Stem Cell Clinic

Australian cell therapy company Mesoblast joins forces with Germany’s Grnenthal to tackle low back pain – Endpoints News

While the FDA reviews a rolling application for its experimental stem cell therapy for acute graft versus host disease (GVHD), Australias Mesoblast has tied up Germanys pain specialist Grnenthal to develop a treatment for chronic low back pain caused by degenerative disc disease.

Under the deal, Grnenthal will gain exclusive commercialization rights to the drug, MPC-06-ID, for Europe and Latin America. Mesoblast, which is testing the drug in a US late-stage study that is expected to readout next year, is getting $150 million in upfront and milestone payments, in addition to potential royalties. Cumulative milestone payments could surpass $1 billion, the companies said on Monday.

A single injection of the drug, which is being developed for use in patients who have not benefited from existing options, resulted in over a three-fold increase, versus placebo, in successfully achieving a composite goal in a 100-patient mid-stage study. The endpoint consisted of 50% improvement in low back pain and 15 point improvement in function at both 12 and 24 months with no treatment or surgical interventions at the treated level through 24 months. Overall, 37% of patients treated with MPC-06-ID compared with 10% in the control group met the composite endpoint over two years, Mesoblast said.

In the United States, the FDA has amped up its scrutiny of stem cell clinics promising wonder cures without a shred of evidence. Mesoblast is putting its technology which is based on mesenchymal lineage adult stem cells (rare cells found in the vicinity of blood vessels that promote tissue repair and modulate immune responses) through the clinic to test its viability across a variety of conditions such as GVHD, heart failure, pain, rheumatoid arthritis and Crohns disease. The companys off-the-shelf GVHD cell therapy, Temcell, has been approved in Japan (and is licensed by partner JCR Pharmaceuticals in the region).

MPC-06-ID is designed as an intra-discal injection engineered to degenerate intervertebral discs to allow the mesenchymal lineage adult stem cells (MLCs) to secrete biomolecules involved in enhanced migration and proliferation of intervertebral disc progenitor cells, in a bid to strengthen the load-bearing function of the disc by improving its stability and enhancing its water content, while also reducing inflammation and pain.

Grnenthal and Mesoblast have agreed on an overall development plan for MPC-06-ID to meet European regulatory requirements, and the two partners will accordingly work on a late-stage design for Europe. The results of the European and US trials are expected to support regulatory approvals in both regions, the companies said.

Among key characteristics of Mesoblasts technology is that the companys products are fertile for significant expansion in culture, Mesoblast chief executive Silviu Itescu noted. These properties facilitate their use as allogeneic, or off-the-shelf, therapeutics with well-defined release criteria and batch-to-batch reproducibility that meet stringent regulatory requirements.

These are some of the challenges faced by the first generation of cell therapies which are autologous CAR-T therapeutics Novartis $NVS Kymriah and Gileads $GILD Yescarta which secured FDA approval in 2017. These products require the manufacturers to first isolate cells from the patient, manipulate them in the lab by adding chimeric antigen receptors to direct T cells to snuff out cancer cells and then re-infuse them back into the patient. This process requires enormous manufacturing heft a challenge that has triggered relatively muted sales for Kymriah, with Novartis struggling to meet demand.

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Australian cell therapy company Mesoblast joins forces with Germany's Grnenthal to tackle low back pain - Endpoints News

Reprogramming the Human Computer: Silicon Valley Meets Cell and Gene Therapy – BioBuzz

How will Cell and Gene Therapy Usher in a New Industrial Revolution?

Cell and gene therapy companies are on the verge of transforming how we treat unmet medical needs such as cancer, rare diseases, genetic disorders and diabetes, to name only a few. By using a patients own cells and genes to fight disease, cell and gene therapy treatments deliver highly-targeted therapies and cures for unserved and underserved patients in need of solutions to a wide variety of intractable diseases.

What was once relegated to science fictionlike the miniaturized heroes sent into a human body in the Fantastic Voyage sci-fi movie of 1966has, to a large degree, become reality with modified cells being put into patients to act as virtual bots capable of clearing diseaseseven cancersfrom the body. New technologies are empowering science to do what would never have been thought possible years ago.

Understanding the complexities of how these novel personalized medicines actually work can be daunting and confusing for many people, especially for the patients who are in need of these new medicines and those who are non-scientific minded.

