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Thursday, June 27, 2019
Functional hair follicles grown from stem cells
Scientists from Sanford Burnham Prebys have created natural-looking hair that grows through the skin using human induced pluripotent stem cells (iPSCs), a major scientific achievement that could revolutionize the hair growth industry. The findings were presented today at the annual meeting of the International Society for Stem Cell Research (ISSCR) and received a Merit Award. A newly formed company, Stemson Therapeutics, has licensed the technology.
More than 80 million men, women and children in the United States experience hair loss. Genetics, aging, childbirth, cancer treatment, burn injuries and medical disorders such as alopecia can cause the condition. Hair loss is often associated with emotional distress that can reduce quality of life and lead to anxiety and depression.
“Our new protocol described today overcomes key technological challenges that kept our discovery from real-world use,” says Alexey Terskikh, Ph.D., an associate professor in Sanford Burnham Prebys’ Development, Aging and Regeneration Program and the co-founder and chief scientific officer of Stemson Therapeutics. “Now we have a robust, highly controlled method for generating natural-looking hair that grows through the skin using an unlimited source of human iPSC-derived dermal papilla cells. This is a critical breakthrough in the development of cell-based hair-loss therapies and the regenerative medicine field.”
Terskikh studies a type of cell called dermal papilla. Residing inside the hair follicle, these cells control hair growth, including hair thickness, length and growth cycle. In 2015, Terskikh successfully grew hair underneath mouse skin (subcutaneous) by creating dermal papilla derived from human pluripotent stem cells—a tantalizing but uncontrolled process that required further refinement.
“Our new protocol described today overcomes key technological challenges that kept our discovery from real-world use,” says Terskikh. “Now we have a robust, highly controlled method for generating natural-looking hair that grows through the skin using an unlimited source of human iPSC-derived dermal papilla cells. This is a critical breakthrough in the development of cell-based hair-loss therapies and the regenerative medicine field.”
The approach detailed in the ISSCR presentation, which was delivered by lead researcher Antonella Pinto, Ph.D., a postdoctoral researcher in the Terskikh lab, features a 3-D biodegradable scaffold made from the same material as dissolvable stitches. The scaffold controls the direction of hair growth and helps the stem cells integrate into the skin, a naturally tough barrier. The current protocol relies on mouse epithelial cells combined with human dermal papilla cells. The experiments were conducted in immunodeficient nude mice, which lack body hair.
The derivation of the epithelial part of a hair follicle from human iPSCs is currently underway in the Terskikh lab. Combined human iPSC-derived epithelial and dermal papilla cells will enable the generation of entirely human hair follicles, ready for allogenic transplantation in humans. Distinct from any other approaches to hair follicle regeneration, human iPSCs provide an unlimited supply of cells and can be derived from a simple blood draw.
“Hair loss profoundly affects many people’s lives. A significant part of my practice involves both men and women who are seeking solutions to their hair loss,” says Richard Chaffoo, M.D., F.A.C.S., a triple board-certified plastic surgeon who founded La Jolla Hair MD and is a medical adviser to Stemson Therapeutics. “I am eager to advance this groundbreaking technology, which could improve the lives of millions of people who struggle with hair loss.”
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New research raises prospect of better anti-obesity drugs
Effective weight-loss strategies call for eating less food, burning more calories—or ideally, both. But for the more than 90 million Americans who suffer from obesity, a disease that contributes to conditions ranging from cancer to heart disease, behavioral change is hard to accomplish or not effective enough—which is why scientists have long sought drugs that would help people shed pounds. Yet effective, long-lasting treatments have thus far eluded them.
In a new report published in Cell, researchers in the laboratory of Jeffrey M. Friedman propose a new avenue in the search for anti-obesity drugs. Collaborating with a Princeton University team, they have found that a group of brain cells previously shown to regulate hunger also controls energy expenditure. And since our body weight depends both on the calories we consume and the energy we burn, these findings could lead to a new type of weight-loss medication that acts on both sides of the energy equation.
Energy in, energy out
Thus far, most obesity research has focused on the biological mechanisms that govern how much we eat. Yet manipulating the neural circuits that control our calorie intake hasn’t led to broadly successful anti-obesity drugs. So, Marc Schneeberger Pané, a postdoctoral Kavli NSI fellow in Friedman’s lab, launched a project to investigate the processes by which we burn energy instead.
Mammals like mice and humans expend energy in many ways, and producing heat is among most important. When the ambient temperature drops, we burn more fuel to maintain a steady body temperature; when it rises, we burn less. We even possess a special form of fat tissue, known as brown fat, that is burned to produce heat directly.
