Monday, March 10, 2008

Needles and Such

Not long after our boys came home, we learned that Jellybean (age 5) has a form of Sickle Cell Disease. Before we got the boys in to see our pediatrician I knew there was something going on with him...something just wasn't 'right' with him, but I couldn't put my finger on it. So when the doctor called and asked if we could make an appointment to come in to talk to her about test results, I wasn't terribly surprised. But I wasn't necessarily expecting to hear that his issue was Sickle Cell. I didn't know much of anything about sickle cell. I knew how it is inherited, because I've taken classes in genetics and sickle cell is always used as an example of genetic inheritance in textbooks; I knew that people with sickle cell (or people who are carriers) are less susceptible to malaria...but I had no idea what having sickle cell would be like.

I know that some of you are thinking "Didn't you know before they got home that he had sickle cell? Isn't this something they would have tested him for?" And the answer typically is that yes, children are tested for this before they are referred to a family for adoption....and that means that typically this is something that would be known ahead of time. But, for one reason or another it was missed. Perhaps his blood sample was never actually taken (this poor boy screams at the top of his lungs when a needle is in sight); or perhaps his sample wasn't actually tested; or perhaps his sample was tested incorrectly; or the results were interpreted incorrectly by the doctor who looked at them. It doesn't really matter - it is what it is. And the truth is, I know that if we knew he had sickle cell it wouldn't have made a bit of difference anyway. He still would be our son and we wouldn't have thought twice about it. The only difference would have been that we could have prepared ourselves for it a bit more by reading about sickle cell and contacting doctors in our area who know more about it. In some ways, though, I am glad that I didn't know. If I had known about it earlier, I would have worried MUCH more about him and would have gotten far more impatient than I already was to get him home. And many of my worries would have been warrantless. After all - we were already working as fast as possible to get them home - worrying more wouldn't have made a bit of difference.

Am I upset that our agency didn't catch this? No...without a second Adoption in Ghana is still new; our agency is still in the pilot stages and they are doing GREAT things. Our boys were two of the very first children to come into their care. The in-country staff was still learning about what clinic to visit; what doctors to see for the children in their care (they have since switched to a different clinic). No child ever comes with a guarantee (even birth children). Z and I would take the risk again in a heartbeat....and we'd use the same agency all over again to do it. In fact, we hope to start the process again in a couple years or so (but don't tell anyone just yet). ;)

On Friday we had our first specialist visit with a doctor at the University of Iowa Children's Hospital. The PA and the doctor we saw were both great. They explained everything in detail, told us what to watch out for, how to deal with things, and they answered all of our questions. They told us we will have to be pro-active with his health; we'll have to educate our local doctors on how to treat him (and they told us our local doctors will probably not take these "lessons" very well - but that we'll have to be persistant and insist they do certain things). They also gave us some websites to check out as well as some pamphlets and a book to read. They are now going to refer us to a doctor who practices at a children's hospital located closer to our home town, so that future specialist visits will not require a 2 1/2 hour trip.

I am still coming to terms with what this means for my little boy. I'm learning about what this means for his everyday life and for his life in the long-term. Some of what I'm learning is scary...and all of it makes me sad for him. I know this means that his life will be a little bit harder....a little more painful...and perhaps a little shorter than the average person.

One thing that I do know is that he is an incredibly strong and resilient little boy. The fact that he made it to age 5 in less than optimum conditions (and is so healthy) is proof of this. Now that he is here, he has access to emergency rooms at any time of the day/night that he might need them; he has access to antibiotics that he can take daily and immunizations that can help boost his immune system; he has access to specialists who can prepare his parents for what's ahead and how to deal with things; and he has access to pain medication when he needs it. For all of these things I am grateful.


jen said...

I am sorry about this news and what it means for Jellybean. You are handling it with the grace and strength of a mother. We should talk about it in our group as I know my little one has the trait and it was something I researched as it seems kind of common. I would be interested in hearing more about his symptoms and how you knew something was wrong. Thankfully you took him in and were able to figure this out so he can get the care he needs. I will keep you all in my prayers...

Anonymous said...

