MedulloBlastoma (2 year old girl)

Little Emily has been battling MedulloBlastoma for the past year. Diagnosed at the age of one with stage 3 MedulloBlastoma, Emily’s parents faced different opinions and had to make tough choices in order to provide her the best treatment. Eventually and against all odds they have traveled from Israel to NYC for a clinical trial, not even sure they will get accepted to the trial.

If your child has MedulloBlastoma, this half-hour interview may help you save his/her life. Learn about the challenges of parents dealing with brain tumors, including the medical, logistic and financial aspects.

 

About Mice and cancer-treatment

champions

This link is to an article that Suzie Siegel wrote about the upcoming clinical trial using mice to help doctors choose the right treatment for someone with advanced soft-tissue sarcoma: http://sarcomaalliance.blogspot.com/
champions
Cancer treatments have been tested on mice for years.

Now a new technology uses mice to determine which treatments may best fight an individual patient’s sarcoma.

 

 

The TumorGraft technology “provides options for patients in difficult situations when, often as doctors, we are not sure how to treat people” said medical oncologist Justin Stebbing, a professor of cancer medicine and oncology at London’s Imperial College. “In doing so, it advances the frontiers of science and medicine, but in a practical way, giving clinicians information on treatment choices – what is likely to work, and as importantly, what is likely to not work.”

 

Posted: May 2015

 

MedulloBlastoma (2.5 year old girl)

 

Gali has been battling MedulloBlastoma for the past 4 years. Diagnosed at the age of 2.5 with stage 3 MedulloBlastoma, Gali’s parents faced different opinions and had to make tough choices in order to provide her the best treatment. Eventually they have traveled from Israel to L.A and from there to NYC, balancing between the recommendations of their doctors in Israel and in several different institutions in the U.S.A on one hand, and different treatment protocol on the other. 

If your child has MedulloBlastoma, this one-hour interview may help you save his life. Learn about the challenges of parents dealing with tumors in the brain & MNS of their 2.5 year old daughter, including the medical, logistic and financial aspects.

 

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My child’s name is Gali, now she is six years old.

When she was diagnosed she was two years and four months old.

She was diagnosed with medulloblastoma stage 3. She had a brain tumor in her small brain and then we also realized that she had some tumors around the back, on the spinal cord.

The first sign we noticed was that her eyes went inside, in what is called strabismus. We took her to an eye doctor who said immediately that she had fluid in her brain and on the same day we went to the hospital and did an MRI of the brain. Two days later she was operated on and they were able to remove the entire tumor from her brain.

The operation was done by Dr. Shalom Michovic of Schneider hospital in Israel. He did a great job, and we thank him today because Gali came out of the operation after eight hours with no neurological damage at all, which is practically a miracle. When you have to remove a tumor from the brain it’s very dangerous. The healing process for the operation took us about a month.

Since she also had some tumors on the spinal cord, of course, this was not enough and we also had to do something to make sure that it will not come back, so we started chemotherapy and a long process of treatment which lasted about one year.

We were treated in the Schneider Hospital for Children in Israel. We had a treatment that started with chemotherapy because she was too young to get radiation, although in this type of tumor radiation is considered to be more appropriate or more successful, but we had to make sure the radiation will not hurt her too much because of her young age by giving controlled doses of radiation. So, we went through a protocol of the Children’s Oncology Group (COG). I don’t remember the exact number but the protocol included a high dose of chemotherapy, and three mini bone marrow transplants. The whole process was about eight months.

We consulted several doctors before we started and we understood that chemotherapy is something that you can do everywhere actually, because it’s like a recipe of how to do a cake and you don’t have to be a specialist. You just have to give the medicine, and hope that it works. It’s not something special and we talked with all the doctors abroad, we talked with at least three doctors and they all confirmed that this is a good protocol, so we gave the okay to do this protocol.

After eight months in Israel, we modified the protocol. We added Methotrexate, a very strong medicine, which is not part of the protocol. The doctors in Israel recommended Methotrexate even though it’s not in the protocol. It is in the head start protocol in the United States, but not in the COG protocol. Due to her tumors on the spinal cord we had to take one step further with the chemotherapy and we also needed to wait more before giving her radiation because she was too young and all the doctors said that three years old is the minimum age to start radiation.

We actually finished the bone marrow transplants when she was exactly three years old, and then we did another MRI to review the results and luckily the brain was still clean however the tumors in the spinal cord remained. Even though they shrank a little bit, and they were like spots around the spinal cord they unfortunately didn’t disappear, and you can’t operate on the spinal cord. The spots were in many places. So we were at a time when we had to choose what to do next.

The doctors in Israel recommended that we go straight to radiation and because there were still spots on the spinal cord. They said, “You have no other choice but to give the full dose radiation.

She is three years old. It’s not the best, but that’s what you have.

We were also in touch with several doctors in the United States and Canada. We had the impression that it’s not that easy to make a decision so I decided to go and meet with all these doctors. The first doctor who I’ve met, who followed us through the entire process, was Dr. Jonathan Finlay from The Children’s Hospital of Los Angeles (CHLA). He’s a great person and a great doctor. He’s also the person that invented the Medulloblastoma head start protocol for infants. I would say his goal in his life is to find a cure for brain tumors in children without using radiation, and he’s doing all that he can to make this possible. I went to see him. I went alone, without Gali, with all the information that I had, with the MRIs, with a summary of everything, and all other information about the entire situation.

Approaching all the doctors was a process. In some hospitals we had to send all of the information beforehand, some were less formal, some required going through international department, and with others we went directly to the doctor. With Jonathan Finlay it was all very informal. I went to see him and he gave me the impression that although he’s really against radiation in Gali’s case we had no other chance but to give her radiation; however, we had to make all efforts to do it the lowest dose possible.

Then I went to see other doctors. The first was Eric Bouffet, head of pediatric brain tumors at the Hospital for Sick Children in Toronto. After that I went to see Dr. Roger Packer at the Washington Hospital for Children, and then I went to see a doctor in Boston.

We gathered all the information from all the doctors and created a plan. The plan was to go to Los Angeles to have the radiation treatment. There were two main reasons why we went to Los Angeles for the radiation treatment instead of doing it in Israel: The first reason was that we got the impression that in Israel they do not have a lot of experience in radiation in such young children. It has to be really precise, on the millimeter, because the spinal cord is so thin and the brain in such little children is so small that it really is important to be precise by using people with the greatest experience, and most knowledge of physics. The second reason was that we didn’t feel that they would cooperate with us in Israel to give the lowest dose of radiation. In Israel they are not familiar with this; they only know to give high dose or nothing.

So we went to Los Angeles where they did the radiation therapy with the lowest possible dose. It was 1,800 for the spinal cord, the brain, and 2,300 for the bed of the tumor, they call it CUYG. What professor Finlay did is that towards the end of the radiation therapy he performed an MRI to see if there was any reaction to the radiation. He saw that in a very short period of time there was a positive response, and the spots reduced in size. He said that we can stop here, so we stopped. I have to say that during the radiation treatment he also gave Gali Vorinostat, which is a biological cancer stopping or killing drug.

After the radiation therapy, our doctors thought that the low dosage wasn’t enough. So we had to complete the treatment using an alternative method.

We went to Memorial Sloan-Kettering Cancer Center (MSKCC) in New York. At MSKCC we met Doctor Ira Dunkel, head of the pediatric brain tumor department, and we applied to join phase two of the clinical trial of iodine monoclonal injection. It’s a special clinical trial that is performed for brain tumors, Neuroblastomas, and other central nervous system tumors. We had to be accepted to the trial by going through tests to make sure that Gali met the requirements for the treatment. Luckily, she was accepted to the trial, and we did it.

The treatment involves five radioactive injections directly to the brain. The radiation goes to the fluid of the brain which goes through the spinal cord. They injected the radioactive material which is attached to blood, or other material from white rats which attaches to tumors. The radiation was attached directly to the tumors if there were any. They still didn’t know if there were any tumors left so they had to attach radioactive material directly to them. This method avoids the long term difficulties that you would expect from getting a high dose of radiation because this is not on the entire brain, or the entire spinal cord. We completed this process. The whole trip from Los Angeles to New York took us about six months.

