Category Archives: Cell Therapy

Scientists from Sweden say they have made significant progress in the search for a new treatment for Parkinsons disease.

Though the research, published in Nature Biotechnology, is still preliminary and the therapy not yet ready to be tested in humans, experts say it could one day help the millions of people living with the neurodegenerative disease.

Researchers from the Karolinska Institute tested whether certain brain cells could be manipulated to take on the role of those destroyed by Parkinsons.

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They first showed in laboratory experiments that it was possible to convert non-neural human brain cells called astrocytes into dopamine neurons, which degenerate and die in the brains of people suffering from Parkinsons disease.

These are two specialized cells that do not spontaneously convert into one another, study author Ernest Arenas, a professor at Karolinska Institutes Department of medical biochemistry and biophysics, told CBS News. However, when we used diverse chemicals and genes important for the development of immature brain cells into functional dopamine neurons, we found that it was possible to convert astrocytes into dopamine neurons.

The researchers then tested whether this could be done in mice with Parkinsons and if the therapy would improve their condition.

After two weeks, they reported that astrocytes in the brains of the mice started to become dopamine neurons. At five weeks, the mice recovered some of their motor functions such as posture, motility and walking pattern.

Current treatments for Parkinsons only address symptoms, not the cause of the disease itself.

While much more research is needed before the treatment can be tested in humans, Arenas says it could one day lead to an approach to change the course of disease and halt or even reverse motor deficits in Parkinsons disease patients.

Aside from being in early stages, the research is limited in several ways, the study authors say.

First, Arenas notes that although dopamine neurons are the main cell type affected in Parkinsons disease -- and those responsible for the characteristic motor symptoms -- other cell types are affected, particularly as the disease progresses. Therefore, additional strategies to treat these other cell types will be needed in the future.

Additionally, this type of therapy would involve surgery, and therefore could be riskier compared to other treatments on the market. However, with people living longer in most societies, more severe forms of disease are currently being seen, Arenas said, and people are suffering longer.

We thus think that cell replacement therapies, because of its potential to change the course of disease, may become the method of choice in the future, he said.

The authors say now that they know the treatment technique is possible, future research will concentrate on making it safer and developing it into a method that could be applied in a clinical setting.

Our goal and hope is that all these studies will lead to the development of a safe and efficient cell replacement therapy for Parkinsons disease in which no cell transplantation or immunosuppression is necessary, Arenas said.

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Brain cell therapy offers hope for Parkinson's patients - CBS News

Mumbai: Regenerative Medical Services Pvt. Ltd on Wednesday said that the Drug Controller General of India (DCGI) recently approved its stem cell-based therapy to treat cartilage defects.

Chondron, its cartilage repair procedure, uses the bodys own cartilage cells that are cultured, multiplied and implanted into the patients damaged joint leading to new cartilage regeneration and avoiding the need for early joint replacement. This is the first stem cell-therapy product to be approved in India.

We are creating new age cartilage regeneration procedure which optimizes the chances of healing due to the use of the bodys own cells. We will tie up with hospitals to promote it; we have received interest from around 200 hospitals, chief executive officer and managing director Yash Sanghavi said.

The company has capacity to culture 1,000-1,200 patient samples annually, which it plans to enhance to 10,000-12,000 samples in next three years, chief scientific officer Satyen Sanghavi, said.

The Mumbai-based company, started in 2009, has invested around Rs70-75 crore on development of Chondron and conducted clinical trials on 350 patients. It is looking to raise funds for capacity expansion and marketing of the cell therapy.

We are looking to raise Rs40-50 crore through equity dilution and have already appointed bankers, Sanghavi said. The treatment, he said, is likely to cost around Rs2 lakh, which is almost the same or slightly lower than the price of a knee replacement surgery.

First Published: Wed, Apr 12 2017. 09 11 PM IST

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DCGI approves Regenerative Medical's stem cell therapy for cartilage defects - Livemint

For the past five decades pharmaceutical drugs like levodopa have been the gold standard for treating Parkinsons disease. These medications alleviate motor symptoms of the disease, but none of them can cure it.

