With support from The Hartwell Foundation, I will design new anti-inflammatory molecules to treat Crohn’s Disease in children. While its cause is unknown, the symptoms of Crohn’s Disease include chronic swelling and irritation (inflammation) of the intestines. Approximately 80,000 children are affected by this disease, and numbers are increasing yearly. Direct medical costs range from $11-$28 billion annually within the United States. Treatments range from oral medications to injectable agents that circulate widely throughout the body. When these treatments eventually fail, surgery is performed to remove parts of the diseased intestine, causing multiple clinical issues. These include the inability to absorb nutrients from foods, growth delays, bone brittleness, and numerous other morbidities. More than two-thirds (70%) of those with Crohn’s Disease will require surgery during their lifetime, drastically affecting quality of life. Pervasive challenges include depression, negative body image, social stigma, and a negative impact on family lifestyle. The current battery of injectable drugs has multiple side effects, including an increased risk of cancer. Moreover, treatments for children with Crohn’s Disease are based on the adult population. The important unmet need is for safe and effective treatments that specifically meet the needs of pediatric patients. I have recently developed anti-inflammatory molecules that improve wound healing. I aim to adapt these to develop effective new treatments for children with Crohn’s Disease. My proposed innovation is to engineer select anti-inflammatory agents packed together closely on the molecules’ surface. These molecules will specifically target inflamed areas in the intestinal tract so that a high proportion of the treatment goes directly to the affected area. This approach will provide much greater coverage than oral medications. Moreover, the anti-inflammatory molecules will disrupt the pathways that lead to intestinal inflammation. These molecules are non-toxic, so they avoid the harmful side effects of current treatments. My approach represents a novel and highly innovative platform to deliver agents to combat short- and long-term inflammation in pediatric Crohn’s Disease patients. If successful, I will have created a highly translatable treatment option for pediatric Crohn’s Disease patients. This will manifest itself in the form of either direct application of the molecules to the point of tissue injury, targeted system-wide delivery of these anti-inflammatory molecules, or both. This will be achieved through a pre-clinical animal model (termed the SAMP1/YitFcsJ mouse model) of Crohn’s Disease, which is very similar to its human counterpart and can be manipulated to mimic the pediatric condition
|Effective start/end date||4/1/16 → 3/31/19|
- Hartwell Foundation (Correspon. 04/01/2016)
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