Autolus Therapeutics Plc (AUTL) Q3 2019 Earnings Call Transcript – The Motley Fool

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Autolus Therapeutics Plc(NASDAQ:AUTL)Q32019 Earnings CallNov 07, 2019, 8:30 a.m. ET

Operator

Hello, ladies and gentlemen, and welcome to the Autolus Therapeutics 3Q 2019 financial results conference call. [Operator instructions] As a reminder, this conference call is being recorded. I would now like to turn the conference over to your host, Ms. Silvia Taylor, vice president, global corporate affairs and communications.

Ma'am, please go ahead.

Silvia Taylor -- Vice President, Global Corporate Affairs and Communications

Thank you, Joanna. Good morning or good afternoon, everyone, and thank you for taking part in today's call on the financial results and operational highlights for the third quarter of 2019. I am Sylvia Taylor, vice president of global corporate affairs and communications, as Joanna just introduced me. With me today are Dr.

Christian Itin, our chairman and chief executive officer; and Andrew Oakley, our chief financial officer. Before we begin, I would like to remind you that during this call, we will be making forward-looking statements. All statements other than statements of historical facts contained in this presentation are forward-looking statements. Our actual results, performance or achievements may be materially different from those expressed or implied by the forward-looking statements.

For a discussion of the risks and uncertainties relating to our business and other important factors, any of which could cause our actual results to differ from those contained in the forward-looking statements, please see the section titled Risk Factors in our annual report on Form 20-F filed on November 23, 2018, as well as discussions of potential risks, uncertainties and other important factors in our other periodic filings with the SEC. The forward-looking statements contained in this presentation reflect the company's views as of the date of this presentation regarding future events, and the company does not assume any obligation to update any forward-looking statements. You should, therefore, not rely on these forward-looking statements as representing the company's views as of any date subsequent to the date of this presentation. On Slide 3, you will see the agenda for today, and it is as follows: Christian will provide a brief introduction, and that will be followed by our operational highlights for the third quarter of 2019.

Andrew will next discuss the company's financial results, and then Christian will conclude with upcoming milestones and other concluding comments. And of course, we will welcome your questions following our remarks. So with that, I'd now like to turn the call over to Christian.

Christian Itin -- Chairman and Chief Executive Officer

Thank you, Sylvia, and good morning to all of you, and thank you for joining us. I'm pleased to review our progress in the third quarter of 2019, as well as some recent company highlights. On Slide 5, and as reported yesterday, we will have four oral and two poster presentations at ASH in December in Orlando. We are pleased that we will be presenting data across the majority of our clinical programs.

The focus will be on AUTO1 with three oral presentations alone. This coming weekend also, we will have a poster presentation with preclinical data on our first solid tumor program, AUTO6NG, at SITC. Turning to Slide 6. Let's start the discussion of our Q3 operational highlights with our highest priority program, AUTO1 in adult ALL.

We're pleased that earlier this week, the U.S. FDA granted AUTO1 orphan drug designation for treatment of acute lymphoblastic leukemia. Relapse and refractory B-cell acute lymphoblastic leukemia represents a significant commercial opportunity both in terms of the potential market size, as well as the high level of unmet need in the management of the disease. Worldwide, approximately 8,400 patients are diagnosed every year with about 6,000 of those patients coming from the U.S.

and the EU5 European countries. While response to initial combination chemotherapy regimen is encouraging, only 30 to 40% of adult ALL patients will achieve long-term remissions, and the median survival for adult patients with relapsed/refractory ALL is less than one year. While Kymriah, a CD19-targeting CAR-T therapy was approved for pediatric ALL patients in 2017, no CAR-T therapy has been approved for adult ALL patients to date. The only redirected T-cell therapy approved for adult ALL is blinatumomab or BLINCYTO, a bispecific CD19-targeting T-cell engager.

Blinatumomab has a 42 response rate -- 42% response rate, yet the durability of the responses is limited and its event-free survival is 31% at six months. Slide 7 shows that data from ASH abstract on ALLCAR19, our AUTO1 study in adults with recurrent refractory ALL. As of the data cutoff, July 24, 83% of the 12 of adult patients achieved MRD-negative molecular complete response at one month. In April of this year at the AACR Annual Meeting reported that a median follow-up of five months, six out of 10 patients were alive and disease-free.

As of July 24th, 2019, data cutoff, that number remains consistent with seven of 12 patients or 58% remaining in MRD-negative remission at a median follow-up of nine months. This MRD response is measured by both flow cytometry, as well as PCR. As reported, AACR also -- and also summarized on Slide 8, none of the adult patients and none of the pediatric patients developed high-grade CRS, although in our adult patients, half of them had 50% or higher blast counts in the bone marrow at the start of therapy, which puts them at high risk for developing severe cytokine release syndrome. By ASH, we will have four additional months of follow-up and additional patients evaluable.

As reported at AACR, we only have patients -- only -- had only one patients transplanted post therapy and no further patient received transplant since. During our oral presentation at ASH next month, Dr. Claire Roddie will present additional follow-up data, including safety and efficacy. On Slide 8, I would like to provide some context on how this data fits into the landscape of adult ALL therapy.

As you can see in both adult and pediatric ALL, AUTO1 is differentiated and has the potential to be best in class. I'd like to highlight the consistency between the pediatric and adult data sets we've seen so far. Both show high molecular complete remission rate without inducing Grade 3 or higher cytokine release syndrome or requiring admission to the ICU for treatment of high-grade CRS. Also, the level of high-grade neurotoxicity is low.

While this is especially significant for the adult population who cannot tolerate high levels of toxicity, it is also significant in the pediatric population due to the high rates of severe CRS seen with Kymriah in these patients. With respect to efficacy in adult ALL, the complete response rate of 83% and the 58% rate of patients who remain in molecular remission at nine months for AUTO1 as detailed in the ASH abstract compared well to blinatumomab. This suggests a product profile that is emerging to be clearly differentiated from Blincyto and from other CD19 CAR-T approaches. If these findings are confirmed in our registration trial, AUTO1 has the potential to set a new standard of care in adult ALL.

On Slide 9, I'd like to summarize where we are with AUTO1 in adult ALL. This program will be the first Autolus program to move to pivotal stage. We have received feedback on our current study design from both the EMA and the FDA, and we will file a clinical trial authorization or CTA in the U.K. this month.

The IND is expected to be filed in the U.S. in the first quarter. The trial will be a single-arm study of approximately 100 patients in morphological relapse among sites in the U.S. and Europe.

The primary end point will the overall complete response rate, including complete response and complete response with incomplete hematologic recovery. Secondary end points will include MRD-negative complete response and event-free survival. And based on this design, we're targeting the second half of 2021 for a BLA filing. Moving on to pediatric ALL on Slide 10.

As a reminder, pediatric ALL is the most common cancer diagnosed in children with about 3,400 new cases diagnosed in the U.S. every year. While pediatric patients respond well to first-line treatment, 10 to 20% relapse or are refractory to treatment. Our development track in pediatric ALL will focus on AUTO1NG or next generation, and the pediatric investigational plan are paid for AUTO1.

The data from our AMELIA trial of AUTO3 in pediatric ALL has informed us on the encouraging role of dual-target antigen targeting. With AUTO3, as you recall, we have had robust clinical efficacy, yet the durability of such responses required further improvement. Thus, we will be moving forward in pediatric ALL using the AUTO1 construct through the development of AUTO1NG, which incorporates the CD19 CAR of AUTO1 and a novel CD22 CAR. The hypothesis for this next-generation version is to combine the favorable persistence properties observed in AUTO1 with the promising effect of dual targeting observed in AUTO3.

We will be presenting data from our trials of both AUTO1 and AUTO3 in the pediatric population next month at ASH. Additionally, we expect to initiate clinical evaluation of AUTO1NG in pediatric ALL in the first half of 2020. Moving to Slide 11 on our program in diffuse large B-cell lymphoma. We believe that DLBCL is a large commercial opportunity, given the market size and the aggressive nature of this disease.

DLBCL is the most common type of non-Hodgkin lymphoma. Approximately 24,000 patients are diagnosed every year in the U.S. alone. High-dose chemotherapy, combined with a monoclonal antibody led to remission in about 50% to 60% of patients.

Thus, we expect that addressable population to be approximately 10,000 patients in the U.S. and EU5 combined. DLBCL represents an aggressive -- and is an aggressive and rapidly progressing cancer. For patients who relapse or are refractory to first-line therapy, the current standard of care for second-line therapy consists of platinum-based chemotherapy regimen with rituximab.