Jeff Galvin, CEO of American Gene Technologies (AGT), has helped a wide variety of audiences understand this new technology by explaining the similarities between the human cell and an organic computer. He explains that DNA is the operating system of the human cell. It contains commands for the cellular machinery called genes, which are coded in four symbols: A, G, T, and C (for the nucleotides adenine, guanine, thymine, and cytosine). The order of these nucleotides determines the instruction that a gene provides to the machine just like the order of 1s and 0s in your personal computer or cell phone determine the commands to be executed by the device. Galvin coined this human computer analogy to describe the work that his company is doing and what is actually taking place with cell and gene therapies to make new solutions for formerly unaddressable human diseases.

Galvin describes gene and cell therapy as the software revolution for the next 100 years: reprogramming DNA in cells to improve health. Scientists have long understood that viruses infect cells and hijack them with new instructions (viral DNA) to cause disease. Over the last few decades, methods have been developed to crack open viruses, scoop out their bad instructions that make cells sick, and replace them with new good instructions that improve the operation of the cell. In a way, the gene and cell therapy industry is hijacking the hijacker, says Galvin. We are taking viruses and converting them to updates to fix bugs or improve the operating system (DNA) of human cells. Just like viruses and updates on your computer work. The possibilities are endless. We can use these updates to repair a broken gene that is the root cause of a disease, insert new instructions into cells to improve their operation allowing them to do their jobs better, clear or protect the body from disease, or even change the operation of cells to make them little bots that can carry out functions that they would not normally do in your body, like clearing cancer. All of these things are within the scope of reprogramming the human computer.

American Gene Technologies (AGT), a biotech company located in Rockville, Maryland, is a company where Silicon Valley tech innovation and the human computer mindset is converging to rethink the approach to developing medicine.

AGTs CEO Jeff Galvin has shared that he and other cell and gene therapy leaders are on a mission to reprogram the human computer to save lives and improve outcomes for patients fighting infectious diseases, monogenic disorders, cancer and other devastating illnesses.

A serial tech innovator, Galvin made his mark as a Silicon Valley entrepreneur. He retired in his early 40s in 2002. His retirement was short-lived. When Galvin discovered the groundbreaking viral vector work of the National Institute of Healths (NIH) Dr. Roscoe Brady, he exited retirement to do what he always does: Vigorously pursue what he loves.

Galvin leapt out of retirementin 2007 to start AGT, and to continue developing Bradys technology.

I was very lucky to meet Roscoe Brady. When he showed me viral vectors and I realized we now had the ability to modify DNA with viruses, I immediately felt that this is the future of pharmaceuticals, stated Galvin. I could see the inherent power in this approach and how viral vectors could bring opportunities to new biotech companies to cure diseases that were formerly untreatable or incurable.

Bradys commitment to solving diseases and improving lives was also infectious. Dr. Brady was retiring, and it looked like this brilliant work was going to be shelved. explains Galvin. I saw it as too valuable and potentially world-changing to ignore. Dr. Brady agreed to stay on as a scientific advisor and I founded AGT with the mission to find the most efficient, effective, and fastest ways to bring this cutting edge technology to patients in need.

A lot of what I imagined as a computer programmer and software/IT specialist coming from the West Coast is actually coming true for this technology. This vision has fortunately attracted a lot of great scientists to our company and they are doing all of the rocket science. The concept is correct: We can use viral vectors to hijack the viruses that have been hijacking our cells for 1.5 billion years, using the inherent ability of viruses to infect cells and carry malevolent genetic code to deliver benevolent code instead,. Galvin stated.

What I am witnessing in our part of this industry is that high tech has come to drug development. The capabilities of everything we are doing is doubling every yearits like the computer revolution; every year youre getting more power while the price of the technology is going down, so youre getting exponential growth in power and value, Galvin commented.

Our approach is a major shift from old-style drug development, which relies on the random generation of thousands or tens of thousands of molecules. And then you try to screen them down via a cell model that gives you the effect youre looking for. And generally after two years and $10 million of investment, you have a few (drug programs) you can test in a mouse, and then one in 19 get into the clinic, stated Galvin.

What were doing at AGT and in the cell and gene therapy field is just different. Gene and cell therapy is about directed development. You can create highly targeted drugs that hit a specific cellular pathway and you can even narrow that down using specific promoters or chimeric envelopes so you can direct that drug to a certain cell type or even to a certain disease indicator, stated Galvin. By narrowing the drug to the particular tissue or particular disease indicator you are sparing all the healthy tissue, which is the main reason drugs drop out of clinical development, the side effects. Gene and cell therapy largely avoids this issue.

