Cold agglutinin disease (CAD) occurs when the body’s antibodies destroy their own red blood cells. It is a form of hemolytic anemia, meaning the body can’t produce enough blood cells fast enough to make up for the ones being killed. About 10,000 people across the U.S. and Europe are diagnosed with the disease.
CAD is chronic and there are currently no approved treatments for the condition. Many patients attempt to manage the disease through blood transfusions. Some also use rituximab with or without chemo, however it has very limited success.
Thankfully, researchers are hopeful that they have found a new effective treatment for CAD.
The Drug
The drug is called sutimlimab, a C1s inhibitor and it has shown extremely positive results for CAD patients.
To examine the drug, a study was composed of 10 patients who were between the ages of 56 and 76. All of the participants had previously undergone various unsuccessful treatments for their condition. These individuals all had low levels of hemoglobin, an essential component of red blood cells responsible for delivering oxygen to vital cells and organs. 6 of the individuals in the study were undergoing blood transfusions regularly.
After just one week of treatment with sutimlimab, patients experienced a significant increase in hemoglobin and a complete halt in the destruction of red blood cells. Within 6 weeks, four of the patients achieved completely normal levels of hemoglobin. When researchers stopped the treatment, the destruction of cells resumed and hemoglobin levels dropped. The drug was then given again and these symptoms reversed. Throughout 18 months of treatment, the 6 patients who had previously been reliant on blood transfusions did not require a single one.
The positive effects of sutimlimab were evident quickly for patients and the drug had no serious adverse effects. The FDA has already given the drug Breakthrough Therapy Designation.
The findings of this study were published in Blood, which is the most frequently cited peer-reviewed journal within the field.The American Society of Hematology, the largest professional society in the world for hematology, established the journal as part of their efforts to support the advancement of research for blood disorders.
Phase 3 studies
Following the positive results from this study, two Phase 3 clinical trials were initiated. Both Phase 3 studies are currently going on, aiming to confirm the safety and efficacy of sutimlimab. One of these trials is still enrolling patients.
The first is examining the effect of the drug in patients who do not have a recent history of blood transfusions. It is currently recruiting and you can find more details on it here.
The second is examining the drug for patients who do have a recent history of blood transfusions. You can read more about this study here.
If these Phase 3 trials reiterate the same positive results seen in the earlier study, this may be the first approved therapy for cold agglutinin disease!
You can read more about these Phase 3 trials and previous research of sutimlimab here!
