MDA Grant Will Support Development of ALS Biomarkers as a Predictive Test

Robert Bowser, Ph.D., founded Iron Horse Diagnostics in 2012.

The Muscular Dystrophy Association has awarded an MDA Venture Philanthropy (MVP) grant totaling $233,200 to Scottsdale, Ariz.-based Iron Horse Diagnostics to support development of a prognostic (predictive) test for ALS (amyotrophic lateral sclerosis).

Such a test could help improve and accelerate clinical trials and speed the development of life-saving drugs to individuals with ALS.

Iron Horse Diagnostics is an early-stage biotech company focused on developing diagnostic and prognostic tests for neurological conditions with high unmet medical need. Founded in 2012 by Dr. Robert Bowser, the company has developed assays for ALS, traumatic brain injury/concussion and for multiple sclerosis relapse.

Identifying the body’s key signs and measurements

The prognostic test under development by Iron Horse will measure specific protein-based biomarkers in blood and cerebrospinal fluid that indicate the presence of neurodegenerative disease.

Biomarkers are biological indicators or measurements that doctors or researchers can objectively measure and evaluate to determine the state of an individual’s health, confirm disease onset and progression, or gauge whether an experimental treatment is working or not. Some common examples of biomarkers include measurements such as blood pressure, heart rate or the presence of particular proteins in the blood or urine.

Biomarkers are often used to form the basis of diagnostic tests, which are used to identify, confirm or rule out a diagnosis, and prognostic tests, which may be used to predict the odds of certain outcomes (for example, the likelihood that a disease such as ALS will progress at a certain rate).

Iron Horse has developed, validated and patented tests to measure protein-based signatures of ALS. The company, which already has launched its diagnostic test in Europe, in partnership with Euroimmun, is working to determine whether levels of two proteins, called phosphorylated neurofilament heavy chain (pNFH) and neurofilament light chain (NFL), could help predict the disease course in ALS.

“MDA is excited to support this work at Iron Horse Diagnostics, with the goal to address one of the most pressing challenges to ALS drug development — the need for prognostic biomarkers,” said MDA Scientific Program Officer Amanda M. Haidet-Phillips, Ph.D.

The U.S. launch for the ALS diagnostic test is set for late 2017.

Facilitating clinical trials

Not only could the test be important in helping physicians predict the likely course of ALS — for example, fast versus slow progression — in individuals with ALS, but it could also allow physicians to enroll a more homogenous, or similar, group of individuals with ALS into a clinical trial. The ALS disease course varies greatly from one individual to another, creating a challenge for clinical trials since it can be difficult to discern a true change in the disease course (such as one caused by a drug) from all the normal noise and variability seen in the typical disease course. Having a prognostic biomarker could help reduce this noise, decrease the number of individuals needed for trials, and ultimately, accelerate the development of effective drugs for ALS.

“Our prognostic test will be an essential tool to enhance drug development for ALS,” said Iron Horse Diagnostics Chief Scientific Officer Andreas Jeromin, Ph.D.

Planning purposes

In addition to its usefulness in planning and conducting clinical trials, Iron Horse’s prognostic test could provide important information for individuals and families affected by ALS, informing discussion on, for example, how soon to shop for a power wheelchair; when a feeding tube or breathing assistance might be needed; and timing for everything from home modifications to assistive communication devices, to financial and end-of-life plans and decision-making.

“More accurate prediction of disease milestones could prove helpful for financial and life planning purposes for those individuals who wish to have this additional information,” Haidet-Phillips said.

In addition to its work on the prognostic test, Iron Horse has launched its first biologic ALS diagnostic test in Europe. The test is expected to yield results that could help confirm an ALS diagnosis within days, not months, allowing for quicker therapeutic intervention.

MDA is committed to finding treatments and cures for ALS

MDA Venture Philanthropy is MDA’s drug development program, which is exclusively focused on funding the discovery and clinical application of treatments and cures for neuromuscular disorders. MVP evaluates and makes targeted investments in for-profit and not-for-profit companies and academics developing therapeutics.

MDA has dedicated more than $363 million to ALS research and support services since 1950 and currently is funding 38 active ALS grants with a total funding commitment of $9.6 million. This year, MDA is funding 150 different research projects around the world.