被治愈的镰状细胞病患者Christelle Salomon在临床试验中建立信任

By Emily Moskal

In her first year as a newborn, Christelle Salomon was diagnosed with sickle cell disease. The fact that, as a baby, Salomon would only stop crying from pain at the peak of a swing on the swing set tipped off one of her six siblings to the debilitating illness that Salomon would live with — until being functionally cured of the disease at 28 years old.  

Inconsolable crying is one of the first obvious yet general symptoms of sickle cell disease. The disease, which affects about 100,000 people in the U.S., is punctuated by an array of symptoms, including pain crises which often result in emergency room visits multiple times per year. This is because the red blood cells are shaped like a sickle, elongated and curved, unlike the round, disc-shaped red blood cells found in individuals without sickle cell disease. The sickle cells can’t travel efficiently through vessels and instead cluster, blocking the flow of oxygen throughout the body.

Now, almost five years after a bone marrow transplant that cured her sickle cell disease — knowing what it’s like to be a patient and a scientist — Salomon bridges communication gaps between patients, providers, and scientists. She ultimately improves trust within the sickle cell community, particularly regarding cell and gene therapy clinical trials at UC San Francisco’s Sickle Cell Center of Excellence.

“I’ve come full circle,” Salomon said. “It’s a perfect blend of having had clinical training and exposure to the disease to be able to break down complex information — once for myself, now for others.” 

A balanced view of treatment

Salomon attributes her current work supporting sickle cell clinical trial recruitment and other work as a health policy and education analyst for UCSF’s Sickle Cell Center of Excellence to her early positive exposure to the medical profession in addition to the transplant. 

“When I was young, my providers were good at giving me the pathophysiology lessons for children and telling me why I feel the way that I feel,” Salomon said. “I felt empowered by them and now want to do that for other sickle cell warriors.” 

To enter the medical field, she got a bachelor’s degree in biology from Tufts University near Boston and then worked as a clinical scribe and clinical research coordinator, very patient-facing jobs that taught her how much she loved working with patients.

Hard decisions

In 2019, she started medical school at the Western University of Health Sciences near Los Angeles, after a one-year, fast-tracked master’s program in medical sciences at the same university. Shortly after starting medical school, she realized that her health was severely holding her back from her dreams.

“At the time, being a doctor was not something I could see myself sustaining long term,” Salomon said. “How am I going to take care of my patients when I can’t do a physical exam without causing myself pain?” 

She decided to take a medical leave of absence and tried multiple disease-modifying therapies available at the time. She maxed out on the dose of hydroxyurea and had bad side effects with L-glutamine. At this point, she was at her most desperate, seeking alternate treatments. She tried three of the four FDA-approved therapies for sickle cell disease at the time. One wasn’t recommended for her type of sickle cell disease. Of the ones she tried, none seemed to benefit her and sometimes worsened her pain.

Salomon eventually decided on a bone marrow transplant at the age of 28 — after writing four pages of pros and cons — under the care of Mark Walters, MD, at UC San Francisco. During the transplant, her unhealthy bone marrow cells were destroyed and replaced by an infusion of her brother’s blood-forming stem cells. Now, she’s living a new normal. 

“I never imagined a life with no pain,” said Salomon. 

Communicating plainly 

The transformation left her wanting to give back in a way that enhanced the doctor-patient relationship. She started volunteering with the transplant team that cared for her. She served on the UCSF’s Sickle Cell Center of Excellence community advisory board and created graphics explaining the disease for social media. After a stint working at two lab jobs eight months post- transplant, she then started her current consulting role as a health policy and education analyst at the center.

Her work involves creating educational content that provides balanced information about the potential risks and benefits of cell and gene therapy. Her goal is to help patients make informed decisions, Salomon said.

“Providers, sickle cell patients and their families, scientists, we all want the same thing, but sometimes we’re speaking different languages,” Salomon said. “My goal is to translate across those personal and professional borders.”  

