Not the Greatest News for the NPC Community

Well today there was a conference called held by the NIH and NNPDF to update the community on the Cyclodextrin trial.  The news was not what I was hoping for.  They had to put the trial on a pause status due to an infection at the ommaya reservoir site in two patients and another (who is not in this trial) suffered from a brain bleed.  The ommaya reservoir is like a port for a chemo patient but it is surgical placed in the front of the brain.  The infection was cause by bacteria that sits on the skin that causes acne.  At this time they are not sure if NPC patients are at a higher risk since this is a lipids disease or not.  But they knew they couldn't continue with the trial with so many problems and not enough answers.

They put in on hold and got in contact with the FDA who in turn put an official hold on the trial and will send the NIH a letter stating what has to be done to get the trial off the hold status.  The FDA was very pleased with the quick action by the team at the NIH for putting the trial on pause right away without first contacted them.  The NIH will receiver the letter sometime next week and will inform the NPC community once they have a plan of action in place.

Dr. Porter did add that they did collect some promising results from the trial thus far.  The bio-markers looked good and they got goo PK data.  These results where from the patients receiving a very small dose of the cyclodextrin.  Dr. Porter thought they would see nothing with this low of a dose, so that in itself is very hopeful!!

They do think that the infection was not drug related but device related.  So he did go on to say that they will be looking at a lumbar injection as means to administer the drug.  They do have concerns with how much of the drug will actually reach the brain but with the data collected from the trial he believes that they will be able to administer enough of the drug to reach the brain without causing toxicity. 

There is a lot of work ahead for the NIH team but they are committed to get this trial back up and running as soon as they can.  They could not give any kind of time line or dose levels at this time.  I will keep everyone posted as information comes to me.  Please say an extra prayer for the NPC community especially those in the trial that suffered an infection and the child who suffered a brain bleed.  Thank you and God Bless!!

NPC Press Release on the Cyclodextrin Trial

Tylor is happy to announce along with the NIH........Drum Roll Please

Dear Families and Friends,
 

The NNPDF central office received the following press release from the National Institute of Health (NIH) NPC Clinic from Dr. Forbes "Denny" Porter with an update on the Cyclodextrin Trial.

Click Here to see the press release.

NIH Clinic Trial is a GO!!!

UPDATE: FDA Approves NIH TRND Team for NPC Cyclodextrin Clinical Trial!

Posted: 11 Jan 2013 02:42 PM PST

Dear Families and Friends,

The NNPDF central office received the following update from the National Institute of Health (NIH) NPC Clinic from Dr. Forbes "Denny" Porter.

"We were informed today that the FDA has removed the clinical hold on the hydroxypropyl-β-cyclodextrin trial. We are planning to enroll the first patient in two weeks. This trial is a major step in trying to determine if this is a safe and biochemically effective drug for NPC. Our goal is to use data from this trial to optimize the design of a larger second trial focused on clinical efficacy. Thank you for your help and support!
The TRND Team"

To follow updates and breaking news visit the Cyclodextrin page on the NNPDF web site.

More on the Cyclodextrin Trial

Below you will find the lastest on the Cyclodextrin Trial at the NIH.  Unfortunately Tylor is not a candidate for this first trial because he is on more than one anti-seizure medication.  We will be following this trial closely and we are hopeful that Tylor will be able to participate in the upcoming trial involving Cyclodextrin.