We later went back to Israel and continued to take three oral medications. The first is Temodal, which is a chemotherapy drug. We took Temodal for six months instead of a year because her immune system couldn’t handle more chemotherapy along with the two other medications. Vorinostat was the second one and the third one was Roacutan, which we took for a whole year.

The entire treatment after the one year of medications ended two years ago, we are still under a very severe follow-up. We do MRIs every 12 weeks because there are still some spots on the spinal cord, and they have to make sure that they don’t grow because as long as there are spots they could be left over scar tissue or tumors. If they suddenly grow, God forbid I hope it will never grow, then it means it’s something else.

The MRI results are sent to Dr. Jonathan Finlay in CHLA, and to the doctor who did head radiation to her, Dr. Kenneth Wong. They are both amazing. They continue to review the MRIs and to compare it to previous MRIs every three months. The Schneider team also waits to give us the results until all doctors review the materials. It is a long week for us but at least I know that many doctors are looking at the MRI. The fact that it’s been two years from the oral medication is a good sign, but there’s never a guarantee.

In addition to the periodic MRI, we also do a hearing test, an eye test, and an endocrine test. We have a lot of follow-up because of the treatment’s side effects and we hope that it’s over but there’s never any guarantee that it is over. We continue to pray that it’s truly over. We see some long term effects from the treatments.

I have to give a general statement that we heard a lot of opinions throughout the process from all the doctors, and we had to, as the parents, do what we believe is right, but we didn’t know what was right. The doctors were in disagreement about the issues, so we had to make some tough decisions throughout the process.

For example, after we did the MRI, when we finished the chemotherapy, and saw that there were still spots in the spinal cord (before we started the radiation) some doctors said, that it may be scar tissue and not the tumor itself, so we did a biopsy, here in Israel to see what it was and then we found out that it is a tumor. Some doctors said not to do the biopsy, because you can’t do a biopsy for each and every spot, and if for example you do it for one spot which is scar tissue, but then the other spot is a tumor you would’ve missed the tumor. We decided to do a biopsy, from the main part of the spot, and we found out that it was a tumor.

There were arguments about this biopsy, about the radiation treatment and about several other topics. In order to deal with these decisions that we had to make, we had a peer group of our close family, I have a cousin who is scientist, and she helped me. We chose my father, my brother, and five-six other people that follow up with us all the time.

When I went to see all the doctors, it was five doctors in five days, I summarized every meeting with them, wrote everything they told me, and then I sent the entire summary to the peer group of the family and friends, the doctors in Israel, and the other doctors. I had everybody in line. Everybody knew what everyone else said, and everyone had all of the information. It was easier to make a decision with many people. We didn’t know if it was the right decision, but at least I knew that everybody knew what I knew. I think it really helped. I also didn’t forget what they told me. I think that the point that I was the connector between everyone is maybe one of the most important things a parent can do because many times the doctors aren’t communicating with each other.

Parents can stay inactive and passive, and do whatever the doctor says, but active parents, proactive parents, take steps to get things done. One of the things to do is to make sure that all the doctors communicate, and document everything. I think it’s really important. For the first eight months, when we were doing chemotherapy we were not very active by the fact that we made sure they give her the right medicine, and the right dosage of the medicine, and we were with her all the time, but we were not active in the parts because it was a protocol, and you had to go through the protocol. Before the protocol had started we made sure it was the right protocol, but that’s it.

My recommendation to parents dealing with situations like ours is to get prepared, meaning if you decide to go through with a protocol of the chemotherapy, it’s good. If you made a decision – do it, don’t look around. Follow the protocol, and hope for the best. Hope that when you finish going through the protocol you are done, and you can go home and be healthy and forget about it. But prepare for a situation in which something goes wrong, and you have to make a quick decision and you don’t have the time to do a research. Therefore, it would be wise if while you follow the protocol to make all the preparations that you can, find out which doctors are the right doctors to consult with, read information about the disease and its treatment, and sign up for newsletters from related health associations etc. Be prepared for the worst, and hope that you won’t need to use this information, but at least for the moment that you need to be active, which I highly recommend because even if you have the best doctor they don’t have the time to learn about your child’s disease as much as you have the time, patience, or passion to do it because it’s your child. I think it’s our duty to learn for our children. Sometimes you don’t have the capability to do it because either your English is not good enough, or mentally it’s difficult for you, so you can ask someone else from your family to do it for you because you have to be detached when you do it. When I went to speak with the doctors I said to myself I am speaking about a child, not my child. I cannot think about my child because sometimes you hear difficult things from the doctors.

When you go to speak with them they say everything, so you have to be prepared. I remember one time when I went to see the doctor I was so shocked, and because of that I was late to the plane. It is not an easy process. You can do it alone, or you can ask someone for help. My brother, for example, joined me at some of the meetings. You can bring some of your family, or you can ask someone else that you trust to come with you, but you also have to do it because otherwise you won’t get the whole picture.

So basically my suggestion to other parents that are starting the process now is:
Be active or proactive,
Get on top of things,
Learn about it so that you will be able to communicate with doctors,
and to get involved.

I don’t think that you have to be nagging the doctors too much or ask them to spend more time on your case because they don’t have the time, and they won’t do it. My experience is that doctors love to hear the information that you bring from other doctors. We are working with, and are being followed by Dr. Helen Toledano from the Schneider Children Hospital in Israel. She is great. Throughout the entire process she always told me that she learned a lot from the process, and that she kept all the information that we sent to her from the U.S. She wants to learn from our process, and then she uses the information that she gets with other kids. If she has something to say, she says it, if not, she doesn’t. Even though she’s a great doctor, she can’t do the same process that we had. She has dozens of other kids to take care of, and she can’t give all her focus to one child. You have to remember that she wants to do it, but doesn’t have the time. We are lucky that we can do it because we have the time to do it, and if we don’t have the capability to do it then we can use somebody else, but it’s something that is important. Also I think that cancer is an illness that is so complicated, and is so different from one form to the other that everyone has to learn about his or her own cancer because it is impossible for the doctors to have all of the information for so many different kinds of cancers because each type of cancer is a different disease. Each type of cancer requires different treatments, and different doctors. Therefore pediatric cancer is not one disease but many, and you need to learn about your specific type and sub type, and get all possible information and this is exactly the point. The age of the child is important as well because treating Medulloblastoma for a six year old child is different than treating a two and a half year old child. For example, if Gali was six when she was diagnosed then she would have started radiation immediately.

xxx

Regarding diagnosis: We know that it’s very challenging sometimes to diagnose cancer in children because you don’t connect the dots. Please take us through the first signs, you mentioned something with the eyes, and through the diagnosis process. How long did it take? How do you diagnose medulloblastoma?

In our case there were signs that we could see, and signs that we couldn’t see. There were two signs that we could see. One was the eye that went inside. It happened suddenly that her eye moved inside however this could’ve been for several different reasons. The second sign was that she was occasionally falling. At first we thought that this was a problem with her leg, but later we learned it was caused by the cancer. She was two years and four months when we started notice things. She was walking fine, and she had been walking since she was 11 months then suddenly she started to fall. It took about one week of falling before we thought that something was wrong. After one week I took her to the doctor to see if something was wrong with her leg, and then we saw the eye so I said maybe she falls because she doesn’t see very well. This caused me to go to the eye doctor immediately which was where we found everything. The doctor found the brain pressure and the tumor by doing a pupil dilation (when you enlarge the pupil), and she saw that there was pressure in the brain which means that something is wrong. She couldn’t immediately say what was wrong, and she said it’s either a brain tumor or something else, but I think she knew that it was probably a brain tumor. It wasn’t ocular pressure, but rather brain pressure that can be seen through the eye. It’s very important that in a case like this that you insist on doing the dilation because this is the only way you can see, and it only takes about 30 minutes until the pupil fully dilates. These were the signs that caused us to find the cancer, and that brought us to do something.

Following the visit to the eye doctor we went immediately to the Schneider Children Hospital in Israel. We decided to go there because I have a friend that works there and also my parents’ friends work there. So we went there, and a neurologist checked her. The neurologist also believed that the brain pressure was caused by a brain tumor. It was morning and about six hours later they did an MRI and told us that it is a malignant brain tumor. We had to decide if we wanted to stay there to do the operation or go to a different doctor because there are other doctors in Israel that operate on brain tumors.

