ALK Treatment in the US

Smiling in the US
Smiling in the US

Hi James here,

After such a chaotic lead up to our departure, it was a relief to actually walk onto the plane.  Whilst we had initially been devastated by Pfizer denying us access to the drug in Australia, it was surreal that the option of free treatment in the USA arose that same day.  There were so many hurdles to overcome to actually get to the USA, and we knew that we would still face further hurdles prior to actually being accepted onto the trial.  Then there is no guarantee that the treatment will actually work.  However as we left Canberra, we knew that we were doing everything we possibly could to try to save our gorgeous girl.

Of course, given our recent luck, even getting to the airport wasn’t without drama.  Luckily, I went into the garage early in the morning, as I was greeted by the sight of the Kluger leaning heavily on a completely flat front tyre!  Of course, our spare was also partially flat, so it took me almost an hour to get the car sorted.  Thankfully, we still made it to the airport with time to spare.

We only had a short stopover in Sydney, where my best friend Tony and our friend Deanna (whose wedding we unfortunately will now miss) came to see us off.  A special thanks to Tony for picking up copies of all of Olivia’s previous scan results from Sydney Children’s Hospital for us, which we were requested to bring with us to the US. 

A special thanks to Qantas who gave us an amazing deal with our US airfares, considering that they were only booked less that 2 weeks before we departed.  Furthermore, Natasha, the mother of another neuroblastoma child who works for Qantas, advised us that our Sydney to LA flight had many vacant seats.  Kirsty thought that she had nothing to lose in asking for an upgrade at the counter, and after quite a lengthy wait was blown away to be told that we were being given complimentary upgrades to Business Class!

We were initially seated in a two plus two configuration, with the two girls next to each other, behind Kirsty and myself.  After take-off, Kirsty and I each chose a movie, put the headphones on and sat down to enjoy a peaceful relaxing flight.  Then reality hit home, when approximately every ten minutes one of the girls would yell out MUM……MUM…….MUM! or DAD……..DAD……..DAD!  Because the reclining seats are capsules, it’s not possible to turn around and see the seats behind, so we had to pause our movie, take the headphones off, get up and walk over to where the girls were to see what they wanted.  After a few hours of this calling out, much to the amusement of (or was it annoyance to) other passengers, Kirsty and Olivia swapped places.  It was a real treat to have beautiful meals, great service, all the extra space and a bed to lie on.  

We stopped overnight in LA and stayed near Santa Monica.  To our surprise, it was very windy and really quite cold and we were just wearing shorts and T-shirts.  The girls really enjoyed going on some rides at the pier, though we ended up having to drag them away as Kirsty in particular was really feeling the cold. 

After a bit of a rough night’s sleep, we headed off to LAX in two taxis, as we had so much luggage and our booked van didn’t turn up!  It was around 5 hours flight time across to Washington D.C., so with the time difference, we didn’t actually arrive until around 6pm and were all feeling pretty exhausted.  Departing the plane, we were shocked by the change in climate between LA and Washington D.C. –  It was very hot and quite humid.

Whilst waiting at the car rental depot, we noticed that the inside area near Olivia’s right eye seemed to be developing a bruise and also appeared partially swollen, consistent with a tumour growing in that area.  We wondered if that was being caused by a new tumour or the one in her sinus (that had been previously irradiated) coming back? 

We had pre-booked a car for six weeks at a discounted rate through my work, but the guy at the counter was hell bent on selling us an upgrade.  It took almost an hour of being mucked around before we finally got our original car – not a pleasant experience at all!  By this time, we are all very tired and frustrated.

We arrived at our new home at The Children’s Inn at the NIH (National Institutes of Health) at Bethesda, Maryland around 8pm.  However, it took us a while to get through the incredibly tight security, including being issued passes and having our car and luggage searched.  The Inn is huge, about four times the size of Ronald McDonald House in Randwick.  Our room is like a typical 3 star hotel room with 2 double beds.  Unlike our room in Germany, there is no separate bedroom, which means that we can’t watch TV or make any noise until the girls go to sleep, which has been taking them ages.  This is partly due to jet lag and partly due to them sleeping together in a double bed.  My feet hang about a foot over the end and I can’t really move without disturbing Kirsty – we really miss our King bed!  Thankfully, we only expect to be here six weeks. 

The Inn is very conveniently located only 200m from NIH where Olivia is being treated. However, it is up a steep hill and we have had some scorching hot days here.  The hospital itself is huge and quite modern. 

I think that when the staff at NIH first met Olivia they were very surprised at how good she looked and how much energy she had.  Obviously, her scans and blood test results made her out to be a very sick girl, though the only visible sign of her disease was her eye.  Just prior to leaving Australia, Olivia’s LDH, a non-specific tumour marker, had risen to a very high level, indicating rapid disease progression.  Also, a couple of her liver enzymes were abnormally high before we left so she had to take some medication to help protect her kidneys.

