Surgeon banned over ‘quack’ procedures and ‘reckless disregard’ for safety – Sydney Morning Herald

Dr Bright, who voluntarily surrendered his registration on August 31, was banned from reapplying for seven years.

He was also ordered to pay the costs of the Health Care Complaints Commission (HCCC), which filed the complaints against him in January.

Dr Bright founded Macquarie Stem Cells in Liverpool, which on its website spruiks its experimental treatments as "lead[ing] the way" and "our way of giving back to the community".

Dr Bright was referred to the HCCC by deputy state coroner Hugh Dillon in 2016 over the "preventable" and "unnecessary" death of an elderly patient after an experimental liposuction stem cell procedure to treat her dementia.

The coroner found the 75-year old died due to a "cluster of errors", including a failure to stop taking blood thinning medication prior to surgery, resulting in uncontrolled blood loss.

The tribunal agreed with Coroner Dillon that the treatment bore the hallmarks of "quack medicine".

It also accepted the evidence of Professor Colin Masters from the University of Melbourne, who said there was no evidence stem cell therapy for dementia patients was safe and it was "completely inappropriate and unethical" on a person who was frail, in poor health and in an advanced stage of dementia.

The tribunal found no proper therapeutic basis for Dr Bright administering the same stem cell treatment on a "very vulnerable" patient suffering bilateral vestibular deficiency, a condition where there is difficulty maintaining balance.

The tribunal accepted the patient - who was allegedly told by Dr Bright the procedure was "100 per cent safe" - has been adversely impacted.

The other complaints upheld related to Dr Bright's prescribing of peptides to three patients, including a woman with terminal motor neurone disease.

The tribunal found Dr Bright did not conduct a proper assessment on a patient before prescribing peptides and failed to take adequate steps to obtain informed consent, including an acknowledgement of the lack of clinical data proving the effectiveness of peptides and potential side effects.

Dr Bright's extensive self-prescribing of peptides was "improper and unethical", the tribunal found.

Dr Bright denied the allegations through his lawyers earlier in the year, but instructed his legal team to cease acting for him in August.

He did not attend the tribunal's hearing and did not respond to the orders it handed down.

Carrie Fellner is an investigative reporter for The Sydney Morning Herald.

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Surgeon banned over 'quack' procedures and 'reckless disregard' for safety - Sydney Morning Herald

Five-year-old leukaemia patient Zachary White spots Nessie on dream-come-true Scotland trip – Extra.ie

Five-year-old leukaemia patient Zachary White realised the dream of a lifetime when he saw the Loch Ness Monsterafter a childrens charity stepped in to help.

Rays of Sunshine teamed up with Loch Ness Cruises to give the youngster, from Bracknell, in Berkshire, the chance to spot the mysterious creature.

Zachary, who has had stem cell treatment for acute myeloid leukaemia, told the charity he wanted to watch an ice hockey game in Scotland, hear bagpipes played live and hunt for the Loch Ness monster.

When he arrived at Loch Ness, the youngster was given binoculars, a torch and a compass to hunt for the enigmatic Nessie.

Zachary said: I spotted Nessie I cant believe that I got to see the Loch Ness Monster. Not many people get to see her and I got to see her.

She was green and scaly, a bit like a dinosaur, but a friendly monster.

His parents, Katie and Mark, said: Seeing him back to his old self, so excited and smiling, has been fantastic.

Earlier in the day, the devoted ice hockey fan also got the chance to lead the celebrations for the Glasgow Clan, starting a thunderclap at Braehead Arena in front of 3,000 fans.

Leukaemia patient Zachary Whites dad told the Glasgow Evening Times: It was just the most amazing day, it was really emotional to see everybody come together, people you dont know, people you have never met, all coming together to give our little boy a once in a lifetime experience.

I am so thrilled that we were able to make Zacharys wish come true and in such a spectacular style.

This wish clearly came from Zacharys own imagination, inspiring the Rays of Sunshine team to go above and beyond to create the most magical experience possible, said charity head Jane Sharpe.

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Five-year-old leukaemia patient Zachary White spots Nessie on dream-come-true Scotland trip - Extra.ie

An Expert Discusses PARP Inhibition, Vaccines and Emerging Therapies in Kidney Cancer – Curetoday.com

The field of kidney cancer treatment could soon be expanding thanks to the inclusion of PARP inhibitors, vaccination-based strategies and emerging therapies such as Fotivda (tivozanib), according to research presented at the 2019 Kidney Cancer Research Summit.

In an interview with OncLive, CUREs sister publication, Dr. Sumanta K. Pal, a medical oncologist at City of Hope, discussed the developing findings in these areas and what they mean for patients with renal cell carcinoma (RCC).

CURE: PARP inhibitors are used in treating ovarian, breast and prostate cancers, but what role do they play in RCC? Pal: One of the things that was demonstrated (during the summit) was that it looks as though PARP-1 tends to be expressed at higher levels in RCC, which is a concept that was somewhat foreign to me, but it does jive with some of the recent work thats being done by the Memorial Sloan Kettering group led by Ari Hakimi, suggesting the role of DNA damage-repair mutations in RCC. So when you put those two elements together, it really does set the stage for the potential utility of PARP inhibitors. And one question that came from the audience, asking is this something thats been done before in RCC, and everybody just really drew a blank. Its certainly an area of therapeutic need.