~ November 28th, 2012 ~

        Cyclodextrin (HP-β-CD) for NPC1 Disease

  ~ Clinical Trial Strategy ~

UPDATE ~ November 28th, 2012 ~ UPDATE

Therapeutics for Rare and Neglected Diseases (TRND)

National Institutes of Health ~ Bethesda, MD 

Dear National Niemann-Pick Disease Foundation Family Members,
The latest release pertaining to the upcoming NIH NPC clinical trial has been made available to the NNPDF. To view the NIH NPC Cyclodextrin Clinical Trial Flyer ~ dated: 11/28/12 ~ click here.
This study titled: 2-hydroxypropyl-B-cyclodextrin (HP-B-CD) in Niemann-Pick Disease, type C1, is in the process of being reviewed by the FDA and the team of researchers and physicians associated with the Therapeutics for Rare and Neglected Disease ~ Niemann-Pick Type C Disease Team (TRND NPC Team) at the NIH are hopeful that they will be able to begin enrolling patients in January of 2013.
The National Niemann-Pick Disease Foundation is pleased that we are able to forward this information along to our family membership. The NIH attached flyer specifies that interested parties should note your interest in possible trial participation by e-mailing a representative at the NIH: nichdnpc1@mail.nih.gov
Please note: If you do NOT have access to the internet or an e-mail account please contact the NNPDF Central Offices at the 920-563-0930 and we will assist you in reaching the appropriate contact individual(s) at the NIH for more information.

This is indeed, a very exciting time for all of our NNPDF family community and, more importantly, all of our precious loved ones diagnosed with Niemann-Pick Disease Type C

.

We WILL Persevere in our Quest for a Cure!

Kind Regards, Nadine M. Hill

Executive Director; National Niemann-Pick Disease Foundation

For a historical timeline on the Cyclodextrin (HP-β-CD) for NPC1 Disease Clinical Strategy ~ outlined by the NNPDF ~ please follow the link above.

Cyclodextrin Trial News

Latest Update on Planning for NIH's Clinical Trial of Cyclodextrin

~ November 2012 ~

Cyclodextrin (HP-β-CD) for NPC1 Disease

~ Clinical Strategy ~

Nuria Carrillo, MD

Therapeutics for Rare and Neglected Diseases (TRND)

National Institutes of Health ~ Bethesda, MD 

Please follow this link to view of PowerPoint presentation titled:
Cyclodextrin (HP-β-CD) for NPC1 Disease ~ Clinical Strategy ~
 
Created and presented by:

Nuria Carrillo, MD; Staff Clinician

Therapeutics for Rare and Neglected Diseases (TRND)

Division of Preclinical Innovation

National Center for Advancing Translational Sciences

National Institutes of Health ~ Bethesda, MD

  

Another NIH Update

Tylor had a bit of a rough morning. He had 14 seizures this morning because his appointments so the swallow study was a little hard as well as the speech appointment. So we will be going back tomorrow to redo those and see a neurologist. We also had our wrap up meeting with Dr. Porter and Nicole which went well. We will have to come back in a year for a follow up. Please pray that the seizure calm down so we can have a good trip home!

NIH Update

My computer has been down so I haven't really been able to keep everyone up on what is going on with Tylor.

Here is a little update about what has been going on at the NIH.


Tylor is fast asleep with Jill at the hospital...only one of could stay so we filpped a coin....Jill won so that means a night out on the town for me! Just kidding the NIH campus is locked down tighter that Fort Knox!! The security is crazy to get on campus. It's like the airport. But anyway Tylor is great today. He had a two hour hearing test which he was very good and then medical history with Dr. Porter and Nicole for a few hours. Tomorrow....Spinal tap, MRI, skin biopsy, and more hearing tests. They want to do more hearing because Tylor is losing some of his high frequency hearing which is normal for NPC. That's all for now...will update tomorrow.

Exciting News

Members of the NPC team....THANK YOU!

Dear families and friends of the NPC community,

There has been a large, collaborative effort to initiate a cyclodextrin clinical trial at the National Institutes of Health (NIH) to systematically evaluate the safety and efficacy of cyclodextrin therapy for the treatment of Niemann-Pick type C (NPC) disease. As many of you are aware, we met with the Food and Drug Administration (FDA) this past Tuesday, November 1, 2011, to discuss the development program for cyclodextrin

The exceptional work that has been done in NPC animal models has guided the design of a human clinical trial. Together with the Therapeutics for Rare and Neglected Diseases (TRND) group at the NIH, as well as several NPC researchers, Johnson & Johnson, and consultants from RRD International, LLC, we are working to submit an Investigational New Drug (IND) application to FDA.

