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 some patients living with NF.
Koselugo (selumetinib) is approved only for NF1 patients with inoperable plexiform neurofibroma tumors. It is an incredible first step. But the devastating effects of NF go far beyond this type of NF, and far beyond this one type of NF tumor. Patients like 7-year-old Cataleya Castanon, who has an optic glioma tumor, are still waiting for breakthroughs in drug development and FDA-approved treatment options. At just two-years-old, Cataleya was diagnosed with bilateral optic gliomas that caused irreversible vision loss. There is new hope for Cataleya because researchers believe that there are drug options that might work for optic glioma tumors the way they do for plexiform neurofibromas. Cataleya just joined an exciting selumetinib clinical trial for patients with optic gliomas, and her family hopes that it will preserve what sight she has left.
When Cataleya was first diagnosed, there were no drug therapy options available, and now there are multiple options; the amount of progress in such a short amount of time for optic pathway gliomas is incredible, and it’s due in part from investments made by the Children’s Tumor Foundation.
In this video that first aired during the Shine a Light NF Walk in Seattle this past summer, Cataleya takes us through what it’s like taking her “chemo,” how it makes her feel, and her advice for other kids who may need to take selumetinib.
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 Cataleya.
