Category Archives: Cell Therapy

Masayo Takahashi (second from left) treated macular degeneration with retinal tissue grown from iPS cells.

Kyodo News/Contributor/getty images

By Dennis NormileMar. 15, 2017 , 5:00 PM

Its official: The first use of induced pluripotent stem (iPS) cells in a human has proved safe, if not clearly effective. Japanese researchers reported in this weeks issue of The New England Journal of Medicine (NEJM) that using the cells to replace eye tissue damaged by age-related macular degeneration (AMD) did not improve a patients vision, but did halt disease progression. They had described the outcome at conferences, but publication of the details is an encouraging milestone for other groups gearing up to treat diseased or damaged organs with the versatile replacement cells, which are derived from mature tissues.

This initial success is pretty momentous, says Alan Trounson, a stem cell scientist at the Hudson Institute of Medical Research in Melbourne, Australia. But the broader picture for iPS therapies is mixed, as researchers have retreated from their initial hopes of creating custommade stem cells from each patients tissue. That strategy might have ensured that recipients immune systems would accept the new cells. But it proved too slow and expensive, says Shinya Yamanaka of Kyoto University in Japan, who first discovered how to create iPS cells and is a co-author of the NEJM paper. He and others are now developing banks of premade donor cells. Using stocks of cells, we can proceed much more quickly and cost effectively, he says.

Even so, clinical work is progressing more quickly than I had expected, says Yamanaka, who did his groundbreaking work just a decade ago. His collaborator on this trial, Masayo Takahashi of the RIKEN Center for Developmental Biology in Kobe, Japan, had a head start. An ophthalmologist, Takahashi was familiar with the ravages of AMD, a condition that progressively damages the macula, the central part of the retina, and is the leading cause of blindness in the elderly.

Takahashi started investigating treatments for AMD in 2000, a time when the only cells capable of developing into all the tissues of the body had to be extracted from embryos. But she was stymied by immune reactions to these embryonic stem (ES) cells. When Yamanaka announced that he could induce mature, or somatic cells, to return to an ES celllike state, Takahashi quickly changed course to develop a treatment based on iPS cells.

Her team finally operated on the first patient, a 77-year-old Japanese woman with late-stage AMD, in September 2014. They took a sample of her own skin cells, derived iPS cells, and differentiated them into the kind of retinal cells destroyed by the disease. A surgeon then slipped a small sheet of the cells into the retina of her right eye.

An operation on a second patient was called off because a number of minor genetic mutations had crept into his iPS cells during processing, and uncontrolled growthcancerhas been a worry with such cells. These changes do not directly induce cancer, but we wanted to make safety the first priority, Yamanaka says. Also, Takahashi says, AMD drugs had stabilized the patients condition so there was no urgency in subjecting him to the risks of surgery, which include hemorrhaging and retinal damage.

Immediately after surgery the first patient reported her eyesight was brighter. Takahashi says the surgery halted further deterioration of her eye, even without the drug injections still being used to treat her other eye, and there were no signs of rejection of the graft as of last December.

Clinical work is progressing much more quickly than I expected.

The result is a proof of principle that iPS cellbased therapy is feasible, says Kapil Bharti, a molecular cell biologist at the U.S. National Institutes of Healths National Eye Institute in Bethesda, Maryland, who is also developing iPS cells for treating AMD. Takahashi says once her team gains more experience with the technique they will extend it to patients with earlier-stage AMD in an effort to preserve vision.

Last month, Takahashi won approval to try the procedure on another five patients with late-stage AMD. But this time, instead of using iPS cells derived from each patient, the team will draw on banked cells from a single donor. It takes time to create iPS cells, and a lot of time for the safety evaluation, Yamanaka says. It is also costly, at nearly $900,000 to develop and test the iPS cells for the first trial, Takahashi adds.

Using donor cells to create the iPS cells will make it more difficult to ensure immune compatibility. But Yamanaka says that donor iPS cells can be matched to patients based on human leukocyte antigen (HLA) haplotypessets of cell-surface proteins that regulate immune reactions. HLA-matched cells should require only small doses of immunosuppressive drugs to prevent rejection, Takahashi saysand perhaps none at all for transplantation into the immune-privileged eye.