So within a few hours from our visit to the eye doctor because Gali was falling we got the news that Gali had a brain tumor that needed to be operated on immediately.

You already knew that it was cancer, but you didn’t know what type of cancer, and I guess the doctors told you that it’s very urgent to do the surgery immediately, and now you as a parent, you started to ask questions: Where am I going to do it? Who is the best doctor? etc.
Due to the fact that it’s a brain tumor and it’s so critical, tell us a bit about this dilemma and how you made the decision that you made.

For us it was not a very difficult decision because we understood the urgency, and we said that it is very important for us to make sure she gets operated on immediately. We didn’t think about

taking her abroad for two reasons. First, we were too shocked to think about it. Second we saw parents there that already took their kid abroad, and they told us that the operation in the U.S. was not good, and they had to come back anyway. They recommended Dr. Michovic because he did a much better job for their kid. We didn’t have time, and we had to move fast. We also thought about the process after that, because you have to go through the operation, and then you have to go to the oncology department. So we asked my friend that is a doctor at Schneider, and we were told the following. She said that in Ichilov there is a very good surgeon, Prof. Constantini, but that the oncology department there is not as good as the one in Tel Hashomer, however, there aren’t any surgeons that are as experienced as Dr. Michovic, and in Schneider we understood that Dr. Michovic is great and that the department of oncology is also great. So we said it’s better to stay in a place where you have everything that you need. This was what we considered and this is why we decided to do it there.

There were three signs that we actually could have seen before, but didn’t. I don’t know if we could have seen them, but at least in retrospect we can. One was the fact that Gali was a very active girl, she went to all the parks, playgrounds, etc., and when she was two and two months, she stopped playing. I think a month passed from the time she stopped being active until we diagnosed her. I thought she stopped being active because at the age of two you start to get scared and everything, but that wasn’t the reason.

The other sign was that there were like two or three days that she vomited in the morning. I thought it was because she was still drinking the formula so I stopped giving her the formula, and then she stopped vomiting in the morning. It is also a very scary sign when you vomit in the morning. Since she vomited only two or three times it was not something that scared us. Vomiting in the morning is one of the signs or symptoms of a brain tumor in children and in adults. When you feel sick in the morning and vomit, it is a very bad sign. I didn’t know that beforehand. After I read about it, I understood, and when I stopped giving her the formula and she didn’t vomit in the morning I lost the chance to see it. It was about two weeks after the vomiting before we diagnosed her.

The third sign was that her head was slightly tilted.

So I would say that in Gali’s case the signs didn’t last long. Brain tumors are very aggressive, and when it grows, it grows fast. You can only see the signs when the tumor is big.

What are the methods of diagnosing medulloblastoma?

Medulloblastoma can be found by an MRI scan. If a tumor is found in the brain with the MRI a biopsy can be done by taking a sample from the tumor, or the tumor can be checked after the resection of the whole tumor to figure out what type of cancer it is.

Biopsies aren’t usually done to the brain because of how hard it it to do brain surgery. Usually Medulloblastoma is accessible because the tumor is located in the medulla which is usually accessible. If it’s a different type of brain tumor then it may be difficult to get to. In Gali’s case we only did an MRI of the brain before the operation, and only after the surgery we did an MRI of the spine, which was a mistake. I recommend to do both before the surgery, but I am not sure why the doctors chose not to do the full body MRI. I think it’s not in the hospital’s protocol here in Israel to do the full MRI. In the U.S. I’m sure it is part of the protocol. Only after they diagnosed it as medulloblastoma, and they knew that medulloblastoma can spread to the spine, did they do the full MRI. If it was Ependymoma or something else where you know that it doesn’t spread they wouldn’t have done the full MRI. So they wanted to identify the type of cancer before they did extra tests. I think that it isn’t a very smart thing to do even though it wouldn’t have had an effect on our choice to do the brain surgery.

Had they done the MRI before the surgery I would’ve known if the tumor was on the spinal cord before the surgery or not. Now that the MRI was done after the surgery it may have moved there during the surgery or it could’ve already been there. In many cases, also in ours, there is a challenge between the decision to do things quickly and to do all the tests possible, and in many cases during the surgery stuff happens, and you don’t know what’s caused it.

Brain surgery is very complicated, you decided to do it at Schneider with Doctor Michovic. Tell us a bit about the doctor and the surgery, how it went, the risks and the end result of it.

We met Dr. Michovic before the brain surgery. He came to meet with us in the middle of the night because they did the MRI arround 6PM, and we got the results which made us really scared so he came at about 10PM to meet with us and to explain to us what would happen. He was very calm, he said that he does brain surgeries daily, and he was very optimistic. He said that he believes that he can remove the whole tumor. He was certain that it was a malignant cancer, and he said that it’s either medulloblastoma, or ependymoma. Before he did the surgery we saw that he was very knowledgeable about everything, and then we had to get prepared.

She couldn’t have food for six hours before the anesthesia. After that they did the operation. It is very difficult. The operation lasted for eight hours. He told us that he had to scrape the tumor off of the stem of the brain because it was attached to it, and he had to do it very gently so as not to damage anything. Damaging the brain is the biggest risk with this type of operation.

After the operation it was difficult for her to start walking. When she finished the surgery there was a lot of fluid in the brain that had to be removed. She couldn’t stand the fluid so it was removed. Later she got sick with meningitis and then they had to take her to the hospital again to clean everything and to close it again. It took about one month before she learned to walk because she was very imbalanced. The part of the brain that was operated on controls balance in the body. Her eyes corrected themselves about a year after the surgery. She couldn’t speak after the surgery either. Before that she said a few words, even though she never really spoke much, but after the operation she couldn’t speak at all for at least a month. There was a regression in all of her development. This was a shock! I know about kids, her situation, that it takes them six months before they can walk, or they can’t speak, or they can’t move. I think Gali had a quick recovery, but it takes a lot of time, and you have to be patient.

After the brain surgery, Gali had a month to recover and then you started chemo in Schneider in Israel for eight months. Please tell us about the chemo treatment.

The protocol was the COG protocol, Children’s Oncology Group, for infants with medulloblastoma, infants meaning kids below the age of three years old. The protocol in Israel is similar to the one in the East Coast of the United States where they do the COG protocol. In the West Coast they do the head start protocol and I think they also do the COG in Europe. The head start and the COG protocols are very similar with the only difference being methotrexate. Methotrexate is a medicine that is more severe, and it can cause learning and other problems in the future.

In Israel we followed the COG protocol, but then we added methotrexate because we didn’t see a lot of progress with the spots on the spinal cord throughout the process. It’s a decision that was made during the protocol.

After about five months she finished the chemotherapy, and then she had the mini stem cell transplant which took about two months. She had a stem cell transplant because she had a very high dose of chemotherapy, but they used her own blood, and her own stem cells. They harvested the stem cells before the chemotherapy, and then they gave her the high dose of chemotherapy, which killed the stem cells, and she then needed a set of three mini transplants. We had to go to do the transplant for three times, and each transplant lasted for a week. It was not like a whole month inside the room and everything like in the regular transplants.

We did some MRIs during the protocol, and saw progress, but not much. After the transplant

we couldn’t see any change from before the transplant. The spots were the same size, and so the mini-transplant didn’t help, but they didn’t grow as well. After that it was stable, but we didn’t know if the spots were scar tissue or something else, or the tumor itself, so then we did the biopsy.

Before that there was a decision not to use radiation because in Israel under the age of three they tend not to radiate. When I went to speak with the doctors everybody told me that they don’t give radiation treatment for the brain and spine for anyone under the age of four because it would negatively affect the brain, and they would lose their ability to be independent. We decided we don’t want to do that to her.

