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

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.
*

Part 2 - Stem Cell Transplant

Part 2 - Anyone Want a Bone Marrow Transplant or
maybe a Stem Cell Transplant?

I couldn’t sleep thinking about this “cure”. I was wondering is there is a rush to yell cure?

Researchers gave 10 patients ages 16 to 45 bone marrow Stem Cells. They gave them low levels of radiation, chemotherapy and high levels of immune suppressant drugs.

People with SCD normally have spleen problems; this makes them more prone to infection. Think about the infection possibilities when you give a person, who’s already catching everything that blows by, an immune suppressant drug. You may have received a cure for sickle cell, but now you may get cancer to replace it.

Researchers said, “Though most patients in the study are still taking immune-suppressant drugs, researchers hope to eventually wean them off the medications.” Check out the side effects of Alemtuzumab, a drug used to suppress immune system T-cells, and Sirolimus, an immune suppressant to fight rejection.

Let’s not run to the hills crying CURE until you can give a person a better quality of life, not replace their problem with another one.

Stem-Cell Transplantation for Sickle Cell Disease

Anyone Want a Bone Marrow Transplant or
maybe a Stem Cell Transplant?

The airways are popping. The media blitz is in full affect. Massive editorial posts have been sent all over the internet. The word is out. There MAY be a cure for Sickle Cell Disease.

According to a New England Journal of Medicine report dated December 10, 2009, researchers at the U.S. National Institutes of Health say that a new method of bone marrow transplantation cured nine out of 10 adult patients with sickle cell disease.

NEW…hmmmm. I’ll take a big mac, fries and a bone marrow transplant…. please.

Here’s what I’ve gathered from reading the media stuff.

Old Bone Marrow Transplant Method - In conventional bone marrow transplants, high doses of chemotherapy drugs and radiation were given. Chemotherapy and radiation is used to wipe out the person's own bone marrow, which makes the faulty red blood cells. There are many complications including destroying fertility

Adults were usually not good candidates for bone marrow transplants because they were thought to be too sick to handle the high doses of chemotherapy and radiation needed to prep the body for the procedure.

New Bone (Stem Cell) Marrow Method - Senior study author, Dr. John Tisdale, a senior investigator at the U.S. National Institutes of Health explained the new method allows for less grueling pre-transplant routines, which adults with severe sickle cell can tolerate.

10 patients ages 16 to 45 with severe sickle cell disease received bone marrow from donors that were siblings with matched HLA (human leukocyte antigen) in their blood.

Lower levels of radiation were used in the new method which does not seem to destroy fertility. The bone marrow is replaced with stem cells from a donor's marrow, which then takes over and begins to produce new, healthy red blood cells.

NOTE: When doing the new bone marrow transplants, the researchers noted that not all of the patient's own marrow was wiped out. Some remained and seemed to co-exist with the donor marrow without causing problems. Dr. Tisdale said, "That meant we didn't necessarily have to kill the entire bone marrow of the patient to make this work." Tisdale said, this opens the possibility of using an even less toxic means of preparing the body for transplant.

Patients in the new method study were also given:

Alemtuzumab, a drug used to suppress immune system T-cells
Side Effects of Alemtuzumab - events include hypotension, rigors, fever, shortness of breath, bronchospasm, chills, rash, syncope, pulmonary infiltrates, ARDS, respiratory arrest, cardiac arrhythmias, myocardial infarction, and cardiac arrest. Some cases of cardiac adverse events have resulted in death. http://www.drugs.com/sfx/alemtuzumab-side-effects.html

Sirolimus, an immune suppressant to fight rejection
Side Effects of Sirolimus – acne, back pain, constipation, diarrhea, headache, joint pain, nausea, trouble sleeping, vomiting and weakness. http://www.drugs.com/cdi/sirolimus.html#side-effects

After 30 months, all 10 patients are alive, and nine of the patients had successful grafts where none of the patients experienced graft-versus-host disease (where the body rejects the new bone marrow). They are also considered cured of sickle cell disease, according to the study. Though most patients in the study are still taking immune-suppressant drugs, researchers hope to eventually wean them off the medications.

The new procedure doctor’s say is promising, especially since it could eventually include those who don't have an HLA-matched sibling. Dr. Tisdale said, "These were the sickest of the sick patients. Some were in the hospital every other week for pain or other crises. Today, some have gone back to school and to work. One patient had a baby."

In the past, these older/sicker patients were excluded from transplant studies as they are very poor candidates for high dose chemotherapy regimens. This study makes it possible to offer patients with severe sickle cell disease stem cell transplants."

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My conclusion: This is a “study”….also know as a “trial”. We won’t see this stem cell treatment given to “us” for a while. But, I’m hopeful.
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Links related to this report:

Bone Marrow Transplants May Cure Sickle Cell in Adults http://www.dreddyclinic.com/forum/viewtopic.php?f=25&t=22080&p=34580#p34580

Bone Marrow Transplants May Cure Sickle Cell in Adults - http://www.nlm.nih.gov/medlineplus/news/fullstory_92818.html

Stem Cell Researcher Dr. MM Hsieh – Co-author of report http://www.stemcellscience.org/showauthor.php?surname=Hsieh&initials=MM

New England Journal of Medicine report dated December 10, 2009
“Allogeneic Hematopoietic Stem-Cell Transplantation for Sickle Cell Disease”
http://content.nejm.org/current.dtl

National Institute of Health

http://www.nih.gov/

Calling All People With SC Disease

Calling all people with Sickle Cell Disease to participate in a research study.

The study is sponsored by Ms. Phyllis Bazen, MSN, FNP-C, a Doctoral Candidate at the University of Rochester School of Nursing. Ms Bazen is the Study Coordinator.

