Hope for Marian’s primary initiative is to keep life-saving medications available for all NPC patients.
These medications work to DRAMATICALLY SLOW OR EVEN FREEZE THE DISEASE SYMPTOMS for years. While nothing is a cure, it allows patients to live healthier, fuller, happier, longer lives with more independence, engagement, and connection, and to spend time with their loved ones WHILE OTHER MEDICINES MOVE THROUGH THE DEVELOPMENT PIPELINE.
Because these medications are still considered experimental by the FDA, patient access to them is an ongoing struggle. Please click the link below to read through individual stories of the people this affects.
Areas of Focus
REGULATORY ADVOCACY FOR SECURE ACCESS TO THERAPIES FOR THIS GENERATION OF PATIENTS WITH NPC
ADVOCACY FOR ETHICAL AND PATIENT CENTERED CONSIDERATIONS IN CLINICAL TRIALS AND ACCESS PROGRAMS
ADVOCACY FOR UTILIZING EXISTING NATURAL HISTORY AND PLACEBO DATA THAT PATIENTS HAVE LOST THEIR LIVES AND ABILITIES TO
Transcript from the Capital Hill Briefing on NPC, organized by Hope for Marian, with bipartisan collaboration from Congresswoman Lesko and Congresswoman Sewell on October 13, 2022
Woodrow was diagnosed with NPC at 3 months old. This year, he declined rapidly. His doctor recommended an existing experimental treatment that many other NPC patients have taken for a decade.
For reasons that I still can’t understand, we were refused access. Meanwhile, at the same hospital that recommended Woodrow’s treatment, there was another child receiving the exact same medicine.
Throughout our country almost every other NPC patient seeking treatment was receiving it, except for Woodrow and a few other newly diagnosed infants and toddlers. We felt abandoned. Woodrow wasn’t crawling anymore, he stopped saying the few words he had, he was choking for the first time.
As if the diagnosis of this terrible disease wasn’t bad enough, we were scared that we might never gain access to treatment. Thanks to Congresswoman Lesko and my Senators, Woodrow is now on Adrabetadex. And he is now stable, even getting stronger, he’s speaking, he’s able to eat safely again. But this story is not over. The next chapter is being written. Our family remains in crisis.”
- DENISE MILLER, MOM OF WOODROW MILLER
“Don’t want to see her become filled with sadness…we aren’t asking for something to be created out of nothing, we’re just asking for it not to be taken away.”
SARA, MARIAN’S MOM
A TIMELINE OF OUR RECENT INITIATIVES
January 2021 to present
Collaborate with C-PATH (Critical Path Institute, an FDA group that helps accelerate and meet unmet needs in complex diseases for drug development) and community partners to focus on NPC specifically; engaging ongoing dialogue and interest.
The Full Scope of Our Work
Hope For Marian seeks to support patient advocacy and access to medicine through a broad range of outreach, including:
We help newly diagnosed navigate a health system that is unfamiliar with this disease and providing financial support for those in need to ensure they have access to experimental medicines.
As an ultra-rare disease community we are our only advocates. We provide resources to help patients and caregivers become strong and confident advocates so they can confidently speak to their elected officials, healthcare providers, and many other healthcare decisionmakers.
We’re cultivating a supportive community of patients and caregivers through email exchanges, parent group chats, regular Zoom meetings, and social media channels to ensure no one feels alone.
SCIENTIFIC & REGULATORY INNOVATION
We encourage investment from industry and scientific agencies to better understand NPC, invest in medicines, and ensure FDA uses regulatory flexibilities and innovative clinical development to quickly approve new treatments.
CONGRESSIONAL OUTREACH & EDUCATION
We worked with the NPC community to orga-nize a virtual Capitol Hill briefing to educate members of Congress about urgent NPC issues.
PATIENT ADVOCACY & AWARENESS
We help bring the challenges of our NPC community into national awareness and conversation through local and national media, most recently Scary Mommy, Dr. Oz, Patient Worthy, Stat, Today.com. We also advocate for ethical and patient-centered considerations in clinical trials and access programs.
The groundwork has been laid, with scientific meetings, community advocacy, the NPC community is excited for companies to be taking part in meetings with FDA to discuss timely submission and resubmission for therapies to be approved for NPC.
We believe the opportunity exists with continued advocacy and collaboration to turn the page and potentially see the total number of approved therapies for NPC go from 0 to multiple.
But the work is far from over. There are hundreds of children and adults alive today whose lives depend on advancing effective investigational therapies to full approval.
Today, patients in the United States are grateful to have therapeutic access via expanded access protocols. Yet, the nature of compassionate use limits patients and their doctors to a single experimental therapy. While this interim approach has at its heart the patient’s best interest, NPC is a relentless, variable, progressive disease that attacks multiple systems in the body. With multiple experimental therapies that address different disease pathways, each year that passes without approvals that allow doctors to treat multiple pathways, patients suffer more irreversible debilitating disability and ultimately death.
The importance to the community of leveraging existing, real world data for regulatory purposes cannot be overstated. In addition to the lives and abilities captured in decades of natural history and trial data; it also cannot be recreated.
We are optimistic. In many ways, perhaps, for the first time. The last few years have seen significant engagement from industry and scientific experts, positive trial data, interest from the Critical Path Institute in NPC, important scientific discussions at the Duke Margolis meeting and recognition from our elected leaders in Congress on the importance of advancing therapies for this generation of NPC patients.
My daughter is alive today because of early expanded access to experimental therapy at 19 months old. But she and her peers will only stay alive by the advancement of these safe and effective therapies for NPC to full approval. This will allow her and other patients with NPC the tools that exist to fight back this disease that takes and takes and will never stop.
I hope you will agree – we are all ready to turn the page and find solutions for the children and adults who need secure access to these medicines to live.