Foundation Aims For Virtual Trial Registry to Help Patients With Brain Cancer Get Drugs Sooner

The approval of new agents to treat cancer can take years to pass all the regulations of the Food and Drug Administration (FDA), but for patients with brain cancer there isn’t time to wait.

Al Musella, D.P.M., understands this firsthand as he lost both his sister-in-law and father to glioblastoma (GBM) — an aggressive and deadly form of brain cancer. He watched as they struggled to pay for the treatments that were keeping them alive.

Their journeys led him to become a patient advocate in 1992, then forming The Musella Foundation For Brain Tumor Research & Information, Inc., a nonprofit organization, in 1997.

Since then he has helped countless members of the brain cancer community gain access to co-payment assistance, clinical trials and support groups.

But it’s his latest project that sets The Musella Foundation apart from other groups which are advocating on behalf of patients with brain cancer. His goal: Gain conditional approval from the FDA with mandatory participation in a “virtual trial.”

“I see the pace of this progress and it’s going way too slow,” said Musella in an interview with CURE. “I see how things are heading. For instance, trials started 10 years ago and we still can’t get those drugs yet.”

He went on to explain that the groups of patients in those trials did very well. “Over 20 percent of the people in those trials are still alive right now, seven to 10 years later. That’s unheard of,” said Musella. “And these people are holding down real jobs.”

Musella’s plan for the Brain Tumor Virtual Trial Registry is to get patients access to more drugs at a faster pace, while collecting treatment and efficacy information that can later be used by oncologists across the country to treat others.

For the Brain Tumor Virtual Trial Registry to work, conditional approvals would have to be proven safe for standard treatments in at least 50 to 100 patients and have shown biologic activity — an improvement in a biomarker, brain scan, progression-free survival or overall survival.

Patients would be required to participate in the registry for the duration of the conditional approval. The patient’s oncologist would be responsible for submitting treatments, side effects, testing results, dates of progression or death.

Oncologists would then be able to access this information so they can evaluate the risks and benefits of the conditional treatment and combinations of treatments.

“A doctor could look into the virtual trial database to see which treatments are being used for a specific profile, which combinations have worked and, if a patient is on two drugs, which third drug would be good to add to the combination,” he said.

The FDA would conduct periodical reviews, according to Musella. In addition, the conditional approval would be removed if the side effects become too severe. But, if the drug is seen to be safe and effective, the FDA could upgrade it to a full approval.

Another aspect of the registry is that it would eliminate the limitations that exist currently in clinical trials. For example, patients must meet certain criteria before they are allowed to participate in clinical trials, such as being a specific age or having a distinct tumor size. Musella said that his virtual trial registry would represent all patients, even those who are excluded from trials, like patients with large or multiple tumors, or those who have cancer plus other health issues.

Safety is a major concern for the FDA, and Musella agrees, explaining that there needs to be steps in place to keep patients protected.

“If you have a 95 percent chance of dying, most patients would be willing to accept a 1 to 2 percent chance of a side effect that you didn’t foresee,” said Musella.

In addition, Musella explained that this plan would reduce costs of drugs and create competition. “If you have five drugs that got the same results, the companies could compete on price. We don’t have that now,” he said.

He feels timing is crucial and with President Donald Trump’s goal to develop drugs faster and make them less expensive, in addition to hiring a new FDA Commissioner, Musella said that now is that perfect time.

“I think with our plan, and the whole key to the virtual trial, is the conditional approval and to be able to get to the results immediately,” said Musella.

Musella presented the plan to the FDA in June. However, the administration feels, at this time, it’s too radical and may not be legal without writing a new law. The FDA asked Musella to start with a smaller step, which he already has in place.

He will soon be presenting another proposal to the FDA that calls for accelerated approval of what he calls “one of the most promising experimental drugs.” Musella hopes that beginning here will lead him to his ultimate endpoint: getting life-saving drugs to patients in the quickest and safest way possible.