FAQ on Stem cell therapy to Muscular Dystrophy
Muscular dystrophy: how could stem cells help?
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This section offers answers to your questions and fears about applying stem cell therapy to Muscular Dystrophy patients. These are not questions and answers picked up from secondary sources. But are questions raised by muscular dystrophy patients and parents in person and responses provided by Dr. Gururaj A Rao, Research Director of Stem Cell Services International Limited, Bangalore, during an interactive workshop organized by Muscular Dystrophy Foundation India on 14th August, 2010 at Hotel GRT Regency, Madurai. However, please be aware that the questions and responses are presented almost in the same manner in which they were asked and responded. Therefore, mdfindia would not be made liable for misinterpretations and misrepresentations, if any.
Questions and answers were compiled by Mr. P. Jeyaprakash and Mr. R. Vignesh, Research Scholars from the Depart of Micro-biology, Madurai Kamaraj University, Madurai. They did help the patients/parents and Dr. Gururaj in communicating with each other by translating questions and responses in Tamil. We gratefully acknowledge their selfless services.
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Q.1 How many numbers of injections of stem cells are required to treat Muscular dystrophy?
It is difficult to interpret. The therapeutic efficacy depends on the patient conditions including the uptake of the stem cells, the severity of the disease and the age.
Q.2 Initial biopsy taken from the patient shows that there is tissue degeneration (with posture instability) and after subsequent biopsies muscle regeneration was observed. Is there any further treatment needed?
If the posture (back) of the patient is really distorted then the surgery is needed along with the continuous physiological observance.
Q.3 Does the continuous physical exercise will improve steroid based drug therapy in Muscular dystrophy?
Physical exercise is necessary but not in a continuous and severe manner. The type of physical exercise must be practised upon the physician’s advice. Steroid therapy is subjective and limited in usage. The main goal of the steroid therapy is to reduce the inflammation during muscular dystrophy. However, the severe exercise will have impact on bringing the side effects.
Q.4 The patient is 10 year old girl, upon the physical examination it was noted that her legs become oversized and she was not able to lift her legs. She has more muscle mass compared to normal. So, is there any link to disease?
If she is diagnosed to be DMD, hypertrophy could be the cause of muscle mass. In such situation the movement of the body is difficult. Based on the patients report, it cannot be DMD.
Q.5 A twelve year male child has disability in leg movement but sensation is normal. Could it possible to improve the condition by using the iron leg braces along with drug therapy?
Physical exercises will improve the condition along with iron leg braces. Forcing the child to walk under pressure may lead to negative effect on health. It is strictly that physical therapy must be done with the doctor’s advice. Prescription of the suitable drug is difficult for this condition however anti-inflammatory drugs can be recommended under physician’s advice.
Q.6 A patient with the defect is able to walk now and in future is it good to give chemotherapy along with the stem cell therapy?
The drug (anti-oxidants) therapy can improve the muscle strength and be used in association with stem cell therapy. However, the approach is in practise only in developed countries.
Q.7 In a family, the elder brother (age – 12) has DMD. Will my younger son get DMD? If so, is that possible to treat with stem cell therapy?
The chance of younger son getting DMD is 50%. However, it has to be confirmed by molecular diagnosis. The stem cell therapy works in younger patients than the older one.
Q.8 We (DMD patients) like to opt for stem cell therapy. So is that possible to go for stem cell therapy as a group? What is the cost of stem cell therapy?
It is difficult to treat multiple patients at the same time due to the difficulty in cultivating enough stem cell for therapy. However, it is possible to conduct the therapy for the patients in collaboration with the Muscular Dystrophy Foundation India. A single session (injection) uses 20 million cells and the cost may come around Rs. 50,000 per session. The number of sessions (doses) depends on the status of the patients. In general, five sessions are considered to be sufficient and markedly reduce the CPK level.
Q.9 I am a driver. I have become weakened and also observe the same condition with my uncle’s son. In order to be active, I am taking Vitamin tablets. Whether due to the DMD, is there any possibility for the development of heart attack and diabetics?
