Generating 70% remission for previously untreatable cancers. Markwin Velders of Kite Pharma discusses

‘It’s amazing to think about the opportunity we create at Kite, at this moment, with other colleagues in the T-CAR field where we see patients who have no options – refractory tumour patients where a doctor tells them we cannot do anything else for you. We can come in with a new therapy.’ – Markwin Velders, Vice President of Operations of Kite Pharma discusses the use of CAR-T therapy as a revolutionary treatment for populations of patients who were previously untreatable.

The word ‘breakthrough’ when used to describe cancer treatment often denotes a positive development, and Markwin Velders of Kite Pharma is confident in using it to describe the use of CAR-T therapy in cancer patients. He speaks of ‘patient populations’, such as those with non-Hodgkinson Lymphoma, which can ‘now be treated effectively and can even reach 60-70% remission’, a group ‘who never had that option before.’ He describes it as ‘an unmet medical need where we make a difference’, which offers a hopeful insight into the progress Kite Pharma are currently making in the field.

Velders highlights the current status that the use of T-Cell Immunotherapies as a successful treatment of haematological, or liquid tumours is currently at. He draws upon the successful results Kite Pharma have seen treating this demographic of illness. However, it is in the much ‘wider field’ of solid tumours that he feels they should be looking to make progress, and improve on current therapies. Solid tumours make up the largest number of cancer sufferers, and Kite Pharma hope to ‘embark on the next phases’ for treating these ‘which is where there are real chances for the therapy as well as other opportunities.’

In terms of the public opinion towards cell therapy, Velders describes it as a ‘double-edged sword’ as often people met with experiences of being charged ‘large amounts of money’ for ‘therapies which aren’t very effective.’ He understands that this can often give the industry an unfavourable image, but notes ‘it is important and…I think generally people look at it as a pretty advanced field. It has a lot of opportunity, but it comes at a cost.’ If this is understood, and people continue to look towards ‘these new therapeutic routes and medicines’, it will expand the possibility of using cell therapies as viable treatment options for cancer patients.

Velders also offers advice to biotech start-ups seeking to take T-Cell Immunotherapies to market, and stresses the need for finding ‘something that can make the difference, and try to get a reliable investor’. He suggests, as he did for altering public opinion, to look forward ‘to the next level of therapeutic approaches’, but underlines the importance of having a reliable platform of scientific expertise.

It with this he draws upon his own experiences at Kite with the ZUMA-1 Phase 2 trial. Kite have been met with a response rate of up to 70% in patients with Diffuse Large B-cell Lymphoma. He says of their findings:

‘About 50% of the patients have a complete response, that’s a durable response in 40% of the patients after 3 months. We’re hoping to get data out in Q1 2017 to really confirm these responses and to see that they are long lasting responses in this population.’

The positive results they have seen so far provide a hopeful platform on which Kite Pharma can build an effective cell therapy model.

Watch the full interview with Markwin Velders, filmed at Cell Therapy Manufacturing & Gene Therapy Congress,  above or here.

Proving the efficacy of cell therapy treatments to convince investors

‘What convinced investors and what convinced our alliance partners was the clinical result we had in chronic lymphatic leukaemia, I think that’s important. I think with animal models, they are very helpful in a sense, in just the bare bones test system, but to prove efficacy, in the end is to run clinical trials.’ Jan Joseph Melenhorst, Director of Product Development & Correlative Sciences Laboratory from the University of Pennsylvania, discusses the necessary steps that must be taken for their cell therapy to become a viable treatment option.

Within his department at the university, Melenhorst examines the post infusion specimens of cells to better understand the potency and toxicity of the cells they infuse into patients. To enhance the potency of cells, additional gene editing tools can be included to modify the genome of the T-Cells and further enhance the expression of the targeting gene. Melenhorst speaks of his own experience with the CRISPR-Cas9 gene editing tool, but also draws upon the use of TALENs, stressing its importance to ‘the future of autologous and allogeneic cell therapies’. It is the autologous therapies Melenhorst believes are the current future of CAR-T therapy. Continue reading “Proving the efficacy of cell therapy treatments to convince investors”

Get Your Complimentary e-Pass to the Cell & Gene Therapy Bioprocessing & Commercialization Digital Week!