Kyoto Universitys Center for iPS Cell Research and Application, which Yamanaka heads, has been developing an iPS cell bank. Just 75 iPS cell lines will cover 80% of the Japanese population through HLA matching, he says. Trounson, a past president of the California Institute for Regenerative Medicine, a stem cell funding agency, says banked iPS cells have advantages. Donor iPS cells may be safer than cells derived from older patients, whose somatic cells may harbor mutations. And Jordan Lancaster, a physiologist at the University of Arizona in Tucson, likes the speed of the approach. He is devising patches for heart failure patients based on iPS-derived myocardial cells that will be premanufactured, cryopreserved, and ready to use at a moments notice.

Patient-specific iPS cells will still have clinical uses. For one thing, Bharti says it will be difficult for cell banks to cover all HLA haplotypes. And a patients own iPS cells could be used to screen for adverse drug reactions, says Min-Han Tan, an oncologist at Singapores Institute of Bioengineering and Nanotechnology, who recently published a report on the approach.

Other human trials are not far behind. Yamanaka says his Kyoto University colleague Jun Takahashi (Masayo Takahashis husband) will launch trials of iPS-derived cells to treat Parkinsons disease within 2 years. Bharti hopes to start human trials of iPS cells for a different type of macular degeneration next year. And as techniques for making and growing iPS cells improve, researchers can contemplate treatments requiring not just 100,000 cells or sothe number in Takahashis retinal sheetsbut millions, as in Lancasters heart patches.

As clinical use approaches, Takahashi cautions that researchers need to keep public expectations realistic. For now, iPS treatments may help but wont fully reverse disease, she says. Regenerative medicine is not going to cure patients in the way they hope.

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Cutting-edge stem cell therapy proves safe, but will it ever be effective? - Science Magazine

New treatments and advances in research are giving new hope to people affected by Osteoarthritis pain and symptoms. Dr. Theofilos provides stem cell therapy for osteoarthritis to help those achieve better health and live life in motion.

Stem cell therapy for osteoarthritis is being studied for efficacy in improving the complications in patients through the use of their own stem cells. These procedures may help patients who dont respond to typical drug treatment, want to reduce their reliance on medication, or are looking to try stem cell therapy due to pain or discomfort.

Osteoarthritis, also known as degenerative arthritis or degenerative joint disease, is a group of mechanical abnormalities involving degradation of joints, including articular cartilage and subchondral bone. Symptoms may include joint pain, tenderness, stiffness, locking and sometimes an effusion. When bone surfaces become less well protected by cartilage, bone may be exposed and damaged. As a result of decreased movement secondary to pain, regional muscles may atrophy, and ligaments may be affected.

Stem cell treatment is designed to target these areas within the joints to help with the creation of new cartilage cells. Mesenchymal stem cells are multipotent and have the ability to differentiate into cartilage called (chondrytes). The goal of each stem cell treatment is to inject the stem cells into the joint to create cartilage.

Its expected that results of the therapy will vary depending upon the many factors of the severity, patients overall health, nutritional state and immune function. Stem cell therapy is safe and effective in reducing pain and improving function for many patients.

Voted as one of America's Top Surgeons, Charles S. Theofilos, MD, Neurosurgeon and Founder of The Spine Center is a leading provider of the state-of-the-art, most comfortable and effective surgical, minimally invasive and non-surgical treatment options for a full range of cervical and spinal ailments, including stem cell therapy and artificial disc replacement. He was among a field of 20 top neuro and orthopedic surgeons in the U.S. chosen to participate in the groundbreaking Artificial Disc Study, which compared the clinical outcome of disc replacement versus traditional spinal fusion. A widely sought after educator and lecturer, Dr. Theofilos has offices in Palm Beach Gardens and Port St. Lucie.

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11621 Kew Gardens Ave., Suite 101; Palm Beach Gardens

*In an effort to maintain and honor the commitment to our patients, we will continue to accept Medicare and Medicare Advantage insurance plans for all new and follow up appointments.

