.
Replacement therapy, in the form of dialysis or kidney transplant, can prolong life, but quality of
life is severely affected, supplies are limited, and costs of long term maintenance are exorbitant.
Additionally, as the kidney is one of the more anatomically complex organs, it has proven
refractory to stem cell-based regenerative techniques to date.
According to the U.S. National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK),
in 2007, over 500,000 Americans alone suffered from End-Stage Renal Disease (ESRD), with over
111,000 new cases diagnosed and 90,000 deaths annually.
Over $30 billion in care was spent on these patients, with 370,000 on dialysis (cost
-$70,000/year/patient), and 17,000 kidney transplants performed (cost - $116,000 year
1; annual cost of immunosuppression - $20,000/pt).
The first organ that Bioquark Inc. is focusing on in its clinical development program for BQ-A,
based on a combination of substantial unmet medical need and minimal competitive landscape, is
the human kidney.
While humans can live normally with just one kidney, when the amount of functioning kidney
tissue is greatly diminished by disease or damage, chronic kidney disease will develop leading
towards a progressive loss in function. This loss of kidney function leads to a downward spiral
throughout the body including negative effects on the cardiovascular system, nervous systems,
and endocrine function.
Replacement therapy, in the form of dialysis or kidney transplant, can prolong life, but quality of
life is severely affected, supplies are limited, and costs of long term maintenance are exorbitant.
Additionally, as the kidney is one of the more anatomically complex organs, it has proven
refractory to stem cell-based regenerative techniques to date.
According to the U.S. National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK),
in 2007, over 500,000 Americans alone suffered from End-Stage Renal Disease (ESRD), with over
111,000 new cases diagnosed and 90,000 deaths annually.
Over $30 billion in care was spent on these patients, with 370,000 on dialysis (cost
-$70,000/year/patient), and 17,000 kidney transplants performed (cost - $116,000 year
1; annual cost of immunosuppression - $20,000/pt).
Kidney Clinical Indication - Focal Segmental Glomerulosclerosis (FSGS)
Focal Segmental Glomerulosclerosis (FSGS), an important cause of kidney failure in adults and
children, is a disorder in which damage occurs to the glomerular epithelial cell (podocyte),
causing leakage of large amounts of protein from the blood into the urine (proteinuria), as well
as scar formation.
Men and boys are affected slightly more often than women and girls, and it also occurs more
frequently in African-Americans. FSGS is often a primary disease, but it can be secondary to
other conditions, and is found in nephropathy associated with HIV infection, heroin abuse, and
obesity.
The goal of therapy is on decreasing proteinuria, slowing disease progression, and preventing
the occurrence of ESRD. Corticosteroids, immunomodulators, and ACE inhibitors / angiotensin
receptor-blockers are mainstays of treatment in patients with FSGS, but used with very limited
success.
Approximately 50% of patients with FSGS progress to ESRD. The prognosis is particular grim in
patients with massive proteinuria, with ESRD occurring within 3 years in the majority of patients.
Thus, there remains significant unmet medical need for a novel therapy to address podocyte
injury, restore glomerular function, induce remission, and prevent the occurrence of ESRD.
Ongoing clinical investigations in patients with FSGS are focused on immunomodulatory therapy
or prevention of fibrosis.
Treatment of FSGS with a novel regenerative biologic agent such as BQ-A, that repairs
and/or regenerates podocytes as demonstrated by complete remission in proteinuria,
would be a significant advance in the standard of medical care.
Focal Segmental Glomerulosclerosis (FSGS), an important cause of kidney failure in adults and
children, is a disorder in which damage occurs to the glomerular epithelial cell (podocyte),
causing leakage of large amounts of protein from the blood into the urine (proteinuria), as well
as scar formation.
Men and boys are affected slightly more often than women and girls, and it also occurs more
frequently in African-Americans. FSGS is often a primary disease, but it can be secondary to
other conditions, and is found in nephropathy associated with HIV infection, heroin abuse, and
obesity.
The goal of therapy is on decreasing proteinuria, slowing disease progression, and preventing
the occurrence of ESRD. Corticosteroids, immunomodulators, and ACE inhibitors / angiotensin
receptor-blockers are mainstays of treatment in patients with FSGS, but used with very limited
success.
Approximately 50% of patients with FSGS progress to ESRD. The prognosis is particular grim in
patients with massive proteinuria, with ESRD occurring within 3 years in the majority of patients.
Thus, there remains significant unmet medical need for a novel therapy to address podocyte
injury, restore glomerular function, induce remission, and prevent the occurrence of ESRD.
Ongoing clinical investigations in patients with FSGS are focused on immunomodulatory therapy
or prevention of fibrosis.
Treatment of FSGS with a novel regenerative biologic agent such as BQ-A, that repairs
and/or regenerates podocytes as demonstrated by complete remission in proteinuria,
would be a significant advance in the standard of medical care.
