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PROGRESS 2019: A tradition of giving | News, Sports, Jobs – Alpena News

Posted on December 31, 2019 by Danzig

News File PhotoIn this photo from June 2016, Alpena resident Jim Park, right, receives the Michigan Association of School Administrators Champions for Choice Award from then-Alpena Public Schools Superintendent Brent Holcomb.

ALPENA The Besser tradition was started by Jesse and Anna Besser and the drive of Phil Park, Jim Park says.

Jim Park and the Park family have for decades donated to the Northeast Michigan community to help improve the areas health, education, and recreational opportunities, and that initiative started in the early 20th century.

The traditions provided by Jesse and Anna Besser, the foresight and drive of Phil Park, as well as some good experience overseas and in Washington, D.C. and Tampa were all instrumental in building the quality of leadership that a person with a great staff could accomplish, Jim Park said.

Phil Park, who is a former president of Besser Co., is Jim Parks father and nephew of Jesse Besser. Jim Park is himself a former Besser president.

Jesse and Anna Besser started the Besser Foundation 75 years ago. Following that lead, Jim Park and his family created the Park Family Foundation.

The Besser Foundation was created in 1944 to receive and administer funds for scientific, educational, religious, and charitable purposes, according to the Besser Co. website.

The Park Family Foundation was created to help meet athletic and educational needs of the communities.

The Park Foundation has three active trustees: Jim Park, his daughter, Kim, and son, Scott.

The Park family has provided funding for the Alpena Community College gymnasium, which was named Park Arena in the 1990s, the Besser Museum, and scholarships for Northeast Michigan high school students.

This year, funding was provided for the new surface for the field at Alpena High School, ACC renovation, and several scholarships for local students through the Community Foundation of Northeast Michigan, Jim Park said.

Jim Park said its hard to put an approximate number on how much the Park Family Foundation and Besser Foundation have each provided to the area over the years.

He said the Besser Foundation provides over $1 million a year. This year, that amount was doubled to celebrate the 75th anniversary of the fund.

About $2.4 million in grants were allocated in September by the Besser Foundation to the Boys and Girls Club of Alpena, Alpena Public Schools, the Friends of Thunder Bay National Marine Sanctuary, and others.

It is rewarding to see the great facilities used and busy, Jim Park said. Our community is generous.

Jim Park received both a bachelors and masters in business administration from the University of Michigan after graduating from Alpena High School.

He returned to Alpena in 1970 and took a position with Besser. He was then named president of the organization in 1977. He was president until 1999.

He was inducted into the Alpena Sports Hall of Fame in 1995, named the Alpena Chamber of Commerce Citizen of the Year in 1999, the president of the Alpena Rotary Club for a year, and inducted into the Alpena High School Alumni Hall of Fame in 2003.

While Jim Park was Besser president, the company expanded to different areas in the country and world. The company has locations in Alpena, Iowa, California, Canada, Russia, and others.

Several significant additions were made to the manufacturing area, as well as the World Headquarters in Alpena, Jim Park said. Major additions were also made to the World Center for Concrete Technology and the number of graduates blossomed.

Jim Park, who spends part of the year in Alpena and the other half in Fort Myers, Florida, is currently working on two books, The Besser/Park Era and Management Principles for Medium Sized Business.

Julie Goldberg can be reached at 989-358-5688 or jgoldberg@thealpenanews.com. Follow her on Twitter @jkgoldberg12.

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PROGRESS 2019: All in the family | News, Sports, Jobs – Alpena News

Posted on December 31, 2019 by Danzig

News Photo by Steve Schulwitz Over the years, the Townsend company has morphed from a coal business to be an industry leading distributor of plumbing, heating and air conditioning products. Its current location is on Bagley Street, but there also showrooms in Burton and Warren.

ALPENA Family, hard work, and the ability to adapt to changing market trends have been the recipe for success for R.A. Townsend in Alpena, which is quickly closing in on its 100th anniversary in business.

It has been a long road for the Townsend family, which grew its business from a meager beginning as a small coal delivery company to a large distributor of plumbing, heating, and air conditioning products from various locations in Michigan.

The story began in 1928, when Russell Ambrose Townsend Sr. established the R.A. Townsend Coal Co., which delivered coal to residents and businesses from its shop at the corner of 9th Avenue and Ripley Boulevard. From there, the business was moved near the Thunder Bay River to the end of 3rd Avenue, near where the Holiday Inn Express & Suites is today. The move allowed ships easy access to the business and easy unloading of the coal.

In 1947, Russ Townsend Jr. took the reins of the company and, later that decade, the company began offering contracted services for heating, in addition to coal delivery.

In the 1950s, Townsend, his family, and brother-in-law Bernie Lamp realized there was a change in the way people were heating their homes. So they decided to transition from a coal business and heating retail service to become a wholesale business. In 1962, the company officially became incorporated and the coal business was abandoned all together.

Today, the family has locations in Alpena, Saginaw, Burton, and Warren.

Co-owner Tom Townsend said both his grandfather and dad were versatile businessmen, even during times of economic uncertainty, and were always looking for a way to change and adapt when needed. He said their foresight helped the business grow.

One thing our father was was always early on the scene, and a real visionary, Tom Townsend said. Sometimes, the changes were made too quick and before any of us were ready to do things.

Jim Townsend said he and his brother, Tom, and sister, Therese Shaw, all were raised learning about the business and every aspect of its operations, which benefits the company today. From the warehouse to the administrative offices, his father wanted to be sure they learned the importance of each job and the benefits of hard work, he said.

That practice is still utilized today with all of Townsend employees.

We always start people in the warehouse, just like we did, Jim Townsend said. That is a great way to learn the product and business. They learn every aspect of this building, from purchasing, to the counter, inside sales, outside sales. They all started there in the warehouse.

Tom Townsend said the business is still very family-oriented and that Bernie Lamp is still regularly at the facility, making sure things are in order. Over the years, a staff has been created that has lots of experience, which will help move the company into the future.

We have a really good group of young people who are working with us now, and they have consistently grown in what they can do, and we continue to challenge them, Tom Townsend said. A few of them started here 20 or 25 years ago, when they were 18 years old, working part-time while going to Alpena Community College. We also have fifth-generation family working here, so family will still be involved.

To ship product farther than they are able to now, Jim Townsend said more locations would need to be open.

He said that is a future goal.

That has to be one of our goals, and that is where our future growth will come from, he said. I think having more operations in other parts of the state will make up our future growth.

Shaw said there are no plans to move far from Alpena when the business expands. Unlike her two brothers, who moved away from home for a period before returning home, she said there is one main reason she hasnt.

Family, she said. We have nine kids in our family and eight live here. I was born and raised here, love the area. I cant fathom living anywhere else.

Steve Schulwitz can be reached at 989-358-5689 at sschulwitz@thealpenanews.com. Follow him on Twitter @ss_alpenanews.com.

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PROGRESS 2019: All in the family | News, Sports, Jobs - Alpena News

PROGRESS 2019: Going above and beyond | News, Sports, Jobs – Alpena News

Posted on December 31, 2019 by Danzig

News Photo by Jonny ZawackiAlison and Steve Jakubcin stand in front of a water tower in Alpena near the skate park, while displaying a United Way banner. The Jakubcins have both been an instrumental part in giving back to the community of Alpena through United Way of Northeast Michigan.

ALPENA It is safe to say that Alpena has its own power couple.

Though maybe not as famous as they want to be, Steve and Alison Jakubcin are accomplished and well-known not just around Alpena, but all of Northeast Michigan and for all the right reasons.

The Jakubcin family has been a driving force behind the ongoing success that is the United Way of Northeast Michigan, a nonprofit responsible for coordinating and fundraising money to help other various nonprofits make the area a better place.

Both Steve and Alison were once co-chairs and got involved with United Way four years ago.

The community has always been very supportive of us and I feel like its a great community, Alison said. Theres so many great people, and we just wanted to be a small part in helping Northeast Michigan grow and help these agencies that United Way supports thrive a little more.

The Jakubcins have helped raise hundreds of thousands of dollars while working with United Way and giving back to the community.

Not only does the couple enjoy giving back, but their work with United Way gave them an opportunity be a part of fundraisers of all different kinds, meet new people, and raise money for a community with which the couple loves being so heavily involved.

I think we make a pretty good team, Alison said. Weve been able to feed off of each other and be supportive. Its really nice to have a partner going into it that is as passionate about it as you are. I feel like (Steves) connections and my connections are what made it really successful for us.

The Jakubcins were both busy, yet flexible with their daily schedules and received tremendous support from their employers, which made being such a big part of United Way possible.

Steve is employed by Cliff Anschuetz Chevrolet in Alpena, and Alison works for the Alpena Alcona Area Credit Union. Despite busy work schedules and the involvement with United Way taking up much of their time, the Jakubcin family always makes time for different events going on the community and taking time to travel and support the rest of their family in the process.

We both have the backing of our employers that is huge, Steve said. Not everyone has that, so were able to be flexible with us and both of our employers are also heavily involved with United Way of Northeast Michigan, which makes it that much more special for us. Its great to know that not only do we as a couple have each others back, but our employers are behind us and support us. It does get busy, it does get hectic, but the responsibility never gets lost in that.

There has never been a goal or an amount of money that needed to be raised that the Jakubcin family couldnt reach. In fact, more often than not, Steve and Alison went above and beyond the goal that was set for them.

There was pressure on us, and it could have been easy for the goals to remain the same, but we always felt like we wanted to improve and do the absolute best we could, Steve said. All were doing when we go beyond our set goals is raising more money for our agencies. Were competitive and took making a difference in this community we love a big deal.

The biggest enjoyment that the Jakubcin family gets out of being such an important part of United Way is the fact that theyre helping everyone in the community, from senior citizens, children, and people in need.

Along with Alpena, the Jakubcins work has made an impact in Alcona and Montmorency counties. The couple has also helped with mini grants in Presque Isle County, and they will help communities outside of those areas and get support for many other surrounding counties, as well.

The biggest benefits from working with United Way has been the people weve met, stories we heard and the impact its made, Alison said. Weve met some of our good friends through getting involved.

The Jakubcin family believes it is important to stay involved not only because they are making a difference through their involvement with United Way, but because the couple believes in Alpena.

