A look at breakthrough treatment for sickle cell anemia 

I’m a Black American molecular biologist. I 1st learned of the CRISPR technique back in 2011, and immediately understood its potential for a Sickle Cell Disease cure. I’m so glad the science has finally made it from the laboratory to the clinic. I once met a 30 year old SCD patient in the midst of a crisis…I was absolutely moved to tears by his cries of agony; made worse by the reticence of his healthcare providers to administer the necessary pain relief for fear he was “faking it” and simply “drug-seeking”. People don’t understand how painful this disease is…and the fact the vast majority of patients affected by the disease are Black has led to a gap in understanding, empathy and research dollars. It’s my wish that everyone who has this horrific illness gets access to this gene-editing therapy as soon as possible, no matter their location or ability to pay.

This condition is entirely overlooked. Grateful for this development.

If this is made freely available to patients, this would change the planet 🙏🏽🙏🏽

What I want to know is will this treatment be covered by insurance? I hope it is.

My daughter has sickle cell and this is very overlooked. People don't even understand what these people go through on a daily basis 😭 I'm so happy this is available.

My mom's best friend died from sickle cell disease (drépanocytose in French) in their childhood. My sister's classmate died from it in middle school. So many people die from it in Benin (west Africa). I hope this treatment will soon be available on the African continent as it is VERY much needed. Thanks for the groundbreaking research.

I wish my sister was alive to have had this groundbreaking therapy. Rest in peace Kamara.

I watched an interview of a young man in Kenya on Tuko who applied for Euthanasia in Belgium because he couldn't stand the pain anymore and didn't have the money to seek advanced treatment.I can only imagine him getting access to this treatment

My 6 year old son has sickle cell anemia. His first crisis was at 8 months old. This disease is so underrated and I’m so happy that they are bringing more awareness to this. I totally empathize with these stories and the untold stories of individuals who struggle with SCD❤❤

"I had no time to wait for someone else to be brave" powerful 🙏🏼🙏🏼

Needed badly in Nigeria 🇳🇬

My daughter has Sickle Cell SS. I hope that this treatment proves to be remarkable for young people and adults. 🙏🏾

Wish they had this back then before my father passed away due to this disease 😢!! They said he wouldn’t make it to 8 yrs old!! He made it to 56!! Soo young RIP Lamar Kimble ❤❤❤

Now here’s to hoping insurance will fully cover the drug… or atleast slash the million + dollar list pricing so patients can afford it..

I’ve had 2 close cousins who were sisters pass away from this disease. I’m glad to hear there are advancements being made in the treatment of this disease 👏🏾

This is great news and thank you for highlighting it! *Now we need the $2 MILLION treatment covered by insurance companies at more than 50%!* The average person cannot afford that.

This is wonderful news. Sickle Cell Disease is definitely horrific for the patient and family. Prayers that things continue to improve for these patients and hopefully, many more to come. Thank you for sharing this story.

Thank you Good Morning America - I appreciate you for this broadcast. I receive this research result on Sickle Cell Disease (SCD) therapy with thankful tears and trust in God that it will be a blessing to the millions of people and their loved ones suffering because of SCD all over the world.

I'm 35 and live in Nigeria, I was diagnosed when I was 10. Wish this remedy was available here.

Need this here in Jamaica

Currently pregnant with a little boy who has the possibility of being born with the disease. I pray that he isn’t born with it . Praying for all the scd warriors

This is so needed in Guyana and thank God for this breakthrough! Both me and my son have the trait; my son get pains from time to time however, I don't.

People who have sickle cell trait provides malarial protection. But of course those that have sickle cell anemia, you do not. I work in a blood bank and this is exciting and great news for these kind patients. I wonder what the limitations are and what are the criterias to this kind of therapy. So many questions for long term use. Where I work we have a large population that uses rare antigen negative blood for our sickle cell patients. Fortunately, where I live, we have a large African American population to provide blood for our sickle cell patients. Since this therapy is new it may be years before doctors will use this to treat their patients, it is extremely important for African American population to donate blood. Your blood could save someone life who is suffering from sickle cell anemia.

My brother died with it november 24 2022 at the age of 50 2 days before his 51st birthday

I’m a pediatric hematology/oncology RN and I see on a weekly basis the pain my sickle cell kids go through. They miss out on school and other activities bcs their pain is that debilitating. What’s more upsetting is that there are disparities in funding and outputs for SCD compared to Cystic Fibrosis. SCD has DRAMATICALLY lower research and FDA approved drugs and therapies than cystic fibrosis (CF), which is also genetic. But it made sense when I realized that CF is predominantly in whites and SCD in blacks…

This is an incredible breakthrough. I truly hope this treatment is covered by insurance so that it can reach as many people as possible.

