It’s been a while since Katie and I have updated all of you who are supporting Vivian. This morning I was texting information to my extended family and realized we needed to update everyone. So, here is where we stand today:

Vivian has completed the only standard therapy for Diffuse Intrinsic Pontine Glioma, which was 6 weeks of daily radiation therapy. She simultaneously took part in a clinical trial which was testing the combination of radiation and an experimental chemo drug. It was a challenging 6 weeks but overall she tolerated this well and since the conclusion of daily radiation/chemo her energy levels are returning and she is definitely improved. Her facial droop has mostly resolved and the eye deviation has also improved significantly. Her zest and vibrancy is back as the weight and worry of daily hospital visits has evaporated. We hadn’t really appreciated how all of that was affecting Viv, but since her last day of therapy, when she woke up knowing she wouldn’t have to do it again, she’s been alive!

Since the completion of that round of treatments Katie and I have been working hard to determine what comes next. There are no known, effective treatments moving forward, but around the country physicians and researchers are working hard to find a cure for DIPG. There are a variety of experimental treatments being tested around the country and we are spending lots of time investigating those options and trying to determine if one seems more promising than another…. then determining if Vivian qualities for the given trial. So far one company has agreed to give Vivian an unproven device she will wear on her head. In the lab and even in animals its electrical impulses prevent tumor cell division, but it’s so new there is no human data and she will be one of the first children to try it.(Thank you, Nativis, for offering this to Vivian free of charge.) We are awaiting FDA clearance for use under “compassionate use” guidelines. We are hoping to combine this device with a medication trial, but getting in touch with key researchers and clinicians is slow and trials have stringent requirements that often exclude patients. Some of the newest meds can only be dispensed if the FDA allows for compassionate use in Viv’s specific scenario because they are untested and could potentially be harmful or deadly. In these cases we need multiple physicians to write letters of support agreeing that the risk of use does not exceed the risk of alternatives or the risk of doing nothing. Given that DIPG is uniformly fatal we can make a strong case for compassionate use. It’s a slow process, but we are pushing forward and slowly making headway.

New drugs and new data are coming out all the time as teams work on this in the US and Europe. The landscape changes regularly as new information becomes available. Today I will be on the phone trying to contact researchers in Boston and in Georgia, trying to determine if Viv might be a candidate for one of two early stage drugs that have shown some promise. These drugs are so new and experimental that one has no pediatric data, the other has data from 6 children… a minuscule number of patients, but you have to start somewhere. A brand new study out of Stanford was just published, and if you are interested in reading the type of data we are pouring through I’ve include the summary at the end of this post. This is exciting information, but no human trials are active yet. We hope they are forthcoming!

So, this is where we are today. We are praying for God’s guiding hand as we seek further treatment and we continue to pray for Vivian’s healing in whatever form it comes. Vivian is doing well right now. She is happy, she remains unaware that she is bald except for the top half of her head, and although we discuss her diagnosis and consistently pray as a family for her healing she only grasps the gravity of the situation superficially. We love her immensely and are constantly amazed at her resilience, her strength and her trusting nature.

Thank you all for your continued prayers and support. We are thankful.

Diffuse intrinsic pontine glioma (DIPG) and other diffuse midline gliomas (DMGs) with mutated histone H3 K27M (H3-K27M)1–5 are aggressive and universally fatal pediatric brain cancers6. Chimeric antigen receptor (CAR)-expressing T cells have mediated impressive clinical activity in B cell malignancies7–10, and recent results suggest benefit in cen- tral nervous system malignancies11–13. Here, we report that patient-derived H3-K27M-mutant glioma cell cultures exhibit uniform, high expression of the disialoganglioside GD2. Anti-GD2 CAR T cells incorporating a 4-1BBz costimulatory domain14 demonstrated robust antigen-dependent cytokine generation and killing of DMG cells in vitro. In five indepen- dent patient-derived H3-K27M+ DMG orthotopic xenograft models, systemic administration of GD2-targeted CAR T cells cleared engrafted tumors except for a small number of residual GD2lo glioma cells. To date, GD2-targeted CAR T cells have been well tolerated in clinical trials15–17. Although GD2-targeted CAR T cell administration was tolerated in the majority of mice bearing orthotopic xenografts, peritumoral neuroinflammation during the acute phase of antitumor activ- ity resulted in hydrocephalus that was lethal in a fraction of animals. Given the precarious neuroanatomical location of midline gliomas, careful monitoring and aggressive neuroin- tensive care management will be required for human trans- lation. With a cautious multidisciplinary clinical approach, GD2-targeted CAR T cell therapy for H3-K27M+ diffuse glio- mas of pons, thalamus and spinal cord could prove transfor- mative for these lethal childhood cancers.