Scientists knew that some populations of temperature-sensitive neurons in the hypothalamus region of the brain play a role in regulating heat production and, therefore, energy expenditure. But they did not know exactly how those neurons exerted their influence, or if other cells outside the hypothalamus might play a similar role.
Double duty
Schneeberger Pané and his colleagues, including graduate student Luca Parolari, began by mapping the brain regions activated by a rise in ambient temperature. They used an advanced 3-D-imaging technique called iDISCO, developed at Rockefeller, to scan the brains of mice exposed to hot temperatures, looking for signs of neuronal activity.
As expected, the team saw activity in the hypothalamus. But they also saw activity among a particular group of neurons in a part of the brainstem known as the dorsal raphe nucleus—and, to the researchers’ surprise, these happened to be the very same neurons that, just two years earlier, the lab had found to be crucial for controlling hunger.
“Our new findings demonstrate that these neurons regulate energy balance by modulating both food intake and energy expenditure through partially overlapping circuit mechanisms,” says Alexander R. Nectow, who led the Princeton University team.
The possibility that these cells, which the lab had previously dubbed “hunger neurons,” might regulate both hunger and energy expenditure raised the prospect that they could serve as powerful levers for managing body weight.
“We were very excited,” recalls Schneeberger Pané, who adds that he and his colleagues view these multitalented neurons as “a new horizon in obesity research.”
The researchers used sophisticated biochemical techniques to alternately turn these temperature-sensitive brainstem neurons on and off. They discovered that activating the neurons reduced the temperature of the animals’ brown fat, which is burned to generate heat, core body temperature as well. Suppressing the neurons, meanwhile, amped up heat production—and, as the scientists had shown previously, it also made the animals less hungry.
Eat less, burn more
But generating heat by burning brown fat isn’t the only way to expend energy. Physical activity burns calories too, as do all the basic tasks that keep the body alive: breathing, digesting food, and so on. So the researchers put the mice in special cages tricked out with sensors to track their movements and gauge how much carbon dioxide they produce and how much food, water, and oxygen they consume. The goal was to see if the temperature-sensitive hunger neurons dorsal raphe nucleus could control energy expenditure not only by regulating temperature, but by other means as well.
Again, the results were clear: just as activating the neurons caused heat production to plummet, so too did it cause movement, metabolic activity, and overall energy expenditure to nosedive. Suppressing the neurons, on the other hand, caused all of them to rise.
The team has already begun searching for unique receptors in these dual-purpose neurons that govern both hunger and energyexpenditure. The idea is to identify targets that can be used to create novel anti-obesity drugs capable of delivering a one-two punch.
“When you inhibit these neurons, they suppress food intake and increase energy expenditure at the same time,” says Schneeberger Pané. Figuring out how to quiet them in people could enable a doubly effective assault against a massive public health problem.
More information: Marc Schneeberger et al, Regulation of Energy Expenditure by Brainstem GABA Neurons, Cell (2019). DOI: 10.1016/j.cell.2019.05.048
Pfizer names former FDA chief Scott Gottlieb to its board
Pfizer Inc said on Thursday it has named former U.S. Food and Drug Administration Commissioner Scott Gottlieb to its board of directors, effective immediately.
Gottlieb stepped down abruptly as the FDA chief in March this year, a role he had held since May 2017.
He was well-regarded by public health advocates and won bipartisan support for his efforts https://www.reuters.com/article/us-usa-fda-gottlieb/u-s-fda-chief-tough-on-e-cigarettes-steps-down-abruptly-idUSKCN1QM2GZ to curb use of flavoured e-cigarettes by youths.
Unlike his predecessors, who said drug pricing was not the purview of the FDA, Gottlieb waded into the intensifying debate about the high cost of medicines for U.S. consumers and had the agency actively looking into possible solutions.
Gottlieb ran into fierce opposition from anti-regulation groups such as Americans for Tax Reform and former FDA officials, who said the agency’s regulatory efforts would destroy thousands of jobs.
Pfizer has also appointed Gottlieb to the board’s regulatory and compliance as well as the science and technology committees.
Karuna Therapeutics IPO: What You Need To Know
Biotech investors seeking a play on the lucrative, yet hard-to-crack central nervous system disorder therapy market have an opportunity this week.
The IPO Terms
Boston, Massachusetts-based Karuna Therapeutics, Inc. plans to offer 4.375 million shares in an IPO, with the pricing estimated between $15 and $17, according to the preliminary prospectusfiled with the SEC.
