By Don C. Reed
“Do you know about sickle-cell disease (SCD)?”, I once asked an African-American lady.
“Not much”, she responded, “Just that my Auntie died of it.”
It was one of those open-mouth-insert-foot moments, but I went ahead anyway, talking about the California stem cell program, to me the greatest medical accomplishment on Earth.
I will talk stem cells with anybody, any time. I spread the good word any way I can, like one of those people who wear a big sign board, a walking advertisement.
Social distancing makes the process more difficult, but still doable. If I stand in a line at the grocery store, I can talk to the person six feet in front of me. Of course I tailor the message to any disease they show interest in—and for African-Americans, that means sickle-cell.
Sickle Cell Disease Fact Sheet
One in every 500 African-Americans has sickle cell. It is an inherited disease, with a single gene mutated. That mutation causes the blood cells to warp at certain times, called crises, during which red blood cells change from soft and round to hard and sickle-shaped. Crises are agony: pain like broken glass in the veins. During the hours or days they may last, crises shut off oxygen flow to the organs, damaging them, which may subtract decades off a person’s life.
Sickle cell patients live an average 36 years, half a healthy person’s life span.
And these are not gentle years.
“By age 20, about 15% of children with SCD suffer major strokes….(of those who reach) 40, almost half suffer significant mental dysfunction, as well as recurring damage to lungs and kidneys, and severe chronic pain…”
https://www.cirm.ca.gov/our-progress/awards/clinical-trial-stem-cell-gene-therapy-sickle-cell-disease
The costs of medical care for a lifetime of sickle cell? Nine million dollars.
And as if all that was not bad enough, SCD patients are more susceptible to attacks of COVID-19. As patient advocate Adrienne Shapiro said:
“Sickle cell patients are at risk of dying from the virus as many have no spleens, are immune-compromised, and suffer from weakened lung function…”
--An advocate’s support for CIRM’s COVID-19 funding, Kevin McCormack, April 1, 2020
How can this terrible disease be fought?
That is the part where the California stem cell program (CIRM, the California Institute for Regenerative Medicine) comes in. Here are three different methods of CIRM-funded attack.
First, remember the 50 children who had the usually fatal bubble baby disease (Severe Combined Immunity Disorder, SCID) but whose lives were saved by CIRM-funded therapy?
Don Kohn, the UCLA scientist who developed that therapy, is adapting it for use against sickle cell disease.
How will it be done? In my non-scientist understanding, the procedure has three steps: remove, repair, replace;
1. Remove some of the patient’s bone marrow (where the blood is made);
2. repair the gene that is causing the trouble;
3. replace the now corrected bone marrow, where it will hopefully get rid of the bad genes in bone and bloodstream.
Mark Walters, of the Children’s Hospital of Oakland Research Institute, is using a similar approach, but with a microscopic scissor-like tool called Crispr CAS-9 to remove the damaging gene.
Importantly, Dr. Walters not only received a grant of $4.49 million from CIRM, but his project will be eligible for additional funding – due to “the landmark agreement between CIRM and the National Heart, Lung and Blood Institute…under the NIH “Cure Sickle Cell” Initiative.
Joseph Rosenthal, of City of Hope, is trying a different approach. The patient will first receive a “milder, less toxic chemotherapy treatment that removes some, but not all of (the) diseased…cells.” Then, a donor’s cells are transplanted. Hopefully, this will allow “the donor stem cells to engraft and create a healthy supply of non-diseased blood cells without causing an immune reaction…”
-- https://www.cirm.ca.gov/about-cirm/newsroom/press-releases/02222018/californias-stem-cell-agency-invests-stem-cell-based
What is the best path to cure? I am no scientist to tell you that.
But I do know the California stem cell program is trying hard, and has spent forty-one million dollars ($41,010,680.00) working to make an end to sickle-cell
One vital aspect of the struggle is the advocacy of African-Americans themselves: speakers, scientists, doctors and patients, bringing change to the research world.
My candidate for most cheerful advocate is Dr. Ted Love, formerly a board member of the California stem cell program.
He enjoys his name, and you can hear the smile in his voice when he answers the phone—“this is Dr. Love!”
But he is deadly serious when it comes to fighting sickle-cell. In fact, he came out of retirement to lead a biomed corporation to do just that.
His company is Global Blood Therapeutics (GBT), and their anti-sickle cell product is called Voxelotor, or Vox for short.
The product had a 274 patient clinical trial, and the results (which to my non-scientist eye seem positive) can be read in the New England Journal of Medicine:
https://www.nejm.org/doi/full/10.1056/NEJMoa1903212
I love to meet advocates—like Ade Adeyokunnu. Ade (Ah-day) has the condition himself, so he knows sickle-cell all too well. He can also express himself clearly, which is vital. He has a website, Sikcell.com, set up so that people living with sickle cell can share information with others who understand the struggle.
Naturally I wanted him to meet Dr. Love.
But would a CEO of a major corporation take the time to meet someone who might possibly be helpful somewhere down the road?
“Of course,” said Dr. Love.
I did not hear from Ade for a while, and I worried he had dropped the ball.
But when I called up Dr. Love, he said, “Oh no, we talked—but Ade is in the hospital right now, with a sickle-cell crisis…”
Advocates continue on, no matter their personal pain.
Listen to another champion advocate, Adrienne Shapiro, recent recipient of Bernie Siegel’s World Stem Cell Summit Advocate of the Year Award:
“It’s my belief that I will be the last woman in my family to have a child with sickle cell disease; and that she, Marissa, is 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.”
May that great dream come true, and soon.
https://www.cirm.ca.gov/our-progress/stories-hope-sickle-cell-diseases;2014