AuguryMJFF launches a focused funding push to turn promising Parkinson’s targets into real treatments
This article was written by the Augury Times
A swift funding move aimed at moving lab findings toward real treatments
The Michael J. Fox Foundation (MJFF) this month announced the first awards in a program meant to push promising biological targets for Parkinson’s disease from early discovery into tests that show whether those targets could become the basis of a drug or diagnostic. It is a deliberate move toward what researchers call “target validation”: building the evidence that a target actually matters for the disease and is worth investing in by drug makers or clinical teams. For patients, the hope is that this step will shorten the time between an exciting lab result and a real clinical test.
What Targets to Therapies aims to do and why target validation matters
Targets to Therapies (T2T) is MJFF’s program to speed up the path from basic science to therapies by focusing resources on the hardest early step: proving that a biological target — a gene, protein, cell type or pathway — plays a clear role in Parkinson’s and can be altered safely and effectively.
In plain terms, a target is something in the body you can aim a drug at. Many labs find potential targets in cells or animals, but those findings don’t always translate into human benefit. Target validation means testing the target with better experiments, biomarkers and models so the evidence becomes strong enough for a company to take the next, expensive step: making and testing a drug in people.
This phase sits between discovery and drug development. Discovery asks “what might be involved in disease?” Target validation asks “is this something we can target to change the disease in people?” By funding that middle ground, MJFF hopes to reduce risky guesswork and free up biotech partners to focus on the leads with real promise.
How the new awards will work and who’s expected to take part
MJFF described these first awards as competitive grants and collaborative project funding aimed at groups that can show a clear plan to move a target toward clinical readiness. The foundation said it will support a mix of academic labs, translational teams and collaborations with industry partners, although it did not list specific companies tied to the initial awards in the announcement.
The mechanics are straightforward: MJFF will pick proposals that include rigorous experiments, plans for relevant biomarkers, and a path to show target engagement in human samples or early clinical studies. The grants are intended to cover experiments that are often too risky for early-stage investors but crucial for deciding whether a target is worth a drug campaign.
Selection will be based on scientific merit and on whether a project can produce clear, de-risking data within a defined timeline. MJFF also emphasized collaboration and data sharing: projects are expected to deliver openly usable evidence to the wider Parkinson’s research community so others can build on successful work quickly.
Why proving a target matters for patients and drug development
Validating targets is a gatekeeper step for new therapies. When a target is well validated, companies can design molecules — small chemicals, antibodies, gene therapies — with more confidence that changing the target will affect disease. Without that validation, drug programs can fail late and expensively because the target simply wasn’t central to human disease biology.
Targets come in many forms. Some are genetic: a change in a gene increases risk and points to a protein or pathway to fix. Others are pathway-based: processes like protein clumping, inflammation, or mitochondrial dysfunction that seem to drive neurons to fail. Validation work tests these ideas in human-relevant systems and looks for markers that show a drug is hitting the target.
The challenges are real. Human biology is messy, and what works in a dish or mouse can fail in people. Building biomarkers that reliably report target engagement and early signs of benefit is often the make-or-break factor for whether a target attracts industry development and clinical trials.
What this means for patients, researchers and the wider research ecosystem
For people living with Parkinson’s, this funding is not an immediate promise of a new therapy. It is, however, a practical investment in the steps that make therapies possible. Better-validated targets mean fewer dead ends and—over time—more efficient drug development pipelines.
Academic researchers stand to gain clearer routes to translate findings into human tests. The emphasis on collaboration and shared data should reduce duplication of effort and let promising signals move faster from one lab to the next. Biotech firms benefit too: when a target arrives at industry with validated human biomarkers, those companies can take on clinical development with less scientific uncertainty.
Collectively, that lowers the overall cost and risk of trying new approaches — which, in a crowded and expensive drug-development world, can be decisive for whether a therapy ever reaches patients.
Limits, realistic timelines and what to watch next
This announcement is a start, not a cure. Target validation can take years; even when a target clears validation, turning that into a safe, effective drug usually requires multiple rounds of development and human testing. Expect a multi-year timeline from these awards to any mid-stage clinical trials, and longer still to wide availability if a program succeeds.
Signals to watch in future updates include: replicated preclinical results in human-relevant models, validated biomarkers that show target engagement in human samples, new public data releases from funded projects, and any partnering announcements with biotech firms that would take validated targets into drug development. Those milestones will be the practical signs that the program is shifting the needle from promising science toward testable therapies.
In sum, MJFF’s move is pragmatic: it aims to fill a funding gap that has long slowed Alzheimer’s and Parkinson’s drug discovery. It won’t create immediate treatments, but it can make the path to them clearer and less risky — and that incremental progress is precisely what the field has been missing.
Photo: Edward Jenner / Pexels
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