We really, honestly believe in Alpena, Steve said. And its not just the fun events, but its just these crazy little things that people are trying to do that make our community just a little bit better. It makes it fun for us to support all of our friends and all of the different people around town that are trying to make Alpena a better place. Its easy to support a community that supports you.

Were both very invested in our community, Alison said. Its a special place. Our families are from here, we found each other here, and its just a great place to live and its important for us to get involved and give back. Were so fortunate to be a reason why the community of Alpena is such a special place.

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PROGRESS 2019: Going above and beyond | News, Sports, Jobs - Alpena News

PROGRESS 2019: The state of the economy today? Alpena’s stock is rising | News, Sports, Jobs – Alpena News

Posted on December 31, 2019 by Danzig

To provide you with a snapshot of where our economy stands today, it is important to look back at where we were not all that long ago.

In the winter of 2010, our unemployment rate hovered around 13.5%, there was little investment being made on any level, and our communitys general attitude reflected that.

Understandably, the counties surrounding Alpena were in even worse shape, as that is how the trends flow. If Northeast Michigan was a stock, it wouldve been classified at that time as a hold or a sell.

Through the work of many people, our economy has improved dramatically over the past eight years. Keep reading to learn how we have gotten to where we are currently.

Prior to 2011, most of the economic development efforts that were conceived and implemented relied on ideas from outside organizations and regional initiatives but didnt focus on the engine. Alpena is the engine that provides for the health and wellbeing of a large swath of geography in Northeast Michigan. How Alpena goes, so goes the rest of the region.

Localized strategies were conceptualized, designed, and put into motion, which focused on improving the health of the engine and, if successful, would in turn drive improvement through the entire economic trade area that we serve, and for the long term.

What really got the ball rolling was the unofficial coordination of the activities amongst many stakeholders. That included the Chamber of Commerce, Convention and Visitors Bureau, Downtown Development Authority, along with the unwavering support of our businesses and local government entities. It also included growing our industrial presence, our commercial presence, our tourism industry, and our entrepreneurial support ecosystem.

There are a lot of extensions from each word in that last sentence, but it was the start of something remarkable. Our strengths were marketed to a thirsty world, weve reinforced our position as the economic hub of the region, and we invested heavily in the engine that is Alpena.

Wins started stacking up. Our undercapitalized or vacant buildings and empty lots started being developed or redeveloped, more people were finding opportunities for employment, visitor imaginations were being stirred, new small businesses began to emerge, and a concerted effort was made to align and improve upon our sense of place and our workforce development initiatives with the talent needs of our major employers.

While some may see the reemergence of our sense of place and self as a localized initiative, the impact isnt. Throw a rock in the water and watch the ripples from the impact move outward thats a good visual of our strategy.

Unemployment is just one of many measuring sticks, but it can show us how far weve come in just nine years:

Alcona County: January 2010, 22.6%; August 2019, 6.8%

Alpena County: March 2010, 11.5%; August 2019, 4.8%

Montmorency County: March 2010, 23.8%; August 2019, 6.5%

Presque Isle County: February 2010, 15.5%; August 2019, 5.8%

Each passing year has brought increasing investment to our community, and, with it, more opportunities to Alpena and our surrounding counties. Our work isnt confined to Alpena and we are both cognizant and motivated to make certain that opportunities arent missed in our adjacent counties.

The improvement in economic conditions in Alpena and Northeast Michigan have undoubtedly placed higher demands on our employers and our workforce. The ability for any employer to diversify, expand, or grow, is directly linked to the available workforce and that can be summed up in one word competency.

Is there an ample current and future supply of competency in specific disciplines which will help accelerate business diversification and growth? No, not yet. But are we getting better? Yes.

The Alpena Chamber of Commerce, along with economic development, our businesses, schools, and Alpena Community College, are working together and have made concerted efforts and developed coordinated programs designed to better serve both the futures of the students and the talent needs of our employers and are used as a model for other communities to follow.

Sometimes, when youre standing waist-deep in something, its hard to gauge where youre standing. Over the past few years, weve had visits from numerous communities from across the state asking us what our recipe is and if we could help them or perform community assessments for them. The fact that they are taking notice of our progress certainly validates our work.

Recently, we had an out-of-region developer with a long history of working with our community in town for a tour. And he said it best when he said, Your stock is no longer a hold or sell, youre considered a buy.

Jackie Krawczak is president and CEO and Jim Klarich is the former economic development director of the Alpena Area Chamber of Commerce.

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PROGRESS 2019: The state of the economy today? Alpena's stock is rising | News, Sports, Jobs - Alpena News

Progress’ 19 most read stories of ‘ – Claremore Daily Progress

Posted on December 31, 2019 by Danzig

This past year was a whirlwind of Rogers County news.

Below is a breakdown of the most-read stories on claremoreprogress.com for the year.

"NO REMORSE: State says Barrett showed no remorse for murder"

In January, the Rogers County District Attorney submitted evidence and testimony with suggestions that Chance Barrett "is never released from prison." Barrett, who was 19 at the time of arrest, was accused of murdering Helen Pasdach in cold blood, in her home, on Aug. 21, 2017.

"Couple arrested after infant tests positive for amphetamines"

Jared and Kyley Spears were arrested after their infant displayed symptoms, then tested positive for amphetamines. Police described details of unsafe living expenses with drugs, drug paraphernalia, and sex toys scattered across the baby's bed.

"Woman whose father traveled Trail of Tears turns 100"

Geraldine Henson Birdsbill turned 100 in March of 2019. In sharing memories of her life, she talked about her father's experience on the Trail of Tears and the life that followed.

"Man arrested on rape charges tells cops 'stop doesn't always mean stop'"

Investigators report that Travis Carl Condry's cell phone contained over five minutes of audio from a reported rape. First degree rape charges were filed April 12 and Condry, 26, of Claremore, was arrested on the charges days later.

"A SMALL FORTUNE: Claremore man offers $10,000 for rare penny"

Claremore businessman Richard McPheeters sent the town into a frenzy when he announced he was willing to pay as much as $10,000 to the person who can help him complete his rare coin collection.

"Rogers County residents should take flood precautions now"

It was a severe weather event that rocked the entire county. In May, those living around the Verdigris River were urged to take flood precautions including evacuation.

"SUBMERGED: Claremore homes engulfed in Verdigris River"

On the far west end of Blue Starr Drive, the property of Melinda Knight, her daughter Brandi Gatzemeyer and her brother Larry Washom were submerged beneath the crest of the Verdigris River. All the while, the family business and Claremore staple, Dot's Cafe, remained open.

"Police: Woman strikes boyfriend with car, arrested"

Claremore Police Department arrested Raychel Higeons for striking her boyfriend with a vehicle in the Ne-Mar shopping center parking lot

"$2M investment at Cedar Point will give north Claremore a face lift"

Cedar Point Lead Pastor Rick Burke said that over the next two years the church will be renovated in two phases, costing approximately $2 million. They said they hope to have phase 1 complete by Easter of 2020.

"Walkingstick:Citizens should be deeply disturbed by Cherokee Nation pay hikes"

Cherokee Nation Tribal Councilor David Walkingstick released a statement in response to the nation's proposal to provide pay raises of elected officials.

"40 Under 40 honorees announced"

Once again the Claremore Progress and community celebrated forty Rogers County residents who were nominated as being top in their field or a shining star in the community.

"Suspect arrested in Claremore armed robbery"

Police arrested 25-year old Joshua Jones in connection to a Quick Mart robbery that took place in Claremore on Sept. 8

"City returns $1.2 million in electric credits"

In September, Claremore city leaders announced that credits would be made to all customers during the months of October and November totaling some $1.2 million as a result of electric overpayment.

"Stage closing, converting to Gordman's Department store"

The Ne-Mar shopping center announced plans to convert Stage to a Gordman's Department store in the spring of 2020.

"Injury accident forces vehicle through wall at Pixleys"

An accident at the intersection of Owalla Avenue and Will Rogers Boulevard forced a vehicle through the wall and into a meeting room at Pixley Lumber on Oct. 25. The driver and passenger sustained minor injuries.

"'I came back different': Governor signs pardon for Rhonda Bear"

The community turned out in full support of She Brews owner Rhonda Bear as Governor Kevin Stitt made the trip to Claremore to sign her pardon nearly 20-years after she was released from prison.

"Police: High speed pursuit ends in drowning"

Zeth Patrick Daly, 23, from Claremore, drowned while attempting to flee from police, according to police records. The driver of the vehicle, Nazra Daly, was arrested for second degree murder and possession of a stolen vehicle.

"Chelsea trash service loses entire fleet"

Chelsea-area trash service, Johns Refuse Service LLC, lost all but one of their trash trucks in a fire over Thanksgiving weekend. The family-owned business has been in operation for over 40 years.

"RCSO, FBI search for possible body in Oologah"

An 18-person FBI team, along with Rogers County Sheriff's Office searched a pond on a residential Oologah property for a possible dead body after receiving a tip on an

Continued here:

Progress' 19 most read stories of ' - Claremore Daily Progress

A year of transition and progress for the KBC in 2019 – Kentucky Today

Posted on December 31, 2019 by Danzig

By MARK MAYNARD, Kentucky Today

The year 2019 was one of transition and forward progress for the Kentucky Baptist Convention.

The commonwealth's largest protestant denomination welcomed a new executive director-treasurer in a year where Kentucky Baptist churches redoubled their evangelistic efforts to introduce communities to Jesus, planted churches in record numbers and generously gave in abundance to the Cooperative Program.

Gray to lead KBC

Todd Gray became the new executive director-treasurer in July following a seven-month search. He came in with a passion for evangelism and has stressed to the more than 2,500 churches in the KBC to engage with the lost and reach every home in the commonwealth with the gospel.

Were commanded to go anywhere we can go with the good news of the gospel, he said. Take the gospel of Jesus Christ to those who are lost. Weve been found because we know the Savior. We know how a lost person can become a found person. So the onus is on us to take that message.

Gray led by example by traveling to communities and knocking on doors with pastors from small churches and big churches throughout Kentucky. Regional consultants were actively on the road, visiting pastors and helping churches with next steps in the process of starting a new program, finding staff or dealing with controversial issues.

An emphasis on church planting

The KBC partnered with local churches and associations to work with more than 60 church planters across Kentucky. The group effort is striving to see churches into planted and revitalized in more communities than ever. One great example is Highview Baptist in Louisville as they recently launed their Valley Station campus an autonomous church.