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Stem cell therapy helps patients with osteoarthritis - Palm Beach Post - Palm Beach Post

SALT LAKE CITY Relief for chronic back and neck pain may be on the horizon, thanks to emerging science technology under development at the University of Utah.

Bioengineering researchers have discovered a technique to control chronic pain by regulating genes that reduce tissue- and cell-damaging inflammation.

This has applications for many inflammatory-driven diseases, said assistant professor Robby Bowles, who led the research. It could be applied for arthritis or to therapeutic cells that are being delivered to inflammatory environments that need to be protected from inflammation.

In laymens terms, the therapy has the potential to treat chronic pain by relieving swelling in affected areas and healing the tissue, he said.

For instance, chronic pain in slipped or herniated discs result from damaged tissue when swelling causes cells to create molecules that break down tissue, he explained. Inflammation is natures way of alerting the immune system to repair tissue or fight infection, but chronic inflammation can lead to tissue degeneration and pain, he said.

Bowles team uses the Clustered Regularly Interspaced Short Palindromic Repeat system known as CRISPR a new technology that modifies human genetics to halt cell death and keep cells from producing molecules that damage tissue and result in chronic pain, he said.

This is something that could be injected into your (damaged) discs to stop the signaling that is driving disc degeneration and the painful signaling, Bowles said. It would keep you from getting worse and it would stop the pain.

But Bowles said the therapy does not edit or replace genes, which is what CRISPR tools are typically used for. Instead, the therapy modulates the way genes turn on and off in order to protect cells from inflammation.

So they wont respond to inflammation. It disrupts this chronic inflammation pattern that leads to tissue degeneration and pain, he said. Were not changing what is in your genetic code. Were altering what is expressed. Normally, cells do this themselves, but we are taking engineering control over these cells to tell them what to turn on and turn off.

He said now that researchers know they can do this, doctors would be able to modify the genes using direct injection into the affected area which delays tissue degeneration. In the case of back pain, a patient may get a discectomy to remove part of a herniated disc to relieve the pain, but tissue near the spinal cord may continue to break down, leading to future pain, he said. This method could stave off additional surgeries by stopping the tissue damage, he noted.

The hope is that this stops degeneration in its tracks, and the patient could avoid any future surgeries, Bowles said. But its patient to patient. Some might still need surgery, but it could delay it.

So far, the team has developed a virus that can deliver the gene therapy and has filed for a patent on the system with the hope of moving to human trials after collecting initial data. One caveat Bowles noted was that there are currently no gene therapies approved for use by the U.S. Food and Drug Administration, so it may take some time to receive necessary acceptance.

So long term there are technological and regulatory hurdles to (overcome), he said. It could be about 10 years before this method is ready for use in patients.

Despite the regulatory issues, Bowles was optimistic about the long-range prospects for treating pain using this new therapy.

The CRISPR systems give us control that would allow us to begin treating these diseases in ways we couldnt treat them before, Bowles said. Over the next 10 to 15 years, were going to see a lot of these CRISPR technologies change these debilitating conditions.

The teams discovery was published in a new paper this month in a special issue of Tissue Engineering. The study was co-authored by University of Utah bioengineering doctoral student Niloofar Farhang and several other researchers in a collaborative project between the U., Duke University and Washington University in St. Louis.

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U. scientists develop cell therapy for chronic disc pain - Deseret News

I just read a concerning paper about an experimental stem cell treatment for children with autism.

The authors are Himanshu Bansal and colleagues of India. The senior author, Prasad S Koka, is the Editor-in-Chief of the Journal of Stem Cells where the paper appeared, which raises questions about whether the manuscript received a thorough peer review. Koka is actually an author on all five of the research papers published in that issue of the journal. But thats a minor issue compared to the content of the paper.

Bansal et al. describe a procedure in which they extracted fluid from the bone marrow of each child. This fluid (bone marrow aspirate) was treated in the laboratory to purify the stem cells within, and then injected into the childs spinal canal. The whole operation took place under general anaesthesia. 10 autistic children aged 4-12 were treated.

I found this pretty shocking. An invasive procedure involving general anaesthesia should only be performed if its medically justified especially in children as young as 4! Bansal et al. provide no scientific explanation for why they thought this treatment was suitable for these patients. They vaguely name immunological and neural dysregulation believed to underlie autism as the target of the cells.

