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Interview
Interview with Katarzyna Kozłowska, President of the Polish Neuromuscular Diseases Association.
Interview with Katarzyna Kozłowska, President of the Polish Neuromuscular Diseases Association.
Whom is your organization’s help directed towards?
Polish Neuromuscular Diseases Association (formerly Society for Fighting Muscular Diseases) active from the year1989 is an organization which primarily comes to the aid of the people suffering from all kinds of neuromuscular diseases. The best known are the Duchenne muscular dystrophy, Becker dystrophy and spinal muscle atrophy, SLA. There is also a commonly used term „muscle atrophy”, which is caused by a deterioration of muscle strength. All suffering from those conditions will sooner or later lose muscle strength. Their physical condition will deteriorate, they will become disabled, dependent on their family or caregivers.
The association’s current register of members lists over 1200 people. Depending on the financial resources we try to help as many of the sick as possible. We provide rehabilitation for 35 children and 45 adults in Warsaw. Headquarters of Association’s Board is located in Warsaw at 10 Św. Bonifacego street, where the Warsaw Branch is situated. We do not employ anyone full-time, but work on voluntary basis whenever possible.
How does modern medicine deal with muscular diseases?
Conventional medicine is increasingly better at diagnosing neuromuscular diseases, i.e. determining their types, degree and expected course. At present an effective pharmacological treatment is not available. Most neuromuscular diseases are genetically conditioned and so far incurable. One of the ways to stop the progress of the disease, losing muscular strength is consistent and regular rehabilitation conducted by physiotherapists. The rehabilitation consists in doing regular stretching exercises, preventing contractures of the Achilles tendon, knees and hips and helps strengthen natural muscular belt of stomach and the back, which is very important in slowing down rapid scoliosis and lordosis, resulting in breathing problems and premature death.
What is the most difficult about those diseases?
Neuromuscular diseases are terrible not because they exist but because their progress is so rapid. A child can walk, and then problems climbing the stairs occur, getting up from the floor becomes impossible, the same goes for getting out of a bathtub, then the child sits in a wheelchair, which later he or she has no strength to drive. Such are the consequences of a progressing loss of muscular strength. In Duchenne muscular dystrophy there are cardiological problems and they cause breathing problems, which is the most common cause of death. It is crucial to early introduce preventive, non-invasive breathing aid. In Poland respirators which support breathing are not refunded and that’s why the lifespan of the boys with Duchenne muscular distrophy is low compared to the United States or Europe.
Is there a system in Poland, created for the aid of the people with neuromuscular diseases?
The problem starts with diagnosis. If somebody finds out that their child, husband or wife has a problem with a neuromuscular disease, there is a series of tests confirming the diagnosis, and what is left is only rehabilitation.
Polish Neuromuscular Diseases Association (formerly Society for Fighting Muscular Diseases) active from the year1989 is an organization which primarily comes to the aid of the people suffering from all kinds of neuromuscular diseases. The best known are the Duchenne muscular dystrophy, Becker dystrophy and spinal muscle atrophy, SLA. There is also a commonly used term „muscle atrophy”, which is caused by a deterioration of muscle strength. All suffering from those conditions will sooner or later lose muscle strength. Their physical condition will deteriorate, they will become disabled, dependent on their family or caregivers.
The association’s current register of members lists over 1200 people. Depending on the financial resources we try to help as many of the sick as possible. We provide rehabilitation for 35 children and 45 adults in Warsaw. Headquarters of Association’s Board is located in Warsaw at 10 Św. Bonifacego street, where the Warsaw Branch is situated. We do not employ anyone full-time, but work on voluntary basis whenever possible.
How does modern medicine deal with muscular diseases?
