Voices From the Frontlines

ASH is reaching out to patients, caregivers, clinicians, scientists, and other stakeholders dealing with blood diseases and disorders to learn about their experiences and shed light on the importance of federal programs that either support scientific research or provide access to health care. Here are their stories.

A Holiday Diagnosis Upends a Young Man’s World

One week before Christmas 2023, Rohan, a resident of Mountain View, California, received a diagnosis that turned his world upside down: acute myeloid leukemia. He had been feeling rundown and fighting an ear infection, so he visited an urgent care center “because, you know, it’s Christmas and the holidays. No one wants to be sick.”

Thirty minutes after he came home, the doctor at the urgent care center called and told him to go to Stanford Hospital immediately. His holidays were spent in the hospital. “I had to pause my career, put my social plans on hold, and learn to navigate a world of transplants, immunosuppression, and survival,” he said.

Following an emergency chemotherapy regimen, Rohan was initially in remission and on track for an allogeneic transplant. But his leukemia relapsed, and his intended donor fell ill. Rohan, who is Indian American, had a unique human leukocyte antigen (HLA) type, making it extremely hard to find another donor.

After consulting with his doctor, Rohan decided to undergo a double cord blood transplant. “I was given a novel combination of therapies pre-transplant, including FLAG-IDA with venetoclax, followed by an aggressive transplant strategy using double cord blood due to my racial background and being unable to find a donor,” Rohan said. “These approaches weren’t standard a decade ago. They represent real progress thanks to research.”

His treatment was not easy and there were complications, including graft-versus-host disease (in which the donor cells attack the recipient’s body), but Rohan is in remission and “regaining strength every day.” Rohan said he has a newfound appreciation for the role of scientific research, including the National Institutes of Health’s funding of basic research in areas such as stem cell transplantation.

“Investing in research is not optional; it’s lifesaving,” he said. “I’m alive today because of advances in stem cell transplants and targeted therapies. Federal programs that support patients, caregivers, and equitable access to care are the backbone of recovery. Every dollar toward science is a vote for survival, for hope, for the future.”

Living With Sickle Cell Disease, Researcher Finds a Lifeline in Medicaid

Ronisha, a researcher and advocate, was diagnosed with sickle cell disease (SCD) at birth through newborn screening. As a child, she depended on Medicaid to get access to the care she needed.

Once she turned 18, Ronisha lost her Medicaid coverage and went uninsured for two years. She did her best to manage her health on her own, but it wasn’t enough, as a severe pain crisis forced her into the emergency department and nearly cost her life. This experience pushed her to re-enroll in Medicaid. For the next five years, with reliable access to medications and specialists, her health stabilized and improved.

“I don’t know if I would still be alive if I hadn’t had access to Medicaid when I needed it.”

Ronisha Edwards-Elliott

Now, Ronisha leads research focused on improving the transition from pediatric to adult care for people with SCD. She helps adolescents understand their disease, the health care system, and how to advocate for themselves. Ronisha’s program was originally supported through funding connected to Medicaid, but with upcoming cuts, her current grant is set to expire at the end of 2025. Reductions to NIH research funding coupled with heightened scrutiny of issues related to health equity further threaten her program’s future.

Medicaid has played an essential role in Ronisha’s life and career as she works to improve the experiences of others living with the disease. Today, more than half of people living with SCD in the U.S. depend on Medicaid or CHIP for the care they need. Cuts to Medicaid not only threaten their health and well-being, but also programs such as Ronisha’s that help them better understand and manage their symptoms.

“Without access to insurance, SCD can not only be costly, but life-threatening,” Ronisha said.

NIH Pulls Funds, Putting Program’s Future in Question

For all of her professional life, Charity Oyedeji, MD, an assistant professor at a leading academic medical center, has dedicated herself to studying sickle cell disease (SCD) and helping individuals who suffer from it. In people with SCD, the red blood cells become rigid and shaped like a sickle, impeding their ability to flow through the body – a condition which brings with it significant pain and a host of life-threatening complications.

Dr. Oyedeji wants to improve the physical agility and quality of life of people with SCD through personalized strategies to manage frailty. This past June, however, the National Institutes of Health (NIH) notified her that a grant supporting her Sickle Cell Disease Functional Assessment Validation and Refinement Study and the SickleFit Exercise Program was being terminated, claiming a “low return on investment” and alleging the study supported racial discrimination. (More than 90% of patients with SCD in the U.S. are non-Hispanic Black or African American.)

“I was in year two of a five-year grant. The funding was terminated, and we’re not even halfway through.”

Charity Oyedeji, MD

“I was in year two of a five-year grant,” Dr. Oyedeji said. “The funding was terminated, and we’re not even halfway through.” An earlier pilot study had helped to shed light on the importance of Dr. Oyedeji’s work.

“We found that people in their 50s actually were functioning more like people in their 70s and 80s,” Dr. Oyedeji said. “We know that physical activity is associated with longevity. People with SCD have a shorter lifespan by about 20 years, compared to Blacks in the general population. We really need a program focused on developing a lifestyle of exercise and building confidence — tailoring the exercise to each person’s impairment.”

