He aha ʻo Atidarsagene Autotemcel: Nā hoʻohana, ka lāʻau, nā hopena ʻaoʻao a me nā mea hou aku

ʻO Atidarsagene autotemcel he lāʻau lapaʻau gene i hoʻolālā ʻia e mālama i ka metachromatic leukodystrophy (MLD), he maʻi genetic kakaʻikahi e pili ana i ka ʻōnaehana nerve. Hoʻohana kēia lāʻau lapaʻau hou i kāu mau cell stem ponoʻī i hoʻololi ʻia i ka genetic e hana i ka enzyme e pono ai kou kino akā ʻaʻole hiki ke hana iā ia iho.

Inā ua ʻike ʻia ʻoe a i ʻole kekahi mea aloha me MLD, hiki iā ʻoe ke manaʻo nui ʻia e ka paʻakikī o kēia koho lapaʻau. E hele kākou i nā mea a pau e pono ai ʻoe e ʻike e pili ana i kēia lāʻau lapaʻau ma nā huaʻōlelo maʻalahi a maopopo i hiki iā ʻoe ke hana i nā hoʻoholo i ʻike ʻia e pili ana i kāu mālama.

He aha ʻo Atidarsagene Autotemcel?

ʻO Atidarsagene autotemcel he lāʻau lapaʻau gene e pani i nā genes hewa i kou kino me nā mana olakino, hana. E noʻonoʻo e like me ka hāʻawi ʻana i kāu mau cell i nā ʻōlelo aʻo hou e hana i kahi enzyme nui i kapa ʻia ʻo arylsulfatase A (ARSA) ʻaʻole hiki i ka poʻe me MLD ke hana pono.

Ua ʻike ʻia kēia lāʻau lapaʻau ma ka inoa inoa ʻo Libmeldy. ʻO ia ka mea a nā kauka e kāhea ai i kahi

Ua ʻae ʻia ka lāʻau lapaʻau no nā keiki me ka MLD i hoʻomaka mua ʻaʻole i hōʻike i nā hōʻailona, a i ʻole i nā wā mua loa o ka maʻi. ʻOi aku ka maikaʻi ke hāʻawi ʻia ma mua o ka pōʻino nui o nā aʻalolo, ʻo ia ke kumu he mea nui ka ʻike mua a me ka mālama ʻana.

E loiloi pono kāu kauka inā kūpono kēia lapaʻau no kāu kūlana. E noʻonoʻo lākou i nā mea e like me kou makahiki, ka holomua o ka maʻi, a me ke olakino holoʻokoʻa ma mua o ka ʻōlelo ʻana i kēia lāʻau lapaʻau.

Pehea e hana ai ʻo Atidarsagene Autotemcel?

Hana kēia lāʻau lapaʻau gene ma ka hāʻawi ʻana i kou kino i ka hiki ke hana i ka enzyme ARSA i nalowale. Me ka ʻole o kēia enzyme, e hōʻiliʻili nā mea ʻino i loko o kāu mau cell nerve a luku mālie iā lākou, e hana ana i nā hōʻailona o MLD.

Hoʻomaka ke kaʻina hana lapaʻau me ka hōʻiliʻili ʻana i kāu mau cell stem mai kou iwi iwi. A laila, hoʻouna ʻia kēia mau cell i kahi hale hana loea kahi e hoʻokomo ai nā ʻepekema i kahi kope olakino o ka gene ARSA me ka hoʻohana ʻana i kahi maʻi i hoʻololi ʻia ma ke ʻano he ʻōnaehana hāʻawi. Ua hana ʻia ka maʻi i mea palekana a ʻaʻole hiki ke hoʻoulu i ka maʻi.

Ke mākaukau nā cell i hoʻololi ʻia, e loaʻa iā ʻoe ka chemotherapy e hoʻomākaukau i kou iwi iwi no nā cell hou. A laila, hoʻokomo ʻia nā cell stem i hoʻoponopono ʻia i kou kahe koko ma o ka IV. Hele kēia mau cell i kou iwi iwi a hoʻomaka e hana i ka enzyme i nalowale.

