American Society of Gene and Cell Therapy

05/09/22 • 37 min

Advances in gene and cell therapies are enabling researchers, clinicians, families, and regulators to work together in incredible new ways to treat previously untreatable conditions.

Listen to Christina Mayer share her efforts to advance policies that help to realize a future where gene and cell therapies are available to all individuals for all diseases. Christina is currently the Senior Manager of Government Affairs at the American Society of Gene and Cell Therapy in Milwaukee, Wisconsin. She works with federal government agencies and decision-makers to impact key components of gene and cell therapies like NIH research funding, genetic testing and screening, payment policy and patient access to approved therapies. She also contributes to the Society’s work on other policy priorities, such as regulatory oversight and the responsible use of new genetic technologies. Christina has a Master of Public Administration from the University of Nebraska-Omaha.

Listen with us as we imagine a future where the availability and equitable use of gene and cell therapies helps to realize the promise of a healthy future for all.

Podcast Interview Questions with Christina Mayer:

1. You are currently the Senior Manager of Government Affairs at the American Society of Gene and Cell Therapy. Can you tell us about the mission of the American Society of Gene and Cell Therapy? What is your role there?

2. What an exciting and rewarding time to advocate for gene and cell therapies! ASGCT reminds us all that there are literally 1000s of clinical trials for novel therapies and over the next decade there will be about 30 approved therapies for genetic disease, not counting cancer. What can our listeners do to become more aware of these efforts and to become advocates for continued advancement and access to these life-saving and in many cases disease curing therapies?

3. In its 2020 – 2022 strategic plan, ASGCT identified access to genetic testing and screening as one of its core patient access priorities. What efforts have been made by ASGCT to advance access to genetic testing and screening in newborns? Can you describe ways that the current approach to newborn screening in the United States could be improved to enable the use of gene and cell therapies? What are ways that our listeners can get connected to your organization?

4. In Feb 2021, ASGCT provided a public comment to Advisory Committee on Heritable Diseases in Newborns and Children (ACHDNC) on the newborn screening process.In this letter, ASGCT stated its support of the Newborn Screening Saves Lives Act. Can you tell us listeners more about the history of this act? Why is it important that it get passed? What would happen if it doesn’t?

5. Also, in the letter, ASGCT offers three recommendations to the ACHDNC. They are: 1) Ensure the RUSP keeps pace with treatment approvals, 2) Collaborate with and rely upon the FDA, and 3) Ensure the process to advance a disorder through the ACHDNC is transparent, predictable,and timely. Would you mind sharing the evidence that supported each of recommendations? (Perhaps, discuss the problem and why this recommendation would solve that problem)

6. ASGCT has worked partner organizations to support other NBS and you are hosting a workgroup and symposium in May to discuss advancements in NBS. Thank you for inviting Dr. Brower and NBSTRN to present during the workshop. Please tell us more about these important events and how they can participate.

7. You and Dr. Brower serve on the planning committee for an effort by EveryLife Foundation to develop actionable policy solutions aimed at ensuring newborn screening continues to advance. This includes the research facilitated by NBSTRN and ASGCT, as well as the policies that you and your team champion. Can you describe why these types of efforts to build coalitions and collaborations across different stakeholder groups are so important?

8. You have a very interesting career path and your work inspires many of us to achieve meaningful change and work to advance discoveries that save and improve lives. Can you share with our listeners what sparked your interest in the revolution that is gene and cell therapy as well as newborn screening research?

9. What does NBS research mean to you?

Learn more about ASGCT annual meeting at https://annualmeeting.asgct.org

To learn more about newborn screening research data tools and resources, visit www.nbstrn.org

Advances in gene and cell therapies are enabling researchers, clinicians, families, and regulators to work together in incredible new ways to treat previously untreatable conditions.

Listen with us as we imagine a future where the availability and equitable use of gene and cell therapies helps to realize the promise of a healthy future for all.

Podcast Interview Questions with Christina Mayer:

9. What does NBS research mean to you?

Learn more about ASGCT annual meeting at https://annualmeeting.asgct.org

To learn more about newborn screening research data tools and resources, visit www.nbstrn.org

Previous Episode

SCID Angels for Life Foundation

Listen as Barb Ballard shares her long history of advocacy and innovative efforts in newborn screening. Barb is currently the Director of SCID Angels for Life Foundation. Her involvement with the non-profit sector began after her son, Ray, born in 1994, was diagnosed at 10 1⁄2 months old with X-Linked Severe Combined Immune Deficiency, commonly known as the Bubble Boy Disease. Barb served on the Board of Trustees for The Immune Deficiency Foundation for 18 years, where she advocated for the interests of parents, families and individuals living with immune deficiency at numerous state, and federal committees, while developing the SCID Initiative Program. While her son was hospitalized as a baby at Duke University, Barb developed the first list-serv for families of children diagnosed with SCID. Be inspired by Barb’s story of perseverance, determination, and dedication in supporting families facing genetic disease.

