Updated September 18, 2019 06:37:06

It is a quiet Monday morning, and as people are returning to desks and lining up for coffee, Doreen Deede is zig-zagging between bars, collecting cans and bottles emptied over the weekend.

Cashing in refundable containers, hosting charity events, even selling donuts at a local market Ms Deede tried all options as she inched towards a fundraising goal of $35,000 to fund overseas medical treatment.

"For the past nine years I've been Emma's sole carer," she said.

"I call myself her fundraising manager because that's what I've been doing."

Emma, aged nine, suffered a brain injury at birth and has been living with cerebral palsy and dystonia a condition where her muscles contract uncontrollably.

Ms Deede's social life all but vanished as her daughter grew up and significant problems with her mobility, eating, and speech began to register.

"That's when I decided that I wanted to be a mum that gives everything to my daughter," she said.

"The choice included that I live for her, that I do everything in my power to give her all the chances that she can get to one day live independently."

This week, the small family will take another of those chances when they travel from Cairns to the United States for a stem cell treatment to be administered to Emma via two lumbar punctures.

Even with Ms Deede's exhaustive fundraising regime the procedure is so expensive she has had to take out a bank loan and then there is the fact that the procedure has not been clinically proven.

"Everyone that is a parent will know that you do everything for your child," Ms Deede said.

"I would feel guilty if I don't try."

In Australia, the only stem cell treatments classed by the Therapeutic Goods Administration as safe and effective have a narrow application. They generally involve the use of umbilical cord blood or bone marrow to treat disorders of the blood or immune system.

These treatments rely on the function of stem cells, which are unallocated, to replace cells that may be lost or damaged.

Also seizing on that potential has been a burgeoning market of private clinics offering solutions to disorders broadly ranging from neurological conditions to ageing.

As the deputy director of the University of Melbourne's Centre for Stem Cell Systems, part of Megan Munsie's job has been to help inquisitive families understand the sometimes significant gap between what these clinics offered and what was actually sold.

"One of my biggest challenges in this field is the fact that stem cells are somehow seen as magical," she said.

"It's an example of non-evidence-based practice that's gone mainstream."

Ten years ago, this practice sometimes termed stem cell tourism was confined to a handful of clinics in exotic locations.

Since then Dr Munsie has witnessed growth in the number of clinics offering these treatments around the world.

In some cases, they may have been supported only by effusive patient testimonies or materials that blurred the line between medical science and marketing sometimes at the expense of communicating that these procedures were invasive and potentially harmful.

"Our responsibility as a research community is to try to contextualise the science and paint a clearer picture about where exactly we are," Dr Munsie said.

"Unfortunately that means that most of those conversations start with a disclaimer that we don't have any proven treatments for that condition."

On the other side of the equation were families like Ms Deede's, buoyed by hope and leaving no stone unturned.

Success stories may add to the guilt some parents feel.

Even in Cairns, another local family has credited two rounds of treatment with helping their son, who also lives with cerebral palsy, learn to speak and improve his fine motor skills.

"What we do know is there's certainly a lot of promise in this area, but we do still need more data to answer some of the many questions that remain," said Megan Finch-Edmondson, a stem cell researcher with the Cerebral Palsy Alliance Research Institute.

"It's a very interesting and complex question."

Like Dr Munsie, Dr Finch-Edmondson has been in frequent contact with families keen to explore their options.

An Australian-first trial conducted by Melbourne's Murdoch Children's Research Institute has been investigating the use of umbilical cord blood to treat cerebral palsy.

Even though it was only a trial and the selection criteria was rigorous, the limited spaces were highly coveted.

"That is one of the reasons that we are so committed to trying to progress this research and conduct more rigorous clinical trials in Australia," Dr Finch-Edmondson said.

"We do need to operate within [rigorous regulatory guidelines] to get any new therapy, or treatment that's like a drug, approved for use in Australia.

"It does require those very rigorous trials that can show enough data to those regulatory bodies to warrant that this treatment should be available."

Until then, families hamstrung between a long regulatory process and the promises of unproven treatments may continue to take the leap.

"I'm doing it because I love my daughter and I want to give her the best life and the best chance of improvement," Ms Deede said.

"I don't want to be in a situation where I haven't done everything I could and in 10 years' time I might be thinking 'What if?'."

Topics:medical-sciences,health-policy,medical-procedures,healthcare-facilities,medical-research,cerebral-palsy,stem-cells,cairns-4870,university-of-melbourne-3010,melbourne-3000,united-states

First posted September 18, 2019 06:34:23

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Unproven stem cell treatments provide expensive last resort in families' search for hope - ABC News

When Constance Benson* was 25 years old, she was living her dream as a model with a major agency. One day, while receiving treatment for a pain crisis in the hospital, she realized she wasnt well enough to travel any longer so she gave up her modeling dream. As a sickle cell warrior, Constance had already left college because she was unable to keep up with school work as a result of her unpredictable pain crises.