Patients who respond to second-line therapy may go on to receive autologous hematopoietic stem cell transplantation or HSCT. Patients who are not candidates for HSCT or those who do not respond to second-line therapy or who relapse after HSCT are typically treated with a third-line salvage chemotherapy. These patients have a poor prognosis, and treatment is generally palliative to try to prevent further cancer growth without the intent to cure. On Slide 12, our DLBCL product candidate, AUTO3, is a dual-targeting CD19, CD22 CAR-T therapy.

The ASH abstract published this week shows that based on interim Phase 1 data, AUTO3 is active and well tolerated with no high-grade CRS observed. We plan to present additional interim Phase 1 data at ASH. The first U.S. patient has been enrolled in this study, and product has been delivered from our new manufacturing operation at the Cell and Gene Therapy Catapult at Stevenage to both U.S.

and U.K. clinical sites. Our AUTO3 program is on track for decision mid next year to advance the program to Phase 2. Slide 13 and our -- describes our multiple myeloma program.

As reported in Q2, we have stopped AUTO2 and will now move to a next-generation program. The Phase 1 experience will be presented in a poster. We aim to initiate clinical testing with a new program in the second half of 2020. On Slide 14, finally, I would like to conclude with a brief discussion of two other programs in our pipeline because they have the potential to bring additional value inflection in 2020.

Slide 14 talks about our T-cell lymphoma program. Patient enrollment in our Phase 1 study with AUTO4 will continue in the first quarter of next year with supply from the Catapult. As a result, we expect to present initial Phase 1 data in the second half of 2020. Finally, on Slide 15, to our lead program in solid tumors.

At our R&D Day in March, we focused on the heterogeneity of the solid tumor microenvironment and how the complexity and dynamic nature of these tumors pose particular challenges for effective therapies. T-cell therapies can be tailored to combat tumor complexity, and programming modules can be added to enhance activities in solid tumors. At SITC this Saturday, we will now present preclinical data on our AUTO6NG program designed to target GD2-positive tumors. This abstract is important because it shows the impact of advanced cell programming technologies in a solid tumor setting.

By adding IL-7 receptor chimeric protein, AUTO6NG demonstrated improved CAR persistence, and by adding dominant-negative TGF-beta receptor II protein and the truncated SHP2 protein, modified T cells were better able to combat the immunosuppressive tumor environment. The abstract also shows that in vivo delivery of AUTO6NG in a challenging mouse model exhibited potent antitumor activity and extended survival, whereas the clinical activity shown with -- while the clinically active AUTO6 could not do that. Based on these encouraging results which demonstrate the feasibility, safety and efficacy of AUTO6NG, we plan on initiating a clinical study in patients with refractory/relapsed neuroblastoma in the second half of next year. We're looking forward to discussing these results with those of you who will be at SITC this weekend.

On Slide 16, I want to share a few other updates before I turn the call over to Andrew to discuss our financials. On the manufacturing side, the Catapult site is fully operational and delivering all our clinical products for patients in both Europe and the U.S. In September, PPF Group announced that they had acquired mainly from Woodford Investment Management, an approximate 19% holding of Autolus. And control of all the remaining shares of Autolus by Woodford Investment Management are in the process of being transferred to Schroder UK Public Private Trust plc.

Finally, with regards to organizational changes, we announced last month that David Brochu has been named senior vice president, head of product delivery, to lead the transition of the company's manufacturing organization to deliver products for registration studies and ultimately commercial sale. Dave has 30 years of technology operations and engineering management expertise in the biopharmaceutical industry. He joined Autolus in March 2019 as vice president, technical operations. In addition, Vishal Mehta was named vice president and head of clinical operations throughout the transition of the company to move into the registration studies.

Vishal joined Autolus in January 2019 from Celgene, where he had the planning and execution of multiple clinical studies for CAR-T products. We're happy to be working with both of them in these expanded capacities. With that, I will turn the call over to Andrew for our third-quarter 2019 financial update. Andrew?

Andrew Oakley -- Chief Financial Officer

Thanks, Christian, and good morning or good afternoon to everyone. It's my pleasure to review our financial results for the three-month period, July through September of 2019. On Slide 18, net total operating expenses for the three months ended 30 September 2019 were $35.6 million. That was net of grant income of $0.3 million, and that compares to net operating expenses of $17.1 million, also net of grant income of $0.3 million, but that's the same period in 2018.

The increase was due, in general, to the increase in development activity, increased head count primarily in our development and manufacturing functions and the cost of being a public company. Research and development expenses increased to $27.3 million for the three months ended 30 September 2019 from $10.1 million for the three months ended 30 September 2018. Cash costs, which exclude depreciation, as well as share-based compensation, increased to $21.6 million from $9 million. The increase in research and development cash costs of $12.6 million consisted primarily of an increase of compensation-related costs of $5.2 million due to an increase in employee head count to support the advancement of our product candidates and clinical development, an increase of $3.6 million in research and development program.

Expenses related to the activities necessary to prepare, activate and monitor clinical trial programs, including the manufacturing and technical transfer activities required for AUTO1 to enable the commencement of the registration study in adult ALL and an increase of $2.6 million in facilities costs supporting the expansion of our research development, translational science capability and investment in manufacturing facilities and equipment, and lastly, an increase of $0.7 million in telecom software costs, as well as an increase of $0.5 million elsewhere. General and administrative expenses increased to $8.6 million for the three months ended 30 September 2019 from $7.3 million for the three months ended September 30, 2018. Cash costs, which again exclude the depreciation expense and share-based compensation, decreased to $5.6 million from $5.7 million. Compensation-related expenses decreased by $0.6 million.

IT, communication, general office expenses decreased by $0.7 million, and that was offset by legal and professional fees of 0.9 million and an increase of 0.3 million in very preliminary commercial expenses. Net loss attributable to ordinary shareholders was $27.2 million for the three-month period compared to $12.9 million for the same period in 2018. The basic and diluted net loss per ordinary share for the three months ended 30 September 2019 totaled $0.61 or 61 cents compared to a basic and diluted net loss per ordinary share of $0.33 for the three months ended 30 September 2018. Cash and cash equivalents at the end of the period totaled $229.4 million, and that compares with $247.1 million at the end of September in 2018.

And we anticipate that cash on hand provides us with the runway into the second half of 2021. With that, I will now hand the call back to Christian to give you a brief outlook on our expected upcoming milestones. Christian?

Christian Itin -- Chairman and Chief Executive Officer

Thank you, Andrew. Let me conclude this part of the management discussion with a review of the upcoming milestones and news flow through 2020. Let's move to Slide 20. The upcoming 15 months will be an eventful period for us with multiple clinical milestones and opportunities for value creation.

Our chief operational focus will be our moving AUTO1 in adult ALL into registration trial in the U.K. and U.S. We also expect to report data across various programs and to progress a number of our other clinical candidates, specifically updates on our ongoing clinical trials, initiation of a Phase 1 study of AUTO1NG in pediatric ALL in the first half of next year, a go/no-go decision on Phase 2 initiation of AUTO3 in DLBCL middle of 2020, initiation of a Phase 1 study of AUTO6NG in neuroblastoma in the second half of 2020 and initiation of a Phase 1 study in the next-generation program in multiple myeloma also in the second half of 2020. In conclusion, on Slide 23, I'd like to recap the major messages from today's call.

First, AUTO1 is our foundational program and the first Autolus program expected to move into pivotal stage. Given the positive safety and efficacy profile today, we believe that AUTO1 has the potential to be a best-in-class CD19 CAR T in ALL. Secondly, our next priority is on AUTO3 in DLBCL with AUTO3NG as a next-generation opportunity. We expect to report full Phase 1 data for AUTO3 in middle of 2020 to reach a decision point on Phase 2 trial initiation thereafter.

Third, in the pediatric ALL, we have transitioned our focus on AUTO1 and AUTO1NG. While AUTO3 data confirmed the dual-targeting hypothesis, we believe the excellent persistence with AUTO1 is likely to drive long-term remissions. Looking ahead to 2020, we see opportunity for additional value steps for multiple myeloma, T-cell lymphoma and the GD2-positive tumor programs. The company has a strong balance sheet with 230 million in cash, which provides a run rate to the second half of 2021.