After the chemotherapy we flew to the East Coast, and to the West Coast, and we consulted with a few doctors, and the decision was to give a very low dose of radiation even though she was less than four because we had no other choice. Professor Finlay was the one that told me that I had no other choice. I then understood that she would have to get the radiation therapy because Professor Finlay is usually against radiation in infants. He said to me, if I tell you to do radiation because there is no other choice then you have to do it. He said that he will make sure that it will be the lowest dose possible. He said that if you don’t do it to her then it will not end well. I then decided that him and Dr. Kenneth Wong, head of the radiation oncology department, would do the radiation treatment. The radiation treatment lasted for two and a half weeks, and it was done every day except for Saturday and Sunday. It’s all done under anesthesia so every day she had to be under anesthesia in the morning, for them to do it.

How did you deal with your insurance?

Our insurance covered the radiation and the clinical trial even though the radiation could’ve been done in Israel. The insurance didn’t cover the clinical trial, the clinical trial done at MSKCC.

The HMO covered the clinical trial and the private insurance covered the radiation. We had private insurance and an HMO, but they did not want to pay for anything, but we told them that in the case of Gali’s tumor that there is just as much chance of being cured associated with the clinical trial and the protocol. There was no difference between doing the clinical trial and the regular protocol because there was no guarantee that any of them would work. If this was a treatment in Israel then they would tell me to go do it, and that your daughter will be fine, and I would have done it, but that’s not the case. I explained that I didn’t have any other solution, because an experiment is not covered by the insurance if there is an alternative treatment that works, but we didn’t have any treatment that we knew would work. The success rate of the protocol wasn’t high in Gali’s case, and it didn’t work anyway.

For the radiation we explained that in Israel they don’t have a lot of experience, and that they don’t believe in giving a low dose of radiation.

Also, I truly believe that Professor Finlay was our only method of doing the clinical trial because he was previously the head of the brain tumor department at MSKCC. We had to go to the next stage of the treatment for Gali after the chemotherapy. We explained that the only option was to go the United States. Eventually they agreed with us.

About The Clinical Trial

The clinical trial that we used is called “3F8”, with Iodine I 131 Monoclonal Antibody 3F8. It is used for treating patients with Central Nervous System Cancer.

The basic idea of the clinical trial is that there are cells that they know how to find tumor cells and carry tumor-killing substances to them without harming the normal cells.

In our case, before Gali could get the treatment they had to put a device called an Ommaya in the brain. The Ommaya, is like a portacath, but in the brain. Through the Ommaya they inject the medicine or antibodies which travel to the brain fluid which later travels to the spine and attaches to the tumors. They also had to do a test to see if fluid in the brain flows to the spine because sometimes it doesn’t flow, and if it wasn’t flowing between the brain and the spine then the treatment wouldn’t work. The Ommaya insertion was also a brain surgery which could’ve been done either in New York or in Israel. We had an agreement with the insurance company to do the Ommaya surgery in Israel because it would have cost $50,000 to do it in the U.S. Remind you that at that point in time we still didn’t know if we would even get accepted to the trial.

So we went back from the U.S. to Israel to do the Ommaya surgery which was done by Dr. Michovic. He knew how to insert the Ommaya, but it wasn’t the best device, and he didn’t put it in the optimal location. After that we went back to New York to do the clinical trial. I didn’t know there was a problem with the Ommaya until after we went back to New York when Gali got meningitis, and they had to remove the Ommaya in New York. They then replaced the Ommaya with a new one and inserted it correctly. Only then I understood how much they don’t know in Israel about the Ommaya, and where it has to be put.

To this day we didn’t remove the Ommaya after we finished the treatment because the removal would be another brain surgery. It was put in a very delicate place and it isn’t visible.

So the lesson is that we should’ve stayed in New York and done the Ommaya insertion there, but we had a cost issue, and we had no choice. We could have paid it, and we could have argued with the insurance company, but at the end we would still end up paying for it because they did the Ommaya surgery in New York and not Israel.

The insurance didn’t cover the second Ommaya surgery, the meningitis, and the hospitalization due to the meningitis. Two weeks in the hospital exceeded the maximum amount of money the insurance would pay for. After that we tried to get Medicaid in the United States, and I had to work a lot to get the approval for it. It took about a year to give them all of our information about our bank accounts, salaries, and everything else they wanted from the last five years. It took me a lot of time to get everything for them; all the receipts, all the visas, all the bank accounts, and everything else. Everything had to be in English and in the end they approved it. They approved $100,000 for Medicaid to pay.

My experience here is that when you have to do something that is not usually done in Israel, like the Ommaya, just don’t do it. Only do it with a doctor that is highly experienced with it. This is of true not only for Israel, but for any country, or for any doctor or hospital where they don’t have the same experience because as we know, especially in pediatric cancer experience makes all the difference.

Side effects and long term implications

There were possible side effects or long term implications for everything that was done.

For chemotherapy especially you have to pay attention to the possible short term risks; hygiene, making sure you have the right dose, and making sure you have the right medicine because of the lowered amount of white blood cells. It’s very important to pay attention to everything. These are the effects of chemotherapy, and everybody knows about them.

I also heard that the bone marrow transplant has an effect, but it isn’t as bad as chemotherapy.

For the radiation the risks are different. When you do the radiation treatment to the brain and the spine there are two major long term risks. One is damage to the brain. It progressively lowers the IQ level slightly. If you do the high dose of radiation at the age of three then the IQ can be decreased by I would say 20 to 30 points. If you give a lower dose of radiation than there is less damage.

When you do radiation on the spinal cord it can change the body, and the spinal cord. The spine will be shorter than it should have been and it also decreases the height that you can grow to.

So these are the risks, there is not much to do about it.

To summarize, the side effects of radiation to the brain would be brain development, IQ, and maybe functionality.

The side effects of radiation to the spine would be development of the spine, skeleton, and maybe being imbalanced. Another side effect of the radiation is that Gali doesn’t have a lot of hair.

These are the risks, and there is not much to do about it besides knowing about them.

What we did for Gali for the past years is that we gave a lot of help with cognitive issues, speech therapy, occupational therapy, and art therapy. Any therapy that you can think of is very helpful because when you put the brain to work, the cells that were damaged during the treatment can grow back.

We are constantly trying to stimulate Gali’s brain because it is like a muscle and if you train it, it will be able to develop. Now she is six, and she just started first grade. Even though she regressed she didn’t regress too far.

She is not at the top of her class and I don’t think that she ever will be at the top of her class because of the regression, and her emotional problems. She’s not like kids her age cognitively. It’s not easy. Due to the fact that she doesn’t have much hair and she has scars and she’s a bit smaller, she looks a little different. She has some emotional problems because of her lack of hair, smaller size, and from going through everything that she went through. She still remembers, and still has medical follow-ups. She’s not an ordinary girl, and she knows it which makes it difficult for her to accept everything.

Her studies studies are difficult for her because she needs to put in a lot of effort to study. It’s not an easy process, but we see a lot of progress. As much as we put more time into it, and we bring good people to help her you have to give a lot of support to a kid like this for emotional, and for cognitive progress. Her grandmother and everybody around her comes to help her. It’s not like you can say okay, the illness is gone, she’s now healthy, that’s it. If you want her to be able to have her own life when she grows up, then you have to give her everything she needs to do it, and this is done through the constant help that is given when she is ready.

For the first two years when she was in kindergarten, we did not put any pressure on her, and we just let her be a very happy and smiling kid. She’s happy, she’s very friendly, and kids love her which is more important than the cognitive issues, but to get prepared to join school is important. We thought about postponing school for a year, but we didn’t because she wanted to be like a normal girl, and start school with kids of her age. We said that it’s better to give her more confidence to go to school, and to be with her friends, even though she won’t be the smartest kid in the class, which is fine.

If you could go back three or four years, what advice would you give to yourself a week after the your daughter was diagnosed?

I think the fact that you have to be proactive is the advice that I give to any parent that calls me. I would also say to constantly believe. It’s possible.

{/sliders}

ChondroSarcoma (19 years old girl)

Daniela Tendler, 24, shares her 5+ years journey fighting cancer. Diagnosed with ChondroSarcoma – a very rare type of cancer, Daniela has overcome 3 relapses and now battling relapse #4, going through treatments which are not part of the familiar protocol. Watch this video to learn more about the latest and greatest in ChondroSarcoma treatment.
If you have information that can help Daniela (new treatment, best doctor etc.), please contact us: MyChildCancer@gmail.com

At MyChildCancer.info we interview parents and young patients from all over the world in order to share life-saving information on rare types of cancer.

