A Letter to our Supporters

We would like our supporters and the Niemann-Pick type C (NPC) community to see some of the amazing progress that is being made to positively impact the lives of those afflicted by this horrible disease.

First, we thank all who support the efforts to find treatments and cure for NPC disease.  It is the goal of the Ara Parseghian Medical Research Fund (APMRF) and the NPC community to collaborate with each other, and bring together clinicians, researchers, and industrial partners to find a cure for this devastating disease that steals the lives of these precious children.

Because of the support of so many, the perseverance of many NPC families, and the dedication of our corporate and organizational partners, there has been a lot of progress over the past year with these efforts. In 2017, the APMRF funded 9 grants ranging from $60,000 – $250,000 and there was an equal amount of funding awarded from other patient groups such as the SOAR-NPC collaborative, the National Niemann-Pick Disease Foundation and others.  Working together, our community invested more than $2.5 million to support NPC research. Research areas range from fundamental science of the NPC pathway, to the development of new therapeutics for NPC disease, to the support for new gene-therapy efforts, and every year more scientists and clinicians join our team and dedicate their research efforts to this cause.

Additionally, on March 15, 2017, Vtesse, Inc. announced full enrollment in the Phase 2b/3 clinical trial of VTS-270, a well-characterized complex mixture of 2-hydroxypropyl-b-cyclodextrin (HPβCD) in Niemann-Pick Type C1 (NPC) disease. The investigational drug candidate showed promise in pre-clinical and early clinical studies for NPC disease. Of recent, Sucampo Pharmaceuticals, Inc. a global biopharmaceutical company, announced on April 3, 2017 that it has acquired Vtesse. Then on December 22, 2017, Mallinckrodt Pharmaceuticals acquired Sucampo.  This is an incredible opportunity because it connects us with an established, global partner with a similar mission.

Beyond research funding, groups of dedicated partners including NPC families and organizations, industry, clinicians, and researchers work together to solve other critical needs.  We are now accelerating the development of a newborn screen to detect children with NPC at birth.  Our community is developing an NPC patient registry to help streamline information for clinicians, researchers, and industry as they race to find a cure.  And every year, over 100 scientists, experts, and family members gather in Tucson for the Annual Michael, Marcia and Christa Parseghian Scientific Conference for Niemann-Pick Type C Research.  It is largely unheard of to have this level of participation for such a rare disorder, and is an opportunity for collaboration amongst our researchers and partners, and inspires hope to all of those effected by this disease.

Everyday our knowledge of NPC disease grows.  Every day we move closer to a cure. Because of our supporters, our first approved treatment is around the corner and families whose children are diagnosed with this dreadful disorder have hope that their children may be the first kids to beat NPC disease.  Thank you again for all you do to help us find a cure and we will leave you with our favorite quote from Ara Parseghian who passed away on August 2nd, 2017:

“All of you in one form of another will share when we cross that goal line and win the fight against Niemann-Pick Type C. It’s not going to be ‘One More Victory For Ara,’ but it will be a victory for every child afflicted with Niemann-Pick Type C, for every parent and for every grandparent.”