Ella's Medical History
Ella was diagnosed with
medulloblastoma five days before she turned
two. Medulloblastoma is the most common childhood
brain tumor, but it is still a rare diagnosis;
most pediatricians will only see one or two
cases of childhood brain cancer in their entire
careers. Ella contracted a very rare, more aggressive
variant, called large cell anaplastic medulloblastoma.
Two days after she was diagnosed
a neurosurgeon removed the primary brain tumor,
but the cancer remained over a large portion
of the surface of her brain and in her spinal
column. To battle the remaining cancer, Ella
began chemotherapy on a Children's Oncology
Group (COG) treatment protocol called ACNS0334,
undergoing two rounds of Methotrexate, Leucovorin,
Vincristine, VP16/Etoposide, Cytoxin, MESNA,
Cisplatin and G-CSF. She also had stem cells
harvested with the intention of using them in
the second part of the protocol.
The chemotherapy worked
initially, with over a 50% reduction in cancer
after the first round of treatment. But the
cancer adapted to the drugs, and an MRI after
the second round of treatment showed that the
cancer was returning quickly. Ella slipped into
a coma within weeks, and passed away a couple
of days thereafter when we removed her from
Disease and Treatment
The name medulloblastoma
refers to the location and classification of
the cancer. The tumor originated in the medulla
oblongata, which is located adjacent to the
fourth ventricle of the brainstem. The classification
of cancer, blastoma, is of unknown genetic origin,
but it can occur in the center of the brain
(pineoblastoma), in the nervous system (neuroblastoma)
and even in the retina (retinoblastoma). There
is evidence that the genetic markers for
these types of cancer exist in the patients
at birth and work in concert with the genes
that control initial brain growth, thus their
unusually early presentation in human life.
Pediatric brain tumors are
rare, but extremely dangerous to children under
three. For instance, standard-risk medulloblastoma
is overcome in more than 80% of cases in older
children, although the treatments may cause
significant permanent side effects. For children
under three, such as Ella, the prognosis is
much worse. The primary reason is that young
children cannot tolerate radiation, and suffer
tremendous brain damage from radiotherapy compared
to older patients.
Ella's oncologist told us
that treatment with the standard dose of radiation
would eventually render Ella to a permanent
infantile state, likely unable to tie her own
shoes and limited to a vocabulary of only a
few words for the rest of her life. In addition,
the radiation was not a sure cure, and the damage
it would cause to non-cancerous cells could
cause new cancers that would emerge later in
Because of these consequences,
high-intensity chemotherapy is the standard
line of defense prescribed by physicians to
patients like Ella, even though it's not nearly
as effective as radiation and chemotherapy combined.
While it offers virtually the only hope in established
treatments for curing the cancer, its toxicities
have brutal side effects on young children.
Oncologists and parents have to decide if the
tremendous suffering under chemotherapy and
its lower chances of successful outcome outweigh
the longer-term, severe, permanent side effects
of radiation, which offers a higher chance of
The Situation Today
Tragically, there is no
third, better choice, unless if you believe
in some of the alternatives offered by the shadowy
world of south-of-the-border treatment centers
and by other "cures" that have no
peer-reviewed, scientific substantiation.
Why is there no better choice?
Part of the answer is that the rarity of these
cancers in children three and younger means
that comparatively little attention has been
paid to developing new treatments for them.
For instance, the main chemotherapy drug that
Ella was being treated with, Methotrexate, was
first used in the 1950s. While some advancements
in brain tumor treatment have been made in recent
decades, particularly with stem cell transplants,
much needs to be done. For all the danger and
side effects we put children through in treatment,
we should be able to guarantee them a higher
Another reason there is
no better choice is that the cure for almost
all cancers remains elusive. The study of childhood
brain tumors would help to further the understanding
of all cancers, since many adult cancer treatment
strategies are also stuck in antiquated chemotherapy
and radiation strategies that work by poisoning
good cells as well as the bad.
Unfortunately, the incentives
may not be there to find the best cures quickly,
or even to pay attention to promising therapies
that do not offer possibilities for high profit.
The drug companies make
millions of dollars from the sales of chemotherapy
drugs, and as evidenced by a May
9, 2007 NY Times article, oncologists are
not above taking incentive payments for administering
drugs, even when those drugs are considered
For instance, according
to the NY Times article, doctors have been accepting
millions each year in kickbacks to prescribe
Aranesp, Epogen and Procrit, drugs that the
FDA have shown are not proven to be effective
and may shorten patients lives when prescribed
at high doses, which is a common practice. At
one clinic in the Pacific Northwest, a group
of six cancer doctors received $2.7 million
from Amgen for prescribing $9 million worth
of Aranesp and Epogen last year. Johnson &
Johnson has been accused of making similar payments.
In addition, even non-profit,
advocacy organizations may not be representing
patients' interests as objectively as they should.
In a January
24, 2008 article, the NY Times raised a
question regarding whether the American Heart
Association was acting in the public's best
interest regarding the drug Vytorin. A press
release from the association criticized a study
of Vytorin, but did not state that the organization
receives nearly $2 million a year from Merck/Schering-Plough
Pharmaceuticals, the joint venture that markets
Vytorin. How does this apply to cancer patients?
