Meet Dr. Renee Wegrzyn, the first Director of President Biden's new health agency, ARPA-H
In today’s podcast episode, I talk with Renee Wegrzyn, appointed by President Biden as the first director of a health agency created last year, the Advanced Research Projects Agency for Health, or ARPA-H. It’s inspired by DARPA, the agency that develops innovations for the Defense department and has been credited with hatching world-changing technologies such as ARPANET, which became the internet.
Time will tell if ARPA-H will lead to similar achievements in the realm of health. That’s what President Biden and Congress expect in return for funding ARPA-H at 2.5 billion dollars over three years.
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How will the agency figure out which projects to take on, especially with so many patient advocates for different diseases demanding moonshot funding for rapid progress?
I talked with Dr. Wegrzyn about the opportunities and challenges, what lessons ARPA-H is borrowing from Operation Warp Speed, how she decided on the first ARPA-H project that was announced recently, why a separate agency was needed instead of reforming HHS and the National Institutes of Health to be better at innovation, and how ARPA-H will make progress on disease prevention in addition to treatments for cancer, Alzheimer’s and diabetes, among many other health priorities.
Dr. Wegrzyn’s resume leaves no doubt of her suitability for this role. She was a program manager at DARPA where she focused on applying gene editing and synthetic biology to the goal of improving biosecurity. For her work there, she received the Superior Public Service Medal and, in case that wasn’t enough ARPA experience, she also worked at another ARPA that leads advanced projects in intelligence, called I-ARPA. Before that, she ran technical teams in the private sector working on gene therapies and disease diagnostics, among other areas. She has been a vice president of business development at Gingko Bioworks and headed innovation at Concentric by Gingko. Her training and education includes a PhD and undergraduate degree in applied biology from the Georgia Institute of Technology and she did her postdoc as an Alexander von Humboldt Fellow in Heidelberg, Germany.
Dr. Wegrzyn told me that she’s “in the hot seat.” The pressure is on for ARPA-H especially after the need and potential for health innovation was spot lit by the pandemic and the unprecedented speed of vaccine development. We'll soon find out if ARPA-H can produce gamechangers in health that are equivalent to DARPA’s creation of the internet.
Show links:
ARPA-H - https://arpa-h.gov/
Dr. Wegrzyn profile - https://arpa-h.gov/people/renee-wegrzyn/
Dr. Wegrzyn Twitter - https://twitter.com/rwegrzyn?lang=en
President Biden Announces Dr. Wegrzyn's appointment - https://www.whitehouse.gov/briefing-room/statement...
Leaps.org coverage of ARPA-H - https://leaps.org/arpa/
ARPA-H program for joints to heal themselves - https://arpa-h.gov/news/nitro/ -
ARPA-H virtual talent search - https://arpa-h.gov/news/aco-talent-search/
Dr. Renee Wegrzyn was appointed director of ARPA-H last October.
New Podcast: Jessica Malaty Rivera Talks Vaccine Hesitancy
"Making Sense of Science" is a monthly podcast that features interviews with leading medical and scientific experts about the latest developments and the big ethical and societal questions they raise. This episode is hosted by science and biotech journalist Emily Mullin, summer editor of the award-winning science outlet Leaps.org.
Listen to the episode:
Stacey Khoury felt more fatigued and out of breath than she was used to from just walking up the steps to her job in retail jewelry sales in Nashville, Tennessee. By the time she got home, she was more exhausted than usual, too.
"I just thought I was working too hard and needed more exercise," recalls the native Nashvillian about those days in December 2010. "All of the usual excuses you make when you're not feeling 100%."
As a professional gemologist, being hospitalized during peak holiday sales season wasn't particularly convenient. There was no way around it though when her primary care physician advised Khoury to see a blood disorder oncologist because of her disturbing blood count numbers. As part of a routine medical exam, a complete blood count screens for a variety of diseases and conditions that affect blood cells, such as anemia, infection, inflammation, bleeding disorders and cancer.
"If approved, it will allow more patients to potentially receive a transplant than would have gotten one before."