Record-setting Cooperative Program giving

It became a year where Cooperative Program giving set records as churches gave more money than was expencted to the partnership effort. Half of the money given by churches to the Cooperative Program stays in Kentucky. The other half is forwarded to the Southern Baptist Convention for efforts such as the North American Mission Board and International Mission Board.

Sunrise reaches milestone in adoptions

Sunrise Children's Service, an agency of the Kentucky Baptist Convention, celebrated their 500th adoption. The agency has been serving children and families in Kentucky since 1869. Sunrise serves Kentucky through adoption, foster care, therapeutic residential care for children and more.

More than 600 lives transformed by the Gospel at Hillvue Heights

Hillvue Height Church in Bowling Green shared in God's faithfulness as they baptized more than 600 people in the fiscal year that ended in 2019. Pastor Steve Ayers said the congregation set and prayed toward a goal that only God could achieve.

Kentucky Baptist DR continues to lead the way

Kentucky Baptist Disaster Relief work remained the gold standard in the country and brought relief both in the United States and abroad behind able director Coy Webbs guidance. Hundreds of people received prayer, food and more through the work of KBC DR workers in 2019. Dozens came to a saving faith in Jesus Christ through the compassion of the ministry.

KBC Church takes on drug epidemicLed by Pastor Casey Carver, Inez Baptist Church stood up to the drug epidemic plaguing the eastern Kentucky community. Carver helped the church launch a Christian-based drug rehab facility in a local hotel that was once a hot-spot for drug dealers.

Tent revival on the banks of the Ohio

Members of Pleasant Valley Church prayed and planned a tent revival on the banks of the Ohio River in Owensboro. Little did they know how God was going to move. Church leaders say God used the multi-night event in September to bring dozens of people to faith in Jesus Christ.

Seven years of debt eliminated in just 24 months

The members of Versailles Baptist Church initially planned to take down their $1.6 million debt over seven years. God has different plans. Through the faithful and generous giving of church members, the debt was eliminated after 24 months in April.

Patterson to lead KBC's evangelism team

When Todd Gray became the executive director-treasurer of the KBC there was a need for someone to lead the KBC's Evangelism Team. The position was filled in December by Rob Patterson, a former IMB missionary, associate pastor at a number of churches and, most recently, as the KBC's regional consultant in central Kentucky. He officially begins in the role on January 1. One of Patterson's first roles will be to help facilate a major outreach event in Owensboro in November in connection with the Crossover events prior to the 2020 KBC Annual Meeting there.

To learn more about the work of the Kentucky Baptist Convention and her 2,350 churches, find them online here.

Looking back on a year of great progress for our industry – InvestmentNews

Posted on December 31, 2019 by Danzig

When we look at back at the biggest changes in our industrys regulatory and legislative landscape in 2019, the headline items will no doubt be the SECs Regulation Best Interest and passage of the SECURE Act.

We are pleased to have worked with the SEC to help shape Reg BI, which increases transparency and protection for investors while maintaining advisers ability to effectively serve their clients.

More recently, the passage of the SECURE Act is a testament to the power of grassroots engagement. This crucial victory for Main Street Americans was achieved after a year-long engagement with lawmakers, during which our members met with their legislators to underscore the importance of this measure for their clients.

With these victories capturing so much attention, it would be easy to think of other regulatory and legislative developments in 2019 as footnotes. We are continually mindful, however, that laws and rules passed by state lawmakers and regulators have just as profound an impact on our industry as the measures that grab the biggest headlines.

With that in mind, we are pleased to report meaningful progress on the following issues in 2019:

Independent contractor status. Educating policymakers on the centrality of the independent contractor model to our industry is constantly ongoing, as new legislators are elected and court rulings create shifts in various independent contractor standards.

Our effectiveness in this mission of education was on display this year in the battle over Californias Assembly Bill 5, which will reclassify hundreds of former contractors in the state as full-time employees in 2020. Thanks to our productive dialogue with legislators and our members engagement, we secured a carve-out in the legislation for independent financial advisers.

State fiduciary rules. Several states advanced rules this year that demonstrated the damage that could occur if advisers are subjected to an inconsistent and confusing patchwork of different standard-of-care obligations across the country. Such an outcome would severely restrict investors access to financial advice and cause excessive complications and uncertainty for advisers.

Massachusetts Securities Division has proposed a rule requiring advisers to make only the best available recommendations to clients based on currently available information without providing clear definitions and standards for such recommendations, nor identifying which body will make these determinations.

New Jersey is moving ahead with a rule that would create a fiduciary obligation for advisers who provide even a single recommendation to an investor, even for nothing more than a one-time transaction.

We remain dedicated to combating these proposals, and we were pleased to help defeat a similar rule this year in Maryland.

Pushing back against regulation by enforcement. The SECs Share Class Selection Disclosure initiative represented the most harmful example of a trend that has been building for years the tendency of the SEC and other regulators to substitute ad hoc enforcement actions for transparent and consistent rule-making procedures.

This practice makes effective planning and compliance vastly more difficult for advisers and firms across the country. We are leading the charge to end regulation by enforcement through ongoing constructive dialogue with regulators and by educating members of Congress on the threat it represents to our members businesses.

[More: Working to get Reg BI implemented]

Dale Brown is president and CEO of the Financial Services Institute.

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Looking back on a year of great progress for our industry - InvestmentNews

Cleveland Browns ownership admits a mistake, & thats what passes for progress in BereaBud Shaws Sports Spin – WKYC.com

Posted on December 31, 2019 by Danzig

CLEVELAND A NFL team never looks as bad as it does when it fires a head coach after one season.

But keeping him and firing him in his third season after hes jumped in the lake to pay off a betwe can say from experienceisnt exactly a reputation saver either.

The Haslams still dont appear to have any earthly idea how to build an organization, but theyre closer Monday than they were Sunday to at least not letting one wallow in the shallow hope that continuity alone is the answer.

So maybe there was residual value to the Hue Jackson mistake, mainly a realization that the biggest blunder was keeping him for as long as they did.

Freddie Kitchens wont get a second year not because the Haslams are suddenly smarter about what they want in a coach. We dont have that answer yet, and if they throw this decision open and create a power struggle between GM John Dorsey and VP Paul DePodesta we may not get the answer then, either.

But they recognized Kitchens wasnt what they hoped for, and wasnt going to become what this team needs just because he was likable inside the building.

Conclusions are never hard and fast in Berea, partly because the shifting seat of power belongs to whomever was not proven wrong most recently (In this competition, DePodesta might have an edge).

But for now, moving on from Kitchens passes for progress.

Firing him Sunday wasnt nearly the stretch hiring him based on a three-month apprenticeship as offensive coordinator was last year. They thought it made sense to put him in charge of a team ready to win, even though he wasnt prepared to do anything more than give Baker Mayfield confidence operating the offense.

When that even failed to materialize, when Mayfield significantly regressed, there wasnt much to recommend the Kitchens hire.

The offense and quarterback were worse than last year despite far better talent (offensive line excluded) in the huddle. Kitchens didnt exactly impress as a CEO or as a game manager.

What else did that leave? Not enough.

Cleveland Browns head coach Freddie Kitchens answers questions during a news conference after the Bengals defeated the Cleveland Browns 33-23 in an NFL football game, Sunday, Dec. 29, 2019, in Cincinnati.

Bryan Woolston/AP

His Browns were undisciplined, sometimes unprepared. It couldnt have helped that so late in the season they took a delay of game penalty after an Arizona kickoff three weeks ago, melted down at halftime against Baltimore and then allowed a Cincinnati rushing touchdown with only 10 defenders on the field Sunday.

The statement the Haslams' released carried telling words about the need for a "strong head coach" and an "exceptional leader." Kitchens might be inclined to throw those words right back at the Haslams, who blow with the wind as owners and dont stand for anything identifiable all these years later.

But its hard to argue with the line in the statement about the belief Kitchens didnt offer "opportunities for improvement."

If this first head coaching job was about a learning curve, we wouldve seen more sustained improvement, however small. Other than special teams and a mid-season stretch of reduced penalties, there wasnt much else.

Better game management as the season went along wouldve helped his case. A player-coach dynamic that didnt make him look as if he were herding cats wouldnt have hurt either.

A change was necessary. If players dont say it, that doesnt mean they dont believe it.

What happens next is a guess. Since its Berea, throw in some prayers just to be safe.

The Haslams were right to admit a mistake.

The hard part for them, though, never changes: Fixing it.

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Cleveland Browns ownership admits a mistake, & thats what passes for progress in BereaBud Shaws Sports Spin - WKYC.com

Ted Phillips’ slow recovery from a stroke: Some bumps in the road, but steady progress – theday.com

Posted on December 31, 2019 by Danzig

Ichi! ... Ni! ... San! ... Shi ...

As karate sensei Chad Merriman barked out the numbers one through four in Japanese, Ted Phillips let go of a cane in his left hand and lurched barefoot across a rubber mat.

... Ku! ... Juu! After calling out nine and 10, Merriman paused while Phillips caught his breath.

OK, now we go backwards, Merriman directed.

Eyes focused like laser beams, Phillips set his jaw, slowly elevated his left leg, swung it a few inches behind him, and gently lowered the limb. He then halted, repeated the motion with his right leg, and began shuffling heel first.

Excellent! Excellent! Merriman exclaimed.

Just then Phillips stumbled and would have toppled if trainer Rich Cochrane, poised alongside, hadnt grabbed his arm. A moment later, Cochrane released his grip and Phillips resumed trudging across the padded floor of Merrimans Niantic dojo.

In many ways, the December workoutreflects Phillipss slow, labyrinthine route to rehabilitation after he suffered a hemorrhagic stroke on July 15, 2017: Sometimes forward, sometimes backward, with a few missteps along the way, including a fractured hip from a tumble this past July but always with fierce determination.

Its tough, he said after the 45-minute karate session, but I just keep going.

Phillips, 78, once attained a black belt in karate. He also ran marathons, played the bagpipes, guitar, mandolin and a veritable orchestra of other instruments, competed in a vintage baseball league (no gloves), earned a doctorate and spoke several languages.

We called him the renaissance man because he did a number of things, all of them well, Tom Amanti, a retired principal at Montville High School, where Phillips had served as head of the counseling department, told The Day a year ago.