For what its worth, the results showed a slight improvement in autism symptoms after the treatment. However, there was no control group, so placebo effects are likely, and were told that the patients were also given speech therapy, occupational therapy and psychological intervention which might account for the benefits.

So who gave the green light to this project? Well, remarkably, Bansal et al.s paper contains no information about which ethics committee reviewed and approved the study. I dont know about the laws in India, but in the UK or the USA, conducting even the most benign research without the proper ethical approval is serious misconduct. Most journals absolutely wont publish medical research without an ethics statement.

The paper also contains no mention of conflicts of interest another thing that most medical journals require. I believe that financial conflicts of interest are likely to exist in this case because Bansal gives his affiliations as Mother Cell, his own private venture, and RegennMed, who sell various stem cell treatments.

Overall, to say that this paper is ethically questionable is an understatement, and it would have been rejected by any real journal.

This isnt Dr Himanshu Bansals first foray into the amazing world of dodgy stem-cells. He briefly made headlines around the globe last year when he announced his ReAnima project to bring a brain dead woman back to life (with stem cells). Indian authorities eventually blocked his resurrection attempt. Theres some more interesting dirt on Bansal on this forum.

This is also not the worlds first stem cells for autism trial. For example, Duke University launched a $40 million trial in 2014. The treatment in that trial was a blood infusion, so it was pretty non-invasive: no bone marrow, spinal needles, or general anaesthesia. However, critics argue that its pure speculation to think that stem cells would help in autism. Then again, the same could be said about a great many stem cell therapies.

Bansal H, Verma P, Agrawal A, Leon J, Sundell IB, Koka PS. (2016). A Short Study Report on Bone Marrow Aspirate Concentrate Cell Therapy in Ten South Asian Indian Patients with Autism Journal of Stem Cells, 11 (1)

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Unethical Stem Cell Therapy for Autism In India? - Discover Magazine (blog)

American Academy of Ophthalmology Statement on Stem Cell Therapy for Treating Eye Disease PR Newswire (press release) The Academy has previously issued clinical guidance that covers the appropriate use of stem cell therapy in eye care. These recent cases confirm the Academy's belief that the FDA should thoroughly investigate and level regulatory action against ...

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American Academy of Ophthalmology Statement on Stem Cell Therapy for Treating Eye Disease - PR Newswire (press release)

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3 Women Blinded After Stem Cell Therapy Newser CORRECTS FROM MD ANDERSON HOSPITAL TO MD ANDERSON CANCER CENTER -Senior Clinical Cell Therapy Specialist Megan Raggio prepares stem cells from bone marrow before they are transplanted into sportscaster... (AP Photo/David J. Phillip). Stem Cell Therapy Blinds Three Patients at Florida ClinicMedscape Cutting-edge stem cell therapy proves safe, but will it ever be effective?Science Magazine Unproven stem cell 'therapy' blinds three patients at Florida clinicStanford Medical Center Report NPR -New Vision -CNN -The New England Journal of Medicine all 131 news articles »

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3 Women Blinded After Stem Cell Therapy - Newser

David Sourdive, co-founder of Cellectis, discusses his pioneering work developing off-the-shelf CAR-T for cancer and the future directions of cell therapy.

Last week I had the opportunity to attend the World ADOPT Summit and hear from some of the world leaders in cell therapy. There I met David Sourdive, co-founder and VP of Cellectis, a French company that is leading the development of the first off-the-shelf CAR-T therapy.

CAR-T therapy consists in the infusion of T-cells engineered to better identify and kill cancer cells. The most advanced approaches, which could be approved as soon as by the end of this year, use an autologous approach by engineering cells from the own patient.Cellectis is taking the technology a step further and developing an allogeneic CAR-T therapy that uses cells from healthy donors instead.

Cellectis uses TALEN gene editing to develop off-the-shelf products that could make this miracle cancer therapy faster, cheaper and accessible to a wider patient population. Its first therapy, UCART19, now licensed to Servier and Pfizer, has shown striking results in two babies with leukemia. A second one, UCART123, recently received approval from the FDA to start clinical trials.

