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Home/People In The News/‘Engineering’ OA Treatment Lands $31M Research Award
People In The News

‘Engineering’ OA Treatment Lands $31M Research Award

May 8, 2024 2 min read Premium comments

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‘Engineering’ OA Treatment Lands $31M Research Award
Farshid Guilak, Ph.D. and Christine T. N. Pham, M.D. / Source: Washington University in St. Louis
#washingtonuniversity#advancedresearchprojectsagencyforhealth

The Advanced Research Projects Agency for Health (ARPA-H) has awarded up to $31 million to researchers at Washington University in St. Louis to support the development of non-surgical osteoarthritis treatment.

The award is part of the Advanced Research Projects Agency for Health’s program for novel innovations for tissue regeneration in osteoarthritis (NITRO). The Advanced Research Projects Agency for Health is part of the U.S. Department of Health and Human Services. In 2022 it was created to enhance the government’s ability to, per the press release, “accelerate biomedical and health solutions.”

The research will be led by Washington University Mildred B. Simon Professor of Orthopaedic Surgery and Shriners Children’s St. Louis Director of Research, Farshid Guilak, Ph.D. Dr. Guilak’s research has focused on advancing therapeutics to prevent or reverse OA progression. The funding will enable a team of scientists to focus on, per the press release, developing “a single-injection treatment that promotes tissue regeneration and restores joints.”

In the press release, Dr. Guilak commented, “Osteoarthritis has one of the greatest disease burdens of any disease in the world. But we have no drugs that can reverse the joint damage it causes.”

Dr. Guilak continued, “This award is a moonshot initiative, funding high-risk projects with the goal of developing a single-injection treatment or even a cure for osteoarthritis. If successful, we could potentially affect the quality of life for millions of people and lessen the economic impact due to the billions of dollars spent treating pain caused by osteoarthritis.”

The project will be driven by a multidisciplinary team focused on developing the single-injection treatment. The team’s investigators will include the following: Christine T.N. Pham, M.D., the Guy and Ella Mae Magness Professor of Medicine and director of the Division of Rheumatology within the university’s Department of Medicine; Hua Pan, Ph.D., an associate professor of medicine; Xiaoxia Cui, Ph.D., an associate professor of genetics; Lori Setton, Ph.D., the Lucy and Stanley Lopata Distinguished Professor and chair of the Department of Biomedical Engineering; and Erik Herzog, Ph.D., the Viktor Hamburger Distinguished Professor in Arts & Sciences in the Department of Biology.

The award will fund a team effort to “develop advanced nanoparticles intended to deliver snippets of genetic code into human joint cells and treat osteoarthritis with a single, yearly joint injection.” In the press release, Dr. Herzog explained, “We are putting knobs on the cells that will allow the body to self-tune drug production according to the severity of the inflammation, degree of mechanical load and the time of day.”

OTW had the opportunity to speak with Dr. Guilak about the award. Dr. Guilak explained that his team “was selected by ARPA-H for funding based on the innovation of our approach to develop a new treatment for osteoarthritis, and the potential to move it to clinical trials within five years.”

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Dr. Guilak continued, “The goal of this is study is to reprogram the cells in the knee joint to be ‘smart,’ meaning we will use advanced bioengineering methods to rewire their genes so that they automatically produce biologic drugs to fight pain and inflammation and regenerate the cartilage and bone in the joint that was lost to arthritis.”

“The team is currently testing the ability of these cell reprogramming methods in the lab to show that they produce enough biological drugs on demand, whenever the cells sense inflammation or arthritic changes in the joint.”

React:

Discussion

14
DS
Dr. Sarah MitchellOrthopedic Surgeon · Mayo Clinic

This is a fascinating development. In my practice we've seen similar outcomes with the revised protocol. The key differentiator seems to be patient selection criteria. Has anyone else noticed the correlation with BMI thresholds?

8
JT
James Thornton, MDSpine Fellow · HSS

Great point. I'd push back slightly on the conclusion, the sample size in the cited study is too small to draw population-level inferences. That said, the directional signal is compelling and worth a larger RCT.

5
RP
R. PatelSports Medicine · Stanford

We implemented a similar approach last year. Early results are promising but we're still gathering 12-month follow-up data. Happy to share our protocol if anyone is interested.

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