RT..CONSUMER HEALTH: Regenetive Medicine Becoming More Mainstream
Last Update: 3/10/2011 9:02:27 AM
By Kristen Gerencher
A DOW JONES COLUMN
Two years ago, doctors closed a longstanding hole in Susan Dennington's right
foot using a skin graft manufactured from infant boys' donated foreskin.
Dennington, 52 years old, suffers from neuropathy, so she couldn't feel the pain
from her diabetic foot ulcer, but the gaping wound made it hard for her to
function. She spent much time and money keeping it clean and bandaged to protect
it from infection. Several infections happened anyway during the 10 years her
ulcer persisted, and there were times she thought the ulcer was finally going
away only to have it return again.
"It was completely frustrating," she said. "My wound was on my mind every day."
Her doctor, Quyen Ha of Ada, Okla., said at first Dennington was skeptical about
a wound-care product called Apligraf but was determined not to face amputation.
"My job as a wound doctor is to save as many legs as I can," he said.
So Dennington had several outpatient procedures to apply Apligraf, followed by
six weeks of recovery. While some patients with similar medical problems have to
come back for more procedures because of their underlying disease, she has not
returned for more treatments, said Ha, who doesn't receive any fees from the
product's maker, Organogenesis of Canton, Mass.
"Since I've had the Apligraf, there's been no treatment and it's never come
back," Dennington said of her foot ulcer. "My wound is closed and I haven't worn
a bandage in two years. That feels like freedom."
Dennington is the beneficiary of a quiet movement focused on shifting medical
care to living-cell-based therapies that stimulate the body's tissue to
regenerate and heal itself, as opposed to more traditional approaches focused on
managing symptoms. Regenerative medicine, as it's known, encompasses a variety of
scientific disciplines and includes stem-cell technologies, medical devices,
biotechnology and even pharmaceutical companies.
Hoping To Cure Wide Range Of Diseases
Some forms of it, such as orthopedic bone grafts and skin grafts for burn
patients, have been around for years. Apligraf itself has been on the market for
more than a decade. But interest in regenerative medicine is picking up as many
ambitious new therapies aim to overcome some of the biggest causes of illness,
disability and death, including heart failure, diabetes, Parkinson's disease,
spinal-cord injury and macular degeneration that can lead to blindness.
A few examples: Advanced BioHealing makes a similar product for diabetic foot
ulcers called Dermagraft. Last month, it filed for a $200 million initial public
offering. Another company, Tengion Inc. (TNGN), is developing methods to repair
failing organs such as bladders with a combination of biocompatible materials and
the patient's own cells.
Some traditional drugmakers want in as well. In January, pharmaceutical giant Eli
Lilly & Co. (LLY) and the Juvenile Diabetes Research Foundation announced they
are teaming up to fund research aimed at helping patients with type 1 diabetes
regenerate insulin-producing cells destroyed by the disease.
Investors Take Note
The market capitalization of 22 publicly traded companies in the cellular therapy
business hit $2.5 billion this year, up 40% from last year, said Robin Young,
chief executive of data firm Pearl Diver and organizer of the annual New York
Stem Cell Summit. He projects U.S. sales will grow to about $1 billion by 2015.
Cell-based approaches involve changing the course of diseases, said Morrie
Ruffin, managing director of the Alliance for Regenerative Medicine, a trade
group of 80 companies based in Bethesda, Md., that launched in 2009.
"One of the ways we're going to have to address the long-term cost increase we
face in health care is by focusing on innovation and getting out of this cycle of
looking at very small incremental gains in treating diseases to finding
transformative solutions," Ruffin said. "That's why regenerative medicine is so
exciting. It takes us in that direction."
The industry wants Congress to reintroduce the Regenerative Medicine Promotion
Act of 2010, which would provide federal funds for research and development and
streamline the approval process for products deemed safe and effective.
Meanwhile, Wall Street wants to see signs of clinical viability, said Mark
Monane, managing director of equity research at Needham & Co. in New York.
"Investors are not giving these companies the benefit of the doubt," he said.
"They trade at a discount relative to other like-stage companies. For the most
part, the jury is still out on whether this can be a product in the real world or
if they are doing a science experiment."
Even so, investors appear increasingly interested. In January, the Alliance for
Regenerative Medicine held its first annual "state of the industry" briefing
during the Biotech Showcase and the J.P. Morgan Healthcare Conference in San
Francisco. The turnout of attendees forced administrators to shift the event to a
bigger room as executives gave company overviews and took questions.
Tapping Embryonic Stem Cells
Earlier this year, the U.S. Food and Drug Administration cleared Advanced Cell
Technology Inc. (ACTC) of Marlborough Mass., to pursue a multicenter clinical
trial to treat dry age-related macular degeneration, which affects 10 million to
15 million Americans. The treatment involves inducing a cell line grown from
human embryonic stem cells to become retinal pigment epithelial cells, which sit
at the base of the retina on top of what's called the Bruch's membrane.
"It's the death of these RPE cells that leads to a variety of retinal disease,
the biggest of which is dry age-related macular degeneration," ACT Chief
Executive Gary Rabin said.
In the upcoming trials, eye surgeons will inject the new cells into patients'
diseased eyes with the aim of having the cells graft and reverse the damage in an
area of the body known for being insulated from typical immune responses that
would reject the new cells, he said.
Embryonic stem cells are much more malleable than adult stem cells, Rabin said,
and he expects ACT's method to gain acceptance no matter how the politics of
embryonic stem cells evolves.
In February, the company received a patent on an extraction technique called
"single blastomere" that doesn't harm the two-day-old embryos. Advanced Cell
Technology acquires them from vitro fertilization clinics with patients' consent,
uses the embryos to start cell lines and then returns them to the clinic for
their original purpose--for making babies or storing for the future.
"The extraction method we use is used in the birth of thousands of babies each
year using PGD [pre-implantation genetic diagnosis]," Rabin said. "We're not
changing the fate of the embryo we borrowed."
Another company, Kinetic Concepts Inc. (KCI), acquired a regenerative-medicine
outfit called LifeCell in 2008. One of its flagship products, Strattice
Reconstructive Tissue Matrix, used mostly for breast and abdominal wall
reconstruction, is changing how surgeons practice, said Dr. Ron Silverman, a
plastic surgeon and chief medical offer for Kinetic Concepts in San Antonio.
"LifeCell's approach has been to provide a natural matrix, like scaffold, and
allow your own cells go into the scaffold," he said. "Within a day there are
already cells within the product. When you have your own cells, there's no
rejection."
Strattice provides an alternative to the synthetic mesh often used in hernia
surgery, which can cause suffering and complications if infection develops, he
said. With plastic, a second surgery would be required to remove the mesh whereas
regenerative tissue doesn't require invasive correction, Silverman said. "We see
this as tremendously economically advantageous," he added.
It's an example of how regenerative medicine facilitates the body's ability to
grow new cells, Silverman said. "If you don't have regeneration, then the only
thing the body knows how to do is heal things by scarring. Regeneration allows
not just scarring but replacement of natural tissue."
For companies struggling to bring products to market, collaboration will be key,
said Monane of Needham & Co.
"There's so much opportunity to win," he said. "They're all at a stage where
they're learning from each other." |
