NewsLine

National Niemann-Pick Disease Foundation, Inc.

Last Update: Saturday, March 7, 2005

NewsLine contains the latest information concerning
the Niemann-Pick Foundation, medical research, support groups, members, etc.

Featured News


Janet Pease informed the foundation that Amanda Ward passed away from respiratory complications of NPC. Amanda is the daughter of Jim and Missy Ward and attended a number of family conferences. Her aunt wrote "Amanda will always be remembered as a fighter and a child of exceptional good humor. She survived a liver transplant at the age of 3 months and was later diagnosed with cystic fibrosis as well as NPC. For many years, Amanda took her disabilities in stride as she tagged along with her big sister, Chelsea, not willing to be left out of anything. She formed a special bond with her brother, Adam, who also had NPC, & would bring toys to him when he could no longer get around on his own. Many of Amanda's cataplexy episodes came about when she joined in the laughter she caused in all of us. In spite of the fact that, for most of her life, Amanda could not speak, she communicated her determination to take part in life to everyone who knew her." [ Dec. 28, 2004 ]


James Brown and the Fox NFL Sunday team will be wearing the NNPDF lapel pins for their December 26th pregame show. The pregame show begins at Noon (EST) and the explanation for the pins is expected to be the first item discussed. Please let your friends, family, and supporters know about this event so they can watch also. NNPDF is working to get additional local coverage from Fox affiliates. If you can assist with media contacts or would like more information, please contact John Taft or Hunt Ozmer.

Fox NFL Sunday team with Hunt, Annette, and Hunter Ozmer
Fox NFL Sunday team wearing the NNPDF lapel pin

[ Dec. 2, 2004 ]


The Foundation is saddened to report the loss of Erin Roberts. Erin, the daughter of Michael and Holly Roberts, passed away at home surrounded by family and friends. Erin's website includes a message from the family. Our condolences and prayers go out to the Roberts and all of Erin's friends. [ Dec. 1, 2004 ].


Vicki Wiese, of the Frith Law Firm, has joined the Hunter's Hope Board of Directors. The firm has issued a press release which includes links about the Ozmer family and JB's work with charities. [ Oct. 23, 2004 ]


"Our little darlin' and brave girl Stacey Lynn died peacefully in her sleep this morning surrounded by family and friends. We know she is finally laughing and singing and dancing her heart out with the other angels in heaven.

As we begin the process of contacting everyone .... it is overwhelming to realize the number of people she has touched during her short life. She will continue giving even in her death with a gift to the "children of NPC" by donating her brain and tissue to Research.

I will not say she lost her battle to Niemann-Pick Disease, because even though this disease took everything else from Stacey, it could not squelch her tremendous spirit, her joy for life or her contagious smile. She did overcome this disease with courage and determination. She is our Hero!

She was our world, our inspiration..... We will miss her with every breath we take.

Barb, Gary and Kimberly Vorpahl"

The Vorpahl's were one of the families that founded the National Niemann-Pick Disease Foundation. Gary and Barb have been instrumental in guiding the Foundation and helping other families cope with the disease. Kimberly has been a pillar of support for her parents and her sister. Stacey has been an inspiration and beacon for many of us. Together they have touched thousands of lives for the better. We all grieve with you and send our deepest condolences and our prayers to Stacey's family and friends. [ Oct. 9, 2004 ]


The Queens Museum of Art in New York City is hosting a "Kids Collectors" day. The museum is selecting kids from all over New York to explain what items they collect and what kind of enjoyment they get from this hobby. The museum will then invite children to bring their collections for display at the museum on Sunday, October 10th.

The museum will also accept applications from Niemann-Pick kids from across the country and will prepare a special display to highlight the children and their collections. You can find more information at www.queensmuseum.org under the link to "Junior Collector's Day". Within the text there is a link to an application form to be completed. Be certain to list that you are a member of the National Niemann-Pick Disease Foundation.

If your child has any type of collection, we encourage you to submit an application. Don't wait to apply - there isn't much time left. What a great opportunity this is for our kids to participate in something really special! [ Sept 26, 2004 ]


A reminder that October is National Niemann-Pick Disease Awareness Month. Everyone’s involvement, be it big or small is so important. Every dollar raised, each raffle ticket sold, letter sent out, sign posted, each interview with the media, gathers support. You never know where the interest of a donor of a small gift might go after he/she gets involved and committed to this great cause. We ask each and everyone to please join us in our efforts.

Contact your local media with information to make them aware of NPD Awareness month and your family’s efforts. Include information on Niemann-Pick Disease and your family’s personal experience. Stress how each dollar your family has raised is being used to make a difference in fighting this disease.

Be sure to keep the NNPDF Office up to date on your plans, so we can update the October is Awareness Month page.

