At the Forefront of Cell Therapy in Boston: Oren Levy of Karp Lab

Oren Levy from Karp Lab gave an excellent talk called “MSCs on Steriods” last month at Biotech Week Boston. Oren's research focus is "investigating the roles of signal transduction pathways in hMSC physiological processes, specifically, the involvement of the JAK/STAT cascade in hMSC proliferation and osteogenic differentiation". His research also focuses on "hMSC homing and engraftment to various sites in the body". Karp Lab is located in the Cambridge/Boston Biotech Hub and works closely with Brigham and Womens' Hospital, MIT, Harvard Medical School and Harvard-MIT Health Sciences and Technology.

Levy started his discussion with this unfortunate fact - so common with so many potential biopharma cures being researched right now – that “MSCs clinical endpoints have not been met and there’s not a single approved FDA product”. He then also shared the impressive stat that “MSCs are used in 600 clinical trials worldwide”; with so much research happening right now, the industry is hopeful that some breakthroughs are on the horizon.

What are MSCs and Why Are They on Steroids? 

The NIH gives a good definition of MSCs on their website: “Mesenchymal stem cells (MSCs) are adult stem cells which can be isolated from human and animal sources”. MSCs are being studied for bone, cartilage, heart and blood vessel repair, as well as inflammatory and autoimmune diseases. Why are MSCs on steroids? If you check out their website, Karp Lab has a great sense of humor (and it’s an understatement to say the Lab’s work is “creative”) which makes sense that the title of the talk was playful. But in all seriousness, MSCs have the potential to be very powerful; researchers such as Oren Levy and Karp Lab have a real sense of urgency about getting MSC therapies to work. So much so that they embarked on a very ambitious project: with the help of Sanofi, Levy screened over 10,000 small molecules to improve cell targeting.

Our Goal is to Improve Control Over Cell Fate 

Oren Levy mentioned two disease areas where his team was studying the use of MSCs: prostate cancer and multiple sclerosis. In fact, Levy mentioned that their multiple sclerosis study was about to be submitted for publication, so we have that to look forward to soon. For prostate cancer, the screening of the small molecules Levy’s team did with Sanofi comes into play. Levy discussed how they use “drug loaded MSCs to kill prostate cancer cells”. How they’re trying to make this happen is by “small molecule pretreatment to give MSCs the homing mechanisms they lack”. Essentially, they’re “using engineering strategies to improve MSCs targeting to tumor sites”. The combination of engineering and life sciences – bioengineering – is a major trademark of what Karp Lab does. Their website relates one of their mission statements: “Our lab firmly believes that innovation occurs at the interface of disciplines”. You can read an in-depth interview of Jeff Karp of Karp Lab produced by Biotech Week Boston and written by journalist Nick Paul Taylor here.

Don't forget to follow Biotech Week Boston on Twitter for news on innovation in biotech and medicine. Each year passionate scientists and innovators converge on Boston to share ground breaking data, research and ideas - don't miss our next event in September 2017!

Learn more about #BiotechWeekBoston

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Today's Challenges in Cell Therapy

 

Joining us in this Cell Therapy Bioprocessing & Commercialization Podcast is Marty Giedlin, VP of Development at Sangamo BioSciences, Inc. Marty discusses key industry challenges, the evolution of the cell therapy field and where it is headed in the next five years, advancements in technology and much more. Below is a brief excerpt from the podcast, follow the links below to access the complete podcast and transcript.

Listen to the podcast | Access the complete transcript

What are the challenges that you’re experiencing in your industry?

Well, I guess it really depends on what kind of cell therapy you’re talking about. We at Sangamo are processing both autologous T-cells and autologous stem cells. We have the most experience in our T-cell program where we knock out the CCR-5 receptor for HIV in HIV-positive patients. So, the challenges are trying to find that patient population that’s most amenable to this type of therapy, both in viral load, how many years they’ve been in retroviral therapy and that sort of thing. And also finding out which of the CD4 cells – which sub-population – is most important in controlling HIV. So, those are some of the challenges that are facing us there.

With our stem cell program, we have a partnership with CIRM (California Institute for Regenerative Medicine) in California with the City of Hope where we are looking at using CD34 stem cells either from peripheral blood or from bone marrow. What sub-population of those cells are really those that have the capacity for self-renewal over time? Those are the cells that we really want to target to do our gene modification technology.

Then, there are culture conditions to minimize replication, retain stemness and maximizing engraftment with respect to CD34s. And then we’re looking at ways of mobilizing peripheral blood CD34s and how does that affect stemness with respect to CD34s? And then also looking at different ways of bone marrow harvest. So, are there better ways of getting maximal yield to make sure we get enough cells to give back to the patient?

So, for us in the adoptive cell therapy, those are some of the challenges that we’re looking at over the next three to five years.

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The Greatest Challenges in Cell Therapy Today

Joining us in this Cell Therapy Bioprocessing & Commercialization Podcast is Jeff Karp - Associate Professor of Medicine at Brigham and Women's Hospital, Harvard Medical School. Jeff talks about the challenges facing the cell therapy space, how to improve cell homing following transplantation, and control cells among other things.