Now, Ted continues putting the pieces of his life back together, relying on a method that has always served him well.

I study, study, study. Work, work, work, he said.

During the past year Ted and his wife, Pat, have continued supplementing regular doctor visits with an exhaustive regimen of treatments and therapies, many of which have morphed from such sports as swimming, weightlifting, cardio training, treadmill workouts, bike riding and even boxing. He also has incorporated music classes, tai chi, acupuncture, hypnosis and speech therapy into his ever-evolving routine. Some appointments are in southeastern Connecticut, others are more than an hours drive away.

Ted and Pat additionally are considering therapeutic horseback riding, and this past summer acquired a three-wheeled bicycle that Ted can pedal, a battery-powered arm brace controlled by Teds neurological impulses, and a Rube Goldberg-like contraption that attaches to a shoelace, allowing Ted to strum a guitar by tapping his foot.

Initially Teds medical insurance helped pay for much of this treatment and equipment, but when some coverage lapsed the family got busy. Pat, along with sons Brennan and Simon, lined up free and lower-cost treatment provided by the University of Connecticut, local and state governments, and such institutions as Gaylord Specialty Healthcare in Wallingford, which Ted regularly visits for pool therapy, and treadmill and stationary bike exercises.

Although Ted has shown steady progress with mobility, cognitive function and speech, the slow pace occasionally hasproved frustrating for him and Pat, a gregarious couple whose default attitude has always been upbeat.

If you had told me wed only be this far along after two-and-a-half years, Im not sure ... Pat said, her voice trailing off.

Then she broke into a smile, not needing to finish her thought. She would have responded exactly the same way: with love for and devotion to the man she married 48 years ago, while applying skills developed from her background in special and early education.

A former teacher and assistant principal in the East Lyme public school system, Pat, 72, received a doctorate in educational administration and served as head of the preschool program at Mitchell College in New London before retiring to help care for her husband.

The stroke also forced Ted in 2017to leave his jobas a guidance counselor at St. Bernard School in Montville. Previously he had been a Spanish instructor at Mitchell College, taught English and then became a guidance counselor at East Lyme High School, and served as headmaster of Waterford Country School before taking the position at Montville High School.

Though Teds recovery has been gradual, his improvement is readily apparent to a visitor who hadnt seen him in several months.

His gait, though halting, is more assured; his smile, broader, his speech more fluid. Teds struggle to articulate the stroke created lesions in his brains left hemisphere, resulting in a communication disorder known as aphasia has been one of his most nettling challenges.

You can see by looking in his eyes that Ted knows what he wants to say, but cant always get the words to come out.

Hearing this observation, Teds face brightened, and he took a moment to formulate a response.

Thats it! Exactly! he blurted.

He can be full of surprises. This past summer, he stunned guests at a family wedding by reciting from memory Lewis Carrolls poem Jabberwocky, which has nonsensical verses capable of twisting the tongue of even those without speech limitations:

Twas brillig, and the slithy tovesDid gyre and gimble in the wabe:All mimsy were the borogoves,And the mome raths outgrabe ...

Merriman, whose weekly one-on-one karate classes began about a month ago, said Teds relentless drive is tempered by the methodical approach of a longtime educator.

He gets mad at himself, but then he works through it, he said.

Merriman, a seventh-degree black belt, is the son of karate legend Chuck Merriman, who started teaching in New London in 1970 before moving to Niantic, went on to attain the disciplines highest level, 10th-degree, served as head coach of the AAU National Karate Team, and is credited with helping popularize karate throughout the Western world.

Chuck Merriman, now 86 and living in Arizona, was among the first people to call Ted after his stroke. His simple message: You can get through this.

Tami McGunnigle, an occupational therapist with the Visiting Nurse Association of Southeastern Connecticut in Waterford, was one of Teds first caregivers after his stroke.

I could tell right away he was going to be a fighter, McGunnigle recalled. He and Pat, working as a team, really stood out. You just want to help them.

McGunnigle also treated Ted after he fell and fractured his hip. It was heartbreaking at first, she said, upon seeing Ted back in the hospital after he had made so much initial progress.

But he overcame it, she added.

In 30 years of helping patients recover from devastating injuries and medical events, McGunnigle has never treated anyone quite like Ted, she said. He is among the rare patients who refuse to give in to disability, who dont accept thats the way life is going to be.

Originally posted here:

Ted Phillips' slow recovery from a stroke: Some bumps in the road, but steady progress - theday.com

Teammates see Roundtree ‘more open’ with recovery, progress – 247Sports

Posted on December 31, 2019 by Danzig

SAN FRANCISCO, Calif. Jamal Woods had a choice. Take the long trip home and spend a few days with his family in Alabama, or take a short car ride from Champaign and visit Illini teammate Bobby Roundtree at his rehabilitation center in downtown Chicago before traveling to California for the Redbox Bowl.

So I went up there with Bobby, Woods told Illini Inquirer between quarters of watching the Golden State Warriors take on the Phoenix Suns on Friday. And he looked really good.

Woods and several of his teammates (Kenyon Jackson, Tymir Oliver and Dele Harding) passed on a rare opportunity to head home to see their families on Christmas so Roundtree who suffered a severe spinal injury last spring in a swimming accident near his hometown of Largo (Fla.) would feel closer to family during the holidays.

Hes far away from Florida, so he aint really got much family up here either, said Woods, who used to be Roundtrees roommate. We are his family. I just wanted to go up there and spend Chrismas with him. We help him out with his rehabilitation period, and it was really very good.

Roundtree, a star defender for Illinois during his 2018 sophomore season, had kept his arduous recoveryfrom his unfortunate May injury mostly private duringthe last year. But he recently has publicized his progress. He made his first return trip to campus during the Illinis final game of the regular season on Nov. 30.

Since then, the usually effervescent Roundtree has been more boisterous on social media. On Dec. 20, he showed a video of him doing push-ups. The video received more than 3,000 likes on Twitter. The following day, he shared a video of him exercising with ropes, which also received more than 3,000 likes on Twitter.

Woods said he and Roundtree discussed a few weeks ago that he should share his journey with the public and provide inspiration to others who are going through similar physical struggles. Roundtree plans to start a YouTube channel toshare his journey.

Hes more open now than what he was in the past, Woods said. He listened to what I said.

Woods and the Illini hope they give Roundtree extra energy and motivation in his rehab. When Woods visited Roundtree on Christmas, Woods saw the same energy and competitive fire Roundtree brought on the field.

We went to the hospital with him a few days ago, and they said we could watch him do rehab. We could watch him or we could help him out, Woods said. We just want to do whatever we can to help him out. Hes so competitive. He was doing some weights, and the lady was trying to hold him back with how much he could do. He was like, No, lets add five more pounds. Then lets add 10 more pounds."

Woods said he's seen Roundtree's positivity increase during the last few weeks as his body, especially his upper-body strength, has shown improvement.

When it first happened, he has that hard-working mentality, but I know he was down when it first happened," Woods said. "But so much progress is being brought forth upon him, and hes just taking it to the next level. He already knows the outcome is going to be what he wants, so he just knows he has to work hard every day and the results will come to him.

Illinois plans to bring Roundtree back to the Illinois campus in January to continue his recovery and studies. Hes come a long way since Woods received that devastating May phone call.

Bobbys been my roommate since freshman year. When coach told me, I broke down in tears, Woods said. I was at home watching TV in the living room. I broke down in tears. But now, Bobby knows everything is going to be all right. So Im uplifting with him. Im happy about how his progress is going. Everything is going to be all right.

The Illinis visits to Roundtree provide healing for not only Roundtree but also for themselves. That makes the choice to make the trip even easier.

Going up to see Bobby, man, every time it brings joy to me, Woods said. It also brings joy to him.

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Teammates see Roundtree 'more open' with recovery, progress - 247Sports

Victory at Celtic shows the progress made by Steven Gerrards Rangers – The Guardian

Posted on December 31, 2019 by Danzig

The nature of Rangers success over Celtic continues to resonate more than the fact it transpired at all. Steven Gerrards team were comprehensive in their dismissal of Celtic away from home, too in an encounter which has fuelled theories of Rangers claiming a first top-flight title since 2011.

For Gerrard this marked a significant moment, arriving a season and a half into his management career. The former Liverpool captain has revealed detail of the message delivered to his players at half-time at Celtic Park, with the game level at 1-1.

I just said to them to go and play the second 45 minutes and reward yourself for the last six months of hard work, Gerrard said. Go back to the first day of pre-season and how do you want to feel walking out of this stadium? Do you want to reward yourself for six months of real hard graft and get a result that the majority of the first half deserved? And that the previous 90 minutes against this team deserved. I think they went out and delivered.

Gerrard resisted calls to declare his team as potential champions. Instead he insisted progressing to the next round of the Scottish Cup, which will be Rangers task after the winter break now under way, is a priority. The 39-year-old admits, though, that his team have progressed.

The plan is to try and make it go the distance; that is the plan, Gerrard said of the league. But there is a lot of football to be played and a lot of challenges, big challenges, home and away. There are two other competitions in amongst that and that is for me to try and navigate through with the players and try and do the best we can. But the plan is to try and put in the best fight we can until the end.

What Gerrard cannot affect, of course, is Celtics response. Rangers are now two points behind their oldest foes with a game in hand. Celtic, though, are expected to invest to a higher degree than their rivals during the transfer window.

Listen, the fans are going to be the fans, Gerrard said. I can only say that by no way, shape or form do we in that dressing room think that we have achieved anything [against Celtic] besides a big three points and a big win. We deserve credit for that and we will enjoy that, but that is all it is. We respect the challenge.

I have got characters. The team needed a spine, a lot of change, it needed big players who werent scared of the big occasion. It had some and it had a few that were coming to the end that I wished were 10 years younger, like Kenny Miller. But there were a lot of changes that needed to happen. We had to virtually wipe it clean over two or three transfer windows. We are still not finished. We need to keep building, keep getting stronger, keep learning. But now I look at this team and it is different from the one I inherited by a long way.

Gerrard defended the actions of Michael Beale, his first-team coach, who was sent off in stoppage time after an altercation with the Celtic assistant manager, John Kennedy. I think you have got to respect the position that we are in as well, Gerrard said.

