Category Archives: Colorado Stem Cells

CSU president Tony Frank: Well be moving things from the bench or laboratory into the hospital, from theory to practice, and patients from disease into health.

Ground has officially been broken on the new $US65 million translational medicine facility at Colorado State University (CSU), to be named in honor of acclaimed New Zealand-born equine arthroscopic specialist Wayne McIlwraith.

The ceremony took place on Friday at the site of the C. Wayne McIlwraith Translational Medicine Institute.

The facility, which required years of planning and record fundraising to reach this point, promises medical innovations by harnessing the bodys healing powers to help animals and people suffering from a wide range of diseases.

Dr. David Frisbie, the institutes interim operations director and a CSU professor of equine surgery, hailed the milestone event in his opening remarks.

As he welcomed those in attendance some 150 faculty, staff, clinicians and donors he described the journey that led to the groundbreaking near the Diagnostic Medicine Center.

This building will be a central focus of scientific advancement as well as research, Frisbie said. The teaching and technology resources will be a beacon to great minds so that they can come together in developing healing technologies for not only people but animals as well.

The facility is named in honor of a veterinarian who has built a remarkable clinical and research enterprise in orthopaedic medicine for horses during nearly 40 years at the university.

McIlwraith, a University Distinguished Professor and founding director of CSUs Orthopaedic Research Center, is an international pioneer in equine arthroscopic surgery.

He has also pushed the boundaries of research into biological therapies based on living cells and their products, including novel protein and stem-cell therapies that help heal injured and degraded joints.

Many of McIlwraiths findings regarding the diagnosis, prevention and treatment of equine joint injury and disease have been translated into orthopaedic advancements for people the succession known as translational medicine.

CSU president Tony Frank said the use of the word translational was an appropriate and important description of what would take place in the building.

Well be moving things from the bench or laboratory into the hospital, from theory to practice, and patients from disease into health, he said.

The word transformational also came up quite a bit in conversations with the lead donors, John and Leslie Malone, according to Frank.

The idea of changing something completely is a daunting one, he explained. With the new institute, the university would completely change the way we go after disease problems, and the way we put teams together, looking across biology and into engineering.

He continued: Changing something completely and making efforts this large are heady conversations. Theyre not new to the people who had the vision for this building, Frank said.

The Malones provided the lead gift of $US42.5 million to establish the research institute, prompted by their interest in the regenerative power of stem-cell therapies for horses and humans.

The Malones raise world-class dressage horses and Thoroughbred racehorses. They became intrigued by the concept of the Translational Medicine Institute after their horses at Harmony Sporthorses near Denver were successfully treated with orthopaedic procedures developed by McIlwraith and his colleagues.

John Malone said that he and his wife are fortunate to have the opportunity to support efforts such as the new research institute. This one, for us, really checked all the boxes: horses, education and research, he said. He added relentlessness, stem cells, and orthopedics to that mix.

As you get older, you appreciate stem cells and orthopedics, both in your horses and in your neck, in my case, he said.

Malone described the university as a practical, pragmatic place where researchers produced real-world results.

He also hailed the man after whom the building is named. If you could extract the source of Waynes energy and drive and put it in a bottle, that is an entrepreneurship Id invest in, he said.

Meeting McIlwraith and working with him had been one of the highlights of this effort, Malone added.

Adding to the Malones gift, Princess Abigail K. Kawananakoa of Hawaii, a direct descendant of the Hawaiian royal family and celebrated breeder of racing American Quarter Horses, donated the institutes naming gift of $US20 million.

McIlwraith has contributed to the success of Princess Abigails stable by supporting the orthopaedic health of her racehorses, inspiring her to give generously and to ask that the new facility be named for her longtime friend and colleague.

In his remarks, McIlwraith relayed his heartfelt thanks to the donors and acknowledged them as terrific philanthropists and visionaries.

The renowned surgeon said the idea for the institute was an evolutionary step beyond the work being conducted at the Orthopaedic Research Center, and would expand the mission and research focuses to cut a wider swath.

He said he was still getting used to the idea of having his name on the building.

Its an incredible honor, he said, choking up a bit with emotion. The thing thats touched me the most is all the people whove commented that its deserved or appropriate or they agree with it. Its humbling. I wasnt looking for a legacy, but I obviously have a fantastic one.

University officials estimate that the C. Wayne McIlwraith Translational Medicine Institute will open its doors in late fall 2018.

Reporting: Mary Guiden, Coleman Cornelius and Dell Rae Ciaravola

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Ground broken on new $US65 million facility at Colorado State University - Horsetalk

COLORADO SPRINGS, Colo. (Cheyenne Mountain Zoo) - The Zoos veterinary and animal care teams have utilized both stem cell transfusion therapy and custom-made urethane sneakers to treat giraffe here at the Zoo. The efforts are led by Dr. Liza Dadone, vice president of mission and programs and head veterinarian for Cheyenne Mountain Zoo.

Dr. Dadone and staff of the Colorado State University James L. Voss Veterinary Teaching Hospital were able to grow stem cells from giraffe blood to then inject back into the giraffe a treatment for giraffe that is believed to be the first of its kind in the world.

Giraffe "sneakers, Cheyenne Mountain Zoo."

Mahali, the 14-year-old male giraffe treated, suffered from chronic lameness and had not been moving well, despite a number of medications and additional treatments the animal care and veterinary teams gave him. Dr. Dadone decided on a ground-breaking stem cell injection treatment plan. In scientific studies, stem cell therapy has proven to repair damaged tissue at the cellular level.

Its been nearly a month since the procedure, when Dr. Dadone and the Zoo team, along with the partnership of the CSU veterinary medicine program, injected Mahali with around 100 million stem cells. The success of the procedure was determined by Dr. Dadone when she reviewed and compared thermographic images taken of Mahalis front legs before and after the procedure. The photos show a considerable decline in inflammation in Mahalis front left leg, which is the one he had been having issues with for some time.

This is meaningful to us not only because it is the first time a giraffe has been treated with stem cells, but especially because it is bringing Mahali some arthritis relief and could help other giraffe in the near future, Dr. Dadone said.

Dr. Dadone said she is not sure if Mahalis positive results are simply due to the stem cell therapy or are a combination of different treatments, but shes pleased and assured his quality of life has dramatically improved.

Prior to the procedure, he was favoring his left front leg and would lift that foot off the ground almost once per minute, Dr. Dadone said. During the immobilization, we did multiple treatments that included hoof trims, stem cell therapy and other medications. Since then, Mahali is no longer constantly lifting his left front leg off the ground and has resumed cooperating for hoof care. A few weeks ago, he returned to life with his herd, including yard access. On the thermogram, the marked inflammation up the leg has mostly resolved.

