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🔢 Key Takeaways

  1. Cancer is not just one disease, but hundreds or thousands, each with unique treatment needs. Basic science has been crucial, but more progress is needed to combat the leading cause of death. Delays from Covid-19 are exacerbating the problem.
  2. The pandemic has caused delays in cancer treatment, but it has also brought about positive changes like telehealth and faster funding development. The National Cancer Institute is dedicated to identifying promising research and prioritizing funding for the best science.
  3. Cancer death rates are down by 30% in the last 30 years in the US, with a record 2.4% decline in 2018. New immuno-oncology drugs offer hope for cancer treatment.
  4. Lung cancer remains a leading cause of death despite advancements in treatment. Tobacco control helps reduce risk, but other factors also contribute. Early detection and a multidisciplinary approach are key in fighting this complex disease.
  5. Obesity can cause chronic inflammation and hormonal imbalances that increase the risk of certain types of cancer. Pancreatic cancer is a particularly concerning type, with a low survival rate and difficulties in both detection and treatment.
  6. Detecting pancreatic cancer early is difficult due to vague symptoms and physical examination challenges. Surgical resection has a high success rate for small tumors, but few patients are eligible. Currently, no effective therapies or screening methods exist.
  7. Dr. Diane Simeone advocates for collaborative research to overcome the challenges posed by pancreatic cancer. Her Precision Promise initiative aims to bring about a new era in clinical trials for a cure to the disease.
  8. Collecting and analyzing patient data from various sources like medical records, risk factors, and imaging can provide valuable insights for cancer treatment. Big data teams are working towards this to assess potential treatments and improve cancer research.
  9. Precision Promise is an adaptive-platform clinical trial for testing experimental pancreatic cancer treatments. It allows for less redundancy, quicker F.D.A. approval, and the possibility of multiple successful therapies.
  10. To improve treatments for pancreatic cancer, clinical trials must be smaller and more personalized, based on the cancer's mutations and molecular genetics. The FDA must navigate when to trust unrandomized designs while considering costs and public outrage.
  11. The high cost of cancer drugs presents a difficult tradeoff between innovation and patient costs. While new drugs are expensive, the need for generic alternatives and early detection methods is crucial for improving survival rates.
  12. Learning from failed trials can lead to breakthroughs in cancer research and other medical fields. The Covid-19 pandemic has spurred impressive advancements in vaccine development, which may have positive effects on the treatment of other diseases. Advances in clinical trial infrastructure and innovation are key to continuing progress.
  13. Covid-19 spurred unprecedented collaboration in the scientific community, allowing for efficient drug testing with multiple candidates and a shared placebo arm. Moderna's mRNA vaccine success paves the way for future advances in mRNA medicines. Continued collaboration is crucial for success in medicine.

📝 Podcast Notes

The Complexity of Cancer and the Need for Continued Progress

Cancer research has shown that the disease is complex, with hundreds or thousands of diseases that each require unique treatment and understanding. The paradigm shift from seeing cancer as a couple of diseases to a multitude has led to progress against certain types of cancer, but not all, creating unevenness in treatment. Basic science and biology have been crucial to understanding the intricacies of cancer and providing effective treatments. However, with nearly 10 million people dying from cancer annually and the Covid-19 pandemic causing delays in treatment and screenings, more needs to be done to continue progress against this leading cause of death.

The Impact of the Pandemic on Cancer Treatment and Research

Delayed cancer treatments due to the pandemic are expected to be costlier and more advanced by the time they are detected. However, the pandemic has also brought benefits in terms of adapting to telehealth, remote clinical trials, and the possibility of faster funding development for cancer treatment due to the fast development of COVID-19 vaccines. The National Cancer Institute, with bipartisan support and separate funding of $6 billion a year, is committed to identifying and conducting the most promising cancer research. Despite the challenges of fragmentation in medical data, the pace of progress in cancer research is exceptional, with opportunities for faster and effective treatment due to the disruption caused by the pandemic. Ned Sharpless, the Director of the N.C.I., brings a unique perspective with experience as a researcher, entrepreneur, and regulator of drugs and medical devices, focused on prioritizing funding for the best science.

Marked Progress in Fighting Cancer

Despite some areas where less progress is being made, the decline in cancer rates and improvement in survival rates for younger cancer patients is really remarkable. In fact, the death rate from all cancers in the U.S. has declined roughly 30 percent in the past three decades, with a 2.4 percent decline in 2018 alone - the biggest single-year decline ever recorded. Immuno-oncology drugs are also offering new hope for cancer treatment, despite initial skepticism from the medical community. While there is still much work to be done, the progress made so far is truly impressive and gives cause for hope.

The Challenges of Treating and Understanding Lung Cancer

Despite progress made in treating lung cancer with new therapies, it still kills more people in the United States than breast, prostate, and colon cancer combined. Tobacco control has had some success in reducing lung cancer, but behavioral and environmental factors also play a role in the development of the disease. Cancer is not a single disease, but a collection of individual diseases, making it difficult to overcome. Additionally, not all treatments work for all types of cancer and they often have unpleasant side effects. Detecting cancer early is also challenging and we do not fully understand all the causes of cancer. Overall, a multidisciplinary approach is needed to better understand and treat this complex disease.

