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CureCaps Hat Project Makes First Medical Research Grants To Leading Alzheimer’s and Athersclerosis Researchers

July 25, 2011 by  
Filed under Featured Stories

Maike and Shelly of Johnson & Johnson sell hats to raise money for CureCaps project

Soon after Addi and Cassi were diagnosed with a fatal genetic cholesterol disease called Niemann Pick Type C disease, we organized a grassroots effort to collect “CureCaps” from volunteers around the world and sell them to help raise money for research into brain diseases that impact millions of people worldwide.

We want to report to our CureCaps supporters that we have made our first medical research grants of $15,000 to two world-renowned Swedish researchers, who are working in collaboration to study neurodegenerative disorders like Niemann Pick Type C.

CureCaps Research Award Recipient #1:  Dr. Kaj Blennow, University of Gothenburg, Gothenburg, Sweden

The first researcher to receive a CureCaps grant of $7,500 is Dr. Kaj Blennow, University of Gothenburg, Gothenburg, Sweden. Dr. Blennow is one of the leading Alzheimer’s researchers in the world who has spent years working to find biomarkers for Alzheimer’s disease and other neurodegenerative diseases.  Currently, there are no blood or routine clinical tests (“biomarkers”) to detect if someone has Alzheimer’s, Niemann Pick Type C disease, etc.  You can’t treat a disease if you have no way to detect it or track it over time. Dr. Blennow is developing reliable tools for diagnosis, prognosis and monitoring of therapy through the analysis of cerebrospinal fluid (CSF) biomarkers such as A-Beta and Tau.

  • “In the field of brain disorders, the development of good biomarkers can enhance the possibilities for early diagnostics and the measurement of treatment effects. In the future, this could enable clinicians to diagnose and treat Alzheimer’s disease before the person experiences any symptoms” – Dr. Kaj Blennow

Dr. Blennow and his team have taken CSF samples from Addi and Cassi and other children afflicted with Niemann Pick Type C and analyzed them for ‘biomarker signatures’ including Tau and A-Beta levels.  What they discovered in Niemann Pick Type C children is that amyloid metabolism is highly disturbed just like in Alzheimer’s disease, creating an even stronger correlation between the two neurological diseases.  Dr. Blennow and his team are looking for further correlations between the two neurological diseases and are exploring how a compound called cyclodextrin (we are treating the twins with this!) may alter amyloid metabolism.

CureCaps Research Award Recipient #2:  Dr. Ingemar Bjorkhem, Professor emeritus of Biochemical Arteriosclerosis, Karolinska Institute, Stockholm, Sweden

The second researcher to receive a grant of $7,500 is Dr. Ingemar Bjorkhem, Professor emeritus of Biochemical Arteriosclerosis, at the Karolinska Institute.  The Karolinska Institute is a medical university located in Stockholm, Sweden, and is one of Europe’s largest medical universities. It is well known because the Institute appoints the laureates for the Nobel Prize in Physiology or Medicine.

Dr. Bjorkhem studies cholesterol homeostasis in the human body.  Most people do not know this, but approximately 25% of the total amount of the cholesterol present in humans is localized to brain, most of it present in myelin. Cholesterol levels and cholesterol turnover are affected in many neurodegenerating disorders, and the capacity for cholesterol transport and recycling in the brain seems to be of importance for the development of these neurodegenerative diseases such as Niemann Pick Type C.

Dr. Bjorkhem is well known for his groundbreaking work on oxysterols, which are oxidized derivatives of cholesterol. Oxysterols are important in many biological processes, including cholesterol homeostasis, sphingolipid metabolism, platelet aggregation and apoptosis. Dr. Bjorkhem believes he has found a new type of oxysterol circulating in Addi and Cassi bloodstream.  This oxysterol is potentially related to iron metabolism so he currently looking into oxysterols in patients with other disturbances in iron metabolism (hemochromatosis).  Addi and Cassi’s may be getting iron overload and this may be a key clue not only for NPC disease, but other neurodegenerative diseases as well.

