Good Day BIO: The role of patient advocacy in R&D

Today’s new episode of the I am BIO Podcast features two patient advocates who have played a role in bringing potential treatments to rare disease patients. We recap Bayer’s event yesterday exploring how biotech can help farmers reduce their footprint AND increase their yields. (729 words, 3 minutes, 38 seconds)

The surprising role of patient advocates in drug development

The FDA’s approval of a new drug to treat ALS partially funded by $2 million from the Ice Bucket Challenge is just the latest example of the power of the patient voice—but there’s more work to do. Today’s new episode of the I am BIO Podcast features two patient advocates who have worked tirelessly to assist in drug development for rare diseases—and they’re seeing results.

“The minute my daughter was born, I looked at my mom and said, ‘That’s it. She’s not going to suffer the way that you and I have,’” says Susan Ruediger, Chief Mission Officer of the CMT Research Foundation, which she founded to fight the genetic nerve disease affecting 3 million worldwide—including 18 members of her family today.

The power of partnership: At the 2019 BIO International Convention, the CMT Research Foundation partnered with a small startup and invested an initial $125,000. The company has since raised $100 million. The foundation has funded 15 projects in four years, with two heading to clinical development and one in a phase 3 trial.  

Another challenge: “a lack of overall understanding of the natural progression of this disease,” says Nasha Fitter, Co-Founder and CEO of the FOXG1 Research Foundation, whose daughter has the debilitating genetic neurological disorder.

“How can we use tech to solve this problem?” she asked. After studies weren’t getting the enrollment needed, she tapped into her background in ed tech to develop a natural history study with 100 patients and over 10 years of data—in just one year. As a result, the first clinical trial specifically for FOXG1 is enrolling patients at NYU.

The bottom line: “As patients, especially with a progressive disease, we don’t have time to waste, we don’t have money to waste,” says Ruediger. “Our job is to make it easy for the drug developers, the drug hunters, to be attracted to our disease.”

Listen: The I am BIO Podcast is available on Apple, Google, Spotify, or wherever you get your podcasts.

Join patient advocates in Washington, D.C., next week! The BIO Patient and Health Advocacy Summit returns to an in-person event October 27-28 in Washington, D.C.—learn more and register now!

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How biotech sows ‘fields of opportunity’

Farmers don’t have to choose between crop yield and sustainability—with biotechnology, they can achieve both, as BIO member Bayer highlighted at an event yesterday at a working farm in Germany.

The context: Climate change already affects 21% of agricultural production, equivalent to losing seven years of productivity growth—and it will get worse. Meanwhile, the population is growing—and world hunger is on the rise, too.

There’s a role and an opportunity for farmers—to reduce “their ecological footprint while also increasing their yields,” said Bayer ahead of the event at the Bayer ForwardFarm in Rommerskirchen, Germany, a 115-acre, six-generation family farm utilizing the latest agricultural biotechnology. 

It’s possible with biotech solutions like…

• Hybrid wheat developed with “genomic tools,” which can “provide higher yield and yield stability,” while also helping farmers “save inputs like fuel, fertilizer, and crop protection,” says Bayer. With the growing risk of drought, gene-edited wheat is gaining acceptance, we’ve reported.
• Carbon farming, such as “low-till farming and the use of cover crops help improve soil health and reduce soil erosion.” (This can have nutrition benefits, too.)
• Biological crop protection, like the next-generation “biologic” pesticides made from peptides several biotechs are developing.

What they’re saying: “In the future, farmers will measure the success of their farms as much by the terabytes of data captured from their fields or the tons of CO2 sequestered, as by the yield produced,” said Rodrigo Santos, President of Bayer’s Crop Science Division. 

Policy is needed to help get these tools to farms—from proposed legislation to create voluntary certification programs to help farmers take advantage of carbon farming, to a regulatory framework that enables farmers and ranchers to adopt tools like precision plant breeding, biostimulants, and more. (BIO’s on it.) 

More Reading: New study shows biotech boosts agricultural outputs, sustainability

President Biden’s Tuesday: He will speak at a DNC event at the Howard Theatre in Washington, D.C., today at 12:15 pm ET, where he’s expected to focus on abortion rights.

What’s Happening on Capitol Hill: Recess.