Join us for the four days of learning at the Cell & Gene Therapy Bioprocessing & Commercialization Digital Week, a complimentary webinar series focused on the innovative science and technologies needed to overcome bottlenecks and pain points in the manufacture and commercialization of cell, gene and immunotherapies.

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What’s on the agenda?

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How GE Healthcare are implementing new cell therapy technology to develop a fully automated and integrated system

‘The Xuri Cell Expansion System W25…is heavily adopted within the T-Cell Expansion System which enables our customers to grow up significant doses of T-Cells in a functionally closed environment. We are working with our customers to further connect the Upstream and Downstream.’ George White, Commercial Leader of Cell Therapy from GE Healthcare, outlines the positive acquisitions they have recently made that will assist their customers in implementing successful cell therapy systems.

GE are currently looking to the future of T-Cell therapy technologies to develop harmonised partnerships that will facilitate and allow their customers to implement fully automated and integrated systems. White highlights the ‘amount of effort and resources [GE] put into developing protocol and applications, specifically for T-Cell manufacture,’ that lead to the close relationships they have with their customers who look for a ‘complete solution.’ Continue reading “How GE Healthcare are implementing new cell therapy technology to develop a fully automated and integrated system”

FDA First Generic Drug Approvals List 2017

Keeping up with all of the new first generic drug approvals can be a tricky business. On this page, we are dedicated to keeping the list updated and you informed.

(Updated 09/02/17)

27 January 2017 – Amneal Pharmaceuticals LLC, Namzaric (memantine and donepezil hydrochlorides)

 A first generic drug approval has been granted to Amneal Pharmaceuticals LLC to produce Namzaric for the treatment of moderate to severe dementia of the Alzheimer’s type.

Click here to find out more 

23 January 2017 – Actavis Laboratories FL, Inc. & Aurobindo Pharma Limited, Ampyra (Dalfampridine)

 For those suffering with multiple sclerosis (MS) the first generic drug approval for Ampyra has granted to help improve walking for the patients of the disease.

Click here to find out more

17 January 2017 – Roxane Laboratories Inc., Xyrem (Sodium Oxybate Oral Solution)

The first generic drug approval for Xyrem come to the aid of those suffering of cataplexy in narcolepsy; excessive daytime sleepiness (EDS) in narcolepsy.

Click here to find out more

4 January 2017 -Aurobindo Pharma Limited,  Ampyra (Levitracetam in Sodium Chloride Injection)

This new FDA first generic drug approval granted to Aurobindo Pharma Limited for an anti-epileptic drug indicated for the adjunct therapy in adults >16 and 16 years of age with certain seizure types when oral administration is temporarily not feasible.

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BioPhorum Operations Group’s (BPOG) Best Practices Guideline for Mitigating Risk from Leachables

The BioPhorum Operations Group’s (BPOG) Best Practices Guideline for Mitigating Risk from Leachables in Polymeric Single-Use Components Used in Biomanufacturing will be presented at BioProduction, Dublin in October 2016.  This leachables guidance builds upon the success of the BPOG Extractables Protocol to form a completed picture of the impact of using Single Use Systems (SUSs) for the biopharmaceutical industry.

Continue reading “BioPhorum Operations Group’s (BPOG) Best Practices Guideline for Mitigating Risk from Leachables”

Leachables Testing Methods & Best Practices

Author: Nick Hutchinson

BPOG’s Best Practice Guide

The biopharmaceutical industry is increasingly using Single-use systems (SUS) for the manufacturing of medicines. Furthermore, companies are applying the technology in applications that are more critical to patient safety. Methods for assessing risks associated with using SUS in bioprocessing need to be developed.

Miriam Monge, Marketing Director for Integrated Solutions at Sartorius Stedim Biotech moderated a session at the BioProcess International Conference & Exhibition, 2016 in which representatives from the BioPhorum Operations Group (BPOG) discussed best practices for leachables testing when implementing SUS. During her introduction, Monge highlighted the need for the various stakeholder in the industry to work together to find consensus on testing methods.

Continue reading “Leachables Testing Methods & Best Practices”