Rapid Methods to Measure the Progression of Fibrodysplasia Ossificans Progressiva (FOP)

37 Submissions
171 Views
$25,000 USD
Challenge under evaluation

Challenge overview

OVERVIEW

The International Fibrodysplasia Ossificans Progressiva Association (IFOPA), the Seeker for this Wazoku Crowd Challenge, is looking for improved methods to rapidly assess the progression of Fibrodysplasia Ossificans Progressiva (FOP) in patients. The primary outcomes that indicate disorder progression are new bone growth as indicated by measuring new bone lesions and/or increased total body bone volume.

FOP is a rare genetic disorder where affected patients suffer from bone growth in muscles, tendons, ligaments, and other tissues where bone should not grow. A key outcome of progression of the disorder is the growth of new bone caused by FOP, which can be measured by detecting internal lesions signifying the start of new bone formation or increased total body bone volume through scans/imaging.

Currently available methods to measure new lesions and increased bone growth are expensive, require high degrees of training to perform and analyze, and can often be inaccessible for FOP patients with permanent changes in their mobility and body positioning caused by the disorder. Even more importantly, current methods’ sensitivity is such that it requires a minimum of 6 to 12 months to detect a statistically significant change in the number of new lesions or total bone volume, which results in very long clinical trials for potential drugs that slow or stop ectopic bone growth in FOP.  

The IFOPA is looking for novel—more efficient, safe, noninvasive, and user-friendly—approaches that should be capable of measuring new bone formation (or lack thereof) in no more than 3-6 months.

This is a Prize Challenge which requires a written proposal to be submitted. Awards will be contingent upon the theoretical evaluation of the proposal by the IFOPA, followed by experimental validation of a few, most promising, proposals. To receive an Award for this Prize Challenge, Solvers are required to transfer non-exclusive rights to the Intellectual Property (IP) in their proposed solution. Solvers will retain all rights to any proposal not Awarded. The IFOPA is also willing to consider partial awards for theoretically evaluated proposals or knowledge of commercially available solutions.

Solvers can win from the award pool of $25,000, with staged awards for solutions that meet all requirements, as solely determined by the IFOPA. In the first stage, $10,000 is reserved for the best solution(s) that meets all solution requirements following theoretical evaluation. Then, the most promising proposals will undergo experimental validation with FOP researchers and a further $15,000 is reserved to award solutions contingent on this experimental validation.

Submissions to this Challenge must be received by 11:59 PM (US Eastern Time) on April 21st, 2024.

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ABOUT THE SEEKER & ELIGIBILITY

The International Fibrodysplasia Ossificans Progressiva Association (IFOPA) is a 501(c)3 nonprofit organization that provides hope to individuals with Fibrodysplasia Ossificans Progressiva (FOP) and their families through education and support programs while funding research to find a cure for FOP.

The IFOPA is one of the largest funders of FOP research in the world with more than two-thirds of the annual budget dedicated to funding open-source research tools, individual research grants, providing research infrastructure, hosting scientific meetings, and engaging the FOP community in clinical studies and trials. 

With multiple assets in development, the IFOPA is using open innovation to maintain momentum and continue to work for one or more of these treatments to prove successful. Those with FOP need reliable, accessible, short-term measurements to assess the efficacy of drugs in development for FOP. For more information go to www.ifopa.org.

Employees of ­­­­the IFOPA are ineligible to receive an award for this Challenge.

Find out more about participation in Wazoku Crowd Challenges.

 

BACKGROUND

 Fibrodysplasia Ossificans Progressiva (FOP) is an ultra-rare (with approximately 900 cases confirmed worldwide) genetic disorder characterized by abnormal bone growth in places where it’s not supposed to occur: muscles, cartilage, tendons, and ligaments. This growth is called “heterotopic ossification” (HO) to differentiate the new bone growth caused by FOP from normal, non-FOP, bone structures.

Due to the HO, patients with FOP suffer from limited mobility and altered posture and positioning of the body, which causes pain and discomfort. Sufferers often cannot fit into traditional imaging/scanning methods and tools or the process of a scan is painful, difficult, and unsustainable as the disease progresses.

As of now, there is one newly approved treatment, Sohonos (palovarotene) for the reduction in volume of new bone in adults and children aged 8 years and older for females, and 10 years and older for males with FOP. There are no treatments for young children for a disease that progressively steals their mobility beginning as young as a few months old. A few other clinical trials are underway with more in preparation. Without cost-effective, accessible, and efficient means to measure new bone formation in ultra-rare FOP, ongoing and emerging trials will run out of available patients, those in trial will endure lengthy trials with uncomfortable and sometimes untenable, measurement procedures, and the pipeline of future treatments will dry up. To learn more about FOP see this recent review.

Figure 1: A photographed individual with FOP (left), an example scan of an individual with FOP (center), and a skeletal representation (right). Sourced from Medizzy.com.