The size of the offering is estimated at $70 million at the midpoint of the estimated price range.
The company has applied for listing the shares on the Nasdaq under the ticker symbol “KRTX.”
Since Karuna qualifies as an “emerging growth company,” it has elected to comply with some reduced public company reporting requirements.
Goldman Sachs, Citigroup, Wells Fargo Securities and Wedbush PacGrow are the underwriters for the offering.
The Company
Karuna focuses on developing novel therapies to address disabling neuropsychiatric conditions characterized by significant unmet need.
The company’s lead product candidate KarXT is in a midstage trial for the treatment of acute psychosis in patients with schizophrenia. It expects to release top-line data from the trial in late 2019.
Karuna is also planning to initiate clinical trials of KarXT to evaluate its therapeutic benefit in other central nervous system disorders, including psychosis in Alzheimer’s disease as well as pain.
Despite being a highly generic market, worldwide sales of antipsychotic drugs are estimated to exceed $14 billion by 2025, the company said.
The Finances
Karuna reported a pretax loss of $17.51 million in fiscal 2018 and $12.01 million for the three-month period ending in March 2019.
Realtor® Support for Association Health Plans Grows Nationally
Over 200 state and local Realtor®associations from across the country have proclaimed their support for the National Association of Realtors®‘ effort to protect access to association health plans. Earlier this year, a federal court held that provisions of the Department of Labor’s AHP rule were unlawful, a ruling which is currently being appealed by the Department of Justice. NAR recently filed an amicus brief in support of the DOJ’s appeal as it continues to work to increase and improve health care options for America’s independent contractors.
“We’re encouraged to see so many associations from across the country demonstrate their public support of association health plans,” said NAR President John Smaby, a second generation Realtor® and broker at Edina Realty in Edina, Minnesota. “The DOL’s rule opens the door for Realtors® to access affordable, high-quality health care options that can be so difficult for independent contractors to find. Moving forward, we’re hopeful that our work alongside these local partners will help secure the health care solutions that our members and their families deserve.”
Over 1.15 million Realtors® are represented by these 200 plus associations offering their support of AHPs. While some regulatory changes stemming from the Patient Protection and Affordable Care Act have benefited Realtors®, others have led to increased health care costs, leaving many individuals to drop insurance coverage and jeopardize the health, safety and financial stability of their families. Representing one of the best possible solutions to this hurdle, AHPs have proven to generate more affordable and better overall coverage.
To date, over 3,000 Realtors® and their families have found cost-effective health insurance solutions through the AHPs currently operated by a handful of local Realtor® associations across the country. Countless additional associations are also exploring AHP options but have been delayed due to uncertainty surrounding federal litigation. The more than 200 associations which have proclaimed their support of AHPs are illustrated here, along with an official, comprehensive list which will be updated as new supporters are added.
Actuaries: Health Care Costs, Public Policy Among Factors Shaping Premiums
A new issue brief from the American Academy of Actuaries provides consumers and public policymakers with an actuarial perspective on key factors involved in setting 2020 health insurance premiums.
“Steadily increasing health care costs; the possibility of ongoing adverse selection caused by the draw of potentially cheaper, non-Affordable Care Act compliant plans; and the effects of other federal and state public policy changes are among the biggest drivers of 2020 premium changes,” said Academy Senior Health Fellow Cori Uccello. “Premiums reflect federal and state rules and recent public policy actions affecting them as well as local market conditions, so the premium adjustments that individual policyholders see will vary considerably across the country.”
The key factors shaping 2020 individual market premiums, according to Drivers of 2020 Health Insurance Premium Changes authored by the Academy’s Individual and Small Group Markets Committee, include:
- The underlying growth in health care costs, or medical trend, which is expected to be consistent with that for 2019, ranging from about 5 to 8 percent.
- Recent and ongoing policy changes regarding the expanded availability of short-term limited duration plans, association health plans, and health reimbursement arrangements, as well as the elimination of the individual mandate penalty.
- The possibility of issuers incorporating extra conservatism in their premiums based on uncertainties regarding expected risk adjustment transfers.
- Adjustments to assumptions used to build the cost of cost-sharing reduction subsidies into premiums.
- State actions to implement reinsurance programs, impose individual mandate penalties, or enact rules that would facilitate or prohibit the availability of alternative coverage options.
- The reinstatement of the health insurance provider fee.
Read the issue paper here and learn more about the Academy’s health policy work under the “Public Policy” tab at actuary.org.
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