Dr. Sidney M. Wolfe, the head of the Public
Citizen's Health Research Group, a consumer
advocacy group, asserts that the drug industry
underwrites many professional medical organizations,
which would undoubtedly include some cancer
This situation can leave
cancer patients and their families feeling as
if no one is watching out for them. On one side,
the doctors appear to be using treatments that
are outdated, inhumane and ineffective. On the
other side, there are entrepreneurs and fanatics
who are ready and willing to offer their own
scientifically and statistically unproven "cures,"
which are hawked chiefly through personal testimonials
and pyramid schemes.
Most oncologists also appear
to have no opinion or even interest in nutritional
or supplemental treatments, although many parents
and adult patients instinctively feel these
are important. Instead, patients must wander
in a thicket of information mostly found on
the Internet and often on sites run by entrepreneurs,
some of who are clearly unscrupulous.
Many hospitals have nutritionists
on staff, but their knowledge specific to cancer
may be limited. Patients can hire independent,
professional nutritionists who specialize in
cancer treatment, but their fees are generally
not covered by insurance.
Finding a Cure?
Government and consumer
monitoring of non-profits accepting donations
toward cancer research can be insufficient.
While Americans pour money into cancer research
through direct donations, fundraising events,
the Combined Federal Campaign and other avenues,
organizations may not be forthright, or even
honest, about disclosing such things as where
these donations go.
Recently, the Chronicle
of Philanthropy reported on nonprofit managers
who exploit the Combined Federal Campaign (CFC),
which is a giving program for government employees
that generates millions of dollars for non-profits
each year. One consultant
runs a site that to us appears to look as if
it is an official government site: www.bestcfc.org.
This site purportedly reveals the "very
best cancer charities," but the
only charities listed are those of the consultant's
clients, who are certified as the "very
best" through a
certifying organization affiliated with
the consultant. Meanwhile, a network of non-profit
administrators exploit the CFC by creating
shell organizations to increase CFC visibility
and then inaccurately represent where donations
The Chronicle has also shown
reporting by fundraising organizations can be
so inconsistent that it is difficult to
know the administrative fees associated with
running fundraising events, and whether these
events are worth the organizations' and volunteers'
There is Hope
Despite these hurdles, there
is hope. Research on medulloblastoma falls under
roughly two categories: clinical research and
Clinical research is meant
to save the lives of patients using tools already
in existence, which for pediatric brain tumor
patients usually means experimenting with various
surgery, radiation and chemotherapy combinations.
So, for instance, although Ella was treated
in a "new" study, it really was just
a new combination of existing chemotherapy drugs.
A more radical example includes the daughter
of basketball star Derek Fisher, who underwent
an experimental treatment in which the chemotherapy
was delivered directly into her eye. Another
example would be the use of focused radiation,
such as a gamma knife, in which cancer cells
are specifically targeted instead of the shotgun
approach of regular radiation, in which healthy
cells are as vulnerable as cancer cells.
These studies are important,
and especially in the case of radiation there
is great progress being made. But there is also
a certain stagnant quality about them, because
they keep drawing from a limited deck of cards
to treat symptoms. The surgery/radiation/chemo
mix offers methods of suppression that sometimes
eliminate all cancer cells, but they do not
offer a cure that gets to the root of the problem.
That said, they can help
to save patients immediately and sometimes yield
great gains, and therefore should continue to
be pursued. But basic science is where the real
key to the cancer cure lies.
Basic science is a misnomer;
it's basic because it goes to the very fundamental
core of the disease and its relationship to
human physiology: it's the study of the disease's
genesis and method of dissemination. When that
knowledge is furthered, it can lead to what
is called "translational research,"
which takes the new basic science knowledge
to lead to a drug or other cure.
Cancer researchers face
two very large hurdles: the disease itself can
be difficult to understand, and because they
involve cells in humans, they are usually very
sophisticated at resisting cures. So, for instance,
when you hear about someone taking an HIV "cocktail"
of drugs, they are actually taking multiple
medications that attack the virus in different
ways. The virus is plastic enough that if you
just presented one attack, it would figure out
its way around the roadblock and continue to
adapt and reproduce. With a cocktail, you are
presenting enough different methods of attack
that you essentially back the virus in a corner.
Cancer cells act with the
same resilience. They reproduce quickly and
therefore adapt quickly. The more basic science
that is done, the better researchers will understand
the disease, and the more possibilities they
will have for actually eliminating it. But right
now, basic science research funding in childhood
brain cancers is scarce. In large part, this
is because childhood brain cancers are rare
and therefore not a national priority, but the
system is not operating as well as it should,
either. The National Institutes of Health, which
includes the National Cancer Institute, has
had cuts to its programs under the Bush administration,
and funding is much scarcer than it should be.
How You Can Help
Clearly, the system
is not working optimally. But the goal is worthy-to
cure cancer in children and adults. Please do
what you can toward this end by helping
in these powerful ways.