Stem Cell Cure For Sickle Cell (Or NOT?)

I have read headlines that say, “Cure for Sickle Cell Disease!!!!!!” Then I asked myself, Why am I NOT cured????

I looked around for first-hand reports of people who were “cured,” but no one would speak after getting the cure. One year ago, after an “adult” stem cell clinical trial, I tried to speak to one participant. She wouldn’t (or couldn’t) talk to me. It all seemed a little too fishy.

I needed an answer. Is there a cure for sickle cell disease, or not? In my search, I found a lot of information. It is very complex, but I believe WE need to understand what doctor’s are doing for us AND to us.

I found one good resource in California, a presentation by Dr. Donald Kohn, Director of the Human Genome Medicine Program at University of California at Los Angeles (UCLA).

Based on Dr. Kohn’s presentation, I will write a three-part blog on the subject of “Is there a cure for sickle cell disease?” I will try to break the information into bite size pieces, so WE can understand and digest it all.

First, I’m going to address bone marrow stem cell background, second, what doctor’s are doing for children today, and lastly, what’s coming in the future for adults.

Here goes.

According to Dr. Kohn - 150 newborn cases per year (in Calif.), 9000existing (Calif.), 80,000 in USA, current medical cost = $10,000 - 15,000 per person (per year), and most sickle cell related medical cost are paid by Medicaid or Medical. (really???)

I guess there is a financial reason for the medical community to find a cure for us. And, that’s good!!!!

Part I - Is there a cure for sickle cell? (background)

1. What is the biology of sickle cell? Our red blood cells are packed with Hemoglobin (the oxygen carrying protein). It has 4 proteins, (2) Beta and (2) Alpha chains. On one of the Beta chains (the 6th amino acid) it is mutated (HbS). In conditions of low oxygen (one trigger), the mutated hemoglobin molecule (HbS) attracts to another mutated (HbS) molecule and they stick together. This causes the hemoglobin to go from round flexible shape to rigid sickle shape.

2. In 1982, the first (accidental) cure for sickle cell disease occurred by a bone marrow stem cell implant to treat a patient who had leukemia AND sickle cell disease. The resulting stem cell implant cured the “child” of sickle cell disease by accident. NOTE: I have no information on what ever happened to that patient. If the “cure” lasted, what their quality of life was, nothing.

3. How is bone marrow stem cell transplant treatments used to “cure” sickle cell?
The body makes red blood cells from stem cells in bone marrow or cord blood. Stem cells are transplanted from the bone marrow or cord blood of a donor (hopefully, completely matched to us). The goal is for our bodies to accept the transplant AND make our bone marrow produce (good) red blood cells without mutated HbS hemoglobin.

End Part I

The following video is hard to watch, but necessary to understand how much bone marrow donors go through for us.
How Bone Marrow is taken from donor.
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Guest Blog by Jenna Walters

I believe in supporting people who want to advocate for sickle cell disease awareness and education.

Jenna Walters wrote the following blog,"Experimental Treatments for Sickle Cell Anemia" and I support her efforts to participate in this important work.

"Sickle cell anemia is an inherited disorder where the usually round red blood cells become sickle shaped. This causes blockages in the capillaries which leads to episodes, called “crises,” of severe pain in the joints, abdomen, chest and back. The disorder affects mostly people of African descent. Over the years experimental treatments have been used to help ease the symptoms of sickle cell anemia. They include:

Nitric Oxide TherapyInhaled nitric oxide is used to help capillaries to dilate, or open up. This lets the blood flow more easily because it keeps the sickled blood cells from sticking together and forming clots, which can lead to sickle cell crises.

Stem Cell Transplants
Stem cells are cells that can turn into any sort of cell in the body, including blood cells. This sort of stem cell transplant is basically a bone marrow transplant. A donor is found for the patient and their own bone marrow is destroyed through courses of chemotherapy or radiation. The donor’s stem cells are then intravenously fed into the patient in the hopes that they’ll create new, healthy red blood cells. This procedure is usually done on people whose sickle cell anemia has become unmanageable, because it's frankly risky. The patient’s body can reject the donor's cells, which will lead to complications.

Gene Therapy
Gene Therapy is now being explored for diseases ranging from sickle cell to peritoneal mesothelioma. Medical researchers now believe it may be possible to replace a patient’s defective sickle cell gene with healthy genes. They can do this by putting the healthy gene into the bone marrow of the patient. They can also find a way to “switch off” the sickle cell gene while turning on a gene that causes the production of fetal hemoglobin, which would stop the blood cells from sickling. Researchers are also looking for drugs that can support the production of fetal hemoglobin. One of the drugs now under study is Vorinostat.

Possible Clinical Trials - MP4CO therapy
Some researchers believe that combining the drug MP4CO with more traditional therapies can dilate the blood vessels and get oxygen quickly into the cells. This can either stop or shorten the length of a sickle cell crisis."

sources:
mayoclinic.com,
Sciencedaily.com,
Possible Upcoming MP4CO Clinical Trials

Supporting Sickle Cell Support Groups

I recently read a story on a facebook sickle cell group page about a young woman with sickle cell who went to the doctor with her younger sister. Her younger sister was newly diagnosed with sickle cell AND was having a pain ‘crisis’. The older sister was shocked at the doctor’s response when he referred them both to a hematologist and then said, “I didn’t know what to do with sickle cell.”