The first step in submitting the IND application to FDA (the perquisite to an initial clinical trial in patients) was to request a pre-IND meeting with FDA to receive the Agency’s feedback on our development program before the IND application is officially submitted. On November 1 we met with the FDA review division staff to discuss the proposed development plan for cyclodextrin and needs for the IND application package. The meeting was positive and the Agency provided helpful feedback focusing on the drug safety and toxicology data. We will have an additional meeting with FDA to focus on the clinical trial design, and FDA is working with us to get that meeting scheduled before the end of the year.

We view this as a very positive step toward pursuing cyclodextrin as a potential treatment for NPC disease. We are planning a scientifically rigorous trial that will allow us to test cyclodextrin in our patients safely and in a way that will provide as much information as possible. While specific details of the trial will not be available until we have agreement from FDA and approval from the NIH ethics review board, we will share information with the NPC community as it is available.

We continue to work toward our goal of starting the trial next year and feel that with the recent FDA feedback, we are on track to do so.

Thank you for your continued support and encouragement as we work together to find a treatment for NPC disease. This fight would not be possible without all of you.

Sincerely,

The TRND Team

NIH Cyclodextrin Trial

National Institues of Health Announces Cyclodextrin Clincial Trial For Fatal Cholesterol Disease Called Niemann Pick Type C


August 16, 2011 by Chris Hempel
Over the past three years, many Niemann Pick Type C families as well as physicians treating NPC patients worldwide have contacted Dr. Caroline Hastings and I regarding Addi and Cassi’s treatments with hydroxypropyl-beta-cyclodextrin.

Since sharing our intravenous (IV) and intrathecal (IT) cyclodextrin treatment protocols with NPC families and doctors in the U.S. and other countries, many NPC patients have started cyclodextrin therapy.

Currently, I know of 11 NPC patients in various countries receiving cyclodextrin treatments – six are receiving intrathecal (IT) treatments to allow cyclodextrin to reach the brain.

With many Niemann Pick Type C families scattered around the world, it’s difficult for people to obtain information on cyclodextrin and determine how they might be able to get started with treatments. In the past month alone, I have been contacted by families located in Italy, Spain, Germany, Dubai and Japan and all are interested in cyclodextrin treatments.

I am writing this blog in the hopes it can help NPC families in other countries who are seeking information on cyclodextrin and help them understand how they may be able to treat their loved ones with this potentially life saving sugar compound.

NIH Announces Intracerebroventricular Clinical Trial with Cyclodextrin
In the United States, the National Institutes of Health (NIH) has announced an intracerebroventricular (ICV) clinical trial with cyclodextrin. Dr. Forbes “Denny” Porter is running the clinical trial with an NIH Bench-to-Bedside Grant and with the help of the NIHs Therapeutics for Rare and Neglected Diseases (TRND) program and the National Center for Advancing Translational Sciences (NCATS).

Details are limited on what the inclusion/exclusion criteria will be for the NIH cyclodextrin clinical trial and they are still preparing their IND for the FDA and conducting pre-clinical studies.

The exciting news for NPC families is that the NIH has announced that they will pursue delivering cyclodextrin directly into the brains of patients using an Ommaya reservoir. An Ommaya reservoir is an intraventricular catheter system – a catheter is implanted into the brain and it is attached to a reservoir implanted under the scalp.

Dosing is still being determined by work done on NPC animal models, including the naturally occurring NPC cat model. I am sure dosing will be quite similar to what we are already giving to Addi and Cassi (possibly every two week cycle) as the FDA likes to base clinical trials from animal data. The NPC cats receive bi-monthly doses of cyclodextrin and Dr. Hastings based our initial bi-monthly IT treatment plan from the same cat studies.

