According to a story from Financial Buzz, the pharmaceutical company Elsayls Biotech is planning to submit a proposal to the European Medicines Agency (EMA) in order to conduct a clinical trial with pediatric patients. This trial will test inolimomab, a treatment for steroid resistant graft-versus-host disease. The company recently presented preliminary data for the drug, which was available via an early access program in France. In a ten year period, from 2005 to 2015, more than 250 children were treated with inolimomab. The data from this period highlighted the potential of this experimental drug.
Graft-versus-host disease (GvHD) is a potentially deadly complication that can occur after stem cell transplants, particularly from bone marrow. White blood cells from the transplanted tissue identify the host body as a foreign attacker, and therefore begin to attack the recipient’s cells. The disease can compromise the survival of the recipient, and graft-versus-host disease can affect a diverse array of the body’s organ systems, such as the digestive tract, liver, skin, and mucosa. The immune system itself can also become a target. Glucocoritcoids, a type of steroid, are the most common treatment, but they can suppress the immune system to the point that there is an increased risk of infection and, for cancer patients, relapse. In addition, graft-versus-host disease can also develop resistance to steroids, which is why treatments like inolimomab have a lot of potential value. To learn more about graft-versus-host-disease, click here.
Treatment options for children with GvHD are very limited, and this becomes an even greater problem if the case indicates resistance to steroids, which are still the primary treatment route. Without better options, these patients do not have very good prospects for treatment. The proposal suggests a multicentric study, with study centers in North America as well as Europe. It is projected to involved anywhere from 60 to 80 patients without a control group.
