A year ago, Sam and his wife knew little about clinical trials, experimental therapies, or a rare, aggressive brain cancer known as diffuse midline glioma.
Since their 6-year-old daughter was diagnosed a few months ago, they’ve become experts.
“I wouldn’t have known about any of it,” said Sam. “It’s so rare — maybe 300 cases a year — that I would have thought it was not something we ever have to worry about.”
The cost of participating in clinical trials
Now he and his wife — they asked that we not use their full name to protect their daughter’s privacy — are fluent in explaining not just diffuse midline glioma but the ins and outs of finding and enrolling in clinical trials. Their daughter just began treatment in a CIRM-supported clinical trial at Stanford. Beyond learning about the complex behavior of the immune system or how scientists are engineering patients’ immune cells to target cancer tumors, Sam and his wife had to untangle the logistical problems of getting their daughter into the trial itself.
While worrying about their daughter, Sam and his wife had to juggle keeping their jobs in Southern California, caring for their 3-year-old son, and scratching out a plan for how they’d afford not just travel to Stanford but the costs of a hotel and food for two months while their daughter participated in the clinical trial.
Sam’s concerns are echoed by others participating in clinical trials, who not only face the existential fear of their diagnosis but the stress of juggling their day-to-day responsibilities with getting treatment.
“We knew we had to do this trial (for our daughter),” he said. “It was pretty much our only option, but we didn’t know how we’d afford 60 days in a hotel in Palo Alto.”
CIRM Patient Support Program
That’s where the California Institute for Regenerative Medicine (CIRM) Patient Support Program comes in. Funded by royalties from the commercialization of therapies in CIRM-supported clinical trials, the fund assists under-resourced patients or families in California participating in CIRM-supported clinical trials. This shows CIRM fulfills its Proposition 14 commitment to help people in California access regenerative medicine clinical trials and therapies. The agency also supports a Clinical Network that provides clinical trials statewide, close to where people live.
The Patient Support Program helps California residents enrolled in CIRM-funded clinical trials with financial assistance for flights, lodging, food, and transportation they may need during clinical trial appointments. About 30 patients and their families are now being helped through the program in clinical trials across the state.
“It’s been amazing,” said Sam, who said he and his wife wouldn’t have been able to afford the costs of hotel rooms near Stanford for 60 days. “It just takes a lot of the pressure off.”
That allows the couple to focus on their daughter, who, after the first infusion of the therapy, had a few days of very high fevers and had to have fluid drained from her brain. That has passed, but they still have weeks more of treatments and observation before they can go home. It’s too soon to tell if it will work, and even this initial treatment will require follow-up visits and infusions.
“This is about access”
Sam and his family are ready for the long haul, but without assistance like this, it would be a struggle. Studies have shown that as many as 30 percent of participants in clinical trials drop out before completing treatment. Sometimes it’s related to the treatment itself. However, travel costs, lodging, day care, lost wages, and other logistical problems are common issues, according to research published in the journal Applied Clinical Trials.
The drop-off undercuts the effectiveness of the trials, which are an essential step in developing new medications.
This CIRM program addresses that, said Nimit Ruparel, senior manager for Patient Access & Support at CIRM.
“This is about access,” said Ruparel. “CIRM is good at funding research and clinical trials. This program helps ensure that Californians who need these treatments can access those trials and get those treatments.”
Built to Reach More Californians
California voters created CIRM to help accelerate the delivery of gene and cell therapies to patients who need them. The voter-supported ballot measures that created CIRM also funded the infrastructure needed for these potential cures. That meant creating labs and clinical trial sites, as well as training the next generation of scientists and researchers.
CIRM doesn’t just help fund basic research, but also clinical trials like the one at Stanford where Sam’s daughter is being treated. The agency also created a clinical network with about a dozen sites across California, bringing clinical trials closer to where people live.
For Sam and his wife, that support has meant fewer hours worrying about hotel bills and more time sitting beside his daughter through treatment. They still have weeks ahead and return visits after that. It’s too soon to know if the therapy will work. But Sam said the assistance has given them something they didn’t expect alongside hope: room to focus.
“We can be here for her,” he said.