Tuesday, August 9, 2022

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.






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.



Cholesterol and Autism – Can Eggs In The Diet Save the Autistic Brain? Will SLOS Provide Clues To Autism?

April 2, 2011 by  
Filed under Featured Stories

Will it be the Mom’s and Dad’s with children with autism who will finally help educate the public on how serious cholesterol is when it comes to brain function in children?

This week researchers at The Ohio State University Medical Center announced they are studying whether a simple nutritional intervention – adding cholesterol to the diets of children with autism spectrum disorders after a test to see if they need it – can improve core autism symptoms. These studies are being led by Dr. L. Eugene Arnold, a child psychiatrist at Ohio State’s Nisonger Center who specializes in researching and treating autism.

According to the article, Arnold is teaming up with Dr. Elaine Tierney of Johns Hopkins University/Kennedy Krieger Institute and Dr. Forbes D. Porter of  the National Institutes of Health to conduct a phase I/II double-blind study for children ages 4-11 who have been diagnosed with autism spectrum disorders: autistic disorder, Asperger’s disorder, or pervasive developmental disorder – not otherwise specified (PDD-NOS).

60 children who are found to have abnormally low cholesterol will participate in a 12-week double-blind study in which they will be fed cholesterol …. or a placebo … to see in increasing dietary cholesterol has an impact on brain function.

I first heard about the role of cholesterol in autism from Dr. Porter a few years ago. Unfortunately, I was at the NIH for a week long visit with my identical twins, Addi and Cassi, who are suffering from a fatal brain disorder called Niemann Pick Type C.  Addi and Cassi have a genetic cholesterol defect on chromosome 18 and don’t process any cholesterol in their cells.  As a result this causes a “childhood Alzheimer’s” type of condition.  My twins appear to make cholesterol fine — but cholesterol gets stuck inside their brain cells, destroying their neurons, and causing this fatal condition.

During my visit, I was taking with Dr. Porter about dietary cholesterol concerns I had with the twins and that I put my twins on a low cholesterol diet.  Dr. Porter mentioned a genetic cholesterol condition called SLOS — or Smith-Lemli-Opitz syndrome. SLOS is a metabolic disorder caused by a genetic mutation in the DHCR7 (7-dehydrocholesterol reductase) gene on chromosome 11. This gene codes for an enzyme that is involved in the production of cholesterol.  Dr. Porter treats both NPC and SLOS kids and explained that SLOS kids have a high degree of autism and many SLOS kids with low levels of cholesterol.

I have heard from Dr. James Hildreth, a leading HIV-AIDS researcher I know,  that the expression of a key transcription factor could be disrupted in both NPC and Autism.  Dr. Hildreth has established a connection between Niemann Pick Type C and  HIV.  Basically cells affected by Niemann Pick Type C, which have the disrupted cholesterol trafficking I mentioned, were unable to release HIV, suggesting NPC cells would not spread the virus.  As it turns out, cholesterol is somehow essential to HIV because HIV relies on specialized structures known as lipid rafts, which are rich in cholesterol, to infect new cells.  Maybe this transcription factor is another key to the puzzle?

I hope this simple nutritional intervention of adding cholesterol into the diet has a positive effect on some of these kids’ brain function.  But if I was in the Autism Community, I wouldn’t be putting all my eggs in one basket just yet.

Novartis Phase 3 cancer drug Panobinostat may correct Niemann Pick Type C cholesterol defect through inhibiting histone deacetylase (HDAC)

March 21, 2011 by  
Filed under Featured Stories

I almost fell off my chair a few minutes ago. I just posted a blog on news from Notre Dame and Cornell about HDAC inhibitors correcting the Niemann Pick Type C defect in cell culture.

I just read on PNAS that the Novartis cancer drug Panobinostat (LBH-589), a Phase 3 drug developed for the treatment of cancer, may be able to halt the progression of Niemann Pick Type C disease, a fatal genetic cholesterol metabolism disorder that affects my twins. Panobinostat inhibits one or more of the histone deacetylase (HDAC) enzymes, a mechanism leading to apoptosis of malignant cells via multiple pathways.

Here is the abstract — I don’t have the full paper.

