INITIAL clinical trial results are indicating mesenchymal stem cell (MSC) infusion is a viable treatment for patients suffering moderate to severe Crohnâs disease who are unresponsive to conventional therapy.
One-third of patients with Crohnâs disease are unresponsive, or develop resistance to, conventional biological treatment, resulting in a need for surgical removal of the bowel.
A phase II clinical trial aimed at establishing preliminary data for the alternative MSC infusion treatment, is being undertaken between several Australian medical institutes including the Cell and Tissue Therapies WA (CTTWA) and the Department of Gastroenterology at the Royal Perth Hospital.
The trial results show a high response rate with a decrease in the symptoms and manifestations of Crohnâs disease.
Out of 15 patients (aged 21â55 years) that completed the trial, 12 demonstrated a response to the alternative treatment and disease remission occurred in eight.
Clinical Professor Richard Herrmann from CTTWA at Royal Perth Hospital says so far 21 patients have been recruited with a goal to reach 30 in total.
In this study, MSCs were isolated from the bone marrow of volunteer donors and prepared for transplantation within a government-funded facility at Royal Perth Hospital.
Cells were injected into the veins of patients weekly for four weeks via infusions of two million cells per kilogram of bodyweight.
Following on from the phase II trial, the group has planned a phase III clinical trial comparing MSC treatment with standard therapy (anti-TNF) in patients with advanced Crohnâs disease.
Long-term efficacy and safety will then need to be addressed in a similar patient group and in those with early-stage disease.
Prof Herrmann says currently research groups do not have the capacity to manufacture stem cells for large-scale commercial use.
âThere is still a need to overcome the technical and commercial challenges of manufacturing MSC preparations, including standardisation and quality control,â he says.
Previously, bone marrow or peripheral blood stem cell transplantation was used to treat active Crohnâs disease and was successful in some patients, but unpleasant chemotherapy (myeloconditioning) was required prior to cell transplantation.
Prof Herrmann says the benefit of using MSCs is that they home to sites of inflammation and have a regulating effect on the immune system.
In addition, they escape immune recognition and therefore transplant rejection; precluding the need for chemotherapeutic marrow conditioning.
The potential use of MSCs in regenerative medicine has been extensively studied over the last 10 years to treat various conditions including graft versus host disease, multiple sclerosis, and diabetes.