The following promotional items are available through the website or by calling the NNPDF office. [ 09-11-04 ]


Nikki Harrison, the clinical trial manager in the UK, announced the close of recruitment for the adult Zavesca clinical trial. There are 29 patients enrolled in this part of the trial. Recruitment for the pediatric arm (ages 12 and under) will remain open until the end of September. For further information, contact Darleen Vecchio (United States) at 212-305-4136 or Helena Prady (United Kingdom) at 44 (0) 161 727 2967. The full announcement is on our OGT-918 page. [ July 2, 2004 ]


Michael Edwards died of complications from Niemann-Pick Type C today. His parents, John and Cindy Edwards, said "The past week had brought many who loved him to visit, and his family was with him when his unbelievable spirit left to join those that have gone before. His smile will be remembered, his memory cherished, and the lessons he taught us will be unforgettable." Michael was well known to members of the Foundation from his visits to the family conference. He recently celebrated his 19th birthday. Our sympathies are extended to the Edwards family and to Michael's many friends on their loss. [ June 22, 2004 ]


Andre and Rosemarie Kipper have informed us that Aide aux Familles Niemann-Pick has been disbanded. AAFNP was the French Niemann-Pick support group. In their message the Kippers said "We thank you very warmly and wish to express our deep appreciation for all those years of collaborative work between our associations and the ties of friendship between our family and yours." The AAFNP website has also been discontinued. However, the website content has been transferred to the NNPDF. In the near future, we hope to put selected pages back on the Internet for French speaking families. [ June 18, 2004 ]


Doug Pease was elected as Vice Chairman of the National Niemann-Pick Disease Foundation. He has been on the Board of Directors since 1998. Doug is also the webmaster for the Foundation and will continue doing that. [ June 16, 2004 ]


Austin Cordell Morando died of pneumonia on June 5th. He would have been five years old on June 11th. Our condolences to the Morando family on their loss. Austin had Niemann-Pick type C. [ June 16, 2004 ]


Several families have been in the local news lately. You can read about them below.

Sam Roberts was visited in the hospital by Simon Goodwin of the Adelaide Crows. You can read the Adelaide Advertiser article here. Sam's father informed us that the Adelaide Crows are the local Australian Football team and that a "footy guernsey" is a team jersey.


Ryan Richardson was in the hospital earlier this month in critical condition. But the "little fighter" recovered and returned home in time for his fourth birthday. You can see the article and photos from the Florida Times here.


Hunter Ozmer and Chris Blixt attended Hunter's senior prom on April 24th . The Ozmer and Blixt families have long been active in the Foundation. You can read the article from the Roanoke Times here. The Ozmer's provided us a photo of the handsome couple. [ May 31, 2004 ]


Packets for the 2004 Family Conference in Orlando have been mailed. If you did not receive a packet and wish to register, an electronic copy is available in PDF format. You need Acrobat Reader to view and print the document. Registration Packet with Forms     Registration Forms Only   [ May 2, 2004 ]


Researchers at the M.D. Anderson Cancer Center at the University of Texas have caused stem cells in muscle tissue to develop into cells that look and act like neurons. The research was published in the April 15, 2004 issue of Genes and Development. The researchers created an artificial gene to activate other genes in the muscle tissue that are normally "turned off". The resulting cells show the biochemical, physiological, and structural properties of neurons according to the M.D. Anderson press release.

The work was done on cell cultures of mouse tissue. The researchers did not test whether the resulting cells were functional neurons but did note that they had been injected into mice brains with no ill effects. A follow up study will assess whether the new neurons might be able to replace damaged neurons. [ May 2, 2004 ]


Bernadette Dunlap, loving sister of Julie Laskoskie, notified us of her sisters death. Julie died peacefully in the early hours of Wednesday morning - April 21st, 2004. Julie is survived by sisters, Mary and Bernadette and a brother Charles along with many loving family members, nieces and nephews. If you wish to contact the family ~ correspondence may be sent to:

Bernadette and Dennis Dunlap
95 County Road 322
Cleveland, Texas 77327

Our condolences are extended to the family. [ April 25, 2004 ]


Dana Rae Cross, daughter of Dale and Ginny Cross, lost her fight with Niemann-Pick on April 20th. The family is requesting that in lieu of flowers memorials be made to:

Dana's Star Trust
c/o Sandra Bowen
4185 Leitches Wharf Road
Prince Frederick, Maryland 20678

The memorials will then be distributed to causes that had supported Dana and given her joy during her lifetime. The Foundation extends its sympathies to Dana's family and friends. [ April 25, 2004 ]


Art and Nancy Sullivan have informed us of the loss of their daughter, Karen, to NPC. The Sullivan's wrote "Karen could neither walk nor talk but now she is dancing and singing (two activities she dearly enjoys) again with the other Angels in Heaven. She is our joy and it was a privilege and blessing to be her parents. We will miss her terribly and are saddened by her loss but are Happy and at Peace knowing that she is free of the pain and restrictions that NPC had imposed on her."

