be effective in the 5% of pediatric and 25% of adult acute
lymphoblastic leukemia (ALL) patients who also have this
Further, 2 FDA-approved kinase inhibitors are now the
standard of care for non-small cell lung cancer; 4 are now
used to treat renal cell carcinoma; 2 each are used to treat
gastrointestinal stromal tumors and pancreatic
neuroendocrine tumors; and 1 each are used to treat
metastatic breast cancers and medullary thyroid cancers.
Additionally, a very unique small molecule inhibitor, called
bortezomib (Velcade), is now the standard of care for
patients with multiple myeloma. This interesting drug works
by blocking the breakdown of proteins, which leads to the
disruption of multiple pathways that are necessary for tumor
The small molecule inhibitors, like imatinib (Gleevec),
dasatinib (Sprycel), and nilotinib (Tasigna) have rendered all
but a few blood cancers chronic rather than lethal
conditions. Unfortunately, for many patients with solid
malignancies, these new precision drugs are used to treat
their disease after it has already progressed on less precise
therapies (see Table 2, pp. 40-41). However, these new,
more precise drugs provide new hope for long-term survival.
With more progress, it is likely that, in the near future,
precision therapies like the ones described here will be the
first choice of treatment for all appropriate patients.
Short Hills, N.J.
In 2005, I was diagnosed with melanoma. It happened after my doctor
found a protruding lesion on my chest and reluctantly had it biopsied.
After it proved to be melanoma, he referred me for surgery and my first
consultation at Memorial Sloan-Kettering Cancer Center. At this point, my
scans did not show signs of any metastases in my body.
A month later the lesion was surgically removed, and I spoke with
multiple doctors to get opinions on which therapy to pursue. The data
were confusing, but I made my decision: interferon. The first night after
the initial drug infusion was the most painful and difficult I would
experience over the next six years. However, the hospital staff learned
how to reduce the side effects, and after a month as an outpatient, I
continued with a year of interferon self-injection and scans.
By 2007, however, scans showed lesions on my lung, and I consulted
chest surgeons. Surgery revealed additional disease cancer in nearby
lymph nodes as well. Still grasping for any way to try to slow the
disease, I went on a medication that stimulates the production of blood
cells that would hopefully fight off the melanoma, even though it is not
FDA approved by the U.S. Food and Drug Administration (FDA) for the
treatment of melanoma. I did well for several months on the therapy,
known as granulocyte-macrophage colony-stimulating factor, or GM-CSF, until additional lesions on my lungs were detected.
In early 2008, things started to look up. I began four rounds of
interleukin- 2, or IL- 2, and the lesions shrank measurably. I was generally
encouraged. The constant therapy was very demanding, but at the time
few other treatment options were available. Meanwhile, talk of a new
drug, ipilimumab (“ipi”), was spreading. Fortunately for me, the
melanoma was not.
But by 2009, scans showed new tumors in my lungs, and for the first
time, a brain lesion. At my doctor’s suggestion, I immediately joined a
small clinical trial to study the effectiveness of ipi on brain lesions.
I experienced side effects and actually missed a round of treatment as
a consequence, but scans quickly showed that my lung metastases
had stabilized, even though ipi was ineffective on my brain lesion.
I underwent radio-surgery for the brain lesion and the ipi has kept the
metastases in my lungs stable with manageable side effects. Going into
the last few ipi infusions on my two-year trial I remain tremendously
encouraged. Because of my experience and the experience of other
patients like me, ipilimumab (Yervoy) was FDA-approved in April.
Ipilimumab is an antibody that re-activates your immune system so
that it can help clear your melanoma, see Harnessing the Patient’s
Immune System, p. 59.
I could easily dismiss my three prior treatments as unsuccessful
because my disease kept progressing. However, they helped me get to
ipi. One major take-away from my experience is that patients should
absolutely accept the argument that even in the face of a tough
prognosis, the situation can change very quickly. And treatments that
offer what is seemingly only incremental survival might actually be the
ticket to longer-term success, because they may get you to the
treatment that ultimately works.