Interview Questions with Barb Ballard:

1. What is the mission of your organization (perhaps, here you can discuss your grant and scholarship program, community engagement activities, and resources on clinical trials)?

2. Barb had a child with SCID who also touched many lives and his story directly and indirectly led to changes in the way newborns are screened, diagnosed, and treated for immune deficiencies. Can you tell our listeners about his life and your hopes for his legacy?

3. Before Ray was diagnosed with SCID were you aware of newborn screening? What do you think prospective parents should know about newborn screening – not only for SCID but other conditions?

4. Because SCID is a spectrum of different genetic variations (or changes, we used to call them mutations) and needs of patients with SCID vary drastically, how do you engage clinical research partners to understand the needs and concerns of the patient population?

5. What are some of the current efforts of SCID Angels for Life organization in disseminating information on new clinical research?

6. What role does your Facebook or other social channels help to support families whose newborn screens positive for SCID? Are there things that national groups like NIH, CDC and HRSA could be doing to support new families?

7. Your organization also currently manages three scholarship programs to support families: Family Scholarship, Travel Scholarship, and Education Scholarship. How could our listeners learn more about these programs? (perhaps you talk about the application process, deadline, qualifications)

8. You have hosted several Town Halls and other informational webinars on new information or clinical trials where the patients and their families are looking to have their questions answered directly. What are some of the common frequently asked questions?

9. What does NBS research mean to you?

To learn more about SCID Angels for Life, visit http://www.scidangelsforlife.com

If you have a story on how rare disease research has impacted your life and family, please contact NBSTRN to share your story on our podcast. Visit www.nbstrn.org

Next Episode

Congenital Cytomegalovirus (CMV)

Listen as Dr. Megan Pesch shares her journey to becoming a parent advocate and a researcher in Cytomegalovirus (also called CMV). Dr. Pesch is an Assistant Professor of Developmental and Behavioral Pediatrics at the University of Michigan where she is the Director of the Congenital CMV Developmental Follow-up Clinic. Dr. Pesch completed her medical school training, residency, and fellowship at the University of Michigan. She is board certified in Developmental and Behavioral Pediatrics and serves as the President-elect of the National CMV Foundation.

Dr. Pesch’s youngest daughter has a profound bilateral sensorineural hearing loss from congenital CMV and this led to her involvement in national advocacy efforts to ensure that all newborns receive CMV screening. Dr. Pesch’s clinical interests include the early diagnosis and treatment of congenital CMV using a multidisciplinary approach with a focus on family care and support, while her research focuses on healthcare provider practices around congenital CMV diagnosis and management and understanding the relationship between autism and CMV.

The month of June is CMV awareness month. Please visit the National CMV foundation to learn more about advocacy efforts in CMV in your area and how you can support NBS for CMV.

Learn more about Dr. Megan Pesch and her advocacy effort on newborn screening for CMV:

https://ihpi.umich.edu/our-experts/pesch

https://www.nationalcmv.org

Podcast Interview Question with Dr. Megan Pesch.

You are currently an Assistant Professor of Developmental and Behavioral Pediatrics and the Director of the Congenital CMV Developmental Follow-up Clinic at the University of Michigan. Can you tell our listeners more about CMV and how it impacts mothers, their babies and families? (perhaps, talk about the differences between prenatal CMV and neonatal congenital CMV)?
You are a clinician and a researcher, and lead efforts in the Pesch Lab at Michigan Medicine at the University of Michigan! One of your projects brings together a multidisciplinary group of health care providers to refer infants who fail their newborn hearing screen for congenital cytomegalovirus testing. Tell us more about this important project and explain how you got involved in newborn screening research?
Currently, there is no standard of care or routine screening for newborns for congenital cytomegalovirus at birth. What can parents do if they suspect their baby has CMV? (perhaps discuss the Alethia CMV Assay Test System)
What is hearing targeted early cytomegalovirus (HT-CMV) screening?
One of your current studies seeks to understand the possible connection between exposure to CMV during pregnancy and the later risk of autism. Can you tell us more about this effort? What are you hoping to learn? What is the biological pathway?
You are also the President-elect of the National CMV Foundation. What are some of the current activities or programs that people can get involved in your advocacy efforts? What are the recent advocacy efforts to support newborn screening for CMV? Is it currently being reviewed to be added to the RUSP?
Are you involved in training the next generation of pediatricians, and what do you tell them about newborn screening research?
You are busy as a clinician, researcher, advocate, and parent. Do you have any stories of inspiration that keep you going?
What does NBS research mean to you?

To learn how NBSTRN can help your research in newborn screening, visit www.nbstrn.org