Ua manaʻo ʻia he lāʻau lapaʻau ikaika a ikaika e pono ai ka nānā pono ʻana. Hiki i ke kaʻina hana holoʻokoʻa mai ka hōʻiliʻili ʻana i nā cell a hiki i ka hoʻōla ʻana ke lawe i kekahi mau mahina a pono i ka mālama olakino loea.

Pehea e lawe ai au i ka Atidarsagene Autotemcel?

Hāʻawi ʻia ʻo Atidarsagene autotemcel ma ke ʻano he infusion intravenous hoʻokahi manawa ma kahi kikowaena lapaʻau loea. ʻAʻole hiki iā ʻoe ke lawe i kēia lāʻau ma ka home, a pono ia i ka hoʻomākaukau nui a me ka nānā ʻana e kahi hui o nā loea lapaʻau.

Ma mua o ka loaʻa ʻana o ka lāʻau lapaʻau, pono ʻoe e hele i nā pule he nui o ka hoʻomākaukau. Aia kēia i ka hōʻiliʻili ʻana i kāu mau cell stem ma o ke kaʻina hana i kapa ʻia ʻo apheresis, e like me ka hāʻawi ʻana i ke koko akā lōʻihi ka lōʻihi. Pono paha ʻoe e lawe i nā lāʻau lapaʻau e kōkua i ka hoʻonui ʻana i ka helu o nā cell stem i loko o kou kahe koko.

ʻO ka lā hoʻokomo maoli e pili ana i ka loaʻa ʻana o ka chemotherapy mua e hoʻomākaukau i kou iwi iwi, a laila e hahai ʻia e ka hoʻokomo ʻana i ka gene therapy. Pono ʻoe e noho i ka haukapila no kekahi mau lā a i nā pule no ka nānā pono ʻana. E nānā kāu hui lapaʻau i nā pilikia a hōʻoia i ka ʻae ʻana o kou kino i ka lāʻau lapaʻau.

I kou noho ʻana, e loaʻa iā ʻoe ka mālama kākoʻo me nā lāʻau lapaʻau e pale ai i nā maʻi, ke kākoʻo meaʻai, a me nā lāʻau lapaʻau ʻē aʻe e like me ka mea e pono ai. Hoʻomalu ʻia ke kaiapuni o ka haukapila e pale iā ʻoe i ka wā e ola hou ai kāu ʻōnaehana pale.

Pehea ka lōʻihi e lawe ai au i ka Atidarsagene Autotemcel?

Hāʻawi ʻia ʻo Atidarsagene autotemcel ma ke ʻano he lāʻau lapaʻau hoʻokahi, ʻaʻole ma ke ʻano he lāʻau lapaʻau e hoʻomau nei. Ke loaʻa iā ʻoe ka hoʻokomo ʻana i ka gene therapy, pono e hoʻomau nā cell i hoʻololi ʻia i ka hana ʻana i ka enzyme e pono ai no nā makahiki e hiki mai ana.

Eia naʻe, hoʻomau ka hoʻōla a me ka nānā ʻana no nā mahina he nui ma hope o ka lāʻau lapaʻau. E hahai pono kāu hui lapaʻau iā ʻoe no ka liʻiliʻi o ʻelua mau makahiki e nānā i kāu holomua a nānā i nā pilikia. Aia kēia i nā hoʻokolohua koko maʻamau, nā loiloi neurological, a me nā loiloi ʻē aʻe.

Ua manaʻo ʻia nā hopena o kēia lāʻau lapaʻau e lōʻihi, e hāʻawi ana i nā pono i ke ola. Pono paha kekahi poʻe maʻi i nā lāʻau lapaʻau kākoʻo hou a i ʻole nā lāʻau lapaʻau i ko lākou ulu ʻana a me ka hoʻomohala ʻana, akā ʻaʻole e hana hou ʻia ka gene therapy.