And finally, we're looking forward to seeing many of you at the upcoming SITC and ASH Annual Meetings. We'd now like to take your questions. Operator, please open the line.

Operator

[Operator instructions] Your first question comes from the line of Gil Blum from Needham & Company. Your line is open.

Gil Blum -- Needham and Company -- Analyst

Thank you for taking my question. Just a quick one about -- so AUTO6NG data that's coming out at SITC, we know that this is kind of a mix of T cells that were transected with two different vectors. What kind of analysis would you have to do in a product that's this complex before using it in human? Like, how would the product be defined?

Christian Itin -- Chairman and Chief Executive Officer

Well, first of all, thanks for joining, and thanks for your question. Obviously, what we're doing with AUTO6 is we're introducing a substantial amount of genetic information into a single cell. And that is actually a level of genetic information that you cannot deliver with a single vector. So you have to use two vectors to do that.

And this is now actually an approach that's been used in a number of programs that have gone through regulatory review and are actually currently the clinic for other types of indications and obviously have gone through the normal regulatory process and are active in development. Ultimately, what you have to show is you have to demonstrate the activity of the product as is and you design your safety studies, etc, to really understand the activity of the product as a whole. We have to understand, obviously, also that even when you look at a product that is transduced to the single vector that we have multiple types of differentiation state of T cells in there, which gives you quite a wide range of properties of these cells, just based on the differentiation state. So the products are complex to begin with.

And the programming on itself, we don't believe will add a significant element on top by adding the two vectors in of themselves. Vectors are designed to -- all of them actually recognize the target antigen so that the basic activity is actually shared among all transduced cells.

Gil Blum -- Needham and Company -- Analyst

All right. Maybe a bit of an odd question, but if Kymriah or ever used off-label in adults ALL? Is that -- anyone does that?

Christian Itin -- Chairman and Chief Executive Officer

Well, what we do know is that the products, obviously, are not part of the normal payment process that you can actually get -- actually have them used in. If they're used off label or not, that's difficult for us to tell. There's certainly a possibility within oncology for products to be used off label, but it's something we can't judge. And I don't think there's any information out there on what it might be and how many patients might actually be impacted.

Gil Blum -- Needham and Company -- Analyst

Got you. And just the last one. I know we're getting an update on AUTO2, but when could we expect updates on the new program in multiple myeloma?

Christian Itin -- Chairman and Chief Executive Officer

Yes. So the next-gen version for the multiple myeloma program we expect to update when we're actually entering into clinical trials and obviously, during the course of next year have opportunity to provide an update on the design of the program, and we'll do it at that point in time.

Gil Blum -- Needham and Company -- Analyst

Thank you for taking my questions and congrats on the quarter.

Christian Itin -- Chairman and Chief Executive Officer

Thank you so much. Thanks for joining.

Operator

Your next question comes from the line of Jim Birchenough from Wells Fargo. Your line is open.

Jim Birchenough -- Wells Fargo Securities -- Analyst

Hi guys, thanks for the call and congrats on the progress toward the AUTO1 pivotal. Just on that pivotal question, could you maybe speak to, No. 1, are there going to be any entry criteria whether it's tumor burden or other features that will reduce the risk of severe CRS or neurotoxicity? And then maybe if you could discuss what the efficacy hurdle is there, if there's some lower bound of the confidence interval you need to shoot for at some point estimate of response, just so we have a frame of reference. And then I've got a follow-up.

Christian Itin -- Chairman and Chief Executive Officer

Yes. First of all, thanks for joining, Jim. With regards to the pivotal study that we're planning to do here, obviously, when you look at the inclusion criteria, we're including patients that actually are in formal relapse. And in other words, these are patients that have more than 5% blasts in the marrow, which is kind of when you have a morphological relapse.

So all these patients have manifest disease, and obviously, given the speed at which the disease moves, can actually have quite a range. And as you see with the data that we have shown at AACR and we'll update at ASH, and approximately half the patients have a massive level of tumor burden, but all patients are in formal relapse.

Jim Birchenough -- Wells Fargo Securities -- Analyst

And just in terms of the efficacy hurdle?

Christian Itin -- Chairman and Chief Executive Officer

In terms of the efficacy hurdle, when we look at the current programs, you'd expect and you'd see kind of well from our own programs that we've shown today is that you want to show a robust molecular complete remission rate because that's your entry ticket for a transformational activity. And then, obviously, you want to have durability of effect. So, we would clearly want to improve substantially over and above the durability of effect that we've seen with blinatumomab in this patient population. Remember that was 31% at six months.

We believe that we want to see somewhere in the range of the double of that at that point in time.

Jim Birchenough -- Wells Fargo Securities -- Analyst

Terrific. And then just on AUTO3 and the go/no-go decision in DLBCL in mid-2020, is durability of response going to be the key there? Or maybe you could speak to what the criteria are for go/no-go. And I guess if there was a durability that was adequate in ALL, is there something different about DLBCL where you might still get away with a win there? Just trying to understand the go/no-go.

Christian Itin -- Chairman and Chief Executive Officer

Yes. The disease settings are quite different between leukemia, acute leukemia and DLBCL. What we need to be able to do in leukemia is we need to be able to put pressure on the tumor for very long periods of time. We're talking 18 months, 24 months to get to transformational activity, and we're actually going to be showing kids that are now really long-term -- have long-term observation on the AUTO1 experience.

When you look at DLBCL, it is quite different. What you need to induce is you need to induce a complete remission. And typically, the complete remission that you can induce and observe at three months or at the latest at six months depending on the program is a very good measure for long-term benefit in that patient population. And the effect that it can induce is, obviously, one that happens relatively quickly, usually within three months' period of time.

Most of those patients have accomplished or have achieved their CR. And at that point, most of the patients, if you look at the Yescarta data, the JCAR-17 data, the Kymriah data, this can actually sustain that activity. And so what we want to see is, we want to see a robust CR rate, as well as, obviously, have a good sense for the durability of those CRs as well at that point in time.

Jim Birchenough -- Wells Fargo Securities -- Analyst

Just a final question, Christian. Reimbursement -- inpatient reimbursement for Kymriah and Yescarta has been difficult from what we've heard, and we've heard from a number of consultants that the move to outpatient is going to be really important in advance of getting some CAR-T specific reimbursement code for the inpatient setting. And so how important is it to you to have features in your cell that lend themselves to outpatient delivery? And is there some way to incorporate that into a trial design?

Christian Itin -- Chairman and Chief Executive Officer

I think it is important when you think about the overall cost of therapy, obviously, there is the actual cost of the therapeutic itself, of the drug itself, but there's also a significant cost associated with managing the patients. And obviously, the more severe your adverse events are, the higher the costs are for the management of the patients. And what cause challenges, particularly in DLBCL reimbursement in the U.S. and elsewhere, is that that portion of patient management cost was initially not properly covered.

And that caused the major issue for the hospitals who were treating these patients. That is now actually being resolved. It's also in part resolved for the Medicare patient, but it remains a significant driver of the overall cost of therapy. So actually, having products that have no high-grade CRS, cytokine release syndrome, that have limited or minimal neurotoxicity is important because it allows you to actually consider giving the patient the therapy and then actually have the patient in an outpatient setting from there on forward.

And as you see with all the programs, it is a progression in terms of the information and the experience you have with the product. What you want to make sure is that it captures much information related to that intensity of patient management during the course of your pivotal study even if your patients are initially mostly treated as inpatients, and then obviously with increased experience of the products, will move more toward an outpatient setting. But it's absolutely crucial to collect that information also when you have conversations with payers because it is a key element of the value assessment as well.

Jim Birchenough -- Wells Fargo Securities -- Analyst

Well, thanks for taking the questions.

Christian Itin -- Chairman and Chief Executive Officer

Thank you very much.

Operator

Your next question comes from the line of Matt Phipps from William Blair. Your line is open.

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Autolus Therapeutics Plc (AUTL) Q3 2019 Earnings Call Transcript - The Motley Fool

Stem Cell Therapy Market Trends, Secondary Research With Geron Corporation, Vericel Corporation, Pluristem Therapeutics, Cytori Therapeutics, Fate…

Stem Cell Therapy Market research analysis and insights displayed in this report are very thoughtful for the businesses to make enhanced decisions, to build up better strategies about production, marketing, sales and promotion of a particular product. Stem Cell Therapy market report also takes into consideration several major factors such as revenue, cost, gross and gross margin while analysing market data. Various markets at local, regional and international level are thought of in this Stem Cell Therapy report. All this helps in extending their reach towards the success. The use of advanced tools and techniques applied for this report makes it the premium in the class. By understanding clients needs precisely, this report merges business and product information for the sustainable growth in the market. Geron Corporation, Vericel Corporation, Pluristem Therapeutics, Cytori Therapeutics, Fate Therapeutics are some players grooming the market.