NICOSAN, FDA ORPHAN Drug for the Treatment of Sickle Cell Disease

There is a relatively new treatment for sickle cell being
produced in Nigeria by an American company called NICOSAN®,
it’s proprietary name is NIPRISAN® . It was developed on the
premise of traditional Nigerian plant based medicinal practices
for the treatment of sickle cell disease. Although this drug
was developed in Nigeria, the man behind it, Dr. Ramesh Pandey
is a distinguished biochemist who has worked for the
National Cancer Institute's (NCI) Frederick Cancer
Research Center as a Senior Scientist, Head of the
Chemistry Section, Abbott Pharmaceuticals and
produced the first commercially viable generic version
of Vancomycin for Lyphomed Inc., a Visiting Professor
at the Waksman Institute of Microbiology at Rutgers,
the State University of New Jersey, holds patents for
biotechnology analysis and rare drug production
processes. He also holds several US and international
patents for paclitaxel and its new analogs. He is a
member of the Editorial Board of the International
Journal of Antibiotics and of several professional
societies. He has been awarded several grants from
NASA, NCI and NIH. The drug NICOSAN has been granted
Orphan Drug Status by both the FDA and E.U..

It has been tested through phase IIb clinical trials and
found to be highly efficacious. Phase III trials have yet
to be completed however it was approved for sale in Nigeria
based on phase IIb trials and toxicity studies which showed
it to be safe and non-toxic.

Double-blind, placebo-controlled, randomised cross-over
clinical trial of NIPRISAN® in patients with Sickle Cell

Efficacy of Niprisan in the prophylactic management of
patients with sickle cell disease

NIPRISAN — Nix-0699 Toxicity Studies
Niprisan (Nix-0699) improves the survival rates of
transgenic sickle cell mice under acute severe hypoxic

NIPRISAN Case, Nigeria
A Report for GenBenefit (2007)

This drug is a major advancement in the treatment of sickle
cell disease unfortunately it is not available in the U.S..
Although the compound has been granted orphan drug status
by the FDA and the regulatory body of the European Union,
to date investigational drug applications for the approval
process have yet to be submitted. Getting a drug approved
in either area is extremely expensive. Until there is
funding available to proceed with the FDA and EU
applications it will be difficult for non-Nigerians to
obtain the drug.

I do say difficult but it is not impossible. If you have a
hematologist or hemoncologist who is willing to put fourth
the effort there are special dispensations available
through the FDA for the importation of unapproved drugs on
a compassionate use basis.

“Expanded access program (EAP). EAPs are typically designed
to provide widespread access to a drug that has proven
efficacy in clinical trials but is still awaiting FDA
approval. They’re similar to standard clinical trials with
a specific treatment plan and certain FDA requirements, but
they have looser patient eligibility criteria. More than
23,000 U.S. cancer patients enrolled in an EAP for Iressa
before it was FDA-approved, for example.”

“Single patient use. This program offers an experimental
drug to an individual patient, rather than a group. The FDA
approves these uses on a case-by-case basis. Decisions are
based on other treatments already available and information
about the drug’s efficacy and potential toxicities.”

To date I have no knowledge that anyone has sought any
single use or expanded access from the FDA for Nicosan.
Unfortunately regardless of the dissemination of this
information thus far no one has put forth the effort to
obtain the drug for use.

If just one person would start the ball rolling with a
caring and concerned medical practitioner it could open up
the drug for wide spread use by tens of thousands of
patients across the U.S. Unfortunately thus far the general
response I receive is that people don’t believe that their
physician would be interested in going to this sort of
effort nor do they themselves seem to be inclined to seek
the use of a treatment that could potentially end their

There has to be at least one physician out there who has
enough care and concern for his patients to be willing to
put forth the effort necessary to obtain this medication
legally. I urge anyone who is effected by sickle cell to
approach their physicians with this information and attempt
to obtain this treatment not only for themselves but for
all patients who could potentially benefit from it’s use.
We already know the benefits of the treatments available in
the U.S. and the E.U.. In many cases they are only
marginally effective or in the case of hydroxyurea cause
side effects so serious that many choose not to use it as
treatment. Here we have an opportunity to use a treatment
that has been shown to be highly effective, eradicating
crises in the majority of patients and reducing crises by
50% in the most refractory cases.