He aha nā hopena ʻaoʻao o Atidarsagene Autotemcel?

E like me nā lāʻau lapaʻau koʻikoʻi a pau, hiki i ka atidarsagene autotemcel ke hana i nā hopena ʻaoʻao. ʻO ka hoʻomaopopo ʻana i kēia mau hopena e kōkua iā ʻoe e hoʻomākaukau no ka lāʻau lapaʻau a ʻike i ka mea e manaʻo ai i ka wā o kou ola hou ʻana.

ʻO nā hopena ʻaoʻao maʻamau e pili ana i ka chemotherapy āu e loaʻa ai ma mua o ka hoʻokomo ʻana i ka gene therapy. He manawa pōkole ka hopena o kēia mau mea akā hiki ke koʻikoʻi a komo pū:

• Hoʻonui ʻia ka pilikia o nā maʻi ma muli o ka nāwaliwali o ka ʻōnaehana pale
• Haʻahaʻa nā helu koko (anemia, haʻahaʻa platelets, haʻahaʻa keʻokeʻo koko)
• Nausea, luaʻi, a me ka nalowale o ka ʻai
• Nā ʻeha waha a me nā pilikia digestive
• Ka hāʻule ʻana o ka lauoho
• Luhi a me ka nāwaliwali

ʻO nā hopena ʻaoʻao koʻikoʻi akā ʻaʻole maʻamau e komo pū me nā maʻi koʻikoʻi, nā pilikia koko, a me nā pilikia e pili ana i ka laina waena i hoʻohana ʻia no ka mālama ʻana. Mālama pono kāu hui lapaʻau iā ʻoe no kēia mau pilikia a hāʻawi i ka lāʻau lapaʻau koke inā loaʻa.

Ke aʻo ʻia nei nā hopena lōʻihi mai ka mea he lāʻau lapaʻau hou. Hiki i kekahi poʻe maʻi ke hoʻomohala i nā pilikia i nā mahina a i ʻole nā makahiki ma hope o ka lāʻau lapaʻau, ʻo ia ke kumu he mea nui ka mālama ʻana.

ʻO nā pilikia laha ʻole akā koʻikoʻi paha e komo pū me ka hoʻomohala ʻana i nā maʻi maʻi koko, ʻoiai ke ʻike ʻia nei kēia pilikia he haʻahaʻa loa ma muli o ka ʻikepili o kēia manawa. E kūkākūkā kāu kauka i nā pilikia hiki ke loaʻa me ʻoe ma mua o ka hoʻomaka ʻana o ka lāʻau lapaʻau.

ʻO wai ka mea ʻaʻole pono e lawe iā Atidarsagene Autotemcel?

ʻAʻole kūpono ʻo Atidarsagene autotemcel no nā mea a pau me MLD. E loiloi pono kāu kauka inā kūpono kēia lāʻau lapaʻau iā ʻoe ma muli o kekahi mau mea nui.

ʻAʻole ʻōlelo ʻia kēia lāʻau lapaʻau inā loaʻa iā ʻoe ka MLD holomua me nā hōʻailona nui, no ka mea, hana maikaʻi loa ia ke hāʻawi ʻia ma mua o ka pōʻino nui o ke aʻalolo. ʻAʻole kūpono hoʻi no kekahi mau ʻano genetic o MLD i hiki ʻole ke pane i kēia ʻano lāʻau lapaʻau.

ʻO nā kūlana ʻē aʻe e hiki ke hoʻolilo iā ʻoe i mea kūpono ʻole no kēia lāʻau lapaʻau e komo pū ana:

• Nā pilikia nui o ka puʻuwai, ka māmā, a i ʻole nā puʻupaʻa
• Nā maʻi maʻi e hana nei a i ʻole nā maʻi o ka ʻōnaehana pale
• Ka maʻi ʻaʻai a i ʻole nā maʻi koko i hala
• ʻAʻole hiki ke hana i ka hoʻomākaukau chemotherapy e pono ai
• ʻAʻole hiki ke kiʻi i ka mālama ma hope

Hiki nō hoʻi ke lilo ka makahiki i mea nui, no ka mea, ua ʻae ʻia kēia lapaʻau i kēia manawa no nā keiki a me nā ʻōpio. He mea nui ka maikaʻi o kou olakino a me kou hiki ke ʻae i ke kaʻina hana lapaʻau ikaika.