Another Zoo giraffe, 14-year-old female Twiga, has advanced arthritis and osteoporosis in her feet. Dr. Dadone and the veterinary team have been monitoring and treating her condition for some time, but were hopeful when they heard of a farrier specialist who had an idea to make custom shoes for her. Weve had Twiga on medicine to help reverse her osteoporosis, but we wanted to do more to protect her feet. So with the help of the farriers, we gave her giraffe sneakers to help give her some extra cushion, said Dr. Dadone.

To get the sneakers onto Twigas feet, the keepers cued Twiga to place her hoof on a specially-designed hoof block, then farriers Steve Foxworth and Chris Niclas of the Equine Lameness Prevention Organization (ELPO) did a routine hoof trim to the foot, a procedure Foxworth performs monthly. Once her foot was clean and ready, the shoe was placed on her sole by Niclas with quick-drying glue. The sneakers are divided on the undersides and were designed by Niclas to adjust to Twigas individual digits.

Dr. Dadone said the change in Twigas behavior was immediate. Twiga instantly shifted her weight off of her right foot, indicating she was comfortable and her pain had considerably lessened. The shoes help to stabilize Twiga and will likely stay on for around six weeks. Dr. Dadone says they will reassess Twigas progress at that time. She is eager to share information regarding this treatment option so that other veterinary teams at fellow zoos can use this technique to help benefit their animals as well.

Large animals like giraffe are susceptible to issues like arthritis and osteoporosis, mainly stemming from their sheer size. Like all animals, these issues are exacerbated as they age. So much of it just relates to the pure mechanics of weighing a ton, Dr. Dadone said.

Other regular veterinary treatments include X-ray imaging, laser therapy, hoof care and more.

Cheyenne Mountain Zoo is not only a leader in the training and health of giraffe in human care, but they are also making a huge difference in conservation of giraffe in the wild. The status of giraffe was recently changed by the International Union for Conservation of Nature (IUCN) from least concern to vulnerable, acknowledging the fact that their population in the wild has plummeted by 40 percent in the last 30 years.

Last year, Cheyenne Mountain Zoos guests and members used their Quarters for Conservation (Q4C) admission contributions to send $26,000 to the Giraffe Conservation Foundation (GCF) and its efforts to help the Rothschilds giraffe in Murchison Falls National Park in Uganda.

Cheyenne Mountain Zoo is home to the worlds most prolific captive reticulated giraffe herd, with 199 births at the Zoo since 1954. Guests can get up close and hand-feed them on special indoor and outdoor elevated platforms anytime during the day, 365 days a year.

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Giraffe 'sneakers' in use at the Cheyenne Mountain Zoo! - KKTV 11 News

Caption + Shutterstock By SebGross

In two separate studies, researchers have successfully created blood stem cells in a laboratory setting for the first time. These types of cells are found in bone marrow and can be depleted by diseases like leukemia and even by the treatments for those diseases, such as chemotherapy.

George Daley, Dean of the Faculty of Medicine at Harvard, and his team started with pluripotent stem cells, which can give rise to just about any type cell in our anatomy. By looking at what proteins controlled the genes in bone marrow cells, they were able to isolate several that were essential to cell differentiation (the process by which stem cells become a specific kind of cell). They then applied them to the pluripotent cells in order to encourage them to turn into the cells found in bone marrow.

Another team lead by Raphael Lis, Instructor in Medicine at Weill Cornell Medical College, took cells from the lungs of animals, and found four factors that encourage the lung stem cells to make blood stem cells. In their report they demonstrate a tractable approach for fully reprogramming adult mouse endothelial cells to haematopoietic stem cells. The next steps for Lis are to streamline the conceived [] reproducible approach to manufacture engraftable durable blood cells, so they can be produced on a larger scale.

Read the full story at Futurism.com.

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For the first time ever, researchers produced lab-grown blood stem cells - Colorado Springs Gazette

Most of us like to give gifts to others. Sometimes theyre small or sentimental-but they can be once in a lifetime events. At the top of the list is the gift of life, and a UTC student had the chance to do that. Page McCoy had developed leukemia-some didnt give her much hope.

M.C. She needed a stem cell transplant and fortunately there was exactly one match in the world. And it was a donor in the Gift of Life register. Her transplant took place in January 2016 in Denver. Colorado. She had a long and difficult recovery but today she is here and she is real excited to meet the donor who saved her life.

That was the way this story unfolded last week in New York as the Gift of Life foundation honored Americans who were registered donors and the people they helped. The donor in this case was a 22-year old student at UTC who registered two years ago in Knoxville two years ago, and promptly forgot all about it. Thomas Davis was surprised to get the call but he didnt hesitate to go through with the STEM cell transplant.

THOMAS DAVIS, UTC STUDENT AND DONOR Followed all the steps, and eventually I was outside D.C.with two needles in the arms, donating the stem cells.

Thomas was invited to the Gala last week where he would actually meet Page for the first time.

(WALKS UP AND HUGS HER )

THOMAS DAVIS out of nowhere, hearing, o.k. Shes on track to recovery..its awesome and the opportunity to actually meet heruhit was wonderful.

Both Thomas and Page McCoy got to make a few comments on stage.

THOMAS Thank you, to the generous men and women whose financial contributions make Gift of Life possible, really..I wouldnt be on stage without you guys, we wouldnt be here together if it wasnt for you guys who made Gift of Life a reality.

PAGE MCCOY, STEM CELL RECIPIENT My donor gave me back my life..but he also gave me my future. Im leukemia free and 100% engrafted because of himapplause.

Thomas wants to build that donor list for Gift of Life.

THOMAS Theres nothing to be afraid of..no reason not to.

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Chattanooga Student Surprised to be a Gift of Life Donor - WDEF News 12

FitzGerald, Susan

doi: 10.1097/01.NT.0000520472.01901.8f

Features

Two new reports on autologous hematopoietic stem cell transplantation (AHSCT) for multiple sclerosis (MS) indicate that the therapy may benefit some MS patients. But whether AHSCT is viable is a matter of debate among some MS experts, who contend that the regimen could be toxic, leading to infection and death.

Two new reports on autologous hematopoietic stem cell transplantation (AHSCT) for multiple sclerosis (MS) indicate that the therapy may benefit some MS patients. But whether AHSCT is viable is a matter of debate among some MS experts, who contend that the regimen, which uses a combination of cytotoxic drugs to ablate the immune system in an attempt to reset the immunological memory could be toxic, leading to infection and death.

Experts who were not involved with the study said that newer, second generation MS drugs may be safer options, though few studies comparing the method with these drugs have been undertaken.

The first new report, published in the April 28 online edition of Neurology, provided a meta-analysis of 15 studies involving 764 MS patients who underwent AHSCT. The report found that the risk-benefit profile of the therapy makes it best suited for patients who have aggressive, relapsing-remitting MS who have not yet become highly disabled.

