Frequently asked questions
Neurofibromatosis & NTAP
The Neurofibromatosis Therapeutic Acceleration Program (NTAP) is a research enterprise focused on NF1 developing therapies for two common tumors in NF1 – cutaneous and plexiform tumors – and attracting more audacious clinician-researchers into the field. NTAP was founded in 2012 at Johns Hopkins University School of Medicine to work with academics and industry alike.
Neurofibromatosis type 1 (NF1) is a genetic condition of the nervous system. NF1 causes tumors to grow on nerves, it can result in altered organ function and NF1 increases the risk for several cancers. The occurrence of NF1 is fewer than 200,000 cases per year in the USA.
There are three types of neurofibromatosis (NF). Neurofibromatosis type 1 (NF1) results from an inherited or spontaneous alterations in the neurofibromin gene (NF1). The frequency of NF1 is 1 in 3,000 people worldwide. An alreration in one germ line allele of NF1 is sufficient to result in the clinical syndrome of NF1. However, tumor formation requires loss of function in the second allele. The fact that biallelic loss of NF1 is required for tumor formation defines NF1 as a tumor suppressor gene. Manifestations of NF1 include skin markings (café-au-lait spots and freckling), Lische nodules in the eye, bone or vascular malformations, learning and cognitive difficulties, and commonly tumors of the nerves throughout the body called neurofibromas.
Cutaneous neurofibromas (cNFs) are the most common skin tumor in people with NF1. cNF affects >95% of people with NF1. The number of cNF varies and can count in the hundreds or even thousands throughout the body in a given individual. These tumors cause disfigurement, pain and itching and are a major unmet need.
Plexiform neurofibromas (pNFs) are tumors that form in individual or multiple deep nerves. Up to 50% of people with NF1 develop pNF. pNFs are histologically benign, but can cause major neurologic and organ dysfunction and in up to 15% of tumors, transform into an aggressive form of cancer known as malignant peripheral nerve sheath tumor (MPNST). Selumetinib is the first FDA-approved oral drug for pNF in children with NF1.
NTAP does not engage in patient support or advocacy as we are solely dedicated to research and bringing new leaders into the field. However, we do encourage patients and others interested in these important activities to reach out to the Children’s Tumor Foundation who do an outstanding job at these two activities.
Applying for a Grant
1. In general
Currently, NTAP is strictly focused on the peripheral nerve tumors of NF1 (Cutaneous, Plexiform and Atypical or ANNUBP).
NTAP has the three programs: The Open Program, The Francis S. Collins Scholars Program and topic specific requests for applications. The Open Program is open all year on a rolling basis. Applications must focus on cutaneous, plexiform or atypical neurofibroma. The Francis S. Collins Scholars Program has an annual application deadline in December. NTAP announces Request for Applications (RFA) on specific topics periodically.
The submission of a grant application is through ProposalCentral (PC).
The lead PI can upload biosketch and other support from the Key Personnel/Collaborators page after entering the PI’s email address and role on the project.
A full grant application to NTAP requires signatures. After validating your application, you and your institute signing official may do the electronic signature.
The first review is completed within two weeks. Peer review is generally completed within one month. For applications that are reviewed favorably and are eligible to proceed to funding, there is typically a process of discussion and revision. Proceeding to contracting depends on the length of this process. Generally, from receipt to final decision is <90 days.
The submitted proposal will be reviewed internally by NTAP leadership to determine if it is in scope with NTAPs mission and of appropriate caliber to convene a peer review panel. Applications selected for progression to peer review will be reviewed by at least three peer reviewers (including NF and subject matter experts).
The review criteria vary and depend on the initiative. Please see more detailed information for a specific initiative. This information will be listed on the announcement and ProposalCentral.
NTAP uses a milestone and deliverable-based funding model. If a proposal is selected for funding, the investigator is asked to finalize a milestone and deliverable schedule in collaboration with NTAP leadership. Each milestone has a budget amount associated with it. NTAP makes payment to the investigator/institution based on evidence that the milestone has been completed.
No cost extension (NCE) request is permitted by PI with a research plan and budget justification during the NCE period. NTAP leadership will review the NCE request and work with the investigator to create a plan.
No. Each NTAP-awarded project stands alone.
No. Each NTAP-awarded project stands alone.