E hana kāu hui lapaʻau i nā hoʻokolohua nui ma mua o ka hoʻoholo ʻana i kou kūpono. Pākuʻi ʻia kēia i ka hoʻokolohua genetic, nā loiloi neurological, a me ka loiloi o kou kūlana olakino holoʻokoʻa.

Inoa Brand Atidarsagene Autotemcel

Hoʻolaha ʻia ʻo Atidarsagene autotemcel ma lalo o ka inoa brand Libmeldy. ʻO kēia inoa ka mea āu e lohe ai i nā kamaʻilio me kāu hui mālama ola a ʻike ma nā mea lapaʻau.

Hana ʻia ʻo Libmeldy e Orchard Therapeutics, kahi hui e loea ana i nā lāʻau gene no nā maʻi laha. Ua loaʻa i ka lāʻau lapaʻau ka ʻae mai nā ʻoihana hoʻoponopono ma ʻEulopa a me nā wahi ʻē aʻe no ka mālama ʻana i ka MLD.

Ke noiʻi a kūkākūkā paha i kēia lapaʻau, hiki iā ʻoe ke ʻike i nā inoa ʻelua i hoʻohana ʻia. Hiki i nā mea mālama ola ke kuhikuhi iā ia ma kona inoa maʻamau (atidarsagene autotemcel) a i ʻole kona inoa brand (Libmeldy).

Nā koho ʻē aʻe o Atidarsagene Autotemcel

I kēia manawa, he kakaikahi wale nō nā koho lapaʻau i loaʻa no ka MLD, kahi e waiwai ai ʻo atidarsagene autotemcel no nā mea maʻi kūpono. ʻO ke ala lapaʻau ʻē aʻe nui ʻo ka hoʻololi ʻana i nā cell stem hematopoietic (HSCT), i ʻike ʻia hoʻi ʻo ka transplant iwi iwi.

Hoʻopili ʻo HSCT i ka loaʻa ʻana o nā cell stem mai kahi mea hāʻawi olakino ma mua o ka hoʻohana ʻana i kāu mau cell i hoʻololi ʻia i ka genetic. Hiki i kēia ala ke hāʻawi i ka enzyme nalowale, akā lawe pū ia i nā pilikia hou e like me ka maʻi graft-versus-host a me ka pono no nā lāʻau immunosuppressive no ke ola.

ʻO nā lapaʻau kākoʻo ʻē aʻe e kālele ana i ka mālama ʻana i nā hōʻailona a hiki ke komo pū me:

• Ka lāʻau lapaʻau kino e mālama i ka neʻe a me ka ikaika
• Ka lāʻau lapaʻau ʻōlelo e kōkua me nā pilikia kamaʻilio
• Ka lāʻau lapaʻau hana e kōkua me nā hana o kēlā me kēia lā
• Nā lāʻau lapaʻau e mālama i nā hopu ʻana, ka ʻeha, a i ʻole nā hōʻailona ʻē aʻe
• Ke kākoʻo meaʻai a me ke kōkua ʻana i ka hānai

ʻO ke koho ma waena o ka lāʻau lapaʻau gene a me nā lapaʻau ʻē aʻe e hilinaʻi ana i nā mea he nui me kou makahiki, ke kahua o ka maʻi, nā mea hāʻawi i loaʻa, a me nā kūlana olakino pilikino. E kōkua kāu hui lapaʻau iā ʻoe e hoʻomaopopo i nā koho a pau i loaʻa.