Stem Cell Therapy Market is expected to reach USD 15.63 billion by 2025, from USD 7.72 billion in 2017 growing at a CAGR of 9.2% during the forecast period of 2018 to 2025. The Stem Cell Therapy market report contains data for historic year 2016, the base year of calculation is 2017 and the forecast period is 2018 to 2025 (Updated values listed in sample report).

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Stem cell therapy is the therapy which uses stem cells for the treatment or prevention of a disease. Bone marrow transplant is the widely applicable therapy which is followed by umbilical cord blood. Research is going on to develop various sources (such as cord blood cells, bone marrow and skin) to use these cells for treatment of various disorders like neurodegenerative diseases and conditions such as heart disease, diabetes and other conditions. Some of the major players operating in the global stem cell therapy market are

Others: ViaCyte, Inc, AbbVie, Mesoblast Ltd., Roslin Cells, Regeneus Ltd, ReNeuron Group plc,, International Stem Cell Corporation, Aastrom Biosciences, Inc., Advanced Cell Technology, Cryo Cell International, Cytori Therapeutics, Inc., Geron Corporation, and Invitrogen and others. The global stem cell therapy market is highly fragmented and the major players have used various strategies such as new product launches, expansions, agreements, joint ventures, partnerships, acquisitions, and others to increase their footprints in this market. The report includes market shares of the global stem cell therapy market for global, Europe, North America, Asia Pacific and South America.

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Major Market Drivers and Restraints:

Drivers:

Restraints:

Segmentation:

The global stem cell therapy market is segmented based on

Type

Product

Application

End Users

Geographical Segments

On the basis of type, the market is segmented into

Allogeneic stem cell therapy

Autologous stem cell therapy

The allogeneic stem cell therapy segment is expected lead the market because of commercialization of allogeneic stem cell therapy products and wide application with easy scale up process.

Based on products, the market is segmented into

Adult stem cells

Human embryonic stem cells

Induced pluripotent stem cells and others

The adult stem cells accounts highest share in market due to ability to generate trillions of specialized cells which may lower the risks of rejection and repair tissue damage.

Based on application, the market is segmented into

Musculoskeletal disorders

Wounds and injuries

Cardiovascular diseases

Surgeries

Gastrointestinal diseases, and other applications

The musculoskeletal disorders segment leads the market due to availability of stem cell-based products for the treatment of musculoskeletal disorders, high prevalence of musculoskeletal disorders and bone & joint diseases.

Based on end users, the market is segmented into

Therapeutic companies

Cell and tissues banks

Tools and reagent companies

Service companies

The growing number of stem cell donors, improved stem cell banking facilities and because of the research and development therapeutic companies held the largest share in stem cell therapy.

By Geography

North America (U.S., Canada, Mexico)

South America (Brazil, Argentina, Rest of South America)

Europe (Germany, France, United Kingdom, Italy, Spain, Russia, Turkey, Belgium, Netherlands, Switzerland, Rest of Europe)

Asia-Pacific ( Japan, China, South Korea, India, Australia, Singapore, Thailand, Malaysia, Indonesia, Philippines, Rest of Asia Pacific)

Middle East & Africa (South Africa, Egypt, Saudi Arabia, United Arab Emirates, Israel, Rest of Middle East & Africa)

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Stem Cell Therapy Market Trends, Secondary Research With Geron Corporation, Vericel Corporation, Pluristem Therapeutics, Cytori Therapeutics, Fate...

Huntington’s Marked by Inflammation and Changes in Brain’s Striatum Before Symptoms, Study Finds – Huntington’s Disease News

Massive inflammation and other changes in the striatum, an area of the brain selectively destroyed in Huntingtons disease (HD), are already present before patients develop any symptoms, a study has found.

These findings could help understand how the disease unravels and why this brain region is particularly sensitive to degeneration in individuals with Huntingtons.

The study, The caudate nucleus undergoes dramatic and unique transcriptional changes in human prodromal Huntingtons disease brain, was published in the journal BMC Medical Genomics.

Huntingtons is a neurodegenerative disease caused by mutations in the huntingtin(HTT) gene and marked by trouble in controlling movement, a progressive loss of thinking ability, and psychiatric problems.

Symptoms, which typically begin when people reach their 30s and 40s, stem from a selective degeneration of certain brain regions, particularly two areas: the basal ganglia, a region deep in the brain thats responsible for functions including movement coordination; and the cortex, the outer and highly twisted layer of the brain which controls thought, behavior, and memory.

Within the basal ganglia, HD targets nerve cells (neurons) of the striatum, especially in two areas known as the caudate nuclei and putamen. These regions can shrink and suffer massive damage as a result of disease progression.

Very little is known about the active disease processes leading to such debilitating symptoms. Obtaining post-mortem brain samples from people with disease-causing mutations who have not yet developed symptoms in other words, still have a largely intact striatum is very rare.

Researchers atBoston University School of Medicine (BUSM) had the opportunity to analyze samples from the striatum more precisely, the caudate nucleus of two HD-positive individuals who had no symptoms at the time of their death.

To pinpoint early drivers of disease, the team compared the activity (expression) of genes those turned on and turned off in the caudate nucleus of these asymptomatic individuals to the prefrontal cortex of 26 symptomatic Huntingtons patients and 56 healthy controls.

Researchers used a high throughput sequencing technology called RNA-Seq to determine gene expression profiles, and a preformed bioinformatics analysis to understand which genes and biological processes were altered.

Our data suggest that the striatum experiences massive inflammation in HD even before symptoms appear, and exhibits a similar gene expression pattern to that observed in prefrontal cortex. Patterns unique to the striatum are also observed, Adam Labadorf, PhD, director of BUs Bioinformatics Nexusand the studys senior author,said in a news release.

In addition to extensive inflammatory processes, the data also suggested that over the diseases course, the striatum undergoes some form of neurogenesis, or the generation of new nerve cells.

While these are only trends that warrant further investigation, researchers propose that active production of neurons could be happening in the striatum during the prodromal phase (before symptom onset) to compensate for the nerve cell loss that precedes symptoms.

The idea that active neurogenesis occurs in the adult brain is controversial, but could lead to exciting discoveries into the innate regenerative capabilities of the central nervous system, Labadorf said.

According to researchers, these findings provide clear evidence that the caudate nucleus is strongly affected in people positive for Huntington disease, before the emergence of any symptoms.

This study presents the most detailed analysis to date of the active disease process in the primarily affected brain region of HD, and although these results do not directly suggest any novel therapies, a better understanding of these processes is likely to lead to them, Labadorfadded.

An important observation was that some genes, like HSPA6, were perturbed across all HD patient samples relative to healthy brains. This set of genes may provide an opportunity to develop prognostic tests for disease progression, the researchers noted.

A robust clinical test measuring disease progression will likely take the form of a panel of key inflammatory and possibly developmental genes measured in the blood or cerebrospinal fluid (the liquid surrounding the brain and spinal cord), they wrote.

Ana is a molecular biologist enthusiastic about innovation and communication. In her role as a science writer she wishes to bring the advances in medical science and technology closer to the public, particularly to those most in need of them. Ana holds a PhD in Biomedical Sciences from the University of Lisbon, Portugal, where she focused her research on molecular biology, epigenetics and infectious diseases.

Total Posts: 79

Ana holds a PhD in Immunology from the University of Lisbon and worked as a postdoctoral researcher at Instituto de Medicina Molecular (iMM) in Lisbon, Portugal. She graduated with a BSc in Genetics from the University of Newcastle and received a Masters in Biomolecular Archaeology from the University of Manchester, England. After leaving the lab to pursue a career in Science Communication, she served as the Director of Science Communication at iMM.

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Huntington's Marked by Inflammation and Changes in Brain's Striatum Before Symptoms, Study Finds - Huntington's Disease News

In 2030, Our Protein Will Come From a Laband We’ll All Be Better Off For It – Singularity Hub

Could a hamburger grown in a lab from Kobe beef stem cells be cheaper, better tasting, and healthier for you? Can you imagine a future where millions of square miles of pastoral land are reclaimed by nature, creating new forests and revitalizing the Earths vital carbon sinks?