The study and program were intended to accomplish two things: create sound assessment tools and develop interventions to reduce impairments and improve physical functionality.

All of that is now up in the air as Dr. Oyedeji searches for alternative sources of funding. Beyond the impact on her own work, Dr. Oyedeji said she worries that NIH cuts could have a chilling effect on researchers, perhaps prompting some to leave the field.

“A lot of researchers are scared, especially those from underrepresented backgrounds,” Dr. Oyedeji said. “Black and brown people are being targeted and they’re the most at risk of dropping out.”

A Pediatric Pioneer Says Research and Science Saved Her Life

One night in 2010 when she was 5, Emily was taking a bath at home when her mother noticed a multitude of bruises on her body — including 21 on her legs alone. That night, she woke up screaming in pain, and her parents took her to the emergency room. In short order, she was diagnosed with acute lymphoblastic leukemia, a type of cancer of the blood and bone marrow, and began chemotherapy.

“After three relapses and 22 months of failed chemotherapy, they told my parents that it was time to take me home for hospice care,” Emily said. But her parents refused to give up, instead taking Emily to the Children’s Hospital of Philadelphia just as a clinical trial for chimeric antigen receptor (CAR) T-cell therapy was to begin. CAR T-cell therapy — developed in part with research funding from the National Institutes of Health — is a type of immunotherapy in which a patient’s T cells are genetically modified to fight cancer cells.

“I became the first child in the world with an immune system trained to eliminate my cancer.”

Emily Whitehead

“I became the first child in the world with an immune system trained to eliminate my cancer,” Emily said. With the dosage for pediatric patients far from perfected, Emily wound up in a 14-day coma because the treatment was so taxing on her body. However, she pulled through.

Today, Emily is considered cured and is a student at the University of Pennsylvania where she studies English, creative writing, and photography. She is also the co-founder of the Emily Whitehead Foundation, which seeks to raise awareness about less toxic treatment options, such as CAR-T, for childhood cancers.

“Without the research, funding, and science that went into making CAR-T cell therapy, I wouldn't be here today,” Emily said. “It is so important to support scientific research and federal health programs to create new treatments like CAR-T, which mean spending less time in the hospital and have little-to-no side effects post-treatment."

Emily also knows firsthand the importance of access to health care and how programs such as Medicaid can make a crucial difference in whether patients get the care they need.

“I have depended on Medicaid throughout my treatment,” she said. “One of the only side effects from CAR-T was immunosuppression, which I continue receiving infusions for and which are covered by Medicaid. The planned cuts to the program mean that I, and millions of others, could lose access to life-saving treatments and medications.”

Sobering Diagnosis Comes Crashing Ashore

For most people, a fractured neck would hardly qualify as a lucky break. For Michael Riotto, it may well have saved his life.

In the summer of 2011, Michael, then 51, was bodysurfing waves with his 13-year-old son on the Jersey Shore when one wave slammed him to the ground. Still sore the next day, he went to the local hospital where he learned he had fractured his neck. The CT scan also revealed something else — that his bones, in the words of the attending physician, were “moth-eaten.” The doctor sent him that same night to a larger hospital in Philadelphia where more tests, scans, and a bone marrow biopsy resulted in a sobering diagnosis: multiple myeloma (MM), an incurable blood cancer that develops in plasma cells in the bone marrow.

“The research that goes into all these different drugs is really, really critical to people’s survival.”

Michael Riotto

Specifically, he was in stage III of the disease, and pamphlets shared with him and his wife that night forecast a life expectancy of no more than five years. “Mind you, I had no symptoms leading up to that fracture on the beach,” Michael recalled, noting he had a demanding career as a manager of a large department store. “No fatigue, no bone pain, no aches in my back, no anemia, none of the typical symptoms.”

Were it not for his fractured neck, Michael’s MM might not have been detected until it advanced even further. With his diagnosis, Michael began chemotherapy. Within a few months, Michael’s hematologist recommended an autologous stem cell transplant, a procedure in which a patient’s healthy stems cells are collected, stored, and infused back into the patient after treatment to replace faulty bone marrow. The National Institutes of Health (NIH) has long been a leader in funding research into stem cell transplantation, a key treatment in fighting blood cancers. MM is not (yet) curable and ongoing research includes looking at how to improve immunotherapies, such as chimeric antigen receptor (CAR) T-cell therapy, and drugs that modify the immune system.

Michael had the procedure a few days after Christmas. After about 24 days in the hospital (due largely to a serious infection), Michael returned home to recover and continue with maintenance treatment, specifically with lenalidomide, a drug that helps the immune system. He continued it until the end of 2019, when his doctor advised a break because Michael had contracted the flu. When tests showed that Michael’s MM remained at bay during his initial break from lenalidomide, his doctor extended the break several more times.

It's been six years since Michael has been on any drug for his MM. He has blood work every quarter, scans at least once a year, and regular visits with his hematologist. Michael still deals with pain and irreversible damage to his bones, but he says he is in the best shape of his life. He is grateful this his stem cell transplant has given him more time with his wife, son, and daughter. His own experience has led him to become a patient advocate, and he is now a regular visitor to Capitol Hill where he speaks about the importance of federal investment in scientific research and support for health care programs.