Conventional medicine is increasingly better at diagnosing neuromuscular diseases, i.e. determining their types, degree and expected course. At present an effective pharmacological treatment is not available. Most neuromuscular diseases are genetically conditioned and so far incurable. One of the ways to stop the progress of the disease, losing muscular strength is consistent and regular rehabilitation conducted by physiotherapists. The rehabilitation consists in doing regular stretching exercises, preventing contractures of the Achilles tendon, knees and hips and helps strengthen natural muscular belt of stomach and the back, which is very important in slowing down rapid scoliosis and lordosis, resulting in breathing problems and premature death.
What is the most difficult about those diseases?
Neuromuscular diseases are terrible not because they exist but because their progress is so rapid. A child can walk, and then problems climbing the stairs occur, getting up from the floor becomes impossible, the same goes for getting out of a bathtub, then the child sits in a wheelchair, which later he or she has no strength to drive. Such are the consequences of a progressing loss of muscular strength. In Duchenne muscular dystrophy there are cardiological problems and they cause breathing problems, which is the most common cause of death. It is crucial to early introduce preventive, non-invasive breathing aid. In Poland respirators which support breathing are not refunded and that’s why the lifespan of the boys with Duchenne muscular distrophy is low compared to the United States or Europe.
Is there a system in Poland, created for the aid of the people with neuromuscular diseases?
The problem starts with diagnosis. If somebody finds out that their child, husband or wife has a problem with a neuromuscular disease, there is a series of tests confirming the diagnosis, and what is left is only rehabilitation.
There are three types of the spinal muscular atrophy. The most serious form of this disease is SMA I, called the Werding-Hoffman disease. Symptoms appear at 6 months of age. The diagnosis is made very early. Such children don’t sit up on their own, breathing problems occur at an early stage. Stage SMA II (transitory form) is diagnosed between the first and the second year of age, when children sit on their own, but are unable to walk. In their case a spine operation is necessary, because of a rapid development of scoliosis. The lightest course of spinal muscle atrophy occurs in the case of SMA III, called Kugelber-Welander disease.
Duchenne muscular distrophy is diagnosed in Poland between the fourth and sixth year of age. Prenatal diagnostic tests are recommended. Doctors can present to the parents of children with Duchenne muscular distrophy an anticipated course of the disease - what they can expect - but except for the diagnosis, they do not have a therapy to offer. Children with Duchenne muscular distrophy are given steroids which can slow down rapid progress of the disease.
Unfortunately, in Poland we do not have a guaranteed system of rehabilitation and medical care refunded by the system. The same goes for appropriate orthopedic equipment. All depends on the determination of the parents and their financial situation. There is no satisfactory social support for the families which face a dilemma of which parent will give up his or her job? Taking into consideration the fact, that the disease is a very serious one, usually one of the parents doesn’t work in order to take proper everyday care of the sick child.
What is needed most?
First of all, there is no system making it possible to refund rehabilitation. We receive signals that it is possible to have access to rehabilitation directly in children hospitals, but later it all ends. Our children should undergo regular rehabilitation. In practice it should be conducted at least three, four times a week. If there are no medicines, there should be such a system of medical care created, that would secure access to rehabilitation and ortopedic equipment. National Health Fund refunds ortopedic equipment (orthesis, corsets, parapodia) but unfortunately you can receive it only once two, three years, whereas children grow fast. So we have to fight for new standards of care and aid, which should be adjusted to the real needs of the sick.
Do stem cells give a chance of recovering from this disease?
Stem cells are our new and great hope. We all believe that someday they will prove to be a real help in the diseases of our children. However, we still do not know enough about them. Nevertheless, centers in Ukraine offer implanting stem cells, but there is no scientific evidence that it helps. We offer this information to our sick on the home page of the Association at: www.ptchnm.org.pl
We publish opinions of our Polish neurological authorities. We still do not have enough knowledge and confirmed information about implating stem cells. We do not have access to clinical tests, medical protocols and no possibility of monitoring a child’s condition before the treatment and after the stem cells have been implanted.
What is your opinion on the subject?