They have only previously had one ALK positive patient like Olivia on the ALK Inhibitor trial at the NIH, and unfortunately that child didn’t respond to the drug.  However, they were quick to point out that they have two patients on the trial in CHOP in Philadelphia who are currently doing well on the drug.

Although Olivia theoretically qualified for the trial based on her test results in Australia a week prior, she was still required to undergo three days of re-testing here to determine her current eligibility.  Needless to say this was a very anxious period for Kirsty and I. 

On the first day of testing, one of the nurses came in to advise us that her blood test indicated that she was neutropenic (meaning that she was very susceptible to infection) so we needed to make her wear a face mask and keep her away from other people as much as possible.  More importantly, her neutrophil count at 110 was significantly below the required level of 750 to enter the trial.  We had struggled so much to get her here and travelled so far, only to be likely turned around – this was turning into our worst nightmare!  Such low counts would also mean that the flight home would present a very high risk of infection for her.

After about half an hour contemplating the ramifications of this bad news, the Dr came in to see us and indicated that something didn’t add up with the blood test results so he rang the lab to check.  It turns out that of all the many numbers in the blood test report, there was a typo on the one that mattered the most to us.  Her neutrophil could was actually 1100, not 110, meaning that she was now back in consideration for the trial! This was just so typical of the emotional rollercoaster ride that we have been on over the last month.  

Olivia underwent several different scans, some taking hours.  She was a champion and lay so still for each one.  They did have trouble finding a vein to inject contrast for her head MRI, which caused her a lot grief.  Once she recovered and dried her tears, she actually apologised to us for making such a fuss.  This, practically brought Kirsty and I to tears.  It is so unfair that she has had to deal with this for most of her life!

We went in with Olivia for her MIBG scan, which we knew would show extensive disease.  They indicated that they do one full body scan and then do a spinning scan of one or two of the worst areas.  After the first scan, the technician indicated that she was going to consult with the radiographer about which area to re-scan.  She was gone for ages.  By this time we were becoming very concerned that there was so much disease that they couldn’t determine which parts to re-scan!  Finally the technician came back in, apologised for the delay and advised us that there were no particular issues.  We never actually saw the results of the scan, though were advised that she had very extensive bone marrow disease. Whilst that is obviously not good news, it was not unexpected. 

They did note some spots on one of her lungs from the CT, though this was not confirmed on the MIBG scan.  It is possible that there are tumours starting to develop there, which could become very dangerous.  The MIBG scan revealed a small tumour near her eye, which explains the bruising and swelling, as well as a couple of bony lesions but it mostly conveyed extensive bone marrow disease.   Most importantly, her brain remained free of tumours and she qualified for the trial.  The biggest risk to Olivia’s health remains the disease suppressing her blood counts and associated problems such as anaemia, internal bleeding and infection risk.

Olivia qualified for the trial and commenced taking the trial drug crizotinib on the Friday.  She is required to take an oral solution of the drug (she refuses to take the capsule after some bad experiences in the past) and needs to take it twice a day, 12 hours apart.  Although she clearly hates it, she always eventually takes it.  She didn’t stomach the first dose of the medication very well, throwing it back up 15 minutes later.  Apparently, this is not uncommon as the nurse later told us that every patient had vomited the first dose.  Since that first dose, we have given her an anti-nausea drug half an hour beforehand.  According to Olivia, the medication is a very sweet but foul tasting syrup and continues to make her feel a little nauseous.  She rinses her mouth out afterwards with water and then has a mouthful of something nice to counter the bad taste.  We have been giving her ice cream, but unfortunately, she is now turned off ice cream.  We can add that to the list of foods including apple sauce, yoghurt and juice, that she has been put off by taking them with different treatments over the years.  We don’t normally let her have soft drinks back home, but we have just tried giving her lemonade instead.  She may end up being turned off soft drinks too, which is probably a good thing, as ‘soda’ is everywhere over here and is hard to avoid. 

The whole time we have been here, we have all been going to bed quite late and sleeping in.  This has been mostly caused by Olivia having to take her medication quite late at night.  Olivia seems to take a long time to get to sleep each night, which may be combination of a possible side effect of the medication and the fact that she usually sleeps in the afternoon when receiving a transfusion as she receives pre-medication that makes her very drowsy.

Other than that one huge mistake with the blood count mentioned above, we have been very happy with the level of care over here.  The doctor/nurse to patient ratio is so much higher than in the public system back home and they are always willing to sit down and discuss anything relating to her treatment and arrange any further testing/investigations necessary. 