We can test some of these in cell models at the bench, then in animal models and be able to get to a go-no-go decision after $100K in investment. We might be able to characterize it so well at the bench that the clinical development becomes highly predictable. This turned out to be true with our HIV therapy, stated Galvin.

Galvin believes that AGTs cell and gene therapy platform will help contributeas part of a wider cell and gene therapy revolutionto the eradication of the $2 to $4 trillion in palliative care treatment costs replaced with one-and-done cures. Viral-vector based drug development platforms like the one AGT deploys will help find new gene and cell therapy treatments and cures for the approximately 7,000 rare diseases that impact approximately one in ten people across the globe.

The future of drug development, in my mind, is that the toolset will keep evolving like computers and software. Software developers used creativity to leverage a limited toolset to create value in the market. At AGT, were thinking about correcting DNA to improve human health and mitigate disease. Everything about technology is playing in our favor. If you were in computers a while ago and you saw mainframes turn into mini-computers and then micro computers you would have said, Eventually we will have these things in our pocket and you would have been right. The same is now true about drug development, stated Galvin, Nearly anything will be possible in the future as this technology continues to exponentially improve. In this high-tech revolution of gene and cell therapy, if you can dream it, you will eventually be able to do it!

AGT is part of an evolving, growing and groundbreaking cell and gene therapy cluster thriving in the BioHealth Capital Region.

Maryland is at the epicenter of the gene and cell therapy technological explosion Its all about the resources. They are starting to hit a critical mass here, added Galvin.

Steve has over 20 years experience in copywriting, developing brand messaging and creating marketing strategies across a wide range of industries, including the biopharmaceutical, senior living, commercial real estate, IT and renewable energy sectors, among others. He is currently the Principal/Owner of StoryCore, a Frederick, Maryland-based content creation and execution consultancy focused on telling the unique stories of Maryland organizations.

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Reprogramming the Human Computer: Silicon Valley Meets Cell and Gene Therapy - BioBuzz

For women in CAR-T, diversity means finding ‘a new normal’ – FierceBiotech

The observations were unsurprising. Panelists convened by the CAR-TCR Summit last week compared statistics of female representation in their companies, finding that the lower ranks were filled pretty evenly by men and women but that this ratio skewed dramatically the higher up they looked.

From those bottom three levels, manager, director and VP, we were at about 40% to 50% women ... But the stark contrast is our C-suite which is only 20% women, said Kimberly Freeman, Ph.D., vice president of commercial strategy and planning at Adaptimmune. Granted, its one out of five, but its still a trend Ive seen across the industry.

Freeman was joined by colleagues from across the chimeric antigen receptor T-cell (CAR-T) and T-cell receptor (TCR) space, ranging from Claudia Zylberberg, Ph.D., CEO of Akron Biotech, a supply chain manufacturer for companies developing cell and gene therapies, and Barbra Sasu, Ph.D., the chief scientific officer at Allogene, to Isabelle Rivire, Ph.D., director of the cell therapy and cell engineering facility at Memorial Sloan Kettering Cancer Center.

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RELATED: 2018's Fiercest Women in Life Sciences

The panelists saw a similar attrition in female representation going up the ranks in their own organizations, even though, as Zylberberg put it, knowledge has no gender.

The truth of the matter is, when in a room where you are speaking the same language, gender disappears. Thats my experience having been in boards that are majority male, she said.

I was struck by what Claudia said: Knowledge has no gender, said Miguel Forte, M.D., Ph.D., CEO of Zelluna. I also think competence has no gender, but currently, opportunities have a bit of a gender and I think thats something we should be aware of.

To address the gender imbalance in the life sciences industry, Nina Bauer, the chief commercial officer at Flodesign Sonics who worked on cell therapies at Lonza, said it has to be done from within, even if its hard to find women who are willing to put up with whats happening, with the politics that are going on at that high level.

Maybe some of us will have to bite the bullet and put up with it and change it from within so that [diversity] does become the new normal. I think one way to do it is indeed with laws, Bauer said.

Industry groups like the Biotechnology Innovation Organization and states such as California are working on this by implementing rules and laws to increase the proportion of women on corporate boards. The consensus on the panel was that these rules should serve as stepping stones to a new normal.