It is difficult to interpret based on observation and diagnosis needs to be done to confirm the disease in your uncle’s son. There is no reports says that DMD can accelerate heart disease and diabetics. However, cardiomyopathy can lead to heart attack.
Q.10 Is there any stimulus that can induce the cells to become mesenchymal cells (trans-differentiation) and can we use the trans-different cells for the therapy?
This is in research level. It was noted that paracrine factors such as TGF-beta will be useful in transforming other cells into mesenchymal stem cells (MSC). Also it is reported that MSC therapy is safe to use and the injection of MSC in the muscle will transform the stem cells into muscle cells. The safety of usage needs to be considered because if there is any non -specific transformation happens, it will be unsafe.
Q.11 Practically did any of DMD patient is been cured?
Unfortunately, none of the patients are cured. Aware it is not like viral or bacterial disease which occurs due external pathogens. But the scenario is different for muscular dystrophy. Since it is a genetic disease and the defect is in each cell, it is difficult to cure.
Q.12 Can 5 doses of drug completely cure DMD?
It just improves the patient condition but it cannot cure the disease.
Q.13 Can the chemotherapy cure the disease?
Drug therapy is practised to just reduce the inflammations and pain during the disease progressions and it is just used to improve the condition. Since DMD is a complicated genetic disease, chemotherapy has no effect in curing the disease. However, the curing of the disease is possible when the number of unaffected cells is more in the patients.
Q.14 A patient is diagnosed with LGMD. In considering the age criteria of 40-50 years, how stem cell therapy can improve the patient health?
Stem cell therapy for LGMD needs more cells for the treatment. With the continuous treatment with stem cell therapy can improve the health condition of the patient. This depends on the status of the patient, age and the severity of the disease condition. The treatment efficiency can be assessed by muscle fibrosis level.
Q.15 A six year old boy is diagnosed with DMD. Subsequently, he has lost his ability to walk and Is it possible to make him walk again?
It is difficult to make him walk again by stem cell therapy. Preferentially, a closed observation is needed to muscle strength with recommended physical therapy.
Q.16 Is it possible to treat a patient with his destabilized back with stem cell therapy along with routine physiotherapy to recover his normal posture?
The stabilization of the back needs the accessories muscle to give a physical support to the back. Once these muscles are lost, it is difficult to bring back the normal posture. Since the muscle cells are low, it needs more number of cells for the therapy and prolonged treatment. Even after the multiple doses the improvement is the question. However, the back can be strengthened.
Q.17 A patient affected severely with DMD (with huge muscle loss). Will the stem cell therapy improves the health and what is the cost of the patient?
The doctor has to work out on cost and also it is important to assess the number of doses depends on the patient observance.
Q.18 An 8 year old boy is affected with DMD. In order to treat the boy, cord blood cell was collected from his aunt and stored in the repository cell line bank in US. Will the stem cell compatible for the boy? If compatible, can the stem cell therapy conducted in US or in India?
Whether the father’s stem cell will be compatible to his son?
As a preliminary assessment, the cell matching needs to be done. If it matches, the stem cell therapy can be carried out in India. And we need to consider the stem cells obtained from the cord blood are not enough for the therapy. Stem cell obtained from the father will give 50% of the match and that is enough to go for therapy if the father’s health condition is proper. Preferentially, stem cells derived from the adipose will be used for the therapy.
Q.19 A Prenatal diagnosis in a conceived women reports that foetus has the genetic defect muscular dystrophy. Whether is it possible to prevent the disease at the embryonic stage?
It is an ethical issue and it is possible to prevent by consulting the gynaecologist to decide about the abortion stage. A foetus with 11-20 weeks of development can be aborted.
Q.20 A patient with 25 years age reported for DMD in NIMHANS, Bangalore. The report says that the patients with DMD around the age of 25 will never survive. But the reported patient still survives. Is it necessary to re-confirm the disease (DMD)?
Molecular diagnosis is really needed to confirm the disease and such kind of molecular diagnosis will be done at Reliance clinical service in Mumbai, Gangaram hospital in Delhi and Vishwanath hospital, Avinashilingam hospital in Coimbatore.
Q.21 Is the muscular dystrophy genetic disease will be transmitted to the progenies?
