Our story & mission
NTAP accelerates the development of effective therapeutics for plexiform and cutaneous neurofibromas.
Beyond NF1: The potential for impact
The connection between NF1 and Ras means that unlocking therapies for NF1 will have direct impact on unlocking therapies for a broad range of common cancers driven by alterations in the NF1 gene as well as the many conditions associated with dysregulation of RAS.
Breakthrough treatments are now within reach thanks to scientists and clinicians who are making this their life’s work.
Relentlessly Focused on Delivering Results
In 10 years, NTAP has revolutionized the NF1 field by supporting researchers and clinicians interested in NF1 with funding, open access to data, critical resources and connections. We have strengthened the development and expansion of the global scientific community dedicated to advancing and sharing discoveries within and beyond the NF1 field. NTAP collaborates with everyone invested in NF1 research including industry, government, academic institutions, and patient support organizations to reach the goal of meaningful therapies for people with NF1 and beyond.
Our Cutting Edge Approach
Focus on therapeutics
NTAP drives the most promising ideas for plexiform and cutaneous neurofibroma into tangible therapeutics. In order to achieve this, NTAP condenses the journey from lab to clinic. This translates discoveries into treatments both faster and with a higher rate of success.
NTAP recognizes that developing treatments requires effective collaboration. We foster relationships across a broad research community including the pharmaceutical industry, regulatory agencies, private foundations and academic centers to increase the pace at which promising research can be converted into patient therapies. Advisory boards with diverse scientific, medical and business expertise ensure that we follow the best in class scientific approaches and that our work is connected to the latest developments within and outside of the NF1 field.
Promote open, timely sharing of data
We require that data from funded projects be shared in real time. NTAP is led by scientists who monitor research progress, facilitate communication among collaborators, and make sure data is disseminated so that projects with the most potential for translational success are advanced.
We identify critical gaps in knowledge and actively recruit scientists with innovative ideas to address these gaps. The grants application and approval process is devoid of bureaucracy and provides quick feedback and funding. Follow-on grants are common, and investigators receive additional resources that can help turn basic science discoveries into effective therapeutics.
NTAP was inspired by two parents whose daughter was diagnosed with NF1 in 2007.
Shocked to learn to learn that despite NF1 being one of the world’s most common rare diseases there were no medical answers or approved drug therapies, they set about to create an organization that would focus on two of the most devastating symptoms of NF1: plexiform and cutaneous tumors. It was their hope that their own daughter would not have plexiform tumors but knew that she, like 99% of the people with NF1, would develop cutaneous tumors.
From their own experiences, they also knew the importance of leadership programs and they were committed to establishing a program to train the next generation of clinician/scientists in NF1.
After a series of twists-and-turns, they knew they had their answer when they met Dr. Jaishri Blakeley at The Johns Hopkin University. Dr. Blakeley y’s was creative, driven and kind. Her desire to find therapies was matched only by them. Thus, modeling collaboration from the start, these parents, Dr. Blakeley, and a small group of trusted industry and academic advisors, established NTAP in 2012.
It is this vision and collaboration that continues to inspire NTAP’s work each day.