The Health Ministry has agreed to let the Sourasky Medical Center in Tel Aviv charge foreign patients for treatment with an experimental drug not approved for use yet anywhere in the world, to treat arterial lateral sclerosis. The payment for use of the experimental therapy is supposed to fund the treatment of Israeli patients and also, treatment with the drug will not be part of the clinical trials it is undergoing.
The treatment is called NurOwn and is being developed by the Israeli firm BrainStorm. It is now undergoing Phase III clinical trials, which examine its medical efficacy. Normally drugs are not given to patients before their safety and efficacy are thoroughly tested, but the Health Ministry has approved limited use: eight Israeli ALS patients may have it, for free, and five patients from abroad may also receive it — but each will pay more than $300,000 for the treatment.
Last year, the Health Ministry had appointed an advisory committee to examine the issue. But the entire committee resigned in May before completing its task or submitting recommendations. Sources close to the issue said that a dispute developed between the committee members and the ministry over charging money for the experimental treatment. Ultimately approval was granted without the committee's input.
Phase 3 clinical trials for the therapy, NurOwn, which uses a patient’s own stem cells to treat ALS, are being done in the United States as of 2017, with 200 patients. Typically for clinical trials, half of the patients receive the treatment and half receive a placebo.
Last year BrainStorm announced that it would allow ALS patients in the U.S. to try the experimental therapy for $300,000 per patient, under the controversial right—to—try law the U.S. passed in May.
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But the company backtracked after taking intense fire over that announcement. Regarding the present case, the company says the project is a not-for-profit humanitarian one. "The company’s previous trials included one-time injections of the cells and showed potential efficacy, which in some patients manifested itself in improved functioning. In light of the positive results we have moved to phase III with FDA approval, which is now underway, in which patients receive three injections of stem cells at intervals of two months. Since the trial is not in Israel, and following requests from many patients in Israel, BrainStorm agreed to try to make the treatment accessible outside of the trial to Israeli patients.”
According to the Health Ministry’s original approval, BrainStorm said, the treatment was to have been given to 10 patients for a fee, two of whom were to be foreigners and eight Israelis. “Because BrainStorm wants to make the treatment accessible to Israelis at no cost, and such a program requires funding, we asked for approval to treat eight Israeli patients for free and five wealthy foreigners who would be charged a fee that would also cover the treatment for the Israelis. To the best of our knowledge, such approval in principle has been given and we are still waiting for final approval. BrainStorm is a public company that operates and will always operate only within the law, in accordance with the required ethical rules.”
It was in February a year ago that the Health Ministry empaneled the committee to discuss allowing use of a treatment still undergoing clinical trials. The committee included senior doctors and legal experts but no ethics expert. Sourasky made its request while the committee was still deliberating but the disagreements that developed led the committee to disband, leaving no minutes of its meetings, let alone recommendations.
According to a source involved in the process, the discord between the committee and the ministry revolved around the number of patients to be eligible for the treatment. Sourasky and BrainStorm sought approval for 20 patients – 10 Israelis to be treated for free and 10 foreigners who would pay (to fund the treatment for the Israelis). The sources said the committee thought eight Israelis and two foreigners should be treated. Sourasky and BrainStorm then suggested a compromise – eight Israelis treated for free, and five paying foreigners, which the committee rejected, but the Health Ministry accepted. That was the reason the committee members resigned, according to the source.
A month later the Health Ministry gave Sourasky approval in principle to begin the experimental therapy on eight Israelis and five foreigners, and charge the foreign patients for it.
So far the drug has undergone Phase II testing, completed in mid—2016, for safety and efficacy in 48 subjects, 36 of whom received the treatment and the rest, as a control group, received a placebo. At the end of the study, BrainStorm reported that 40 percent of the patients showed a 50 percent improvement 12 weeks after the stem cells had been injected.
The technology is based on research by Tel Aviv University professors Daniel Offen and Eldad Melamed and uses stem cells harvested from the patient’s own bone marrow (autologous transfer). The cells are isolated, propagated in the lab and appropriate ones are injected back into the patient and excrete large quantities of proteins, which are responsible for the growth and survival of nerve cells and their interaction between nerves and muscles. The treatment aims to slow the degeneration of brain tissue.
Some European nations enable drugs that haven't been approved for general use, under special circumstances, and since last year, the U.S. has followed suit with the right—to—try law. President Donald Trump signed a law allowing terminally ill patients who have exhausted all known medical options to avail themselves of unapproved treatments if these treatments have passed their first phase of clinical trials. The law sparked controversy and many ethical questions, which arose because the companies developing the drugs are allowed to select the patients to be treated, and since no restrictions have been placed on how much they can charge.
Since September 2016, use approved by the Health Ministry of drugs not fully tested yet has been legal in Israel as well.
But selling these last—ditch therapies enables wealthy patients to ensure they get the drug without participating in medical trials that could risk winding up with placebo, an expert in stem cell therapy told Haaretz. “We fear that this route will become the preferred route of the companies and will become a parallel and less strict road to marketing drugs and therapies."
Another source said charging patients for such treatment was cynical exploitation of anxious sick people and their families.
The Health Ministry stated that Sourasky has not yet received the final word on the project , but remains is conditional on the hospital completing procedures. It agrees that the hospital proceed with the said limited number of patients and pointed out that the law does not prohibit charging patients for such treatment. The ministry added that its approval didn't require the okay from the defunct advisory committee in any case, only the hospital’s Helsinki Committee (which reviews proposals for clinical human trials), and that approval had been obtained.