Ep164: Ted Love and Alan Anderson on Raising the Bar for Sickle Cell Disease Patients

Episode Summary

Executive Summary: This episode centers on sickle cell disease as a global, historically neglected condition and on a biotech-led campaign to expand newborn screening and basic treatment in Africa. Ted Love and Alan Anderson discuss advances in U.S. therapies, the power of low-cost diagnostics, the Sickle Forward initiative, and how philanthropy, biotech, and clinical partnerships can save lives now while advancing long-term research.

Main Topics: Sickle cell disease as a global neglected disease (Priority: 5/5): Alan explains sickle cell as the most common inherited red blood cell disorder, concentrated in malaria-endemic regions, with a heavy burden in Africa and major disparities in diagnosis, care, and funding. Scientific and therapeutic progress in the U.S. (Priority: 5/5): Ted and Alan review the shift from hydroxyurea to newer oral small molecules, gene therapy, and gene editing, emphasizing that the field is entering a period of rapid iteration and possible functional cures. Newborn screening and basic interventions in Africa (Priority: 5/5): The conversation highlights a practical care model: diagnose early, then provide antibiotics, malaria prevention, and vaccinations to reduce childhood mortality in low-resource settings. Sickle Forward and the Kilimanjaro fundraising campaign (Priority: 4/5): Luke describes the Timmerman Traverse for Sickle Forward, a biotech-community effort to climb Kilimanjaro and raise funds for screening and treatment programs across Africa. Access, affordability, and implementation (Priority: 4/5): The guests stress that expensive, logistically complex therapies cannot reach most patients globally, so low-cost point-of-care screening and scalable interventions are the most immediate path to impact. Changing perceptions and raising the bar for patients (Priority: 4/5): The discussion closes on shifting expectations for sickle cell patients, from survival-only framing toward long, productive lives with expanding options and more ambitious goals.

Key Arguments: Sickle cell is not only a U.S. disease; it is a global condition with the highest burden in Africa and substantial underdiagnosis. Research and industry investment in sickle cell has historically lagged far behind diseases like cystic fibrosis, but that is beginning to change. Therapies such as voxelotor/Oxbryta and newer agents like GBT601 show that oral small molecules can meaningfully alter disease biology and may eventually become affordable worldwide. Gene therapy and gene editing are transformative but are too expensive and complex to serve the millions of patients in low-resource settings. The fastest way to reduce mortality in Africa is not advanced curative therapy but early newborn screening followed by low-cost preventive care. A one-dollar screening test can enable systematic newborn diagnosis and connect infants to interventions that save lives. Partnership with local clinicians and ministries of health is essential because outside actors cannot sustainably impose change without local leadership. The success of the biotech community fundraising campaign demonstrates that sickle cell can attract investment and enthusiasm when people see both the science and the human need.

Data Points: U.S. sickle cell population: approximately 100,000 - Estimated number of people with sickle cell disease in the United States. Africa sickle cell burden: about 6 million - Estimated number of affected individuals in continental Africa. Funding disparity vs. cystic fibrosis: 1:100 to 1:1000 less investment - Ted cites reported comparisons showing sickle cell has received far less funding than cystic fibrosis across NIH, industry, and charity sources. GBT funding raised: over $1.5 billion - Ted says Global Blood Therapeutics raised substantial capital to develop sickle cell therapies and became a major investor in the field. GBT acquisition value: $5.4 billion - Luke describes Pfizer’s acquisition of Global Blood Therapeutics in 2022. Additional matching gift: $1 million - Ted Love and his wife Joyce pledged an extra million to UAB sickle cell research after the team reached its fundraising goal. Total campaign amount: more than $2 million - Combined total from the Sickle Forward campaign plus the matching research gift. Newborn screening test cost: $1 per test - The point-of-care hemotype SC test is described as a very low-cost diagnostic for newborn screening in Africa. Screening turnaround time: 10 minutes - The point-of-care test can provide results quickly enough to support immediate counseling and follow-up. Hospital screening coverage: 99% of babies born at one rural Mali hospital - Since 2019, the program has screened nearly all newborns delivered at the partner hospital. Infants screened: over 20,000 - Alan presented data on the cumulative number of infants screened at the rural Mali hospital. Mortality reduction from early intervention: 30% reduction - Alan cites U.S. newborn screening plus penicillin and vaccination as having reduced mortality substantially. Childhood survival without diagnosis in Africa: 50% to 80% die before age 5 - Alan notes that many undiagnosed children in continental Africa die from preventable complications. Botwana work period: 2015 to 2017 - Alan lived and worked in Botswana for two years to gain experience in global sickle cell care. Kilmanjaro event dates: September 10 to 17 - The team plans to gather for the climb during National Sickle Cell Awareness Month. Individual fundraising target: $50,000 - Some participants are still working to meet their personal fundraising commitments.

Pivotal Quotes: "the single largest investor in fundamental research for sickle cell disease in the world" — Ted Love: Ted describes Global Blood Therapeutics’ role in advancing sickle cell research and changing the ecosystem. "we are now resetting the bar for success" — Alan Anderson: Alan explains how improved therapies are changing expectations for the life outcomes of patients with sickle cell disease. "every baby is screened for sickle cell disease in the United States" — Alan Anderson: Used to contrast U.S. newborn screening norms with the lack of screening in many African settings.

Implications: The episode argues that near-term impact will come from low-cost screening and basic care, while advanced U.S. therapies continue to expand. For industry and donors, the message is clear: sickle cell is scientifically tractable, globally urgent, and worth sustained investment.