Earlier this year, the world as we knew it changed forever. My husband Steve suffered a seizure that led to the discovery of a tumor and subsequent surgery. The diagnosis was as unexpected as it was devastating - a rare brain cancer. While the puzzle pieces of his tumor still don't quite fit together, what is clear is that after standard radiation and chemotherapy treatments, there is one drug that could potentially help Steve and others like him - ONC201.

ONC201 has shown promise in treating patients with H3k27m mutations found in malignant brain tumors. However, it's currently awaiting FDA approval while patients like Steve are running out of time.

We understand the need for rigorous testing and safety measures but believe there should be an accelerated approval process for critical treatments such as ONC201 when they show significant potential benefits for patients with rare conditions. 

Imagine a rare brain cancer that affects only a limited number of people each year, mostly children and young adults. Despite this, there is some hope - a proven treatment could potentially prolong life through a clinical trial.

Although the ONC201 trial has shown remarkably promising results that surpass those of previously FDA-approved drugs in initial trial phases, it remains inaccessible to all those in need. A physician we consulted with said the following: 

“Despite Phase I and II data showing significant promise not to mention significant anecdotal experience from clinicians across the country (and verifiable data from the EAP), the FDA insisted that a randomized, placebo-controlled design be used for this trial. This is unheard of in high grade glioma due to significant ethical concerns. However, the FDA felt there was ‘no standard of care at this time and thus placebo control is appropriate.’ In this day of customized, precision medicine, when the WHO 2021 criteria has entirely changed the way we diagnose these tumors and how we target them with agents, this seems at best a naïve, uninformed assumption.  Patients would not go to their oncologists and be offered ‘nothing.’ They would get temodar if methylated. If they have concurrent BRAF mutations, they will get targeted therapy. They would at least get bevacizumab and cytotoxic chemotherapy. We would not just direct a high functioning individual with a family, hopes, dreams, plans, and goals – while they’re feeling well (which is often the case with our patients when they are diagnosed) – to simply go home and wait to die because ‘there is no standard of care.’

Yet, this is what the FDA assumes when it requires a placebo-controlled trial. They expect the physicians on the ground to offer placebo, especially when there is constantly new data, new articles, anecdotes of the notable benefit of ONC201 in this patient population. The FDA does not have to have these conversations with these patients and their families. 

As a clinician I am being asked to offer a double blinded (with no plans to unblind) placebo-controlled trial to this patient – especially when I have seen ONC201 work in patients, give them their lives back, shrink the tumor, improve clinical and functional status. Meanwhile, drugs with a smaller body of literature and much less data (and much more side effects, including life threatening side effects and death) are getting FDA approval because of larger, more powerful lobbyists and money. DMG is rare – H3K27M DMG is even smaller. This translates to a smaller population of voices – but this does not make them less worthy of treatment, especially when we’re considering something that is potentially lifesaving and life extending.”

This trial invites patients with a rare, incurable brain cancer to take part in a program where they may or may not receive a life-altering treatment. One-third of participants will not receive the drug but will instead receive a placebo.

If the existing trial data holds such promise, why can't we offer deserving patients the treatments that could extend their lives and provide hope to those who need it most?

The FDA requires more - more participants, more data. Other treatments have been approved with less.

Time is critical for us and families facing similar circumstances. Waiting for more patients to enroll in the trial is not an option. Time is precious for these patients, for Steve, and for all the children, partners, siblings, friends, and families affected by this devastating disease.

We urge the FDA to immediately approve and grant ONC201 access for all individuals diagnosed with H3k27m so that we can all have the precious gift of time.