The second report, which provided long-term outcomes for 281 MS patients from an observational, retrospective study, found that almost half of the patients remained free from neurological progression five years after AHSCT. The study, published in the April edition of JAMA Neurology, reported that younger age, relapsing form of MS, fewer prior immunotherapies, and lower baseline EDSS [Expanded Disability Status Scale] score were factors associated with better outcomes.

Maria Pia Sormani, PhD, professor of biostatistics at the University of Genoa in Italy, and lead author of the report in Neurology, told Neurology Today that skepticism about the treatment approach is likely due to multiple factors.

MS is not a lethal disease, and this procedure is very invasive and has a non-negligible mortality risk, said Dr. Sormani, who also was a study author on the JAMA Neurology study. The lack of data from a rigorous clinical trial of AHSCT for MS has also been problematic.

To gain a clearer picture of what the current evidence shows, her team's meta-analysis pooled data from 15 studies, mostly open label, from January 1991 to July 2016. The researchers found that treatment-related mortality (TRM) declined during the period covered by the review, likely a result of improvements in transplant techniques, more clinical experience, and better patient selection, Dr. Sormani said. Overall TRM was 2.1 percent, but after 2005 it was 0.3 percent.

The meta-analysis found that the rate of disease progression in patients was 17.1 percent at two years following AHSCT and 23.3 percent at five years. The analysis also found that 83 percent of patients had no evidence of disease activity (NEDA) at two years, and 67 percent had no evidence at five years. Doing the transplant earlier, before the patient develops much disability seems advantageous, Dr. Sormani said.

The meta-analysis had the usual limitations of such reviews, she noted. The original studies were not all designed or executed in the same way, patient selection and study methodology were not uniform, and transplant techniques and protocols varied.

Even with advanced immunotherapy, such as natalizumab or alemtuzumab, only 32-39 percent maintained NEDA at two years in the phase II clinical trials, wrote Joachim Burman, MD, PhD, of Uppsala University in Sweden and Robert Fox, MD, of the Cleveland Clinic, in the editorial accompanying the paper. They agreed with the research team that the approach is more likely to benefit those with RRMS, not those with progressive forms of MS.

The report in JAMA Neurology included data on 281 patients from 25 centers who underwent AHSCT between January 1995 and December 2006. Seventy-eight percent of the patients had progressive forms of MS. The median follow-up was 6.6 years, with some patients followed for as long as 16 years

The five-year probability of progression-free survival was 46 percent and overall survival was 96 percent, the research team headed by Paolo A. Muraro, MD, a clinical reader in neuroimmunology and deputy head of the division of brain sciences at Imperial College London.

Factors associated with neurological progression after transplant were older age, progressive (versus relapsing) form of MS, more than two previous disease-modifying therapies, and higher baseline EDSS scores.

An accompanying editorial coauthored by Michael K. Racke, MD, professor of neurology and neuroscience at Ohio State University, noted that while the transplant therapy appears to favor those with RRMS with aggressive breakthrough disease, it Z

Dr. Racke told Neurology Today in an interview that he is currently planning a multicenter randomized controlled trial, which will include 55 RRMS patients in each arm. The study will compare AHSCT using what is considered a medium-intensity myelobation (BEAM) technique to best available drug treatment (whatever treatment a given patent is taking).

Dr. Racke said one question that needs to be further considered is, When is the best time to do a transplant? He said drug therapies need to be given a chance, but earlier might be better than later because once you start getting damage to the central nervous system we can't really fix that.

He said the upcoming trial will likely include cost analyses to compare the cost of long-term drug therapy to the mostly upfront costs of transplant, which is thought to be a once-and-done procedure.

Commenting on the two studies, Timothy L. Vollmer, MD, FAAN, professor of neurology at University of Colorado Health Sciences Center and co-director of the Rocky Mountain MS Clinic at Anschutz Medical Center, expressed skepticism about using AHSCT, particularly in light of effectiveness of the second-generation MS drugs that have come into use, such as natalizumab for JCV negative patients, fingolimod, dimethyl fumarate, and ocrelizumab.

Dr. Vollmer said most studies of AHSCT for MS were done before the newer drugs were available. He is concerned about both the immediate risks (infection, death) and potential long-term consequences of undergoing a toxic regimen to eradicate the immune system, noting that it could cause brain atrophy, already a concern for MS patients.

Mark S. Freedman, MD, professor of neurology at the University of Ottawa, senior scientist at The Ottawa Hospital Research Institute, and director of the Multiple Sclerosis Research Unit at The Ottawa Hospital-General Campus, is more sanguine about the procedure.

In a 2016 report in The Lancet, he and a colleague described outcomes for 24 RRMS patients who underwent transplant after failing drug therapy. Dr. Freedman said he has done about 25 more cases since the study came out. He said no patient has experienced a clinical relapse following transplant, none has evidence of new brain lesions on MRI, and none requires disease-modifying medication.

Dr. Freedman said there is a high level of interest in the procedure among MS patients, but it isn't for everyone. Patients must be carefully selected for the procedure, and undergo an aggressive chemotherapy regimen to eliminate their immune system, he said, noting that those with a high inflammatory component to their disease are ideal. Harvested stem cells undergo a special sorting technique at his center before being infused into the body to make sure that no previous disease-causing lymphocytes are accidentally included.

We're taking away immunologic memory, Dr. Freedman said. The new immune system is learning all over again what it should and shouldn't be doing.

He said that while the procedure is only done in patients who have not fared well with drug therapy, the best timing for this treatment would be as early as possible, when disability is minimal.

Probably doing it within five years from the onset of illness would give the optimal results, he said.

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Is Autologous Heamatopoietic Stem Cell Transplantation Still Viable for MS? - LWW Journals

BY FRAN KRITZ

High doses of chemotherapy drugs followed by an infusion of a patient's own stem cells may result in remission for people with remitting-relapsing multiple sclerosis (MS), according to a study published online in Neurology on February 1, 2017.

The new results are from a small clinical trial, called HALT-MS, which was sponsored by the National Institute of Allergy and Infectious Diseases (NIAID) at the National Institutes of Health (NIH). Earlier results were published in the third year of the clinical trial while this new study looked at five years of data. The clinical trial included patients with relapsing-remitting MS, which is the most common form of the disease and often involves through long periods with no or only mild symptoms and occasional flare-ups or relapses. Over years, though, symptoms can worsen and progress.

Study Basics

Twenty four patients, ages 18 to 60 (17 women and 7 men) who had not responded to other MS medications, were included in the trial, which involved high-dose immunosuppressive therapy and autologous hematopoietic cell transplant (HDIT/HC.) The high-dose chemotherapy weakens the immune system while transfusions of the patient's own blood stem cells can "reset" the immune system and potentially knock out MS.

Promising Results

At the end of the trial, 91 percent of patients had no disease progression, 87 percent did not relapse, and 86 percent had no signs of new lesions (scars that can indicate MS) on their brain or spinal cord. "These extended findings suggest that one-time treatment with HDIT/HCT may be substantially more effective than long-term treatment with the best available medications for people with a certain type of MS," says NIAID director Anthony S. Fauci, MD.