Applying for a Grant
2. The Open Program Initiative
Open Proposals support independent research projects accelerating the effective treatment development for plexiform neurofibromas (pNFs), cutaneous neurofibromas (cNFs) and atypical neurofibromas (aNF). This initiative aims to broaden and diversify the strategies applied to pNF, cNF and aNF research. These applications can be for small, highly focused projects or complex program project grants.
Open Program’s proposal must focus on the peripheral nerve tumors of NF1. The proposal must be focused on NF1 plexiform, cutaneous or atypical neurofibromas. However, there is no limitation in funding amount, duration or complexity of the study as long as all of the requests are expressly justified for the success of the project. There is equal opportunity for funding for all investigators indepdent of geography, institution or background.
NTAP’s Open Proposal program is open to investigators without limitation. Investigators from any part of the world and from any scientific background are eligible for NTAP awards.
The five pages proposal (single-spaced in typeface no smaller than Arial 11-point and 0.5” margins) does not include a bibliography. The bibliography and proposed Milestones/Deliverables are two additional documents (no page limit).
Unlike the NIH grant application, we do not request the vertebrate animal or human subjects section. We will request a copy of an approved IACUC/IRB for awarded proposals when proceeding to contracting. However, investigators must describe and justify animal or human study details in the proposal including species, strain name/number, source, sex, age, number/group.
There is no specific requirement for the number of support letters. Generally, your application should include letters of support from your institution (e.g., core facility director, department chair if the PI is a junior investigator), key personnel, collaborators, and other significant contributors, if you think these letters support/help your proposed research and proposal.
If the proposal is updating attachments only, you can do so without unsubmitting the application. If you need to upload a new file, you can send it to NTAP directly by email (email@example.com).
There is no limit for direct and total budget but the direct costs should be appropriate and well justified to perform the proposed research.
Applying for a Grant
3. The Francis S. Collins Scholars (FCS) Program
The Francis S. Collins Scholars Program in Neurofibromatosis Clinical and Translational Research is designed to build a community of exceptional clinician-scientists who will lead NF1 research and clinical care.
FCS is open to anyone with a health-professional degree that permits patient care and is willing to commit a minimum of 75% of professional effort to the NF1 for a minimum of two years and a maximum of three years. The applicants be in the last stages of post-doctoral training or a junior faculty member within seven years of the first faculty appointment. Please see the detailed eligibility requirements in the program guidelines or contact NTAP by email at firstname.lastname@example.org.
Following documents are required for the full application of the FCS program. i) professional history (1 page maximum), ii) career development plan (4 page maximum), iii) research project description (10 page maximum), iv) NIH formatted biosketch, v) mentorship plan (3 page maximum), vi) budget and budget justification, vii) letters of support (a minimum of three letters of support are required). Please see the detailed eligibility requirements in the program guidelines.
In addition to the salary support for the Collins Scholar (based on NIH salary cap) the following funds are available: i) mentor stipend ($10,000 per year), ii) research costs (up to $20,000 per year), iii) travel costs (up to $10,000 per year), and iv) education expenses (up to $10,000 per year). Among those funds, PI’s salary and mentor stipend are fixed costs. Research costs, travel costs, and education expenses are reimbursement costs..
Yes. Indirect costs are permitted a maximum of 10% of direct costs. However, no indirect costs are permitted on patient care-associated costs.
All applications undergo an initial review by NTAP staff and if the application is complete, it is forwarded to a pre-selected review panel for detailed review. Reviewers are senior medical and scientific leaders recruited across disciplines pertinent to NF1 research that have expertise in clinical or translational science.
FCS reviewers will evaluate each application on the following aspects: i) applicant’s research history, ii) career development plan, iii) merit and feasibility of research proposal, iv) mentorship plan, and v) budget and budget justification.
III. JHU NF1 Biorepository
The program is to establish a comprehensively annotated biorepository of tissue and blood fractions for use in NF1 research. The goal of the program is to increase availability of these tissues to the NF1 research community through scientific resource and data sharing.
The repository has collected NF1-associated tumors including cutaneous neurofibroma, diffuse infiltrating neurofibroma, plexiform neurofibroma, atypical neurofibroma, and malignant peripheral nerve sheath tumors (MPNST), and blood fractions from patients with NF1. The bank also collected and banked any malignancy from a patient with NF1 including breast cancer, GIST, and brain cancer. A current inventory of available specimens can be found here.
The following specimens are available – frozen tissue, patient derived xenografts, cell lines, and blood normal. The bank contains clinical information and genomic data such as RNAseq and whole exome sequencing (WES). The genomic data can be obtained through Synapse/NF Data Portal upon request.