Yes, the genetic disease will be transmitted to the progeny. Boys have the 50% of chance to get the disease while the girls act as carriers. Usually boy affected with muscular dystrophy dies earlier.
Q.22 If we introduce stem cells into the body, is it distributed evenly in the body and promotes the muscle growth?
It depends on the type of stem cells which are used for the treatment. In case of MSC, animal studies showed that it is transformed into muscle tissue. It is not sure that the distribution is even in the body.
Q.23 Whether the stem cell therapy is approved by ICMR?
The stem cell therapy will be conducted under the guidance of Institutional committee for Stem Cell therapy with ICMR regulations.
Q.24 Does stem cell therapy needs a specific type of stem cell (eg: muscle stem cells)?
Stem cell therapy does not need a type specific stem cell. Generally, any type of stem cell can be used.
Q.25 Any patient in India had undergone stem cell therapy?
Yes, many patients had been treated with stem cell therapy but their clinical improvement and case studies have not been reported in India.
Q.26 If father is not alive, can the stem cells from mother used for stem cell therapy?
Usually, stem cells from the males are highly preferred since the females can act as a carrier. So, prior molecular diagnostics must be done with mother before isolating the stem cells.
Q.27 Is there any preventive measure in practise that can prevent the death muscle cells during the disease progression?
Unless the disease is treated at the genetic level (gene therapy), the death of the muscle cells cannot be controlled. However, anti-inflammatory drugs can be used to prevent the death of muscle cells due to the immunological response.
Q.28 How many patients have undergone stem cell therapy in your institutions?
Around 30 patients have undertaken the stem cell therapy and around 100-200 stem cell injections have been done.
Q.29 Does the stem cell therapy authenticate the safety of the patients?
The clinical data of the patients who have undertaken the stem cell therapy for 3-4 years are available and there were no complications reported. But there is no long time record available. In animal studies, it was confirmed that stem cell therapy is 100% safe if it is done with safety guidelines.
Q.30 Can physiotherapy alone recover the patient from back problem?
It is unlikely. However, physiotherapy will bring down the further complications.
Q.31 Can stem cell be injected through veins although arterial injection in practise?
Injection of stem cells can be done through vein but the number of cells goes to the muscle will be very less since the venous blood will go across the liver and heart and the stem cells will be eventually lost and that will give minimal beneficial effects. Eg: if you inject 10 million cells through vein around 1 million cells alone will reach the muscle. In some cases, intravenous will be done with some considerations. Even intramuscular can be done but still multiple doses are needed however it can leads to other complications.
Q.32Can a patient affected with DMD go for marriage?
Yes. But after the marriage genetic counselling must be done and the baby needs to be checked by genetic testing.
Q.33 A patient with BMD is undertaking the nutrition therapy at the age of 18. Can the nutritional treatment be continued?
Yes. The patient can continue the treatment along with the anti-inflammatory drug therapy.
Q.34 A 11-year old child was reported for DMD and there is no deletion was reported in the dystrophin gene. Can the child go for stem cell therapy?
Yes. But the immunohistochemical report showed that the child is affected with BMD but not DMD. Since it is BMD no need to fear and stem cell therapy can be used.
Q.35 Does consignous marriage will bring high risk of muscular dystrophy transmission?
Yes, consignous marriage usually enhances the risk of genetic defect accumulation. In order to prevent this, the family history genetic counselling must be followed.
Q.36 Can a DMD patient donate his/her organs after the death?
Yes, it is possible to donate some of the organs like eyes and kidney but definitely heart will not be donated because of the inefficient heart muscle (cardiomyopathy) unless it is muscular related, the organs can be donated since eyes and kidney are non-muscular organs.
Q.37 A female has been reported for muscular dystrophy. Can she give birth to her child through surrogate mother?
If the sperm of the male is not affected with DMD, it can be done.
Q.38 What is the average time course required for 5-6 doses of stem cell injections? Should it be continued throughout the life time?
It is not possible to conclude anything with 5-6 doses. Unfortunately, the muscle satellite cells will die during the aging. Until, the defective cells will be replaced with the normal cells the therapy needs to be continued.