And beyond PARP, I can see us using other novel classes of drugs: ATR inhibitors, ATM inhibitors, and other things that really help in DNA repair, and those sorts of agents that are directed at those pathways may really play a role in this disease.

Are we still looking into the potential of vaccines in kidney cancer?Theres definitely a role for vaccination-based strategies. I think in terms of novel immunotherapy techniques, were going to hear a lot at this meeting thats centered around CAR technologies in RCC. I myself have been working with CRISPR in the development of CD-70 allogeneic CAR-T cell. We discussed that last year at the Kidney Cancer Association meeting for the first time, and were approaching a timepoint where we might ultimately see studies based on that technology evolve, which is very exciting.

Its always been challenging in RCC to pinpoint one particular antigen to go after with CAR-based technologies. In the case of the CRISPR-based technology, we have a unique target that we are going after. And I think that it really stands to benefit a broad swath of patients with RCC based on expression of this unique target.

Can you discuss the recent data involving other therapies such as Fotivda?Tivozanib has a really interesting history. If you look back a couple of years, youll probably recall the discussions we were having around the TIVO-1 clinical trial, where there was a real discordance because we saw a benefit in terms of progression-free survival, but a lack of benefit, maybe even a trend in the opposite direction, for overall survival, and that caused a lot of pause.

In the TIVO-3 study, which was recently announced via press release, we really had a chance, again, to demonstrate the activity of tivozanib, this time in the third- and fourth-line setting. We had already reported out the data at ASCO GU this year, suggesting what I think is a pretty impressive improvement in progression-free survival in these heavily pre-treated patients.

What we had a chance to do more recently, in the context of the press release, is outline the fact that there seemed to be no significant difference in OS (overall survival), not that trend towards worse survival with tivozanib as wed seen previously. So I think that allays a lot of our fears that there might be some downstream impact.

And, of course, the thing to keep in mind is that we have very little control as clinical trialists over what patients are going to get in studies beyond their prescribed therapies in the trial. With that in mind, accounting for all of those characteristics, OS was a bit of a wash in the study.

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An Expert Discusses PARP Inhibition, Vaccines and Emerging Therapies in Kidney Cancer - Curetoday.com

Leukaemia: what is it, how to spot the warning signs and who is at risk? – The Telegraph

How isleukaemia treated?

The treatment of leukaemia varies depending on the patient and type of leukaemia they have.

Acute leukaemia (fast developing) is usually curable with standard treatments, such as chemotherapy.

Chronic leukaemia (slow developing), is often incurablebut treatable. For CLL (a form of chronic leukaemia) some patients are not given treatmentstraight away;however if they do require treatment it will often involve chemotherapy.

The main treatments for leukaemia are:

Chemotherapy: This treatment involves theuse ofdrugs.Chemotherapy drugs either kill cancerous cells or stop them from dividing; they can also kill normal blood cells as a side effect.The type of leukaemia you have will depend on the amount and strength of chemotherapy you are offered, along with other factors such as your age and fitness.

Radiation therapy:Similar to chemotherapy, radiation therapy can be used to destroy the cancerous cells but using radiation waves rather than drugs.Again, the type of leukaemia you have will determine what treatment you're offered. External beam radiation therapy (EBRT) is often used for CLL.It is a fast, painless procedure which usually lasts just a few minutes.

Targeted therapy:Drugs are used to block the growth of cancer cells by disturbing specific molecules in the cells. Targeted therapy can also kill cancer cells by stimulating the patient's immune system to recognise the cells as a threat and consequently kill them.

Biological therapy:This treatment does not target the cancer cells directly, but instead helps to stimulate the body's immune system to act against the cancer. It is also often referred to as "immunotherapy". It is often usedfor patients with CML.

Stem cell or bone marrow transplant: Transplants for stem cells or bone marrow are commonly carried out for patients withacute leukaemia,if chemotherapy does not prove effective.By undergoing a stem cell or bone marrow transplant it can help replenish the healthy bone marrow in patients, and stimulate new growth that restores the immune system. It is usually given to younger, or more healthy patients.

Leukaemia Care, which provides support to individuals and families affected by blood cancer, is one ofthree charities supported by this years Telegraph Christmas Charity Appeal. Our two other charities are Wooden Spoon, which works with Britains rugby community to raise money for sick, disabled and disadvantaged children; and The Silver Line, a 24-hour helpline and support service for lonely elderly people. To make a donation, visit telegraph.co.uk/charity or call 0151 284 1927

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Leukaemia: what is it, how to spot the warning signs and who is at risk? - The Telegraph

Is blood cancer curable at an early stage? – The Indian Awaaz

If yes, what are the treatment options available?

Health Desk

Cancer is that one deadly disease that every single person dreads.If you are witnessing persistent symptoms in your body that isnt normal, it isvery important to consult a doctor. Lack of persistence is what causes theproblem to become big and spread around. If that is the case, you must focus onfinding cures for the condition. Blood cancer is one such condition. Therecould be anything causing this disease, gene mutation or unknown factors.