Last week, we discussed the hyper-efficient food production systems of 2030. This week, we continue that discussion, but from a different perspectivebecause by the end of the next decade, we will witness the end of industrial animal agriculture as we know it.

Through the convergence of biotechnology and AgTech, we will witness the birth of the most ethical, nutritious, and environmentally sustainable food system ever devised by mankind.

Lets dive in.

Meat production is problematic, to say the least. A quarter of the planets available landmass is currently used to keep 20 billion chickens, 1.5 billion cattle, and 1 billion sheep alivethat is, until we can kill them and eat them. The suffering quotient is through the roof. As is the waste.

Worse is the water involved. Meat production accounts for 70 percent of global water use. Compared to 1,500 liters required to produce a kilogram of wheat, it takes 15,000 liters to produce a kilogram of beef, meaning theres enough water in an adult steer to float a US Navy destroyer.

Meat is also responsible for14.5 percentof all greenhouse gases and a considerable portion of our deforestation problem. In fact, we are in the midst of one of the largest mass extinctions in history, and loss of land for agricultural production is currently the largest driver of that extinction.

Enter cultured meat: meat that is grown from a few cells into a full-blown steak.

Take a few stem cells from a live animal, typically via a biopsy so the animal isnt harmed. Feed these cells a nutrient-rich solution. Power the whole process in bioreactors. Give the industry a few years to mature and the technology a few years to shed costs and, finally, we can produce an infinite number of steaks to feed an increasingly carnivorous population.

There are still numerous hurdles to overcome in the process, but we are fast approaching the point at which converging exponential technologies will enable this transformation of todays food system.

Environmental issues aside, cultured meat has the potential to become far more cost-effective than conventional meat. It will soon compete with the latter on almost every market-oriented criteria in existence.

For starters, cultured meat production is mostly an automated process without much need for land or labor. Plus, it takes a few years to grow a cow in the wild, but only afewweeksto grow a cows worth of steak in the lab.

And its more than just steak. The meats in development range from pork sausage and chicken nuggets to foie gras and filet mignonit all depends on which stems cells you start out with.

In late 2018, for example, Just Foods announced a partnership with Japanese Wagyu beef producer Toriyama to develop cultured meat from the cells of what has long been the rarest and most expensive steaks on Earth.

And whats true for meat is also true for milk.

Perfect Day Foods, a Berkeley, California-based company started by two founders with a passion for cheese, has figured out how to make the animal-derived product without any involvement from cows. Combining gene sequencing with 3D printing and fermentation science, theyve created a line of animal-free dairy products.

So what does this all mean? A fundamental reconfiguration of the way we source, consume, and pay for foodnot to mention the environmental costs that are often written off as externalities.

Such a transformation will revamp our world in ways we have only begun to imagine.

The decimation of resources alone is considerable. Cultured meat uses 99 percent less land, 82-96 percent less water, and produces 78-96 percent less greenhouses gases. Energy use drops somewhere between 7 and 45 percent depending on the meat involved (traditional chicken ranching is much more energy-intensive than traditional beef ranching).

And by liberating a quarter of our landmass, we can also reforest, providing sufficient habitat required to halt the biodiversity crisis and revitalize tremendous natural carbon sinks needed to slow global warming.

While thats a haze of numbers to consider, what they add up to is astounding: An ethical and environmental solution to world hunger.

Its also a muchhealthiersolution. As we will soon be growing steak from stem cells, we can do the impossible: Make fast food hamburgers that are actually good for you. Well be newly able to increase helpful proteins, reduce saturated fat, even add vitamins.

Another huge win: cultured meat requiresno antibiotics. Given the danger of diseases like mad cow disease, next-gen meat consumption will be far safer for humans, reducingif not eliminatingthe food industrys industrial hygiene challenges.

In fact, as a high percentage of emerging diseases come from livestock, by turning to cultured meat, were both lowering the global disease burden and decreasing our risk of pandemic.

Over the last two weeks, we have explored how converging exponentials will revolutionize one of humanitys most basic needs. By the end of the next decade, anyone anywhere will have on-demand, ultra-cheap access to lab-grown meatfar more nutritious than livestock-derived products, with a near-zero carbon footprint and safety guarantees.

Dont want to leave home? The rise of vertical farming, autonomous drone networks, and last-mile delivery advancements will make food deliverable to your doorstep, sourced from a low-land-use food production center. Local foods will be truly local. Either that, or download physiology-optimized recipes to your in-home food 3D printer.

While traditional agriculture has experienced shifts and industrialization, growing food has roughly been the same since10,000 BC.

Soon to undergo one of the most monumental technological revolutions in history, our food system is about to be leagues more efficient, ethical, and sustainable than ever beforenot to mention far healthier.

In just a few years, humans will become the first animal that derives our protein from other animals, yet doesnt harm anyone in the process. Meat milesthat is, the transportation costs involved in moving meat aroundwill nearly disappear. Slaughterhouses will become a ghost story we tell our grandchildren.

And a planet that is already significantly strained under the weight of seven billion will have a fighting chance as we grow to ~10 billion by 2050.

Image Credit: Photo byCarolien van OijenonUnsplash

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In 2030, Our Protein Will Come From a Laband We'll All Be Better Off For It - Singularity Hub

BrainStorm Cell Therapeutics to Present at the Dawson James Securities 5th Annual Small Cap Growth Conference – BioSpace

NEW YORK, Oct. 25, 2019 (GLOBE NEWSWIRE) -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leader in the development of innovative autologous cellular therapies for highly debilitating neurodegenerative diseases, today announced that it will be presenting at the Dawson James Securities 5th Annual Small Cap Growth Conference, being held on October 28-29, 2019 at the Wyndham Grand Hotel in Jupiter, Florida.

Preetam Shah, PhD, MBA, Chief Financial Officer is scheduled to present on Tuesday, October 29th at 3:40 p.m. Eastern Time, in Track 2 - Preserve Ballroom B, with one-on-one meetings to be held throughout the conference.

Chaim Lebovits, President and CEO of BrainStorm said, We are pleased to have the opportunity to have Dr. Shah present at the Dawson James Small Cap Growth Conference. Dr. Shah, joined BrainStorm in September 2019, and we look forward to having him present the Companys growth strategy and future to a wide audience of accreditied investors.

About NurOwnNurOwn (autologous MSC-NTF cells) represent a promising investigational approach to targeting disease pathways important in neurodegenerative disorders. MSC-NTF cells are produced from autologous, bone marrow-derived mesenchymal stem cells (MSCs) that have been expanded and differentiated ex vivo. MSCs are converted into MSC-NTF cells by growing them under patented conditions that induce the cells to secrete high levels of neurotrophic factors. Autologous MSC-NTF cells can effectively deliver multiple NTFs and immunomodulatory cytokines directly to the site of damage to elicit a desired biological effect and ultimately slow or stabilize disease progression. NurOwn is currently being evaluated in a Phase 3 ALS randomized placebo-controlled trial and in a Phase 2 open-label multicenter trial in Progressive MS.

About BrainStorm Cell Therapeutics Inc.BrainStorm Cell Therapeutics Inc. is a leading developer of innovative autologous adult stem cell therapeutics for debilitating neurodegenerative diseases. The Company holds the rights to clinical development and commercialization of the NurOwn Cellular Therapeutic Technology Platform used to produce autologous MSC-NTF cells through an exclusive, worldwide licensing agreement. Autologous MSC-NTF cells have received Orphan Drug status designation from the U.S. Food and Drug Administration (U.S. FDA) and the European Medicines Agency (EMA) in ALS. BrainStorm has fully enrolled the Phase 3 pivotal trial in ALS (NCT03280056), investigating repeat-administration of autologous MSC-NTF cells at six sites in the U.S., supported by a grant from the California Institute for Regenerative Medicine (CIRM CLIN2-0989). The pivotal study is intended to support a BLA filing for U.S. FDA approval of autologous MSC-NTF cells in ALS. BrainStorm received U.S. FDA clearance to initiate a Phase 2 open-label multi-center trial of repeat intrathecal dosing of MSC-NTF cells in Progressive Multiple Sclerosis (NCT03799718) in December 2018 and has been enrolling clinical trial participants since March 2019. For more information, visit the company's website.