Getting Cyclodextrin Into The Brain and Past Blood Brain Barrier
In my previous posts, I have detailed our plans with trying to get cyclodextrin into the twins’ brains. In October 2010, Dr. Hastings received FDA approval to start the twins on intrathecal injections of cyclodextrin into their spines. Since Addi and Cassi were the first in the world to ever receive this treatment, safety was a primary concern.

We initially started with intrathecal injections into the spine because we had to prove that the twins would not have an adverse reaction or die from an injection of cyclodextrin into their central nervous systems. Also, intrathecal treatment was less invasive. Per our FDA approved protocol, the twins must be hospitalized and monitored after intrathecal treatments. At first, they were monitored for 24 hours. Currently, we’re down to three hour observations following IT treatments.

Since October 2010, Addi and Cassi have received over 20 intrathecal injections of cyclodextrin into their spines as we work towards a permanent solution. The twins were going deaf before we initiated intrathecal cyclodextrin therapy. We have now restored their hearing to normal levels which is quite encouraging.

We also continue with our weekly IV infusions of cyclodextrin into their bloodstreams and are also looking for ways to deliver cyclodextrin into the lung as IV and IT treatments do not appear to reach the lung.

Medtronic SynchroMed Pump To Deliver Cyclodextrin
Over the past year, we have been creating an intrathecal protocol with the help of Medtronic and Johnson & Johnson and plan to implement a Medtronic SynchroMed pump system to deliver cyclodextrin into the brains of the twins. Medtronic is currently conducting final pump studies with cyclodextrin and we hope to re- submit data to the FDA and our hospital Institutional Review Board (IRB) in September.

We are working towards a SynchroMed pump solution because the NPC mice data shows that low continuous dosing is effective in completely stopping NPC. NPC mice are now being treated like diabetics. Cyclodextrin may need to be given on a frequent basis (perhaps daily or even weekly). We simply don’t know. We will start with a weekly bolus of cyclodextrin through the pump.

The Ommaya port does have many advantages, however, it does not offer frequency of dosing — it’s an access port versus an automatic system. In addition, it is my understanding that the Ommaya requires a patient to be in a hospital setting to receive a treatment (in the United States). SynchroMed pump refills can be made in an outpatient setting once the pump is installed and the pump lasts about seven years.

Choosing Between Ommaya and SynchroMed
The beauty of the Ommaya system is that you know for certain that cyclodextrin is reaching the brain. I believe the NIHs plan to pursue the Ommaya option is the right decision for the clinical trial and to determine efficacy. I hope many NPC patients will be eligible to participate in the clinical because I believe cyclodextrin could be life saving.

Since Addi and Cassi have been receiving cyclodextrin treatments for almost three years, they will not be eligible for the NIH trial. Many other NPC patients, especially those in foreign countries, will also not be able to participate in a U.S. clinical trial and therefore may want to pursue treatments in their individual countries.

Hugh and I continue to look at a longer term and permanent solution for cyclodextrin treatments as it appears cyclodextrin will be a life-long treatment. We are looking for a solution that provides flexibility on dosing and also keeps our kids out of a hospital setting as much as possible.

Medtronic’s data shows high catheter placement into the spine will allow cyclodextrin to reach brain and we will be taking this chance. We know that our intrathecal treatments in the lower spinal area are reaching the twins’ brains as we have restored the twins’ hearing to normal levels. But there are still lots of unknowns and many risks too. There is no easy decision and no right answer.

Also, I am not sure if an infant can get a SynchroMed pump placed so the only option may be an Ommaya. The NPC animal data shows that the sooner the animals start cyclodextrin treatment (BEFORE symptoms are evident) the healthier the animals stay.

Exciting Treatment Options
The good news for NPC patients and families worldwide is there are now promising treatment options to pursue which were not available before. Doctors can look at both Ommaya or SynchroMed solutions to get cyclodextrin into the brain and consider these options today for their patients.

