A Pivotal Moment in Medicine: Stem Cell Research

Ryan Custer was a freshman basketball player at Wright State University in Dayton, Ohio, when he cracked his C5 vertebra in a pool at a house party in April 2017. Custer, paralyzed from the chest down, enrolled in a Chicago clinical study for an experimental stem cell treatment that might restore limited nerve function one centimeter at a time – shoulder shrug, bicep motion, tricep usage –

Cheryl Wiers, a mother in her forties, has had her severe non-lymphoma Hodgkin’s recur twice. Chemotherapy had failed, but a clinical study for a stem cell transplant at City of Hope Medical Center in Duarte, California, provided hope.

Andrew Caldwell, an HIV-positive man in San Francisco, underwent an experimental therapy in which his own genetically modified stem cells were transfused back into his body; if the modified cells produced enough HIV-resistant fighter cells, known as T-cells, the treatment could functionally cure HIV.

All three are forerunners in the field of stem cell therapy, which may one day provide relief from illnesses including cancer, Type 1 diabetes, lupus, and other auto-immune disorders. And in the film Ending Disease, a collection of intimate portraits of experimental medicine under the culturally fraught, politically vulnerable, and extremely promising umbrella of stem cell research, all three, along with several others, share their emotional, idiosyncratic, and casually radical stories.

Such research has long been a game of possibilities – treatments that could cure a wide range of incurable diseases and conditions, from HIV to certain causes of blindness to quadriplegia; research whose funding could be slashed by political whims; treatments that could become available to the general public but are only available to a small number of clinical trial participants.

Ending Disease, a documentary that followed many trial participants from 2016 to 2019, draws its title from the greatest extent of that potential: “we are on the verge of a tremendous number of cures,” filmmaker Joe Gantz remarked.

For each of the case studies in Ending Disease, stem cell therapies have incalculably large potential that is unclear and uncertain. It’s a chance for Rosie Barrero, who became legally blind in her 20s due to retinitis pigmentosa, to see a little better and rely less on her family. The resumption of any movement below the neck for Custer.

The chance for Erica Billy’s toddler daughter, Ava, who was born without an immune system, to go to school, leave seclusion, and avoid the terrible procedures – chemotherapy rounds, a bone marrow transplant – that characterized her infancy.

The 138-minute film provides a general overview of stem cells – which are effectively blueprint cells that can transform into any other kind of cell in the body – as well as a high-level explanation of how treatments function on a scientific level. The portraits, on the other hand, are hyper-specific, ordinary, and emotional, providing a clear insight into the time-sensitive implications of decades of study, which are sometimes compromised by political headwinds.

“It’s not critical for people to understand exactly how stem cells or CAR-T cells work,” Gantz added. Instead, the video emphasizes how “this is such an important moment and to follow these patients, scientists, and doctors,” many of whom have spent decades researching cures for extremely particular disorders.

Ending Disease, like another Gantz film, The Race to Save the World, which focuses on the personal lives of several climate activists rather than macro-level arguments for change, captures “the tremendous emotion that these families are dealing with, hoping for a cure for their loved ones” rather than technical explanations of how stem cell therapies work.

There are appointments, tentative conversations with doctors about scans, needle injections, and families crammed around a hospital bed, a playful fight between Custer and his father over a disappointing college grade, a nurse playing with an immunocompromised Ava in a full bodysuit, Caldwell’s mother recalling his coming out, a conversation between Wiers and her husband, both Catholics, over their initial reluctance and eventual support of therapies involving embryonic steroid therapy.

Between the episodes, specialists like Irving Weissman, the head of Stanford University’s Institute for Stem Cell Biology and Regenerative Medicine, provide some academic perspective. Weismann, a pioneer in cancer stem cell research, attests to the tumultuous cultural battles that have stifled scientific advancement. Many people will be reminded of the arguments in the early 2000s, when George W. Bush’s government openly prohibited the use of fetal tissue, such as embryonic stem cells, in scientific research.

California voters took things into their own hands in 2004 when they approved state financing for the California Institute for Regenerative Medicine’s stem cell research (CIRM).

Weissman added, “Since then, we’ve been able to develop purified blood-forming stem cells to rescue people from high-dose chemotherapy with their own stem cells.” Scientists have demonstrated that such treatment is possible for years, but it takes years of FDA-approved clinical trials and additional years of research, which can be jeopardized by political allegiances to anti-abortion groups opposed to the use of fetal tissue for scientific research, which is sometimes obtained from abortions but is not used in all stem cell therapies.

With a surprising executive order in June 2019, Donald Trump halted government funding for fetal tissue research, appeasing the anti-abortion factions his administration hoped to pacify. The decision, which was purportedly based on the whims of an obscure but tenacious West Wing official called Joe Grogan, stopped HIV drug studies and pushed several laboratories to lay off employees and cancel projects. (Efforts to discover alternative Covid-19 therapies were likewise postponed until March 2020.)

“Rather than having a ban that precludes any of this scientific and medical development,” Weissman said, “it would be great if we could get rid of that political and religious aspect of looking at science and let science at least make its case and be adjudicated by the other bodies.”

The key roadblocks to stem-cell therapeutic innovation and, more importantly, accessibility remain “short-term politics, a lack of understanding of what pharmaceuticals do and can do, and how they must operate” from a commercial perspective. Furthermore, there is the “valley of death” gap between discovery and real treatment, which Weissman describes as “our responsibility to look at, find out what the gap is, and fill the gap so that we can advance medicine for people.”

For the patients in Ending Disease, the chasm is palpable, a sliver of hope. Not all of the studies were successful; the video is dedicated to Steven Sharpling, who died in 2018 after stem cell injections failed to completely eliminate anaplastic astrocytoma, a kind of brain cancer. However, even little victories have a significant impact on daily life.

Custer was able to use his arms again within months, and he even passed a driving test in a customized automobile. Wiers’ therapy, which retrained her own white blood cells to locate and kill cancer cells, saved her life; she is now cancer-free. Caldwell’s T-cell counts had improved to the point that he could stop using anti-viral HIV drugs.

Ava George’s stem cell treatment retrained her immune system enough for her to attend school with other children. These are future possibilities, not silver bullets, but avenues out of sheer incurability — if we choose to support them.

Thanks to Adrian Horton at The Guardian whose reporting provided the original basis for this story.