Safe-Harbor Statements Statements in this announcement other than historical data and information, including statements regarding future clinical trial enrollment and data, constitute "forward-looking statements" and involve risks and uncertainties that could cause BrainStorm Cell Therapeutics Inc.'s actual results to differ materially from those stated or implied by such forward-looking statements. Terms and phrases such as "may", "should", "would", "could", "will", "expect", "likely", "believe", "plan", "estimate", "predict", "potential", and similar terms and phrases are intended to identify these forward-looking statements. The potential risks and uncertainties include, without limitation, BrainStorms need to raise additional capital, BrainStorms ability to continue as a going concern, regulatory approval of BrainStorms NurOwn treatment candidate, the success of BrainStorms product development programs and research, regulatory and personnel issues, development of a global market for our services, the ability to secure and maintain research institutions to conduct our clinical trials, the ability to generate significant revenue, the ability of BrainStorms NurOwn treatment candidate to achieve broad acceptance as a treatment option for ALS or other neurodegenerative diseases, BrainStorms ability to manufacture and commercialize the NurOwn treatment candidate, obtaining patents that provide meaningful protection, competition and market developments, BrainStorms ability to protect our intellectual property from infringement by third parties, heath reform legislation, demand for our services, currency exchange rates and product liability claims and litigation,; and other factors detailed in BrainStorm's annual report on Form 10-K and quarterly reports on Form 10-Q available at http://www.sec.gov. These factors should be considered carefully, and readers should not place undue reliance on BrainStorm's forward-looking statements. The forward-looking statements contained in this press release are based on the beliefs, expectations and opinions of management as of the date of this press release. We do not assume any obligation to update forward-looking statements to reflect actual results or assumptions if circumstances or management's beliefs, expectations or opinions should change, unless otherwise required by law. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance or achievements.

CONTACTS

Corporate:Uri YablonkaChief Business OfficerBrainStorm Cell Therapeutics Inc.Phone: 646-666-3188uri@brainstorm-cell.com

Media:Sean LeousWestwicke/ICR PRPhone: +1.646.677.1839sean.leous@icrinc.com

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BrainStorm Cell Therapeutics to Present at the Dawson James Securities 5th Annual Small Cap Growth Conference - BioSpace

NASA plans to cut cost of space exploration by commercializing low-earth orbit – Digital Trends

The Artemis 1 Orion Crew Module. NASA

In a presentation at the 2019 International Astronautical Congress in Washington, D.C., NASA Administrator Jim Bridenstine spoke about the agencys plans for the Artemis mission to the moon and then on to Mars. And he laid out how NASA plans for pay for this new era of manned space exploration: By encouraging the commercialization of low-earth orbit in order to cut costs for the agency.

Were not going back to the moon, Bridenstein promised. Were going forward to the moon. The aim for the 2024 mission is for astronauts to stay on the moon for a longer period of time than ever before, and for the mission to be sustainable in a new way by using resources found on the moon itself.

Of course, sending humans off the planet is expensive, especially when trying to take them farther than any man has traveled before. To achieve its aims, NASA intends to double down on partnerships with commercial companies and to encourage even more commercial activities in space.

Currently, NASA has commercial space programs such as the commercial resupply missions for the International Space Station and the upcoming commercial crew program. It wants to expand these programs to include commercial habitation programs too, especially in low-earth orbit where the International Space Station (ISS) resides.

Earlier this year, NASA caused some controversy when it announced that the ISS would begin accepting space tourists as early as 2020. Experts worried about the precedent of allowing wealthy individuals to visit a scientific research outpost and about the potential environmental costs of transporting those passengers into space.

Others have raised concerns that NASA may be putting itself in competition with private companies, but Bridenstine doesnt see it that way. NASA wants to be a customer, he explained. One of many in a very robust marketplace in low-earth orbit. The type of work that can be done in low-earth orbit includes bio-medicine projects like the 3D printing of organs using adult stem cells, or the creation of artificial retinas. This work cant be performed on Earth because of the way materials are layered, but it can be done in the microgravity of space.

The hope is that the proliferation of commercial programs will lower costs for NASA: We want those companies to go out and get customers that arent us, to drive down costs, Bridenstine said.

An example of this commercialization in action will be the delivery of the VIPER rover, the lunar rover that should land in 2022. The rover will be built by NASA, but it will be delivered by a private company through the Commercial Lunar Payload Services, or CLPS, program.

Partnerships between NASA and private companies have not always worked out smoothly, however. Bridenstein publicly criticized SpaceX CEO Elon Musk recently for showing off the companys new Starship rocket while its contributions to the commercial crew program are years behind schedule. And similar scheduling issues have beset Boeings crew capsule, the Starliner, which has also been delayed.

Time will tell if the involvement of private companies is the key to a more affordable space program, or whether a focus on profits over exploration will undermine the efforts of NASA and other government space agencies.

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NASA plans to cut cost of space exploration by commercializing low-earth orbit - Digital Trends

Artificial Early Mouse Embryos Created In The Lab Using Stems Cells Rather Than Sperm Or Eggs – IFLScience

A groundbreaking study has seen scientists create the early stages of artificial mice embryos in a lab using stem cells, rather than sperm or eggs, for the first time.

Its still very early days for the research, and it has some big problems to iron out, but itcould potentially pave the way towards creating viable embryos just from cultured cells. It could also provide scientists with an indefinite source of blastocysts to study the early stages of development, without the need for sperm, eggs, or natural embryos.

These studies will help us to better understand the very beginnings of life; how early on in life a single cell can give rise to millions of cells and how they are assembled in space and time to give rise to a fully developed organism. Importantly, this work avoids the use of natural embryos and is scalable, Juan Carlos Izpisua Belmonte, study author and professor in Salks Gene Expression Laboratory, explained in a statement.

Reporting in the journal Cell, researchers at the Salk Institute and the University of Texas Southwestern Medical Center created structures resembling mouse blastocysts, known as blastoids.

Blastocysts are structures made up of 100+ cells or so, found in the very early stages of development in mammals. After the blastocyst implants itself into the uterus, it can develop into an embryo.Typically, the blastocyst forms around five days after a sperm has fertilized an egg.

However, the researchers managed to create an early form of blastocyst using pluripotent stem cells, master cells that can turn into almost any kind of tissue in the body, obtained from adult mouse cells.The stem cells were placed together and nurtured in a special culture medium. They soon formed connections with each other, eventually forming a ball of cells with an inner and outer layer.

The structure of this, according to the researchers, was remarkably like a natural blastocyst. The artificial blastoids contained the same three primordial cell types found in natural blastocysts, along with being a similar size and similar gene expression.

Other researchers working in the field have described the research as wonderful, significant, and outstanding, although they warn there are still many hurdles to overcome before we see this research being applied.

For example, its noteworthy that the blastocyst-like structures showed signs of malformation and disorganization, meaning they wouldnt be able to develop into a fully viable embryo or mouse.

I would caution against interpreting the current study as showing that embryos can be made from adult tissues this has the potential to raise undue ethical concerns, the structures made are not embryos, and the research is in mice not humans, said Dr Harry Leitch, a stem cell biologist at the MRC London Institute of Medical Sciences and Imperial College London who was not involved in the study.

In reality, this study reports incremental findings in a pre-existing model system, and makes a valuable contribution to ongoing research in the field.

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Artificial Early Mouse Embryos Created In The Lab Using Stems Cells Rather Than Sperm Or Eggs - IFLScience

Healthcare Terms Are Out of Touch, Says Mom Advocate – Medscape

This transcript has been edited for clarity.

Eric J. Topol, MD: Hello. I'm Eric Topol, editor-in-chief for Medscape. I'm thrilled to speak with Hala Durrah today, who I got to know about through a remarkable essay she wrote for Health Affairs back in March. Hala, welcome to our program.

Hala H. Durrah, MTA: Thank you for having me.

Topol: It's a real pleasure and this an important and critical topic. Your essay was "My Child Is Sick; Don't Call Her a 'Consumer.'"[1] There is so much to learn here for Medscape folks. Can you tell us a little bit about your background and your family?

Durrah: Sure. I am a patient/family engagement consultant by profession, which basically means that I work with healthcare organizations and systems around the concept of patient/family engagement, spanning anywhere from patient-centered measurements to quality improvement, and so on. I came to the work, though, not as part of a plan, but by fate and destiny. My firstborn, whose name is Ayah, was born with a very rare liver disease which we knew would require liver transplantation. She is 16 years old now, but as you can imagine, we've been on quite a journey in the healthcare system. In addition, I have four other children and my husband is an adult medical hospitalist at a major academic center on the East Coast, so healthcare is a big piece of our lives.