Now a study from the Karolinska Institute in Stockholm shows it is possible to coax the brains own astrocytescells that typically support and nurture neuronsinto producing a new generation of dopamine neurons.

The reprogrammed cellscould alter the course of Parkinsons, according to the researchers. You can directly reprogram a cell that is already inside the brain and change the function in such a way that you can improve neurological symptoms, says senior author Ernest Arenas, a professor of medical biochemistry at Karolinska.

Directly converting astrocytes already present in patients brains could eliminate the need to search for donor cells[and the treatment may] be less likely to cause side effects compared with current drugs. This is like stem cell 2.0. Its the next-generation approach to stem cell treatments and regenerative medicine, says James Beck, vice president and chief scientific officer, for the nonprofit Parkinsons Disease Foundation.

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The GLP aggregated and excerpted this blog/article to reflect the diversity of news, opinion, and analysis. Read full, original post:Cell Therapy 2.0: Reprogramming the Brains Own Cells for Parkinsons Treatment

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Parkinson's stem cell therapy 2.0: New treatment coaxes the brain to repair itself - Genetic Literacy Project

Cell therapy is revolutionizing medicine, heres how the latest technology can help overcome the major challenges stopping it from taking over the market.

Offering unprecedented possibilities to treat some of the most challenging diseases, cell therapy is stealing the show in the biotech space. Strimvelis, the first hematopoietic stem cell (HSC) gene therapy is already treating rare genetic diseases. In less than a year, CAR-T therapy is expected to hit the market and revolutionize the treatment of cancer. And in the not-so-far future, cell therapy could even eradicate HIV or put an end to diabetes, which is reaching epidemic proportions in Western countries.

The first cell therapies have already been on the market for a few years, and analysts are confident that the numbers will quickly grow over the years. The stem cell therapy market alone is expected to hit 57Bn ($61Bn) by 2022, and the upcoming CAR-T technology will reach an impressive 8Bn ($8.5Bn) in the next decade.

Although the potential is definitely there, researchers are still looking for ways of making these therapies cheaper, safer and more effective. Currently, not all patients are suited to receive cell therapy due to scientific or economic challenges. The advent of allogeneic therapies is addressing the financial obstacles, but what about efficiency?

One of the key elements to building a successful cell therapy are viral vectors, which are used to deliver the necessary DNA sequences to engineer the cells. Lentiviral vectors are a common choice because they have a rather larger capacity and enable long-lasting genetic expression.

They are specially researched for ex-vivo treatment of hematopoietic stem cells and primary T-cells. The percentage of cells that can be reached, however, often remains low, and the number of gene copy numbers per cell can be extremely variable.

Finding a solution to this challenge is unfortunately not as simple as increasing the virus load. Cells that carry a surplus of copies integrated into their DNA are more prone to mutations that affect their survival and put the safety of the patient in danger. This is an essential factor taken into account during the regulatory phase to determine whether a therapy can make it to the market or not.

How can we then improve the efficacy of transduction without affecting the health of the cells and the safety of the therapy? In the lab, researchers often use enhancers such as polybrene, a polymer that can increase the efficacy of transduction of viral DNA. However, this substance is not applicable in a clinical context because of its heightened cell toxicity.

To overcome these challenges, scientists at SIRION Biotech got down to work and screened for compounds that could improve the fusion of the viral and cell membranes. The result was a technology with the potential to solve a major problem in the development of cell therapies, with DNA delivery reaching an impressive 80% of hematopoietic stem cells while keeping the copy numbers down to the ideal value of 3 to 5 per cell.

This technology, called LentiBOOST, works its magic simply by adding a non-toxic polymer to the mixture during transduction. It has already been accepted as an element of clinical trials from phase I to phase III and its possibilities seem unlimited. One of SIRIONs partners, the Heinrich Pette Institute in Hamburg, is studying the potential of LentiBOOST to improve transduction in a therapy intended to actively remove HIV from infected blood cells and induce long-lasting resistance against the virus.

With applications ranging from cancer to infectious disease, cell therapy is definitely going to change medicine. Technology like LentiBOOST is helping these amazing developments materialize with a boost to both their efficacy and safety.