Some Adverse Events

Three deaths were reported among the trial participants, but they were unrelated to the transplants. Two were the result of MS progression and one was due to cardiovascular disease. The most common side effect of the treatment was infection.

More Studies Needed

More trials are needed before this can be considered a treatment for relapsing-remitting MS, say experts. "Although further evaluation of the benefits and risks of HDIT/HCT is needed, these five-year results suggest the promise of this treatment for inducing long-term, sustained remissions in patients with relapsing-remitting MS, who have a poor prognosis," says Richard Nash, MD, a hematologist with the Colorado Blood Cancer Institute and the principal investigator of the HALT-MS study.

The new clinical trial is "an important study" that "contributes to the accumulating knowledge of the possible benefits and risks ofstem cell transplantation in relapsing MS," says Bruce Bebo, PhD, executive vice president of research at the National MS Society. He added that "larger, well-controlled trials are needed to better understand who might benefit from this procedure and how it compares to the benefits of powerful immune-modulating therapies now available."

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Stem Cell Transplants May Work for MS, Study Says - LWW Journals (blog)

Stanford University scientists have identified a protein that, given before an injury, boosts stem cell response and improves healing. Priming with this protein, called hepatocyte growth factor activator (HGFA),could eventually speed recovery in cases where injury is expected, such as patients undergoing surgery.

Senior author Thomas Rando and lead author Joseph Rodgers previously showed that injury to one leg caused stem cells in the other leg to become alert. This state is different from the fully resting or fully active phases that stem cells usually assume.

To pinpoint the cause of this alert state,the researchers injected uninjured mice with blood serum from mice with a muscle injury. While this serum had the same level of HGFas serum from uninjured mice, it had higher levels of HGFA, a protein that activates HGF.Once HGF is activated, it, in turn, activates a signaling pathway in stem cells that produces proteins that make them alert,according to a statement.

In another experiment, they dosed mice with HGFA two days before injury. The treated mice scampered around on their wheels sooner than untreated mice, indicating faster muscle recovery. Their skin also healed faster than that of untreated mice.

Our research shows that by priming the body before an injury you can speed the process of tissue repair and recovery similar to how a vaccine prepares the body to fight infection, said Rodgers in the statement.

Finding new ways to stimulate stem cells is a major focus of tissue-repair research. Scientists at New York University and the University of Colorado at Boulder, for example, recentlyfound a gene that prompts adult stem cells to repair injured muscle in mice. They successfully used a drug to boost this gene in mice that did not have it.

While the direct implications of Stanford's discovery are obviousthe treatment could become a way to boost recovery for people in combat orsports, or those who have undergone surgerythe team is also interested in the role of HGF and HGFA in aging.

Stem cell activity diminishes with advancing age, and older people heal more slowly and less effectively than younger people, Rando said. The researchers want to look into the possibility of restoring youthful healing rates by activating this pathway, he said.

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'Priming' protein boosts stem cell response to injury, promotes healing - FierceBiotech

Heidi Wyandt, 27, puts on her coat to leave the Altoona Center for Clinical Research in Altoona, Pa., on Wednesday, March 29, 2017, where she is helping test an experimental non-opioid pain medication for chronic back pain related to a work related injury she received in 2014. With about 2 million Americans hooked on opioid painkillers, researchers and drug companies are searching for less addictive drugs to treat pain. (AP Photo/Chris Post)

Tummy tucks really hurt. Doctors carve from hip to hip, slicing off skin, tightening muscles, tugging at innards. Patients often need strong painkillers for days or even weeks, but Mary Hernandez went home on just over-the-counter ibuprofen.

The reason may be the yellowish goo smeared on her 18-inch wound as she lay on the operating table. The Houston woman was helping test a novel medicine aimed at avoiding opioids, potent pain relievers fueling an epidemic of overuse and addiction.

Vicodin, OxyContin and similar drugs are widely used for bad backs, severe arthritis, damaged nerves and other woes. They work powerfully in brain areas that control pleasure and pain, but the body adapts to them quickly, so people need higher and higher doses to get relief.

This growing dependence on opioids has mushroomed into a national health crisis, ripping apart communities and straining police and health departments. Every day, an overdose of prescription opioids or heroin kills 91 people, and legions more are brought back from the brink of death. With some 2 million Americans hooked on these pills, evidence is growing that they're not as good a choice for treating chronic pain as once thought.

Drug companies are working on alternatives, but have had little success.

Twenty or so years ago, they invested heavily and "failed miserably," said Dr. Nora Volkow, director of the National Institute on Drug Abuse.

Pain is a pain to research. Some people bear more than others, and success can't be measured as objectively as it can be with medicines that shrink a tumor or clear an infection. Some new pain drugs that worked well were doomed by side effects Vioxx, for instance, helped arthritis but hurt hearts.

Some fresh approaches are giving hope:

"Bespoke" drugs, as Volkow calls them. These target specific pathways and types of pain rather than acting broadly in the brain. One is Enbrel, which treats a key feature of rheumatoid arthritis and, in the process, eases pain.

Drugs to prevent the need for opioids. One that Hernandez was helping test numbs a wound for a few days and curbs inflammation. If people don't have big pain after surgery, their nerves don't go on high alert and there's less chance of developing chronic pain that might require opioids.

Funky new sources for medicines. In testing: Drugs from silk, hot chili peppers and the venom of snakes, snails and other critters.

Novel uses for existing drugs. Some seizure and depression medicines, for example, can help some types of pain.

The biggest need, however, is for completely new medicines that can be used by lots of people for lots of problems. These also pose the most risk for companies and patients alike.

ONE DRUG'S BUMPY ROAD

In the early 2000s, a small biotech company had a big idea: blocking nerve growth factor, a protein made in response to pain. The company's drug, now called tanezumab (tah-NAZE-uh-mab), works on outlying nerves, helping to keep pain signals from muscles, skin and organs from reaching the spinal cord and brain good for treating arthritis and bad backs.

Pfizer Inc. bought the firm in 2006 and expanded testing. But in 2010, some people on tanezumab and similar drugs being tested by rivals needed joint replacements. Besides dulling pain, nerve growth factor may affect joint repair and regeneration, so a possible safety issue needed full investigation in a medicine that would be the first of its type ever sold, said one independent expert, Dr. Jianguo Cheng, a Cleveland Clinic pain specialist and science chief for the American Academy of Pain Medicine.

Regulators put some of the studies on hold. Suddenly, some people who had been doing well on tanezumab lost access to it. Phyllis Leis in Waterfall, a small town in south-central Pennsylvania, was one.

"I was so angry," she said. "That was like a miracle drug. It really was. Unless you have arthritis in your knees and have trouble walking, you'll never understand how much relief and what a godsend it was."

