DIPG Family Help
Our son Simon Aliotta was diagnosed with Diffuse Intrinsic Pontine Glioma
(DIPG) on 12/26/2006 and then passed away on 10/13/2007. Neither my wife nor I are doctors but we thought
we would share our thoughts to help other families in similar situations.
This section was written for parents of DIPG children. We have no
qualifications except our time researching and talking to doctors about
Simon. We write this section --- not for you to believe us --- but to
provide you with enough background so that you can ask intelligent questions
of the doctors.
We would like to collect; however, the treatments that each family is
trying and has tried. If you have been diagnosed with DIPG, since July
2006, we are interested in the treatment that you have tried and the
results.
If you have a child who is diagnosed with DIPG, we would be happy to speak with you and answer
any questions contact me at
jeff@aliotta.com.
FAQ
What does 'Diffuse Intrinsic Pontine' Glioma mean?
Will my child live?
Where should I go for
treatment?
What treatments are available?
Is it
reasonable to just do radiation therapy?
How are the other children in the clinical trials doing?
Can the cancer spread?
What does 'Diffuse Intrinsic Pontine Glioma' really mean?
Here is my definitional breakdown. For more
definitions, go to the National
Cancer Institute
Diffuse - Widely Spread; not localize or confined. not
focal.
Intrinsic - Located inside the structure; not external
or extrinsic to structure.
Pontine - Located in the Pons of the Brainstem; The
brain stem has three parts (Pons,
midbrain and
the
medulla). The Pons controls critical functions such as
breathing making surgery extremely dangerous.
Glioma - type of primary
central nervous system (CNS)
tumor
that arises from
glial cells. The most common site of involvement of gliomas
is the brain, but they can also affect the spinal cord or any
other part of the CNS, such as the optic nerves
In English terms, the way to think about it is that 'Glioma'
is the noun and 'Diffuse', 'Intrinsic' and 'Pontine' are the
adjectives. So first, the word 'Glioma' tells you that it
is a central nervous system tumor. The word 'Pontine'
describes the location of that tumor which is in the
pons of the
brain stem. The word 'Intrinsic' tells us that the Glioma
is inside the pons
as opposed to attached externally to it. Finally, the word
'Diffuse' tells us that the Glioma is widely spread within the
pons as opposed
to being located in one spot.
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Will my child live?
Research Summary
According to the
Pontine Glioma Disease summary on St. Jude's website
survival past 12-14 from diagnosis date months is 'uncommon'.
The National Cancer Institute estimates survival after 18 months
at only 10%
(click here to see NCI reference). The Journal of Clinical Oncology published a
summary on DIPG that put 1 year survival between 30 and 50%, but
3 year survival near 5%.
As for progression of the disease,
the initial treatment of radiation often shows significant
improvement with the tumor shrinking dramatically and symptoms
often disappearing. The tumor though soon starts to grow
again. Once the growth returns, the National Cancer
Institute does not recognize any treatment that has extended
survival and recommends entry into clinical trials. (Click
here to see NCI recurrent reference)
The general consensus is that
survival is very poor; however, some children do survive for at
least a few years. I though have not found anyone
who has survived greater than 5 years.
What we believe!
Bottom line, life can be extended and perhaps long enough for a
cure. Actual survival rates
though are quite ambiguous due to the rare nature of the
disease. Furthermore, estimates of length of survival
(i.e. 30 to 50% 1 year survival rate) vary widely due to the wide
variation of when symptoms appear that would lead to an MRI or
PET scan. Personally, we think we were fortunate with Simon
that such an obvious symptom (his eye) indicated that an MRI
should be performed. Regardless, all length of survival
data starts with diagnosis date so hopefully, your child was
diagnosed early.
Anecdotally and according to the National
Cancer Institute, we believe that if after
the initial radiation treatment the tumor continues to grow
(often 4-6 months later), there is almost nothing to do that can
stop the growth and comfort care needs to be considered..
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Where should I go for
treatment?
Research Summary
The hospitals associated with the
Pediatric Brain Tumor Consortium (PBTC) provide the greatest range
of treatment options as they are conducting almost all of the
clinical trials which provides access to pre-FDA drugs.
Non-member hospitals do NOT have access to pre-FDA drugs
or all possible clinical trials. The following is a list
of these hospitals (in alphabetically order) within the PBTC.
All of these hospitals
participate in the joint clinical trials
particularly due to the rare nature of DIPG, in
order to get enough patients in a trial, they need
each hospital to include some children. St.
Jude collects the data from all the hospitals and is
the 'PBTC Operations & Biostatistics Center'.
Most importantly! If you child has just
been diagnosed, DO NOT begin treatment at another
hospital until you at least speak to one of these
institutions. If you begin treatment somewhere
else, you will NOT be allowed to participate in any
clinical trial for newly diagnosed patients.
