Patient Voices

Bringing Awareness to Sickle Cell Disease: A Fireside Chat with Mapillar Dahn

January 26, 2021

Sickle cell disease (SCD) is an inherited blood disorder in which the body makes “sickle-shaped” red blood cells that tend to clump and restrict blood flow, causing severe pain, organ damage, strokes, anemia, and a heightened risk of infections.

Although the genetic mutation causing SCD has long been known, research into new therapies was woefully lacking, in large part because the disease disproportionately affects people of African descent. That is starting to change, with the approval of a new medicine from Global Blood Therapeutics (GBT) and other therapies in development. Now in collaboration with GBT, Syros is working to develop a novel oral medicine with the aim of providing a functional cure for SCD.

Syros CEO Nancy Simonian held a fireside chat with Mapillar Dahn to better understand the experiences of families affected by SCD. Dahn, who has three daughters with SCD, was motivated by her experiences to found an advocacy group, MTS Sickle Cell Foundation, to raise awareness about the disease and support families affected by this life-threatening disease.

A Stigmatized and Misunderstood Disease

Nancy Simonian: Does SCD run in your family?

Mapillar Dahn: SCD does run in my family, but before I had my first daughter, I didn’t know that because people didn’t talk about it. I found out I had the sickle cell trait in high school, but there wasn’t a sense of it being a serious thing. There was no education around it.

There is a common misconception about SCD skipping generations, or that if you have one child with the disease, your other children won’t get it.

The disease also ran in my children’s father’s family. He had relatives who were always in the hospital and one of his sisters passed from it. When my first daughter was diagnosed with SCD, I was crushed because I imagined she would suffer the same things.

Almost 17 years later, with three beautiful daughters who all inherited the disease, SCD is my life. My girls are so full of life and have taught me so much about living life to the fullest in spite of adversities.

NS: You mentioned that your family didn’t talk about SCD. Is there a stigma attached to it?

MD: Absolutely. I am an African from Liberia. We don’t talk about medical conditions, especially a disease like SCD. Even in America, many Africans who are impacted don’t talk about SCD, especially not about their kids who have it.  This means that there are many families who suffer in silence.

NS: When were your daughters diagnosed and when did they begin to experience symptoms?

MD: All three were diagnosed through routine newborn screening.

My oldest daughter, Tully, who is now 16, started to have severe bouts of pain, known as pain crises, when she was 6 months old. Until a year ago, she was having two to three crises a year, which for her means being in the hospital for at least a week. About a year ago, after a blood transfusion, she developed antibodies to the blood group antigen Jsb, which means she can only get transfusions from the 1-2% of donors who lack this antigen.

My other daughters have not had pain crises, but my middle daughter, Khadeejah, who is 15, had a stroke when she was 7 years old. Since then, she’s had to have monthly blood transfusions and multiple surgeries, including brain surgery.

My youngest daughter, Hajar, who is 11, struggles with the disease in her own ways. She doesn’t have obvious symptoms or require regular blood transfusions, but she has cognitive challenges that I believe are likely the result of “silent” mini-strokes associated with SCD.

NS: Your family shows how the symptoms and experiences of every patient can be different.

MD: Yes, and that’s part of the conversation I bring to the pharma industry, which is very pain-centric in terms of developing SCD therapies. Not everyone struggles with pain. We have to look at individual patients and treat the disease more broadly.

NS: Why do you think the disease has been treated so narrowly?

MD: Lack of education and training of ER doctors and other healthcare providers. Many doctors don’t know much about SCD because there is very little in the medical textbooks. One way to shift understanding is to update those books – and if nothing else, add that SCD impacts everyone differently.

Racial Inequities and Sickle Cell Disease

NS: Can you speak to the role race has played in stigmatizing the disease and affecting research and treatments for it?

MD: SCD is seen as a Black disease. Is that why SCD isn’t taken as seriously? Is that why patients are not getting proper care? Is that why some healthcare workers believe patients are faking their pain to get medications? I think so. And that’s why we are raising our voices to not only educate, but to also help dispel those myths and misconceptions that stand in the way of SCD patients receiving timely and adequate care.

NS: Have you experienced skepticism from doctors about your oldest daughter’s pain?

MD: No, but I know families who have had medical professionals say, “Your child is addicted to pain meds.” In fact, of all the things that can be done while we’re waiting for a cure, the main one is improving how SCD pain is handled in the ER.

For example, patients with the SS form of the disease supposedly have the severest pain, but many patients who have the SC form also experience pain. Doctors who don’t realize this will say to an SC patient, “You shouldn’t be in so much pain,” and that brings a stereotype into the hospital room.

Patients are experts in their own disease, but when it relates to SCD patients in the medical setting, this isn’t respected or recognized. Patients are not seen as partners in their own care. If a patient says, “This drug doesn’t work for me, but that one does,” what the doctor hears is, “This person has a drug of choice,” instead of, “This person has lived with their disease and knows what works for them.”

More needs to be done to build communication, sensitivity and awareness. If we don’t change the culture around how the medical community interacts with the SCD community, companies like Syros can come up with the best medications and it will all be for nothing because a lack of trust presents a very real barrier to care.

NS: Do you think the SCD community distrusts the medical community and clinical trials because of the terrible history of medical experiments on Blacks?

MD: Yes, but we have to push against it because our community needs these new advances. We need to educate the community about the safety of clinical trials. We need patients to get involved in the clinical trial process by not only participating, but also sharing their opinions. If there is one thing a patient will be comfortable with, it’s the opinions of another patient – that’s even more important than a doctor’s opinion.

Building Trust in the SCD Community

NS: As a drug company that’s working on new treatments for SCD, what can we do to build trust with the SCD community, especially given how long drug development takes?

MD: First, I want you to know that patients are rooting for you – and not just in this country.  Right now, there are people in African villages and remote places around the world who have SCD but do not know it. They have pain and experience the complications that are associated with this disease, but they don't have a name for what's ailing them. They were not fortunate, like my children, to have been screened at birth. They do not have access to care. Some rock themselves to sleep many nights to distract themselves from the excruciating pain they suffer. They are depending on you to help them someday.

Beyond working on new therapies, the most important thing you can do is to respect the community. Ask what you can do to help us. For example, GBT didn’t say to our foundation, “Come sit at our table.” They asked us, “May we have a seat at your table?”

Make sure the community knows what you’re doing. Continue to promote education about the disease because that shows the community that we can rely on you. What you’re doing might not be the cure just yet, but we can trust that you are there for us.

You can also help other organizations and efforts that aim to support the community. Often, we stop at awareness, but this community also needs a lot of support.

Supporting Families Affected by SCD

NS: What kind of support, and how does the MTS Foundation provide it?

MD: People might hear about the symptoms of SCD, but they don’t see the socio-economic effects of this disease. One minute everything is OK and the next minute you’re in the hospital with your child – wondering how you’re going to pay your rent and bills. It can be hard to keep a job. I’ve been in that position myself. I’m fortunate to be able to freelance and build my work schedule around my daughters’ needs. My mother also lives with us so I can be at the hospital with one daughter and not worry about my other two. But not all families have those options.

I want to talk about these socio-economic issues and do something about them. The MTS Sickle Cell Foundation helps families pay for rent, food, transportation to the doctor’s office, and even burials. I don’t have a template because everyone’s needs are different – just like the disease. It impacts every aspect of your life. What I say is, “If we can help, we will.”