Pediatric Endocrinology Use of Biologic and Chemotherapeutic Agents Pediatric Endocrinology Use of Biologic and Chemotherapeutic Agents

TYPE 1 DIABETES Life long autoimmune condition which can occur in both children and adults This occurs when the body attacks and destroys the cells in the pancreas that make insulin. This process usually begins years before the condition is diagnosed. Since the body requires insulin to use food for energy, insulin replacement is required. When a patient is first diagnosed they often still have some functioning beta cells and therefore researchers are searching for a way to protect the remaining beta cells before the body destroys then and stops producing any insulin.

TYPE 1 DIABETES

STATISTICS 18.2 million people in the US have been diagnosed with diabetes. 5 to 10% of the people have type 1 diabetes About 176,500 people under age 20 have diabetes Approximately one in every 400 to 600 children and adolescents has type 1 diabetes Occurs equally in males and females More common in whites than in nonwhites Relatives of people with type 1 diabetes are at a 10 to 15 % greater risk for developing diabetes

CURRENT TREATMENT Combination of taking insulin everyday along with healthy eating and physical activity The goal is to keep the blood sugar level as close to normal as possible Recent advances include newer types of insulin and creation of insulin pumps both of which allow for more flexibility in eating patterns and lifestyle, as well as fewer injections.

GLUCOSE MONITORING

RITUXIMAB STUDY GOAL: To find out if rituximab can prevent further destruction of beta cells and thereby allow them to continue producing some amount of insulin. Although patients will still require some insulin injections, hopefully their glucose will be easier to control and patients will have lower incidence of long term problems: blindness, kidney failure, nerve damage, stroke or heart attacks.

ELIGIBILITY CRITERIA Diagnosed with type 1 diabetes in the past 3 months Are 8 to 45 years of age Have blood tests that show your body is still making insulin Have antibodies present in blood that are present in people with type 1 diabetes Are willing to use birth control and take urine pregnancy tests at regular intervals (if female and sexually active) Agree to manage your diabetes daily which means checking your blood sugar 3 to 4 times each day and taking multiple insulin injections or using an insulin pump

RITUXIMAB THERAPY Rituximab is given once a week for 4 weeks as an intravenous infusion. After this initial treatment, patients are examined by the MD every three months for the next two years and will periodically have labs drawn.

ABATACEPT STUDY GOAL: To find out if abatacept can prevent further destruction of beta cells and thereby allow them to continue producing some amount of insulin. Although patients will still require some insulin injections, hopefully their glucose will be easier to control and patients will have lower incidence of long term problems: blindness, kidney failure, nerve damage, stroke or heart attacks. This is a double blinded study – 2/3 of patients will receive active drug while 1/3 will receive a placebo.

ELIGIBILITY Between 4 to 45 years of age Diagnosed with type 1 diabetes less than 3 months ago Screening labs to confirm diagnosis and presence of functioning beta cells Agree to manage your diabetes daily which means checking your blood sugar 3 to 4 times each day and taking multiple insulin injections or using an insulin pump

ABATACEPT THERAPY Monthly intravenous infusions over 30 minutes each of abatacept for 2 years. This is followed by addition visits and labs every 6 months for an additional 2 years.

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