Stem Cell Cure For Sickle Cell (Or Not?) Part II

I am finally home from travelling and speaking on behalf of people living with Sickle Cell Disease. My conclusion is not good. We (people with Sickle Cell) have the same concerns....and it's not looking good.

I will address these issues soon, but I wanted to finish my series on "Stem Cell Cure For Sickle Cell (Or Not?) Part II"
We hear in the news "Sickle Cell Cured!!!!" then.....nothing. We don't hear about complications, infections, secondary diseases caused by "treatment" or the true life-span of those "cured" children AND adults.

This is why I needed to finish exploring this "cure" subject. Here goes:

Stem Cell Cure For Sickle Cell (Or Not?) Part II

1. CHILDREN - Today, in children 250-300 stem cell bone marrow transplants are done in US. Doctors report a 90% success rate “IF AND ONLY IF” done on children with “perfectly matched sibling stem cell bone marrow donor”.

(Personally, I have NOT seen any long-term results data on these children’s quality of life, future complications from the aggressive chemotherapy treatment or immune suppressing medications, or the mortality rate after their “successful” stem cell bone marrow transplant)

I’m just saying.

In children, when there is a non-sibling stem cell donors (from the limited supply of National Bone Marrow Donor Program or Cord Blood Bank) there is a higher risk of immune diseases and transplant rejection which causes the body to attack itself. Over 5% reject the graft (transplant) & sickle cell comes back. National bone marrow donor register is also limited.

Cord blood stem cell donors are usually from a family member. This is very expensive, does not always cure sickle cell, and some patients die in the process with immune diseases caused by the aggressive chemotherapy and immune suppressing drugs.

My conclusion: Parents (and) patients must weight the benefits vs. risk with a FULLY INFORMED decision.

2. ADULTS - In adults, they have higher risk of side effects (immune diseases) and existing complications like previous strokes, eye damage, kidney damage, and bone decay issues like Avascular Necrosis will not be reversed if stem cell transplant is offered.

Answer: Cure in Adults has not proven effective, however future research & clinical trials will include (Genetically) Modified Stem Cell Bone Marrow Transplants.

According to a presentation (2012) by Dr. Donald Kohn at the Human Genome Medicine Program at UCLA, a recent $9 million dollar grant to California Institute of Regenerative Medicine (CIRM) was issued for stem cell “gene therapy” for curing sickle cell disease (in adults). Preparation for clinical trials (getting volunteer subjects) will begin soon.

(The requirements for this clinical trial are: 18 years or older, SCD (SS SThal), have few existing complications (that offset the risks of treatments), no existing matched donor, no existing matched sibling, pass state of health criteria.)

Stem cell “gene therapy” means bone marrow is removed (taken from hip), then “genetically modified” while patient is given high doses of chemotherapy to kill their existing bone marrow, then the modified bone marrow is given back to patient through transfusion directly into blood stream.

MY CONCLUSION:Sickle cell patients are being poked and prodded, clinical trialed and drug tested. In the meantime, we have to LIVE with sickle cell disease. I wish someone would fund Comprehensive SCD Care Centers again…..and help us “live” while you search for a “cure”.

Sources:
Dr. Donald Kohn at the Human Genome Medicine Program at University California at Los Angeles (UCLA):
Sickle Cell Anemia Stem Cell Gene Therapy
and Bone Marrow Stem Cells

The Hina Patel Foundation Cares

The Hina Patel Foundation in Bakersfield, CA works to support Sickle Cell Disease awareness and screening programs in India. The quote below is from their recent trip to India 2012:

“Sickle Cell camp in India was a huge success. We went to 5 villages (Zankhari, Chakara, Ambach, Mahuvaria, and Surali) and took samples of 3,732 people.

851 had sickle cell trait, 67 had sickle cell disease and approximately 40% of them were newly diagnosed.

They had pain crisis but due to poverty and living far from the city without transportation. These people usually stayed home and suffered with the pain.

We diagnosed a girl about 8 years of age that could not stand or walk due to multiple pain crisis. This was very sad. Her brother also was diagnosed with the disease.
Our foundation provided education about sickle cell, gave each tested person a personal report about sickle cell with explanations. We provided blood grouping to all 3,732 people. Healthy individuals received a “normal” report card with their blood type and on back of the card it tells them the importance of donating blood (a live blood bank for sickle cell patients).

The 851 sickle cell trait individuals got a card indicating they were a “carrier” and on back of the card it explained what it means to be a carrier. The card also explained that these “carriers” should seek genetic counseling when reaching appropriate age to get married and have children.


The 67 individuals with sickle cell disease were given a card that explains what they must do to take care as far as taking medicine, drinking 10 cups of water, dress warm, do not over exert, etc. Each person was given Folic Acid, Larium and Toradol for pain for 1 month. We also set up a program where they will receive these three medications once a month for entire year along and 4 times per year, they will receive CBC (funded by our foundation).

The doctor I worked with also gave check-ups.


Our goal (Hina Patel Foundation) is to continue screening once a year and to keep funding for medication. There are roughly 8 million people that live in this tribal region of Gujarat who mostly carry the sickle cell gene.”

The Hina Patel Foundation is doing their part to support sickle cell disease awareness, treatment and care. They are an example of doing something……anything for your fellow man, woman and child with sickle cell disease.

Thank you Hina Patel Foundation, the angels in heaven are smiling down on you.

Visit The Hina Patel Foundation website to find out more and to donate to their efforts.

****** Sickle Cell Awareness ******* One Reader At A Time

I have two important blogs to write.

(1) Is there a cure or NOT? part 2.

(2) The Hina Patel Foundation's work in America and India.

First, I thought I'd add a video about the recent sickle cell awareness & book signing event at Russo's Books in Bakersfield, CA. March 24, 2012

Fallen Soldier

Today I was reminded why I speak out about sickle cell disease. I found out that Phyllis Zachery-Thomas, a long-time sickle cell disease advocate and founder of SCD Soldier Network recently passed away.

Phyllis was an inspiration to me and one of the first voices on the internet, that I knew of, telling the world about sickle cell disease. She united people and inspired them to do something....anything.
God bless Phyllis Zachery-Thomas. Her memory lives on right here.

Sincerely,
P.Allen Jones

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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