We
will also mention a controversial alternative therapy clinic
called the Burzynski
Clinic. To be frank, there is wide variety of comments of
Dr. Burzynski from genius to quack. We provide information
only because he is running a clinical trial (Clinical
Trials: Antineoplastons in Treatment of Brainstem Glioma)
that is monitored by the FDA.
What I believe
This is a very difficult decision for everyone
particularly if travel far from home is required.
Although if you live near one of these institutions,
we believe it is an easier decision. For Simon,
we decided to go to Duke (~130 miles away from where
we live) and participate in a clinical trial.
We are happy with our decision and would do it
again.
Regardless of your decision, we would
suggest you have one of the PBTC institutions look at your case prior to
beginning treatment at a non-PBTC hospital.
You do NOT have to necessarily travel there, simply
have the hospital that diagnosed your child send the
MRI/PET Scan results to the appropriate contact at
one of these hospitals. The PBTC doctor's will
review and at least speak to you prior to having to
go visit.
The other advantage of these hospitals
is that they have much more experience dealing with
children, particularly children with brain cancer.
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What treatments are
available?
We will repeat my earlier statements that we have neither
been trained nor are we doctors. We first encourage the family to
go to the National Cancer
Institute; however, the information is sparse. We also
encourage you again to go to one of the
PBTC Hospitals.
Finally, while all Diffuse Intrinsic Pontine Gliomas (DIPG)
are Brain
Stem Gliomas, not all Brain Stem Gliomas are DIPGs. This
only applies to Diffuse Intrinsic Pontine Gliomas (DIPG).
For a newly diagnosed Diffuse Intrinsic Pontine Glioma, the
initial treatment will be a combination of Radiation (also known
as XRT) and chemotherapy (i.e. a drug). Please
note, I understand that for some patients the DIPG tumor has
grown so fast that no therapy is available. Additionally,
if the DIPG is a result of a spread of cancer from another
location, this information will probably not be of help to you. The Radiation Therapy is standard for all cases (whether you are
in a clinical trial or not) and can be given at either one of
the PBTC hospitals or at a large metropolitan hospital.
The radiation is the standard treatment (also known as protocol)
that usually shows good, though short-term, results.
The chemotherapy portion is the addition to the Radiation
Therapy and usually begins simultaneously with the radiation
therapy and continues with some breaks after radiation therapy
is finished. As of Simon's diagnosis in December 2006, the
standard (non-clinical trial drug) is
Temozolomide
(also known as
Temodar) for DIPG. The results from
DIPG Temozolomide Clinical Trial are not formally published
(as far as I could tell).
Due to this Temodar's ineffectiveness for
DIPG patients, the PBTC is now also conducting
other trials for DIPG including pre-FDA drugs. Our son
Simon is participating in the clinical trial "PBTC-0014 - Phase I/II
Trial of R115777 and XRT in Pediatric Patients with Newly
Diagnosed Non-Disseminated Intrinsic Diffuse Brainstem Gliomas".
The drug for this clinical trial is a called
Zarnestra (also known as
Tipifarnib).
For parents, please understand that for
childhood diseases, the ethical boundaries are very clear but
lead to different suggestions of treatment from a local doctor
vs. a doctor at a brain cancer institute. The local doctor is
likely to recommend the standard Radiation Therapy plus Temodar
because that is considered standard even though the chemotherapy
(i.e. Temodar) is considered completely ineffective.
If
Temodar (aka
Temozolomide) showed any effectiveness even in extending life just a
few months, all doctors would be ethical bound to treat our
children with it. The fact that there are other clinical
trials attempting other drugs with the Radiation tells us (the
parents) that Temodar is most likely completely useless.
In other words, the FDA would not allow doctors to enroll
children into a clinical trial if another treatment option was
available that showed success. I
also will say when I spoke to Duke University about the
treatment with Temodar,
Dr.
Friedman, Director of the
Duke's Brain Cancer Institute
and Clinical lead for the Temodar clinical trial,
told me that Temodar does not work.
Finally,
again think of the ethics if the tumor returns (which is likely)
post-radiation. Because there is no treatment that has
been proven to extend life, the doctors are free to enter the
children into new clinical trials. If any treatment out
there were proven to extend life, then the doctors would be
ethically bound to provide that treatment before allowing a
child to enter a clinical trial. This is true of the Burzynski
Clinic which we mentioned above is called by many a quack
though it does run one clinical trial. The FDA allows this
trial only after the tumor returns following radiation therapy.
Additionally,
we understand that some patients
particularly those who have recurring tumors are now trying
bevacizumab (also known as
Avastin). The clinical trial is called "Phase
II study of Bevacizumab plus Irinotecan (Camptosar™) in Children
with Recurrent, Progressive, or Refractory Malignant Gliomas
And Diffuse/Intrinsic Brain Stem Gliomas".
A
recent article states that there is some evidence that
Avastin has shown the ability to extend quality of life of
adults with brain cancer although still ending in death.