The condition of blood cancer isnt likeany normaldiseasethat normal treatment can cure. You need propertreatment methods implemented to recover and get rid of the side effects thatit comes with. In several cases, you must focus on finding better treatmentoptions. Some of the types of blood cancer are treatable while some are not.

But, is it treatable at an early stage?

The answer to this question is Yes. You can treat the condition ifit is detected at an early stage among blood cancer stages. Theonly thing that you need to pay attention to is to acknowledge the symptoms youare experiencing.

Seeing a doctor get the symptoms checked at an early stage canrule out a lot of the risks that the majority of the people face.

What are the treatmentoptions?

As we did mention before, it is possible to cure cancer if it isdetected at an early stage.

Some of the possible treatment options include:

Intensive treatment

Even though this is secured for the last resort, these forms ofaggressive treatment in the initial stage can eradicate the condition from theroot. Some of the treatment types in such cases include chemotherapy or stemcell transplant. Often, the doctors even opt for radiotherapy if the cancer islocalized in a specific area instead of being spread out. Surgery is also partof the high-intensity treatment option but that is always the last resort.

Low intensity

If the doctors find that blood cancer is still at its initialstage and hasnt spread across, the best bet to fight that is withlow-intensity treatment options such as respite care.Apart from a low dosage of chemo and radiotherapy, immunotherapies are also anadded benefit that can help the immune system fight back the cancerous cellsfor the better.

Curative and non-curative options

These kinds of treatment approaches are often tough to understandand depend on the prognosis and come as a secondary step after the intensive orlow-intensity treatment is done. If the patient is in remission, they will findother ways to help keep them in that condition with consistent treatment alongthe way.

Blood cancer can be cured if detected at an early stage. Knowing the symptoms can help you seek immediate medical attention that later can save your life as well. If you feel like something is wrong, dont keep it to you, but speak to a doctor.

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Is blood cancer curable at an early stage? - The Indian Awaaz

Restore your crowning glory with recombinant DNA tech – The New Paper

Hair care is being taken to the next level, by utilising recombinant DNA technology to restore one's confidence and crowning glory.

Such hair restoration products or treatments are made using recombinant DNA - or DNA cloning - where selected pieces of DNA from different organisms are combined to construct artificial DNA.

At Ageless Medi-Aesthetics, its latest AnteAge MD Hair Treatment is a non-invasive procedure that involves applying the AnteAge MD Hair Growth Factor solution or serum - made from potent recombinant growth factors and cytokines - onto skin prepared with microneedling.

Dr Lam Bee Lan, director of Ageless Medi-Aesthetics, told The New Paper: "Recombinant DNA technology is more efficient in producing large amounts of artificial messenger proteins effective for skin and hair renewal compared with stem cells derived from plants."

Methods of hair restoration are often divided into two broad categories - invasive techniques and topical and/or oral solutions. They can either be expensive or linked to side effects such as erectile dysfunction, ejaculatory dysfunction and loss of libido.

But Dr Lam cautioned that before treatments are prescribed, patients must consult with a physician to ascertain if they are suitable for them.

"Treatments based on recombinant DNA technology should be worked in as a first-line treatment when you start to experience more hair loss than usual, or as part of a regular routine in maintaining a full head of hair.

"For more severe hair loss, patients should consider a hair transplant," she said.

While there are minimal side effects such as occasional soreness and redness that will resolve within one to two hours, Dr Lam noted that most patients will experience slowing down of hair loss after the first session, while new hair will grow after the second session.

Home-grown scalp specialist PHS Hairscience has also explored stem cell technology and cell signalling technology since 2014 to treat hair loss or greying hair on the cellular level.

Ms Anita Wong, its chief executive and founder, told TNP: "As the body ages or changes due to reasons such as stress or lifestyle choices, cell functions can deteriorate, and cell activity that directly impacts new hair growth or melanin (hair pigment) production becomes less than optimal."

PHS Hairscience's marquee treatment, Miracle Stem Cell Solution, leverages on stem cell science and cell signalling to reactivate dormant follicle cells to promote hair growth. At $297 a session, it can be complemented with the FEM/HOM Thickening range of products.

She said: "These active botanical stem cells also work to increase the life span of hair follicles so your hair can remain in the anagen (growth) phase of the hair growth cycle for a longer period of time.

"Keeping the hair in this growth phase will maximise the length and thickness of new hair, as well as stop the existing strands from shedding."

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Restore your crowning glory with recombinant DNA tech - The New Paper

Stem Cell Therapy: What’s Real and What’s Not at California’s For-Profit Clinics – UCSF News Services

Many for-profit stem cell clinics advertise therapies that are not backed by science and may actually cause harm.

For-profit stem cell clinics have popped up around California in recent years, advertising that they can treat everything from arthritis to Alzheimers, without FDA approval.

They claim that injections of stem cells (naturally occurring blank slate cells that can grow into any type of cell) can help alleviate pain or illness by replacing or regenerating diseased tissue claims that are not supported by existing research. The procedures can cost thousands of dollars out-of-pocket, and regulators have warned that patients have developed tumors, suffered infections and even lost eyesight after unapproved procedures.

