One of the most exciting areas in healthcare today is regenerative medicine. This simple term encompasses a rapidly expanding revolution in medical research, which in its application by physicians is allowing the body to heal itself in ways never before seen.
Hundreds of companies and universities around the world are working on regenerative technologies to cure leukemia, hemophilia and other devastating diseases or repair injured organs and tissues such as spinal cords, retinas and hearts.
Of the 946 regenerative clinical trials going on at the end of 2017, more than half were in cancer. Nearly 10% were related to heart disease and 6.5% were addressing diseases of the brain and spinal cord.1
By exploring the mysteries of DNA, scientists are successfully engineering stem cells and tissues that are then injected into patients and actually fight diseases at their roots or "regenerate" tissue to heal patients fundamentally advancing medicine beyond its traditions of just treating symptoms.
FDA promises support
The Food and Drug Administration is promising to support accelerated paths for product approvals in many fields.
"We're at a key point when it comes to cell and gene therapy. These therapies have the potential to address hundreds, if not thousands, of different rare and common diseases," FDA Administrator Dr. Scott Gottlieb told the Alliance for Regenerative Medicine's (ARM) annual board meeting in Washington on May 22.
"For a long time, they were largely theoretical constructs. Now they're a therapeutic reality. And it's my expectation that they will soon become the mainstay of how we treat a wide range of illnesses."
In the fall of 2017, the FDA for the first time approved not one but two of the new gene therapies for general use.
Novartis's Kymriah, used to attack leukemia, became the first gene therapy available in the United States. That was followed by Kite/Gilead’s Yescarta to fight non-Hodgkin's lymphoma. Both involve genetically altering a patient's own immune cells to fight a disease, known as CAR T-cell therapies.
No longer science fiction
ARM CEO Janet Lambert says 2017 was groundbreaking for the sector and trends continue to be strong. Global financings for IPOs in the sector at the end of first quarter 2018 were already 90% of the investment seen for all of 2017. First-quarter mergers and acquisitions were also strong, nearly 70% of full-year 2017.
"It's scientifically disruptive that we can now tackle the cause of the disease," Lambert says. "Once these products get to market you’re in a whole new world, greatly expanding their access beyond the limited number of patients participating in trials."
"Some of the most exciting are the CAR-T focused therapies and cell-based immune oncology," she says, adding there has been much success in fighting "liquid" tumors such as blood-based cancers. Hopes are high that regenerative medicine will soon be successful battling "solid" tumors like those of the lung, prostate, breast and colon.
"These concepts are no longer the stuff of science fiction, but rather real-life science," Gottlieb said at the time of the first 2017 approvals.
Big pharma and biotech deals
The applications of this scientific revolution have grabbed the attention of big pharma in particular. Just from August of last year to this April, there have been three multibillion-dollar acquisitions centered on regenerative medicine made by Gilead, Celgene and Novartis.
Swiss drugmaker Novartis’s purchase of AveXis, an Illinois biotech company, closed in May for $8.7 billion—a 72% premium to AveXis’s 30-day volume-weighted average stock price.
These therapies have the potential to address hundreds, if not thousands, of different rare and common diseases.
"There's significant excitement about the potential of cell and gene therapies to transform the treatment paradigm for several diseases, and that's reflected in these valuations," says William
Blair analyst Raju Prasad, who covers biotech companies including AveXis.
Novartis was particularly interested in AveXis’s new gene therapy to treat spinal muscular atrophy (SMA), a disease that prevents the body from producing muscle and is the leading genetic cause of infant deaths.
AveXis engineered a gene that is missing in children suffering from SMA, then delivered it to them using a virus. Of the 15 patients in the Phase I clinical trial, all were alive after 24 months and the young children achieved many important motor nerve milestones after getting treatment.
AveXis is currently enrolling a Phase III trial, with early results from that study presented at the American Academy of Neurology annual meeting in April 2018, corroborating the impressive results seen from the Phase I trial.2
Revolution in its early stages with hurdles ahead
Such promise of medical miracles drives both the basic science and the applied medicine of regenerative therapies. Scientists say the revolution is still in its early stages with next-generation therapies primed for FDA-approved clinical trial.
Practical hurdles remain. Hopes are soaring—but so are costs. How many people can pay for a million-dollar injection? What will be government’s role? For now, Gottlieb promises a sharp eye to protect consumers and researchers.
The FDA is cracking down on touted but unregulated and unapproved procedures or claims it says put patients at risk, as it did at stem cell clinics in Florida and California in the past year.
"This field is moving really fast but there’s still going to be a lot of challenges and missteps along the way," said Matt Phipps, a William Blair analyst who covers the biotech sector.
"People get very exuberant on the prospects. But at the end of the day, biology is really complex. On top of that, the thing people worry about is our healthcare system really isn’t set up to pay for one-time cures," he adds.
Still, challenges on how treatments will be paid for have not yet slowed interest in the burgeoning regenerative medical revolution.
"We're just beginning to scratch the surface that this sector is going to be able to deliver for patients and families," Lambert says. "The most profound story is curing one individual at a time."
1 Alliance for Regenerative Medicine
2 AveXis study