Academy eBriefings

eBriefings are online multimedia reports documenting recent Academy events. Additional reports may be found on the Academy website at: nyas.org/ebriefing.

Protein Folding in Human Health: 2019 Dr. Paul Janssen Award Symposium

Mammalian cells can make up to 20,000 different proteins, responsible for a wide range of cellular functions, including structure, catalysis, transport, and signaling. Proteins are synthesized as linear chains, but to carry out their myriad roles, they must then fold into complex three-dimensional configurations. Franz-Ulrich Hartl, M.D., of the Max Planck Institute of Biochemistry and Arthur Horwich, M.D., of Yale School of Medicine and Howard Hughes Medical Institute, are working to better understand the molecular machinery that drives protein folding, and the implications when a protein misfolds. They discovered a new class of proteins, part of the chaperone family, responsible for protein folding. Chaperones bind to peptide chains as they are being transcribed to prevent them from aggregating and to give them an isolated space, shielded from the hubbub of the crowded cytoplasm, in which to fold properly. Misfolded proteins are associated with aging and diseases including Alzheimer’s disease, Parkinson’s disease, Huntington’s disease, and prion disease.

On October 4, 2019, prominent scientists gathered at the New York Academy of Sciences to grant the 2019 Dr. Paul Janssen Award to Hartl and Horwich for their groundbreaking insights into chaperone-mediated protein folding.

Symposium Highlights

• While studying mitochondrial protein import, Horwich and Hartl hypothesized that the process may not be spontaneous but dependent on cellular machinery. They discovered a new class of proteins responsible for protein folding.
• Hsp60, its bacterial homolog GroEL, and its eukaryotic homolog TRiC have a double ring structure that forms a chamber in which a peptide substrate can fold into its proper shape.
• The unfolded protein response of the endoplasmic reticulum responds to the presence of misfolded proteins, which accrue with age. The response itself declines with age.
• Hsp70 is a diverse family of monomeric chaperones that binds to polypeptide chains as they’re being translated or when they misfold from mutation or stress and prevents them from collapsing into aggregates.
• Clinically relevant receptors that have been difficult to treat require specific chaperones that may provide more easily druggable targets for neurological and psychiatric disorders.

Full eBriefing by Diana Gitig

Solving Malnutrition Through Food Systems and Nutrition Programs

Malnutrition, in all its forms, is a global challenge. As a key modifiable risk factor for chronic and infectious diseases, it has far-reaching health, economic, and social consequences. On September 13, 2018, the New York Academy of Sciences hosted a discussion of two contrasting, yet not mutually exclusive, strategies to meet the global population’s nutritional needs. Barbara Burlingame, Ph.D., of Massey University, New Zealand, and Kathryn Dewey, Ph.D., of the University of California Davis, presented their views on food systems and nutrition programs.

Highlights:

• According to Dewey, filling nutrient gaps with supplementation, fortification, bio-fortification, and fortified products may be an appropriate complement to improve food systems as a whole.
• Burlingame argued that food systems can adequately support healthy diets for the population through sustainable
• diets and food biodiversity already present in the ecosystem.
• Both Dewey and Burlingame agree on the importance of finding mutually agreeable strategies and exploring areas of complementarity using multisectoral involvement.

Full eBriefing by Saima Ahmed

The Future of Complex Medicines: Development and Regulation

Advances in biomedical research, biotechnology, and nanotechnology are yielding innovative medicines that can change the course of a disease in ways never before possible. These life-saving new medicines — biologic compounds such as gene therapies and recombinant proteins, as well as non-biological complex drugs such as liposomes and iron-carbohydrate complexes — are so diverse, and often difficult to characterize, that regulatory agencies around the world have struggled to figure out what kinds of data should be submitted to approve them.

To advance research and build consensus, it is necessary to engage together key stakeholders from academia, regulatory bodies, industry, and drug manufacturing. On May 13, 2019, the New York Academy of Sciences, the Nanotechnology Characterization Laboratory, and the Non Biological Complex Drugs Working Group hosted a conference to stimulate this discussion. A follow-up to a 2016 convening on the same topic, this event featured presentations on best scientific approaches for the development and regulation of complex medicines, current challenges in the assessment of equivalence, and methods to improve timely patient access for new medicines.

Symposium Highlights:

• There is no standard definition of what constitutes a complex medicine — it could be a product containing a complex active ingredient or formulation, or even a simple medicine enabled to access a complex environment in the body.
• Regulatory agencies around the world are still in the
• process of developing the framework for approving complex medicines. That process is especially problematic for follow-on medicines, and it will have to evolve as increasingly different types of complex medicines emerge.
• Real-world data generated when people use the healthcare system can boost regulatory efficiency for complex medicines.
• The approval of biosimilars would significantly decrease healthcare costs and make the most powerful medicines more accessible.
• As complex medicines drive up the cost of healthcare, governments and regulators will also have to confront inequality in access to medicines.

Full eBriefing by Alla Katsnelson

The New Transformers: Innovators in Regenerative Medicine

The human body regenerates itself constantly, replacing old, worn-out cells with a continuous supply of new ones in almost all tissues. The secret to this perpetual renewal is a small but persistent supply of stem cells, which multiply to replace themselves and also generate progeny that can differentiate into more specialized cell types. For decades, scientists have tried to isolate and modify stem cells to treat disease, but in recent years the field has accelerated dramatically.

A major breakthrough came in the early 21st century, when researchers in Japan figured out how to reverse the differentiation process, allowing them to derive induced pluripotent stem (iPS) cells from fully differentiated cells. Since then, iPS cells have become a cornerstone of regenerative medicine. Researchers can isolate cells from a patient, produce iPS cells, genetically modify them to repair any defects, then induce the cells to form the tissue the patient needs regenerated.

On April 26, 2019, the New York Academy of Sciences and Takeda Pharmaceuticals hosted the Frontiers in Regenerative Medicine Symposium to celebrate 2019 Innovators in Science Award winners and highlight the work of researchers pioneering techniques in regenerative medicine. Presentations and an interactive panel session covered exciting basic research findings and impressive clinical successes, revealing the immense potential of this rapidly developing field.

Symposium Highlights:

• New cell lines should reduce the time and cost of developing stem cell-derived therapies.
• The body’s microbiome primes stem cells to respond to infections.
• iPS cell-derived therapies have already treated a deadly genetic skin disease and age-related macular degeneration.
• Polyvinyl alcohol is a superior substitute for albumin in stem cell culture media.
• A newly isolated type of stem cell reveals the stepwise process driving early embryo organization.

Full eBriefing by Alan Dove

Why STEM Professionals are Valuable Across Industries

According to the Pew Research Center, employment in science, technology, math and engineering (STEM) occupations has grown 79% since 1990. But STEM expertise is vital to many

industries, some of which you’d never associate with those skills. As companies large and small work to find and retain top talent who can identify emerging trends to propel their businesses forward, they are actively investing in professionals with STEM skills.

In a new webinar series, A Recruiter’s Perspective, the Academy’s Science Alliance talks to companies about how and why they’re recruiting talented STEM professionals. On September 26, 2019, PepsiCo, the first partner in the series, shared their creative approach to recruiting top talent in data science and research and development. Learn more from the leading global food and beverage company in this summary.

Webinar Highlights

• A STEM-proficient workforce helps PepsiCo identify emerging scientific trends and advance innovative efforts.
• Their educational programs encourage employees to sharpen their skills and develop new ones across disciplines.
• Applicants should convey their hard and soft skills to PepsiCo recruiters, as communication, flexibility, and intellectual curiosity are critical.

Full eBriefing by Lauren Savage