It is an emotional game; the referee ends up playing more than the four minutes that were on there. We get a red card [for Alfredo Morelos] and there is one [against Morelos] that was blatant. Understand being in our shoes, it is an emotional time. I am not going to make any apologies. This is new to some of us so we are not going to get everything perfect, not going to get everything right. My staff and my players have been fantastic and that is all I want to say.

The fallout from Moreloss sending off continued on Monday as Rangers, while also repeating calls for VAR to be introduced in Scotland, hit out at alleged racist abuse of the striker. We believe Alfredo was racially abused and we would expect now that all measures will be taken to identify and deal with those responsible.

Celtic responded with a statement which read: We are aware of the gestures made by the player towards Celtic supporters. We have received no reports of any racial abuse but would fully investigate any should these be brought to our attention.

Morelos made some gestures with his hand across his throat as he made his way off and up the tunnel. Rangers insisted the strikers actions had been misinterpreted.

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Victory at Celtic shows the progress made by Steven Gerrards Rangers - The Guardian

Amherst’s new form of government a work in progress – GazetteNET

Posted on December 31, 2019 by Danzig

AMHERST One year into the Town Council becoming Amhersts legislative body,goals to confrontclimate change have been adopted, projects to provide more affordable housing are in progress and the foundation for a series of municipal building projects is set.

Even with these successes highlighted in her state of the town address earlier this month, Council President Lynn Griesemer didnt shy away from observing that a significant challengeconfronts the councilas 2020 nears: making the councilors jobs more manageable.

For both those who supported and opposed adopting the charter crafted by the Charter Commission, there is agreement that the amount of timethe 13 councilors are spending on town affairs needs to be addressed. The first-ever Town Council was sworn in on Dec. 2, 2019.

Mandi Jo Hanneke, the only commissioner whoran for and won a seat on the Town Council,said she expected significant work, understanding that the jobwould be to keep the town running, adopting budgets and making decisions on spending while also setting up the processes for operating the council fairly, effectively and efficiently.

My personal experience as a councilor has generally been what I imagined it might be when I was on the Charter Commission, Hanneke said.

As Lynn mentioned, many councilors, including myself, want to work towardmaking the time commitment more manageable, Hanneke added. This is an especially important goal for me, due to my work on the Charter Commission.

Thecommissioners, Hanneke said, hoped that any resident would be willing and able to serve, including those with full-time jobs or young children at home.

I still believe that as the council moves beyond setting up its operating structures, we will be able to achieve that goal, Hanneke said.

Former commission member Meg Gage, who opposed the charter and replacingrepresentative Town Meeting, said she feels the council hasnt done much over the past 12 months, in part because of how time-consuming its work has been.

As should have been expected, setting things up has dominated all the council does, Gage said. We need people to run for reelection for continuity, and its a real concern that people may not want to run.

A Town Meeting member who opposed the charter,Maurianne Adams said the results have not met what she described as overly optimistic predictions, though she said its not forlack of working hard.

It has turned out to be far more daunting than the proposers had suggested to create a new council while also trying to divvy up executive-legislative roles, and all of this while running the government, meeting needs and deadlines, and making difficult decisions, Adams said.

Former commissioner Nick Grabbe said he worries about regular meetingsdragging on for four to five hours, though he still supports the charter and new form of government because of the accountability it brings.

We want councilors to seek reelection so voters can decide if theywant to keep them or not, Grabbe said.

Michael Greenebaum, another former Town Meeting member opposed to the charter, said the length of meetings is denying residents the chance to fully participate. Thats an inadvertent way of separating the council from the public, which is disturbing to me, Greenebaum said.

Andy Churchill, who chaired the Charter Commission, said the analogy of building the airplane while flying it is appropriate.

They are trying to establish procedures at the same time as they are trying to run the town, thats going to be more work, Churchill said.

Town Manager Paul Bockelmanpraises the councilors for their commitment.

Its more workbecause its a new entity finding its pathway. Its a lot of time for the council to determine the process for making decisions, Bockelman said.

He also praised town staff and the more rigorous analysis and improved presentations demanded.

Its forced all of us toup our gameto meet expectationsof the council, Bockelman said.

Aside from the workload, supporters are pleased with just how will the council has functioned.

Katherine Appy, a leader of the Amherst Forward political action committee that endorsedcandidates, said the past year has seen an incredible effort to get structures in place and to govern.

Across the board, they have shown themselves to be responsive to community needs while keeping the big picture in sight for what's best for the town, Appy said.

Thenext challenges are clear for the council,Appy said, which is to develop and enact a strategic capital plan.

Hanneke, too, said she believes that residents have been well served by thecharter, with easy access to councilors and a government that is flexible enough to act, pointing to the recent decision on spending for a playground at Kendrick Park. Under Town Meeting, providing such a match for a state grant would have been almost impossible due to timing issues.

Churchill said the government model is akin toa CEO and board of directors, and from that view, the council has done exactly what was anticipated

I feel like theyve done a pretty good job representing the perspectivesof the town and working together, Churchill said.

Gage agreed with that. Im very impressed with the cohesion that seems to exist and the general respect everyone has for everyone else, Gage said.

Greenebaum said thediversity of the council has been demonstrated in the few unanimousvotes it has taken.

Grabbesaid the new government means residents know and elect their councilors.

It is wonderful that the decision-makers in Amherst, all of them now have accountability to voters, Grabbe said. For me, its all about democracy. Town Meetingwasnt democratic and didnt legitimately have accountability to voters.

People feel theyknow who to call to be responsive, Churchill said.

Griesemer said this is the compliment she hears the most, that residents can easily get in touch with their district representatives to make their views known.

Gage, though, said she has seen a council focused on neighborhoods rather than the full town, pointing to the decision to install a temporary bridge on Station Road after District 5 residents putpressure on the council.

Im worried that people are participating around neighborhood issues rather than whats best for the whole town, Gage said.

Many of the fears opponents expressed, such as that the council would cause rampantdevelopment to take place in town due to developers buying elections, has not come to pass, Grabbe said.

Still, Greenebaum said heworries about the northern end of downtown where long-established businesses like The Pub and Cousins Market may be closing to make way for a new project.

Im in great distress with whats happening in town and would like the council to indicate how it feels about that, Greenebaum, said.

One area that Hannekesaid has been less successful and will need work to match the ideals in the charter isthe annual public forums on the schools, master plan, and town budget.

While they haven't been as well attended as the commission had hoped, they have served their purpose for those individuals that came out, Hanneke said. The council has a lot more work ahead of it to make these events into the robust discussions the commission envisioned.

Gene Therapies Make it to Clinical Trials – Discover Magazine

Posted on December 31, 2019 by Danzig

After years of ethical debates and breakthroughs in the lab, CRISPR has finally made its way to clinical trials. Researchers are now looking at whether the DNA-editing tool, as well as more conventional gene therapies, can effectively treat a wide array of heritable disorders and even cancers.

Theres been a convergence of the science getting better, the manufacturing getting much better, and money being available for these kinds of studies, says Cynthia Dunbar, a senior investigator at the National Heart, Lung, and Blood Institute. Its truly come of age.

CRISPR formally known as CRISPR-Cas9 has been touted as an improvement over conventional gene therapy because of its potential precision. CRISPR (clustered regularly interspaced short palindromic repeats) is a genetic code that, contained in a strand of RNA and paired with the enzyme Cas9, acts like molecular scissors that can target and snip out specific genes. Add a template for a healthy gene, and CRISPRs cut can allow the cell to replace a defective gene with a healthy one.

In April, scientists at the University of Pennsylvania announced they had begun using CRISPR for cancer treatments. The first two patients one with multiple myeloma, the other with sarcoma had cells from their immune systems removed. Researchers used CRISPR to genetically edit the cells in the lab, and then returned them back into their bodies.

On the other side of the country, Mark Walters, a blood and bone marrow transplant specialist at the University of California, San Francisco, Benioff Childrens Hospital in Oakland, is gearing up for trials that will use CRISPR to repair the defective gene that causes sickle cell disease. With CRISPR, once youve made that type of correction, [that cell] is 100 percent healthy, says Walters.

Another team is tackling the same disease using a type of hemoglobin, a protein in red blood cells, thats normally made only in fetuses and newborn babies. Researchers found that some adults continue to produce these proteins throughout their lives, and when those adults also have sickle cell disease, their symptoms are mild. So the international team used CRISPR to disable the gene that interferes with production of this hemoglobin, resuming its production and protecting the adult patients against sickle cell disease.

Several other CRISPR studies are in the works to treat a range of inherited disorders, including hemophilia and SCID-X1 (also known as X-linked severe combined immunodeficiency, the so-called bubble boy disease in which babies are born without a functioning immune system).

At St. Jude Childrens Research Hospital, a gene therapy trial cured Gael Jesus Pino Alva (pictured with his mother, Giannina) of SCID-X1, the bubble boy disease. (Credit: St. Jude Children's Research Hospital/Peter Barta)

The past year also saw success in a handful of experiments on conventional gene therapy. Instead of using CRISPR to repair disease-causing genes, these treatments use hollowed-out viruses to ferry healthy versions of genes into cells. Millions of these altered cells are released into the bloodstream or bone marrow in hopes that enough will land in the right places. But because scientists cant predict where the circulating genes may end up, this shotgun approach has had unintended, sometimes fatal, consequences including, in an earlier study, inadvertently activating leukemia-causing genes in patients treated for SCID-X1.

But in 2019, researchers learned that using a different type of virus one related to HIV to transport the genes may prevent these side effects. In an April study, researchers at St. Jude Childrens Research Hospital in Memphis, Tennessee, and UCSF Benioff Childrens Hospital in Oakland collected bone marrow from eight newborns with SCID-X1. They loaded corrective genes into the disabled HIV-related virus, which carried them into the patients bone marrow stem cells. The infants also received low doses of busulfan, a chemotherapy that gave the doctored stem cells room to grow. So far, we havent seen anything worrisome, says Ewelina Mamcarz, a pediatric oncologist at St. Jude who led the research team. The study recently added its 12th patient.

Gene therapy does have its momentum [back], says Mamcarz, reflecting on the fields setback after the earlier studys leukemia side effects. Theres so much that still needs to be done, and so many questions, she says. [But] this is how medicine evolves. We always want to be better than we were a week ago.

In the future, the hope is that gene therapy technologies will move beyond mending simple genetic mistakes and be used to combat big killers like diabetes or heart disease. [Those diseases are] more challenging, but a lot of them would benefit from knocking out a bad gene, says Dunbar.