“Oxysterols are oxidized metabolites of cholesterol. These steroids are formed both enzymatically and non-enzymatically in all cells and have been ascribed a number of regulatory effects. They have been suggested to play a role in connection with atherosclerosis and neurological diseases. During the last three decades our laboratory has been involved in research on the role of the oxysterols in connection with the above diseases and we have developed a number of sensitive and accurate methods for identification and quantification of oxysterols.

Thank you to all volunteers who make and sell these hats.  While these are small grants, they are making a big impact and there is a huge return on investment.

We are making a difference not only in the lives of kids like Addi and Cassi, but millions of others too!  Winter is coming and we’re hoping to sell a lot more of the hats we have in stock!  Please keep them coming!

Procter & Gamble’s Febreze Product May Contain Active Drug Compound That Can Enter Human Cell

July 19, 2011 by  
Filed under Featured Stories

Something does not smell right over at Procter & Gamble. For more than a year, I have been trying to reach the proper people at the company  (ie. Sr. Execs) in order to get them to pay attention to the fact that one of their key products has a potential drug in it that millions of people are inhaling.

The potential drug is called hydroxy propyl beta cyclodextrin (HPBCD) and this compound is used in many of Procter & Gamble household products, including their Febreze air freshener.  You can read about how P&G uses HPBCD in Febreze on their website.  HPBCD is the active ingredient which helps Febreze do its odor reduction magic!

What the top people at P&G may or may not know is that it has recently been discovered that cyclodextrins have an affinity for cholesterol, especially forms of HPBCD.  For decades, HPBCD was thought to be an inactive and non toxic ingredient but in fact HPBCD may act as very powerful drug in the human body.  When cyclodextrin enters into the body, researchers believe it has the ability to penetrate into the lysosome of a human cell and interact with cholesterol and potentially other things as well.

I am currently trying to save my twins’ lives with HPBCD as they are afflicted with one of the worst cholesterol diseases on the planet — it’s called Niemann Pick Type C and the condition is often referred to as “childhood Alzheimer’s.”  I have been working on trying to figure out how make an HPBCD aerosol that the twins can breath in — essentially a Febreze without all the scents.  While we currently have FDA approval to give Addi and Cassi intravenous and intrathecal treatments of HPBCD, the compound does not appear to penetrate the lung through these routes of administration.  So I need to look into making an aerosol to reach the lung.

I contacted Procter & Gamble when I learned about HPBCD being in their product line.  I was hoping they could help me with some data on HPBCD, but more importantly, I wanted to alert them of the fact that HPBCD is a potential active drug and I believed further studies on HPBCD needed to be carried out.

What really stinks is that Procter and Gamble ignores me regarding this issue with HPBCD being a potentially active drug  — it’s truly an unbelievable situation.  The product manager I was routed to last year who is apparently responsible for the Febreze product line does not respond to me nor do their top PR people who I have contacted numerous times.  Maybe the lawyers are involved at this point?  I don’t know ….. but something about them ignoring me stinks to high heaven.  The whole nightmare with them completley blowing me off for months has sent me completley over the edge.

Two weeks ago, I forwarded them patent information on HPBCD as a new therapy to threat asthma and COPD — essentially researchers are looking to create an HPBCD that can be inhaled through the nose and mouth to treat these lung conditions  — just what I am looking for!   No response.

I do not think HPBCD is harmful.  It’s a non toxic sugar compound and it has a great safety profile.  Obviously, we are putting the compound into my twins’ brains to try and save their lives and it could be used to treat lung diseases.  But given that HPBCD is a potential active drug, is seems like a fair request to ask Procter & Gamble to take what I am saying seriously given millions of people are inhaling Febreze (even if it is in very small amounts or it’s a small exposure).

If Febreze is going through your nose, it’s potentially getting into your bloodstream and could even cross the blood-brain barrier through the nasal passage.  People have a right to know that they could be breathing in a drug that could interact with their cholesterol, even if it is completely safe.  Surely more studies need to be conducted now that we have new information that cyclodextrin is a potential active pharmaceutical ingredient (API).