I believe we’re ultimately going to need combination therapy to treat NPC as the disease impacts every cell in the body. We’ll need ICV or IT to reach the brain, IV to reach the organs and tissues and probably some type of inhalable HPBCD to reach the lung.

These are the options we are pursuing to try and save Addi and Cassi’s lives. We hope this information will help other families as they look for any possible way to save their loved ones from this wretched disease.

For more on Addi and Cassi go to their website.

More news on Cyclodextrin

Results in Mice May Help Shape Clinical Trial for Children With Rare Fatal Disease

By Amy Dockser Marcus

It’s a well-known maxim in science that curing a disease in a rodent doesn’t guarantee the same results in a human being. But new research on using the drug cyclodextrin in mice is likely to help shape a clinical trial being planned for children with a rare and fatal genetic disease.

Scientists led by John M. Dietschy at the University of Texas Southwestern Medical School just published a paper in the Journal of Neuroscience on continuous cyclodextrin treatment for Niemann-Pick Type C, a fatal neurodegenerative condition in which cholesterol builds up in tissues and organs. The treatment not only kept mice alive but prevented the cognitive decline that is one of the hallmarks of the illness.

When delivered directly into each rodent’s central nervous system via the spinal column in order to better reach the brain, cyclodextrin “completely prevented the expected neurodegeneration,’’ the authors wrote.

The paper’s publication is especially propitious because the NIH announced earlier this year that it plans to start a clinical trial treating NPC patients with cyclodextrin, hopefully some time in 2012. One of the key issues still being discussed is whether to deliver the drug directly into the central nervous system (as was done with the mice in the paper) or through an infusion into a vein that carries the drug into the general circulatory system.

The new paper will fuel and shape that ongoing debate. It found that delivering cyclodextrin directly to the brain requires lower doses and appears to be more effective in reversing the cholesterol defect than infusions into a vein.

“It will be a very influential paper in the field,” scientist Daniel Ory tells the Health Blog. Ory ought to know: he is the principal investigator on an NIH grant focused on getting cyclodextrin from the lab into NPC patients. He’s also working closely with NIH’s Therapeutics for Rare and Neglected Diseases program, which selected NPC and cyclodextrin as one of its pilot projects to attempt to repurpose drugs for use in rare diseases.

Dietschy, who has published a number of previous papers on cyclodextrin and NPC, acknowledged that continuous, life-long administration of the drug into the central nervous system using a pump isn’t easy — even in a mouse. In children, surgery is required to install, remove, and replace pumps, and the devices can become infected. Still, Dietschy says, if more data and studies back up the findings, he believes the technical obstacles can be overcome by surgeons.

Rumors about the paper’s findings were already creating a buzz among some in the NPC patient advocacy community even before it was published. In May, the National Niemann-Pick Disease Foundation, an advocacy group, sponsored a teleconference with Ory and NIH clinician Denny Porter to discuss the planned cyclodextrin trial.

In response to a question by a parent asking about the possibility of doing a trial using the delivery method described in the Dietschy paper, Porter said that there would be additional regulatory hurdles involved since it is a “huge jump in risk or risk-benefit ratio” to think about delivering the drug directly to the brain rather than through a vein.

Ory tells the Health Blog that investigators want the most efficient delivery method, but also one that can be rapidly moved into the clinic. He says the risks of injections into the spinal column, could potentially slow down the opening of a trial. Still, he agreed that the paper’s findings will inform the discussion.

One human experiment is already underway in twin girls with NPC, who have been receiving cyclodextrin infusions under the FDA’s compassionate use program.

Chris Hempel, the mother of the girls, Addison and Cassidy, tells the Health Blog they will submit a protocol for hospital approval to install a pump in each girl’s spine that will start delivering continuous cyclodextrin infusions to the brain.