Medical Centers

For sources of information about a specific type of cancer please go to our cancer types page

LEADING HOSPITALS & CANCER CENTERS

In the United States

Boston Children’s Hospital, Boston, MA

Located in Boston, this award-winning hospital is one of the best pediatric hospitals in the US. You can visit their Dana Farber Cancer Center for more information about your child’s cancer and suggested treatment.

Children Hospital of Philadelphia (Chop), Philadelphia, PA

Located in Philadelphia, this is one of the best children hospital on the east coast. Visit their Pediatric Cancer Center for more information about your child’s cancer and suggested treatment.

Memorial sloan Ketering Cancer Center (MSKCC), NYC, NY

Located in New York, USA, this is one of the leading cancer treatment and research centers in the world. Visit their Pediatric Cancer Care for more information about your child’s cancer and suggested treatment.

M.D Anderson Cancer Center , Houston, TX

Located in Texas, USA, this is one of the world’s largest cancer centers. You can visit the Children Cancer Hospital for additional information on Pediatric cancers and treatments.

St. Jude Children’s Research Hospital , Memphis, TN

This Children’s Research Hospital is internationally recognized for its pioneering research and treatment of children with cancer. It is one of the best pediatric cancer hospitals in the U.S and is the first and only National Cancer Institute designated Comprehensive Cancer Center devoted solely to children.

St. Jude is the only pediatric cancer research center where families never pay for treatment not covered by insurance: No child is ever denied treatment because of the family’s inability to pay.

*******

National Cancer Institute (NCI)

As part of the National Institute of Health (NIH), this institute’s website offers a lot of information on the different types of cancer and the available treatments for them, nationwide. In addition, there are many links to other information centers.

If you heard about a new/experimental treatment or drug and you are not sure whether it is under an official study or not, you can visit the NCI’s Clinical Trials page and look it up. Remember that an “experimental treatment” may use as a cover for a doubtful activity purposed to get money from the families without having any medical basis or any formal acknowledgment form official institutes.

In the NCI’s website you can also have a live chat with the institute’s advisors.

In Europe

Manhaim radiation center

In Israel

Schneider Children’s Medical Center, Petah Tikva, Israel

Schneider Children‘s is the only comprehensive, highly specialized care hospital of its kind in the country and in the Middle East, dedicated exclusively to the well-being of all children and adolescents

Dana Dwek Children’s Hospital (at Tel Aviv Sourasky Medical Center), Tel Aviv, Israel

The Pediatric Hemato-oncology Department at Dana’s Children Hospital specialize in Pediatric bone marrow transplantations. They perform a full array of bone marrow transplants (BMT) for children, sourced by self-donated (autologous) marrow and peripheral blood donations and donations from a related or unrelated donor (allogeneic).

 

 

NeuroBlastoma (9 months old boy)

This testimonial is about a Neuroblastoma first diagnosed at the age of 9 months old. Today this boy is 4.5 years old and fought successfully 2 relapses.
This is an amazing testimonial from the relapse master, Muli’s mom.

In this interview, MyChildCancer’s founder, Oded Grinstein, is interviewing Myriam Safrai from Israel about the treatment and challenges her family went through with her son’s Neuroblastoma cancer. Her son, Muli, was diagnosed with Neuroblastoma when he was 9 months old. After early signs were misdiagnosed for a couple of months by the family’s pediatrician, he was diagnosed with a stage 4 NB. On the time this interview was made Muli was 4.5 years old, with no evidence of cancer however learning from past experience his parents were just about to start yet another clinical trial in an effort to prevent a third(!) relapse.

Watch the amazing story of Muli. 4.5 years old including 4 years of treatment, 3 different types of treatment 3 different locations on the globe, 2 relapses and 1 amazing and forth-thinking mother.

This interview is part of MyChildCancer’s mission to extract hidden life-saving information and make it easily accessible to other parents all over the world. Because knowledge saves lives.

Text version:

 

–>Please, state your boy’s name, age, and type of cancer.

Muli, his real name is Shmuel, was diagnosed when he was one year and four months. Now he’s four years and six months. He was diagnosed with NeuroBlastoma high risk, with MYC amplified.

Our story in a Nutshell:

We did all the front-line therapy in Israel, which is the SIOPEN (SIOP-Europe) protocol, after he had the first relapse. He gets two more cycles of chemotherapy, TVD (Topotecan, Vincristine, Doxorubicin) X 2 and TVC X 2 cycles.

He had a huge fungal infection, so we stopped all the treatment, and it was recommended that we do a big trip and enjoy the time that we have left. We moved to Hershey in PA (Pennsylvania, U.S.A) for a medical trial. It was a vaccine trial, with Decitabine. We were in Hershey for six months, and he was still clear after the trial.

I was flying back and forth to Michigan, for the DFMO trial of Dr. (Giselle) Sholler, for nine cycles. Then, he relapsed again, and he had two cycles of MIBG in San Francisco at UCSF Medical Center without any chemotherapy or any other drug, just MIBG, and he was NED (No Evidence of Disease) after the first cycle. We did the second round as a consolidation.

Now, we are in Memorial Sloan Kettering and the purpose is to do the antibodies – one course at least, and after that to do the vaccine trial with Dr. (Brian) Kushner.

–> Could you please tell us about the first signs, and the diagnosis process?

Muli had Anemia. They tried to treat it with Serotonin but it didn’t work. He also had a little bit of hypotonia in the legs and we took him to the pediatrician who said everything was fine. One night, it was difficult for Muli to sleep so I took him to the emergency center because I had a bad feeling. I was feeling something in my stomach, and I am a doctor, I thought he had an infection. When we did the ultrasound, they saw a huge mass in his abdomen. Afterward they did an x-ray and everything began. We did an MIBG and we saw many spots metastasized everywhere in the bone. And so, we started the protocol.

–> You mentioned that he had a pre-condition – Anemia. What were the first signs that you can now relate to the cancer? How long between these first signs and the actual cancer diagnosis?

I think the anemia was the first thing. He was also FTT (Failure To Thrive) so he was not growing so well. I have been to the doctor many times, and I told him everything that he was eating and he said that everything was fine.

So, I think the failure to treat the FTT was the first sign, there was also the anemia. And he also had the hypotonia in the leg, which today we know was caused by the tumor which was compressing his core. These were the three signs that we had. And it’s not that we didn’t notice this signs however it took months to diagnose. We went to doctors but they failed to diagnose. I have been to the doctor just 3 days before I had to take him to the E.R., and he said everything was fine.

–> How old was Muli then?

One year and four months when he was diagnosed and started treatment. Maybe six or nine months when the first signs were noticed. I can’t be sure because it was a growing process.

–> So once you took him to the E.R. and they did the scans, how did they identify the cancer, and how did they identify the type of cancer?

They made an ultrasound first of all and they reviewed the mass. The differential diagnosis for a mass in the abdominal at this age was to find what it is. So they did the biopsy, for two purposes: they needed tissue before the final diagnosis, and they needed also to decide if it’s a high risk or an intermediate risk, since before the age of one year and six months it matters if there is MYC amplification or not. Muli had MYC amplification.

–> You have mentioned that you are a doctor. Most of the people who will see this are not doctors. Please explain about the disease, the different subtypes – you mentioned MYC: is it rare?

In Neuroblastoma there are five stages: 1, 2, 3, 4, and 4S. If a child is diagnosed before the age of one year old, despite if he has metastasis, he would usually have a very good prognosis, and it would be stage 4S.

After that age, the child is usually stage 3 if it a local disease or stage 4 if the patient has a metastatic disease. Muli had metastasis, so he was stage 4.

After that doctors need to decide if he’s high risk or intermediate risk. There are few things that will help to decide if the child is high risk, which is a less good prognosis, or intermediate risk. Within stage 3 and stage 4, and you need to decide if it’s high risk or not. The age is a factor: if more than one year and six months, it’s high risk.
Now within the high risk, you have an equivalent of “very high” risk, and doctors need to look for the factors that will make the prognosis more difficult. One of the most important factors is the MYC mutation: The MYC mutation is a mutation in a specific chromosome. When doctors see in the gene of the cancer cell that amplification exist, and if they see that there is more than ten copies of the gene MYC, it’s “very” high risk.

