Gamida Cell - Cord Blood Transplant

Clinical Need

Blood Cancers like leukemia and lymphoma

According to the World Marrow Donor Association (WMDA),   annually, there are approximately 60,000 blood cancer patients indicated and eligible for blood stem cell transplantation.  Of this, only 15% will find a matched family related donor; the rest, around 50,000 patients, start a formal search for a matched unrelated donor.  The lack of a suitable donor and the prolonged search results in a transplantation of only approximately 18,000 of these patients (36%).

The Center for International Blood and Marrow Transplant Research (CIBMTR), reports that the number of transplants from unrelated donors has increased in the last 10 years by approximately 7.7% in the US.  Of the patients who receive a blood stem cell transplantation, only approximately 3,000 are transplanted with blood stem cells sourced from umbilical cord blood, most of them are children.  For more than 20 years umbilical cord blood has been an accepted alternative to a bone marrow graft. Umbilical cord blood has two major advantages: (1) it does not require a full tissue matching between the patient and the umbilical cord blood, therefore most of the patients who were not successful in finding a fully matched donor will find at least one unit of umbilical cord blood that matches them well enough; and (2), umbilical cord blood is a readily available tissue, with more than 700,000 units cryopreserved in public cord blood banks worldwide. The limitation in using umbilical cord blood is associated with the fact that there are not enough blood stem cells in a unit of umbilical cord blood to effectively treat adolescents and adults who have a larger body weight. The challenge, therefore, is to increase the number of stem and progenitor cells in a unit of umbilical cord blood such that it will become a good graft option for adolescents and adults.  The accepted approach to overcome this challenge has been to expand (increase) the number of stem cells. The problem: it is very difficult to expand cells without changing their properties, which can lead to loss of functionality and delayed engraftment, associated with longer hospitalization stays and increased morbidity and mortality.

Gamida Cell has been able to effectively expand the number of stem cells while preserving their initial properties and functionality to create a therapeutic product for transplantation in patients with blood cancers such as leukemia and lymphoma.  The product, called NiCord, is currently being tested in clinical trials in patients with leukemia and lymphoma. It is intended to provide a transplantation solution for patients who do not have a fully matched donor.

Non-malignant Diseases: SCD and Thalassemia

Sickle cell disease (SCD) is a rare genetic disease, affecting approximately 100,000 patients in the United States. SCD is a group of hereditary, recessive blood disorders  where a person’s red blood cells become rigid and sickle-shaped. Sickled cells break down sooner and block blood vessels. Symptoms range in type and severity such as pain and organ damage.  SCD patients have a relatively low life expectancy. The cost of medical treatment of SCD patients during their lifetime is approximately $9 million in the US (cdc.gov).

To date, other than symptomatic treatments, the cure for a SCD patient is stem cell transplantation.  However, HSC (blood stem cell) transplantation is used in rare cases when patients have a healthy, fully matched family related donor. Matched unrelated donors are almost never available to this patient population. Due to the nature of SCD, there is a high rate of engraftment failure, meaning the transplant isn’t successful. HSC transplantation from umbilical cord blood has also been unsuccessful to date, with a more than 50% rate of engraftment failure, mainly because there just aren’t enough stem cells in a unit of umbilical cord blood to make it work.   As a result, only approximately 450 transplants have been reported in the past 30 years.

Thalassemia is another genetic disease where the body either partly or completely suppresses the normal production of hemoglobin (a protein in red blood cells that carries oxygen). About 60,000 symptomatic individuals, on average 1 in 100,000 people, are born annually with β thalassemia, mostly in the developing world.  Thalassemia (beta) is prevalent in Mediterranean countries, the Middle East, Central Asia, India, Southern China, and the Far East as well as countries along the north coast of Africa and in South America.

 

 

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