Precision Medicine vs Lily’s fight for survival

It’s true that we often focus on the successes of Precision Medicine, as it is extremely important in the battle against rare diseases when other therapies have failed.

However, it is also key to consider that due to the financial strain of Precision Medicine treatment it is simply out of reach for many who desperately need the treatment as their final resort. On top of that, there are not enough clinical trials that specifically target children.

In this story, Arnaud Papin, Global Marketing Director, of Precision Medicine at Merck explains how 7-year-old Lily and her family have fought tirelessly to find clinical Precision Medicine trials for the girl, and thus far, failed.

“Look at this picture. At first glance, we see a beautiful young girl having a blast doing a fun activity.

Yet, she, Lily, is the saddest story you could think of. She represents the absolute failure of Precision Medicine, the one we aren’t talking about.

I will remember that particular Saturday this summer. My dog had some neurological issues and we had to bring him in as an emergency to the veterinary hospital. After the first diagnosis, the vet told us it was probably hydrocephalus, a condition that would give him less than 60% chance to see his second birthday. I was devastated. Since then, he has been perfectly fine and a cuddle buddy. I really congratulate the vet hospital. They have been terrific!

But that day, while waiting to hear more from the vet, I had a text from my friend Jing as she was sitting at the hospital across the Atlantic. Not a veterinary hospital – a human one.

Lily, her 7 year-old daughter, had just been diagnosed with Diffuse Intrinsic Pontine Glioma (#DIPG). It is a disease I have not heard of before, but somehow it felt like bad news.

While progressing through my internet search, I felt sadness taking over my entire body: the child had less than a year to live and there is no treatment available. Terminal. The end.

At that particular time, while coping through my own pain with the diagnosis of my dog less than an hour ago, I was trying to apprehend how Jing could go through this. The idea of losing my dog was incredibly painful, but seeing my own child going away? I don’t know how I could go through this. It felt like I would immediately collapse.

Yet, whoever knows Jing will tell you she is a rock, and as fierce as a bull. She won’t just sit and do nothing. No, she contacted her network in Precision Medicine to see what could be done. Lots of friends and colleagues contributed, and the media got involved. TV, local journal, everything we could think about.

In the end, we were able to find some potential clinical trials. And this is where I discovered a completely different side to Precision Medicine. The one I wish never existed.

  • There are not enough clinical trials targeting children. But in the end, aren’t they the one that would most benefit from it? If we could trade, how many octogenarians should a child be worth?
  • The clinical trials we identified were not recruiting in the UK. This means Lily would need to travel to the US or Switzerland to get a chance. Beside the challenge of travelling while having a brain tumor, think about doing this during the current pandemic.
  • While sites abroad would potentially accept her, the out-of-pocket costs would still be in the range of half million British pounds.
  • It took 1 1/2 months to get the result back from her brain biopsy, to be contrasted with her 6 months life expectancy.
  • The results from the brain biopsy test the NHS performed aren’t accepted for the clinical trials as the method was not the official companion diagnostic. She was therefore asked to re-perform a brain biopsy – as if this was just a simple procedure, and tissue was unlimited.
  • The assay the NHS used is not broad enough to identify mutations that are required to get potentially considered for some clinical trials. These are missed opportunities that you can’t get back.
  • To enroll into a promising clinical trial in the US, she must have been through >54 Gy of radiation therapy, but Lily “only” had 52.5 Gy, one session short. When asked to “just” do another one, they require another full plan, delaying the process by another month.

See Jing’s post here, the twitter account here, the Sun article here and the ITV coverage here.

Finally, here is the GoFundMe page to help the family to cover the treatment costs. Please consider giving anything -everything count- to allow Lily to laugh and smile for a bit more time. When considering it, think how much you would give for your dog, for your own child. Be thankful life sparing you from this experience and support Lily, Jing and Vineet to go through this, this time not in the dark side, but through the #LightsForLily.”

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