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One thing that really frustrates people with diabetes mellitus is the biopharma industry's focus on treatments rather than cures. A cure is what the diabetes community wants, not another band-aid. So the existence of a biopharma company that calls itself "CureDM" is promising, and its first product, Pancreate, seems to be on its way to fulfilling that promise.
CureDM started with the information that in most cases, the mass of pancreatic islets drops by 80 percent in type 1 patients and 50 percent in type 2 patients. They also knew from recent research that the adult human pancreas contains an abundance of pancreatic progenitor cells. Like stem cells, progenitor cells have the capacity to differentiate. Unlike stem cells, however, they are not able to become any type of cell. Instead, they differentiate only into their "target" cell, in this case, islets.
In adults, however, pancreatic progenitor cells rarely make the change into islets. Under normal conditions, islets differentiate only during fetal development, when the pancreas is first powering up. When islets do form in adults, it is usually in response to pancreatic injury and stress.
Scientists knew this fact way back before insulin was discovered, when surgeons performed partial pancreatectomies on children with diabetes in hopes of triggering islet regeneration. Rather than hacking off pieces of pancreases, however, CureDM turned to the modern study of genes, called genomics, and proteins, called proteomics. Using these approaches, they were able to identify the key that unlocks the pancreatic progenitor cells, causing them to differentiate into islets. That key is Human proIslet Peptide (HIP), christened Pancreate by CureDM.
HIP is a peptide, or small piece of a protein, made of 14 amino acids (the building blocks of proteins). It is a segment of a large protein that is created by a gene called regenerating islet-derived 3 alpha, or the REG3a gene. HIP stimulates the pathways that cause adult pancreatic progenitor cells to differentiate into functioning islets, fully equipped with alpha, beta, gamma, and delta cells. Because of the scarcity of the REG3a protein after fetal development, CureDM believed that a lack of HIP was the critical element preventing new islet formation, or neogenesis, in adults.
CureDM discovered that the sequence of amino acids in HIP is very similar among many species. When they made a three-dimensional model of the human REG3a protein, they found the HIP part is exposed on the outer surface of the protein, not folded deep within it, making it available to bind with the other proteins that go on to stimulate islet differentiation. And CureDM has successfully stabilized HIP to improve its availability in the body. Recent studies indicate that the dose of HIP required to stimulate islet neogenesis may be 100 times lower than the concentration required by naturally produced HIP.
So far, HIP has been producing some hopeful results. In cultures of human pancreatic ductal tissue, treatment with HIP increases insulin secretion four-fold. In diabetic mice, it triples the number of islets, essentially reversing the disease. Diabetes-related biomarkers normalize in as few as 10 weeks, and diabetic animals no longer need extra insulin after only 21 days of treatment.
CureDM is currently completing the toxicological studies required before filing an Investigational New Drug application, or IND, for Pancreate. An IND is a request for permission from the FDA to administer an investigational drug to humans. The company expects to begin clinical trials in both type 1 and type 2 diabetes in early 2010.
Patients will probably take Pancreate once or twice daily for a number of months, during which time their insulin injections will be gradually eliminated as Pancreate stimulates islet neogenesis. Type 1 patients would require simultaneous treatment with an immune tolerance agent to protect the new islets from autoimmune attack. Type 2 patients could receive Pancreate along with existing therapies to boost their islet numbers, and patients with pre-diabetes could use Pancreate to alleviate the stress on their existing islets.
In recognition of CureDM's work with HIP, the company recently received the 2009 North American Excellence in Healthcare Product Innovation of the Year Award for diabetes mellitus therapeutics, presented by Frost and Sullivan.
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