80% of children are cured of their cancer, but 20% aren’t. The most important thing we can do is find
new treatment modalities for those 20% of children who die of their cancer. One of the important
things about Markey Cancer Center is we have a very comprehensive program for pediatric cancers.
We're providing that really critical leading care to children in Kentucky and
very much supported by the Pediatric Cancer Research Trust—is our research programs. So we
are doing groundbreaking research in pediatric cancers by identifying new mechanisms that are
causing these cancers and identifying new treatment modalities for these cancers.
We are the first state institution to be hosting a molecular tumor board
focused on pediatric brain tumors. And so, what the Molecular Tumor Board does,
is it brings together a team of interprofessional experts to interpret the tumor genomics, and
once those tumor genomics are discussed, they can help us pick the best treatment for that patient.
About 10% of pediatric cancers have a known genetic cause, but we're really still in the
infancy of figuring out what the other genetic causes are. As we assimilate data and understand
the genomic makeup of these tumors, we will build data resources and informatics tools around those
to provide better decision-making tools for our clinicians. So a couple of years ago,
we noticed that childhood cancer rates in Kentucky appear to be higher than the national rates.
With help from the Kentucky Cancer Registry’s Virtual Tissue Repository, we actually achieved
obtaining 258 specimens. And so, that involved contacting eleven different pathology facilities
throughout Kentucky and three facilities outside of the state to obtain these specimens.
The great thing about collaborating with CHOP is that we also have access to the Children's
Brain Tumor Network data and they have literally thousands of cases. One particular
mutation that we’ve observed—KN2C—appears to be more prevalent in our cases. That's a common
mutation that we find in breast cancer. And so, it’ll be interesting to determine if this actual
mutation or others are occurring at a higher rate in our kids compared to other children.
The response rate from that study has been phenomenal.
Of all the patients and families that we’ve contacted so far, about 80% of those have
agreed to participate in the study, which is really remarkable. That’s probably the
highest response rate that we’ve ever seen in a study of Kentucky cancer patients.
So one of the little-known facts about the immune system is that it attacks bacteria and other
foreign things, but it also attacks cancer cells. And one of the main players in this response
are macrophages. They can be pro-cancer or they can be anti-cancer. We are developing
a method for changing the cancer-supporting macrophages to anti-cancer macrophages that
can then attack that tumor and get rid of it. We take them into the lab and we polarize them. So,
the real benefit of these polarized macrophages is they go right to the cancer. They also should
cause less adverse effects and they should go directly to the tumor and have the effects there.
So artesunate is a drug that actually comes from a plant called Artemisia annua,
and Artemisia annua is typically grown in India or China. However—interestingly enough—it is
cultivated almost exactly like tobacco. So, we are growing Artemisia annua in Kentucky. And as part
of that project, we are evaluating the anti-cancer activity of constituents of that plant.
We have isolated different chemicals. We found out that they work better than artesunate.
They’re actually stronger. And so right now, we’re testing them in preclinical
models with the anticipation of moving on to animal models. They’re very non-toxic,
so we can give them to patients, and they won't have as many side effects. Trust fund funding
has really been essential to this work. I’m not sure we could have done any of this without some
financial support that covers staff and material expenses. And we’re already seeing a return
on that investment. And so, we’ve obtained funding for two additional studies—one related
to participating in the National Childhood Cancer Registry. Another study was funded to collect
pathology slides and scan those to create digital images of slides for childhood tumors.
This funding is instrumental to our work. Pediatric cancers are very rare. It’s very hard to get
funding for pediatric cancers. So having this type of support that is focused on these cancers that
affect children is critical for us to continue our work and make these groundbreaking discoveries.