–> So MYC is a part of a gene?
Yes. And you see the genome and you see that there’s an amplification of the genome of this part of the mutation. Muli had so many that it was very clear.

–> But Muli was under the age of 18 months?

Yes. It was the only good thing, but if you take into consideration all the factors and see what is most important in the staging, the thing that has the most accurate prognosis is the MYC. If you have MYC, it’s not good.

–> So Muli was a year and four months when he was diagnosed, and he had metastasis, so it was stage 4, and he had MYC amplified?

Yes: Neuroblastoma, stage 4, high risk, MYC amplified.

–> Tell us about the SIOPEN (SIOP-European) protocol.

The SIOPEN protocol has five parts. (1) First of all, eight rounds of chemo (2) Afterward a surgery. (3) Then you have a self-bone marrow transplant. (4) After that you get radiation. (5) The fifth and last step is immunotherapy.
For us, it was not too bad. Muli went through the whole protocol very fine. He was clear of all the metastasis after the eight rounds of chemo and before the surgery.
He had the surgery and he recovered very well. After only one week we were at home. After that he had a bone marrow transplant that was very fine. Twenty-six days we were in the isolation room but it’s very short. It was hard, but it was fine. Then he had radiation. It was fine – he was just tired. For the radiation he needed anesthesia every day because he was small. And lastly we had immunotherapy.
For that last part we had two possibilities for immunotherapy; CH18.14 with or without I02. We get it with I02, and it was very hard. Muli had a huge reaction the first night and he almost died. He had a capillary leak, which means that fluids are not staying in the blood vessels and are leaking into the small tissue. The blood pressure dropped to 30 or 50 and he stopped giving urine. We stopped everything. They took us to give him dopamine for the blood pressure, and then we decided instead of giving him the infusion of the antibodies in eight hours, as it should be in the protocol, we gave in about twelve hours. It was also hard and we have done the treatment in the ICU. We finished the first cycle in the ICU, and after that we moved to a different hospital that was more accurate for this kind of treatment and we did it, as I said, in twelve hours. It was hard. It was a hard treatment.
It was very painful. Muli all the time had some complications that were manageable. He had once a Pericardial effusion (fluid around the heart), which was hard. He had pneumonia once. Each time something else, but it was very manageable. for example, his weight was fluctuating: he was keeping so much of the fluid, he gained like 3 kilos in one week. I needed to buy him new clothes towards the end of the treatment, because all the clothes were too small. After that he would just urinate a lot and one week after we got back the Muli that we knew, the small one. We did everything we could. He had the Acutan as well, which makes the skin very dry, and we finished the protocol. We were very hopeful and very happy.
In the last scan, before we were scheduled to move to the follow-up part, we saw a relapse: one relapse in the Humerus bone. He relapsed.

–> The protocol that you were treated under, is it an Israeli protocol or a European protocol?

It’s a European protocol. We had few randomizations, no one immunization. Back then you had two options of drug / chemotherapy before the transplant. We received the CEM, but it was not the best. The BuMel is the best, and not everybody is getting the BuMel.

–> Where were you treated in Israel?

Jerusalem Hadassah Hospital.

–> And then the second hospital?

Schneider Children Hospital. In Hadassah Muli was the first kid to get the immunotherapy.

–> So, the immunotherapy is an experimental treatment?

Everything is experimental. Every protocol has some randomization so it’s all experimental because you have the immunotherapy with or without the IO2. You have few randomizations to the protocol.

–> How many children do you know in Israel that got the same treatment as Muli?

Well it depends if we are referring to all exactly the same or just the same SIOPEN protocol. Muli was I think one of the last SIOPEN chemotherapy before the transplant. Today everybody is getting the BuMel, but I know many children are getting the SIOPEN treatment.

–> Who is the best doctor you could recommend parents in Israel to visit?

We have been in two hospitals in Israel: Hadassah and then Schneider, and then we’ve returned to Hadassah.
I think the doctor who knows the most about NB is the head of the Children Oncology department in Schneider. Dr. Itzhak Yaniv. That being said, we decided to go back for treatment to Hadassah in Jerusalem since it was easier for Muli. the protocol was the same, and one can always take a second opinion. We got a second opinion with Schneider at the beginning, but I don’t think there’s much different between these two hospitals.
The treatment in Hadassah was much smoother for us. The day care was much more convenient for us, and the hospitalization as well.

–> How long was the treatment in Israel, from the beginning and until the relapse?

One year and one month.

–> After one year and one month of treatment in Israel, there was the relapse. Muli was, by then two years and five months old. What’s next?

We saw the relapse and I wanted to do the MIBG therapy because I read about it a lot.
There was a misunderstanding, and we didn’t do the MIBG therapy in Israel because it’s not a treatment we could do in Israel, and we didn’t really know how to get it. They gave him chemotherapy – two TVC and two TVD, plus local radiation, and Muli was clear at the end of the treatment, so Muli was not approved for the MIBG therapy.
When you are clear, there’s a paradox, because everybody knows that you shouldn’t treat a child with no evidence of the disease (NED), but it doesn’t mean that for 100% there is no disease; it just means that you can’t see the disease, so it’s like you have minimal disease and it’s better to treat when you have minimal disease. Everything is more effective. The problem is that the clinical trial, most of the clinical trials are usually open for childrenwith signs of disease, so there are very few treatments that you can have at this stage.
For Muli we knew that the prognosis was very bad. Muli was high risk with one relapse. In all the papers, the five-year survivor rate after one relapse of Neuroblastoma practically zero. I wanted him to get the treatment, so we went to get the treatment that was open for us – which was the vaccine therapy, in Hershey hospital in Pennsylvania U.S.

–> After the first cycle ended and they saw the relapse, what were the considerations, and how did you make the decision to go to Hershey, from all the options? Where did you apply, and what was the process?

Well there was not so much applying, because Muli already got antibodies. We had two options: a treatment in Sloan-Kettering (NY) and the one in Hershey (PA).
The CH14.18 that he already got during the treatment was an anti GD2 (antibody treatment). The GD2 is something on the cell of the Neuroblastoma, and the antibodies were an anti GD2. The Sloan-Kettering antibodies were also anti GD2, so we went for a different approach, in Hershey.
There weren’t so many options because Muli was clear of diseased, and for the research purpose it is always better if you have an active disease with evident signs, so you can measure everything.

We were recommended by our doctors to stop everything and enjoy the time we have left, so we didn’t have a lot of choices, but we decided to continue the fight and we moved to Hershey.

–> Was there any other place in the world that you checked?

We checked, yes, we checked a lot of others, but there was no trial for children without evidence of disease.

–> All the other places were accepting only kids with evidence?

Yes. You may have more options when you have evidence, but these clinical trial are closing and opening all the time. I wanted to try a vaccine trial for him, but it was closed.
It’s a matter of timing. Timing plays a big role in the options you get to choose from.

–> For better or worse, the only option you could find was in Hershey.

Yes.

–> Did you check also other countries, other than the U.S?

Yes. We checked in Europe for MIBG therapy, but we already got the European protocol, so what they recommended was chemo. Muli had the fungus infection during his treatment, from which he almost died. He had three surgeries in four days to treat it, and a lot of treatment after that, and we could not give him any more chemo, so it also closed some options for us. Chemo is not curable for Neuroblastoma high-risk relapse.

–> Is the Israeli protocol equivalent to the European protocol?

Yes.

–> And this is why it made less sense for you to go to Europe?

Yes but also because in Europe there are not so many clinical trials like in America. He could not get any more chemo at that time and the European protocol is all about chemotherapy.

–> Tell us about the second phase in Hershey: after you got there, how did you get along, how did you make the connection? Tell us also about the treatment itself.

