THINK BIG

As stem cells emerge as treatments for a variety of currently incurable diseases, Canada needs a co-ordinated strategy to score economic goals

time was slipping away for Dan Muscat, a 49-year-old St. Thomas, Ontario jeweler who has systemic scleroderma. The rare and painful autoimmune disease hardens the skin before invading the internal organs, eventually leading to death.

Energy had never been a problem for the self-motivated Muscat who, aside from running his jewelry business, loves fishing, collects sports cars and built his own log home. Then scleroderma struck him so hard he could barely get out of bed. It turned his skilled hands to stone.

Muscat underwent an experimental stem cell transplant at The Ottawa Hospital over the summer that could extend and significantly improve his life. So far so good: “I feel optimistic again,” he said after his release from hospital.

For Muscat, stem cells signify -- at the very least -- hope and -- at the very best -- a second chance at life. He is far from alone in his search for stem cell treatments for conditions that continue to frustrate modern medicine. People across Canada have been touched by the story of Jonathan Pitre, the remarkably upbeat 16-year-old Ottawa boy who traveled to Minnesota for a stem cell treatment to spare him from some of the ravages of his skin-blistering epidermolysis bullosa (EB). At the time this was written his first stem cell transplant had failed to engraft and he and his mother were pondering whether to attempt a second one.

James Price, president & CEO of the Canadian Stem Cell Foundation (CSCF), has a twoand-a-half-inch- thick stack of email printouts on his desk, each sheet representing someone seeking help for themselves or a loved one for conditions or diseases ranging from Alzheimer’s to spinal cord injury. “And those are just the email inquiries from the past 14 months,” says Price, who has been working to advance stem cell research and development in Canada for the past 14 years. “Then there are the many phone calls and enquiries and comments left on our blog each week.”

Most often, responding to these pleas for stem cell help means explaining that the science isn’t there yet, which comes as cold comfort to someone who is suffering a severely life-limiting disorder. But little by little, things are inching toward clinical trials for a variety of diseases for which there currently are no cures.

Muscat contacted the CSCF in January, desperately seeking help.

“Fortunately, we were able to put him in touch with Dr. Harry Atkins at The Ottawa Hospital,” said Price, “because we know Harry’s doing remarkable things with autoimmune diseases like multiple sclerosis (MS), Crohn’s disease and Stiff Person’s Syndrome.”

Dr. Atkins, whose June Lancet paper described his transplant team’s success with MS, had never treated a scleroderma patient but was intrigued.

Muscat underwent an experimental stem cell transplant at The Ottawa Hospital over the summer that could extend and significantly improve his life.

Dan Muscat with Brenda Allen (sister)

“The Government can see how the Strategy would not only lead to cures and save lives, but also generate thousands of good jobs and boost the economy.”

— James Price, President & CEO of the Canadian Stem Cell Foundation (CSCF)

“There was a randomized trial in Europe that had very good results,” says Dr. Atkins. “I know the doctors involved and their methods have been published.”

After five months of treatment, including several weeks in hospital and plenty of physiotherapy, Muscat reports increased range of motion and significant lessening of the pain, which previously was “like hitting your finger with a hammer, where you want to put your hand between your legs and cry.”

Dr. Atkins, however, urges caution. “We got him through this (the stem cell/chemo combination treatment) and will have to wait and see until his rheumatologist assesses him. This is not a curative treatment. It doesn’t make scleroderma go away and never come back.”

Not every systemic scleroderma patient is a good candidate for the stem cell treatment, especially if their lungs and other internal organs have been affected. Still, a treatment is now possible for some, which is better than being told to go home and get your affairs in order. There is also evidence that stem cell transplants can induce sustained remissions in patients with other severe autoimmune diseases such as lupus, rheumatoid arthritis and juvenile arthritis. At Northwestern University in Illinois, Dr. Richard Burt is investigating stem cell treatments for more than 20 autoimmune diseases. He, too, has had some success treating MS.