You can find more information on LentiBOOST at SIRION Biotechs website!

Images from Montri Thipsorn, vchal /Shutterstock; SIRION Biotech

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Taking Cell Therapy one Step Further with this Boost Reagent - Labiotech.eu (blog)

Frequency Therapeutics raised $32 million in a Series A financing to support development of its Progenitor Cell Activation (PCA) platform. The technology uses small molecules to trigger the division and differentiation of tissue-specific Lgr5+ progenitor cells and is initially being developed to generate new sensory cells in the inner ear as a treatment for noise-induced hearing loss. The Massachusetts-based firm says other potential applications of the platform could span skin disorders, muscle regeneration, and gastrointestinal diseases.

Frequencys Series A fundraising round was led by Cobro Ventures. Other investors included Morningside Ventures, Emigrant Capital, Korean Investment Partnership, Alexandria Real Estate Equities, and additional U.S. and international investors.

The PCA platform was developed by Robert Langer, Sc.D., and Jeffrey Karp, Ph.D., at the Massachusetts Institute of Technology (MIT), and Harvard Medical School. Frequency was established in 2015 to develop the platform for therapeutic indications and has an exclusive global license to relevant IP from MIT and Partners Healthcare. Bob Langer's and Jeff Karps vision is to gain much of the same effect as gene therapy and CRISPR by using small molecules, which we believe are safer and allow for easier delivery, stated Marc Cohen, co-founder of Cobro Ventures and chairman of Frequencys board of directors, which was established earlier this year. Our data is very compelling and we are excited to be moving to the clinic in the next 12 to 18 months.

Frequencys co-founders published in vitro research demonstrating use of their small-molecule approach to trigger the differentiation of Lgr5-expressing progenitor cells into sensory hair cells, in the February 21 issue of Cell Reports in a paper titled "Clonal Expansion of Lgr5-Positive Cells from Mammalian Cochlea and High-Purity Generation of Sensory Hair Cells."

Commenting on the initial, hearing loss indicaiton for the PCA platform, David Lucchino, Frequencys co-founder, president and CEO, added, With no effective therapy available, this presents an enormous market opportunity and we believe there is even broader potential in indications beyond hearing loss with the further development of the PCA platform.

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Frequency Raises $32M in Series A to Develop Progenitor Cell Therapy for Hearing Loss - Genetic Engineering & Biotechnology News

GE Healthcare said today it has acquired Asymptote for an undisclosed price in a deal that the buyer said would strengthen its cell therapy portfolio with technologies designed to enable the cryopreservation of cellular materials.

Asymptote specializes in cryochain technology for sensitive cellular therapies, with the goal of significantly lowering the risk of contamination found in conventional processes.

The companys integrated suite of cryochain hardware, software, and consumables is designed to support cGMP and maintain the potency of cellular therapies by enabling ultra-low-temperature freezing during production, followed by thawing prior to administering to patients in clinical settings.

Asymptotes VIA FreezeTM range delivers liquid nitrogen-free cryopreservation, while the soon-to-be released VIA ThawTM series thaws deeply frozen cells using a dry conduction (water-free) process. The companys web-based my.Cryochain software platform is designed to support cell therapy companies as they scale their cryopreservation and thawing processes by synchronizing with the VIA Freeze and VIA Thaw products to standardize freezing and thawing programs across multiple sites, improving the visibility of remote processes.

Asymptotes high-quality offering takes us another step forward in our vision to industrialize cell therapy, and in providing reliable and high-quality services for our customers and patients around the world, Ger Brophy, Ph.D., general manager of GE Healthcares cell therapy business, said in a statement.

Added Asymptote founder and CEO John Morris, Ph.D.: The acquisition gives us the opportunity to reach a larger audience through the local specialist GE Cell Therapy team, provides us with a strong platform for our product launches, and allows us to significantly scale up our product development.

GE Healthcare reasons that demand for manufacturing and clinical delivery will increase as the cell therapy market grows and develops. The company cited statistics from the Alliance for Regenerative Medicine showing 804 clinical trials underway by the end of last year for cell therapies, as well as gene-modified cell therapies, gene therapies, and tissue-engineering therapies.

