A recent discussion between Dr Crystal Mackall and Dr Lynn Zydowsky explored the research, challenges, and future of cancer gene therapies. Dr Mackall is a doctor and renowned cancer researcher who specialises in childhood oncology and haematology, and is the founding director of the Stanford Center for Cancer Cell Therapy; she was asked questions by the audience and Dr Zydowsky, the president of the Alexandria Summit. A video of the full conversation (about one hour long), released by the Alliance for Cancer Gene Therapy Foundation, can be found here, on YouTube.
The Need for Advances in Paediatric Cancer
In the video (at 20:42), Dr Mackall talks about the impact of paediatric cancer and the need for better treatments. One of the challenges the field faces is the ‘rare disease aspect’ – children’s cancer makes up a small percentage of total cancer cases. However, she says, if you consider the years of life lost, “It ranks about 8th.” Despite this, the advances in treatments for childhood cancer have been limited compared to those for other cancers. Dr Mackall said that “It’s crazy when you consider all the technological advances” that it’s still common to use treatments from the early 80s (around the time the first Mac computer came out), and new developments in this area are desperately needed.
The Importance of Non-Profit Funding
Around twenty-seven minutes into the talk, Dr Mackall talks about the “absolutely essential” role of non-profit funding in pushing scientific discoveries forward in paediatric cancer research. She says that academic institutions and the NIH companies often fund the initial lab discoveries, and drugs closer to being approved are likely to be backed by big pharma. However, when it comes to the research in-between that helps make a drug work better, “this is where the non-profit sector really comes in.”
On Designing Clinical Trials
Once funding has been secured, the key to designing a good clinical trial, according to Dr Mackall, is writing them so that the patients who would benefit from taking part the most are eligible. She also says that collaboration between researchers and patients is crucial to developing better drugs. Drugs that are developed using models in a lab often aren’t as effective when given to human patients, and the process of working out why some patients respond to an investigational drug while others don’t, and then going back to the lab and building on those findings, is how researchers are often able to make more effective drugs. For example, Dr Mackall and her team have gone through this lab-patient-lab cycle several times in the research process for a CAR T cell cancer therapy.
The Future of Cancer Treatments
While Dr Mackall emphasised that it’s not possible to know where research and treatments will go in the future, she did share some of her speculations with the audience. She said that CAR T cells, a research area that she is currently working on, may become more frequently used as a treatment in combination with existing methods, such as chemotherapy and surgery. In addition, the field may see more approvals for checkpoint inhibition and combination therapies for highly mutated cancers, such as melanomas, lung, and oesophageal cancers.
To find out more about the great work Dr Mackall is doing, and hear her answer questions, you can follow the link at the top of the article to view her talk in full.