{"id":179783,"date":"2025-09-25T01:33:07","date_gmt":"2025-09-25T01:33:07","guid":{"rendered":"https:\/\/www.newsbeep.com\/us\/179783\/"},"modified":"2025-09-25T01:33:07","modified_gmt":"2025-09-25T01:33:07","slug":"experts-highlight-medical-innovations-and-challenges","status":"publish","type":"post","link":"https:\/\/www.newsbeep.com\/us\/179783\/","title":{"rendered":"Experts highlight medical innovations and challenges"},"content":{"rendered":"

Earlier this year, gene-editing methods Liu\u2019s lab invented were used to treat KJ Muldoon, a baby born with a rare genetic disorder called CPS1 deficiency. Half of all babies with the disorder die in infancy, said Liu, a core member of the Broad Institute of MIT and Harvard and a professor at Harvard University.<\/p>\n

Instead, KJ received the first-ever bespoke gene editing treatment, which was designed to edit the single-letter mutation that causes the disease. <\/p>\n

Now, just over a year old, KJ is thriving \u2013 meeting developmental milestones and eating a normal diet, Liu said.<\/p>\n

\u201cMaybe for the first time, DNA is not destiny,\u201d Liu said. \u201cIf you happen to have a misspelling in your DNA that would normally create a life for you that is full of hardship or shorten your life, there are things that you can now do in some cases to depart from that fate.\u201d<\/p>\n

The technology, first developed in Liu\u2019s lab in 2016, is called base editing. It works by using CRISPR, the gene editing tool that won its inventors the Nobel Prize in 2020. But instead of cutting the DNA strand like the first CRISPR approaches, it flips the mutated letter \u2014 also known as a base \u2014 to the correct type. <\/p>\n

Liu\u2019s lab has since developed another gene editing tool, called prime editing, which is a \u201ctrue search and replace,\u201d he said, able to make virtually any kind of local DNA change.<\/p>\n

The technologies have enormous potential to treat rare genetic diseases, Liu said, which affect as many as 400 million people globally but receive less per capita investment because of the specificity of the conditions.<\/p>\n

Liu called for a streamlining of the regulatory process so patients can access treatments like KJ\u2019s that target different diseases.<\/p>\n

\u201cThe system needs to keep up with the science,\u201d he said.<\/p>\n

Other panelists highlighted the capability of GLP-1 drugs for obesity, such as Ozempic, in helping patients lose weight. <\/p>\n

Dr. Fatima Cody Stanford, an obesity medicine physician scientist at Massachusetts General Hospital and associate professor at Harvard Medical School, emphasized the importance of thinking about obesity as a chronic disease that requires long-term treatment, including lifestyle modification, medications, or a surgical intervention. <\/p>\n

Despite these advancements, high costs still present a challenge.<\/p>\n

Health insurance companies, including Blue Cross Blue Shield of Massachusetts<\/a> and Harvard Pilgrim Health Care<\/a>, said they will stop covering popular GLP-1 drugs for weight loss starting next year. Both will continue to cover patients who are using the drugs for diabetes treatment.<\/p>\n

Stanford said she has spent entire appointments with patients focused on this loss of coverage.<\/p>\n

\u201cMany of these patients have lost between 15 and 20 percent of their total body weight,\u201d Stanford said. \u201cWhen we pull back these medications, we see that 85 to 90 percent of these patients will regain their weight.\u201d<\/p>\n

Without access to GLP-1 drugs under insurance, Stanford worries that patients who cannot afford to pay will indeed regain weight.<\/p>\n

\"Dr.Dr. Fatima Cody Stanford<\/p>\n

massgeneral.org<\/p>\n

Misinformation and federal funding cuts have hit universities and hospitals across Massachusetts, panelists said.<\/p>\n

Earlier this week, President Trump touted an unproven link between Tylenol use in pregnancy and autism. That, as well as long-debunked claims about the risks of vaccines promoted by the White House, are contributing to a dangerous loss of public understanding of science, said Dr. Jeremy Faust, an emergency physician at Brigham and Women\u2019s Hospital and assistant professor at Harvard Medical School.<\/p>\n

\u201cThere is now a vast percentage of this country that from this day forward will always believe that Tylenol increases the rate of autism in pregnancy, period,\u201d said Faust. \u201cWe\u2019re heading in the wrong direction because we\u2019re replacing the scientific process.\u201d<\/p>\n

Also undercutting scientific progress, experts say, is a funding freeze on grants to Harvard imposed on the Trump administration earlier this year and an overall reduction in the number of grants awarded by the National Institutes of Health. <\/p>\n

\u201cI know colleagues who have lost a third, or half, or two-thirds of their lab\u2019s funding,\u201d said Liu of the Broad Institute. \u201cAnd as you might imagine, that creates a situation where you suddenly have to decide which projects, which people, which mouse colonies, which monkey colonies, you are going to continue to advance.\u201d<\/p>\n

Other panels focused on increasing rates of colorectal cancer in young people, the latest science on aging and a discussion about a career in biotechnology with Robert Langer, co-founder of Moderna and Institute Professor at the Massachusetts Institute of Technology.<\/p>\n

Katie Muchnick can be reached at katie.muchnick@globe.com<\/a>.<\/p>\n","protected":false},"excerpt":{"rendered":"Earlier this year, gene-editing methods Liu\u2019s lab invented were used to treat KJ Muldoon, a baby born with…\n","protected":false},"author":2,"featured_media":179784,"comment_status":"","ping_status":"","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[59],"tags":[97,252,253],"class_list":["post-179783","post","type-post","status-publish","format-standard","has-post-thumbnail","category-health-care","tag-health","tag-health-care","tag-healthcare"],"_links":{"self":[{"href":"https:\/\/www.newsbeep.com\/us\/wp-json\/wp\/v2\/posts\/179783","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.newsbeep.com\/us\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.newsbeep.com\/us\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/us\/wp-json\/wp\/v2\/users\/2"}],"replies":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/us\/wp-json\/wp\/v2\/comments?post=179783"}],"version-history":[{"count":0,"href":"https:\/\/www.newsbeep.com\/us\/wp-json\/wp\/v2\/posts\/179783\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/us\/wp-json\/wp\/v2\/media\/179784"}],"wp:attachment":[{"href":"https:\/\/www.newsbeep.com\/us\/wp-json\/wp\/v2\/media?parent=179783"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.newsbeep.com\/us\/wp-json\/wp\/v2\/categories?post=179783"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.newsbeep.com\/us\/wp-json\/wp\/v2\/tags?post=179783"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}