 

HO bone growth—measured as a fraction of total body bone volume—is one primary outcome measure used to monitor the progression of FOP. Currently, this is being done by the whole body computed tomography (WBCT) method, a method approved by regulatory agencies.

WBCT is a noninvasive imaging procedure that uses a combination of X-rays and computer technology to produce horizontal, or axial images (often called slices) of the body. In WBCT, an X-ray beam moves in a circle around the whole body to create many different views of the same organs and structures. The X-ray information is sent to a computer that determines the total volume of bone surface changes in comparison with previous WBCT scans and displays the information in a two-dimensional form on a monitor.

The FDA in the United States have begun to use number of new lesions as a primary outcome measure for progression of the disorder, also determined using WBCT or positron emission tomography (PET) scan. Rapid methods to detect the number of new lesions and their location would be an acceptable solution, showing the IFOPA and clinical trial providers where new bone is beginning to form.

Although WBCT and PET scans show detailed images of bone that are more detailed than a standard X-ray analysis, the WBCT method has several shortcomings:

  • Timing: Most importantly, it takes a minimum of 6 to 12 months to see a statistically significant difference in total body volume between different WBCT scans to see if a potential treatment is working or not. This makes clinical trials unacceptably long.
  • Accessibility: The WBCT test lasts 30-60 minutes, which makes it difficult, if not outright impossible, for many FOP patients with some body shapes to take the test.
  • Availability: WBCT and PET scans are complicated procedures requiring expensive equipment and highly skilled staff to perform. This makes the test prohibitively expensive and therefore inaccessible in many places around the world. For example, the typical cost of one single WBCT test in the United States may approach $2,000 without healthcare or insurance deductions. PET scans are more expensive.

Other methods can potentially be used to monitor FOP progression too: the whole-body X-ray scan, radionuclide bone scan, dual energy X-ray absorptiometry, CT scout scan, ultrasound, and MRI. However, these methods either lack sufficient sensitivity (whole-body X-ray scan) or prohibitively expensive (CT scout scan, MRI), or include using rather high levels of radiation (whole-body X-ray scan, radionuclide bone scan), which can be dangerous to patients, especially for children. Ultrasound approaches are more accessible but lack the ability of other methods to measure new bone growth on top of old bone growth and scan/image the entire body.

Cost estimates for these other modalities vary widely, depending on the institution provider, region, country, and geography. For further information on the cost of imaging, scanning, and detection methods currently used in U.S. healthcare, visit Medicare’s Procedure Price Lookup for estimates (use the dropdown ‘more cost information’ to get the total cost for the procedure comparison).

 

THE CHALLENGE

IFOPA is therefore looking for a new innovative test that will overcome the shortcomings of the existing tests, including WBCT and PET scanning. The expectations are that the proposed test will be capable of accurately measuring new lesions and/or abnormal (HO) bone growth in clinical trials designed to assess the efficacy of new FOP medications to inhibit abnormal bone growth. More frequent higher resolution outcome measurements would help to reduce the wait faced by clinicians, patients, and families alike.

Solvers from the public or private sectors are invited to develop new and improved, low or no-dose radiation, more cost-effective, non-invasive methods to measure HO in clinical trials to assess the efficacy of new medications to inhibit the formation of HO. This measurement modality could include: detecting total body volume of the new bone caused by FOP, detection of lesions that signify the start of new bone formation, or improved, non-invasive imaging to more accurately measure and picture patients’ bones.

New approaches and improvements to current methods could bring greater comfort with non-invasive scanning methods, shorter time frames for measurement that will speed up clinical trial timelines, and improved worldwide access to regulatory-approved outcome measurements in FOP.

IFOPA is open to solutions that would propose modifications of the existing methods but only if a significant improvement is clearly demonstrated.

 

SOLUTION REQUIREMENTS

IFOPA poses no restrictions to the nature of any proposed solution as long as it meets the following major requirements:

  • The proposed method should allow the detection of a significant change in total body bone volume and/or detection of new lesions, taking place in 3-6 months between separate measurements and, ideally, in 1-2 months.
  • The proposed method should be able to reliably detect HO growth anywhere in the body, even if it overlaps with the normal (old) bone growth. Where your solution detects new lesions and the number of lesions, it should be able to reliably detect that the lesions are caused by new HO bone growth, not pre-existing lesions with no new activity.
  • The proposed test should be non-invasive. More specifically, it shouldn’t require introduction of instruments into the body; however, approaches such as dyes or digestible medicine will be considered.
  • The proposed test should accommodate all FOP patients regardless of their specific body-shape deformity or inability to move.
  • Ideally, the proposed test should use no radiation. If radiation is proposed, its dose shouldn’t exceed 1 mSv per test.
  • The proposed test should comply with major requirements of regulatory agencies for imaging tests.
  • The cost of the proposed test should not exceed $2,000 and, ideally, result in a yearly cost of < $250 per patient per scan.
  • The proposed test should be simple and intuitive enough to be performed in a standard clinical imaging laboratory.

