Medical Innovation Provides Hope to Pediatric Sarcoma Patients

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 Today’s post was written by Bert E. Thomas IV, PhD, MBA, CEO of the Sarcoma Foundation of America.

SFA CEO Bert Thomas, PhD, MBA
SFA CEO Bert Thomas, PhD, MBA

As I write this blog, we find ourselves in the midst of Childhood Cancer Awareness Month, a time to educate the public about the realities of childhood cancer and to draw attention to the need for more pediatric cancer research.  For the Sarcoma Foundation of America, Childhood Cancer Awareness Month is also a time to remind everyone that sarcomas are not just a cancer of adults, but also of children and young adults.  Sarcomas account for 15 – 20% of all childhood cancers, a number that is far too large.  Unfortunately, there has been very little hope for many children with sarcoma who rely on therapies approved more than 30 years ago to treat their disease.

But I believe that there is now cause for HOPE!  And that hope comes in the form of innovative sarcoma research – the type that the Sarcoma Foundation of America funds every year.  The timeline from new discoveries to new therapeutics is long as it can take 10, 20, even 30 years for these discoveries to turn into new therapeutics that save lives.  But if we don’t fund innovative sarcoma research every year, with as much passion and effort as the sarcoma community can muster, who else is going to do it for us?

There is a payoff, even if the timelines are long.  We are in a time where there are more innovative therapeutics being explored in the clinic for pediatric sarcomas than in any other period in the past.  Let me tell you about a few.

There is HOPE in the form of immunotherapy.

The first experimental therapeutic I would like to tell you about is based on an immunotherapy platform called Vigil®.  Vigil® utilizes the patient’s own cancer cells to create a fully personalized cancer immunotherapy.  The goal of the platform is to activate the patient’s T-cells against their own unique tumor cells.   This immunotherapy approach is currently being tested in Ewing’s Sarcoma patients in a Phase 2 clinical trial.

The second experimental therapeutic I would like to tell you about is also based on an immunotherapy approach.  ADXS-HER2 is based on the Lm Technology which uses live, attenuated bioengineered Listeria monocytogenes (Lm) bacteria to stimulate the immune system to view tumor cells as potentially bacterial-infected cells and target them for elimination.  ADXS-HER2 was developed to target human epidermal growth factor receptor 2 (HER2)-expressing cancers which is expressed in certain solid-tumor cancers, including osteosarcoma. A Phase 2 clinical trial is currently being planned in collaboration with the Children’s Oncology Group.

The last experimental immunotherapy I will share is being developed for Synovial Sarcoma, which occurs about 1/3 of the time in childhood patients.   The therapeutic is called NY-ESO TCR and it targets the NY-ESO peptide which is present across multiple cancer types. NY-ESO-1 is one of the best-characterized and most immunogenic cancer testis antigens, and is frequently expressed by tumors of different origins and in advanced tumors, including synovial sarcoma.  NY-ESO TCR is currently being evaluated in a Phase 1/2 open-label clinical trial of NY-ESO therapeutic candidates in patients with synovial sarcoma.

There is HOPE in the form of new targeted agents.

There are a few experimental targeted agents that may have a role in treating pediatric sarcoma patients.  The first experimental targeted agent that I would like to share is specifically for Ewing’s Sarcoma.  TK216 is a small molecule that inhibits the biological activity of ets-family transcription factor oncoproteins in a variety of tumor types including Ewing’s Sarcoma, stopping cancer cell growth and tumor formation.  TK216 is currently being studied in a Phase 2 clinical trial in Ewing’s Sarcoma patients.

Other experimental targeted therapeutics are designed to inhibit the tropomyosin receptor kinases (TRK) family.  Growing research suggests that the NTRK genes, which encode for TRKs, can become abnormally fused to other genes, resulting in growth signals that can lead to cancer in many sites of the body.   These gene fusions can cause sarcomas in pediatric and adult patients.  While there are differences between LOXO-101 and Entrectinib that are beyond the scope of this blog, they are two more examples of how innovative targeted therapies are being developed for pediatric sarcoma patients.  Both are being evaluated in early stage clinical trials.

There is HOPE! 

While all of these experimental therapies may not make it through clinical trials to our pediatric sarcoma patients, they all represent the efforts of many scientists over the years to better understand sarcoma, the associated molecular pathways and the immune system, and translate those understandings into innovative experimental therapeutics that have the potential to save lives. The more research we fund, the more potential we have to find even more innovative therapeutics that can save the lives of our pediatric sarcoma patients.