Ke hoʻomau nei ka noiʻi ʻana i nā lapaʻau hou no MLD, me nā ala lāʻau lapaʻau gene ʻē aʻe a me nā lāʻau lapaʻau pani enzyme. Hiki ke loaʻa nā hoʻokolohua lapaʻau no nā maʻi ʻaʻole i kūpono i nā lapaʻau i ʻae ʻia i kēia manawa.

Ua ʻoi aku ka maikaʻi o Atidarsagene Autotemcel ma mua o ka Bone Marrow Transplant?

Hiki i nā atidarsagene autotemcel a me ka bone marrow transplant ke lilo i mau lapaʻau maikaʻi no MLD, akā he mau mea maikaʻi a me nā mea maikaʻi ʻole. ʻO ke koho maikaʻi loa e hilinaʻi ana i kou kūlana pilikino a me ka mea nui loa no kāu hihia.

Hāʻawi ʻo Atidarsagene autotemcel i nā mea maikaʻi he nui ma luna o ka bone marrow transplant kuʻuna. No ka mea e hoʻohana ana i kāu mau cell ponoʻī, ʻaʻohe pilikia o ka maʻi graft-versus-host, kahi pilikia koʻikoʻi kahi e hoʻouka ai nā cell donor i kou kino. ʻAʻole pono ʻoe e ʻimi i kahi mea hāʻawi kūpono, hiki ke paʻakikī a me ka manawa.

Hiki nō hoʻi ke ʻoi aku ka maikaʻi o ke ala lāʻau lapaʻau gene i ka hāʻawi ʻana i ka enzyme e pono ai i ka lolo a me ka ʻōnaehana nerve. Hōʻike nā haʻawina mua e hiki ke hāʻawi i nā hopena lōʻihi maikaʻi aʻe no kekahi poʻe maʻi, ʻoiai e pono ana ka noiʻi hou e hōʻoia i kēia.

Akā, ua hoʻohana ʻia ka hoʻololi ʻana i ka iwi iwi no ka manawa lōʻihi a loaʻa nā ʻikepili palekana lōʻihi. He koho maikaʻi paha ia no kekahi poʻe maʻi, ʻoiai ʻo ka poʻe i kūpono ʻole i ka lāʻau gene a i ʻole i nā kūlana i loaʻa koke nā cell donor.

E noʻonoʻo kāu hui lapaʻau i nā mea e like me kou makahiki, ka holomua o ka maʻi, ka mōʻaukala ʻohana, a me nā makemake pilikino i ka wā e ʻōlelo ai i ke ala lapaʻau maikaʻi loa no ʻoe.

Nā Nīnau i Nīnau Pinepine ʻia e pili ana iā Atidarsagene Autotemcel

Q1. He palekana anei ʻo Atidarsagene Autotemcel no nā keiki?

ʻAe, ua ʻae ʻia ʻo atidarsagene autotemcel no ka mālama ʻana i nā keiki me ka MLD mua a ua hōʻike ʻia he palekana maʻamau ke hāʻawi ʻia e nā hui lapaʻau i ʻike ʻia. ʻO ka ʻoiaʻiʻo, ʻoi aku ka maikaʻi o ka lapaʻau ʻana i nā keiki i loaʻa ʻole nā hōʻailona nui.

Eia naʻe, e like me nā lāʻau lapaʻau koʻikoʻi, loaʻa nā pilikia e pono e kaupaona ʻia me nā pōmaikaʻi hiki. Pono nā keiki e loaʻa ana i kēia lāʻau lapaʻau i ka nānā ʻana a me ka mālama kākoʻo i ka wā o ke kaʻina hana lapaʻau. E hana pū ka hui lapaʻau me ʻoe a me kou ʻohana e hōʻoia i ka ʻike palekana loa.

Q2. He aha kaʻu e hana ai inā loaʻa nā pilikia i ka wā o ka lapaʻau?

Inā kū mai nā pilikia i ka wā a i ʻole ma hope o ka lapaʻau ʻana iā atidarsagene autotemcel, ua mākaukau kāu hui lapaʻau e pane koke. E noho ʻoe i kahi kikowaena lapaʻau kūikawā me ka nānā ʻana 24/7 a me ke komo ʻana i ka mālama ulia pōpilikia i kou noho ʻana.