I realized a few years into the journey with my daughter that I wanted to become more engaged in improving healthcare and partnering with our care teamthe physicians, nurses, pharmacists, and the larger group of specialists that she was seeing on a regular basis. I had the opportunity to volunteer at a local hospital that was looking for more patient/family involvement in some of their quality improvement work, and I decided that this was definitely something I wanted to pursue further, so I did. But my daughter's healthcare journey continues and I continue to see the good, the not so good, and everything in between.

In addition, I think I have a unique perspective because my husband is a physician and works in the hospital setting. I understand and can empathize with the real challenges facing physicians that are constantly placing pressure on them and demanding their time. I am very cognizant of that, so in my work I really try to balance the voice of the patient or caregiver, and the voice of the physician or nurse, because I can definitely see all of the different perspectives. At the end of the day, I think we all really want these meaningful relationships where we communicate and empathize with one another, and at the same time, we all want the best care for each other. That is my goal and the work I've been doing for the past several years.

Topol: That is terrific. You have a panoramic view of the healthcare landscapethat's for sure.

Topol: You wrote about your daughter; it is a miracle story in so many respects. Her name, Ayah, means "sign of God"is that right?

Durrah: Yes. We named her that before I knew she was going to be born with this illness, and it's pretty remarkable that she fit that name and the meaning of her name since she was born. Her first liver transplant, unfortunately, failed within the first 24 hours. As you all know, a liver does not wait, so we were preparing to say goodbye to her when she was about 5 years old.

Subsequently, by miracle, she got another liver 2 days later. It was from the same hospital where she was at, which was even more amazing because her surgeons were prepared to travel anywhere to get another liver. She was placed number one on the nation live listing after that first transplant failed, and it's remarkable that she got another one.

Topol: After she had these two liver transplants at age 5, she got Burkitt lymphoma.

Durrah: Unfortunately, because of her therapies, and also being Epstein-Barr viruspositive, she developed stage III Burkitt lymphoma 2 years after the liver transplant, which was quite devastating, to say the least.

Topol: It required another type of transplant, a bone marrow transplant.

Durrah: Yes. Unfortunately, she had several months of a pretty intense chemotherapy regimen that ultimately failed, and we had to move to an autologous bone marrow transplant. She was not able to have donor cells because she was already an organ transplant recipient. She sat one day in the hospital with a catheter, and they took her stem cells and then gave them back to her about a month later after some more intense chemotherapy.

In all our experiences, I don't believe that physicians and nurses ever defined my daughter as a consumer.

Obviously, she has been through quite a bit. A lot goes along with being a patient of liver disease or a patient who survived cancer with all of the chemotherapy, so while her health is stable, things are constantly moving in the background that we're watching. I tell people that I sometimes look at it like a radar screen. You kind of see the little red dots bleeping and some of them get brighter and some of them get dimmer. A lot of people are monitoring those lights, but I'm the chief monitor of all of those lights and the coordinator of making sure we stay on top of them.

Topol: You are quite an advocate and it is just an amazing story. I want to get into this message that you have about using the term "consumer," because I have hated that term for years. Why do we use this term when we're talking about patients in the health world? You wrote, "I share our story because I am becoming increasingly troubled by a trend in healthcaretoward thinking of patients as 'consumers' but not actually engaging communities in healthcare improvement and innovation." Tell us a bit about this objection of this term, because I think if anybody has a right to object to the use of that term, it would be you.

Durrah: The term definitely does not resonate with me, and I share your strong feelings against the utilization of this term. Overall, I think it continues to underline and push the business imperative of healthcare versus the humanity imperative in which healthcare was initially built upon. I reject in some ways that this notion of consumerism will improve healthcare because healthcare did not start as a business imperative, nor was the framework built to support such a term.

In all of our experiences, I don't believe that physicians and nurses ever defined my daughter as a consumer. I believe [the term] distances us from one another and does not amplify or support that relationship-based care that we all seek to have and to improve. I also don't think "consumer" empowers us, although I understand why some believe it may, because the framework of healthcare is not set up in such a way where "consumer" denotes choice. A lot of healthcare is dictated to the patient or to the caregivers by their insurance companies or their lack thereof, or the communities in which they live, or by lots of different other parties that are involved in it. I'm not quite sure where the choice comes in, especially because no one chooses to be sick. A consumer has choices, but we don't have that choice.

It does not make sense to use that term. I understand where people come from wanting to empower individuals by changing the terminology [to a less] "passive" role of the patient, but I'm not quite sure that changing the terminology we use makes us equals, gives us any more choices, or improves the care we will receive.

Topol: Words are important. This term is hackneyed, it's pervasive, and I think you made the most eloquent case in the history of the medical literature because your intersection with the healthcare world is extensive.

Durrah: Oh my gosh. I'm humbled by that. I'm not sure if I deserve all that, but I appreciate it.

Topol: You have a master's degree from George Washington and you worked in tourism, and you made some interesting parallels between a consumer of tourism versus a patient. Can you talk to us about that?

Durrah: Sure. My master's is in tourism and administration, with a focus in meeting and event management, and here I am in healthcare advocacy now. But I found that it's been very useful because you do see in healthcare this shift of trying to create a tourism-type experience for healthcare. And generally speaking, tourism is a choice; we all choose to travel or to use a certain hotel or rental car company because we've had experiences. Most of the time, you come back from a vacation with great memories, and when you share those memories with others, they may want to have the same experiences as you.

But I don't believe that we can really equate healthcare to a vacation. I'm not quite sure who would utilize that same experience as a vacation. Things that healthcare has that no other industry has are the trauma, pain, and anguish that go into being either a patient or the caregiver of someone. I think physicians and nurses have a lot of emotions in the healthcare experience that you could never find in tourism. And tourism tries to have a constant feedback loop with their customers or consumers; healthcare does not. Yes, we tout these wonderful surveys that are supposed to increase patient satisfaction, but were any of them co-designed with the communities the health systems serve? The answer is no. Do they allow you to really respond in such a way that will provide meaningful data using stories of your experiences in the system? No. So again, trying to equate the healthcare experience with the lessons learned from tourism is a mismatch.

Topol: Right. Even that term "medical tourism" is about a different concept.

There are many parts of your daughter's story that were really touching and deep, but one of the things that stood out was how her liver doctor would hold your hand, and how that was not about consumerism but, as you say, a choice driven by humanity and compassion. This, I think, speaks to the stark dramatic differences between the terms.

Topol: Another term I'm interested in asking you about is "providers." It's another term I don't care for. Should that term be used?

Durrah: It's interesting, because I believe that in the article I do reference "care providers" at one point. Initially when I started in this work, I always [used the specific terms of] "physicians," "nurses," "pharmacists," and so on. All of a sudden that term "provider" got thrown into the mix, and I adapted it because it seemed that everyone was using it to cover all the members of the care team that might interact or touch a patient. I'm guilty of using that term and I got a lot of response after my article came out, particularly from physicians, about how the term "provider" was not liked. I believe the term came about with this whole business imperative/push in healthcare. Insurance companies use that term and healthcare systems have now adopted that term as well, and it probably is in play because of this whole consumerism push as well.

I did prefer saying "physicians" or "nurses" or "pharmacists" and that we're all part of the "care team." I prefer that terminology, just as I prefer to be called a "partner" in my care versus a "consumer" or a "customer." I think that the term does not fit the role that physicians and nurses play. They are not just a provider. I look at them as part of my team and I'm on their team, so we're all working together. I think "provider" also sounds a little bit like a hierarchy. It does not suggest that notion of that partnership and just reinforces a distance. Consumerism is kind of defined by this transactional relationship, so you would use that term to reinforce that. I see how it has developed and how it's being pushed, and I think it's all related to the business of medicine versus the actual human experience and the humanity of medicine.

We so largely lost the 'care' in 'healthcare.

Topol: These terms got adopted when healthcare was transformed to a business, and now we're relooking at this.

Your work and eloquence and what you continue to do as an advocate in patient care will help us get back on track. It's just one of many things we need to do.

Topol: You also brought up the term "lean principles." It would be interesting for you to just touch on this as well.

Durrah: I've noticed in my work in healthcare thus far that health systems are adopting a lot of frameworks. One of these is becoming "lean" or utilizing "lean principles," which are driven by efficiency and cost savings.