Although the clinical trial group was what the casual
reader might interpret as quite small it is common for
drugs which fall into the orphan drug category to use small
sample groups. Many orphan drugs have been approved based
on very small phase II and phase IIb clinical trials in the
U.S. In the case of FDA fast track status, a drug may be
approved during phase II trials if the drug shows
significant advantage over current approved therapies for
life threatening illness.

Fast Track Designation is a program that, if granted, is
designed to facilitate the development and expedite the
review of new drugs, thereby allowing the FDA to approve
drugs used to treat a serious condition or a
life-threatening disease with less safety data following
the conclusion of phase II studies, rather than phase III,
the normal practice.

The main criterion for a Fast Track Designated drug is the
potential to treat a life-threatening illness or fill a
major unmet medical need. Fast Track may be submitted with
the IND or at any time during the clinical development of
the drug. The Fast Track designation may allow a company’s
application to follow Priority Review, Standard Review, or
a Rolling Review of the application.

Nicosan by Western standards is an extremely inexpensive
drug. It is available in Nigeria without prescription at
$23/month for adults and child doses at $18/month.
Here is a link to the company and product website.

I sincerely hope that you find this information helpful. I
would encourage you to to forward and post this information
to any person, blog or website where persons effected by
sickle cell anemia can have access to this information.
Feel free to write me with any questions you may have.

Anonymous said...

United Nations Economic Commission For Africa

Book Of Abstracts

Science With Africa Conference

March 3-7, 2008

page 30

Evaluation of Niprisan (Herbal Medicine) for the Management of Sickle Cell

Charles Wambebe and Hadiza Khamofu, International Biomedical Research in Africa, Abuja,
Nigeria,, Joseph Okogun, Nathan Nasipuri and Karynius Gamaniel,
National Institute for Pharmaceutical Research and Development, Abuja, Nigeria.

About 70% of all sickle cell anemia (SCA) subjects reside in Africa, estimated at over 12 million. The prevalence of SCA is estimated at over 2% while infant mortality is about 8% and survival rate of SCA babies in rural areas by five years of age is about 20%. These statistics indicate that SCA is probably the most neglected (and sometimes forgotten by health authorities) serious public health disorder with serious mortality and morbidity rates in Africa. The objective was to undertake pre-clinical and clinical assessments of a herbal extract vis-à-vis management of sickle cell anemia using Good Laboratory Practice and Good Clinical Practice principles respectively. In Africa, there is no standard treatment for sickle cell anemia, only palliative management is generally available. In view of this situation, most SCA subjects use herbal medicines. NIPRISAN is a standardized extract from four medicinal/food plants: Piper guineenses seeds, Pterocarpus osun stem, Eugenia caryophyllum fruit and Sorghum bicolor leaves. Short term toxicity study indicated that NIPRISAN was safe in laboratory animals. Bio-activity guided fractionation show that vanillin and aromatic aldehydes may be the bioactive moieties. NIPRISAN reversed sickled red blood cells and protected them from being sickled when exposed to low oxygen tension. NIPRISAN dose- dependently delayed polymer formation of haemoglobin S. NIPRISAN induced 85% increased solubility of deoxy haemoglobin S. The in vivo efficacy study was undertaken at Children Hospital of Philadelphia, USA. Histological examination of lungs of control Tg transgenic mice carrying human sickle haemoglobin showed entrapment of massive numbers of sickled cells in alveolar capillaries. NIPRISAN significantly cleared the lungs of sickled cells. Furthermore, NIPRISAN induced profound effect on the survival time of Tg mice under hypoxic conditions (p<0.0001). The phase II clinical data indicated that all the subjects benefited from NIPRISAN with no serious adverse effect. About 80% of the subjects did not experience any crisis during the study (12 months). The subjects experienced significant reduction in hospital admission while attendance at school profoundly increased. Furthermore, there was no evidence of kidney or liver damage. NIPRISAN has been patented, licensed to an American company, registered and being manufactured at Abuja for
global market.

Leslie said...

That is wonderful that Jellybean is getting the care he needs--I am praying for God to make all grace abound to you and to him.