Monday, October 10, 2016

Demystifying The Cure! by Marie Ojiambo

I am super excited to feature my very first guest blogger, my very dear sister warrior Marie Ojiambo on this page. She is also a Sickle Cell warrior and a global advocate for sickle cell disease awareness. She was diagnosed at the age of 1 and has since been living with the condition.
In the year 2013, she founded the Sickle Strong Initiative (SSI) a not for profit whose mandate is to raise awareness around sickle cell disease and to advocate for better healthcare opportunities for patients suffering the condition in Kenya.
Trained at the University of Nairobi, School of Pharmacy, Marie Ojiambo is a Pharmacist by profession. Marie recently graduated with her Master’s degree in Industrial and Physical Pharmacy from St. John’s University, College of Pharmacy and Health Sciences in Queens, New York. She is specializing in drug research and development, with a focus in formulation sciences.
Through SSI, Marie has been able to host medical training and awareness campaigns for patients suffering the disease in Kenya. She is also the proprietor of the annual sickle cell public awareness forum, Ongea (which means speak out in Swahili). It is a platform that gives sickle cell warriors an opportunity to speak out about their condition, share and interact with each other. It also provides an opportunity for warriors to engage with and challenge the government, the pharmaceutical industry, insurance companies, other stakeholders and the public on what they are doing to improve the health care and environment that these patients are exposed to. Her initiative hosted the third edition of Ongea on the 18th of June this year that brought together 300 warriors and their families. 
Marie is also former Miss Kenya USA (2013), the People’s Princess Miss Africa USA (2014), first runners up and Face of Kenya USA (2016) platforms that she has used to raise public awareness on the disease.

 Demystifying The Cure!

After over a century since the discovery of sickle cell disease, the only available, FDA approved and marketed medication for the treatment of Sickle Cell Disease is Hydroxyurea commonly referred to as Hydrea. Hydroxyurea whose primary indication or primary use is for the treatment of myeloproliferative disorders (cancers of the blood) was found to increase the levels of fetal hemoglobin in patients who took it.

Red blood cells containing Fetal hemoglobin or as I like to call it ‘good hemoglobin’ cannot enter the deoxy sickle cycle that cells containing abnormal sickle cell hemoglobin undergo when subjected to oxidative stress. What this simply means is that cells containing fetal hemoglobin do not sickle. Fetal hemoglobin induction has been the preferred treatment modality for sickle cell disease. Hydroxyurea, a fetal hemoglobin inducing agent went through a fast tracked clinical trial when it was discovered that the drug could be used in the management of this otherwise life threatening orphan disease. Sickle Cell is classified as an orphan disease in the US sighting that the financial incentive for the research and development of drugs used to treat this disease is not significant as the disease isn’t prevalent in this country. Given the current statistics, this statement is easily arguable.
Hydroxyurea has been grossly underused and/or misused by the patient population suffering sickle cell disease. Maybe because of the lack of knowledge on its benefits but I’d say mostly because of the misinformation and negative connotations associated with the side effects that manifest from patient to patient when one uses the drug. In some countries, the lack of access to the drug and unaffordability are other causes for its underuse.  

So with only one FDA approved treatment option, which may present with moderate to severe side effects coupled with a lack of information on the benefits of the drug, diminished accessibility, unaffordability and a plethora of other issues, patients suffering sickle cell disease have reached a dangerous level of desperation and need for a different and/or better treatment option, better yet… for a cure.

The Internet and patients within the sickle cell disease space have been buzzing with excitement over the news of a cure for the disease as reported by various articles. I remember this one particular headline that read ‘Relief as doctors find a permanent cure for sickle cell disease’ that threw everyone into a state of uninformed euphoria. Many friends slipped inbox messages to me through my social media channels to inform me of this new ‘light’ at the end of the tunnel. Fortunately, due to my undying curiosity and thirst for updates on current research efforts and medical breakthroughs within the sickle cell disease space, I was well informed on this procedure even before it made news.
The skeptic in me decided to read through and understand what this cure was all about, how the medical intervention was performed and what it meant in terms of follow up, treatment, financial burden as well as its impact on quality of life before joining in the somersault fest that most seemed to be undertaking. 


The new treatment option, a haploidentical bone marrow transplant procedure, also commonly referred to as a half matched bone marrow transplant was the talk of hematology. A procedure developed at John Hopkins, was now the hope of many.