The acquisition of Asymptote is GE Healthcares latest move toward growing its cell therapy offerings. In July 2016, GE acquired Biosafe Group, a supplier of integrated cell bioprocessing systems, for an undisclosed sum. Three months earlier, GE Ventures teamed up with Mayo Clinic Ventures to launch Vitruvian Networks,a collaboration providing cloud-based software systems and manufacturing services for cell and gene therapies.

And in January 2016, GE Healthcare and the Government of Canada each committed C$20 million ($15 million) to launch the [emailprotected] Cell Therapy Centre of Excellence, created to promote new technologies for the production of cellular therapies in Toronto.

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GE Healthcare Adds to Its Cell Therapy Portfolio by Acquiring Asymptote - Genetic Engineering & Biotechnology News

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Brain cell therapy 'promising' for Parkinson's disease BBC News Scientists believe they have found a way to treat and perhaps reverse Parkinson's disease, by making replacement cells to mend the damaged brain. They say human brain cells can be coaxed to take over the job of the ones that are destroyed in Parkinson's. Cell Therapy 2.0: Reprogramming the Brain's Own Cells for Parkinson's TreatmentScientific American Brain Cell Therapy Reverses Parkinson's-Like Symptoms In MiceHuffington Post UK Brain cells reprogrammed to make dopamine, with goal of Parkinson's therapyThe San Diego Union-Tribune STAT -NHS Choices all 67 news articles »

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Brain cell therapy 'promising' for Parkinson's disease - BBC News

At the halfway point, a phase 3 trial of Mesoblast's off-the-shelf cell therapy for chronic heart failure is on track and should continue to completion, say advisers.

Shares in the Australian biotech hit a 12-month high on the update from the trial, which came after it enrolled 270 out of a planned total of 600 patients with moderate CHF andcruciallyrevealed no safety issues with Mesoblast's MPC-150-IM candidate.

The trial is still blinded so there is now way to tell if the therapy is having an effect, but it's a case of so far, so good, as the biotech looks ahead to completing the study next year.

The trial is seeing whether delivery of mesenchymal precursor cells (MPCs) via a catheter into the left ventricular heart muscle, to see if it can reduce non-fatal heart failure-related major adverse cardiac events (HF-MACE), such as death, needing a pacemaker implanted or undergoing a heart transplant.

Last year, Israeli drugmaker Teva returned rights to MPC-150-IM to Mesoblast, which it had inherited through its takeover of Cephalon. Mesoblast however decided to go it alone, a decision helped by a recent $40 million placement intended to help bring the CHF trial to fruition.

Analyst John Savin at Edison said in a recent note that the biotech may not have to wait for its own trial to complete before filing for U.S. approval. That could happen before the end of the year if a National Institutes of Health-sponsored trial of the therapy in end-stage heart failure patients hits the target.

The 159-patient NIH study is expected to complete enrolment in the first half andwith luckcould report top-line data before year-end, according to Savin. He reckons that could "lead to an application for accelerated approval" under the U.S. 21st Century Cures Act, which provides a speedy route to market for regenerative medicines.

"Passing this interim futility analysis for MPC-150-IM is an important milestone for Mesoblast and our cardiovascular disease program," commented CEO Silviu Itescu. "This validates our strategy and our prioritization of this valuable program." Analysts at Credit Suisse have previously suggested that that drug could be worth $4.1 billion in peak annual sales.

Mesoblast is not the only biotech looking at a stem cell approach to cardiovascular diseases. BioCardia is developing CardiAMP for CHF, reporting positive data from small phase 2 trial last year, while CardioCell presented mixed results on its candidate at the 2016 ESC Congress in Rome, and Celyad's C-Cure product failed to meet its objectives in its phase 3 CHART trial. Meanwhile, Belgium's TiGenix said recently its acute myocardial infarction therapy AlloCSC-01 hit its targets in a phase 1/2 trial.

The Australian biotech claimed an FDA green light to start trials of an MPC therapy given alongside corrective heart surgery for children with hypoplastic left heart syndrome (HLHS).