As a mother of a sick child, who travels at her own expense around the world seeking hope in various clinical centers, mainly in Italy, I wonder why there are no foreigners in those Ukrainian centers. Why don’t people from Israel, Italy, France, Germany and even United States go there? For them the cost of such a treatment, 10 – 15 thousand euro, is insignificant.
If those treatments really helped, the hospitals there couldn’t rid themselves of the parents from all over the world. There are too few contacts in Poland with parents, who have already been there. It turns out, that the ones who were there are quite secretive about it. Isn’t this suspicious? I attended professor’s G. Novelli lectures in Italy and he explained that scientists all around the world are at the stage of isolating those cells. They know how those cells function and they know that those cells can help. But they still do not know, what those stem cells can transform into and how long they can function as new cells, which are the ones that we want to obtain. In case of such rare genetic diseases, parents of the children who suffer from the lack of pharmacological treatment, put great hopes on gene therapy and the use of stem cells, but we have to wait for clinically confirmed evidence.
What differences do you see when you travel around the world and have contacts with families of the sick children,?
Poland differs most of all in the treatment standards. Neuromuscular diseases are not listed in the register of chronic diseases. This is scandalous and should be changed as soon as possible. In the European Union neuromuscular diseases are considered chronic diseases and therefore can be treated in a comprehensive way, with application of appropriate medical standards. The sick require systematic, specialist care: of a neurologist, orthopedist pulmonologist, physiotherapists. In EU countries there are also standards of orthopedic care. Boys have their Achilles tendons cut. This surgery can give two extra years of walking and postpone the moment of confiding the boys to wheelchairs. As far as European standards of treatment are concerned, it is virtually impossible to compare them with ours, because our children are left alone and if they have strong parents, the ones who go beyond standards that are in effect, they can count on the parents to find financing for private rehabilitation. The same goes for medical attention standards. They cannot be compared. In Holland and in Italy, children with Duchenne muscular dystrophy are given electric wheelchairs without any costs, they get orthopedic equipment, patient lifts , whereas in Poland we get patient lifts once every three years. In Poland, parents must cover a part of the cost of an electric wheelchair. There is also a great difference in the quality of our corsets and the ones produced in the West. Respirators for non-invasive breathing support are not refunded either.
What is the biggest financial obstacle for Polish parents?
Our children die because of the lack of respirators. One of the cheapest respirators costs about 35 thousand PLN. Fortunately, recently companies selling equipment for non-invasive breathing support have begun selling the equipment in a form of medical leasing, so instead of a new car you can buy a respirator for a child in installments. But how many parents can afford it? Should it be like that? I am not saying that our children are entitled to something, but if it’s available in the European Union, which we fortunately joined, the problem of refunding and access to respirators and the equipment for non-invasive breathing support should be solved.
The Association makes attempts at introducing standards...
What differences do you see when you travel around the world and have contacts with families of the sick children,?
Poland differs most of all in the treatment standards. Neuromuscular diseases are not listed in the register of chronic diseases. This is scandalous and should be changed as soon as possible. In the European Union neuromuscular diseases are considered chronic diseases and therefore can be treated in a comprehensive way, with application of appropriate medical standards. The sick require systematic, specialist care: of a neurologist, orthopedist pulmonologist, physiotherapists. In EU countries there are also standards of orthopedic care. Boys have their Achilles tendons cut. This surgery can give two extra years of walking and postpone the moment of confiding the boys to wheelchairs. As far as European standards of treatment are concerned, it is virtually impossible to compare them with ours, because our children are left alone and if they have strong parents, the ones who go beyond standards that are in effect, they can count on the parents to find financing for private rehabilitation. The same goes for medical attention standards. They cannot be compared. In Holland and in Italy, children with Duchenne muscular dystrophy are given electric wheelchairs without any costs, they get orthopedic equipment, patient lifts , whereas in Poland we get patient lifts once every three years. In Poland, parents must cover a part of the cost of an electric wheelchair. There is also a great difference in the quality of our corsets and the ones produced in the West. Respirators for non-invasive breathing support are not refunded either.