The IFOPA wants the Solvers to be aware of two additional important points:

  • The IFOPA would like to establish, as soon as possible, proof of concept of the most promising solutions. Proposals that include a detailed description of experimental validation steps of any innovative approach will therefore be given higher consideration.
  • The IFOPA is aware of programs like Lightship that offer access to clinical trials to everyone with at home, near home, and in-clinic visits. If Solvers suggest proven ways to improve access to traditional FOP imaging (WBCT/MRI/X-rays/ultrasound/nuclear medicine, etc.) or proven ways to mobilize these open resource approaches for use in further clinical trials or by industry, the IFOPA would consider a partial award.

This Challenge has a staged award structure. Solvers are asked to provide a written proposal, consisting of a technical description of how their theoretical solution or improvement to current modalities could be validated for use with human subjects with FOP. All proposals will undergo theoretical evaluation of the proposal by the IFOPA, with a portion of the award pool ($10,000 USD) committed for the best solution(s) at this stage that meet all solution requirements. Solvers are required to transfer non-exclusive rights to the Intellectual Property (IP) in their proposed solution, to be awarded.

Then, a few, most promising proposals will undergo experimental validation – FOP researchers will reach out to and work with Solvers in this stage to validate the solution. In this stage, a further portion of the award pool ($15,000 USD) is reserved to award solutions that demonstrate promise in experimental validation.

The IFOPA is also willing to consider partial awards in the first stage of theoretical evaluation for knowledge of commercially available solutions.

Solutions with Technology Readiness Levels (TRLs) 4-9 are invited; proof of concept solutions detailing HOW to solve the solution requirements, or production ready collaboration proposals about existing technologies and/or WHO can provide these.

This is a Prize Challenge, which has the following features:

  • The best solution(s) in this Prize Challenge have the opportunity to win some or all of the award pool of $25,000 USD if you meet all requirements, as solely determined by the IFOPA.
  • Awards will be contingent upon the theoretical evaluation of the proposal by the IFOPA with awards up to $10,000 reserved to this first stage, followed by experimental validation of a few, most promising, proposals, with awards of up to $15,000 reserved to this second stage.
  • The IFOPA is also willing to consider partial awards for theoretically-evaluated proposals or the knowledge of commercially-available solutions.
  • To receive an Award for this Prize Challenge, Solvers are required to transfer non-exclusive rights to the Intellectual Property (IP) in their proposed solution. Solvers will retain all rights to any proposal not Awarded.
  • The IFOPA may also issue “Honorable Mention” recognitions for notable submissions that are not selected for monetary awards.

 

YOUR SUBMISSION

Please login and register your interest, to complete the submission form.

The submitted proposals must be written in English and can include:

  1. Participation type – you will first be asked to inform us how you are participating in this Challenge, as a Solver (Individual), Solver (Organization), or Expert.
  2. Solution Stage - the Technology Readiness Level (TRL) of your solution, TRL1-3 ideation stage, TRL4-6 proof of concept stage, TRL7-9 production ready stage.
  3. Problem & Opportunity - highlight the innovation in your approach to the Problem, its point of difference, and the specific advantages/benefits this brings (up to 500 words).
  4. Solution Overview - detail the features of your solution and how they address the SOLUTION REQUIREMENTS (up to 500 words, there is space to add more in the summary field, and add supporting data, diagrams, etc. as attachments).
  5. Experience - Expertise, use cases and skills you or your organization have in relation to your proposed solution (up to 500 words).
  6. Solution Risks - any risks you see with your solution and how you would plan for this (up to 500 words).
  7. Timeline, capability and costs - describe what you think is required to deliver the solution, estimated time and cost - bearing in mind IFOPA's solution requirement that the cost of the proposed test should not exceed $2,000 and, ideally, result in a yearly cost of < $250 per patient per scan. (up to 500 words).
  8. References - provide links to any publications or press releases of relevance (up to 500 words).

Wazoku encourages the use by Solvers of AI approaches to help develop their submissions, though any produced solely with generative AI are not of interest.

Find out more about participation in Wazoku Crowd Challenges.

Submissions to this Challenge must be received by 11:59 PM (US Eastern Time) on April 21st, 2024.

Would you like to learn more about the Evaluators' requirements in this Challenge? Watch our Challenge Space for the Rapid Methods to Measure the Progression of Fibrodysplasia Ossificans Progressiva (FOP) Challenge now:

Late submissions will not be considered.

Your submission will be evaluated by the evaluation team first reviewing the information and content you have submitted at the submission form, with attachments used as additional context to your form submission. Submissions relying solely on attachments will receive less attention from the evaluation team.

After the Challenge submission due date, the IFOPA will complete the review process and make a decision with regards to the winning solution(s) according to the timeline in the Challenge header. All Solvers who submit a proposal will be notified about the status of their submissions; however, no detailed evaluation of individual submissions will be provided.

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