It's also in the build-up to a decision by option partner Mallinckrodt on its phase 3 MPC for chronic low back paindue in or before Septemberand a graft-versus-host disease (GVHD) candidate that should see pivotal results before year-end.

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Mesoblast takes off as cell therapy for heart failure passes interim test - FierceBiotech

Making CAR T-Cell Therapy Safer The Scientist JCAR015 is not the first CAR T-cell therapy to have been associated with patient deaths. In fact, even those trials considered a success sometimes have troubling safety profiles. For example, Novartis's lead candidate, the CD19-targeting CLT019, ...

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Making CAR T-Cell Therapy Safer - The Scientist

Fred Hutchinson Cancer Research Center immunotherapy researchers Drs. Kristin Anderson and Philip Greenberg and their colleagues are working on ways to tweak their team's early successes with T-cell therapy for leukemia to apply to solid tumors.

In a presentation on April 4 at the annual meeting of the American Association of Cancer Research in Washington, D.C., Anderson will describe preclinical research on T-cell therapy for ovarian tumors and the particular tumor microenvironment factors that any clinical version of this therapy will need to take into account.

For some patients, certain forms of immunotherapy are showing promise in treating previously difficult-to-treat cancers. In the case of T-cell therapies, though, most of the early experimental successes have been seen in blood cancers. Solid tumors, like breast, lung, ovarian and pancreatic cancers, pose a tougher nut to crack for this new wave of cancer therapies.

There are a number of additional hurdles T-cell therapy has to overcome to reach these cancers, which kill more people in the U.S. than blood cancers, according to the American Cancer Society. There's the simple issue of access -- patients with leukemia or lymphoma can receive an infusion of engineered T cells directly into their bloodstream, but it can be more difficult to tweak the cells to traffic to a tumor tucked away in the body. A major roadblock to adopting T-cell therapy to solid tumors is what's known as the tumor microenvironment, the local milieu of non-cancerous cells and molecules in and around the tumor.

Anderson and her colleagues have identified proteins overproduced by ovarian cancer cells, known as WT1 and mesothelin, and have found that T cells engineered to specifically recognize these proteins can kill both human and mouse ovarian cancer cells in the lab. They've also found that the T cells significantly extend survival in a mouse model of the cancer, but there's a ways to go before this therapy is ready for clinical trials in humans, Anderson said.

"Tumor microenvironment issues come hand-in-hand with working on solid tumors," she said.

In her presentation, Anderson will describe three types of roadblocks to an effective ovarian cancer T-cell therapy -- and how the research team is working to overcome each. They are:

Immunosuppressive cells and proteins in the microenvironment that can signal the engineered T cells to shut down or ignore tumors. Existing checkpoint inhibitor drugs could circumvent this problem, Anderson said, and the Fred Hutch team is also exploring engineering the therapeutic T cells to block those immunosuppressive signals. A "death signal" produced by both ovarian tumor cells and nearby blood vessels on their surfaces. This molecular signal causes T cells coming to the tumor from the bloodstream to commit suicide before they can fight the cancer. Dr. Shannon Oda in the Greenberg lab is working on a new type of fusion protein the engineered T cells will carry that will rewire their internal circuitry, causing the death signal to instead boost their anti-tumor activity. The tumors' low-sugar environment. Fast-growing ovarian cancer cells churn through the glucose in their environment -- the same energy source engineered T cells need to do their work. Researchers in the Greenberg lab are working to re-engineer the therapeutic T cells to process other sources of energy.

Although her current work focuses on ovarian cancer, a particularly difficult-to-treat solid tumor, Anderson hopes the work will shed light on new therapeutic avenues for other solid tumors as well.

"If we can solve some of the issues that really plague us with these hard ones, then we can more readily apply them to some of the cancers that have fewer of these hurdles," she said.

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Materials provided by Fred Hutchinson Cancer Research Center. Original written by Rachel Tompa. Note: Content may be edited for style and length.

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Developing adoptive T-cell therapy for ovarian cancer -- ScienceDaily - Science Daily