Hempel says she closely follows Dietschy’s research, reading each paper. “As more research comes out, we can modify’’ the approach,” she says.

Dietschy says it’s too early to tell how or if the new results will be applied to patients. In the meantime, he says he plans to observe a colony of mice receiving continuous infusions to see how they progress. Researchers are still working on the technical issues but Dietschy says he can’t help but wonder: Will mice treated this way go on to live a normal life? And if so, could children?

Photo: Associated Press
**Taken from the Wall Street Journal Health Blog**

Rare Diseases: Will push for new drugs pay off?

Cassidy Hempel, 6, waves at hospital staff with the help of her mother, Chris, at the Children's Hospital and Research Center in Oakland, Calif., Friday, March 18, 2011. Cassidy and twin sister, Addison, are being treated for a fatal disorder called Niemann Pick Type C disease. (Credit: AP Photo)

(CBS/AP)Call it the rare disease gap. Scientists have identified more than 7,000 diseases that affect fewer than 200,000 people, but treatments are available for just 200 of the diseases.


But now there's a move to close the gap. The National Institutes of Health this fall will open a center to speed genetic discoveries into usable therapies, doing some of the riskiest early-stage research in hopes companies then will step in.

A new International Rare Diseases Research Consortium is pushing for at least 200 more treatments by 2020, in part by pooling the work of far-flung scientists and families.

Rather than starting from scratch, the FDA is pointing the way for manufacturers to "repurpose" old drugs for new use against rare diseases, publishing a list of those deemed particularly promising.

And legislation recently introduced in the Senate, called the Creating Hope Act, would offer drug makers another financial incentive - a voucher promising fast FDA evaluation of their next blockbuster drug in return for developing a therapy for a rare or neglected disease that disproportionately affects children.

"We have to give drug companies a reason to go into this market," says Nancy Goodman of Kids v Cancer, a group pushing the legislation. Her son Jacob died at age 10 from a type of brain cancer that has no good treatment.

Pharmaceutical giants are starting to show some new interest in rare diseases, traditionally a niche market for small biotech companies. The practical reason: Blockbusters are drying up, says Dr. Ed Mascioli of Pfizer Inc., the world's largest drug company.

Some other companies, including Novartis AG and GlaxoSmithKline PLC, also have begun rare-disease programs.

But NIH Director Dr. Francis Collins says all the activity reflects a larger promise. "Getting a home run with a rare disease sometimes points you in a direction that will be beneficial for common diseases," he told The Associated Press.

That's the argument put forth by Chris Hempel, of Reno, Nev. Her 7-year-twin girls have been getting injections of an experimental drug for Niemann-Pick Type C (HPC), a disease that causes cholesterol and other fats to build up inside cells, harming the brain and other organs until patients lose the ability to talk, walk and swallow. Only 500 children worldwide are known to have it. But a drug that could flush out that build-up, Hempel contends, just might point to a new route to fighting heart disease or Alzheimer's.

Hempel isn't alone in her quest to repurpose common drugs. Consider progeria, a disease that rapidly ages children until they die of a heart attack or stroke, usually before their teens.

Collins' lab at NIH uncovered the gene defect behind progeria, research that he says he pursued only because of meeting another mom, Dr. Leslie Gordon, founder of the Progeria Research Foundation, and her son, Sam, who has the disease. Today, clinical trials are under way using a failed cancer drug named lonafarnib that promises to block some of the progeria mutation's effect.

There are an estimated 150 progeria patients worldwide, but Gordon points to growing evidence that the culprit protein may play a role in the heart disease that comes with regular aging, too.

** Taken from CBS News Health Watch **

Rare Diseases in the Spotlight

By Marissa Cevallos, HealthKey

1:55 p.m. EDT, April 27, 2011

Rare diseases are likely to get more attention now that an international consortium of patient advocacy groups and research funders has vowed to deliver 200 new therapies by 2020. For people with these diseases, such attention must seem long overdue.