Her doctor, Alan Kivitz of Altoona Center for Clinical Research, has helped run hundreds of pain studies and consults for Pfizer and many other companies. "You rarely get people to feel that good" as many of them did on the nerve growth factor drugs, he said.

A drug with that much early promise is unusual, said Ken Verburg, who has led Pfizer's pain research for several decades.

"When you do see one, you fight hard to try to bring one to the market," he said.

An independent review ultimately tied just a few serious joint problems to tanezumab and the suspension on testing was lifted in August 2012. But a new issue nervous system effects in some animal studies prompted a second hold later that year, and that wasn't lifted until 2015.

Now Eli Lilly & Co. has joined Pfizer in testing tanezumab in late-stage studies with 7,000 patients. Results are expected late next year about 17 years after the drug's conception.

AVOIDING PAIN TO AVOID DRUGS

What if a drug could keep people from needing long-term pain relief in the first place? Heron Therapeutics Inc. is testing a novel, long-acting version of two drugs the anesthetic bupivacaine and the anti-inflammatory meloxicam for notoriously painful operations like tummy tucks, bunion removal and hernia repair.

Company studies suggest it can numb wounds for about three days and cut patients' need for opioids by 30 to 50 percent.

There's a good chance of preventing brain responses that lead to chronic pain if patients can get through that "initially very rough period," said Dr. Harold Minkowitz, a Houston anesthesiologist who consults for Heron and treated Hernandez in the tummy tuck study.

Hernandez was part of an experiment testing the drug versus a placebo and doesn't know whether she got the drug or a dummy medicine. But she hurt less than she expected to and never filled a prescription for pain pills.

"The goal would be to have half or more of patients not requiring an opiate after they go home," said Heron's chief executive, Barry Quart. "You have far fewer opiates going out into society, far fewer opiates sitting in medicine cabinets that make their way to a high school."

Studies so far are mid-stage too small to prove safety and effectiveness but Heron plans more aimed at winning approval.

ON THE HORIZON

Many companies have their eyes on sodium channel blockers, which affect how nerves talk to each other and thus might help various types of pain. Others are testing cell therapies for nerve pain. Stem cells can modulate immune responses and inflammation, and may "overcome a raft of problems," said Cheng of the pain medicine academy.

Some companies, including Samumed, Centrexion Therapeutics and Flexion Therapeutics, are testing long-acting medicines to inject in knees to relieve arthritis pain. Samumed's aims to regenerate cartilage.

And then there's marijuana. A cannabis extract is sold as a mouth spray in Britain for nerve pain and other problems from multiple sclerosis. But cannabinoid research in the U.S. has been hampered by marijuana's legal status. A special license is needed and most researchers don't even try to obtain one, said Susan Ingram, a neurosurgery scientist at Oregon Health & Science University.

She is studying cannabinoid receptors in the brain, looking at how pain affects one type but not another. Such work might someday lead to drugs that relieve pain but don't produce a high or addiction.

Selective activity has precedent: The drug buprenorphine partially binds to opioid receptors in the brain and has become "an extraordinarily successful medication" for treating addiction, said Volkow, of the national drug institute.

"It has shown pharmaceutical companies that if you come up with a good intervention, there is an opportunity to recover their costs," she said.

___

Marilynn Marchione can be followed at http://twitter.com/MMarchioneAP

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Overcoming Opioids: The quest for less addictive drugs - Colorado Springs Gazette

JP and Annamarie Kealy plan to climb to Mt. Everest Base Camp in Nepal to raise money for the Multiple Myeloma Research Foundation. Jeannette Ross photo

Three years ago, JP Kealy was lifting weights and hurt his back. But it was more than just your typical pain, so he went to an orthopedist, who ordered an MRI.

What they found was a surprise. His bones were untypically brittle for a 48-year-old man. Blood work confirmed he had multiple myeloma, a cancer formed by malignant plasma cells.

Although the diagnosis on April 21, 2014, was devastating, Kealys wife, Annamarie, said the couple consider themselves lucky.

Most people are not diagnosed until the disease has progressed and they are in kidney failure, she said at the couples home in Wilton. We had the gift of time, spending the last three years waiting and watching.

Now they are fighting the disease head-on from two fronts: personally and publicly. Next March, JP and Annamarie will climb to Mt. Everest Base Camp in the Himalayas in a fund-raising program for the Multiple Myeloma Research Foundation called Moving Mountains for Myeloma.

The point of the expedition, Annamarie said, is to show that patients can climb mountains literally and figuratively.

Multiple myeloma is a very complicated cancer, she said, and when Kealy was diagnosed they saw four doctors and got four opinions for going forward. Kealy received monthly IV treatments to strengthen his bones, but last year his numbers reached a point where he needed chemotherapy. He recently completed a six-month course of drugs at Stamford Hospital, which he tolerated very well.

But that is not all Kealy needs.

A stem cell transplant is the best chance for remission, Annamarie said, and so on April 17 they will go to Mount Sinai Hospital for a transplant.

Doctors have already collected 20 million stem cells from Kealy through a process that took four eight-hour days. Kealy lay with his arms straight out and with one needle as big as a turkey baster, Annamarie said, that drew his blood. The stem cells were then collected and the rest of his blood was returned through another needle into his other arm.

The stem cells enough for four treatments are now frozen. They can last in that state 10 to 15 years.

When he goes to Mount Sinai, Kealy will be given a high-dose chemotherapy treatment to reduce his immunity to zero. Then his stem cells will be thawed and returned.

He will remain in a sterile environment for three weeks, with Annamarie as a caregiver, before returning home for two to three months. When home, he will be on an anti-microbial diet that Annamaria must prepare for him. That means, among other things, no raw fruits or vegetables except fruits with thick skins like bananas and oranges or red meat.

He will also need all new immunizations.

Since there is no cure as yet, the goal is complete remission, but for how long is not known.

When the Kealys first learned of his diagnosis they connected with the Multiple Myeloma Research Foundation (themmrf.org), formed in New Canaan in 1998 by twin sisters Kathy Giusti and Karen Andrews soon after Giusti was diagnosed. She was given two years to live, Annamarie said, but that was almost 20 years ago.

The support we have received from them is incredible, Annamarie said. For the last three years, the couple has participated in the organizations fund-raising 5K walk, but they wanted to do more.

Last year they started Moving Mountains and the first trip was to Mt. Kilimanjaro, Annamarie said. Typically, only one person per family is accepted for an expedition, but as Annamarie said, Ive been with him throughout. Its been such a life-changing experience. Our motto has always been JP Strong.

She wrote a letter to the organization explaining why they should go as a patient-caregiver team, and they were accepted. Because the expedition is a fund-raiser, with proceeds going to research, they were required to raise $10,000. In seven weeks they have raised more than $25,000. To contribute, visit https://endurance.themmrf.org/2018Everest/jpstrong.