I gave my brother bone marrow blood to cure his sickle cell disease in Atlanta ga I was 13 years of age now Iam 44 my brother is 37 years of age sickle cell never came back he was 2nd child to have it done at children hospital in Atlanta Ga

I need to figure out how I can get this stem cell transplant! I will DO anyything to get my blood corrected to live a normal life without pain. In NC if anyone has advice 😢

I hate having sickle cell...its too painful and sucks when even yhr best meds don't work

My best friend had sickle cell she was not expected to make it through her childhood. But she died 10yrs ago at the age of 42. She was in horrible pain most of her life and doctors called her a drug seeker. She was not. When she was passing away she just moaned and screamed in pain all day none of the pain medicine gave her relief. I’m so glad they will use this because those with sickle cell really do some suffering we can’t even imagine.

We thank God for this hope we wil receive this in kenya families are suffering due to this disease and many have die too.

i thank God for this break through, it's an answer to many peoples prayer.please make the this treatment available I in Africa.

We are a sickle cell white American/Italian family that have lost several of family members. Both my Italian grandparents were from Sicily! Caucasian people suffer this disease as well! I am a carrier, and my son is a carrier!! His both girls are carriers of trait!! Thank God this has been discovered to help this disease carrier!!!😢😂

My son lives with this disease, and this gives me so much hope. It’s true it’s such an OVER LOOKED DISEASE. God is so good !!!!

Both my kids have the trait. I want them to be very careful with who they decide to have a child with . It’s such a horrible disease 😢😢😢😢 I pray this cure works , my neice has it

What are the long term side effects/benefits of using this gene therapy ?

this is remarkable work…. RIP DAVIDA BANKS of Chicago, IL… 9years too late for her… knew her since 1st grade….her journey and fight ended 2015…

As a healthcare professional who has taken care of sickle cell patients it’s a terrible disease and I applaud the bravery of Victoria to open the door for many other deserving patients.

I have Sickle Cell Disease and would love to get this treatment.

Have lost dear family to this. So happy to see this progress.

It’s sad the donations for research often follow demographics versus need. I’m excited that so many black lives will be positively changed by this, and I hope this will lead to more empathy when patients with sickle enter into the hospital system.

Please it's needed in Nigeria. I am one and would really love to get more information on this please.

We need this treatment in Zambia.

Bring to Caribbean

This is good news. My first girl friend suffered from this. She went through so much pain. I started crying many times just watching her. Some good news in this hard world.

Well I've battled 57 years with it.... and is prepared to battle on until such time.....

Who else thought it was a video about Kelly Rowland?

Wow... I'm a 39 yr old that lives with this horrible/painful disease. I most definitely will look into this with my doctor. 🙏🏽❤️

Amazing!! Thanking God for people who care!!

I hope they'll consider making it available affordably for Africans too...& Our insurances cover it since the ppl affected most by this disease, at least I'm my country, are marginalized. I am so surprised they are this old with the disease, but I also happy . I hope we won't have to see children having their lives cut short....twas at 11 that I saw a child like me in a coffin an it still haunts me.

This is phenomenal. Thank goodness for the researchers and doctors developing this astounding treatment!! Bless the patients for having the courage and getting better.

Thank you for sharing this. I have a beautiful friend in London, England who suffers from this and wants nothing more than to be cured and be there for her 2 kids well into old age. She has been through so much as well as in and out of the hospital more times that I can count. Yes this needs to reach the rest of the world and fast. Lives are depending on it. I pray that somehow this becomes available for those who have little to no insurance.

I wouldn't risk having kids with inheritable genetic diseases. I'm not a selfish pos. My x bf parents KNEW they had a risk of this before they had a kid and they did it anyways 2X! MY X SUFFFFFEEERED and it stole his potential and future. The pain I watched him endure made me hate his religious zealot parents who didn't believe in science. Even if there is a treatment now nobody should have to be born REQUIRING medical intervention in order not to suffer when you know you are a carrier!

With this “FDA Approved” now and this being such a Amazing breakthrough it’s not that impressive They only showed two people whose received this quite extraordinary one time procedure .

My daughter has the trait and so does both of my grandsons. This is great news!

This is great. Unfortunately this is America. Which means that the high price of this treatment means that most people with sickle cell will never be able to utilize it.

Thank you Lord Jesus for this life saving treatment

Is this type of therapy used for MS disorders?

Can this get to Nigeria and west Africa? The people with SS cells really look like it. Same disease,. different environment.