For now, though, researchers are optimistic about the progress thats already been made. All of this has been very encouraging, says Dunbar. [And] for sickle cell in the U.S. and hemophilia in the developed world, these diseases may soon be solved.

[This story originally appeared in print as "Gene Therapy Gets Clinical."]

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Gene Therapies Make it to Clinical Trials - Discover Magazine

2019: The year gene therapy came of age – INQUIRER.net

Posted on December 31, 2019 by Danzig

For decades, the DNA of living organisms such as corn and salmon has been modified, but Crispr, invented in 2012, made gene editing more widely accessible. Image: YinYang/IStock.com via AFP Relaxnews

In the summer, a mother in Nashville with a seemingly incurable genetic disorder finally found an end to her suffering by editing her genome.

Victoria Grays recovery from sickle cell disease, which had caused her painful seizures, came in a year of breakthroughs in one of the hottest areas of medical research gene therapy.

I have hoped for a cure since I was about 11, the 34-year-old told AFP in an email.

Since I received the new cells, I have been able to enjoy more time with my family without worrying about pain or an out-of-the-blue emergency.

Over several weeks, Grays blood was drawn so doctors could get to the cause of her illness stem cells from her bone marrow that were making deformed red blood cells.

The stem cells were sent to a Scottish laboratory, where their DNA was modified using Crispr/Cas9 pronounced Crisper a new tool informally known as molecular scissors.

The genetically edited cells were transfused back into Grays veins and bone marrow. A month later, she was producing normal blood cells.

Medics warn that caution is necessary but, theoretically, she has been cured.

This is one patient. This is early results. We need to see how it works out in other patients, said her doctor, Haydar Frangoul, at the Sarah Cannon Research Institute in Nashville.

But these results are really exciting.

In Germany, a 19-year-old woman was treated with a similar method for a different blood disease, beta thalassemia. She had previously needed 16 blood transfusions per year.

Nine months later, she is completely free of that burden.

For decades, the DNA of living organisms such as corn and salmon has been modified.

But Crispr, invented in 2012, made gene editing more widely accessible. It is much simpler than preceding technology, cheaper and easy to use in small labs.

The technique has given new impetus to the perennial debate over the wisdom of humanity manipulating life itself.

Its all developing very quickly, said French geneticist Emmanuelle Charpentier, one of Crisprs inventors and the cofounder of Crispr Therapeutics, the biotech company conducting the clinical trials involving Gray and the German patient.

Cures

Scientists practicing the technique insert a normal gene into cells containing a defective gene.

It does the work the original could not such as making normal red blood cells, in Victorias case, or making tumor-killing super white blood cells for a cancer patient.

Crispr goes even further: instead of adding a gene, the tool edits the genome itself.

They join several other gene therapies bringing the total to eight approved in recent years to treat certain cancers and an inherited blindness.

Serge Braun, the scientific director of the French Muscular Dystrophy Association, sees 2019 as a turning point that will lead to a medical revolution.

Twenty-five, 30 years, thats the time it had to take, he told AFP from Paris.

It took a generation for gene therapy to become a reality. Now, its only going to go faster.

Just outside Washington, at the National Institutes of Health (NIH), researchers are also celebrating a breakthrough period.

We have hit an inflection point, said Carrie Wolinetz, NIHs associate director for science policy.

These therapies are exorbitantly expensive, however, costing up to $2 million meaning patients face grueling negotiations with their insurance companies.

They also involve a complex regimen of procedures that are only available in wealthy countries.

Gray spent months in hospital getting blood drawn, undergoing chemotherapy, having edited stem cells reintroduced via transfusion and fighting a general infection.

You cannot do this in a community hospital close to home, said her doctor.

However, the number of approved gene therapies will increase to about 40 by 2022, according to MIT researchers.

They will mostly target cancers and diseases that affect muscles, the eyes and the nervous system.

Bioterrorism

Another problem with Crispr is that its relative simplicity has triggered the imaginations of rogue practitioners who dont necessarily share the medical ethics of Western medicine.

Last year in China, scientist He Jiankui triggered an international scandal and his excommunication from the scientific community when he used Crispr to create what he called the first gene-edited humans.

The biophysicist said he had altered the DNA of human embryos that became twin girls Lulu and Nana.

His goal was to create a mutation that would prevent the girls from contracting HIV, even though there was no specific reason to put them through the process.

That technology is not safe, said Kiran Musunuru, a genetics professor at the University of Pennsylvania, explaining that the Crispr scissors often cut next to the targeted gene, causing unexpected mutations.

Its very easy to do if you dont care about the consequences, Musunuru added.

Despite the ethical pitfalls, restraint seems mainly to have prevailed so far.

The community is keeping a close eye on Russia, where biologist Denis Rebrikov has said he wants to use Crispr to help deaf parents have children without the disability.

There is also the temptation to genetically edit entire animal species malaria-causing mosquitoes in Burkina Faso or mice hosting ticks that carry Lyme disease in the US.

The researchers in charge of those projects are advancing carefully, however, fully aware of the unpredictability of chain reactions on the ecosystem.

Charpentier doesnt believe in the more dystopian scenarios predicted for gene therapy, including American biohackers injecting themselves with Crispr technology bought online.

Not everyone is a biologist or scientist, she said.

And the possibility of military hijacking to create soldier-killing viruses or bacteria that would ravage enemies crops?

Charpentier thinks that technology generally tends to be used for the better.

Im a bacteriologist weve been talking about bioterrorism for years, she said. Nothing has ever happened.IB/JB

Experimental Gene Therapy Shows Promise for Preventing and Treating Lou Gehrig’s Disease in Mice – BioSpace

Posted on December 31, 2019 by Danzig

Amyotrophic lateral sclerosis (ALS), sometimes called Lou Gehrigs disease, is a neurodegenerative disease affecting nerve cells in the brain and spinal cord. Researchers at the University of California San Diego School of Medicine published research describing a new way to deliver a gene-silencing vector to mice with ALS. The therapy resulted in long-term suppression of the disease if the treatment was given before the disease started. It also blocked disease progression in the mice if symptoms already appeared.

The study was published in the journal Nature Medicine.

At present, this therapeutic approach provides the most potent therapy ever demonstrated in mouse models of mutated SOD1 gene-linked ALS, said senior author Martin Marsala, professor in the Department of Anesthesiology at UC San Diego School of Medicine. In addition, effective spinal cord delivery of AAV9 vector in adult animals suggests that the use of this new delivery method will likely be effective in treatment of other hereditary forms of ALS or other spinal neurodegenerative disorders that require spinal parenchymal delivery of therapeutic gene(s) or mutated-gene silencing machinery, such as in C9orf72 gene mutation-linked ALS or in some forms of lysosomal storage disease.

ALS appears in two forms, sporadic and familial. The most common form is sporadic, responsible for 90 to 95% of all cases. Familial ALS makes up 5 to 10% of all cases in the U.S., and as the name suggests, is inherited. Studies have shown that a least 200 mutations of the SOD1 gene are linked to ALS.

In healthy individuals, the SOD1 gene provides instructions for an enzyme called superoxide dismutase. This enzyme is used to break down superoxide radicals, which are toxic oxygen molecules that are a byproduct of normal cellular processes. It is believed that the mutations in the gene cause ineffective removal of superoxide radicals or potentially cause other toxicities resulting in motor neuron cell death.

The new research involves injecting shRNA, an artificial RNA molecule that can turn off, or silence, a targeted gene. This delivers shRNA to cells by way of a harmless adeno-associated virus (AAV). In the research, they injected the viruses carrying shRNA into two locations in the spinal cord of adult mice expressing an ALS-causing mutation of the SOD1 gene. They were performed just before disease onset or after the laboratory animals started showing symptoms.

The researchers have tested the approach in adult pigs, whose have spinal cord dimensions closer to those in humans. They found that by using an injector developed for adult humans, the procedure could be performed without surgical complications and in a reliable fashion.

The next step will be more safety studies with a large animal model.

While no detectable side effects related to treatment were seen in mice more than one year after treatment, the definition of safety in large animal specimens more similar to humans is a critical step in advancing this treatment approach toward clinical testing, Marsala said.

About 5,000 people are diagnosed with ALS in the U.S. each year, with about 30,000 people living with the disease. There are symptomatic treatments, but no cure. Most patients die from the disease two to five years after diagnosis.

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Experimental Gene Therapy Shows Promise for Preventing and Treating Lou Gehrig's Disease in Mice - BioSpace

First Alzheimer’s Disease Gene Therapy Human Study Provided by Maximum Life Foundation, Offers 10 Free Therapies for Qualifying Patients – Benzinga

Posted on December 31, 2019 by Danzig

Maximum Life Foundation ("MaxLife"), is rapidly transforming the way we treat aging diseases. MaxLife plans to use a promising gene therapy offered by Integrated Health Systems to give free access to ten (10) early to mid-stage Alzheimer's Disease (AD) patients. David Kekich, MaxLife's CEO, stated "MaxLife will grant 100% of the therapy costs to help bring pioneering gene therapy to cure this disease and make Alzheimer's Disease a thing of the past."

NEWPORT BEACH, Calif. (PRWEB) December 30, 2019

Cure Now Instead of Palliative Care

According to the Alzheimer's Association:

Alzheimer's costs Americans $277 billion a year and rising. Sharp increases in Alzheimer's disease cases, deaths and costs are stressing the U.S. healthcare system and caregivers. About 5.7 million Americans have Alzheimer's disease. To date, no one has survived it.

Improvements of AD symptoms and the recovery of normal brain functions have been demonstrated in-vivo in mouse experiments, and in-vitro in human cell experiments through the rejuvenation of microglia (the brain's first line of defense against infection) and neurons as well as stimulating mitochondrial function using the telomerase reverse transcriptase (TERT) protein.

One human patient received a lower dose therapy in August 2018 with no adverse side effects. To date, the patient's disease has not progressed. MaxLife hopes to see symptom reversals in the next patients.

"If we can prove a benefit to patients that have no other option now, we can potentially treat Alzheimer's Disease in people in early to mid-stage Alzheimer's, finally creating effective medicine at the cellular level," states Kekich. "If successful, this treatment could potentially be used on other diseases such as Parkinson's and ALS."