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Is it reasonable to just do radiation therapy?
Yes. The only proven beneficial treatment is the
radiation. In almost all cases, the radiation
significantly shrinks the tumor for a period that is measured in
months providing a high quality of life for those months.
Furthermore, unlike many other cancers the radiation therapy is
isolated to a very small portion of the body thus the side
effects of the radiation are usually minimal (or at least much
less than other cancers).
There is no chemotherapy that has really proven significantly
beneficial, thus, all the clinical trials. It is both very
reasonable and perhaps in the child's best interest to not
subject the trial to additional experimental drugs or treatment.
What I believe
This is a choice best left to the parents -- as long as the
parents are comfortable with the choice than I believe the
choice is correct. The facts are that radiation is the
only proven (although temporary) therapy. All other
treatments, particularly chemotherapy, have little proof of
improvements so it is given the less than one year life
expectancy.
We chose to enter a clinical trial to get access to a new
type of chemotherapy as despite the odds, we
wanted to try everything. Did it help? Who knows -
probably not significantly. We also take some comfort in
moving the overall knowledge base forward for all future
diagnosed children
If every parent only did radiation, then we will never cure
this.
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How are the other children in the clinical trials doing?
This list is for those children that I know that have
participated in the
Zarnestra clinical trial. Kyle Roger is the only child
still alive from the trial though fighting a recurrence and the
drug did not save any child.
The jury though is still out if this extends life more than just
radiation alone. As of 2-25-2008, my understanding is that
the trial is no longer taking any new children. If your child is or was in the
trial, please e-mail me at
jeff@aliotta.com
| Child |
Diagnosed Date |
Zarnestra Trial Status (as of
10-20-08) |
|
Elena Desserich |
11/28/06 |
Elena passed away on August 11, 2007 |
|
Sam Jones |
12/06/06 |
Sam's tumor showed signs of growth and reappeared in
August. Trial discontinued. Sam then
tried the
Avastin Trial. Sam passed away on Feb 25th,
2008. |
| Simon Aliotta |
12/26/06 |
Simon's tumor both returned and spread to spine.
Simon passed away on Oct 13th, 2007. |
|
Madeline Adams |
01/04/07 |
Madeline tumor exhibited growth.
She then tried the
Avastin Trial.
Madeline passed away on November 30, 2007 |
|
Kyle Roger |
02/08/07 |
First MRI showed 80%
decrease. Tumor showed regrowth in spring in 2008.
As of October, Kyle continues to battle putting his
survival at an unusual 18 months after diagnosis. |
|
Sophie Quayle |
02/21/07 |
Sophie passed away on October 6th, 2007. |
|
Chance Mitchell |
05/21/07 |
Chance's initial MRI
showed decrease in tumor size. Tumor showed
re-growth in mid-November 2007. Chance then
tried
etoposide. Chance passed away on December 10, 2007 |
|
Caitlyn Churak |
05/23/07 |
Caitlyn's initial MRI
showed decrease. Trial stopped in October, 2007.
Caitlyn then tried
Avastin Trial and is has now stopped avastin.
Caitlyn passed away in June, 2008; |
|
Lindsay Adams |
07/19/07 |
Lindsay's first MRI
post-Radiation showed decrease in tumor size. In May
2008, the tumor showed re-growth and she is no longer on
trial. Lindsay passed away in June 2008. |
|
Raymond Vaneman |
08/11/07 |
Raymond's first MRI
showed decrease but many of the symptoms remained.
Raymond and his family decided to discontinue the trial
in January, 2008. Raymond passed away on
02/18/2008. |
I will also add that while that the radiation therapy alone
usually decreases the size of the tumor initially, the success
of Zarnestra will really be determined by the limiting of the
return of the tumor growth.
I have added another list of two
children who are doing well on
Iressa (aka gefitinib, ZD1839). As with Zarnestra, my
understanding is that this trial is no longer taking any
children. Please note: other
children did not see such good results, but again I pass this
along to provide as much information as possible to parents.
| Child |
Diagnosed Date |
Iressa Trial Status (as of
10-20-07) |
|
Dasia Atkinson |
03/23/2005 |
Dasia showed significant tumor shrinkage and
continues to do well |
|
Aidan Zaugg |
04/14/2006 |
In summer 2008, Aidan's tumor
returned and Aidan continues to battle. Note:
though that Aidan's tumor was held off for nearly two
years from diagnosis |
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Can the cancer spread?
Yes.
The cancer can spread to other glial cells and according to Duke
spreads to the spine about 20% of the time. The cancer is
not known though to spread to other organs. I personally
recommend that you check the spine with an MRI at least every
other MRI. At this time, there is little to do regarding
the spine spread, but knowing can help you make other decisions
about the welfare of the child. Simon, our son, did have
the tumor spread to his spine. Another case was Katie
Metz.
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