Looking beyond autoimmune disorders, stem cells are now being explored -- with promising early results -- as treatments for baffling diseases such as amyotrophic lateral sclerosis (ALS or Lou Gehrig’s disease) in the U.S. Here in Canada, stem cells are being tried to treat septic shock, osteoarthritis and to prevent scarring after heart attacks. Trials are underway in Alberta to test the use of progenitor-cell implants to eliminate the need for insulin injections for diabetics. In the U.S., Japan and the United Kingdom, stem cells are being tried as a sight-restoring treatment for macular degeneration.

Dr. Duncan Stewart, head of the Ontario Institute for Regenerative Medicine, likens the current state of cell and gene therapy to the early days of the personal computer when “it was very frustrating -- you did it for fun, really, for word processing, but it was hardly earth-shaking. Now you look at a PC and it can do what NASA used to do with a great big mainframe. A smartphone can do absolutely everything. The technology has evolved to the point where it’s changed the whole landscape.”

Dr. Stewart, himself a leading researcher (he heads the aforementioned heart attack study), would like to see more movement from research labs to clinical trials “in a safe and responsible way, to get the experience in the context of the patient population that has the disease. Because you can never model all this completely in the lab.”

The problem with clinical trials is they are very costly. And, says the CSCF’s Price, it can be hard to get big pharma interested in investing in stem cell solutions that often involve a process, like the treatments Muscat received, and not a product.

“While there are definite drugs and products waiting to be developed, there is no Viagra equivalent that will earn hundreds of millions of dollars,” says Price. “That’s why, to make this happen, we need to think differently -- and to think big.”

The CSCF led the coalition of researchers, clinicians, industry partners, health charities and philanthropists that crafted the Canadian Stem Cell Strategy, a $1.5-billion plan to deliver five to 10 curative therapies to the clinic within 10 years. Already one-third of the funding has been pledged from philanthropists and non-federal sources.

The CSCF has approached the Federal Government to integrate the Strategy into its Innovation Agenda and provide one-third of the funds -- roughly $50-million a year for 10 years. So far, says Price, cabinet ministers and ministerial executives have been receptive to the idea, which has been encouraging. “The Government can see how the Strategy would not only lead to cures and save lives, but also generate thousands of good jobs and boost the economy.”

The potential economic impact could be as attractive as the treatments and cures that could come from intensified investment in stem cell/ regenerative medicine R&D. Bloomberg News reported in July that Japan is preparing to tap a global market for regenerative medicine that will grow to $120-billion by 2030 and set off “a medical and industrial revolution.” The Centre for the Commercialization of Regenerative Medicine (CCRM) predicts the Strategy could generate in excess of 12,000 jobs for Canadians via the growth of existing companies and the development of new enterprises.

Recent investments suggest the Trudeau Government recognizes the potential, with a $20-million commitment to the CCRM to advance cell manufacturing and a $12-million, two-year extension to the Stem Cell Network that synergizes the efforts of stem cell researchers across Canada. Looking forward, the Government has asked the Council of Canadian Academies to study the state of regenerative medicine in Canada and report back in early 2017, identifying “the opportunities that exist and barriers that must be overcome for Canada to ensure that it can excel in regenerative medicine in the international arena.”

The stem cell community asserts that the time to act is now. A September iPolitics piece declared that “Canada has a deep well of innovative talent in a range of sectors. But there is fierce international competition for Canadian expertise and the jobs and economic rewards that flow from cutting-edge ideas; just look at the 250,000-plus Canadians living and working in Silicon Valley. The pressure to move quickly and effectively — to avoid being left behind — is enormous.” Along with Price, the iPolitics piece was co-authoured by Dr. Molly Shoichet, named the 2015 North American winner of the L’Oréal-UNESCO Awards for Women in Science, and Peter MacKinnon, former president and vice-chancellor of the University of Saskatchewan.

Ultimately, though, the true beneficiaries of the Strategy and a co-ordinated, big-think approach to stem cell/regenerative medicine R&D will be people like Dan Muscat and the millions of others like them across Canada and around the world looking for a way to get better.

“I know I still have the remnants of the disease,” said Muscat, “but I want to regain everything I had before. I want to get back to my business. I want to spend time with my son. And I have a lot more fishing to do. I feel like I have a second chance at life.”

To see this story online visit http://biotechnologyfocus.ca/comes-stemcells-canada-needs-think-big/