She was uncertain of her future. She recalls feeling tormented by the response of medical professionals who doubted the severity of her condition with one nurse telling her she was too pretty to be sick. Constance considered it divine intervention when she learned that a blood stem cell or marrow transplant could cure her from sickle cell disease. Her parents are both nurses, but it wasnt until her mom was caring for a patient whose son had just had a transplant to cure his sickle cell disease that she learned this was even a treatment option.

To date, hundreds of people in the USA and around the world have been cured of sickle cell disease following blood stem cell or marrow transplants from compatible donors. Like Constance, too often people are unaware that transplant is even an option.

A new day has arisen for sickle cell disease treatment. For the first time, increasingly more medical and research efforts are directed at better understanding and treating sickle cell disease. Historically, progress in developing new treatments for sickle cell disease had been slow. This has been in part due to the complexity of sickle cell disease itself and underfunding compared to other potentially life-threatening genetic diseases.

Among children under the age of 16 battling sickle cell disease, about 95% were cured from sickle cell disease after a transplant from a matched sibling. A blood stem cell or marrow transplant from a matched sibling is now considered a pediatric standard of care by many doctors, and transplant from matched unrelated donors are becoming more common.

Too often people suffering from severe sickle cell disease are unaware of treatments that exist and may be available to them. For example, clinical trials that offer cutting-edge treatment options like blood stem cell or marrow transplantation may be an appropriate path for someone who is not responding well to prescribed therapies. Clinical trials are highly regulated and administered by medical experts. They are designed to assess the safety and effectiveness of new treatments. A benefit of clinical trials includes access to specialized care for your condition with built-in safety measures.

With a transplant, blood stem cells or marrow is extracted from a compatible donor and given to a sickle cell disease patient via IV. The donors healthy blood-forming stem cells, which create healthy red blood cells, replace a patients unhealthy stem cells that made sickled red blood cells. For the person with sickle cell disease, a successful transplant can mean no more sickled red blood cells with no more blocked blood vessels and no more damage from ruptured cells. Some people will even see existing damage improve with the introduction of healthy blood stem cells. Risks do exist for transplant, but doctors can inform a patient about the benefits and risks of such a procedure so that the patient and their family can make an informed decision about proceeding.

Transplant too often is dismissed as an option for people with sickle cell disease by themselves, or by their families, or even by their physicians because of concerns that the financial cost would be prohibitive. There are financial resources available to assist families considering transplant as an option, so it should not be ruled out as a treatment for a person battling advanced sickle cell disease.

As representatives from Be the Match and the Sickle Cell Transplant Advocacy and Research Alliance (STAR), we want to encourage people with sickle cell disease and their loved ones to be empowered to educate themselves about new treatment options, to ask questions of their primary care or hematology providers and to consider participating in clinical trials if other treatments are not working.

Constances younger sister was a perfect match for her. Today, Constance is living free from sickle cell disease after a successful marrow transplant seven years ago. She was able to return to college and obtained her bachelors degree, and she is committed to spreading awareness of the need for more diverse donors on the Be The Match Registry.

Nearly 1 out of 5 people with sickle cell disease will find a match within their family. Those without a match in their family turn to Be The Match, the national marrow donor program, to try to find an unrelated donor willing to help a complete stranger find their cure.

When searches are performed for Black patients, there is a 23% chance of finding a compatible donor on the Be The Match Registry. This is because race and ethnicity play a role in finding a matched blood stem cell or marrow donor. The makeup of each of our cells is as diverse as the places our grandparents and ancestors came from and people of African descent have more unique and complex genes than other races. Currently, only 4% of the 20 million donors on the worlds largest registry are Black or African American. Given all the diversity among persons of African descent, more donors are needed.

This underrepresentation can be improved by more Black donors joining the registry and it only takes a cheek swab to get started. Its important to note that not only people with sickle cell disease, but patients battling blood cancers like leukemia or serious blood disorders like aplastic anemia, also are searching for matches on the registry. There is an urgent need for more racially and ethnically diverse donors to join the registry. Visit http://www.endsicklecell.org to learn how.

A diagnosis of sickle cell disease can mean frequent emergencies, life threatening infections, irreversible organ damage, and even early death. Sickle cell disease frequently denies patients and families what any of us would want comfort, time, growth and financial stability. Sickle cell disease causes excruciating acute pain in children and, as patients age, the pain becomes chronic and debilitating for adults. STAR is comprised of hematologists and supporters of the sickle cell community who are dedicated to advancing research that will help lead to a cure for sickle cell disease. We have partnered with Be The Match to ensure that people with sickle cell have access to free resources, including information about clinical trials, access to certified nurses and patient navigators to learn more about transplant as an option, and a new Peer Connect program that will match existing patients with sickle cell transplant recipients. For more information, call the Be The Match Patient Support Center at (888) 999-6743 or visit http://www.bethematch.org/sicklecell. To learn more about research initiatives and success stories being supported by STAR, visit http://www.curesicklenow.org.