What is the biggest financial obstacle for Polish parents?
Our children die because of the lack of respirators. One of the cheapest respirators costs about 35 thousand PLN. Fortunately, recently companies selling equipment for non-invasive breathing support have begun selling the equipment in a form of medical leasing, so instead of a new car you can buy a respirator for a child in installments. But how many parents can afford it? Should it be like that? I am not saying that our children are entitled to something, but if it’s available in the European Union, which we fortunately joined, the problem of refunding and access to respirators and the equipment for non-invasive breathing support should be solved.
The Association makes attempts at introducing standards...
With this aim we organize all kinds of trainings. I, myself participated in such trainings, for example the one in Bologna, where doctor John Bach, who specializes in non-invasive breathing support in neuromuscular diseases was invited. The training was dedicated to instrumental and manual techniques of breathing support in neuromuscular diseases, how to help a child to breathe, to clear his/her throat of discharge. I came up with an idea to organize such training in Poland. We managed to do it thanks to financing from PFRON. The training was organized for doctors and physiotherapists from all over Poland.
How about Poland?
Currently we have active branches in Gdańsk, Łódź, , Wałbrzych, Poznań. We have doctors, who in spite of the lack of effective pharmacological treatment, monitor information about new clinical trials. We’re in contact with physiotherapists from all over Poland, who know the specificity of rehabilitation in the case of neuromuscular diseases. We do as much as we can. Anyone can be useful and helpful. I am responsible for contacts with doctors in Poland and abroad. We organize support groups, so that someone can call us. We put information on our website precisely because the moment when a person is given diagnosis is the most difficult one. However, we do not have enough money for psychological help. We talk on a daily basis about somebody dying or somebody’s death and that the rest is looking for help. There should be a standard worked out for psychological care for the times, when diagnosis is made, the disease of a child is confirmed and when a member of a family, suffering from incurable neuromuscular diseases dies.
What is the most mentally difficult for parents?
The moment, when diagnosis is made and the gradual deterioration of a child’s muscular strength. You virtually have to help a child to live, overcome everyday problems, explain many issues, like why a child cannot ski, walk, why the friends he made at rehabilitation courses die, why there is no assistant available like in the European Union countries? We are only now fighting for it. Other organizations train assistants, but so far there is no center available, where one could call and at a small cost ask for help and having an assistant sent. In the West, 30-40 year old disabled people live on their own, they have an assistant eight hours a day, whereas here, parents of such disabled children, regardless of the type of their disability wonder what will happen to their children in the future, when they no longer are able to provide help.
If each mother has a child with a different type of dystrophy, how to establish standards for medical attention?
Unfortunately, in Poland the medical attention system does not offer any programs for taking care of the children with such serious diseases, where a cooperation of a team of doctors is necessary, as opposed to visiting a neurologist once a year. There is no information available about the necessity to adjust Polish medical attention system to the standards of the treatment and care of the disabled children, suffering from all kinds of diseases, which are already binding in the EU. We have to fight ourselves to have them introduced.
Was that the case with, for example, respirators?
We managed to obtain 30 respirators from Holland. Our sick breathe thanks to the respirators, which were written off a long time ago in Holland. They can still work, though, and help the sick, who have to use the non-invasive breathing support. I’ve recently attended a conference in the Hague and an organization similar to ours, dealing with problems of people with neuromuscular diseases promised us, that they will continue to collect respirators for the sick in our country.
I think that sooner or later the people responsible for those matters in the National Health Fund, in the Ministry of Health will have to precisely calculate costs of a one-day stay of each person suffering from the Duchenne muscular dystrophy in the Intensive Care ward and compare them with the costs of refunding respirators and this will help to significantly reduce costs and make it possible for the sick to stay home. There are ways to reduce costs of the medical attention of the sick and not block hospital wards. It is being practiced all over the world.