Art and Nancy have been hosts of the NNPDF Board Meeting for several years. Each year, Karen would stop by to say hello to everyone. Her visits will be missed. Our condolences to the Sullivans on their loss. [ April 12, 2004 ]


A conference on Lysosomal Diseases and the Brain will be held May 14 and 15 in Bethesda, Md. The conference is for physicians and other health professionals involved in the treatment and care of patients with lysosomal diseases affecting the brain. Five of the presenters have close ties to Niemann-Pick research. Families wishing to inform their doctors of this conference can find more information at at www.lysosomal-brain-conf.org.

There are many lysosomal storage diseases, including Niemann-Pick, Gaucher's, Tay-Sachs, and Fabry's. These disease have certain common characteristics and most have neurological involvement. Advancements in treatment of any of the lysosomal storage diseases can have great benefit for others as well. NNPDF has recently agreed to collaborate with other lysosomal support and research organizations to expand awareness of these diseases and to pursue increased research funding by the Federal government. [ March 27, 2004 ]


CellTech announced today that the NPC pediatric trial for OGT-918 has been approved in the United States. Applications for the trial are now being taken. A mailing has been sent to NPC families about this trial. To apply or to obtain further information about the pediatric trial, contact Darlene Vecchio at Columbia University. A total of 12 pediatric patients will be included in the trial (six from the U.S. and six from the U.K.). All pediatric patients in the trial will receive OGT-918 (no control group is planned).

Please note that the adult clinical trial has not filled all available placements. If you are interested in participating in the adult trial, you should also contact Darlene. [ March 22, 2004 ]


Research published in the Feb. 20th issue of Science magazine shows that the NPC1L1 protein is critical to the absorption of cholesterol by the intestine (dietary cholesterol). The NPC1L1 protein is structurally similar to the NPC1 protein found in Niemann-Pick Type C. Mice without the protein had a substantial reduction in absorbed cholesterol. The drug ezetimibe, which reduces cholesterol absorption, had no effect on these mice, indicating the NPC1L1 protein is part of the same pathway.

This research is particularly noteworthy as the work was conducted by Schering-Plough, a major pharmacuetical company. In the past few years, Niemann-Pick has begun to be recognized as a disease that may have major implications for the treatment of heart disease, Alzheimer's, and other conditions. This is leading to new and unexpected research, such as that done by Schering-Plough. [ Feb. 24, 2004 ]


We have recently been informed of the passing of several members of the Niemann-Pick family. Our sincere sympathies are extended to each family. [ Feb. 24, 2004 ]


The NNPDF Board Meeting was held at the beginning of February. It was a busy meeting with much accomplished. [ Feb. 20, 2004 ]


Robert Whitehead, son of Leonard and Peggy Whitehead, passed away on February 4th. "Robert's heart was pure gold and his smile was 24 carat." The family has requested memorial donations be made to the Whitehead Fund at Washington University. Donations may be sent to:

Department of Neurology
Attn: Brenda McCall
St. Louis Children's Hospital
One Children's Place
St. Louis, Missouri 63110

Our condelences are extended to to Robert's family and friends. [ Feb. 5, 2004 ]


Thad and Leah Limer have informed us that their newborn daughter Rebecca Marye Limer passed away on January 16th. Our sympathies to the Limer family on their loss. [ Jan. 21, 2004 ]


James Brown and the Fox Sports team wore their Niemann-Pick lapel pins during the play off game between Green Bay and Philadelphia today. This is the second year that Niemann-Pick has gotten national publicity from Fox Sports. James Brown is the national spokesperson for the National Niemann-Pick Disease Foundation. We thank him for his efforts in publicizing our cause. [ Jan. 11, 2004 ]


Genzyme has obtained encouraging results testing gene therapy for Niemann-Pick Type A in animals. Mice were injected with corrected genes which resulted in an increase in acid sphingomylinase (ASM) activity. Lack of ASM is the basic cause of Niemann-Pick Type A. The study concluded that ASM activity increased in multiple regions of the brain. Dr. Gregory Stewart, Directory of Neuroscience for Genzyme stated "We clearly saw an improvement, and in some cases, a return to normal anatomy".

This is very good news. For many other diseases, gene therapy research has only been able to slow the progression of the disease. This research indicates it may be possible to reverse the effects of Niemann-Pick Type A in the brain. However, much more work is needed before a therapy can be developed.

Gene therapy has been successfully tested on many neurological diseases in animals. Unfortunately, the effects usually only last a short time. In the few trials that have been conducted in humans, serious side effects have been reported. The techniques used to deliver corrected genes in humans need to be greatly improved before general use is possible. [ Jan. 11, 2004 ]

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