I contacted the FDA about this issue since HPBCD is a potential drug but I was told by FDA that this is an issue for the Consumer Products Safety Commission (CPSC).  I am planning on contacting Inez Tenenbaum who is the Chairman of the Commission because it’s not right that a company like P&G can simply ignore something like this.  Certainly a company should report whether they have been notified that their product’s main ingredient is a potential drug that could get into a person’s cells.

What is the public’s right to know in a situation like this?  I don’t know the answer but I am going to sniff it out.






Mother of All Lies – Casey Anthony. RIP Caylee. We won’t forget you, Little Sweetheart.

July 5, 2011 by  
Filed under Featured Content

Note: I normally don’t post anything on Addi and Cassi’s website that is not about their disease or something that is medical related. Hugh would say to leave it to Facebook or Tweet about it.

But the case with Casey Anthony and her little daughter Caylee has captured my attention….and my heart.

When you’re in so much pain about losing your own kids to a terrible disease, you can’t imagine a mother suffocating an innocent child and discarding her in a swamp to be eaten by animals.

Peter Gelzinis of the Boston Herald said it all for me!  His commentary is below.

Jury swayed by
mother of all lies

The O.J. Simpson case introduced us to the idea of jury nullification, but it took a 25-year-old bar-hopping party girl, bored by motherhood, to refine the concept.

Yesterday, a Florida jury acquitted Casey Anthony on all three counts of murdering her 2-year-old daughter, Caylee.

Then, this very same jury went on to return four guilty counts of lying to police about . . . that’s right, the murder of her daughter.

Shame on this jury for denying the obvious.

If the O.J. debacle proved anything, it was that the chances of killing your wife and beating the rap are better in California.

Likewise, what we take away from this sad, slimy soap opera in the Sunshine State is that a bimbo looking to detach herself from the burdens of her 2-year-old daughter has a good chance of getting away with murder in Florida.

All you needed to know about the absurdity of yesterday’s verdict was uttered by Casey’s victorious defense lawyer, Jose Baez, who reminded the public that he had never denied his client was a liar.

Quite a victory declaration.

Ah, but then what else could this lawyer say? Even though Casey Anthony was the last person to see her daughter alive, she spent a month denying Caylee’s disappearance to everyone, including her own parents.

Even when the trunk of her car reeked with the stench of death left by a decomposing body, Casey kept lying. She couldn’t even bring herself to conduct a sincere vigil. While the cops searched by day, Casey was hitting the bar scene at night.

But as this trial unfolded, it was clear that such damning “circumstantial evidence” was dwarfed by the suntanned dysfunction of a family who were all more than somewhat wacky.

O.J. was helped tremendously by the behavior of a white LAPD detective branded a racist.

To plug the huge holes in Casey Anthony’s story, she and her lawyers put her father on trial. All of this woman’s malignant deficiencies and narcissism could be explained by the alleged molestation she suffered at the hands of her father, a former homicide detective.

Casey’s lawyers tried to pin Caylee’s death on Dad, suggesting he was the one who put the duct tape over her mouth after she drowned in the family’s pool.

Apparently, that seemed to qualify as reasonable doubt in this jury’s collective consciousness.

And yet they weren’t troubled by a young mother who gets the two-word credo “belle vita,” or beautiful life, tattooed on her body while her dead child is rotting away in the woods.

The old saying is that it only takes one to hang a jury. (You just know Sal DiMasi was on his knees lighting vigil candles for that one dissenting juror.)

It takes 12 knuckleheads to nullify a jury. And yesterday, those 12 people managed to say that, yes, Casey Anthony lied about what happened to her child. But no, she had nothing to do with Caylee’s horrible death.

It’s a verdict that simply does not make any sense. It’s a verdict that is, in itself, a lie.