The purpose of the study is to explore the most common stressors that adults with SCD cope with and to explore the effects that these stressors have on mood and/or quality of life perceptions.


People with Sickle Cell Disease have many things that bring stress; pain, depression, hopelessness, etc. etc. etc. We have to live on despite these stressors and try to have a meaningful, productive lives...anyway.

The best way to contact Ms. Bazen is the web site contact page, her cell phone, email, or 1-800-464-8668 (in US) See card below for information.

If you decide to participate in this study, you will be speaking with Ms. Bazen who conducts a one-on-one private (confidential) phone interview. In this interview, Ms. Bazen will ask a series of questions related to living with Sickle Cell Disease and related stresses.

The outcome of this study is to gather data related to the stress that Sickle Cell patients endure and find ways to better treat them (us).

Check out the web site at http://www.scdstresstudy.com/

We can all have our voice heard on this one.




.

BCL11A

Researchers have finally identified the genetic address of Sickle Cell Disease. It’s BCL11A, that wonderful alphanumeric.

In a report from the National Institute of Health on Friday, December 19, 2008, they report, “Researchers have identified a gene that directly affects the production of a form of hemoglobin that is instrumental in modifying the severity of the inherited blood disorders sickle cell disease and thalassemia. The discovery could lead to breakthrough therapies for sickle cell disease and thalassemia, which could potentially eliminate the devastating and life-threatening complications of these diseases, such as severe pain, damage to the eyes and other organs, infections, and stroke."

This is only the beginning.

Researchers report that by suppressing the gene BCL11A, fetal hemoglobin (HbF) production could improve dramatically. They say, “New therapies targeting BCL11A would be the first to directly affect the natural processes involved in increasing HbF.”

They’re not there yet.

So, what does this mean? I don’t know, but what I do know is that if researchers can “target” the gene, then help is on the way.

I hope I live to see the day.

Source:
http://public.nhlbi.nih.gov/newsroom/home/GetPressRelease.aspx?id=2607

NICOSAN vs. HYDROXYUREA

There has been a lot of talk about drugs that treat Sickle Cell Disease. (Not to mention cures that work for a few people but not all????) Currently, I’m not taking any drugs but I really wanted more information about the two drugs available, Nicosan and Hydroxyurea.

Here’s what I found:

The first drug I’ll discuss is the only US Food and Drug Administration (FDA) approved drug for the treatment of Sickle Cell Disease. It is Hydroxyurea, which is sold in the US under the trade name Hydrea(TM). It has also been used in the treatment of leukemia and certain other cancers.

Hydrea induces the synthesis of fetal hemoglobin, which inhibits the production of the abnormal sickle cells (what the heck does that mean?). I believe that means; this drug causes the production of healthy fetal hemoglobin without the sickle cell abnormality. In other words, it makes your body make good hemoglobin. Why do we care? Well, hemoglobin carries oxygen inside the red blood cell. One little change causes the hemoglobin to form long rods in the red cell (when it gives away oxygen). These rods change the red cell into the sickle shape…and we don’t want that.

The reports on this drug are mixed. I’ve read that not all patients respond to this treatment, and I’ve read of cases of leukemia being developed in patients…now, that’s scary.

Second, there’s Nicosan. Formally know as Niprisan, and in America it’s known as Hemoxin. This drug is not approved by the FDA. It is manufactured in Nigeria, Africa and is supposedly in clinical trials there, but none have begun in the United States. This drug is a plant base drug which means it’s made from organic plant material…that’s gotta be good, right? Some side affects reported are rash, headache, and weight gain.

XECHEM, the company that makes Nicosan in Nigeria, Africa says “Though NICOSAN/HEMOXIN does not cure Sickle Cell Disease (SCD), the medicine greatly reduce the degree of "sickling" of the affected red blood cells, which in turn eliminates or greatly reduces the devastating secondary consequences most patients succumb to, including strokes, kidney and liver failure, and extremely painful episodes known as "crises."

The problem with this drug is complicated.

It’s not FDA approved. Now, to get a drug tested and approved by the FDA takes loads of red tape (aka requirements). These red tape requirements could take a drug many, many years to get approved for use(in the US). This does not mean that a drug is not good or that it doesn’t work (look at Chinese medicines that are not FDA approved…where was the FDA in the first century anyway????)…..get it….the FDA is an American drug law/rule/red tape. But somehow, it makes me feel safer to take a drug that’s approved, and tested, with side affects known.

In addition, XECHEM (the company that produces Nicosan in Nigeria), said in a report to the SEC, “There is a lack of data to document the influence of raw materials (i.e. plant material quality, age, time of harvest, location, soil quality, preparation, handling, etc.) on the production of NICOSAN/ HEMOXIN.”
I have a problem with this lack of “data”.

Also, I’m not sure about the future of Nicosan because, according to a report to the Securities and Exchange Commission (SEC) by XECHEM, they are having severe financial troubles and may file bankruptcy.

So, in conclusion, I don’t know anything. All I know is that there are people suffering with Sickle Cell Disease, like me, and I would love to take a pill and send it far, far away.

Would You Participate in Clinical Trials?

According to the Washington Post, May 29,2008, Johns Hopkins Institute researchers developed a human stem cell line containing the mutation associated with sickle cell anemia.

They say, "One challenge to studying blood diseases like sickle cell anemia is that blood stem cells can't be kept alive for very long in the lab, so researchers need to keep returning to patients for more cells to study."

Linzhao Cheng, an associate professor of gynecology and obstetrics, medicine and oncology, and a member of the Johns Hopkins Institute for Cell Engineering said, "Having these new cell lins available might enable some bigger projects, like screening for potential drugs."

Would you get involved in clinical trials if you thought it could help find a cure for Sickle Cell Disease?
Check out the link http://www.clinicaltrials.gov/.

There may be something you could do to help researchers.