The shocking part about this story is that the young girl’s pain ‘crisis” was not addressed. This was shocking because of the basic requirement for medical professionals to "treat" sick people.

What does this say about sickle cell treatment today? There is still a lot of work to be done.

We still have to participate in educating the medical community, informing the public, and advocating for respect, good treatment and compassion. We (people with sickle cell) have to support each other.

Sickle Cell Support Groups are a united voice for people suffering with this disease. Together health care leaders, SCD patients, medical staff, friends and family do make a difference.

Together, they encourage us to live well and live long. They inform us and answer questions that relate to our experiences (bad or good) with health care providers. They plan events in the community to bring awareness and put a face on sickle cell. They work to encourage the political process related to sickle cell policy, research and funding. They also empower us to take charge of our health.

Sickle Cell Support Groups are everywhere.

Search the internet for “sickle cell support groups” for your city or state. Ask your hospital’s education or patient services department, look for clinics, medical groups or sickle cell advocacy groups that meet on a regular basis.

Get involved and have your say. Be apart of the bigger fight because there is power in numbers. Together, we will win this battle against sickle cell disease.

If you can't find a support group, go to on-line groups like People Living With Sickle Cell.

Or try Facebook. There are a lot of facebook group links where people with SCD share information and encourage each other. (My facebook page is just one example).

Let’s not tolerate bad medical care, medical disrespect, or ignorance to go unchecked. We can (together) address our needs one doctor at a time, one hospital at a time, one city at a time and one nation at a time.

A New Day Has Come For Children With Sickle Cell Disease

(photos by Denise Truscello/ WireImage)

On January 15, 2012 Play Without Pain: Children’s Hospital Sickle Cell Benefit at The Colosseum in Caesars Palace began with Gary W. Loveman, Chairman, Chief Executive Officer and President of Caesars Entertainment Corporation thanking everyone for supporting this life-changing event. He explained the alarming statistics of children suffering and dying from the complications of sickle cell disease, and the important work being done at Children’s Hospital of Boston to find a cure. “Children’s physician-researchers have done what others have tried to do for decades; they’ve found a way to override the sickle cell defect,” he said. Mr. Loveman then introduced the team of doctors led by Dr. Stuart Orkin and Dr. David A. Williams, who are behind the breakthrough strategies to “flip the switch” on Fetal Hemoglobin F (HbF) and cure sickle cell disease once and for all. “Hope,” he said, “is in sight.”

Enter Celine Dion and Rene Angelil, AEG Live, Children’s Hospital Boston, Southern Wine & Spirits, Angel Management Group, Restaurant Guy Savoy, and countless other sponsors too numerous to name. Only heaven bestows such star power. The rest was history in the making.

As Celine walked onto the stage, a new day dawned for children with sickle cell disease. When she began to sing angels smiled, clouds parted and the rain of hope fell like a beautiful waterfall. With the voice of perfection, accented with flawless wardrobe changes, and make-up sprinkled with tears, Celine’s heart was fully exposed.

While I listened to Celine perform, like only Las Vegas could contain, I looked around the Colosseum in amazement. I was witnessing history. When Celine cried, I cried. God allowed me to live to see the dawning of this new day.
(photo of Celine and A.J. Green, young man with sickle cell)

As Celine sang, my heart sang,

“I see a light in the sky
Oh, it’s almost blinding me
I can’t believe I’ve been touched by an angel
With love

Let the rain come down and wash away my tears
Let it fill my soul
And drown my fears
Let it shatter the walls
For a new sun
A new day has come
A new day has come.”
(song by Aldo Nova & Stephen Moccio)

I may not see a cure for my sickle cell disease; I’m too old for these new procedures. I see, however, the light shining for children with sickle cell disease…………can you see it?

Thank you Melanie, Caesars Palace, Celine and Rene, Children’s Hospital of Boston, and the numerous sponsors of Play Without Pain: Children’s Sickle Cell Benefit. You are all partners in giving the gift of life. Put on your sun glasses, it’s gonna get bright.

(photos by Denise Truscello/WireImage)

2012 - The Year of SCD Awareness

All I can say about the coming year of 2012 is WOW!!!!!.

I have heard more about Sickle Cell Disease in the last six months than I have in the past five years. That is partly due to the tireless voices of SCD patients speaking out, SCD Associations not giving up the fight, and a renewed national commitment from American government agencies like National Institute of Health (NIH/NHLBI)) and Center for Disease Control (CDC).

What the focus seems to be leading toward is psycho-social help and improved care/treatment for adults with SCD. That is a good thing.

Another good thing is the 2012 calendar filling up with events, support services, conferences, and meetings about Sickle Cell and related blood disorders.

Our Voice, the voice of people living with sickle cell disease, will continue to speak out bringing information and education globally. I'm excited about the future so stay tuned. I'll post events as they come my way.

One big event coming January 15, 2012 is Celine Dion at Caesars Palace. She will play a charity show to support Children's Hospital Boston "Play Without Pain Children's Sickle Cell Benefit."

This is a wonderful thing!!!!!



Click here for ticket information & concert link