Dr. Wilky’s new clinical trial likely available early 2016

Upon Manny’s diagnosis, I’m sure many of you – his friends, family, and aquaintances – resorted to “googling” ASPS because you had no idea what it was. I’m also sure you were all disappointed by what you saw.

Manny’s oncologist, Dr. Breelyn Wilky, always encouraged us to avoid searching information about ASPS because there is so little confusing, misleading information because of a lack of knowledge on the topic.

“You can’t get discouraged by looking at the statistics on sarcoma,” Wilky said. “There are some patients that do very well, and we can’t explain it, but someone has to figure it out.”

Manny was Dr, Wilky’s first ASPS patient, so when she immediately began to explore his limited treatment options, she realized she needed to do something. Since Manny’s diagnosis, she has made her mission “figuring it out.”

Dr. Wilky representing #MAF at the Capitol with her #SpearSarcoma band. She paid a visit to DC in May to fight for more funding for Sarcoma research.

Dr. Wilky representing #MAF at the Capitol with her #SpearSarcoma band. She paid a visit to DC in May to fight for more funding for Sarcoma research.

“Very few oncologists know much about sarcoma, and the known treatments are elaborate and confusing – ” Wilky said. “If I figured out anything for sarcoma it would mean that much more because nobody else wanted to take them on.”

Dr. Wilky instantly felt a special connection with Manny – one that would lead to her creation of a clinical trial that is tailored to treat ASPS. It will be available out of the Sylvester Comprehensive Cancer Center beginning in 2016. This could very-well be a break-through in treatment options for patients who typically have few to none.

Wilky first consulted with her colleague, Dr. John Goldberg, who had done research on ASPS several years prior and had treated children in his clinical trial. Goldberg’s theory was to boost their immune systems to help their bodies fight the cancer on their own, Wilky said.

While the trial was not ultimately effective, Goldberg did find that the children’s bodies responded to the treatment by creating antibodies to fight the protein that makes these abnormal blood vessels that allow ASPS to grow.

Referred to as VEGF, which stands for vascular endothelial growth factor, this protein and others related were proven to be recognized by the immune system; however, just this boost to the immune system wasn’t enough to kill the tumors.

Another issue with treating ASPS and other cancers is that some tumors use a protein “flag” known as PD-L1 that tells the immune system to not attack it.

In recent studies, medicines have been found to be somewhat successful at blocking VEGF, Wilky said. Many ASPS patients, including Manny, are given these drugs to stop the growth of and stabilize the disease, but patients are not cured of it.

Wilky’s new clinical trial, which she admits entirely came from Manny’s diagnosis, takes both concepts – boosting the immune system by blocking PD-L1 and treating with a VEGF-blocking drug – and combines them to create what she believes may be the cure for ASPS.

Her argument is that “using the stabilizing effect of a VEGF-blocking drug will buy the immune system more time to kick in.”

The VEGF-blocking drug also creates holes in the blood vessels that will allow the patients’ boosted immune systems to get to the core of larger tumors, Wilky said.

The clinical trial, which Dr. Wilky began working on in November 2014, is currently being reviewed by the University of Miami Institutional Review Board. Although this process may take up to a few months, she is hopeful that the trial will begin by February 2016.

As soon as the trial is approved, Manny will begin treatment. Until then, however, Dr. Wilky has plans for other similar treatment options.

We’ll catch you up on what’s going on with Manny lately in our next post.

Victoria Verdeja