RELATED: MassBio kicks off internship program to boost socioeconomic diversity in life sciences

There are many countriesand I even see it in the U.S. todaywhere you need to have a law for equal opportunity because equal opportunity maybe doesnt exist, Zylberberg said.

The laws are meant to guide us in thinking about diverse candidates and improving diversity until one day, the behavior becomes second nature, she said.

Other ways to get to the new normal involve thinking about diversity on teams the same way one would consider any other business decision.

Anyone who is building a team knows that diversity of any kind will get a better outcome from the team, Forte said. I think [improving gender diversity] is something we should consider, if not for the sake of rebalence, then for the sake of being effective and getting good teams.

Rather than pointing out the negative, we should be saying this is good business sense. It's a way more compelling argument that we can have rather than saying its so unfair, Bauer said.

Of course, unless theyre terrible humans, people dont mean to discriminate.

I try and think of this in terms of conscious and unconscious bias. I dont think anybody out there means to discriminate. There are things in their heads that they dont realize that theyre doing, Sasu said.

We have to give people a break and understand where their unconscious bias is and help to point it out in a kind and helpful way, she added.

And as companies, industry groups, states and countries are working to boost diversity, individuals can do their part both for their own careers and to be a trailblazer, in Bauers words, that helps drive change in the industry.

The universal piece of advice was to find a little village of sponsors and mentors who know who you are and where you want to go, said Andrea Moore, director of analytical development at Tmunity Therapeutics.

Having people you trust that genuinely have your success at heart is very important, said Ariane Hamaide, vice president of corporate development at Cellectis. It doesnt have to be a lot of people, but people you truly trust and can count on, who will be there to help, to advise and to tell you the hard things as well. Its not about having someone who always agrees; its about having someone who will tell you when youre wrong and when youre not doing the right thing.

The panels advice for women who may feel intimidated by gender imbalance at any level of the hierarchy? Fake it till you make it.

Its all about finding a way to find your voice and build that confidence, and maybe express a bit more confidence than you feel until you have that level of confidence because people respect people who respect themselves, Sasu said.

Something I have always told myself is be proud: youre here. Youre just as good as anybody elsemaybe betterand dont be intimidated, Hamaide said.

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For women in CAR-T, diversity means finding 'a new normal' - FierceBiotech

TrakCel partners with McKesson – BioPharma-Reporter.com

The collaboration agreed between the supply chain management services providers, McKesson and TrakCel, comes after the companies have jointly taken on the market entrance of a T-cell based allogeneic therapeutic product of an undisclosed late-stage biopharmaceutical developer.

The commercial launch of this product, which will be the first off-the-shelf product available, is expected in the fourth quarter of 2020, Akshay Peer, VP of sales and account management at TrakCel, told us.

Under the partnership and in order to serve this commercialization, as well as other future product launches, the two companies will develop a combined program, utilizing McKessons patient services alongside TrakCels data management software platform.

The special supply chain requirements of personalized treatments, such as cell and gene therapies, are due to the developers need to manage multiple players and organizations concurrently, according to Peer.

This includes different teams within site of care, shipping and logistics, manufacturing and storage, Peer added.

More specifically, according to TrakCels VP, the product journey consists of the below stations:

Layne Martin, VP of specialty distribution solutions at McKesson Life Sciences, commented that, as the wave of over 900 cell and gene products currently under review by the FDA come to market, supply chain service providers can begin to offer some standards to improve outcomes.

As the products evolve to the latest stages of development,a number of processes including patient scheduling and care have to be mapped, said Peer, adding that the case management teams that are in-charge of communicating with the patient and healthcare professionals need a dashboard view of the entire lifecycle development of the therapy.

According to the companies, the integrated platform is expected to enable the scale-up of products towards market delivery, for the increasing number of developers approaching the commercial launch of cell and gene therapies.

The platform will include automatic scheduling of product-specific workflows across multiple supply chain partners and care team members, and validated chain-of-identity tracking to guarantee correct drug product delivery.These capabilities ensure that the patient receives the correct, uncompromised treatment at the right time, McKesson stated.

Therefore, the integrated suite which will result from the collaboration is expected to provide a control tower view of the product distribution.

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TrakCel partners with McKesson - BioPharma-Reporter.com

Michigan Health Care Regulators Just Restricted Access to Promising New Cancer Treatments – Reason

A state commission, acting at the behest of Michigan's largest hospital chain, voted on Thursday to restrict cancer patients' access to promising, potentially lifesaving treatments.

