In April 2020, NF patients and families received the news of a lifetime — the United States Food and Drug Administration (FDA) announced the approval of Koselugo (selumetinib). Finally, for the first time ever, there is an FDA-approved drug treatment for patients living with NF. Early-stage discoveries from Children’s Tumor Foundation-funded researchers proved that MEK inhibitor drugs have the potential to affect the size of NF tumors, bringing us to this moment. More than 70% of NF patients taking selumetinib in a clinical trial had shrinkage of 20 to 60% in the size of their tumors. Koselugo has helped so many patients living with inoperable plexiform neurofibromas. Patients like Lily Childers.
Now four-and-a-half years old, young Lily’s parents first met her when she was six months old in China. Her father Patrick was working for a nonprofit that connected doctors in the U.S. with sick orphans and underserved kids overseas. When her caregivers reached out to Patrick’s nonprofit organization for help, the news was that Lily had a tumor that was growing really fast, and no one knew how to help her. Patrick and his wife Lauren fell in love with Lily, and soon adopted her. They charged ahead searching for answers, looking for a treatment that might stop her tumor from so dangerously growing. More than a year and several surgeries later, Lily has now started taking Koselugo (selumetinib), and her parents and her doctors are looking forward to the promise that this new FDA-approved treatment might have in store for this sweet young girl. “Lily is such a bright little girl. It’s been really exciting to see her have these opportunities for the future, as we look at all the progress in NF research and care. We just know that things are going to be completely different as she grows and changes, and we’re excited to see what tomorrow holds for Lily, and all people with NF,” said Lauren, Lily’s mother.
In this video, Lauren, Patrick, and Lily share their NF journey:
Koselugo (selumetinib) is approved only for NF1 patients with inoperable plexiform neurofibroma tumors. But the devastating effects of NF go far beyond this type of NF, and far beyond this one type of NF tumor. There is still no approved treatment option for NF patients with disfiguring cutaneous neurofibromas, life-threatening meningiomas, or painful schwannomas. We cannot stop until there are treatments for all forms of neurofibromatosis – NF1, NF2, and schwannomatosis. In subsequent blog posts, we’ll meet more NF Heroes waiting their turn for a life-changing treatment.
Through the end of the year (December 31, 2020), every donation made at ctf.org/donate will be matched dollar for dollar. Help us help more NF Heroes like Lily.