With his company positioned at the forefront of innovation in cell therapy, I decided to ask David Sourdive how he thinks new technologies will change the scene.

Cell therapy is now transitioning from the world of grafts, where it has been confined for decades, to the world of products. This happened with antibodies. People used to give their blood and antibodies. Then it became possible to manufacture antibodies from cells in an industrial setting, and then it became a product. I think that were going in that direction with cell therapy.

In the coming decade, off-the-shelf cell therapy will become a reality. That is a revolution and is going to change a lot of things. We will need to establish standards and regulations.Cells usually have not been regarded as products, so its a big leap. We have to discuss with regulators how to define the products.

The immune system is going to start being tweaked like it has never been and we will be able to take advantage of it to address very difficult diseases. For example, solid tumors.We think UCARTs can also target solid tumors. To that end, we work with two pharma partners that have strong capability to tackle these complicated tumors.

We are the first ones to do [allogeneic CAR-Ts] and theres no precedent. We have to invent everything. We have to figure out all the possibilities, all the solutions, and theres no real way to predict how things are going to behave in patients because historically, the one kind of cell nobody has ever used is an allogeneic unmatched situation is T-cells, by definition.

Its very exciting because every week we have to do something for the first time, its challenging.I think this year is the year when we will know better how allogeneic products behave in patients.

We have shown that gene editing can really be transformative in the CAR-T space. With gene editing, you can make the cells overcome the defense mechanisms of tumors. You can, for example, knock out genes such as PD1 so that CAR T-cells become capable of killing PDL1-bearing tumors.

Tumors that succeed in the body are tumors that have found a way to evade the immune system.You need to put additional features to empower the T-cells to do what normal T-cells fail to do. People will realize the transformative power of gene editing. We think it will take over the industry.

I think globally, gene editing is going to be a strong power because we are going to be able to tweak cells like weve never done before.We will be able to program cells to deliver a treatment locally. This is happening. Its not futuristic. Its now.

This is definitely going to be an exciting year, with the first CAR-T therapies approaching market approval and the first results from allogeneic CAR-Ts due soon. Keep tuned for the latest updates!

Images from Cellectis; Tyler Olson, ImageFlow /Shutterstock

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In the Coming Decade, Off-the-Shelf Cell Therapy will Become a Reality - Labiotech.eu (blog)

Scientists have long hoped that stem cells might have the power to treat diseases. But it's always been clear that they could be dangerous too, especially if they're not used carefully.

Now a pair of papers published Wednesday in the New England Journal of Medicine is underscoring both the promise and the peril of using stem cells for therapy.

In one report, researchers document the cases of three elderly women who were blinded after getting stem cells derived from fat tissue at a for-profit clinic in Florida. The treatment was marketed as a treatment for macular degeneration, the most common cause of blindness among the elderly. Each woman got cells injected into both eyes.

In a second report, a patient suffering from the same condition had a halt in the inexorable loss of vision patients usually experience, which may or may not have been related to the treatment. That patient got a different kind of stem cell derived from skin cells as part of a carefully designed Japanese study.

The Japanese case marks the first time anyone has given induced pluripotent stem (iPS) cells to a patient to treat any condition.

"These two reports are about as stark a contrast as it gets," says George Q. Daley, Harvard Medical School's dean and a leading stem cell researcher. He wrote an editorial accompanying the two papers. "It's really striking."

The report about the three women in their 70s and 80s who were blinded in Florida is renewing calls for the Food and Drug Administration to crack down on the hundreds of clinics that are selling unproven stem cell treatments for a wide variety of medical conditions, including arthritis, autism and stroke.

"One of the big mysteries about this particular case and the mushrooming stem cell clinic industry more generally is why the FDA has chosen to effectively sit itself out on the sidelines even as this situation overall grows increasingly risky to patients," says Paul Knoepfler, a University of California, Davis, stem cell researcher who has studied the proliferation of stem cell clinics.

"The inaction by the FDA not only puts many patients at serious risk from unproven stem cell offerings, but also it undermines the agency's credibility," Knoepfler wrote in an email.

