The tumor has spread in the brain of P, a 6.5 year old girl. The feeling was that the girl was lost, but one doctor has refused to give up. Prof. Miriam Ben Harush from Rambam has convinced an international pharmaceutical company to give the girl an experimental drug – which saved her life.
Published on July 5, 2014 Yediot Achronot
There are several heroes in this amazing story: a dedicated doctor, who thought “out of the box” and saved a life. Determined parents who refused to give up even when everything seemed to be lost, and an international pharmaceutical company, which made efforts to save the life of one Israeli girl.
The battle for the life of P, a 6.5 year old girl from Haifa, began at the end of last summer. At the end of her first day at school, in the first grade, the girl complained of severe headaches. “We thought that she was excited due to the transition to school. The pediatrician was also not impressed by her symptoms,” her mother tells. But the headaches have not resolved, and in several weeks, the girl has started experiencing photosensitivity. This time she was referred to computerized imaging, which revealed a tumor in her brain. “We hoped that the biopsy taken from the tumor will show that it is benign, but we received terrible news: our daughter was diagnosed with an aggressive and rare brain tumor”, the father recalls.
P underwent surgery for tumor removal and a series of radiations. For a while, it seemed that she is recovering, and she even returned to school. However, in January, 2 months following the completion of radiotherapy, the headaches returned. The physicians performed CT imaging and when they examined it, they were terrified. “The tumor has spread to the brain meninges and along the spinal cord. In this situation, the medicine usually gives up. It has nothing more to offer to the patient,” based on the explanation of Prof. Miriam Ben Harush, Director of the Department of Hemato – Oncology, Mayer Children Hospital at Rambam Medical Center.
“The girl screamed due to pain”.
In March, P’s condition has rapidly deteriorated. She was hospitalized in a life threatening condition. “The girl screamed due to extreme pain which could not be relieved even by morphine. He legs were paralyzed, she could not raise her head, she stopped eating. We were forced to feed her via a tube inserted into her stomach. It was heart breaking to look at her, lying in bed and suffering, so little and helpless”, Ben Harush describes.
P’s parents refused to give up. With the assistance of the expanded family, they did everything they could do to find medical treatment for her. An then, when there was almost nothing else to do, Prof. Ben Harush offered a test which paved the way to effective medication therapy. Based on her recommendation, the girl underwent genetic testing which revealed that in one of her genes, she has a mutation which has been also discovered in patients with melanoma (skin cancer).
Ben Harush turned to a large pharmaceutical company conducting clinical trials with a new drug in patients who carry the same genetic mutation. The doctor hoped that this drug may also relieve the suffering of P. However, the pill developed by the company was too big to swallow, and it was impossible to dissolve it without losing its efficacy.
Having no other choice, the doctor and family members turned to Doron Shalit, CEO of the GSK Israel pharmaceutical company. The company is conducting trials with a new drug named Dabrafenib, intended for adult patients with advanced melanoma. Shalit was requested to find a way to dissolve the drug in order to enable P to receive it via the feeding tube. “Their request was irrational. This was complete gambling. But I have immediately decided that we will cope with the challenge to save the girl’s life”, Shalit recalls.
The CEO delegated the mission to Dr. Einat Dotan, Medical Director of GSK in the field of oncology. “This story has completely shaken me”, Dotan testifies. “From the first moment, I felt that I am professionally and morally obligated to do everything I can to adjust Dabrafenib for the girl. I asked the scientists of the US company to dissolve the drug in a manner that will maintain all its components.”
“We prayed for her to hang on”
However, the challenge was extremely complicated: first, the drug is not intended for treating brain tumors. Second, it has never been tested in children, and it was not clear what would be the dosage suitable for P. This effort has been made under the shadow of a race against the clock, and it was not clear who will win it: science or the angel of death. They all knew that P may die any day.
In parallel, people at Rambam made efforts to receive the approvals required in order to import the experimental drug to Israel and to give it to the dying child. “Each second acted against us, because her condition had extremely deteriorated. We just prayed for P to hang on until the drug will be prepared for her,” Ben Harush said. “We signed the consent for our daughter to receive this experimental drug, we had nothing to lose at this point. The little girl was lost anyway”, the father recalls.
In April, the drug arrived to Rambam. The girl, who was terminally ill, started receiving it twice a day – and then the unbelievable miracle occurred: P. came back to life. “After 3 days, we noticed improvement in her condition. The headaches have gradually decreased and P. started talking again. After some time, she could sit,” Ben Harush told, “Today, 3 months after treatment initiation, the tests show that the tumors in her head have shrunk, and most of the metastases in the spinal cord have disappeared”.
P has recently started solving arithmetic exercises, and she even enjoys the hospital’s children’s center. In the beginning of next week, she is finally expected to return home. The girl will continue taking the drug which saved her life for all her life, at the expense of the pharmaceutical company. “The nightmare we’ve gone through has taught us never to give up”, her parents told yesterday. “We want to tell other parents in such situations: do not give up, do not lose hope”.