Drug companies currently don’t have much incentive to develop drugs for diseases that affect fewer than 200,000 people, but almost 7,000 rare diseases exist affecting a total of about 25 million Americans.

Many are caused by mutations in a gene. The National Institutes of Health is opening a center in the fall to translate research findings in genetics to usable therapies, the Associated Press reports.

The NIH already has grant programs to spur research in rare diseases. The NIH's Therapeutics for Rare and Neglected Diseases program has a pipeline of projects. Its pilot projects offer a glimpse into some of the diseases that, though rare, can nonetheless have debilitating consequences.

—Schistosomiasis (also known as bilharzia or snail fever): Infection begins when a parasitic worm carried by freshwater snails penetrates the skin and lays eggs in blood vessels. First come rashes, then fever and chills, followed by liver and other organ damage over time. Researchers recently decoded the genomes of two schistosomiasis-causing parasites, which may allow researchers to find ways to inhibit the parasites’ growth. About 200 million people worldwide have the disease, and 280,000 die from it each year.

—Niemann-Pick Type C: In this condition, fatty deposits accumulate in the spleen, liver, lungs, bone marrow and brain. Type A, the most common, is fatal in infants. Type C can appear early in life or in young adulthood; it causes brain damage and ultimately can affect walking, swallowing, seeing and hearing. Only about 500 children in the world are known to have Type C. Researchers have found two genes that can contribute to Type C and Type D, but progress is slow.

—Hereditary inclusion body myopathy: Usually starting in young adulthood, the disease causes muscle-wasting, leading to severe disability in 10-20 years. A clinical trial in 2006 found mild benefits from intravenous immune globulin, essentially antibodies from blood plasma. A small gene therapy trial is underway, and stem cell therapies are being considered.

—Sickle cell disease: Crescent, or sickle-shaped, blood cells block blood flow in vessels, and can lead to stroke, organ failure or death. The disease affects about 70,000 to 100,000 people in the U.S., mostly African Americans. Only one effective medication exists to help prevent deaths. But a few children and adults have been cured by blood and bone marrow transplants.

—Chronic lymphocytic leukemia : This is the most common type of leukemia, a cancer of the bone or blood, found in adults. About 15,000 people are diagnosed each year (and about 101,000 people live with it).

The new consortium’s goal is to have 200 new therapies in nine years. Many people, in seemingly isolated disease groups, are waiting.

** Taken from the Baltimore Sun **

Promising Trial

NIH to Develop Clinical Trial Utilizing Cyclodextrin
Informational Conference Call to be Scheduled


The National Institutes of Health (NIH), in collaboration with the Therapeutics for Rare and Neglected Diseases Program (TRND), is developing a clinical trial utilizing cyclodextrin for Niemann-Pick Type C patients.

The clinical trial is in the planning phase and many criteria must be met and numerous approvals granted before the trial can take place. Dr. Porter, a Senior Investigator at the NIH, and Dr. Ory, NNPDF Scientific Advisory Board Chair, are working collaboratively to bring this trial to our NPC patient community.

In early May, the NNPDF will host a conference call with key constituents and researchers, for all interested parties in the NPC community to learn more about the work being done at the NIH. This conference call will include information pertaining to the development of plans for a cyclodextrin trial.

As a date and details for the conference call are confirmed, the NNPDF will update and inform our NPC family membership with the call-in information, agenda outlines and topics of discussion. We anticipate that after the presentation, the conference call format will allow participants to submit questions to the speakers/researchers.

Further updates on the clinical trial will be presented at the NNPDF Family Support and Medical Conference in Norfolk, Virginia, July 28th - 31st. Dr. Porter and Dr. Ory will answer questions pertaining to the clinical trial and will report up-to-date information about the trial at the conference.

For more information about Niemann-Pick Disease and the National Niemann-Pick Disease Foundation, visit http://www.nnpdf.org/.