No one knows how many clinics there are, but California reportedly has more than any other state. We asked Arnold Kriegstein, MD, PhD, director of the UC San Francisco Developmental & Stem Cell Biology Program, about whats real and whats not in stem cell medicine.

How do these clinics operate?

There has been an explosion of so-called clinics offering stem cell treatments for a wide range of ailments, none of which have been shown to be effective. They are largely unregulated. Many clinics claim that they can treat untreatable illnesses like Alzheimer's disease, autism, muscular dystrophy, or stroke. The list is quite extensive.

The majority are using fat tissue for their stem cells, obtained through liposuction. These are usually autologous cells, which means that they are taking the patient's own tissue and extracting cells to re-administer to the same patient, usually through an intravenous route. In addition to fat cells, some clinics administer bone marrow stem cells or umbilical cord or placental stem cells, which come from unrelated donors.

The clinics often advertise through testimonials from patients who've received their therapies. Many of the conditions that the testimonials address are the kinds that normally improve or fluctuate over time, such as joint pain, low back pain, arthritis, or multiple sclerosis.

The problem is that patients will receive a treatment, and then, within a month or two, they'll notice that the aches and pains in the joints are improving, and they will attribute the improvement to the stem cell therapy, when in fact it would've happened regardless.

What is the risk of trying an unproven stem cell treatment?

Reports of physical harm have included infections and the development of tumors. When using cells that are not the patients own, umbilical cord cells for example, immune responses can occur often triggering inflammatory conditions.

In cases where stem cells have been delivered into the eye, blindness has been reported, and when they have been delivered to the central nervous system through lumbar puncture (spinal tap), adverse outcomes including serious infections of the central nervous system and tumors have occurred.

Then there's the emotional cost associated with raising false hope, and the financial loss that comes from exorbitant fees charged for ineffective, potentially harmful therapies.

Why arent there more legitimate stem cell therapies available?

Stem cells have been in the news so much over the last decade or so that I think it has created the impression that therapies are already on the market. The reality is that it is very early days for the science. The most interesting, most promising animal studies are only now beginning to be translated into clinical trials, and the process for approval of therapies takes many years and very few are likely to succeed.

Unfortunately, the public needs to be patient, but the good news is that potential treatments are progressing along the pipeline.

What are some examples of proven stem cell therapies?

For the last 50 years or so, there have been countless patients successfully treated with hematopoietic stem cells, commonly known as bone marrow transplants. This remains the prototype for how a stem cell therapy can work. Other successful examples include corneal stem cell grafts for certain eye conditions, and skin grafts for burn victims.

There are efforts to see if stem cells could successfully treat diseases like Parkinson's and diabetes, particularly type 1 diabetes. There are clinical trials testing whether stem cell therapy might work against macular degeneration, a blinding disease that is very common as people age. There are also early stage clinical trials for nervous system disorders including stroke, spinal cord injury, and ALS (Lou Gehrigs disease).

All of these examples are still at a very early stage, where the primary goal is to make sure that the approaches are safe. To determine if they are effective will require large, well-controlled, relatively long-term clinical trials.

What will it take to advance stem cell therapy into more real treatments?

This is where basic research comes in. The field is evolving quickly, there's much to be done, and there's still a huge amount of promise in stem cell therapies down the road. But it's going to take a lot of very careful and very laborious research before we get there.

Link:
Stem Cell Therapy: What's Real and What's Not at California's For-Profit Clinics - UCSF News Services

The Science Of Stem Cell Research Hits A Snag With Bad Batch – Forbes

Bad Batch logo

Dr. Death was a brilliant series about a rogue doctor that was unqualified at his job and made a lot of bad decisions that injured or killed many people. This terrifying tale of medical malpractice also shone a light on the problems with the medical system that could allow such an individual to bounce around from office to office and keep his medical license.

Laura Beil, host and reporter of Dr. Death, did an amazing job on a series that at times felt like a horror movie, but she just may have topped herself with her new six-part series, Bad Batch, that doesnt just call into question medical practices, but an entire largely unregulated industry - stem cells.

Heres the show summary from Wondery:

Patients are offered a miracle cure, but instead they end up rushed to the hospital in critical condition. The race is on to track down what went wrong with several patients in Texas before more people get hurt. The trail leads to a stem cell company with a charismatic CEO, and to an entire multibillion dollar industry where greed and desperation collide.

The bulk of the show is about John Kosolcharoen, the charismatic CEO of Liveyon, an ambitious stem cell company that raced ahead of existing research to provide cure-alls to patients who can afford the $5000 injections. After successfully injecting himself with stem cells and later his mother, he was convinced that he had found the answer that people were looking for, along with a way to make a lot of money.

After people starting getting sick, all the bad batch of vials of stem cells were destroyed and the finger-pointing began on who was to blame. John blamed the doctors who purchased the stem cells from him for improper handling of the vials even though the FDA didnt share his conclusions.