The unique difference is developing treatments against the cellular degeneration caused by aging as the root cause of most major diseases. Studies have proven aging is the leading risk factor for many life-threatening diseases, including Alzheimer's.

With a world class Scientific Advisory Board, MaxLife is ready to push forward into practical solutions. A gene therapy facilitator, Integrated Health Systems plans to treat other adult aging-related diseases with no previous cure such as Sarcopenia, Atherosclerosis, Chronic Kidney Disease (CKD) and even aging itself with gene therapies.

"This technology could halt many of the big age associated killers in industrialized countries'" states Kekich. "Compassionate care helps patients with no other option to get access to experimental therapies that may benefit both themselves and society as a whole."

MaxLife also seeks grants and donations for human gene therapy studies for atherosclerosis, sarcopenia and chronic kidney disease as well as for human aging. The protocols have already been developed. Please Click Here and scroll to the bottom of the page to see how to donate.

To apply for a free therapy or for more information, see http://www.maxlife.org/alzheimers-disease/ and https://maxlife.org/how-to-register-and-qualify-for-the-alzheimers-human-study/.

For Further Information, Contact: David Kekich, CEO Maximum Life Foundation.

Maximum Life Foundation is a 501(c)(3) Not-For-Profit corporation founded in 1999.

Tax I.D. #31-1656405. David A. Kekich Tel. #949-706-2468. Info@MaxLife.org

For the original version on PRWeb visit: https://www.prweb.com/releases/first_alzheimers_disease_gene_therapy_human_study_provided_by_maximum_life_foundation_offers_10_free_therapies_for_qualifying_patients/prweb16809113.htm

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First Alzheimer's Disease Gene Therapy Human Study Provided by Maximum Life Foundation, Offers 10 Free Therapies for Qualifying Patients - Benzinga

BLA Submitted for Gene Therapy to Treat Hemophilia A – Monthly Prescribing Reference

Posted on December 31, 2019 by Danzig

Home News Drugs in the Pipeline

BioMarin has submitted a Biologics License Application (BLA) to the Food and Drug Administration (FDA) for valoctocogene roxaparvovec (BMN 270) for the treatment of hemophilia A in adults. This is the first marketing application submission for a gene therapy product for any type of hemophilia.

Valoctocogene roxaparvovec is an investigational adeno-associated virus (AAV) gene therapy that is administered as a single infusion to produce clotting factor VIII. The BLA submission is supported by interim analysis of a phase 3 study and 3-year phase 1/2 data. Results from the ongoing phase 1/2 study showed that bleed rate control and reduction in factor VIII usage was maintained for a third year following a single administration of valoctocogene roxaparvovec.

The FDA previously granted Breakthrough Therapy and Orphan Drug designations to valoctocogene roxaparvovec. The Company anticipates the BLA review to commence in February 2020.

We look forward to working with the FDA as we seek marketing authorization for the potential first gene therapy for hemophilia A, said Hank Fuchs, MD, President, Global Research and Development at BioMarin. Our hope is one day very soon to deliver a transformative treatment that has the potential to change the way hemophilia A is treated.

For more information visit biomarin.com.

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BLA Submitted for Gene Therapy to Treat Hemophilia A - Monthly Prescribing Reference

Gene therapy to conquering hepatitis C: A decade of medical breakthroughs – Business Standard

Posted on December 31, 2019 by Danzig

For all the flak the pharmaceutical industry has taken for its exorbitant pricing practices, there's no getting around the fact that it's been a pretty stunning decade for medical progress.

Multiple new categories of medicines have moved from dreams and lab benches into the market and peoples lives, and investors who came along for the ride often reaped extraordinary profits. The Nasdaq Biotech Index is up 360% over the last 10 years to the S&P 500's 190%. And thats without mentioning the hundreds of billions of dollars in takeovers that rewarded shareholders with windfalls.

As 2020 approaches, it's worth highlighting how far we've come in the past 10 years in developing new therapies and approaches to treating disease, even as politicians grapple with how to rein in health-care costs without breaking an ecosystem that incentivizes the search for new discoveries. Here are some of the decades biggest medical breakthroughs:

Cell therapies: First approved in the U.S. two years ago, these treatments still sound like science fiction. Drugmakers harvest immune cells from patients, engineer them to hunt tumors, grow them by the millions into a living drug, and reinfuse them. Yescarta from Gilead Siences Inc. and Novartis AGs Kymriah the two treatments approved so far can put patients with deadly blood cancers into remission in some cases. At the beginning of the decade, academics were just beginning early patient tests.

Its still early days for the technology, and some issues are holding these drugs back. There are significant side effects, and the bespoke manufacturing process is expensive and time-consuming. That has contributed to a bruising price tag: Both of the approved medicines cost over $350,000 for a single treatment. And for now, cell therapy is mostly limited to very sick patients who have exhausted all other alternatives.

Luckily, more options are on their way. Some drugmakers are focused on different types of blood cancers. Others hope to mitigate side effects or create treatments that can be grown from donor cells to reduce expenses and speed up treatment. In the longer run, companies are targeting trickier solid tumors. Scientists wouldn't be looking so far into the future without this decades extraordinary progress.

Gene therapies: Researchers have spent years trying to figure out how to replace faulty DNA to cure genetic diseases, potentially with as little as one treatment. Scientific slip-ups and safety issues derailed a wave of initial excitement about these therapies starting in the 1990s; the first two such treatments to be approved in Europe turned out to be commercial flops.

This decade, the technology has come of age. Luxturna, a treatment developed by Spark Therapeutics Inc. for a rare eye disease, became the first gene therapy to get U.S. approval in late 2017. Then in May came the approval of Novartis AGs Zolgensma for a deadly muscle-wasting disease. The drugs have the potential to stave off blindness and death or significant disability with a single dose, and, unsurprisingly, Big Pharma has given them a substantial financial endorsement. Roche Holding AG paid $4.7 billion to acquire Spark this year, while Novartis spent $8.7 billion in 2018 to buy Zolgensma developer Avexis Inc.

Dozens of additional therapies are in development for a variety of other conditions and should hit the market in the next few years. They offer the tantalizing potential not just to cure diseases, but to replace years of wildly expensive alternative treatment. If drugmakers can resist the temptation to squeeze out every ounce of value by doing things like charging $2.1 million for Zolgensma, theres potential for these treatments to save both lives and money.

RNA revolution: The above treatments modify DNA; this group uses the bodys messaging system to turn a patients cells into a drug factory or interrupt a harmful process. Two scientists won a Nobel Prize in 2006 for discoveries related to RNA interference (RNAi), one approach to making this type of drug, showing its potential to treat difficult diseases. That prompted an enormous amount of hype and investment, but a series of clinical failures and safety issues led large drugmakers to give up on the approach. Sticking with it into this decade paid off.

Alnylam Inc. has been working since 2002 to figure out the thorny problems plaguing this class of treatments. It brought two RNAi drugs for rare diseases to the market in the past two years and has more on the way. The technology is also moving from small markets to larger ones: Novartis just paid $9.7 billion to acquire Medicines Co. for its Alnylam-developed drug that can substantially lower cholesterol with two annual treatments.

Ionis Pharmaceuticals Inc. and Biogen Inc. collaborated on Spinraza, a so-called antisense drug that became the first effective treatment for a deadly rare disease. It was approved in late 2016 and had one of the most impressive drug launches of the decade. And Moderna Therapeutics rode a wave of promising messenger RNA-based medicines to the most lucrative biotechnology IPO of all time in 2018. From pharma abandonment to multiple approvals and blockbuster sales potential in under 10 years. Not bad!

Cancer immunotherapy: Scientists had been working on ways to unleash the human immune system on cancers well before the 2010s without much luck. Checkpoint inhibitors drugs that release the brakes on the body's defense mechanisms have since produced outstanding results in a variety of cancers and are the decades most lucrative turnaround story.

Merck got a hold of Keytruda via its 2009 acquisition of Schering-Plough, but it was far from the focus of that deal. Once Bristol-Myers Squibb & Co. produced promising results for its similar drug, Opdivo, Merck started a smart development plan that has turned Keytruda into the worlds most valuable cancer medicine. Its now available to treat more than 10 types of the disease, and has five direct competitors in the U.S. alone. Analysts expect the category to exceed $25 billion in sales next year.

If anything, the drugs may have been too successful. Copycat efforts are pulling money that could fund more innovative research. There are thousands of trials underway attempting to extend the reach of these medicines by combining them with other drugs. Some are based more on wishful thinking than firm scientific footing. Still, the ability to shrink some previously intractable tumors is a considerable advance. If drugmakers finally figure out the right combinations and competition creates pricing pressure that boosts access, these medicines will do even more in the years to come.

Conquering hepatitis C: From a combined economic and public-health standpoint, a new group of highly effective hepatitis C medicines may outstrip just about anything else on this list so far. Cure rates for earlier treatments werent especially high; they took some time to work and had nasty side effects. The approval of Gileads Sovaldi in 2013, followed in time by successor drugs such as AbbVie Inc.s Mavyret, have made hepatitis C pretty easily curable in a matter of weeks. For Gilead, getting to market rapidly with its drug proved enormously profitable; it raked in over $40 billion in revenue in just three years.

Hepatitis C causes liver damage over time that can lead to transplants or cancer. The existence of a rapid cure is a significant long-term boon even if the initial pricing on the drugs made them, in some cases, prohibitively expensive. Sovaldi notoriously cost $1,000 per pill at launch and over $80,000 for a course of treatment. The good new is, treatments have become a lot more affordable, which should allow this class of drugs to have a broad and lasting positive health impact.

Hepatitis C is one of the relatively few markets where the drug-pricing system has worked well. As competing medicines hit the market, the effective cost of these treatments plummeted. That, in turn, made the drugs more accessible to state Medicaid programs and prison systems, which operate on tight budgets and care for populations with higher rates of hepatitis C infection. Louisiana has pioneered the use of a Netflix model, under which the state paid an upfront fee for unlimited access to the drug. Its an arrangement that will help cure thousands of patients, and other states are likely to follow its lead.

Many of the medicines highlighted in this column have list prices in the six figures, a trend thats helped drive up Americas drug spending by more than $100 billion since 2009. Building on this decades medical advances is going to lead to even more effective medicines that will likely come with steeper prices. Id like to hope that policymakers will come up with a solution that better balances the need to reward innovation with the need to keep medicines accessible. That would really be a breakthrough.

