Final patient receives treatment in stem cell clinical trial for severe blood cancers

MONTREAL, June 27, 2018 (GLOBE NEWSWIRE) -- ExCellThera Inc., a biotechnology company delivering molecules and bioengineering solutions to expand blood stem cells for therapeutic use, announced today the close of its initial, 25-patient clinical trial for its lead technology, ECT-001. The final patient in this phase of the study, led by Dr. Sandra Cohen at the Maisonneuve-Rosemont Hospital in Montreal, received treatment late last week. Preliminary findings from the trial have shown that stem cells expanded with ECT-001 are both safe and feasible.

“We’re very encouraged by what we’ve seen in this trial thus far,” said Dr. Cohen a haematologist and clinical researcher at the Maisonneuve-Rosemont Hospital. “The majority of the 25 patients responded positively to the cell graft and we are seeing reductions in post-transplant complications such as duration of fever and incidence of severe graft versus host disease. As a result, these patients have shorter length of hospital stays when compared to conventional cord blood transplants. We’re optimistic about the longer-term outcomes for these patients and it is important to test ECT-001 in more patients to confirm these promising results.”

Dr. Cohen indicated that the patients from this initial study will continue to be followed for a period of three years.

ECT-001 was used in this Phase I/II multicentre trial to expand blood stem cells from the umbilical cord, which have been shown to be better tolerated by the patient than stem cells from a family member or unrelated donor. This higher tolerance is associated with lower incidence of chronic graft versus host disease, a life-threatening complication in which the grafted stem cells attack the patient’s body. Risk of disease relapse is also less with cord blood. Despite these advantages, use of cord blood under current standards of care has been limited by the low number of stem cells in a single unit. ECT-001 allows safe and rapid expansion of cord blood units to enable rapid blood cell recovery for patients, thereby making cord blood better suited and safer for transplant.

“This is the best possible news for our work with ECT-001,” said Dr. Guy Sauvageau, CEO and Chief Scientific Officer of ExCellThera. “Results from this trial are impressive and will enhance our knowledge about the cell types being expanded in these cultures, including immune cells that may have contributed to these remarkable outcomes. This will help us reach our goal of clinical approval for ECT-001 for the benefit of patients.”

“I am thrilled with the robustness of the cell manufacturing outputs we have seen in this trial. This result provides us with a great foundation to expand our offering in Canada and internationally,” said Dr. Peter Zandstra ExCellThera's Chief Technology Officer.

ExCellThera acknowledges funding support for this trial from the Canadian Cancer Society, Canadian Institutes of Health Research and the Stem Cell Network. Cell manufacturing and additional clinical support were provided by the Center for Commercialisation of Cancer Immunotherapy and the Maisonneuve-Rosemont Hospital.

This was the first of a series of clinical trials using ECT-001 to treat patients with severe blood disorders. A second ongoing Phase I/II clinical trial is using the technology to expand cord blood stem cells for treatment in patients with multiple myeloma. Other multicentre trials using ECT-001 are expected to be announced later this year.

About ECT-001
The ECT-001 technology is a combination of a small molecule, UM171, and an optimized culture system. The technology is capable of expanding the number of therapeutic stem and immune cells in a single unit of cord blood in excess of 35 times in as little as seven days, twice as fast as any other solution. ExCellThera holds the exclusive rights to molecule UM171, initially discovered at the Institute for Research in Immunology and Cancer of the Université de Montréal.

About ExCellThera Inc.
ExCellThera is a biotechnology company delivering molecules and bioengineering solutions to expand blood stem cells for therapeutic use. ExCellThera’s lead technology, ECT-001, combines a proprietary small molecule, UM171, and an optimized culture system. In pursuit of better treatments for patients, the company is building out its portfolio of products, as well as supporting best-in-class clinical trials. 

About the CIUSSS de l’Est-de-l’Île-de-Montréal
The Centre intégré universitaire de santé et de services sociaux de l’Est-de-l’Île-de-Montréal (CIUSSS-Est) includes the Maisonneuve-Rosemont Hospital, the Santa Cabrini Hospital, the Canadian-Polish Welfare Institute, the Institut universitaire en santé mentale de Montréal, as well as the CSSS de Saint-Léonard et Saint-Michel, the CSSS de la Pointe-de-l’Île, and the CSSS Lucille-Teasdale. Affiliated with Université de Montréal, its two major research centres are nationally and internationally recognized for their expertise in mental health, immunology and oncology, vision health, nephrology and cell therapy.


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