Russian Minister of Health Mikhail Murashko announced the creation of a Russian analogue of the drug for the treatment of spinal muscular atrophy (SMA) Zolgensma, which until 2023 was the most expensive drug in the world, and which was denied to Russian children due to an unspoken ban by the Ministry of Health. SMA is a rare genetic disease that affects the motor neurons of the spinal cord, leading to muscle atrophy and paralysis. In Russia, dozens of children received refusals from the medical commission to purchase Zolgensma, even if the funds for the drug were collected by philanthropists, and not allocated by the state. According to Murashko, the domestic analogue of the drug is now undergoing clinical trials: several children received the first injections. The minister did not specify the specific number, as well as information about the children on whom the tests are being carried out. According to media reports , we are talking about a drug with the working name Bluebell from BIOCAD, information about the trials is in the public domain, but their results are also not disclosed.
According to neurologist Alexander Kurmyshkin, who is a confidant of a large number of families whose members live with SMA, not a single family from the Russian Federation sent their child to the drug trials. His words are quoted by Sota. The neurologist clarified that children from countries where the Zolgensma drug is not registered or its use in the country “is associated with obstacles” take part in the trials. As an example, he cited the annexed territories of Ukraine. In 2021, Kurmyshkin attributed the reluctance to purchase Zolgensma and the delay in registering the drug to the fact that the Russian BIOCAD is developing a domestic drug for the treatment of SMA.
The Insider asked experts to explain whether the domestic analogue differs from the expensive foreign drug and what they think about trials on children. They clarified that there is not a single drug in the world that can completely cure spinal muscular atrophy, and data on the effectiveness and safety of the Russian drug have not yet been presented. At the same time, the new drug has risks, the final decision should be made at a meeting of the Ethics Committee of the Russian Ministry of Health. If the study has begun, then the committee has already approved it.
Olga Germanenko, founder and director of the SMA Families Foundation
“Any new drug, if it passes clinical trials of its efficacy and safety, will bring certain benefits to the center as a whole. To date, there is no drug that can completely stop or cure spinal muscular atrophy, so each new development is important both in terms of increasing the availability of therapy, and in terms of the fact that something can work better. It is too early to talk about this topic here, because clinical studies have just begun, and we do not yet have any data on efficacy and safety. This is a question to be answered later.
If we talk about access to therapy in Russia, given that gene therapy drugs are being developed for young patients, almost all children in Russia are provided with one of two drugs. From the point of view of access to this or that type of treatment, this will not affect the situation. Here the question is rather about the choice of the drug, because now there are three of them, and families can choose the best product together with their doctors.
From the point of view of conducting clinical trials, it is clear that any development is carried out only with the subjects, otherwise it is impossible to obtain data on the safety and efficacy of the drug. In my opinion, it would be unethical to conduct trials with a placebo: if there were a study in which an active drug and a placebo would participate, this is a completely unacceptable situation. Because some children would not receive treatment and would be doomed to the natural course of the disease, which in SMA has disastrous consequences. There is no placebo group in this study, so all children receive the active drug. It is up to each family to make a decision and use already registered drugs with proven effects, or to consider participating in a clinical trial. It is the right of every family to decide, but the study site staff should inform the family of the drugs available and the risks of participating in the study so that the family can make an informed decision.”
Ilya Yasny, head of scientific expertise of the pharmaceutical fund LanceBio Ventures
“The Russian drug is very similar to Zolgensma, that is, it is fundamentally the same – it is a viral vector, adeno-associated virus 9, which carries the gene. There are also differences in which genetic construct is contained in the vector. Certain improvements have been made that may improve the efficacy and safety of the drug, but this needs to be proven in clinical trials. This is promising, although the patient population is not very large – several thousand. Clinical trials can last a year or two, at least, depending on the rate of recruitment and the design of the clinical trial.
There are three aspects – efficiency, safety and cost. There are certain prerequisites that the drug may be better than Zolgensma. “Zolgensma” is dear, and now the children are provided for by her, because there are not so many of them. It is possible to provide everyone with treatment, although I heard that there are problems with this, too, because it is terribly expensive. This is an important milestone and an end in itself – a way to prove that the company can produce and bring such products to market. And this will not be limited to muscle atrophy – if the product is successful, then others will follow.
The question of the ethics of conducting research on children is very acute. Adults can answer for themselves, but here you also have to decide for children. The question is fair, because if there is a therapy, is it necessary to explore a new one? In this case, there are prerequisites that it can be more effective and safe, so there are fewer questions about ethics. Of course, there are risks, because the drug is new, but the risks should not exceed the benefits. This is decided by a meeting of the Ethics Committee of the Ministry of Health of Russia, and in this case, since the study began, the committee made a positive decision. Unfortunately, this is not always the case in Russia. We published an article in a scientific journal analyzing the fact that many studies in Russia are carried out without the approval of the Ethics Committee or even without the approval of the Ministry of Health. This is a special case because this is a new therapy that has received attention and BIOCAD expects to continue to work and expand to other markets, so I think the procedures have been followed in accordance with international rules.
A difficult situation would be if the drug was completely similar to the original. Such a question will sooner or later arise in gene-cell therapy, because it is a risky and difficult therapy. They try to minimize the number of clinical studies and show that the drugs are similar to each other in terms of the production process and properties. On the other hand, there is also the other side of the coin – it is unethical to release a completely untested drug on the market, therefore "it is better to put a certain number of people at risk than tens and hundreds later." It is also wrong to think so. The benefit to society should never justify the risk to one person. This is also one of the principles of conducting clinical trials.”