It's another example of the problems caused by little-known state-level health care regulations known as Certificate of Necessity (or, in some states, Certificate of Public Need) laws. These laws are supposed to slow down increasing costs, but they often end up being used to restrict competition, often at the request of powerful hospital chains.

That's exactly what seems to have happened in Michigan, where the state's Certificate of Need Commission voted Thursday to impose new accreditation requirements for health care providers who want to offer new immunotherapy cancer treatments. Those treatments attempt to program the body's own immune system to attack and kill cancer cells, and they have become an increasingly attractive way to combat cancer alongside more traditional methods, such as surgery, chemotherapy, and radiation.

One particularly promising type of immunotherapy involves literally bio-engineering T-cellsthe foot-soldiers of the body's immune systemand equipping them with new Chimeric Antigen Receptors that target cancer cells. This so-called "CAR T-cell therapy" is every bit as badass as it sounds:

But under the new rules adopted by the Michigan Certificate of Need Commission, hospitals will need to go through unnecessary third-party accreditation processes before being able to offer CAR T-cell therapies. Even after obtaining that additional accreditation, hospitals would have to come back to the CON commission for another approvala process that effectively means only large, wealthy, hospital-based cancer centers will be able to offer the treatments.

The new rules were "opposed by cancer research organizations, patient advocates and pharmaceutical companies, who argue it would add an unnecessary level of regulation and deny many patients access to potentially life-saving treatment," reportsMichigan Capital Confidential, a nonprofit journalism outfit covering Michigan politics.

In favor of the new rules? The University of Michigan Health System, the state's largest hospital system, which argues that the new rules are necessary for patient safety.

To be clear: It's not a question of patient safety. In 2017, the Food and Drug Administration (FDA) approved two CAR T-cell therapies for children suffering from leukemia and for adults with advanced lymphoma. Although the technology is still being developed and other uses of T-cell therapies are yet to be approved by the FDA, the Michigan CON Commission does not do medical testing. Like similar agencies in other states, the extent of its mandate is purely economic, not medical.

Anna Parsons, a policy coordinator with the American Legislative Exchange Council, points out that the safe administration of CAR T-cell therapy does not require hospitals to make new capital investmentswhich is the only time CON laws should apply. Literally any FDA-certified hospital should be capable of offering these treatments, since all the high-tech bioengineering is done at other locations. The only thing that happens at the hospital is a simple blood transfusion.

Though the specific applications of CON laws differ from state to state, their stated purpose is to prevent overinvestment and keep hospitals from having to charge higher prices to make up for unnecessary outlays of capital costs. But in practice, they mean hospitals must get a state agency's permission before offering new services or installing new medical technology. Depending on the state, everything from the number of hospital beds to the installation of a new MRI machine could be subject to CON review.

As part of that review process, it's not uncommon for large hospital chains to wield CON laws in order to limit competition, even at the expense of patient outcomes.

From 2010 to 2013, for example, the state agency in charge of Virginia's CON laws repeatedly blocked attempts by a small hospital in Salem, Virginia, to build a neonatal intensive care unit (NICU), in large part because a nearby hospitalwhich happened to have the only NICU in southwestern Virginiaobjected to the new competition. Even after a premature infant died at the Salem hospital, state regulators continued to side with the Salem hospital's chief competitor, against the wishes of doctors, hospital administrators, public officials, and patients who repeatedly testified in favor of letting the new NICU be built.

Even when the outcomes aren't as tragic as dead babies or untreated cancer patients, CON laws have adverse consequences. In 2016, reseachers at the Mercatus Center at George Mason University found that hospitals in states with CON laws have higher mortality rates than hospitals in non-CON states. The average 30-day mortality rate for patients with pneumonia, heart failure, and heart attacks in states with CON laws is between 2.5 percent and 5 percent higher even after demographic factors are taken out of the equation.

When it comes to CAR T-cell therapy, there does not seem to be any compelling reason for Michigan regulators to use CON laws except to explicitly limit which hospitals can provide those treatments.

"We will never know how many more lives this therapy could have saved if the added time and expense these onerous regulations put in place discourage hospitals and clinics from providing treatment in the first place," Parsons wrote this week in The Detroit News.

Under Michigan law, the legislature has 45 days to review and overturn the decisions of the CON Commission. Here is one situation where that is exactly what it should do.

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Michigan Health Care Regulators Just Restricted Access to Promising New Cancer Treatments - Reason