In our second week here, Olivia started to experience tooth pain, due to one of her adult teeth pushing in front of her baby tooth, in a similar fashion to what happened several months ago.  Olivia’s oncologist booked her in at short notice to see the dentist.  They explained that provided the pain doesn’t get any worse the tooth could stay in to see if it will fall out on its own accord.  As part of the investigation, they conducted a panoramic mouth x-ray.  We were surprised to be told that Olivia has not developed her adult molars for whatever reason.  It is unclear if this is due to a treatment side effect or just an abnormality that Olivia was born with.  It just means that she will have to live with her baby molars for life and will probably require extensive dental work.  This is merely one of many issues that we will happily deal with in the future if we can cure her of this dreaded disease.
We were warned by the doctors that one of the potential side effects of the ALK inhibitor was light visual disturbances.   After only a few days on the crizotinib (the trial drug), Olivia started experiencing slight blurring vision at night, when in darkness.  The doctors decided that it would be best to get her eyes checked out.  Her vision was okay, but the test indicated that she has swollen optic nerves.  This was initially a concern as this can often indicate pressure in the brain.  However, her recent head MRI didn’t show anything.  We had a follow up appointment with the ophthalmologist/neurologist, who confirmed that both optic nerves were swollen though could not find an immediate cause.  He ordered another MRI just to be sure, which also came back clear. It was suggested that one possible cause is her low haemoglobin levels.  We just need to ensure that she is monitored carefully as this can lead to further eye problems. 

Overall, Olivia remains quite well.  We were initially hopeful that the treatment is working.  Given that it has been four weeks since we left Australia, we expect that she would be much more unwell if the treatment wasn’t doing anything.    There seems to be several contradictory signs with her blood test results, for example, the tumour marker, LDH, has been fluctuating when we were hoping that it would just continue to decrease.  Most of Olivia’s blood levels are still quite abnormal as well as some of her liver enzymes, suggesting that the cancer is still significantly affecting her bone marrow.   As such, we are not under any illusion that the treatment is rapidly killing off most of her disease.  It may be doing something, but at this point, we can’t really be sure how effective it is. 

The bruising and swelling around her right eye seemed to subside within a week of starting the treatment, filling us with hope that the treatment is working.  In fact, the bruising had completely healed.  However, she has since developed a new bruise and her eye is again swollen, appearing quite misshapen. 

Olivia was originally needing red blood cell transfusions once a week, though her last one lasted her just over two weeks.  However, she still requires frequent platelet transfusions, averaging about every three days.  If we were back in Australia, we might be able to boost her platelets by giving her back some of her own stem cells (that we recently collected), however, we can’t do this whilst she is on this trial. 

Thankfully, it is pretty easy to tell when Olivia needs platelets, as she normally wakes up with bleeding gums.  The hospital still has to do a full blood count and await for the results before transfusing her.  She also requires prophylactic pre-medication as she can have a severe allergic reaction to platelets so, all up, it can take around 5 or 6 hours.  We are hopeful that her platelets will start to last longer as the treatment continues, so she won’t need to spend much time in hospital.  Other than transfusions and scans, the treatment itself actually requires very little hospital time.

Whilst we can walk to hospital from the Inn, we are so glad that we have a car, as the nearest supermarket is about a ten minute drive.  Given that we only have one shelf in the shared refrigerator, we find ourselves going to the shops every second or third day.   We have also done a bit of exploring Bethesda and downtown DC, though the traffic here can get pretty congested.  From what we have seen so far, this is a really nice part of the US.  We had originally planned one or two overnight trips to surrounding attractions, though this will have to wait until Olivia’s blood levels stabilise some more, so that we can be sure that she won’t need a transfusion (which we would have to pay for) whilst we are interstate.  We also have been told that she should not be going on any rollercoaster rides or the like that could possibly cause internal bleeding.  Olivia was not happy at all to hear that.  Hopefully, if everything goes well, we would love to take the girls to DisneyWorld on one of our future trips when we return for scans. 

Olivia has three days of scans and procedures this week, which will show us if the treatment is working.  On the one hand, we are scared to see how much disease she has, but on the other we are so hopeful that there is a significant reduction and that we can return home with the medication knowing that we have bought our gorgeous girl some more time. 

We should mention that there  is another treatment option available to Olivia at the NIH, should the current ALK inhibitor not work and Olivia remains well enough.  It is a customised treatment, based on testing of her disease’s response to numerous different agents in the laboratory.  This seems like a logical approach, given that different types of neuroblastoma respond to different treatments.  This will require taking a tumour sample for testing and can take a minimum of three weeks to analyse.  Unfortunately, as Olivia’s disease is in her bones so it has never been easy to obtain tumour samples.

When we were in Germany, we became friends with a Scottish family who also have a daughter called Olivia, who is one year younger than our Olivia.  In what was a shocking week for all of us in Jan 2011, both Olivias relapsed within a few days of each other.  Unfortunately, she has been constantly fighting the disease ever since.  In a desperate attempt to help her, they recently travelled to Mexico for a some alternative treatment.  Unfortunately, Olivia’s health has been deteriorating and they are now desperately trying to get her back to Scotland so that she can be at home.  We pray that her condition will improve enough to allow her to make the journey home and that they can raise the required funds to make it possible.






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