What is Langerhans Cell Histiocytosis (LCH)?
LCH is a rare blood cancer that occurs in approximately 3 – 9 cases per million children <15 years of age. Although LCH can occur at any age, including in newborns and adults, it is most prevalent in toddlers 1-3 years old.
LCH is caused by an overaccumulation of Langerhans cells, a type of white blood cell that normally helps fight against infection. In LCH, immature Langerhans cells grow and divide uncontrollably, building up in different locations throughout the body where they can form tumors or damage organs, including the skin, brain, liver, and bones. Complications of LCH are numerous and include musculoskeletal disability, retardation of growth, diabetes, hearing impairment, pulmonary impairment, mood disturbance, skin scarring, and liver cirrhosis. Conditions such as these have devastating, life-long impacts on the quality of life of affected children and their families.
The Stella Hope Fund is committed to raising $2 million to support a clinical trial for a new therapeutic approach to treating LCH. Since 2019, the Stella Hope Fund has raised more than $500,000 toward this goal - a major milestone. Help us reach $2 million to find a cure for LCH!
We believe finding a new, more effective, and less toxic treatment for LCH is possible. Your donation can make this a reality.
Dr. Barbara Degar and Dr. JonathanPaolino
Treatments for LCH vary widely depending on how many and which organs are affected. Frontline care can include surgery, radiation, chemotherapy, and other medications…all bringing with them side-effects that are particularly debilitating for children. While chemotherapy is often effective for LCH, children treated with chemotherapy experience significant side effects. Additionally, some children do not respond to treatment, and many relapse once treatment is stopped.
A team of leading doctors at the Dana Farber Cancer Institute (DFCI), spearheaded by Drs. Barbara Degar, Jonathan Paolino and Barrett Rollins, have identified a family of proteins inside Langerhans cells that they can ‘target’ with a drug that has already shown remarkable activity against certain leukemias with very mild side effects. They have evidence that targeting these proteins with a ‘rationally-designed’ drug will cause Langerhans cells to die with fewer side effects than current treatments.