Currently, the only cure for sickle cell is a blood stem cell or bone marrow transplant, but new methods of gene therapy are now also being tested. With more than 100,000 persons with sickle cell disease in the U.S. today, the need for safe and effective treatment options and the need for more donors is high.

We hope that EBONY readers will take action to help address sickle cell disease in the Black community:

Disclosure: Constance Benson and her family have given consent to Be The Match to share her story.

In the USA, approximately 1 in 365 people of African descent will be born with sickle cell disease, an inherited blood disorder where red blood cells are abnormal. Healthy red blood cells are soft and oval-shaped as they travel throughout the body delivering oxygen to organs. Red blood cells that contain sickle hemoglobin can become stiff and crescent-shaped. Sickled cells block blood flow, causing excruciating pain, lung damage and potential strokes. They also rupture, releasing debris that causes damage to blood vessels.

Some people with sickle cell disease manage symptoms as they come. Other patients take a daily medication that decreases the likelihood of forming sickled red blood cells. Still others might receive chronic red blood cell transfusions to reduce the number of sickled cells in their bloodstream. Additional patients choose to participate in clinical trials to pursue new treatments that offer a chance at a cure. Visit http://www.Clinicaltrials.gov to learn about qualifying to participate in a trial.

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Hope for Sickle Cell Warriors: A Cure Exists and Support from the Black Community Could Help Save More Lives - EBONY

SUMMARY: Researchers are learning how best to use this powerful modality while avoiding serious toxicities.

Since it was approved by the U.S. Food and Drug Administration (FDA) in the fall of 2017, a form of the powerful and promising therapy known as CAR T-cell therapy has been used to treat certain young patients with B-cell acute lymphoblastic leukemia (ALL) who have relapsed or didnt respond to standard regimens.

Today, researchers and physicians at Dana-Farber/Boston Childrens Cancer and Blood Disorders Center say they are learning how best to use the specific CAR T-cell treatment known as Kymriah for which patients, and at what point in their cancer treatment and how to predict and treat the serious toxicities that can occur. Theyre also laser-focused on another question: Can CAR T-cell therapy work for other pediatric patients?

CAR T-cell therapy is a form of gene therapy in which a patients own immune cells are removed, engineered in a lab to hone their cancer-fighting abilities, and returned to the body. In clinical trials, Kymriah showed encouraging remission and survival rates in B-cell ALL patients who had few other options leading to its FDA approval.

Kymriah is approved for patients 25 years old and below whohave B-cell ALL expressing the CD19 protein, and who have not responded tostandard therapy or are in a second or later relapse. The great majority ofpatients with B-cell ALL are successfully treated with standard methods, butthose who dont respond have an unfavorable prognosis. Kymriah has been shownto be effective in many of these cases.

CAR T-cell therapy is a complete paradigm shift, says Christine Duncan, MD, a senior physician at Dana-Farber/Boston Childrens. It started with CD19, which is a specific form of pediatric ALL. Now that it has expanded, were learning a lot more about the obstacles to therapy and how we need to pick the right patients for the treatment, so we can move forward to other high-risk populations.

Some patients receive CAR T-cell therapy and then undergo stem cell transplantation, while for others its the reverse, say Duncan and Steven Margossian, MD, PhD, who is a senior physician in the stem cell transplant program at Dana-Farber/Childrens.

Generally, we are using CAR T as a bridge to a transplant;CAR T gets the patient into a good remission and then we take them totransplant, says Margossian. They do well with a transplant.

But some patients whose leukemia relapses after a stem celltransplant are treated with Kymriah as definitive therapy.

Kymriah treatments are custom-made for each individual. Initially, the patient undergoes apheresis to remove lymphocytes known as T cells, which are frozen, packaged, and sent to a pharmaceutical laboratory. There, the T cells are equipped with whats known as a chimeric antigen receptor (CAR) so that they will home in on the CD19 protein on the patients leukemia cells. This takes about 24 to 28 days; meanwhile, the patient may receive a cycle of chemotherapy to combat the leukemia. When the CAR T cells are returned to Dana-Farber/Boston Childrens, they are reinfused into the patient, where they seek out and destroy the cancer cells.

CAR T-cell therapy can trigger serious side effects,including cytokine release syndrome. There are medications that can block thisreaction, and we are researching the correct way to give the medication: shouldit be as a rescue medicine or can you give it pre-emptively? Margossian says. Neurologicaltoxicities are also possible.

