Power to the Patient: Meet Xconomy’s Patient Partnership Award Finalists

Xconomy Boston — 

It’s clear at this point that patients are no longer just subjects in clinical trials. Thanks to the growing power of patient advocacy groups—and an FDA eager to hear their perspective—patients and drug developers are increasingly finding creating ways to work together, as we’ve seen with the four finalists in the Patient Partnership category of the Xconomy Awards. From live storytelling to online platforms and face-to-face interactions at the earliest stages of R&D, these partnerships are showing how patients can influence the way drugs are developed and tested, help accelerate scientific research, and also get a sense of ownership in the process. Here are the finalists. Winners will be announced at the Xconomy Awards Gala on September 26 at the Hynes Convention Center.

Fulcrum Therapeutics – Collaborating From the Start

The founders of Fulcrum reached out to patient groups to figure out which diseases to go after, even before the company was officially formed in 2016. Those early conversations led the company to focus from the get-go on developing drugs for two rare genetic disorders: Fragile X, which causes intellectual disabilities, speech problems and other symptoms, mostly in males; and facioscapulohumeral (FSH) muscular dystrophy, which is marked by muscle degeneration that starts in the face, back, and upper arms. While many drug developers connect with patient groups as they approach late-stage clinical trials or commercialization, Fulcrum is unique in that it forged these relationships as an early-stage company, says Michael Tranfaglia, medical director, chief scientific officer, and co-founder of the FRAXA Research Foundation, which is focused on Fragile X and is one of Fulcrum’s partners.

Boys with Fragile X and their families were recently hosted by Fulcrum in the foyer of its Cambridge, MA, headquarters one Sunday afternoon. The company had set up play areas for the kids so that Fulcrum scientists could meet with patients and their parents. “It puts a human face on what we’re trying to do, which is hugely motivating,” says Robert Gould, Fulcrum’s CEO and president. These sorts of interactions also give hope to parents of children with Fragile X, says Tranfaglia. “Families get to feel like they’re participating in the work [towards a drug].”

Fulcrum works with another patient group called the FSH Society to obtain muscle tissue samples from people with the disease who are undergoing surgery as a part of their treatment. Fulcrum scientists have already received two samples, growing muscle cells from the tissue and using those cells to test drug candidates. Daniel Perez is the society’s president, CEO and co-founder and is so severely affected by the disease that he relies on an electric wheelchair. He says he has confidence in Fulcrum based in part on the interactions he’s had with the company since its earliest days. “It’s a unique relationship, and there’s a lot of respect for one another,” he says.

Nikhil Wagle – Metastatic Breast Cancer Project

Early engagement with patients was also key for Nikhil Wagle’s Metastatic Breast Cancer Project, an effort at the Broad Institute to try to understand why some patients with metastatic breast cancer respond better to drugs than others, and why some have more aggressive disease. To find an answer, Wagle, a breast cancer oncologist and researcher at the Dana-Farber Cancer Institute, came up with a patient-centric plan. The idea: Collect and sequence the DNA from patients’ tumors and look for genetic changes that are associated with how those patients responded, or didn’t respond, to treatment. In the year leading up to the launch of the project in late 2015, Wagle and his team worked with patients and patient organizations to hone the mission, work out the details, and build the website. “There was an enthusiastic response, but also a lot of caution and we got a lot of advice, which we took to heart,” says Wagle.

He heard loud and clear that patients want the data made public so that researchers in industry and academia can use it. Wagle and his team listened. In the next couple of months, they’ll release data from 125 sequenced tumor samples along with the relevant clinical information.

Now Wagle is starting similar projects for other cancers. He launched the Angiosarcoma Project several months ago, and initiatives for prostate and other cancers are on the way. Wagle says working with patients so closely has changed the way he thinks about research. “The value patients can contribute to research has been really underestimated,” he says.

Shire – Online Platform for Rare Diseases

Shire (NASDAQ: SHPG) is working to create online communities for people affected by two conditions that the big rare disease drugmaker has approved or experimental treatments for: Hunter Syndrome, and complications from being born extremely premature. To do this, the company last year partnered with PatientsLikeMe, which has worked with several other drugmakers and runs a website where patients can create profiles, connect with one another, and share their health information. Shire paid PatientsLikeMe to build the platforms, and the plan is for them to launch this fall. “We have a history of working in rare disease so we understand how difficult it can be for patients and caregivers to find information and other people going through the same thing,” says Dawn Irish, Shire’s head of digital experience and brand. “This is an opportunity for us to support community groups.”

Tesaro – Survey and Storytelling About Ovarian Cancer

In 2013, around the time Tesaro (NASDAQ: TSRO) was starting a phase 3 clinical trial for its now-approved ovarian cancer drug, niraparib (Zejula), the company began to realize from discussions with patient groups how little patients with ovarian cancer knew about what to expect after diagnosis—in particular, what would happen if the disease came back after surgery and chemotherapy (about 80% of women with late-stage ovarian cancer will experience recurrence). So Tesaro decided about a year ago to team up with the National Ovarian Cancer Coalition (NOCC) and the Ovarian Cancer Research Fund Alliance to conduct a survey of patients and doctors. Nearly 500 oncologists and women with ovarian cancer took the online survey earlier this year, and the results showed a wide gap in perception between doctors and patients. Although 90 percent of doctors felt they had often talked about what their patients should expect with treatment, only 40 percent of patients said that they heard those discussions. The findings were surprising, says Mary Lynne Hedley, Tesaro’s president and chief operating officer. “There seems to be this chasm of information,” she says.

Tesaro worked with the two patient groups to create a website to share the survey results. From the site, which went live in May, users can download that information and a 2-page PDF discussion guide intended to help patients ask their doctors the right questions about their treatment. Hedley says that more resource guides and other content will be added to the site in September, which is Ovarian Cancer Awareness Month. Although patients can already find information online, Tesaro provided more about how to plan for, and deal with, a possible recurrence of the disease, says Megan Murphy, NOCC’s director of business development.

In early June, on the first day of the American Society of Clinical Oncology’s annual meeting in Chicago, Tesaro hosted a live storytelling event with The Moth, which produces storytelling events and a podcast. Three women told stories about their experiences with ovarian cancer. Murphy, who was at the event, said the stories were inspiring. “Any opportunity that our community has to be in the spotlight to highlight these stories is critical to advancing the cause,” says Murphy.

This is the fifth in a series of articles highlighting the Xconomy Award finalists. See previous stories on the CEO, Startup, Young Innovator, and Commitment to Diversity categories. The winners will be announced at the Awards Gala on September 26 at the Hynes Convention Center.