Histone deacetylase inhibitor treatment dramatically reduces cholesterol accumulation in Niemann-Pick type C1 mutant human fibroblasts

  • Niemann-Pick type C (NPC) disease is predominantly caused by mutations in the NPC1 protein that affect intracellular cholesterol trafficking and cause accumulation of unesterified cholesterol and other lipids in lysosomal storage organelles. We report the use of a series of small molecule histone deacetylase (HDAC) inhibitors in tissue culture models of NPC human fibroblasts. Some HDAC inhibitors lead to a dramatic correction in the NPC phenotype in cells with either one or two copies of the NPC1I1061T mutation, and for several of the inhibitors, correction is associated with increased expression of NPC1 protein. Increased NPC1I1061T protein levels may partially account for the correction of the phenotype, because this mutant can promote cholesterol efflux if it is delivered to late endosomes and lysosomes. The HDAC inhibitor treatment is ineffective in an NPC2 mutant human fibroblast line. Analysis of the isoform selectivity of the compounds used implicates HDAC1 and/or HDAC2 as likely targets for the observed correction, although other HDACs may also play a role. LBH589 (panobinostat) is an orally available HDAC inhibitor that crosses the blood–brain barrier and is currently in phase III clinical trials for several types of cancer. It restores cholesterol homeostasis in cultured NPC1 mutant fibroblasts to almost normal levels within 72 h when used at 40 nM. The findings that HDAC inhibitors can correct cholesterol storage defects in human NPC1 mutant cells provide the potential basis for treatment options for NPC disease.

Interestingly, Gleevec, another cancer drug developed by Novartis also works in the NPC mouse model (modestly) and was reported by Paul Greengard to have an effect in the Alzheimer’s model.

Wow. Panobinostat may also work in other neurodegenerative diseases as well such as Alzheimer’s, Huntington’s, ALS, SMA……many others where HDAC inhibition is showing some success!


HDAC Inhibition Therapy to Stop Neurodegeneration? Notre Dame and Cornell Scientists PNAS Paper To Show How Histone Deacetylase Inhibitor (HDIs) Corrects Niemann Pick Type C Cholesterol Defect

March 21, 2011 by  
Filed under Featured Stories

Notre Dame and Cornell researchers are reporting a major breakthrough in Niemann Pick Type C disease research that has major relevance to other progressive neurological conditions such as Alzheimer’s, Huntington’s, and ALS.

A paper coauthored by Olaf Wiest and Paul Helquist of the University of Notre Dame’s Department Chemistry & Biochemistry and Frederick Maxfield, Chair of Biochemistry at Cornell University Weill College of Medicine, is apparently going to appear in the Proceedings of the National Academy of Sciences this week according to a press release issued by Notre Dame.

The paper apparently shows how the use of a histone deacetylase inhibitor corrects the damage done in Niemann Pick Type C,  a fatal genetic cholesterol metabolism disorder, and allows once-diseased cells to function normally. The press release discusses the “effectiveness of the drug” but no compound is named.

Histone deacetylase inhibitors (HDIs) have a long history of use in psychiatry and neurology as mood stabilizers and anti-epileptics.  In more recent times, HDIs are being studied as a treatment for neurodegenerative diseases so the fact these researchers are having such success in NPC cells is very promising.

Interestingly, our neurologist Dr. Daniel Birnbaum at Children’s Hosptial Research Center Oakland started Addi and Cassi on Valproic Acid, an anti-seizure medicine that is also classified as an HDAC inhibitor.  We have had good success with Valproic Acid over the past three months.

Trichostatin A, suberoylanilide hydroxamic acid (SAHA), Phenylbutyric Acid and Butyric Acid can all act as HDAC inhibitors as well.  Maybe it’s one of these compounds that is showing success in the Niemann Pick Type C cells?

The press release does not say whether testing was done in NPC mice or NPC cats or if the HDAC inhibitors worked in NPC animal models.  I hope the paper discusses animal work.  The good news is that many HDAC inhibitors are in advanced clinical trials or even approved drugs.  I hope these compounds can also easily cross the blood brain barrier!

HDAC inhibition therapy — is this the miracle we have all been waiting for?   I hope so and if  it is Notre Dame and Cornell researchers will be heros for their in work that will extend beyond NPC disease.

More information on Histone deacetylase is here.



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