Obesity is one of the major drivers of cancer and is associated with chronic inflammation which can lead to carcinogenic effects. Alterations to certain hormone levels also contribute to the development of cancer. As obesity becomes a greater problem in the United States, there has been an increase in certain types of cancer including breast, liver, gallbladder, and stomach cancer. Pancreatic cancer is particularly concerning as it has two major problems: early detection is difficult, and treatment is not very successful. Moreover, pancreatic cancer incidence is going up, unlike lung cancer, which is going down. Despite recent advancements in therapies and approaches, pancreatic cancer continues to have a single-digit survival rate of 9 percent.

The Challenge of Detecting and Treating Pancreatic Cancer

Pancreatic cancer is difficult to detect early on due to vague and nonspecific symptoms and the difficulty in physically examining the pancreas. Screening for pancreatic cancer is complicated due to the challenge of balancing overdiagnosis and overtreatment with early detection and effective treatment. Surgical resection is the most effective treatment for pancreatic cancer, with a 60 to 70 percent five-year survival rate for tumors less than one centimeter. However, only 15 percent of patients have a surgically resectable tumor and pancreatic cancer often spreads before detection, making surgery not always an option. Pancreatic cancer is a particularly deadly disease with no effective therapies or screening methods currently available.

Dr. Diane Simeone's Revolutionary Approach to Pancreatic Cancer Research

Dr. Diane Simeone is a renowned pancreatic cancer surgeon and researcher, who aims to create hope for patients by changing the conversation around the disease. She advocates for a collaborative effort in research to overcome the challenges posed by pancreatic cancer, which is hard to detect and treat. One of her major initiatives is Precision Promise, an adaptive-platform trial that aims to bring together researchers, hospitals and patient advocacy groups to share data and collectively work towards finding a cure for pancreatic cancer. The success of this initiative could redefine how clinical trials are done and bring about a new era for pancreatic cancer research.

The Need for Aggregating Siloed Data for Improving Cancer Research

Medical data is fragmented and not easily searchable, leading the National Cancer Institute to create data sets like SEER to aggregate the national cancer story. However, more information is needed to fully understand cancer patients and their treatments, such as medical records and risk factors. Aggregating siloed data can provide valuable insights for cancer treatment and research, especially for uncommon cancers where large randomized trials are difficult. Big data teams are being formed to collect and analyze data from every patient, including imaging, blood-based tests, patient activity data, and artificial intelligence to assess risk factors and potential treatments. Overall, there is a need for more patient data to improve cancer research and treatments.

Precision Promise: A New Approach to Clinical Trials in Pancreatic Cancer Treatment

The Precision Promise platform is an adaptive-platform clinical trial designed to test experimental treatments in pancreatic cancer patients. This platform allows for less redundancy and the ability to swap out failed treatments for newer ones, potentially leading to more successful therapies. The F.D.A. and pharmaceutical companies are involved in the platform, which has the potential to cut the timing of F.D.A. approval in half. The randomized trial has 30% of patients in the control arm and 70% in the experimental arm, allowing for the possibility of multiple therapies that can count towards F.D.A. approval. This new approach to clinical trials may change the course of pancreatic cancer treatment.

How clinical trials need to change to advance pancreatic cancer therapies

In order to de-risk the pharmaceutical industry from investing in new therapies for pancreatic cancer, changes need to be made in the clinical trial process. The cardiology paradigm of large randomized trials is not suitable for cancer research due to the variation in cancers and the cause of each cancer. Instead, smaller, nimble, and sometimes unrandomized trials are needed, which allocate therapy based on the mutations and molecular genetics of the cancer. This poses a challenge for the F.D.A., which is used to seeing large randomized trials. They must decide when to trust these unrandomized designs and when to insist on the gold standard of purity. Equipoise is a key factor in this decision-making process. The high cost of treatments, especially for rare cancers, is still a source of public outrage.

The Complex Dilemma of Drug Pricing and Cancer Treatment

Drug pricing is a complicated issue with no simple solution. Expensive drugs for cancer patients create a tradeoff between stifling innovation versus immense costs to patients. Generic medicines are a potential solution, but it takes time. Making new drugs is very expensive, but having an expensive drug that works is better than having no drug at all. Making the drug cheaper becomes an engineering challenge. Early detection is the holy grail for pancreatic cancer. Small efforts to develop a test need to be expanded. The goal for the field is to have a 50 percent survival rate for pancreatic cancer in 10 years by developing more effective therapies and better testing for susceptibility genes, and putting people in screening programs.

The Importance of Failure in Cancer Research and Breakthroughs

Negative results in cancer research are important and are being more widely shared, allowing for more informed and successful results. Decades of failed trials ultimately led to the development of an effective new therapy for neurofibromatosis. This example emphasizes the need to learn from failures in science, as they often lead to breakthroughs. The Covid-19 pandemic has also spurred impressive advancements in medical research, particularly in vaccine development, which may have positive effects on the treatment of other diseases. Advances in clinical trial infrastructure and innovation are key to continuing progress in cancer research and other medical fields.

Collaboration and Efficiency in Drug Testing during Covid-19

Testing multiple drugs against one placebo arm is an efficient way to learn about potential candidates quickly and give more people access to them. Covid-19 brought the scientific community together in unprecedented collaborations that should continue to foster advances in medicines. Moderna's vaccine success has implications beyond Covid-19, as it proves the ability to scale mRNA technology for manufacturing. This success also translates to increased confidence and success for other mRNA medicines. The pandemic has highlighted the need for continued collaboration amongst companies and government agencies, leading to a positive change in relationships and interactions moving forward.