On May 23rd, Friends of Cancer Research hosted a public webinar, “Friends’ Cell Therapies Webinar for Advocates,” spotlighting the pivotal role of patient advocacy and cell and gene therapies in cancer care. Dr. Alex Marson from Gladstone Institutes presented CRISPR’s potential in cancer immunotherapy, while Chris White, author and patient advocate, shared his journey with metastatic melanoma and the success of a tumor-infiltrating lymphocytes (TIL) therapy trial, emphasizing the impact of cell therapies in remission. The webinar bridged the gap between research and tangible patient outcomes by facilitating an open dialogue and an engaging Q&A session between the live audience and panelists.
Presentation by Dr. Alex Marson, Gladstone Institutes
(Click here to watch the presentation)
During his presentation, Dr. Alex Marson detailed his research using CRISPR technology to genetically engineer T cells by precisely editing their DNA to introduce new instructions, transforming them into effective cancer-fighting agents. He highlighted the success of CAR T-cell therapy in treating leukemia and the development of CRISPR as a tool for genetic editing. Dr. Marson discussed his lab’s approach to large-scale testing of gene modifications to enhance T cell efficacy, aiming to develop safer and more effective immunotherapies, and demonstrated the progression of these therapies from the lab to clinical trials.
Chris White’s Cell Therapies Experience and Advocacy Story
(Click here to watch Chris’s Story)
Q&A Session
(Click here to watch the Q&A session)
Why have we seen cell and gene therapies largely only for the treatment of blood cancers and what is being done to expand them into treatment of more solid tumors?
Alex Marson highlighted two challenges to developing cell therapies for solid tumors:
- While there are good targets for cell therapies to treat blood cancers there are fewer known targets for solid tumors. He noted there is a lot of work being done to identify additional targets that would enable the treatment of solid tumors.
- Second, he explained how solid tumors can evolve to “fight back” against T cells. So, even if a good target exists to help the T cells identify the solid tumor, the tumor may suppress the T cells ability to attack it. He explained how gene editing approaches are being used to address this issue.
What are some of the things being done to improve cell therapies?
Chris shared some of the advancements being made to improve cell therapies:
- Companies are exploring alternative approaches to activating cell therapies after they are infused.
- To create a cell therapy, developers must take a patient’s cells and “grow” them. Chris noted there are efforts to improve blood and tissue donations to be used as feeder cells to aid in this process.
- There are also things being explored to improve the safety of these therapies.
Are CAR T-cell therapies always patient-specific or is there a possibility to create generic/ “off-the-shelf” versions that can be used more universally?
Alex Marson noted there are a lot of efforts to make “off-the-shelf” cell therapies. He highlighted how the process of manufacturing patient-specific T cells can be time-consuming and expensive. He emphasized the need to make these treatments more accessible by simplifying the manufacturing process and potentially creating off-the-shelf versions.
What kinds of things are being done to expand access to cell therapies?
Chris shared several things he views as critical to expanding access to and knowledge around cell therapies:
- Advocates can help improve access to cell therapies by sharing their experiences and knowledge on various social media platforms. Different demographics use different social media channels, so it’s essential to reach out to various communities.
- Given the risks associated with administering cell therapies and requirements for storing them, there are only 30-40 places that are approved cell therapy centers. Chris noted there are efforts to expand the number of centers that can administer these therapies. He emphasized the importance of establishing cell therapy centers in closer proximity to patients.
Chris also shared his experience navigating barriers to accessing the cell therapy trial he participated in. He explained how much travel and time was required to participate. He also noted how the trial adapted when COVID began. For example, use of decentralized elements such as getting scans at local hospitals and using telehealth to review results with trial physicians and investigators. Chris emphasized the importance of collaborative partnerships between oncologists, patients, and other stakeholder organizations to make the system more accessible and equitable. He also noted the need for resources to help patients identify trial opportunities.
Given the risk and novelty of these therapies, what are some considerations for ensuring research is being conducted in an ethical way?
Dr. Marson noted a critical element of ethics in cell and gene therapies is ensuring patients with the greatest need can access these treatments. For example, gene therapies have curative potential for treating sickle-cell disease. Dr. Marson noted the need to ensure any patient with sickle-cell disease can benefit from this therapy. He also emphasized the need to keep a patient-centered focus when weighing risks and benefits and noted it is critical to involve patients in these conversations.
Chris noted that costs for patients, financial and otherwise, must be considered. He explained that many patients are willing to do anything to live and may face significant financial burdens for treatments, which could cost upwards of half a million dollars. He also noted the sooner patients can undergo cell therapy, the more beneficial it may be, as their bodies are healthier and better equipped to handle potential side effects. He explained how critical it is to ensure consent forms clearly communicate risks, known and unknown, including possible side effects and follow-up treatments that may be needed to address side effects.
Click here to watch the full meeting.
