Newfound Hope with CAR-T Treatment – Mylène’s Heartwarming Story
In January 2016, a 15-year-old Swiss girl named Mylène was diagnosed with acute lymphoblastic leukemia (ALL). The shock and sadness of this leukemia diagnosis was only the beginning of a heartwarming story full of hope, setbacks, and compassion. After a course of chemotherapy lasting five months at Lucerne State Hospital, Mylène’s leukemia relapsed early in July 2016. It then became clear that Mylène had a very resistant type of leukemia with a poor prognosis.
Next, doctors referred Mylène to the University Children’s Hospital in Zurich for treatment with high-dose chemotherapy. Prof. Dr. Jean-Pierre Bourquin, a leading Swiss expert in pediatric leukemia, took charge of Mylène’s medical treatment. But even high-dose chemotherapy could not bring Mylène into complete remission, and her disease took hold again.
Blinatumomab, a relatively new biopharmaceutical T-cell engager, in combination with a successful bone marrow transplant finally helped Mylène accomplish complete remission in December 2016. By Christmas Eve of 2016, Mylène was full of hope. Unfortunately, Mylène had the fourth relapse of her leukemia only three months later.
Mylène was treated again with two cycles of blinatumomab, resulting in negative residual disease. Again, Mylène suffered a fourth relapse, though this time her leukemia was extramedullary, meaning clusters of abnormal cells were found outside her bone marrow. This was a worst-case scenario. Dr. Bourquin immediately contacted his colleagues, Prof. Shai Izraeli and Dr. Elad Jacoby, at Sheba Medical Center in Tel Aviv, Israel.
New Therapy, New Hope
The last chance for Mylène was the pioneering new immunotherapy called CAR-T (chimeric antigen receptor). Unfortunately, this ground-breaking therapy was not available in Switzerland. Furthermore, Mylène did not have access to promising clinical studies using treatments like Kymriah from Novartis, and CAR-T therapy in the United States is very expensive at around half a million dollars.
CAR engineered T-cells were originally invented by Prof. Dr. Zelig Eshhar from the Weizmann Institute of Science in Rehovot, Israel. Decades later, several institutions in the United States established CAR-T as a therapy for ALL.
Dr. Elad Jacoby, trained at the National Cancer Institute in the US, established a CAR-T program for ALL in Israel. Since 2016, the Pediatric Hemato-Oncology Department at Sheba Medical Center has treated 12 cases successfully with CAR-T therapy.
Arriving at Sheba
Mylène’s situation was now a race against time. Her entire family prepared for her stay and treatment in Israel. Finally, Mylène and her parents flew to Tel Aviv. They arrived at Sheba on May 31, 2017, and Mylène’s parents arranged hotel accommodations for themselves nearby.
Mylène had a difficult beginning at Sheba as she had intense abdominal pain. Around midnight, she went to the emergency room at Sheba’s Safra Children’s Hospital and was admitted to the Pediatric Hemato-Oncology Department for a few days. Finally, her abdominal pain abated after the interdisciplinary efforts of Sheba’s gastroenterologists and oncologists.
At first, Mylène did not have sufficient T-cell levels to begin CAR-T therapy. Waiting for her T-cell amounts to rise was one of the most difficult parts of her treatment course.
The Start of CAR-T Therapy
Finally, on June 12, Mylène’s T-cells were extracted. On-campus laboratory personal engineered and enhanced her T-cells with specific chimeric antigen receptors so that these T-cells could be directed against the leukemia. On June 22, Mylène received 35 million CAR-T cells.
Following the introduction of the CAR-T cells, Mylène experienced moderate cytokine release syndrome with high fever, chills, back pain, and neurologic symptoms such as tremor and seizure. Fortunately, she did not need intensive care.
Mylène’s family relates, “Without Dr. Elad Jacoby and the volunteer medical staff, Zeev Greenberg and Hasia Wolpin, and our medical coordinator, Yana Boguslavsky, we never would have survived this turning point in our life.”
A Happy Ending
Doctors treated Mylène for seven weeks at Sheba Medical Center but eventually overcame her complicated course of progressive resistant leukemia.
CAR-T therapy at Sheba made the difference for Mylène’s health and survival. Mylène and her family left Sheba for Switzerland on July 22. Medical investigations by PCR and PET MR at University Children’s Hospital Zurich confirmed the success of the CAR-T treatment at Sheba. CAR-T cells eliminated leukemia in Mylène’s bone marrow, central nervous system, and extramedullary lesions, giving this heartwarming story a happy ending.
Mylène subsequently had a second bone marrow transplant in Zurich. Currently, she is doing well and only needs a weekly consultation at University Children’s Hospital Zurich.
A Shining Endorsement
Mylène’s family speaks about their experience at Sheba: “We can strongly recommend Sheba Medical Center to people who find themselves or their loved ones needing medical treatment. The medical staff in the Pediatric Hemato-Oncology Department was competent and fluent in English. With the support of our compassionate medical coordinator, Yana, we managed visits to the Day Care Center. We were impressed by the volunteers providing us with food, beverages, and sweets in the Pediatric Hemato-Oncology Department and Day Care Center.
Both medical competency and compassion abound at Sheba.”