Host and researcher Laura Beil expanded the basic news story to look at the science and regulation behind stem cells and explain it in an easy to understand way. John K, as hes referred to in the podcast, was more than happy to talk to her because he did not see himself as the villain, which made the story more complicated.

At a Wondery listening party Q and A for Bad Batch, Laura told me that to tell a big story like this narratively You have to pick out the details that matter most, but leave in enough to tell the story. You have one central character, the industry itself, the science of it, and the regulations.

The main reason that any of this happened, according to Beil, is that stem cells are not treated as drugs and the FDAs attitude is that its not a drug to regulate since youre just moving parts of your own body around.

There is a legitimate scientific supported use for stem cells, and the promise is legitimate but theyre being abused. Liveyon spent a million dollars on a commercial seminar for doctors who were pitched on how much money they could make and then given a script of exactly what to say to patients.

Liveyon was preying on people for whom the promise of a miracle cure from stem cells was their last hope for a better life and they were happy to pay whatever it took. Their ads for patients were like siren songs that caused people to want to believe that its going to work and that belief creates a placebo effect.

Ive been writing about medicine for 25 years, Laura says, and the placebo effect is powerful. The more you pay for something the stronger the placebo effect is.

Hernan Lopez, the CEO of Wondery, was at the Q and A and asked Laura, Should the fact that the treatments are cash only be a red flag?

Yes!, Laura responded, but there are patients that have the idea that medical research is too slow. For those who still want to try stem cells please do your research first.

The scary part is that theres still a lot we dont know about stem cell treatments. As Laura pointed out, we dont know whose a good candidate and we dont know whats a safe treatment. Its all about informed consent but people arent getting it, she says.

Lauras advice for people who are desperate for something to happen is to join a clinical trial where you are monitored by doctors with an expected outcome. Unfortunately, theres evidence that 90 percent of drugs that go into clinical trials dont work and the general public is often misled by slick advertising that often uses exaggerated or misleading claims.

And beyond all the science and the explanations, theres the story of the man behind it all. John K. is a quintessentially American figure, a smooth talker who got in over his head Liveyon and he comes across as a very sympathetic character which makes him hard to hate.

Its an utterly devastating story that hopefully will cause people to think twice before paying cash for the dream of an instant fix to all their problems.

The first four episodes of Bad Batch are available now or you can sign up for Wondery Plus and listen to all six.

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The Science Of Stem Cell Research Hits A Snag With Bad Batch - Forbes

Mallinckrodt Announces UVADEX (Methoxsalen) Approved in Australia for use with the THERAKOS CELLEX Photopheresis System for Treatment of Chronic Graft…

STAINES-UPON-THAMES, United Kingdom, Oct. 31, 2019 /PRNewswire/ -- Mallinckrodt plc (NYSE: MNK), a global biopharmaceutical company, today announced thatUVADEX(methoxsalen) has received regulatory approval in Australia by the Therapeutic Goods Administration (TGA) for extracorporeal administration with the THERAKOSCELLEXPhotopheresis System. The treatment is indicated for steroid-refractory and steroid-intolerant chronic graft versus host disease (cGvHD) in adults following allogeneic hematopoietic stem cell (HSC) transplantation. The TGA also approved Uvadex in conjunction with the THERAKOS CELLEX Photopheresis System for the palliative treatment of skin manifestations of cutaneous T-cell lymphoma (CTCL) that is unresponsive to other forms of treatment.

The TGA approval marks the first combined indication label and the first regulatory approval in the world for UVADEXin conjunction with the THERAKOS Photopheresis System for the treatment of chronic graft versus host disease in adults.

"The TGA approval of UVADEX with the Therakos ECP platform opens up new treatment options for patients with these challenging conditions," said Steven Romano, M.D., Executive Vice President and Chief Scientific Officer, Mallinckrodt. "The cGvHD indication is also an important milestone for Mallinckrodt, confirming the potential benefit of this therapeutic option for patients who are refractory to or intolerant of steroid treatments."

About Chronic Graft Versus Host Disease (cGvHD)Graft-versus-host-disease is a common complication of hematopoietic stem cell (HSC)transplantation resulting in significant morbidity and mortality.1 It can be classified as acute or chronic based on the clinical presentation and the time of occurrence after the transplantation. Signs and symptoms of cGvHD nearly always occur within the first year post transplantation but can occasionally happen several years later.2 In cGvHD, the skin is the most frequently affected organ with manifestations of itchy rash, hyper or hypopigmentation and changes in texture. However, the disease can affect multiple sites, which may have a major impact upon a patient's quality of life.2,3Chronic GvHD can lead to debilitating consequences, such as joint contractures, loss of sight, end-stage lung disease, or mortality resulting from profound chronic immune suppression leading to recurrent or life-threatening infections.1

About Cutaneous T-Cell Lymphoma (CTCL) Cutaneous T-cell lymphoma (CTCL) is an umbrella term for a group of non-Hodgkin lymphomas involving T lymphocytes that localize in the skin. It is a relatively rare cancer, with 2,500 to 3,000 new cases per year in the United States.4 The age of onset of the condition is typically greater than 50 years, with the incidence rising significantly in the later decades of life.5 CTCL causes visible skin symptoms ranging from a small rash to extensive redness, peeling, burning, soreness, and itchiness all over the body.6,7 CTCL falls into different categories based on the severity of the disease and symptoms.8

Minimum Product Information: UVADEX(methoxsalen) Concentrated Injection for extracorporeal circulation via photopheresis (ECP)

This medicinal product is subject to additional monitoring in Australia. This will allow quick identification of new safety information. Healthcare professionals are asked to report any suspected adverse events at http://www.tga.gov.au/reporting-problems.