Investigators interested in requesting specimens for their research can complete a specimen request form found here. Completed sample request form should be sent to Dr. Pratilas (email@example.com) & Stavriani Makri (firstname.lastname@example.org). Each request will be reviewed by an internal scientific review committee and JHU human Institutional Review Board (IRB). Investigators will be notified of a decision within approximately two months.
The short and best link to the JH NF1 biobank is https://bit.ly/nf1bank.
IV. ATCC cell line reimbursement
The reimbursement program was initiated by NTAP in 2018 to promote plexiform neurofibroma research and to foster collaboration.
NTAP’s current cell line reimbursement program is for the immortalized human plexiform neurofibroma cells and Schwann cells of ATCC. i) hTERT ipNF05.5 (mixed clones) (CRL-3387), ii) hTERT ipNF 05.5 (single clone) (CRL-3388), iii) hTERT ipNF95.6 (CRL-3389), iv) hTERT ipNF95.11b C (CRL-3390), v) hTERT ipNF95.11c (CRL-3391), and vi) hTERT ipNF02.3λ (CRL-3392).
Anyone who uses the ordered ATCC’s immortalized human plexiform neurofibroma cells for NF1-related research is eligible. The basic premise is that reimbursement is supported if your research efforts are intended to advance NF1-related discovery and therapeutics.
Yes. To be eligible, requests for reimbursement need to be received by NTAP within 90 days of the executed purchase.
If you are seeking reimbursement, it is advised to contact NTAP (email@example.com) for guidance and details about the cell line reimbursement program. To request a reimbursement, you need to submit the following forms to NTAP: i) a filled application form, ii) a receipt from ATCC associated with the cell lines purchase, and iii) proof of payment.
NTAP initiated the 100% reimbursement program for the immortalized human plexiform neurofibroma cells of ATCC to promote plexiform neurofibroma research. Although the reimbursement program is active, we do not reimburse those who ordered only healthy Schwann cell lines (either CRL-3391 or CRL-3392, or both CRL-3391/CRL-3392).
Bulk ordering means when you order at least five cell lines (CRL-3387, -3388, -3389, -3390, -3391, -3392) in the panel. Bulk ordering has discounted price. The price for bulk ordering is $2,800 for academics and $11,250 for industries.
Yes, a cell line price is $625 for academics and $2,500 for industries. The price of the cells for academics is 25% of the industry price.
i. For actual costs incurred for the purchase of the product (including taxes and shipping fees). Payments will not be made for indirect costs.
ii. Capped at $3,500 for not-for-profit entities and $12,000 for commercial (i.e., profit) entities within 24 months (based on purchase date) per primary investigator.
iii. Reimbursements for multiple orders from the same primary investigator within 24 months are allowed. However, it cannot exceed the cap limits noted above.
The reimbursement form is on NTAP’s website here. The document explains the requirements.
NTAP supports the FAIR Data Principles (Findable, Accessible, Interoperable, and Reusable) by sharing all NTAP-funded project data.
NTAP engages a third party, Sage Bionetworks (“Sage”), to provide data coordinating services for all NTAP-funded research. PIs are required, at the direction of NTAP-leadership, to provide Sage with all key data generated via NTAP support into a centralized database run by Sage Bionetworks.
NTAP will decide with the PI when the project is funded. Data contributors should upload the key raw data from their research to enable the data to be analyzed by a third party. This includes any information necessary to interpret the data, such as a description of the cohort or the model system, experimental protocols, descriptions of study instruments and equipment, and code used to generate processed data.
Yes. Uploaded data will be embargoed from the public and accessible only to the data contributor, the NF-OSI data curation team at Sage Bionetworks, or NTAP staff and consultants, during the award period.
This data shall be embargoed and not released by Sage. After the embargo period (18 months after the end date of the award), we will ask that you provide approval for the data to be available on an open platform. However, data will not be released to open access without PI’s approval. The embargo period will end earlier if the data is published or at the data contributor’s discretion.
Yes. Any requests by an investigator for an extension of the embargo period (e.g., due to a delay in primary publication) must be made in writing to the NTAP.
Synapse is a set of web services and tools that make it easier for researchers to aggregate, organize, analyze, and share scientific data, code, and insights.
The NF Data Portal is designed to help openly explore and share NF datasets, analysis tools, resources, and publications related to neurofibromatosis.