We are very good at managing the patients through the complications we always have regular meetings for every CAR T patient that comes in, and we are always prepared to transfer the patient to the ICU if needed, Margossian notes.

The long-term effects of CAR T-cell therapy are also still unclear,but doctors are working to understand them.

It took many years to figure out how to correctly followpatients who received a stem cell transplant, says Duncan. Now were tryingto figure out how to follow patients who received CAR T-cell therapy. This issomething well learn over time.

Meanwhile, variations on the approved use of Kymriah are being studied in new clinical trials. Margossian says one trial will be for patients with ALL who are in remission but have high levels of detectable cancer cells. Another trial will test Kymriah in pediatric B-cell lymphomas. Also on the horizon is a clinical trial of CAR T-cells as a bridge to transplant for patients with acute myeloid leukemia (AML).

Farther off are CAR T-cell therapy trials for solid tumorssuch as bone cancers and neuroblastoma, which present unique challenges, accordingto Margossian. One hurdle is that the cancer-specific molecules that CAR Tcells bind to are often inside the cancer cells of solid tumors, not on thesurface, as they are in blood cancers, and therefore are more difficult totarget.

Its exciting to see CAR T-cell studies developing andexpanding to very high-risk patient populations, says Duncan. Were veryhopeful that CAR T-cell therapy will expand to entirely new populations.

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CAR T-Cell Therapy for Pediatric Patients: The Latest Updates - Dana-Farber Cancer Institute

The legal and regulatory tools designed to protect the public from the marketing of unproven stem cell therapies will remain ineffective without bureaucratic will and grassroots efforts, according to a University of Alberta health law expert.

There's this perception that stem cells are revolutionizing science and they have transformed medicine already, but that's just not the case, said Timothy Caulfield.

You see the word stem cells being used to sell everything from skin cream to sports recovery tools to supplements, it's absolutely everywhere.

Caulfield, who refers to the marketing of spurious stem cell treatments as scienceploitation, explained there are actually only a handful of such therapies that have been approved for use in a clinical setting.

The most well known is probably the use of stem cells in bone marrow transplants and certain kinds of leukemiabut these therapies have been around for decades, he said, adding other stem cell therapies have shown some effectiveness in the treatment of bad burns and blindness.

But that's it.

In a paper outlining a strategy to combat the spread of misrepresentation within this field, Caulfield and Health Law Institute research associate Blake Murdoch argued the first step is to leverage the powers wielded by the provincial colleges of physicians and surgeons.

We need a more robust response from them because they have the power to stop their members from marketing treatments inappropriately and from offering services that are unproven, said Caulfield.

We haven't seen that, and it really is their role to protect the public.

He added organizations aimed at stopping the spread of misinformation and inaccurate marketinglike Ad Standards, Canadas advertising industry's non-profit self-regulating body, or Competition Bureau Canada, the federal advertising regulatorcan also be more involved.

While the Competition Bureau can only prohibit clinics from using misleading advertising and

not the provision of unproven interventions, this would help to stop the spread of misinformation, which may curtail public interest, said Caulfield.

He added political pressure on federal and provincial lawmakers could encourage change and allow a more comprehensive response, but noted that targeting the marketing of these treatments might be the more politically palatable course of action.

I think a really good logical first step is if you're going to market this stuff, if you're going to offer these services, the information you're using to market the services has to be accurate.

Even as the paper was being published, Caulfield said Health Canada weighed in by stating stem cell therapies need its approval.

Basically, if youre an MD, and you're providing stem cell therapy, you need to get it approved, said Caulfield. In the paper, we said Health Canada has got to get more aggressive, and thankfully, we're starting to see some action in that space.

He said ultimately, however, responses from Canadas regulatory bodies are often triggered by complaints from the public.

I've actually spoken to regulators, and theyre not hearing complaints about people being injured by stem cell treatment, said Caulfield. Of course, just because something is safe, doesn't mean it's a good idea.

Not only have some of these treatments shown to have caused real harm while offering little more than hope, Caulfield said there is a financial exploitation element, all of which can only leave a black mark on the science.

The spread of clinics marketing these interventions may, over the long term, damage public trust in legitimate regenerative technologies, thus adversely impacting their future development, he said. It confuses what is an incredibly promising field.

The most perplexing element of the proliferation of these treatments is the involvement of medical professionals who should know better, Caulfield said.

The team went into the analysis with the hypothesis that alternative practitioners were the ones providing and marketing stem cell therapies. This was true, but Caulfield said they were surprised to find that an MD was often involved.

I've been in the room with these health providers, and you get the sense that many of them believe it works.

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Regulatory heft needed to curb false promises on stem cells, says health law expert - Folio - University of Alberta