An unprecedented protest in the history of the health system in Israel:
about 80 parents of children with the severe and fatal genetic disease Duchenne are threatening to abandon treatment at the Schneider Children's Hospital in Petah Tikva, and to exclude dozens of their children from all the research and medical experiments in which they participate, all because of the parents' claims that Prof. Yoram Nevo, one of the most senior pediatric neurologists in Israel, and until recently the director of the neurological institute at the "Schneider" Children's Hospital in Petach Tikva, supports and influences the general health insurance fund in its resolute opposition to the inclusion of a modern and expensive genetic drug in the drug basket as part of the discussions of the basket committee that have been going on for the past few weeks.
The Duchenne Children - Tzipi Karlik's docu-film (archive) // Mia Tanner
The Genetic Institute at "Schneider" is one of the largest and most central in Israel and treats approximately 9,000 children with neurological disorders, diseases and injuries every year.
Such protest measures by parents in the context of the struggle for medicine basket revenues have not happened in Israel since the Medicine Basket Committee began its deliberations about 25 years ago.
The Schneider Hospital is one of the largest and most important in Israel for the treatment of children, and is fully owned and responsible by the General Hospital Fund, the largest fund in the country whose representatives have a very large influence on the decisions of the Medicines Basket Committee which determines which additional new medicines will enter the basket this year with a budget of approximately NIS 650 million.
The children who are diagnosed with the genetic disease Duchenne suffer from a cumulative and continuous loss of muscle functions, and their condition may reach severe disability and early death at a young age, already in the third decade of their lives, mainly due to heart failure and difficulty breathing.
However, this year marked the breakthrough of the first gene therapy, approved by the American Food and Drug Administration (FDA), which allows the insertion and construction of an alternative and shortened gene into the children's muscle cells, and restores to them the ability to produce a shortened and functional version of the original protein that was damaged due to the disease .
Capricorn and Mitra Rose.
One injection will change a child's life.
Cost: NIS 12 million, photography: none
The treatment is given with only one injection as a one-time treatment, and is approved at this stage only for children aged 4 and 5.
The treatment that allows children for the first time the ability to produce shortened and functional protein in the muscles has been proven in studies as a treatment that preserves and even improves the children's functional abilities, such as walking and standing.
These results may indicate a halt in the deterioration of the disease for many years.
According to estimates, there are about 15 children who are suitable for treatment in Israel.
The current cost in the US of the treatment is approximately 12 million shekels for the one-time treatment for each child, with this cost defining the drug as the most expensive ever submitted to the basket. The gene therapy was developed by the American company Sarpeta and the Swiss pharmaceutical giant Roche entered into a collaboration in the research and marketing of the drug.
In the unprecedented letter of protest that the parents wrote yesterday to Prof. Nebo, with copies to Eli Cohen, CEO of Klalit, and Dr. Efrat Baron Harlev, director of Schneider Hospital, they wrote that "the time has come to stop the experiments on our children and stand behind our demand and All the experts to include in the basket of medicines the groundbreaking genetic therapy for our child.
We turn to you, in these fateful days when the lives of our little children - the 4- and 5-year-old Duchenne children - await the verdict of the Medicine Basket Committee."
Professor Yoram Nevo, photo: Dodi Vakanin
"Who like you knows that these children's time is running out, that in the coming years the steep deterioration will begin, their muscles will deteriorate until they reach a wheelchair and cardiac support, then respiratory support will also come, and the end is known - this is a fatal disease.
And here, for the first time after years of hope, we were informed of a groundbreaking gene therapy approved by the FDA, completely changing the rules of the game, and giving life to our children's muscles.
For us, this is a miracle, albeit a scientific one, but definitely one that saves lives."
"Gene therapy is a breakthrough, a one-time injection that penetrates with a virus that has been emptied and loaded with an alternative and shortened gene into the children's muscle cells. The treatment gives them for the first time the ability to produce a shortened and functional version of the original protein, known as micro-dystrophin. We were full of hope until, to our astonishment, we learned , according to the allegations that were brought before us, that the general representative on the drug basket committee, the one that is supposed to approve funding for the treatment for our child, not only does not support the fact that our children will receive the medicine funded by the state, he is the one who leads the opposition in the committee. This is a treatment that was ranked first by the neurology association Children as the most essential for inclusion in the 2024 drug basket - so how can a Klalit representative oppose it so strongly? But the answer we received completely shocked us, and we hope it is not true that 'Klalit's neurologists do not support the inclusion of the drug in the 2024 basket', as we were told by Some sources we spoke with.'
Demonstration of parents of children with Duchenne disease at Sheba Tel Mehshomer Hospital (archive), photo: Gideon Markowitz
The parents also wrote that "If these things are indeed true, then this is a complete loss of trust on the part of us, the parents, in you, Prof. Nebo, and in your team.
After many years, in which we are full partners in the clinical trials you led and are still leading today on Duchenne children, the time has come to stop the trials on our children, and allow them to receive life-saving medicine now!.
Our children have no time!
The time has come to stand behind the demand of all the experts in the Pediatric Neurology Association (of which you are also a member) and of us parents to include in the medicine basket the groundbreaking genetic therapy for our children - the Duchenne children.
To introduce the first treatment for Duchenne muscular dystrophy into the Israeli medicine basket."
"We are full of hope that these things are not true. Prof. Nebo, we have followed you in many studies that you have led over the years and you are for us and for our children a first-rate professional authority. Our complete loss of trust in you will inevitably lead to the cessation of our children's participation in all the studies that you or the clinic at the Schneider Medical Center leading as well as a general request to transfer the treatment of our children to other medical centers. It is important for us to note that although the genetic therapy is currently only intended for children aged 4 and 5, the letter here is signed by parents of children with Duchenne of all ages."
The Medicine Basket Committee (archive), photo: Yossi Zeliger
"All of us as one as a whole community are mobilized for the inclusion of basket therapy - they and the children we see in the US walking, jumping, playing at the age of 12 and older after receiving the treatment - they are the hope, they are the light of all of us to save all children's lives."
The response of the Schneider Hospital and the General Health Insurance Fund has not yet been given in the matter.
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