Allan Appel Photo
Smith makes her case.
In the early 2000s, a Yale scientist discovers why cutting your arm or finger can result in nerve cells regenerating, but not so if your injury is in the spinal column. He figures out how to inhibit the inhibitors of regeneration for the central nervous system.
In 2010, a company is formed to research the drug, and it passes tests with mice, rats, and monkeys.
In early 2019, human clinical trials are all set to go, giving hope to nearly 17,000 people a year — many young adults — with paralyzing spinal injuries, for whom there’s no reversing treatment, only physical therapy.
Then comes the federal government shutdown.
Today, on the cusp of submitting for Federal Drug Administration (FDA) approval to proceed to human trials, the company has discovered that due to the shutdown, 41 percent of the FDA isn’t reporting to work.
What’s worse, due to the shutdown the FDA staff is no longer accepting submissions for an “IND,” or investigational new drug, the process for a pharmaceutical company to get permission to move ahead to human trials.
Erika R. Smith, CEO of ReNetX Bio, Inc, told that story Wednesday to Connecticut’s two U.S. Senators, Richard Blumenthal and Chris Murphy.
The solons came to the company on the 19th floor of the financial building at 157 Church St. for a press conference a day before they return to Washington. On Thursday the senators are due to vote on two bills which, they said, give at least some minimal hope to move the government and its agencies out of legislative paralysis.
Blumenthal and Murphy Wednesday at ReNetX Bio.
Smith’s company consists of five staffers in the business office high above the Green plus dozens of scientists and lab connections around the country sub-contracted by ReNetX Bio to work on the project.
She reported that the company has received hundreds of emails from hopeful patients and their families dealing with paralysis of an individual — often a young man — from athletics or an auto accident. The contacts come her way from blogs on new research in the field of spinal cord injuries as well as from people who have been following the work of Yale School of Medicine’s Dr. Stephen Strittmatter. His pioneering neurological research in the early 2000s is the scientific basis for the company, and its drug — an injection into the lumbar area of the spine — called AXER-204
Smith called the situation “heartbreaking” not only for people who communicate with her company for hope, but also for a start-up company that has surmounted fundraising, legal, and testing hurdles with positive results in animal trials,now to have to overcome the unnecessary barriers resulting from the government shutdown.
“Every day this goes on puts uncertainty in our minds,” she told the senators. “We work with five clinical sites who are poised to go with human trials across the country, but they cannot hold that space forever.”
Then there are also materials that have a shelf life, and the salaries of the employees and subcontractors to pay.
“We are concerned with peoples’ lives,” she said.
The FDA’s inaction has resulted in the company’s having to come up with a back-up plan: to explore proceeding in Canada or another country. Smith said she hopes it won’t get to that point.
“Are there competitors in other countries?” asked Blumenthal.
“We want to believe we’re it,’” replied Smith. “But we’re not giving up. We’re committed to making this happen.”
“If investors are faced with regular FDA shutdowns , they’ll take their investment dollars elsewhere,” Murphy added.
“At Least Release These Hostages”
Smith with company Chief Science Officer George Maynard.
“We’re asked what is the damage: of the shutdown, Blumenthal said, “and it’s not just to the TSA. It’s a ripple effect for the whole economy. In Connecticut, biotech is part of our DNA. We don’t have gold or diamond mines. We have smart people who are inventing things.”
She said that even after the drug goes to human trials, those trials will last two years approximately with the aim to verify safety and efficacy in humans.
Even then, results might not be miraculous. However, as a result of this new treatment, if someone who can’t move a muscle, is now able able to feed or dress himself, that counts certainly as a minor miracle. Currently the only therapy for paralysis due to spinal cord injury is physical therapy, Smith said. About 300,000 people nationwide have spinal cord injuries.
“Life-transforming medicine that can change lives should not be hostage” to the shutdown and the president’s demand for a wall, Blumenthal concluded. “When life-saving drugs can’t come to patients, it’s unconscionable.”
He and Murphy both offered sober if slim hope that tomorrow’s votes on Senate bills might begin a dialogue leading to the opening of the government and its agencies like the FDA, while a larger more comprehensive bill on immigration is wrangled over.
“What does this company,” Murphy asked rhetorically, “have to do with building a wall! Why is [President Trump] not funding the FDA and other agencies?
“At least release these hostages.”
“I feel they heard us,” Smith said of the visit. “I’m very appreciative. They understand the wider implications.”
The FDA's drug approval folks are funded by fees paid by the drug companies. Congress should authorize the FDA to continue to collect those fees, and use them for their intended purpose, just as the TSA and major National Parks can be funded through user fees. "FDA will continue to support activities funded by carryover user fee balances, which allows us to continue to bring new therapeutic options to the patients that need them. However, during the lapse period, the FDA will not have legal authority to accept user fees assessed for FY 2019 until an FY 2019 appropriation or Continuing Resolution for the FDA is enacted. This will mean that the FDA will not be able to accept any regulatory submissions for FY 2019 that require a fee payment and that are submitted during the lapse period." https://www.fda.gov/AboutFDA/WorkingatFDA/ucm629100.htm