Leading Voices on Protein Misfolding Diseases

Many proteinopathies develop silently for years before symptoms appear. Identifying them early can make a big difference, giving patients a better chance to slow or stop disease progression. However, early detection is only part of the solution. Treatments also need to be practical, safe enough for long-term use, and affordable so they are within reach for everyone who needs them.

Dr. Birgit Strodel, who combines lab experiments and computational modeling to study amyloid diseases, explained the importance of finding these problems before major damage occurs.

“The most difficult part is that the disease starts 10 to 20 years before it’s diagnosed. We need better diagnostics so people in their 50s and 60s know they are at risk.”

She also highlighted that treatments must work in real life, not just in theory.

“Treatments must be affordable and have few side effects as patients will be taking them for many years.”

Dr. Strodel believes that scientists working together and sharing ideas is the most effective way to speed up progress.

“There are many great minds in the field, and we can move forward a lot faster if we work together.”

The Role of Curiosity and New Ideas

Science moves forward when researchers are curious and willing to explore unexpected directions. Staying engaged and asking new questions can lead to discoveries that might not come from simply following established paths. Creating new methods and tools can also help answer questions that once seemed impossible.

Dr. Bernd Reif, an expert in protein structure and NMR spectroscopy, believes that real progress begins with genuine interest in the work.

“Staying productive begins with being interested in your direction of research.”

Dr. Reif emphasizes that even small, incremental findings can shape the future of the field. These “little breakthroughs” might not make headlines but often enable larger advances later. Moreover, he points out the competitive advantage of developing and refining your own tools:

“New discoveries happen when you have new equipment, but if you can develop the equipment yourself, you’re ahead of others.”

The Value of Open Thinking and Sharing Ideas

Proteinopathies are complex and often overlap with other diseases. To fully understand them and create better treatments, researchers and clinicians need to share information and learn from each other. Keeping an open mind and avoiding rigid thinking is essential.

Dr. Rakez Kayed, who studies toxic protein clumps in neurodegenerative diseases, spoke about the importance of ongoing communication.

“Cross talk is key for both clinicians and researchers. If a disease overlaps with other diseases, commonly, the diagnosis is not changed.”

He believes that introducing protein chemistry earlier in education could help bring more young scientists into this field.

“Protein chemistry is not a taught topic. If it is introduced at the undergrad level, it may widen the interest in this field.”

Dr. Kayed also encourages scientists to trust solid data and their own instincts instead of relying only on simplified textbook explanations.

“Don’t listen for the oversimplifications in textbooks; go with the data and your gut instincts. There will always be new things; you must be open-minded.”

New Treatments and Global Collaboration

New therapies, including antibody-based approaches, are giving patients and researchers new hope. At the same time, scientists worldwide are working together more than ever, sharing results and learning from each other. This kind of teamwork helps move ideas forward faster and leads to new solutions that might not be possible working alone.

Dr. Ehud Gazit, a leader in amyloid research and treatment development, discussed how encouraging it is to see progress with antibody therapies.

“Antibodies are already in the market to target amyloid diseases; these have proved that the amyloid hypothesis is valid by being able to affect the disease by amyloid treatment.”

He also shared his view on the power of international scientific cooperation.

“Despite the world’s struggles, researchers are still working together to advance science. Bringing people together from different backgrounds is fruitful for scientific activity and discussion.”

Inspiring the Next Generation

Introducing students to research early helps them see what science really looks like and sparks curiosity that can last a lifetime. Giving young people a chance to work in labs and ask their own questions helps them see themselves as part of the scientific community.

Dr. Thomas L. Rothstein, known for his work in immunology and B-cell biology, spoke about the importance of hands-on experience for students.

“Promoting undergraduate students to learn and gain lab experiences may encourage students to become interested in research.”

Looking to the future, he shared a realistic but hopeful outlook for new treatments. While a 10 to 15 year timeline is common, he pointed out that major breakthroughs can sometimes come faster than expected. The rapid development of the COVID-19 vaccine is an example of what is possible when there is focus and collaboration.

“For a 10-year timeline from now, a treatment would have to be in the lab being developed and tested now. 15 years away would be more realistic, however, watching the COVID-19 vaccine be developed so quickly gives hope for a faster treatment development process.”

Embracing Science as a Shared Opportunity

Science should feel open to everyone. Showing people that it is something they can be part of, regardless of background, helps bring in fresh ideas and different ways of thinking. Accepting that mistakes and setbacks are part of the journey makes for stronger scientists and better discoveries.

Dr. Oleta Johnson, a researcher focused on chemical biology who uses chemical and biophysical tools to study and adjust molecular chaperone proteins in protein misfolding diseases, spoke about making science more accessible.

“Getting people excited about science and making science more available allows people to understand that it is an opportunity that is also available to them.”

She also reminded young researchers not to fear mistakes or failure because these experiences are a natural part of learning.