Indicationsin Australia: UVADEX (methoxsalen) is indicated for extracorporeal administration with the THERAKOS CELLEX Photopheresis System for the:

Contraindications:History of idiosyncratic or hypersensitivity reaction to methoxsalen, psoralen compounds or any excipients of UVADEX; coexisting melanoma, basal cell or squamous cell skin carcinoma; lactation; aphakia. ECP procedure contra-indications: Photosensitive disease; inability to tolerate extracorporeal volume loss; WBC count > 25,000 mm3; previous splenectomy; coagulation disorders. Special warnings and precautions:Only physicians who have special competence in the diagnosis and treatment of cGVHD and CTCL who have special training and experience with the THERAKOS CELLEX Photopheresis System should use UVADEX. Men and women being treated with UVADEX should take adequate contraceptive precautions both during and after completion of photopheresis treatment. Exposure to large doses of UVA causes cataracts in animals, an effect enhanced by the administration of oral methoxsalen. The patient's eyes should be protected from UVA light by wearing wraparound, UVAopaque sunglasses during the treatment cycle and during the following 24hours. Exposure to sunlight or ultraviolet radiation (even through window glass) may result in serious burns and, in the longterm, "premature aging" of the skin therefore patients should avoid exposure to sunlight during the 24hours following photopheresis treatment. Thromboembolic events, such as pulmonary embolism and deep vein thrombosis, have been reported with UVADEX administration through photopheresis systems for treatment of patients with graft versus host disease. This product contains 4.1% w/v ethanol and each 1 mL of UVADEX contains 40.55 mg of ethanol. Caution is advised in patients with liver disease, alcoholism, epilepsy, brain injury or disease. No specific information is available for use in renal or hepatic impairment and there is no evidence for dose adjustment in the elderly. The safety and efficacy of UVADEX have not been established in children. Use in pregnancy: Category D. Use in Lactation: UVADEX is contra-indicated. Interactions with other medicines: Effects on P450 system metabolism may affect clearance / activation of other drugs (caffeine, paracetamol) or may extend the methoxsalen half-life leading to prolonged photosensitivity in patients. Methoxsalen binding to albumin may be displaced by dicoumarol, warfarin, promethazine and tolbutamide with potential for enhanced photosensitivity. Caution when treating with concomitant photosensitising agents. Adverse effects: In the clinical trials, published information and postmarketing surveillance of UVADEX/ECP, adverse events were usually mild and transient and in most cases, related to the underlying pathology. Verycommon: diarrhoea, anaemia, nausea, headache, hypertension, sinusitis, upper respiratory tract infection, fatigue, pain in extremity, pyrexia, cough, dyspnoea, cushingoid, dry eye, photophobia, toothache, anorexia. Common: depression, lacrimation increased, abdominal pain, hypokalaemia, paraesthesia oral, pharyngolaryngeal pain, tachycardia, conjunctivitis, eye pain, visual acuity reduced, dysphagia, chills, mucosal inflammation, nasopharyngitis, contusion, blood pressure diastolic decreased, haemoglobin decreased, hyperglycaemia, hypocalcaemia, neuropathy peripheral, tremor, rash, hypotension. Additional adverse effects seen in clinical trials include vomiting, infections. Adverse events related to the ECP/CELLEX procedure thromboembolism and severe allergic reactions, vascular access complication, vasovagal spasm, hickman catheter infection/thrombosis, headache, hypercoagulability, haemolysis.Additional adverse events identified post-marketing: anaphylactic reaction, allergic reaction, dysgeusia, exacerbation of congestive heart failure, sepsis, endocarditis, and vomiting. Dosage and Administration: Chronic Graft versus Host Disease: Three ECPtreatments in the first week then two ECP treatments per week for at least 12 weeks, or as clinically indicated. Cutaneous T-cell Lymphoma: ECP treatment on two successive days each month for six months. Patients who show an increase in skin scores after eight treatment sessions may have their treatment schedule increased to two successive days every two weeks for the next three months. Refer to full Product Information and THERAKOS CELLEX Operator's Manual for information regarding administration.

Store below 25C. Date of first approval: 16 September 2019. Date of revision: 11 October 2019.

Indications and Prescribing Information for Uvadex vary globally. Please refer to the individual country product label for complete information.

Before prescribing Uvadex, please refer to the full Product Informationalso available by calling+ 1 800.778.7898.

ABOUT THERAKOSMallinckrodt is the world's only provider of approved, fully-integrated systems for administering immunomodulatory therapy through ECP. Its Therakos ECP platforms, including the latest generation THERAKOS CELLEX Photopheresis System, are used by academic medical centres, hospitals, and treatment centres in nearly 40 countries and have delivered more than 1 million treatments globally. For more information, please visitwww.therakos.co.uk.