The bureaucracy was very fast. Hershey was not used to having international patients; I think Muli was one of the first. We got a paper saying “for the Israeli patient”. It was funny — there’s no international department in the hospital or anything.
It was very, very fast, and they were very, very nice and helpful, and when we decided and they checked everything and say that we can only after we were there with Muli. For ten days, me and my husband, we made a peripheral blood, like before the bone marrow transplant, they harvest peripheral cells. We take the cells and after we go back to Israel and for three weeks and they prepare the cells for the vaccine. After we move, we have a daughter, we move together, we lived in the Ronald McDonald house for five and a half months, and Muli gets the treatment.
It’s four courses of treatment.
There is one week of chemotherapy, low-dose Decitabine chemotherapy, two injections. In weeks number 2 through 4 it’s only blood tests. Everything was outpatient, unless he had a fever or neutropenia or something, but it’s not lowering the count so much.
It was for courses like this, and we had one delay because the count was too low to begin the Decitabine.
It was fine. It was an easy treatment – only the shot were not so nice.

–> So it was five months?

Yes. Roughly between the age of three and three and a half, approximately, because he had chemotherapy in Israel. After they finished and after they saw the relapse, they gave Chemotherapy — two TVD and two TVC. Plus local radiation.

–> Local radiation. That was after the relapse?

Yes. Then, he was clear, and then we got to the Decitabine treatment.

–> Tell us a bit about the treatment in Hershey. Was it biological? How did it work etc.

There’s two parts. There’s the Decitabine, the chemotherapy, with a very low dose, which just felt like fine-tuning of the white cells and red cells to increase the immunity response. They took some cells from Muli and they worked with them in the labs, and we injected them back into the body. The body does not fight these cells because it’s cells from the body, and it showed in the lab that the cells fight against Neuroblastoma.
For us the treatment was good and NED – almost six months after, he was continuing to stay clear. For us, it was very good; we were very happy. It was hard to think that he will still be clear. It was good.
He was at the age of three and a half when he finished.

–> The tests, what are the tests they did at the end?

We did MIBG in the middle and at the end, and we saw there was no relapse.

In the MIBG Test, as oppose to the MIBG treatment, they inject something radioactive. The cells are absorbing it – it’s radioactivity plus iodine, and the Neuroblastoma cells absorb iodine. After that the child is lying in the machine and a radioactive sensor scans the body and you can see if there are any spots.

–> It’s like PET-CT?

It’s exactly like PET-CT, but PET-CT is with glucose and the Neuroblastoma cell is avid to iodine. So MIBG test is like PET-CT, but they switch the glucose with iodine.

–> Did you have insurance for this clinical trial?

NO. We paid out of pocket in Hershey Medical Center, and I think it’s important for people to know the costs, out of pocket. The Decitabine treatment was almost $60,000.

–> That’s for the six months in Hershey?

Only the medical treatment, yes – not including flying back, hotel, food, car etc. Only the medical treatment. All the other flights in the U.S, the visits, the meetings with the doctor, we had to pay for each of the meetings.

–> Why was the Hershey treatment not covered by your insurance in Israel? Everyone in Israel has medical insurance from the state, and I assume that your treatment in the Israeli hospitals was all covered. Why wasn’t the Hershey treatment covered by the insurance?

Because it was a medical trial, and the only thing in Neuroblastoma that we knew that the medical insurance in Israel would cover only a surgery in the case it cannot be done in Israel, or MIBG treatment. At that stage, Muli was not a candidate for the MIBG treatment because he was without any evidence of disease.

–> After five months in Hershey, the MIBG test showed no evidence of cancer. What’s next?

After the treatment in Hershey, before we came back to Israel, I wanted Muli to be in some of the computers in the States because of bureaucracy. During the first relapse, the time it took us to get any answers was too long and I didn’t want to spend any more time, if something should happen again.
We got a second opinion meeting at CHOP (Children Hospital Of Philadelphia) with Dr. John Maris. He had a medical appointment there, so he was in the computer already.
Also, I sent many e-mails to the head of five of the most important doctors for Neuroblastoma who I knew about.
We also went to UCSF Medical Center in San Francisco. We flew all the way back there because the head of the entire COG (Children’s Oncology Group) was there.
It was a thing that was very important for us to do, to try to skip all the bureaucracy, in case there’s going to be another relapse because in Israel where we live, if there’s a relapse, you speak to your doctor and the doctor speaks to another doctor and it takes time which we don’t have. So now, if the child is already in the computer, you can speak directly to your doctor which is overseas.

–> Amazing; genius! For one visit to the doctor, you’re already in the hospital’s system. You now know the doctor, and from now on you have a direct channel of communication. Wow!

Yes, it’s a non-writing contract. Your child is a patient of him, so you can be in contact with him.
It took almost two months to have the appointment in CHOP, and one month to the appointment at UCSF. It’s time that you don’t have when your child relapses. It’s completely different to do it when you have time. Also, I sent a few emails and I had some calls with other doctor. I knew that there’s going to be a treatment which was about to open with Dr. Sholler for maintenance treatment. I was looking for a magical drug that will keep Muli clear, because Muli is without any evidence of disease.
So I was at the medical conference in Texas, and I spoke with Dr. (Giselle) Sholler from Michigan, Grand Rapids. Helen DeVos Hospital. We saw that the trial was not yet open but I was in touch with her, and ten days after I came back to Israel I flew with Muli to Michigan to be with seven kids to be enrolled in a new treatment of maintenance for Neuroblastoma, the DFMO trial in Grand Rapids, Michigan.

–> Thanks to your visit to the conference in Texas..

Yes, I was there because I knew they were going to speak about the DFMO trial. It’s the reason I went to the conference, and to meet with her and to know her. I wrote her an email before.

–> You established contact, and then once you finished the tour in the U.S, you went back to Israel and ten days afterward you came back to Michigan for this trial.

Yes.

–> Tell us, please, about the Michigan chapter.

The drug is called DFMO and is for maintenance. The purpose is to prevent any relapse. It’s a drug that you take twice a day orally.
One course of treatment is one month, and every three months you have an MIBG scan, a CT scan, and any other special tests you may need.
The periodic tests (blood etc.) you can do in Israel however you need to be in Michigan to get the drug because they cannot ship the drugs overseas. They need to see you, check on the kids, and after that you can fly back with the medical drugs. This treatment was very easy on Muli. We didn’t see any side effect.
After nine months he relapsed, so we stopped the treatment. Usually, the protocol for this treatment is two years.

–> Where was the relapse?

There were two sites of relapse: one in the first rib: a bony relapse with a little mass around it, and one in the leg. Only bones. It metastasized in the bone.

–> Who manages the DFMO?

Dr. Giselle Sholler in Van Handel Hospital in Grand Rapids, Michigan.

–> Were you happy with the treatment in Michigan?

Yes, very happy.

–> What happened after the relapse? I assume the trial in Michigan had to stop?

Yes. Before the relapse we were always thinking and discussion what we will do in case of a relapse, because we knew that when the relapse happens you don’t have time. So we already knew and the doctor knew as well that in case of a relapse we want to do an MIBG therapy. I’ve been in touch with CHOP, where my child was already in the system, and with UCSF. The treatment in both hospitals is similar so we wanted to go to CHOP because it’s much closer; Philadelphia to Israel than San Francisco, but the bureaucracy there was too long. So eventually, after one month we began the treatment in UCSF (University of California, San Francisco).
In CHOP the bureaucracy took too long. They asked for many things and it was just.. We didn’t have the time to spend, so we flew to UCSF. It was a bureaucracy decision between the hospitals because the treatment would have been the same.

–> How long have you been in Israel before you went to UCSF?

One month. He got a little dose of some chemo just to prevent it from spreading anywhere else. We also needed to harvest stem cells since in order to make the MIBG therapy, you need to have stem cells.
If you want to make it a full dose of MIBG therapy, you need to have bags of stem cells, so we harvested some stem cells. We’d already harvested in the past, but after MIBG therapy, you cannot harvest any more stem cells, so we harvested another bag of stem cells. And then we flew to San Francisco to do the MIBG therapy.

–> Tell us a bit about the MIBG therapy please.

The MIBG therapy is like the MIBG scan, but it’s a much higher dose of radioactivity, so it just will burn the disease. It will go to the disease and burn it. The child is radioactive after the infusion so you cannot be with him for about one week. He’s in a special room and there’s a shield, a special led shield. If you need to help him with urinating or changing diapers or anything else you can go to the other side of the shield for a short period of time but normally you would stay outside the room. It was not an easy week for Muli, but thanks to an iPad that we got him it was fine. If you have a child that doesn’t know how to use an iPad and you are about to do an MIBG therapy, teach him how to use an iPad! Because we wrapped it a lot and he was just playing with the iPad for hours. It was very good.