In response to a query from Shots, an FDA spokeswoman wrote in an email that the agency is in the process of finalizing four new guidelines aimed at clarifying how clinics could use stem cells as treatments. The agency also noted that it had previously issued a warning to patients.

In the meantime, "consumers are encouraged to contact FDA and the appropriate state authorities in their jurisdictions to report any potentially illegal or harmful activity related to stem cell based products," the FDA email says.

Other researchers say the cases should stand as a warning to patients considering unproved stem cell treatments, especially those tried outside carefully designed research studies.

"Patients have to be wary and tell the difference between the snake oil salesmen who are going to exploit them and the kind of slow, painstaking legitimate clinical trials that are also going on," Daley says.

The New England Journal of Medicine report did not name the Florida clinic, but noted that the treatment was listed on a government website that serves as a clearinghouse for research studies. The sponsor is listed as Bioheart, Inc., which is part of U.S. Stem Cell Inc. in Sunrise, Fla.

Kristen Comella, the scientific director of U.S. Stem Cell, would not discuss the cases. "There were legal cases associated with eye patients that were settled under confidentiality, so I am not permitted to speak on any details of those cases due to the confidentiality clause," Comella said by phone.

She acknowledged, however, that the clinic had been performing the stem cell procedures. They were discontinued after at least two patients suffered detached retinas, she says.

But Comella defended the use of stem cells from fat tissue to treat a wide variety of other health problems.

"We have treated more than 7,000 patients and we've have had very few adverse events reported. So the safety track record is very strong," Comella says. "We feel very confident about the procedures that we do, and we've had great success in many different indications."

According to the New England Journal of Medicine report, The Florida clinic was using adult stem cells, which circulate in various parts of the body, including in fat tissue. While those cells may someday be turn out to be useful for treating disease, none have been proven to work.

The body produces a variety of stem cells. The kind that have generated the most excitement and controversy are human embryonic stem cells, which are derived from early human embryos and can be coaxed to become any kind of cell in the body.

Scientists are also excited about iPS cells, which can be made in the laboratory by turning any cell in the body, such as skin cells, into cells that resemble embryonic stem cells.

Those are the cells that were tested by the Japanese scientists. The stem cells were converted into retinal pigment epithelium (RPE) cells, which are the cells that are destroyed by macular degeneration.

"This represents a landmark," says Daley. "It's the first time any patient has been treated with cellular derivatives of iPS cells. So it's definitely a world first."

Daley noted that the scientists only treated one of the patient's eyes in case something went wrong, to ensure remaining vision would not be threatened in the other eye.

After at least a year, no complications had occurred and the patient had not experienced any further deterioration of vision in the treated eye. While that is promising, more patients would have to be treated and followed for much longer to know whether that approach is successful, Daley says.

"Given that macular degeneration is the most frequent cause of vision loss and blindness in the elderly and our population is aging, the prevalence of macular degeneration is going up dramatically," Daley says. "So to be able to preserve or even restore sight would be a really remarkable medical advance."

Despite the potentially encouraging results with the first patient, Daley noted that the Japanese scientists decided not to treat a second patient and suspended the study. That's because they discovered worrisome genetic variations in the RPE cells they had produced for the second patient.

"They weren't certain these would cause problems for the patient, but they were restrained enough and cautious enough that they decided not to go forward," Daley says. "That's what contrasts so markedly with the approach of the second group, who treated the three patients with an unproven stem cell therapy that ended up have devastating effects on their vision."

In this case, the New England Journal of Medicine report says, patients paid $5,000 each to receive injections of solutions that supposedly contained stem cells that were obtained from fat removed from their abdomens through liposuction.

Even though the safety and effectiveness of this procedure is unknown, all three patients received injections in both eyes.

"That's what led to these horrible results," says Thomas Albini, a retina specialist at the University of Florida's Bascom Palmer Eye Institute, who helped write the report.

Before the procedure, all three women still had at least some vision. Afterwards, one woman was left completely blind while the other two were effectively blind, Albini and his colleagues reported.

The cases show that patients need to be warned that something that "sounds too good to be true may indeed be too good to be true and may even be horrible," Albini says.

