Connor Webb developed a lump on his right thumb that raised concerns with his mother, Kim, but when queried about it, doctors told her not to worry about it and to just “take care of it whenever.”
Eventually though, the lump doubled in size, prompting surgery to remove it, but the subsequent phone call from the doctor stunned Kim – Connor had been diagnosed with epithelioid sarcoma, a slow-growing soft-tissue cancer.
After a month of tests and scans, Connor underwent a full resection of the tumour plus a bit of surrounding tissue to prevent recurrence and regained full use of his thumb. Later CT and MRI scans show no evidence of disease (NED).
This has, however, not put his mother at ease, as epithelioid sarcoma has a very ugly secret…

Epithelioid sarcoma is exceedingly rare – it is estimated that at most, there are no more than around 100 cases per year. Of those, 10% occur in children and adolescents.
Unfortunately, this cancer often comes back later – even decades later, and when it returns, it does so extremely aggressively, and there are no drugs to stop it.
The most unfortunate part about such rare cancers is that it takes a long time to find enough patients to test new therapies, and even worse is the fact that because of the small patient numbers, there is less motivation to allocate resources to study the diseases and develop potential drugs.
For families like the Webbs, whose cancers have no approved treatments beyond surgery, radiation and chemotherapy, it can feel as though there is nothing that they can do to help their children, but some of these families have found a new ally in their fight: the Children’s Cancer Therapy Development Institute, or cc-TDI, a nonprofit biotech lab that brings families and researchers together to push the field forward.
In 2016 the Creating Hope Act introduced incentives for industry to invest in rare childhood diseases. Paediatric cancers, however, offer little payoff for the investment in drug development compared to breast or colon cancer, for example, where patient numbers reach “hundreds of thousands, rather than hundreds,” says Jim Geller, an oncologist at Cincinnati Children’s Hospital.
Geller adds that because there are so few cases, “trials cannot be conducted quickly, nor can you conduct multiple trials on the same disease as easily as in adult cancer.”
With so few new treatments in the pipeline, families with children with cancer can find themselves out of options very quickly.
Over the past 40 years, the Food and Drug Administration has only approved six drugs for treating children’s cancer, as opposed to the dozens approved annually for more common cancers that affect adults.
 

The Children’s Cancer Therapy Development Institute, just outside of Portland, Oregon, channels families’ desperation to quietly transform pediatric cancer research. In less than three years after the Institute began operating out of a remodeled paint factory, it has pushed new drug candidates into three clinical trials.
The Institute was launched to help fill what founder Dr. Charles Keller calls the “preclinical gap” in childhood cancer research – where potential drugs languish in lab freezers because scientists aren’t incentivised to do the laborious experiments needed to translate promising lab findings into viable compounds for drug trials.
All the papers and grants in the world don’t mean anything if we can’t get drugs into clinical trials,” says Keller, an expert on the biology of childhood sarcomas who left his faculty position at Oregon Health & Science University in 2014 to start cc-TDI. “We’ve used our relationships with families to reboot a broken cycle.”
The Institute is equipping small armies of parents to help shape the research agenda by sharing what they know from first-hand experience with these diseases. “The more you talk with people, the more you realize they have some insights,” says Keller. “They want a way to participate.”
cc-TDI offers a paediatric cancer “nanocourse” – a weeklong crash course offered each summer for families, students and other lay people to learn from world experts, shadow scientists in the lab, collaborate with other participants to brainstorm the road to a cure and publish those plans in peer-reviewed journals.
Participants leave the nanocourse equipped to raise funds, educate their communities, and help arrange tumour tissue donations to labs that can create cell cultures and mouse models. The Institute runs its own program, called CuRe-FAST, for families to contribute tumour samples to a registry that scientists around the world can access.
The Institute’s patient-centric approach combines the rigour of an academic lab with the flexibility of a startup. Instead of depending solely on academic grants which require scientists to apply the funding toward specific research programs, cc-TDI also receives money from foundations and other philanthropic sources that do not carry such restrictions. This allows the research to move nimbly — to “go with what’s working and pivot from what’s not working,” says Keller.
The families that the Institute works with are often key to getting that funding.
A lot of foundations are started by parents,” says Geller. “And the finances they provide is incredibly meaningful.”
Last year, more than half of cc-TDI’s $1.9 million budget came from foundations.
Attending a course at cc-TDI gave Webb new knowledge and a new network. As most parents of children with cancer do, she did a lot of research on the web and also joined two Facebook groups dedicated to epithelioid sarcoma.
I had to research everything. I needed to understand what was going to help my son. But this isn’t my field,” says Webb, who hadn’t studied science since high school. “And so I’m like, where do I even go?”
One of those groups was created by Andy Woods, a Montana tile contractor whose 11-year-old daughter suffered a dire form of Wilms’ tumour, a childhood kidney cancer. Woods had become active in researching and sharing information about other rare cancers. Through Facebook messages and a frenzy of texting, he taught Webb a lot and eventually connected her with cc-TDI, where he now works.
Webb rallied three other families to join her at this summer’s nanocourse, which focused on hepatoblastoma and epithelioid sarcoma (EpS), and after the course they all had a shared mission: better treatments for epithelioid sarcoma, and ultimately a cure.
I learned more about epithelioid sarcoma and other rare cancers in one week at cc-TDI than in the last ~2.5 years since my son’s diagnosis,” Jill Cook, a mother from central Iowa, wrote in an email.
Since the nanocourse, the EpS families have set up a fundraising platform, created a research-focused Facebook group, and are holding weekly conference calls to build a website. Most importantly, they found each other.
All of us were out there by ourselves. How do you conquer something like that — researching a rare cancer — when you’re by yourself?” Webb says. “But now, from that course, we’re able to form our army. That’s the biggest thing.”
 
 

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