Being a person that LIVES and SUFFERS from Sickle Cell Disease what they said that definitely hits the nail on the head is that it’s overlooked. It’s DEFINITELY overlooked and it shouldn’t be. People don’t get and understand what it’s like to live with this HORRIBLE SERIOUS DISEASE on a daily basis. But instead they look at it and see it as “the black disease.” So grateful this is in the making.

Who owns these GMO stem cells??? Africa stay far please. After COVID anything approved by FDA these days should be considered dangerous. Just my opinion!!!

Amazing. God answers prayers.I hope my best friend in Uganda will have access to this ❤.

Thank You for covering and educating others about sickle cell disease

There's hope. Thanks for sharing this story 🙏🏾.

Praise God

Thank you for your bravery. As we speak today my little warrior is six year old and she is going under the clinical trial for her age group, she is the youngest one in her group. As a mother I am grateful for my daughter, being chosen for this treatment. We are having the treatment at VCU in Richmond. The best part is that her older brother who has no trait or the actual disease was not a match for her to have bone marrow transplant. Since I was notified at 10days postpartum, I have been fighting to find a way to cure her. I did not want sickle disease damage her before something can be done. These past six years have been rough. But the almighty Allah knows best and he has the final word. Please keep us in your prayers.

I cried seeing the look on his face before getting up to hug her😭😭😭

hay hay thalassemia trait affect all people in asia they looks like die due to this desease

God bless 🙌🏽 🙏🏽 every one with this disease. I had a beautiful friend named Keisha who had the disease 🕊🙏🏽

Thank you Doners, scientists, engineers, nurses, doctors, patients…everyone. This is a game changer and will heal other diseases.

I PRAY THAT THIS WILL BE COVERED BY HEALTH INSURANCE AND AVAILABLE FOR ANYONE WHO IS WILLING TO HAVE IT. ASAP 🙌🏿🙏🏾🙌🏿

Thank you so much, it gives me hope for someone I love

Thank you for this video

It is incredible how medicine and science can team up to learn how to conquer chronic disease symptoms. Now, if only the United States could join other advanced nations by offering the taxpayers universal healthcare. . .

This needs to be accessible to minority groups- they are the ones really affected by sickle cell disease. Sickle cell is a chronic debilitating illness. Very difficult for patients to live a meaningful life. This therapy once widely available will be a miracle for all affected patients and their families.

It’s not rare. It’s just less common here in America. But not in other places in the world.

This is absolutely amazing 👏🏿 👍🏿🙏🏽.

Congratulations guys. I am so happy for you guys

I read an article years ago that claimed that those who suffered from sickle cell - I forget in which African country - ate African yams (not American sweet potato/yam) during the rainy season to prevent their cells from sickling. Perhaps someone could look into it.🙏🏾🙏🏾🙏🏾

I hope my daughter get this as a Yale patient.

Thank you for covering this.

Waiting for this in India but can't afford 😢

This is such great news. Sufferers go through agonising and incessant pain.

Needed in Jamaica .is it free in the USA

Great! FDA just approved another groundbreaking med for melanoma cancer - Iovance Bio therapeutics inc. IOVA

This is extraordinary. The medical science, the bravery of these patients. This disease is terrible and so debilitating.

I hate living with Sickle Cell 😢

literally, your veins are blocked... that pain is unfathomable. My best friend goes through hell but we are not in America, we need this for every country. I hope that the therapy lasts and doesn't add complications.

This is wonderful

CRISPER finally at work !! It’s effect in the life of those affected is indescribable. Forever grateful for this breakthrough knowing many have perished in agonizing pain at such a young age.

There is nothing to try here, THIS THING IS WORKING, START TREATING PEOPLE. WE THANK GOD FOR this discovery 🙏🙏🙏🙏🙏

My first born was born with Sickle cell anaemia{ss}, He was placed in medical 🏥 induced coma when he was just 22years old and at 8years old he developed a mini-stroke . We live in the United Kingdom. Sickle cell is a global medical emergency.

We need this in Zambia

Great News, I'm 60 years old and has suffered my entire life. I would like to know if it's good for those who are up in age?

Rest In Heaven to my niece! She died of sickle cell complications! 💜💜💜💜

Congratulations to the two.I wish I could get a chance for my son who is a sickle cell patient

King Tut also had this dis ease....makes me wonder ..WHO AND WHERE DOES THIS BLOODLINE COME FROM....IT SEEMS TO BE OF A ROYAL LINEAGE...TALK BLAK TO ME

Awwww I'm so grateful to God for this research 😢😢😢😢. May God bless whoever did this research.🎉

My daughter is 24 years and has SCD. It is very painful to see her go through the pain and the cost is very high. But also it is a disease that will disable your loved one as you look on. This disease can cause multiple strokes

We thank God. Let's share this so save loves of people in our community suffering from this.