If there are no standards of treatment for such diseases, how are families dealing with the situation?
A disabled child will be receiving a pension, but Polish pensions do not secure an independent living.
A reasonable movement of mothers should therefore be created, which would jointly fight for their children’s right to a dignified life. We should fight for establishing treatment standards and listing rare genetically conditioned diseases in the register of chronic diseases. But if we keep on making divisions and entering into disputes about what is important and necessary to whom and who is more entitled to what, we will not achieve anything. First we must cause a change in the current system of health care, so that it has worked out methods of supporting families, and provide a sense of security both to the disabled and their direct caregivers. As far as the parents are concerned, the most difficult moment is certainly hearing the diagnosis and the anticipated course of a given disease. I will never forget waiting for the results of genetic tests, confirming my daughter’s spinal muscular atrophy. In the first two weeks I was looking everywhere for information about possibilities of treating this disease. I couldn’t accept the fact, that my daughter is sick, that she will have problems walking. I still can’t accept the fact that spinal muscular atrophy is an incurable disease, that medicine cannot offer any effective pharmacological treatment.
Everyday I monitor information available in English and Italian on various pages, related to spinal muscular atrophy. I am a member of many newsgroups in Europe. I maintain contacts with parents from all over the world. I try to share all the information about the conducted new clinical trials with other parents in Poland. I still believe that the development of the genetic engineering will make it possible in the next couple of years to create an effective medicine for slowing down progress of the disease.
When looking for information about all new medicines tested on children suffering from spinal muscular atrophy, I pay special attention to potential side effects. I do hope that Polish children will be able to participate in the tests evaluating effectiveness of new, experimental drugs. I am counting on the National Health Fund to find additional resources allowing Polish children suffering from rare, genetically conditioned diseases to participate in the European clinical trials that are being planned. I believe, that the Polish health care system will soon have to be adjusted to the standards of treatment and medical attention for the disabled, binding in European Union.
If there are no standards of treatment for such diseases, how are families dealing with the situation?
A disabled child will be receiving a pension, but Polish pensions do not secure an independent living.
A reasonable movement of mothers should therefore be created, which would jointly fight for their children’s right to a dignified life. We should fight for establishing treatment standards and listing rare genetically conditioned diseases in the register of chronic diseases. But if we keep on making divisions and entering into disputes about what is important and necessary to whom and who is more entitled to what, we will not achieve anything. First we must cause a change in the current system of health care, so that it has worked out methods of supporting families, and provide a sense of security both to the disabled and their direct caregivers. As far as the parents are concerned, the most difficult moment is certainly hearing the diagnosis and the anticipated course of a given disease. I will never forget waiting for the results of genetic tests, confirming my daughter’s spinal muscular atrophy. In the first two weeks I was looking everywhere for information about possibilities of treating this disease. I couldn’t accept the fact, that my daughter is sick, that she will have problems walking. I still can’t accept the fact that spinal muscular atrophy is an incurable disease, that medicine cannot offer any effective pharmacological treatment.
Everyday I monitor information available in English and Italian on various pages, related to spinal muscular atrophy. I am a member of many newsgroups in Europe. I maintain contacts with parents from all over the world. I try to share all the information about the conducted new clinical trials with other parents in Poland. I still believe that the development of the genetic engineering will make it possible in the next couple of years to create an effective medicine for slowing down progress of the disease.
When looking for information about all new medicines tested on children suffering from spinal muscular atrophy, I pay special attention to potential side effects. I do hope that Polish children will be able to participate in the tests evaluating effectiveness of new, experimental drugs. I am counting on the National Health Fund to find additional resources allowing Polish children suffering from rare, genetically conditioned diseases to participate in the European clinical trials that are being planned. I believe, that the Polish health care system will soon have to be adjusted to the standards of treatment and medical attention for the disabled, binding in European Union.
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