E hōʻike koke i nā hōʻailona e pili ana i kāu hui mālama ola, me ke kuni, ke kahe koko ʻano ʻē, ka pilikia o ka hanu ʻana, a i ʻole nā loli i ka hana neurological. Ua aʻo ʻia nā limahana lapaʻau e ʻike a mālama i nā pilikia me ka wikiwiki. Mai kānalua e nīnau i nā nīnau a i ʻole e hōʻike i nā hopohopo i kēlā me kēia manawa i kāu huakaʻi lapaʻau.

Q3. He aha kaʻu e hana ai inā haʻalele au i nā manawa e hahai ai?

He mea nui nā manawa e hahai ai ma hope o ka mālama ʻana me atidarsagene autotemcel no ka nānā ʻana i kou holomua a me ka ʻike ʻana i nā pilikia hiki ke hiki koke. Inā ʻoe e hala i kahi manawa, e hoʻokaʻaʻike koke i kāu hui mālama olakino e hoʻonohonoho hou i ka manawa hiki.

Aia i loko o kēia mau manawa nā hoʻokolohua nui e like me ka hana koko, nā loiloi neurological, a me nā loiloi ʻē aʻe e kōkua ana e hōʻoia i ka hana pono ʻana o ka mālama. Pono kāu hui lapaʻau i kēia ʻike e hāʻawi i ka mālama maikaʻi loa a me ka hana ʻana i nā hoʻoponopono e pono ai i kāu hoʻolālā mālama.

Q4. I ka manawa hea e hiki ai iaʻu ke hoʻi i nā hana maʻamau ma hope o ka mālama ʻana?

He ʻokoʻa ka manawa hoʻōla no kēlā me kēia kanaka, akā pono ka hapa nui o nā maʻi i kekahi mau mahina ma mua o ka hoʻi ʻana i nā hana maʻamau. E nāwaliwali ana kāu ʻōnaehana pale no nā pule a i ʻole mau mahina ma hope o ka mālama ʻana, no laila pono ʻoe e mālama kūikawā e pale aku i nā maʻi.

E hāʻawi kāu hui lapaʻau i nā alakaʻi kikoʻī e pili ana i ka manawa e hiki ai iā ʻoe ke hoʻi i ke kula, ka hana, a i ʻole nā hana ʻē aʻe. Aia kēia hoʻoholo i nā mea e like me kāu helu koko, ke olakino holoʻokoʻa, a me ka maikaʻi o kou hoʻōla ʻana. Ma keʻano laulā, e hoʻonui mālie ʻoe i kāu pae hana i ka wā e hoʻōla ai kou kino a hoʻōla hou kāu ʻōnaehana pale.

Q5. E pono anei iaʻu i nā mālama hou aʻe ma hope o ka gene therapy?

ʻOiai ua hoʻolālā ʻia ʻo atidarsagene autotemcel e lilo i hoʻokahi manawa mālama, pono paha ʻoe i ka mālama kākoʻo hou aʻe i kou ulu ʻana a me ka hoʻomohala ʻana. Hiki i kēia ke komo i ka physical therapy, speech therapy, a i ʻole nā hana ʻē aʻe e kōkua iā ʻoe e hiki i kou hiki piha.

Pono nō hoʻi ʻoe i ka nānā ʻana i ka lāʻau lapaʻau maʻamau no nā makahiki ma hope o ka mālama ʻana e hōʻoia i ka hana mau ʻana o ka therapy a me ka nānā ʻana i nā hopena lōʻihi. Hiki i kekahi poʻe maʻi ke pōmaikaʻi mai nā lāʻau lapaʻau hou a i ʻole nā mālama ʻana e hoʻoponopono i nā hōʻailona kikoʻī a i ʻole nā pilikia, akā hana ʻia kēia mau hoʻoholo e pili ana i kāu mau pono pilikino a me ka holomua.