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Wednesday, January 20, 2010 12:00:48 PM
http://addiandcassi.com/family-of-twins-with-rare-and-fatal-cholesterol-disease-to-seek-fda-approval-to-deliver-cyclodextrin-into-the-brain/
Family of Twins with Rare and Fatal Cholesterol Disease To Seek FDA Approval To Deliver Cyclodextrin Into the Brain
January 15, 2010 by Chris Hempel
The tagline of the new movie, Extraordinary Measures, about John Crowley’s fight to save his children from the rare and fatal Pompe disease is….Don’t Hope For a Miracle – Make One!
We are now working on making our own miracle – a new compassionate use investigational new drug (IND) protocol and application that we plan to submit to the FDA by April 2010. We are seeking to add intrathecal and/or intraventricular cyclodextrin delivery into Addi and Cassi’s brains to treat Niemann Pick Type C disease.
We realize this sounds radical to some people but we believe that based on the data and the twins’ cyclodextrin treatment throughout 2009, we can show the FDA that doctors can administer cyclodextrin into the brain safely and that the treatment could be life-saving.
Our efforts to alter our treatment protocol from bloodstream to brain may create some amount of debate. We are prepared to fight the next set of roadblocks in our efforts to save Addi and Cassi and other children suffering from this fatal cholesterol disorder.
Addi and Cassi have been receiving cyclodextrin intravenous treatment (IV into bloodstream) for the past nine months. In September 2009, we stopped our weekly dose escalations and remain constant at a dose of 2500mg/kg, 8 hour treatments two times per week via IV.
Since putting cyclodextrin into the brain in humans has never been attempted before, it’s going to take a monumental effort to pull together the IND application. We remain hopeful that with the world-class team of experts we are assembling to write the protocol, we can administer cyclodextrin safely into Addi and Cassi’s brains.
Research funded by The Addi and Cassi Fund and Hadley Hope Fund with Dr. David Begley of Kings College of London has shown that cyclodextrin does not cross the blood brain barrier (which was under debate). However, Dr. Begley has found that cyclodextrin does not cross it may bind to the blood brain barrier which could be a key clue into how it is working. Dr. Begley will be presenting data at the Lysosomal Disease Network World Conference being held in Miami Feb 10-12, 2010.
The bottom line is that cyclodextrin is not getting into the brain. However, cyclodextrin’s ability to possibly bind to the blood brain barrier or it’s impact in teh bloodstream may be causing the positive effects we are seeing in pur twins such as:
Reduction in the size of their lysosomes
Reduction of plasma oxysterols which appear to be a new biomarker for Niemann Pick Type C disease
Improved balance and stability, less ataxia and better head control
Improved mood and stronger emotions (happier, smiling and laughing more)
Much more affectionate, better eye contact
Increased appetite (better swallowing and chewing with Cassi in particular!)
More overall awareness to their surroundings, less “flat” and distant
Making more sounds and more volume to sounds
Increased concentration and ability to sit for longer periods
Not as floppy and jello-like, seem to have more muscle strength
Following directions better, seem to understand more what we’re saying even if they can’t talk
Improved general health status
We do not see any visible outward side effects – no vomiting, hair loss, skin rashes, dizziness – nothing. The most concerning and serious side effect with cyclodextrin appears to be pulmonary issues based on the animal studies conducted on NPC cats and mice. Apparently, in cats and mice they see increase macrophage development in the lungs but researchers do not know why.
We are keeping a close eye on Addi and Cassi’s pulmonary function. Last month, the twins underwent bronchoscopes at Children’s Hospital Oakland to look inside their lungs. I have posted the lung procedures on Vimeo for anyone to view (Addi – Cassi).
According to our pulmonologist, the girls have slight scattered nodules in their lungs. Doctors believe these nodules may be xanthomas. Xanthomas can occur in people with high blood lipids but usually appear on the elbows, joints, tendons, knees, hands, feet or buttocks.
Xanthomas are not a common feature of Niemann Pick Type C disease, however, there has been a case of suspected xanthomas with a child with NPC mutations. If the nodules are xanthomas, they are either as result of Addi and Cassi’s rare and private mutations or from the cyclodextrin IV treatment. Since the girls are identical twins, the only logical conclusion is genetic or cyclodextrin. Interestingly, researchers do not see xanthoma development in the lungs of cyclodextrin treated mice or cats.
In addition, we had CT scans done on Addi and Cassi’s spleens and livers and there was no change in size. We are told by experts that this does not mean that the cyclodextrin is not benefiting their organs. On the other hand, we may not be achieving the right concentrations of cyclodextrin in the plasma. We are still trying to develop the pharmacokinetics around cyclodextrin. They don’t exist and we have to create everything from scratch.
We have been in contact with top metabolic doctors in Japan and they are treating little girl with cyclodextrin. The good news is they have seen a reduction in the child’s liver and spleen size. Her liver and spleen size were 4-5cm and 7cm below the coastal margin before cyclodextrin treatment and today are 1cm and 3-4cm below the coastal margin. She showed some improvement on her EEG and has not had any adverse effects with the IV treatment. However, she is progressed and they are not seeing major neurological benefits.
It is clear that we must design further treatment options with cyclodextrin to try and improve neurological function. This means delivering cyclodextrin into the brain through the intrethecal and/or intraventricular route of administration. We will never know if cyclodextrin can save our girls unless we try.
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