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3 Women Blinded By Unproven Stem Cell Treatments - NPR

This morning, Australian biotech company Mesoblast Ltd (NASDAQ:MESO) announced encouraging phase 2 trial data regarding its regenerative therapy for chronic lower back pain (CLBP). Mesoblasts proprietary allogeneic Mesenchymal Precursor Cells (MPCs) are given to patients via a single injection and, in the clinical trial, have proved to spark significant improvement in both lower back pain and function that lasted for at least 36 months.

The sustained benefits on pain and function over three years seen with a single injection of Mesoblasts cell therapy have the potential to transform the treatment paradigm for chronic low back pain due to disc degeneration, said Dr. Hyun Bae, the trial investigator and Director of the Spine Institute.

According to Bae, there is growing evidence that disc degeneration can be healed instead of needing to be replaced or fused. Researchers are quickly arriving at this inflection point in the treatment of lower back pain, which is especially critical given the ongoing opioid abuse epidemic sweeping the nation.

Researchers believe that MPCs are turned on by signals in damaged tissues that spark factors to block damaging inflammation and begin repairing the afflicted area.

The phase 2 trial in question involved 100 patients, who were treated with either six million or 18 million MPCs via one intra-discal injection or a placebo of either saline or hyaluronic aid. The primary endpoints were a 50 percent drop in Visual Analog Scale (CAS), which is a pain metric, and a 15-point reduction in Oswestry disability index (ODI) at both the one and two year marks.

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The primary endpoint composite at the two-year mark was met by 41 percent of patients in the 6 million MPC arm, 35 percent in the 18 million arm, with just 18 percent and 13 percent in the placebo groups. The phase 2 results support the companys criteria for its ongoing phase 3 program.

Mesoblast is confident that the durable treatment outcomes in lower back pain are part of an overarching mechanism that might also have a positive effect in other types of chronic conditions where a single injection of MPCs can lead to sustained benefits. Two of these potential indications, according to Mesoblast, are chronic heart failure and biological-resistant rheumatoid arthritis.

Following the trial data being released, Maxim Groups Jason Kolbert reaffirmed a Buy rating on Mesoblasts stock and maintained a price target at $14.00. The upside indicated is 81 percent.

The market opportunity is significant and the treatment fits ideally within the existing paradigm the analyst told investors this morning. Injecting cells versus steroids with a better AE profile and now a better long-term result.

Earlier this month, Mesoblast announced that its MSC-100-IV therapy had been given Fast Track designation by the Food and Drug Administration (FDA) for its use in children with steroid refractory acute Graft Versus Host Disease (aGVHD). This type of designation shortens the FDA review path from ten months to six months, and certain portions of the Biologics License Application (BLA) can be sent in on a rolling basis rather than when everything is finished.

The author of this article holds no position in any of the stocks mentioned above.

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Mesoblast's (MESO) Regenerative Cell Therapy Proves Itself in Back Pain Trial - Economic Calendar

Thinly traded nano cap ReliCel Life Sciences (OTCQB:REPCF -5.1%) slips after it announced data from a five-year Phase 1 study assessing the safety of its autologous cell therapy for the treatment of androgenic alopecia (pattern baldness), RCH-01. The results were fine, but the timeline for the next study has apparently dampened investors' enthusiasm.

The 19-subject trial met its endpoints thereby confirming the complete safety profile of a high dose of dermal sheath cup cells, administered via injection, for patients with pattern baldness.

Although not powered for efficacy, a treatment effect was observed. The seven best responders experienced at least a 10% increase versus baseline in hair density six months after injection. At month 24, the average hair density increase in the same seven subjects was 8.3%. The top responder showed a 21% increase in hair density at month 24.

The company intends to advance RCH-01 into a Phase 2 study in 160 healthy men with mild-to-moderate pattern baldness. Dermal sheath cup cells will be isolated from a small punch biopsy taken from the back of the subject's scalp. The cells will be replicated and then reintroduced into balding areas. Participants will remain on study for ~39 months.

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RepliCel's autologous cell therapy candidate to treat hair loss safe in long-term study; shares ease 5% on long ... - Seeking Alpha