Thursday, 20 October 2016

Peptide Vaccine for 5 year old with GBM


Thanks to everyone for contributing to this great resource.  First a profile of our daughter's condition and then a question at the end.


Our 5 year old:

DOB 9/2/2011
Brooklyn, NY

9/9/2013 admission to:
Weill-Cornell Medical Center
Right neck abscess lanced and drained

March 18th, 2016 admission to:
Weill-Cornell Medical Center
Mass discovered in head after experiencing headaches for a few weeks
Left occipital/parietal brain tumor
Operated 3/19/2016
"Full Gross Resection"
Pathology determines Glioblastoma Multiforme "methylated" 

Transferred to Memorial Sloan Kettering
Photon Radiation treatment for 33 days (Dr. Suzanne Wolden)
Temozolomide chemotherapy for 43 days
MRI on 7/8/2016 'clear' 

'Maintenance' regime of temozolomide prescribed for 12 cycles (5 days chemo/23 days off)
Currently in 5th? cycle 
MRI on 10/5/2016 'clear'
Genetic sequencing/testing
• Cornell _ Precision Medicine
• MSK _ Methylation array
• MSK _ Impact Testing
• Caris Test

Other Diagnosis via 3rd party (non-MSK) bloodwork
• Positive (via Plasma test) for CMV antibodies
• Positive (via DNA test) for Herpes-6 active case

Other Treatments
• Cronaxal (
• Cannibas oils (THC/THCa/CBD)
• Chinese Herbs (prescription pending)
• Supplements- Vitamin B, C, D, L-Lysine, Curcumin, sodium selenite, paw paw, cayenne pepper w/garlic, green tea extract, etc.
• Ketogenic diet since appx. June 2016

We are so grateful that our daughter is NED to-date but are considering engaging in an off-label customized peptide vaccine treatment coming out of Tubingen Germany via CeGaT.  Has anyone in this community had any experiences with this?

Treatment description from their literature:

Our personalized peptide vaccination approach aims to train the immune system to recognize
specific “non-self” antigens (neoantigens) which are predicted to be specifically presented by the
patient’s tumor cells. In order to identify tumor-specific mutations, the patient’s tumor and normal
tissue sample (mostly blood) are analyzed using the modern next-generation sequencing technology.
Many of the identified tumor-specific mutations lead to alterations in proteins. Bioinformatic
prediction algorithms are subsequently applied to select those mutated peptides which are most
probably presented on the surface of the tumor cells by the patient-specific HLA molecules and
which have the potential to elicit a strong immune response. These selected peptides are synthesized
and injected as a vaccine in order to stimulate the immune system and to enhance the propagation
and activation of immune cells that specifically attack tumor cells presenting the mutated peptides.
In order to further boost the vaccine specific immune response, an immune stimulatory adjuvant (Leukine) is

co-applied at the injection site.

While they have had good results to-date they have not treated any children.  This obviously concerns us.  Does anyone have any experience with peptide vaccines in children?  The doctors say that theoretically there shouldn't be any difference beyond dosage/body mass.  What are people's thoughts on:

1) The logic of this treatment approach

2) Experiences?

3) Peptides in children

4) Experiences with FDA Personal Use Exemption program / importing off-label drugs from overseas

Again, thanks to everyone for help!

Brooklyn, NY


  1. Hi Winston, and welcome.
    I'd be interested to know more about the results of the genetic testing, as pediatric gliomas typically have a different genetic and epigenetic background vs. adult gliomas. Specifically was there mutation detected in histone 3.3 [H3F3A], or ATRX mutation? p53 mutation?

    I feel that looking at vaccines at this time is indeed logical. Vaccines are typically more effective when there is minimal residual tumor, as an actively growing tumor puts out immunosuppressive cytokines that makes any effective anti-tumor immune response less likely.

    I'm curious where you found this description of the CeGaT vaccine approach. I've heard through word of mouth that this company has created vaccines for patients on a Compassionate Use basis, but there is no information on their website about it that I can see. The company seems to be more about diagnostics than treatment.

    I'm not sure that peptide vaccination in pediatric patients would bring up any different issues versus other types of vaccines (such as tumor lysate-pulsed dendritic cell vaccines). There is some information on SL-701, a peptide vaccine, in pediatric glioma:

    "Peptide-based vaccination has been well tolerated in children with
    gliomas, with no > Grade 3 non-CNS toxicities."