Max Nisen at mnisen@bloomberg.net

@2019Bloomberg

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Gene therapy to conquering hepatitis C: A decade of medical breakthroughs - Business Standard

Year in Review: Gene Therapy Technology and a Milestone 2019 for Medical Research – News18

Posted on December 31, 2019 by Danzig

In the summer, a mother in Nashville with a seemingly incurable genetic disorder finally found an end to her suffering -- by editing her genome. Victoria Gray's recovery from sickle cell disease, which had caused her painful seizures, came in a year of breakthroughs in one of the hottest areas of medical research -- gene therapy. "I have hoped for a cure since I was about 11," the 34-year-old told AFP in an email.

"Since I received the new cells, I have been able to enjoy more time with my family without worrying about pain or an out-of-the-blue emergency." Over several weeks, Gray's blood was drawn so doctors could get to the cause of her illness -- stem cells from her bone marrow that were making deformed red blood cells. The stem cells were sent to a Scottish laboratory, where their DNA was modified using Crispr/Cas9 -- pronounced "Crisper" -- a new tool informally known as molecular "scissors." The genetically edited cells were transfused back into Gray's veins and bone marrow. A month later, she was producing normal blood cells.

Medics warn that caution is necessary but, theoretically, she has been cured. "This is one patient. This is early results. We need to see how it works out in other patients," said her doctor, Haydar Frangoul, at the Sarah Cannon Research Institute in Nashville. "But these results are really exciting." In Germany, a 19-year-old woman was treated with a similar method for a different blood disease, beta thalassemia. She had previously needed 16 blood transfusions per year.

Nine months later, she is completely free of that burden. For decades, the DNA of living organisms such as corn and salmon has been modified. But Crispr, invented in 2012, made gene editing more widely accessible. It is much simpler than preceding technology, cheaper and easy to use in small labs. The technique has given new impetus to the perennial debate over the wisdom of humanity manipulating life itself. "It's all developing very quickly," said French geneticist Emmanuelle Charpentier, one of Crispr's inventors and the cofounder of Crispr Therapeutics, the biotech company conducting the clinical trials involving Gray and the German patient.

Cures

Crispr is the latest breakthrough in a year of great strides in gene therapy, a medical adventure started three decades ago, when the first TV telethons were raising money for children with muscular dystrophy. Scientists practising the technique insert a normal gene into cells containing a defective gene. It does the work the original could not -- such as making normal red blood cells, in Victoria's case, or making tumor-killing super white blood cells for a cancer patient. Crispr goes even further: instead of adding a gene, the tool edits the genome itself.

After decades of research and clinical trials on a genetic fix to genetic disorders, 2019 saw a historic milestone: approval to bring to market the first gene therapies for a neuromuscular disease in the US and a blood disease in the European Union. They join several other gene therapies -- bringing the total to eight -- approved in recent years to treat certain cancers and an inherited blindness. Serge Braun, the scientific director of the French Muscular Dystrophy Association, sees 2019 as a turning point that will lead to a medical revolution. "Twenty-five, 30 years, that's the time it had to take," he told AFP from Paris.

"It took a generation for gene therapy to become a reality. Now, it's only going to go faster." Just outside Washington, at the National Institutes of Health (NIH), researchers are also celebrating a "breakthrough period." "We have hit an inflection point," said Carrie Wolinetz, NIH's associate director for science policy.These therapies are exorbitantly expensive, however, costing up to $2 million -- meaning patients face grueling negotiations with their insurance companies. They also involve a complex regimen of procedures that are only available in wealthy countries.

Gray spent months in hospital getting blood drawn, undergoing chemotherapy, having edited stem cells reintroduced via transfusion -- and fighting a general infection. "You cannot do this in a community hospital close to home," said her doctor. However, the number of approved gene therapies will increase to about 40 by 2022, according to MIT researchers. They will mostly target cancers and diseases that affect muscles, the eyes and the nervous system.

Bioterrorism

Another problem with Crispr is that its relative simplicity has triggered the imaginations of rogue practitioners who don't necessarily share the medical ethics of Western medicine. Last year in China, scientist He Jiankui triggered an international scandal -- and his excommunication from the scientific community -- when he used Crispr to create what he called the first gene-edited humans. The biophysicist said he had altered the DNA of human embryos that became twin girls Lulu and Nana.

His goal was to create a mutation that would prevent the girls from contracting HIV, even though there was no specific reason to put them through the process. "That technology is not safe," said Kiran Musunuru, a genetics professor at the University of Pennsylvania, explaining that the Crispr "scissors" often cut next to the targeted gene, causing unexpected mutations. "It's very easy to do if you don't care about the consequences," Musunuru added. Despite the ethical pitfalls, restraint seems mainly to have prevailed so far.

The community is keeping a close eye on Russia, where biologist Denis Rebrikov has said he wants to use Crispr to help deaf parents have children without the disability. There is also the temptation to genetically edit entire animal species -- malaria-causing mosquitoes in Burkina Faso or mice hosting ticks that carry Lyme disease in the US. The researchers in charge of those projects are advancing carefully, however, fully aware of the unpredictability of chain reactions on the ecosystem.

Charpentier doesn't believe in the more dystopian scenarios predicted for gene therapy, including American "biohackers" injecting themselves with Crispr technology bought online. "Not everyone is a biologist or scientist," she said. And the possibility of military hijacking to create soldier-killing viruses or bacteria that would ravage enemies' crops? Charpentier thinks that technology generally tends to be used for the better. "I'm a bacteriologist -- we've been talking about bioterrorism for years," she said. "Nothing has ever happened."

Get the best of News18 delivered to your inbox - subscribe to News18 Daybreak. Follow News18.com on Twitter, Instagram, Facebook, Telegram, TikTok and on YouTube, and stay in the know with what's happening in the world around you in real time.

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Year in Review: Gene Therapy Technology and a Milestone 2019 for Medical Research - News18

What were the biggest biopharma stories of 2019? – MedCity News

Posted on December 31, 2019 by Danzig

Like any reporting beat, biopharma has its slow news days. But its rare to find a whole year when few events of note occur, and 2019 was no exception. Indeed, the last year of the 21st centurys second decade saw its fair share of major mergers and acquisitions, controversies and historical milestones across the medical biotechnology and pharmaceutical industries.

The year kicked off with a mega-merger, when on Jan. 3, New York-based Bristol-Myers Squibb said it would acquire Summit, New Jersey-based Celgene for $74 billion. A wave of mergers and acquisitions across the biopharma industry soon followed. This was a decade after another wave of biopharma consolidation, when 2009 kicked off with Pfizers acquisition of Wyeth, followed by Merck & Co. buying Schering-Plough and Roche buying Genentech. The next large-scale deal happened in June, when Chicago-based AbbVie said it would spend $63 billion to acquire Allergan.

Other than the two aforementioned large deals, however, most M&A activity this year has taken the form of large biotechnology and pharmaceutical companies buying much smaller, but still large-cap players. Earlier this month, Roche concluded its $4.3 billion acquisition of gene therapy maker Spark Therapeutics, originally announced in February. That deal was dogged by a 10-month investigation by U.S. and U.K. regulators into the question of whether the Roche might have a disincentive to develop Sparks Phase III gene therapy candidate for hemophilia A, given that the Swiss drugmaker already makes a drug for the disease. Indianapolis-based Eli Lilly & Co.s $8 billion acquisition of Loxo Oncology days after the BMS-Celgene deal did not face such hurdles, though Loxo handed off its one approved product, the cancer drug Vitrakvi (larotrectinib), to development partner Bayer. Numerous other deals have followed, including Novartis deal to acquire The Medicines Co. for $9.7 billion, Astellas announcement that it would buy Audentes Therapeutics for $3 billion, Mercks move to buy ArQule for $2.7 billion, among others.

While dwarfed by its counterpart in 2009, the current M&A wave has not been without controversy. In September, a group of eight Democratic senators and one independent several of whom are or had been running for president wrote to the Federal Trade Commission urging greater scrutiny over such deals amid concerns about competition and high drug prices. While its uncertain if the FTC was responding to their concerns, the agency said Dec. 17 that it would seek to block genomic sequencing company Illumina from buying a smaller firm, Pacific Biosciences of California, in a previously announced $1.2 billion deal.

Another controversy that arose in 2019 was on the regulatory front. In August, the Food and Drug Administration said it was looking into a disclosure from Novartis subsidiary AveXis that mouse data from a disused assay used in its application for the gene therapy Zolgensma (onasemnogene abeparvovec-xioi) in spinal muscular atrophy which the FDA approved in May had been manipulated. The scandal, which predated Novartis acquisition of AveXis in 2018, led to two of the gene therapy developers executives being fired and created a publicity crisis for the drugmaker.

Yet, while the Zolgensma scandal did not affect the FDAs overall position on the product itself, another FDA decision still raised eyebrows. The approval of Karyopharm Therapeutics Xpovio (selinexor) in highly refractory multiple myeloma patients attracted significant criticism from physicians, who pointed both to what some called a low response rate in the Phase IIa study on which the approval was based and the FDA using Phase III data that were not disclosed to the public. The approval also happened despite the FDAs Oncologic Drugs Advisory Committee voting not to recommend it.

2019 also saw some important milestones on the regulatory front. Nine months after Scott Gottliebs resignation as commissioner of the FDA, the Senate confirmed Dr. Stephen Hahn, a radiation oncologist and chief medical executive at The University of Texas MD Anderson Cancer Center, to take his place. And the agency approved the first ever drug to tackle the root cause of sickle cell disease and its first vaccine against the Ebola virus.

In addition to specific events, many trends that have garnered increasing public and political attention moved to front and center in 2019 as well. The national conversation about drug pricing showed no signs of quieting down, as the Trump administration rolled out various programs designed to tackle high drug prices, and the House fired a shot across the bow with a bill this month that would require the Centers for Medicare and Medicaid Services to negotiate prices for some drugs.

Many of these events will continue to play out in 2020, as a new FDA commissioner takes the helm of the agency and large companies see their acquisitions bear fruit, for good or ill. And the conversation about drug pricing will only get louder amid the 2020 presidential election, which in turn could lead to greater scrutiny over biopharma industry consolidation.

Photo: klenger, Getty Images

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What were the biggest biopharma stories of 2019? - MedCity News

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