Who:  Nathalie Traller (15) has been diagnosed with Stage IV ASPS – (Alveolar Soft Part Sarcoma), a rare pediatric sarcoma without known chemotherapy.

What:   Since Nathalie’s diagnosis in April 2012, her parents have explored each clinical trial and treatment option available. She has valiantly come back from eight surgeries and continues to surprise her doctors on the east and west coast. Knowing last September that the cancer was continuing to spread, sarcoma experts around the country pointed Nathalie’s family towards emerging anti-PD1 immunotherapy. Because of age, she is unable to participate in at least six recruiting clinical trials she otherwise meets criteria for. Her oncologist requested Compassionate Use of anti-PDL1 immunotherapy trial meds from Genentech. Further requests to Merck and Bristol-Myers Squibb for their anti-PD1 compounds have also been denied.
Now, with the cancer unchallenged and spreading, her family is calling out for help in sharing her urgent story with the public.  
*For information on anti-PD-1 immunotherapy — the breakthrough treatment that Nathalie is seeking — see this New York Times article.

Why:    The goal is simple: start treating Nathalie with anti-PD-1 immunotherapy from either Bristol-Myers Squibb, Merck, or Genentech before time runs out.

How:  There are two ways for Nathalie to receive the treatment she needs: clinical trials and compassionate use.
Clinical trials are being actively pursued, however Nathalie’s age as a teen keeps her from qualifying (a systemic problem for teens fighting cancer). An age exception could help her. Pediatric trials of these medicines are planned, but as usual lag way behind and could take up to another year to come online.
Under the FDA’s laws, compassionate use is for patients with life-threatening illnesses who cannot access the breakthrough treatments they need via clinical trials. This process requires that the patient have an oncologist willing to oversee the treatment and a pharmaceutical company willing to provide the drug. Nathalie’s oncologist is willing to oversee the course of treatment; also, the family is working with a non-profit experienced in navigating the FDA approval process, and they tell him that he can expect FDA approval within a week of submitting an application.


Nathan Traller
Thank you for your help. Please invite others to support 4Nathalie as we walk forward.