Perhaps the only sorry truth to emerge from this hellacious modern-family fable is that Caylee Anthony was probably doomed by the accident of her birth into such a heinous household … and then further betrayed by 12 strangers who were unable to see the truth before their eyes.

FDA Filing Made Requesting Use of Medtronic SynchroMed Pump To Deliver Cyclodextrin To Brain

April 13, 2011 by  
Filed under Featured Stories

To say I am pumped up  today is an understatement!  We’ve reached another historic milestone in our effort to treat Addi and Cassi with cyclodextrin!  Dr. Caroline Hastings at Children’s Hospital Research Center Oakland filed a 400+ page document with the FDA yesterday on behalf of the twins seeking a permanent solution to deliver hydroxyl-propyl-beta-cyclodextrin (HPBCD) past the blood-brain barrier and into the cerebrospinal fluid (CSF) by utilizing a Medtronic SynchroMed® II Drug Infusion Pump System.

We believe that combination therapy of intravenous (IV) and intrathecal (IT) HPBCD is the most effective way to target both the brain (IT) and organs/peripheral tissues (IV) in Niemann Pick Type C disease.

The FDA has been very helpful throughout this process but the FDA filings are incredibly time consuming and take months and months of work.  The amount of detail necessary for a new drug treatment protocol is mind blowing — even if it’s for two children with an ultra rare disease.  The reason is everything we do has implications for all other children with Niemann Pick Type C, not to mention other diseases where cyclodextrin may work.  I think it could work in the ‘Lorenzo’s Oil’ disease called adrenoleukodystrophy.  Check out this paper where cyclodextrin is having an effect in ALD:  ADRENOLEUKODYSTROPHY Cyclodextrin Paper.  I hope  ALD families are making their researchers pump this into their mice models.  HPBCD is safe — if it has an effect on VLCFA, it could go into ALD kids as well with this pump idea!

We’re proposing to the FDA to use a new drug (hydroxyl-propyl-beta-cyclodextrin) in an existing device (a SynchroMed pump).  This requires two FDA divisions to be involved in the approval process — the Division of Gastroenterology Products (DGP) which is part of the Center for Drug Evaluation and Research (CDER) and Center for Devices and Radiological Health (CDRH), which is responsible for regulating firms selling medical devices in the United States.

I am sure anyone who works in the drug development field can appreciate the complexities involved when seeking FDA approval for a new drug in a device.  We don’t have time on our side and getting agreement from two FDA divisions is not easy.  But our plan is solid and makes sense and I am confident we’ll get the approval to move forward.

Once we get the Medtronic pump solution approved by FDA, Dr. Peter Sun, Chief of Pediatric Neurosurgery at Children’s Hospital & Research Center Oakland, will surgically implant a SynchroMed® II Drug Infusion System into Addi and Cassi.  We are proposing placing the catheter in the mid-thoracic region of the spine at the T6—T7 level rather than at the typical T11—T12 level.  Then HPBCD will be pulsed in bolus doses into the twins’ central nervous system so it can reach their brains.

To understand how this works, think about how a diabetic pump works.  Instead this pump is surgically implanted on inside of the body and is programmable from outside the body.  Generally, Synchromed pumps are used to deliver a drug called Baclofen used to control pain or spasticity.  In our case, we want to fill the pump with HPBCD monthly and deliver it just like you would Baclofen.

We have now completed 11  intratehcal injections of hydroxyl-propyl-beta-cyclodextrin into the twins’ spines via lumbar punctures. Intrathecal treatments are going very well but the travel from Nevada to California every other week is exhausting.  In addition, the sedations with propofol (yes, the one Michael Jackson used) take a toll on the twins. We really need a permanent solution and we believe SynchroMed is it!

Thanks to the amazing doctors and medical pros at Johnson & Johnson and Medtronic who have donated their time pro-bono to help us create this ground breaking treatment protocol.  The support we have received from people outside the Niemann Pick Type C community has been remarkable to say the least.

Now we wait to hear from the FDA to find out when we can schedule a teleconference call to discuss our proposal.