Terumo BCT is the exclusive distributor of the Therakos ECP platform in Australia, as well as Latin America and select countries in Europe. To learn more about Terumo BCT, visit http://www.terumobct.com.

UVADEX (methoxsalen) and THERAKOS CELLEX Photopheresis Systems are separately approved in a number of global markets. Please refer to your local approved labelling for Uvadex and the Operator's Manual for CELLEX for more information on approved uses for specific indications.

Before administering therapy using the THERAKOS CELLEX Photopheresis System, please refer to the Operator's Manual available at +61 2 9429 3600or +1 (800)778-7898.

ABOUTMALLINCKRODTMallinckrodt is a global business consisting of multiple wholly owned subsidiaries that develop, manufacture, market and distribute specialty pharmaceutical products and therapies. The company's Specialty Brands reportable segment's areas of focus include autoimmune and rare diseases in specialty areas like neurology, rheumatology, nephrology, pulmonology and ophthalmology; immunotherapy and neonatal respiratory critical care therapies; analgesics and gastrointestinal products. Its Specialty Generics reportable segment includes specialty generic drugs and active pharmaceutical ingredients. To learn more about Mallinckrodt, visit http://www.mallinckrodt.com.

Mallinckrodtuses its website as a channel of distribution of important company information, such as press releases, investor presentations and other financial information. It also uses its website to expedite public access to time-critical information regarding the company in advance of or in lieu of distributing a press release or a filing with theU.S. Securities and Exchange Commission(SEC) disclosing the same information. Therefore, investors should look to the Investor Relations page of the website for important and time-critical information. Visitors to the website can also register to receive automatic e-mail and other notifications alerting them when new information is made available on the Investor Relations page of the website.

CAUTIONARY STATEMENTS RELATED TO FORWARD-LOOKING STATEMENTSThis release includes forward-looking statements for Mallinckrodt concerning THERAKOS Photopheresis including potential benefits associated with its use. The statements are based on assumptions about many important factors, including the following, which could cause actual results to differ materially from those in the forward-looking statements: satisfaction of regulatory and other requirements; actions of regulatory bodies and other governmental authorities; changes in laws and regulations; issues with product quality, manufacturing or supply, or patient safety issues; and other risks identified and described in more detail in the "Risk Factors" section of Mallinckrodt's most recent Annual Report on Form 10-K and other filings with the SEC, all of which are available on its website. The forward-looking statements made herein speak only as of the date hereof and Mallinckrodt does not assume any obligation to update or revise any forward-looking statement, whether as a result of new information, future events and developments or otherwise, except as required by law.

CONTACTSMedia InquiriesJames Tate The Henley Group+1 44 1491 570 971james@henley.co.uk

Investor RelationsDaniel J. Speciale, CPAVice President, Investor Relations and IRO314-654-3638daniel.speciale@mnk.com

Mallinckrodt, the "M" brand mark and theMallinckrodt Pharmaceuticalslogo are trademarks of aMallinckrodtcompany. 2019Mallinckrodt. 10/19. FR-1900004

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SOURCE Mallinckrodt Pharmaceuticals

http://www.mallinckrodt.com

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Mallinckrodt Announces UVADEX (Methoxsalen) Approved in Australia for use with the THERAKOS CELLEX Photopheresis System for Treatment of Chronic Graft...

Q&A: Draper’s Jenna Balestrini on the future of CAR-T – – pharmaphorum

As exciting as CAR-T is, there are still myriad challenges preventing it from reaching its full potential. Jenna Balestrini, head of strategy and business development at Draper, gives us her views on the biggest barriers to wider usage of these innovative treatments and how to overcome them.

Is CAR-T living up to its promise yet?

The promise of CAR-T is that we can have a single-dose curative solution to treat cancer, and in some ways that is true. There are some cases where a patient (such as Emily Whitehead) was dosed with an autologously-sourced cellular therapy after traditional approaches of radiation and chemo failed, and that cell therapy eradicated cancer in their body.

In that way CAR-T has and is living up to its promise as a curative solution. However, this is not the case for the majority of patients who receive this therapeutic. There are a number of issues including relapse, lack of specificity, limited response, toxicity and side effects. Also, there is a general lack of patient access, especially outside of the indications already approved for the two available cell therapies (Yescarta and Kymriah). Unless a patient is lucky enough to get into a certain trial, under certain care, with a certain kind of cancer, and with a certain kind of genetic predisposition, CAR-T may not work for them.

What needs to be done to fulfill the promise of CAR-T are two things. As a scientific community, we need to think more critically about ways we can broaden the abilities of these biologic agentsthese living cell therapiesto treat not just the immediate hematologic blood cancers but also those patients who have gone through multiple rounds of chemotherapy and radiation, which can seriously damage their initial blood material.

We also need to make a system that is much more one-size-fits-all in terms of its capability of treating cancer. So, the starting material needs to be better, and the biology needs to be more specific. For example, in the case of solid tumors, you need to have a scenario where the cell-based therapy doesnt introduce rejection via either graft-versus-host or host-versus-graft disease. We need to have a scenario where if you have a patient population that doesnt have, say, enough healthy T-cells to begin with, or have enough healthy blood material to work with, we will have a source for that starting material.