–> So, the MIBG treatment is basically trying to burn the cancer cells by identify them with the iodine and then burn them with the radiation?

Yes. They have the iodine plus radiation. They inject them together. It’s going everywhere in the body, but it only stays in the tumor.
It’s basically a radioactive treatment. The tough part about MIBG treatment is that the kid is radioactive and you cannot get next to him. He was for almost a week alone in a room. Parents can go back and forth for short periods of time. They don’t do the treatment if you don’t have two caregivers. We took three caregivers: I, my husband, and my mother-in-law. When you are three caregivers you can win more minutes inside, because everyone has an amount of minutes. The hardest part is only for the first three days, after which you can basically go back and forth. It’s not so radioactive;
All in all the treatment was fine. It’s not a painful treatment.

–> How many cycles of MIBG treatment?

You can do two MIBG cycles. After the first one we have done we saw that Muli was NED, however we decided to do another one. We weren’t sure what to do, but every doctor that we asked told us that if the disease responds now for MIBG, it’s not sure that it will have a similar influence in the future however on the flip side it may be good to ‘keep’ the second round as a backup measure in case another relapse happens.
We were certain that there were cells that we didn’t see, and decided it would be better to give him another cycle and try to kill it. So we did another cycle.

To summarize the UCSF chapter: we have been for one month in San Francisco. Starting with the bone marrow biopsy, CT scan and MIBG test for the purpose to get into the trial. When Muli was in, we did the trial. It was one week in the room. Then we waited for two weeks to get the stem cells back in America. We chose to stay there to get the stem cells in San Francisco because in Israel, nobody gets MIBG therapy and we didn’t want to fly with a child overseas if there was any allergic reaction, any side effects – we wouldn’t have known what to do with him. After they gave him the stem cells back, we flew back to Israel where we did blood count twice a week.
In Israel he gets platelets and whatever else is needed. After another a month and a half we flew back to UCSF and we made an MIBG scan (Not therapy) and we saw that there is no disease left, we decided then that we will do another cycle of MIBG therapy to ‘consolidate’. Again, we stayed one month, and we flew back to Israel where he gets blood count, platelets and whatever he needs.

And now we came to Memorial Sloan Kettering Cancer Center (MSKCC). We made the scan on purpose to get him into another study, and we saw that Muli is still NED 🙂

–> How old was Muli when you finished the second MIBG?

Four years and four months.

–> The MIBG treatment; who was running it and what can you tell other parents about this treatment?

Dr. Katherine Matthay was our doctor. A very, very good doctor, very nice. We stayed there in front of the hospital in the family house. It was very nice because it was just in front of the hospital so it was easy for us to go back and forth.

–> Tell us a bit about the Memorial Sloan-Kettering chapter.

We just began, so we don’t know a lot, but we hope to do the mouse vaccine or mouse antibody, one course, and see if we can treat the HAMA (Human Anti-Mouse Antibodies).
They treat the antibody against the antibodies, so then if you have HAMA you cannot continue the treatment but you can lower the HAMA. We will see; we don’t exactly know. The purpose is to make some antibodies, whatever we can, and then to do the vaccine trial.

–> What is HAMA?

HAMA stands for Human Antibody Against Mouse Antibodies.
It’s the antibody that your body is doing against the antibody that we’re trying to give you.

–> They’re going to take something and put it in mice?

They already have the antibodies and they give it to you, but if your body is making antibodies against the antibodies that they try, you cannot treat the disease.
He will get on course, and then we will try to manage it. And if no we will go straight to the vaccine. If you get any other antibodies before, you will get antibodies before and after.

–> At Memorial Sloan Kettering, they accept patients without any evidence of disease?

Yes, after relapse. After a relapse, you can get this treatment.

Lessons learned

–> Looking back, in retrospection, if you could go back in time, to which point in your amazing journey would you go, and what would you have done differently?

I would have harvested more stem cells in the beginning, before any treatment, because it’s like gold down the road and it’s very different to harvest cells after transplants. I would definitely try to harvest more cells in advance. I think that’s the only thing that I would have done differently. That’s the most important thing.

–> May a doctor have any objection to doing it?

No. In Israel, we harvest much more cells.

–> Didn’t they harvest the maximum possible?

No. They harvested just for the transplant, but now they know that it’s important for the future. If I could go back, I’d ask them to harvest much more.

–> What would you advise other parent who are now starting to deal with Neuroblastoma in a very young child?

I think it’s important to know exactly what your child is getting, and to check what works and what is available even if it’s not your field.
You need to know what medicines and procedures should come and check that it’s coming. It’s important because there are mistakes in the hospital all the time.
Also, it’s important to know what is going to be your next step, hoping you don’t really need to use it but to know, because when a big mess happens like a relapse you don’t really have the time to think. I think it’s important to know who the best doctor is. If you can be in contact with them, it’s important.

–> For a parent who is just now starting the Neuroblastoma journey, please mention a couple of names and hospitals that you recommend he should get in touch with.

In CHOP (Children’s Hospital of Philadelphia), Dr. John Maris and Dr. Yael Mossé.
In UCSF (University of California, San Francisco), Dr. Katherine Matthay and Dr. Steven DuBois.
In Hellen Devos hospital (Grand Rapids, Michigan), Dr. Giselle Sholler.
In Chicago, Dr. Susan Cohn.
In Boston, Dr. Lisa Diller.
In Israel: Dr. Iris Fried in Hadassah and Dr. Shifra Ash and Dr. Itzhak Yaniv in Schneider.
There are others which I don’t know.

–> Should a parent get in touch with all of them or only some of them?

It depends. The treatment you can have in CHOP, in Boston, in San Francisco and in Chicago is under the same protocol. So, it’s not important to know everybody, However sometimes it made the difference for us with the bureaucracy and the time frame you can get your child into one of the trials.

–> Tell us a bit about the side effects of the treatment in Muli.

Muli has very low side effects, no real problems. Despite all the chemo that he gets, it’s completely fine. The only thing is, his blood count needs to recover after a huge treatment. That’s the only side effect. For example, he needs to get platelets. But it’s a matter of time and it will pass. Everything that we can have in my pocket and an able to give to him I don’t really care. It will pass.

–> How about growing, the radiation effect on his internal organs?

Nothing, nothing, nothing, nothing. We are lucky.

–> How many surgeries did he undergo?

One big one for the removal of a tumor. He had a smaller one for the removal of the fungus. He had port insertion. Three surgeries. He also had biopsies, but only one big surgery.

–> Did you ever use alternative medicine?

Yes. Anthroposophy medicine, in the beginning, and also for the side effect we used reflexology for the appetite and the nausea.

–> Anything you can recommend? Did you see any evidence?

I think the reflexology was helping for the appetite.

–> How many kids do you know that had a similar journey to you?

Nobody.

–> How many children with Neuroblastoma do you know?

In Israel about Fifteen or so. Here in the U.S I know many others.

–> Could you please mention websites and sources of information that a parent could use?

NIH (National Institute of Health): For the clinical trial, there’s a list of all the clinical trials. I think it’s an important site. That’s our main source of information for clinical trials.

–> Do you know of any communities or Facebook groups that you can recommend to parents to check out?

There’s a Facebook group for Neuroblastoma, and there’s also a list in the INRC, a very good list of emails. If somebody is sending an email it goes to all the people. You respond and you can see. It’s very good. It’s www.BlastNB.com and they have all types of cancer, not only Neuroblastoma. You can choose what kind you want to be in touch with. This can enable a parent to get in touch with another parent of the same type of cancer.

–> How did it affect the family?

It’s hard. It’s very hard. It’s not what we planned. We’re not making the career that we wanted. We are not with our friends and family. The life is completely different.

–> And you have another baby?

Yes, we have two children.

–> How old is the younger baby?

One year and four months.

–> So he was born into..

Yes. When I flew back when we were in Hershey, I did the delivery and came back.

–> Thank you very much for sharing.

Thank you!