For me, its something were doing thats proactive. We have four kids. I want them to see they can be resilient, she said. Their children are Tommy, a junior at the College of William and Mary; Brendan, a student at Dickinson College; Jake, a senior at Fairfield Prep; and Lily, a sophomore at Wilton High School.

Assuming he will be well enough by then, the couple will go on a training climb in Colorado in July, where they will surmount a 14,000-foot peak and meet their fellow travelers.

Next March, they will fly into Kathmandu and then to Lukla, Nepal. They will stay at Nepalese teahouses to get acclimated, and then with yaks to carry their gear and Sherpas to guide them, the group of 16 doctors, patients and caregiver will ascent to Mt. Everest Base Camp at 18,519 feet in altitude.

Its a small group of people, but the impact will be huge, she said. They will carry a banner proclaiming the Bennett Cancer Center at Stamford Hospital.

Kealy said he wasnt surprised at his wifes plan for them to participate in the climb.

I wasnt surprised she did it, he said. Theres no talking her out of it. Im glad were doing it together.

Annamarie and JP arent the only Kealys working to support the MMRF. Daughter Lily will hold a bake sale at the Village Market on Sunday, April 23. Last year she raised more than $1,000 for the organization.

This wasnt the master plan, Annamarie admitted, but added there has been an upside. Theres been a lot of good people whove come out of the woodwork. We are so positive because we have such a team rallying behind us.

Link:
Climbing mountains for cancer research - The Wilton Bulletin

Suspended from a tree in the wilds of Tennessee, the remains of his hang-glider entangled in the branches above, his lower left leg pulverised and his chest badly bruised from his dramatic fall into the forest canopy, Alan Mackay-Sim felt hyper-alert from the electricity of adrenalin, the clarity of shock. Only the wind was audible, softly rustling the branches around him as he sucked in the forest air, perfumed with poplar and sweet-gum.

Knowing that the adrenalin coursing through his veins would soon give way to an agonising and possibly debilitating pain, the 28-year-old used these precious minutes to assess his predicament, to figure it out coolly like a man of science.

A broken leg, no doubt shattered in multiple places. Possibly hours before his fellow hang-gliding friends would be able to locate him; if they didn't reach him by nightfall, he could be dangling here until the next morning. Unfastening his harness and climbing down to the ground five metres below was not an option, at least, not without incurring further injury. To prevent blood from pooling and to save his leg, he quickly concluded, he'd have to carefully oh-so carefully free the hang-glider's stirrup bar and one of the ropes from his harness, create a splint for his injured left leg, secure it to his right leg and hoist up both limbs while hanging there like a gammy fruit bat.

Mackay-Sim had only arrived in the US a few weeks before, a post-doctoral researcher from the University of Sydney eager to extend his studies into the olfactory system specifically, what the nose tells the brain at the University of Philadelphia. But on that blustery October day back in 1979, when a freak wind gust whooshing around Lookout Mountain near Chattanooga sent a promising young Australian scientist nosediving into the forest, before a rescue team found himhanging in the tree just before sunset, both legs securely elevated, Mackay-Sim was set to gain some useful insights that would become valuable to him in his later life. Insights that would be peculiarly relevant to his work as a pioneering stem cell researcher specialising in the treatment of spinal cord injuries.

So badly broken was his leg that Mackay-Sim spent more than six months in a wheelchair, and many more months afterwards receiving intensive physiotherapy.

"It gave me some insight into what life's like in a wheelchair, and it stayed with me," says Mackay-Sim, settling into a chair in his office at the Institute for Drug Discovery at Griffith University, just down the corridor from the laboratory where he spent years toiling over petri dishes of nasal stem cells, in his life's mission to treat spinal injuries, hereditary spastic paraplegia and diseases like Parkinson's.

A photo of the late actor Christopher Reeve is pinned on a noticeboard behind him. "I met Christopher in 2003 when he came out for a conference; he was interested in our clinical trials," Mackay-Sim says, looking at the photo. "Then in the following year I spent some time at his home in New York, and we talked a lot about spinal cord injury repair, and his own personal story."

As Mackay-Sim explains, the higher up the spinal cord an injury is, the more severe the effects. "As we know, Christopher fell off a horse and became a full paraplegic on a respirator, but in fact he suffered only a small injury; the problem was that the bleed went straight into his spinal cord. It only takes a very small injury to stop transmission; you can have large injuries to the chest and not suffer long-term repercussions but here, in the neck, a small event can change your life."

Back in the late 1980s, after he started at Griffith University, Mackay-Sim became interested in a set of extraordinary busy-bee cells in the human nose called olfactory ensheathing cells nerve cells that regenerate every single day to recreate our sense of smell. If these wonder cells are continually regenerating, he kept asking himself, could they not be transplanted to another part of the body where cells don't regenerate, like the spinal cord?

Years of scientific slog followed until 2002, when Mackay-Sim was the first researcher in the world to remove cells from the nose of a patient paralysed in a car accident, grow them in a cell culture and then, with the help of surgeons at Brisbane's Princess Alexandra Hospital, implant them in the same patient's spinal cord. "By the time Christopher died in 2006, we'd transferred stem cells from the nose into three patients and shown it was safe to do so," he says. "One of the patients recovered some sensation above the injury, which was hopeful, but one person does not make real scientific evidence."

For Mackay-Sim, the importance of scientific breakthroughs in the treatment of life-threatening illnesses is deeply personal. In 2014, he was diagnosed with multiple myeloma, an incurable form of leukaemia. As a result of the illness, which breaks down bones in an advanced form of osteoporosis, and the punishing series of treatments that followed his diagnosis, involving radiation, chemotherapy and stem cell therapy (albeit a very different form from the one the scientist was researching), Mackay-Sim lost nine centimetres in height and shed more than 15 kilograms of body weight. "I became extremely sick from the chemotherapy just prior to the bone marrow transplant," the 65-year-old recalls. "It was the worst experience of my life."

There was also the initial shock of the diagnosis, and grief for the loss of his health after a highly active life, from football and rowing in his teens to distance cycling, scuba diving and hang-gliding, which he took up while atuniversity. "Both my parents lived into their 80s and 90s and I'd been cycling up to 200 kilometres a week for decades, so I wasn't anticipating something like this."

Still, as a scientist he couldn't help but observe the trajectory of his illness with stricken fascination. "I had some good conversations with my oncologist," he smiles. "As a biologist examining my own biology, it did demystify lots of things. One minute I was a grieving patient, the next an interested scientist."

Above all, Mackay-Sim refuses to sentimentalise his battle with the illness and asks that I don't embroider it in this story by turning it into some kind of triumph of personal will power over disease. "My survival is determined by the vagaries of the particular cancer I've got," he says matter-of-factly. "Some people have nasty genetic diseases that mean they die earlier. For the moment, I feel very healthy."