Michael said he’s alarmed by the cuts at NIH: “The research that goes into all these different drugs is really, really critical to people’s survival.”

Michael said he believes it’s simply a matter of time before his remission ends, and he’s already discussed with his doctor what treatments might be appropriate when that happens. He thinks some form of CAR T-cell therapy would be likely, and he has followed with interest research developments on that front.

Michael said that one of his doctors told him that federal cuts to scientific research may discourage scientists from pursuing their studies.

“He said, ‘People dedicate years, or even their entire lives, to a certain project. The funding may be here now, but will it be here next year?’ As a patient, that’s really scary.”

For a Woman of Faith, Mixed-Match Bone Marrow Transplant Is an Answer to Prayers — and a Result of Research

In September 2017, Debra was busy. A social worker with the state of Delaware, she was running a unit within her department, active in alumni and community organizations, and living a full life with her husband and three adult children. So, it wasn’t too alarming when she started to feel a bit tired. But then the fatigue got extreme: “I’d walk down the hall and feel winded.”

A few days before she was to leave for a conference in Florida, Debra visited a walk-in clinic, thinking she might get a prescription and travel as planned. But after taking blood samples, the doctor told Debra that she was seriously ill — saying the results showed low counts for her blood cells and platelets. An ambulance was called to take her to the hospital immediately.

“The science around transplant itself, the understanding of genetics, all these things and more led to me having the option to receive a transplant, especially when the immunosuppressant therapy route failed. My prayers for recovery were answered.”

Debra

A few days later and after a flurry of additional blood tests, Debra received a diagnosis of severe aplastic anemia, a rare and serious disorder in which the bone marrow does not make enough red blood cells, which transport oxygen through the body, white blood cells, which fight infections, and platelets, which are crucial for clotting. Her medical treatment began with blood transfusions and immunosuppressants, along with the use of eltrombopag, which increases the production of platelets. While there was initial improvement, by early 2019 Debra’s blood levels were showing signs of trouble. A bone marrow biopsy confirmed that she had relapsed. After discussions with her doctor and family, Debra opted for a bone marrow transplant, which involves infusing healthy blood-forming stem cells to replace diseased bone marrow.

Moving forward with a transplant had its own challenges. An unrelated surgery and recovery delayed things, and the search for a donor was daunting. (Debra was adopted as an infant and had no biological siblings that she knew of, and her antibodies against her adult children’s antigens ruled them out as possible donors. Individuals of color have less success in finding non-biological matching donors.)

A mixed-match unrelated donor (a female from Germany) was identified, and in May 2020, Debra had her bone marrow transplant at Johns Hopkins Hospital in Baltimore, where she spent the subsequent three months getting her post-transplant treatment. She went home two days before her birthday in late July, fighting her way through a long recovery marked by nausea, fatigue, and other health challenges.

Debra, who is doing well today, attributes her success with the transplant to prayer and the research that preceded her case. The National Institutes of Health has long been a leader in funding research into bone marrow transplants, including those involving mixed-matched donors.

“The science around transplant itself, the understanding of genetics, all these things and more led to me having the option to receive a transplant, especially when the immunosuppressant therapy route failed,” she said. “My prayers for recovery were answered. I’m a recipient of that science and the work of skilled trained medical professionals. I’m five years post-transplant because people did work years before I arrived.”

After Seeing Years of Progress in Treatments, Patient With Sickle Cell Disease Worries About Future

As a baby, Erica tears and cries of pain alerted her parents that something was very wrong. Their search for answers resulted in multiple visits to different doctors. The answer came when Erica was 2. “They tested me for everything and that’s when they discovered I have sickle cell anemia, and it’s the very severe kind.” Erica was diagnosed with hemoglobin SS, which means most, if not all, of her hemoglobin is abnormal, resulting in significant pain, chronic anemia, and strokes. “It’s been a challenge,” said Erica.

“It has taken years to get even this far with treatments for sickle cell disease. And now, it's like we don't know what we'll face tomorrow.”

Erica

The severity of her condition meant Erica often missed school while growing up, and she has faced frequent hospitalizations throughout her childhood and into adulthood. While she graduated college with a Bachelor of Science degree in biology, Erica has not been able to pursue a full-time career because of her health challenges. For Erica and other SCD patients in similar situations, anticipated Medicaid work requirements are a concern.

Erica, the mother of a teenage daughter, relies on Medicaid for her access to care, which includes a regimen of 12 pills in the morning and four at night. She also undergoes apheresis, an automated red blood cell exchange, every three to four weeks. “If it wasn’t for Medicaid, I wouldn’t be able to afford the treatment. Medicaid is very important to me, as well as others like me. We have to have it to live.”

Erica is also unnerved by the cuts at the National Institutes of Health, which has funded research into a host of blood diseases and disorders, including SCD. .

“Honestly, I'm afraid. I'm scared,” she said. “It has taken years to get even this far with treatments for sickle cell disease. And now it's like we don't know what we'll face tomorrow.”