Specialists from the Polish Mother’s Memorial Hospital – Research Institute have developed and adapted an innovative method of administering one of the most expensive drugs in the world. They spoke about it during a press conference.
PMMH-RI participates in the program of spinal muscular atrophy treatment (SMA) financed by the National Health Fund. This is a severe, genetically determined disease that leads to muscle and nerve degradation. Firstly, the arms and legs weaken, then the patient stops swallowing, and problems with breathing appear -breathing support is necessary. Untreated, the disease leads to physical disability and even death.
The Ministry of Health decided to fully refund Nusinersen – a very expensive drug that stops the disease from progressing. All patients in Poland (the number estimated at about 700) are to have the access to this effective treatment.
At PMMH the drug has already been given over 50 times. 22 children from the łódzkie and świętokrzyskie voivodships benefited from the therapy.
– We administer Nusinersen to the dural sac through lumbar puncture – says Łukasz Przysło MD, PhD, the head of the Department of Neurology – It’s simple if the patient’s anatomy is correct. But sometimes it is very difficult or impossible to administer the medicine this way. With the most seriously ill patients who have a deformed spine we cannot get into the canal.
So neurologists asked specialists from the Department of Neurosurgery for help, and they have proposed an innovative solution.
– A device that is used in intracerebral surgery, the so-called chamber access – the Rickham tank – says prof. Krzysztof Zakrzewski, the head of the Department of Neurosurgery.
Neurosurgeons decided to implant it in the lumbar region. It is connected to the spinal canal with a silicone drain and Nusinersen is injected to the tank located under the skin.
What does the drug administration look like with patients with a tank? – You have to feel it with your finger and pierce through the skin – says Bartosz Polis, PhD, a neurosurgeon. – By puncturing the skin layers, we take cerebrospinal fluid (its analysis is necessary before the drug is given), and we give the medicine. The tank can be punctured repeatedly. Enough for several hundred punctures. it is constructed so that it seals up itself.
A 14-year-old Nikola from Łódź was the first to get the medicine this way. Two patients more were qualified for implantation of the Rickham tank. – This is a small group, but after hard ordeals, and long-lasting attempts of preparation’s administration via standard, intrathecal, route would be an additional factor reducing the quality of their life – adds Dr. Przysło. – Thanks to the use of the Rickham tank, administration of Nusinersen takes several dozen seconds.
More about this innovative method:
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Lekarze z ICZMP stosują innowacyjną metodę podania leku na SMA