Cynata Therapeutics, an Australian stem cell and regenerative medicine company, has announced positive results from a Phase 1 clinical trial of CYP-001, the CymerusTM mesenchymal stem cell product candidate. The study investigated it as an experimental treatment for steroid-resistant acute graft-versus-host disease. For more detailed information you can view the source press release here, at Globe Newswire.
About Graft-Versus-Host Disease (GvHD)
Graft-versus-host disease (GvHD) is a possible complication after a bone marrow or stem cell transplant from someone else (a donor or allogeneic transplant). GvHD occurs when the graft (donated marrow or stem cells) reacts against the host (the transplant recipient). Typically this happens when a type of white blood cell in the donated graft, called T cells, see the patient’s body cells as foreign and attack them.
GvHD can vary in severity and symptoms, and can be chronic, acute, late acute, or overlap syndrome. Acute GvHD tends to first occur within one hundred days of the transplant, although this is not always the case. Early symptoms of acute GvHD may include a rash on areas such as the palms of hands, soles of feet, ears, and face, and it can be itchy and painful. It may also affect the digestive system and/or liver, which can cause sickness and affect appetite, amongst other symptoms.
Sometimes steroids may be prescribed by a doctor for the treatment of GvHD, however, according to the source press release, these may only be effective in approximately half of patients and new treatment options are needed.
This information on GvHD was sourced from Cancer Research UK.
About the Clinical Trial
The Phase 1 clinical trial was designed to evaluate the safety and efficacy of the investigational drug CYP-001 in patients with steroid-resistant acute GvHD. The results of the study showed that the investigational drug met all the clinical endpoints of the trial.
Patients who took part in the study were divided into two groups; group A received a lower dose level, and group B received a higher dose level. All treated patients were given two infusions.
After 100 days, patients in group A (the lower dose group) found that all eight patients showed an overall response (improvement of GvHD by one or more grades from baseline), and half showed a complete response (the absence of GvHD signs/symptoms). The results were similar in group B (the higher dose group), in which six out of seven participants showed an overall response, and four out of seven showed a complete response. However, patients in group B appeared to respond to the therapy more quickly, with a 57% complete response rate after 28 days, compared to 12.5% in group A.
For more information, you can view the original press release here.