Insulin Therapy and Alzheimer’s – Could Cyclodextrin Sugar Act on Insulin and Glucose Metabolism?

April 10, 2011 by  
Filed under Uncategorized

Recently reports in the news shows how insulin therapy may be a treatment for Alzheimer’s.  Basically, a low dose of insulin has been found to suppress the expression in the blood of four precursor proteins involved in the pathogenesis of Alzheimer’s disease, according to research by University at Buffalo endocrinologists.  Paresh Dandona, MD, PhD, University at Buffalo is a senior author on the study.

I am very interested in insulin therapy for Addi and Cassi and I have been looking into this along with a drug called Rosiglitazone.  I wonder if I am already doing insulin therapy by giving the twins the sugar compound cyclodextrin into their bodies and brains?  I think I’ll email Paresh Dandona about it.

Addi and Cassi have major disruption in glucose metabolism in their brains. Dr. Harry Chugani and his team at Children’s Hospital of Michigan discovered this a few years ago in the twins and it is spreading out of control based on our last PET scan.  Dr. Chugani and team are publishing a paper about the glucose metabolism PET based brain biomarker in Niemann Pick Type C disease.

For over a year, I have been looking into whether or not hydroxy-propyl-beta-cyclodextrin (HPBCD) might have some sort of effect on insulin and glucose metabolism in the body and brain.

I found these very interesting references:

  • International Journal of Molecular Sciences (2009): The objective of this study was to elucidate the effects of hydroxylpropyl- B-cyclodextrin (HPBCD) on the in vitro stability of insulin. It was found that HPBCD had positive effects on the stability of insulin in acid and base and under high temperature conditions. Furthermore, use of HPBCD could also increase the stability of disulfide bonds which are important to the conformation of insulin. Through 1H-NMR experiments it was found that the protective effect of HBPCD was due to complexation with insulin. The results suggest that the presence of HPBCD could improve the stability of insulin in different environments.
  • Abulrob et.al. (J Neurochem. 2005 Mar;92(6):1477-86) demonstrated the neuroprotective activity of some cyclodextrin derivatives against oxygen-glucose deprivation (OGD), N-methyl-D-aspartic acid (NMDA) and glutamate in cortical neuronal cultures. Although all Cyclodextrin complexed with NMDA or glutamate, only beta-, methylated beta-and sulfated beta-CDs displayed neuroprotective activity and lowered cellular cholesterol.

I have been asking our researchers to see if they can conduct microPET on the Niemann Pick Type C mice to see if they can find a hypometabolism issue in the brains of the mice like they find in my identical twins.  If  found, researchers could give the NPC mice the cyclodextrin and test as to whether or not it has an effect on insulin and glucose metabolism in the brain.  If so, this could be a very important finding that could bolster the University at Buffalo’s work.

Also, one of the things I find really interesting is there is a rare disease called Ataxia – telangiectasia that I think provides further clues to neurodegeneration.   In Ataxia – telangiectasia, there are issues with glucose and insulin receptors: http://www.ncbi.nlm.nih.gov/pubmed/651946

Both Niemann Pick Type C and Ataxia – telangiectasia  there is massive loss of Purkinje cells in the brain — loss of Purkinje cells in one of the first signs in NPC disease.  Why the massive loss of Purkinje neurons in Niemann Pick Type C and Ataxia – telangiectasia?  What is the connection point causing loss of Purkinje neurons?  I think it might be glucose metabolism or insulin utilization.

Given the glucose metabolism issue we have found in Addi and Cassi’s brains and the ataxia which is one of the first symptoms of the disease, maybe glucose metabolism and insulin issues are a core issue in NPC?  I read that the cerebellum is largely fueled by glucose and the cerebellum is where Purkinje neurons are located!

Steve Walkley’s work shows that cyclodextrin rescues Purkinje neurons in the NPC mouse model  – could it be acting on glucose metabolism or insulin?  Could cyclodextrin rescue Purkinje cells in Ataxia – telangiectasia?

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