Skip to main content

Diffuse Intra-Axial expansive Lesion of the Enchephalic Trunk not surgically investigated _2

Short summary

7-year-old boy was diagnosed with diffused intra-axial brainstem expansive lesion. He underwent radiation therapy but his physical condition is worrisome. His relatives ask the expert's opinion about experimental therapy consists of combination Antineoplaston and radiation therapy for the treatment of gliomas in children, that is now in clinical trial. The expert doesn't recommend it.

Patient's questions
Amidst our growing confusion, we would be infinitely grateful if you could comment and provide any insight on this therapy and on the clinic itself.
Medical Background
Patient's History
We have submitted the case of my 7-year old nephew, to your attention in the past. On November 2008 he was diagnosed with a diffused intra-axial brainstem expansive lesion which to date has not been surgically investigated.
Perusing the internet we saw that researchers apparently made breakthroughs in the treatment of brainstem gliomas at X Clinic in Houston. We also learned that the FDA recently granted clearance for "Phase III Trial of Combination Antineoplaston and Radiation Therapy" for the treatment of those gliomas in children. Wishing to understand more about this experimental therapy, we tried to gather information on this from various sources and came up with opposing opinions on Dr. X. and his clinic, some even questioning the physician’s dependability . Amidst our growing confusion, we would be infinitely grateful if you could comment and provide any insight on this therapy and on the clinic itself.
The patient is currently attended to at Milan's Istituto Nazionale dei Tumori where he underwent radiation therapy from 11/2008 up to 01/2009.
Follow-up MRI is scheduled for 02/2009. Despite not having severe head pain, Patient's physical condition is worrisome. The left side of his body seems pretty impaired (he drags his left leg and finds it hard to use his left arm and hand). We expected Patient would partially regain his physical strength by the end of radiation therapy but our expectations did not materialize. Rather, his condition worsened and we are left wondering what the best way ahead might be. From here our need to find out what all can before the follow-up MRI in early February. We fear we might not have much time left to decide on Patient's future.
Medical opinion
  1. I have seen, and have in my custody, a copy of the January 2009 press release from the X Research Institute, announcing the formation of a multi-site phase III trial to test antineoplastons A10 and AS2-1 plus radiation versus radiation alone in the treatment of newly diagnosed, diffuse intrinsic pontine glioma.  Indeed, Stanford University has been invited to participate, and our group is debating this offer—we were invited 30 January.
  2. Patient would not be eligible for this trial, as the study is for children newly diagnosed with pontine glioma.
  3. Yes, the X Clinic is still and has been controversial, featured on United States newscasts, and has during the 1990s come under close scrutiny by the United States Food and Drug Administration for accusations of overstated successes of antineoplastons in cancer.
  4. To date, there is no peer-reviewed, solid, published or medically accepted evidence that these substances improve outcomes from brain tumors, including pontine gliomas.
  5. Yes, many practitioners in the United States are highly critical of the Clinic and antineoplastons, as good evidence has been lacking, despite multiple word or mouth stories and internet testimonials.  Doctors have witnessed notable side-effects arising from the high use of steroids (dexamethasone) employed in conjunction with the antineoplastons.  In addition, many United States physicians are further skeptical because of the high monthly price paid for this as yet unproved treatment, generally $10,000 to $15,000 a month. 
  6. I have contacted just today 5 colleagues at leading United States Centers, like Stanford, to see their response to any invitation to participate in the phase III trial.  Their response has been flat out no, to very muted enthusiasm to test this treatment rigorously once and for all (with the anticipation that no effect will be seen), to simply uncertain.
  7. Overall, there is no doubt that the condition of Patient is fragile and his future uncertain, worrisome, with very limited treatment options.  However, almost all oncologists in the United States would not endorse antineoplastons, though it will be interesting to see how many centers will entertain this study and try to see if this treatment has any effect.  Almost everyone would agree that the treatment is not curative.