Children are suffering from rare diseases, a new law will help

• Senator Josh Harkins writes that a new law designed to allow for experimental gene therapy treatments could have a significant impact for patients who have exhausted other options.

Today in Mississippi, thousands of our friends and neighbors are suffering from a rare disease or a serious form of cancer. About 30 million Americans – half are children – are afflicted by rare diseases for which there are no treatments or cures.

Such conditions include: L-CMD (a rare form of muscular dystrophy); Barth Syndrome; Fabry Disease; and Batten Disease. Unfortunately, there are many more. And, of course, many of us have a close relative or friend who has died from cancer.

On behalf of these children and cancer patients, I worked with my colleagues in the Mississippi Legislature to pass a bill (SB 2858) that creates new options for patients seeking treatments that are out-of-reach because of bureaucratic red tape.

This innovative concept is also law in Arizona and Nevada, where patients suffering from rare diseases may access cutting-edge, personalized medical treatments. Thanks to SB 2858, these treatments will soon be available in Mississippi.

Medicine is making incredible breakthroughs, tailoring treatments based on our individual genes and DNA structure. It’s truly amazing, but these innovative ideas are only helpful if patients can actually utilize them.

Unfortunately, the federal government’s drug approval process isn’t designed for the latest medical advances. It’s a one-size-fits-all approach, based on an industrial model, that takes years for new treatments to get approved and, literally, costs hundreds of millions of dollars.

Here’s the problem. The U.S. Food and Drug Administration (FDA) requires new medicines and treatments to go through a series of clinical trials – tested on a large population suffering from the same disease – in order to receive approval. Those trials can take, on average, ten years to complete.

Rare diseases don’t afflict a large population, and individualized treatments are custom made for only one person. That means there aren’t enough people in a clinical trial to meet the FDA’s standards; and, of course, a treatment designed for one person can’t be tested on hundreds or thousands of people.

SB 2858 offers a tool in the fight.

There is already precedent for signaling to the FDA that a change is needed. In 2015, I sponsored a bill, called Right to Try, that provides terminally ill patients access to drugs and therapies that have been approved by FDA safety trials, but not FDA efficacy trials.

Today, for example, patients suffering from glioblastoma, a fast-growing and aggressive type of brain cancer, are using Right to Try to access a new treatment that is working.

Over the years, we updated Right to Try in Mississippi, providing momentum to pass a federal version in 2018, signed by President Donald Trump. We helped start a movement that is saving lives around the country. With SB 2858, we are doing it again.

With new advances in medicine, and the emergence of treatments that don’t fit into the FDA’s regulatory framework, SB 2858 expands Right to Try by allowing patients access to personalized gene therapies.

There’s a story out of Arizona that shows just why this law is needed in Mississippi. The Riley family have two daughters who suffer from a genetic brain disorder called metachromatic leukodystrophy (MLD). The disease is fatal, but there is hope in an individualized gene therapy if the patient receives treatment in time.

The therapy was approved by the European Union in 2020, but was not approved in the United States until recently. By the time the Rileys learned of treatment alternatives in Europe, it was too late for their second daughter, Olivia, to get help. But there was still time for her younger sister, Keira.

The Rileys did what many Mississippi families would have done. They raised hundreds of thousands of dollars and moved to Italy so they could access this lifesaving gene therapy for their daughter.

Their story is not unique. I personally know two people in Mississippi who have done much the same thing to get personalized cancer treatment in other countries, like Switzerland and Germany.

Today, the Riley’s third daughter, Keira, is a healthy little girl, thanks to the treatment she obtained in Italy.

Tragically, her sister, Olivia, is in hospice care and fighting to survive through childhood. Had the Rileys waited for the FDA to approve their daughter’s treatment, Keira would be suffering the same fate as her older sister. No family should be forced to suffer this tragedy or to travel across the ocean to receive lifesaving treatment that should be available in the United States.

We can’t let red tape stop our children, parents, family and friends from getting lifesaving care. Other states, and other countries, are using individualized treatments to offer hope to patients with cancer and rare diseases. I’m proud to say that here in Mississippi we are doing the same by enacting SB 2858.