While she was in the hospital, a bone marrow biopsy revealed that Khoury had acute myeloid leukemia, or AML, a high-risk blood cancer. After Khoury completed an intense first round of chemotherapy, her oncologist recommended a bone marrow transplant. The potentially curative treatment for blood-cancer patients requires them to first receive a high dose of chemotherapy. Next, an infusion of stem cells from a healthy donor's bone marrow helps form new blood cells to fight off the cancer long-term.
Each year, approximately 8,000 patients in the U.S. with AML and other blood cancers receive a bone marrow transplant from a donor, according to the Center for International Blood and Marrow Transplant Research. But Khoury wasn't so lucky. She ended up being among the estimated 40% of patients eligible for bone marrow transplants who don't receive one, usually because there's no matched donor available.
Khoury's oncologist told her about another option. She could enter a clinical trial for an investigational cell therapy called omidubicel, which is being developed by Israeli biotech company Gamida Cell. The company's cell therapy, which is still experimental, could up a new avenue of treatment for cancer patients who can't get a bone marrow transplant.
Omidubicel consists of stem cells from cord blood that have been expanded using Gamida's technology to ensure there are enough cells for a therapeutic dose. The company's technology allows the immature cord blood cells to multiply quickly in the lab. Like a bone marrow transplant, the goal of the therapy is to make sure the donor cells make their way to the bone marrow and begin producing healthy new cells — a process called engraftment.
"If approved, it will allow more patients to potentially receive a transplant than would have gotten one before, so there's something very novel and exciting about that," says Ronit Simantov, Gamida Cell's chief medical officer.
Khoury and her husband Rick packed up their car and headed to the closest trial site, the Duke University School of Medicine, roughly 500 miles away. There they met with Mitchell Horowitz, a stem cell transplant specialist at Duke and principal investigator for Gamida's omidubicel study in the U.S.
He told Khoury she was a perfect candidate for the trial, and she enrolled immediately. "When you have one of two decisions, and it's either do this or you're probably not going to be around, it was a pretty easy decision to make, and I am truly thankful for that," she says.
Khoury's treatment started at the end of March 2011, and she was home by July 4 that year. She say the therapy "worked the way the doctors wanted it to work." Khoury's blood counts were rising quicker than the people who had bone marrow matches, and she was discharged from Duke earlier than other patients were.
By expanding the number of cord blood cells — which are typically too few to treat an adult — omidubicel allows doctors to use cord blood for patients who require a transplant but don't have a donor match for bone marrow.
Patients receiving omidubicel first get a blood test to determine their human leukocyte antigen, or HLA, type. This protein is found on most cells in the body and is an important regulator of the immune system. HLA typing is used to match patients to bone marrow and cord blood donors, but cord blood doesn't require as close of a match.
Like bone marrow transplants, one potential complication of omidubicel is graft-versus-host disease, when the donated bone marrow or stem cells register the recipient's body as foreign and attack the body. Depending on the severity of the response, according to the Mayo Clinic, treatment includes medication to suppress the immune system, such as steroids. In clinical trials, the occurrence of graft-versus-host disease with omidubicel was comparable with traditional bone marrow transplants.
"Transplant doctors are working on improving that," Simantov says. "A number of new therapies that specifically address graft-versus-host disease will be making some headway in the coming months and years."
Gamida released the results of the Phase 3 study in February and continues to follow Khoury and the other study patients for their long-term outcomes. The large randomized trial evaluated the safety and efficacy of omidubicel compared to standard umbilical cord blood transplants in patients with blood cancer who didn't have a suitable bone marrow donor. Around 120 patients aged 12 to 65 across the U.S., Europe and Asia were included in the trial. The study found that omidubicel resulted in faster recovery, fewer bacterial and viral infections and fewer days in the hospital.
The company plans to seek FDA approval this year. Simantov anticipates the therapy will receive FDA approval by 2022.
"Opening up cord blood transplants is very important, especially for people of diverse ethnic backgrounds," says oncologist Gary Schiller, principal investigator at the David Geffen School of Medicine at UCLA for Gamida Cell's mid- and late-stage trials. "This expansion technology makes a big difference because it makes cord blood an available option for those who do not have another donor source."
As for Khoury, who proudly celebrated the anniversary of her first transplant in April—she remains cancer free and continues to work full-time as a gemologist. When she has a little free time, she enjoys gardening, sewing, or maybe traveling to national parks like Yellowstone or the Grand Canyon with her husband Rick.