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UM171: A small molecule with big potential Dr. Guy Sauvageau discovered the molecule named UM171, after optimization of a hit for multiplying primitive CD34+CD45RA- stem cells. The new molecule causes hematopoietic stem cells (HSCs) to expand in greater numbers, while still expressing the CD34+ protein, which allows for more rapid neutrophil/platelet recovery in the clinic. UM171 has been shown to expand CD34+ 100 fold, outperforming all other published strategies.

Expansion of CD34+cells using UM171

Expansion of CD34+cells using UM171

Fed-Batch Culture System: An innovative solution

This innovative bioreactor system incorporates a continuous feeding scheme that dilutes endogenously produced inhibitory factors. The Fed-Batch system enables a 10-fold expansion of HSCs and 50-fold expansion of CD34+ cells. This system, developed by Dr. Peter Zandstra, can be fully integrated into a closed manufacturing system. Together, ExCellThera’s two core technologies offer the ideal process for sourcing and producing HSCs from umbilical cord blood (UCB) for treatment of blood cancers.

CD34+ HSC expansion using Fed-Batch technology

CD34+ HSC expansion using Fed-Batch technology

  • Enables maintenance of HSC in culture
  • Minimizes cost of expansion
  • Integrates a closed culture system

Why Umbilical Cord Blood

Blood cancers, such as acute myeloid leukemia, are commonly treated using HSCs derived from bone marrow transplants. Bone marrow transplants, while effective in many ways, present several challenges to health care providers, including a painful and risky donor process, occurrence of infection after transplant and difficulty finding matches for ethnic minorities. Umbilical cord blood (UCB) provides an ideal source of HSCs that solves these challenges while delivering better clinical outcomes and reduced health care costs. ExCellThera’s technology overcomes the barriers that previously limited the use of UCB as an effective source of HSCs. Our innovative expansion process dramatically increases the number of HSCs per unit, with the outcome that adult transplants can now be achieved with a single UCB unit.


Process for using umbilical cord blood as a source of HSCs for transplant

Treating patients with UCB is more clinically effective and economically efficient. Put simply, ExCellThera makes it easier to bank, store and use HSCs for transplant in blood cancers. This means more patients can be treated with fewer UCB units, while increasing the amount of HSCs administered and decreasing complications before and after transplant. Using UCB as the source for hematopoietic stem cell transplant (HSCT) means:

  • No risk to donor
  • Rapidly available matched blood
  • Human leukocyte antigen (HLA) incompatibilities allowed
  • Reduced risk of graft-versus-host disease
  • Improved accessibility for ethnic minorities
  • Low risk of infection transmission (Cytomegalovirus or CMV)

Therapeutic Areas

Acute Myeloid Leukemia Leukemias are cancers that start in cells that would normally develop into different types of blood cells. Acute means that this leukemia can progress quickly if not treated, and would probably be fatal in a few months. Myeloid refers to the type of cell this leukemia starts from. Acute Myeloid Leukemia (AML) develops when blood stem cells in the bone marrow change and no longer grow or behave normally. It is one of the more common types of leukemia in adults. AML starts in the bone marrow (the soft inner part of certain bones, where new blood cells are made), but in most cases it quickly moves into the blood. It can sometimes spread to other parts of the body including the lymph nodes, liver, spleen, central nervous system (brain and spinal cord), and testicles. Acute Lymphocytic Leukemia Acute lymphocytic leukemia (ALL) is also called acute lymphoblastic leukemia. The term acute means that the leukemia grows quickly, and if not treated, could be fatal in a few months. Lymphocytic means it develops from early forms of lymphocytes, a type of white blood cell. This is different from acute myeloid leukemia (AML), which starts in other blood cell types found in the bone marrow. Non-Hodgkin’s Lymphomas Non-Hodgkin lymphoma (also known as non-Hodgkin’s lymphoma, NHL, or sometimes just lymphoma) is a cancer that starts in cells called lymphocytes, which are part of the body’s immune system. Lymphocytes are in the lymph nodes and other lymphoid tissues (such as the spleen and bone marrow). Lymphoid tissue is found in many places throughout the body; therefore, lymphomas can start almost anywhere. There are over 30 types of non-Hodgkin lymphoma. Myelodysplastic Syndromes Myelodysplastic syndromes (MDS) are a group of diseases in which the bone marrow doesn’t produce enough healthy mature blood cells. The immature blood cells, called blasts, do not function properly and die either in the bone marrow or blood. As a result, there are fewer healthy red blood cells, white blood cells and platelets. MDS can turn into a fast-growing cancer of bone marrow cells called acute myeloid leukemia. This happens in about 1 out of 3 people with MDS. Sources: www.cancer.org and www.cancer.ca