Typically, those who advocate for lean principles within health systems say, "This is going to benefit everyone: physicians, nurses, patients and families, and the communities we serve." But if you really dig down deep, I don't think it has trickled down at all to any cost savings for patients or families. I don't think it's improved quality for communities which the healthcare system serves. And I certainly don't see physicians and nurses not being more burned out or more extended. They get affected by lean principles, where staffing is cut in order for that cost savings to occur. They are being told to do 150 things versus 100 things a day.

When I first learned about "lean" and read about it, I thought it sounded great. But then when I got deeper into this work, I thought, "Wait a minutewho is this benefiting other than the bottom line of healthcare systems?" It's really interesting how healthcare tends to cherry-pick principles or terminology from other industries with the goal of trying to improve healthcare, when it's really not doing that. It's simply putting a Band-Aid on one problem and creating a new one.

Topol: We so largely lost the "care" in "healthcare." You referred to the "care team." You undoubtedly experienced that with your daughter, and we want to bring it back. We should be more precise about language and avoid business terms, and talk about clinicians as doctors, nurses, pharmacists, physical therapists, or whoever. We should be more specific than using this "provider" term which obviously could be a relative or a friend.

Topol: What response did you receive from the essay you wrote in Health Affairs? It clearly resonated, and when I posted it on Twitter, there was quite a bit of response.

Durrah: I got an incredible response from the article, and I appreciate you tweeting and retweeting it because a lot of people saw that and reached out to me and started retweeting as well. A number of people sent me personal emails and messages, and I really was humbled by the response. I was quite nervous to write the article I did because in my work, there is kind of a fine line that you can only push the boundary so far, and then if you push them too far you get a lot of pushback. I sometimes feel like I'm this one little patient advocate voice among all these other voices. The article really resonated with physicians in particular, and they really understood the story which I shared about my daughter's liver doctor.

When we found out that she was diagnosed with cancer, she asked to sit in the meeting with the oncologists. She didn't have to do that, but she wanted to. I said, "Of course you can join us." She sat next to me and held my hand the entire meeting. Every time they said something distressing, she would just squeeze my hand, but she didn't utter a word. I think that encapsulates the relationship that patients and caregivers have with their physician or nurse. It's such a special bond, and the term "consumer" could never define that particular bond; she would never look at this as a transactional relationship. Would "consumer" teach her to do that? No. As you said before, it is the humanity and compassion of medicine, and that is why most everyone who comes into the profession really wants to help humanity and give that empathy and compassion.

We have a lot to learn, and it's going to take many voices to speak up and push back against consumerism because some pretty large organizations and groups are trying to push consumerism and continue this business imperative of healthcare that is motivated by bottom lines. I think we're going to get there. Maybe we're already there. We're at a tipping point. There is so much dissatisfaction, and so many parts of the system are broken. To say that consumerism will give you more choices, shorter wait times, and maybe even price transparency, even though you still have to deal with your insurance provider or lack thereof, makes me giggle because I don't think that is going to be the solution.

The only solution I see is that we have to partner with one another and co-create solutions to improve healthcare as teamsequal voices at the tableand begin this pushback. If we don't, I fear what is ahead because I've already had a glimpse of it. I'm nervous because as my daughter slowly moves into adulthood, and she is almost there, these were the things I hoped we would have conquered before she got to that point. A lot of this is pushing us away from one another and distancing ourselves from one another, and that is not going to change healthcare.

Topol: You said it so well. I've been eager to meet you since I read your work months ago, and I wanted the whole Medscape audience to get to know you and your story. Most of them are not on Twitter and most don't read Health Affairs. Your story is so darn important, and you convey it in a powerful way.

I'm so glad to know that your daughter is okay, having gone through 16 years of rough times, especially in her earlier years. I just want you to carry on. You are an important voice. You may qualify yourself as only one, but it's a powerful one, and very few people have had an experience like yours. You're in a rarified group and can transmit the emotion and the sense of caring. Let's get these words right, and let's zap "consumer" from this story of future healthcare.

Thanks so much for joining us today on Medscape, and we will look forward to following you and learning more from you in the future.

Durrah: Thank you. I look forward to partnering with your audience on improving healthcare.

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Healthcare Terms Are Out of Touch, Says Mom Advocate - Medscape

A stop-motion movie of the whole brain, starring new neurons – Spectrum

Cells on the move: A new technique helps reveal patterns in the migration of neurons (green) to their destination (red).

A new method visualizes nascent neurons in the mouse brain and follows them as they migrate to their final destinations.

The method essentially a time series of 3D images may shed light on processes that unfold during brain development.

We take snapshots at different time points, then match them together to see dynamic processes, says Alexander Lazutkin, senior scientist at the P.K. Anokhin Institute of Normal Physiology in Moscow, Russia. Lazutkin presented the work yesterday at the 2019 Society for Neuroscience annual meeting in Chicago, Illinois.

Researchers can use the method to study the birth of neurons and the complex ways in which they migrate through the brain. Abnormalities in these processes have been implicated in autism.

In the weeks after a mouse is born, neural stem cells in its brain continue to divide and generate neurons. Lazutkins team marked these dividing cells throughout the brain by adapting a fluorescent labeling technique typically used on thin sections of tissue. (The labeling method was published 12 September in MethodsX1.)

In the new work, they labeled cells in the brains of adult mice. They used a fluorescent microscope to take a snapshot of the whole brain of one set of animals, and then a different set of animals one day older the next day, and so on. They built an algorithm to stitch the images together and fill in the gaps to form a time series.

The result is a movie that tracks cells locations as they migrate toward their destination. The cells originate in the subventricular zone, one of two places where cells continue to divide in the adult brain. The movie shows the migrating cells reaching the olfactory bulb in about 10 days.

It also shows cells forming a stripe pattern in the subventricular zone.

We suppose these are distinct migration streams, Lazutkin says. You cant see this pattern on a conventional [2D] slide. But when you have the full picture, you can recognize them.

Lazutkin and his colleagues plan to use the method to look at differences in early brain development between typical mice and mouse models of autism particularly those with mutations that lead to altered brain growth.

For more reports from the 2019 Society for Neuroscience annual meeting, please click here.

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A stop-motion movie of the whole brain, starring new neurons - Spectrum

Humans evolved to think faster by slowing down brain development – New Scientist News

By Clare Wilson

D. ROBERTS/SCIENCE PHOTO LIBRARY

How did humans get to be so much cleverer than other apes? One counter-intuitive idea is that it was made possible by a slowdown of our brain growth during fetal development.

The suggestion comes from a relatively new approach of growing embryonic-like stem cells in a dish and coaxing them to turn into nervous system cells until they form pea-sized three-dimensional clumps known as organoids.

These mini brains seem to replicate real neuron behaviour more closely than when brain cells are grown in a flat layer. They have nothing like the complexity needed for thought or consciousness, but do develop into different kinds of brain tissue and display patterns of electrical activity that have some similarities with real brains.

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Human brains are certainly bigger than those of our nearest primate relatives, but there are surprisingly few differences in structure. So it is unclear what gives rise to the huge differences in our mental abilities.

Gray Camp at the Max Planck Institute for Evolutionary Anthropology in Leipzig, Germany, and his colleagues used stem cells from humans, chimpanzees and macaque monkeys to make mini brains for each species. After four months, a key difference was that nerve cells in the chimp and monkey organoids were more mature.

Identifying such differences may be a step towards explaining why humans are more intelligent although the team doesnt speculate on exactly how their findings might relate to this puzzle.

Until now it wasnt possible to compare human and chimp organ development, says Camp. The organoids, some made from stem cells that can be generated directly from adult cells, offer a way of making that comparison.

Camp and his team also delved into another long-standing puzzle: why there are so few differences between the protein-coding genes of humans and the other apes, considering the huge disparity in our intellects.

A recent technique for analysing which genes are turned on or off in individual cells, known as single cell RNA sequencing, has suggested the answer might lie in differences in which genes are turned on at different times.

In the latest study, Camps team charted which genes are turned on in different brain cells over four months of mini brain development, comparing the results across humans, chimps and macaques, to make a database other researchers can also use.

Organoids are most useful for society in letting us understand disease, says Camp. But its also very interesting to think about where our species came from and how we became uniquely human.

Journal reference: Nature, DOI: 10.1038/s41586-019-1654-9

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Humans evolved to think faster by slowing down brain development - New Scientist News