In this particular procedure, rather than totally wiping out a patients immune system before transplanting a donors bone marrow, a process done for all transplant procedures, doctors administer a lower dose of immunosuppressive drugs. Just enough to prevent rejection of the donors bone marrow by the recipient’s immune system. As a result of using lower doses of immunosuppressive agents, the side effects are reported to be much milder thus greatly reducing the time patients spend in hospital and recovery. Normally, doctors look for a donor who matches a patient's tissue type, specifically their human leukocyte antigen (HLA) tissue type. HLA’s are proteins or markers found on most cells in the body. The immune system uses these markers to recognize cells that belong in the body versus those that are foreign. The closer the match between a patient's HLA markers and the donor’s, the higher the probability of a successful transplant. In most cases of sickle cell disease, for example, doctors looked for a nearly full match prior to bone marrow transplantation. This was extremely difficult because in many cases, the person with the closest match, such as a sibling, may also have carried the sickle cell gene. The Hopkins procedure requires just a half-match, meaning that a patient’s parents or children could be suitable donors. With this option, doctors estimate that more than half of sickle cell patients, and nearly all patients with other blood cancers or autoimmune disorders, have potential matches.

The procedure thus greatly increases the potential donor pool, an issue that most transplant recipient’s face, which delays their treatment. The increase in the number of potential donors means a decrease in the number of patients excluded for this treatment. A treatment option primarily designed yet again for myeloproliferative disorders or cancers of the blood found a niche in sickle cell disease.

I talked to a parent who had his sickle cell warrior go through the traditional bone marrow transplant procedure. Yes, his son is now completely cured and leads a normal, healthy life. This parent however abhorred the transplant process. He said it was painful to watch his child suffer during the recovery period when he had to be on chemotherapy to keep his body from rejecting the donor’s bone marrow. The total recovery period he said was 2 years. Luckily, the financial obligations were covered by the teaching hospital at which his son underwent the transplant procedure. His advice to me was that and I quote ‘if you can manage the pain of sickle cell, you’d rather find a way to cope with having the disease as compared to going through a bone marrow transplant’. From the look on his face I could tell that the road to recovery for his dear boy and himself as a parent was not an easy one and may have left pain and an emotional scar that only time would somehow heal.

So the half match bone marrow transplant procedure is now highly researched and currently in clinical use.

I had the privilege of talking to a patient within my networks who has actually undergone the Haploidentical bone marrow transplant procedure. Though his first trial with the procedure wasn’t successful, he said he’d go through it again and again until he is sickle cell free. His hope in this new procedure was so infectious though his account seemed extremely challenging and grueling even for me who was only reading what he had to go through. I share his account in my next post.
Stay informed, Stay positive!
To be continued...

2 comments:

  1. Am Alex from USA,
    There is a cure for Sickle Cell Disease,
    My Son once had sickle cell Disease he was always sick every month, and his growth was also very slow. he was very intelligent in school and all his teachers loves him for that. when ever he is sick, I feel the whole world is falling on me.
    I got the permanent cure from Dr Alegbe, for my son. He took the medication for good one month, and from then still now he does not get any fever again. and his body system is strong. he does not get sick easily. Dr Steve has really made me happy in life again. and I promise to tell the whole world about him. I never believed my Son can live long to this extent, but now his is living fine and healthy like the other kids. Now I believed Sickle cell Disease can be cured. any one with such issues can contact the Doctor on dralegbe@gmail.com or Whats-app him on +1(518)-675-6082

    ReplyDelete
  2. THERE IS A CURE TO SICKLE CELL ANEMIA, AM A LIVING TESTIMONY.

    I write to you with great joy in my heart how Dr Alegbe John turned my life around. I was born a sickle cell patient through the gene- type of my parents and became a carrier which led to immense crises all through my growing up years. This particular ailment was called rheumatism, the pain of the bone which I was told was caused by difficult circulation of blood in the vein axis.

    Growing up was like hell because apart from the pain and the fear of it, I also lived in bondage for years before Dr Alegbe intervention.

    I was restricted from doing what my mates could do, there was a lot of don’t touch, don’t eat, don’t go, don’t wear by the doctor and I lived all through this period on drugs. I was made to know that without this pill, my life will would finally come to an end. It was total bondage.

    During this period of pain, I would cry, shout, throw myself to the ground, destroy things I could find around me just because of restlessness the pain caused. My Dad got the contact of Dr Alegbe from the internet and he emailed him told him about me and made purchase from his product. I too the medication for one month and he always call to know how am feeling, that was how i was cured completely. any one can reach him On his gmail address at dralegbe@gmail.com

    I stopped taking my drugs because Dr Alegbe John has made me completely cured, I no longer feel pain neither do I remember how it feels.

    ReplyDelete

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