What are the biggest remaining unmet needs for patients when it comes to CAR-T?

There are both scientific and business needs that must be understood for these therapies.

The unmet scientific need is understanding why some of these CAR T-cell therapies work and why some dont. As scientists, we need to understand why certain patients respond better, and why we have scenarios in which we can readily adapt these therapeutics for some kinds of cancer but not others. Finally, we need to be able to create biologics that are readily available so that patients can get them when they are initially sick and not after multiple rounds of other options, like chemotherapy and radiation.

There are a large number of studies that show once a patient completes one to two rounds or more of chemotherapy or radiation, their cells are unable to efficaciously be changed into a cell-based therapy. Getting a patients materials earlier would be beneficial. For example, the first time you, as a patient, are diagnosed with cancer, you could have your cells removed and made into a therapeutic, and that would give you a much greater chance for the therapy to be successful. It would also avoid the current situation where your starting material is damaged through rounds of chemotherapy.

In terms of unmet business needs, it is safe to say that these customised immunotherapies are on the brink of revolutionizing how we treat cancer. The commercially approved applications of CAR-T therapies are currently limited to treating just a few kinds of blood cancers, and even these indications require a patient to have undergone several other therapies unsuccessfully. The reality is that when we do develop therapies for larger patient indications, the current healthcare system cannot afford them. Consider the cost of treating 300,000 cancer patients a year, roughly half of those that die from their disease. At $373,000 per patient, the increased burden to the healthcare system would not be sustainable. This cost does not include other associated costs from providing the therapy to the patient hospitalisation, medical support, etc. The unmet need is having a means to manufacture therapeutics at high volume, in significantly less time (i.e. less than 3 days), and at significantly lower cost per therapy. Patient access to these potentially life-saving therapies will require that the costs do not significantly increase the burden on the established healthcare systems.

What are some other barriers to widespread adoption of CAR-T and accessibility for patients?

Other barriers center on patient access to the therapy. For instance, one barrier is how clinical trials are set up, which patient cohorts are chosen and therefore are the contingency that are approved for the therapy use, and ultimately what patients are allowed to have access to them.

Another barrier is the current approach to introducing CAR T-cell therapy only after other treatment options are exhausted. This is a big challenge because its not just about the patients starting material but its about how sick the patient is once they get to you. A patient may have gone through seven rounds of chemotherapy and radiation, and now they are compromised. If we can make CAR-T a front-line therapy, it would make it more accessible and affordable.

What makes CAR-T treatments so expensive? Are there any ways to reduce costs?

One of the reasons CAR-T treatments are so expensive is that the industry does not have an automated, systematic manufacturing process, and because of that, there is an over-reliance on using instrumentation and processes that have been borrowed from other therapies, like blood banking or stem cell transplantation. Most cell therapy manufacturing processes came out of academic institutions. In academia, your touch labor is typically grad students and therefore is inexpensive, while the touch labor in industry is trained professionals and comes at a much higher cost. The high degree of touch labor also contributes to a breakdown in therapy development and hinders its scalability. Tissue engineering, as a field, suffered broadly for these exact reasons.

What difficulties exist in manufacturing CAR-T therapies? How are they affecting access?

CAR T-cells are a promising therapy, but the current state of biomanufacturing is very complex, expensive and time-consuming, often requiring multiple rooms, highly skilled technicians and many instruments. The process for developing CAR T-cell therapies involves gathering T cells from the cancer patients blood, re-engineering them in a lab so they will recognise cancer cells as an enemy and then reintroducing these enriched T cells to the patients body. The current price tag for a single treatment is $450,000. The typical timeframe is 12-17 days.

One reason for this bioprocessing bottleneck is that manufacturers are using equipment thats been retrofitted from other purposes, and then strung them together. With each step in the process, manufacturers typically have to conduct extra quality control measures, which can result in lost time and material and significant expense.

Whats needed is a bioprocessing platform that can help cell therapy developers bring their therapies to market more quickly, safely and affordably.

Draper is working to develop microfluidic platforms that will perform key steps of the CAR T-cell manufacturing process, as well as processes for other types of cell therapies. Innovations include a technique known as acoustophoresis for separating and enriching the immune system cell population that includes T cells using sound energy, as well as a platform for accelerating the process for transferring genetic material into T cells from the current one-to-three days to less than a day.

The goal of these microfluidic technologies is to create improvements in the cell therapy manufacturing process by combining fluidic control, precision and the ability to scale to higher throughput systems for easy integration with existing manufacturing equipment.

What are your future plans for the technology?

I can imagine a scenario where Draper is not just making devices that manufacture cell-based therapies but going further by incorporating artificial intelligence (e.g., machine learning) into these devices. This intelligence would enable us to not just study and learn from the data that is being extracted from our devices but also inform the process. I think thats the future of really smart engineering.

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Q&A: Draper's Jenna Balestrini on the future of CAR-T - - pharmaphorum