Cord blood in patients with blood cancer has been shown to be a viable alternative source of hematopoietic stem cell transplantation. However, not unlimited numbers of useful cells and slow immune reconstitution should be considered, which may pose a risk to patients infection.
Researchers at the University of Montreal identified a small molecule drug that could help increase stem cell counts in umbilical cord blood. Early Clinical Data published in The Lancet Hematology shows rapid expansion of blood cells and a low complication rate.
Montreal-based biotech company ExCellThera, founded by senior study author Guy Sauvageau, MD, Ph.D, holds a license to the drug and has received an FDA award for novel therapies for regenerative medicine equipped studies in Canada and America with patients at high risk of leukemia.
Stem cells harvested for transplantation in blood disorders currently come mainly from the blood itself or from the bone marrow, although umbilical cord blood offers benefits. "Although the rate of graft-versus-host disease (GVHD) is low when using cord blood stem cells, these cells are rarely used because the umbilical cords are small and do not contain enough cells to treat an adult." explained Sauvageau in a statement.
In this study, UM171 (ECT-001) was used to culture umbilical cord blood before it was infused into 22 patients with blood cancer Some had previously attempted unsuccessful stem cell transplantation trials He hopes that the expanded UM171 blood cells will quickly become established. During a median follow-up period of 18 months, none of the patients developed severe chronic GVHD, and despite the high risk, only 1 patient died due to treatment-related bleeding.
"The most impressive result is the low mortality rate of UM171 transplants compared to conventional umbilical cord transplants," said co-senior author Sandra Cohen, Ph.D., in a statement.
"Not a single patient required immunosuppression at 13 months, whereas in normal transplants, 50% of patients needed such treatment at that time," added Sauvageau. "No other biotechnological process has yielded such results."
According to the researchers, UM171 caused stem cells to multiply 30-fold on average in just 7 days and increase the number of major immune cells in the transplant blood, such as antigen-presenting dendritic cells and mastocytes.
Stem cell transplantation is promising for cancer patients, although harvesting cells can be difficult and sometimes complications such as GVHD and infection can pose a serious risk. Therefore, scientists are working to find ways to improve the efficiency of the treatment. Previously programmed cell death was prevented in donated hematopoietic stem cells via the blocking of BIM and BMF proteins for a short period of time, as described in a study published in the Journal of Experimental Medicine .
Another study published in the journal Stem Cell Reports of the University of California revealed that Viagra may assist blood stem cells in bone marrow removal Mozobil could reduce the number of stem cells involved in the bloodstream reach, significantly increase.
Umbilical cord transplantation has the advantage over other transplantation methods that it is available to patients who do not have a matching donor. The UM171 may offer some additional benefits, such as: Facilitating rapid and permanent transplantation, speeding up the recovery of the immune system, and the added benefit of a lower mortality rate.
As the stem cell count is increased, the candidate can also help to increase the number of umbilical cords that can be used to benefit even more patients. "While only 5% of umbilical cords can be used with the conventional method, our method increases this proportion to 50%", the researchers found. "This allows us to increase the availability of genetically compatible grafts by 50% to 80%."
"We will soon conduct a Phase 3 clinical trial, hoping to obtain approval from the Food and Drug Administration, which would then make this promising treatment available to the population as soon as possible" Dr. Sauvageau and dr. Cohen.