Often her task is to address public misinformation or hype around sickle cell treatment. For example, viral vectors used to deliver therapies won’t transmit diseases. What about the doctor who recommended a gene or cell therapy in haste without providing information on the potential risks? There’s more to it than that, Salomon said.

“Unexpected and serious complications, identity crisis, and navigating the financial process are all challenging aspects of the transplant process that warriors may not know about when considering cell and gene therapy,” said Salomon. “My goal is to empower patients with the information they need to make informed decisions by bridging the gap between expert providers and warriors and their families.”

Understanding treatment options

She does this by explaining what happens at the cellular level and how that translates into real-life implications, such as isolating for 100 days after a transplant when the immune system is most vulnerable.

One of the two FDA-approved gene therapies for sickle cell disease, Lyfgenia, adds a gene for modified anti-sickling hemoglobin—the protein in red blood cells that carries oxygen from the lungs to the rest of the body—providing relief for the patient. The treatment uses a viral vector to deliver the gene addition and, Salomon said, there remain many misconceptions around this.

People also often need more information to know which treatment is best for them. Although several treatments are now available, some are still in clinical trials, and not everyone with sickle cell disease will qualify. Or, even if it’s not a straight disqualification, factors can affect treatment success. For example, transplants involving a donor and recipient tend to be riskier as recipients and donors get older. That was a key determining factor in Salomon choosing to pursue a transplant when she did.

The future is patients 

The research landscape is evolving, and treatments are coming on and off the market, Salomon said during a CIRM Trainee Networking Conference in July. But there remains a hesitation by many patients living with sickle cell to join clinical trials.

Many people remain distrustful of new treatments from doctors that may have dismissed them previously. According to Salomon, trust remains an “x factor” of sickle cell disease treatment. There are risks such as infertility, infection, hair loss, donor rejection, and death, but the treatments have the potential to be lifesaving, according to Salomon.  

Rather than have a balanced view of the disease’s effects and its treatment, providers may be uninformed, underinformed, or misinformed about the disease, she said. That’s partly because medical school doesn’t address it the way it does more common conditions, Salomon said.

Winning back trust

The disease disproportionately affects Black people (1 in 365). Also, some doctors have biases, including rejecting opioid pain medicine because they assume their Black patients are “drug seekers,” Salomon said. Sickle cell patients simply want to alleviate excruciating pain that comes with their tissues not receiving oxygen.

“Mistrust that sickle cell warriors have in the system is earned,” said Salomon. “Time and time again we have been let down. So, don’t be surprised if someone doesn’t want to hear about new treatment options.” 

Salomon said this is a chance to understand patients’ hesitations, fears, and frustrations. Rebuilding trust is one of the first steps to helping patients access clinical trials. Without trust, clinical science cannot move forward.

Providers can start by genuinely caring about patients’ well‑being and doing what matters most when it matters most. Even if patients are not actively dying, warriors may miss meaningful parts of life because of their condition. Support them however you can, Salmon said. That includes researching a clinical trial or simply learning what they miss, Salomon said. When patients and providers work together, discovery begins.

“Patients are the drivers of innovation. Without a patient, there is no problem,” said Salomon during the talk. “And without a problem, there is nothing to solve or innovate.”

CIRM’s Support of Sickle Cell Research

To date, CIRM has invested over $65 million in sickle cell research. That includes investments in basic discovery, clinical trials, and everything in between.

As of June, CIRM is supporting two clinical trials recruiting patients in California. Both trials use the patients’ own bone marrow stem cells and are co-funded by the National Heart, Lung, and Blood Institute (NHLBI), a division of the National Institutes of Health (NIH). 

One of the two projects is a Phase 1 clinical trial led by Dr. Mark Walters at UCSF. That clinical trial uses the gene-editing tool CRISPR to directly correct the beta‑globin mutation that causes sickle cell disease.

Related Posts

• A patient advocate’s take on sickle cell disease

• An open leyter to CIRM on World Sickle Cell Day

• Progress on sickle cell disease

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