Yesterday’s and Monday’s posts were so long that this one will be a bit shorter than usual (probably). (It will also look at a case that I had meant to write about when it hit the news right before Christmas but for some reason never got around to.) I’ve written a lot about DIPG (diffuse instrinsic pontine glioma). DIPG is an incurable form of brain cancer involving the brainstem. Part of the reason it’s so difficult to treat and almost always incurable is because, given that it involves the brainstem (by definition) it is almost always unresectable and is rarely responsive to chemotherapy. Basically, for the vast majority of patients with DIPG, the recommendation is palliative radiation therapy, which reliably temporarily shrinks the tumor. Unfortunately, radiotherapy generally adds at best a few months to a patient’s life expectancy. Worse, DIPG disproportionately affects children, which makes its effects on families even more tragic. Indeed, cancer quack Stanislaw Burzynski treats a lot of children with DIPG. My most recent major series on a new group of cancer quacks in Monterrey, Mexico featured mostly children with DIPG being victimized by pseudoscience. One of the most common claims of quacks treating DIPG (particularly Stanislaw Burzynski) is that no one ever survives this cancer and that therefore the quackery must have cured it when it’s likely that long term survivors were as a rare result of the conventional treatment. Enter an 11 year old girl named Roxli Doss in Texas, who was diagnosed in DIPG in June after having complained for weeks about headaches.
Here’s the tale on her CaringBridge page:
Roxli has been experiencing headaches for a couple of weeks now. In the past week they got much worse and after numerous doctor visits with no clear answers as to why, they got an MRI done. On the morning of June 21st they received a phone call to go to Dell children’s hospital.
The news is not good. Roxli has an inoperable tumor on the back of her brain that is pushing on her spinal chord. It is called Pontine tumor or also called DIPG.
Last night, they started steroids for her to help relieve the pressure. They will check the tumor again in a couple of days. The doctor is giving them the full schedule today. If the tumor hasn’t shrunk they will consider putting in a stent to redirect the fluid.
She will stay in the hospital for a couple of days and then the family is going to the beach for a vacation.
When they return she will have radiation treatments Monday – Friday for 6 weeks to hopefully slow the growth.
How many times has a post by on this blog about a family of a child with DIPG started out with a story like this? More times than I can remember dating back at least nine years. In the case of Roxli Doss, however, something different happened. Yes, her family was trying to raise money on GoFundMe and CaringBridge, but, unlike the vast majority of cases that I write about, they weren’t raising money to go to Houston to have Roxli treated by Stanislaw Burzynski or heading to Monterrey to have her treated at Clínica 0-19 by Dr. Alberto Siller and Dr. Alberto Garcia of of Instituto de Oncología Intervencionista. No, her family took her to Dell Children’s Medical Center in Austin.
Dr. Harrod said the now 11-year-old went through weeks of radiation, even though there is no cure. The family held a benefit for her in August, and the Buda community responded in a big way. At that point, all Gena and Scott Doss could do was pray for a miracle.
“And we got it,” said Gena.
“Praise God we did,” said Scott.
Now, they cry tears of joy.
“When I first saw Roxli’s MRI scan, it was actually unbelievable,” said Dr. Harrod. “The tumor is undetectable on the MRI scan, which is really unusual.”
Doctors can’t explain why the tumor disappeared.
“At Dell Children’s, Texas Children’s, at Dana-Farber, at John Hopkins, and MD Anderson, all agreed it was DIPG,” said Scott.
As of now, doctors are following Roxli Doss to look for recurrence and are administering immunotherapy to try to prevent recurrence. As an aside, I hope they’re doing this on a clinical trial, because there really isn’t any good evidence that immunotherapy is effective at preventing DIPG recurrence. However, in this case, I can understand the desire to do something, particularly in the face of not a lot of evidence given how uncommon DIPG is.
I’ve pointed out before that, even though the reported survival rate for DIPG is dismal, survival after treatment for DIPG is, while pretty rare, not zero and spontaneous regression has even been reported.
Of course, a Roxli’s response is incredibly dramatic and quite rare. However, it demonstrates that, even in the case of the most deadly cancers, spontaneous regression or far better-than-expected response to conventional therapy is possible, albeit rare. I also note that Roxli’s case is not the sort of case where the parents will be tempted to seek alternative cancer quackery, like that at the Burzynski Clinic or Clínica 0-19. Usually, families seeking such quackery have a child who has either not responded so dramatically to radiation therapy or who have had recurrence.
Complicating the issue is a well-described radiologic phenomenon known as pseudoprogression, which I’ved described several times before in the context of Stanislaw Burzynski. in which a treated DIPG tumor will appear to increase in size after radiation therapy, thus appearing to progress. The “false” part comes in because this is a radiological phenomenon. MRIs image blood flow, and the inflammation provoked by the dying cancer cells will result in increased blood flow to the area of the cancer. If a treatment, like Burzynski’s antineoplastons or other alternative cancer treatments, is initiated during pseudoprogression, when the inflammation subsides the cancer will appear to shrink, and it’s easy to be fooled into thinking this tumor shrinkage is due to treatment effect. When the tumor disappears so quickly and completely, there’s no room to be fooled by pseudoprogression.
Of course, Roxli’s family is attributing her outcome to God:
Although I can understand why they would do this, it still irks me when God is given credit instead of the medicine. I also can’t help but wonder: If God healed Roxli, why did he give her a deadly brain cancer in the first place? (I know, I know, I’m not the only one who asks such questions.) Also, I hate to be a downer, but Roxli is not out of the woods yet. She might have had a radiological complete response, but that doesn’t mean that there aren’t microscopic tumor cells left that could start growing again. I sincerely hope that’s not the case and that Roxli lives a happy life to a ripe old age, but it’s only been four months. Fingers crossed, as they say.
Roxli’s case is an example of a cancer that scientists really need to study, because she is the very definition of an “exceptional responder“:
Exceptional responders are patients who met the following criteria:
- Received a treatment in which fewer than 10% of patients had a complete response or a durable (lasting at least 6 months) partial response based on clinical trial data or extensive historical experience in the context of the patient’s tumor type
- Achieved either a complete response (CR) or a partial response (PR) with duration of at least 6 months as defined by RECIST (Response Evaluation Criteria in Solid Tumors) criteria for solid tumors or response criteria as defined where RECIST is not commonly used
- Sustained a complete or partial response for at least three times longer than the median duration of response from literature resources for that treatment
Finding out what is different about Roxli’s DIPG compared to the vast majority of DIPG treated. DIPG can be quite responsive to radiation therapy, but such dramatic complete responses are very uncommon, and long term survival is rare. That’s why I hope there is tissue available to study. Historically, suspected DIPG has not always biopsied, because sticking large needles into the brainstem is hazardous (although biopsy is becoming more common with safer techniques). The reason is that DIPG can be pretty reliably diagnosed by its clinical characteristics and its features on MRI. In any case, this is where science-based medicine comes in.
In the meantime, I wish Roxli and her family only the best and hope to see an update on her several years from now in which she’s in high school and thriving.