Surely his extreme fitness at least helped him to survive the ravages of chemo? "I think being fit and active all my life has given me a higher quality of life after treatment," he acknowledges. "But one doctor put it to me that I probably would have sought out treatment earlier if I wasn't so fit, because I dismissed the symptoms as simple back pain from the cycling. It took two years after the chemo and radiation for the pain to go away. 2016 was a year of normality for me my back became stable enough for me to get on a road bike again."

The diagnosis added poignancy to the evening in Canberra in late January when Mackay-Sim, out of 3000- plus nominations, was crowned Australian of the Year. Sitting alongside him were his American-born wife of nearly 34 years, Lisa Peine, a retired primary school teacher, their 28-year-old daughter Matilda, a trainee psychiatrist, and 25-year-old son Callum, an engineer.

Mackay-Sim with wife Lisa Peine in North Queensland in 1983. Photo: Courtesy of Alan Mackay-Sim

Perhaps no Australian of the Year is better placed to recognise just how precious a year can be, and more determined to seize the moment to put science and innovation at the top of the national conversation. A former Queenslander of the Year, Mackay-Sim sees science as vital to our future national wellbeing, especially after the recent wake-up call in international school education rankings, which placed Australia behind Kazakhstan and Slovenia in maths and science.

Mackay-Sim agrees unequivocally with Michelle Simmons, professor of quantum physics at the University of NSW, who drew headlines recently when she declared that the "feminised" nature of Australia's high school physics curriculum (emphasising the sociology of science with essays and theory instead of rigorous lab experiments and mathematical problem-solving) had been an unmitigated failure. Introduced in the 1980s, the approach had resulted in a long, slow decline in standards.

"Scientific understanding comes from learning the processes; it can be hard work but is absolutely essential," Mackay-Sim insists. "The key to a good science education in schools is to get well-trained teachers." (Mackay-Sim has been deeply encouraged by some of the science teachers he's met since winning the award.)

The choice of Mackay-Sim the first scientist honoured as Australian of the Year since immunologist Ian Frazer in 2006 was met with near-universal applause by Australia's scientific community, who no doubt feel dispirited in this post-truth world of climate-change denial, cuts to the CSIRO and the growing view by government agencies that basic research isn't worth it.

"We need to invest in young scientists," Mackay-Sim declared in his acceptance speech, adding that the discovery of new medical treatments can reduce the strain on health budgets. "More than 10,000 Australians live with a spinal cord injury a new person is added to this tally every day." But politicians need to take a long-term view of the benefits of basic research, he tells me, "a view much longer than the political horizon".

The announcement also gave the image of the Australian of the Year awards a much-needed polish. The 2016 winner, Lieutenant-General David Morrison, drew criticism for charging up to $15,000 a pop forpublic speaking engagements, as well as grandstanding about sexism in the military despite his own handling of the army's "Jedi Council" sex scandal, in which demeaning sex videos of women were distributed among a group of soldiers. (It was revealed that Morrison's office knew of the scandal 11 months prior to the former Chief of Army releasing a now-famous condemnation on YouTube of those involved.)

Will Mackay-Sim accept speakers' fees? "I knew nothing about speakers' fees when I accepted the award," he says crisply. "I'm not pursuing money after all, I've spent my life doing public research."

Although he hasn't received any fees to date, Mackay-Sim insists that if they are offered, the funds will be donated to the Hereditary Spastic Paraplegia Research Foundation, his charity of choice.

Mackay-Sim only had a day or so to bask in the glow of being named Australian of the Year before there was a claim his scientific achievements had beenoverstated in the application. A Polish scientist, Professor Pawel Tabakow, after being approached by an Australian journalist in Europe, declared that Mackay-Sim had nothing to do with the world-first surgery using olfactory stem cells that enabled a Polish paraplegic, Darek Fidyka, to walk again. "It is not our business who should be Australian of the Year," Tabakow told The Weekend Australian. "But it is our business when his work is being linked to the surgery of Fidyka. He has no link whatsoever."

The scientific hullaballoo arose from the submission to the Australia Day Council (ADC), which states that Mackay-Sim's research "helped play a central role in proving the safety of science that was a precursor to Dr Tabokow in Poland undertaking the first successful restoration of mobility in a quadriplegic man".

Although Mackay-Sim didn't write the submission to the ADC, doesn't know who did, and never claimed to be involved in Tabokow's work, an artificial straight line was drawn between the two scientists, especially when the word "precursor" was dropped from condensed versions of the ADC's quote in multiple news stories (we'll examine the fallout from the controversy a little later).

Padding amiably about his large, multi-room laboratory, past refrigerator-sized storage cabinets containing cell cultures, past white-coated scientists peering into microscopes, Mackay-Sim seems to be in his element, with every second person saying "Hi", "Hello", or "How are you?" If stem cells are indeedthe microscopic building blocks of the world, this is the tiny universe the scientist feels most comfortable in. But it's a laboratory that now has to hum along without him Mackay-Sim retired late last year, his duties now limited to popping into the university once a week as an emeritus professor.

Later in the day, Professor George D. Mellick, head of Clinical Neurosciences at Griffith, tells me that Mackay-Sim has always set aside time to mentor younger scientists, and to explain sometimes hideously complicated science to a lay audience, but would be the last person to crow about his own scientific achievements.

"One of the things that isn't highlighted very much about Alan's work is his research into Parkinson's. We've been able to learn a lot about Parkinson's by studying cells from people with the disease, and the information coming out of this research will hopefully lead to better treatments."

Back in his office, Mackay-Sim gives me a quick rundown, 101-style, on the human nose. No, the human sense of smell doesn't necessarily decline with age, unless illness or disease set in, and it is astonishingly adept at distinguishing hundreds of thousands of different odours. Yes, women do have a superior sense of smell to men, but the difference is surprisingly only slight. Yes, the first symptom of Parkinson's, before the typical tremors set in, is a reduced sense of smell, as it is with those sufferers who will go on to develop dementia. And yes paws down dogs do have a vastly more powerful sense of smell than humans, although it's impossible to quantify by exactly how much (Mackay-Sim has been known to hide from his spoodle Henry, to measure how long it takes for the dog to find him).

As he relays all this, Mackay-Sim's eyes twinkle and a smile lights up his face: it's easy to see how he'd be the perfect academic for Griffith to call on to schmooze a government minister or potential philanthropist and secure desperately sought-after funding. I ask him about his trademark moustache, which he's had since the early 1990s, when he shaved off a beard. "My wife wouldn't recognise me without it," he jokes. "She says that a small mammal could roost beneath my mouth."

Mackay-Sim, whose double-barrelled surname comes from his paternal grandfather, grew up in middle-class Roseville, on Sydney's leafy North Shore, the third of four brothers. His mother Lois was a nurse during World War II and later a full-time mum while his father Malcolm ran a hardware importing and distributing business, Macsim Distributors (now Macsim Fasteners, owned by Alan's eldest brother, Fraser). At North Sydney Boys' High he was "the opposite of a shit-stirrer. I was vice captain, head of the cadets, played football, was in the rowing team, had a shot at athletics, sang in the choir I did it all."

