Montreal, March 12, 2020 – Capping a medical adventure that began in 1993, CHU Sainte-Justine children's hospital has successfully performed its 1,000th bone-marrow transplant, on a 12-year-old girl.

The important milestone marks a highpoint in the evolution of treatment methods and the use of transplantation at the Université de Montréal-affiliated hospital to address the scourge of childhood cancers. 

"Transplantation was originally a very new technique, and the constant hard work of our teams to perfect it and improve its effectiveness for the well-being of young patients has paid off, and we are very proud of this achievement", said Caroline Barbir, president and CEO of CHU Sainte-Justine

"As we celebrate our 1,000th transplant – a young girl with a red blood cell disorder who received bone marrow from her little brother – we give thanks for how far we have come since 1993," Barbir said.

"We would like to thank our patients' families for their trust, our care teams for their dedication throughout Sainte-Justine and its supporting foundations, and to Quebec in general for people's commitment to the best possible care for children."

27 years and counting

Over its 27-year history, the transplant program at CHU Sainte-Justine has undergone profound changes that have transformed the care of children and the promise of a better life through transplantation.

In its first two years, the program was limited to autologous transplants, using marrow from the patient's body. Then, in 1995, came the first transplants from compatible siblings, followed in 1996 by the first transplant using an internationally registered donor.

For a quarter of a century, the search for donors suitable for all patients – and indeed, the possibility of a universal donor – has become the goal of doctors and families, with promising results.

Through international mobilization, donor registries have been greatly enriched with people from diverse backgrounds, increasing the chances of finding a donor to almost 90 per cent in the case of Caucasians and almost 50 per cent for people from other backgrounds.

At the same time, medicine has made tremendous progress in the use of otherwise  incompatible donors. For example, it has become possible to use blood from the placenta of newborns, which is usually discarded with the placenta after birth, to perform marrow transplants.

CHU Sainte-Justine has always been at the forefront of the quest for donors. The first cord-blood transplant took place there in 1997 and the procedure remained important until around 2010, when transplants from international donors improved and gained priority. The first parent-to-child transplant took place in 2001, but the method was rarely used until 2016, when new transplant protocols made it much more reliable.

Hope for a cure

For a long time, hope for a transplant and hope for a cure co-existed but never coalesced. For children with cancer or leukemia – half of all child transplant recipients – the hope was overshadowed by the very real fear that the disease would return after the transplant.

There was also the danger that the transplant itself would be life-threatening, due to the toxicity of the chemotherapies or radiotherapies given to prepare for the transplant, or to infectious complications during the first weeks before immune defences were restored.

In the early years of the marrow transplant program at Sainte-Justine, the chances of survival and cure were only 40 to 50 per cent. Although these numbers were daunting, transplantation represented the only hope for survival for these patients.

Over the past 25 years, several developments have improved the outcomes for children who receive transplants.

• In 2002, the first donor lymphocyte infusion was performed at Sainte-Justine to fight cancer relapses after transplantation.
• In 2010, the first treatments of the graft-versus-host-disease using mesenchymal stem cells were successful at Sainte-Justine. This changed the prognosis for the survival of children with severe forms of this complication.
• In 2011, new transplant preparation protocols with reduced doses of chemotherapy were introduced for children who underwent transplants for non-cancerous diseases.
• In 2012, Sainte Justine implemented a new technology for the early detection of leukemia relapses after transplantation, to detect relapses as early as possible and to be able to treat them by activating the transplant's immune system against leukemia cells.
• Meanwhile, new treatments to better detect and treat infections have been developed.

All of these improvements, combined with the experience of caregivers, have greatly reduced the risk of death from transplant complications to 10 per cent or less for cancerous diseases and 5 per cent or less for non-cancerous diseases. Nevertheless, the risk of the cancer returning is still very present. One in three children undergoing transplants for leukemia or cancer will ultimately pass away because of relapse after transplantation.

Throughout these years, the children and their families have never had to face their ordeals alone. They have always been supported by the unfailing generosity of the people of Quebec through the Charles Bruneau Foundation, the Sainte-Justine Foundation and the Leucan Foundation.

A new chapter in therapies

In 2015, the old dream of being able to use immune cells to attack cancer cells without attacking the patient's healthy cells became a reality thanks to chimeric antigen receptor T-cell (or CAR T-cell) therapy, which consists of introducing into immune cells (T-cells) a new gene that will give these cells the ability to recognize and attack cancer cells specifically.

This therapy is currently only available for patients with B lymphoblastic leukemia, but it will someday be possible for such CAR therapies to be used for many different types of cancer. As of 2019, it is now possible to virtually eliminate the risk of graft-versus-host disease. Lastly, for certain non-cancerous blood diseases, the arrival of gene therapies offers renewed hope.

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