SMA Scientific Committee members, Ministry officials and SMA associations attended the Workshop on Current Treatments in Spinal Muscular Atrophy (SMA), organized by the Ministry of Health via video conference method.
SMA Scientific Committee members shared data on the SMA drugs used at the meeting. In addition, they made presentations about the most up-to-date scientific studies on possible gene therapy, which has come to the fore recently.
In the presentations made by the scientific board; until recently, without any treatment of SMA for the first time in 2016 that a drug's exit, Turkey the same year was expressed that the drugs in the world as one of the first countries all SMA type 1 patients have access to the free offer. It was also emphasized that shortly thereafter, our type-2 and type-3 patients, which are the milder forms of the disease, which are not included in the reimbursement scope of the majority of the world states, have free access to the drug in our country.
Pointing out that the claims that these patients do not have treatment and that they will die if they do not receive gene therapy until the age of 2 do not reflect the truth, the Scientific Committee shared the following information:
“Our SMA patients, whose application evaluation has been completed, still benefit from the treatment, which is known to be efficacy and side effects, and which is successfully applied in our country.
The data on gene therapy, which has been developed recently and is on the agenda, was immediately and meticulously examined by our scientific committee, as in the first process. In the last 2 months only, our scientific committee has gathered 5 times and examined the data about the drug.
The evidence published in scientific platforms on the efficacy of gene therapy is not yet sufficient and there is no evidence that it is superior to the currently administered therapy. Some studies have reported serious side effects, especially liver failure and low platelet count (bleeding tendency).
In addition, as part of the application procedure of gene therapy, the immune system needs to be suppressed for at least one month, especially in some patients with higher weight, this process may take up to 1 year. In our already fragile SMA type-1 patients, infections and suppression of the immune system pose a greater risk, and the course of any disease can be fatal regardless of the disease. "
Scientific board members, considering all these scientific data; He stated that the application of gene therapy is not suitable for now and that developments will be followed on the grounds that the current data are insufficient in terms of benefit-harm ratio, that a treatment with known efficacy is already being applied, and immunosuppression during the Covid-19 epidemic may increase the risk of death.
The SMA Scientific Committee convenes every month to follow developments in new treatment alternatives. NGOs and families are regularly informed.
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