With wife, Lisa Peine, in Sulawesi, Indonesia, 2007. Photo: Courtesy of Alan Mackay-Sim

After graduating with honours in science from Macquarie University, Mackay-Sim picked up tutoring work in the department of physiology at the University of Sydney, where he completed a PhD on the brain's visual system. Two academic stints in the US followed, first at the University of Pennsylvania from 1979 until 1981, followed by two years at the University of Wyoming, during which time he met his wife Lisa, then living in northern Colorado.

The pair married in 1984, by which time Mackay-Sim had been offered a research role in the department of physiology at the University of Adelaide. He started at Griffith University in 1987, where his research concentrated on the biology of nasal cells.

At the height of the heated moral debate over the use of embryonic stem cells whether the therapeutic potential of stem cells could justify destroying human embryos to extract them Mackay-Sim met Pope Benedict XVI at a Vatican conference in 2005. The Pope congratulated him on his exclusive use of adult stem cells.

"I wasn't avoiding embryonic stem cells for religious reasons," Mackay-Sim explains. "It just so happenedthat I was working with adult stem cells at the time and the conference was looking at alternatives to using embryonic stem cells. But it was a scientific conference and I was impressed with its calibre; the only difference was that men in purple robes were sitting at the back asking questions."

Later in the same trip, Mackay-Sim was invited, along with a host of others, to the Apostolic Palace at Castel Gandolfo the Vatican summer palace. "You feel the history of the Roman Catholic Church, with the Pope coming in with his cardinals and the Swiss Guards," he says. "I'm not a believer, but it was a very powerful experience."

In 2006, the debate over embryonic stem cells virtually vanished when scientist Shinya Yamanaka from Japan's Kyoto University stunned the world by proving that stem cells needn't come from human embryos adult cells can be reprogrammed to act like stem cells, to be returned to an embryo-like state (Yamanaka's discovery won him the Nobel Prize in 2012). "Yamanaka worked out how to genetically engineer any cells so that they had the properties of embryonic stem cells," says Mackay-Sim, who nonetheless continued to focus on adult stem cells only.

Mackay-Sim accomplished his own world first in 2002 when, with the assistance of doctors at Brisbane's Princess Alexandra Hospital, he transplanted olfactory stem cells into the spinal cord of a man crippled in a car accident. The procedure was repeated with two other paraplegic patients at the same hospital and the study wrapped up in 2007.

While the procedures didn't result in any of the patients regaining useful movement in their legs, the results of Mackay-Sim's clinical trials, published in 2005 and 2008, paved the way for further development of olfactory stem cell transplantation.

One researcher who followed Mackay-Sim's trials closely was Geoffrey Raisman from University College London, who visited the Australian team shortly after the first operation in Brisbane to study their work. Raisman later led the British team who worked with Polish surgeon Tabakow on Darek Fidyka in 2012.

Tabakow deployed 100 separate micro-injections of olfactory sheathing cells above and below Fidyka's spinal injury, with the hope these cells would provide a skeleton for nerve fibres to grow and reconnect. A former volunteer firefighter, Fidyka had become paralysed in 2010 after a severe knife attack by the jealous ex-husband of his girlfriend. The repeated stab wounds to Fidyka's back severed his spinal cord, paralysing from the waistdown. (Fidyka's attacker, a fellow firefighter, committed suicide shortly afterwards.)

There's no doubt Tabakow's work was a major advance on Mackay-Sim's research. Tabakow's strategy was to extract ensheathing cells specifically from the olfactory bulbs in Fidyka's nose, grow them in a culture, while also extracting nerve cells from his ankle in a multi-pronged attempt at spinal cord reconstruction. After a series of operations, Fidyka can walk with the assistance of a frame, has regained some bladder control and sexual function, and can ride a tricycle.

Raisman described their new stem cell procedure as "more impressive than man walking on the moon", but it will have be tested on other paraplegics, including those with more severe injuries than Fidyka's, such as car accident victims who have had more of their spinal cord damaged, before it can be declared a reliable method of restoring mobility. As impressive as Tabakow's achievement is, it has still only worked on one patient.

Nobody, however, disputes Mackay-Sim's immense contribution to stem cell transplantation; his work is unimpeachable. If nothing else, he was at the forefront of the science showing that restoring the ability to walk to paraplegics is no longer science fiction. "What I've always said is that we did the first phase of clinicaltrials with olfactory stem cells, and the aim of those trials was to show they were safe," says Mackay-Sim. "That was the first important step."

Mackay-Sim wrote to Tabakow shortly after the controversy blew up, explaining that he didn't write the submission to the Australia Day Council, and was in no way claiming credit for Fidyka's remarkable recovery. "He wrote back a very nice email," says Mackay-Sim. "I believe I've given credit to other scientists in every interview I've given to journalists. I feel comfortable in my behaviour and ethics."

With Prime Minister Turnbull in January this year. Photo: Elesa Kurtz

Mackay-Sim can remember the day when he felt something was wrong terribly wrong. He'd been suffering back pain for months, but dismissed it as old age, or strain from bending over on his bicycle on long rides, and stocked up his pantry with painkillers. "I was in Colorado with Lisa visiting her family, and the pain became so bad I couldn't walk very far. I found the pain eased when I got on my bicycle. I flew home a week before she did; the plane trip back was absolute hell."

What followed was a swift diagnostic journey from his GP to specialists at Brisbane's Wesley Hospital, resulting in a devastating diagnosis. "They suspected something cancerous quite quickly. I didn't realise how ill I was; by this stage, my kidneys weren't coping at all with the antibodies released from my white blood cells, which were going berserk trying to fight the disease. I was at risk of kidney failure and my bones were becoming very fragile. I started therapy almost immediately, in June 2014. Then began the cycles of chemotherapy and stem cell treatment in December."

Since the beginning of last year, however, Mackay-Sim's health has dramatically improved, and even though he's retired to his beachside home in Currimundi on the Sunshine Coast, he is still active in university affairs. He concedes that his health may prevent him from being as active as Rosie Batty, perhaps our most vigorous Australian of the Year to date. But he's already spoken at functions in Brisbane, Sydney and Perth, and will be attending the national March for Science on April 22, which coincides with Earth Day. He moves with the speed and fluidity of a man 10 or 15 years younger.

"I feel very healthy, very energised at the moment," says Mackay-Sim, who is planning a bicycle ride in Italy's Dolomites in July with a couple of mates. (Last year he and his wife went on the Great Victorian Bike Ride, a seven-day ride averaging 85 kilometres a day.)

"I do need to be selective with the number of invitations around Australian of the Year," he concedes, "but I'll do everything I can. After all, what more exciting time could you have to talk about science?"

Continued here:
Australian of the Year Alan Mackay-Sim on the advantage of being 'an interested scientist' - The Sydney Morning Herald