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Thanks, Science!

The closing plenary session of the 2021 PDA/FDA Joint Regulatory Conference, “Thanks Science,” examined the strides science and technology have made to improve public health, even though the public has seemingly become less trusting of scientists.

In his presentation, “Vaccine Diplomacy in a Time of Anti-Science,” Peter Hotez, MD, PhD, shared his long history of advocating the benefits of vaccinations, particularly for children. As the Dean of the National School of Tropical Medicine at Baylor College of Medicine, Hotez has mostly been involved with the development of vaccines for parasitic diseases throughout his career and has been working with coronaviruses over the last 10 years. He is also head of the Texas Children’s Center for Vaccine Development. These groups have made enormous strides in vaccinating the world’s children and reducing the number of deaths of children under the age of five, with the help of the U.S. FDA, the Gavi Alliance and other global health organizations.

Hotez points to many drivers behind the formation of new hot zones of preventable disease globally, including political instability, climate change, urbanization, deforestation, and the very aggressive rise in anti-science.

Hotez appealed for more international help to effect technology transfer of COVID vaccines to lower middle-income countries. “Vaccine Diplomacy” is an effort to counter these drivers by gaining international cooperation to develop vaccines and expanding access to vaccinations globally. Whether it be new vaccine technology or traditional, the focus is on meeting the needs of individual countries.

For over two decades, Hotez has used good science to improve communications about the benefits and safety of MMR vaccines to “combat” misinformation, particularly the unfounded belief they cause autism.

Each time antivaccine voices have “moved the goal posts,” the scientific community has responded with evidence against it. Today, Hotez sees the politicization of antivaccination viewpoints with calls for “health freedom,” which is causing new outbreaks of measles and propagating the anti-COVID-19 vax and mask movement.

Gene Therapy Treats Rare Spinal Disease

In recent years, gene therapy has become more of a reality, according to Peter W. Marks, MD, PhD, Director of CBER, U.S. FDA, during his presentation “Bringing Bespoke Therapeutics into Being.”

Marks shared the remarkable results of the Onasemnogene clinical trial, a drug that treats patients with infantile onset spinal muscular atrophy (SMA). While most SMA patients die before the age of two, 14 of the 15 trial patients are now in the normal range. To emphasize the power of this therapy, Marks showed an image of a three-year-old patient, Evelyn, running around and playing; “Something never seen in untreated children,” he commented.

Gene therapy, Marks said, could be used to address hundreds of other disorders that affect thousands of people, and edited molecular defects could possibly relegate common diseases to the level of very rare diseases.

Personalized medicine is transitioning to individualized medicines, and the FDA is very interested in facilitating created products as a way to see more gene therapies come about.

To help distinguish among the various kinds of gene therapies, Marks compared them to men’s suits: personalized meds are “ready-to-wear/off the rack,” customized therapies are “made-to-measure that can be fitted,” and bespoke therapies are customized suits created for the individual.

In a hypothetical example, he theorized about how the challenges of creating individualized medicine might be overcome in the future:

  • Manufacturing—An automated device that could perform large parts of processing to surmount the costs of producing only a few doses of medicines for rare disorders
  • Nonclinical Development—Use of such new model systems as organoids and humanized mice to facilitate safety testing
  • Clinical Development—Creating templates for collecting baseline and historical data and Bayesian clinical trial designs to help determine efficacy in small populations
  • Product Access—Public-private partnerships could enable product access in diverse populations and create a system where manufacturers could envision this working (see Figure 1)
Bespoke Gene Therapy Consortium chart. Showing the steps of the gene therapy manufacturing process under the umbrella of a non-profit organization. Figure 1 Concept of public-private partnership enabling product access to diverse populations

One concept in development FDA is looking into is making a “cookbook” for the development and manufacturing of bespoke therapeutics, so one vector of a product could be changed without changing the entire product. Standardization among academic institutions would make it easier to transfer technologies to commercial manufacturing, he said, and regulatory aspects would need to be developed to leverage nonclinical and manufacturing data from one application to another. In this and other ways, FDA is committed to advancing the development of cell and gene therapies for all populations.

About the Experts

Peter HotezPeter Hotez, MD, PhD, is a professor of pediatrics and molecular virology at Baylor College of Medicine, where he also co-directs the Texas Children’s Center for Vaccine Development as an Endowed Chair in Tropical Pediatrics. Hotez obtained his undergraduate degree from Yale University and his MD and PhD from Weil Cornell Medical College and Rockefeller University. A vaccine scientist who has led the development of vaccines to prevent and treat neglected tropical diseases and coronavirus infections, he has authored four books and more than 550 scientific articles promoting global health, vaccines and immunizations. Hotez is also a vocal proponent countering antivaccine and anti-science movements.

Peter MarksPeter W. Marks, MD, PhD, was named Director of the Center for Biologics Evaluation and Research for the U.S. Food and Drug Administration in January 2016 after serving as the Center’s Deputy Director for four years. The center is responsible for assuring the safety and effectiveness of biological products, including vaccines, allergenic products, blood and blood products, and cellular, tissue, and gene therapies. Marks received his graduate degree in cell and molecular biology and his medical degree at New York University and completed Internal Medicine residency and Hematology/Medical Oncology training at Brigham and Women’s Hospital in Boston. He has worked in academic settings teaching and caring for patients and in industry performing clinical development of hematology and oncology products.

Mary FarbmanMary E. Farbman, PhD, as Executive Director of Global Quality Compliance at Merck & Co., Inc., leads a team of GMP experts responsible for compliance support at Merck’s manufacturing facilities around the globe. She previously worked at FDA CDER’s Office of Compliance authoring and reviewing warning letters and compliance documents, conducting microbiological reviews of therapeutic protein manufacturing processes, and performing pre-license inspections of BLA products. In industry, Farbman has worked in various compliance and auditing roles at both the site and global levels as well as in the R&D arena. Her areas of expertise include biotechnology, sterile products, and analytical techniques.

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