Listen to the Podcast | Access the complete transcript

What are some of the greatest challenges in cell therapy today? 

I think that we’re getting to a point in time where we can obtain almost any cell type in unlimited quantities with a few exceptions. This is using reprogramming, programming, different differentiation, protocols that have recently been worked out. So, I really think we are getting to that point where we can obtain nearly any cell type that can then be delivered to patients for treatment. 

While I think we’re there, I think one of the greatest challenges that remains is that once we transplant cells, we lose control over the cells. So, when we’re working with cells in a Petri dish, for example, we can pattern the media, we can put cells on all different types of textured substrates, we can control the media and the environment exquisitely. But, when we transplant cells, they are entirely at the mercy of the biological. So, if they end up in different tissues in the body, they are going to behave completely differently. So, we lose control of those cells following transplantation.

So, I think one of the greatest challenges is how can we now take the cells that we worked so hard to derive in vitro and then transplant those cells into patients and exhibit control so that the cells get to the right location and can perform their function when they get there.

[The above was an excerpt from the podcast]

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The Cell Therapy Commercialization Summit is next week!

The Cell Therapy Commercialization Summit is only one week away!

Are you confident that your current clinical development plan will take your product from approval to successful commercialization?

Are you looking for a roadmap to ensure financial solvency throughout clinical development?

Do you want to maximize your valuable time away from the office at a minimum of expense?

In three days you will learn strategies to:
1. Manage Costs to Control the Spending Burn and Remain Self-Sustaining
2. Choose the Right Design Parameters for Trial Efficiency and Sustainability
3. Minimize Waste, Maximize Efficiencies, and Speed Up Timelines During the Clinical Trial Transition
4. Optimize Cell Product Quality of Ancillary Materials
5. Incorporate the Concepts of Pharmaceutical Quality Systems into Cell Therapy Product Development
6. Leverage Manufacturing Process Technologies to Maximize Capacity and Minimize Cost-of-Goods

For more information on the event, download the brochure.  We hope to see you next week at the Clinical Business Expo!

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South Korea could be the first country to approve stem cell therapy

The Korea Food and Drug Administration (KFDA) could be the first in the world to approve stem cell therapy as a medicine in the world.  According to the Yonhap News Agency, the therapy would treat acute myocardial infarction.  The stem cells are harvested from the person's body then treated and injected back into the heart. It has passed the safety and validity tests needed for approval.

This September, The Cell Therapy Commercialization Summit will be join by AON Insurance, Argenta Advisors and others to identify how companies can ensure reimbursement for cell therapy treatments long before they are approved by the US FDA. For more about the Cell Therapy Commercialization Summit, download the brochure.

What could approval of a stem cell therapy in South Korea mean for other pending approvals across the world?

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Stem cells have potential for Type I Diabetes

ViaCyte has released news indicating that preliminary studies have shown that mice with Type I Diabetes have been cured when islet cells were injected into the mices' skin to function as pancreatic cells. According to the LA Times, Type I Diabetes is ideal for this type of treatment because it only needs to have one type of cell replaced. By inserting the pancreatic cells into the skin, the cells function as a new pancreas, thereby potentially curing the disease. Funding from the California Institute of Regenerative Medicine made this study possible.

At the Cell Therapy Commercialization Summit, Chad Green, Associate Director for Device Engineering & Manufacturing will be presenting the session, “Identify and Establish Optimal Delivery Systems for Your Cell Therapy Product.” In this session, hear about how Viacyte is pioneering delivery systems for cell therapies such as their Type 1 Diabetes study that has the potential to revolutionize the treatment of diabetes.  Download the brochure to find out more about this session and this year's agenda.

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Stem Cell research will continue to be funded by tax dollars

It’s been a rocky road for stem cell researchers in the United States, with both Presidential administrations and the courts going back and forth on whether federal funding can be used to support embryonic stem cell research. According to a recent Reuters report, the latest round went in favor of supporters of the use of ESCs, as an appeals court ruled that the Obama administration can continue to use federal tax dollars for this research.

NIH Director Francis Collins stated, "This is a momentous day -- not only for science, but for the hopes of thousands of patients and their families who are relying on NIH-funded scientists to pursue life-saving discoveries and therapies that could come from stem cell research.”

Executives at the Cell Therapy Commercialization Summit look to rulings such as these to know the barometer for where support is for cell therapy research, and particularly research using ESCs. With this recent victory under their belts, they can continue move forward with their R&D, preclinical and clinical efforts at producing viable cell therapy products, capable of mass commercialization in the coming years.

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Stem cells show promise for hearing loss

A recent study by Dr. Kazuo Oshima from Standford University took two stem cells and used them to regenerate hearing in mice. The stem cells were coaxed into becoming hair cells in the middle ear that are key in the hearing process. Their next step is to try with human cells. Should this work, which Dr. Oshima points out there are still many hurdles to overcome and there are a few years before this could be a viable option. But the study shows success. Read the full article here.

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