Introducing Mira

In January 2021, Ina and Flo from Aachen in Germany, were delighted to welcome their second child Mira into the world. However, just two months later, their world was turned upside-down when Mira was diagnosed with stage IV neuroblastoma, a rare type of cancer that develops from specialised nerve cells or neuroblasts left behind from a baby’s development in the womb. Mira’s mother Ina describes how a whole family is affected by such a serious diagnosis. Ina also shares their journey and how today her daughter is thriving, thanks to the discovery that Mira’s neuroblastoma is ALK-positive which opened up new treatment lines. 

“In January 2021, we were thrilled that our second child finally arrived, and our family’s happiness was complete,” shares Mira’s mother Ina. “But when Mira was three weeks old, we began noticing some small nodules under the skin. These started increasing in number and began turning a blue-like colour. We sought advice from several doctors who each diagnosed different, but always harmless skin diseases. At one stage, she was thought to have Blueberry Muffin Syndrome, a rare condition characterised by non-specific lesions of blue-red colour which can be associated with various conditions.”

Finally in March, an abdominal ultrasound was performed and Ina was told there is something that doesn't belong here. That ‘something’ turned out to be a huge malignant tumour on the adrenal gland that had metastasized all over her body, including skin, bone marrow, liver, kidneys, eyes and brain. It turned out that the visible blue nodules were skin metastases. After these investigations, Ina and Flo received the devastating news that their two-month-old baby daughter Mira had stage IV neuroblastoma. The tumour was deemed to be inoperable because it was wrapped around the aorta. Ina and Flo were devastated.  

Shortly after this, Mira received intravenous chemotherapy to shrink the cancer. Sadly, Mira’s first post-chemo MRI scan in May showed that the tumour was not responding sufficiently to chemotherapy. Ina adds, "We had already been through so much. This news felt like someone pulled the rug right out from under our feet. But we were also given a glimmer of hope when biomarker testing identified an ALK mutation in the tumour.”

ALK (or anaplastic lymphoma kinase) mutations are more common in lung cancer, where it is found in approximately 3-5% of all non-small cell lung cancers. There are more treatment options for ALK-positive cancers and patients receive targeted therapies – oral medications which inhibit ALK. Ina adds, “Our team in Germany proposed Mira become the first infant treated with a third-generation ALK-targeted inhibitor called Lorlatinib – Mira's lifesaver.”

From May 2021, Mira was alternately given chemotherapy and the ALK inhibitor, Lorlatinib, in blocks.  Ina adds, “By August, her skin was much better. We saw the first results after a few days, and we began noting how Mira’s skin metastases were decreasing each day. Scans in August confirmed what we could see, and it turned out that the primary tumour and the other metastases had all shrunk at breakneck speed. We then received the good news that the tumour resection could take place.

By August, just three months after starting Lorlatinib, a nuclear medicine scan that detects living cancer cells (called MIBG), was negative and in November various tests indicated that Mira was in remission. "We were completely overjoyed”, Ina said.

Following the experts’ advice and best-in-class practices, Mira’s parents were informed she would continue to take the ALK inhibitor daily for two years. "So, we were hoping to stop Lorlatinib by the end of the year." Ina adds, “Mira tolerated it very well, but she did have a few side effects, such as constant hunger, severe obesity and poor liver values. But overall, she did very well with it.” 

In March 2023, Mira stopped taking Lorlatinib earlier than planned because of her weight. Ina adds, “We had MIBG and MRI scans in April and Mira remains cancer free.”

Reflecting back on the incredibly intense time, Ina shares, “When your child is diagnosed with cancer, you suddenly live in a parallel world. From one day to the next, the whole lives of all family members change. When a child gets cancer, the whole family symbolically has cancer. It is primarily the parents who look after the child day and night; they only meet in the clinic for the handover of care. They somehow coordinate the sibling and are dependent on an incredible amount of help from the outside. They must face not only their own fate, but also that of the other families’ processing somehow. You get to know other people who are affected and make the deepest connections in a very short amount of time. You tremble, laugh, and mourn together; but you also lose friends. Often you think that you will probably never find your way again. For the first time, you see what real bravery looks like and you are amazed at the strength of the little patients who always surprise you. Often things will be fine again, but nothing will ever be the same again.”

The value of medical research is not lost on the family. Ina adds, “At the end of treatment, one of Mira’s doctors said to me that if she had been born two years earlier, Mira would not have been able to receive the right therapy. For me this underscores that cancer research is survival. Also, funding is so important, especially in paediatric oncology.”

Published in May 2023.