  2. Stephen-

    Thanks for your quick response.

    In reviewing the sequencing documents we have seen (difficult to interpret!) we don't see the mutations you reference but that doesn't mean they are not there. The German Whole Exhome sequencing may reveal more.

    The description I pasted is from some PDFs they sent us as part of becoming enrolled in their compassionate use vaccine program.

    I'll take a look at the link you sent but that sounds reassuring.

    Thanks again

    1. I'd be happy to look at the molecular pathology results and give a shot a interpretation. My email address is on the User Information page, at the top of the blog.

  3. Winston,
    This is a devastating, terrifying disease, and the challenge is all the more magnified when the afflicted is a child.

    My own feeling is that a clinic offering an unapproved therapy should be viewed skeptically.

    A clinic that offers a treatment they've never tried on a child before should be viewed *very* skeptically.

    A clinic that suggests treating a young child as if he/she were just a small adult should not be trusted at all. Pediatric care is a specialty for a reason. Young children are not simply small adults, not biologically, not psychologically, not in terms of treatment systems or approaches needed.

    Your situation is both enviable and especially challenging. With no measurable tumor present any more, you can never know if *any* intervention is responsible for delaying/preventing relapse, or if somehow an intervention might have paradoxically hastened progression.

    As long as this remains the situation, I might suggest limiting interventions to ones that are low risk, low-impact, relatively reliable, and affordable.

    Perhaps focusing on allowing your child to live a relatively normal, happy childhood may be prudent. So something like an Optune helmet might *biologically* be a wonderful intervention, but might be a dreadful burden on a child who might (or might not) actually benefit from it.

    Meanwhile, a "methylated" tumor, generally, is known to be relatively responsive to radiation and temozolomide. Continuing with standard treatment protocols may be prudent.

    I'm a big believer in participating in well-designed clinical trials. My own wife is receiving nivolumab/OpDivo in a trial, and gets to stay on it (protocol permitting) indefinitely, at zero cost to us. Hard to beat something like that.

    Of course, almost any trial of interest won't admit her unless there is measurable tumor to gauge progress--that is, she might not qualify until new tumor tissue is detected (though not necessarily so in all trials).

    So, I'd recommend studying the available pediatric GBM trials at

    You might sort out which ones seems most promising (perhaps only to use when relapse is detected) and review these options with your oncologist (and/or folks here). That way, if and when that dreadful day happens, you might be prepared with a plan already in place.

    Meanwhile, low-risk interventions that aren't a massive burden on anyone seems reasonable. I might suggest curcumin and mushroom extracts (PSP/PSK, etc) as benign additions. Many others are worth considering. Clicking on terms in the column on the right will yield many potentially valuable leads.

    Best wishes,


    1. Just a quick comment on Optune; you won't be permitted to use it as it's not approved for people below 21 years of age. This is because it affects fast growing cell, which a child has in his/her brain naturally as the brain is still growing.

      That is has NED is wonderful. I agree with everything that Steve has said in proceeding cautiously and judiciously.

      All the best, this is a beast at any age but certainly more so for a child.

    2. Thanks for the clarification. You're quite right about Optune, I hadn't thought of that when I raised it as a hypothetical example.
      Really, this is an example of how children can't be treated as if they were just small adults.

  4. Immunotherapy is at best at minimal residual disease. One can fear that there are still some silenct glioma cancer stem cells residing that will restart growing at a certain time point. These cells are not sensible to chemo- nor radiotherapy. Immunotherapy can still target them. There are some publications in the internet on immunotherapy for children with malignant glioma. Geiger JD et al. Cancer Res 2001;61:8513, Ardon H et al. Pediatr Blood Cancer 2010;545:519. So there is experience with tumor lysate pulsed DCs. The Pittsburg group generated results in children on vaccinations with peptides as well. The proposed approach for defining tumor-specific peptides by comparing tumor material with healthy material is a well known strategy of current research for vaccines in adults. Early clinical experiences show feasibility without major toxicity. In part with this methodology one escapes to the MHC restriction of most classical defined peptide vaccines. As this is a known immunology and immunotherapy methodology my guess is that this methodology can in theory also be applied to children. Administration to a child will be under the condition of "Individuelles Heilversuch".