Imagine pain, excruciating pain, like broken glass in your veins. Now imagine, on top of that, being insulted — at the hospital? Hold that thought.

First, the enemy: sickle cell disease (SCD) which means a change in the shape of your blood cells. Instead of being round and soft, the cells become hard and c-shaped, like a sickle. These hard sticky cells can clog the blood vessels, an agonizing experience called a “crisis”.

Because these crises slow the blood’s ability to transport oxygen, sickle cell anemia can damage organs in your body: liver, lungs, heart, brain: by the time an afflicted child is ten, he/she may have had a major stroke; by 20, there is a strong possibility of brain damage.

This alone is reason to take joy in the California stem cell program’s funding of research like Dr. Mark Walters’ efforts at the Children’s Hospital of Oakland Research Institute (CHORI).

Dr. Walters hopes to use a gene-editing tool called CRISPR/Cas9 to fix what’s wrong in the body’s cells: remove, repair, replace.

Imagine if he was successful. In financial terms alone, the benefits would be extraordinary.

“The estimated lifetime cost of care is $9 million per person. (emphasis added) The project aims to improve SCD therapy by preparing for a clinical trial that might cure SCD after giving back sickle gene-corrected (blood) stem cells to a person with SCD. If successful, this would be a universal life-saving and cost-saving therapy.”

https://www.cirm.ca.gov/our-progress/awards/curing-sickle-cell-disease-crispr-cas9-genome-editing

But there is something else: a sense of wrongs, needing to be made right.

Remember those previously-mentioned insults? Imagine going to the hospital, in agony, and then when you get there, you are treated like a drug addict?

Unfortunately, this prejudice appears to be real. I interviewed four African-Americans with sickle cell knowledge; each mentioned the insults as a problem.

“Those with sickle cell see going to the hospital as going into battle, “said patient advocate Nancy Rene, “They “gear up” with copies of medical records and NIH guidelines, make sure they have a diary to record inappropriate remarks from medical staff, ask a friend to come along as an advocate to help them withstand the implied racism…with which they are confronted.”

https://blog.cirm.ca.gov/2016/09/29/a-patient-advocates-take-on-sickle-cell-disease-the-pain-and-the-promise/

Ms. Rene’s grandson, now 14, had a sickle-cell caused stroke — at the age of nine months.

This experience with “implied racism”, unfortunately, is not unique.

“Studies show if I as a black man and you as a white person go into the ER with severe pain, you are far more likely to be supplied with rapid pain relief treatment than me”, said Dr. Ted Love, former member of the California stem cell Board of Directors: also a Harvard/Yale physician-scientist and CEO of a major corporation.

Suggestion: California was the first state to mandate sickle cell screening at birth, mandatory now in every state. So why not have a medical card saying “SICKLE CELL PATIENT”? We already have the information, gathered by screening at birth — maybe a card could reduce the hassle at the Emergency Room?

Dr. Love had tried to retire, but fortunately, it did not stick.

I asked him how such a young man could even consider retiring, and he replied with a laugh: “Well, there is a lot of mileage on me!” But when a friend, asked him to consider coming out of retirement to become the Chief Executive Officer (CEO) of Global Blood Therapies, (GBT), he could not say no — because GBT is focused on fighting sickle cell.

“Ever since I was ten, kids called me Dr. Love,” he said, and I could hear the smile in his voice over the phone. “I worked with patients directly for a lot of years, and it hurt to walk away from them, as if I was letting them down. But to work for therapies and cures would benefit more people around the world.”

Now, Dr. Love’s company is working on a new drug: Voxelotor.

There is also the CIRM-funded effort of Dr. Donald Kohn, famous for his work to defeat Severe Combined Immune Deficiency (SCID), which technique has cured nearly two dozen children who had “bubble baby syndrome”.

Perhaps a similar technique might work in the fight against sickle cell?

To remove some bone marrow from the patient, reconfigure the DNA by removing the bad mutation, and then putting the improved blood cells back — remove, repair, replace — if it works, that would be wonderful indeed.

But we cannot forget the advocate: without whom the scientist often stands alone.

When I first talked on the phone with Ade Adeyokunnu, I had a sense of recognition, as if I had known him before.

It was like a story I read about a Hell’s Angel motorcycle leader, who was asked how they recruited members. He said, “We don’t recruit them, we recognize them.” And that is how it works for advocates. You meet someone with a disease, and they talk cheerfully and knowledgeably about it, and how it is best fought? That is an advocate, and we recognize each other.

Ade (Ah-day) Adeyokunnu is an advocate fighting sickle cell.

If you visit Sikcell.com, you can see his community website: designed so people living with sickle cell can share information with others who understand.

He should talk to Dr. Love, I thought. But would a CEO of a major corporation have time to talk with someone who might be helpful somewhere down the road?

“Of course,” he said, after we had gone through the difficulties of squeezing in a half hour on his schedule to talk, and I followed up with Ade.

However — I did not hear from Ade for a while. I worried he had dropped the ball.

“No, no”, said Dr. Love — “Ade is in the hospital, with an SCA crisis.”

He told me a story of another person with SCA who had gone to a medical conference and then seemingly disappeared. His friends searched for him, and finally located him in a hospital, where he had gone into a coma….

You will be relieved to know that Ade is past the crisis now. He continues to work on his Master’s Degree in Business Administration — and to fight sickle cell.

I want to close with a message from one more outstanding advocate.

Adrienne Shapiro is a stem cell Ambassador for Americans for Cures Foundation. Adrienne is a great talker, very likeable, and can put into words the urgency of the need to defeat Sickle Cell.

She was just recognized as Patient Advocate of the Year by Bernard Siegel’s World Stem Cell Summit.

“It’s my true belief that I’m going to be the last woman in my family to have a child with sickle cell disease; and that she, Marissa’s going to be the last child to suffer, and that her younger daughter Casey is going to be the last one to fear. Stem cells are going to fix this for us and many other families.”

https://www.cirm.ca.gov/our-progress/stories-hope-sickle-cell-diseases;2014

Thank you, Adrienne: you speak for us all, every parent, and every child: for anyone who suffers an “incurable” condition, and who will fight back.

This post originally appeared on Medium. 

Don C. Reed is Vice President of Public Policy for Americans for Cures, and he is the author of the forthcoming book, CALIFORNIA CURES: How California is Challenging Chronic Disease: How We Are Beginning to Win—and Why We Must Do It Again! You can learn more here.